2023 April Quarterly Pipeline

Table of CONTENTS

EDITORIAL STAFF Maryam Tabatabai, PharmD Editor-in-Chief Vice President, Clinical Information Carole Kerzic, RPh Executive Editor Clilnical Pharmacist, Drug Information

EDITOR-IN-CHIEF'S MESSAGE

2

Consultant Panel

PIPELINE DEEP DIVE

3

Robert Greer, RPh, BCOP Vice President, Clinical Strategy and Programs

KEEP ON YOUR RADAR

29

PIPELINE DRUG LIST

30

GLOSSARY

49

Michelle Pannone-Booth, PharmD Director, Specialty Clinical Solutions

Andrea Henry, PharmD, MBA, BCPS Specialty Drug Information Pharmacist Katie Lockhart Senior Manager, Forecasting and Pharmacoeconomics Simone Ndujiuba, PharmD, BCOP Director, Clinical Strategy and Innovation, Oncology Olivia Pane, PharmD, CDCES Drug Information Pharmacist Nothing herein is or shall be construed as a promise or representation regarding past or future events and Magellan Rx Management expressly disclaims any and all liability relating to the use of or reliance on the information contained in this presentation. The information contained in this publication is intended for educational purposes only and should not be considered clinical, financial, or legal advice. By receipt of this publication, each recipient agrees that the information contained herein will be kept confidential and that the information will not be photocopied, reproduced, distributed to, or disclosed to others at any time without the prior written consent of Magellan Rx Management.

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Editor-in-Chief's MESSAGE Welcome to the MRx Pipeline. This quarterly publication offers clinical insights and competitive intelligence on anticipated drugs in development, so you are well-sourced on the drug pipeline.

MRx Pipeline, our universal forecast, addresses trends applicable across market segments. Traditional and specialty drugs as well as agents under the pharmacy and medical benefits are featured. Also profiled in the report are new molecular entities, pertinent new and expanded indications for existing medications, and biosimilars.

Clinical analyses, financial outlook, and pre-regulatory status are considered. The products housed in the MRx Pipeline have been researched in detail. They have been developed in consultation with our internal team of clinical and analytics experts.

METHODOLOGY

Emerging therapeutics continue to grow and influence the clinical and financial landscape. Therefore, Magellan Rx Management has developed a systematic approach to determine the products with significant clinical impact. For the in-depth clinical evaluations, the products’ potential to meet an underserved need in the market by becoming the new standard of care, and the ability to replace existing therapies were investigated. The extent to which the pipeline drugs could shift market share on a formulary and their impact on disease prevalence were also important considerations. In order to assist payers with assessing the potential impact of these pipeline drugs, where available, a financial forecast has been included for select products. Primarily complemented by data from EvaluateTM, this pipeline report looks ahead at the 5-year projected annual US sales through the year 2027. These figures are not specific to a particular commercial or government line of business; rather, they look at forecasted total US sales. Depending on a variety of factors, including the therapeutic categories, eventual FDA-approved indications, populations within the plan, and other indices, the financial impact could vary by different lines of business.

REFLECTION

Thus far in 2023, the agency has approved 13 novel drugs. Notably, most of the approvals so far in 2023 use at least one of the FDA’s expedited approval methos and the majority are designated as Orphan Drug. Some of the products include first-time approvals for rare diseases, a new agent for Alzheimer’s disease, new oncology drugs, a new option for the treatment of anemia caused by chronic kidney disease, among others.

While numbers do not tell the entire story, they do represent significant innovation in patient care and advance public health for the American public.

ON THE HORIZON

As we look ahead, and with the COVID-19 Public Health Emergency (PHE) ending on May 11, 2023, there is a continued trend towards the approval of specialty medications and drugs for rare conditions, with 65% and 33% of approvals expected, respectively, for agents with applications submitted to the FDA. There are 2 agents seeking FDA’s Accelerated Approval, which allows for earlier drug approval for serious conditions that fill an unmet need based on a surrogate endpoint reasonably likely to predict a clinical benefit. Approval of 2 new vaccines for RSV prophylaxis in older adults is expected with a biologic for younger ages on the horizon. Several injectable gene therapies are awaiting FDA decision for hemophilia A, sickle cell anemia and thalassemia, Duchenne muscular dystrophy, as well as a topical gene therapy for a rare dermatologic condition. Other noteworthy pipeline trends to watch include the development of complex therapies, cell therapies, oncology, immunology, immunotherapy, and therapeutic options for ultra-rare hereditary diseases. The launch of several Humira® biosimilars is anticipated to in July 2023. Moreover, sprouting products for obesity and women’s health are being actively monitored through MRx Pipeline.

The drug pipeline ecosphere will continue to evolve as it faces challenges and successes. Innovative agents that show positive results without compromising patient safety and access offer true therapeutic advances and hold the promise to alter the treatment paradigm.

Maryam Tabatabai, PharmD Editor-in-Chief, MRx Pipeline

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Pipeline DEEP DIVE Objective evidence-based methodology was used to identify the Deep Dive drugs in the upcoming quarters. This section features a clinical overview and explores the potential place in therapy for these agents. Moreover, it addresses their FDA approval timeline and 5-year financial forecast.

SPECIALTY

PRIORITY REVIEW

BREAKTHROUGH THERAPY

86%

14%

18%

BIOSIMILAR

ORPHAN DRUG

57%

11%

pecialty drug names appear in  S magenta throughout the publication.

ONCOLOGY

avasopasem manganese IV Galera PROPOSED INDICATIONS

Radiotherapy (RT)-induced severe oral mucositis (SOM) in patients with head and neck cancer (HNC)

CLINICAL OVERVIEW

Avasopasem manganese is a selective dismutase mimetic designed to convert RT-induced bursts of superoxide to hydrogen peroxide in normal cells and cancer cells.

The randomized, double-blind, placebo-controlled, phase 3 ROMAN trial (NCT03689712) evaluated avasopasem manganese in 455 patients with HNC who were receiving intensity-modulated RT (IMRT) plus cisplatin chemotherapy. The study demonstrated a 16% relative reduction in the incidence of SOM (primary endpoint) through the end of the study treatment period (approximately 7 weeks) with avasopasem manganese compared to placebo (54% versus 64%, respectively; p=0.045) and a 56% relative reduction in SOM duration (median, 8 versus 18 days, respectively; p=0.002). In addition, after 1 year of follow-up, tumor outcomes and OS were comparable between the avasopasem manganese and placebo groups. Notably, according to a predefined exploratory analysis, after 1 year of post-treatment follow-up, significantly fewer patients in the avasopasem manganese group developed CKD, a known risk of cisplatin, compared to those in the placebo group (10% versus 20%, respectively; p=0.0043).

Avasopasem manganese 90 mg was infused IV over 60 minutes before each daily (Monday–Friday) RT fraction.

PLACE IN THERAPY

Chemotherapy and RT create reactive oxygen species within cells leading to cell damage and mucositis.

Mucositis typically presents during the second or third week of RT and is characterized by erythema and ulceration of the mouth, but can occur throughout the entire GI tract. Approximately 70% of patients with HNC treated with cisplatin plus RT experience SOM (grade 3 or 4). Patients may experience pain and difficulty eating and swallowing warranting the need for enteral/parenteral nutrition, opioids for pain control, and interruption of cancer therapy, if severe. In addition, in immunocompromised patients, SOM may lead to infections, including bacteremia, resulting in hospitalization and increased mortality.

Symptomatic management of mucositis includes basic oral hygiene, dietary modifications, topical agents, and analgesics. Various types of mouthwash found in the literature are suggested to alleviate pain due to oral mucositis (OM). These include oral solutions of doxepin, hydrogen peroxide, normal saline, and baking soda, as well as “miracle” mouthwashes containing combinations of an antacid, diphenhydramine, viscous lidocaine, dexamethasone, nystatin, and/or an antibiotic. The Multinational Association of Supportive Care in Cancer (MASCC) recommends benzydamine mouthwash (anti-inflammatory; not available in the US), intraoral photobiomodulation (PBM) with low-level laser therapy, oral glutamine (off-label), and honey for the prevention of OM in patients with HNC receiving RT and chemotherapy. Topical morphine 0.2% mouthwash is also suggested to treat pain associated with OM. Several mucoadhesive products, considered devices by the FDA, are approved to manage OM symptoms, including Gelclair®, Mugard®, Mucotrol™, and Caphosol®; however, these agents have not been adequately evaluated in well-designed clinical trials.

If approved, avasopasem manganese will be the first systemic therapy for RT-induced SOM. Initial approval is expected for use in patients with HNC. Dusquetide, an innate defense regulator administered via a 4-minute IV infusion every 2 weeks, is in phase 3 trials for OM in patients with HNC receiving RT plus chemotherapy.

FDA APPROVAL TIMELINE August 9, 2023

 Breakthrough Therapy

 Fast Track

 Priority Review

FINANCIAL FORECAST (reported in millions)

The financial forecast for avasopasem manganese is not currently available.

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HEMATOLOGY

concizumab SC Novo Nordisk PROPOSED INDICATIONS

Hemophilia A and B prophylaxis in patients with inhibitors

CLINICAL OVERVIEW

Concizumab, an anti-tissue factor pathway inhibitor (TFPI) monoclonal antibody, improves thrombin generation.

The randomized, open-label, parallel-group, phase 3 EXPLORER 7 trial (NCT04083781) evaluated prophylactic treatment with concizumab in 132 male patients ≥ 12 years of age with hemophilia A or B with inhibitors.

The study reported an estimated mean annualized bleeding rate (ABR) of 1.7 with concizumab compared to 11.8 with no prophylaxis (ABR ratio, 0.14; p<0.001). The median ABR was 0 for concizumab and 9.8 with no prophylaxis. Notably, the EXPLORER clinical program, including the EXPLORER 7 trial, was temporarily placed on hold due to reports of 3 non-fatal thrombotic events in patients who had risk factors at baseline and had used concomitant hemostatic medication (recombinant factor VIIa or VIII). The program resumed after new safety measures were adopted to mitigate this risk. No thrombotic events or other safety concerns were reported after EXPLORER 7 was restarted.

Concizumab was self-administered as a SC injection via a prefilled pen. It was administered as a loading dose of 1 mg/kg on day 1, followed by 0.2 mg/kg once daily starting on day 2. Over the next 5 to 8 weeks, the dose could be adjusted to 0.25 mg/kg or 0.15 mg/kg depending on the concizumab plasma concentrations.

PLACE IN THERAPY

Hemophilia is an X-linked congenital bleeding disorder that affects up to 33,000 males in the US. Hemophilia is characterized by chronic spontaneous bleeding into muscles and joints that can progress to debilitating arthropathy. Hemophilia A and B exhibit low or missing levels of clotting factors VIII and IX, respectively.

Hemophilia A and B comprise about 80% and 20% of all cases of hemophilia, respectively. Approximately 35% of patients with hemophilia A and 3% with hemophilia B will develop neutralizing antibodies, or inhibitors, to factor products, which make the condition more difficult to treat.

The bypassing agents, recombinant factor VIIa (e.g., Novoseven RT®, Sevenfact®) and activated prothrombin complex concentrates (e.g., Feiba®), are SOC for managing hemophilia in patients with inhibitors. A hemostatic efficacy of about 80% has been reported with these agents but may vary and change over time. The first FDAapproved non-factor product, emicizumab-kxwh (Hemlibra®), mimics factor VIIIa cofactor and is indicated for routine prophylaxis for patients (newborns through adulthood) with hemophilia A with or without inhibitors.

This bispecific factor IXa/X antibody is self-administered SC every 1, 2, or 4 weeks.

If approved, concizumab will be the first anti-TFPI antibody available in the US. In clinical trials, it significantly reduced ABR compared to no prophylaxis. Market uptake could be a challenge due to the need for daily SC injections and by reports of non-fatal thrombosis that led to the halting of early studies. Although, concizumab could find a niche in patients with hemophilia B – a population in which emicizumab-kxwh is not indicated.

Novo Nordisk plans to submit concizumab for FDA approval for hemophilia A and B without inhibitors in 2023.

Other non-factor products in phase 3 studies for both hemophilia A and B include the anti-TFPI antibody marstacimab and the small interfering RNA fitusiran. In addition, the first gene therapy for adults with severe hemophilia A, valoctocogene roxaparvovec, is awaiting the FDA decision by June 30, 2023, as a 1-time dose.

FDA APPROVAL TIMELINE September 2023

 Breakthrough Therapy

 Orphan Drug

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$8

$21

$35

$49

$59

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ONCOLOGY

Large B cell lymphoma epcoritamab SC Genmab/Abbvie PROPOSED INDICATIONS

Relapsed/refractory (R/R) large B cell lymphoma (LBCL) as ≥ 3rd-line therapy

CLINICAL OVERVIEW

Epcoritamab is a cluster of differentiate 20 (CD20) x cluster of differentiate 3 (CD3) T cell engaging bispecific antibody.

The open-label, phase 1/2 EPCORE NHL-1 trial (NCT03625037) evaluated epcoritamab in 157 adults with R/R LBCL, including diffuse LBCL (DLBCL), high-grade B cell lymphoma, primary mediastinal LBCL, or follicular lymphoma. Enrolled patients received ≥ 2 prior lines of systemic therapy (median, 3; range, 2 to 11), including an anti-CD20 antibody. Among the patients in the study, 39% received prior CAR T cell therapy and 20% had prior autologous SCT. In the phase 2 period, at a median follow-up of 10.7 months (range, 0.3 to 17.9), the ORR (primary endpoint) was 63%, with a complete response rate of 39%. A response was seen in patients with and without prior CAR T therapy (ORR, 54%, and 69%, respectively; complete response rate, 34%, and 42%, respectively). The median time to complete response was 2.7 months (range, 1.2 to 11.1). The estimated median DOR was 12 months (range, 0.3 to 17.9). Among patients who achieved a complete response, 89% maintained that response at 9 months, and the PFS was not reached. CRS occurred in 49.7% of patients, with 2.5% of patients experiencing grade 3 CRS. In the phase 2 portion of the EPCORE NHL-1 trial, to reduce the risk of serious CRS, patients received priming and intermediate doses of epcoritamab followed by full doses of 48 mg in 28-day cycles. The dosing frequency in cycles 1 through 3 was once weekly, in cycles 4 through 9 was every 2 weeks, and in cycles 10 and beyond was every 4 weeks. Treatment was continued until disease progression or unacceptable toxicity occurred. To further mitigate CRS severity, patients were hospitalized after epcoritamab doses 1 through 4.

The ongoing, open-label, phase 3 EPCORE DLBCL-1 study (NCT04628494) is comparing epcoritamab with investigator’s choice of rituximab ± bendamustine or gemcitabine + oxaliplatin in patients with R/R DLBCL who failed or are ineligible for autologous SCT. Primary data for the study are anticipated in mid-2023.

FDA APPROVAL TIMELINE May 21, 2023

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$47

$176

$331

$476

$623

.

glofitamab IV Genentech PROPOSED INDICATIONS

Relapsed/refractory (R/R) large B cell lymphoma (LBCL) as ≥ 3rd-line therapy

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glofitamab cont.

CLINICAL OVERVIEW

Glofitamab is a CD20 x CD3 T cell engaging bispecific antibody.

An open-label, phase 1/2, dose-escalation study (NCT03075696) evaluated glofitamab monotherapy in 154 adults with R/R DLBCL who had received ≥ 2 prior lines of therapy. Approximately one-third of patients had prior CAR T cell therapy. At a median follow-up of 12.6 months (range, 0.1 to 22.1 months), 39% of patients achieved the primary endpoint of complete response. Among those with prior CAR T cell therapy, 35% achieved complete response. Patients achieved a complete response in a median of 42 days. The ORR was 52%, and the 12-month PFS and OS were 37% and 50%, respectively. The most common TEAEs were CRS (63%), neutropenia (38%), anemia (31%), and thrombocytopenia (25%). Serious (grade ≥ 3) events were reported in 62% of patients, including CRS (4%) and neurologic events (3%).

Glofitamab monotherapy was administered IV every 2 or 3 weeks for up to 12 cycles. Patients received stepup doses of 2.5 mg and 10 mg, followed by 30 mg on day 1 of cycles 2 through 12. In addition to step-up dosing, patients received a single dose of obinutuzumab (1,000 mg) 7 days prior to glofitamab treatment to mitigate CRS

FDA APPROVAL TIMELINE July 1, 2023

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$0

$96

$136

$202

$245

PLACE IN THERAPY

DLBCL is the most common lymphoid malignancy in adults with over 25,000 new cases diagnosed in the US each year. It is an aggressive mature B cell lymphoma that involves the lymph nodes and often affects the spleen, liver, bone marrow, and other organs. The average age at diagnosis is 64 years. The 5-year survival rate is 64.6%.

Approximately 30% to 40% of DLBCL cases will relapse or progress after initial SOC therapy (R-CHOP). The choice of 2nd-line pharmacotherapy depends on the response to initial therapy, the timing of relapse, and eligibility for HSCT. Preferred 2nd-line regimens may contain platinum-based chemotherapy (± rituximab), CAR T cell therapy, the CD79b-directed ADC polatuzumab vedotin-piiq (Polivy®), or the CD19-directed cytolytic antibody tafasitamab-cxix (Monjuvi®). Current 3rd-line and subsequent therapy options include CAR T therapy, the CD19-directed ADC loncastuximab tesirine-lpyl (Zynlonta®), and the nuclear export inhibitor selinexor (Xpovio®).

HSCT and CAR T cell therapy are proven advancements in the treatment of R/R DLBCL; however, some patients may again relapse or may be ineligible for these therapies. SC-administered epcoritamab and IV glofitamab are bispecific antibodies that bind to CD3 on T cells and CD20 on B cells to induce T cell-mediated tumor cell death. If approved, epcoritamab and glofitamab will provide important targeted therapies for use in heavily treated patients with R/R LBCL. Unlike CAR T, both agents require more than 1 dose. These off-the-self agents are also distinguished from CAR T in that they do not require the collection and genetic modification of patient cells; further, patients are not burdened with traveling to a major academic center for treatment as with CAR T. Other CD3/CD20 bispecific agents that are in late-phase development for R/R LBCL include mosunetuzumab (phase 3), odronextamab (phase 2), and plamotamab (phase 2).

