Keyword: Alnylam Pharmaceuticals
Alnylam is gearing up to move RNAi therapies for central nervous system disorders into the clinic.
The initiative taps into the networks Invitae has built up to connect sponsors to patients and the clinicians responsible for their care.
Akcea suffered the setback after the FDA asked for more time to review its response to requests for information on the rare disease antisense drug.
Details on Westphal's new venture are sketchy, with a corporate filing saying the company is "developing immunotherapy treatments for cancer."
The success positions Alexion to file for approval and shield itself from an anticipated assault on its paroxysmal nocturnal hemoglobinuria franchise.
The new angle comes from an exploratory post hoc analysis which linked the RNAi therapy to a 50% drop in hospitalization and mortality over placebo.
Dicerna and Alnylam are settling a yearslong spat over trade secrets relating to gene-silencing technology.
Pfizer reported positive results from a phase 3 trial of its tafamidis in transthyretin cardiomyopathy, a form of ATTR.
If the pair finds potential treatments in the discovery deal, they will ink a new collaboration to advance them.
The French pharma’s decision gives Alnylam control of a phase 3-ready rare disease drug with an FDA breakthrough therapy designation.