MRx Pipeline - January 2021

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MRx PIPELINE A VIEW INTO UPCOMING SPECIALTY & TRADITIONAL DRUGS JANUARY 2021


EDITORIAL STAFF Maryam Tabatabai, PharmD Editor-in-Chief Vice President, Clinical Information Carole Kerzic, RPh Executive Editor Drug Information Pharmacist Consultant Panel Michelle Booth, PharmD Director, Medical Pharmacy Strategy Lara Frick, PharmD, BCPS, BCPP Drug Information Pharmacist Robert Greer, RPh, BCOP Senior Director, Clinical Strategy and Programs Katie Lockhart Manager, Forecasting and Pharmacoeconomics Brian MacDonald, PharmD Senior Manager, Specialty Clinical Programs

Table of CONTENTS

Troy Phelps Senior Director, COAR - Analytics

EDITOR-IN-CHIEF'S MESSAGE

2

PIPELINE DEEP DIVE

3

KEEP ON YOUR RADAR

20

PIPELINE DRUG LIST

21

GLOSSARY

41

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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.


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 2025. 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.

LOOKING BACK

Despite the pandemic's unprecedented challenges, in 2020, the US FDA approved 53 novel drugs, making it the second highest number of approvals in 10 years. Notably, 58% of these approvals were for patients with Orphan conditions. Of note, the all-time record approvals was in 2018 with 59 novel drugs approved. Furthermore, the last 3 years share the distinction of having the highest number of approvals in the last 10 years. Remarkably, in late 2020, two life-saving COVID-19 vaccines received Emergency Use Authorization (EUA). While numbers do not tell the entire story, they do represent incredible advances in patient care and hope for the American public.

ON THE HORIZON

As we look ahead, there is a continued trend toward the approval of specialty medications and drugs for rare and ultra rare diseases, with 61% and 34% of approvals expected, respectively, for agents with applications submitted to the FDA. The growth of biosimilars, new treatment modalities using gene therapy, and additional CAR T therapies are expected. A public health arsenal of COVID-19 therapeutics and vaccines, including a single-dose shot, will make 2021 a pivotal year in combating this deadly infection. Other noteworthy pipeline trends to watch include the development of complex therapies, oncology, immunology, therapeutic options for rare hereditary diseases, and a possible new therapy for Alzheimer’s disease. Moreover, sprouting products for hematology, ophthalmology, and diabetes await on the horizon. 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

90%

24%

10%

BIOSIMILAR

ORPHAN DRUG

62%

29%

pecialty drug names appear in ï‚« S magenta throughout the publication.


IMMUNOLOGY

avacopan oral Chemocentryx PROPOSED INDICATIONS

Antineutrophil cytoplasmic antibodies (ANCA)-associated vasculitis (AAV)

CLINICAL OVERVIEW

Hyperactivity of the complement pathway activates neutrophils causing damage to small blood vessels. Avacopan selectively inhibits the complement 5a receptor (C5aR) that is present on neutrophils. The double-blind, phase 3 ADVOCATE trial evaluated safety and efficacy of avacopan in 330 patients with AAV (granulomatosis with polyangiitis or microscopic polyangiitis). Patients were randomized 1:1 to avacopan 30 mg twice daily or standard prednisone therapy. Both groups also received either cyclophosphamide followed by azathioprine, or rituximab. The primary endpoint of disease remission was achieved in 72.3% and 70.1% of patients who received avacopan and prednisone, respectively, at week 26 (p<0.0001 for non-inferiority) and in 65.7% and 54.9% of patients, respectively, at week 52 (p=0.0066 for superiority). Avacopan resulted in longer remission compared to prednisone (HR, 0.46). In addition, among patients with renal impairment at baseline, greater improvement in function at week 52 was reported with avacopan compared to prednisolone (eGFR increase, 7.3 versus 4.1 mL/min/1.73 m2, respectively; p=0.029). Overall, fewer serious adverse events occurred with avacopan than with prednisone (25 versus 31 events, respectively). This included WBC decline, which was reported in 2.5% and 4.9% of patients, respectively. Conversely, liver function test increases were reported more often with avacopan than with prednisone (5.4% and 3.7%, respectively).

PLACE IN THERAPY

The estimated annual incidence of AAV in Europe and North America is 20 per million people. AAV encompasses a group of diseases including granulomatosis with polyangiitis (GPA), eosinophilic granulomatosis with polyangiitis (EGPA), and microscopic polyangiitis (MPA). The rare autoimmune disease occurs when neutrophils attack small blood vessels causing inflammation, damage, and possible necrosis. AAV affects several organs and signs and symptoms range from skin rash to fulminant multisystem disease. Staphylococcus aureus infection, select medications, and exposure to environmental toxins may trigger an AAV episode. If left untreated, AAV leads to significant morbidity and mortality, with a 75% survival rate at 5 years. Standard induction therapy for severe AAV episodes includes high-dose glucocorticoids plus immunosuppressive therapy (cyclophosphamide or rituximab). In patients with clinical decline despite ongoing induction therapy, plasma exchange may be added. For non-severe AAV cases, methotrexate, mycophenolate mofetil, or azathioprine may be used, depending on the patients’ clinical status. Overall, remission is typically maintained with azathioprine or methotrexate. In addition, the interleukin-5 (IL-5) inhibitor mepolizumab (Nucala®) is approved for SC administration in combination with oral corticosteroids in adults with EGPA. Avacopan offers a new approach to treat AAV that targets the C5aR. It demonstrated improvement in achieving and maintaining remission over oral steroid therapy when coupled with standard immunosuppressant therapy. Avocopan will compete with cyclophosphamide, rituximab (Rituxan®, biosimilars), and mepolizumab (Nucala) in the AAV space. Moreover, avacopan may decrease the need for steroid therapy and the incidence of steroidrelated adverse effects. The novel C5aR inhibitor is also in phase 2 trials for C3 glomerulopathy (C3G) and hidradenitis suppurativa (HS).

FDA APPROVAL TIMELINE July 7, 2021

 Orphan Drug

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$25

$111

$253

$391

$532

The forecast is a projection of total US sales per year.

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IMMUNOLOGY

belumosudil oral Kadmon PROPOSED INDICATIONS

Chronic graft versus host disease (cGVHD)

CLINICAL OVERVIEW

Belumosudil is a Rho-associated coiled-coil kinase 2 (ROCK2) inhibitor. ROCK2 has been shown to play a critical role in the regulation of proinflammatory cytokines, including interleukin 17 (IL-17) and interleukin 21 (IL-21). It is also involved in regulation of interferon gamma, T follicular helper and regulatory cells, and signal transducer and activator of transcription (STAT) 3 and 5 that are believed to be involved in development of cGVHD. The ongoing, open-label, phase 2 ROCKstar trial evaluated 2 doses of belumosudil in patients with cGVHD who had received ≥ 2 prior lines of systemic therapies, including prior ibrutinib or ruxolitinib. Among enrollees, 67% had severe cGVHD and 52% had involvement of > 4 organs. Patients were randomized 1:1 to receive oral belumosudil 200 mg either once or twice daily. In the 12-month interim analysis (median follow-up, 8 months), the primary endpoint of ORR was 73% with once-daily dosing and 77% with twice-daily dosing (p<0.001 for each). Median time to response was 4 weeks. Overall, secondary endpoints demonstrated a median DOR of 50 weeks, and 58% of patients were failure-free at 12 months. Improved QOL, as measured by the Lee Symptom Scale, was reported in 60% of patients. Corticosteroid use was decreased or discontinued in 64% and 21% of patients, respectively. Belumosudil was well tolerated. Adverse effects were similar to those observed with corticosteroids and other immunosuppressants used for cGVHD.

PLACE IN THERAPY

Chronic GVHD is a complication of allogenic HSCT in which the transplanted cells attack host cells resulting in inflammation and fibrosis. Chronic GVHD can be life-threatening, affecting multiple organ systems (e.g., skin, lungs, GI tract, liver, eyes, joints), and is reported in > 50% of allogenic HSCT recipients. Kadmon reports approximately 14,000 patients in the US are currently living with cGVHD. High-risk cGVHD requires systemic therapy and is associated with involvement of ≥ 3 organs, severity score > 2 in any single organ, persistent thrombocytopenia, or cGVHD that has progressed from acute GVHD. The mainstay of systemic therapy for moderate to severe cGVHD is glucocorticoids as well as supportive care to specific sites. If patients are unresponsive to glucocorticoids, the addition of a calcineurin inhibitor (e.g., cyclosporine, tacrolimus) may be considered. Ibrutinib (Imbruvica®) is also FDA-approved for cGVHD after failure of systemic therapy. Off-label use of imatinib, mycophenolate mofetil, rituximab, ruxolitinib, or sirolimus has also demonstrated benefit. Notably, patients with cGVHD are encouraged to enroll in a clinical trial. Belumosudil may inhibit key fibrotic processes associated with cGVHD. It demonstrated significant response in treating patients with cGVHD, and lacks significant safety concerns at this time. If approved, it will provide an oral option for patients with cGVHD who have failed ≥ 2 prior lines of systemic therapies. Belumosudil is also in phase 2 trials for idiopathic pulmonary fibrosis and cutaneous scleroderma. Ruxolitinib (Jakafi®) has also been submitted to the FDA for cGVHD; the agency's decision is anticipated in 2H 2021.

FDA APPROVAL TIMELINE May 30, 2021

 Breakthrough Therapy

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$43

$137

$205

$274

$321

The forecast is a projection of total US sales per year.

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 Project Orbis

 RTOR


IMMUNOLOGY

bimekizumab SC UCB PROPOSED INDICATIONS

Moderate to severe chronic plaque psoriasis (PSO)

CLINICAL OVERVIEW

Bimekizumab is a monoclonal IgG1 antibody that inhibits interleukins 17A and 17F (IL-17 and IL-17F). The efficacy of bimekizumab was demonstrated in 4 pivotal randomized, double-blind, phase 3 trials in over 2,200 adults with moderate to severe PSO. Effectiveness was measured based on the primary endpoints of improvement in the PASI and IGA scores. » B E SURE (n=478): Bimekizumab was superior to adalimumab based on PASI 90 (86.2% versus 47.2%, respectively; p<0.001) and an IGA of 0 (clear) or 1 (almost clear) (85.3% versus 57.2%, respectively; p<0.001) at week 16. In addition, at week 24, an increase in skin clearance was observed after switching from adalimumab to bimekizumab. » B E RADIANT (n=743): Bimekizumab was superior to secukinumab based on PASI 100 at week 16. Superiority to secukinumab was also observed based on the secondary endpoints of PASI 75 at week 4 and PASI 100 at week 48. » B E VIVID (n=567): Superiority of bimekizumab compared to ustekinumab was demonstrated based on PASI 90 and IGA at week 16. Bimekizumab was also superior to ustekinumab based on the secondary endpoints of PASI 100 at week 52 (64.2% versus 38%, respectively; nominal p<0.001) and in achieving IGA of 0 or 1 and PASI 90 at week 52 compared with ustekinumab (IGA, 77.9% versus 60.7%, respectively; PASI 90, 81.6% versus 55.8%, respectively; p<0.001 for both). » B E READY (n=435): PASI 90 was maintained at 56 weeks in 86.8% of patients treated with continuous bimekizumab every 4 weeks and in 91% of patients who were switched to bimekizumab every 8 weeks compared to 16.2% who were switched to placebo. Across the studies, bimekizumab was generally well-tolerated. The most common TEAEs were nasopharyngitis, oral candidiasis, and upper respiratory tract infection. The overall rate of TEAEs were comparable with bimekizumab and adalimumab; however, serious TEAEs were reported more often with adalimumab than bimekizumab (3.1% versus 1.6%, respectively). No suicidal ideation/behavior, inflammatory bowel disease, or major adverse cardiac events were reported with bimekizumab (BE SURE). In BE VIVID, serious TEAEs were reported in 6.1% of patients treated with bimekizumab versus 7.4% treated with ustekinumab (at week 52).

