Earnings updates, regulatory decisions, and pipeline developments continue to drive activity in the biotech sector. Several companies have recently announced significant updates impacting their stock performance and future prospects.
Moderna Beats Earnings Expectations, Awaits RSV Vaccine Approval
Moderna (MRNA) reported Q4 earnings of 55 cents per share, exceeding the Zacks Consensus Estimate of a loss of 78 cents per share. The company's revenues of $2.8 billion also surpassed expectations, primarily driven by sales of its COVID-19 vaccine, Spikevax. Moderna reaffirmed its product sales guidance of approximately $4 billion for 2024 and anticipates initial regulatory approvals for its RSV vaccine (mRNA-1345) in the first half of 2024.
Iovance's Amtagvi Receives Accelerated FDA Approval for Melanoma
Iovance Biotherapeutics, Inc. (IOVA) saw its shares surge following the FDA's accelerated approval of lifileucel suspension for intravenous infusion, marketed under the brand name Amtagvi. This tumor-derived autologous T cell immunotherapy is indicated for the treatment of adult patients with unresectable or metastatic melanoma who have been previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor. The approval was based on safety and efficacy data from the phase II C-144-01 study, which demonstrated deep and durable responses in this patient population. Iovance is currently evaluating Amtagvi in frontline advanced melanoma in the phase III confirmatory study, TILVANCE-301, as well as in additional solid tumor types. The accelerated approval was based on overall response rate and duration of response.
RAPT Therapeutics Faces Clinical Hold on Zelnecirnon Trials
RAPT Therapeutics, Inc. (RAPT) experienced a significant stock decline after the FDA placed a clinical hold on its phase IIb trial of zelnecirnon (RPT193) in atopic dermatitis and its phase IIa trial in asthma. The hold was prompted by a serious adverse event of liver failure in one patient in the atopic dermatitis trial. While the cause is currently unknown, it has been characterized as potentially related to zelnecirnon. Dosing of zelnecirnon has been halted in both clinical trials, and enrollment of new trial participants has been paused. The clinical hold does not apply to RAPT’s ongoing trial of tivumecirnon (FLX475) in oncology. According to the company, approximately 350 patients have been enrolled across three clinical studies evaluating zelnecirnon. No evidence of liver toxicity has been observed with any other trial participant, nor in nonclinical studies.
Sarepta's Elevidys BLA Supplement Granted Priority Review
Sarepta Therapeutics (SRPT) saw its shares rise after the FDA accepted its efficacy supplement to the biologics license application (BLA) seeking to expand the treatment label for its Duchenne muscular dystrophy (DMD) drug, Elevidys. The FDA has assigned Priority Review status to the filing, with a review goal date of June 21, 2024. Elevidys was initially approved in June 2023 under the accelerated pathway to treat ambulatory pediatric patients aged four to five years with DMD. The efficacy supplement seeks to expand the labeled indication for Elevidys to treat all DMD patients, irrespective of age and ambulation status. Subject to approval, it will also convert the Elevidys accelerated approval to a traditional approval. The FDA has confirmed that it will not hold any advisory committee meeting to discuss the efficacy supplement to the Elevidys BLA.
Regeneron's Myeloma Drug Gets Priority Review
Regeneron Pharmaceuticals, Inc. (REGN) announced that its biologics license application (BLA), seeking approval of linvoseltamab for the treatment of adult patients with relapsed/refractory (R/R) multiple myeloma (MM) that has progressed after at least three prior therapies has been accepted by the FDA. The regulatory body has granted Priority Review to the same with a target action date of Aug 22, 2024. The BLA is supported by data from a phase I/II pivotal trial (LINKER-MM1) investigating linvoseltamab in R/R MM. The linvoseltamab clinical development program includes a phase III confirmatory trial in patients with R/R MM (LINKER-MM3) that is currently enrolling. Additional trials in earlier lines of therapy and stages of disease are planned or underway, including a phase I/II study in the first-line setting, a phase II trial in high-risk smoldering MM and a phase II trial in monoclonal gammopathy of undetermined significance. A phase I study of linvoseltamab, in combination with a Regeneron CD38xCD28 costimulatory bispecific in MM, is also planned.
