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Mirum Pharmaceuticals announced that Livmarli oral solution met its primary endpoint in the Phase III MARCH study in young patients with progressive familial intrahepatic cholestasis.
BioMed X and Sanofi have inked an R&D collaboration to use digital data and artificial intelligence to predict the efficacy of first-in-class drug candidates using virtual patient populations.
PhaseBio plans to file Chapter 11 bankruptcy and sell off its bentracimab assets under a stalking horse arrangement with an unnamed “large pharmaceutical company.”
The FDA greenlit AstraZeneca’s tremelimumab in combination with its checkpoint inhibitor Imfinzi as a treatment for patients with unresectable hepatocellular carcinoma.
Alpine Immune Sciences has voluntarily terminated enrollment in two clinical trials of its immuno-oncology asset, davoceticept, after a second patient death.
Top-line data from Tricida’s VALOR-CKD trial showed veverimer failed to meet its primary endpoint and does not slow progression in patients with metabolic acidosis and CKD.
In a Phase III trial comparing Novartis’ experimental iptacopan against AstraZeneca’s Soliris and Ultomiris in paroxysmal nocturnal hemoglobinuria, iptacopan demonstrated superiority.
Myovant Sciences agreed to be acquired by Sumitovant Biopharma for $2.9 billion - three weeks after turning down a previous offer.
New data from Scholar Rock’s Phase II TOPAZ trial showed its investigational antibody apitegromab improves quality of life in types 2 and 3 spinal muscular atrophy patients.
The FDA released guidelines Thursday in an attempt to clarify processes for assessing a drug candidate’s efficacy while examining several endpoints, simultaneously.
Shares of Immunic stock have plunged more than 70% in premarket trading after an early peek at its Phase Ib psoriasis drug candidate has so far failed to distinguish itself from placebo.
Johnson & Johnson will lay off 64 employees from its Manhattan-based pharmaceutical supplies business in the early part of 2023.