AI-Designed Drug AH-001 Complete U.S. Phase I Trial, Demonstrating Excellent Safety and Tolerability

AnHorn Medicines Achieves Key Milestone in AI-Discovered Therapeutics; Advances Toward Global Licensing and Phase II Development

TAIPEI, Oct. 29, 2025 /PRNewswire/ -- AnHorn Medicines, a biotech company leveraging generative AI to design small molecules and protein degraders, today announced positive results from its U.S. 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 U.S. 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.

Novel Topical Protein Degrader Targets the Root Cause of Hair Loss

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.

Global Market Demand Highlights Urgent Need for Innovation

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.

Existing treatments — finasteride and minoxidil — require continuous, long-term use, often deliver limited efficacy, and may cause undesirable side effects such as sexual dysfunction, hormonal imbalance, and scalp irritation. Surveys show that only 35–40% of patients are satisfied with current therapies, underscoring the unmet need for safer, more effective, and sustainable solutions.

AI-Powered Drug Design Opens New Era for Precision Therapeutics

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 20–50, offering an effective, convenient, and side effect sparing solution for hair restoration.

Next Steps: Global Partnerships and Phase II Development

With the global hair loss therapeutics market estimated to grow at a compound annual growth rate (CAGR) of 8.45%, reaching USD 4.76 billion 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 U.S. Phase I trial demonstrates that AI-designed drugs can achieve real-world clinical validation—not just computational promise," said Dr. 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."

About AnHorn Medicines

AnHorn Medicines, founded in 2020, is an AI-driven biotech company specializing in the development of novel small molecules and protein degraders for aesthetic medicine, oncology, and cancer supportive care. The company's proprietary AIMCADD AI platform integrates generative AI and molecular simulation to design highly potent, safe, and patentable drug candidates with unprecedented efficiency.

AnHorn is redefining how AI integrates into drug discovery—accelerating innovation, advancing precision medicine, and enabling a more sustainable and effective therapeutic development ecosystem.

Website: www.anhornmed.com

Linkedin: https://www.linkedin.com/company/anhorn-medicines/

SOURCE AnHorn Medicines