PLACE IN THERAPY

Psoriasis is a chronic, multisystem, immune-mediated, inflammatory disease involving the skin and joints and characterized by hyperproliferation of epidermal keratinocytes. It affects an estimated 8 million people in the US. Median age at onset is 28 years. Treatment for PSO is based on severity of the condition. Targeted immunomodulators are indicated to treat moderate to severe PSO after failure of topical therapy alone when phototherapy is not available. Immunomodulators include injectable monoclonal antibodies which reduce the level of pathogenic cytokines (TNFα, IL-23, and IL-17), and the oral phosphodiesterase 4 (PDE4) inhibitor apremilast (Otezla®), which reduces the production of proinflammatory mediators. Bimekizumab is a monoclonal IgG1 antibody that inhibits IL-17A and IL-17F. In clinical trials, bimekizumab was generally well tolerated, but it lacks safety and efficacy data beyond 56 weeks. Although no safety concerns were identified with bimekizumab, safety signals have been associated with the anti-IL-17 class of medications. Brodalumab (Siliq®) carries a boxed warning for suicidal ideation, and inflammatory bowel disease exacerbations have been reported with secukinumab (Cosentyx®). If approved, bimekizumab will be

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bimekizumab cont. PLACE IN THERAPY cont.

the first monoclonal IgG1 antibody targeting IL-17A and IL-17F in the US to treat PSO. Effectiveness of every 4 week and every 8 week dosing were comparable; therefore, bimekizumab could be a self-administered option that is dosed as infrequently as every 8 weeks. Bimekizumab will compete with several biological agents that are currently available to treat PSO, particularly the TNFα inhibitor adalimumab (Humira®), IL-17A inhibitor secukinumab (Cosentyx®), and IL-12/23 inhibitor ustekinumab (Stelara®). In clinical trials, bimekizumab demonstrated superiority to all 3 of these agents. Other investigational biologics in late-stage development for moderate to severe PSO include mirikizumab (IL-23 inhibitor; Eli Lilly), spesolimab (IL-36R inhibitor; Boehringer Ingelheim), as well as biosimilars for ustekinumab. Bimekizumab is also in phase 3 studies for the treatment of psoriatic arthritis, axial spondyloarthritis, and hidradenitis suppurativa.

FDA APPROVAL TIMELINE July 2021

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$44

$205

$399

$601

$815

The forecast is a projection of total US sales per year and reflects future potential indications for bimekizumab.

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CARDIOLOGY

evinacumab IV Regeneron PROPOSED INDICATIONS

Homozygous familial hypercholesterolemia (HoFH) as adjunct to other lipid-lowering therapies

CLINICAL OVERVIEW

Evinacumab is a monoclonal antibody that inhibits the function of the angiopoietin-like 3 (ANGPTL3) protein. ANGPTL3 is expressed naturally in the liver and acts as a partial inhibitor of lipoprotein lipase and endothelial lipase. ANGPTL3 plays a key role in lipid metabolism by increasing the levels of triglycerides and other lipids. Inhibition of ANGPTL3 is associated with reduced LDL-C levels independent of the LDL receptor. The phase 3, double-blind, parallel-group ELIPSE HoFH trial is evaluating the efficacy and safety of evinacumab in 65 patients aged ≥ 12 years with HoFH who have achieved LDL-C ≥ 70 mg/dL on stable maximally tolerated lipid-lowering therapy. Interim data revealed that after 24 weeks of therapy, evinacumab resulted in a 47.1% reduction in LDL-C from baseline (primary endpoint) compared to a 1.9% increase with placebo (difference, -49%; 95% CI, -65 to -33.1; p<0.001). The LDL-C reduction with evinacumab was reported as early as week 2 of the study. Notably, LDL-C reductions were similar among those with null-null and non-null variants. LDL-reductions were also similar regardless of background anti-lipid therapies (e.g., statins, ezetimibe, lomitapide, PCSK9 inhibitors, apheresis). Serious adverse events reported in the evinacumab group were urosepsis and suicide attempt (1 case each). No deaths occurred. In the clinical study, evinacumab was administered as 15 mg/kg IV every 4 weeks.

PLACE IN THERAPY

HoFH is an autosomal dominant disorder that results in severe elevations in total cholesterol and LDL-C. In individuals with HoFH, cardiovascular disease and cerebrovascular disease as well as cutaneous xanthomas may emerge during childhood. It is estimated that HoFH occurs in 1 out of a million people in the US. Life expectancy of individuals with HoFH is approximately 30 years unless treated with a liver transplant, lipoprotein apheresis, or ileal bypass surgery. LDL-C lowering is a main objective of HoFH treatment, which consists of lifestyle modifications, pharmacotherapy with statins, and lipoprotein apheresis. Other LDL-lowering pharmacotherapies, such as ezetimibe, niacin, lomitapide, mipomersen, PCSK9 inhibitors, and bempedoic acid, may also be employed to achieve target LDL-C levels. If approved, evinacumab will be the first treatment that targets ANGPTL3 to lower LDL-C levels. Efficacy was similar regardless of genetic variants that result in virtually absent (null–null) or impaired (non-null) LDL-receptor activity. The initial application for approval in the US is for use in patients with HoFH and elevated LDL-C despite maximally tolerated lipid-lowering therapy. Evinacumab is also being studied in adults with persistent hypercholesterolemia and severe hypertriglyceridemia (both phase 2 trials). Subcutaneous administration of evinacumab is also being investigated. Evinacumab could compete with lomitapide in the HoFH space as it has a more desirable safety and tolerability profile compared to lomitapide, which is associated with hepatotoxicity. PSCK9 inhibitors are administered SC every 2 or 4 weeks and demonstrate higher LDL-C lowering (range, 55% to 59%) compared to evinacumab; therefore these agents could be preferred over evinacumab in the majority of HoFH patients who have residual LDL receptor activity.

FDA APPROVAL TIMELINE February 11, 2021

 Breakthrough Therapy

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$17

$44

$65

$88

$154

The forecast is a projection of total US sales per year.

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ENDOCRINE

lonapegsomatropin SC Ascendis PROPOSED INDICATIONS

Pediatric growth hormone deficiency (GHD)

CLINICAL OVERVIEW

Lonapegsomatropin is long-acting prodrug of the human growth hormone (hGH) somatropin. Interim data from two phase 3 trials demonstrated safety and efficacy of lonapegsomatropin for GHD. In the randomized, open-label, HEIGHT trial, 161 treatment-naïve children 3 to 12 years of age with GHD were randomized to receive either SC injections of once-weekly lonapegsomatropin or daily somatropin (Genotropin®, 34 μg/kg/day = 0.24 mg/kg/week) for 52 weeks. Lonapegsomatropin was superior to somatropin in the primary endpoint of annualized height velocity (AHV) at 52 weeks (11.2 versus 10.3 cm/year, respectively; treatment difference, 0.86 cm/year; 95% CI, 0.22 to 1.5; p=0.0088). Rate of poor response (AHV < 8 cm/year) was lower with lonapegsomatropin compared to somatropin (4% versus 11%, respectively). The 26-week, open-label, FLIGHT trial demonstrated safety and tolerability of lonapegsomatropin in 146 participants 6 months to 17 years of age with GHD. No serious adverse events related to the study drug were observed in either trial. The ongoing ENLIGHTEN trial is evaluating use of an auto-injector for SC administration of lonapegsomatropin. Lonapegsomatropin was studied at a dosage of 0.24 mg/kg administered SC once weekly.

PLACE IN THERAPY

GHD is a rare disorder characterized by a lack of growth hormone (GH) production in the anterior pituitary gland. Causes of childhood-onset GHD may be considered congenital, acquired (e.g., brain trauma, CNS infection, tumor), or idiopathic. GHD results in growth retardation, short stature, and delays in lengthening of the bones of the extremities. Current treatment for GHD includes daily SC injections of somatropin (various brands). Lonapegsomatropin uses the proprietary TransCon platform to delay the release and clearance of somatropin in a predictable manner. If approved, lonapegsomatropin will be the first once-weekly hGH approved for use in pediatric patients with GHD. Ascendis is also developing an auto-injector to administer lonepagsomatropin that is stable at room temperature. Pfizer and Opko Health submitted an application for the long-acting hGH somatrogon for once-weekly administration for the treatment of pediatric GHD; an FDA decision is expected in October 2021. In addition, Novo Nordisk’s hGH analog somapacitan-beco (Sogroya®), which received FDA approval as a once-weekly SC injection for adult GHD in August 2020, is currently in phase 3 trials for pediatric GHD (ages 2 to 11 years). Lonapegsomatropin is in phase 2 trials for the treatment of GHD in adults.

FDA APPROVAL TIMELINE June 25, 2021

 Orphan Drug

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$67

$197

$413

$583

$766

The forecast is a projection of total US sales per year.

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ONCOLOGY

loncastuximab tesirine IV ADC Therapeutics PROPOSED INDICATIONS

Relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL)

CLINICAL OVERVIEW

Loncastuximab tesirine is an antibody drug conjugate (ADC) that contains a humanized monoclonal antibody that targets the cluster of differentiation 19 (CD19) antigen found on B cells. Once loncastuximab tesirine enters the cell, it blocks DNA strand separation, ultimately resulting in cell death. The phase 2, single-arm LOTIS-2 trial evaluated loncastuximab tesirine in 145 adults with R/R DLBCL, including patients with poor prognosis (double/triple hit [10.3%], double/triple expressor [13.8%], transformed disease [20%]). Patients had received ≥ 2 prior therapies (range, 2 to 7); 24 patients received prior SCT, and 13 received prior CAR T therapy. Loncastuximab tesirine was administered IV over 30 minutes every 3 weeks as 150 mcg/kg during the first 2 cycles and 75 mcg/kg thereafter. After 1 year, doses were repeated every 12 weeks for up to 3 years. After a mean 4.3 treatment cycles (range, 1 to 15), loncastuximab tesirine demonstrated an ORR of 48.3% and a CR rate of 24.8%. Median DOR was 12.58 months, median PFS was 5.09 months, and overall survival was 9.53 months. TEAEs were considered manageable. The most common grade ≥ 3 TEAEs were neutropenia (26.2%), thrombocytopenia (17.9%), gamma-glutamyl transferase (GGT) increases (17.2%), and anemia (10.3%). The incidence of febrile neutropenia was low (3.4%). Following loncastuximab tesirine treatment, 15 patients in the trial received CD19-directed CAR T therapy with an investigator-assessed ORR of 46.7%, and 9 patients proceeded to SCT as consolidation after responding to loncastuximab tesirine. The phase 2, single-arm LOTIS-3 trial, combined loncastuximab tesirine with daily oral ibrutinib in adults with DLBCL who had failed or were intolerant to SOC therapy, including those with poor prognosis. Patients had received 1 to 6 prior therapies, including 4 patients who received prior SCT. During the 1-year trial, loncastuximab tesirine was administered IV as 60 mcg/kg every 3 weeks for 2 cycles during the first 14 weeks, after which the dose was 60 mcg/kg IV every 4 weeks for cycles 5, 6, 9, and 10. Oral ibritinib was given daily throughout the year. Among 37 evaluable patients in an interim analysis, ORR was 62.9% and CR rate was 31.4%. Grade ≥ 3 TEAEs included anemia (10.8%), neutropenia (10.8%), thrombocytopenia (5.4%), and fatigue (5.4%).

PLACE IN THERAPY

Over 25,000 new cases of DLBCL are diagnosed annually in the US. While some aggressive forms are often curable with intensive chemotherapy, others are less responsive. An estimated 30% to 40% of cases relapse or progress after SOC therapy (R-CHOP). Few options remain for patients who are refractory to or relapse after chemotherapy or who are ineligible for SCT. Recently approved therapies include medications that target specific biomarkers on tumor cells, as well as immunotherapies that stimulate the body’s immune response against malignancy. The CD19 protein expressed on B cells has become a very viable target for treating DLBCL. Current therapies for R/R DLBCL that are directed toward CD19 include the IV monoclonal antibody tafasitamab-cxix (Monjuvi®), used with lenalidomide in patients ineligible for autologous SCT, and single-dose CAR T therapies axicabtagene ciloleucel (Yescarta®) and tisagenlecleucel (Kymriah®). In general, ADC therapies produce deep and durable responses. Loncastuximab tesirine has exhibited a manageable safety profile, a significant ORR, and a durable response of up to 1 year or more in adults with R/R DBLCL, including patients at high risk. Moreover, loncastuximab tesirine has been effective when used after SCT or CAR T therapy and does not eliminate the possibility of subsequent SCT or CAR T; clinical data reports an ORR of 50% and CR rate of 43% in those who received CAR T therapy post loncastuximab tesirine. If approved, loncastuximab tesirine will be the second ADC available to treat R/R DBLCL. It may compete with the ADC polatuzumab vedotin-piiq (Polivy®), which targets CD79b. Although loncastuximab tesirine reported a lower CR rate than polatuzumab vedotin-piiq (used in combination with bendamustine and rituximab) in

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PLACE IN THERAPY cont.

non-comparative clinical trials (CR, 24% versus 40%, respectively), it has the advantage of being a chemotherapy-free option. Loncastuximab tesirine also has a potentially more desirable safety profile, as polatuzumab vedotin-piiq is associated with grade ≥ 3 neuropathy (incidence, 42%). Loncastuximab tesirine could also compete with tafasitamab-cxix, particularly in heavily pretreated patients with R/R DBLCL who cannot tolerate more intensive therapies. Use of loncastuximab tesirine in combination with rituximab as second-line therapy for R/R DLBCL is also being investigated in the phase 3 confirmatory LOTIS-5 trial. In addition, the product is in phase 2 evaluation for mantle cell lymphoma.

