CAMBRIDGE, Massachusetts, January 7, 2024
Insilico Medicine, a clinical-stage leader in generative AI-driven drug discovery and development, has announced promising topline results from two Phase I clinical trials for ISM5411, a novel gut-restricted PHD-specific inhibitor targeting inflammatory bowel disease (IBD). The trials, conducted in Australia and China, confirm the safety and pharmacokinetics (PK) profile of ISM5411, marking a significant step forward in the development of AI-designed therapeutics (ClinicalTrials.gov Identifier: NCT06012578).
Key Findings
The Phase I studies assessed the safety, tolerability, PK, and food effects of ISM5411, enrolling 124 healthy volunteers across both sites. The trials included single ascending dose (SAD), multiple ascending dose (MAD), and food effect (FE) components.
- Safety: ISM5411 was well tolerated across all dose groups, with no serious adverse events or discontinuations due to treatment. Most treatment-emergent adverse events (TEAEs) were mild (Grade 1) and resolved by the study’s conclusion.
- Pharmacokinetics: The drug exhibited a gut-restrictive PK profile with minimal systemic exposure, a high fecal-to-plasma ratio, and no drug accumulation after 14 days of dosing — validating its potential for localized treatment with reduced systemic side effects.
Accelerated AI-Driven Drug Development
ISM5411 was developed using Insilico’s proprietary AI-driven Chemistry42 platform, which enabled the rapid synthesis and screening of 115 molecules, culminating in its preclinical candidate nomination within just 12 months. The development process was highlighted in a Nature Biotechnology publication, setting a new industry benchmark for accelerated AI-powered drug discovery.
Strategic Advancements in IBD Treatment
Current therapies for IBD, including ulcerative colitis and Crohn’s disease, often face limitations in efficacy and significant side effects. ISM5411 aims to address these gaps by reducing inflammation and promoting epithelial repair in the gastrointestinal tract.
“These results reinforce the potential of ISM5411 as an innovative solution for IBD,” stated Dr. Philip Ryan, Principal Investigator for the Australian trial.
Dr. Carol Satler, Vice President for Clinical Development at Insilico, emphasized, “Given the unmet needs in IBD management, ISM5411’s gut-restrictive properties represent a significant advancement. We are eager to advance this candidate into Phase II trials to evaluate its therapeutic impact on ulcerative colitis patients.”
Insilico Plans Phase II Trial in 2025
Insilico plans to initiate a Phase II proof-of-concept trial for ISM5411 in active ulcerative colitis patients in late 2025. This milestone builds on the company’s track record of leveraging generative AI in drug discovery:
- 22 preclinical candidates nominated since 2019
- 10 programs advancing to clinical stages
- 1 program completing Phase IIa
AI’s Expanding Role in Drug Development
“IBD represents a substantial healthcare challenge globally,” said Alex Zhavoronkov, PhD, Founder and CEO of Insilico Medicine. “Our commitment to leveraging generative AI allows us to pioneer therapies that minimize disease burden, reduce adverse effects, and enhance patient quality of life. ISM5411 exemplifies the transformative potential of AI in creating life-changing medicines.”
For more information about Insilico’s clinical programs, visit: ClinicalTrials.gov – Study NCT06012578.
About Insilico Medicine
Insilico Medicine is a trailblazer in AI-driven drug discovery, bridging biology, chemistry, and clinical research. With its proprietary platforms, the company develops breakthrough solutions for a broad spectrum of diseases, including cancer, fibrosis, autoimmune conditions, and age-related diseases.
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