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 strong to get the process going. Sometime in the first half of 2025, Uniqure and the FDA will meet again to talk about a statistical analysis plan and CMC details. The nod lit the fuse on the company’s stock (NASDAQ:QURE) was up 110% at midday, with shares going for $15.39 each. Bayer AG threw up its hands in frustration regarding its own gene therapy for Huntington’s in November by discontinuing development of BV-101.

Chimerix accelerates US filing for ONC-201 in glioma; stock soars

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. The company expects to seek priority review, which, if granted, would place a PDUFA action date, potential approval and launch in the third quarter of 2025, about the same time interim overall survival results read out from the phase III confirmatory Action trial. It is also a year ahead of when investors expected an FDA approval decision. “If approved, dordaviprone would be the first FDA-approved therapy specific to this devastating disease, as well as one of the first molecularly defined approvals for any high-grade glioma, providing a critical new treatment option for patients,” said CEO Mike Andriole during an investor call. Chimerix’s stock, which reached a 52-week high of $3.39 early on Dec. 10, was trading midday at $2.64, up 207%.

Relation inks two GSK deals tackling osteoarthritis, fibrotic disease

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. To kick off the partnership, GSK is making an up-front payment of $25 million, of which $15 million is an equity investment. In addition, Relation is eligible for research costs of up to $63 million to generate validated target packages that GSK will then have the right to license. The $200 million headline figure refers to any programs that subsequently go all the way to market, at which point Relation also will be eligible to receive tiered royalties on net sales of products. The collaborations, in osteoarthritis and fibrotic diseases, will each involve multiple targets.

ASH 2024: Cstone’s ROR1 ADC chases Merck’s, plus China biotech data

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. Along with phase Ia/Ib data of Cstone’s ROR1 ADC, CS-5001, early study data from Chinese biopharmas posted Dec. 9 to Dec. 10 include: Abbisko Therapeutics Co. Ltd.’s phase II-stage small-molecule CSF-1R inhibitor, pimicotinib (ABSK-021), in chronic graft-vs.-host disease; Jacobio Pharmaceuticals Group Co. Ltd.’s phase I-stage BET inhibitor, JAB-8263, for myelofibrosis; and Dizal Pharmaceutical Co. Ltd.’s pooled analysis of ongoing phase I/II studies of DZD-8586, an oral dual LYN/Bruton’s tyrosine kinase inhibitor for B-cell lymphomas.

ASH 2024: BTK inhibitor roundup

From large pharmaceutical companies like Astrazeneca plc and Eli Lilly and Co. to smaller companies like Beigene Co. Ltd. and Newave Pharmaceutical Inc., there were plenty of companies highlighting their drugs targeting Bruton tyrosine kinase (BTK) in patients with blood cancers at the 66th American Society of Hematology Annual Meeting. BioWorld offers a roundup of companies looking to compete with currently approved BTK inhibitors, Calquence (acalabrutinib, Astrazeneca), Imbruvica (ibrutinib, Johnson & Johnson and Abbvie) and Brukinsa (zanubrutinib, Beigene).

Bionomics progresses BNC-210 to phase III in PTSD

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 were presented at the American College of Neuropsychopharmacology meeting in Arizona on Dec. 9, which showed that BNC-210 improved PTSD symptom severity at week 12 with efficacy observed as early as week four. Data were also published in NEJM Evidence.

US lawmakers launch year-end push for PBM reforms

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. Led by Rep. Buddy Carter, R-Ga., 120 House members signed on to a Dec. 6 letter asking House and Senate leadership to ”prioritize PBM reform during end-of-year negotiations and ensure that the bipartisan efforts we have worked on throughout the 118th Congress are enacted into law.”

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