Bristol Myers Squibb presents interim Phase 1 data for zola‑cel CAR‑T therapy in multiple sclerosis
Bristol Myers Squibb disclosed interim safety and efficacy results from the Phase 1 Breakfree‑2 study of its CD19‑directed CAR‑T cell therapy, zolacabtagene autoleucel (zola‑cel), in 66 patients with highly active relapsing and progressive multiple sclerosis. The primary safety endpoint showed that 42.4% of patients experienced cytokine release syndrome, mostly Grade 1, and 7.6% had ICANS, with one Grade 4 event; one patient died from septic pneumonia. Efficacy signals included all 60 patients treated at dose level 2 remaining off disease‑modifying therapies and 92% of progressive‑MS patients showing stable or improved disability.
Why it matters
The company says the data suggest a potential new treatment approach for multiple sclerosis, which could expand its CAR‑T pipeline beyond oncology if later trials confirm efficacy. Investors will watch how the safety profile and early efficacy influence future trial designs and regulatory prospects.
Key facts
- 1The Breakfree‑2 study enrolled 66 patients (30 with haRMS and 36 with PMS). finance.yahoo.com
- 2Patients had a median of two prior disease‑modifying therapies (range 1‑8 haRMS; 1‑6 PMS). finance.yahoo.com
- 3Overall, 42.4% of patients (28/66) experienced cytokine release syndrome, mostly Grade 1 (36.4%) and some Grade 2 (6.1%). finance.yahoo.com
- 47.6% of patients (5/66) experienced immune effector cell‑associated neurotoxicity syndrome, including one Grade 4 event. finance.yahoo.com
- 5One patient with haRMS died of septic pneumonia 44 days after infusion, the only Grade 5 event reported. finance.yahoo.com
- 6All patients treated at dose level 2 (60/60) remained off disease‑modifying therapies at last follow‑up. finance.yahoo.com
Summary written by AlphAI AI Desk from 7 of 7 sources. Not investment advice. Figures are as stated by the linked sources.