Ionis Pharmaceuticals’ olezarsen has Phase 3 results showing the therapy handily beat a placebo at reducing fat levels in the blood due to a rare, inherited metabolic disorder with no FDA-approved drugs. Ionis plans early 2024 submissions for what could become the first medicine it commercializes without a partner.

Novartis and Ionis Pharmaceuticals, already partners in cardiovascular disease R&D, are expanding their alliance to see if they can develop a next-generation therapy offering efficacy and dosing advantages. But competitors are already in the mix with programs addressing the same cardiovascular disease target.

An Ionis Pharmaceuticals heart disease drug beat a placebo in a mid-stage clinical trial, but partner AstraZeneca has decided it won’t advance the genetic medicine to a larger Phase 3 test. The results were apparently not enough to set the Ionis therapy apart from commercialized cholesterol-lowering drugs that address the same protein target.

An Ionis Pharmaceuticals drug has positive Phase 2 results in immunoglobulin A nephropathy, leading Roche to exercise its option to license the rights to the molecule. The pharmaceutical giant gets a contender in the chase to win the first regulatory approval of a therapy for this rare kidney disease.

The planned FDA submission follows the report from AstraZeneca and Ionis Pharmaceuticals that their partnered drug, eplontersen, met the main goals of a pivotal study in treating nerve pain caused by hereditary transthyretin-mediated amyloidosis. The data come one week after rival Alnylam Pharmaceuticals won FDA approval for its second drug for this rare disease.

The INVEST Precision Medicine conference cast a spotlight on the role of children’s hospitals, biopharma companies, diagnostic companies and startups in biopharma innovation. The video recordings of the conference sessions are now available to view.

Targeted therapies for certain types of cancer and rare disease offer new hope for these patient populations. But how can we ensure that these treatments are also developed with minority communities in mind?

Register for the INVEST Precision Medicine conference, which kicks off today. Over the next three days industry experts will talk about precision medicine developments across pediatrics, bioinformatics, startups and more.

French firm to start trial of Dyn101, in-licensed from US-based Ionis Pharmaceuticals, in centronuclear myopathy next year.

Ionis and Biogen have secured FDA approval for their spinal muscular atrophy drug, working closely with the FDA and tapping into a trifecta of incentives — including fast-track designation, priority review and orphan drug designation.