Significant improvements observed in primary endpoints, paving the way for regulatory submission later this year. The study demonstrated robust efficacy and a favorable safety profile.
BOSTON, Mass. — Nexalife Pharmaceuticals today announced positive top-line results from the Phase 3 clinical trial evaluating NX-202 for the treatment of rare neurogenetic disorders. The study met its primary endpoint, demonstrating a statistically significant improvement in motor function compared to placebo.
NX-202 represents a novel approach in our precision medicine pipeline, targeting the underlying genetic mechanism of the disease rather than just managing symptoms. The trial enrolled 120 patients across 15 sites globally, with patients receiving either NX-202 or placebo over a 52-week period.
"These results represent a transformative moment for patients who have had limited options for too long. The data not only validates our precision medicine approach but also brings hope to families affected by this devastating condition."
Key Clinical Data Summary
The primary efficacy endpoint was the change from baseline in the Motor Function Score (MFS) at Week 52. Patients treated with NX-202 showed a mean improvement of 4.5 points, compared to a decline of 1.2 points in the placebo group.
| Endpoint | NX-202 (n=60) | Placebo (n=60) | P-Value |
|---|---|---|---|
| Primary: MFS Change | +4.5 | -1.2 | < 0.001 |
| Secondary: Biomarker A | -45% | -2% | < 0.01 |
| Secondary: Quality of Life | +12.3 | +1.1 | < 0.05 |
Safety data indicated that NX-202 was generally well-tolerated. The most common adverse events were mild to moderate injection site reactions. No serious adverse events related to the drug were reported. Nexalife plans to submit a New Drug Application (NDA) to the FDA in the first half of 2024.