The U.S. FDA has greenlit the first steps of Uniqure NV’s accelerated approval pathway for gene therapy AMT-130 to treat Huntington’s disease. The agency said data from the ongoing phase I/II studies compared to natural history external control are muscular enough to get the process going without having to dive into additional studies. Read More
In a surprise move that drove its stock up by 292% in early trading, Chimerix Inc. revealed plans for a U.S. NDA filing by year-end, seeking accelerated approval of dordaviprone (ONC-201) to treat recurrent H3 K27M-mutant diffuse glioma, a highly aggressive tumor with limited treatment options. Read More
Relation Therapeutics Ltd. has delivered the first commercial validation of its combined wet lab/in silico platform for homing in on disease-related genes in the thick of the genome, signing two collaboration agreements with GSK plc, with a potential value of $200 million per target. Read More
Four Chinese biopharmaceuticals unveiled early data on respective cancer therapies at the American Society of Hematology (ASH) 2024 meet in San Diego, including Cstone Pharmaceuticals Co. Ltd.’s receptor tyrosine kinase-like orphan receptor 1 (ROR1)-inhibiting antibody-drug conjugate (ADC) in phase I study for lymphomas. Read More
At the 66th American Society of Hematology Annual Meeting, a plethora of companies presented clinical trial data highlighting their drugs targeting Bruton tyrosine kinase (BTK) in patients with blood cancers. Read More
Bionomics is progressing BNC-210 to phase III trials in post-traumatic stress disorder (PTSD) following positive feedback from the U.S. FDA. Final data from the phase II Attune study showed that BNC-210 improved PTSD symptom severity at week 12 with efficacy observed as early as week four. Read More
If a bipartisan group of U.S. House members has its way, at least some of the legislation congressional committees have passed to target pharmacy benefit manager (PBM) practices could yet make it into law this year. That is if congressional leaders listen to the rank-and-file members. Read More
Cervomed Inc. executives said they intend to scrutinize low plasma drug concentrations that appeared to spoil results from the phase IIb Rewind-LB trial testing neflamapimod in patients with dementia with Lewy bodies. Read More
The U.S. SEC filed charges against David Banister and The Market Analysts Group LLC, which Banister controls, alleging that they conducted a fraudulent scheme to promote long-term investment in Biovie Inc. without disclosing that Banister was actively selling his own shares in the biopharma company. Read More
New hires and promotions in the biopharma industry, including: Bicycle, Camp4, Corrixr, Eikonizo, Incarda, Kling, Klotho, Newbiologix, Proqr, Silexion. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Astrazeneca, Chiesi, Crinetics, Daiichi, GSK, Protalix. Read More
