A top-line readout of the 26-week phase IIa Cbeyond trial showed nimacimab, Skye Bioscience Inc.’s peripherally restricted CB1 inhibitor antibody for weight loss, fell short of statistical significance as a monotherapy vs. placebo on the primary endpoint of weight loss, sending the company’s shares down 60%. Skye executives, however, offered a more optimistic outlook for the findings, which they said provide clear direction for moving forward.
Patients with chronic spontaneous urticaria who remain symptomatic despite H1 antihistamine treatment now have a new oral treatment option, with the U.S. FDA’s approval of Novartis AG’s Rhapsido (remibrutinib), a Bruton’s tyrosine kinase inhibitor administered by pill twice daily.
Pfizer Inc. has become the first drugmaker to agree to provide its products at most-favored nation (MFN) pricing, an effort aimed at lowering the costs of U.S. drug prices by bringing them in line with the prices paid in other developed nations.
Genmab A/S is paying $8 billion to acquire Merus NV, gaining rights to the latter’s phase III-stage petosemtamab, a bispecific antibody initially in development for head and neck cancer, and continuing the firm’s strategy to transition from a royalty-based operation to a fully integrated biopharma.
As the first – and so far only – drug to enter clinical testing for the rare neurogenetic disorder Alexander disease, there were some unknowns heading into the readout of the pivotal study testing Ionis Pharmaceuticals Inc.’s zilganersen in children and adults. But the top-line data yielded a clear win for the antisense oligonucleotide candidate, which demonstrated a disease-modifying impact, including statistical significance on the primary endpoint of gait speed as assessed by the 10-Meter Walk Test vs. control at week 61.
Renewing hopes of restoring the rare pediatric disease priority review voucher (RPD PRV) program that expired at the end of 2024, the House Energy and Commerce Committee voted 47-0 Sept. 17 to advance the Give Kids a Chance Act of 2025 (H.R. 1262), one of six pieces of legislation slated to move to the full U.S. House for consideration.
With positive phase III results in hand, Roivant Sciences Ltd. and Priovant Therapeutics Inc. anticipate an NDA filing in the first half of 2026, seeking approval for brepocitinib, a dual inhibitor of tyrosine kinase 2 and JAK1, in dermatomyositis, a rare autoimmune disease with limited treatment options.
To say the team at Ollin Biosciences Inc. has some experience in ophthalmology would be an understatement. The company, which emerged from stealth with $100 million in venture capital and two in-licensed assets with plans to add more, brings to the table years of expertise from work on blockbuster retinal disease drugs Lucentis (ranibizumab, Roche AG) and Vabysmo (faricimab, Roche AG).
When it won U.S. FDA accelerated approval more than eight years ago, Intercept Pharmaceuticals Inc.’s Ocaliva (obeticholic acid) was viewed as a breakthrough, becoming the first new treatment in 20 years for rare, progressive liver disease primary biliary cholangitis (PBC) and, for several years, the only second-line treatment for PBC patients failing to respond to ursodeoxycholic acid. More recently, however, Ocaliva has faced regulatory and safety stumbles, with Intercept now voluntarily pulling the farnesoid X receptor activator from the U.S. market.
PMV Pharmaceuticals Inc.’s rezatapopt yielded impressive overall response data in ovarian cancer, prompting the firm to aim for a potential accelerated approval filing in early 2027. The news caused the firm’s stock (NASDAQ:PMV) to jump in premarket trading, but shares ended the day at $1.27, down 20%, as investors signaled they are clearly holding out for further data confirming broader market potential for the p53-targeting small molecule.
