Shares in Uniqure NV fell sharply on inconclusive interim data from a U.S. phase I/II trial of its gene therapy for Huntington’s disease. The stock (NASDAQ:QURE) hit a six-year low during trading on June 21, ending the day at $11.62, down 40%. Read More
Following a three-month delay, the U.S. FDA approved under priority review a subcutaneous formulation of Argenx SE’s efgartigimod, offering patients a much more convenient way to treat generalized myasthenia gravis (gMG). Read More
In its newly issued complete response letter (CRL) to Aldeyra Therapeutics Inc., the U.S. FDA said there’s just not enough evidence of efficacy right now to approve ADX-2191, an injectable vitreous-compatible formulation of methotrexate to treat primary vitreoretinal lymphoma (PVRL). Read More
Empress Therapeutics Inc., a Flagship Pioneering firm, has come out of stealth with $50 million in initial funding, a new chief scientific officer and an ambitious plan to file multiple INDs for novel small-molecule compounds over the next two years. Read More
Many of the available treatments for the autoimmune conditions ulcerative colitis (UC) and Crohn’s disease come with drawbacks such as safety issues or inconvenient dosing regimens. Describing itself as a biomarker-driven immunoregulatory therapeutics company, Nimmune Biopharma Inc. is striving to fulfil what it perceives as a high need for safer, more effective options with candidates that target the lanthionine synthetase C-like 2 (LANCL2) pathway. Read More
The floodgates have opened for challenges to the new U.S. drug price negotiation process laid out in the Inflation Reduction Act (IRA) that was narrowly passed last year. Read More
The World Health Organization (WHO) recently gave an emergency use listing (EUL) for SK Bioscience Ltd.’s COVID-19 vaccine called Skycovione, a self-assembled nanoparticle vaccine that targets the SARS-CoV-2 spike protein. Read More
Shenzhen Salubris Pharmaceuticals Co. Ltd. received marketing approval from China’s NMPA for enarodustat as a treatment for anemic patients with chronic kidney disease (CKD) that are not on dialysis. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: GSK, Neogap, Novo Nordisk, Ose, Scynexis, Veru, Virpax. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Aardvark, Advantage, Agepha, Akebia, Aldeyra, Amarin, Amgen, Arcellx, Argenx, Aridis, ARS, Biologix, Boehringer Ingelheim, Geron, Pfizer, Precigen, Replay, Sonnet. Read More
