iPS Cell-Based Drug Discovery Reveals Efficacy of an ALS Drug Candidate
First written in FY2023-Q3. 3 of 4 entries say "Cited as achieved", first in FY2023. Latest entry: FY2025.
No other company in this theme has enough history to compare yet.
- FY2025Cited as achievedAnnual report
「On 18 August 2025 (U.S. time), the paper “Ropinirole functions through a dopamine receptor D2-independent mechanism to ameliorate amyotrophic lateral sclerosis phenotypes in TARDBP-mutant iPSC-derived motor neurons” was published in the international academic journal Journal of Neurochemistry.」
The company reports publication in an international academic journal of joint research on the mechanism of action of ropinirole hydrochloride in ALS. - FY2024Cited as achievedAnnual report
「Median survival was 50.3 weeks in the ropinirole hydrochloride group (active-treatment group) and 22.4 weeks in the placebo group. Over the one-year trial period, the results indicated that ropinirole hydrochloride might delay disease progression by 27.9 weeks, or approximately 7 months, compared with placebo.」
The company reiterates the prior Phase I/IIa trial results regarding safety, tolerability and efficacy. - FY2023Cited as achievedAnnual report
「In the physician-initiated clinical trial (Phase I/IIa trial) conducted by Keio University, all patients were able to take the maximum dose of 16 mg, most adverse events were mild, and no patient discontinued treatment because of an adverse event. These results confirmed the safety and tolerability of ropinirole hydrochloride in ALS patients.」
The company cites the prior Phase I/IIa trial as evidence of safety and tolerability, as well as the potential to delay disease progression in the active-treatment group by 27.9 weeks. - FY2023-Q3First statedAnnual report
「We discovered, using iPS cells, that ropinirole hydrochloride is effective against ALS pathology. This represents the world's first successful identification through iPS cell-based drug discovery of a drug that may provide greater clinical suppression of disease progression than existing drugs. The results were published on 2 June 2023 (Japan time) in Cell Stem Cell, an international scientific journal published by Cell Press that specializes in research using iPS cells and other stem cells.」
The company states that its research findings on ropinirole hydrochloride were published in Cell Stem Cell on 2 June 2023.