FDA APPROVAL TIMELINE May 21, 2021

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$48

$132

$210

$373

$662

The forecast is a projection of total US sales per year.

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IMMUNOLOGY

pegcetacoplan SC Apellis PROPOSED INDICATIONS

Paroxysmal nocturnal hemoglobinuria (PNH)

CLINICAL OVERVIEW

Pegcetacoplan is a complement 3 (C3) inhibitor designed to regulate hyperactivity of the complement pathway and destruction of RBCs associated with PNH. The phase 3 PEGASUS trial compared safety and efficacy of pegcetacoplan and eculizumab for the treatment of PNH. It enrolled 80 adults with PNH who were on stable eculizumab therapy and had a Hb level < 10.5 g/dL at screening. During a 4-week run-in period, all patients received pegcetacoplan in addition to their current eculizumab dose. Patients were then randomized to pegcetacoplan or their current eculizumab dose for 16 weeks, after which 77 patients entered an open-label period receiving pegcetacoplan until week 48. At week 16, pegcetacoplan resulted in a mean increase from baseline in Hb (primary endpoint) by 2.9 g/dL, which was sustained through week 48. In addition, 73% of patients in the pegcetacoplan group remained transfusion free. This was an increase from the year prior to the study, in which only 25% of the patients were transfusion free while on eculizumab. The TEAEs reported more often with pegcetacoplan than eculizumab included injection site reactions (36.6% versus 2.6%, respectively), any infection (29.3% versus 23.1%, respectively), and diarrhea (22% versus 0%, respectively). Meningitis was not reported in either group. The rate of discontinuations due to TEAEs was 7.3% with pegcetacoplan compared to 0% with eculizumab. In PEGASUS, pegcetacoplan 1,080 mg was administered SC twice weekly.

PLACE IN THERAPY

PNH is a rare blood disorder that is estimated to occur in 0.5 to 1.5 per million people worldwide. Diagnosis is typically made during the fourth decade of life. In PNH, the bone marrow produces abnormal RBCs that prematurely hemolyze, resulting in hemolytic anemia and chronic hemoglobinuria that can worsen during illness, trauma, or stress. Most patients experience fatigue and difficulty breathing. Individuals with PNH are at risk of developing potentially life-threatening blood clots. Allogeneic SCT is the only cure for PNH. Eculizumab (Soliris®) was the first drug available in the US to manage PNH. This was followed by the approval of ravulizumab-cwvz (Ultomiris®) in 2018. Both complement inhibitors target complement protein C5, while pegcetacoplan targets C3, which is upstream in the complement pathway and may improve efficacy. Pegcetacoplan and ravulizumab-cwvz have shown non-inferiority to eculizumab in treatment-experienced and treatment-naïve patients, respectively, based on the proportion of patients who remained transfusionfree in clinical trials. Pegcetacoplan also showed improvement in Hb over eculizumab. Eculizumab and ravulizumab-cwvz carry boxed warnings for reports of serious meningococcal infections. To date, the PEGASUS trial has not reported meningitis with pegcetacoplan. If approved, pegcetacoplan will provide an administration method that does not require an HCP visit. While eculizumab is dosed IV once every 2 weeks and ravulizumab-cwvz is dosed IV once every 8 weeks, pegcetacoplan can be self-administered SC twice weekly. Pegcetacoplan will compete with eculizumab and ravulizumab-cwvz, both of which also hold indications for atypical hemolytic uremic syndrome (aHUS).

FDA APPROVAL TIMELINE May 14, 2021

 Fast Track

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$19

$76

$151

$206

$245

The forecast is a projection of total US sales per year. 12 | MAGELLANRX.COM


INFECTIOUS DISEASE

taurolidine/heparin/citrate IV Cormedix PROPOSED INDICATIONS

Prevention of catheter-related blood stream infections (CRBSI) in hemodialysis (HD) patients

CLINICAL OVERVIEW

Cormedix’s proprietary catheter lock solution contains taurolidine 1.35%, citrate 3.5%, and heparin 1,000 units/mL. Taurolidine is an amino acid derivative that denatures surface proteins and chemically alters membrane lipids. It has shown in vitro activity against gram-positive and gram-negative bacteria, including antibiotic resistant strains, and activity against mycobacteria and clinically relevant fungi, including Aspergillus. Heparin provides anticoagulation activity and citrate is a pH buffer. In the double-blind, phase 3 LOCK-IT-100 trial, patients with ESRD receiving HD ≥ 2 times per week were randomized 1:1 to taurolidine/heparin/citrate or the SOC catheter lock heparin 1,000 units/mL. Based on an interim analysis that included 653 patients, the study revealed a 72% reduction in CRBSIs (primary endpoint) with taurolidine/heparin/citrate compared to heparin alone (incidence, 6 versus 22 events per 1,000 catheter days, respectively; p=0.0034). Among the secondary endpoints, the incidence of catheter removal due to CRBSI was lower with taurolidine/heparin/citrate versus heparin (2% versus 7.3%, respectively). Loss of catheter patency was reported in 16% of patients in the taurolidine/heparin/citrate group compared to 12% in the heparin group, but the difference was not significant (p=0.12). Adverse events that were reported more often with taurolidine/heparin/citrate than with heparin included cardiac failure (3% versus 1.8%, respectively), fluid overload (3.5% versus 3%, respectively), and hyperkalemia (2.5% versus 2%, respectively). Taurolidine/heparin/citrate is instilled in the arterial and venous lumens of the HD catheter after each HD session. The catheter lock solution is not intended for systemic administration. Prior to the next use of the lumen, the catheter lock solution is aspirated from the lumen.

PLACE IN THERAPY

It is reported that over 450,000 individuals in the US are HD dependent. Based on 2008 data, an estimated 37,000 CRBSIs occurred among those who received outpatient HD. Tunneled dialysis catheters provide immediate vascular access and are often used for months or years. Proper catheter management to maintain patency and prevent infection is imperative. Currently, no pharmacological agent is FDA-approved for the prevention of CRBSIs. Heparin is the SOC to maintain catheter patency. In addition, the CDC recommends applying topical povidone iodine ointment or bacitracin/gramicidin/polymyxin B ointment at the catheter exit site after each HD session, if compatible with the catheter material. In general, risk factors for developing CRBSI include duration of catheterization, catheter material, insertion conditions and skill of inserter, catheter site care, increased manipulation of catheter, and catheter thrombosis. Indwelling catheters in particular, including those used for HD, are at risk for CRBSI. In clinical studies, taurolidine/heparin/citrate resulted in significantly fewer cases of CRBSI and displayed a similar safety profile when compared to heparin alone in HD patients. If approved, it will be the first antibacterial and antifungal catheter lock solution in the US to prevent catheter-related infections in this patient population. Minocycline/edetate/ethyl alcohol catheter lock solution, by Citius Pharmaceuticals, is also in phase 3 investigation for the treatment of catheter-related or central line associated bloodstream infection (CRBSI/CLABSI), including in hemodialysis patients.

FDA APPROVAL TIMELINE February 28, 2021

 Fast Track

 QIDP

 Priority Review

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$18

$50

$95

$140

$185

The forecast is a projection of total US sales per year. 13 | MAGELLANRX.COM


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. Many controversies surround 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 FDA is still considering how to implement the nomenclature for previously approved biosimilar products. 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 will focus on providing flexibility for 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. Several states had already enacted biosimilar substitution legislation. Biosimilars are expected to receive full extrapolation for the eligible indications of the reference products without requiring additional trials. Nevertheless, as each biosimilar comes to market, it will likely need to be considered individually. Insulins were historically regulated by the FDA 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 biologics 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) improving the biosimilar development and approval process; (2) maximizing scientific and regulatory clarity for sponsors; (3) providing effective communications for patients, clinicians, and payers; and (4) reducing unfair tactics that may delay market approval and entry. The BAP strives to promote access to biosimilar products and reduce healthcare costs.

14 | MAGELLANRX.COM


To date, a total of 29 biosimilars have received FDA approval. Of these, only 19 have entered the market. APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Zarxio® (filgrastim-sndz)

Novartis/Sandoz

March 2015

Inflectra® (infliximab-dyyb)

Pfizer/Celltrion

April 2016

Erelzi™ (etanercept-szzs)

Novartis/Sandoz

August 2016

Amjevita™ (adalimumab-atto)

Amgen

September 2016

Renflexis® (infliximab-abda)

Samsung Bioepis/ Merck

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

December 2017

Retacrit (epoetin alfa-epbx)

Pfizer/Hospira

May 2018

Fulphila® (pegfilgrastim-jmdb)

Mylan

June 2018

Nivestym® (filgrastim-aafi)

Pfizer

July 2018

Hyrimoz™ (adalimumab-adaz)

Novartis/Sandoz

October 2018

Udenyca® (pegfilgrastim-cbqv)

Coherus

November 2018

Truxima® (rituximab-abbs)

Celltrion/Teva

November 2018

Herzuma® (trastuzumab-pkrb)

Celltrion/Teva

December 2018

Ontruzant® (trastuzumab-dttb)

Samsung Bioepis/ Merck

January 2019

Trazimera™ (trastuzumab-qyyp)

Pfizer

March 2019

Eticovo™ (etanercept-ykro)

Samsung Bioepis/ Merck

April 2019

Kanjinti™ (trastuzumab-anns)

Amgen

June 2019

Zirabev™ (bevacizumab-bvzr)

Pfizer

June 2019

Hadlima™ (adalimumab-bwwd)

Samsung Bioepis/ Merck

July 2019

Ruxience™ (rituximab-pvvr)

Pfizer

July 2019

Abrilada™ (adalimumab-afzb)

Pfizer

November 2019

Ziextenzo® (pegfilgrastim-bmez)

Novartis/Sandoz

November 2019

®

15 | MAGELLANRX.COM

Commercially Available

  -

 -

 -

    -

     -

  -

 -

Originator Product (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) Humira (Abbvie) Neulasta (Amgen)


APPROVED BIOSIMILARS continued APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Avsola™ (infliximab-axxq)

Amgen

December 2019

Nyvepria™ (pegfiltrastim-apgf)

Pfizer

June 2020

Hulio® (adalimumab-fkjp)

Mylan

July 2020

Riabni™ (rituximab-arrx)

Amgen

December 2020

Commercially Available

 -

Originator Product (Manufacturer) Remicade (Janssen) Neulasta (Amgen) Humira (Abbvie) Rituxan (Genentech)

* 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 biosimilar agent to Sanofi’s Lantus®, and Sanofi’s Admelog® insulin lispro, approved as a biosimilar product to Eli Lilly’s Humalog®. In June 2020, the FDA approved insulin glargine (Semglee™) by Mylan/Biocon under an abbreviated 505(b)(2) New Drug Application (NDA) pathway; the reference product was Lantus. Semglee is considered a biologic under section 351(a) rather than a biosimilar. A host of factors will contribute to market acceptability and the potential success of biosimilars. Payers, pharmacies, prescribers, and patients each play a role in market adoption of biosimilars. While < 2% of Americans use biologics, they account for almost 40% of all prescription drug spending. Moreover, they comprised 70% of growth in drug spending from 2010 to 2015. Not surprisingly, there is a growing body of evidence on predicted biologic spend and potential biosimilar savings. A 2020 report by IQVIA forecasts that biosimilars are on track to reduce overall US drug spend by $100 billion over the next 5 years. In fact, the next 5 years are expected to have an estimated 5-fold increase in savings relative to the past 5 years as more biosimilars launch and existing biosimilars see more utilization and reductions in price. Three biosimilar launches in 2019 saw substantial uptake within the first year of commercialization, these are: bevacizumab (42%), trastuzumab (38%), and rituximab (20%). In the US, it is estimated that biosimilars will cost 15% to 35% less than the originator product, although price dynamics vary. The potential cost savings, however, can vary based on the market segment where brand contracts can play a role. A 2017 report by the RAND Corporation estimates a $54 billion cost savings from biosimilars between 2017 and 2026. In July 2018, an FDA analysis reported that if Americans had access to FDA-approved biosimilars in 2017, it would have resulted in a $4.5 billion savings. A 2017 analysis by the Moran Company projects biosimilars could save the government an estimated $11.4 billion by 2027, but it would require the Centers for Medicare and Medicaid Services (CMS) to revise its reimbursement policy for biosimilars. Subsequently, in November 2017, the CMS revised its reimbursement policy. The CMS now issues a unique Healthcare Common Procedure Coding System (HCPCS) code to each individual biosimilar. Under this rule, Medicare Part B separately codes and pays for biosimilars and no longer groups them into a common payment code with originator agents. A June 2018 infliximab case study by the Pacific Research Institute forecasts annual savings of up to $465 million from increased use of biosimilars to replace a single biologic for commercial payers and Medicare. Biosimilars are paving the way for increased access to important biologic therapies that may increase survival and/ or QOL for many patients with difficult-to-treat diseases while also reducing costs.