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IMMUNOLOGY

etrasimod oral Pfizer PROPOSED INDICATIONS

Moderate to severe ulcerative colitis (UC)

CLINICAL OVERVIEW

Etrasimod is a selective sphingosine-1-phosphate (S1P) receptor modulator.

Etrasimod was evaluated in the double-blind, placebo-controlled ELEVATE UC 12 (NCT03996369; n=354) and ELEVATE UC 52 (NCT03945188; n=433) trials in patients ≥ 16 years of age with moderate to severe UC. Approximately one-third of patients in both trials had prior treatment with a biologic agent or Janus kinase (JAK) inhibitor. Patients were randomized 2:1 to etrasimod or placebo. The primary endpoint in both trials was clinical remission as measured by the modified Mayo score (MMS), a composite score consisting of participantreported symptoms (stool frequency [SF] and rectal bleeding [RB]), and a centrally read endoscopic score (ES). Clinical remission was defined as an SF subscore = 0 (or = 1 with a ≥ 1-point decrease from baseline), RB subscore = 0, and ES ≤ 1. In ELEVATE UC 12, at 12 weeks, clinical remission of UC was achieved in significantly more patients treated with etrasimod (25%) than placebo (15%; p=0.026). ELEVATE UC 52 reported significantly higher remission rates with etrasimod compared to placebo at week 12 (27% versus 7%, respectively; p<0.0001) and at week 52 (32% versus 7%; p<0.0001). TEAEs reported more often with etrasimod than placebo were headache, UC worsening, COVID-19 infection, dizziness, pyrexia, arthralgia, abdominal pain, and nausea. No serious bradycardia and atrioventricular block were reported. Two events of symptomatic bradycardia were reported and resulted in study withdrawal.

In both studies, the dosage of etrasimod was 2 mg orally once daily.

PLACE IN THERAPY

UC is a chronic inflammatory disease affecting the large bowel (colon and rectum) that is characterized by superficial infiltration of the bowel wall by inflammatory WBCs, resulting in mucosal ulcerations and crypt abscesses. Nearly 1,000,000 people in the US are affected by UC. Onset can occur at any age, but peaks between the ages of 15 to 30 years. Patients experience symptoms such as bloody diarrhea, mucous, bowel urgency, abdominal pressure and cramping, and weight loss that may take on a relapsing/remitting pattern.

Moderate to severe UC is considered when patients are dependent on or refractory to corticosteroids, exhibit ulcers upon endoscopic assessment, or are at high risk for colectomy. Based on current guidelines, 1st-line treatment for moderate to severe UC includes injectable biologic agents, such as TNF-inhibitors (adalimumab [Humira, biosimilar], golimumab [Simponi®], infliximab [Remicade®, biosimilars]), the integrin receptor antagonist vedolizumab (Entyvio®), and the IL-12/IL-23 antagonist ustekinumab (Stelara®). The oral JAK inhibitor tofacitinib (Xeljanz®, Xeljanz® XR) is also used as 1st-line treatment. Long-term management of patients with moderate to severe disease can include biologic agents, tofacitinib, or immunomodulators (e.g., azathioprine, methotrexate).

The S1P receptor modulator ozanimod (Zeposia®) was FDA-approved in 2021 for moderate to severe UC in adults and is not addressed in current guidelines.

If approved, etrasimod will compete directly with ozanimod in the UC setting. Both agents are administered orally once daily. Notably, ozanimod requires dose titration to prevent bradycardia and atrioventricular block. It is also contraindicated in patients who experienced prior select CV events (e.g., MI, stroke, unstable angina). In clinical trials, etrasimod was not associated with serious bradycardia or atrioventricular block, and dose titration was not required. In non-comparative clinical trials, 24.8% to 27% of patients treated with etrasimod achieved clinical remission of UC (at week 12) compared to 18% of patients treated with ozanimod.

FDA APPROVAL TIMELINE July to December 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$21

$120

$226

$363

$497

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IMMUNOLOGY

lebrikizumab SC Eli Lilly PROPOSED INDICATIONS

Moderate to severe atopic dermatitis (AD)

CLINICAL OVERVIEW

Lebrikizumab is an interleukin-13 (IL-13) inhibitor.

The double-blind, placebo-controlled, phase 3 ADvocate 1 (NCT04146363; n=424) and Advocate 2 (NCT04178967; n=472) trials evaluated lebrikizumab monotherapy in patients ages ≥ 12 years (weighing ≥ 40 kg) with moderate to severe AD. Patients were randomized 2:1 to lebrikizumab 500 mg SC initially and at 2 weeks, followed by lebrikizumab 250 mg SC or placebo every 2 weeks. Co-primary endpoints in each trial were an IGA of 0 or 1 (clear or almost clear skin) with a reduction of ≥ 2 points from baseline and EASI-75.

In both trials at week 16, significantly more patients on lebrikizumab achieved the IGA endpoint compared to those given placebo (ADvocate 1: 43.1% versus 12.7%, respectively; ADvocate 2: 33.2% versus 10.8%, respectively; p<0.001 for both). Similarly, lebrikizumab led to higher rates of EASI-75 response at week 16 (ADvocate 1: 58.8% versus 16.2%, respectively; ADvocate 2: 52.1% versus 18.1%, respectively; p<0.001 for both). Significant improvements in skin clearing and itching were seen as early as week 4. At week 16, patients who responded to lebrikizumab were re-randomized to lebrikizumab 250 mg every 2 weeks or every 4 weeks or placebo for an additional 36 weeks. At week 52, the studies demonstrated similar and durable responses with lebrikizumab given every 2 and every 4 weeks (IGA 0/1 response rate: ADvocate 1, 76% versus 74%, respectively, and ADvocate 2, 65% versus 81%, respectively; EASI-75 response rate: ADvocate 1, 79% versus 79%, respectively, and ADvocate 2, 77% versus 85%, respectively). Lebrikizumab was well tolerated. The most common TEAEs were conjunctivitis, nasopharyngitis, and headache.

The double-blind, parallel-group, phase 3 ADhere trial (NCT0425033; n=211) evaluated every-2-week SC dosing (after an initial loading dose) of lebrikizumab in combination with a topical corticosteroid (TCS) compared to TCS therapy alone in patients ages ≥ 12 years (weighing ≥ 40 kg) with moderate to severe AD. Medium potency (triamcinolone 0.1%) and low potency (hydrocortisone 1%) TCS creams were used in the study. The patients could taper, stop, and resume TCS therapy as needed. At week 16, significantly more patients achieved an IGA of 0/1 and EASI-75 (co-primary endpoints) in the lebrikizumab/TCS group compared to the placebo/TCS group (IGA 0/1: 41.2% versus 22.1%, respectively [p=0.01]; EASI-75: 69.5% versus 42.2%, respectively [p<0.001]). Lebrikizumab plus a TCS was well tolerated.

PLACE IN THERAPY

AD affects an estimated 31.6 million people in the US, including 9.6 million children and adolescents.

Approximately 30% and 40% of cases in pediatrics and adults, respectively, are moderate to severe. The onset of AD occurs before age 6 years in about 80% of cases. While several systemic DMTs are available to treat moderate and severe AD, topical emollients, corticosteroids, and immunomodulators remain important options for treating AD; however, long-term continuous use of TCSs and immunomodulators is limited by TEAEs. If approved, lebrikizumab will be the second biologic for AD that solely targets IL-13, following tralokinumab’s (Adbry™) approval in December 2021 for use in adults only. Lebrikizumab will compete with tralokinumab and dupilumab (Dupixent®). Dupilumab inhibits IL-4 and IL-13 signaling and is indicated for moderate to severe AD in patients ≥ 6 months of age. Lebrikizumab, tralokinumab, and dupilumab have similar maintenance dosing regimens (every 2 or 4 weeks). Non-comparative clinical trials report greater responses with lebrikizumab monotherapy (IGA difference from placebo range, 22% and 30%; EASI-75 difference from placebo range, 43% and 33%) compared to tralokinumab monotherapy (IGA difference from placebo range, 9% to 12%; EASI-75 difference from placebo range, 12% to 22%). Similar responses were reported in non-comparative trials with lebrikizumab and dupilumab (dupilumab IGA difference from placebo, 28%; EASI-75 difference from placebo range, 32% to 36%).

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lebrikizumab cont.

FDA APPROVAL TIMELINE September 2023  Fast Track

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$107

$347

$548

$762

$976

.

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OPHTHALMOLOGY

lotilaner ophthalmic Tarsus PROPOSED INDICATIONS Demodex blepharitis

CLINICAL OVERVIEW

Lotilaner ophthalmic solution is a gamma-aminobutyric acid-gated chloride channel (GABA-Cl) inhibitor designed to eradicate Demodex mite infestation.

The randomized, vehicle-controlled, double-masked, phase 2b/3 Saturn-1 (NCT04475432; n=421) and phase 3 Saturn-2 (NCT04784091; n=412) trials evaluated lotilaner in patients with Demodex blepharitis. Enrolled participants displayed > 10 collarettes (cylindrical dandruff) per eyelid, at least mild upper eyelid margin erythema, and ≥ 1.5 mites/lash on upper/lower eyelids. In both trials, significantly more patients treated with lotilaner achieved the primary endpoint of complete collarette cure (0 to 2 collarettes per upper eyelid) at day 43 compared to patients who received vehicle (Saturn 1: 44% versus 7.4%, respectively; Saturn 2: 56% versus 13%, respectively; p<0.0001 for both). In addition, significantly more patients achieved the following secondary endpoints with lotilaner compared to the vehicle: mite eradication (Saturn 1: 67.9% versus 17.6%; respectively; Saturn 2: 52% and 14%, respectively; p<0.0001 for both), complete eyelid erythema cure (Saturn 1: 19.1% versus 6.9%, respectively; Saturn 2: 31.1% versus 9%; p<0.0001 for both), and complete composite (collarette + erythema) cure (Saturn 1: 13.9% versus 1%, respectively; Saturn 2: 19.2% versus 4%, respectively; p < 0.0001 for both). The Saturn 1 study also reported that significant improvement was observed as early as day 8 in the primary and some secondary endpoints. Lotilaner was well tolerated. The most common TEAE was instillation site pain/burning/stinging (7.9%). In a phase 2 trial, collarette grade and mite density improvements were maintained 2 months after stopping therapy.

Lotilaner 0.25% ophthalmic solution was administered as 1 drop in each eye twice daily for 43 days, with no eyelid intervention such as eyelid hygiene.

PLACE IN THERAPY

Demodex is a common parasite of the skin and inhabits the eyelashes, eyebrows, and face. As detected on slit-lamp examination, the presence of waxy deposits called collarettes, or cylindrical dandruff or “sleeves,” on eyelashes is an indicator of Demodex infestation. Collarettes contain lipids, keratin, Demodex eggs, and/ or dead Demodex mites. Common symptoms of Demodex blepharitis include swollen, red, irritated eyelids, and ocular itching. Patients may also experience eyelash loss, dry eyes, and recurrent styes or chalazia. It is estimated that Demodex blepharitis affects 10 million people in the US. If not properly diagnosed, patients may be prescribed medications to treat ocular sequalae of the condition (e.g., dry eye), rather than the underlying cause, and if left untreated it can lead to serious corneal conditions.

There are no FDA-approved treatments for Demodex blepharitis. Therapies cited in the literature provide mixed results. The topical application of tea tree oil (weekly scrub or daily shampoo) or its active ingredient, terpinen-4-ol (wipe), is commonly used. Off-label use of oral or topical ivermectin, topical metronidazole, permethrin, or crotamiton has also appeared in the literature.

Lotilaner is a topical antiparasitic agent that paralyzes and eradicates Demodex mites. If approved, it will be the first agent indicated in the US to treat Demodex blepharitis. In clinical studies, it resulted in a clinical cure and a favorable safety profile. Notably, the active ingredient lotilaner is approved as oral tablets (brand Credelio®) for the prevention of fleas and ticks in dogs and cats. Oral or other systemic formulations of lotilaner are not approved for use in humans.

FDA APPROVAL TIMELINE August 25, 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales*

$3

$74

$180

$297

$414

* Forecasted sales reported for all investigational indications

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ONCOLOGY

momelotinib oral GlaxoSmithKline PROPOSED INDICATIONS Myelofibrosis (MF)

CLINICAL OVERVIEW

Momelotinib is an inhibitor of Janus kinase (JAK) 1 and 2 and activin A receptor, type 1 (ACVR1). JAK 1/2 inhibition is designed to improve myelofibrosis symptoms and splenomegaly, and ACVR1 inhibition decreases circulating hepcidin, which contributes to anemia.

The randomized, double-blind, parallel-group, phase 3 MOMENTUM trial (NCT04173494) compared momelotinib and danazol in 195 symptomatic, anemic (Hb < 10 g/dL) adults with primary myelofibrosis (PMF), post-polycythemia vera (PV) myelofibrosis, or post-essential thrombocythemia (ET) myelofibrosis. All patients were previously treated with a JAK inhibitor (ruxolitinib) and had palpable splenomegaly at baseline. In the momelotinib and danazol groups, 72% and 58% of patients, respectively, completed 24 weeks of treatment, with adverse reactions as the most common reason for discontinuation. At 24 weeks, momelotinib proved to be superior to danazol in the primary endpoint of the proportion of patients achieving a ≥ 50% reduction from baseline in the Myelofibrosis Symptom Assessment Form Total Symptom Score (MSA TSS) (25% versus 9%, respectively; p=0.0095) and the secondary endpoints of ≥ 25% reduction in spleen volume (40% versus 6%, respectively; p<0.0001) and ≥ 35% reduction in spleen volume (23% versus 3%, respectively; p=0.0006). In addition, momelotinib was non-inferior to danazol, based on transfusion independence at 24 weeks (31% versus 20%, respectively; p=0.0064). The most common serious (grade ≥ 3) TEAEs in each group were anemia (61% versus 75%, respectively) and thrombocytopenia (28% versus 26%, respectively).

The randomized, double-blind, phase 3 SIMPLIFY-1 trial (NCT01969838) compared momelotinib and the JAK inhibitor ruxolitinib in 432 JAK inhibitor-naïve adults with PMF, post-PV myelofibrosis, or post-ET myelofibrosis.

Enrollees had anemia (Hb < 10 g/dL), symptomatic splenomegaly or hepatomegaly, and/or were unresponsive to available non-JAK inhibitor myelofibrosis therapy. At week 24, momelotinib was shown to be non-inferior to ruxolitinib, based on the proportion of patients who achieved the primary endpoint of ≥ 35% reduction in spleen volume (26.5% and 29%). However, fewer patients who received momelotinib compared to those given ruxolitinib achieved a ≥ 50% reduction in the MSA TSS, a secondary endpoint (28.4% versus 42.2%, respectively). Notably, more patients in the momelotinib group were transfusion independent at week 24 (66.5%) compared to the ruxolitinib group (49.3%; nominal p<0.001). The median rate of RBC transfusion through week 24 was 0 units/month with momelotinib compared to 0.4 units/month with ruxolitinib (nominal p<0.001).

The randomized, open-label, phase 3 SIMPLIFY-2 trial (NCT02101268) compared momelotinib to the best available therapy (BAT) for MF, which included hydroxyurea, anagrelide, corticosteroids, hematopoietic growth factors, immunomodulating agents, androgens, and interferon. Patients (n=156) enrolled had PMF, post-PV myelofibrosis, or post-ET myelofibrosis with palpable splenomegaly and either anemia or thrombocytopenia.

All patients were previously treated with ruxolitinib. At 24 weeks, 7% of patients in the momelotinib group and 6% in the BAT group achieved the primary endpoint of ≥ 35% reduction in spleen volume from baseline (p=0.9); momelotinib was not superior to BAT. In the phase 3 trials, the doses studied were momelotinib 200 mg orally once daily, ruxolitinib 20 mg orally twice daily, and danazol 300 mg orally twice daily. Dose interruptions or reductions due to thrombocytopenia, neutropenia, or other toxic effects were permitted.

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momelotinib cont.

PLACE IN THERAPY

Myelofibrosis (MF) is a rare, myeloproliferative neoplasm (MPN) characterized by the overproduction of abnormal hematopoietic stem cells and scarring (fibrosis) within the bone marrow. Anemia and thrombocytopenia are usually present, particularly as the disease progresses. Notably, a Hb < 10 g/dL is an unfavorable prognostic marker. Splenomegaly is also common. It is estimated that 13,000 people in the US have MF. The median age at diagnosis is 65 years, although it can occur at any age. Most often (> 50%) MF is idiopathic (primary) in nature, but it can also be secondary to a malignant or hematologic condition, such as polycythemia vera and essential thrombocythemia (10% to 15%). Primary MF may progress slowly, and patients may be asymptomatic for many years; however, some cases may transform into acute myeloblastic leukemia (AML). Several gene mutations have been identified with MPNs. Approximately 60% of patients with primary MF have a V617F JAK2 gene mutation, which is associated with a median survival period of 9 years.

In asymptomatic MF patients, clinicians may adopt a watch-and-wait approach. In early primary MF, pegylated interferon may reduce bone marrow fibrosis and spleen size in low-risk patients. Allogeneic HSCT may be curative in patients with advanced MF, but the associated morbidity and mortality risks limit its use in patients with advanced disease. Palliative therapy for MF includes androgens, erythropoietin, hydroxyurea, thalidomide, lenalidomide, corticosteroids, radiation therapy, chemotherapy, splenectomy or splenic embolization, and RBC and/or platelet transfusions. The only FDA-approved treatments for MF are the oral JAK inhibitors. This includes ruxolitinib (Jakafi®; JAK1/2 inhibitor; therapy of choice) and fedratinib (Inrebic®; selective JAK2 inhibitor), both of which are indicated for the treatment of intermediate- or high-risk primary or secondary MF in patients with platelet count > 50,000/µL, and pacritinib (Vonjo™; JAK2 and FLT3 inhibitor), which is approved for use in patients with platelet count < 50,000/µL. For patients with anemia associated with MF, after ruling out other causes for anemia (e.g., bleeding or iron, vitamin B12, or folate deficiencies), the NCCN recommends erythropoietin stimulating agents (ESAs), danazol, lenalidomide, or thalidomide, depending on serum erythropoietin levels.

