The opening plenary abstract session at the 2020 annual meeting of the American Society of Human Genetics (ASHG) began with the definition of a new disease, identified through a new approach, and possibly leading to a new way to think about rheumatic diseases.
New data from a global phase II trial of Kiniksa Pharmaceuticals Ltd.'s monoclonal antibody, mavrilimumab, in the rare chronic inflammatory disease giant cell arteritis showed a 62% lower risk of flare in patients receiving the candidate vs. those given a placebo.
With a just-completed $100 million common stock financing, Flame Biosciences Inc. plans to target the inflammasome, a protein complex that sets off innate immune responses by activating the cytokines IL-1beta and IL-18.
DUBLIN – Roche Holding AG is taking a second shot on the NLRP3 inflammasome by acquiring Dublin-based Inflazome Ltd. for €380 million (US$447.8 million) up front plus undisclosed milestones linked to the progress of its two clinical-stage candidates inzomelid and somalix. In terms of cash on the table, the deal is the largest yet for a portfolio of drug candidates that acts on the NLRP3 inflammasome, and it represents a profitable exit for Inflazome’s investors, who put €55 million into the company over two funding rounds, in 2016 and in 2018.
Amytrx Therapeutics Inc., of Nashville, Tenn., founded in 2014 but coming out of stealth now, is developing a therapeutic for treating multiple inflammatory, autoimmune and metabolic diseases.
An FDA rejection of Galapagos NV's rheumatoid arthritis (RA) drug filgotinib, announced Aug. 18, was "disappointing and unexpected," CEO Onno van de Stolpe said, adding that "there is so much more" to his company's story and pipeline. Gilead Sciences Inc., the Belgian company's partner and a substantial backer of both the drug and the pipeline, said it would "evaluate the points raised" by the CRL, but continued to believe in the drug.
Shares of Unity Biotechnology Inc. (NASDAQ:UBX) fell 66.6% to $4.15 on Aug. 17 after UBX-0101, its one-time lead candidate, failed to best a placebo on an established measure of osteoarthritis pain in a phase II study.
The industry's expanding quest for protein degraders grew a little larger on July 9 with Sanofi SA tapping Kymera Therapeutics Inc. to advance two new protein degrader programs, one of which will target IRAK4 in patients with immune-inflammatory diseases and another for an undisclosed target. Each program could yield multiple therapies.
DUBLIN – Gesynta Pharma AB raised SEK190 million (US$20.6 million) in new funding to move GS-248, a selective inhibitor of microsomal prostaglandin E synthase-1 (mPGES-1), into a phase IIa trial in systemic sclerosis. The study is due to get underway toward the year end. “It’s going to be in the second half of the fourth quarter,” said Patric Stenberg, CEO of Lund, Sweden-based Gesynta.
PERTH, Australia – Melbourne-based regenerative medicine company Cynata Therapeutics Ltd. is gearing up to take its Cymerus mesenchymal stem cell (MSC) product candidate, CYP-004, into a phase III trial in osteoarthritis.
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