RESTEM, a clinical-stage biotechnology company, is advancing its lead candidate, Restem-L, into a potentially registrational Phase 2/3 clinical trial (IIMPACT) for Idiopathic Inflammatory Myopathy (IIM) in Q1 2025. This follows promising Phase 1 data demonstrating clinical improvements in a significant portion of treated patients.
Restem-L Shows Promise in Phase 1 for IIM
In 2024, RESTEM reported Phase 1 data for Restem-L in adults with polymyositis/dermatomyositis (PM/DM), now classified as IIM by the FDA. The results indicated that 78% of patients (7 out of 9) experienced clinical improvements, with an average total improvement score (TIS) in the range of 30-35% sustained for one year. Additionally, there was a 35% reduction in steroid usage within six months among these patients. These findings were presented at the American College of Rheumatology's (ACR) annual meeting, ACR Convergence 2024.
Advancing Restem-L to Phase 2/3
The upcoming Phase 2/3 trial is designed as a randomized, double-blinded study, initially enrolling 40 participants to assess the safety and efficacy of Restem-L. The study may expand to include up to 150 patients, with Phase 3 enrollment commencing at eight months. Restem-L has the potential to benefit from accelerated approval pathways, priority reviews, and rolling submissions, potentially expediting its availability to patients with IIM.
Regulatory Support and Manufacturing Expansion
RESTEM has received IND approval from the FDA for the Phase 2/3 clinical study in IIM, as well as a Phase 2 study in rheumatoid arthritis. The FDA has also granted Fast Track Designation and Orphan Drug Designation to Restem-L for the treatment of IIM. To support clinical development and potential commercialization, RESTEM has increased its Restem-L working bank to approximately 3 million treatment doses, each containing 100 million cells of the final product.
Expanding Pipeline: aNK Cells and Restem-X
In addition to Restem-L, RESTEM is advancing its activated NK (aNK) cell program to develop allogeneic aNK cells and aNK-derived exosomes for age-associated disorders and diseases. The company also continues to develop Restem-X, a secretome derived from Restem-L, for orthopedic conditions. RESTEM has increased autologous aNK cell production by 50%, enabling the capability for multiple doses and has commenced allogeneic aNK production for individuals lacking functional NK cells.
Management Perspective
"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," said Andres Isaias, Chief Executive Officer of RESTEM.
