Amid the increasingly competitive myasthenia gravis drug development space, siRNA candidate cemdisiran met phase III endpoints, with the monotherapy showing numerically higher results than a combination product. Regeneron Pharmaceuticals Inc., which has a worldwide license to cemdisiran from Alnylam Pharmaceuticals Inc., plans to file for U.S. approval of the monotherapy in the first quarter of 2026. In the phase III Nimble trial, cemdisiran monotherapy reduced circulating levels of complement factor 5 by 74%, while also showing a 2.3-point placebo-adjusted improvement in Myasthenia Gravis Activities of Daily Living total score. If approved, original developer Alnylam Pharmaceuticals Inc. would be entitled to tiered double-digit royalties of up to 15% on net sales.

Lilly scores again in phase III with oral GLP-1 orforglipron

Eli Lilly and Co. made known positive top-line results from the phase III Attain-2 trial testing orforglipron, an oral glucagon-like peptide 1 (GLP-1) receptor agonist, in adults with obesity or overweight and type 2 diabetes. All three doses met the primary and all key secondary endpoints, delivering significant weight loss, meaningful A1C reductions and improvements in cardiometabolic risk factors at 72 weeks. For the primary endpoint, orforglipron 36 mg taken once per day without food and water restrictions lowered weight by an average of 10.5% (22.9 lbs.) compared to 2.2% (5.1 lbs.) with placebo, using the efficacy estimand. It’s the second successful phase III study with Lilly’s GLP-1 prospect.

Osivax awarded $19.5M by BARDA for universal influenza A vaccine

With U.S. vaccines developers facing disruption over shifts in government policy, French vaccines specialist Osivax SAS has been awarded $19.5 million by the U.S. Biomedical Advanced Research and Development Authority (BARDA), to fund work on its universal influenza A vaccine. The product, OVX-836, is due to enter a phase IIb trial in Europe during the northern hemisphere 2025-2026 flu season, and the BARDA award will shape up product for stockpiling for pandemic preparedness.

Beone sells off Amgen Imdelltra royalties for $950M

Beone Medicines Ltd. (formerly Beigene Ltd.) is selling its worldwide royalty rights on Imdelltra (tarlatamab) sales, excluding China, to Royalty Pharma for up to $950 million. Under terms of the deal, Royalty pharma will pay Beone $885 million up front, and Beone has the option to sell the remaining royalties within a year for up to $65 million. Beone will share in a portion of the royalties on annual sales above $1.5 billion and will maintain royalties and all other rights to other assets under the terms of its existing collaboration with Amgen Inc.

Akeso’s gumokimab, manfidokimab meet phase III endpoints

Akeso Pharmaceuticals Inc.’s gumokimab (AK-111) and manfidokimab met all primary efficacy endpoints in its phase III trials in active ankylosing spondylitis and in moderate to severe atopic dermatitis, respectively. Gumokimab already had shown positive results in a phase III trial for moderate to severe psoriasis, and China’s National Medical Products Administration accepted Akeso’s NDA in January 2025. The NDA submission is Akeso's third non-oncology drug to enter the NDA review process.

Newco news: Arnatar emerges with RNA approach to both silence and restore

A few years after it was founded with the aim of taking RNA therapies to the next level, Arnatar Therapeutics Inc. emerged from stealth, disclosing a $52 million series A round raised in 2024 as well as U.S. FDA orphan and rare pediatric disease designations for ART-4, an antisense oligonucleotide candidate targeting the root cause of Alagille syndrome.

Also in the news

Akeso, Amgen, Argenx, Arnatar, Artelo, Ayrmid, Beone, Catalyst, Eli Lilly, Hutchmed, I-Mab, Immuneering, Incyte, ITF, Lupin, Mannkind, Oculis, PDS, Scpharmaceuticals, Serb, Sonoma, Valneva