Novartis Deepens Argo Collaboration with $5.2B siRNA Pact
Summary:
Novartis has struck a new deal with Shanghai-based Argo Biopharma, investing $160 million upfront and committing up to $5.2 billion in combined milestone and option payments for multiple cardiovascular-focused siRNA assets, plus tiered royalties. This latest deal builds on a January 2024 agreement, which included $185 million upfront and up to $4.165 billion in milestones.
Encoded’s ETX101 Earns FDA RMAT Designation for Dravet Syndrome
Summary:
Encoded Therapeutics’ gene therapy ETX101, designed to boost SCN1A expression in GABAergic neurons for SCN1A-positive Dravet syndrome, has received FDA RMAT designation based on preliminary seizure reduction data from its ongoing Phase 1/2 POLARIS trial. ETX101 already holds Fast Track, Rare Pediatric, and Orphan Drug designations from the FDA, plus Orphan Drug status from the EMA. Encoded has also opened a GMP facility in North Carolina to produce clinical supply and plans to share interim efficacy data later this year.
Nanoscope’s MCO-010 Wins Key FDA and EMA Support
Summary:
Nanoscope’s gene therapy MCO-010 secured FDA RMAT designation for Stargardt disease and five EMA Orphan Drug designations spanning multiple retinal dystrophies. These milestones strengthen its global regulatory path as a potential one-time, in-office treatment for vision restoration.
Cytokinetics’ Aficamten Delivers Best-in-Class Phase III Results
Summary:
In the Phase III MAPLE-HCM trial presented at the European Society of Cardiology Congress, Cytokinetics’ investigational drug aficamten increased patients’ peak oxygen uptake (pVO2) by 1.1 mL/kg/min after 24 weeks, while standard beta-blocker metoprolol caused a 1.2 mL/kg/min decline. Analysts at Truist Securities labeled these outcomes “robust” and suggest aficamten could emerge as a potential best-in-class first-line therapy for obstructive hypertrophic cardiomyopathy. Aficamten is under FDA review, with a decision expected by December 26, 2025.
Trump’s API Push Welcomed, But Questions Remain
Summary:
President Trump ordered the U.S. to build a six-month stockpile of key drug ingredients, prioritizing domestic sources. Industry groups applaud the move for strengthening supply chains, but call for payment reforms to ensure access. Analysts warn costs of reshoring APIs may be high and details on implementation remain unclear.
