The U.S. FDA provided Sarepta Therapeutics Inc. with the best possible scenario in the full approval of gene therapy Elevidys (delandistrogene moxeparvovec) for Duchenne muscular dystrophy (DMD). Shares of the Cambridge, Mass.-based firm were trading at $165.36, up $41.87, or almost 34% on the news. The compound is Sarepta’s fourth in DMD, which causes skeletal and heart muscle weakness that quickly gets worsens with time, afflicting mainly boys.

BioWorld Biopharmaceutical Index outpaces market benchmarks with an 8% rise

The BioWorld Biopharmaceutical Index saw an 8.15% increase at the close of May, outperforming the Dow Jones Industrial Average, which rose by 2.64%, and the Nasdaq Biotechnology Index, which was up by 0.7%. Eli Lilly and Co. emerged as the top performer in the first five months of 2024, with a 41% increase.

Aprilbio, Evommune strike $475M deal for autoinflammatory drug 

Aprilbio Co. Ltd. saw its stock surge 30% June 20 after announcing a potential $475 million (₩655.88 billion) out-licensing deal with Evommune Inc. for autoimmune disease drug candidate APB-R3. Under the terms of the deal, Gangwon-do, South Korea-based Aprilbio will out-license exclusive global rights of APB-R3 to Palo Alto, Calif.-based Evommune for a nonrefundable $15 million up-front payment, additional potential development milestone payments reaching $82.5 million and commercial milestone payments of up to $377.5 million, along with royalties based on net sales. APB-R3 is a recombinant anti-serum albumin Fab-IL-18 binding protein currently in an Australia-based phase I study for patients with adult-onset Still’s disease.

US FDA warning letter spells out expanded access duties 

Communication missteps, overreliance on a contract research organization and lack of clarity of U.S. FDA expectations for an expanded access protocol for emergency use resulted in a warning letter posted June 18 that highlights potential pitfalls for sponsor/investigators conducting the individualized studies.

Newco news: Yellowstone emerges with new T-cell receptor approach in AML

Newco Yellowstone Biosciences Ltd. has been formed to develop soluble bispecific T-cell receptors against a novel class of tumor-specific antigens it has discovered in leukemia patients who were cured by a donor stem cell transplant.

Two Eylea biosimilars among 11 FDA drug approvals in May 

In May, the U.S. FDA approved 11 new drugs, a significant drop from the 26 approvals in April, which was the sixth-highest monthly total since 2016. This also falls short of March’s record-setting 30 approvals, the highest number recorded by BioWorld. For context, the FDA approved an average of about 16 drugs per month in 2023, 12.5 per month in 2022, and 17 per month in both 2021 and 2020.

Also in the news

Aphaia, Āshibio, Astrazeneca, Atai, Aurion, Avextra, Cessation, Clearmind, Coeptis, Gilead, Innocare, Jazz, J&J, Kiromic, Medigene, Mendus, Mustang, Nephrodi, Neurotech, Pharmaessentia, PTC, Rapafusyn, Sab, Sanofi, Silence, Synqube, Tharimmune, Vanda, Vaxxinity