BioRestorative Therapies (BRTX) Status Update summary

FDA clearance and clinical trial updates

• FDA cleared the IND for a phase II clinical trial of BRTX-100 for chronic cervical discogenic pain, making it the first stem cell product candidate cleared for this indication globally.

• Clearance allows direct entry into phase II without animal studies or a phase I, saving significant time and costs.

• IND clearance broadens the clinical pipeline, now targeting both chronic lower back and neck pain with BRTX-100.

• Fast Track designation was granted by the FDA for the lumbar program, potentially enabling priority review and accelerated BLA approval.

• The cervical trial will enroll about half as many subjects as the lumbar trial, with a two-to-one randomization.

Product and program overview

• BRTX-100 is an autologous mesenchymal stem cell therapy formulated from the patient’s bone marrow.

• Proprietary process involves bone marrow collection, stem cell isolation, culturing, and cryopreservation.

• The lumbar phase II trial is enrolling up to 99 subjects at 16 U.S. sites, randomized two-to-one to BRTX-100 or sham procedure.

• The cervical trial will use a lower injection volume (0.5 cc vs. 1.5 cc for lumbar) due to anatomical differences.

• The smaller cervical disc space may offer a more favorable environment for cell retention and efficacy.

Market opportunity and medical context

• Musculoskeletal conditions affect 1.7 billion people worldwide, with lower back pain as the leading cause of disability in over 160 countries.

• Chronic neck pain is a major public health issue, with cervical discogenic pain affecting 16% to 41% of patients.

• In the U.S., 80% of adults experience lower back pain, and cervical pain is among the top five chronic pain conditions.

• The cervical disc replacement market is projected to grow from $430 million in 2023 to $8.5 billion in 2032.

• BRTX-100 aims to address the large unmet need for non-surgical and effective pain relief in both cervical and lumbar disc disease.