2023 July Quarterly Pipeline

Table of CONTENTS

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

EDITOR-IN-CHIEF'S MESSAGE

2

Consultant Panel

PIPELINE DEEP DIVE

3

Katie Lockhart Senior Manager, Forecasting and Pharmacoeconomics

KEEP ON YOUR RADAR

29

PIPELINE DRUG LIST

30

GLOSSARY

49

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

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

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

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

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

METHODOLOGY

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

REFLECTION

Thus far in 2023, the agency has approved 29 novel drugs, roughly 45% more approvals compared to about the same time last year. Notably, most of the approvals so far in 2023 use at least one of the FDA’s expedited approval methods and the majority of drugs are designated as Orphan Drugs. Approvals included many firsts. Among them are first-time approvals for gene therapies for hemophilia A and Duchenne muscular dystrophy, first once-weekly topical gene therapy for a rare dermatologic condition, first vaccines for RSV prevention in older adults, first single-injection monoclonal antibody for RSV prevention in infants, first cell-based therapy for type 1 diabetes, and a full traditional approval for an Alzheimer’s anti-amyloid antibody. Several long-awaited biosimilars to adalimumab (Humira®), including citrate-free and high-concentration formulations plus an interchangeable product, have launched.

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

ON THE HORIZON

As we look ahead, there is a continued trend towards the approval of specialty medications and drugs for rare conditions, with 74% and 36% of approvals expected, respectively, for agents with applications submitted to the FDA. There is 1 agent seeking FDA’s Accelerated Approval, which allows for earlier drug approval for serious conditions that fill an unmet need based on a surrogate endpoint reasonably likely to predict a clinical benefit. Approval of 2 new gene therapies for sickle cell anemia and thalassemia are on the horizon. FDA decision for a biosimilar to Neulasta® Onpro® is anticipated. Moreover, sprouting products for obesity, NASH, and Alzheimer's disease are being actively monitored through MRx Pipeline. Other noteworthy pipeline trends to watch include the development of complex therapies, cell and gene therapies “CGT,” oncology, immunology, and therapeutic options for ultra-rare hereditary diseases.

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

97%

20%

9%

BIOSIMILAR

ORPHAN DRUG

69%

26%

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

OPHTHALMOLOGY

avacincaptad pegol intravitreal Astellas PROPOSED INDICATIONS

Geographic atrophy (GA) secondary to age-related macular degeneration (AMD)

CLINICAL OVERVIEW

Avacincaptad pegol is a pegylated RNA aptamer that inhibits complement C5.

The safety and efficacy of avacincaptad pegol were evaluated in the randomized, double-masked, shamcontrolled, phase 2/3 GATHER1 (NCT02686658; n=286) and GATHER2 (NCT04435366; n=448) trials in adults ≥ 50 years of age with GA secondary to AMD. Enrolled patients had no foveal involvement and a best-corrected visual acuity (BCVA) between 20/25 and 20/320 in the study eye. In both trials, at month 12, avacincaptad pegol 2 mg doses led to a significant reduction from baseline in the primary endpoint of mean change in the GA area compared to sham (GATHER1: 0.668 mm2 [35.4%]; p=0.005; GATHER2: 0.376 mm2 [17.7%]; p=0.0039). Avacincaptad pegol was generally well-tolerated. Notable ocular TEAEs were conjunctival hemorrhage and choroidal neovascularization (CNV). No cases of endophthalmitis or ischemic optic neuropathy were reported. Data also showed a 56% relative risk reduction in persistent vision loss (over 2 consecutive visits) with avacincaptad pegol 2 mg compared to sham.

Avacincaptad pegol was administered as monthly intravitreal injections in the affected eye(s). In GATHER2, at month 12, patients in the avacincaptad pegol arm were re-randomized to receive avacincaptad pegol 2 mg once monthly or once every other month through month 24. Results are pending.

PLACE IN THERAPY

GA is an advanced form of nonexudative (dry) AMD, which is the leading cause of blindness in the US. GA typically occurs in both eyes and is characterized by chronic, progressive atrophy of outer retinal tissue, retinal pigment epithelium, and choriocapillaris that lead to marked decline of visual acuity. As the condition advances, GA lesions can expand into the fovea, resulting in loss of central vision. The rate of progression of GA varies. The reported median lesion growth rate is 2.1 mm2/year, but may range up to 10.2 mm2/year.

Some data suggest that over 31% of patients with GA will lose ≥ 3 lines of vision in 2 years. GA affects approximately 20% of individuals with AMD. It is estimated that 1 million people in the US have GA and the incidence will likely rise as the age-burden increases. Risk factors for GA include age ≥ 60 years, family history of AMD, and current or past smoking.

The complement cascade has been implicated in the development of GA. This system is important for tissue homeostasis and can lead to excessive cell death when unregulated. If approved, avacincaptad pegol will be the second agent in the US to treat GA secondary to AMD, following the February 2023 approval of pegcetacoplan (Syfovre™). Both agents are administered via intravitreal injection. While avacincaptad pegol inhibits complement C5, located distally in the complement cascade, pegcetacoplan inhibits complement C3, which is centrally located in the cascade. Pegcetacoplan has demonstrated a reduction in the mean GA lesion growth through 24 months (range, 17% to 22%) with doses administered every 25 to 60 days in patients with or without foveal involvement. In clinical trials, avacincaptad pegol demonstrated mean GA lesion reduction at 12 months (range, 17.7% to 35.4%) when given once monthly and has the potential for every-other-month dosing (pending study results). Post hoc analyses for each drug showed slowing of decline in visual acuity.

FDA APPROVAL TIMELINE August 19, 2023

 Breakthrough Therapy

 Fast Track

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$8

$126

$297

$534

$765

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ONCOLOGY

Multiple myeloma elranatamab SC Pfizer PROPOSED INDICATIONS

Relapsed or refractory multiple myeloma (RRMM)

CLINICAL OVERVIEW

Elranatamab is an off-the-shelf B cell maturation antigen (BCMA) CD3-targeted bispecific antibody. It binds to and bridges BCMA on the surface of myeloma cells and CD-3 receptors on T cells. This leads to T cell activation and myeloma cell death.

The ongoing, multicenter, open-label, single-arm, phase 2 MagnetisMM-3 study (NCT04649359) evaluated the efficacy and safety of elranatamab monotherapy in 123 patients with RRMM. Enrolled patients had received prior treatment with ≥ 1 proteasome inhibitor, ≥ 1 immunomodulatory agent, and ≥ 1 anti-CD38 antibody. After a median follow-up of 10.4 months, the ORR for elranatamab was 61%, with 55% of patients achieving at least a very good partial response (VGPR). It was estimated that 84% of patients would maintain the response at 9 months. In addition, an analysis of pooled data from 3 MagnetisMM studies (MM-1 [phase 1; NCT03269136], MM-3, MM-9 [phase 2; NCT05014412]) included data from 86 patients (64 from MM3) whose prior therapy included BCMA-directed therapy (ADC and/or CAR T). After a median follow-up of 10.3 months (range, 0.3 to 32.3), the ORR was 41.4% and 52.8% in patients with prior BCMA-directed ADC and CAR T therapies, respectively. Among the responders, the median time to achieving an objective response was 1.9 months. At 9 months, the DOR rate was 67.3% and 78.9% in patients with prior ADC and CAR T therapy, respectively.

In the MagnetisMM-3 study, elranatamab was administered as 76 mg SC once weekly on a 28-day cycle with a step-up priming dose regimen of 12 mg and 32 mg administered on day 1 and day 4, respectively, during cycle 1.

FDA APPROVAL TIMELINE August to September 2023  Breakthrough Therapy

 Fast Track

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$25

$66

$193

$285

$490

talquetamab SC Janssen PROPOSED INDICATIONS

Relapsed or refractory multiple myeloma (RRMM)

CLINICAL OVERVIEW

Talquetamab is an off-the-shelf bispecific T cell engager antibody. It binds to G protein-coupled receptor class C group 5-member D (GPRC5D) on the surface of myeloma cells and CD-3 receptors on T cells. This leads to T cell-mediated myeloma cell death.

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Multiple myeloma (cont.) talquetamab (cont.)

CLINICAL OVERVIEW (cont.)

An ongoing, open-label, phase 2 study (NCT04634552) evaluated the efficacy and safety of talquetamab in patients with RRMM who received ≥ 3 prior lines of therapy, including ≥1 proteasome inhibitor, ≥1 immunomodulatory drug, and ≥1 anti-CD38 antibody. Data from 288 patients have been reported, of whom 51 received prior T cell-redirection therapy (CAR T cells, bispecific antibodies). The study demonstrated an ORR of 71.7% and 74.1% in patients who received talquetamab dosed once weekly (at median follow-up of 12.7 months) and every other week (at median follow-up of 18.8 months), respectively. In each dosing group, approximately 60% achieved a VGPR or better, approximately 9% achieved a complete response, and about 24% and 30%, respectively, had a stringent complete response. Among patients with prior T cell-redirection therapy, at a median follow-up of 14.8 months, the ORR was 64.7%, with a VGPR or better of 54.9%. The DOR was ≥ 9 months in all groups, with the longest among those who experienced a complete response.

The median PFS was 7.5 and 11.9 months in the weekly and every-other-week dosing cohorts, respectively, and 5.1 months among the prior T cell-redirection cohort. Most adverse events were mild to moderate and manageable. CRS occurred in 75% to 79% of patients across the 2 study cohorts. The incidence of infections, a common comorbidity in patients with multiple myeloma, was 58% to 71% (grade 3/4, 16% to 26%) across the cohorts. No deaths occurred related to talquetamab therapy.

In the phase 2 study, talquetamab is given SC as 0.4 mg/kg once weekly or 0.8 mg/kg every other week with step-up doses. Treatment is continued until disease progression.

Talquetamab is also being studied in an ongoing phase 3 study (NCT05455320) in combination with daratumumab ± pomalidomide. Encouraging results were also seen with talquetamab plus teclistamab (ORR ≥ 85%) in a phase 1b/2 dose escalation study (NCT04586426).

FDA APPROVAL TIMELINE December 11, 2023

 Breakthrough Therapy

 Orphan Drug

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$8

$30

$80

$125

$163

PLACE IN THERAPY

MM is a malignancy of plasma cells that accumulate in bone marrow resulting in marrow failure. It accounts for about 18% of all hematologic malignancies in the US. Approximately 35,730 new cases of MM and 12,600 deaths due to the condition are predicted in the US in 2023. MM is usually diagnosed in adults ≥ 65 years of age and is twice as likely to occur in African Americans than Caucasians. Risk factors may include obesity/ overweight, other active plasma cell diseases, and a family history of MM. There is no cure for MM. While patients typically respond to initial therapy, eventual treatment-resistant relapse usually occurs, including after HSCT and radiation therapy.

Triplet therapy is the standard pharmacotherapy for previously treated MM. Preferred regimens consist of dexamethasone plus 2 of the following: a proteasome inhibitor, an immunomodulatory agent, or a monoclonal antibody. Newer alternatives for patients who have received ≥ 4 prior lines of therapies include agents that target BCMA, which is present in 60% to 70% of patients with MM. BCMA-directed treatments include monotherapy with the ADC teclistamab-cqyv (Tecvayli™) and the CAR T therapies idecabtagene vicleucel (ide-cel; Abecma®) and ciltacabtagene autoleucel (cilta-cel; Carvykti®). Notably, the ADC belantamab mafodotin-blmf (Blenrep®) was withdrawn from the US market based on poor confirmatory trial results in patients with RRMM.

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Multiple myeloma (cont.) talquetamab (cont.)

PLACE IN THERAPY (cont.)

If approved, elranatamab and talquetamab will be off-the-shelf (ready-to-use), SC-administered, T cellredirecting bispecific antibody options for RRMM, in addition to the currently available teclistamab-cqyv.

Initial approvals for elranatamab and talquetamab are expected to be in heavily pretreated patients. In non-comparative monotherapy trials, the ORR for talquetamab, but not elranatamab, was higher compared to teclistamab-cqyv (ORR, >71% and 61%, versus 63%, respectively) and are lower than the ORR for CAR T cell therapies (ORR, 95% with Carvykti and 72% with Abecma). However, bispecific antibody therapies have an overall more favorable tolerability profile compared to CAR T therapies, including a lower incidence and severity of CRS. Elranatamab, talquetamab, and teclistamab have the potential for use in combination with other agents (e.g., daratumumab, pomalidomide, dexamethasone) in earlier lines of therapy.

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HEMATOLOGY/GENE THERAPY

exagamglogene autotemcel IV Vertex/CRISPR PROPOSED INDICATIONS

» Transfusion-dependent beta-thalassemia (TDT) » Severe sickle cell disease (SCD)

CLINICAL OVERVIEW

Exagamglogene autotemcel (exa-cel) is an autologous, ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene-edited therapy. With exa-cel, a patient’s hematopoietic stem cells (HSCs) are engineered to produce RBCs with high levels of fetal hemoglobin (hemoglobin F; HbF). Increased levels of HbF may reduce vaso-occlusive episodes (VOEs) in patients with SCD and the need for blood transfusions in those with TDT. The single-arm, open-label phase 1/2/3 trials CLIMB-THAL-111 (NCT03655678) and CLIMB-SCD-121 (NCT03745287) evaluated the efficacy and safety of exa-cel in patients 12 to 35 years of age with TDT and severe SCD, respectively. At the time of data cut-off in CLIMB-THAL-111, 27 patients with TDT had received exa-cel and had evaluable data. Among these patients, 88.9% achieved the primary endpoint of transfusionindependence for ≥ 12 months. The mean duration of transfusion independence was 20.5 months (maximum 40.7 months). Rapid and sustained increases in total Hb (≥ 11 g/dL) were seen as early as month 3. In CLIMBSCD-121, at data cut-off, 17 patients with SCD who received exa-cel had evaluable data. The primary endpoint of freedom from VOEs for ≥ 12 months was achieved by 94.1% of the patients. The mean duration of freedom from VOEs was 18.7 months (maximum 36.5 months). Early and sustained increases in total Hb and HbF were demonstrated within the first few months. In both studies, the mean proportion of edited BCL11A alleles in the bone marrow and peripheral blood was stable over time, demonstrating a successful permanent editing in the long-term hematopoietic stem cells. Two patients with TDT experienced serious TEAEs; 1 instance each of hemophagocytic lymphohistiocytosis (HLH), acute respiratory distress syndrome, headache, and idiopathic pneumonia syndrome was reported. No serious TEAEs were reported in patients with SCD. In both trials, after packed RBC transfusions and stem cell mobilization, CD34+ hematopoietic stem and progenitor cells (HSPCs) were collected from patients via apheresis. Collected cells were then edited with CRISPER-Cas9 technology. Patients received myeloablative therapy before receiving a single, 1-time IV infusion of the edited HSPCs (exa-cel).