Momelotinib is a first-in-class inhibitor of both JAK1/2 and ACVR1. If approved, it will be the first JAK inhibitor to target myelofibrosis in patients with anemia. In clinical trials, momelotinib led to greater improvements in MF symptoms and spleen volume compared to danazol, but not compared to ruxolitinib. A greater improvement in anemia (transfusion independence) was reported with momelotinib over both danazol and ruxolitinib.

FDA APPROVAL TIMELINE June 16, 2023  Fast Track

 Orphan Drug

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$11

$52

$90

$130

$169

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INFECTIOUS DISEASE

nirsevimab IM AstraZeneca/Sanofi PROPOSED INDICATIONS

Prevention of respiratory syncytial virus (RSV) lower respiratory tract infection (LRTI) in newborns and infants during their first RSV season and in children ≤ 24 months of age who are at risk for severe RSV disease through their second RSV season

CLINICAL OVERVIEW

Nirsevimab is a long-acting recombinant human IgG1kappa monoclonal antibody that binds the F1 and F2 subunits of the RSV fusion (F) protein, thereby blocking viral entry into the host cell.

The international, double-blind, placebo-controlled, phase 3 MELODY trial (NCT03979313) evaluated nirsevimab in healthy infants born at a gestational age (GA) of ≥ 35 weeks 0 days who were ≤ 1 year of age and who were entering their first RSV season. Patients were randomized 2:1 to a single IM dose of nirsevimab or matching placebo that was administered before the start of the RSV season. The primary endpoint was medically attended RSV-associated LRTI through 150 days after the dose. Based on published interim data that included 1,490 patients, the primary endpoint occurred in 1.2% of patients in the nirsevimab group and 5% in the placebo group, demonstrating a 74.5% efficacy against medically attended RSV-associated LRTI for nirsevimab. Nirsevimab was generally well tolerated; however, serious adverse events were reported in 6.8% and 7.3% of patients in the nirsevimab and placebo groups, respectively. Three deaths occurred between day 140 and 361, all among patients in the nirsevimab group; 2 deaths were due to gastroenteritis and 1 death due to an unknown cause. An updated analysis that included data from a total of 3,012 patients reported similar findings, with a 76.4% efficacy against medically-attended RSV-associated LRTI with nirsevimab. In the full cohort, nirsevimab also demonstrated a 76.8% efficacy against RSV LRTI-related hospitalization, a 78.6% efficacy against very severe medically attended RSV LRTI, and a favorable tolerability profile.

In a post hoc analysis of pooled data from the MELODY trial and a phase 2b trial, RSV neutralizing antibodies were detected in blood from nirsevimab-treated infants 151 days after the dose. Levels were approximately 50 times higher than at baseline and remained over 19 times higher than levels in placebo-treated infants through day 361.

The double-blind, phase 2/3 MEDLEY trial (NCT03959488) compared the safety and pharmacokinetics of nirsevimab and palivizumab in a total of 925 preterm infants (born at GA ≤ 35 weeks) OR infants with chronic lung disease (CLD) or congenital heart disease (CHD) of prematurity. All patients were at high risk for RSV LRTI and eligible for palivizumab therapy. Prior to RSV season 1, all patients were randomized 2:1 to nirsevimab (1 weight-based dose followed by 4 monthly placebo doses) or palivizumab (15 mg/kg for 5 monthly doses).

Patients with CLD or CHD also received the study drug prior to RSV season 2. Those originally assigned to nirsevimab continued with nirsevimab, and those originally assigned to palivizumab were re-randomized to either nirsevimab or palivizumab. The study showed similar safety and tolerability profiles between both agents. However, 2 serious TEAEs were reported in the nirsevimab group; heparin-induced thrombocytopenia in an infant with CHD and maculopapular rash following a placebo dose. In addition, 4 (0.6%) infants receiving nirsevimab and 3 (1%) receiving palivizumab had medically attended RSV LRTI. In both clinical trials, nirsevimab was administered IM as 50 mg in patients weighing < 5 kg or 100 mg in patients weighing ≥ 5 kg prior to the start of the RSV season.

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nirsevimab cont.

PLACE IN THERAPY

RSV is a common contagious respiratory virus that usually causes mild, cold-like symptoms lasting 1 to 2 weeks. In the US each year, RSV infection leads to approximately 2.1 million outpatient visits, 58,000 to 80,000 hospitalizations, and 100 to 300 deaths in children < 5 years of age. Children who are at particular risk for serious RSV infections are premature infants, children < 2 years of age with CLD or CHD of prematurity, and children who are immunocompromised or have neuromuscular disorders.

Typically, the RSV season in the US occurs during approximately November through April but may vary by region. However, the regular RSV circulation pattern was disrupted by the COVID-19 pandemic, leading to interseasonal variability. There is no FDA-approved medication to treat RSV or vaccine to prevent RSV illness in the pediatric population. Treatment of mild cases consists of symptom management, including use of antipyretics and analgesics; however, palivizumab (Synagis®), an RSV F protein inhibitor monoclonal antibody, is the only agent FDA-approved for the prevention of RSV. It is indicated for use only in infants and young children who are at high risk for serious RSV LRTI (preterm infants < 6 month of age and children ≤ 24 months of age with CLD or CHD of prematurity). Palivizumab is typically administered once monthly per IM injection for 5 consecutive doses beginning just before the start of the RSV season, as based on the American Academy of Pediatrics guidelines. However, during the period of an unusual RSV circulating pattern observed during the COVID-19 pandemic, additional or off-season doses may have been warranted.

If approved, nirsevimab will be the first single-dose option to prevent RSV LRTI. It could also provide protection for a broader pediatric population, including healthy infants, whereas palivizumab requires monthly dosing for use only in high risk patients. Nirsevimab demonstrated comparable safety and tolerability as palivizumab and may provide enhanced viral efficacy against RSV-related hospitalization in at-risk children, as suggested in the MEDLEY trial and non-comparison study data.

Other products in the pipeline for RSV include Merck’s monoclonal antibody clesrovimab, which is in phase 3 trials for use in patients < 1 year of age who are at increased risk for severe RSV infection. Pfizer’s RSV vaccine candidate (PF-06928316) has been submitted to the FDA for maternal administration during pregnancy to protect the infant from RSV after birth and has the potential to be the first RSV vaccine approved to protect infants from RSV LRTI. Novavax’s RSV vaccine is also in phase 3 research for maternal administration.

RSV can also cause serious illness in older adults. Two RSV vaccines by GlaxoSmithKline (GSK3844766A) and Pfizer (PF-06928316) have been submitted to the FDA for prevention of RSV in older adults (≥ 60 years of age). The FDA decisions for these vaccines are anticipated in May 2023.

FDA APPROVAL TIMELINE July to September 2023

 Breakthrough Therapy

 Fast Track

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$144

$364

$547

$718

$823

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INFECTIOUS DISEASE

olorofim oral F2G

PROPOSED INDICATIONS

Invasive fungal infections in patients who have limited or no treatment options

CLINICAL OVERVIEW

Olorofim is an orotomide antifungal. It inhibits the dihydroorotate dehydrogenase (DHODH) enzyme in the fungal pyrimidine synthesis pathway resulting in cell death. It has shown in vitro activity against Aspergillus species (spp). (including azole-resistant and cryptic species), rare molds (e.g., Lomentospora prolificans, Scedosporium spp., Scopulariopsis spp.), and dimorphic fungi (e.g., Histioplasma spp., Blastomyces spp., Coccidioides spp.).

The ongoing, open-label, phase 2b FORMULA-OLS trial (NCT03583164) evaluated olorofim in adults with resistant invasive fungal disease due to Lomentospora prolificans, Scedosporium spp., Aspergillus spp., or other resistant fungi lacking appropriate treatment options. Patients 16 or 17 years of age weighing ≥ 40 kg were also allowed study entry. Patients received olorofim for up to 90 days (median, 84 days). Approximately 75% of the first 100 patients had moderate to high levels of immunosuppression. Data from the first 100 patients revealed that at day 42, the ORR was 69%, including stable response. A complete or partial response was observed in 44% of patients. At days 42 and 84, the rates for all-cause mortality were 15% and 20%, respectively. Olorofim was generally well tolerated. Nausea, vomiting, and diarrhea were reported. Eight patients (8%) experienced drug-induced liver injury, a serious adverse effect possibly related to olorofim; 2 (2%) patients were required to discontinue therapy.

Olorofim was administered orally as an initial loading dose of 150 mg twice daily on day 1, followed by 90 mg twice daily for up to 90 days.

PLACE IN THERAPY

Invasive fungal infections cause considerable morbidity and mortality, particularly among immunocompromised patients. Antifungal agents that have been approved in the past 20 years to fight invasive fungal infections include azoles (e.g., extended-spectrum triazoles, voriconazole, posaconazole, and isavuconazole); amphotericin B lipid formulations; and the echinocandins. However, current antifungals have limitations, including limited dosage forms, drug-drug interactions, and significant adverse reactions. The increase in fungal pathogens resistant to current antifungals adds to the increased need for new treatment options.

Olorofim is a first-in-class orotomide antifungal. Its ability to reversibly inhibit fungal DHODH, a key enzyme in the biosynthesis of pyrimidines, leads to impairment of nucleic acid production and fungal cell lysis.

This mechanism of action allows for activity against isolates that are resistant to current treatment options.

Olorofim is active against many molds and dimorphic fungi, including species that are resistant to azoles and amphotericin B. However, it lacks activity against yeasts, including Candida spp. and Cryptococcus spp., and the Mucorales group. Olorofirm exhibits time-dependent fungicidal activity and wide tissue distribution, including the CNS. Olorofim is metabolized by multiple CYP450 enzymes, including CYP3A4; however, based on current evidence, it does not appear to have any effect on CYP450 enzymes, and therefore, may have limited potential for drug-drug interactions.

If approved, olorofim may be an important oral option for patients with invasive fungal infections, including patients with immunocompromise, limited treatment options, or with difficult-to-treat organisms. Olorofim does not possess broad-spectrum antifungal activity, but it may play a key role in treating multi-drug resistant fungal infections and/or endemic mycoses. F2G has initiated the phase 3 OASIS trial comparing olorofim with liposomal amphotericin B injection (Ambisome®) followed by SOC to treat invasive aspergillosis of the lower respiratory tract.

FDA APPROVAL TIMELINE June 17, 2023

 Breakthrough Therapy

 LPAD

 Orphan Drug

FINANCIAL FORECAST (reported in millions)

The financial forecast for olorofim is not currently available.

16 | MAGELLANRX.COM

 QIPD

BEHAVIORAL HEALTH

zuranolone oral Sage/Biogen PROPOSED INDICATIONS

» Major depressive disorder (MDD) » Postpartum depression (PPD)

CLINICAL OVERVIEW

Zuranolone is a neuroactive steroid that acts as a positive allosteric GABA-A receptor modulator. It is designed to rapidly rebalance dysregulated neuronal networks in the brain that affect mood, arousal, behavior, and cognition.

Zuranolone is being evaluated in adults with moderate or severe MDD across several randomized, doubleblind (unless otherwise noted), placebo-controlled, phase 3 clinical trials. Baseline HAMD-17 scores were ≥ 24 in WATERFALL and CORAL, ≥ 22 in MOUNTAIN, and ≥ 20 in SHORELINE. » The 42-day WATERFALL trial (NCT04442490; n=543) demonstrated significant improvement from baseline with zuranolone 50 mg compared to placebo in depressive symptoms, based on HAMD-17 score at day 15 (primary endpoint; difference, −1.7; p=0.0141). A significant difference compared to placebo was seen as early as day 3 (difference, −3; p<0.0001). A significant improvement in anxiety based on HAM-A score was also reported at day 8 (difference, -1.7; p=0.0011) and day 15 (difference, -1.4; p=0.0199). Numerical improvements were maintained through day 42. » The CORAL study (NCT04476030; n=440) evaluated zuranolone 50 mg co-initiated with a SOC antidepressant (SSRI, SNRI) in adults with MDD with elevated anxiety. The mean change in HAMD-17 score from baseline to day 3 (primary endpoint) was -8.9 with combination therapy, compared to -7 with SOC alone (p=0.0004). The mean changes over the 14-day treatment period were -11.7 and -10.1, respectively (the difference was not statistically significant). » The MOUNTAIN study (NCT03672175) enrolled patients with an HDRS-17 (a.k.a. HAMD-17) total score ≥ 22. Based on a post hoc analysis, patients who received zuranolone 30 mg (n=194) experienced significant improvement in HDRS-17 total score compared to those who received placebo (n=193) at days 3, 8, 12, and 15 (p<0.05 at all time points). In a blinded follow-up, the response was maintained in 74.5% of responders to zuranolone 30 mg at day 182. » The ongoing, open-label, 1-year longitudinal SHORELINE study (NCT03864614) assessed the need for retreatment after a 14-day course of zuranolone. Patients initiated therapy with zuranolone 30 mg (n=725) or 50 mg (n=52), which led to mean changes in their HAMD-17 scores from baseline to day 15 by -14.9 and -16, respectively. Among those who received zuranolone 30 mg, 71.6% achieved a response (HAMD-17 reduced by ≥ 50%) and 39.8% achieved remission (HAM-D ≤ 7). Likewise, initial doses of 50 mg resulted in a response rate of 74.9% and a remission rate of 40.2%. During the year-long study, retreatment was reported in 55.5% (mean, 1.9 treatments per year) of patients who responded to initial treatment with zuranolone 30 mg and 45.2% (range, 1 to 5 treatment courses) of those who responded to initial treatment with zuranolone 50 mg. Updated data revealed that the median times to first retreatment were 135 and 249 days with zuranolone 30 mg and 50 mg, respectively. No significant difference in response was observed between patients who were on pre-existing antidepressants and those who were not taking other antidepressant agents.

The randomized, double-blind, placebo-controlled ROBIN and SKYLARK trials evaluated zuranolone in adult women with PPD (HAMD-17 ≥ 26) that began no earlier than the third trimester and no later than the first 4 weeks following delivery. Additionally, patients were ≤ 6 months (ROBIN) or ≤ 12 months (SKYLARK) postpartum. » In ROBIN (NCT02978326; n=153), a statistically significant improvement in depressive symptoms as based on the HAMD-17 score was observed at day 15 (primary endpoint) with zuranolone 30 mg compared to placebo (−17.8 versus −13.6, respectively; p=0.003). Significant differences in HAMD-17 between zuranolone 30 mg and placebo were reported as early as day 3 (difference, -2.7; p=0.03) and at day 45 (difference, -4.1; p=0.003).

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zuranolone cont.

CLINICAL OVERVIEW cont.

» In SKYLARK (NCT04442503; n=195), a statistically significant and clinically meaningful improvement in the HAMD-17 score was observed at day 15 (primary endpoint) with zuranolone 50 mg compared to placebo (−15.6 versus −11.6, respectively; p=0.0007). More patients in the zuranolone group achieved response (reported at days 3 through 28; p<0.05 at all time points) or remission (reported on day 3 and day 45; p<0.05 at day 45) than in the placebo group. Significant improvements in anxiety were also reported with zuranolone compared to placebo.

In the MDD and PPD studies, zuranolone was dosed orally once daily for 14 consecutive days. The drug was generally well tolerated. The most common TEAEs included somnolence, dizziness, headache, fatigue, diarrhea, nausea, and sedation. In the SHORELINE trial, more frequent TEAEs were reported with the 50 mg dose compared to 30 mg, but the severity of TEAEs was similar between the groups. Weight gain, sexual dysfunction, and sleep disruption were not observed with zuranolone. One patient in the ROBIN trial zuranolone group experienced a serious adverse event of confusion. Similar efficacy and safety were reported in patients ≥ 65 years of age and younger populations with MDD.

PLACE IN THERAPY

MDD is defined as a depressed mood or loss of interest in daily activity with a majority of certain symptoms (e.g., difficulty sleeping, impaired concentration, negative self-worth) lasting ≥ 2 weeks. It is one of the most common mental disorders in the US, with an estimated 21 million adults reporting at least 1 MDD episode in 2020 and 14.8 million reporting severe impairment. Prevalence is higher among females than males (10.5% and 6.2%, respectively) and is highest among those 18 to 25 years of age. First-line pharmacotherapy for MDD includes an SSRI, SNRI, bupropion, or mirtazapine. Select atypical anti-psychotics may be added when an adequate response is not achieved after multiple trials with an antidepressant alone.

The CDC reports that about 1 in 8 women with a recent live birth experience symptoms of PPD, including intense feelings of sadness, anxiety, or despair that interfere with activities of daily living. PPD symptoms may occur up to 1 year after delivery. Primary treatment includes cognitive behavioral therapy and interpersonal therapy, particularly in patients who are breastfeeding; however, antidepressant medication, notably SSRIs in breastfeeding women, may be considered. Sage Therapeutics’ IV-administered neuroactive steroid GABA-A receptor positive modulator brexanolone (Zulresso®) is FDA-approved for the treatment of PPD in females ≥ 15 years of age. Brexanolone (Zulresso) is a schedule IV controlled substance that may cause excessive sedation and sudden loss of consciousness; therefore, it must be administered in a certified healthcare facility to allow for continuous monitoring during the 60-hour single-dose infusion and the patient and facility must enroll in the REMS program.

If approved, oral zuranolone could fill an unmet need in patients experiencing moderate to severe MDD. Its unique dosing, rapid onset (as early as 3 days), short duration of therapy (14 days), and proven safety and efficacy with retreatment could change how patients are treated for depression, as well as how follow-up care is administered. In contrast, currently available antidepressants (e.g., SSRIs, SNRIs) require long-term continuous use and take 6 to 8 weeks to realize their full effect. An exception to this is intranasal esketamine (Spravato®), an N-methyl D-aspartate (NMDA) receptor antagonist indicated in conjunction with an oral antidepressant for resistant MDD or MDD associated with suicidal ideation or behavior. Esketamine has demonstrated an onset of effect for MDD as early as 24 hours. However, unlike zuranolone, the schedule III controlled substance esketamine carries risks of abuse and misuse and may cause sedation and dissociation after administration; therefore, patients must be monitored for at least 2 hours after each dose.

