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.
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.
Interleukin 1 receptor accessory protein (IL1RAP) is a receptor that activates signaling pathways triggered by IL1α/β, IL33 and IL36α/β/γ, which are involved in several autoimmune or inflammatory disorders, including psoriasis, gout or systemic sclerosis. Innovent Biologics Co. Ltd. presented data for their anti-IL1RAP antibody IBI-3011, a fully humanized IgG1 antibody targeting IL1RAP that blocks those pathways.
Shooting for further proof of durable, drug-free, disease-free remission with a single dose of KYV-101 in generalized myasthenia gravis, Kyverna Therapeutics Inc. plans to start phase III work by the end of this year. The Emeryville, Calif.-based firm rolled out positive interim results from the phase II portion of the registrational Kysa-6 clinical trial testing the drug, a fully human, autologous, CD19 CAR T-cell therapy with CD28 costimulation.
Shanghai Institute of Materia Medica of the Chinese Academy of Sciences has disclosed discoidin domain-containing receptor 1 (DDR1) inhibitors reported to be useful for the treatment of systemic lupus erythematosus and rheumatoid arthritis.
Impressive data from an interim readout of Bridgebio Pharma Inc.’s BBP-418 in limb-girdle muscular dystrophy type 2I/R9 has the company prepping to meet with the U.S. FDA to discuss plans for the upcoming NDA filing, including the possibility for seeking full approval for what could be the first therapy for the rare muscular disease.
The $12.5 billion acquisition of Avidity Biosciences Inc. by Novartis AG strengthens the company’s neuroscience pipeline and marks the second biggest deal that’s been announced this year. It also is the fifth M&A deal in the past five weeks to top the $1 billion mark, a sign that the market may be strengthening.
Johnson & Johnson Medtech won out over a jury verdict that found the company’s Depuy Synthes liable for $20 million for infringing a patent claimed by Rasmussen Instruments LLC.
Arcus Biosciences Inc. has announced five new research programs for autoimmune and inflammatory diseases, and is targeting initiation of the first clinical studies next year.
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