FDA APPROVAL TIMELINE SCD – December 8, 2023  Fast Track

 Orphan Drug

 Priority Review

 RPD

 RMAT

TDT – March 30, 2024  Fast Track

 Orphan Drug

 RPD

 RMAT

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$0

$141

$360

$577

$780

Includes projected annual sales for TDT and SCD.

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lovotibeglogene autotemcel IV Bluebird Bio PROPOSED INDICATIONS

Severe sickle cell disease (SCD) in patients with a history of vaso-occlusive events (VOEs)

CLINICAL OVERVIEW

Lovotibeglogene autotemcel (lovo-cel) is a gene therapy that modifies a patient’s hematopoietic stem cells with the addition of functional copies of a modified β-globin gene (βA-T87Q-globin gene). The resulting cells are designed to reduce RBC sickling and hemolysis.

The single-arm, phase 1/2 HGB-206 trial (NCT02140554) evaluated the efficacy and safety of lovo-cel in patients 12 to 50 years of age with severe SCD. The study included patients with ≥ 4 severe VOEs in the 24 months prior to enrollment (cohort C). After receiving 1 dose of lovo-cel, 31 patients (96%) had complete resolution of severe VOEs (VOE requiring ED or hospital visit or priapism requiring medical facility visit; primary endpoint) through 24 months of follow-up. The safety profile in the 24 months following lovo-cel was generally consistent with busulfan conditioning. The most common TEAEs were pain, anemia, opiate withdrawal, nausea, suicidality, and vomiting. Graft failure, insertional oncogenesis, and replication-competent lentivirus were not observed.

The treatment process for lovo-cel involved the following steps: pre-collection preparation, cell mobilization, and apheresis. This was followed by ex vivo transduction of autologous HSPCs with the BB305 lentiviral vector encoding the βA-T87Q transgene. Patients then received myeloablative conditioning followed by IV infusion of a single dose of lovo-cel.

FDA APPROVAL TIMELINE December 20, 2023  Fast Track

 Orphan Drug

 Priority Review

 RPD

 RMAT

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$0

$51

$92

$140

$176

PLACE IN THERAPY

Sickle Cell Disease An estimated 100,000 people in the US are diagnosed with SCD, with the highest prevalence in African Americans. The hereditary condition is characterized by sticky, sickle-shaped RBCs. The abnormal RBCs cause chronic anemia, vaso-occlusion, and eventual multiorgan damage. VOEs (also called vaso-occlusive crises [VOC]) occur when the microcirculation is obstructed by sickled RBC, leading to ischemic injury and pain. VOEs are the most common manifestation of SCD and the most common reason for ED visits. The life expectancy of people with SCD is reduced by ≥ 20 years compared to the general population.

Pharmacologic agents approved for use in patients with SCD include the oral antimetabolite hydroxyurea, the oral amino acid L-glutamine (Endari®), and the IV selectin blocker crizanlizumab-tmca (Adakveo®). In clinical trials, these agents led to a reduction in VOEs. Hydroxyurea reduces the need for blood transfusion, but is associated with leukemogenic and carcinogenic toxicities. The oral hemoglobin S (HbS) inhibitor voxelotor (Oxbryta™) is also approved for SCD. In clinical trials, it increased Hb levels, but did not have a significant impact on VOEs. The only curative treatment for SCD is allogeneic bone marrow transplantation; however, its use is limited by associated risks and lack of matched donors.

The gene therapies, exa-cel and lovo-cel, treat the underlying cause of SCD using a patient’s own hematopoietic stem cells that have been genetically modified. Exa-cel achieves this through CRISPR-Cas-9 technology to produce RBCs with high levels of HbF, while lovo-cel’s process adds functional copies of a modified β-globin gene (βA-T87Q-globin gene). If approved, they will be the first gene therapies to treat SCD. Both have demonstrated safety and efficacy for SCD, based on VOE reduction, in patients ≥ 12 years of age. Ongoing clinical trials are also assessing exa-cel and lovo-cel in patients as young as 2 years of age with SCD. 9 | MAGELLANRX.COM

lovotibeglogene autotemcel (cont.)

PLACE IN THERAPY (cont.)

β-thalassemia β-thalassemia is a chronic, autosomal recessive disorder caused by a mutation in one or both hemoglobin beta (HBB) genes. Symptomatic cases are estimated to occur in approximately 1 in 100,000 individuals in the general population. β-thalassemia is reported most often in populations in the Mediterranean, Africa, India, and Asia, with prevalence reported as high as 10%. The condition is characterized by a reduction in Hb and RBC levels, resulting in anemia. Other complications include iron overload, skeletal changes, and functional changes in the heart, liver, gall bladder, and spleen.

TDT is a severe form of β-thalassemia that requires the use of RBC transfusions (every 2 to 4 weeks) and iron chelation therapy to manage anemia and associated complications. Splenectomy may decrease transfusion requirements in select patients. Allogeneic HSCT may be curative in some cases but requires a well-matched donor (~60% of TDT patients do not have a suitable sibling donor) and carries risks of graft rejection and GVHD. Two pharmacologic treatments are FDA-approved to treat anemia in patients with TDT. They include the erythroid maturation agent luspatercept-aamt (Reblozyl®), which is given by SC injection every 3 weeks by an HCP, and the single-dose IV gene therapy betibeglogene autotemcel (beti-cel; Zynteglo®). In clinical trials, both treatments significantly reduced the need for RBC transfusions in patients with TDT. If approved, exa-cel will be the second gene therapy approved to treat TDT. Using the patient’s own genetically modified hematopoietic stem cells, beti-cel and exa-cel target the underlying cause of β-thalassemia and have demonstrated safety and efficacy in patients ≥ 12 years of age with a single IV dose. In clinical trials, exa-cel led to freedom from transfusions for ≥ 12 months in patients with TDT. An ongoing clinical trial is also assessing exa-cel in patients ages 2 to 11 years with TDT.

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ONCOLOGY

fruquintinib oral Takeda/Hutchmed PROPOSED INDICATIONS

Refractory metastatic colorectal cancer (mCRC) as 2nd-line or later therapy

CLINICAL OVERVIEW

Fruquintinib inhibits vascular endothelial growth factor receptors (VEGFR) -1, -2 and -3, which impedes new blood vessel growth (angiogenesis) of malignant tumors.

The multicenter, double-blind, phase 3 FRESCO-2 (NCT04322539) study evaluated the efficacy and safety of fruquintinib in adults with refractory mCRC who had progressed on or were intolerant to trifluridine/ tipiracil and/or regorafenib. Patients also had prior treatment with standard therapies (e.g., chemotherapy [2 lines], biological VEGF inhibitor, EGFR inhibitor [if RAS wild-type], immune checkpoint inhibitor [if MSI-H/ dMMR], BRAF inhibitor [if V600E mutated]). A total of 691 patients were randomized 2:1 to fruquintinib or placebo in addition to best supportive care (BSC). After a median follow-up of 11 months, the primary endpoint of OS was significantly longer in patients treated with fruquintinib than placebo (median, 7.4 versus 4.8 months, respectively; HR, 0.66; p<0.001). The PFS was greater with fruquintinib compared to placebo (median, 3.7 versus 1.8 months, respectively; HR, 0.32; p<0.001) as was the DCR (median, 55.5% versus 16.1%, respectively). Results were consistent regardless of prior therapy. Grade 3 or 4 TEAEs were reported in 62.7% of patients in the fruquintinib arm and 50.4% in the placebo arm, with the most common being hypertension, asthenia, and hand-foot syndrome.

Fruquintinib 5 mg orally once daily was given on days 1 through 21 of each 28-day cycle.

PLACE IN THERAPY

CRC is the fourth most frequently diagnosed cancer and the second leading cause of cancer death in the US. It is predicted that in 2023, approximately 153,000 new cases of colorectal cancer and 52,550 associated deaths will occur in the US. Risk factors for CRC include inflammatory bowel disease, smoking, diabetes, obesity, low levels of physical activity, alcohol consumption, and diet.

In patients with mCRC, intensive chemotherapy with 5-FU/leucovorin or capecitabine, with oxaliplatin and/ or irinotecan, is the standard for 1st- and 2nd-lines of therapy. The addition of targeted therapies, such as injectable antibodies and oral kinase inhibitors is also typical. These agents target markers such as VEGF, EGFR, microsatellite instability (MSI), HER2 overexpression, and BRAF mutations. For mCRC that is refractory to chemotherapy, trifluridine/tipiracil (Lonsurf®) or regorafenib (Stivarga®) ± bevacizumab is recommended.

Fruquintinib is a selective kinase inhibitor that targets VEGFR-1, -2, and -3, thereby diminishing angiogenesis that may lead to tumor metastasis. If approved, it will likely compete with trifluridine/tipiracil or regorafenib in patients with refractory mCRC. In non-comparative clinical trials, the median OS of fruquintinib, trifluridine/ tipiracil, and regorafenib were 7.4 months, 7.1 months, and 6.4 months, respectively. Notably the fruquintinib clinical trial included patients previously treated with trifluridine/tipiracil or regorafenib. In addition, fruquintinib displays a more favorable safety profile than the other 2 agents. The TEAEs associated with fruquintinib are similar to regorafenib but occurred at a lower incidence, and trifluridine/tipiracil is associated with neutropenia, anemia, and thrombocytopenia, which have not been reported with fruquintinib.

FDA APPROVAL TIMELINE November 30, 2023  Fast Track

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$17

$44

$69

$101

$123

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ONCOLOGY

lifileucel IV Iovance PROPOSED INDICATIONS

Advanced unresectable or metastatic melanoma that has progressed after anti-PD-1/PD-L1 therapy and targeted therapy

CLINICAL OVERVIEW

Lifileucel is an autologous tumor infiltrating lymphocyte (TIL) therapy. It uses T cells extracted from a tumor and expanded ex vivo using a centralized manufacturing process. The T cells are engineered to recognize tumor antigens and are then infused back into the patient.

A multicenter, single-arm, open-label, phase 2 trial (NCT02360579) evaluated lifileucel in 66 patients with advanced unresectable or metastatic melanoma (stage IIIC or IV) that progressed after anti-PD-1/PD-L1 and BRAF ± MEK targeted agents (if BRAF V600 mutation-positive). Patients had received a mean of 3.3 (range, 1 to 9) prior lines of treatment. In the study, patients received a nonmyeloablative lymphodepletion regimen (cyclophosphamide, fludarabine), followed by a single infusion of lifileucel, and up to 6 doses of high-dose interleukin-2. The ORR (primary endpoint) was 36%, with a 3% complete response rate. The DCR was 80%, and after a median 18.7 months of follow-up, the median DOR was not reached; however, the 1-year DOR was 69%. In addition, the median OS was 17.4 months. The best response was reached in a median of 1.4 months after the lifileucel dose. No difference in response was seen based on age, prior anti-CTLA-4 use, BRAF mutation, or PD-1/PD-L1 status. The most common grade 3 or 4 TEAEs were thrombocytopenia (82%), anemia (56%), febrile neutropenia (55%), neutropenia (39%), hypophosphatemia (35%), leukopenia (35%), and lymphopenia (32%).

Lifileucel was administered as a single IV infusion of 1 x 109 to 150 x 109 cells.

PLACE IN THERAPY

While melanoma accounts for about 1% of all cases of skin cancers in the US, it causes the most deaths from skin cancer. It is estimated that nearly 8,000 deaths from melanoma will occur in the US in 2023. Regional disease (stage III) is present in approximately 9% of patients at diagnosis, which is associated with a 71% 5-year survival rate. Approximately 4% of patients have distant metastatic disease (stage IV) upon initial diagnosis, which has an estimated 5-year survival rate of 32%. Tumor thickness, ulceration, and mitotic rate are the most important characteristics to predict treatment outcome. Tumor-infiltrating lymphocytes (TIL) may also help predict clinical outcome.

For 1st-line and subsequent systemic therapies for metastatic or unresectable melanoma, the NCCN recommends an anti-PD-1/PD-L1 antibody; preferred agents include nivolumab (Opdivo®) ± ipilimumab (Yervoy®), nivolumab/relatlimab-rmbw (Opdualag™), or pembrolizumab (Keytruda®) monotherapy. In patients with BRAF V600 mutations, combination therapy with a BRAF inhibitor plus an MEK inhibitor is recommended; regimens include dabrafenib (Tafinlar®)/trametinib (Mekinist®), vemurafenib (Zelboraf®)/cobimetinib (Cotellic®), and encorafenib (Braftovi®)/binimetinib (Mektovi®). For 2nd-line or subsequent therapy, targeted agents may be considered based on mutation status (KIT mutations, ROS1 fusions, NTRK fusions, NRAS mutations).

Lifileucel is a first-in-class TIL therapy. If approved, it will be the only autologous tumor-specific treatment for patients with advanced or metastatic melanoma who have progressed on or after SOC therapies.

FDA APPROVAL TIMELINE November 25, 2023  Fast Track

 Orphan Drug

 Priority Review

 RMAT

 seeking Accelerated Approval

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$1

$119

$264

$489

$693

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ONCOLOGY

motixafortide SC Biolinerx PROPOSED INDICATIONS

Stem cell mobilization for autologous transplantation in patients with multiple myeloma (MM)

CLINICAL OVERVIEW

Motixafortide is a selective cyclic-peptide inhibitor of the C-X-C motif chemokine receptor 4 (CXCR4) with high affinity (Ki, 0.32 nM), long receptor occupancy, and extended clinical activity (> 48 hours).

The international, double-blind, phase 3 GENESIS (NCT03246529) trial evaluated the safety and efficacy of adding motixafortide to G-CSF therapy in patients with MM undergoing HSPC mobilization prior to autologous HSCT. The median age was 63 years and 70% of patients received lenalidomide-containing induction therapy.

Following initiation of G-CSF, randomized patients received either motixafortide (n=80) or placebo (n=42) added to G-CSF. The study demonstrated that significantly more patients in the motixafortide group met the primary endpoint of collecting ≥ 6 x 106 CD34+ cells/kg within 2 apheresis days compared to the placebo group (92.5% versus 26.2%, respectively; OR, 53.3; p<0.0001). Similarly, more patients in the motixafortide group achieved ≥ 6 x 106 CD34+ cells/kg within 1 apheresis day (88.8% versus 9.5%, respectively; OR, 118; p<0.0001). As a comparison, in a prospectively enrolled, demographically similar, contemporaneous cohort that included patients (n=14) who were mobilized with plerixafor plus G-CSF, 50% of patients achieved this amount with 1 apheresis day. The median number of CD34+ cells/kg HSPCs mobilized in 1 apheresis day was 10.8 x 106 with motixafortide, 2.25 x 106 with placebo, and 5.47 x 106 with plerixafor. The mean number of apheresis procedures was 1.23 in the motixafortide group and 3.24 in the placebo group (p<0.0001).