Zuranolone could also be an oral alternative to IV brexanolone (Zulresso) for PPD. Symptoms of excessive sedation and loss of consciousness that are seen with IV brexanolone have not been reported with oral zuranolone. It remains to be seen whether oral zuranolone’s labeling will carry boxed warnings regarding excessive sedation or loss of consciousness, similar to brexalonone, or warnings regarding suicidal thoughts and behaviors, like other oral antidepressants. In clinical trials, women receiving oral zuranolone were not permitted to breastfeed during treatment and for 7 days after the last dose.

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zuranolone cont.

FDA APPROVAL TIMELINE August 5, 2023

The FDA advisory committee is not anticipated to review the NDA for zuranolone.  Breakthrough Therapy

 Fast Track

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$25

$138

$259

$381

$489

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Biosimilar Overview CLINICAL OVERVIEW

Biosimilars are very different from generic drugs in that they are not exact duplicates of their reference biologic product. The FDA approval process for biosimilars is designed to ensure that the biosimilar product is highly similar to the reference product without having any meaningful clinical differences. Moreover, an interchangeable biological product is a biosimilar that is expected to produce the same clinical result as the reference product in any given patient. Switching or alternating between the reference and interchangeable products should have been evaluated and should not negatively impact the safety and efficacy of therapy.

Many controversies have surrounded biosimilars, and regulatory and litigation hurdles remain. The FDA has issued final and draft guidances. Select FDA biosimilar guidances are noted here. In January 2017, the agency issued final guidance on the nonproprietary naming of biologic products, which also applies to biosimilars.

The biological products must bear a core name followed by a distinguishing 4-letter, lowercase, hyphenated suffix that is devoid of meaning. The international nonproprietary name (INN) impacts interchangeability as it affects pharmacists’ ability to substitute an interchangeable biosimilar for the reference product.

The FDA withdrew the September 2017 draft industry guidance on determining similarity of a proposed biosimilar product to its reference product to allow for further consideration of the most current and relevant scientific methods in evaluating analytical data. The agency focuses on providing flexibility for the efficient development of biosimilars while maintaining high scientific standards. In July 2018, the FDA finalized its guidance on labeling biosimilars. The guidance pertains to prescribing information (PI) but does not contain specific recommendations on interchangeability in the labeling. The labeling guidance provides recommendations on how to include, identify, and differentiate the biosimilar and the reference product in various sections of the PI. The basic premise remains that the originator product’s safety and effectiveness can be relied upon for HCPs to make prescribing decisions; therefore, a biosimilar should include relevant data from the originator in its PI. In May 2019, the agency released its final guidance on interchangeability.

Most states have enacted biosimilar substitution laws. An interchangeable product may be substituted for the originator at the pharmacy without the involvement of the prescriber. In December 2022, the FDA announded a pilot regulatory science program that focuses on advancing the development of interchangeable products and improving the efficiency of biosimilar product development. The Purple Book is an FDA database of licensed biological products that lists biosimilar and interchangeable products. The FDA has approved 3 biosimilars for interchangeability to their reference product: insulin glargine-yfgn (Semglee®), adalimumabadbm (Cyltezo®), and ranibizumab-eqrn (Cimerli™).

Biosimilars can receive extrapolation to gain an indication without direct trials of the biosimilar for the eligible indication(s) of the reference products without requiring additional trials. Nevertheless, as each biosimilar comes to market, it will need to be considered individually. The FDA historically regulated insulins as small molecules. However, effective March 23, 2020, drugs such as insulin and growth hormone were deemed biologics and transitioned from the drug pathway to the biologic pathway. Their licensure as biologics allows these agents to be considered in the biosimilar space and promotes competition and access.

PLACE IN THERAPY

The patents of several biologic drugs are set to expire in the next few years, opening the US market for biosimilar entry; however, patent litigation has resulted in significant launch delays of FDA-approved biosimilars. In June 2017, the US Supreme Court issued 2 landmark rulings: (1) allowing a biosimilar manufacturer to provide launch notice of commercial marketing to the originator manufacturer before or after FDA approval of the biosimilar product and (2) eliminating any federal requirement for disclosure, also known as the “patent dance.” Some states, however, mandate disclosure. These decisions may bring biosimilars to the market sooner and potentially create price competition in the marketplace.

In July 2018, the FDA unveiled its Biosimilar Action Plan (BAP), a series of 11 steps to encourage biosimilar market competition, some of which were previously announced or underway. The BAP contains 4 key strategies: (1) improve the biosimilar development and approval process; (2) maximize scientific and regulatory clarity for sponsors; (3) provide effective communications for patients, clinicians, and payers; and (4) reduce unfair tactics that may delay market approval and entry. The BAP strives to promote access to biosimilar products and reduce healthcare costs.

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BIOSIMILAR OVERVIEW continued To date, a total of 40 biosimilars have received FDA approval. Of these, only 30 have entered the market.

APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Zaxio® (filgrastim-sndz)

Sandoz

March 2015

Inflectra® (infliximab-dyyb)

Pfizer

April 2016

Erelzi® (etanercept-szzs)

Sandoz

August 2016

Amjevita™* (adalimumab-atto)

Amgen

September 2016

Renflexis® (infliximab-abda)

Merck/Organon

May 2017

Cyltezo* (adalimumab-adbm)

Boehringer Ingelheim

August 2017

Mvasi® (bevacizumab-awwb)

Amgen

September 2017

Ixifi™ ‡ (infliximab-qbtx)

Pfizer

December 2017

Ogivri® (trastuzumab-dkst)

Mylan/Biocon

December 2017

Retacrit® (epoetin alfa-epbx)

Pfizer/Vifor/ Hospira

May 2018

Fulphila® (pegfilgrastim-jmdb)

Mylan/Biocon

June 2018

Nivestym® (filgrastim-aafi)

Pfizer

July 2018

Hyrimoz®* (adalimumab-adaz)

Sandoz

October 2018

Udenyca® (pegfilgrastim-cbqv)

Coherus

November 2018

Truxima® (rituximab-abbs)

Cephalon/Teva

November 2018

Herzuma® (trastuzumab-pkrb)

Teva

December 2018

Ontruzant® (trastuzumab-dttb)

Merck

January 2019

Trazimera™ (trastuzumab-qyyp)

Pfizer

March 2019

Eticovo™ (etanercept-ykro)

Merck

April 2019

Kanjinti® (trastuzumab-anns)

Amgen

June 2019

Zirabev® (bevacizumab-bvzr)

Pfizer

June 2019

Hadlima™*† (adalimumab-bwwd)

Organon

July 2019

Ruxience® (rituximab-pvvr)

Pfizer

July 2019

21 | MAGELLANRX.COM

Interchangeable -

Commercially Available

  -

 

Originator (Manufacturer) Neupogen® (Amgen) Remicade (Janssen) Enbrel® (Amgen) Humira (Abbvie) Remicade (Janssen)



-

Humira (Abbvie)

-



Avastin® (Genentech)

-

-

Remicade (Janssen)

-

    -

     -

 

Herceptin® (Genentech) Epogen® (Amgen) Procrit® (Janssen) Neulasta® (Amgen) Neupogen (Amgen) Humira (Abbvie) Neulasta (Amgen) Rituxan® (Genentech) Herceptin (Genentech) Herceptin (Genentech) Herceptin (Genentech) Enbrel (Amgen) Herceptin (Genentech) Avastin (Genentech)

-

-

Humira (Abbvie)

-



Rituxan (Genentech)

BIOSIMILAR OVERVIEW continued APPROVED BIOSIMILARS continued APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Abrilada™* (adalimumab-afzb)

Pfizer

November 2019

Ziextenzo® (pegfilgrastim-bmez)

Sandoz

November 2019

Avsola® (infliximab-axxq)

Amgen

December 2019

Nyvepria™ (pegfiltrastim-apgf)

Pfizer

June 2020

Semglee (insulin glargine-yfgn)

Mylan/Biocon

July 2021

Hulio®* (adalimumab-fkjp)

Mylan/Biocon

July 2020

Riabni™ (rituximab-arrx)

Amgen

December 2020

Byooviz™ (ranibizumab-nuna)

Biogen

September 2021

Rezvoglar™ (insulin glargine-aglr)

Eli Lilly

December 2021

Yusimry™* (adalimumab-aqvh)

Coherus

December 2021

Releuko® (filgrastim-ayow)

Amneal

March 2022

Alymsys® (bevacizumab-maly)

Amneal

April 2022

Fylnetra® (pegfilgrastim-pbbk)

Amneal

May 2022

Cimerli (ranibizumab-eqrn)

Coherus

August 2022

Stimufend® (pegfilgrastim-fpgk)

Fresenius Kabi

September 2022

Vegzelma® (bevacizumab-adcd)

Celltrion

September 2022

Idacio® (adalimumab-aacf)

Fresenius Kabi

December 2022

Interchangeable

Commercially Available

-

-



   

-

-

-

Originator (Manufacturer) Humira (Abbvie) Neulasta (Amgen) Remicade (Janssen) Neulasta (Amgen) Lantus® (Sanofi-Aventis) Humira (Abbvie) Rituxan (Genentech) Lucentis® (Genentech)



  

-

-

Humira (Abbvie)

-

-

 -

      -

Lantus (Sanofi)

Neupogen (Amgen) Avastin (Genentech) Neulasta (Amgen) Lucentis (Genentech) Neulasta (Amgen) Avastin (Genentech) Humira (Abbvie)

* Abbvie’s adalimumab (Humira) and adalimumab-adaz (Hyrimoz) are approved as 50 mg/mL (with citric acid/citrate) and 100 mg/mL (citratefree) concentrations. All other biosimilars for Humira are approved as 50 mg/mL concentrations only. † Adalimumab-bwwd (Hadlima) by Organon is also approved as a 100 mg/mL high concentration citrate-free formulation (approved in August 2022). ‡ Pfizer already has Inflectra on the market and has not announced plans to launch Ixifi.

Also available are Eli Lilly’s Basaglar® insulin glargine, a follow-on to Sanofi’s Lantus, and Sanofi’s Admelog® insulin lispro approved as a follow-on to Eli Lilly’s Humalog®.

Specialty medications, which include biologics, continue to grow and constitute a large part of drug spend. In the US, it is estimated that biosimilars will cost approximately 15% to 35% less than the originator product, although price dynamics vary.

Further, the potential cost savings can vary based on the market segment where brand contracts can play a role. A host of factors will contribute to market acceptability and the potential success of biosimilars. Payers, pharmacies, prescribers, and patients each play an important role in market adoption of biosimilars.

The first biosimilar version of Abbvie’s adalimumab (Humira), adalimumab-atto (Amjevita) became available in the US in January 2023. It is anticipated that multiple Humira biosimilars (including citrate-free and high-concentration formulations) will enter the market in July 2023.

22 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

IMMUNOLOGY

adalimumab SC Celltrion is seeking approval for their investigational biosimilar to Abbvie’s citrate-free, high-concentration (100 mg/mL) Humira. Abbvie’s Humira is a tumor necrosis factor alpha (TNF-α) blocker indicated for the treatment of autoimmune disorders, including rheumatoid arthritis (RA), juvenile idiopathic arthritis (JIA), ankylosing spondylitis (AS), plaque psoriasis (PSO), psoriatic arthritis (PsA), Crohn’s disease (CD) in adults and children, ulcerative colitis (UC), hidradenitis suppurativa (HS), and non-infectious uveitis.

Pfizer is seeking interchangeability of FDA-approved adalimumab-afzb (Abrilada) 50 mg/mL.

FDA APPROVAL TIMELINE

50 mg/mL • Pfizer (Abrilada) – Pending for interchangeability 100 mg/mL • Celltrion (Yuflyma) – May 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$11,480

$7,024

$5,194

$4,134

$3,428

The forecast is a projection of total US sales per year for the branded originator product.

OPHTHALMOLOGY

aflibercept intravitreal Biocon/Janssen Biocon/Janssen are seeking approval of their investigational biosimilar (MYL-1701) to Regeneron’s Eylea®, a vascular endothelial growth factor (VEGF) inhibitor indicated for the treatment of neovascular (wet) age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME), and diabetic retinopathy (DR).

FDA APPROVAL TIMELINE Pending

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$6,628

$6,664

$6,126

$5,759

$5,335

The forecast is a projection of total US sales per year for the branded originator product.

23 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

ONCOLOGY

bevacizumab IV Bio-Thera Solutions/Sandoz, Centus, and Samsung Bioepis/Organon are seeking approval for their investigational biosimilars to Genentech’s Avastin, a vascular endothelial growth factor (VEGF)-specific angiogenesis inhibitor indicated for the treatment of metastatic colorectal cancer, nonsquamous non-small cell lung cancer, glioblastoma, metastatic renal cell carcinoma (RCC), and recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer.

FDA APPROVAL TIMELINE

• Bio-Thera Solutions/Sandoz (BAT1706) – Pending • Centus (FKB238) – Pending • Samsung Bioepis/Organon (Aybintio) – Pending

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$543

$458

$404

$363

$327

The forecast is a projection of total US sales per year for the branded originator product.

ENDOCRINE

denosumab SC Sandoz Sandoz is seeking approval for their investigational biosimilar (GP2411) to Amgen’s receptor activator of nuclear factor- κB ligand (RANKL) inhibitor denosumab (Prolia®, Xgeva®). Prolia is indicated for the treatment of osteoporosis in men and postmenopausal women, treatment of glucocorticoid-induced osteoporosis in men and women, and to increase bone mass in women receiving adjuvant aromatase inhibitor therapy or men receiving androgen deprivation therapy; all indicated populations are high risk for fracture. Xgeva is indicated for the prevention of skeletal-related events in patients with multiple myeloma and in patients with bone metastases from solid tumors, treatment of select patients with giant cell tumor of bone, and treatment of hypercalcemia of malignancy refractory to bisphosphonate therapy.

FDA APPROVAL TIMELINE December 6, 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$4,220

$4,467

$4,329

$3,929

$3,366

The forecast is a projection of total US sales per year for the branded originator product.

24 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

BLOOD MODIFIER

filgrastim IV, SC Apotex Apotex is seeking approval of their investigational biosimilar (Grastofil) to Amgen’s Neupogen, a leukocyte growth factor indicated for use in patients with nonmyeloid malignancies who are receiving myelosuppressive anti-cancer drugs; following induction or consolidation chemotherapy for acute myeloid leukemia (AML); with nonmyeloid malignancies in patients who are undergoing myeloablative chemotherapy followed by bone marrow transplantation; to mobilize autologous hematopoietic progenitor cells for collection by leukapheresis; with symptomatic congenital neutropenia‚ cyclic neutropenia‚ or idiopathic neutropenia; and who are acutely exposed to myelosuppressive doses of radiation (hematopoietic syndrome of acute radiation syndrome [HSARS]).

FDA APPROVAL TIMELINE Pending

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$71

$63

$56

$51

$47

The forecast is a projection of total US sales per year for the branded originator product.

NEUROLOGY

natalizumab IV Polypharma/Sandoz Polypharma/Sandoz are seeking approval for their investigational biosimilar (PB006) to Biogen’s Tysabri®, an integrin receptor antagonist indicated for treatment of multiple sclerosis (MS) and Crohn’s disease (CD).

FDA APPROVAL TIMELINE May to June 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$1,079

$1,045

$1,032

$1,023

$951

The forecast is a projection of total US sales per year for the branded originator product.

25 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

BLOOD MODIFIER

pegfilgrastim SC Apotex, Coherus, and Lupin are seeking approval for their investigational biosimilars to Amgen’s Neulasta, a leukocyte growth factor indicated for use in patients with nonmyeloid malignancies who are receiving myelosuppressive anticancer drugs and in patients acutely exposed to myelosuppressive doses of radiation (HSARS).

Coherus is seeking approval of Udenyca OBI®, an investigational biosimilar to Amgen’s Neulasta Onpro®.

FDA APPROVAL TIMELINE

• Apotex (Lapelga) – Pending • Coherus (Udenyca OBI) – October 2023 • Lupin (Lupifil-P) – Pending

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$622

$420

$347

$283

$234

The forecast is a projection of total US sales per year for the branded originator product.

IMMUNOLOGY

ranibizumab intravitreal Stada Arzneimittel/Xbrane Stada Arzneimittel/Xbrane are seeking approval for their investigational biosimilar (Ximluci) to Genentech’s Lucentis, a vascular endothelial growth factor (VEGF) inhibitor indicated to treat wet age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME), diabetic retinopathy, and myopic choroidal neovascularization (mCNV).

FDA APPROVAL TIMELINE January to April 2024

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$752

$505

$361

$275

$223

The forecast is a projection of total US sales per year for the branded originator product.

26 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

IMMUNOLOGY

tocilizumab IV, SC Fresenius Kabi and Biogen/Bio-Thera Solutions are seeking approval for their investigational biosimilars to Genentech’s Actemra®, an interleukin-6 (IL-6) receptor antagonist indicated for the treatment of rheumatoid arthritis (RA), giant cell arteritis, systemic sclerosis-associated interstitial lung disease, polyarticular and systemic juvenile idiopathic arthritis (JIA), and cytokine release syndrome.

FDA APPROVAL TIMELINE

• Fresenius Kabi (MSB11456) – April to June 2023 • Biogen/Bio-Thera Solutions (BIIB800 or BAT1806) – October 9, 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$1,178

$952

$767

$640

$562

The forecast is a projection of total US sales per year for the branded originator product.

ONCOLOGY

trastuzumab IV Henlius/Accord Henlius/Accord are seeking approval of their investigational biosimilar (HLX02) to Herceptin, a HER2/neu receptor antagonist indicated for the treatment of HER2-overexpressing breast cancer and HER2-overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma.

