Ionis Pharmaceuticals

The FDA approved Ionis Pharmaceuticals' drug, olezarsen (Tryngolza), for familial chylomicronemia syndrome (FCS), a rare disorder affecting fat breakdown. It's the first treatment for FCS, aimed at reducing triglycerides alongside a low-fat diet. Affecting fewer than 5,000 in the U.S., the drug's pricing and supply are ready, with peak sales estimated at $341 million.

FDA approved Ionis Pharmaceuticals' Tryngolza (olezarsen) for adults with familial chylomicronemia syndrome (FCS) to reduce triglycerides, a condition leading to acute pancreatitis. Tryngolza, self-administered monthly, significantly lowers triglyceride levels and reduces AP events, based on Phase 3 trial data. It will be available in the U.S. by year-end.

Editas Medicine lays off 65% of staff, shelving its sickle cell disease treatment Reni-Cel; Ionis Pharmaceuticals shifts to in-house drug commercialization; GLP-1 drugs spark debate in Trump's circle; investors sue Bristol Myers Squibb over Padlock Therapeutics milestone payments.

Ionis Pharmaceuticals awaits FDA decision on olezarsen by Dec. 19, a therapy for familial chylomicronemia syndrome with potential for broader use. CEO Brett Monia shifted strategy to develop drugs independently, aiming to maximize value for both company and patients.

UniQure aligned with FDA on accelerated approval pathway for Huntington’s disease gene therapy AMT-130, using Phase I/II studies and composite Unified Huntington’s Disease Rating Scale as primary basis for Biologics License Application (BLA).

PCSK9 inhibitors, targeting cholesterol levels, are gaining traction with market growth anticipated from 2020-2034. Key companies include Innovent Biologics, Amgen, and AstraZeneca, with therapies like Tafolecimab and Evolocumab. The market is driven by increasing prevalence of hypercholesterolemia and cardiovascular diseases, with significant advancements in pipeline products.

Theratechnologies Inc. partners with Ionis Pharmaceuticals to license olezarsen for familial chylomicronemia syndrome and severe hypertriglyceridemia, and donidalorsen for hereditary angioedema, with submissions to Health Canada planned for 2025.

Novartis pays $1 billion upfront to license PTC518, a Huntington's disease drug, with potential for $1.9 billion in milestone payments and royalties. PTC retains 40% of U.S. profits and leads Phase 2 completion and FDA discussions. The deal is expected to close in Q1 2025.

Alex Martinez, CEO of Intrinsic Medicines, is developing human milk oligosaccharide (HMO) drugs to treat gut immune microbiota brain axis disorders, with Parkinson’s and IBS in phase 2 trials. His personal experience with IBS drives his focus on the human, subjective experience. Intrinsic Medicines aims to leverage synthetic HMOs for a breakthrough class of drugs, targeting complex diseases with high unmet needs. The company plans to start a phase 2 trial of its lead candidate OM002 in Parkinson’s disease in early 2024, exploring the drug’s impact on constipation and associated dysbiosis.

Praxis Precision Medicines to present preclinical and clinical data on relutrigine, vormatrigine, and elsunersen at AES 2024, targeting CNS disorders and epilepsy.