BioRestorative Therapies, Inc. has announced promising preliminary data from its ongoing Phase 2 clinical trial of BRTX-100 for the treatment of chronic lumbar disc disease (cLDD). The data, which covers the first 10 patients enrolled in the trial over a 26-52 week period, indicates both safety and potential efficacy of the novel cell-based therapy. The results will be presented at the Orthopaedic Research Society (ORS) Philadelphia Spine Research Society (PSRS) 7th International Spine Research Symposium.
Phase 2 Trial Design and Patient Outcomes
The Phase 2 trial is a prospective, randomized, double-blinded, and controlled study designed to evaluate the safety and efficacy of BRTX-100 in treating cLDD. The trial aims to enroll up to 99 eligible subjects across 16 clinical sites in the United States, with participants randomized in a 2:1 ratio to receive either BRTX-100 or a placebo. The preliminary data reported is from an initial safety run-in with 10 subjects.
Safety Profile
According to the company, no serious adverse events (SAEs) were reported in any of the 10 safety run-in subjects. Furthermore, there was no dose (40X10 cells) limiting toxicity observed at 26-52 weeks, suggesting a favorable safety profile for BRTX-100 at the tested dose.
Efficacy Endpoints
In addition to safety, the trial assessed the secondary efficacy endpoint of at least a 30% decrease in pain, as measured by the Visual Analog Scale (VAS), and at least a 30% increase in function, based on the Oswestry Disability Index (ODI) at week 52. The blinded preliminary efficacy endpoint data demonstrated the following:
- At 26 weeks, 70% of subjects (n=10) reported a >30% improvement in VAS versus baseline.
- At 52 weeks, 100% of subjects (n=4) reported a >30% improvement in VAS versus baseline.
- At 12 and 26 weeks, 70% of subjects (n=10) had a >30% improvement in ODI versus baseline.
- At 52 weeks, 100% of subjects (n=4) had a >30% improvement in ODI versus baseline.
- At 26 weeks, 70% of subjects (n=10) reported a >30% decrease in pain (VAS) and a >30% increase in function (ODI).
About BRTX-100
BRTX-100 is a novel cell-based therapeutic engineered to target areas of the body with limited blood flow. It represents BioRestorative Therapies' lead clinical candidate and is designed to address the underlying causes of chronic lumbar disc disease by promoting tissue repair and regeneration.
