RESTEM Presents Phase 1 Data of its ULSC Program in Polymyositis/Dermatomyositis at ACR Convergence 2024

– Oral presentation at ACR highlights safety and efficacy data from the Phase 1 trial of umbilical cord outer lining-derived stem cells (ULSCs) in patients with Polymyositis/Dermatomyositis (PM/DM), an orphan disease with highly unmet medical need, now referred to by the FDA as Idiopathic Inflammatory Myopathy (IIM)

– Clinical improvements observed in 7 out of 9 PM/DM 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

– Phase 2/3 dose established at 100 million ULSCs

– Phase 2/3 to initiate in 1Q 2025

CORONA, Calif., Nov. 18, 2024 (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 data from its Phase 1 trial of novel umbilical cord outer lining-derived stem cells (ULSCs) program in adults with (Polymyositis (PM)/Dermatomyositis (DM), an orphan disease with highly unmet medical need, now referred to by the FDA as Idiopathic Inflammatory Myopathy (IIM). These data were featured in an oral presentation at the American College of Rheumatology’s (ACR) annual meeting, ACR Convergence 2024, held November 14-19, 2024, in Washington, DC.

The presentation entitled, “Safety and Efficacy Data from a Phase I Trial of Umbilical Lining Stem Cells (ULSCs) in Adult Dermatomyositis/Polymyositis” was delivered by Michael R Bubb, M.D., University of Florida Health Rheumatology.

“We are extremely encouraged by the data presented at this year’s ACR which validates our ULSC approach to targeting the symptoms of PM/DM while reducing detrimental steroid use,” said Andres Isaias, Chief Executive Officer of RESTEM. “The treatment was well tolerated and led to clinically meaningful and durable improvements in the TIS in 78% of patients. In addition, all patients experienced significant improvements in manual muscle testing and extra-muscular disease which were sustained throughout the study. Based on the promising results from Phase 1 we look forward to advancing the study to Phase 2/3 in 1Q 2025.”

“Polymyositis and dermatomyositis (PM/DM) are debilitating inflammatory myopathies that can lead to persistent muscle weakness and disability. Current treatments involve steroids and immunosuppressive drugs that often come with substantial side effects, highlighting the urgent need for safer and more effective therapies,” said Rafael Gonzalez, PhD, Chief Development and Science Officer. “Phase 1 data are highly promising, demonstrating the safety and tolerability of ULSCs, along with encouraging initial clinical outcomes. Notably, treatment with ULSCs resulted in a 35% reduction in steroid dosage within 6 months, indicating its potential to modulate inflammation in the immune system. These results serve as a testament to the significant progress we are making in advancing cell therapies, and we believe that our ULSC therapy has the potential to become a transformative treatment option for PM/DM.”

Dr. Jonathan Krant, Chairman of Rheumatology for Monadnock Health Systems in Peterborough, NH, stated, “These are remarkable clinical data. If validated, a 35% reduction in steroid use has broad implications for PM/DM patients currently treated with standard induction and maintenance regimens”.

The Phase 1 trial (NCT04723303) was an open-label, dose-escalation study investigating a single intravenous infusion of allogeneic ULSCs in patients with PM/DM. The primary endpoint of the trial was to determine the dose-limiting toxicity (DLT) and recommended phase 2 dose. Nine subjects were enrolled, with three participants in each of the 3 dose levels, consisting respectively of a single administration of 50, 100, or 200 million ULSC, with 12 months of follow-up.

Key findings from the phase 1 trial include:

• Efficacy data showed that 6 of 9 patients met the criteria for a moderate improvement with a total improvement score (TIS) of ≥40% – a composite outcome of six core measures of myositis activity within 6 months of the infusion
• The criterion for clinically significant improvement, TIS of ≥20%, was met by 7 out of 9 subjects
• The average TIS was sustained in the 30-35% range over a 12-month period
• Manual Muscle Testing (MMT8) showed a significant improvement in strength between baseline and Month 6 of nearly 10 points, from 59 ± 4 at baseline to 68 ± 4 at Month 6 (mean ± SEM), p< 0.001
• Average prednisone dosage decreased by 35% at 6 months, when excluding data from one subject (a non-responder by TIS) who had a flare treated with corticosteroids at that time point
• Average reduction of nearly 50% of prednisone dosage in 6 of 7 participants that were daily users of prednisone (86%)
• The treatment was well tolerated with one adverse event (AE) of flushing reaction occurring during the infusion in the first patient. The infusion was temporarily paused, 150 mg intravenous hydrocortisone was administered, and the infusion was re-initiated with no recurrence

About PM/DM

Polymyositis is an autoimmune disease that causes inflammation 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. Both disorders can significantly compromise quality of life and are very challenging to treat. Currently, the only treatment for these disorders is immunosuppressive drugs, 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 designed to modulate the immune system. Leveraging our proprietary products, robust clinical development expertise, and cutting-edge-manufacturing capabilities, we advance two potentially groundbreaking programs, umbilical cord lining stem cells (ULSCs) for auto-immune diseases and natural killer cell (NK) 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