RESTEM, a clinical-stage biotechnology company, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Restem-L for the treatment of Idiopathic Inflammatory Myopathy (IIM). Restem-L is the company's umbilical cord outer lining stem cells (ULSCs) program, marking the second regulatory designation for Restem-L in IIM, following the recent Orphan Drug Designation.
Andres Isaias, Chief Executive Officer of RESTEM, stated that this designation underscores the need for novel therapeutic options for IIM patients. He highlighted that Phase 1 data demonstrated a 78% clinical improvement and a 35% reduction in steroid usage within 6 months with Restem-L treatment. The Fast Track designation could lead to accelerated approval, priority reviews, and rolling submissions, potentially expediting the availability of this treatment to patients.
Clinical Efficacy and Safety
Phase 1 trial data, presented at the 2024 American College of Rheumatology (ACR) Convergence, included nine patients. The results showed that 6 out of 9 patients achieved a Total Improvement Score (TIS) of at least 40% within 6 months, meeting the criteria for moderate improvement. Additionally, 7 out of 9 patients achieved a TIS of at least 20, indicating significant improvement. The trial also demonstrated an improvement of almost 10 points from baseline in Manual Muscle Testing (MMT8), increasing from 59 ± 4 at baseline to 68 ± 4 at 6 months post-treatment (p < 0.001). Furthermore, excluding one non-responding patient, the remaining eight patients showed a 35% reduction in average prednisone dosage at 6 months post-treatment.
The Phase 1 trial reported only one treatment-related adverse event (AE), a flushing reaction during the first patient’s infusion, which resolved after pausing the infusion and administering hydrocortisone.
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, leading to muscle inflammation, weakness, and skin rashes, significantly compromising the quality of life. Current treatments primarily involve immunosuppressive drugs, which can have significant toxicity and side effects.
Fast Track Designation
The FDA's Fast Track designation is designed to expedite the development and review of drugs for serious conditions with unmet medical needs. It allows for more frequent meetings with the FDA to discuss the drug's development plan, ensuring the collection of appropriate data for approval. Fast Track designation also provides eligibility for Accelerated Approval, Priority Review, and Rolling Review.
Future Development
RESTEM is planning to initiate IIMPACT, a potentially registrational Phase 2/3 clinical trial in IIM, in Q1 2025 to further assess the efficacy and safety of Restem-L.
