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Ionis

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pharmaceuticals2 years ago

AstraZeneca and Ionis report Phase III success with eplontersen, a potential $3B drug.

AstraZeneca and Ionis have announced positive Phase III results for eplontersen, a drug for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), a fatal neurodegenerative disease. Patients taking eplontersen recorded a 0.28-point average increase from baseline compared to a 25.06-point average increase in the placebo group. The drug is expected to generate over $3 billion in sales.

pharmaceuticals2 years ago

FDA convenes advisory panel to review BrainStorm's ALS therapy NurOwn

BrainStorm Cell Therapeutics' ALS treatment, NurOwn, will be discussed in an FDA advisory committee meeting after receiving a refusal to file letter in November. Ionis and AstraZeneca's rare disease drug, eplontersen, has completed a positive Phase III trial. Codiak BioSciences has filed for Chapter 11 bankruptcy. ArriVent Biopharma has raised $155 million in a Series B round. Endpoints has added three new journalists to their newsroom. Kristen Hege, a cancer researcher, has put her career on hold to hike the Pacific Crest Trail. Another biotech startup has faced a Phase II pileup in Parkinson's disease.