BioRestorative Therapies’ MSC Therapy BRTX-100 Fast Tracked for Chronic Lumbar Disc Disease

Francisco Silva

Credit: BioRestorative Therapies

BioRestorative Therapies’ BRTX-100, an autologous mesenchymal stem cell (MSC) therapy, has received FDA fast track designation for the treatment of chronic lumbar disc disease (cLDD).¹

BRTX-100 is currently being evaluated in a prospective, randomized, and controlled phase 2 clinical trial (NCT04042844). The study is expected to enroll up to 99 patients in total, who will be randomly assigned to receive either BRTX-100 or a placebo in a 2:1 fashion across up to 16 study sites in the United States. BRTX-100 consists of hypoxic, cultured mesenchymal stem cells intended to improve blood flow in impacted areas of the body and is delivered via intradiscal injection.

“We are thrilled that the FDA has granted fast track designation, underscoring the potential of BRTX-100 to fill significant unmet medical needs in the treatment of cLDD resulting from ineffective conservative nonsurgical approaches or failed surgical interventions, and reflecting the comparatively very positive preliminary phase 2 clinical data that we have reported to date,” Lance Alstodt, BA, the chief executive officer of BioRestorative, said in a statement.¹ “Achieving fast track designation is an important milestone for BioRestorative, enabling us to work more collaboratively with the FDA as we continue to advance our lead BRTX-100 clinical program toward biologics license application (BLA) approval to bring this important and novel stem cell therapy to the millions of cLDD patients waiting for effective pain relief and functional improvement.”

Interim safety and efficacy data from the phase 2 clinical trial were recently presented by Francisco Silva, MBRS, the vice president of research and development at BioRestorative Therapies, at the Orthopaedic Research Society (ORS) annual meeting, held February 7 to 11, 2025, in Phoenix, Arizona.^2,3 With regard to safety, it was reported that no serious adverse events (AEs) have occurred. There were 9 AEs observed in 3 of the 10 safety run-in patients. There were 5 AEs deemed related to treatment, which occurred across 2 patients. These included 3 cases of increased post-procedural back pain in 2 patients and cases of worsening disc protrusion and acute Modic Type II changes observed via MRI in 1 patient. One patient experienced cases of ulnar nerve entrapment, trigger thumbs, trigger finger, and nonalcoholic fatty liver disease, all of which were deemd unrelated to treatment.

With regard to efficacy, it was reported that at 26 weeks posttreatment, a greater than 30% improvement in visual analog scales (VAS) score was reported in 70% of patients (n = 10). At 52 weeks posttreatment, a greater than 30% improvement in VAS score was reported in 100% of patients (n = 5). Furthermore, 70% of patients (n = 10) had a 30% or greater improvement from baseline in Oswestry Disability Index (ODI) at 12 weeks and 26 weeks posttreatment. At 52 weeks posttreatment, a 30% or greater improvement from baseline was seen in all patients (n = 5).

“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 end points, and thereby surpass what we believe are the requisite function and pain improvement thresholds for FDA approval,” Alstodt said in a February 10, 2025, statement.² “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. We look forward to updating all stakeholders as we progress.”

REFERENCES
1. BioRestorative receives FDA fast track designation for BRTX-100 chronic lumbar disc disease program. News release. BioRestorative Therapies, Inc. February 20, 2025. Accessed February 20, 2025. https://www.globenewswire.com/news-release/2025/02/20/3029644/0/en/BioRestorative-Receives-FDA-Fast-Track-Designation-for-BRTX-100-Chronic-Lumbar-Disc-Disease-Program.html
2. BRTX-100 Front and Center at ORS 2025. News release. BioRestorative Therapies, Inc. February 10, 2025. Accessed February 20, 2025. https://www.globenewswire.com/news-release/2025/02/10/3023320/0/en/BRTX-100-Front-and-Center-at-ORS-2025.html
3. Silva F. Next generation orthobiologic therapy for chronic lumbar disc disease: initial phase 2 data of hypoxic cultured mesenchymal stem cells. Presented at: ORS annual meeting, February 7-11, 2025, Phoenix, AZ.