On April 23, Steminent Biotherapeutics publicly presented Phase II clinical trial results of its stem cell therapy candidate Stemchymal® for spinocerebellar ataxia (SCA), a form of cerebellar atrophy, at the World Orphan Drug Congress (WODC) in the United States. The data showed that Stemchymal® demonstrated clear disease-modifying effects across multiple clinical evaluation scales, indicating the therapy’s potential to become a new treatment opportunity for patients with cerebellar atrophy.
Invited to speak at WODC, Steminent's clinical and scientific advisor Dr. Kurt Gunter—who brings over 30 years of experience in medical regulatory affairs, clinical development, and senior roles at the FDA—revealed the Stemchymal® Phase II results for the first time.
Dr. Gunter explained that there are approximately 50 known types of SCA. Steminent’s Phase II trial in Taiwan adopted a double-blind, randomized, placebo-controlled design involving 56 patients with moderate to severe SCA3 who scored 9 or above on the SARA (Scale for the Assessment and Rating of Ataxia), which ranges from 0 to 40. Patients received three intravenous injections and were evaluated using both the SARA and the newer fSARA scales. The results showed that the treatment group achieved disease stability and demonstrated significant clinical improvements.
After one year of follow-up, disease progression in the treatment group remained stable, with a noticeable delay in SARA score increases, consistent with the Phase I findings. Some patients even showed more than a one-point improvement. By contrast, untreated SCA3 patients typically experience an annual deterioration of 1.5 to 1.6 points in SARA score. According to U.S. and European data, the average annual increase in SARA score for SCA3 patients is approximately 1.41 points. The improvement seen in the treatment group stands in sharp contrast to the natural disease progression observed in the placebo group, further validating Stemchymal®’s potential to stabilize symptoms and slow degeneration. These findings were also corroborated by the parallel Phase II clinical trial conducted in Japan, reinforcing the consistency of results across international patient populations.
Following the WODC, Steminent will also participate in the 2025 International Society for Cell & Gene Therapy (ISCT) Annual Meeting held in the U.S. in early May. There, the company will present a clinical trial led by another clinical and scientific advisor, Professor Susan Perlman, further confirming Stemchymal®’s feasibility and safety in clinical settings. This paves the way for potential cell therapy applications in cerebellar atrophy and other neurological disorders.
Chairperson Ling-Mei Wang noted that the World Orphan Drug Congress is one of the most influential conferences in rare disease and orphan drug development, while the ISCT Annual Meeting is a key global gathering for leaders in regenerative medicine. Steminent’s presence at both events reflects its strong capabilities in international business development and highlights the global recognition of its R&D efforts. The release of these clinical results will support conditional drug approval applications in both Taiwan and Japan. In addition to previously licensed markets in Japan and South Korea, Steminent is actively pursuing further licensing opportunities in the U.S., Europe, and Southeast Asia, with the aim of expanding its global footprint and driving revenue growth through international technology transfer.
Resource: 仲恩公佈台日二期結果 臨床指標分數優異穩定
