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.
Nervgen Pharma Corp. rolled out expanded findings from the Connect phase Ib/IIa study with NVG-291 in spinal cord injury patients showing that the 35-amino acid peptide derived from the intracellular wedge domain of phosphatase sigma provided durable functional gains that continued at week 16 and after.
Celltrion Inc. announced Oct. 29 the signing of an $87.75 million joint drug R&D agreement with AI and spatial transcriptome-based biotech Portrai Inc.
Five-year follow-up data for Santhera Pharmaceuticals AG’s Duchenne muscular dystrophy (DMD) drug, Agamree (vamorolone), confirm that its efficacy in preserving muscle function is comparable to standard-of-care corticosteroids, but that the overall side-effect profile is more benign. There was less positive DMD news from Sarepta Therapeutics Inc.
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