Yoda Therapeutics Inc. (YODA) has dosed the first patient in its Phase 2 clinical trial of YA-101 for Multiple System Atrophy (MSA) in the United States, the company announced on March 26, 2025. This milestone marks a significant advancement in the development of potential treatments for this rare and debilitating neurodegenerative disorder.
MSA is characterized by severe motor impairment, including parkinsonism or cerebellar ataxia, accompanied by autonomic dysfunction. The disease progresses rapidly and is ultimately fatal, with patients experiencing a significant decline in quality of life. Currently, there are no approved disease-modifying therapies that can reverse or slow the progression of MSA.
"The dosing of our first patient in the Phase 2 clinical trial of YA-101 for Multiple System Atrophy represents a big step forward as we strive to bring a much-needed treatment option to individuals affected by this rare and debilitating neurodegenerative disorder," stated Yufeng Jane Tseng, PhD, Chief Executive Officer of YODA.
Novel Mechanism of Action
YA-101 is an AI-pioneered novel chemical entity (NCE) specifically designed to treat neurodegenerative diseases through a dual modulation mechanism. The compound works by inhibiting neuroinflammation and enhancing neural plasticity, addressing key pathological processes involved in MSA progression.
The drug has already demonstrated promising results in preclinical studies, showing improvements in motor-related behavioral impairments in animal models of ataxia and MSA. Additionally, YA-101 exhibited a favorable safety profile and was well-tolerated in healthy adults during its Phase 1 clinical trial conducted in Australia.
In recognition of its potential, YA-101 received Orphan Drug Designation (ODD) for MSA from the United States Food and Drug Administration (FDA) on July 1, 2022, providing incentives for its development as a treatment for this rare disease.
Phase 2 Trial Design
The Phase 2 trial is designed as a randomized, double-blind, placebo-controlled investigation that will evaluate the safety, tolerability, pharmacokinetics, and efficacy of YA-101 in subjects with MSA. The study will be conducted at multiple sites across the United States and Taiwan.
Eligible participants include MSA patients with walking ability, including those who require walking assistance. The comprehensive assessment will include measurements of clinical symptoms, biomarkers, and imaging to evaluate the drug's effects.
Detailed information about the trial is available on the clinical trials registry (NCT06848231).
The Burden of Multiple System Atrophy
MSA presents with debilitating symptoms that severely impact patients' quality of life. These include severe orthostatic hypotension (a sudden drop in blood pressure upon standing), urinary dysfunction, ataxic gait, dysmetria, scanning speech, and cognitive decline.
Current therapeutic approaches for MSA are limited to symptomatic management, such as dopamine therapy and medications for autonomic dysfunction. However, these treatments often provide inadequate relief and are not practical long-term solutions. The lack of disease-modifying therapies underscores the significant unmet medical need in this area.
About Yoda Therapeutics
Yoda Therapeutics Inc. is an AI-driven pharmaceutical company focused on advancing drug research and development for Central Nervous System (CNS) disorders. Based in Taipei, Taiwan, the company leverages artificial intelligence to pioneer innovative treatments for neurodegenerative diseases.
YA-101 represents the company's flagship drug candidate, now advancing through clinical development. The company's mission centers on addressing critical unmet medical needs by developing innovative, effective, and safe treatment options for patients with serious neurological conditions.
As the Phase 2 trial progresses, the scientific and patient communities will be watching closely to see if YA-101 can demonstrate efficacy in slowing or halting the progression of MSA, potentially offering hope to patients currently facing limited therapeutic options.