In addition, patients in the motixafortide group received 5.26 G-CSF injections compared to 8.12 G-CSF injections in the placebo group (p<0.0001). The median time to neutrophil engraftment, platelet engraftment, graft durability, PFS, and OS were similar between the motixafortide and placebo groups. Compared with plerixafor, motixafortide significantly increased the number of multipotent progenitors (MPPs), common myeloid progenitors (CMPs), common lymphoid progenitors (CLPs), and basophil precursors (BPs) when added to G-CSF. The most common TEAEs reported with motixafortide were mild to moderate local injection site reactions, flushing, pruritis, urticaria, and erythema. Bone pain was reported less often with motixafortide than placebo (18% versus 31%, respectively).

All patients received G-CSF 10 mcg/kg on days 1 through 5 (and 6 through 8, as needed). Motixafortide 1.25 mg/kg was administered SC on day 4 (and day 6, if needed). In the contemporaneous cohort, plerixafor was administered SC at a dose of 0.24 mg/kg on day 4 (and 5 through 7, if needed). Apheresis was performed on day 5 (and 6 through 8, if needed).

PLACE IN THERAPY

MM is a malignant neoplasm of plasma cells that accumulate in bone marrow, leading to destruction and failure of the bone marrow. It is estimated that 35,730 new cases of MM and 12,590 deaths due to MM will occur in the US in 2023. Patients are typically diagnosed with MM between 65 and 74 years of age. The 5-year relative survival rate is 79% for localized disease and 57% for distant disease.

High-dose chemotherapy (e.g., cyclophosphamide, etoposide, doxorubicin, bendamustine), often combined with corticosteroids and immunomodulating agents, followed by autologous HSCT is used to treat MM. In preparing for HSCT, G-CSF therapy is administered to increase (mobilize) peripheral blood progenitor cells (PBPCs) to a minimum of 2 to 5 x 106 CD34 cells/kg for autologous donors; however, in up to 35% of patients, G-CSF alone does not lead to an adequate PBPC level, particularly in those with advanced age, extensive prior therapy, prior bone marrow radiation, and WBC count < 4,000. The addition of the CXCR4 inhibitor plerixafor has been shown to improve PBPC collection yield.

If approved, motixafortide will be the second CXCR4 inhibitor in the US, competing with plerixafor (Mozobil®) for PBPC mobilization in patients with MM. In clinical trials, motixafortide was well tolerated and demonstrated a higher success rate in PBPC collection of ≥ 6 x 106 CD34+ cells/kg compared to placebo and plerixafor (contemporaneous cohort). Motixafortide produced rapid and durable engraftment and led to significantly fewer doses of G-CSF and number of apheresis days required compared to placebo. Notably, several generics for plerixafor were approved in July 2023 and could launch at any time.

13 | MAGELLANRX.COM

motixafortide (cont.)

FDA APPROVAL TIMELINE September 9, 2023  Orphan Drug

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$4

$10

$14

$20

$27

14 | MAGELLANRX.COM

IMMUNOLOGY

pozelimab IV, SC Regeneron PROPOSED INDICATIONS

CD55 deficiency with hyper-activation of complement, angiopathic thrombosis, and severe protein-losing enteropathy (CHAPLE) syndrome

CLINICAL OVERVIEW

Pozelimab is an IgG4 antibody that inhibits complement factor C5.

A multicenter, open-label, single-arm, phase 2/3 trial (NCT04209634) evaluated the safety and efficacy of pozelimab in 10 patients with CD55-mutation confirmed CHAPLE syndrome. Enrolled patients were ≥ 1 year of age. At 24 weeks, all 10 patients achieved the co-primary endpoints of normalization of serum albumin, a marker of disease improvement, and improvement or stabilization of clinical symptoms (e.g., abdominal pain, number of daily bowel movements, and edema). The trial also revealed marked reductions in hospitalization days and albumin transfusions, as well as meaningful increases in body weight and growth.

Seven patients experienced mild to moderate adverse events, including iron deficiency, pyrexia, rhinitis, urticaria, and vomiting (n=2 for each).

Pozelimab was administered as a single 30 mg/kg IV loading dose on day 1, followed by weekly SC weightbased doses.

PLACE IN THERAPY

CHAPLE syndrome is a form of primary intestinal lymphangiectasia (also known as Waldmann’s disease).

The ultra-rare disease (< 100 known cases worldwide) is caused by a defect in the CD55 gene, leading to a deficiency of the CD55 protein in the immune regulating complement pathway. A lack of CD55 results in overactivity of the complement pathway that in turn leads to blood and lymph vessel damage in the GI tract and loss of immune proteins and blood cells. CHAPLE syndrome is associated with severe protein-losing enteropathy, abdominal pain, bloody diarrhea, vomiting, malabsorption, hypoproteinemia, delayed growth, edema, and recurrent infections. Life-threatening infections and thrombosis may also occur.

There is currently no treatment approved for CHAPLE syndrome. Off-label use of the complement inhibitor eculizumab (Soliris®) corrects GI protein loss in patients with CHAPLE syndrome; however, it appears that there are no studies evaluating eculizumab for CHAPLE syndrome in the US. If approved, pozelimab will be the only agent indicated for the treatment of CHAPLE syndrome. In the clinical trial, it demonstrated rapid and sustained clinical improvement (up to 24 weeks) and few side effects with weekly SC maintenance dosing.

Pozelimab is also in phase 3 development for paroxysmal nocturnal hemoglobinuria (PNH).

FDA APPROVAL TIMELINE August 20, 2023  Fast Track

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions)

 RPD

The financial forecast for pozelimab is not currently available.

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MUSCULOSKELETAL

vamorolone oral Santhera PROPOSED INDICATIONS

Duchenne muscular dystrophy (DMD)

CLINICAL OVERVIEW

Vamorolone is a dissociative steroid that binds to the glucocorticosteroid receptor.

The double-blind, placebo-controlled, phase 2b VISION-DMD trial (NCT03439670) evaluated the safety and efficacy of vamorolone in boys 4 to < 7 years of age with DMD who were not previously treated with corticosteroids. A total of 121 patients were randomized 1:1:1:1 to vamorolone 2 or 6 mg/kg/day, prednisone 0.75 mg/kg/day, or placebo. A total of 114 patients completed the initial 24-week treatment period of this 2-part study. At 24 weeks, the study met the time to stand (TTSTAND) velocity endpoints with vamorolone 6 mg compared to placebo (LS mean, 0.05 versus -0.01 m/s, respectively; p=0.002), a primary endpoint, and with vamorolone 2 mg (LS mean difference compared to placebo, 0.05 m/s; p=0.02), a secondary endpoint. The vamorolone 6 mg dose displayed a shorter time to onset of activity than vamorolone 2 mg (approximately < 1 week versus 6 weeks, respectively). At 24 weeks, both doses also met the 6MWT endpoint and the vamorolone 6 mg dose met the time to run/walk 10 meters (TTRW) endpoint. Height percentile increased in patients treated with vamorolone 6 mg and 2 mg, but decreased among those treated with prednisone (3.86 and 0.26 versus -1.88, respectively); the difference was significant between the vamorolone 6 mg dose but not the 2 mg dose when each were compared to prednisone (p=0.02 and p>0.05, respectively). The vamorolone and prednisone treatment groups showed similar overall increase in BMI. Interim data from an 18-month, non-randomized, open-label phase 2a trial (NCT02760277) reported fewer adverse effects (e.g., cushingoid appearance, hirsutism, weight gain, behavior change) with vamorolone than have been reported for treatment with prednisone and deflazacort. In addition, vamorolone did not lead to stunting of growth that is seen with deflazacort or prednisone.

PLACE IN THERAPY

DMD is a rare, X-linked neuromuscular disorder characterized by progressive muscle degeneration and weakness. An estimated 400 to 600 boys are born with DMD each year in the US. In DMD, gene mutations lead to a lack of functional dystrophin protein involved in maintaining muscle fiber integrity. Onset of signs and symptoms of DMD occurs between 3 to 5 years of age. Most boys affected lose the ability to walk by age 12. Moreover, death due to respiratory or cardiac failure typically occurs by the early 30s. Select corticosteroids (prednisone) have been used historically to treat DMD and deflazacort (Emflaza®) is the only steroid FDA-approved for DMD. Corticosteroids may be used in combination with other agents for DMD, including casimersen (Amondys 45), eteplirsen (Exondys 51), golodirsen (Vyondys 53), and viltolarsen (Viltepso®). While corticosteroids have been a SOC in DMD to delay progression of muscle weakness and improve respiratory function, their use is associated with side effects such as weight gain, slowed growth trajectories, bone fractures, and cataracts. Recently, Sarepta’s delandistrogene moxeparvovec-rokl (Elevydis) became the first gene therapy approved, via Accelerated Approval, to treat DMD. If approved, oral vamorolone will provide an additional steroid option for the treatment of DMD. In clinical trials, it led to improvement over placebo in key mobility endpoints and had an improved safety profile (including growth trajectory) compared to prednisone. Vamorolone has the potential to replace other steroids, including deflazacort, as SOC for DMD based on an improved safety profile and potential for dosing flexibility.

FDA APPROVAL TIMELINE October 26, 2023  Fast Track

 Orphan Drug

 RPD

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$7

$70

$117

$237

$308

16 | MAGELLANRX.COM

IMMUNOLOGY

zilucoplan SC UCB PROPOSED INDICATIONS

Myasthenia gravis (MG) that is acetylcholine receptor antibody positive (AChR-Ab+)

CLINICAL OVERVIEW

Zilucoplan is a complement C5 inhibitor.

The double-blind, phase 3 RAISE trial (NCT04115293) evaluated zilucoplan in 174 adults with AChR+ generalized myasthenia gravis (gMG). Enrollees had a Myasthenia Gravis Activities of Daily Living (MG-ADL) score ≥ 6.

Participants were randomized 1:1 to zilucoplan or placebo. At week 12, a greater reduction in the primary endpoint of MG-ADL score from baseline was reported in patients who received zilucoplan compared with those who received placebo (LS mean change, -4.39 versus -2.3, respectively; LS mean difference, -2.09; p=0.0004).

In addition, more patients in the zilucoplan arm achieved a clinically meaningful (≥ 3-point) reduction in the MG-ADL score without the need for rescue therapy, compared with those in the placebo arm (73% versus 46%, respectively; p=0.0005). Greater improvement from baseline in the Myasthenia Gravis Quality of Life (MG-QOL) score was also reported with zilucoplan compared to placebo (-5.65 versus -3.16; p=0.013). The most common TEAE reported with zilucoplan was injection site bruising. Interim data from the ongoing RAISE-XT open-label extension study (NCT04225871) reported continued long-term safety and response through extension week 12 that were maintained through extension week 48.

Zilucoplan was administered as 0.3 mg/kg SC once daily by self-injection.

PLACE IN THERAPY

MG is an autoimmune neuromuscular disorder that affects an estimated 37 per 100,000 people in the US. The mean age at onset is 28 years in females and 42 years in males. This chronic condition is characterized by muscle weakness and fatigue and affects muscles of the arms, legs, neck, face, and eyes. A reported 15% to 20% of patients will experience an MG crisis that may result in respiratory failure requiring emergency care. Anti-AChR antibodies are identified in approximately 85% of patients with gMG and lead to disruption in neuromuscular signaling, primarily through activation of the classic complement pathway.

There is no cure for MG. Plasmapheresis and IV immunoglobulins have provided benefit in severe cases.

Thymectomy may also significantly improve MG symptoms. Pharmacotherapy includes 1st-line use of anticholinesterase inhibitors (e.g., pyridostigmine) followed by immunosuppressants (e.g., corticosteroids, azathioprine, cyclosporine, mycophenolate mofetil, tacrolimus). In recent years, IV-administered biologics have been approved to treat gMG in AChR-Ab+ adults. They include the IV and SC formulations of the neonatal FcRn blocker efgartigimod alfa (Vyvgart®; Vyvgart® Hytrulo), the SC neonatal FcRn blocker rozanolixizumabnoli (Rystiggo®), and the IV complement C5 inhibitors eculizumab (Soliris) and ravulizumab-cwvz (Ultomiris®).

If approved, zilucoplan will likely compete directly with the complement C5 inhibitors. In non-comparative trials, zilucoplan resulted in a greater reduction in the MG-ADL score compared to eculizumab and ravulizumabcwvz (-2.09 versus -1.9 and -1.6, respectively). Zilucoplan may provide a more preferable self-administered SC once-daily regimen, compared to the other complement inhibitors which require IV administration by an HCP at every 2-, 4-, or 8-week maintenance intervals, depending on the product. Eculizumab and ravulizumabcwvz carry boxed warnings for serious meningococcal infections and are available only through a REMS program. No cases of meningococcal infections were reported with zilucoplan in the RAISE trial, which required meningococcal vaccination for all participants. It remains to be seen if zilucoplan will also carry this warning and limited access via REMS.

FDA APPROVAL TIMELINE October to December 2023  Orphan Drug

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$17

$117

$242

$346

$429

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Biosimilar Overview CLINICAL OVERVIEW

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

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

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

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

Most states have enacted biosimilar substitution laws. An interchangeable product may be substituted for the originator at the pharmacy without the involvement of the prescriber. In December 2022, the FDA announced a pilot regulatory science program that focuses on advancing the development of interchangeable products and improving the efficiency of biosimilar product development. The Purple Book is an FDA database of licensed biological products that lists biosimilar and interchangeable products. The FDA has approved 4 biosimilars for interchangeability to their reference product: insulin glargine-yfgn (Semglee®), insulin glargineaglr (Rezvoglar™), adalimumab-adbm (Cyltezo®), and ranibizumab-eqrn (Cimerli™).

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

PLACE IN THERAPY

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

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

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BIOSIMILAR OVERVIEW continued To date, a total of 41 biosimilars have received FDA approval. Of these, 37 have entered the market.

APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Zaxio® (filgrastim-sndz)

Sandoz

March 2015

Inflectra® (infliximab-dyyb)

Pfizer

April 2016

Erelzi® (etanercept-szzs)

Sandoz

August 2016

Amjevita™* (adalimumab-atto)

Amgen

September 2016

Renflexis® (infliximab-abda)

Merck/Organon

May 2017

Cyltezo* (adalimumab-adbm)

Boehringer Ingelheim

August 2017

Mvasi® (bevacizumab-awwb)

Amgen

September 2017

Ixifi™ ‡ (infliximab-qbtx)

Pfizer

December 2017

Ogivri® (trastuzumab-dkst)

Mylan/Biocon

December 2017

Retacrit® (epoetin alfa-epbx)

Pfizer/Vifor/ Hospira

May 2018

Fulphila® (pegfilgrastim-jmdb)

Mylan/Biocon

June 2018

Nivestym® (filgrastim-aafi)

Pfizer

July 2018

Hyrimoz®* (adalimumab-adaz)

Sandoz

October 2018

Udenyca (pegfilgrastim-cbqv)

Coherus

November 2018

Truxima® (rituximab-abbs)

Cephalon/Teva

November 2018

Herzuma® (trastuzumab-pkrb)

Teva

December 2018

Ontruzant® (trastuzumab-dttb)

Merck

January 2019

Trazimera® (trastuzumab-qyyp)

Pfizer

March 2019

Eticovo™ (etanercept-ykro)

Samsung Bioepis

April 2019

Kanjinti® (trastuzumab-anns)

Amgen

June 2019

Zirabev® (bevacizumab-bvzr)

Pfizer

June 2019

Hadlima™* (adalimumab-bwwd)

Organon

July 2019

Ruxience® (rituximab-pvvr)

Pfizer

July 2019

Abrilada™* (adalimumab-afzb)

Pfizer

November 2019

®

19 | MAGELLANRX.COM

Interchangeable -

 -

Commercially Available

  -

    -

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

    

Herceptin® (Genentech)

    

Neulasta (Amgen)

(only HCF)

-

    -

Epogen® (Amgen) Procrit® (Janssen) Neulasta (Amgen) Neupogen (Amgen) Humira (Abbvie)

Rituxan® (Genentech) Herceptin (Genentech) Herceptin (Genentech) Herceptin (Genentech) Enbrel (Amgen) Herceptin (Genentech) Avastin (Genentech) Humira (Abbvie) Rituxan (Genentech) Humira (Abbvie)

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

Manufacturer

Approval Date

Ziextenzo® (pegfilgrastim-bmez)

Sandoz

November 2019

Avsola® (infliximab-axxq)

Amgen

December 2019

Nyvepria™ (pegfiltrastim-apgf)

Pfizer

June 2020

Semglee (insulin glargine-yfgn)

Mylan/Biocon

June 2020

Hulio®* (adalimumab-fkjp)

Mylan/Biocon

July 2020

Riabni™ (rituximab-arrx)

Amgen

December 2020

Byooviz™ (ranibizumab-nuna)

Biogen

September 2021

Rezvoglar™ (insulin glargine-aglr)

Eli Lilly

December 2021

Yusimry™* (adalimumab-aqvh)

Coherus

December 2021

Releuko® (filgrastim-ayow)

Amneal

March 2022

Alymsys® (bevacizumab-maly)

Amneal

April 2022

Fylnetra® (pegfilgrastim-pbbk)

Amneal

May 2022

Cimerli (ranibizumab-eqrn)

Coherus

August 2022

Stimufend® (pegfilgrastim-fpgk)

Fresenius Kabi

September 2022

Vegzelma® (bevacizumab-adcd)

Celltrion

September 2022

Idacio® (adalimumab-aacf)

Fresenius Kabi

December 2022

Yuflyma®* (adalimumab-aaty)

Celltrion

May 2023

Interchangeable -

 -

 -

 -

Commercially Available

                

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

* Abbvie’s adalimumab (Humira), adalimumab-adaz (Hyrimoz), and adalimumab-bwwd (Hadlima) are approved in 50 mg/mL (with citric acid/ citrate) and 100 mg/mL (citrate-free) concentrations. Adalimumab-aaty (Yuflyma) is only approved as a 100 mg/mL high concentration citratefree formulation. All other biosimilars for Humira are approved as 50 mg/mL concentrations only. † Pfizer’s Inflectra is marketed in the US; Pfizer has not announced plans to launch Ixifi.

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

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

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

The first biosimilar version of Abbvie’s adalimumab (Humira), adalimumab-atto (Amjevita) became available in the US in January 2023. Several Humira biosimilars, including citrate-free and high-concentration formulations as well as an interchangeable product, launched in July 2023.

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BIOSIMILAR OVERVIEW continued

IMMUNOLOGY

adalimumab SC Pfizer is seeking interchangeability of FDA-approved adalimumab-afzb (Abrilada) 50 mg/mL biosimilar to Abbvie’s 50 mg/mL Humira. Celltrion is also seeking interchangeability for their FDA-approved adalimumab-aaty (Yuflyma) 100 mg/mL, a biosimilar to Abbvie’s citrate-free, high-concentration (100 mg/mL) Humira. Abbvie’s Humira is a tumor necrosis factor alpha (TNF-α) blocker indicated for the treatment of autoimmune disorders, including rheumatoid arthritis (RA), juvenile idiopathic arthritis (JIA), ankylosing spondylitis (AS), plaque psoriasis (PSO), psoriatic arthritis (PsA), Crohn’s disease (CD) in adults and children, ulcerative colitis (UC), hidradenitis suppurativa (HS), and noninfectious uveitis.

FDA APPROVAL TIMELINE

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

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$11,900

$7,164

$5,480

$4,435

$3,744

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

OPHTHALMOLOGY

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

FDA APPROVAL TIMELINE

• Biocon/Janssen (MYL-1701) – Pending • Celltrion (CT-P42) – June 28, 2024 • Coherus (FYB-203) – June 28, 2024

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$6,266

$6,352

$5,961

$5,271

$4,819

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

21 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

ONCOLOGY

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

FDA APPROVAL TIMELINE

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

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$505

$431

$388

$350

$316

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

ENDOCRINE

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

FDA APPROVAL TIMELINE December 6, 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$4,253

$4,486

$4,578

$3,973

$3,181

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

22 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

IMMUNOLOGY

eculizumab IV Amgen Amgen is seeking approval for their investigational biosimilar (Bekemv) to Alexion’s complement inhibitor eculizumab (Soliris) that is indicated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).

FDA APPROVAL TIMELINE February 2024

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$1,779

$1,402

$1,113

$745

$548

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

BLOOD MODIFIER

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

FDA APPROVAL TIMELINE Pending

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$50

$41

$34

$28

$24

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

23 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

ENDOCRINOLOGY

insulin aspart SC Gan & Lee/Sandoz Gan & Lee/Sandoz are seeking approval for their investigational biosimilar (GL-ASP) to Novo Nordisk’s rapid-acting human insulin analog insulin aspart (Novolog®) that is indicated to improve glycemic control in patients with diabetes mellitus.

FDA APPROVAL TIMELINE April 14, 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$723

$670

$626

$594

$565

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

ENDOCRINOLOGY

insulin glargine SC Gan & Lee/Sandoz Gan & Lee/Sandoz are seeking approval for their investigational biosimilar (Basalin) to Sanofi-Aventis’ long-acting human insulin analog insulin glargine (Lantus) that is indicated to improve glycemic control in patients with diabetes mellitus.

FDA APPROVAL TIMELINE December 23, 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$590

$512

$457

$411

$370

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

24 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

ENDOCRINOLOGY

insulin lispro SC Gan & Lee/Sandoz Gan & Lee/Sandoz are seeking approval for their investigational biosimilar (Prandilin) to Eli Lilly’s rapid-acting human insulin analog insulin lispro (Humalog) that is indicated to improve glycemic control in patients with diabetes mellitus.

FDA APPROVAL TIMELINE April 1, 2024

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$1,032

$850

$781

$723

$672

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

NEUROLOGY/IMMUNOLOGY

natalizumab IV Polypharma/Sandoz

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

FDA APPROVAL TIMELINE Pending

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$1,113

$1,055

$1,039

$1,029

$952

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

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BIOSIMILAR OVERVIEW continued

BLOOD MODIFIER

pegfilgrastim SC Apotex and Lupin are seeking approval for their investigational biosimilars to Amgen’s Neulasta and Coherus is seeking approval for their investigational biosimilar to Amgen’s Neulasta Onpro. Neulasta and Neulasta Onpro contain pegfilgrastim, 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 • Coherus (Udenyca OBI®) – October 2023 • Lupin (Lupifil-P) – Pending

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales Neulasta

$622

$420

$347

$283

$234

Projected Total US Sales Neulasta Onpro

$2,972

$2,576

$2,390

$2,084

$1,833

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

OPHTHALMOLOGY

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

FDA APPROVAL TIMELINE April 21, 2024

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$718

$533

$408

$335

$303

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

26 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

IMMUNOLOGY/ONCOLOGY

rituximab IV Dr. Reddy’s

Dr. Reddy’s is seeking approval for their investigational biosimilar to Genentech’s Rituxan, a CD20-directed cytolytic antibody indicated for the treatment of select patients with non-Hodgkin’s lymphoma (NHL), mature B-cell NHL/ mature B-cell acute leukemia, chronic lymphocytic leukemia (CLL), rheumatoid arthritis (RA), granulomatosis with polyangiitis (GPA)/microscopic polyangiitis (MPA), and pemphigus vulgaris.

FDA APPROVAL TIMELINE July 12, 2024

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$1,133

$919

$766

$685

$611

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

IMMUNOLOGY

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

FDA APPROVAL TIMELINE

• Biogen/Bio-Thera Solutions (BIIB800) – October 9, 2023 • Fresenius Kabi (MSB11456) – Pending

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$1,088

$861

$709

$603

$528

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

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BIOSIMILAR OVERVIEW continued

ONCOLOGY

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

FDA APPROVAL TIMELINE December 15, 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$388

$335

$300

$269

$236

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

IMMUNOLOGY

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

FDA APPROVAL TIMELINE July to December 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$6,036

$4,149

$2,954

$2,243

$1,779

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

28 | MAGELLANRX.COM

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

troriluzole

aficamten

Gastrointestinal

Cardiovascular

$670

$872

Neurology

blarcamesine Neurology

$77

$1,049

tirzepatide (Mounjaro™)

datopotamab deruxtecan

Weight management

Oncology

$4,572

$1,090

tabelecleucel

debamestrocel

$401

$426

Oncology

Neurology

donanemab

sotatercept

Neurology

Cardiology

$1,090

$1,434

EB-101

resmetirom

Dermatology/Gene therapy

Cardiovascular

$129

$1,301

fidanacogene elaparvovec

reproxalap

Hematology/Gene therapy

Ophthalmology

$464

relacorilant Endocrine

$588

giroctocogene fitelparvovec

$187

Hematology/Gene therapy

$131

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

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

APPLICATION APPLICATION SUBMITTED SUBMITTED TO THE FDA

PHASE IN PHASE 33 TRIALS TRIALS

74%

62%

26%

38%

36%

35%

17%

Specialty

17%

15%

19%

7%

Traditional

Orphan Drug

Priority Review

Breakthrough Therapy

Biosimilar

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

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

NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

Submitted (New Drugs) naloxone spray

Harm Reduction Therapeutics

Opioid overdose

Intranasal

Submitted − 505(b)(2) NDA for OTC; Priority Review

July 2023

toripalimab

Coherus

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

IV

Submitted − BLA; Breakthrough Therapy; Orphan Drug

Jul-Sep 2023

donanemab

Eli Lilly

Alzheimer's disease (early)

IV, SC

Submitted − BLA; Breakthrough Therapy

Jul-Dec 2023

etrasimod

Pfizer

UC (moderate to severe)

Oral

Submitted − NDA

Jul-Dec 2023

tislelizumab

Beigene/Novartis

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

IV

Submitted − BLA; Orphan Drug

Jul-Dec 2023

ustekinumab (biosimilar to Janssen’s Stelara)

Alvotech

CD; UC; PSO; PsA

SC

Submitted − BLA

Jul-Dec 2023

risperidone (once monthly)

Laboratorios Farmacéuticos Rovi

Schizophrenia

IM

Submitted − 505(b)(2) NDA

07/27/2023

denileukin diftitox

Citius

Cutaneous T cell lymphoma (persistent or recurrent)

IV

Submitted − BLA; Orphan Drug

07/28/2023

remestemcel-L

Mesoblast

GVHD (acute, steroidrefractory, pediatrics)

IV

Submitted − BLA; Fast Track; Orphan Drug

07/31/2023

chikungunya vaccine monovalent, live attenuated

Valneva

Chikungunya infection prevention (single dose, adults)

IM

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

elranatamab

Pfizer

Multiple myeloma (R/R)

SC

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

Aug-Sep 2023

brimonidine 0.35%

Visiox

Glaucoma/ocular hypertension

Ophthalmic

Submitted − 505(b)(2) NDA

08/04/2023

zuranolone

Sage/Biogen

MDD; Postpartum depression

Oral

Submitted − NDA; Breakthrough Therapy; Fast Track; Priority review

08/05/2023

avasopasem manganese Galera

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

IV

Submitted − NDA; Breakthrough Therapy; Fast Track; Priority review

08/09/2023

melphalan kit for injection/hepatic delivery system

Delcath

Uveal melanoma (hepatic-dominant)

Percutaneous hepatic perfusion

Submitted − 505(b)(2) NDA

08/14/2023

palovarotene

Ipsen

Fibrodysplasia ossificans progressiva

Oral

Submitted − NDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority review; RPD

08/16/2023

avacincaptad pegol

Astellas

Geographic atrophy secondary to AMD

Intravitreal

Submitted − NDA; Breakthrough Therapy; Fast Track; Priority review

08/19/2023

31 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

pozelimab

Regeneron

CHAPLE syndrome

IV, SC

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

08/20/2023

bevacizumab-vikg

Outlook

Wet AMD

Intravitreal

Submitted − BLA

08/29/2023

lebrikizumab

Eli Lilly

Atopic dermatitis (moderate-severe)

SC

Submitted − BLA; Fast Track

September 2023

nedosiran

Novo Nordisk

Hyperoxaluria

SC

Submitted − NDA; Breakthrough Therapy; Orphan Drug; RPD

September 2023

motixafortide

Biolinerx

Stem cell mobilization for autologous BMT for multiple myeloma

SC

Submitted − NDA; Orphan Drug

09/09/2023

atezolizumab SC

Genentech

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

Submitted − BLA

09/15/2023

momelotinib

GlaxoSmithKline

Myelofibrosis

Oral

Submitted − NDA; Fast Track; Orphan Drug

09/16/2023

epinephrine

ARS

Anaphylaxis

Intranasal

Submitted − 505(b)(2) NDA; Fast Track

09/19/2023

metronidazole (tastemasking liquid)

Appili

Parasitic and anaerobic bacterial infection treatment

Oral

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

09/23/2023

phentolamine 0.75%

Ocuphire

Reverse pharmacologicallyinduced mydriasis

Ophthalmic

Submitted − 505(b)(2) NDA

09/28/2023

meningococcal pentavalent vaccine (PF-06886992)

Pfizer

Meningococcal immunization

IM

Submitted − BLA

October 2023

capivasertib

AstraZeneca

Breast cancer (R/R, Oral HR+/HER2-, locally advanced or metastatic, in combination with fulvestrant, prior endocrine-based therapy)

Submitted − NDA; Fast Track; Priority Review

Oct-Dec 2023

sofpironium

Botanix

Axillary hyperhidrosis (severe)

Topical

Submitted − NDA

Oct-Dec 2023

zilucoplan

UCB

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

SC

Submitted − NDA; Orphan Drug

Oct-Dec 2023

tocilizumab (biosimilar Biogen/Bio-Thera to Genentech’s Actemra) Solutions

RA; Polyarticular JIA; Systemic JIA

IV, SC

Submitted − BLA

10/09/2023

tenapanor

Ardelyx

Hyperphosphatemia (associated with CKD)

