Huntington’s filing gets a May date, uniQure says

Five things to know

1. Must readFDA accepted uniQure’s Huntington’s gene therapy filing with Priority Review and set a goal date of 05.01.2027, uniQure says. FDA is tentatively planning an advisory committee, which would test whether a comparison with natural history data can carry an accelerated approval. Acceptance is not approval.Partial NCT04120493 (N=43) and NCT05243017 (N=14) both register safety as the primary; the release’s BLA case rests on 36-month data against an external Enroll-HD control that neither record names, and the re#
2. Must readKaryopharm filed for Chapter 11 and plans a merger with Ayrmid as lead bidder, it says. Lenders committed up to $55M in financing, subject to court approval; no price was disclosed.#
3. Must readA sirolimus-coated balloon was non-inferior to a drug-eluting stent at one year in 1,842 patients, the trial’s release says. Target lesion failure was 2.1% vs 2.4%, but any repeat procedure on the treated lesion or vessel was more than twice as common with the balloon.Partial NCT04893291 registers 1:1 randomization and target lesion failure at 12 months as the primary, N=1,820 at 53 listed sites; the release reports 1,842 patients at 53 centers. The record also lists net a#
4. Know itEurope and Japan accepted filings for savolitinib plus osimertinib in MET-driven lung cancer, HUTCHMED says. The SAFFRON trial beat chemotherapy on overall survival; the release doesn’t say whether FDA has accepted the U.S. filing.#
5. Know itAn oral TYK2 inhibitor, nomelcitinib, met its Phase 3 goals in psoriasis, InventisBio says. More than 70% reached PASI 90 at week 16, but the release gives no placebo rates, doses or safety numbers.#

Bright spot

Eton licensed rights to test mecasermin, a form of the growth factor IGF-1, in babies with infantile spasms. A Phase 2 of up to 20 children is planned for early 2027; so far the support is animal data only.