FDA APPROVAL TIMELINE December 15, 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$418

$357

$317

$286

$259

The forecast is a projection of total US sales per year for the branded originator product.

27 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

IMMUNOLOGY

ustekinumab SC Alvotech Alvotech is seeking approval for their investigational biosimilar (AVT04) to Janssen’s Stelara, an interleukin 12/23 (IL-12/23) antagonist indicated for the treatment of plaque psoriasis (PSO), psoriatic arthritis (PsA), Crohn’s disease (CD), and ulcerative colitis (UC).

FDA APPROVAL TIMELINE July to December 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$6,229

$4,330

$3,042

$2,236

$1,747

The forecast is a projection of total US sales per year for the branded originator product.

28 | MAGELLANRX.COM

Keep on Your RADAR Notable agents that are further from approval have been identified in this unique watch list. These are products with the potential for significant clinical and financial impact. Their development status is being tracked on the MRx Pipeline radar. These pipeline products, their respective class or proposed indication, as well as an estimated financial forecast for the year 2027, are displayed. The financials are projected total annual US sales, reported in millions. zilucoplan

aficamten

Immunology

Cardiovascular

$313

$793

vamorolone Neurology

aprocitentan Cardiovascular

$306

$66

tabelecleucel

avacincaptad pegol

$401

$765

Oncology

Ophthalmology

talquetamab

bimekizumab

$135

$1,085

Oncology

Immunology

resmetirom

blarcamesine

$686

$1,049

Cardiovascular

Neurology

datopotamab deruxtecan

reproxalap

Ophthalmology

Oncology

$442

$1,004 giroctocogene fitelparvovec

exagamglogene autotemcel

Hematology/Gene therapy

$131

Hematology/Gene therapy

fidanacogene elaparvovec

$780

Hematology/Gene therapy

$199 pecialty drug names appear in  S magenta throughout the publication.

Pipeline DRUG LIST The pipeline drug list is an aerial outline of drugs with anticipated FDA approval through 2024. It is not intended to be a comprehensive inventory of all drugs in the pipeline; emphasis is placed on drugs in high-impact categories. Investigational drugs with a Complete Response Letter (CRL).

APPLICATION APPLICATION SUBMITTED SUBMITTED TO THE FDA

PHASE33 IN PHASE TRIALS TRIALS

65%

60%

35%

40%

33%

36%

23%

Specialty

23%

13%

12%

8%

Traditional

Orphan Drug

Priority Review

Breakthrough Therapy

Biosimilar

pecialty drug names appear in  S magenta throughout the publication.

PIPELINE DRUG LIST  Specialty drug names appear in magenta throughout the publication.

Submitted (New Drugs) anthrax vaccine, adjuvanted

Emergent

Anthrax infection (postexposure prophylaxis, ages 18-65 years)

IM

Submitted − BLA; Fast Track; Orphan Drug

April 2023

bimekizumab

UCB

PSO

SC

Submitted − BLA

Apr-Jun 2023

risperidone long-acting

Teva

Schizophrenia

SC

Submitted − 505(b)(2) NDA

Apr-Jun 2023

ritlecitinib

Pfizer

Alopecia areata (ages ≥ 12 years)

Oral

Submitted − NDA; Breakthrough Therapy

Apr-Jun 2023

rozanolixizumab

UCB

Myasthenia gravis

SC

Submitted − BLA; Orphan Drug; Priority Review

Apr-Jun 2023

tocilizumab (biosimilar to Genentech’s Actemra)

Fresenius Kabi

RA; Polyarticular JIA; Systemic JIA

IV, SC

Submitted − BLA

Apr-Jun 2023

epinephrine

ARS

Anaphylaxis

Intranasal

Submitted − 505(b)(2) NDA; Fast Track

Apr-Sep 2023

aripiprazole 2-month

H. Lundbeck

Bipolar disorder; Schizophrenia

IM

Submitted − 505(b)(2) NDA

04/27/2023

naloxone 0.3 mg spray

Harm Reduction

Opioid overdose

Intranasal

Submitted − NDA for OTC use; Priority Review

04/28/2023

palopegteriparatide

Ascendis

Hypoparathyroidism (adults)

SC

Submitted − NDA; Orphan Drug; Priority Review

04/30/2023

adalimumab 100 mg/mL (biosimilar for Abbvie’s Humira)

Celltrion

RA; AS; PSO; PsA; JIA; CD; UC

SC

Submitted − BLA

May 2023

RSV pre-fusion protein vaccine (PF-06928316)

Pfizer

RSV prevention (ages ≥ 60 years)

IM

Submitted − BLA; May 2023 Breakthrough Therapy; Fast Track; Priority Review

tenapanor

Ardelyx

Hyperphosphatemia (associated with CKD)

Oral

Submitted − NDA

May 2023

natalizumab (biosimilar to Biogen’s Tysabri)

Polpharma/Sandoz

CD; MS

IV

Submitted − BLA

May-Jun 2023

RSV pre-fusion protein vaccine, adjuvanted (GSK3844766A)

GlaxoSmithKline

RSV prevention (older adults, ages ≥ 60 years)

IM

Submitted − BLA; Fast Track; Priority Review

05/03/2023

phenylephrine/ tropicamide (2.5%/1%)

Eyenovia

Mydriasis (in-office)

Ophthalmic

Submitted − 505(b)(2) NDA

05/08/2023

pegunigalsidase alfa

Chiesi

Fabry disease

IV

Submitted − BLA; Fast Track

05/09/2023

(vic-) trastuzumab duocarmazine

Byondis

Breast cancer (HER2+, unresectable locally advanced or metastatic)

IV

Submitted − BLA; Fast Track

05/12/2023

beremagene geperpavec

Krystal

Epidermolysis bullosa (dystrophic)

Topical

Submitted − BLA; Fast Track; Orphan Drug; Priority Review; RMAT

05/19/2023

epcoritamab

Genmab/Abbvie

DLBCL (R/R, ≥ 3rd-line)

SC

Submitted − BLA; Priority Review

05/21/2023

dengue tetravalent vaccine, live, attenuated

Takeda

Dengue fever prevention (ages 4-60 years)

SC

Submitted − BLA; Fast Track; Priority Review

05/22/2023

fezolinetant

Astellas

Menopause vasomotor symptoms

Oral

Submitted − NDA

05/22/2023

nalmefene

Opiant

Opioid overdose

Intranasal

Submitted − 505(b)(2) NDA; Fast Track; Priority Review

05/22/2023

31 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

nogapendekin alfa inbakicept

Immunitybio

Bladder cancer (BCG-unresponsive, non-muscle invasive carcinoma in situ, in combination with BCG)

Intravesical

Submitted − BLA; Breakthrough Therapy; Fast Track

05/23/2023

sotagliflozin

Lexicon

HF (in patients with or without T2DM)

Oral

Submitted − NDA

05/26/2023

nirmatrelvir/ritonavir (Paxlovid™)

Pfizer

COVID-19 treatment (high-risk individuals)

Oral

Submitted − NDA

05/28/2023

delandistrogene moxeparvovec

Sarepta/Genentech

DMD (ambulatory patients)

IV

Submitted − BLA; seeking Accelerated Approval; Fast Track; Orphan Drug; Priority Review

05/29/2023

durlobactam/sulbactam

Innoviva

Acinetobacter baumannii infection

IV

Submitted − NDA; Fast Track; Priority Review; QIDP

05/29/2023

cyclosporine A

Novaliq

DED

Ophthalmic

Submitted − 505(b)(2) NDA

06/08/2023

momelotinib

GlaxoSmithKline

Myelofibrosis

Oral

Submitted − NDA; Fast Track; Orphan Drug

06/16/2023

olorofim

F2G

Fungal infections (invasive)

Oral

Submitted − NDA; Breakthrough Therapy; LPAD; Orphan Drug; QIDP

06/17/2023

efgartigimod/ hyaluronidase

Argenx

Myasthenia gravis

SC

Submitted − BLA; Orphan Drug; Priority Review

06/20/2023

methotrexate

Aldeyra

Primary vitreoretinal lymphoma

Intravitreal

Submitted − 505(b)(2) NDA; Orphan Drug; Priority Review

06/21/2023

gepirone

GlaxoSmithKline

MDD

Oral

Submitted − NDA

06/23/2023

perfluorohexyloctane

Bausch

DED (Meibomian gland dysfunction-associated)

Ophthalmic

Submitted − NDA

06/28/2023

carbidopa/levodopa ER

Amneal

Parkinson’s disease

Oral

Submitted − 505(b)(2) NDA

06/30/2023

valoctocogene roxaparvovec

Biomarin

Hemophilia A

IV

Submitted − BLA; Breakthrough Therapy; Orphan Drug; RMAT

06/30/2023

clobetasol proprionate

Laboratorios Salvat

Postsurgical ocular pain and inflammation

Ophthalmic

Submitted − 505(b)(2) NDA

Jul-Sep 2023

clotrimazole

Laboratorios Salvat

Otomycosis

Otic

Submitted − 505(b)(2) NDA

Jul-Sep 2023

nirsevimab

AstraZeneca/Sanofi

RSV prevention (newborns/infants in 1st RSV season, at risk patients ages ≤ 24 months through 2nd RSV season)

IM

Submitted − BLA; Breakthrough Therapy; Fast Track

Jul-Sep 2023

etrasimod

Pfizer

UC (moderate to severe)

Oral

Submitted − NDA

Jul-Dec 2023

ustekinumab (biosimilar to Janssen’s Stelara)

Alvotech

PSO; PsA; CD; UC

SC

Submitted − BLA

Jul-Dec 2023

glofitamab

Genentech

DLBCL (R/R, ≥ 3rd-line)

IV

Submitted − BLA; Priority Review

07/01/2023

cantharidin

Verrica

Molluscum contagiosum

Topical

Submitted − NDA

07/24/2023

32 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

quizartinib

Daiichi Sankyo

AML (in combination with cytarabine &/or anthracycline induction & consolidation, and monotherapy following consolidation)

Oral

Submitted − NDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review

07/24/2023

dehydrated alcohol injection

Eton

Methanol poisoning

SC

Submitted − 505(b)(2) NDA; Orphan Drug

07/27/2023

risperidone (once monthly)

Laboratorios Farmacéuticos Rovi

Schizophrenia

IM

Submitted − 505(b)(2) NDA

07/27/2023

remestemcel-L

Mesoblast

GVHD (acute, steroidrefractory, pediatrics)

IV

Submitted − BLA; Fast Track; Orphan Drug

07/31/2023

chikungunya vaccine monovalent, live attenuated

Valneva

Chikungunya prevention (single dose, adults)

IM

Submitted − BLA; August 2023 Breakthrough Therapy; Fast Track; Priority Review

RSV pre-fusion protein vaccine (PF-06928316)

Pfizer

RSV prevention (maternal IM administration for RSV prevention from birth to 6 months of age)

Submitted − BLA; August 2023 Breakthrough Therapy; Fast Track; Priority Review

elranatamab

Pfizer

Multiple myeloma (R/R)

SC

Submitted − BLA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review

Aug-Sep 2023

brimonidine tartrate 0.35%

Visiox

Glaucoma/ocular hypertension

Ophthalmic

Submitted − 505(b)(2) NDA

08/04/2023

zuranolone

Sage/Biogen

MDD; Post-partum depression

Oral

Submitted − NDA; 08/05/2023 Breakthrough Therapy; Fast Track; Priority Review

avasopasem manganese

Galera

Oral mucositis (severe, radiotherapy-induced, in patients with head & neck cancer)

IV

Submitted − NDA; 08/09/2023 Breakthrough Therapy; Fast Track; Priority Review

melphalan kit for injection/ Delcath hepatic delivery system (HDS)

Uveal melanoma (hepatic-dominant, unresectable)

Percutaneous hepatic perfusion

Submitted − NDA

08/14/2023

palovarotene

Ipsen

Fibrodysplasia ossificans Oral progressiva (prevention of heterotopic ossification)

Submitted − NDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review

08/16/2023

avacincaptad pegol

Iveric Bio

Dry AMD-related geographic atrophy

Intravitreal

Submitted − NDA; 08/19/2023 Breakthrough Therapy; Fast Track; Priority Review

pozelimab

Regeneron

Chaple syndrome

IV, SC

Submitted − BLA; Orphan Drug; Priority Review

08/20/2023

lotilaner

Tarsus

Demodex blepharitis

Ophthalmic

Submitted − NDA

08/25/2023

nirogacestat

Springworks/ GlaxoSmithKline

Desmoid tumors or deep fibromatosis

Oral

Submitted − NDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review; RTOR

08/27/2023

bevacizumab-vikg

Outlook

Wet AMD

Intravitreal

Submitted − BLA

08/29/2023

concizumab

Novo Nordisk

Hemophilia A and B with inhibitors (prophylaxis)

SC

Submitted − BLA; Breakthrough Therapy; Orphan Drug

September 2023

33 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

lebrikizumab

Eli Lilly

Atopic dermatitis (moderate to severe)

SC

Submitted − BLA; Fast Track

September 2023

nedosiran

Novo Nordisk

Hyperoxaluria

SC

Submitted − NDA; Breakthrough Therapy; Orphan Drug; RPD

September 2023

motixafortide

Biolinerx

Stem cell mobilization for autologous BMT for multiple myeloma

SC

Submitted − NDA; Orphan Drug

09/09/2023

atezolizumab SC

Genentech

Hepatocellular carcinoma; SC Melanoma (BRAF-mutant); NSCLC; SCLC; Soft tissue sarcoma; Urothelial cancer

Submitted − BLA

09/15/2023

metronidazole (tastemasking liquid)

Appili

Trichomoniasis infections; Oral Amebiasis; Bacterial infections caused by certain microorganisms

Submitted − 505(b)(2) NDA; Orphan Drug

09/23/2023

phentolamine 0.75%

Ocuphire

Reversal of pharmacologicallyinduced mydriasis

Ophthalmic

Submitted − 505(b)(2) NDA

09/28/2023

meningococcal pentavalent Pfizer vaccine (PF-06886992)

Meningococcal immunization

IM

Submitted − BLA

October 2023

lifileucel

Iovance

Melanoma (advanced unresectable or metastatic)

IV

Submitted − BLA; Fast Oct-Dec 2023 Track; Orphan Drug; RMAT

sofpironium

Botanix

Axillary hyperhidrosis (severe)

Topical

Submitted − NDA

Oct-Dec 2023

zilucoplan

UCB

Myasthenia gravis (acetylcholine receptor antibody positive [AChRAb+])

SC

Submitted − NDA; Orphan Drug

Oct-Dec 2023

tocilizumab (biosimilar to Genentech’s Actemra)

Biogen/Bio-Thera Solutions

RA; Polyarticular JIA; Systemic JIA

IV, SC

Submitted − BLA

10/09/2023

infliximab SC

Celltrion

Inflammatory bowel disease

SC

Submitted − BLA

10/22/2023

vamorolone

Santhera

DMD

Oral

Submitted − NDA; Fast Track; Orphan Drug

10/26/2023

reproxalap

Aldeyra

DED

Ophthalmic

Submitted − NDA

11/23/2023

lovotibeglogene autotemcel

Bluebird Bio

SCD

IV

Submitted − BLA; Fast Dec 2023-Apr Track; Orphan Drug; RMAT 2024

denosumab (biosimilar to Amgen’s Prolia/Xgeva)

Sandoz

Osteoporosis/osteopenia

SC

Submitted − BLA

12/06/2023

talquetamab

Janssen

Multiple myeloma (R/R)

SC

Submitted − BLA; Breakthrough Therapy; Orphan Drug

12/11/2023

trastuzumab (biosimilar to Genentech’s Herceptin)

Henlius/Accord

Breast cancer; Gastric or gastroesophageal junction adenocarcinoma

IV

Submitted − BLA

12/15/2023

roflumilast foam

Arcutis

Seborrheic dermatitis (ages ≥ 9 years)

Topical

Submitted − NDA

12/16/2023

aprocitentan

Janssen

Hypertension (resistant)

Oral

Submitted − NDA

12/20/2023

eplontersen

Ionis/AstraZeneca

Transthyretin amyloid polyneuropathy

SC

Submitted − NDA; Orphan Drug

12/22/2023

34 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

dihydroergotamine nasal powder

Satsuma

Migraine acute treatment

Intranasal

Submitted − 505(b)(2) NDA

January 2024

ranibizumab (biosimilar to Genentech’s Lucentis)

Stada Arzneimittel/ Xbrane

Diabetic retinopathy; DME; Myopic choroidal neovascularization; Macular edema following RVO; Wet AMD

Intravitreal

Submitted − BLA

Jan-Apr 2024

niraparib/abiraterone (dual-action tablet)

GlaxoSmithKline

Prostate cancer (BRCA+, metastatic, castrationresistant, in combination with prednisone)

Oral

Submitted − NDA; Breakthrough Therapy

01/01/2024

pilocarpine 0.4%

Orasis

Presbyopia

Ophthalmic

Submitted − 505(b)(2) NDA

01/03/2024

cosibelimab

Checkpoint

Cutaneous squamous cell carcinoma (metastatic)

IV

Submitted − BLA

01/04/2024

berdazimer gel

Novan

Molluscum contagiosum

Topical

Submitted − NDA

01/05/2024

travoprost implant

Glaukos

Glaucoma/ocular hypertension

Intraocular

Submitted − 505(b)(2) NDA

02/27/2024

fruquintinib

Hutchmed/Eli Lilly

CRC (refractory, metastatic)

Oral

Submitted − NDA; Fast Track

03/29/2024

exagamglogene autotemcel

Vertex

Beta-thalassemia (transfusion-dependent); SCD

IV

Submitted − BLA; Fast 04/03/2024 Track; Orphan Drug; RMAT

aflibercept (biosimilar to Regeneron’s Eylea)

Biocon/Janssen

DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD

Intravitreal

Submitted − BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Bio-Thera Solutions/ Sandoz

Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC

IV

Submitted − BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Centus

Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC

IV

Submitted − BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Samsung Bioepis/ Organon

Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC

IV

Submitted − BLA

Pending

cipaglucosidase alfa

Amicus

Pompe disease (in combination with oral miglustat)

