SNAPSHOT

Star Therapeutics shines bright with early trial results for its bleeding disorder drug

Star Therapeutics' experimental antibody, VGA039, showed up to 87% reduction in bleeding in von Willebrand disease (VWD) patients during a phase 1/2 trial, paving the way for a potentially game-changing phase 3 outcome.

Why it matters: VWD is the most common genetic bleeding disorder, yet treatments often fall short. VGA039 could redefine the standard of care, offering a more effective and less burdensome option for patients. This benefit could also expand to benefit sufferers of other bleeding disorders.

Backstory: VWD occurs when patients lack enough von Willebrand factor, a key clotting protein. Current treatments involve frequent infusions of clotting factor, often weekly or more, which aren’t always effective and are cumbersome.

Zoom in: VGA039 targets protein S, a natural anticoagulant, to help blood clot more easily. Phase 1/2 results showed 73–87% bleeding reduction in high-risk patients. One patient saw 100% bleed elimination after switching from daily infusions. Treatment is a once-monthly self-injection, showing strong tolerability. All treated patients have moved to an open-label extension trial.

Big picture: A successful outcome in the ongoing phase 3 trial could lead to FDA approval, making VGA039 the first treatment in its class and possibly opening doors for other bleeding disorder indications.

What’s next: Star is enrolling 60 patients in its phase 3 Vivid-6 trial, with results expected in 2028. The company plans to commercialize VGA039 independently if approved.

SNIPPETS

What’s happening in biotech today?

✅ Short trial: Praxis Precision Medicines announced positive efficacy results from its phase 2 Embold study of relutrigine, a sodium channel inhibitor targeting SCN2A-DEE and SCN8A-DEE, two rare and severe forms of pediatric epilepsies with no approved treatments. The study, which previously met its safety endpoint, was ended early following a data monitoring committee recommendation after demonstrating clear efficacy in the 16-participant trial. Praxis plans to discuss the data with the FDA in the coming weeks, with an approval application potentially slated for early 2026. The company’s stock surged 30% on the news, and additional trial data will be presented at the American Epilepsy Society meeting.

 🏙️ Shanghai growth: OTR Therapeutics, a Shanghai-based biotech founded in March 2025, has raised $100 million in a Series A financing round backed by Pfizer Ventures, True Light Capital, and Sirona Capital. The company, focused on oncology, immunology, inflammation, and cardiometabolic diseases, is using the funds to expand its R&D hub in Shanghai’s Zhangjiang Hi-Tech Park. Alongside the raise, OTR revealed the acquisition of a preclinical neurological program with high unmet need, though details remain undisclosed.

🧬 Gene delay: UniQure’s efforts to secure FDA approval for its Huntington’s disease gene therapy, AMT-130, have hit a setback, with finalized minutes from an October meeting confirming that current clinical data are insufficient to support a filing. The company had hoped that strong mid-stage results, showing a 75% slowing in disease progression among high-dose patients, would justify an early 2026 submission. However, the FDA’s updated stance has introduced uncertainty, leading UniQure to plan a follow-up meeting in early 2026. The news caused shares to fall further, compounding earlier losses, as analysts now see possibilities ranging from extended trials to the need for a new study.

🏷️ Label looming: Vertex Pharmaceuticals reported promising early results from two phase 3 trials of its CRISPR-based therapy Casgevy in children aged 5 to 11 with sickle cell disease and beta thalassemia. In the sickle cell trial, all four children who completed at least a year of follow-up experienced no vaso-occlusive crises, while in the thalassemia trial, six out of six eligible participants remained transfusion-free for at least 12 months. These findings, presented at the American Society of Hematology meeting, may support a label expansion and faster FDA review, with an approval request planned for early 2026. Casgevy is currently designated a “national priority” drug in the U.S.

🍼 Tiny triumphs: Encoded Therapeutics reported encouraging phase 1/2 data for its gene therapy ETX101 in children with SCN1A-positive Dravet syndrome, showing a 78% median reduction in seizure frequency at the third dose level. No treatment-related serious adverse events were observed across four dose levels in the open-label Polaris study, which enrolled children aged 6 months to 7 years. Neurodevelopmental improvements were noted, particularly in children treated before age 2, with gains in communication, motor function, and cognitive development. These findings support a planned pivotal trial in 2026, though Encoded may need additional funding or a partnership to advance the program.

↪️ Good pivot: Oric Pharmaceuticals presented new data on its EGFR/HER2 inhibitor enozertinib, supporting its decision to abandon development in relapsed non-small cell lung cancer (NSCLC) due to uncompetitive response rates. In HER2 exon 20-mutated relapsed NSCLC, enozertinib achieved confirmed overall response rates (ORRs) of just 26–27%, well below recently approved competitors from Boehringer Ingelheim and Bayer. Shifting focus, Oric is now targeting first-line treatment of EGFR PACC-mutant NSCLC, reporting a preliminary 80% ORR in 10 patients. While this early data offers promise, the small sample size and existing competition from ArriVent and Black Diamond raise questions about long-term positioning.

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