RESTEM Receives FDA Fast Track Designation for Restem-L for Idiopathic Inflammatory Myopathy

• Second regulatory designation for Restem-L, a lead ULSC program, following the recent Orphan Drug Designation
• Recently reported Phase 1 data of Restem-L demonstrated a clinically meaningful efficacy and significant reduction in steroid use within 6 months in Idiopathic Inflammatory Myopathy (IIM)
• Company is on track to initiate IIMPACT, a potentially registrational Phase 2/3 clinical trial in IIM in Q1 2025

MIAMI, Jan. 07, 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 that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for Restem-L, the Company’s umbilical cord outer lining stem cells (ULSCs) program for the treatment of Polymyositis and Dermatomyositis (PM/DM), a rare autoimmune disease, now defined by the FDA as Idiopathic Inflammatory Myopathy (IIM). This is the second regulatory designation for Restem-L in IIM, following the recently granted Orphan Drug Designation.

“Receiving Fast Track designation for our ULSC program in IIM, followed by the recent Orphan Drug Designation, underscores the need for a change in the treatment paradigm and highlights the importance of developing novel therapeutic options for patients with IIM,” said Andres Isaias, Chief Executive Officer of RESTEM. “The recently presented Phase 1 data demonstrated that treatment with our Restem-L led to 78% clinical improvement and a 35% reduction in steroid usage within 6 months. With the Fast Track designation, Restem-L could benefit from accelerated approval pathway, priority reviews, and rolling submissions, allowing us to bring this potentially life-changing treatment to IIM patients sooner. We look forward to advancing the Restem-L program development and initiating IIMPACT, a potentially registrational Phase 2/3 clinical trial in IIM in Q1 2025.”

The FDA’s Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill unmet medical needs. The purpose is to accelerate the development of important new drugs for patients. Drugs granted Fast Track designation are eligible for more frequent meetings with the FDA to discuss the drug’s development plan and ensure the collection of appropriate data needed to support approval, as well as eligibility for Accelerated Approval, Priority Review and Rolling Review if relevant criteria are met.¹

1. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track

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 and dermatomyositis are subtypes of IIM. Polymyositis 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, Restem-L, our umbilical cord lining stem cells (ULSCs) program for auto-immune diseases and our 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 Cabautan
Restem Group, Inc.
+1.800.490.0924
ncabatuan@restem.com