BioRestorative Therapies Inc. has completed patient enrollment in its Phase 2 clinical trial evaluating the safety and efficacy of BRTX-100 for treating chronic lumbar disc disease (cLDD).

BRTX-100 is BioRestorative’s lead clinical candidate—an autologous, hypoxically cultured mesenchymal stem cell therapy engineered for delivery into low-oxygen, low-nutrient disc environments.

With 99 patients enrolled across 15 U.S. clinical sites, the BRTX-100 Phase 2 study represents the largest Phase 2 cell-therapy trial conducted in cLDD under a U.S. Food and Drug Administration FDA) Investigational New Drug (IND) application and ranks among the largest controlled cell-therapy trials in spine medicine to date, according to the company.

“This is a defining operational and clinical milestone for BioRestorative,” BioRestorative Therapies CEO Lance Alstodt stated. “Completing enrollment in what we believe is the largest and most rigorously designed Phase 2 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 randomized, double-blind, sham-controlled, single-disc trial sets a high evidentiary bar and positions the program for meaningful regulatory dialogue and advancement. Based on the preliminary blinded Phase 2 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 3 development and a potential BLA submission. We look forward to providing additional updates as we advance the program in the very near future.”

The trial uses a prospective, randomized, double-blind, sham-controlled design focused on single-disc disease, widely considered the gold standard for clinical rigor in spine intervention studies. Participants were randomized two to one to receive either BRTX-100 or placebo through a minimally invasive outpatient intradiscal procedure.

The primary safety endpoint for the BRTX-100 Phase 2 clinical trial is adverse events (AEs) frequency and severity. The trial’s primary efficacy endpoints are a greater than 30% improvement in function in the Oswestry Disability Index (ODI) and a greater than 30% reduction in pain on the Visual Analog Scale (VAS) vs. baseline at week 52.

Single-disc enrollment criteria are notably restrictive and historically challenging, underscoring the significance of completing full enrollment at this scale.

“Our manufacturing, quality control and clinical teams executed exceptionally well in completing enrollment in a highly selective, technically demanding study design,” BioRestorative Therapies Vice President of Research and Development Francisco Silva said. “Interest from both investigators and patients was driven in part by the preliminary blinded data we presented at multiple scientific conferences, including ISSCR, ISCT, ORS, and the International Spine Research Symposium. We are grateful to the patients and investigators whose participation made this milestone possible.”

Approximately 40% of total enrollment occurred within the past six months, reflecting accelerating site productivity and investigator engagement.

cLDD is a common, often confounding problem for patients and physicians. At least 80% of U.S. adults experience at least one episode of lower back pain during their lifetime. Low back pain is the most common cause of disability among Americans between 45 and 65 years of age and imposes the highest economic burden on the U.S. healthcare system. The standard of care for treating cLDD involves conservative non-surgical approaches or surgical interventions that target symptomatic relief and musculoskeletal stabilization. Currently, there is no clinical therapy for reversing disc degeneration or addressing intervertebral disc cell homeostasis.

BioRestorative Therapies develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Its two core clinical development programs relate to treating disc/spine disease and metabolic disorders, and the company also operates a commercial BioCosmeceutical platform.

Disc/Spine Program (brtxDISC): BRTX-100 is a product formulated from autologous (or a person’s own) cultured mesenchymal stem cells collected from the patient’s bone marrow. The product would be used for the non-surgical treatment of painful lumbosacral disc disorders or as a complementary therapeutic to a surgical procedure. The BRTX-100 production process uses proprietary technology and involves collecting a patient’s bone marrow, isolating and culturing stem cells from the bone marrow, and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patient’s damaged disc. The treatment is for patients whose pain has not been alleviated by non-invasive procedures and who potentially face surgery. The company has obtained FDA Investigational New Drug clearance to evaluate BRTX-100 in treating chronic cervical discogenic pain.

Metabolic Program (ThermoStem): The company is developing cell-based therapy candidates to target obesity and metabolic disorders using brown adipose (fat) derived stem cells (BADSC) to generate brown adipose tissue (BAT) as well as exosomes secreted by BADSC. BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in animals may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes. BADSC secreted exosomes may also impact weight loss.

BioCosmeceuticals: BioRestorative Therapies operates a commercial BioCosmeceutical platform. Its current commercial product, formulated and manufactured using the firm’s cGMP ISO-7 certified cleanroom, is a cell-based secretome containing exosomes, proteins, and growth factors. This proprietary biologic serum has been specifically engineered to reduce the appearance of fine lines and wrinkles and bring forth other areas of cosmetic effectiveness. Moving forward, the company also will explore potentially expanding its commercial offering to include a broader family of cell-based biologic aesthetic products and therapeutics via IND-enabling studies.