Oral

Submitted − NDA

10/17/2023

adapalene/benzoyl peroxide/clindamycin

Bausch Health

Acne

Topical

Submitted − NDA

10/20/2023

pilocarpine 0.4%

Orasis

Presbyopia

Ophthalmic

Submitted − 505(b)(2) NDA

10/20/2023

infliximab SC

Celltrion

Inflammatory bowel disease

SC

Submitted − BLA

10/22/2023

clobetasol propionate

Laboratorios Salvat

Postsurgical ocular pain and inflammation

Ophthalmic

Submitted − NDA

10/25/2023

32 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

vamorolone

Santhera

DMD

Oral

Submitted − NDA; Fast Track; Orphan Drug; RPD

10/26/2023

taurolidine/citrate/ heparin

Cormedix

Reduction of catheterrelated bloodstream infections (CRBSIs) related to chronic hemodialysis

IV

Submitted − NDA; Fast Track; QIDP

11/15/2023

vonoprazan

Phathom

Erosive gastroesophageal reflux disease

Oral

Submitted − NDA

11/17/2023

reproxalap

Aldeyra

DED

Ophthalmic

Submitted − NDA

11/23/2023

lifileucel

Iovance

Melanoma (advanced unresectable or metastatic, after antiPD-1/PD-L1 and targeted therapy)

IV

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

11/25/2023

nirogacestat

Springworks/ GlaxoSmithKline

Desmoid tumors

Oral

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

11/27/2023

repotrectinib

Bristol-Myers Squibb

NSCLC (ROS1-positive, metastatic)

Oral

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

11/27/2023

fruquintinib

Takeda/Hutchmed

CRC (refractory, metastatic, ≥ 2nd-line)

Oral

Submitted − NDA; Fast Track; Priority Review

11/30/2023

denosumab (biosimilar to Amgen’s Prolia/ Xgeva)

Sandoz

Osteoporosis/osteopenia

SC

Submitted − BLA

12/06/2023

debamestrocel

Brainstorm

ALS

Intrathecal

Submitted − NDA; Fast Track; Orphan Drug

12/08/2023

exagamglogene autotemcel

Vertex/CRISPR

SCD (severe)

IV

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

12/08/2023

talquetamab

Janssen

Multiple myeloma (R/R)

SC

Submitted − BLA; Breakthrough Therapy; Orphan Drug

12/11/2023

trastuzumab (biosimilar to Genentech’s Herceptin)

Henlius/Accord

Breast cancer; Gastric or gastroesophageal junction adenocarcinoma

IV

Submitted − BLA

12/15/2023

roflumilast foam

Arcutis

Seborrheic dermatitis (ages ≥ 9 years)

Topical

Submitted − NDA

12/16/2023

aprocitentan

Janssen

Hypertension (resistant)

Oral

Submitted − NDA

12/20/2023

lovotibeglogene autotemcel

Bluebird Bio

SCD (severe)

IV

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

12/20/2023

givinostat

Italfarmaco

DMD

Oral

Submitted − NDA; Fast Track; Orphan Drug; Priority Review; RPD

12/21/2023

eplontersen

Ionis/AstraZeneca

Transthyretin amyloid polyneuropathy

SC

Submitted − NDA; Orphan Drug

12/22/2023

travoprost implant

Glaukos

Glaucoma/ocular hypertension

Intraocular

Submitted − 505(b)(2) NDA

12/22/2023

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PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

insulin glargine (biosimilar to SanofiAventis’ Lantus)

Gan & Lee/Sandoz

T1DM; T2DM

SC

Submitted − BLA

12/23/2023

dihydroergotamine nasal powder

Satsuma

Migraine (acute treatment)

Intranasal

Submitted − 505(b)(2) NDA

January 2024

niraparib/abiraterone (dual-action tablet)

GlaxoSmithKline

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

Oral

Submitted − NDA; Breakthrough Therapy

01/01/2024

cosibelimab

Checkpoint

Cutaneous squamous cell carcinoma (metastatic)

IV

Submitted − BLA

01/04/2024

berdazimer gel

Novan

Molluscum contagiosum

Topical

Submitted − NDA

01/05/2024

zolbetuximab

Astellas

Gastric cancer (unresectable or metastatic, HER2-, CLDN18.2+)

IV

Submitted − BLA; Orphan Drug; Priority Review; RTOR

01/12/2024

apadamtase alfa/ cinaxadamtase alfa

Takada

Thrombotic thrombocytopenic purpura (congenital)

IV

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

01/16/2024

atropine 0.01%

Nevakar

Myopia (pediatrics)

Ophthalmic

Submitted − NDA

01/31/2024

eculizumab (biosimilar to Alexion’s Soliris)

Amgen

PNH; Hemolytic uremic syndrome (atypical)

IV

Submitted − BLA

February 2024

imetelstat

Geron

Myelodysplastic syndrome (transfusiondependent, ESAineligible)

IV

Submitted − NDA; Fast Track; Orphan Drug

Feb-Jun 2024

roluperidone

Minerva

Schizophrenia (negative symptoms)

Oral

Submitted − NDA

02/26/2024

clobetasol propionate

Formosa

Ocular pain &/or inflammation

Ophthalmic

Submitted − 505(b)(2) NDA

03/04/2024

exagamglogene autotemcel

Vertex/CRISPR

Beta-thalassemia (transfusion-dependent)

IV

Submitted − BLA; Fast Track; Orphan Drug; RPD; RMAT

03/30/2024

fidanacogene elaparvovec

Pfizer/Genentech

Hemophilia B (adults)

IV

Submitted − BLA; Breakthrough Therapy; Orphan Drug; RMAT

Apr-Jun 2024

resmetirom

Madrigal

NASH (with liver fibrosis)

Oral

Submitted− NDA; Breakthrough Therapy; Fast Track

Apr-Jun 2024

insulin lispro (biosimilar to Eli Lilly’s Humalog)

Gan & Lee/Sandoz

T1DM; T2DM

SC

Submitted − BLA

04/01/2024

insulin aspart (biosimilar to Novo Nordisk’s Novolog)

Gan & Lee/Sandoz

T1DM; T2DM

SC

Submitted − BLA

04/14/2024

ranibizumab (biosimilar to Genentech’s Lucentis)

STADA Arzneimittel/ Xbrane

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

Intravitreal

Submitted − BLA

04/21/2024

camrelizumab

Jiangsu Hengrui

HCC (unresectable, 1stline, in combination with rivoceranib)

IV

Submitted − BLA; Orphan Drug

05/16/2024

rivoceranib

Elevar

HCC (unresectable, 1stline, in combination with camrelizumab)

Oral

Submitted − NDA; Orphan Drug

05/16/2024

34 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

macitentan/tadalafil

Janssen

PAH (WHO functional class II-III)

Oral

Submitted − NDA; Orphan Drug

05/30/2024

troriluzole

Biohaven

Spinocerebellar ataxia type 3

Oral

Submitted − NDA; Fast Track; Orphan Drug

05/31/2024

cefepime/ enmetazobactam

Allecra

UTI (complicated)

IV

Submitted − NDA; Fast Track; QIDP

06/27/2024

ensifentrine

Verona

COPD

Inhaled

Submitted − NDA

06/27/2024

aflibercept (biosimilar to Regeneron’s Eylea)

Celltrion

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

Intravitreal

Submitted − BLA

06/28/2024

aflibercept (biosimilar to Regeneron’s Eylea)

Coherus

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

Intravitreal

Submitted − BLA

06/28/2024

rituximab (biosimilar to Genentech’s Rituxan)

Dr. Reddy’s

CCL; Granulomatosis with polyangiitis/Microscopic polyangiitis; NHL; Mature B-cell NHL/mature B-cell acute leukemia; Pemphigus vulgaris; RA

IV

Submitted − BLA

07/12/2024

aflibercept (biosimilar to Regeneron’s Eylea)

Biocon/Janssen

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

Intravitreal

Submitted − BLA

Pending

bevacizumab (biosimilar Bio-Thera Solutions/ to Genentech’s Avastin) Sandoz

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

IV

Submitted − BLA

Pending

bevacizumab (biosimilar Centus to Genentech’s Avastin)

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

IV

Submitted − BLA

Pending

bevacizumab (biosimilar Samsung Bioepis/ to Genentech’s Avastin) Organon

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

IV

Submitted − BLA

Pending

bimekizumab

UCB

PSO

SC

Submitted − BLA

Pending

cipaglucosidase alfa

Amicus

Pompe disease (in combination with oral miglustat)

IV

Submitted − BLA; Breakthrough Therapy; Orphan Drug

Pending

filgrastim (biosimilar to Amgen’s Neupogen)

Apotex

Neutropenia/leukopenia

IV, SC

Submitted − BLA

Pending

natalizumab (biosimilar to Biogen’s Tysabri)

Polpharma/Sandoz

MS; CD

IV

Submitted − BLA

Pending

pegfilgrastim (biosimilar Apotex to Amgen’s Neulasta)

Neutropenia/leukopenia

SC

Submitted − BLA

Pending

pegfilgrastim (biosimilar Lupin to Amgen’s Neulasta)

Neutropenia/leukopenia

SC

Submitted − BLA

Pending

tocilizumab (biosimilar Fresenius Kabi to Genentech’s Actemra)

RA; Polyarticular JIA; Systemic JIA

IV, SC

Submitted − BLA

Pending

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

July 2023

Submitted (Supplementals) brentuximab vedotin (Adcetris®)

35 | MAGELLANRX.COM

Seagen

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

IV

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

RSV vaccine (Abrysvo™)

Pfizer

RSV prevention (ages birth-6 months, maternal administration)

IM

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

trifluridine/tipiracil (Lonsurf)

Otsuka

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

Oral

Submitted − sNDA; Fast Track; Priority Review

08/13/2023

daxibotulinumtoxinA (Daxxify™)

Revance

Cervical dystonia

IM

Submitted − sBLA; Orphan Drug

08/19/2023

valbenazine (Ingrezza®)

Neurocrine

Huntington’s disease

Oral

Submitted − sNDA; Orphan Drug

08/20/2023

luspatercept-aamt (Reblozyl)

Merck

Myelodysplastic syndrome

SC

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

08/28/2023

empagliflozin (Jardiance®)

Boehringer Ingelheim/Eli Lilly

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

Oral

Submitted − sNDA; Fast Track

Sep-Oct 2023

letermovir (Prevymis™)

Merck

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

IV, Oral

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

09/07/2023

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

Coherus

Neutropenia/leukopenia

SC

Submitted − sBLA

October 2023

binimetinib (Mektovi)

Pfizer

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

Oral

Submitted − sNDA

Oct-Dec 2023

encorafenib (Braktovi)

Pfizer

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

Oral

Submitted − sNDA

Oct-Dec 2023

vedolizumab (Entyvio®)

Takeda

UC (SC maintenance following IV induction)

SC

Submitted − sBLA

Oct-Dec 2023

patisiran (Onpattro®)

Alnylam

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

IV

Submitted − sNDA; Orphan Drug

10/06/2023

nivolumab (Opdivo)

Bristol-Myers Squibb

Melanoma (completely resected stage IIB or IIC)

IV

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

10/13/2023

pembrolizumab (Keytruda)

Merck

NSCLC (post surgical resection; stage 1B, II, or IIIA)

IV

Submitted − sBLA; Breakthrough Therapy

10/16/2023

roflumilast (Zoryve™)

Arcutis

PSO (ages 2-11 years)

Topical

Submitted − sNDA

10/19/2023

dupilumab (Dupixent®)

Sanofi

Urticaria (chronic, spontaneous, ages ≥ 12 years)

SC

Submitted − sBLA

10/20/2023

vosoritide (Voxzogo®)

Biomarin

Achondroplasia (ages < 5 years)

SC

Submitted − sNDA; Orphan Drug

10/20/2023

36 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

bupivacaine/meloxicam (Zynrelef®)

Heron

Postsurgical pain (soft tissue and orthopedic surgical procedures)

Instillation

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

10/23/2023

secukinumab (Cosentyx®)

Novartis

Hidradenitis suppurativa

SC

Submitted − sNDA

10/25/2023

maralixib (Livmarli®)

Mirum

Progressive familial intrahepatic cholestasisrelated pruritus (ages ≥ 2 months)

Oral

Submitted − sNDA; Breakthrough Therapy; Orphan Drug

12/14/2023

fluticasone propionate (Xhance®)

Optinose

Chronic rhinosinusitis

Intranasal

Submitted − sNDA

12/16/2023

idecabtagene vicleucel (Abecma)

Bristol-Myers Squibb

Multiple myeloma (R/R, prior immunomodulatory, proteasome inhibitor, and anti-CD38 therapy)

IV

Submitted − sBLA; Breakthrough Therapy; Orphan Drug

12/16/2023

pembrolizumab (Keytruda)

Merck

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

IV

Submitted − sBLA; Orphan Drug

12/16/2023

COVID-19 vaccine, bivalent (Omicron BA.4/BA.5) mRNA (Comirnaty®)

Biontech

COVID-19 prevention (bivalent primary series and booster doses)

IM

Submitted − sBLA; Fast Track

12/22/2023

von Willebrand factor/ coagulation factor VIII complex (Wilate®)

Octapharma

von Willebrand disease (routine prophylaxis)

IV

Submitted − sBLA; Orphan Drug

12/22/2023

zanubrutinib (Brukinsa®) Beigene

Follicular lymphoma (R/R, Oral ≥ 3rd-line, in combination with obinutuzumab)

Submitted − sNDA; Fast Track

Jan-Mar 2024

coagulation factor IX, recombinant (Ixinity®)

Pediapharm

Hemophilia B (ondemand, prophylactic, and perioperative treatment, ages < 12 years)

IV

Submitted − sBLA

Feb-Mar 2024

pembrolizumab (Keytruda)

Merck

Biliary tract cancer (locally advanced unresectable or metastatic, in combination with chemotherapy)

IV

Submitted − sBLA; Orphan Drug

02/07/2024

irinotecan liposomal (Onivyde®)

Ipsen

Pancreatic cancer (1stline, in combination with chemotherapy)

IV

Submitted − sNDA; Fast Track; Orphan Drug

02/13/2024

faricimab-svoa (Vabysmo®)

Genentech

Macular edema following RVO

Intravitreal

Submitted − sBLA

03/09/2024

COVID-19 vaccine, monovalent (Omicron XBB.1.5) mRNA (Comirnaty)

Biontech

COVID-19 prevention (ages ≥ 6 months)

IM

Submitted − sBLA; Fast Track

04/23/2024

37 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

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

Pfizer

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

SC

Submitted − sBLA for interchangeability

Pending

adalimumab-aaty (Yuflyma) (biosimilar to Humira 100 mg/mL)