IV

Submitted − BLA; Breakthrough Therapy; Orphan Drug

Pending

debamestrocel

Brainstorm

ALS

Intrathecal

Submitted − BLA; Fast Track; Orphan Drug

Pending

filgrastim (biosimilar to Amgen’s Neupogen)

Apotex

Neutropenia/leukopenia

IV, SC

Submitted − BLA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Apotex

Neutropenia/leukopenia

SC

Submitted − BLA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Lupin

Neutropenia/leukopenia

SC

Submitted − BLA

Pending

tislelizumab

Beigene/Novartis

Esophageal squamous cell carcinoma (unresectable or metastatic, 2nd-line)

IV

Submitted − BLA; Orphan Drug

Pending

tixagevimab/cilgavimab (EvusheldTM)

AstraZeneca

COVID-19 treatment

IM

Submitted − BLA

Pending

35 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

toripalimab

Coherus

DOSAGE FORM

CLINICAL USE Nasopharyngeal cancer (advanced recurrent/ metastatic, 1st-line with gemcitabine & cisplatin, subsequent monotherapy)

IV

DEVELOPMENT STATUS

FDA DECISION

Submitted − BLA; Breakthrough Therapy; Orphan Drug

Pending

April 2023

Submitted (Supplementals) cobimetinib (Cotellic )

Genentech

Histiocytosis

Oral

Submitted − sNDA; Breakthrough Therapy

pneumococcal conjugate vaccine candidate (20-valent)(Prevnar®)

Pfizer

Invasive pneumococcal disease prevention & related otitis media (ages 6 weeks-17 years)

IM

Submitted − sBLA; April 2023 Breakthrough Therapy; Fast Track; Priority Review

dapagliflozin (Farxiga®)

AstraZeneca

Chronic HFpEF

Oral

Submitted − sNDA

Apr-Jun 2023

somapacitan-beco (Sogroya®)

Novo Nordisk

Growth hormone deficiency (pediatric)

SC

Submitted − sBLA

Apr-Jun 2023

elexacaftor/tezacaftor/ ivacaftor (Trikafta®)

Vertex

CF (ages 2-5 years, F508del mutation or responsive mutation)

Oral

Submitted − sNDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review

04/28/2023

durvalumab (Imfinzi®)

AstraZeneca

HCC (unresectable)

IV

Submitted − sBLA; Orphan Drug

04/30/2023

ravulizumab-cwvz (Ultomiris®)

AstraZeneca

Neuromyelitis optica (Devic’s syndrome)

IV, SC

Submitted − sBLA

May-Jun 2023

ivacaftor (Kalydeco®)

Vertex

CF (ages 1 to < 4 months of age)

Oral

Submitted − sNDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review

05/03/2023

brexpiprazole (Rexulti®)

Otsuka

Alzheimer’s disease (neuropsychiatric symptoms)

Oral

Submitted − sNDA; Fast Track; Priority Review

05/10/2023

Abbvie

CD (moderate to severe)

Oral

Submitted − sNDA

05/26/2023

atezolizumab (Tecentriq )

Genentech

Sarcoma (alveolar soft part)

IV

Submitted − sBLA

June 2023

letermovir (Prevymis™)

Merck

CMV prophylaxis (high risk adult kidney transplant recipients [D+/R-])

IV, Oral

Submitted − sNDA; Fast Track; Orphan Drug; Priority Review

06/05/2023

odevixibat (Bylvay®)

Albireo

Alagille syndromeassociated cholestatic pruritus

Oral

Submitted − sNDA; Orphan Drug

06/15/2023

mavacamten (Camzyos®)

Bristol-Myers Squibb

Cardiomyopathy (reduce need for septal reduction therapy)

Oral

Submitted − sNDA; Breakthrough Therapy; Orphan Drug

06/16/2023

obeticholic acid (Ocaliva®)

Intercept

NASH-related precirrhotic liver fibrosis

Oral

Submitted − sNDA; seeking Accelerated Approval; Breakthrough Therapy

06/22/2023

aflibercept (Eylea) 8 mg dose

Regeneron

Diabetic retinopathy; Wet AMD; DME (all with extended dose interval)

Intravitreal

Submitted − sBLA; Priority Review

06/27/2023

brentuximab vedotin (Adcetris®)

Seagen

Hodgkin’s lymphoma (advanced, 1st-line)

IV

Submitted − sBLA; Breakthrough Therapy; Fast Track; Orphan Drug

July 2023

®

upadacitinib (Rinvoq®) ®

36 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

vosoritide (Voxzogo®)

Biomarin

Achondroplasia (ages < 5 years)

SC

Submitted − sNDA; Orphan Drug

Jul-Dec 2023

rucaparib (Rubraca®)

Clovis

Ovarian cancer (1st-line maintenance)

Oral

Submitted − sNDA; Breakthrough Therapy; Orphan Drug

07/12/2023

linaclotide (Linzess®)

Ironwood/Abbvie

Functional constipation (ages 6-17 years)

Oral

Submitted − sNDA; Priority Review

07/14/2023

denileukin diftitox (Ontak®) Citius

Cutaneous T cell lymphoma (persistent or recurrent)

IV

Submitted − sBLA; Orphan Drug

07/28/2023

trifluridine/tipiracil (Lonsurf®)

Otsuka

CRC (metastatic, prior fluopyrimidineoxaliplatin-, and irinotecan-based chemotherapy, anti-VEGF, and anti-EGFR [if RAS wild-type] therapy)

Oral

Submitted − sNDA; Fast Track; Priority Review

08/13/2023

talazoparib (Talzenna®)

Pfizer

Prostate cancer (metastatic, castrationresistant, in combination with enzalutamide)

Oral

Submitted − sNDA; Priority Review

08/16/2023

daxibotulinumtoxinA (Daxxify™)

Revance

Cervical dystonia

IM

Submitted − sBLA; Orphan Drug

08/19/2023

valbenazine (Ingrezza®)

Neurocrine Biosciences

Huntington’s disease

Oral

Submitted − sNDA; Orphan Drug

08/20/2023

empagliflozin (Jardiance®)

Boehringer Ingelheim/Eli Lilly

CKD (reduce the risk of kidney disease progression and CV death)

Oral

Submitted − sNDA; Fast Track

Sep-Oct 2023

letermovir (Prevymis)

Merck

CMV prophylaxis (allogeneic HSCT recipients, days 100-200)

IV, Oral

Submitted − sNDA; Fast Track; Orphan Drug

09/07/2023

pegfilgrastim-cbqv onbody injector (Udenyca OBI)(biosimilar to Amgen's Neulasta Onpro)

Coherus

Neutropenia/leukopenia

SC

Submitted − sBLA

October 2023

binimetinib (Mektovi®)

Pfizer

NSCLC (metastatic, BRAF V600E mutation, in combination with encorafenib)

Oral

Submitted − sNDA

Oct-Dec 2023

encorafenib (Braftovi®)

Pfizer

NSCLC (metastatic, BRAF V600E mutation, in combination with binimetinib)

Oral

Submitted − sNDA

Oct-Dec 2023

patisiran (Onpattro®)

Alnylam

Transthyretin amyloid cardiomyopathy (wild type or hereditary)

IV

Submitted − sNDA; Orphan Drug

10/06/2023

pembrolizumab (Keytruda®)

Merck

NSCLC (stage II, IIIA, or IIIB)

IV

Submitted − sBLA; Breakthrough Therapy

10/16/2023

roflumilast (Zoryve™)

Arcutis

PSO (ages 2-11 years)

Topical

Submitted − sNDA

10/19/2023

dupilumab (Dupixent)

Sanofi

Urticaria (chronic, spontaneous, ages ≥ 12 years)

SC

Submitted − sBLA

10/20/2023

secukinumab (Cosentyx®)

Novartis

Hidradenitis suppurativa

SC

Submitted − sBLA

10/25/2023

37 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

bupivacaine/meloxicam (Zynrelef®)

Heron

Postsurgical pain (soft tissue and orthopedic surgical procedures)

Instillation

Submitted − 505(b)(2) sNDA; Breakthrough Therapy; Fast Track

10/27/2023

maralixibat (Livmarli®)

Mirum

Progressive familial intrahepatic cholestasisrelated pruritus (ages ≥ 2 months)

Oral

Submitted − sNDA; Breakthrough Therapy; Orphan Drug

12/14/2023

fluticasone propionate (Xhance®)

Optinose

Chronic rhinosinusitis

Intranasal

Submitted − sNDA

12/15/2023

idecabtagene vicleucel (Abecma®)

Bristol-Myers Squibb

Multiple myeloma (R/R, prior immunomodulatory agent, a proteasome inhibitor, and an antiCD38 monoclonal antibody)

IV

Submitted − sBLA; Breakthrough Therapy; Orphan Drug

12/16/2023

pembrolizumab (Keytruda)

Merck

Gastric and gastroesophageal junction adenocarcinoma (locally advanced unresectable or metastatic, 1st-line, in combination with fluoropyrimidine & platinum chemotherapy)

IV

Submitted − sBLA; Orphan Drug

12/16/2023

adalimumab-afzb 50 mg/mL (Abrilada) (biosimilar to Abbvie’s Humira)

Pfizer

RA; AS; PSO; PsA; JIA; CD; UC

SC

Submitted − PAS BLA for interchangeability

Pending

norgestrel (Opill®)

Perrigo

Contraception

Oral

Submitted − sNDA Rxto-OTC

Pending

olaparib (Lynparza®)

AstraZeneca

Prostate cancer (mCRPC, Oral in combination with prednisone ± abiraterone)

Submitted − sNDA; Breakthrough Therapy; Priority Review

Pending

Phase 3 − BLA; Orphan Drug

TBD

Phase 3 (New Drugs) AAV8-ranibizumab

Regenxbio

Wet AMD

abelacimab

Anthos

Stroke prevention in atrial SC fibrillation; VTE

Phase 3 − BLA; Fast Track

TBD

abiraterone

Tavanta

Prostate cancer

Oral

Phase 3 − NDA

TBD

acoramidis

Bridgebio

Transthyretin amyloid cardiomyopathy (ATTRCM)

Oral

Phase 3 − NDA

TBD

adagrasib

Mirati

CRC

Oral

Phase 3 − NDA; Breakthrough Therapy

TBD

adintrevimab

Invivyd/Biocon

COVID-19

IM

Phase 3 − BLA

TBD

aficamten

Cytokinetics

Hypertrophic cardiomyopathy

Oral

Phase 3 − NDA; Breakthrough Therapy; Orphan Drug

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Amgen

DME; Wet AMD

Intravitreal

Phase 3 − BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

SamChunDang

DME; Wet AMD

Intravitreal

Phase 3 − BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Samsung Bioepis

DME; Wet AMD

Intravitreal

Phase 3 − BLA

TBD

38 | MAGELLANRX.COM

Subretinal

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

aflibercept (biosimilar to Regeneron’s Eylea)

Sandoz

DME; Wet AMD

Intravitreal

Phase 3 − BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Santo/Formycon

DME; Wet AMD

Intravitreal

Phase 3 − BLA

TBD

aloradine

Vistagen

Social anxiety disorder

Intranasal

Phase 3 − NDA; Fast Track TBD

amubarvimab/ romlusevimab

Brii

COVID-19

IV

Phase 3 − BLA

TBD

anti-betv1 antibody (REGN- Regeneron 5713-5714-5715)

Birch allergy

SC

Phase 3 − BLA

TBD

apolipoprotein A1 (human)

CSL

Atherosclerosis

IV

Phase 3 − BLA

TBD

AR-15512

Aerie

DED

Ophthalmic

Phase 3 − NDA

TBD

arimoclomol

Kempharm

Niemann-Pick disease

Oral

Phase 3 − NDA; Breakthrough Therapy; Fast Track; Orphan Drug; RPD

TBD

ARO-APOC3

Arrowhead

Familial chylomicronemia syndrome

SC

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

asundexian

Bayer

Stroke prevention in atrial Oral fibrillation

Phase 3 − NDA

TBD

ataluren

PTC

DMD

Oral

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

bamlanivimab

Eli Lilly

COVID-19

IV

Phase 3 − BLA

TBD

batiraxcept

Aravive

Ovarian cancer

IV

Phase 3 − BLA; Fast Track

TBD

bentracimab

Phasebio

Ticagrelor (Brilinta®) reversal

IV

Phase 3 − BLA; Breakthrough Therapy

TBD

bevacizumab (biosimilar to Genentech’s Avastin)

Kyowa Kirin

Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC

IV

Phase 3 − BLA

TBD

bimekizumab

UCB

Axial spondyloarthritis; PsA

SC

Phase 3 − BLA

TBD

bis-choline tetrathiomolybdate

AstraZeneca

Wilson’s disease

Oral

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

blarcamesine

Anavex Life Sciences

Alzheimer’s disease; Rett syndrome

Oral

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

botaretigene sparoparvovec

Janssen

Retinitis pigmentosa

Subretinal

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

BPR277

Lifemax

Netherton syndrome

Topical

Phase 3 − NDA; Fast Track; Orphan Drug; RPD

TBD

brensocatib

Insmed

Bronchiectasis

Oral

Phase 3 − NDA; Breakthrough Therapy

TBD

buntanetap

Annovis

Parkinson’s disease

Oral

Phase 3 − NDA

TBD

cannabidiol gel

Zynerba

Fragile X syndrome

Topical

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

cefepime/taniborbactam

Venatorx

UTI (complicated)

IV

Phase 3 − NDA; Fast Track; QIDP

TBD

ceftobiprole medocaril

Basilea

Bacteremia; CAP; Bacterial IV skin and skin-structure infections

Phase 3 − NDA; Fast Track; QIDP

TBD

clobetasol propionate

Formosa

Ocular pain/inflammation

Ophthalmic

Phase 3 − 505(b)(2) NDA

TBD

CM-AT (pancreatic enzyme)

Curemark

Autism spectrum disorders

Oral

Phase 3 − BLA; Fast Track

TBD

39 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

cobitolimod

Index/Merck

UC

Rectal

Phase 3 − NDA; Orphan Drug

TBD

colistimethate sodium

Zambon

Bronchiectasis

Inhaled

Phase 3 − NDA; Breakthrough Therapy; Fast Track; QIDP

TBD

copper histidine

Zydus

Menkes disease

SC

Phase 3 − NDA; Breakthrough Therapy; Fast Track; Orphan Drug

TBD

COVID-19 S-trimer vaccine (SCB-2019)

Clover

COVID-19

SC

Phase 3 − BLA

TBD

COVID-19 vaccine (JNJ78436735; formerly Ad26.

COV2-S)

Janssen

COVID-19

IM

Phase 3 − BLA

TBD

COVID-19 vaccine (MT2766)

Mitsubishi Tanabe

COVID-19

IM, SC

Phase 3 − BLA; Fast Track

TBD

COVID-19 vaccine (SP0253)

Sanofi/ GlaxoSmithKline

COVID-19

IM

Phase 3 − BLA

TBD

COVID-19 vaccine, adjuvanted (NVXCoV2515)

Novavax

COVID-19

IM

Phase 3 − BLA

TBD

crinecerfont

Neurocrine

Congenital adrenal hyperplasia

Oral

Phase 3 − NDA; Orphan Drug

TBD

crovalimab

Genentech

Hemolytic uremic syndrome; PNH

IV, SC

Phase 3 − BLA; Breakthrough Therapy; Orphan Drug

TBD

CTX-009

Compass

Biliary tract cancer

IV

Phase 3 − BLA; Orphan Drug

TBD

cyclobenzaprine

Tonix

Fibromyalgia

SL

Phase 3 − 505(b)(2) NDA

TBD

dabocemagene autoficel

Castle Creek

Epidermolysis bullosa

Intradermal

Phase 3 − BLA; Fast Track; Orphan Drug; RMAT

TBD

danicopan

AstraZeneca

PNH

Oral

Phase 3 − NDA; Breakthrough Therapy; Orphan Drug

TBD

darvadstrocel

Takeda

CD

IV

Phase 3 − BLA; Orphan Drug

TBD

delgocitinib

Leo

Atopic dermatitis

Topical

Phase 3 − NDA; Fast Track TBD

denosumab (Biosimilar to Amgen’s Prolia/Xgeve)

Biocon

Osteoporosis/osteopenia

SC

Phase 3 − BLA

TBD

denosumab (Biosimilar to Amgen’s Prolia/Xgeve)

Celltrion

Osteoporosis/osteopenia

SC

Phase 3 − BLA

TBD

denosumab (Biosimilar to Amgen’s Prolia/Xgeve)

Gedeon Richter

Osteoporosis/osteopenia

SC

Phase 3 − BLA

TBD

denosumab (Biosimilar to Amgen’s Prolia/Xgeve)

Teva

Osteoporosis/osteopenia

SC

Phase 3 − BLA

TBD

dersimelagon

Mitsubishi Tanabe

Porphyria

Oral

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

difamilast

Medimetriks

Atopic dermatitis

Topical

Phase 3 − NDA

TBD

difelikefalin

Cara

Atopic dermatitis; Pruritus Oral

Phase 3 − NDA

TBD

difluprednate XR

Visiox

Ocular pain/inflammation

Ophthalmic

Phase 3 − NDA

TBD

dinutuximab beta

EUSA

Neuroendocrine tumors

IV

Phase 3 − BLA; Orphan Drug

TBD

40 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

dirloctocogene samoparvovec

Genentech

Hemophilia A

IV

Phase 3 − BLA; Breakthrough Therapy; Orphan Drug

TBD

donanemab

Eli Lilly

Alzheimer’s disease (early)

IV, SC

Phase 3 − BLA; Breakthrough Therapy; Priority Review

TBD

donaperminogene seltoplasmid

Helixmith

Diabetic foot ulcers (chronic non-healing)

IM

Phase 3 − BLA

TBD

doravirine/islatravir

Merck

HIV-1 infection treatment

Oral

Phase 3 − NDA

TBD

dust mite immunotherapy tablet

Stallergenes Greer

Allergic rhinitis

SL

Phase 3 − BLA

TBD

EB-101 (gene therapy)

