YOKOHAMA, Japan – April 25, 2025 – REPROCELL Inc. today announced that its partner, Steminent Biotherapeutics Inc. (Headquarters: Taiwan; hereinafter "Steminent"), reported positive results on April 24, 2025, from Phase II clinical trials conducted in Taiwan and Japan for the stem cell therapy "Stemchymal®" (hereinafter "Stemchymal"). REPROCELL holds the exclusive commercialization license agreement for Stemchymal in Japan for the treatment of Spinocerebellar Ataxia (SCA).
Steminent's release: https://steminent.com/news/view2?news_category_id=3&news_id=13&page=1
This announcement was made by Dr. Kurt Gunter (former FDA), Clinical Scientific Advisor to Steminent, at the World Orphan Drug Congress (WODC) USA, held from April 22 to 24, 2025.
Overview of Announced Clinical Trial Results
- Target Disease/Drug: Stem cell therapy Stemchymal® for Spinocerebellar Ataxia (SCA)
- Trial Design (Taiwan): Randomized, double-blind, placebo-controlled comparative study
- Target Patients (Taiwan): 56 patients with moderate to severe SCA Type 3, with a SARA score[1] of 9 or higher
- Administration Method: Stemchymal® or placebo administered intravenously three times
- Evaluation Items: Assessment of efficacy using SARA score and fSARA score[2]
- Key Results:
- The Stemchymal®-treated group clearly demonstrated an effect in suppressing disease progression across multiple endpoints.
- At the one-year follow-up, the increase (worsening) in SARA scores was slower in the Stemchymal® group compared to the placebo group. Some patients showed an improvement of 1 point or more in their SARA score, consistent with observations from the Phase I clinical trial.
- Dr. Gunter pointed out that the SARA score for untreated SCA3 patients worsens by an average of 1.5-1.6 points per year (US and European data show an average of 1.41 points per year), supporting the possibility that Stemchymal® slows the progression of neurodegeneration and stabilizes the condition.
- Similar positive outcomes were also confirmed in the parallel clinical trial conducted with Japanese patients, representing a different ethnic group.
Future Plans
Steminent plans to participate in the annual meeting of the International Society for Cell & Gene Therapy (ISCT) in the US in early May 2025. Additional clinical data, led by Dr. Susan Perlman, Professor of Clinical Neurology at the University of California, Los Angeles (UCLA), will be presented.
Steminent’s Chairperson Ling-Mei Wang emphasized that Steminent’s participation in WODC and ISCT signifies growing global recognition. The clinical data will support temporary drug approval applications in both Taiwan and Japan. In addition to already-licensed markets in Japan and South Korea, the company is actively expanding licensing efforts into the U.S., Europe, and Southeast Asia.
REPROCELL holds the exclusive commercialization rights for Stemchymal® in Japan. We believe that the announcement of these positive Phase II clinical trial results in Taiwan strongly supports our efforts towards the development and commercialization of this drug within Japan.
REPROCELL will continue to work closely with Steminent to advance business development, aiming to deliver this new treatment option to Japanese patients suffering from Spinocerebellar Ataxia as quickly as possible. We will disclose further progress in a timely and appropriate manner.
Glossary
[1.] SARA score (Scale for the Assessment and Rating of Ataxia): An international standard index for evaluating the severity of ataxia. Higher scores indicate greater severity.
[2.] fSARA score: A newer index developed to evaluate the SARA score in more detail from a functional perspective.