BioRestorative Therapies, a clinical-stage biotech company, has presented preliminary data from its Phase 2 trial of BRTX-100, a novel cell-based therapy for chronic lumbar disc disease, at the ORS 2024 Annual Meeting.
What is BRTX-100, and how does it work?
BRTX-100 is the lead therapeutic candidate of BioRestorative Therapies, which develops stem cell-based therapies for various conditions. BRTX-100 is designed to treat patients with chronic lower back pain caused by degenerative disc disease, a common condition that affects up to 80% of the population at some point. Degenerative disc disease occurs when the discs that cushion the spine lose their water content and elasticity, leading to pain, inflammation, and reduced mobility.
It uses the patient’s stem cells, which are harvested from their bone marrow and cultured under low oxygen conditions to mimic the disc environment. The stem cells are then injected into the damaged disc, where they are expected to regenerate the disc tissue and restore its function. BRTX-100 is the first cell-based therapy to use hypoxic cultured stem cells for this purpose, as previous studies have shown that the disc’s harsh microenvironment can affect the viability and efficacy of the cells.
What are the results of the Phase 2 trial?
The Phase 2 trial of BRTX-100 is a randomized, double-blinded, and controlled study that aims to evaluate the safety and efficacy of the therapy in up to 99 patients with chronic lumbar disc disease. The patients are enrolled at up to 16 clinical sites in the United States and are assigned to receive either BRTX-100 or placebo. The trial measures the changes in pain and function using various questionnaires, such as the Visual Analog Scale (VAS), the Oswestry Disability Index (ODI), the Roland Morris Disability Questionnaire (RMDQ), and the Functional Rating Index (FRI), at weeks 2, 12, 26, 52, and 104 after the treatment.
The data presented at the ORS 2024 Annual Meeting is still blinded and at an early stage, but it indicates a positive trend compared to the baseline. The pain and function scores collected at 26 and 52 weeks after the injection show meaningful improvements in the patients enrolled in the study, and no notable safety signals have been observed. The final results of the trial are expected to be available in 2025.
Why is this important for the field of regenerative medicine?
The preliminary data on BRTX-100 is encouraging for the field of regenerative medicine, as it demonstrates the potential of using stem cells to treat chronic and degenerative conditions that have limited treatment options. BRTX-100 is one of the few cell-based therapies that have reached the Phase 2 stage of clinical development, and it could pave the way for more innovative and effective solutions for patients suffering from chronic lower back pain and other musculoskeletal disorders.
BRTX-100 is also a promising candidate for the approval of a biologics license application (BLA) by the U.S. Food and Drug Administration (FDA), which would allow the therapy to be marketed and distributed in the United States. The FDA has granted BRTX-100 the Regenerative Medicine Advanced Therapy (RMAT) designation, which provides expedited review and guidance for the development of regenerative medicine products. The FDA has also granted BRTX-100 the Fast Track designation, which facilitates the development and review of drugs that address unmet medical needs.
“We are thrilled with the progress of our ongoing clinical development programs. Regarding the Phase 2 study investigating the use of BRTX-100 in the treatment of colds, we are strongly encouraged by the preliminary data presented at ORS 2024. The preliminary clinical data shows meaningful signals in patients enrolled in the study and, importantly, no notable safety signals,” said Lance Alstodt, Chief Executive Officer of BioRestorative.
BioRestorative Therapies is a clinical-stage biotech company that is developing cell-based therapies to treat chronic lumbar disc disease and metabolic conditions such as obesity and diabetes. The company is focusing on developing BRTX-100, its lead therapeutic candidate for the treatment of patients with chronic lower back pain caused by degenerative disc disease.
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