Yoda Therapeutics Announces First Patient Dosed in Phase 2 Trial of YA-101 for Multiple System Atrophy

TAIPEI, March 26, 2025 /PRNewswire/ -- Yoda Therapeutics Inc. (YODA), an AI-driven company that focuses on Central Nervous System (CNS) drugs, today announced the dosing of the first patient in the Phase 2 trial of YA-101 for Multiple System Atrophy (MSA) in the United States.

MSA is a rare and rapidly progressive neurodegenerative disorder that causes severe motor impairment (parkinsonism or cerebellar ataxia) accompanied by autonomic dysfunction, leading to a significant decline in patients' quality of life. Currently, no disease-modifying therapies exist to reverse or slow the progression of MSA, and symptomatic management offers limited relief and is not a practical long-term solution.

YA-101, a novel chemical entity (NCE), is designed to treat neurodegenerative diseases by inhibiting neuroinflammation and enhancing neural plasticity. Following the successful completion of safety and tolerability assessments in Phase 1 clinical trial with healthy volunteers in Australia, YA-101 has entered Phase 2 trial, which aims to evaluate the safety, tolerability, pharmacokinetics, and efficacy of YA-101 in subjects with MSA at multiple sites in the United States and Taiwan. This Phase 2 trial is a randomized, double-blind, placebo-controlled investigation for MSA patients with walking ability, including those who need walking assistance. Measurement of clinical symptoms, biomarkers, and imaging will allow a comprehensive assessment. Further details are available at https://clinicaltrials.gov/study/NCT06848231.

"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.

About Multiple System Atrophy (MSA)

MSA is a rare, rapidly progressive, and fatal neurodegenerative disorder characterized by motor impairment (parkinsonism or cerebellar ataxia) accompanied by autonomic dysfunction. Patients experience debilitating symptoms, including severe orthostatic hypotension, urinary dysfunction, ataxic gait, dysmetria, scanning speech, and cognitive decline, significantly impacting their quality of life. Current therapies focus on symptomatic management, such as dopamine therapy and medications for autonomic dysfunction, but often yield disappointing results. To date, no approved agents are available for MSA.

About YA-101

YA-101 is an AI-pioneered NCE with a dual modulation mechanism specifically designed to treat neurodegenerative diseases. YA-101 not only demonstrated improvements in motor-related behavioral impairments in preclinical animal models of ataxia and MSA, but also exhibited a favorable safety profile and was well-tolerated in healthy adults during the Phase 1 trial. On July 1, 2022, it received Orphan Drug Designation (ODD) for MSA from the United States Food and Drug Administration (FDA).

About Yoda Therapeutics Inc. (YODA)

YODA is an AI-driven company advancing drug research and development targeting CNS disorders. YA-101 is the company's flagship drug, currently in Phase 2 clinical trial for the treatment of MSA. Guided by a mission to address critical unmet medical needs, we are dedicated to offering new treatment options that are innovative, effective, and safe. The company is based in Taipei, Taiwan. For more information, please visit the company's website at https://yodapharma.com/.

SOURCE Yoda Therapeutics Inc.