Steminent Stands Ready to Showcase Novel MSC-based Therapy for Spinocerebellar Ataxia at Global Stage

The company’s most anticipated product, Stemchymal®, an allogeneic adipose-derived mesenchymal stem cell (MSC) therapy, has completed Phase 2 clinical trials in Taiwan and Japan for spinocerebellar ataxia (SCA) and has received orphan drug designation in the U.S. and Japan.

Prior to the first-ever public presentation of the positive Phase 2 findings in Taiwan, GeneOnline has recently interviewed Dr. Ling-Mei Wang, Steminent’s Chair and CEO, and Dr. Jonathan Ko, Senior Vice President of the company. The interview focuses on the scientific progress of this groundbreaking product for SCA treatment, the promising clinical results to be debuted in the international arena, plus Steminent’s strategies for advancing global licensing and partnerships.

To date, there are almost 50 known genetic SCA subtypes with varying prevalence across the world. For instance, SCA3 is the most common subtype in Taiwan, while SCA3 and SCA6 are the most prevailing ones in Japan. This progressive disease typically occurs in young adults, resulting in irreversible neuron degeneration that leads to symptoms including unsteady gait, poor coordination of limbs, and speech impediments. SCA is currently incurable, while existing treatments only provide symptom relief.

According to Dr. Ko, Steminent identified SCA as a drug development target not only because of being undruggable, but also because The symptoms onset of SCA patients are usually between the ages of 20 and 40, reaching their prime working and family years, meaning that the disease could pose a significant social and financial burden.

Mutations in the Ataxin-3 gene are the primary culprits for SCA3. Abnormal Ataxin-3 proteins with an expanded polyglutamine tract (PolyQ proteins) formed from these mutations could accumulate in the brain, causing neurotoxicity and ultimately premature apoptotic death of neurons. Based on Steminent’s research findings, apart from neuroprotective effects like other potential SCA therapeutics, the distinctive action of the MSC-based product lies in its ability to clear the pathogenic mutant Ataxin-3.

In their preclinical study in two SCA3 transgenic mice, Steminent’s team showed that the off-the-shelf cell therapy product significantly cleared mutant Ataxin-3 in the mice’s brains and improved their motor function. “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,” said Dr. Ko. The relatively unique mechanism of action has filed a U.S. patent and holds promise for future expansion of indications into other PolyQ disorders such as Huntington’s disease.

On April 23, Steminent officially presented the results of its Phase 2 clinical trial of Stemchymal® in Taiwan at the World Orphan Drug Congress (WODC) USA 2025 in Boston.

The study was conducted in a double-blind, randomized, and placebo-controlled design. Three intravenous injections of the MSC-based product were administered to 56 patients with moderate-to-severe SCA3 with SARA (Scale for the Assessment and Rating of Ataxia) scores of 9 or higher.

The SARA scale consists of eight functional domains, such as gait, stance, sitting, and speech disturbance, with total scores ranging from 0 to 40, where higher scores indicate more severe ataxia. Natural history studies have shown that untreated SCA3 patients deteriorate by an average of 1.5 to 1.6 points per year, while combined data from the U.S. and Europe show an average increase of 1.41 points per year.

After one year of treatment and follow-up, disease stabilization was observed in the Stemchymal® group, showing a marked delay in the increase in SARA scores consistent with the findings obtained in the previous open-label phase 1 trial, while some patients even saw an improvement of more than one point. In terms of changes in total SARA scores, apart from a superior performance than the placebo group, the treatment group also demonstrated a statistically significant difference when compared to the worsening trend seen in the natural history of SCA3, signaling the disease-modifying effects of the MSC-based product.

Notably, the company has also completed a Phase 2 trial for Stemchymal® in Japan, involving 59 SCA3 and SCA6 patients across 10 sites. Compared with both the placebo group and historical control, the treatment group showed positive results highly similar to the above-mentioned Taiwan trial, indicating that the stem cell product is likely to achieve reproducible and consistent efficacy in different patient populations, thus laying the groundwork for further global licensing and applications for multinational marketing approvals.

According to Dr. Ko, while there is a competing experimental drug currently undergoing Phase 3 trials in the U.S., it utilizes the fSARA scale (functional SARA, which is a shortened and modified version of the original scale) to achieve its efficacy goals. Stemchymal® has, however, demonstrated consistent disease-modifying effects when evaluated with the full SARA and fSARA scores, giving it a competitive advantage in treating SCA.

Following the presentation at the WODC USA 2025, Steminent will attend the ISCT 2025 Annual Meeting in New Orleans in early May to unveil a late-breaking case report by Dr. Susan Perlman, a UCLA neurology professor and clinical advisor of the company. As a physician-led observational study involving a U.S. SCA3 patient, its preliminary results also support the feasibility and safety of Stemchymal® for clinical use.

On top of the impressive clinical progress, Steminent keeps expanding its overseas strategic partnerships, including the regional licenses of Japan’s REPROCELL in 2016 and Korea’s SCM Lifescience in 2020, along with discussions with potential commercialization partners. Furthermore, through collaboration with Shibuya Corporation, Steminent has introduced a customized cell processing isolator to create a state-of-the-art cell therapy manufacturing platform, which not only complies with PIC/S GMP standards, but also makes it the only Asian company to be recognized as an “Accredited Foreign Manufacturer” of regenerative medical products by Japan’s Ministry of Health, Labor and Welfare.

CEO Dr. Ling-Mei Wang added that while advancing its clinical programs, Steminent has also recruited world-renowned experts with extensive experience in clinical research, regulatory affairs, and business development to join its management and advisory teams, intending to establish itself as one of the pioneers in Taiwan’s regenerative medicine industry and to enhance its international visibility.

In 2025, Steminent aims to secure conditional approvals for Stemchymal® in Taiwan and Japan and apply for FDA approval for a Phase 2b trial in the U.S. By promoting international financing, licensing and collaborations, the Taiwan-based stem cell innovator aspires to bring the MSC-based product to clinical Phase 3 and expand its indication into Huntington’s disease and other neurodegenerative disorders, thus showing the enormous potential of Taiwan’s biotech companies in the global advanced therapy industry.