Phase II
BridgeBio posted data Monday from its small but highly anticipated Phase II study of infigratinib in achondroplasia - a drug that could give BioMarin a run for its money.
Merck presented Phase IIb data at ACC Monday showing that its hypercholesterolemia hopeful met the primary endpoint.
Moderna may seek the FDA’s accelerated approval for its personalized cancer vaccine, president Stephen Hoge said Monday.
Shares of Praxis Precision Medicines dropped 48% Friday morning following mixed Phase II results for ulixacaltamide (PRAX-944), its candidate being studied in movement disorder essential tremor.
Aptinyx’s NYX-458 fell short of the primary endpoint in a Phase II trial of patients with cognitive impairment associated with Parkinson’s disease and dementia with Lewy bodies.
The hits kept coming for the sickle cell disease space last week as the FDA placed a full clinical hold on Fulcrum Therapeutics’ Investigational New Drug application FTX-6058.
Rezpegaldesleukin, Lilly’s and Nektar Therapeutics’ candidate for systemic lupus erythematosus, fell short of its primary efficacy endpoint in the Phase II ISLAND trial.
Novartis recently announced it is returning a Phase II-ready nonalcoholic steatohepatitis asset to Pliant. The program stemmed from a 2019 collaboration.
There are currently no disease-modifying treatments for Friedreich’s ataxia. That could change when the FDA makes a decision about Reata Pharmaceuticals’ omaveloxolone on Feb. 28.
Novartis has abandoned its ex vivo sickle cell disease program developed using Intellia Therapeutics’ CRISPR gene editing platform, Intellia announced Thursday.
PRESS RELEASES