Phase I
Its reversible nature offers the potential for RNA editing to go beyond rare diseases, eliciting excitement and buy-in from large pharmas like GSK and Eli Lilly.
New Phase I/II trial results show that one more type 1 diabetes patient achieved insulin independence after treatment with Vertex Pharmaceuticals’ investigational stem cell therapy VX-880.
On Monday, Syndax Pharmaceuticals announced that its menin inhibitor revumenib met the goal in a pivotal leukemia study and stopped the trial early. Their stock price still dropped on the news.
Shares of the biotech startup jumped as much as 70% on Friday after the company reported promising early-stage results for a four-week trial that included only 24 patients.
Shares were up over 60% in premarket trading on news that the company’s anti-FcRn antibody exhibited dose-dependent reductions in IgG levels, drivers of inflammation in many autoimmune diseases.
The company has dropped its gene therapy candidate TSHA-120 for giant axonal neuropathy after the FDA reiterated the need for a randomized, double-blind, placebo-controlled trial.
Bristol Myers Squibb’s pipeline cuts, announced Thursday during its R&D Day, include a mid-stage drug candidate for nonalcoholic steatohepatitis and an anti-TIGIT solid tumor program.
During Wednesday’s annual R&D Day, Moderna said it is culling four programs from its pipeline, including two molecules that had been discontinued last year by AstraZeneca.
Biotech Beam Therapeutics has dosed the first patient in a Phase I/II study of its base-edited CAR-T therapy in relapsed or refractory T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma.
Data from the first-in-human trial showed that Eli Lilly’s muvalaplin is safe and can cut significant levels of lipoprotein(a), a risk factor for atherosclerosis and related cardiovascular diseases.
PRESS RELEASES