Celltrion

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

SC

Submitted − sBLA for interchangeability

TBD

Phase 3− BLA; Orphan Drug

TBD

Phase 3 (New Drugs) AAV8-ranibizumab

Regenxbio

Wet AMD

abelacimab

Anthos

Stroke prevention in atrial SC fibrillation; VTE

Phase 3− BLA; Fast Track

TBD

abiraterone

Tavanta

Prostate cancer

Oral

Phase 3− NDA

TBD

acoramidis

Bridgebio

Transthyretin amyloid cardiomyopathy; Transthyretin amyloid polyneuropathy

Oral

Phase 3− NDA; Orphan Drug

TBD

adagrasib

Mirati

CRC

Oral

Phase 3− NDA; Breakthrough Therapy

TBD

adintrevimab

Invivyd/Biocon

COVID-19

IM

Phase 3− BLA

TBD

aficamten

Cytokinetics

Hypertrophic cardiomyopathy

Oral

Phase 3− NDA; Breakthrough Therapy; Orphan Drug

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Amgen

DME; Wet AMD

Intravitreal

Phase 3− BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

SamChunDang

DME; Wet AMD

Intravitreal

Phase 3− BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Samsung Bioepis

DME; Wet AMD

Intravitreal

Phase 3− BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Sandoz

DME; Wet AMD

Intravitreal

Phase 3− BLA

TBD

ALXN1720

AstraZeneca

Myasthenia gravis

SC

Phase 3− BLA

TBD

amubarvimab/ romlusevimab

Brii

COVID-19

IV

Phase 3− BLA

TBD

ANX-005

Annexon

Guillain-Barré syndrome

IV

Phase 3− BLA; Fast Track; Orphan Drug

TBD

apitegromab

Scholar Rock

Spinal muscular atrophy

IV

Phase 3− BLA; Fast Track; Orphan Drug

TBD

AR-15512

Aerie

DED

Ophthalmic

Phase 3− NDA

TBD

arimoclomol

Zevra

Niemann-Pick disease

Oral

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

TBD

ARO-APOC3

Arrowhead

Familial chylomicronemia syndrome

SC

Phase 3− NDA; Fast Track; Orphan Drug

TBD

asundexian

Bayer

Stroke prevention in atrial fibrillation

Oral

Phase 3− NDA; Fast Track

TBD

aztreonam-avibactam

Abbvie

Intra-abdominal bacterial infections

IV

Phase 3− NDA; Fast Track; QIDP

TBD

bamlanivimab

Eli Lilly

COVID-19

IV

Phase 3− BLA

TBD

batiraxcept

Aravive

Ovarian cancer

IV

Phase 3− BLA; Fast Track

TBD

bentracimab

SERB

Ticagrelor (Brilinta®) reversal

IV

Phase 3− BLA; Breakthrough Therapy

TBD

38 | MAGELLANRX.COM

Subretinal

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

bimekizumab

UCB

Axial spondyloarthritis; Hidradenitis suppurativa; PSO

SC

Phase 3− BLA

TBD

blarcamesine

Anavex Life Sciences

Alzheimer’s disease; Rett syndrome

Oral

Phase 3− NDA; Fast Track; Orphan Drug

TBD

botaretigene sparoparvovec

Janssen

Retinitis pigmentosa

Subretinal

Phase 3− BLA; Fast Track; Orphan Drug

TBD

BPR277

Lifemax

Netherton syndrome

Topical

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

TBD

brensocatib

Insmed/AstraZeneca

Bronchiectasis

Oral

Phase 3− NDA; Breakthrough Therapy

TBD

buntanetap

Annovis

Alzheimer’s disease; Parkinson’s disease

Oral

Phase 3− NDA

TBD

cagrilintide/semaglutide Novo Nordisk

T2DM

SC

Phase 3− NDA

TBD

cannabidiol gel

Zynerba

Fragile X syndrome

Topical

Phase 3− NDA; Fast Track; Orphan Drug

TBD

carbachol/brimonidine

Visus

Presbyopia

Ophthalmic

Phase 3− 505(b)(2) NDA

TBD

cefepime/ taniborbactam

Venatorx

UTI (complicated)

IV

Phase 3− NDA; Fast Track; QIDP

TBD

ceftobiprole medocaril

Basilea

ABSSSI; Bacteremia; CAP; HAP

IV

Phase 3− NDA; Fast Track; QIDP

TBD

cemdisiran

Alnylam

Myasthenia gravis

SC

Phase 3− NDA; Orphan Drug

TBD

cenerimod

Idorsia

SLE

Oral

Phase 3− NDA; Fast Track

TBD

clesrovimab

Merck

RSV prevention

IM

Phase 3− BLA

TBD

cobitolimod

Index

UC

Rectal

Phase 3− NDA; Orphan Drug

TBD

colistimethate sodium

Zambon

Bronchiectasis

Inhaled

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

TBD

condoliase

Ferring

Sciatica

Intrathecal

Phase 3− BLA

TBD

copper histidine

Zydus

Menkes disease

SC

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

TBD

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

Clover

COVID-19

SC

Phase 3− BLA

TBD

COVID-19 vaccine (SP0253)

Sanofi/ GlaxoSmithKline

COVID-19

IM

Phase 3− BLA

TBD

COVID-19 vaccine, adjuvanted (NVXCoV2515)

Novavax

COVID-19

IM

Phase 3− BLA

TBD

crinecerfont

Neurocrine

Congenital adrenal hyperplasia

Oral

Phase 3− NDA; Orphan Drug

TBD

crovalimab

Genentech

Hemolytic uremic syndrome; PNH

IV, SC

Phase 3− BLA; Breakthrough Therapy; Orphan Drug

TBD

CTX-009

Compass

Biliary tract cancer

IV

Phase 3− BLA

TBD

dabocemagene autoficel

Castle Creek

Epidermolysis bullosa

Intradermal

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

TBD

danicopan

AstraZeneca

PNH

Oral

Phase 3− NDA; Breakthrough Therapy; Orphan Drug

TBD

39 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

darvadstrocel

Takeda

CD (perianal fistulas)

IV

Phase 3− BLA; Orphan Drug

TBD

denosumab (Biosimilar to Amgen’s Prolia/ Xgeva)

Biocon

Osteoporosis/osteopenia

SC

Phase 3− BLA

TBD

denosumab (Biosimilar to Amgen’s Prolia/ Xgeva)

Celltrion

Osteoporosis/osteopenia

SC

Phase 3− BLA

TBD

denosumab (Biosimilar to Amgen’s Prolia/ Xgeva)

Gedeon Richter

Osteoporosis/osteopenia

IV

Phase 3− BLA

TBD

denosumab (Biosimilar to Amgen’s Prolia/ Xgeva)

Teva

Osteoporosis/osteopenia

SC

Phase 3− BLA

TBD

deoxythymidine/ deoxycytidine

UCB

Thymidine kinase 2 (TK2) deficiency

Oral

Phase 3− BLA; Breakthrough Therapy; Orphan Drug

TBD

deuruxolitinib

Sun

Alopecia areata

Oral

Phase 3− NDA; Breakthrough Therapy; Fast Track

TBD

difamilast

Medimetriks

Atopic dermatitis

Topical

Phase 3− NDA

TBD

difelikefalin

Cara

Atopic dermatitis; Pruritus Oral

Phase 3− NDA

TBD

dinutuximab beta

EUSA

Neuroendocrine tumors

IV

Phase 3− BLA; Orphan Drug

TBD

dirloctocogene samoparvovec

Genentech

Hemophilia A

IV

Phase 3− BLA; Breakthrough Therapy; Orphan Drug

TBD

donaperminogene seltoplasmid

Helixmith

Diabetic foot ulcers (chronic non-healing)

IM

Phase 3− BLA

TBD

doravirine/islatravir

Merck

HIV-1 infection treatment

Oral

Phase 3− NDA

TBD

dust mite immunotherapy tablet

Stallergenes Greer

Allergic rhinitis

SL

Phase 3− BLA

TBD

EB-101 (gene therapy)

Abeona

Epidermolysis bullosa

Surgical application

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

TBD

ebselen

Sound

Meniere’s disease

Oral

Phase 3− NDA; Fast Track

TBD

efzofitimod

Atyr

Sarcoidosis

IV

Phase 3− BLA; Fast Track; Orphan Drug

TBD

elafibranor

Genfit

Primary biliary cholangitis Oral

Phase 3− NDA; Breakthrough Therapy; Orphan Drug

TBD

englustat

Sanofi

Gaucher’s disease

Oral

Phase 3− NDA; Orphan Drug

TBD

enmetazobactam

Allecra

UTI (complicated)

IV

Phase 3− NDA; Fast Track; QIDP

TBD

eplontersen

Ionis

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

SC

Phase 3− NDA; Orphan Drug

TBD

esreboxetine

Axsome

Fibromyalgia

Oral

Phase 3− NDA

TBD

etavopivat

Novo Nordisk

SCD

Oral

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

TBD

etesevimab

Eli Lilly

COVID-19

IV

Phase 3− BLA

TBD

40 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

etrasimod

Pfizer

Atopic dermatitis; CD

Oral

Phase 3− NDA

TBD

etripamil

Milestone

Paroxysmal supraventricular tachycardia

Intranasal

Phase 3− NDA

TBD

evobrutinib

Merck

MS

Oral

Phase 3− NDA

TBD

factor VIII mimetic bispecific antibody

Novo Nordisk

Hemophilia A

SC

Phase 3− BLA; Orphan Drug

TBD

fasedienol

Vistagen

Social anxiety disorder

Intranasal

Phase 3− NDA; Fast Track

TBD

fenebrutinib

Genentech

MS

Oral

Phase 3− NDA

TBD

fexapotide triflutate

Nymox

BPH

Intratumoral

Phase 3− NDA

TBD

fianlimab

Regeneron

Melanoma

IV

Phase 3− BLA

TBD

filgotinib

Gilead

UC

Oral

Phase 3− NDA

TBD

fitusiran

Sanofi

Hemophilia A and B

SC

Phase 3− NDA; Fast Track; Orphan Drug

TBD

garadacimab

CSL

HAE

SC

Phase 3− BLA; Fast Track; Orphan Drug

TBD

gavorestat

Applied Therapeutics Galactosemia

Oral

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

TBD

GBT601

Pfizer

SCD

Oral

Phase 3− NDA; Orphan Drug

TBD

gepotidacin

GlaxoSmithKline

UTI (uncomplicated)

Oral

Phase 3− NDA; QIDP

TBD

giroctocogene fitelparvovec

Pfizer

Hemophilia A

IV

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

TBD

glatiramer depot

Viatris

MS

IM

Phase 3− 505(b)(2) NDA

TBD

glepaglutide

Zealand

Short bowel syndrome

SC

Phase 3− NDA; Orphan Drug

TBD

gold nanocrystal

Clene

ALS

Oral

Phase 3− NDA; Orphan Drug

TBD

granexin

Xequel

Surgical scar formation reduction

Topical

Phase 3− NDA

TBD

ianalumab

Novartis

Autoimmune hemolytic anemia; SLE

SC

Phase 3− BLA

TBD

inavolisib

Genentech

Breast cancer (HR+/HER2, 1st-line)

Oral

Phase 3− NDA

TBD

inclacumab

Pfizer

SCD

IV

Phase 3− BLA; Orphan Drug

TBD

infliximab (biosimilar to Janssen’s Remicade)

Nichi-Iko

RA; AS; PSO; CD

IV

Phase 3− BLA

TBD

influenza nanoparticle vaccine

Novavax

Seasonal influenza prevention

IM

Phase 3− BLA; Fast Track

TBD

insulin aspart (biosimilar to Novo Nordisk’s Novolog)

Amphastar

T1DM; T2DM

SC

Phase 3− BLA

TBD

insulin aspart (biosimilar to Novo Nordisk’s Novolog)

Sanofi

T1DM; T2DM

SC

Phase 3− BLA

TBD

insulin glargine (biosimilar to SanofiAventis’ Lantus)

Genesys

T1DM; T2DM

SC

Phase 3− BLA

TBD

41 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

iptacopan

Novartis

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

Oral

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

TBD

isotretinoin

Timber

Congenital ichthyosis (ages > 6 years)

Topical

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

TBD

itepekimab

Regeneron

COPD

SC

Phase 3− BLA; Fast Track

TBD

ivarmacitinib

Reistone

UC

Oral

Phase 3− NDA

TBD

izokibep

Affibody

Hidradenitis suppurativa

SC

Phase 3− BLA

TBD

JDQ-443

Novartis

NSCLC

Oral

Phase 3− NDA

TBD

Lactobacillus reuteri

Infant Bacterial Therapeutics

Necrotizing enterocolitis

Oral

Phase 3− BLA; Orphan Drug; RPD

TBD

lanifibranor

Inventiva

NASH

Oral

Phase 3− NDA; Breakthrough Therapy; Fast Track

TBD

lazertinib

Genosco/Janssen

NSCLC

Oral

Phase 3− NDA

TBD

lenadogene nolparvovec

Gensight

Leber’s hereditary optic neuropathy

Intravitreal

Phase 3− BLA; Orphan Drug

TBD

leriglitazone

Minoryx

Adrenoleukodystrophy

Oral

Phase 3− NDA; Fast Track; Orphan Drug

TBD

lerodalcibep

LIB

Dyslipidemia/ hypercholesterolemia

SC

Phase 3− BLA

TBD

leukocyte interleukin

Cel-Sci

SCCHN

Peritumoral/ perilymphatic

Phase 3− BLA; Orphan Drug

TBD

levodopa/carbidopa patch pump

Mitsubishi Tanabe

Parkinson’s disease

SC

Phase 3− 505(b)(2) NDA

TBD

ligelizumab

Novartis

Food allergies

SC

Phase 3− BLA

TBD

linerixibat

GlaxoSmithKline

Cholestatic pruritus

Oral

Phase 3− NDA; Orphan Drug

TBD

litifilimab

Biogen

SLE

SC

Phase 3− BLA

TBD

Lyme disease vaccine

Valneva/Pfizer

Lyme immunization

IM

Phase 3− BLA; Fast Track

TBD

magrolimab

Gilead

AML; Myelodysplastic syndrome

IV

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

TBD

marstacimab

Pfizer

Hemophilia A and B

IV, SC

Phase 3− BLA; Fast Track; Orphan Drug

TBD

marzeptacog alfa

Catalyst

Hemophilia A and B (with inhibitors)

SC

Phase 3− BLA; Fast Track; Orphan Drug

TBD

masitinib

AB Science

Alzheimer’s disease; ALS; Asthma; Mastocytosis; MS

Oral

Phase 3− NDA; Orphan Drug

TBD

mavodelpar

Reneo

Primary mitochondrial myopathies

Oral

Phase 3− NDA; Fast Track; Orphan Drug

TBD

mavorixafor

X4

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

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

TBD

meningococcal vaccine (GSK3536819A)

