Daily Snap - 22. September 2025

Author

Joachim Eeckhout
September 22, 2025

 

Good morning! A new study by the Tufts Center for the Study of Drug Development found that roughly one-third of the data collected in 105 phase 2 and 3 clinical trials was unnecessary for supporting primary or secondary endpoints. This “non-core” and “non-essential” data collection contributes to 25–30% of the burden on trial participants, including increased travel and time commitments. The study points to increasing trial complexity over the past 25 years as a key factor behind the rise in unnecessary data. It’s a timely reminder that, even in science, sometimes less really is more.

Enjoy today’s read!

—Joachim E.

SNIPPETS

What’s happening in biotech today?

✅EU greenlight: The European Commission has approved Tryngolza (olezarsen), developed by Swedish Orphan Biovitrum (Sobi) and Ionis Pharmaceuticals, as an adjunct to diet for treating familial chylomicronaemia syndrome (FCS) in adults with genetically confirmed diagnoses. This RNA-based therapy, targeting apoC-III, is administered monthly via an 80-mg subcutaneous autoinjector. It becomes the second FCS treatment approved in the EU, following Sobi’s earlier drug Waylivra. Approval was based on the Phase III Balance study, which showed significant and sustained reductions in triglyceride levels and acute pancreatitis events. Ionis markets Tryngolza in the US, while Sobi holds rights in Canada, China, and other non-US markets.

⚠️Safety scare: Genfit SA has discontinued Phase II development of its lead drug candidate VS-01, a liposomal ammonia scavenger originally developed by Versantis AG for treating acute-on-chronic liver failure (ACLF), due to safety concerns following a serious adverse event in the UNVEIL-IT clinical trial. Despite the independent Data Monitoring Committee allowing the study to proceed with additional oversight, Genfit decided to halt both the ACLF trial and a related proof-of-concept study in hepatic encephalopathy, citing concerns over the benefit-risk ratio. The company will instead reprioritize VS-01 for preclinical development in urea cycle disorder (UCD) and accelerate four other ACLF-focused drug candidates.

💉Keytruda remix: The FDA has approved Merck & Co.'s Keytruda Qlex, a subcutaneous formulation combining pembrolizumab and berahyaluronidase alfa-pmph, for the treatment of solid tumors in adults and children aged 12 and older, matching the indications of the existing intravenous Keytruda. Developed with Alteogen’s berahyaluronidase alfa, the new version must be administered by a healthcare provider and is expected to launch in the U.S. in late September 2025. With Keytruda generating $8 billion in Q2 2025 and facing patent expiration in 2028, the new formulation is seen as essential for maintaining Merck’s revenue stability.

🧬RNA wrangling: Japan-based xFOREST Therapeutics has entered a research partnership with Axcelead Drug Discovery Partners (DDP) to develop RNA structure-targeted small molecule therapeutics, including mRNA candidates. The collaboration will leverage xFOREST’s drug discovery platform, featuring an RNA structure library and high-throughput screening technology, alongside Axcelead’s expertise in small molecule drug discovery. The goal is to identify compounds targeting RNA structures typically deemed undruggable, thereby enhancing research efficiency and increasing the chances of successful candidate development. xFOREST will retain exclusive development and commercialization rights, while Axcelead DDP is eligible for milestone payments. Financial terms were not disclosed.

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