Abeona

Epidermolysis bullosa

Surgical application

Phase 3 − BLA; Breakthrough Therapy; Orphan Drug; RMAT

TBD

ebselen

Sound

Meniere’s disease

Oral

Phase 3 − NDA; Fast Track TBD

eculizumab (biosimilar to Alexion’s Soliris®)

Amgen

PNH

IV

Phase 3 − BLA

TBD

efzofitimod

Atyr

Sarcoidosis

IV

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

enmetazobactam

Allecra

UTI (complicated)

IV

Phase 3 − NDA; Fast Track; QIDP

TBD

ensifentrine

Verona

COPD

Inhaled

Phase 3 − NDA

TBD

ensovibep

Novartis

COVID-19

IV

Phase 3 − BLA; Fast Track

TBD

eplontersen

Ionis

Transthyretin amyloid cardiomyopathy (ATTRCM)

SC

Phase 3 − NDA; Orphan Drug

TBD

esreboxetine

Axsome/Pfizer

Fibromyalgia

Oral

Phase 3 − NDA

TBD

etanercept (biosimilar to Amgen’s Enbrel)

Coherus

RA; Polyarticular JIA; AS; PSO; PsA

SC

Phase 3 − BLA

TBD

etavopivat

Novo Nordisk

SCD

Oral

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

etesevimab

Eli Lilly

COVID-19

IV

Phase 3 − BLA

TBD

etrasimod

Pfizer

Atopic dermatitis

Oral

Phase 3 − NDA

TBD

etripamil

Milestone

Supraventricular tachycardia

Intranasal

Phase 3 − NDA

TBD

evobrutinib

Merck

MS

Oral

Phase 3 − NDA

TBD

factor VIII mimetic bispecific antibody

Novo Nordisk

Hemophilia A

SC

Phase 3 − BLA; Orphan Drug

TBD

fasedienol

Vistagen

Social anxiety disorder

Intranasal

Phase 3 − NDA; Fast Track TBD

fexapotide triflutate

Nymox

BPH

Intratumoral

Phase 3 − NDA

TBD

fianlimab

Regeneron

Melanoma

IV

Phase 3 − BLA

TBD

fidanacogene elaparvovec

Pfizer/Genentech

Hemophilia B

IV

Phase 3 − BLA; Breakthrough Therapy; Orphan Drug

TBD

filgotinib

Gilead

UC

Oral

Phase 3 − NDA

TBD

fitusiran

Sanofi

Hemophilia A and B

SC

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

follitropin alfa (biosimilar to EMD Serono’s Gonal-F®)

Abbvie

Female reproductive disorder

SC

Phase 3 − BLA

TBD

garadacimab

CSL

HAE

SC

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

41 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

gavorestat

Applied Therapeutics Galactosemia

Oral

Phase 3 − NDA; Fast Track; Orphan Drug; RPD

TBD

GBT601

Pfizer

SCD

Oral

Phase 3 − NDA; Orphan Drug

TBD

gepotidacin

GlaxoSmithKline

UTI (uncomplicated)

Oral

Phase 3 − NDA; QIDP

TBD

giroctocogene fitelparvovec

Pfizer

Hemophilia A

IV

Phase 3 − BLA; Fast Track; Orphan Drug; RMAT

TBD

glatiramer depot

Viatris

MS

IM

Phase 3 − 505(b)(2) NDA

TBD

glepaglutide

Zealand

Short bowel syndrome

SC

Phase 3 − NDA; Orphan Drug

TBD

gold nanocrystal

Clene

ALS

Oral

Phase 3 − NDA; Orphan Drug

TBD

hypericin

Soligenix

Cutaneous T-cell lymphoma (CTCL; early stages)

Topical

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

imetelstat

Geron

Myelodysplastic syndrome

IV

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

inavolisib

Genentech

Breast cancer (locally advanced or metastatic, HR+/HER2-, 1st-line)

Oral

Phase 3 − NDA

TBD

inclacumab

Pfizer

SCD

IV

Phase 3 − BLA; Orphan Drug

TBD

infliximab (biosimilar to Janssen’s Remicade)

Nichi-Iko

RA; AS; PSO; CD

IV

Phase 3 − BLA

TBD

influenza nanoparticle vaccine

Novavax

Seasonal influenza prevention

IM

Phase 3 − BLA; Fast Track

TBD

insulin aspart (biosimilar to Novo Nordisk’s Novolog®)

Amphastar

T1DM; T2DM

SC

Phase 3 − BLA

TBD

insulin aspart (biosimilar to Novo Nordisk’s Novolog)

Sanofi

T1DM; T2DM

SC

Phase 3 − BLA

TBD

insulin glargine (biosimilar to Sanofi’s Lantus)

Gan & Lee

T1DM; T2DM

SC

Phase 3 − BLA

TBD

insulin icodec (onceweekly dosing)

Novo Nordisk

T2DM

SC

Phase 3 − BLA

TBD

iptacopan

Novartis

Complement 3 (C3) glomerulopathy; Hemolytic uremic syndrome; Immunoglobulin A nephropathy (Berger’s disease); PNH

Oral

Phase 3 − NDA; Breakthrough Therapy; Orphan Drug

TBD

isotretinoin

Timber

Congenital ichthyosis

Topical

Phase 3 − 505(b)(2) NDA; Breakthrough Therapy; Fast Track; Orphan Drug

TBD

itepekimab

Regeneron/Sanofi

COPD

SC

Phase 3 − BLA; Fast Track

TBD

JDQ-443

Novartis

NSCLC

Oral

Phase 3 − NDA

TBD

Lactobacillus reuteri

Infant Bacterial Therapeutics

Necrotizing enterocolitis

Oral

Phase 3 − BLA; Orphan Drug

TBD

lanifibranor

Inventiva

NASH

Oral

Phase 3 − NDA; Breakthrough Therapy; Fast Track

TBD

lazertinib

Genosco/Janssen

NSCLC

Oral

Phase 3 − NDA

TBD

42 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

lenadogene nolparvovec

Gensight

Leber’s hereditary optic neuropathy

Intravitreal

Phase 3 − BLA; Orphan Drug

TBD

leriglitazone

Minoryx

Adrenoleukodystrophy

Oral

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

leukocyte interleukin

Cel-Sci

SCCHN

Peritumor/ perilymphatic

Phase 3 − BLA; Orphan Drug

TBD

levodopa/carbidopa patch pump

Mitsubishi Tanabe

Parkinson’s disease

SC

Phase 3 − 505(b)(2) NDA

TBD

linerixibat

GlaxoSmithKline

Cholestatic pruritus

Oral

Phase 3 − NDA; Orphan Drug

TBD

litifilimab

Biogen

SLE

SC

Phase 3 − BLA

TBD

magrolimab

Gilead

Myelodysplastic syndrome

IV

Phase 3 − BLA; Breakthrough Therapy; Fast Track; Orphan Drug

TBD

marstacimab

Pfizer

Hemophilia A and B

IV, SC

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

marzeptacog alfa

Catalyst

Hemophilia A and B (with inhibitors)

SC

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

masitinib

AB Science

ALS; Alzheimer’s disease; Asthma (eosinophilic); Mastocytosis; MS

Oral

Phase 3 − NDA; Orphan Drug

TBD

mavodelpar

Reneo

Primary mitochondrial myopathies

Oral

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

mavorixafor

X4

Warts, Oral hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome

Phase 3 − NDA; Breakthrough Therapy; Fast Track; Orphan Drug; RPD

TBD

meningococcal vaccine (GSK3536819A)

GlaxoSmithKline

Meningococcal immunization

IM

Phase 3 − BLA

TBD

metachromatic leukodystrophy gene therapy

Orchard

Metachromatic leukodystrophy

IV

Phase 3 − BLA; Orphan Drug; RMAT

TBD

midomafetamine

Multidisciplinary Association for Psychedelic Studies

PTSD

Oral

Phase 3 − NDA; Breakthrough Therapy

TBD

minocycline

Journey

Rosacea

Oral

Phase 3 − 505(b)(2) NDA

TBD

minocycline/edetate/ethyl alcohol

Citius

Catheter-related bloodstream infection (CRBSI)

IV

Phase 3 − 505(b)(2) NDA; Fast Track; QIDP

TBD

mirikizumab

Eli Lilly

CD

IV, SC

Phase 3 − BLA

TBD

molnupiravir (Lagevrio)

Merck

COVID-19

Oral

Phase 3 − NDA

TBD

mometasone furoate

Lyra

Chronic rhinosinusitis

Implant

Phase 3 − 505(b)(2) NDA

TBD

mosunetuzumab

Genentech

DLBCL

SC

Phase 3 − BLA; Orphan Drug

TBD

nabiximols

Jazz

MS-related spasticity

Oral transmucosal

Phase 3 − NDA

TBD

nalbuphine ER

Trevi

Pruritus

Oral

Phase 3 − NDA; Fast Track TBD

narsoplimab

Omeros

Hemolytic uremic syndrome

IV, SC

Phase 3 − BLA; Fast Track

TBD

navitoclax

Abbvie/Genenetech

Myelofibrosis

Oral

Phase 3 − NDA; Orphan Drug

TBD

nemolizumab

Galderma

Atopic dermatitis

SC

Phase 3 − BLA

TBD

43 | MAGELLANRX.COM

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MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

nipocalimab

Janssen

Autoimmune hemolytic anemia (warm)

IV

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

nitazoxanide ER

Lupin/Romark

COVID-19; Influenza

Oral

Phase 3 − NDA

TBD

nomacopan

Akari

HSCT-associated thrombotic microangiopathy; PNH

SC

Phase 3 − BLA; Fast Track; Orphan Drug; RPD

TBD

obefazimod

Abivax

UC

Oral

Phase 3 − NDA

TBD

ocrelizumab SC (Ocrevus®)

Genentech

MS

SC

Phase 3 − BLA

TBD

olezarsen

Akcea

Familial chylomicronemia syndrome

SC

Phase 3 − NDA; Fast Track TBD

omalizumab (biosimilar for Genentech’s Xolair®)

Teva

Asthma; Nasal polyps; Urticaria

SC

Phase 3 − BLA

TBD

OPT-302

Opthea

Wet AMD

Intravitreal

Phase 3 − BLA; Fast Track

TBD

padeliporfin

Steba

Bladder cancer

IV

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

paltusotine

Crinetics

Acromegaly

Oral

Phase 3 − NDA; Orphan Drug

TBD

pamrevlumab

Fibrogen

COVID-19; DMD; IV Idiopathic pulmonary fibrosis; Pancreatic cancer

Phase 3 − BLA; Fast Track; Orphan Drug; RPD

TBD

parsaclisib

Incyte

Autoimmune hemolytic anemia (warm)

Oral

Phase 3 − NDA; Orphan Drug

TBD

pegadricase

Swedish Orphan Biovitrum

Gout

IV

Phase 3 − BLA

TBD

piclidenoson

Can-Fite

PSO

Oral

Phase 3 − NDA

TBD

PL-9643

Palatin

DED

Ophthalmic

Phase 3 − NDA

TBD

plastoquinone derivative

Mitotech

DED

Ophthalmic

Phase 3 − NDA

TBD

plinabulin

Beyondspring

Chemotherapy-induced neutropenia prevention; NSCLC

IV

Phase 3 − NDA; Breakthrough Therapy

TBD

pollinex quattro grass

Allergy Therapeutics

Allergic rhinitis

SC

Phase 3 − BLA

TBD

pollinex quattro ragweed

Allergy Therapeutics

Allergic rhinitis

SC

Phase 3 − BLA

TBD

posoleucel

Allovir

Virus-associated hemorrhagic cystitis (post allogeneic HSCT)

IV

Phase 3 − BLA; Orphan Drug; RMAT

TBD

potassium citrate/ potassium bicarbonate

Advicenne

Renal tubular acidosis

Oral

Phase 3 − 505(b)(2) NDA; Orphan Drug

TBD

povorcitinib

Incyte

Hidradenitis suppurativa

Oral

Phase 3 − NDA

TBD

pozelimab

Regeneron

PNH

IV, SC

Phase 3 − BLA

TBD

pramipexole/rasagiline

Pharma Two B

Parkinson’s disease

Oral

Phase 3 − 505(b)(2) NDA

TBD

pritelivir

Aicuris Anti-infective Cures

Herpes simplex virus treatment

Oral

Phase 3 − NDA; Breakthrough Therapy; Fast Track

TBD

prothrombin complex

Octapharma

Hemostasis

IV

Phase 3 − BLA

TBD

proxelutamide

Kintor

COVID-19

Oral

Phase 3 − NDA

TBD

ralinepag

United

PAH

Oral

Phase 3 − NDA; Orphan Drug

TBD

rapamycin (high-strength)

Palvella

Pachyonychia congenita

Topical

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

relacorilant

Corcept

Cushing’s syndrome

Oral

Phase 3 − NDA; Orphan Drug

TBD

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MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

remibrutinib

Novartis

Urticaria

Oral

Phase 3 − NDA

TBD

reproxalap

Aldeyra

Allergic conjunctivitis

Ophthalmic

Phase 3 − NDA

TBD

resmetirom

Madrigal

NASH

Oral

Phase 3 − NDA; Breakthrough Therapy; Fast Track

TBD

rexlemestrocel-L

Mesoblast

Chronic low back pain

Intradiscal

Phase 3 − BLA; RMAT

TBD

RGX-121

Regenxbio

Mucopolysaccharidosis II (Hunter syndrome)

Intracisternal

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

ridinilazole

Summit

C. difficile-associated diarrhea

Oral

Phase 3 − NDA; Fast Track; QIDP

TBD

rilzabrutinib

Sanofi

ITP

Oral

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

rocatinlimab

Amgen

Atopic dermatitis

IV

Phase 3 − BLA

TBD

roflumilast foam

Arcutis

PSO

Topical

Phase 3 − NDA

TBD

roluperidone

Minerva

Schizophrenia (negative symptoms)

Oral

Phase 3 − NDA

TBD

RSV pre-fusion F protein vaccine (mRNA-1345)

Moderna

RSV prevention (ages ≥ 60 years)

IM

Phase 3 − BLA; Breakthrough Therapy; Fast Track

TBD

ruxolitinib (deuterated)

Concert

Alopecia areata

Oral

Phase 3 − NDA; Breakthrough Therapy; Fast Track

TBD

sabatolimab

Novartis

Myelodysplastic syndrome

IV

Phase 3 − BLA; Fast Track

TBD

sabizabulin

Veru

COVID-19

Oral

Phase 3 − NDA; Fast Track TBD

saroglitazar

Zydus

Primary biliary cholangitis Oral

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

sebetralstat

Kalvista

HAE

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

seladelpar

Cymabay

Primary biliary cholangitis Oral

Phase 3 − NDA; Breakthrough Therapy; Orphan Drug

TBD

seltorexant

Janssen

MDD

Oral

Phase 3 − NDA

TBD

serplulimab

Henlius

SCLC

IV

Phase 3 − BLA; Orphan Drug

TBD

sotatercept

Merck

PAH

SC

Phase 3 − BLA; Breakthrough Therapy; Orphan Drug

TBD

sulopenem etzadroxil

Iterum

UTI (uncomplicated)

Oral

Phase 3 − NDA; Fast Track; QIDP

TBD

tabelecleucel

Atara

Epstein-Barr virus-associated post-transplant lymphoproliferative disease

IV

Phase 3 − BLA; Breakthrough Therapy; Orphan Drug

TBD

TAK-755

Takeda

Thrombotic thrombocytopenic purpura (TTP)

IV

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

tamibarotene

Syros

Myelodysplastic syndrome

Oral

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

tanfanercept

Hanall

DED

Ophthalmic

Phase 3 − BLA

TBD

45 | MAGELLANRX.COM

Oral

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

tarcocimab tedromer

Kodiak

DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD

Intravitreal

Phase 3 − BLA

TBD

tebipenem pivoxil

GlaxoSmithKline

UTI (complicated)

Oral

Phase 3 − NDA; Fast Track; QIDP

TBD

tecarfarin

Espero

Anticoagulation

Oral

Phase 3 − NDA

TBD

telisotuzumab vedotin

Abbvie

NSCLC

IV

Phase 3 − BLA; Breakthrough Therapy

TBD

tiragolumab

Genentech

Esophageal cancer; NSCLC

IV

Phase 3 − BLA; Breakthrough Therapy; Orphan Drug

TBD

tiratricol

Rare Thyroid Therapeutics

Monocarboxylate transporter 8 (MCT8) deficiency

Oral

Phase 3 − NDA; Fast Track; Orphan Drug; RPD

TBD

tislelizumab

Beigene/Novartis

Gastric cancer; HCC; Nasopharyngeal cancer; NSCLC

IV

Phase 3 − BLA; Orphan Drug

TBD

tixagevimab/cilgavimab (Evusheld)

AstraZeneca

COVID-19

IM

Phase 3 − BLA

TBD

tolebrutinib

Sanofi

MS

Oral

Phase 3 − NDA

TBD

toripalimab

Coherus

Esophageal cancer; HCC; NSCLC

IV

Phase 3 − BLA; Orphan Drug

TBD

tovorafenib

Day One Bio

Brain cancer

Oral

Phase 3 − NDA; Breakthrough Therapy; Orphan Drug; RPD

TBD

tradipitant

Vanda

Atopic dermatitis; COVID-19; Emesis; Gastroparesis; Pruritus

Oral

Phase 3 − NDA

TBD

tramiprosate

Alzheon

Alzheimer’s disease

Oral

Phase 3 − NDA; Fast Track TBD

tusamitamab ravtansine

Sanofi

NSCLC

IV

Phase 3 − BLA

TBD

ulotaront

Sumitomo

MDD; Schizophrenia

Oral

Phase 3 − NDA; Breakthrough Therapy

TBD

upifitamab rilsodotin

Mersana

Ovarian cancer

IV

Phase 3 − BLA; Fast Track

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Amgen

PSO; PsA; CD; UC

IV, SC

Phase 3 − BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Formycon

PSO; PsA; CD; UC

IV, SC

Phase 3 − BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Hikma

PSO; PsA; CD; UC

IV, SC

Phase 3 − BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Intas

PSO; PsA; CD; UC

IV, SC

Phase 3 − BLA

TBD

venglustat

Sanofi

GM2 gangliosidoses (TaySachs disease, Sandhoff disease, AB variant)

