BioRestorative Therapies (NASDAQ:BRTX) presented new preliminary 26–52 week blinded data from the first 15 patients with chronic lumbar disc disease (cLDD) enrolled in the ongoing Phase 2 clinical trial of BRTX-100. The presentation was made as part of the “2025 Winners in Industry Innovations in MSK Health” program at the 2025 Orthopaedic Research Society (ORS) annual meeting this past weekend.
BRTX-100, a novel cell-based therapeutic engineered to target areas of the body that have little blood flow, is the company’s lead clinical candidate.
No serious adverse events (SAEs) were reported, and there was no dose (40X106 cells) limiting toxicity at 26-52 weeks.
In addition to safety outcomes, changes compared to baseline in Visual Analog Scale (VAS), which measures pain, and Oswestry Disability Index (ODI), which measure function, are used by the FDA to help determine whether the Phase 2 BRTX-100 trial will be allowed to proceed and ultimately gain Biologics License Application (BLA) approval.
As presented at ORS 2025, preliminary blinded VAS and ODI data collected at weeks 26 and 52 post-injection demonstrated an exceptionally positive trend compared to baseline. With respect to decrease in pain at 26 weeks, the average improvement in VAS versus baseline was 71.20%, and at 52 weeks, the average improvement was 73.58%. Improvement in ODI versus baseline at 26 weeks averaged 69.04%, and at 52 weeks, the average improvement was 65.16%. These results are significantly better than the requisite FDA minimum threshold of 30% improvement in VAS and ODI, respectively.
Also, in a new analysis reported for the first time, 52 week comparison of MRI images to baseline appear to demonstrate morphological changes, such as increase in T2 signal (hydration), decrease in protrusion size, as well as resolutions of annular tears. This potentially demonstrates disc microenvironment remodeling as a result of cLDD treatment with BRTX-100.
“This news is incredibly energizing; with each new blinded preliminary data analysis, like the one presented this past weekend at ORS 2025, our confidence grows that the ongoing Phase 2 BRTX-100 trial in cLDD will meet its Primary and Secondary endpoints, and thereby surpass what we believe are the requisite function and pain improvement thresholds for FDA approval. In addition, we have, for the first time, observed digital objective improvements in the disc on radiographic measures, which may translate into transformational outcomes never seen before in the industry,” Lance Alstodt, BioRestorative’s CEO said in a statement.
