FDA Grants Fast Track Designation to BRTX-100 to Treat Chronic Lumbar Disc Disease

Key Takeaways

BRTX-100 is a novel cell-based therapy targeting chronic lumbar disc disease, currently in phase 2 trials for safety and efficacy evaluation.
Preliminary data shows BRTX-100 significantly improves pain and function in cLDD patients, with no serious adverse events reported.
Fast track designation by the FDA underscores BRTX-100's potential to address unmet needs in cLDD treatment, offering hope for effective pain relief and functional improvement.

Currently, the agent is undergoing evaluation in a phase 2 clinical trial to determine its safety and efficacy.

The FDA granted a fast track designation to the BRTX-100 program (BioResporative Therapies) for the treatment of chronic lumbar disc disease (cLDD).¹ Currently, it is undergoing evaluation in a phase 2 clinical trial (NCT04042844)² to determine its safety and efficacy when treating the disease.¹

Image credit: JaRiRiyawat | stock.adobe.com

cLDD is a common and often difficult problem for patients and their health care professionals. In the US, at least 80% of adults experience at least 1 episode of lower back pain during their lifetime. Low back pain is the most common cause of disability among Americans who are between 45 and 65 years of age. Currently, the standard of care for the treatment of cLDD involves conservative non-surgical approaches or surgical interventions that target symptomatic relief and musculoskeletal stabilization. There is no clinical therapy that targets the reversal of disc degeneration or that addresses intervertebral disc cell homeostasis.¹

BRTX-100 is a novel cell-based therapeutic that is designed to target areas of the body that have little blood flow. The safety and efficacy of the agent is undergoing evaluation in the prospective, randomized, double-blind, placebo-controlled phase 2 study.^1,2

In this trial, patients were randomly assigned to receive either BRTX-100 or placebo, which is administered as intravenous infusions. Patients will attend study site visits at weeks 2, 12, 26, 52, and 104. The study’s primary outcome measures are frequency of adverse events (AEs) as well as changes in visual analog scales (VAS) and Oswestry Disability Index (ODI) scores. Up to 99 eligible patients from 16 US clinical sites are expected to be enrolled.^1,2

Analysis results from November 2024 show preliminary blinded data from weeks 26 through 52. By week 26, BRTX-100 had helped about 70% of patients have at least a 30% decrease in VAS scores as well as a 30% increase in ODI. At week 52, patients had at least a 30% decrease in pain on the VAS scale, and a 30% increase in function based on the ODI scale.³

“Blinded preliminary clinical data of safety and efficacy end points from the ongoing phase 2 clinical trial of BRTX-100 in the treatment of cLDD are very encouraging, with patient reported pain and function outcomes demonstrating a positive trend,” said Lance Alstodt, CEO of BioRestorative in a news release. “Most importantly, at 26 weeks 70% of the patients are reporting a greater than 30% increase in function and a more than 30% decrease in pain. If data continues with this trend, we are confident that we will hit our efficacy end points for the phase 2 trial.”³

About the Trial

Trial Name: A Single Dose of BRTX 100 for Patients With Chronic Lumbar Disc Disease (cLDD)

ClinicalTrials.gov ID: NCT04042844

Sponsor: BioRestorative Therapies

Completion Date (Estimated): August 31, 2025

Additionally, these data show there were no serious AEs reported by any of the patients. Of note, there was no dose (40 x 10⁶ cells) limiting toxicity between weeks 26 and 52.³

“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 non-surgical approaches or failed surgical interventions, and reflecting the comparatively very positive preliminary phase 2 clinical data that we have reported to date,” said Alstodt in a news release. “Achieving fast track designation is an important milestone…to bring this important and novel stem cell therapy to the millions of cLDD patients waiting for effective pain relief and functional improvement.”¹

REFERENCES

1. GlobeNewswire. BioRestorative Receives FDA Fast Track Designation for BRTX-100 Chronic Lumbar Disc Disease Program. News release. 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. A Single Dose of BRTX 100 for Patients With Chronic Lumbar Disc Disease (cLDD). ClinicalTrials.gov identifier: NCT04042844. Updated April 16, 2024. Accessed February 20, 2025. https://clinicaltrials.gov/study/NCT04042844

3. BioSpace. BioRestorative Therapies Reports Positive Preliminary Phase 2 BRTX-100 Clinical Data. News release. November 13, 2024. February 20, 2025. https://www.biospace.com/press-releases/biorestorative-therapies-reports-positive-preliminary-phase-2-brtx-100-clinical-data