BioRestorative reports Good Preliminary Data for FDA-Fast-Tracked Stem Cell Therapy for Disc Disease
BioRestorative's early Phase 2 trial results show notable improvements in function and pain for chronic lumbar disc disease patients, moving one step closer to a BLA.
BioRestorative Therapies, Inc. announced updated preliminary blinded data from the first 36 patients participating in its ongoing Phase 2 clinical trial of BRTX-100, an autologous (patient-derived) stem cell therapy for chronic lumbar disc disease (cLDD). The data was presented by Francisco Silva, Vice President of Research and Development, at the ISSCR 2025 Annual Meeting in Hong Kong.
The FDA requires a greater than 30% improvement in function, assessed using the Oswestry Disability Index (ODI), and a greater than 30% reduction in pain, assessed using the Visual Analog Scale (VAS), as benchmarks to determine whether the clinical trial will progress and potentially obtain Biologics License Application (BLA) approval.
Key Findings from the Updated Data:
- Increased Subject Enrollment: The number of evaluated subjects has grown from 15 to 36 since the previous data release, moving closer to full enrollment of up to 99 subjects.
- Functional Improvement: More than 74% of subjects achieved greater than 50% improvement in ODI scores by 52 weeks.
- Pain Reduction: Over 72% of subjects reported greater than 50% reduction in pain (VAS) scores by 52 weeks.
- Combined Results: A significant portion of subjects achieved greater than 50% improvement in both ODI and VAS measures across all evaluated time points.
- Safety Profile: No serious adverse events (SAEs) or dose-limiting toxicities have been reported between weeks 26 and 104 at the target dose of 40 million cells.
- Data Trend: Each new data analysis has shown improvement relative to previous analyses, suggesting an upward trend in efficacy.
Detailed breakdown of subjects demonstrating greater than 50% improvement in ODI, VAS, or both:
| Week | % Subjects with >50% Improvement in ODI | % Subjects with >50% Improvement in VAS | Subjects with >50% Improvement in Both ODI & VAS |
|---|---|---|---|
| Baseline | 0.00% | 0.00% | 0/36 |
| 12 | 67.57% | 73.82% | 5/25 |
| 26 | 74.04% | 76.94% | 6/15 |
| 52 | 74.63% | 72.35% | 8/10 |
| 104 | 75.13% | 68.54% | 2/4 |
“With every new analysis, our confidence grows that BRTX-100 is positioned to meet and potentially exceed the FDA’s functional and pain reduction thresholds,” said Lance Alstodt, Chief Executive Officer of BioRestorative. “We are excited by the trajectory of this material milestone and its potential to address a massive unmet need in chronic lower back pain — one of the largest global healthcare burdens. We believe this data moves us one step closer to bringing a much-needed, non-surgical therapeutic option to market and should add to further value enhancing inflection points in the near-term.”
About the BRTX-100 Phase 2 Clinical Trial
BRTX-100 is an autologous (patient-derived) cell-based therapy aimed at treating painful lumbosacral disc degeneration. The ongoing Phase 2 clinical trial is a randomized, double-blinded, placebo-controlled study enrolling up to 99 participants across 16 U.S. sites. Participants are randomized in a 2:1 ratio to receive either BRTX-100 or placebo via a minimally invasive outpatient procedure.
