- MDR-001 achieved statistically significant weight reductions of 8.2% to 10.3% at 24 weeks compared to 2.5% with placebo (p<0.00001), with up to 48.1% of participants achieving ≥10% weight loss.
- No treatment-related serious adverse events were reported. Most treatment-emergent adverse events were mild to moderate gastrointestinal symptoms. Discontinuation rate was 0.8%.
- Preliminary findings support continued development of a once-daily formulation to enhance dosing convenience.
HANGZHOU, China and LONDON, June 24, 2025 (GLOBE NEWSWIRE) -- MindRank, a clinical stage artificial intelligence (AI)-empowered drug discovery company, today announced positive topline results from a Phase 2b clinical trial of its proprietary AI-designed oral GLP-1 receptor agonist (GLP-1RA), MDR-001, in adults with obesity or overweight in China.
In this 24-week, randomized, placebo-controlled study, MDR-001 demonstrated clinically meaningful, dose-dependent weight reduction. 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.
In addition to 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.
MDR-001 was well tolerated, 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.
Importantly, hepatic safety analyses showed no evidence of transaminase elevation, even among approximately 20% of participants with pre-existing liver impairment. In fact, ALT and AST levels were significantly reduced in the MDR-001 treatment groups compared to placebo. Additionally, no clinically relevant increases in heart rate were observed. The overall discontinuation rate due to TEAEs was only 0.8%.
Professor Linong Ji, Leading Principal Investigator of the study, 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.”
Dr. Zhangming Niu, Founder and CEO of MindRank, 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.”
MDR-001 represents MindRank’s lead asset and the first AI-designed oral GLP-1RA to report positive Phase 2b data globally. These results lay a solid foundation for late-stage development and future partnerships aimed at addressing the global obesity epidemic.
About Phase 2b Study Design
The phase 2b clinical trial (NCT06606483) was a multi-center, randomized, double-blind, placebo-controlled study conducted across 19 clinical sites in China, enrolling a total of 317 adult participants, who were either overweight with at least one weight-related comorbidity, or obese individuals with inadequate response to lifestyle interventions such as diet and exercise. 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. Participants were 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.
About MDR-001
MDR-001 is a novel, β-arrestin 2 selective, orally bioavailable small-molecule GLP-1 RA discovered using MindRank's proprietary AI platform, Molecule Pro™. MDR-001 has demonstrated excellent functional potency and selectivity, with favorable ADME properties and oral bioavailability, as well as desirable pharmacokinetics, safety and tolerability, indicating its best-in-class potential for the treatment of obesity and T2DM with large unmet medical needs.
About MindRank
MindRank is an artificial intelligence (AI)-empowered drug discovery company. By leveraging its proprietary AI platforms (PharmKG™, Molecule Dance™ and Molecule Pro™), the company aims to significantly accelerate the drug discovery process and deliver small molecule medicines with differentiations and clinical benefits. Leading asset MDR-001, an AI-designed oral GLP-1RA small molecule, has successfully completed a phase 2b clinical study.
For more information, visit www.mindrank.ai.
For BD inquiries, contact bd@mindrank.ai.