GlaxoSmithKline

Meningococcal immunization

Phase 3− BLA

TBD

42 | MAGELLANRX.COM

IM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

midomafetamine

Multidisciplinary Association for Psychedelic Studies

PTSD

Oral

Phase 3− NDA; Breakthrough Therapy

TBD

minocycline hydrochloride

Journey

Rosacea

Oral

Phase 3− 505(b)(2) NDA

TBD

mirikizumab

Eli Lilly

CD

IV, SC

Phase 3− BLA

TBD

molnupiravir (Lagevrio)

Merck

COVID-19

Oral

Phase 3− NDA

TBD

mometasone furoate

Lyra

Chronic rhinosinusitis

Implant

Phase 3− 505(b)(2) NDA

TBD

MTX-005 (anti-BK polyomavirus)

Memo

BK polyomavirus IV infection (renal transplant recipients)

Phase 3− BLA; Fast Track

TBD

MVA-BN RSV (RSV vaccine)

Bavarian Nordic

RSV prevention

IM

Phase 3− BLA; Breakthrough Therapy

TBD

nalbuphine ER

Trevi

Pruritus

Oral

Phase 3− NDA; Fast Track

TBD

narsoplimab

Omeros

Hemolytic uremic syndrome

IV, SC

Phase 3− BLA; Fast Track

TBD

navitoclax

Abbvie

Myelofibrosis

Oral

Phase 3− NDA; Orphan Drug

TBD

nemolizumab

Galderma

Atopic dermatitis; Pruritus SC

Phase 3− BLA; Breakthrough Therapy

TBD

nipocalimab

Janssen

Autoimmune hemolytic anemia; Myasthenia gravis

IV

Phase 3− BLA; Fast Track; Orphan Drug

TBD

nomacopan

Akari

HSCT-associated thrombotic microangiopathy; PNH

SC

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

TBD

NPC-12Y

Nobelpharma

Tuberous sclerosis complex skin lesions

Topical

Phase 3− NDA

TBD

obefazimod

Abivax

UC

Oral

Phase 3− NDA

TBD

olezarsen

Akcea

Familial chylomicronemia syndrome

SC

Phase 3− NDA; Fast Track

TBD

omalizumab (biosimilar for Genentech’s Xolair®)

Teva

Asthma; Nasal polyps; Urticaria

SC

Phase 3− BLA

TBD

omecamtiv mecarbil

Cytokinetics

Chronic heart failure (with Oral reduced ejection fraction)

Phase 3− NDA; Fast Track

TBD

OPT-302

Opthea

DME; Wet AMD

Intravitreal

Phase 3− BLA; Fast Track

TBD

orforglipron

Eli Lilly

Obesity/overweight

Oral

Phase 3− NDA

TBD

OTL-200 (gene therapy)

Orchard

Metachromatic leukodystrophy

IV

Phase 3− BLA; Orphan Drug; RPD; RMAT

TBD

padeliporfin

Steba

Bladder cancer

IV

Phase 3− NDA; Fast Track; Orphan Drug

TBD

paltusotine

Crinetics

Acromegaly

Oral

Phase 3− NDA; Orphan Drug

TBD

pamrevlumab

Fibrogen/BristolMyers Squibb

COVID-19; DMD; Pancreatic cancer

IV

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

TBD

parsaclisib

Incyte

Autoimmune hemolytic anemia

Oral

Phase 3− NDA; Orphan Drug

TBD

pegadricase

Swedish Orphan Biovitrum

Gout

IV

Phase 3− BLA

TBD

43 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

pegzilarginase

Aeglea

Arginase 1 deficiency

IV

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

TBD

pelacarsen

Novartis

Dyslipidemia/ hypercholesterolemia

SC

Phase 3− NDA; Fast Track

TBD

phentolamine ophthalmic solution 0.75%

Ocuphire

Presbyopia

Ophthalmic

Phase 3− NDA

TBD

PHVS416

Pharvaris

HAE

Oral

Phase 3− NDA

TBD

piclidenoson

Can-Fite

PSO

Oral

Phase 3− NDA

TBD

PL-9643

Palatin

DED

Ophthalmic

Phase 3− NDA

TBD

plinabulin

Beyondspring

Chemotherapy-induced neutropenia prevention

IV

Phase 3− NDA; Breakthrough Therapy

TBD

pollinex quattro

Allergy Therapeutics

Allergic rhinitis

SC

Phase 3− BLA

TBD

pollinex quattro grass

Allergy Therapeutics

Allergic rhinitis

SC

Phase 3− BLA

TBD

posoleucel

Allovir

Virus-associated hemorrhagic cystitis (post allogeneic HSCT)

IV

Phase 3− BLA; Orphan Drug; RMAT

TBD

povorcitinib

Incyte

Hidradenitis suppurativa

Oral

Phase 3− NDA

TBD

pozelimab

Regeneron

PNH

IV, SC

Phase 3− BLA; Orphan Drug

TBD

pritelivir

AiCuris Anti-infective Herpes simplex virus Cures treatment

Oral

Phase 3− NDA; Breakthrough Therapy; Fast Track

TBD

prothrombin complex

Octapharma

Hemostasis

IV

Phase 3− BLA

TBD

pruxelutamide

Kintor

COVID-19

Oral

Phase 3− NDA

TBD

QRX003

Quoin

Netherton syndrome

Topical

Phase 3− NDA

TBD

ralinepag

United Therapeutics

PAH

Oral

Phase 3− NDA; Orphan Drug

TBD

rapamycin (high strength)

Palvella

Pachyonychia congenita

Topical

Phase 3− NDA; Fast Track; Orphan Drug

TBD

ReACT (autologous kidney cells)

Prokidney

CKD

Renal cortex injection

Phase 3− BLA; RMAT

TBD

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

Regeneron

Birch allergy

SC

Phase 3− BLA

TBD

relacorilant

Corcept

Cushing’s syndrome

Oral

Phase 3− NDA; Orphan Drug

TBD

remibrutinib

Novartis

MS; Urticaria

Oral

Phase 3− NDA

TBD

reparixin

Dompé

Transplant Rejection

IV

Phase 3− NDA; Orphan Drug

TBD

reproxalap

Aldeyra

Allergic conjunctivitis

Ophthalmic

Phase 3− NDA

TBD

resiniferatoxin

Grunenthal

Osteoarthritis pain (knee)

Intra-articular

Phase 3− NDA; Breakthrough Therapy

TBD

rexlemestrocel-L

Mesoblast

Chronic low back pain

Intradiscal

Phase 3− BLA; RMAT

TBD

RGX-121 (gene therapy)

Regenxbio

Mucopolysaccharidosis II (Hunter Syndrome)

Intracerebroventricular

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

TBD

rilzabrutinib

Sanofi

ITP

Oral

Phase 3− NDA; Fast Track; Orphan Drug

TBD

ritlecitinib

Pfizer

Vitiligo

Oral

Phase 3− NDA

TBD

rocatinlimab

Amgen

Atopic dermatitis

IV

Phase 3− BLA

TBD

44 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

roflumilast foam

Arcutis

PSO

Topical

Phase 3− NDA

TBD

roxadustat

AstraZeneca

Anemia due to cytotoxic chemotherapy

Oral

Phase 3− NDA

TBD

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

RSV prevention (ages ≥ 60 years)

N/A

Phase 3− BLA; Breakthrough Therapy; Fast Track

TBD

ruxolitinib (deuterated)

Sun

Alopecia areata

Oral

Phase 3 − NDA; Breakthrough Therapy; Fast Track

TBD

sabatolimab

Novartis

Myelodysplastic syndrome

IV

Phase 3− BLA; Fast Track

TBD

sabizabulin

Veru

COVID-19 treatment

Oral

Phase 3− NDA; Fast Track

TBD

saroglitazar

Zydus

Primary biliary cholangitis Oral

Phase 3− NDA; Fast Track; Orphan Drug

TBD

sebetralstat

Kalvista

HAE

Phase 3− NDA; Fast Track; Orphan Drug

TBD

seladelpar

Cymabay

Primary biliary cholangitis Oral

Phase 3− NDA; Breakthrough Therapy; Orphan Drug

TBD

semaglutide

Novo Nordisk

NASH

SC

Phase 3− NDA; Breakthrough Therapy

TBD

serplulimab

Henlius

SCLC

IV

Phase 3− BLA; Orphan Drug

TBD

SkQ 1

Mitotech

DED

Ophthalmic

Phase 3− NDA

TBD

sotatercept

Merck/Bristol-Myers Squibb

PAH

SC

Phase 3− BLA; Breakthrough Therapy; Orphan Drug

TBD

sulopenem

Iterum/Pfizer

Intra-abdominal bacterial infections; UTI (uncomplicated)

IV, Oral

Phase 3− NDA; Fast Track; QIDP

TBD

tabelecleucel

Atara

Epstein-Barr virus-associated post-transplant lymphoproliferative disease

IV

Phase 3− BLA; Breakthrough Therapy; Orphan Drug

TBD

tamibarotene

Syros

Myelodysplastic syndrome

Oral

Phase 3− NDA; Fast Track; Orphan Drug

TBD

tarcocimab tedromer

Kodiak

DME; Diabetic retinopathy; Macular Edema from RVO; Wet AMD

Intravitreal

Phase 3− BLA

TBD

tebipenem pivoxil

GlaxoSmithKline

UTI (complicated)

Oral

Phase 3− NDA; Fast Track; QIDP

TBD

telisotuzumab vedotin

Abbvie

NSCLC

IV

Phase 3− BLA; Breakthrough Therapy

TBD

telitacicept

Rongchang

SLE

SC

Phase 3− BLA; Fast Track

TBD

tiragolumab

Genentech

Esophageal cancer; NSCLC

IV

Phase 3− BLA; Breakthrough Therapy; Orphan Drug

TBD

tiratricol

Rare Thyroid Therapeutics

Monocarboxylate transporter 8 (MCT8) deficiency

Oral

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

TBD

45 | MAGELLANRX.COM

Oral

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

tislelizumab

Beigene/Novartis

Gastric cancer; HCC; Nasopharyngeal cancer; NSCLC

IV

Phase 3− BLA; Orphan Drug

TBD

tixagevimab/cilgavimab (Evusheld)

AstraZeneca

COVID-19

IM

Phase 3− BLA

TBD

tolebrutinib

Sanofi

MS

Oral

Phase 3− NDA

TBD

tovorafenib

Day One Bio

Glioma (relapsed/ progressive)

Oral

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

TBD

tradipitant

Vanda

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

Oral

Phase 3− NDA

TBD

tramiprosate

Alzheon

Alzheimer’s disease

Oral

Phase 3− NDA; Fast Track

TBD

trastuzumab (biosimilar to Genentech’s Herceptin)

Sandoz

Breast cancer; Gastric or gastroesophageal junction adenocarcinoma

IV

Phase 3− BLA

TBD

treosulfan

Medac

Allogenic-HSCT conditioning

IV

Phase 3− NDA; Orphan Drug

TBD

tusamitamab ravtansine

Sanofi

NSCLC

IV

Phase 3− BLA

TBD

ulotaront

Concert

Schizophrenia

Oral

Phase 3− NDA; Breakthrough Therapy

TBD

upifitamab rilsodotin

Mersana

Ovarian cancer

IV

Phase 3− BLA; Fast Track

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Amgen

PSO

IV, SC

Phase 3− BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Formycon

PSO

IV, SC

Phase 3− BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Hikma

PSO

IV, SC

Phase 3− BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Intas

PSO

IV, SC

Phase 3− BLA

TBD

vadadustat

Akebia

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

Oral

Phase 3− NDA

TBD

venglustat

Sanofi

GM2 Gangliosidoses (TaySachs Disease, Sandhoff Disease, AB Variant)

Oral

Phase 3− NDA; Orphan Drug

TBD

viaskin peanut

DBV

Peanut allergy

Transdermal

Phase 3− BLA; Breakthrough Therapy; Fast Track

TBD

von Willebrand factor concentrate

LFB

von Willebrand disease

IV

Phase 3− BLA; Orphan Drug

TBD

VX-121/tezacaftor/ deutivacaftor

Vertex

CF

Oral

Phase 3− NDA; Orphan Drug

TBD

xanomeline/trospium

Karuna

Schizophrenia

Oral

Phase 3− NDA

TBD

zanidatamab

Jazz

Gastric cancer

IV

Phase 3− BLA; Fast Track; Orphan Drug

TBD

zoliflodacin

Innoviva

Gonorrhea

Oral

Phase 3− NDA; Fast Track; QIDP

TBD

46 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

Phase 3 (Supplementals) alpelisib (Piqray )

Novartis

Breast cancer (HER2+, triple-negative); Ovarian cancer

Oral

Phase 3− sNDA

TBD

atezolizumab (Tecentriq®)

Genentech

SCCHN

IV

Phase 3− sBLA

TBD

Eli Lilly

JIA; Uveitis

Oral

Phase 3− sNDA

TBD

benralizumab (Fasenra ) AstraZeneca

ANCA-associated vasculitis; Bullous pemphigoid; Chronic rhinosinusitis with nasal polyp; Esophagitis

SC

Phase 3− sBLA; Orphan Drug

TBD

brexpiprazole (Rexulti®)

Otsuka

PTSD

Oral

Phase 3− sNDA

TBD

canakinumab (Ilaris )

Novartis

NSCLC (adjuvant)

SC

Phase 3− sBLA

TBD

cemiplimab-rwlc (Libtayo®)

Regeneron

Melanoma

IV

Phase 3− sBLA

TBD

deucravacitinib (Sotyktu®)

Bristol-Myers Squibb

SLE

Oral

Phase 3− sNDA

TBD

dupilumab (Dupixent)

Sanofi

Bullous pemphigoid; COPD

SC

Phase 3− sBLA; Orphan Drug

TBD

durvalumab (Imfinzi®)

AstraZeneca

Bladder cancer (1st-line); Gastric cancer

IV

Phase 3− sBLA; Breakthrough Therapy

TBD

efgartigimod (Vyvgart)

Argenx

ITP

IV

Phase 3− sBLA; Orphan Drug

TBD

ferric carboxymaltose (Injectafer®)

Daiichi Sankyo

Anemia in heart failure

IV

Phase 3− sNDA

TBD

ferric derisomaltose (Monoferric®)

Pharmacosmos

Anemia in heart failure

IV

Phase 3− sNDA

TBD

fostamatinib (Tavalisse®) Rigel

Autoimmune hemolytic anemia

Oral

Phase 3− sNDA; Fast Track; Orphan Drug

TBD

guselkumab (Tremfya®)

Janssen

CD; UC

IV, SC

Phase 3− sBLA

TBD

iloperidone (Fanapt®)

Vanda

Bipolar disorder

Oral

Phase 3− sNDA

TBD

immune globulin, Octapharma human 10% (Octagam®)

COVID-19

IV

Phase 3− sBLA

TBD

inebilizumab-cdon (Uplizna®)

Horizon

IgG4-related disease; Myasthenia gravis

IV

Phase 3− sBLA

TBD

lumateperone (Caplyta®)

Intra-Cellular Therapies

MDD

Oral

Phase 3− sNDA

TBD

mepolizumab (Nucala®)

