AnHorn Medicines has announced positive results from its US phase I clinical trial of AH-001, an AI-generated topical protein degrader developed for the treatment of androgenetic alopecia (male pattern hair loss).
The trial confirmed that AH-001 was safe and well-tolerated across all dose levels, with no drug-related adverse events. This achievement marks the first successful completion of a US human clinical trial for an AI-designed new drug originating from Taiwan, underscoring the clinical viability and precision design capability of AnHorn’s proprietary AI platform.
AH-001 is a novel, AI-designed small molecule that works through a targeted protein degradation mechanism to selectively eliminate the androgen receptor (AR) – a key driver in hormone-related hair loss.
Applied topically, AH-001 acts locally at the disease site, directly addressing the root cause of androgenetic alopecia while avoiding the systemic side effects commonly associated with oral hormonal inhibitors such as finasteride.
Preclinical and phase I data demonstrated a strong safety profile and excellent local tolerability, setting the stage for dose optimization and efficacy trials in phase II.
Androgenetic alopecia is a common condition worldwide, including 50% of men by age 50 and 40% of women after menopause or childbirth. Despite its high prevalence, therapeutic innovation in this field has stagnated for over two decades.
Surveys show that only 35–40% of patients are satisfied with current therapies, underscoring the unmet need for safer, more effective, and sustainable solutions.
Developed using AnHorn’s AIMCADD generative AI platform, AH-001 showcases how AI can design clinically viable small molecules with high specificity, safety, and patentability. Its precision-targeted AR degradation introduces a new therapeutic paradigm for hair loss and other hormone-driven diseases.
AH-001 is being advanced as a topical, next-generation therapy for adults aged 20 to 50, offering.
The global hair loss therapeutics market is estimated to grow at a compound annual growth rate (CAGR) of 8.45%, reaching $4.76bn by 2030, according to a report by Grand View Research. AnHorn Medicines is actively preparing for phase II clinical trials and pursuing strategic licensing and co-development partnerships worldwide to accelerate clinical and commercial progress.
“The successful completion of AH-001’s US phase I trial demonstrates that AI-designed drugs can achieve real-world clinical validation, not just computational promise,” said Chu-Chiang Lin, founder and CEO of AnHorn Medicines.
“This milestone highlights the transformative power of generative AI in drug development. We’re now focused on advancing AH-001 into phase II and establishing global partnerships to bring this first-of-its-kind therapy to patients worldwide.”
Jim Cornall is editor of Deeptech Digest and publisher at Ayr Coastal Media. He is an award-winning writer, editor, photographer, broadcaster, designer and author. Contact Jim here.