Steminent Biotherapeutics Unveils Promising Results for Novel MSC-based Therapy in Spinocerebellar Ataxia

Steminent Biotherapeutics, a Taiwan-based stem cell innovator, has revealed groundbreaking results from its Phase 2 clinical trials for Stemchymal®, an allogeneic adipose-derived mesenchymal stem cell (MSC) therapy targeting spinocerebellar ataxia (SCA). The company presented its findings at the World Orphan Drug Congress (WODC) USA 2025 in Boston, marking a significant milestone in the treatment of this rare neurodegenerative disease.

Stemchymal® Shows Disease-Modifying Potential in SCA3 Patients

The Phase 2 clinical trial, conducted in Taiwan, involved 56 patients with moderate-to-severe SCA3. Results demonstrated that Stemchymal® effectively stabilized disease progression, with some patients even showing improvement. The treatment group exhibited a statistically significant difference in Scale for the Assessment and Rating of Ataxia (SARA) scores compared to both the placebo group and the natural history of SCA3.

Dr. Jonathan Ko, Senior Vice President of Steminent, explained the unique mechanism of action: "By releasing a specific protein, Stemchymal® can initiate cellular autophagy to degrade toxic proteins that accumulate in nerves. This goes beyond mere symptom relief and shows the potential for disease modification."

A parallel Phase 2 trial in Japan, involving 59 SCA3 and SCA6 patients across 10 sites, yielded similar positive results, indicating reproducible efficacy across different patient populations.

Global Expansion and Strategic Partnerships

Steminent has been actively pursuing global partnerships to advance its clinical progress. The company has already secured regional licenses with Japan's REPROCELL in 2016 and Korea's SCM Lifescience in 2020. Additionally, Steminent has collaborated with Shibuya Corporation to introduce a customized cell processing isolator, enhancing its manufacturing capabilities.

Dr. Ling-Mei Wang, Steminent's Chair and CEO, highlighted the company's strategic recruitment of world-renowned experts in clinical research, regulatory affairs, and business development to bolster its international presence.

Future Outlook and Expansion Plans

Steminent aims to secure conditional approvals for Stemchymal® in Taiwan and Japan in 2025, while also applying for FDA approval for a Phase 2b trial in the U.S. The company's long-term goals include advancing to Phase 3 clinical trials and expanding indications to include Huntington's disease and other neurodegenerative disorders.

As Steminent continues to make strides in the field of regenerative medicine, it exemplifies the growing potential of Taiwan's biotech companies in the global advanced therapy industry.