16 | MAGELLANRX.COM


BIOSIMILAR OVERVIEW continued

IMMUNOLOGY

adalimumab SC AVT-02 and CHS-1420 are biosimilars to Abbvie’s Humira, a tumor necrosis factor alpha (TNFα) 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.

FDA APPROVAL TIMELINE Alvotech (AVT-02) September 2021

Coherus (CHS-1420) September 2021

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$16,872

$17,387

$12,802

$9,143

$7,085

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

ONCOLOGY

bevacizumab IV Aybintio, Bmab-100, BAT1706, and FKB238 are investigational biosimilars to Genentech’s Avastin, a vascular endothelial growth factor (VEGF)-specific angiogenesis inhibitor indicated for the treatment of metastatic colorectal cancer, non-squamous non-small cell lung cancer (nsNSCLC), glioblastoma, metastatic renal cell carcinoma (RCC), and recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer.

FDA APPROVAL TIMELINE

Bio-Thera Solutions (BAT1706) September 2021 Centus/AstraZeneca (FKB238) Pending Merck/Samsung Bioepis (Aybintio) Pending Mylan/Biocon (Bmab-100) Pending

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$1,369

$1,079

$892

$787

$705

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

17 | MAGELLANRX.COM


BIOSIMILAR OVERVIEW continued

BLOOD MODIFIER

filgrastim IV, SC Apotex and Kashiv are seeking biosimilars 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 in patients who are acutely exposed to myelosuppressive doses of radiation (hematopoietic syndrome of acute radiation syndrome [HSARS]).

FDA APPROVAL TIMELINE Apotex (Grastofil) Pending Kashiv/Amneal Pending

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$110

$95

$85

$78

$72

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

ENDOCRINE

insulin aspart SC Mylan/Biocon Mylan/Biocon is seeking biosimilar approval to Novo Nordisk’s Novolog®, a rapid-acting insulin to improve glycemic control in patients with T1DM or T2DM.

FDA APPROVAL TIMELINE April to June 2021

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$750

$640

$561

$517

$485

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

18 | MAGELLANRX.COM


BIOSIMILAR OVERVIEW continued

BLOOD MODIFIER

pegfilgrastim SC Lapelga, MSB-11455, and TPI-120 are investigational biosimilars to Amgen’s Neulasta, a leukocyte growth factor indicated for use in patients with nonmyeloid malignancies who are receiving myelosuppressive anti-cancer drugs and in patients acutely exposed to myelosuppressive doses of radiation (HSARS).

FDA APPROVAL TIMELINE Apotex (Lapelga) Pending

Kashiv/Amneal (TPI-120) January 2022 Merck/Fresenius (MSB-11455) March 2021

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$1,609

$1,333

$1,132

$985

$876

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

OPHTHALMOLOGY

ranibizumab intravitreal Biogen/Samsung Bioepis Biogen/Samsung Bioepis are seeking biosimilar approval 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 September to October 2021

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$1,727

$1,653

$1,488

$1,278

$1,104

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

19 | 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 2025, are displayed. The financials are projected total annual US sales, reported in millions.

tirzepatide Diabetes

trilaciclib

abrocitinib

$593

$570

Oncology

Dermatology

adagrasib Oncology

$1,404

$1,035

somatrogon

aducanumab

$74

$2,501

Neurology

Endocrine

bardoxolone methyl

NVX-CoV2373 vaccine

Renal

COVID-19

$1,134

$1,637 mirikizumab

betibeglogene autotemcel (Zynteglo)

$324

$582

Hematology/Gene therapy

Immunology

mavacamten

deucravacitinib

$538

$1,305

Immunology

Cardiovascular

efgartigimod

lenadogene nolparvovec (GS010)

Immunology

$1,098

Ophthalmology/Gene therapy

$53

elivaldogene tavalentivec (Lenti-D)

ipatasertib

Neurology/Gene therapy

Oncology

$382

idecabtagene vicleucel

ďŹ rmacute eubacterial spores

$771

$598

Oncology

$65

Gastrointestinal

pecialty drug names appear in ď‚Ť S magenta throughout the publication.


Pipeline DRUG LIST The pipeline drug list is an aerial outline of drugs with anticipated FDA approval through 2022. 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) and those that have been withdrawn from development are also noted. APPLICATION SUBMITTED APPLICATION TO THE FDA SUBMITTED

IN PHASE 3 PHASE 3 TRIALS TRIALS

61%

63%

39%

37%

34%

35%

34%

Specialty

23%

14%

10%

9%

Traditional

Orphan Drug

Priority Review

Breakthrough Therapy

Biosimilar

pecialty drug names appear in ï‚« S magenta throughout the publication.


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

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

Submitted (New Drugs) lamotrigine

Eton

Partial seizures

Oral

Submitted – 505(b)(2) NDA

2021

ansofaxine

Luye

MDD

Oral

Submitted – NDA

January 2021

eflapegrastim

Spectrum

Neutropenia/leukopenia

SC

Submitted – BLA

Jan–Feb 2021

tepotinib

Merck

NSCLC (mesenchymalepithelial transition exon 14 [METex14] skipping)

Oral

Submitted – NDA; Breakthrough Therapy; Priority Review; RTOR

Jan–Mar 2021

inolimomab

Elsalys

GVHD (acute, steroidrefractory)

IM

Submitted – BLA; Orphan Drug; RTOR

Jan–Jul 2021

dostarlimab

GlaxoSmithKline

Endometrial cancer (dMMR/MSI-H, recurrent, 2nd-line)

IV

Submitted – BLA

01/14/2021

evinacumab

Regeneron

HoFH

IV

Submitted – BLA; Breakthrough Therapy; Orphan Drug; Priority Review

02/11/2021

trilaciclib

G1 Therapeutics/ Boehringer Ingelheim

SCLC

IV

Submitted – NDA; Breakthrough Therapy; Priority Review

02/15/2021

umbralisib

TG

Marginal zone lymphoma (≥ 1 prior anti-CD20 based regimen)

Oral

Submitted – NDA; seeking Accelerated Approval; Breakthrough Therapy; Orphan Drug; Priority Review

02/15/2021

casimersen

Sarepta

DMD (amenable to exon 45 skipping)

IV

Submitted – NDA; seeking Accelerated Approval; Orphan Drug; Priority Review

02/25/2021

melflufen

Oncopeptides

Multiple myeloma (triplerefractory)

IV

Submitted – NDA; Orphan Drug; Priority Review

02/26/2021

udenafil

Allergan

Single ventricle heart disease (ages ≥ 12 years)

Oral

Submitted – NDA; Orphan Drug

02/26/2021

paclitaxel/encequidar

Athenex

Breast cancer

Oral

Submitted – NDA; Priority Review

02/28/2021

taurolidine/heparin/citrate

Cormedix

Prevention of catheterrelated blood stream infections (hemodialysis patients)

IV

Submitted – NDA; Fast Track; QIDP; Priority Review

02/28/2021

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Merck/Fresenius

Neutropenia/leukopenia

SC

Submitted – BLA

March 2021

ropeginterferon alfa-2b

Pharmaessentia

Polycythemia vera (in absence of symptomatic splenomegaly)

SC

Submitted – BLA; Orphan Drug

Mar–Apr 2021

budesonide oral suspension

Takeda

Eosinophilic esophagitis

Oral

Submitted – 505(b)(2) NDA; Breakthrough Therapy; Orphan Drug; Priority Review

Mar–Jun 2021

22 | MAGELLANRX.COM


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

d-methylphenidate

Corium

ADHD

Oral

Submitted – 505(b)(2) NDA

aducanumab

Biogen/Eisai

Alzheimer’s disease

IV

Submitted – BLA; Fast 03/05/2021 Track; Priority Review

ponesimod

Janssen

MS (relapsing)

Oral

Submitted – NDA

03/18/2021

roxadustat

AstraZeneca

Anemia due to CKD (dialysis-dependent, dialysis-independent)

Oral

Submitted – NDA

03/20/2021

idecabtagene vicleucel

Bristol-Myers Squibb/ Bluebird Bio

Multiple myeloma (≥ 3 prior therapies)

IV

Submitted – BLA; Breakthrough Therapy; Orphan Drug; Priority Review

03/26/2021

dasiglucagon

Zealand

Hypoglycemia (diabetesrelated)

SC

Submitted – NDA; Orphan Drug

03/27/2021

tivozanib hydrochloride monohydrate

Aveo

RCC (relapsed/refractory)

Oral

Submitted – NDA

03/31/2021

eflornithine/sulindac

Mallinckrodt

Familial adenomatous polyposis

Oral

Submitted – 505(b)(2) NDA; seeking Accelerated Approval; Fast Track; Orphan Drug

Apr–Jun 2021

insulin aspart (biosimilar to Novo Nordisk’s Novolog)

Mylan/Biocon

T1DM; T2DM

SC

Submitted – BLA

Apr–Jun 2021

tanezumab

Pfizer

Osteoarthritis pain

IV

Submitted – BLA; Fast Apr–Jun 2021 Track

tralokinumab

AstraZeneca

Atopic dermatitis

SC

Submitted – BLA

Apr–Jun 2021

brincidofovir

Chimerix

Smallpox

Oral

Submitted – NDA; Fast Track; Orphan Drug; Priority Review

04/07/2021

fosdenopterin

Bridgebio

Molybdenum cofactor deficiency

IV

Submitted – NDA; 04/09/2021 Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatric Disease

estetrol/drospirenone

Mayne

Contraception

Oral

Submitted – NDA

04/16/2021

benzoyl peroxide

Sol-Gel

Rosacea

Transdermal

Submitted – 505(b)(2) NDA

04/26/2021

pegunigalsidase alfa

Chiesi

Fabry’s disease

IV

Submitted – BLA; seeking Accelerated Approval; Fast Track; Priority Review

04/27/2021

abrocitinib

Pfizer

Atopic dermatitis

Oral

Submitted – NDA; Breakthrough Therapy; Priority Review

04/30/2021

bupivacaine/meloxicam

Heron

Postsurgical pain

Instillation

Submitted – NDA; Breakthrough Therapy; Fast Track

05/13/2021

pegcetacoplan

Apellis

Paroxysmal nocturnal hemoglobinuria

SC

Submitted – NDA; Fast Track; Orphan Drug; Priority Review

05/14/2021

23 | MAGELLANRX.COM

03/02/2021


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

avalglucosidase alfa

Sanofi

Pompe disease

IV

Submitted – BLA; Breakthrough Therapy; Fast Track; Priority Review

05/18/2021

loncastuximab tesirine

ADC Therapeutics

DLBCL (relapsed/ refractory)

IV

Submitted – BLA; Orphan Drug; Priority Review

05/21/2021

dehydrated alcohol

Eton

Methanol poisoning

SC

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

05/27/2021

leuprolide mesylate readyto-use, 6-month depot

Foresee

Prostate cancer

SC

Submitted – 505(b)(2) NDA

05/27/2021

zonisamide oral suspension

Eton

Partial seizures

Oral

Submitted – 505(b)(2) NDA

05/29/2021

belumosudil

Kadmon

GVHD (chronic)

Oral

Submitted – NDA; 05/30/2021 Breakthrough Therapy; Orphan Drug; Priority Review; Project Orbis; RTOR

20-valent pneumococcal conjugate vaccine

Pfizer

Pneumococcal pneumonia prevention

IM

Submitted – BLA; Breakthrough Therapy; Fast Track; Priority Review

June 2021

cantharidin

Verrica

Molluscum contagiosum

Topical

Submitted – NDA

June 2021

infigratinib

Bridgebio

Biliary tract cancer

Oral

Submitted – NDA; June 2021 Fast Track; Orphan Drug; Priority Review; RTOR

relugolix/estradiol/ norethindrone

Myovant

Uterine fibroid-related Oral heavy menstrual bleeding

Submitted – NDA

06/01/2021

samidorphan/olanzapine

Alkermes

Bipolar disorder; Schizophrenia

Oral

Submitted – NDA

06/01/2021

plasminogen (human)

Liminal

Congenital plasminogen deficiency

IV

Submitted – BLA; Fast 06/05/2021 Track; Orphan Drug; Rare Pediatric Disease

ibrexafungerp

Scynexis

Vulvovaginal candidiasis

Oral

Submitted – NDA; Fast Track; Orphan Drug; QIDP; Priority Review

06/14/2021

umbralisib

TG

Follicular lymphoma (≥ 2 prior systemic therapies)

Oral

Submitted – NDA; seeking Accelerated Approval; Orphan Drug

06/15/2021

arimoclomol

Orphazyme

Niemann-Pick disease

Oral

Submitted – NDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review; Rare Pediatric Disease