Oral

Phase 3 − NDA; Orphan Drug

TBD

von Willebrand factor concentrate

LFB

von Willebrand disease

IV

Phase 3 − BLA; Orphan Drug

TBD

vonoprazan

Phathom

Non-erosive gastroesophageal reflux disease

Oral

Phase 3 − NDA

TBD

xanomeline/trospium

Karuna

Schizophrenia

Oral

Phase 3 − NDA

TBD

46 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

zanidatamab

Zymeworks

Gastric cancer

IV

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

zolbetuximab

Astellas

Gastric cancer

IV

Phase 3 − BLA; Orphan Drug

TBD

zoliflodacin

Innoviva

Gonorrhea

Oral

Phase 3 − NDA; Fast Track; QIDP

TBD

Phase 3 (Supplementals) alpelisib (Piqray®)

Novartis

Ovarian cancer

Oral

Phase 3 − sNDA

TBD

atezolizumab (Tecentriq)

Genentech

RCC; SCCHN

IV

Phase 3 − sBLA

TBD

baricitinib (Olumiant )

Eli Lilly

JIA; Uveitis

Oral

Phase 3 − sNDA

TBD

benralizumab (Fasenra )

AstraZeneca

ANCA-associated vasculitis; Bronchiectasis; Bullous pemphigoid; Esophagitis

SC

Phase 3 − sBLA; Orphan Drug

TBD

brexpiprazole (Rexulti)

Otsuka

PTSD

Oral

Phase 3 − sNDA

TBD

Novartis

NSCLC

SC

Phase 3 − sBLA

TBD

cemiplimab-rwlc (Libtayo )

Regeneron

Melanoma

IV

Phase 3 − sBLA

TBD

dupilumab (Dupixent)

Sanofi

Allergic fungal rhinosinusitis; Bullous pemphigoid; Chronic rhinosinusitis; COPD

SC

Phase 3 − sBLA; Orphan Drug

TBD

durvalumab (Imfinzi)

AstraZeneca

Bladder cancer; Gastric cancer

IV

Phase 3 − sBLA; Breakthrough Therapy

TBD

efgartigimod (Vygart®)

Argenx

ITP

IV

Phase 3 − sBLA; Orphan Drug

TBD

empagliflozin (Jardiance)

Boehringer Ingelheim/Eli Lilly

Diabetic nephropathy

Oral

Phase 3 − sNDA; Fast Track

TBD

faricimab-svoa (Vabysmo®)

Genentech

Macular edema following RVO

Intravitreal

Phase 3 − sBLA

TBD

ferric carboxymaltose (Injectafer®)

Daiichi Sankyo

Anemia in heart failure

IV

Phase 3 − sNDA

TBD

ferric derisomaltose (Monoferric®)

Pharmacosmos

Anemia in heart failure

IV

Phase 3 − sNDA

TBD

fostamatinib (Tavalisse®)

Rigel

Autoimmune hemolytic anemia (warm)

Oral

Phase 3 − sNDA; Fast Track; Orphan Drug

TBD

guselkumab (Tremfya®)

Janssen

UC

SC

Phase 3 − sBLA

TBD

hydrogen peroxide (Eskata®)

Aclaris

Warts

Topical

Phase 3 − sNDA

TBD

iloperidone (Fanapt®)

Vanda

Bipolar disorder

Oral

Phase 3 − sNDA

TBD

immune globulin, human 10% (Octagam®)

Octapharma

COVID-19

IV

Phase 3 − sBLA

TBD

inebilizumab-cdon (Uplizna®)

Horizon

IgG4-related disease; Myasthenia gravis

IV

Phase 3 − sBLA

TBD

lumateperone (Caplyta®)

Intra-Cellular Therapies

MDD

Oral

Phase 3 − sNDA

TBD

mepolizumab (Nucala®)

GlaxoSmithKline

COPD

IV, SC

Phase 3 − sBLA

TBD

meropenem/vaborbactam (Vabomere®)

Melinta

Bacteremia; HAP

IV

Phase 3 − sNDA; QIDP

TBD

mitapivat (Pyrukynd®)

Agios

Thalassemia; SCD

Oral

Phase 3 − sNDA; Orphan Drug

TBD

®

®

canakinumab (Ilaris ) ®

®

47 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

omalizumab (Xolair)

Genentech

Food allergies

SC

Phase 3 − sBLA; Breakthrough Therapy

TBD

pegcetacoplan (Empaveli®)

Apellis

Autoimmune hemolytic anemia (warm)

SC

Phase 3 − sNDA; Orphan Drug

TBD

ranibizumab port delivery system (Susvimo®)

Genentech

Diabetic retinopathy

Intravitreal

Phase 3 − sBLA

TBD

rimegepant (Nurtec ODT®)

Pfizer

Chronic rhinosinusitis; Nasal polyposis

Oral

Phase 3 − sNDA

TBD

risankizumab-rzaa (Skyrizi®)

Abbvie

UC

IV, SC

Phase 3 − sBLA

TBD

rivaroxaban (Xarelto®)

Janssen

COVID-19

Oral

Phase 3 − sNDA

TBD

roflumilast (Zoryve)

Arcutis

Atopic dermatitis

Topical

Phase 3 − sNDA

TBD

romiplostim (Nplate )

Amgen

Chemotherapy-induced thrombocytopenia

SC

Phase 3 − sBLA; Orphan Drug

TBD

selinexor (Xpovio)

Karyopharm

Endometrial cancer

Oral

Phase 3 − sNDA

TBD

sparsentan (Filspari )

Travere

Focal segmental glomerulosclerosis

Oral

Phase 3 − sNDA; Orphan Drug

TBD

tapinarof (Vtama®)

Roivant

Atopic dermatitis

Topical

Phase 3 − sNDA

TBD

tezepelumab-ekko (Tezspire®)

Amgen

Chronic rhinosinusitis; Esophagitis; Nasal polyposis

SC

Phase 3 − sBLA; Orphan Drug

TBD

tirzepatide (Mounjaro™)

Eli Lilly

Obesity/overweight (weight-related comorbidities)

SC

Phase 3 − sNDA; Fast Track

TBD

upadacitinib (Rinvoq)

Abbvie

Giant cell arteritis

Oral

Phase 3 − sNDA

TBD

vedolizumab (Entyvio)

Takeda

GVHD prophylaxis

IV

Phase 3 − sBLA; Orphan Drug

TBD

venetoclax (Venclexta®)

Abbvie/Genenetech

Mantle cell lymphoma; Multiple myeloma; Myelodysplastic syndrome

Oral

Phase 3 − sNDA; Breakthrough Therapy; Orphan Drug

TBD

®

®

Complete Response Letter (CRL) NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

adalimumab 100 mg/mL (biosimilar to Abbvie’s Humira)

Alvotech

RA; AS; PSO; PsA; JIA; CD; UC

SC

CRL

TBD

levodopa/carbidopa pump

Abbvie

Parkinson’s disease motor SC fluctuations

CRL

TBD

mirikizumab

Eli Lilly

UC

IV, SC

CRL

TBD

naloxone spray

Orexo

Opioid overdose

Intranasal

CRL

TBD

omecamtiv mecarbil

Cytokinetics

Chronic heart failure (with Oral reduced ejection fraction)

CRL

TBD

ruxolitinib XR

Incyte

GVHD; Myelofibrosis, Polycythemia vera

Oral

CRL

TBD

vonoprazan

Phathom

Erosive esophagitis

Oral

CRL

TBD

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GLOSSARY 6MWT 6 Minute Walking Test

CAP Community Acquired Pneumonia

ABSSSI Acute Bacterial Skin and Skin Structure Infection

CAR T Chimeric Antigen Receptor T Cell

ACC American College of Cardiology

CD3 Cluster of Differentiate 3

ACEI Angiotensin-Converting Enzyme Inhibitor ACR20 American College of Rheumatology 20% Improvement ACR50 American College of Rheumatology 50% Improvement

CD Crohn's Disease CD19 Cluster of Differentiate 19 CD20 Cluster of Differentiate 20 CD38 Cluster of Differentiate 38 CD79b Cluster of Differentiate 79b

ACR70 American College of Rheumatology 70% Improvement

CDC Centers for Disease Control and Prevention

ADC Antibody-Drug Conjugate

CHF Congestive Heart Failure

ADHD Attention Deficit Hyperactivity Disorder

CI Confidence Interval

ADL Activities of Daily Living

CKD Chronic Kidney Disease

AED Anti-Epileptic Drug

CLL Chronic Lymphocytic Leukemia

AHA American Heart Association

CML Chronic Myeloid Leukemia

ALK Anaplastic Lymphoma Kinase

CMS Centers for Medicare & Medicaid Services

ALL Acute Lymphoblastic Leukemia

CNS Central Nervous System

ALS Amyotrophic Lateral Sclerosis

COPD Chronic Obstructive Pulmonary Disease

ALT Alanine Transaminase

COVID-19 Coronavirus Disease 2019

AMD Age-Related Macular Degeneration

CRC Colorectal Cancer

AML Acute Myeloid Leukemia

CRL Complete Response Letter

ANCA Antineutrophil Cytoplasmic Antibodies

CRR Complete Response Rate

ANDA Abbreviated New Drug Application

CRS Cytokine Release Syndrome

ARB Angiotensin II Receptor Blocker

CSF Colony Stimulating Factor

ARNI Angiotensin Receptor-Neprilysin Inhibitor

CV Cardiovascular

ART Antiretroviral Therapy

CVD Cardiovascular Disease

ARV Antiretroviral

CYP Cytochrome P-450

AS Ankylosing Spondylitis

CYP3A4 Cytochrome P-450 3A4

ASCVD Atherosclerotic Cardiovascular Disease AST Aspartate Aminotransferase

DAS28-CRP Disease Activity Score-28 with C Reactive Protein

BCG Bacillus Calmette-Guérin

DCR Disease Control Rate

BCVA Best Corrected Visual Acuity

DEA Drug Enforcement Administration

BLA Biologics License Application

DED Dry Eye Disease

BMI Body Mass Index

DLBCL Diffuse Large B Cell Lymphoma

BMT Bone Marrow Transplant

DMARD Disease Modifying Antirheumatic Drug

BPH Benign Prostatic Hyperplasia

DMD Duchenne Muscular Dystrophy

BSA Body Surface Area

DME Diabetic Macular Edema

BsUFA Biosimilar User Fee Act

DMT Disease Modifying Therapy

CABP Community Acquired Bacterial Pneumonia

DNA Deoxyribonucleic Acid

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CF Cystic Fibrosis

GLOSSARY continued DOR Duration of Response

HER Human Epidermal Growth Factor Receptor

DPI Dry Powder for Inhalation

HER2 Human Epidermal Growth Factor Receptor 2

DPP-4 Dipeptidyl Peptidase 4

HF Heart Failure

DR Delayed-Release

HFA Hydrofluoroalkane

EASI-75 Eczema Area and Severity Index ≥ 75% Reduction

HFpEF Heart Failure with preserved Ejection Fraction

ECOG Eastern Cooperative Oncology Group

HIT Heparin Induced Thrombocytopenia

EDSS Expanded Disability Status Scale eGFR estimated Glomerular Filtration Rate EGFR Epidermal Growth Factor Receptor ER Extended-Release ESA Erythropoietin Stimulating Agent ESRD End-Stage Renal Disease EUA Emergency Use Authorization FDA Food and Drug Administration FH Familial Hypercholesterolemia FLT3 FMS-Like Tyrosine Kinase-3 FMS Feline McDonough Sarcoma GABA-A Gamma-Aminobutyric Acid Receptor Type A G-CSF Granulocyte Colony Stimulating Factor GI Gastrointestinal GIST Gastrointestinal Stromal Tumor GLP-1RA Glucagon-Like Peptide-1 Receptor Agonist GM-CSF Granulocyte-Macrophage Colony Stimulating Factor GVHD Graft Versus Host Disease H Half HAART Highly Active Antiretroviral Therapy HAE Hereditary Angioedema HAM-A Hamilton Anxiety Rating Scale HAM-D Hamilton Depression Rating Scale HAMD-17 Hamilton Depression Rating Scale HAP Healthcare-Associated Pneumonia Hb Hemoglobin HbA1c Hemoglobin A1c HBV Hepatitis B Virus HCC Hepatocellular Carcinoma HCP Healthcare Professional HCV Hepatitis C Virus HDRS-17 Hamilton Depression Rating Scale

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HFSA Heart Failure Society of America HIV Human Immunodeficiency Virus HIV-1 Human Immunodeficiency Virus-1 HPV Human Papilloma Virus HR Hazard Ratio HSCT Hematopoietic Stem Cell Transplant HSV Herpes Simplex Virus HTN Hypertension IBS Irritable Bowel Syndrome IBS-C Irritable Bowel Syndrome, Constipation Predominant ICU Intensive Care Unit IDSA Infectious Diseases Society of America IGA Investigator's Global Assessment IgG Immunoglobulin G IgG1kappa Immunoglobulin G1 kappa IL-4 Interleukin-4 IL-12 Interleukin-12 IL-13 Interleukin-13 IL-17 Interleukin-17 IL-23 Interleukin-23 IM Intramuscular IR Immediate-Release IRB Institutional Review Board ITP Immune Thrombocytopenic Purpura ITT Intention-To-Treat IV Intravenous JAK Janus Kinase Inhibitor JIA Juvenile Idiopathic Arthritis LDL Low-Density Lipoprotein LDL-C Low-Density Lipoprotein Cholesterol LPAD Limited Population Pathway for Antibacterial and Antifungal Drugs LVEF Left Ventricular Ejection Fraction mAb Monoclonal Antibody

GLOSSARY continued MACE Major Adverse Cardiovascular Events

PCI Percutaneous Coronary Intervention

MADRS Montgomery – Åsberg Depression Rating Scale

PCSK9 Proprotein Convertase Subtilisin Kexin 9

MAOI Monoamine Oxidase Inhibitor

PD-L1 Programmed Death-Ligand 1

MDD Major Depressive Disorder MDI Metered Dose Inhaler MDR Multi-Drug Resistant MECP2 Methyl-CpG Binding Protein 2 MI Myocardial Infarction mITT modified Intention-To-Treat MRI Magnetic Resonance Imaging MRSA Methicillin-Resistant Staphylococcus Aureus

PD-1 Programmed Death Protein 1 PDUFA Prescription Drug User Fee Application PFS Progression-Free Survival PGA Physician Global Assessment PI3K Phosphatidylinositol-3-kinase PNH Paroxysmal Nocturnal Hemoglobinuria PsA Psoriatic Arthritis PSO Plaque Psoriasis

MS Multiple Sclerosis

PTCA Percutaneous Transluminal Coronary Angioplasty

N/A Not Applicable

PTSD Post-Traumatic Stress Disorder

NASH Non-Alcoholic Steatohepatitis

Q Quarter

NCCN National Comprehensive Cancer Network

QIDP Qualified Infectious Diseases Product

NCT National Clinical Trials

QOL Quality of Life

NDA New Drug Application

R/R Relapsed or Refractory

NHL Non-Hodgkin Lymphoma

R-CHOP Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, Prednisone

NIAID National Institute of Allergy and Infectious Diseases

RA Rheumatoid Arthritis

NSAID Non-Steroidal Anti-Inflammatory Drug

RBC Red Blood Cell

NSCLC Non-Small Cell Lung Cancer

RCC Renal Cell Carcinoma

NYHA New York Heart Association

REMS Risk Evaluation and Mitigation Strategy

ODT Orally Disintegrating Tablet

RMAT Regenerative Medicine Advanced Therapy

OR Odds Ratio

RNA Ribonucleic Acid

ORR Overall/Objective Response Rate

RPD Rare Pediatric Disease

OS Overall Survival

RRR Relative Risk Reduction

OTC Over-the-Counter

RSV Respiratory Syncytial Virus

PAH Pulmonary Arterial Hypertension

RTOR Real-Time Oncology Review

PARP Poly(ADP-Ribose) Polymerase

RVO Retinal Vein Occlusion

PAS Prior Approval Supplement PASI Psoriasis Area and Severity Index

SARS-CoV-2 Severe Acute Respiratory SyndromeAssociated Coronavirus-2

PASI 50 Psoriasis Area and Severity Index 50% Reduction

sBLA supplemental Biologics License Application SC Subcutaneous

PASI 75 Psoriasis Area and Severity Index 75% Reduction

SCCHN Squamous Cell Cancer of the Head and Neck

PASI 90 Psoriasis Area and Severity Index 90% Reduction

SCLC Small Cell Lung Cancer

PASI 100 Psoriasis Area and Severity Index 100% Reduction

SCD Sickle Cell Disease SCT Stem Cell Transplant SGLT2 Sodium-Glucose Co-Transporter 2 SL Sublingual

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GLOSSARY continued SLE Systemic Lupus Erythematosus SLL Small Lymphocytic Lymphoma sNDA supplemental New Drug Application SNRI Serotonin and Norepinephrine Reuptake Inhibitor SOC Standard of Care sPGA static Physician Global Assessment SR Sustained-Release SSRI Selective Serotonin Reuptake Inhibitor SSSI Skin and Skin Structure Infection T1DM Type 1 Diabetes Mellitus T2DM Type 2 Diabetes Mellitus TBD To Be Determined TEAE Treatment-Emergent Adverse Event TNBC Triple Negative Breast Cancer TNF Tumor Necrosis Factor TNFα Tumor Necrosis Factor-alpha UA Unstable Angina UC Ulcerative Colitis US United States UTI Urinary Tract Infection VAS Visual Analog Scale VEGF Vascular Endothelial Growth Factor VTE Venous Thromboembolism WBC White Blood Cell WHO World Health Organization XR Extended-Release

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MRx PIPELINE A VIEW INTO UPCOMING SPECIALTY & TRADITIONAL DRUGS

JANUARY 2022

© Prime Therapeutics LLC | Magellan Rx, a wholly owned subsidiary of Prime Therapeutics LLC. MRX1119_0423