RESTEM Receives FDA Clearance of IND for Phase 1/2 Clinical Trial of Restem-X in Knee Osteoarthritis
Phase 1/2 clinical trial anticipated to be initiated in the first half of 2027 Expands RESTEM’s clinical pipeline from
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Phase 1/2 clinical trial anticipated to be initiated in the first half of 2027
Expands RESTEM’s clinical pipeline from autoimmune disease into orthopedics with an off-the-shelf secretome derived from its umbilical cord lining modified progenitor cell platform
MIAMI, Nov. 10, 2026 (GLOBE NEWSWIRE) — RESTEM, a clinical-stage biotechnology company that develops off-the-shelf, next-generation cell and cell-derived therapies designed to modulate the immune system, today announced that the Company has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to initiate a Phase 1/2 clinical trial evaluating Restem-X, an investigational secretome-based therapy, in patients with symptomatic knee osteoarthritis (OA).
Restem-X is a secretome derived from RESTEM’s proprietary Restem-L umbilical cord lining modified progenitor cell (UMPC) product, with potential applications in joint repair and regeneration. UMPCs are stimulated to secrete an optimized profile of bioactive molecules intended to enhance immunomodulatory as well as anti-inflammatory activity and support tissue repair and joint health. The formulation contains collagen, fibroblast growth factors and other bioactive molecules that play key roles in tissue repair and healing. Restem-X has not been approved by the FDA or any other regulatory authority, and its safety and efficacy have not been established.
The IND clearance expands the clinical development of RESTEM’s UMPC platform from Restem-L in autoimmune, inflammatory and neuromuscular diseases, including idiopathic inflammatory myopathy, rheumatoid arthritis and facioscapulohumeral muscular dystrophy, to Restem-X in osteoarthritis. For patients, the Restem-X study represents an important first step in evaluating a new secretome-based approach with potential supportive and regenerative benefits for orthopedic conditions. The study will be the first prospective clinical evaluation of Restem-X under an FDA-cleared IND.
“We appreciate the FDA’s guidance and clearance of our IND for Restem-X in osteoarthritis and look forward to initiating our Phase 1/2 study in the first half of 2027,” said Andres Isaias, Chief Executive Officer of RESTEM. “This study marks an important milestone in advancing our secretome platform and evaluating Restem-X’s safety, tolerability and preliminary efficacy in patients with symptomatic knee osteoarthritis. Many people living with knee osteoarthritis have few options between pain medication and joint replacement surgery, and they need treatments that can offer durable relief. The data from this study will help us better understand Restem-X’s potential to reduce pain and improve joint function.”
Restem-X Clinical Experience to Date
The IND is supported by preclinical data and by early clinical observations in 14 patients (17 knees) with knee osteoarthritis who received intra-articular Restem-X; these data were submitted to the FDA with the application. In a retrospective review of patient-reported outcomes, Restem-X was generally well tolerated, with no serious adverse events reported; 13 of 14 patients (93%) reported overall improvement, and all 14 (100%) reported reduced use of pain medication, with follow-up ranging from 2 to 25 months. These observations are retrospective, uncontrolled and patient-reported, and are not predictive of results in a prospective clinical trial; the Phase 1/2 study is designed to evaluate Restem-X under controlled conditions.
Phase 1/2 Clinical Trial of Restem-X in Osteoarthritis
Restem-X will be evaluated in a Phase 1/2 clinical trial to assess its safety, tolerability and preliminary efficacy in patients with symptomatic knee osteoarthritis. The study will include an open-label, dose-escalation Phase 1 component followed by a randomized, double-blind, active-controlled Phase 2 component. Patients with Kellgren-Lawrence grade 2 or 3 knee osteoarthritis will be eligible. The primary endpoint of Phase 1 is safety and tolerability. In Phase 2, the primary efficacy endpoint is the change from baseline in overall Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) score at Month 6 for each Restem-X group compared with hyaluronic acid (HA). Secondary endpoints include the proportion of patients achieving at least a 50% improvement in WOMAC score; changes in WOMAC pain, stiffness and function subscores; pain measured on a visual analog scale (VAS); patient-reported outcomes (PROMIS-29); and radiographic progression of osteoarthritis. Matthew Negaard, MD, a fellowship-trained sports medicine physician at Forté Sports Medicine and Orthopedics and the Forté Orthopedic Research Institute in Carmel, Indiana, and a team physician for the Indianapolis Colts, will serve as the principal investigator. Additional information will be available at ClinicalTrials.gov.
About Osteoarthritis
Osteoarthritis is the most common form of arthritis, characterized by the gradual breakdown of cartilage and changes in other joint tissues. It commonly affects the knees, hips, hands and spine, causing pain, stiffness, swelling and reduced mobility. Symptoms often develop over time and can significantly affect daily activities and quality of life. An estimated 595 million people worldwide had osteoarthritis in 2020, and the condition affects more than 32.5 million adults in the U.S. The knee is the most commonly affected joint. There is no approved therapy shown to slow or reverse the disease; current treatments focus on symptom relief, and many patients ultimately require joint replacement surgery.
About RESTEM
RESTEM is a clinical-stage biotechnology company focused on developing off-the-shelf, next-generation cell and cell-derived therapies for autoimmune, inflammatory, and age-related diseases. Leveraging proprietary products, deep clinical expertise, and advanced manufacturing capabilities, RESTEM is advancing three potentially transformative programs: Restem-L, our umbilical cord lining modified progenitor cell (UMPC) therapy for autoimmune and inflammatory diseases; Restem-X, a secretome-based product for orthopedic conditions; and activated natural killer cell (aNK) therapeutics targeting senescence and age-associated disorders. Our therapies are designed to reprogram the immune system rather than focusing solely on symptom management, offering patients with limited options the potential to address underlying disease mechanisms. RESTEM is headquartered in Miami, Florida. For more information, please visit www.restem.com and follow us on X and LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding the timing, design and conduct of the Phase 1/2 clinical trial of Restem-X, the potential safety and efficacy of Restem-X, the potential of RESTEM’s UMPC platform and secretome-based approach, the predictive value of the early clinical observations described above, and RESTEM’s development plans. These statements are based on current expectations and are subject to risks and uncertainties, including those inherent in clinical development and regulatory review, that could cause actual results to differ materially. RESTEM undertakes no obligation to update these statements except as required by law.
Investor Contact
Daniel Ferry
LifeSci Advisors
+1.617.430.7576
daniel@lifesciadvisors.com
Media Contact
Nelson Cabatuan
Restem Group, Inc.
+1.800.490.0924
ncabatuan@restem.com
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