06/17/2021

cyclosporine

Santen

Dry eye syndrome

Ophthalmic

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

06/24/2021

lonapegsomatropin

Ascendis

Growth hormone deficiency (pediatrics)

SC

Submitted – BLA; Orphan Drug

06/25/2021

24 | MAGELLANRX.COM


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

bimekizumab

UCB

PSO

SC

Submitted – BLA

anifrolumab

AstraZeneca

SLE

IV

Submitted – BLA; Fast Jul–Dec 2021 Track

sotorasib

Amgen

NSCLC (KRAS G12Cmutation, locally advanced/metastatic, ≥ 1 prior systemic therapy)

Oral

Submitted – NDA; Breakthrough Therapy; Fast Track; Orphan Drug; RTOR

Jul–Dec 2021

teplizumab

Provention

T1DM (delay/prevention)

IV

Submitted – BLA; Breakthrough Therapy; Orphan Drug; Priority Review

07/02/2021

avacopan

Chemocentryx

ANCA-associated vasculitis

Oral

Submitted – NDA; Orphan Drug

07/07/2021

finerenone

Bayer

Diabetic nephropathy

Oral

Submitted – NDA; Priority Review

07/09/2021

narsoplimab

Omeros

HSCT-associated thrombotic microangiopathy

IV, SC

Submitted – BLA; Breakthrough Therapy; Orphan Drug; Priority Review

07/16/2021

15-valent pneumococcal conjugate vaccine

Merck

Invasive pneumococcal disease prevention

IM

Submitted – BLA; Breakthrough Therapy; Priority Review

07/18/2021

retifanlimab

Incyte

Anal cancer (squamous cell, locally advanced/ metastatic, failed on/ intolerant to platinumbased chemotherapy)

IV

Submitted – BLA; Orphan Drug; Priority Review

07/25/2021

tretinoin/benzoyl peroxide

Sol-Gel

Acne vulgaris

Topical

Submitted – 505(b)(2) NDA

08/01/2021

topiramate oral solution

Eton

Partial seizures

Oral

Submitted – 505(b)(2) NDA

08/06/2021

vosoritide

Biomarin

Achondroplasia

SC

Submitted – NDA; Orphan Drug

08/20/2021

adalimumab (biosimilar to Abbvie’s Humira)

Alvotech

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

SC

Submitted – BLA

September 2021

adalimumab (biosimilar to Abbvie’s Humira)

Coherus

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

SC

Submitted – BLA

September 2021

bevacizumab (biosimilar to Genentech’s Avastin)

Bio-Thera Solutions

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

IV

Submitted – BLA

September 2021

ranibizumab (biosimilar to Genentech’s Lucentis)

Biogen/Samsung Bioepis

Wet AMD

Intravitreal

Submitted – BLA

Sep–Oct 2021

paliperidone palmitate 6-month injectable

Janssen

Schizophrenia

IM

Submitted – NDA

09/02/2021

dihydroergotamine mesylate

Impel Neuropharma

Migraine treatment

Intranasal

Submitted – 505(b)(2) NDA

09/06/2021

risperidone long-acting in situ microparticle

Rovi

Schizophrenia

IM

Submitted – 505(b)(2) NDA

09/24/2021

reltecimod

Atox

Necrotizing soft tissue infection (NSTI)-related organ dysfunction/failure (ages ≥ 12 years)

IV

Submitted – NDA; seeking Accelerated Approval; Fast Track; Orphan Drug

09/30/2021

25 | MAGELLANRX.COM

July 2021


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

cabotegravir long-acting

Viiv

HIV-1 infection preexposure prevention (PrEP)

IM

Submitted – NDA; Breakthrough Therapy

Late 2021– Early 2022

dexamethasone insert

Ocular Therapeutix

Allergic conjunctivitis

Intraocular

Submitted – NDA

October 2021

somatrogon

Pfizer/Opko

Growth hormone deficiency (pediatrics)

SC

Submitted – BLA; Orphan Drug

October 2021

amivantamab

Janssen

NSCLC (EGFR exon 20 insertion mutations, progressed on or after platinum-based chemotherapy)

IV

Submitted – BLA; Breakthrough Therapy

Oct–Dec 2021

efgartigimod

Argenx

Myasthenia gravis

IV

Submitted – BLA; Fast Oct–Dec 2021 Track; Orphan Drug

avapritinib

Blueprint Medicines

Mastocytosis (advanced, systemic)

Oral

Submitted – NDA; Breakthrough Therapy; Orphan Drug

10/15/2021

sodium oxybate (oncenightly)

Avadel

Narcolepsy-related excessive daytime sleepiness and cataplexy

Oral

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

10/15/2021

varenicline

Oyster Point

Dry eye syndrome

Intranasal

Submitted – 505(b)(2) NDA

10/18/2021

phenylephrine 2.5%/ tropicamide 1%

Eyenovia

Pharmacologic mydriasis

Ophthalmic

Submitted – 505(b)(2) NDA

10/29/2021

sulopenem

Iterum

Uncomplicated UTI (quinolone-resistant)

IV, Oral

Submitted – NDA; Fast Track; QIDP

November 2021

difelikefalin

Cara

Pruritus (hemodialysisrelated)

IV

Submitted – NDA; Breakthrough Therapy

December 2021

odevixibat

Albireo

Progressive familial intrahepatic cholestasis

Oral

Submitted – NDA; Fast Track; Orphan Drug; Rare Pediatric Disease

December 2021

oportuzumab monatox

Sesen

Bladder cancer (BCGunresponsive, nonmuscle invasive)

Intravesical

Submitted – BLA; Fast December Track 2021

trivalent hepatitis B vaccine

VBI Vaccines

Hepatitis B infection (prevention)

IM

Submitted – BLA

12/01/2021

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Kashiv/Amneal

Neutropenia/leukopenia

SC

Submitted – BLA

January 2022

testosterone undecanoate

Marius

Hypogonadism

Oral

Submitted – 505(b)(2) NDA

January 2022

daridorexant

Idorsia

Insomnia

Oral

Submitted – NDA

01/08/2022

bevacizumab (biosimilar to Genentech’s Avastin)

Centus/AstraZeneca

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

IV

Submitted – BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Merck/Samsung Bioepis

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

IV

Submitted – BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Mylan/Biocon

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

IV

Submitted – BLA

Pending

26 | MAGELLANRX.COM


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

filgrastim (biosimilar to Amgen’s Neupogen)

Apotex

Neutropenia/leukopenia

SC

Submitted – BLA

Pending

filgrastim (biosimilar to Amgen’s Neupogen)

Kashiv/Amneal

Neutropenia/leukopenia

IV, SC

Submitted – BLA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Apotex

Neutropenia/leukopenia

SC

Submitted – BLA

Pending

romiplostim (Nplate®)

Amgen

Acute radiation syndrome

SC

Submitted – sBLA; Orphan Drug; Priority Review

01/28/2021

Eli Lilly

Atopic dermatitis

Oral

Submitted – sNDA

Feb-Apr 2021

cabozantinib (Cabometyx )

Exelixis

RCC (advanced, in combination with nivolumab)

Oral

Submitted – sNDA; Priority Review

02/20/2021

nivolumab (Opdivo®)

Bristol-Myers Squibb

RCC (advanced, in combination with cabozantinib)

IV

Submitted – sBLA; Breakthrough Therapy; Fast Track; Priority Review

02/20/2021

cemiplimab-rwlc (Libtayo®)

Regeneron

NSCLC (1st-line, locally advanced or metastatic, ≥ 50% PD-L1 expression)

IV

Submitted – sBLA; Priority Review

02/26/2021

valsartan/sacubitril (Entresto®)

Novartis

Heart failure with preserved ejection fraction (HFpEF)

Oral

Submitted – sNDA; Fast Track

02/26/2021

cemiplimab-rwlc (Libtayo)

Regeneron

Basal cell carcinoma

IV

Submitted – sBLA; Priority Review

03/03/2021

axicabtagene ciloleucel (Yescarta)

Gilead

Follicular lymphoma, Marginal zone lymphoma (relapsed/refractory after ≥ 2 lines of therapy for both)

IV

Submitted – sBLA; Breakthrough Therapy; Orphan Drug; Priority Review

03/05/2021

rilonacept (Arcalyst®)

Regeneron

Recurrent pericarditis

SC

Submitted – sBLA; Breakthrough Therapy; Orphan Drug; Priority Review

03/19/2021

mirabegron (Myrbetriq®) tablet, oral suspension

Astellas

Neurogenic detrusor overactivity (ages ≥ 3 years)

Oral

Submitted – sNDA; Priority Review

03/28/2021

pembrolizumab (Keytruda®)

Merck

Breast cancer (highrisk early-stage TNBC, in combination with chemotherapy as neoadjuvant treatment); Breast cancer (adjuvant monotherapy after surgery)

IV

Submitted – sBLA; seeking Accelerated Approval; Breakthrough Therapy; Priority Review

03/29/2021

treprostinil (Tyvaso®) solution

United Therapeutics

Interstitial lung diseaseassociated pulmonary hypertension

Inhaled

Submitted – sNDA

April 2021

dapagliflozin (Farxiga®)

AstraZeneca

CKD (with or without T2DM)

Oral

Submitted – sNDA; Breakthrough Therapy; Fast Track; Priority Review

Apr-Jun 2021

tofacitinib (Xeljanz®/ Xeljanz XR®)

Pfizer

Axial spondyloarthritis

Oral

Submitted – sNDA

Apr-Jun 2021

Submitted (Supplementals)

baricitinib (Olumiant®) ®

27 | MAGELLANRX.COM


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

upadacitinib (Rinvoq™)

Abbvie

PsA

Oral

Submitted – sNDA

04/01/2021

pimavanserin (Nuplazid®)

Acadua

Dementia-related hallucinations and delusions

Oral

Submitted – sNDA; Breakthrough Therapy

04/02/2021

pembrolizumab (Keytruda)

Merck

Esophageal cancer (locally advanced/ metastatic, 1stline, in combination with platinum- & fluoropyrimidine-based chemotherapy)

IV

Submitted – sBLA; Priority Review

04/13/2021

lorlatinib (Lorbrena®)

Pfizer

NSCLC (1st-line, ALK+)

Oral

Submitted – sNDA; Orphan Drug; Priority Review; RTOR

04/27/2021

tenapanor (Ibsrela®)

Ardelyx

Hyperphosphatemia (CKD dialysis-dependent patients)

Oral

Submitted – sNDA

04/29/2021

pirfenidone (Esbriet®)

Genentech

Interstitial lung disease (unclassified)

Oral

Submitted – sNDA; Breakthrough Therapy; Orphan Drug; Priority Review

May 2021

teriflunomide (Aubagio®)

Sanofi

MS (pediatric)

Oral

Submitted – sNDA

May 2021

nivolumab (Opdivo)

Bristol-Myers Squibb

Esophageal/ gastroesophageal junction cancer (resected, adjuvant setting, after neoadjuvant chemoradiation therapy)

IV

Submitted – sBLA; Orphan Drug; Priority Review

05/20/2021

nivolumab (Opdivo)

Bristol-Myers Squibb

Gastric/gastroesophageal junction cancer/ esophageal adenocarcinoma (advanced/metastatic, in combination with fluoropyrimidine- & platinum-based chemotherapy)

IV

Submitted – sBLA; Orphan Drug; Priority Review

05/25/2021

semaglutide (Ozempic®)

Novo Nordisk

Obesity/overweight (≥ 1 weight-related comorbidity)

SC

Submitted – sNDA; Priority Review

06/04/2021

upadacitinib (Rinvoq)

Abbvie

Axial spondyloarthritis

Oral

Submitted – sNDA

06/25/2021

secnidazole (Solosec )

Lupin

Trichomoniasis (adults and adolescents)

Oral

Submitted – sNDA

06/30/2021

mepolizumab (Nucala)

GlaxoSmithKline

Nasal polyposis

SC

Submitted – sNDA

Jul-Aug 2021

ruxolitinib (Jakafi) tablet

Incyte

GVHD (chronic)

Oral

Submitted – sNDA; Orphan Drug

Jul-Dec 2021

upadacitinib (Rinvoq)

Abbvie

Atopic dermatitis (adults & adolescents)

Oral

Submitted – sNDA; Breakthrough Therapy

08/19/2021

empagliflozin (Jardiance®)

Boehringer Ingelheim

Chronic heart failure (reduced ejection fraction)

Oral

Submitted – sNDA; Fast Track

September 2021

®

28 | MAGELLANRX.COM


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

daratumumab/ hyaluronidase-fihj (Darzalex Faspro™)

Janssen

Multiple myeloma (relapsed/refractory, in combination with pomalidomide and dexamethason)

SC

Submitted – sBLA

09/21/2021

sacituzumab govitecanhziy (Trodelvy®)

Immunomedics

Breast cancer (metastatic, TNBC, ≥ 2 prior therapies)

