- Initiation of the IIMPACT study follows promising Phase 1 results where Restem-L demonstrated clinically meaningful efficacy and a significant reduction in steroid use within 6 months in Idiopathic Inflammatory Myopathy (IIM)
- Interim readout expected in 2H 2026
MIAMI, Feb. 03, 2026 (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 it had dosed the first patient in the Phase 2/3 IIMPACT trial of Restem-L, the Company’s, investigational umbilical lining modified progenitor cells (UMPC) program for the treatment of Polymyositis and dermatomyositis (PM/DM), a rare autoimmune disease, now defined by the FDA as idiopathic inflammatory myopathy (IIM).
“The initiation of this study in IIM represents a significant milestone in our Restem-L development program,” said Andres Isaias, Chief Executive Officer of RESTEM. “Restem-L’s potential has been clinically validated in our Phase 1 trial, which demonstrated meaningful clinical improvement in 78% of patients and an average reduction of more than 50% in steroid use within six months of treatment. These results represent an important step in advancing Restem-L development strategy and underscore our commitment to improving outcomes and quality of life for patients with rare and refractory autoimmune disorders. We look forward to sharing the interim readout in the second half of 2026.”
The Phase 2/3 IIMPACT trial (NCT07160205) is an adaptive, double-blinded, randomized, dose-repeating, crossover study evaluating Restem-L in PM/DM. The trial will enroll approximately 80 patients, randomized 1:1 to receive either Restem-L or a placebo. A portion of patients will be included from the Phase 1 trial, who also continue to be followed to reinforce the safety and efficacy data generated to date. The study design includes a planned interim analysis at the midpoint to assess safety and efficacy. Based on the interim findings, the trial may be expanded to enroll up to 150 patients, if necessary, to achieve the appropriate statistical significance and support registration. The participants will then cross over to either placebo or Restem L. This adaptive approach ensures optimal resource utilization and increases the likelihood of obtaining robust clinical data. The primary endpoint is the comparative change in total improvement score (TIS) and safety assessment, while the secondary endpoints include a pre-specified steroid step-down to solidify the concurrent efficacy and steroid reduction findings.
“The IIMPACT trial balances patient-centered care with robust scientific rigor while addressing the unmet needs of IIM,” said Keith March, MD, PhD, Chief Medical Officer of RESTEM. “The incorporation of steroid tapering with a crossover design is intended to generate clear and robust data, offering insights into both the therapeutic potential of Restem-L and the overall strategic execution of the program.”
Restem-L has received Fast Track and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of IIM, underscoring the significant medical need in this patient population. In addition to the newly initiated Phase 2/3 IIMPACT trial, Restem-L is currently being evaluated in a Phase 1/2a study in Facioscapulohumeral Muscular Dystrophy (FSHD).
About Idiopathic Inflammatory Myopathy (IIM)
Idiopathic inflammatory myopathies (IIM) are a group of conditions characterized by inflammation of muscles (myositis) and other body systems. Polymyositis, dermatomyositis, and antisynthetase syndrome are subtypes of IIM. Polymyositis causes inflammation, pain, and weakness of the skeletal muscles responsible for movement. Dermatomyositis is a form of polymyositis that is associated with skin rash, in addition to muscle inflammation. Antisynthetase syndrome is a systemic disease of muscle, but it often includes joint and lung involvement. These disorders can significantly compromise quality of life and are very challenging to treat. Currently, the treatments for these disorders are various immunosuppressive drugs, biologics, and steroids, which can be associated with significant toxicity and other side effects.
About RESTEM
RESTEM is a leading clinical-stage biotechnology company focused on developing off-the-shelf, next-generation cell therapies for autoimmune, inflammatory, and age-related diseases. Leveraging proprietary products, deep clinical expertise, and advanced manufacturing capabilities, RESTEM is advancing two potentially transformative programs: Restem-L, our umbilical cord lining progenitor cells (UMPC) therapy for autoimmune diseases, 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.
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