RESTEM Provides Corporate Update and Announces Key Business Objectives for 2025

MIAMI, Jan. 10, 2025 (GLOBE NEWSWIRE) — RESTEM – a clinical-stage biotechnology company that develops off-the-shelf, next-generation cell therapies designed to modulate the immune system, today announced its key objectives for 2025.

“We have made tremendous progress in the development of Restem-L, our umbilical cord lining stem-cell program, Restem-X, a secretome-based product, derived from Restem-L, as well as our activated NK (aNK) cell program in 2024,” said Andres Isaias, Chief Executive Officer of RESTEM. “We believe that 2025 will be a transformative year for RESTEM, as we advance our mission to unlock the full potential of Restem-L, Restem-X, and aNK cell therapies to address autoimmunity, orthopedic conditions, and age-associated disorders. Building on the excellent progress we achieved in 2024, including highly promising Phase 1 data of Restem-L in IIM, we look forward to multiple milestones that have the potential to bring significant value for both investors and patients. Specifically, we are on track to initiate IIMPACT, a potentially registrational Phase 2/3 clinical trial of Restem-L in IIM in Q1 2025.”

2024 Key Achievements

• Reported Phase 1 data of novel Restem-L program in adults with polymyositis/dermatomyositis (PM/DM), an orphan disease with a highly unmet medical need, (now referred to by the FDA as Idiopathic Inflammatory Myopathy (IIM)) demonstrating clinical improvements in 7 out of 9 patients (78%), with an average total improvement score (TIS) in the range of 30-35% lasting for one year and a 35% reduction in steroid usage within 6 months. These data were featured in an oral presentation at the American College of Rheumatology’s (ACR) annual meeting, ACR Convergence 2024.
• Received IND approval from the FDA for a Phase 2/3 clinical study in IIM, as well as a Phase 2 study in rheumatoid arthritis, another autoimmune indication.
• Received FDA Fast Track Designation and Orphan Drug Designation for Restem-L for the treatment of IIM.
• The executive team has been strengthened with the appointment of life science industry veterans Keith March, MD, PhD as Chief Medical Officer, and Nelson Cabatuan as Chief Financial Officer
• Increased Restem-L working bank up to approximately 3 million treatment doses of 100 million cells of the final product
• Increased autologous aNK cell production by 50%, enabling the capability for multiple doses
• Commenced allogeneic aNK production for individuals lacking functional NK cells

Expected Milestones in 2025

• Advance Restem-L in IIM to a potentially registrational, randomized, double-blinded Phase 2/3 trial in Q1 2025. The study will initially enroll 40 participants to evaluate the safety and efficacy of Restem-L and may be expanded up to 150 patients starting at 8 months for Phase 3 enrollment. Restem-L in IIM could also benefit from an accelerated approval pathway, priority reviews, and rolling submissions, allowing the Company to bring this potentially life-changing treatment to IIM patients sooner.
• Initiate Phase 2 clinical trial in rheumatoid arthritis patients who are refractory to standard of care in the second half of 2025.
• Advance aNK cell program to develop and utilize allogeneic aNK cells and aNK-derived exosomes, aiming to broaden their application for patients with age-associated disorders and diseases.
• Continued development of Restem-X, a secretome derived from a proprietary Restem-L product for orthopedic conditions

About RESTEM

RESTEM is a leading clinical-stage biotechnology company focused on developing off-the-shelf, next-generation cell therapies designed to modulate the immune system. Leveraging our proprietary products, robust clinical development expertise, and cutting-edge-manufacturing capabilities, we advance potentially groundbreaking, wholly owned programs, Restem-L, an umbilical cord lining stem cells (ULSCs) for auto-immune 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 intended to treat a broad range of disabling diseases and are designed to improve patient outcomes, as well as overall health and wellness. RESTEM is headquartered in Miami, Florida. For more information, please visit www.restem.com and follow us on X and LinkedIn.

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