IV

Submitted – sBLA; Breakthrough Therapy; Fast Track

10/08/2021

Phase 3 (New Drugs) abaloparatide-TD

Radius Health

Osteoporosis/osteopenia

Transdermal

Phase 3 – NDA

TBD

abicipar pegol

Allergan

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

acoramidis

Eidos

Transthyretin amyloid cardiomyopathy (ATTRCM)

Oral

Phase 3 – NDA

TBD

Ad26.COV2-S vaccine

Janssen

COVID-19

IM

Phase 3 – BLA

TBD

adagrasib

Mirati

NSCLC

Oral

Phase 3 – NDA

TBD

adalimumab (biosimilar to Abbvie’s Humira)

Coherus

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

SC

Phase 3 – BLA

TBD

adalimumab (biosimilar to Abbvie’s Humira)

Fresenius

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

SC

Phase 3 – BLA

TBD

adalimumab (biosimilar to Abbvie’s Humira)

Mylan/Biocon

Hidradenitis suppurativa; Uveitis

SC

Phase 3 – BLA

TBD

adrabetadex

Mallinckrodt

Niemann-Pick disease

Intrathecal

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug; Rare Pediatric Disease

TBD

aflibercept (biosimilar to Regeneron’s Eylea®)

Mylan/Momenta

Diabetic macular edema

Intravitreal

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Samsung Bioepis/Biogen

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Santo/Formycon

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

AKCEA-TTR-LRx

Akcea

Transthyretin amyloid cardiomyopathy (ATTR-CM, wild-type or hereditary)

N/A

Phase 3 – NDA

TBD

alpha 1 proteinase inhibitor

Kamada

Alpha-1 antitrypsin deficiency-related lung disease

Inhaled

Phase 3 – BLA; Orphan Drug

TBD

antolimab

Allakos

Gastroenteritis (eosinophilic)

IV

Phase 3 – BLA; Orphan Drug

TBD

apolipoprotein A-I (human)

CSL

Atherosclerosis

IV

Phase 3 – BLA

TBD

apomorphine continuous infusion pump

Supernus

Parkinson’s disease

SC

Phase 3 – NDA

TBD

ARO-AAT

Arrowhead

Alpha-1 antitrypsin deficiency-related liver disease

SC

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

asciminib

Novartis

Chronic myelogenous leukemia

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

ataluren

PTC

DMD

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

atogepant

Allergan

Migraine prevention

Oral

Phase 3 – NDA

TBD

29 | MAGELLANRX.COM


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

autologous genetically modified human dermal fibroblasts

Castle Creek

Epidermolysis bullosa

Intradermal

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

TBD

AZD1222 vaccine

AstraZeneca

COVID-19

IM

Phase 3 – BLA

TBD

baclofen/naltrexone/ sorbitol

Pharnext

Charcot-Marie-Tooth disease

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

balixafortide

Polyphor

Breast cancer

IV

Phase 3 – NDA; Fast Track

TBD

bamlanivimab

Eli Lilly

COVID-19

IV

Phase 3 – BLA

TBD

bardoxolone methyl

Reata

Alport syndrome; Polycystic kidney disease

Oral

Phase 3 – NDA; Orphan Drug

TBD

beremagene geperpavec

Krystal

Epidermolysis bullosa

Topical

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

TBD

betibeglogene autotemcel (Zynteglo)

Bluebird Bio

Sickle cell disease; Thalassemia

IV

Phase 3 – BLA; Breakthrough Therapy; Fast Track; Orphan Drug; RMAT

TBD

bevacizumab-vikg

Outlook

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

bexagliflozin

Theracos

T2DM

Oral

Phase 3 – NDA

TBD

bimekizumab

UCB

Axial spondyloarthritis; Hidradenitis suppurativa; PsA

SC

Phase 3 – BLA

TBD

bintrafusp alfa

Merck

Biliary tract cancer

IV

Phase 3 – BLA; Orphan Drug

TBD

BIVV 001

Sanofi

Hemophilia A

IV

Phase 3 – BLA; Orphan Drug

TBD

brensocatib

Insmed

Bronchiectasis

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

budesonide

Calliditas

Immunoglobulin A (IgA) nephropathy (Berger’s disease)

Oral

Phase 3 – 505(b)(2) NDA; Orphan Drug

TBD

cannabidiol gel

Zynerba

Fragile X syndrome

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

capivasertib

AstraZeneca

Breast cancer

Oral

Phase 3 – NDA

TBD

capsaicin

Centrexion

Osteoarthritis

Intraarticular

Phase 3 – 505(b)(2) NDA; Fast Track

TBD

carglumic acid

Recordati

Hyperammonaemia (autosomal disorder related)

Oral

Phase 3 – NDA; Orphan Drug

TBD

CD24Fc

OncoImmune

COVID-19

IV

Phase 3 – BLA

TBD

ceftobiprole medocaril

Basilea

ABSSSI; Bacteremia

IV

Phase 3 – NDA; Fast Track; QIDP

TBD

ceftriaxone wearable micropump

scPharmaceuticals

Gram+/Gram- infection

SC

Phase 3 – NDA

TBD

cenicriviroc mesylate

Allergan

NASH

Oral

Phase 3 – NDA; Fast Track

TBD

ciltacabtagene autoleucel

Janssen

Multiple myeloma (relapsed/refractory)

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

30 | MAGELLANRX.COM


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

cipaglucosidase alfa

Amicus

Pompe disease (in combination with miglustat)

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

clindamycin phosphate gel

Daré

Bacterial vaginosis

Intravaginal

Phase 3 – NDA; Fast Track; QIDP

TBD

CM-AT (pancreatic enzyme)

Curemark

Autism spectrum disorder

Oral

Phase 3 – BLA; Fast Track

TBD

crinecerfont

Neurocrine Biosciences

Congenital adrenal hyperplasia

Oral

Phase 3 – NDA

TBD

crisantaspase

Jazz

ALL; Lymphoblastic lymphoma

IM, IV

Phase 3 – BLA; Fast Track

TBD

crovalimab

Genentech

Paroxysmal nocturnal hemoglobinuria

IV, SC

Phase 3 – BLA; Orphan Drug

TBD

dactolisib

Restorbio

COVID-19

Oral

Phase 3 – NDA

TBD

dalcetrapib

Dalcor

Acute coronary syndrome (ADCY9 AA genotype)

Oral

Phase 3 – NDA

TBD

daprodustat

GlaxoSmithKline

Anemia due to CKD (dialysis-dependent, dialysis-independent)

Oral

Phase 3 – NDA

TBD

dehydrated human amnion chorion membrane

Mimedx

Achilles tendonitis; Plantar fasciitis

IV

Phase 3 – BLA

TBD

denosumab (biosimilar to Amgen’s Prolia®)

Novartis

Osteoporosis/osteopenia

SC

Phase 3 – BLA

TBD

deramanido

Otsuka

Tuberculosis

Oral

Phase 3 – NDA

TBD

dersimelagon

Mitsubishi Tanabe

Porphyria

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

deucravacitinib

Bristol-Myers Squibb

PSO

Oral

Phase 3 – NDA

TBD

dexamethasone SR

Otonomy

Meniere’s disease

Intratympanic

Phase 3 – 505(b)(2) NDA; Fast Track

TBD

dexmedetomidine

Bioxcel

Schizophrenia-related acute aggitation

SL

Phase 3 – 505(b)(2) NDA; Fast Track

TBD

dextromethorphan/ bupropion

Axsome

MDD

Oral

Phase 3 – 505(b)(2) NDA; Breakthrough Therapy; Fast Track

TBD

dociparstat

Chimerix

COVID-19

IV

Phase 3 – NDA

TBD

donaperminogene seltoplasmid

Helixmith

Diabetic foot ulcers

IM

Phase 3 – BLA

TBD

doravirine/islatravir

Merck

HIV-1 infection

Oral

Phase 3 – NDA

TBD

dovitinib lactate

Allarity

RCC

Oral

Phase 3 – NDA

TBD

dust mite immunotherapy

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

eculizumab (biosimilar to Alexion’s Soliris)

Amgen

Paroxysmal nocturnal hemoglobinuria

IV

Phase 3 – BLA

TBD

efgartigimod

Argenx

Immune thrombocytopenic purpura

IV, SC

Phase 3 – BLA; Orphan Drug

TBD

31 | MAGELLANRX.COM


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

elivaldogene autotemcel (Lenti-D)

Bluebird Bio

Adrenoleukodystrophy

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

enmetazobactam

Allecra

UTI (complicated)

IV

Phase 3 – NDA; Fast Track; QIDP

TBD

ensifentrine

Verona

COPD

Inhaled

Phase 3 – NDA

TBD

EP-2101 therapeutic vaccine

OSE Immunotherapeutics

NSCLC

SC

Phase 3 – NDA; Orphan Drug

TBD

episalvan

Amryt

Epidermolysis bullosa

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

eprenetapopt

Aprea

Myelodysplastic syndrome

IV

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

TBD

etanercept (biosimilar to Amgen’s Enbrel)

Coherus

RA; Polyarticular JIA; AS; PSO; PsA

SC

Phase 3 – BLA

TBD

etranacogene dezaparvovec

Uniqure

Hemophilia B

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

etrasimod

Arena

UC

Oral

Phase 3 – NDA

TBD

etrolizumab

Genentech

CD

SC

Phase 3 – BLA

TBD

exebacase

Contrafect

Staphylococcus aureus bacteremia

IV

Phase 3 – BLA; Breakthrough Therapy; Fast Track

TBD

faricimab

Genentech

Diabetic macular edema; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

fasinumab

Regeneron

Osteoarthritis

SC

Phase 3 – BLA

TBD

favipiravir

Dr. Reddy’s

COVID-19; Influenza

Oral

Phase 3 – NDA

TBD

fezagepras

Liminal

Alström syndrome

Oral

Phase 3 – NDA; Orphan Drug; Rare Pediatric Disease

TBD

fezolinetant

Astellas

Menopause vasomotor symptoms

Oral

Phase 3 – NDA

TBD

fidanacogene elaparvovec

Pfizer

Hemophilia B

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

filgotinib

Gilead

RA; CD; UC

Oral

Phase 3 – NDA

TBD

firmacute eubacterial spores

Seres

C. difficile infection (recurrent)

Oral

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

fitusiran

Sanofi

Hemophilia A and B (with and without inhibitors)

SC

Phase 3 – NDA; Orphan Drug

TBD

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

Allergan

Female reproductive disorder

SC

Phase 3 – BLA

TBD

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

Finox

Female reproductive disorder

SC

Phase 3 – BLA

TBD

follitropin delta

Ferring

Female reproductive disorder

IV

Phase 3 – BLA

TBD

32 | MAGELLANRX.COM


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

ganaxolone

Marinus

Status epilepticus; CDKL5 deficiency disorderrelated seizures

IV, Oral

Phase 3 – NDA; Orphan Drug

TBD

gantenerumab

Genentech

Alzheimer’s disease

SC

Phase 3 – BLA

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

givinostat

Italfarmaco

DMD

Oral

Phase 3 – NDA; Orphan Drug; Rare Pediatric Disease

TBD

glatiramer acetate depot

Mylan

MS

IM

Phase 3 – NDA

TBD

glepaglutide

Zealand

Short bowel syndrome

SC

Phase 3 – NDA; Orphan Drug

TBD

glofitamab

Genentech

DLBCL

IV

Phase 3 – BLA

TBD

hydrocortisone granules

Diurnal

Congenital adrenal hyperplasia

Oral

Phase 3 – 505(b)(2) NDA; Orphan Drug

TBD

hypericin

Soligenix

Cutaneous T cell lymphoma

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

idursulfase

Takeda

Mucopolysaccharidosis II Intrathecal (MPS II, Hunter syndrome)

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

infliximab (biosimilar to Janssen’s Remicade)

Nichi-Iko

RA; AS; PSO; PsA; CD; UC

IV

Phase 3 – BLA

TBD

insulin aspart (biosimilar to Novo Nordisk’s Novolog)

Mylan/Biocon

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)

Novo Nordisk

T2DM

SC

Phase 3 – NDA

TBD

insulin tregopil

Biocon

T2DM

Oral

Phase 3 – NDA

TBD

ipatasertib

Genentech

Breast cancer (TNBC/HR+, 1st-line, in combination with chemotherapy); Prostate cancer

Oral

Phase 3 – NDA

TBD

KSI-301

Kodiak

Diabetic macular edema; Macualar edema related to retinal vein occlusion

Intravitreal

Phase 3 – BLA

TBD

L-citrulline

Asklepion

Acute lung injury

IV

Phase 3 – NDA; Orphan Drug

TBD

Lactobacillus reuteri

Infant Bacterial

Necrotizing enterocol

Oral

Phase 3 – BLA; Orphan Drug

TBD

lebrikizumab

Dermira

Atopic dermatitis

SC

Phase 3 – BLA; Fast Track

TBD

lecanemab

Eisai

Alzheimer’s disease

IV

Phase 3 – BLA

TBD

lenacapavir

Viiv

HIV-1 infection (heavily treatment-experienced)

