With Roche's oral spinal muscular atrophy therapy Evrysdi off to a strong start after its recent US launch, Novartis is switching its priorities for rival RNA-splicing drug branaplam and putting it into a Phase IIb trial for Huntington's disease.
Novartis AG is planning to evaluate its investigational oral spinal muscular atrophy (SMA) drug branaplam as a treatment for Huntington's disease (HD), suggesting that the future prospects for the therapy may be best served by focusing on this other devastating inherited neurodegenerative condition instead.
Branaplam, previously known as LMI070, is a once-weekly, orally administered, small molecule RNA splicing modulator that Novartis has been developing...
Biogen hopes to move directly from Phase I to Phase III with salanersen, a higher-potency therapy that uses the same mechanism of action as Spinraza.
Oral RNA splicing modulator has gone into a Phase II/III trial
Wales-based group launches with $140m series A.
Supernus agreed to pay $561m upfront plus a contingent value right that could add $234m to buy Sage, topping a previously rejected offer from Biogen.
The late-stage biotech posted results from the maintenance extension portion of its Phase IIb QUALITY trial in which enobosarm reduced weight regain and helped preserve lean mass in patients who discontinued their semaglutide weight-loss treatment.
GSK tells Scrip about timelines for data expected from two Phase II trials for its Bharat Biotech-partnered shigella vaccine candidate, which puts it ahead of Limmatech/Valneva. Meanwhile, a global-first malaria vaccine, also partnered with Bharat, sees an over 50% price cut
The drugmaker announced positive topline results for the drug in hemophilia A and B patients with inhibitors, on top of the existing approval for patients without inhibitors.
