Phase 1 results demonstrate that SUL-238, a first-in-class, orally administered, mitochondria-directed drug candidate, is safe and well-tolerated in healthy elderly volunteers, showing a favourable pharmacokinetic profile and high brain penetration. These findings support the advancement of SUL-238 into further clinical development for Alzheimer's and other neurodegenerative diseases.
ANKARA, TR / ACCESS Newswire / July 28, 2025 / GEN Pharmaceuticals (GENIL.IS), Türkiye's leading specialty pharmaceutical company, announced positive results from its Phase 1 clinical trial evaluating the safety, tolerability, and pharmacokinetics (PK) of first-in-class and novel orally administered mitochondria-directed drug candidate SUL-238 in healthy elderly volunteers. The findings were presented at the Alzheimer's Association International Conference 2025 (AAIC®) in Toronto.
This single oral ascending dose (SAD) Phase 1, first-in-human, randomized, double-blind, placebo-controlled study was conducted in three parts, involving a total of 53 healthy elderly adults. Part 1 included 6 cohorts (50, 100, 250, 500, 1000, and 2000 mg orally, n=23). In Part 2, the PK of a single 1000 mg oral dose was investigated in 10 healthy elderly adults. In Part 2B, the food effect was assessed using a randomized, single oral 2000 mg dose, two-treatment, two-period, crossover design (n=20).
The trial results showed that single oral doses of 50-2000 mg of SUL-238 were safe and well-tolerated, while demonstrating a favourable PK profile and high cerebrospinal fluid (CSF) penetration. These findings make SUL-238 a promising candidate for further clinical development in neurodegenerative diseases, including Alzheimer's disease.
No adverse effects (AEs) limited dose escalation, AE rates were comparable between SUL-238 and placebo, and all AEs were mild or moderate. The mean terminal elimination half-life was 0.86-3.80 hours, and the time to maximum plasma concentration was 0.50-1.39 hours. Under fed conditions, maximum plasma concentration (Cmax) and area under the plasma concentration-time curve (AUC0-8) decreased by 50% and 60%, respectively. CSF-to-plasma percentages at 2 and 8 hours post-dose were 21.1% (±6.6%) and 74.2% (±46.0%).
Abidin Gülmüs, Chairman of GEN, stated:
"We are very encouraged by these promising first-in-human results, marking an important step forward in our mission to address the underlying biology of Alzheimer's disease."
Nadir Ulu, MD, PhD, Vice President of R&D at GEN, added:
"With its excellent safety and PK profile in this Phase 1 trial, combined with robust preclinical data, SUL-238 represents a strong candidate for further clinical development to meet the critical unmet needs in neurodegenerative diseases, including Alzheimer's disease."
About SUL-238
SUL-238 is a novel, first-in-class, hibernation-derived small molecule that targets mitochondria, the cell's "powerhouse." It supports mitochondrial bioenergetics via complex I/IV activation and has improved mitochondrial function in rodent models of neurodegenerative, cardiovascular, and renal diseases, as well as aging. SUL-238 crosses the blood-brain barrier and has undergone extensive safety evaluation in preclinical and Phase 1 studies. GEN licenses SUL-238 from Sulfateq BV for neurodegenerative disease applications.
About GEN:
Founded in 1998, GEN is Türkiye's leading specialty pharmaceutical company, focused on developing innovative therapies across multiple therapeutic areas. GEN manufactures high-quality, competitive products at its GMP-certified facility and pursues original drug development through two dedicated R&D centers and investments.
About Sulfateq:
Sulfateq B.V. is an early-stage Dutch biotech company that fosters strategic collaborations with academic and industrial research centers to accelerate the development of innovative new medicines. It has developed a novel class of small molecules, the SUL-compounds, that maintain mitochondrial health.
For more information:
www.genilac.com.tr
www.sulfateqbv.com
Contact Information
Bulutay Günes
Sr. Head of Corporate Brand
b.gunes@genilac.com
Ali Ketencioglu
Investor Relations Manager
a.ketencioglu@genilac.com
Kees van der Graaf
Sulfateq CEO
info@sulfateqbv.com
SOURCE: GEN Ilaç ve Saglik Ürünleri A.S.
View the original press release on ACCESS Newswire:
https://www.accessnewswire.com/newsroom/en/healthcare-and-pharmaceutical/gen-and-sulfateq-bv-announce-positive-phase-1-trial-data-on-investiga-1053008