GlaxoSmithKline

COPD

IV, SC

Phase 3− sBLA

TBD

meropenem/ vaborbactam (Vabomere®)

Melinta

Bacteremia; HAP

IV

Phase 3− sNDA; QIDP

TBD

mitapivat (Pyrukynd®)

Agios

SCD; Thalassemia

Oral

Phase 3− sNDA; Orphan Drug

TBD

mosunetuzumab-axgb (Lunsumio™)

Genentech

DLBCL

SC

Phase 3− sBLA

TBD

nitazoxanide ER (Alinia®) Lupin/Romark

COVID-19; Influenza

Oral

Phase 3− sNDA

TBD

obinutuzumab (Gazyva®)

Genentech

Lupus nephritis; SLE

IV

Phase 3− sBLA; Breakthrough Therapy

TBD

ocrelizumab SC (Ocrevus®)

Genentech

MS

SC

Phase 3− sBLA

TBD

®

baricitinib (Olumiant®) ®

®

47 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

omalizumab (Xolair)

Genentech

Food allergies

SC

Phase 3− sBLA; Breakthrough Therapy

TBD

pegcetacoplan (Empaveli®)

Apellis

Autoimmune hemolytic anemia

SC

Phase 3− sNDA; Orphan Drug

TBD

ranibizumab port delivery system (Susvimo®)

Genentech

DME; Diabetic retinopathy

Intravitreal implant

Phase 3− sBLA

TBD

rimegepant (Nurtec ODT®)

Pfizer

Chronic rhinosinusitis

Oral

Phase 3− sNDA

TBD

risankizumab-rzaa (Skyrizi®)

Abbvie

UC

IV, SC

Phase 3− sBLA

TBD

rivaroxaban (Xarelto®)

Janssen

COVID-19 treatment

Oral

Phase 3− sNDA

TBD

roflumilast (Zoryve)

Arcutis

Atopic dermatitis

Topical

Phase 3− sNDA

TBD

romiplostim (Nplate®)

Amgen

Chemotherapy-induced thrombocytopenia

SC

Phase 3− sBLA; Orphan Drug

TBD

satralizumab-mwge (Enspryng®)

Genentech

Myasthenia gravis

SC

Phase 3− sBLA

TBD

secukinumab (Cosentyx) Novartis

Giant cell arteritis

SC

Phase 3− sBLA

TBD

semaglutide (Rybelsus ) Novo Nordisk

Obesity/overweight

Oral

Phase 3− sNDA

TBD

sparsentan (Filspari®)

Travere

Focal segmental glomerulosclerosis

Oral

Phase 3− sNDA; Orphan Drug

TBD

spesolimab-sbzo (Spevigo®)

Boehringer Ingelheim

UC

IV, SC

Phase 3− sBLA

TBD

tapinarof (Vtama®)

Roivant

Atopic dermatitis

Topical

Phase 3− sNDA

TBD

tezepelumab-ekko (Tezspire®)

Amgen

Chronic rhinosinusitis (with nasal polyps); Esophagitis

SC

Phase 3− sBLA; Orphan Drug

TBD

tirzepatide (Mounjaro)

Eli Lilly

Obesity/overweight (weight-related comorbidities)

SC

Phase 3− sNDA; Fast Track

TBD

treprostinil (Tyvaso®)

United

Idiopathic pulmonary fibrosis

Inhaled

Phase 3− sNDA; Orphan Drug

TBD

upadacitinib (Rinvoq®)

Abbvie

Giant cell arteritis

Oral

Phase 3− sNDA

TBD

vedolizumab (Entyvio)

Takeda

GVHD prophylaxis

IV

Phase 3− sBLA; Orphan Drug

TBD

venetoclax (Venclexta®)

Abbvie/Genentech

Mantle cell lymphoma; Multiple myeloma; Myelodysplastic syndrome

Oral

Phase 3− sNDA; Breakthrough Therapy; Orphan Drug

TBD

Non-erosive gastroesophageal reflux disease (NERD)

Oral

Phase 3− sNDA

TBD

®

vonoprazan/amoxicillin/ Phathom clarithromycin (Takecab®)

Complete Response Letter (CRL) NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

aflibercept (Eylea) 8 mg dose

Regeneron

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

Intravitreal

CRL

TBD

carbidopa/levodopa ER

Amneal

Parkinson’s disease

Oral

CRL

TBD

concizumab

Novo Nordisk

Hemophilia A and B with inhibitors (prophylaxis)

SC

CRL

TBD

48 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

CLINICAL USE

DOSAGE FORM

DEVELOPMENT STATUS

FDA DECISION

dasatinib

Xspray

CML

Oral

CRL

TBD

dehydrated alcohol injection

Eton

Methanol poisoning

SC

CRL

TBD

methotrexate

Aldeyra

Primary vitreoretinal lymphoma

Intravitreal

CRL

TBD

nogapendekin alfa inbakicept

Immunitybio

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

Intravesical

CRL

TBD

obeticholic acid (Ocaliva®)

Intercept

NASH-related precirrhotic liver fibrosis

Oral

CRL

TBD

olorofim

F2G

Fungal infections (invasive)

Oral

CRL

TBD

palopegteriparatide

Ascendis

Hypoparathyroidism

SC

CRL

TBD

rucaparib (Rubraca )

Clovis

Ovarian cancer (1st-line maintenance)

Oral

CRL

TBD

(vic-) trastuzumab duocarmazine

Byondis

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

IV

CRL

TBD

®

49 | MAGELLANRX.COM

GLOSSARY 5-FU 5-Fluorouracil

BsUFA Biosimilar User Fee Act

6MWT 6 Minute Walking Test

CABP Community Acquired Bacterial Pneumonia

ABSSSI Acute Bacterial Skin and Skin Structure Infection

CAP Community Acquired Pneumonia

ACC American College of Cardiology

CD Crohn's Disease

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

CAR T Chimeric Antigen Receptor T Cell CD3 Cluster of Differentiate 3 CD19 Cluster of Differentiate 19 CD20 Cluster of Differentiate 20 CD38 Cluster of Differentiate 38

ACR70 American College of Rheumatology 70% Improvement

CD79b Cluster of Differentiate 79b

ADC Antibody-Drug Conjugate

CF Cystic Fibrosis

ADHD Attention Deficit Hyperactivity Disorder

CHF Congestive Heart Failure

ADL Activities of Daily Living

CI Confidence Interval

AED Anti-Epileptic Drug

CKD Chronic Kidney Disease

AHA American Heart Association

CLL Chronic Lymphocytic Leukemia

ALK Anaplastic Lymphoma Kinase

CML Chronic Myeloid Leukemia

ALL Acute Lymphoblastic Leukemia

CMS Centers for Medicare & Medicaid Services

ALS Amyotrophic Lateral Sclerosis

CNS Central Nervous System

ALT Alanine Transaminase

COPD Chronic Obstructive Pulmonary Disease

AMD Age-Related Macular Degeneration

COVID-19 Coronavirus Disease 2019

AML Acute Myeloid Leukemia

CRC Colorectal Cancer

ANCA Antineutrophil Cytoplasmic Antibodies

CRL Complete Response Letter

ANDA Abbreviated New Drug Application

CRR Complete Response Rate

ARB Angiotensin II Receptor Blocker

CRS Cytokine Release Syndrome

ARNI Angiotensin Receptor-Neprilysin Inhibitor

CSF Colony Stimulating Factor

ART Antiretroviral Therapy

CTLA-4 Cytotoxic T-Lymphocyte-Associated Protein 4

ARV Antiretroviral

CV Cardiovascular

AS Ankylosing Spondylitis

CVD Cardiovascular Disease

ASCVD Atherosclerotic Cardiovascular Disease

CYP Cytochrome P-450

AST Aspartate Aminotransferase

CYP3A4 Cytochrome P-450 3A4

BCG Bacillus Calmette-Guérin BCVA Best Corrected Visual Acuity

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

BLA Biologics License Application

DCR Disease Control Rate

BMI Body Mass Index

DEA Drug Enforcement Administration

BMT Bone Marrow Transplant

DED Dry Eye Disease

BPH Benign Prostatic Hyperplasia

DLBCL Diffuse Large B Cell Lymphoma

BRAF B-Raf Proto-Oncogene

DMARD Disease Modifying Antirheumatic Drug

BSA Body Surface Area

DMD Duchenne Muscular Dystrophy

50 | MAGELLANRX.COM

CDC Centers for Disease Control and Prevention

GLOSSARY continued DME Diabetic Macular Edema

HBV Hepatitis B Virus

dMMR DNA mismatch repair

HCC Hepatocellular Carcinoma

DMT Disease Modifying Therapy

HCP Healthcare Professional

DNA Deoxyribonucleic Acid

HCV Hepatitis C Virus

DOR Duration of Response

HDRS-17 Hamilton Depression Rating Scale

DPI Dry Powder for Inhalation

HER Human Epidermal Growth Factor Receptor

DPP-4 Dipeptidyl Peptidase 4

HER2 Human Epidermal Growth Factor Receptor 2

DR Delayed-Release

HF Heart Failure

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

HFA Hydrofluoroalkane

ECOG Eastern Cooperative Oncology Group

HFSA Heart Failure Society of America

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

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HFpEF Heart Failure with preserved Ejection Fraction HIT Heparin Induced Thrombocytopenia HIV Human Immunodeficiency Virus HIV-1 Human Immunodeficiency Virus-1 HPV Human Papilloma Virus HR Hazard Ratio HSCT Hematopoietic Stem Cell Transplant HSV Herpes Simplex Virus HTN Hypertension IBS Irritable Bowel Syndrome IBS-C Irritable Bowel Syndrome, Constipation Predominant ICU Intensive Care Unit IDSA Infectious Diseases Society of America IGA Investigator's Global Assessment IgG Immunoglobulin G IgG1kappa Immunoglobulin G1 kappa IL-4 Interleukin-4 IL-12 Interleukin-12 IL-13 Interleukin-13 IL-17 Interleukin-17 IL-23 Interleukin-23 IM Intramuscular IR Immediate-Release IRB Institutional Review Board ITP Immune Thrombocytopenic Purpura ITT Intention-To-Treat IV Intravenous JAK Janus Kinase Inhibitor JIA Juvenile Idiopathic Arthritis KIT c-KIT Proto-Oncogene

GLOSSARY continued LDL Low-Density Lipoprotein

OTC Over-the-Counter

LDL-C Low-Density Lipoprotein Cholesterol

PAH Pulmonary Arterial Hypertension

LPAD Limited Population Pathway for Antibacterial and Antifungal Drugs

PARP Poly(ADP-Ribose) Polymerase

LS Least Square

PASI Psoriasis Area and Severity Index

LVEF Left Ventricular Ejection Fraction mAb Monoclonal Antibody MACE Major Adverse Cardiovascular Events MADRS Montgomery – Åsberg Depression Rating Scale MAOI Monoamine Oxidase Inhibitor MDD Major Depressive Disorder MDI Metered Dose Inhaler MDR Multi-Drug Resistant MECP2 Methyl-CpG Binding Protein 2 MEK Mitogen-Activated Extracellular Signal-Regulated Kinase MI Myocardial Infarction mITT modified Intention-To-Treat MRI Magnetic Resonance Imaging MRSA Methicillin-Resistant Staphylococcus Aureus MS Multiple Sclerosis MSI-H Microsatellite Instability-High N/A Not Applicable NASH Non-Alcoholic Steatohepatitis NCCN National Comprehensive Cancer Network NCT National Clinical Trials NDA New Drug Application NHL Non-Hodgkin Lymphoma NIAID National Institute of Allergy and Infectious Diseases NRAS Neuroblastoma RAS Proto-Oncogene NSAID Non-Steroidal Anti-Inflammatory Drug NSCLC Non-Small Cell Lung Cancer NTRK Neurotrophic Tyrosine Receptor Kinase NYHA New York Heart Association ODT Orally Disintegrating Tablet OR Odds Ratio ORR Overall/Objective Response Rate OS Overall Survival

PAS Prior Approval Supplement PASI 50 Psoriasis Area and Severity Index 50% Reduction PASI 75 Psoriasis Area and Severity Index 75% Reduction PASI 90 Psoriasis Area and Severity Index 90% Reduction PASI 100 Psoriasis Area and Severity Index 100% Reduction PCI Percutaneous Coronary Intervention PCSK9 Proprotein Convertase Subtilisin Kexin 9 PD-1 Programmed Death Protein 1 PD-L1 Programmed Death-Ligand 1 PDUFA Prescription Drug User Fee Application PFS Progression-Free Survival PGA Physician Global Assessment PI3K Phosphatidylinositol-3-kinase PNH Paroxysmal Nocturnal Hemoglobinuria PsA Psoriatic Arthritis PSO Plaque Psoriasis PTCA Percutaneous Transluminal Coronary Angioplasty PTSD Post-Traumatic Stress Disorder Q Quarter QIDP Qualified Infectious Diseases Product QOL Quality of Life R/R Relapsed or Refractory R-CHOP Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, Prednisone RA Rheumatoid Arthritis RAS Ras Protein Superfamily RBC Red Blood Cell RCC Renal Cell Carcinoma REMS Risk Evaluation and Mitigation Strategy RMAT Regenerative Medicine Advanced Therapy RNA Ribonucleic Acid RPD Rare Pediatric Disease RRR Relative Risk Reduction

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GLOSSARY continued RSV Respiratory Syncytial Virus RTOR Real-Time Oncology Review RVO Retinal Vein Occlusion SARS-CoV-2 Severe Acute Respiratory Syndrome-Associated Coronavirus-2 sBLA supplemental Biologics License Application SC Subcutaneous SCCHN Squamous Cell Cancer of the Head and Neck SCD Sickle Cell Disease SCLC Small Cell Lung Cancer SCT Stem Cell Transplant SGLT2 Sodium-Glucose Co-Transporter 2 SL Sublingual SLE Systemic Lupus Erythematosus SLL Small Lymphocytic Lymphoma sNDA supplemental New Drug Application SNRI Serotonin and Norepinephrine Reuptake Inhibitor SOC Standard of Care sPGA static Physician Global Assessment SR Sustained-Release SSRI Selective Serotonin Reuptake Inhibitor SSSI Skin and Skin Structure Infection T1DM Type 1 Diabetes Mellitus T2DM Type 2 Diabetes Mellitus TBD To Be Determined TEAE Treatment-Emergent Adverse Event TNBC Triple Negative Breast Cancer TNF Tumor Necrosis Factor TNFα Tumor Necrosis Factor-alpha UA Unstable Angina UC Ulcerative Colitis US United States UTI Urinary Tract Infection VAS Visual Analog Scale VEGF Vascular Endothelial Growth Factor VTE Venous Thromboembolism WBC White Blood Cell WHO World Health Organization XR Extended-Release

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MRx PIPELINE A VIEW INTO UPCOMING SPECIALTY & TRADITIONAL DRUGS

JANUARY 2022

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