Yoda Therapeutics Begins Phase 2 Trial of YA-101 for Multiple System Atrophy

TAIPEI, March 26, 2025 – Yoda Therapeutics Inc. (YODA), a company focused on developing drugs for Central Nervous System (CNS) conditions using artificial intelligence, has reached a significant milestone. The organization has administered the first dose in its Phase 2 clinical trial of YA-101, a drug aimed at treating Multiple System Atrophy (MSA), in the United States.

Multiple System Atrophy is a rare neurodegenerative disorder that progresses rapidly, leading to severe motor impairments such as parkinsonism or cerebellar ataxia, along with autonomic dysfunction. These symptoms drastically diminish the quality of life for patients. Currently, there are no therapies available that can alter the course of MSA or sufficiently alleviate its symptoms. Management of the condition focuses on symptomatic relief, but this approach is not sustainable for long-term care.

YA-101 is a novel chemical entity designed specifically to combat neurodegenerative diseases by inhibiting neuroinflammation and promoting neural plasticity. After successfully completing a Phase 1 trial in Australia that assessed the drug's safety and tolerability in healthy volunteers, YA-101 has moved into Phase 2. This stage of the trial will evaluate the safety, tolerability, pharmacokinetics, and effectiveness of YA-101 in patients with MSA across various sites in the United States and Taiwan. The trial is structured as a randomized, double-blind, placebo-controlled study, including MSA patients who can walk independently or require assistance. Comprehensive assessments will be conducted through the measurement of clinical symptoms, biomarkers, and imaging.

Yufeng Jane Tseng, PhD, CEO of Yoda Therapeutics, remarked, "Initiating the Phase 2 clinical trial by dosing the first patient is a significant advancement towards providing a much-needed treatment for individuals suffering from this rare and challenging neurodegenerative disorder."

Multiple System Atrophy is not only rare and rapidly progressive but also fatal. The disorder is marked by motor impairments like parkinsonism or cerebellar ataxia and is often accompanied by autonomic dysfunction. Patients endure debilitating symptoms, such as severe orthostatic hypotension, urinary problems, ataxic gait, dysmetria, scanning speech, and cognitive decline, all of which severely impact their daily lives. Current treatment methods focus primarily on symptomatic management, utilizing dopamine therapy and medications for autonomic issues, but these often produce unsatisfactory results. As of now, there are no approved treatments specifically for MSA.

YA-101, the investigational drug, stands out as an AI-developed novel chemical entity with dual modulation capabilities, engineered for the treatment of neurodegenerative conditions. In preclinical examinations involving animal models of ataxia and MSA, YA-101 showed improvements in motor-related behavioral issues, alongside a promising safety profile. The drug was well-tolerated in healthy adults during the Phase 1 trial. Furthermore, on July 1, 2022, YA-101 was granted Orphan Drug Designation (ODD) for MSA by the United States Food and Drug Administration (FDA).

Yoda Therapeutics Inc., based in Taipei, Taiwan, is an AI-driven enterprise dedicated to advancing drug research and development for CNS disorders. With YA-101 as its leading drug currently in Phase 2 clinical trials for MSA treatment, YODA is committed to addressing unmet medical needs by providing innovative, effective, and safe treatment options.