Oral (lead-in), SC

Phase 3 – NDA; Breakthrough Therapy

TBD

lenadogene nolparvovec (GS010)

Gensight

Leber’s hereditary optic neuropathy

Intravitreal

Phase 3 – BLA; Orphan Drug

TBD

lenzilumab

Humanigen

COVID-19

IV

Phase 3 – BLA

TBD

33 | MAGELLANRX.COM


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

leriglitazone

Minoryx

Adrenoleukodystrophy

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

leronlimab

Cytodyn

HIV-1 infection treatment (in combination therapy with HAART, highly treatment-experienced); COVID-19

SC

Phase 3 – BLA; Fast Track

TBD

levodopa/carbidopa patch pump

Mitsubishi Tanabe

Parkinson’s disease

SC

Phase 3 – 505(b)(2) NDA

TBD

levoketoconazole

Strongbridge

Cushing’s syndrome

Oral

Phase 3 – 505(b)(2) NDA; Orphan Drug

TBD

ligelizumab

Novartis

Urticaria

SC

Phase 3 – BLA; Breakthrough Therapy

TBD

linrodostat

Bristol-Myers Squibb

Bladder cancer

Oral

Phase 3 – NDA

TBD

linzagolix

Obseva

Endometriosis; Uterine fibroids

Oral

Phase 3 – NDA

TBD

lorecivivint

Samumed

Osteoarthritis (knee)

Intraarticular

Phase 3 – NDA

TBD

lucerastat

Idorsia

Fabry’s disease

Oral

Phase 3 – NDA; Orphan Drug

TBD

lutetium 177Lu-PSMA-617

Novartis

Prostate cancer

IV

Phase 3 – NDA

TBD

LYS-SAF302

Sarepta

Mucopolysaccharidosis IIIA (MPS IIIA; Sanfilippo A syndrome)

Intracerebral

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

magrolimab

Forty Seven

Myelodysplastic syndrome

IV

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

TBD

maribavir

Takeda

Cytomegalovirus infection treatment

Oral

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

TBD

masitinib

AB Science

Asthma; MS

Oral

Phase 3 – NDA

TBD

mavacamten

Myokardia

Obstructive hypertrophic cardiomyopathy

Oral

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

MBG453

Novartis

Myelodysplastic syndrome

IV

Phase 3 – BLA

TBD

metachromatic leukodystrophy gene therapy

Orchard

Metachromatic leukodystrophy

IV

Phase 3 – BLA; Orphan Drug; Rare Pediatric Disease; RMAT

TBD

microbiota suspension

Ferring

C. difficile infection (recurrent)

Rectal

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

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

PSO; CD; UC

IV, SC

Phase 3 – BLA

TBD

mirvetuximab soravtansine

Immunogen

Ovarian cancer

IV

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

34 | MAGELLANRX.COM


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

mitapivat

Agios

Pyruvate kinase deficiency

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

mobocertinib

Takeda

NSCLC

Oral

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

Moderna COVID-19 vaccine (mRNA-1273)

Moderna/NIAID

COVID-19

IM

Phase 3 – BLA; Fast Track

TBD

nabiximols

GW

MS-related spasticity

Oral submucosal

Phase 3 – NDA

TBD

nalbuphine ER

Trevi

Pruritus

Oral

Phase 3 – NDA

TBD

napabucasin

Sumitomo Dainippon

CRC

Oral

Phase 3 – NDA

TBD

natalizumab (biosimilar to Biogen’s Tysabri®)

Novartis

MS

IV

Phase 3 – BLA

TBD

nedosiran

Dicerna

Hyperoxaluria

SC

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug; Rare Pediatric Disease

TBD

nemolizumab

Galderma

Atopic dermatitis

SC

Phase 3 – BLA

TBD

nirsevimab

AstraZeneca

RSV infection prevention

IM

Phase 3 – BLA; Breakthrough Therapy; Fast Track

TBD

nomacopan

Akari

Paroxysmal nocturnal hemoglobinuria

SC

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

NVX-CoV2373 vaccine

Novavax

COVID-19

IM

Phase 3 – BLA

TBD

odevixibat

Albireo

Alagille syndrome; Oral Biliary Atresia (post Kasai hepatoportoenterostomy)

Phase 3 – NDA; Orphan Drug

TBD

olipudase alfa

Sanofi

Niemann-Pick disease

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

ondansetron ER once-daily

RedHill

Gastroenteritis

Oral

Phase 3 – 505(b)(2) NDA

TBD

oteseconazole

Mycovia

Vulvovaginal candidiasis

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

OTL-103

Orchard

Wiskott-Aldrich syndrome IV

Phase 3 – BLA; Orphan Drug; RMAT

TBD

oxalobacter formigenes

Oxthera

Hyperoxaluria

Oral

Phase 3 – BLA; Orphan Drug

TBD

pacritinib

CTI

COVID-19; Myelofibrosis

Oral

Phase 3 – NDA

TBD

palovarotene

Ipsen

Fibrodysplasia ossificans progressiva

Oral

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

TBD

pamrevlumab

Fibrogen

DMD; Idiopathic pulmonary fibrosis

IV

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

pegzilarginase

Aeglea

Arginase 1 deficiency

IV

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

TBD

35 | MAGELLANRX.COM


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

pevonedistat

Takeda

Myelodysplastic syndrome

IV

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

PF-06939926

Pfizer

DMD

IV

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

Pfizer-Biontech COVID-19 vaccine (BNT162b2)

Pfizer/Biontech

COVID-19

IM

Phase 3 – BLA; Emergency Use Authorization

TBD

plinabulin

Beyondspring

NSCLC; Neutropenia/ leukopenia

IV

Phase 3 – NDA; Breakthrough Therapy

TBD

pollinex quattro grass

Allergy

Allergic rhinitis

SC

Phase 3 – BLA

TBD

potassium citrate/ potassium bicarbonate

Advicenne

Renal tubular acidosis

Oral

Phase 3 – NDA

TBD

PTI-NC-733

Proteostasis

CF

Oral

Phase 3 – NDA; Fast Track

TBD

ranibizumab (biosimilar to Genentech’s Lucentis)

Coherus

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

ranibizumab (biosimilar to Genentech’s Lucentis)

STADA Arzneimittel/ Bausch

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

ranibizumab intravitreal implant

Genentech

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

REGN-COV2

Regeneron

COVID-19

SC

Phase 3 – BLA

TBD

relacorilant

Corcept

Cushing’s syndrome

Oral

Phase 3 – NDA; Orphan Drug

TBD

relugolix/estradiol/ norethindrone

Myovant

Endometriosis

Oral

Phase 3 – NDA

TBD

reproxalap

Aldeyra

Dry eye syndrome

Ophthalmic

Phase 3 – NDA

TBD

resmetirom

Madrigal

NASH

Oral

Phase 3 – NDA; Fast Track

TBD

ridinilazole

Summit

C. difficile-associated diarrhea

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

rigosertib

Onconova

Myelodysplastic syndrome

IV

Phase 3 – NDA; Orphan Drug

TBD

rilzabrutinib

Principia

Pemphigus vulgaris

Oral

Phase 3 – NDA; Orphan Drug

TBD

ritlecitinib

Pfizer

Alopecia areata

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

rituximab (biosimilar to Genentech’s Rituxan)

Archigen

RA; CLL/SLL; NHL (indolent); ANCAassociated vasculitis

IV

Phase 3 – BLA

TBD

roflumilast cream

Arcutis

PSO

Topical

Phase 3 – NDA

TBD

ruxolitinib cream

Incyte

Atopic dermatitis; Vitiligo

Topical

Phase 3 – NDA

TBD

seladelpar

Cymabay

Primary biliary cholangitis Oral

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

sepofarsen

Proqr

Leber’s congenital amaurosis

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

36 | MAGELLANRX.COM

Intravitreal


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

sodium hyaluronate/ triamcinolone hexacetonide

Anika

Osteoarthritis (knee)

Intraarticular

Phase 3 – NDA

TBD

sodium thiosulfate

Fennec

Chemotherapy-induced ototoxicity prevention

IV

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

TBD

sofpironium

Brickell

Axillary hyperhidrosis

Topical

Phase 3 – NDA

TBD

sotagliflozin

Lexicon

Heart failure in patients with T2DM

Oral

Phase 3 – NDA; Orphan Drug

TBD

sparsentan

Retrophin

Focal segmental glomerulosclerosis; Immunoglobulin A (IgA) nephropathy (Berger’s disease)

Oral

Phase 3 – NDA; Orphan Drug

TBD

tapinarof

Roivant

PSO

Topical

Phase 3 – NDA

TBD

tebipenem pivoxil

Spero

UTI (complicated); Acute pyelonephritis

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

tecarfarin

Espero

Anticoagulation

Oral

Phase 3 – NDA

TBD

teprasiran

Quark

Kidney injury prevention following cardiac surgery

IV

Phase 3 – NDA

TBD

tesetaxel

Odonate

Breast cancer

Oral

Phase 3 – NDA

TBD

tetrathiomolybdate

Alexion

Wilson’s disease

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

tezepelumab

Amgen

Asthma (severe, uncontrolled)

SC

Phase 3 – BLA; Breakthrough Therapy

TBD

timbetasin

Regenerx

Dry eye syndrome

Topical

Phase 3 – BLA

TBD

tiragolumab

Genentech

SCLC

IV

Phase 3 – BLA

TBD

tirzepatide

Eli Lilly

T2DM; Obesity

SC

Phase 3 – NDA

TBD

tisagenlecleucel-t (Kymriah)

Novartis

DLBCL (1st relapse)

IV

Phase 3 – BLA; Orphan Drug

TBD

tofersen

Biogen

Amyotrophic lateral sclerosis

Intrathecal

Phase 3 – NDA; Orphan Drug

TBD

tominersen

Genentech

Huntington’s disease

Intrathecal

Phase 3 – NDA; Orphan Drug

TBD

tonogenchoncel-L

Kolon Tissuegene

Osteoarthritis

Intraarticular

Phase 3 – BLA

TBD

tradipitant

Vanda

Atopic dermatitis; COVID-19; Emesis; Gastroparesis

Oral

Phase 3 – NDA

TBD

trastuzumab (biosimilar to Genentech’s Herceptin)

Novartis

Breast cancer; Gastric/ gastroesophageal cancer

IV

Phase 3 – BLA

TBD

trastuzumab (biosimilar to Genentech’s Herceptin)

Tanvex

Breast cancer; Gastric/ gastroesophageal cancer

IV

Phase 3 – BLA

TBD

trehalose

Seelos

Oculopharyngeal muscular dystrophy

IV

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

triamcinolone acetonide

Bausch Health

Uveitis

Intravitreal

Phase 3 – 505(b)(2) NDA

TBD

trientine tetrahydrochloride

GMP-Orphan

Wilson’s disease

Oral

Phase 3 – NDA

TBD

37 | MAGELLANRX.COM


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

trofinetide

Acadia

Rett syndrome

Oral

Phase 3 – NDA; Fast Track; Orphan Drug; Rare Pediatric Disease

TBD

ublituximab

TG

MS

IV

Phase 3 – BLA

TBD

ublituximab + umbralisib

TG

CLL

IV + Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Amgen

PSO

IV, SC

Phase 3 – BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Formycon

PSO

IV, SC

Phase 3 – BLA

TBD

valoctocogene roxaparvovec

Biomarin

Hemophilia A

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

vazegepant

Biohaven

Migraine treatment; COVID-19

Intranasal

Phase 3 – NDA

TBD

veliparib

Abbvie

Breast cancer; Ovarian cancer

Oral

Phase 3 – NDA; Orphan Drug

TBD

venglustat

Sanofi

GM2 gangliosidoses (TaySachs disease, Sandhoff disease, AB Variant); Polycystic kidney disease

Oral

Phase 3 – NDA; Orphan Drug

TBD

verbrinacogene setparvovec

Freeline

Hemophilia B

IV

Phase 3 – BLA

TBD

veverimer

Tricida

CKD-related metabolic acidosis

Oral

Phase 3 – NDA

TBD

VGX-3100 therapeutic vaccine

Inovio

Cervical dysplasia

IM

Phase 3 – BLA

TBD

(vic-)trastuzumab duocarmazine

Byondis

Breast cancer (HER2+)

IV

Phase 3 – BLA; Fast Track

TBD

volanesorsen

Akcea

Familial chylomicronemia syndrome

SC

Phase 3 – NDA; Orphan Drug

TBD

vonoprazan

Phathom

Esophagitis; H. pylori infection

Oral

Phase 3 – NDA; QIDP

TBD

vutrisiran

Alnylam

Transthyretin amyloid cardiomyopathy (ATTR-CM, wild type or hereditary); Transthyretin amyloid polyneuropathy

