MindRank's AI-Designed Oral GLP-1 Drug Achieves 10.3% Weight Loss in Phase 2b Trial
Key Insights
MindRank (search)'s AI-designed oral GLP-1 receptor agonist MDR-001 demonstrated statistically significant weight reductions of 8.2% to 10.3% at 24 weeks compared to 2.5% with placebo in a Phase 2b trial.
The drug showed excellent tolerability with no treatment-related serious adverse events and only 0.8% discontinuation rate due to adverse events.
MDR-001 represents the first AI-designed oral GLP-1 receptor agonist to report positive Phase 2b data globally, positioning it for Phase 3 development.
MindRank (search) announced positive topline results from a Phase 2b clinical trial of its proprietary AI-designed oral GLP-1 receptor agonist (GLP-1RA), MDR-001, demonstrating clinically meaningful weight reduction in adults with obesity or overweight. The 24-week, randomized, placebo-controlled study represents a significant milestone as the first AI-designed oral GLP-1RA to report positive Phase 2b data globally.
Significant Weight Loss Achieved Across Dose Groups
In the multi-center study conducted across 19 clinical sites in China, participants receiving MDR-001 achieved mean body weight reductions ranging from 8.2% to 10.3% (7.4-9.2 kg) compared to 2.5% (2.4 kg) in the placebo group (p<0.00001). Placebo-adjusted weight loss ranged from 7.1% to 7.8%, with 70.9% to 85.4% of participants achieving at least 5% weight loss and 34.5% to 48.1% achieving at least 10% weight loss.
The study enrolled 317 adult participants who were either overweight with at least one weight-related comorbidity, or obese individuals with inadequate response to lifestyle interventions. Baseline characteristics were well balanced across treatment arms, with a mean body weight of 90.1 kg, BMI of 32.3 kg/m², and 53.5% of participants being female.
Comprehensive Metabolic Benefits Observed
Beyond weight reduction, MDR-001 delivered significant improvements in key cardiometabolic markers, including waist circumference, blood pressure, and lipid profiles, underscoring its potential as a comprehensive metabolic therapy. Hepatic safety analyses showed no evidence of transaminase elevation, even among approximately 20% of participants with pre-existing liver impairment. ALT and AST levels were significantly reduced in the MDR-001 treatment groups compared to placebo.
Favorable Safety Profile with Minimal Discontinuations
MDR-001 demonstrated excellent tolerability with no treatment-related serious adverse events (SAEs) reported. The most common treatment-emergent adverse events (TEAEs) were mild to moderate gastrointestinal symptoms, such as nausea, vomiting, and diarrhea, which were predominantly observed during the initial 6-week dose-escalation period and resolved within 1 to 5 days. No clinically relevant increases in heart rate were observed, and the overall discontinuation rate due to TEAEs was only 0.8%.
AI-Driven Drug Discovery Validation
MDR-001 is a novel, β-arrestin 2 (search) selective, orally bioavailable small-molecule GLP-1 RA discovered using MindRank (search)'s proprietary AI platform, Molecule Pro™. The compound has demonstrated excellent functional potency and selectivity, with favorable ADME properties and oral bioavailability, as well as desirable pharmacokinetics, safety and tolerability.
Professor Linong Ji, Leading Principal Investigator of the study and Director of Endocrinology and Metabolism at Peking University People's Hospital, remarked: "MDR-001 has demonstrated compelling efficacy, tolerability, and metabolic benefit in this trial. Its oral formulation and favorable safety profile make it a highly attractive candidate for long-term obesity management, supports its progression to Phase 3 trials."
Phase 3 Development and Future Directions
Dr. Zhangming Niu, Founder and CEO of MindRank (search), commented: "The outstanding Phase 2b data validate the power of our AI-driven drug discovery engine and our internal development capabilities. We are rapidly advancing preparations for global Phase 3 trials and continue to explore once-daily dosing regimens to enhance patient convenience."
The Phase 2b clinical trial (NCT06606483) involved participants randomized to receive twice-daily doses of MDR-001 at 90 mg, 120 mg, 150 mg or 180 mg, or matching placebo, over a 24-week treatment period, including a structured dose-escalation phase to optimize tolerability. The primary efficacy endpoint was the percent change in body weight from baseline at Week 24.
Preliminary findings support continued development of a once-daily formulation to enhance dosing convenience. These results lay a solid foundation for late-stage development and future partnerships aimed at addressing the global obesity epidemic.