BioRestorative Announces FDA Clearance of IND for Phase 2 Trial of BRTX-100 in Chronic Cervical Discogenic Pain

Rhea-AI Summary

BioRestorative Therapies (NASDAQ:BRTX) has received FDA clearance for its Investigational New Drug (IND) application for BRTX-100, marking a significant advancement in stem cell-based therapies. The treatment, designed to target areas with blood flow, will be evaluated for chronic cervical discogenic pain (cCDP) in a Phase 2 trial.

BRTX-100 represents the first and only stem cell product candidate cleared for evaluation in cervical degenerative disc disease. This expansion builds upon the company's existing FDA Fast Track-designated program for chronic lumbar disc disease (cLDD). The upcoming Phase 2 study will be conducted as a double-blind, sham-controlled, randomized trial to assess safety and preliminary efficacy of intradiscal BRTX-100 injections.

Positive

• First-ever FDA clearance for stem cell therapy in cervical disc disease
• Expansion of clinical pipeline to include both neck and back pain treatments
• FDA Fast Track designation for lumbar disc disease program

Negative

• Phase 2 trials yet to commence
• No efficacy data available yet

Insights

Biotechnology Analyst

BioRestorative's FDA clearance for their BRTX-100 IND in chronic cervical discogenic pain represents a significant clinical and strategic expansion that could substantially enhance the company's market position in regenerative medicine for spinal conditions.

This development is particularly noteworthy for three key reasons:

• BRTX-100 becomes the first and only stem cell therapy cleared for evaluation in cervical degenerative disc disease, establishing a potential first-mover advantage in this specific indication
• The company now has clinical programs targeting both cervical and lumbar spine regions, addressing a comprehensive degenerative disc disease market that affects up to 90% of adults over 50
• The lumbar program's existing Fast Track designation demonstrates regulatory recognition of the unmet medical need, potentially accelerating development timelines

From a mechanistic perspective, BRTX-100's approach of targeting low blood flow regions addresses a fundamental challenge in disc degeneration treatment. Current standard interventions primarily manage symptoms rather than underlying pathology, creating an opportunity for disruptive therapies that could modify disease progression.

The planned double-blind, sham-controlled Phase 2 trial design represents a rigorous approach that, if successful, would provide compelling evidence for efficacy. However, investors should recognize that despite this positive regulatory development, significant clinical hurdles remain before commercialization, including demonstrating safety and efficacy in well-controlled trials.

The company's relatively small $12.6 million market capitalization suggests the market remains cautious about ultimate clinical and commercial success, creating potential asymmetric risk/reward if clinical data proves promising.

– BRTX-100 is the first and only stem cell product candidate cleared to be evaluated in the cervical degenerative disc disease setting –

– IND clearance expands Company’s advanced clinical pipeline for BRTX-100 treatment of both chronic lower back and neck pain –

– Company to host webcasted conference call today at 10:30am EST –

MELVILLE, N.Y., Feb. 27, 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, today announced the U.S. Food and Drug Administration (“FDA”) has cleared its Investigational New Drug (“IND”) application for BRTX-100, a novel cell-based therapeutic engineered to target areas of the body that have little blood flow, for the treatment of chronic cervical discogenic pain (“cCDP”).

“Clearance of this IND for BRTX-100 represents a significant milestone and is a testament to the devotion of our talented scientific team to develop innovative cell-based therapies that fill the significant unmet medical needs in the treatment of degenerative disc disease, one of the leading causes of chronic pain and illness,” said Lance Alstodt, Chief Executive Officer of BioRestorative. “We are taking a comprehensive approach to leveraging our technology throughout the spine to bring effective pain relief and functional improvement to millions of patients. To that end, we are laying plans to evaluate the safety and preliminary efficacy of intradiscally injected BRTX-100 for patients with cCDP in a double-blind, sham-controlled, randomized Phase 2 study as soon as is practicable. In the meantime, we continue to advance our FDA Fast Track-designated BRTX-100 chronic lumbar disc disease (cLDD) program.”

Clinical Pipeline Update Conference Call & Webcast Details

BioRestorative management will host a webcasted conference call today at 10:30am EST to discuss the Company’s pipeline and review some additional positive events related to BRTX-100. To participate in the conference call by telephone, please dial 888-506-0062 (United States) or 973-528-0011 (International), participant access code 440089. The call will also be broadcast live and archived on the Investor section of the Company’s website at https://www.biorestorative.com/investor-relations/.

About Chronic Cervical Discogenic Pain

Chronic neck pain is a considerable public health burden that accounts for one of the top five chronic pain conditions. The pain can range from cervical intervertebral discs, facet joints to atlantoaxial joints. Cervical discogenic pain syndrome is a common source of neck pain with a reported prevalence between 16% to 41%. Cervical discs have a rich supply of nerve fibers that are prone to structural disruption and inflammatory reaction that makes them susceptible to pain. The commonly affected levels are C5/C6 and C6/C7, with C7 being the most common nerve root involved. Initially, the symptoms present proximally, but they can later progress to brachialgia.¹

Source

¹ Saini A, Mukhdomi T. Cervical Discogenic Syndrome. 2023 Apr 3. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2025 Jan–. PMID: 32310420.

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 have also recently begun offering BioCosmeceutical products:

• 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, as amended, and Form 10-Q 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 is the significance of FDA's IND clearance for BRTX-100 in cervical disc disease?

BRTX-100 is the first and only stem cell product cleared for evaluation in cervical degenerative disc disease, representing a potential breakthrough in treating chronic neck pain.

How will BioRestorative conduct the Phase 2 trial for BRTX-100?

The Phase 2 trial will be a double-blind, sham-controlled, randomized study evaluating safety and preliminary efficacy of intradiscal BRTX-100 injections for chronic cervical discogenic pain.

What conditions is BRTX-100 being developed to treat?

BRTX-100 is being developed for both chronic cervical discogenic pain (neck) and chronic lumbar disc disease (lower back), targeting areas with blood flow.

What is the current status of BRTX-100's development programs?

BRTX-100 has received FDA clearance for cervical disc disease trials and holds Fast Track designation for chronic lumbar disc disease treatment.