Formation Bio Inc. acquired ex-China rights to Lynk Pharmaceuticals Co. Ltd.’s oral TYK2-inhibitor, LNK-01006, for up to $605 million. The phase I-ready central nervous system (CNS) candidate will be developed at Formation’s newly formed subsidiary, Bleecker Bio.
Sanofi SA reported more hitches in the development of Bruton’s tyrosine kinase inhibitor tolebrutinib, saying the phase III Perseus study failed to meet its primary endpoint in primary progressive multiple sclerosis (MS) and that the ongoing U.S. regulatory review in non-relapsing secondary progressive MS likely will extend beyond the targeted PDUFA date of Dec. 28.
Formation Bio Inc. acquired ex-China rights to Lynk Pharmaceuticals Co. Ltd.’s oral TYK2-inhibitor, LNK-01006, for up to $605 million. The phase I-ready central nervous system (CNS) candidate will be developed at Formation’s newly formed subsidiary, Bleecker Bio.
Shares of San Diego-based Capricor Therapeutics Inc. (NASDAQ: CAPR) closed Dec. 3 at $29.96, up $23.60, or 371%, as investors cheered top-line data from the pivotal phase III Hope-3 trial testing cell therapy deramiocel in Duchenne muscular dystrophy.
Alebund Pharmaceuticals (Hong Kong) Ltd. has described complement factor B (CFB) inhibitors reported to be useful for the treatment of glomerular disorders and rheumatoid arthritis.
Pseudoxanthoma elasticum (PXE) is an autosomal recessive connective tissue disorder caused by pathogenic variants in ATP-binding cassette subfamily C, member 6 (ABCC6). ABCC6 typically exports ATP, which is then converted by ENPP1 into AMP and pyrophosphate (PPi). Because PPi is a potent inhibitor of calcification, reduced systemic PPi production is a key driver of PXE. University of Pennsylvania investigators and collaborators proposed applying liver-targeted variant correction via genome editing as a single-intervention therapeutic approach for PXE, leading to subsequent restoration of systemic PPi.
Researchers from Stanford University have reported that inhibiting the enzyme 15-hydroxyprostaglandin dehydrogenase (15-PGDH) promoted cartilage regeneration in mouse models of osteoarthritis due to either aging or tissue injury. An oral version of the inhibitor that the team used is in a clinical trial for sarcopenia; it improved muscle mass and strength in preclinical studies. However, the mechanism by which 15-PDGH inhibition works appears to differ in the two conditions.
The U.S. Centers for Medicare & Medicaid Services (CMS) rolled out negotiated costs of the second batch of drugs subject to such bargaining under the Inflation Reduction Act. Wall Street was not surprised to learn that the numbers amount to much greater cuts than the Biden administration managed for 2026. CMS said the adjusted maximum fair prices would have achieved 44% lower net spending had they been implemented in 2024 – 36% if forgiven discounts from the part D redesign of the Medicare prescription drug benefit are figured in. Fifteen drugs are listed.
More than six years after Novartis AG’s Zolgensma was approved for children under 2 with spinal muscular atrophy (SMA) with biallelic mutations in the survival motor neuron 1 gene, the U.S. FDA cleared a new version, under the brand name Itvisma (onasemnogene abeparvovec), for those 2 and older, including teens and adults with the same mutation.
In a recent study published in Cell Reports Medicine, researchers from the Institute of Chinese Materia Medica of the China Academy of Chinese Medical Sciences and Xi’an Jiao Tong University Health Science Center found that Mesothelin (MSLN) was significantly elevated in rheumatoid arthritis (RA) patients and collagen-induced arthritis (CIA) rats and mice, where it drove osteoclast differentiation.
RSS
