Regeneron reports Phase 2 results for baloncibart in POTS, plans Phase 3 trial
Regeneron presented data from its Phase 2 PINNACLE trial of baloncibart, an NPR1‑blocking monoclonal antibody, in adults with postural orthostatic tachycardia syndrome. A single 450 mg dose lowered the standing‑to‑lying heart‑rate increase by 11.4 bpm at day 8 and maintained a 7.4 bpm reduction at day 29, while also improving orthostatic symptoms and physical functioning. The 150 mg dose showed a smaller, non‑sustained effect. The company said a pivotal Phase 3 study is planned for the first half of 2027 (or early 2025 per a separate release).
Why it matters
Regeneron expects the Phase 3 trial to move the candidate toward potential FDA approval, which could create a first‑in‑class therapy for a disorder with no approved treatments. The positive Phase 2 data may also support the company’s broader antibody platform and pipeline narrative.
Key facts
- 1The Phase 2 PINNACLE trial enrolled 27 patients on baloncibart 150 mg, 28 patients on baloncibart 450 mg, and 27 patients on placebo. quiverquant.com
- 2Baloncibart 450 mg reduced the change in heart rate from lying to standing by a least‑squares mean difference of –11.4 bpm at day 8 (nominal p=0.0007). quiverquant.com
- 3At day 29, baloncibart 450 mg showed a mean difference of –7.4 bpm versus placebo (nominal p=0.0124). quiverquant.com
- 4Baloncibart 150 mg reduced the heart‑rate change by –9.5 bpm at day 8 but the effect was not maintained at day 29. quiverquant.com
- 5Patients receiving the 450 mg dose reported improvements in orthostatic symptoms (–12.9 points), physical functioning (–13.8 points), and activities of daily living (–8.7 points) on the LWPQ at day 29. quiverquant.com
- 6The trial reported treatment‑emergent adverse events in 66.7% of the 150 mg group and 67.9% of the 450 mg group, most of which were mild or moderate. quiverquant.com
Open questions
- Material 1 states the Phase 3 trial is planned for the first half of 2027, while Material 2 says the launch is slated for the first half of next year (early 2025).
Summary written by AlphAI AI Desk from 3 of 3 sources. Not investment advice. Figures are as stated by the linked sources.