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

Title

Steminent Unveils Promising Stem Cell Therapy for Spinocerebellar Ataxia with Positive Phase 2 Results at WODC USA 2025

Keywords

Steminent
Stemchymal®
Spinocerebellar Ataxia (SCA)
SCA3
Mesenchymal Stem Cells (MSC)
Cell Therapy
Neurodegenerative Disease
World Orphan Drug Congress (WODC)
Disease Modification
Neuroprotection
Clinical Trial
Autophagy
Ataxin-3
PolyQ Protein Disorders

Key Facts

Steminent presented its novel therapy Stemchymal® at the World Orphan Drug Congress (WODC) USA 2025, showcasing Phase 2 clinical trial data for spinocerebellar ataxia (SCA), specifically SCA3, the most prevalent subtype in Taiwan1 4.
Stemchymal® is an allogeneic, adipose-derived mesenchymal stem cell (MSC) therapy administered intravenously. It is designed to provide neuroprotective effects and address the underlying accumulation of neurotoxic proteins in SCA32 3 4.
The Phase 2 study was double-blind, randomized, placebo-controlled, and included 56 patients with moderate-to-severe SCA3 (SARA score ≥9), who received three intravenous injections over 12 months1.
Results showed statistically significant disease stabilization and, in some cases, clinical improvement, as measured by SARA scores, compared to expected natural disease progression and placebo, indicating potential disease-modifying effects1 4.
The therapy's mechanism is based on inducing autophagy to degrade mutant Ataxin-3, the main pathological protein in SCA3—a unique approach that targets the root cause of the disease, not just symptoms4.
Stemchymal® holds first-in-class potential for both SCA3 and other PolyQ protein-related disorders, such as Huntington's disease, and is protected by U.S. patents4.
There are currently no FDA-approved therapies for SCA. MSC-based therapies are being actively explored due to their neuroprotective, anti-inflammatory, and regenerative properties, with ongoing trials addressing major unmet needs in this field5.