#USA #Boston #Spinocerebellar Ataxia #Steminent Biotherapeutics #Stemchymal

Steminent Biotherapeutics to Showcase MSC Therapy for SCA

Steminent Biotherapeutics Inc., established in 2007, is poised to make a significant impact in the field of regenerative medicine by presenting its flagship investigational therapy, Stemchymal®, at two prominent international conventions: the World Orphan Drug Congress (WODC) USA 2025 and the ISCT 2025 Annual Meeting. This debut marks a crucial step for the company, highlighting its commitment to developing therapies for rare neurodegenerative diseases.

Understanding Spinocerebellar Ataxia
Spinocerebellar Ataxia (SCA) is a group of rare neurodegenerative disorders that lead to progressive degeneration of the cerebellum, leading to loss of coordination and balance. Currently, treatment options for SCA remain limited, particularly for SCA3, the most common subtype. Stemchymal® utilizes allogeneic adipose-derived mesenchymal stem cells (MSCs) and represents a novel approach designed to not only alleviate symptoms but to target the underlying causes of the disease.

Mechanism of Action
What separates Stemchymal® from traditional therapies is its unique mechanism that induces cellular autophagy to degrade mutant Ataxin-3, a protein implicated in the progression of SCA3. This innovative treatment, administered intravenously, promises neuroprotective benefits while also alleviating the accumulation of neurotoxic proteins, thereby addressing the root cause of the disorder rather than just masking its symptoms. With patent protection granted in the U.S., Stemchymal® is setting new standards in the treatment of PolyQ protein-related disorders, including Huntington's disease.

Phase 2 Trials and Promising Data
At the WODC USA 2025, the results from a Phase 2 trial conducted in Taiwan will be showcased. In this trial, patients suffering from moderate-to-severe SCA3 received three doses of Stemchymal® over 12 months. The results indicate statistically significant stabilization of the disease, as measured by SARA and fSARA scores, with some individuals experiencing notable clinical improvement. This data stands in stark contrast to the expected annual deterioration typically observed in placebo-controlled and natural history studies. Furthermore, these encouraging results are supported by another multi-center Phase 2 trial conducted in Japan involving SCA3 and SCA6 patients, reinforcing the therapy's potential efficacy across diverse patient demographics.

Future Outlook
In early May, during the ISCT 2025, Dr. Susan Perlman from UCLA, who serves as the clinical advisor for Steminent, will present a U.S.-based late-breaking case report, which is expected to further substantiate the safety and clinical feasibility of Stemchymal®.

Steminent has positioned itself as a trailblazer in the field, being the sole Asian company recognized as an Accredited Foreign Manufacturer of regenerative medical products by Japan's Ministry of Health, Labour and Welfare (MHLW). Their state-of-the-art facility is equipped for good manufacturing practices (GMP), enabling scalable production ready for export. With regional collaborations established in Japan and South Korea, and ongoing global partnership discussions, the company anticipates conditional approvals within Taiwan and Japan, while also pursuing FDA clearance for launching a Phase 2b trial in the U.S. in 2025.

As Steminent Biotherapeutics continues to advance its innovative therapies, the international spotlight on their work at these forthcoming conferences embodies a promising future for patients battling the challenges of SCA.