BioRestorative’s BRTX-100 Phase 2 Shows 52% ≥50% Pain and Function Gain in 25 Patients
At ISCT 2026, BioRestorative expanded its blinded Phase 2 dataset to 25 evaluable patients at 52 weeks versus 12 previously, showing 52% achieved at least 50% improvement in both VAS pain and ODI function with no dose-limiting toxicities. The trial sustains favorable safety and advances Phase 3 readiness after FDA Fast Track designation.
1. Expanded Phase 2 Dataset
BioRestorative enrolled a 52-week follow-up cohort of 25 patients in its Phase 2 trial of BRTX-100, more than doubling the evaluable population from the previously reported 12 patients, to assess sustained outcomes in chronic lumbar disc disease.
2. Efficacy Outcomes
At 52 weeks, 52% of evaluable patients achieved at least 50% improvement in both Visual Analog Scale pain scores and Oswestry Disability Index function scores. Secondary measures showed 56% of patients with ≥50% VAS pain improvement (average 71.4%), 64% with ≥50% ODI functional gain (average 73.6%), and similar durable gains on FRI and RMDQ metrics.
3. Safety Profile
No dose-limiting toxicities or related adverse events were observed throughout the 52-week blinded dataset, reinforcing a favorable safety profile for autologous hypoxic-cultured mesenchymal stem cell injections in non-surgical regenerative therapy.
4. Regulatory Pathway and Phase 3 Readiness
BRTX-100 holds FDA Fast Track designation for chronic lumbar disc disease and recently secured alignment on Phase 3 design elements during a Type B meeting. The company has initiated Phase 3 enabling activities aiming for an IND submission later in 2026.