GEN Pharmaceuticals (GENIL.IS) and Sulfateq B.V. on Monday announced positive results from a Phase 1 trial of SUL-238, a first-in-class, orally administered, mitochondria-directed drug candidate under development for Alzheimer's disease and other neurodegenerative conditions.

Data presented at the Alzheimer's Association International Conference 2025 in Toronto show that SUL-238 was safe and well-tolerated across doses from 50 to 2000 mg in 53 healthy elderly participants. The investigational compound demonstrated a favourable pharmacokinetic profile, including high cerebrospinal fluid penetration, and no dose-limiting adverse events.

The study comprised three parts, including dose escalation, pharmacokinetics at 1000 mg, and food effect evaluation at 2000 mg. All adverse events were mild or moderate, with similar incidence between SUL-238 and placebo. High brain penetration and consistent tolerability position SUL-238 for advancement into further clinical development.

Licensed by GEN from Sulfateq, SUL-238 is derived from hibernation biology and enhances mitochondrial function via complex I/IV activation. Preclinical data support its potential in treating neurodegenerative, cardiovascular, and renal diseases.

GEN, Türkiye's leading specialty pharmaceutical company, continues to expand its innovation pipeline through its GMP-certified manufacturing and R&D infrastructure. Sulfateq, based in the Netherlands, develops small molecules targeting mitochondrial health through strategic global collaborations.