BioRestorative Concludes Phase II BRTX-100 Trial Enrolment for cLDD

Why It Matters

Successful enrollment validates the trial’s scale and design, positioning BioRestorative for pivotal Phase III data that could reshape regenerative treatments for chronic lumbar disc disease. Positive outcomes may accelerate regulatory approval and expand market opportunities in the growing spine‑care sector.

Key Takeaways

• Phase II trial enrolled 99 patients across 15 U.S. sites.
• 2:1 randomization: two-thirds received BRTX‑100, one-third placebo.
• Primary endpoints: safety, ≥30% ODI improvement, ≥30% pain reduction.
• Autologous hypoxic MSC therapy targets low‑oxygen disc environment.
• Data supports pathway to Phase III and potential BLA submission.

Pulse Analysis

Chronic lumbar disc disease (cLDD) remains a leading cause of disability worldwide, accounting for a substantial share of low‑back pain consultations and driving billions in healthcare expenditures. Conventional interventions—physical therapy, analgesics, and spinal fusion—often provide limited relief and carry risks of complications or long‑term dependency. In this environment, cell‑based regenerative approaches have attracted considerable investor and clinical interest, promising to restore disc integrity rather than merely alleviate symptoms. BioRestorative’s BRTX‑100, an autologous mesenchymal stem cell product cultured under hypoxic conditions, is positioned to address the low‑oxygen, nutrient‑poor niche of the intervertebral disc.

The Phase II trial marks the most extensive, rigorously controlled study of a stem‑cell therapy for cLDD to date. Enrolling 99 patients across 15 U.S. sites, the protocol randomizes two‑thirds of participants to receive a single intradiscal injection of BRTX‑100 while one‑third receive a sham procedure, preserving blinding integrity. Primary safety monitoring tracks adverse events, while efficacy hinges on achieving at least a 30 % reduction in the Oswestry Disability Index and a comparable drop in Visual Analog Scale pain scores at one year. Early blinded data have already demonstrated a favorable safety profile, bolstering confidence in the therapeutic hypothesis.

Regulatory momentum is evident after BioRestorative’s recent FDA Type B meeting, where the agency provided constructive feedback on trial endpoints and manufacturing controls. The company now outlines a clear trajectory toward a Phase III pivotal study and a potential Biologics License Application, which could fast‑track market entry if outcomes meet expectations. Success would not only expand the limited arsenal of biologics for spinal disorders but also set a precedent for sham‑controlled designs in regenerative medicine, influencing future investment and research strategies across the orthobiologic sector.

BioRestorative concludes Phase II BRTX-100 trial enrolment for cLDD

Image: Doctor examining an X‑ray on a tablet and taking notes · Credit: create jobs 51 / Shutterstock.com

BioRestorative Therapies has concluded patient enrolment in its Phase II study evaluating the efficacy and safety of BRTX‑100 for chronic lumbar disc disease (cLDD).

BRTX‑100 is an autologous, hypoxically cultured mesenchymal stem cell therapy designed for delivery into low‑oxygen, low‑nutrient disc settings.

The randomised, prospective, double‑blind, sham‑controlled trial enrolled 99 subjects across 15 sites in the US, focusing on single‑disc disease. Participants were randomised in a 2:1 ratio to receive either the therapy or placebo through a minimally invasive outpatient intradiscal procedure.

The trial’s primary safety endpoint is to assess the severity and frequency of adverse events. Efficacy endpoints include an improvement exceeding 30 % in function as measured by the Oswestry Disability Index and a greater than 30 % decrease in pain on the Visual Analog Scale compared to baseline at week 52.

CEO Comment

“This is a defining operational and clinical milestone for BioRestorative. Completing enrolment in what we believe is the largest and most rigorously designed Phase II cell‑therapy trial in chronic lumbar disc disease represents years of disciplined execution and scientific focus.

This is not only a scale milestone — it is a quality milestone. A randomised, double‑blind, sham‑controlled, single‑disc trial sets a high evidentiary bar and positions the programme for meaningful regulatory dialogue and advancement.”

— Lance Alstodt, CEO, BioRestorative

“Indeed, based on the compelling preliminary blinded Phase II data we have reported to date, together with constructive feedback from our recent FDA Type B meeting, we believe we have a clear and actionable pathway toward Phase III development and a potential BLA submission. We look forward to providing additional updates as we advance the programme in the very near future.”

— Lance Alstodt, CEO, BioRestorative