SC

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

zilucoplan

Ra

Myasthenia gravis

SC

Phase 3 – NDA; Orphan Drug

TBD

ziritaxestat

Galapagos

Idiopathic pulmonary fibrosis

Oral

Phase 3 – NDA; Orphan Drug

TBD

zoliflodacin

Entasis

Gonorrhea

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

Phase 3 (Supplementals) anakinra (Kineret )

Swedish Orphan Biovitrum

COVID-19

SC

Phase 3 – sBLA

TBD

baricitinib (Olumiant)

Eli Lilly

Alopecia areata; COVID-19; SLE

Oral

Phase 3 – sNDA; Breakthrough Therapy

TBD

benralizumab (Fasenra®)

AstraZeneca

Nasal polyposis

SC

Phase 3 – sBLA

TBD

®

38 | MAGELLANRX.COM


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

brexpiprazole (Rexulti®)

Otsuka

Alzheimer’s diseaserelated agitation; PTSD

Oral

Phase 3 – sNDA; Fast Track

TBD

canakinumab (Ilaris®)

Novartis

COVID-19

SC

Phase 3 – sBLA

TBD

dapagliflozin (Farxiga)

AstraZeneca

Diabetic nephropathy; COVID-19

Oral

Phase 3 – sNDA; Breakthrough Therapy; Fast Track

TBD

dehydroepiandrosterone (Intrarosa®)

Millicent

Female sexual arousal disorder

Intravaginal

Phase 3 – sNDA

TBD

dupilumab (Dupixent®)

Sanofi

COPD; Eosinophilic esophagitis; Pruritus, Urticaris

SC

Phase 3 – sBLA

TBD

empagliflozin (Jardiance)

Boehringer Ingelheim

Chronic heart failure; CKD; Diabetic nephropathy

Oral

Phase 3 – sNDA; Fast Track

TBD

hydrogen peroxide (Eskata®)

Aclaris

Warts

Topical

Phase 3 – sNDA

TBD

immune globulin intravenous (human) 10% (Octagam®)

Octapharma

COVID-19

IV

Phase 3 – sBLA

TBD

L-lactic acid/citric acid/ potassium bitartrate (Phexxi®)

Evofem

Chlamydia trachomatis infection; Neisseria gonorrhoeae infection

Intravaginal

Phase 3 – sNDA; Fast Track; QIDP

TBD

lonafarnib (Zokinvy®)

Eiger

Hepatitis D infection

Oral

Phase 3 – sNDA; Breakthrough Therapy; Fast Track; Orphan Drug

TBD

mepolizumab (Nucala)

GlaxoSmithKline

COPD; Nasal polyposis

SC

Phase 3 – sBLA

TBD

meropenem/vaborbactam (Vabomere®)

Melinta

Bacteremia; HAP

IV

Phase 3 – sNDA; QIDP TBD

nitazoxanide (Alinia®)

Lupin

COVID-19; Influenza

Oral

Phase 3 – sNDA

TBD

obeticholic acid (Ocaliva )

Intercept

NASH

Oral

Phase 3 – sNDA; Breakthrough Therapy

TBD

omalizumab (Xolair®)

Genentech

Food allergies

SC

Phase 3 – sBLA; Breakthrough Therapy

TBD

ozanimod (Zeposia®)

Bristol-Myers Squibb

UC

Oral

Phase 3 – sNDA

TBD

patisiran (Onpattro )

Alnylam

Transthyretin amyloid cardiomyopathy (ATTR-CM, wild-type or hereditary)

IV

Phase 3 – sNDA

TBD

risankizumab-rzaa (Skyrizi®)

Abbvie

PsA; CD; UC

SC

Phase 3 – sBLA; Orphan Drug

TBD

rivaroxaban (Xarelto®)

Janssen

COVID-19

Oral

Phase 3 – sNDA

TBD

sacituzumab govitecanhziy (Trodelvy)

Immunomedics

Bladder cancer (3rd-line)

IV

Phase 3 – sBLA; Fast Track

TBD

selinexor (Xpovio®)

Karyopharm

Lipoarcoma

Oral

Phase 3 – sNDA; Orphan Drug

TBD

ticagrelor (Brilinta®)

AstraZeneca

Sickle cell disease

Oral

Phase 3 – sNDA

TBD

upadacitinib (Rinvoq)

Abbvie

CD; UC

Oral

Phase 3 – sNDA; Orphan Drug

TBD

valsartan/sacubitril (Entresto)

Novartis

Post-acute myocardial infarction

Oral

Phase 3 – sNDA

TBD

®

®

39 | MAGELLANRX.COM


PIPELINE DRUG LIST continued

Complete Response Letter (CRL)/Withdrawn Drugs NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

arbaclofen ER

Osmotica

MS-related spasticity

Oral

CRL

TBD

baloxavir marboxil (Xofluza®) oral granules, tablets

Genentech

Influenza (treatment, ages Oral 1-12 years; postexposure prophylaxis, ages 1 year to adults)

CRL

TBD

buprenorphine ER

Braeburn

Substance use disorder

SC

CRL

TBD

furosemide wearable pump Scpharmaceuticals

Congestive heart failure

SC

CRL

TBD

inclisiran

The Medicines Company

Dyslipidemia (for secondary prevention patients with ASCVD and familial hypercholesterolemia)

SC

CRL

TBD

naloxone (high dose)

US WorldMeds

Opioid overdoise

IM

CRL

TBD

sutimlimab

Sanofi

Cold agglutnin disease

IV

CRL

TBD

treprostinil dry powder

Liquidia

PAH

Inhaled

CRL

TBD

viloxazine

Supernus

ADHD (ages 6 to 17 years)

Oral

CRL

TBD

40 | MAGELLANRX.COM


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

ACEI Angiotensin-Converting Enzyme Inhibitor ACR20 American College of Rheumatology 20% Improvement ACR50 American College of Rheumatology 50% Improvement ACR70 American College of Rheumatology 70% Improvement ADHD Attention Deficit Hyperactivity Disorder ADL Activities of Daily Living AED Anti-Epileptic Drug ALK Anaplastic Lymphoma Kinase ALL Acute Lymphoblastic Leukemia ALT Alanine Transaminase AMD Age-Related Macular Degeneration AML Acute Myeloid Leukemia ANCA Antineutrophil Cytoplasmic Antibodies ANDA Abbreviated New Drug Application ARB Angiotensin II Receptor Blocker ARNI Angiotensin Receptor II Blocker – Neprilysin Inhibitor ART Antiretroviral Therapy ARV Antiretroviral AS Ankylosing Spondylitis ASCVD Atherosclerotic Cardiovascular Disease AST Aspartate Aminotransferase BCVA Best Corrected Visual Acuity BLA Biologics License Application BMI Body Mass Index BsUFA Biosimilar User Fee Act CABP Community Acquired Bacterial Pneumonia 41 | MAGELLANRX.COM

CD Crohn's Disease CDC Centers for Disease Control and Prevention CF Cystic Fibrosis CHF Congestive Heart Failure CI Confidence Interval CKD Chronic Kidney Disease CLL Chronic Lymphocytic Leukemia CNS Central Nervous System COPD Chronic Obstructive Pulmonary Disease COVID-19 Coronavirus Disease 2019 CRC Colorectal Cancer CRL Complete Response Letter CSF Colony Stimulating Factor CV Cardiovascular CVD Cardiovascular Disease DAS28-CRP Disease Activity Score-28 with C Reactive Protein DEA Drug Enforcement Administration DLBCL Diffuse Large B Cell Lymphoma DMARD Disease Modifying Antirheumatic Drug DMD Duchenne Muscular Dystrophy DNA Deoxyribonucleic Acid DOR Duration of Response DPP-4 Dipeptidyl Peptidase 4 DR Delayed-Release ECOG Eastern Cooperative Oncology Group EDSS Expanded Disability Status Scale eGFR estimated Glomerular Filtration Rate ER Extended-Release


GLOSSARY continued ESRD End-Stage Renal Disease

IGA Investigator's Global Assessment

FDA Food and Drug Administration

IM Intramuscular

FH Familial Hypercholesterolemia

ITP Immune Thrombocytopenic Purpura

FLT3 FMS-Like Tyrosine Kinase-3

ITT Intent-To-Treat

G-CSF Granulocyte Colony Stimulating Factor

IV Intravenous

GI Gastrointestinal

JIA Juvenile Idiopathic Arthritis

GIST Gastrointestinal Stromal Tumor

LDL Low-Density Lipoprotein

GLP-1RA Glucagon-Like Peptide-1 Receptor Agonist

LDL-C Low-Density Lipoprotein Cholesterol

GM-CSF Granulocyte-Macrophage Colony Stimulating Factor

mAb Monoclonal Antibody

GVHD Graft Versus Host Disease H Half HAART Highly Active Antiretroviral Therapy HAM-D Hamilton Depression Rating Scale HAP Healthcare-Associated Pneumonia Hb Hemoglobin HbA1c Hemoglobin A1c HCC Hepatocellular Carcinoma HCP Healthcare Professional HCV Hepatitis C Virus HER Human Epidermal Growth Factor Receptor HER2 Human Epidermal Growth Factor Receptor 2

MACE Major Adverse Cardiovascular Events MADRS Montgomery – Åsberg Depression Rating Scale MAOI Monoamine Oxidase Inhibitor MDD Major Depressive Disorder MDI Metered Dose Inhaler MRI Magnetic Resonance Imaging MRSA Methicillin-Resistant Staphylococcus Aureus MS Multiple Sclerosis N/A Not Applicable NASH Non-Alcoholic Steatohepatitis NDA New Drug Application NHL Non-Hodgkin Lymphoma

HFA Hydrofluoroalkane

NIAID National Institute of Allergy and Infectious Diseases

HIT Heparin Induced Thrombocytopenia

NSAID Non-Steroidal Anti-Inflammatory Drug

HIV Human Immunodeficiency Virus

NSCLC Non-Small Cell Lung Cancer

HIV-1 Human Immunodeficiency Virus-1

ODT Orally Disintegrating Tablet

HR Hazard Ratio

OR Odds Ratio

HSCT Hematopoietic Stem Cell Transplant

ORR Overall/Objective Response Rate

HTN Hypertension

OS Overall Survival

IBS Irritable Bowel Syndrome

PAH Pulmonary Arterial Hypertension

IBS-C Irritable Bowel Syndrome, Constipation Predominant

PARP Poly(ADP-ribose) polymerase

42 | MAGELLANRX.COM

PASI Psoriasis Area and Severity Index


GLOSSARY continued PASI 50 Psoriasis Area and Severity Index 50%

SCCHN Squamous Cell Cancer of the Head and Neck

PASI 70 Psoriasis Area and Severity Index 70%

SCLC Small Cell Lung Cancer

PASI 90 Psoriasis Area and Severity Index 90%

SCT Stem Cell Transplant

PASI 100 Psoriasis Area and Severity Index 100%

SGLT2 Sodium-Glucose Co-Transporter 2

PCI Percutaneous Coronary Intervention

SL Sublingual

PCSK9 Proprotein Convertase Subtilisin Kexin 9

SLE Systemic Lupus Erythematosus

PD-1 Programmed Death Protein 1

SLL Small Lymphocytic Lymphoma

PD-L1 Programmed Death-Ligand 1

sNDA supplemental New Drug Application

PDUFA Prescription Drug User Fee Application

SNRI Serotonin and Norepinephrine Reuptake Inhibitor

PFS Progression-Free Survival PGA Physician Global Assessment PsA Psoriatic Arthritis PSO Plaque Psoriasis PTCA Percutaneous Transluminal Coronary Angioplasty

SOC Standard of Care sPGA static Physician Global Assessment SR Sustained-Release SSRI Selective Serotonin Reuptake Inhibitor SSSI Skin and Skin Structure Infection

PTSD Post-Traumatic Stress Disorder

T1DM Type 1 Diabetes Mellitus

Q Quarter

T2DM Type 2 Diabetes Mellitus

QIDP Qualified Infectious Diseases Product

TBD To Be Determined

QOL Quality of Life

TEAE Treatment-Emergent Adverse Events

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

TNBC Triple Negative Breast Cancer

RA Rheumatoid Arthritis RBC Red Blood Cell RCC Renal Cell Carcinoma REMS Risk Evaluation and Mitigation Strategy RMAT Regenerative Medicine Advanced Therapy RNA Ribonucleic Acid RRR Relative Risk Reduction RSV Respiratory Syncytial Virus RTOR Real-Time Oncology Review sBLA supplemental Biologics License Application SC Subcutaneous

43 | MAGELLANRX.COM

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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