Phase 2 Trial of BRTX-100 in cLDD Continues to Generate Positive Preliminary Blinded Data
- No serious adverse events (SAEs) reported in the Phase 2 trial
- Trial demonstrates >30% improvements in both ODI and VAS measures, meeting FDA requirements
- 70% of subjects showed >50% improvement in both function and pain reduction at week 52
- Sustained therapeutic effects with 66.66% improvement maintained through week 104
- None.
Insights
BRTX-100 shows strong safety profile and encouraging preliminary efficacy trends in Phase 2 trial for chronic lumbar disc disease.
BioRestorative Therapies' Phase 2 trial of BRTX-100 is generating promising blinded data for chronic lumbar disc disease (cLDD) treatment. The preliminary results from the first 15 patients show no serious adverse events (SAEs) and no dose-limiting toxicity at the 40 million cell dose through 104 weeks of follow-up, indicating a strong safety profile.
The efficacy data is particularly encouraging. The FDA requires >30% improvement in both Oswestry Disability Index (ODI, which measures functional disability) and Visual Analog Scale (VAS, which measures pain) - and the company reports their data shows trends exceeding these thresholds. The most striking finding is the dramatic improvement over time in patients experiencing substantial benefit: while 0% of subjects showed >50% improvement in both measures at 2 weeks, this jumped to 70% of subjects at 52 weeks and remained at 66.66% at 104 weeks.
This response pattern aligns with the expected biological mechanism of mesenchymal stem cells, which typically require time to exert their full regenerative effects on damaged disc tissue. The sustained response at 104 weeks (2 years) is particularly noteworthy as it suggests potential long-term benefits, which would be highly valuable in a chronic condition like cLDD.
For context, cLDD represents an enormous market opportunity with limited effective treatments. Current options like physical therapy offer modest relief, while surgical interventions carry significant risks. A regenerative approach with BRTX-100's apparent durability could position it as a transformative middle-ground therapy that addresses the underlying pathology rather than just symptoms.
MELVILLE, N.Y., May 13, 2025 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (“BioRestorative”, “BRTX” or the “Company”) (NASDAQ:BRTX), a clinical stage regenerative medicine innovator focused on stem cell-based therapies and products, is pleased to announce that preliminary 26-, 52- and 104-week blinded preliminary data from the first 15 patients with chronic lumbar disc disease (“cLDD”) enrolled in the ongoing Phase 2 clinical trial of BRTX-100 was recently presented by Francisco Silva, Vice President of Research and Development, at the International Society for Cell & Gene Therapy (“ISCT”) 2025 Annual Meeting.
No serious adverse events (SAEs) were reported, and there was no dose (40X106 cells) limiting toxicity at 26-104 weeks.
The U.S. Food and Drug Association (FDA) is requiring at least a greater than
| Week | Percentage of Subjects with a Greater than |
| 2 | |
| 12 | |
| 26 | |
| 52 | |
| 104 |
“We were pleased that blinded data from the ongoing Phase 2 trial of BRTX-100 in cLDD continues to be in-line to meet the primary safety endpoint of study, and that the positive preliminary efficacy trends also continue,” said Lance Alstodt, Chief Executive Officer of BioRestorative.
Mr. Silva’s ISCT presentation, titled “Phase 2 Clinical Safety/Efficacy Data of Intradiscal Injection of Hypoxic Mesenchymal Stem Cells for Lumbar Disc Disease,” can be accessed on the Company’s new website under “Scientific Publications” in the Product Candidate section.
About the BRTX-100 Phase 2 Trial in cLDD
BRTX-100, a novel cell-based therapeutic engineered to target areas of the body that have little blood flow, is the Company’s lead clinical candidate. The safety and efficacy of BRTX-100 in treating cLDD is being evaluated in a Phase 2, prospective, randomized, double-blinded and controlled study. A total of up to 99 eligible subjects will be enrolled at up to 16 clinical sites in the United States. Subjects included in the trial will be randomized 2:1 to receive either BRTX-100 or placebo.
About BioRestorative Therapies, Inc.
BioRestorative (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. As described below, our two core clinical development programs relate to the treatment of disc/spine disease and metabolic disorders, and we also operate a commercial BioCosmeceutical platform:
• Disc/Spine Program (brtxDISC™): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a person’s own) cultured mesenchymal stem cells collected from the patient’s bone marrow. We intend that the product will 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 utilizes 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 intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have commenced a Phase 2 clinical trial using BRTX-100 to treat chronic lower back pain arising from degenerative disc disease. We have also obtained FDA IND clearance to evaluate BRTX-100 in the treatment of chronic cervical discogenic pain.
• Metabolic Program (ThermoStem®): We are 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: We operate a commercial BioCosmeceutical platform. Our current commercial product, formulated and manufactured using our cGMP ISO-7 certified clean room, is a cell-based secretome containing exosomes, proteins and growth factors. This proprietary biologic serum has been specifically engineered by us to reduce the appearance of fine lines and wrinkles and bring forth other areas of cosmetic effectiveness. Moving forward, we also intend to explore the potential of expanding our commercial offering to include a broader family of cell-based biologic aesthetic products and therapeutics via Investigational New Drug (IND)-enabling studies, with the aim of pioneering U.S. Food and Drug Administration (FDA) approvals in the emerging BioCosmeceuticals space.
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Company's latest Form 10-K, filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.
CONTACT:
Stephen Kilmer
Investor Relations
Direct: (646) 274-3580
Email: skilmer@biorestorative.com
FAQ
What are the latest Phase 2 trial results for BRTX-100 in treating chronic lumbar disc disease?
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