Aardvark Therapeutics, Inc. issued a press release on October 23, 2026, detailing exploratory efficacy data from its previously terminated Phase 3 Hunger Elimination or Reduction Objective (HERO) trial and the accompanying open-label extension (OLE) trial. The company randomized 67 of the planned 90 participants in the HERO trial before terminating it early, meaning all efficacy data are classified as exploratory.
The data focused on the company's lead candidate, ARD-101, an oral small-molecule therapeutic designed to treat hyperphagia associated with Prader-Willi Syndrome. In the HERO trial, a modest reduction in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score was observed at Week 2 of dose escalation compared to placebo. Upon discontinuation of the treatment, the average HQ-CT score in the ARD-101 group returned to near baseline, while the placebo group showed less rebound.
Results from the OLE cohort indicated that participants who switched from placebo to ARD-101 experienced an average reduction of 6 points in HQ-CT score by Day 28. Similar trends were observed in caregiver and clinician outcome measures, specifically Caregiver Global Impression and Clinician Global Impression Severity scores.
Regarding safety, ARD-101 was generally well tolerated, with most adverse events described as mild to moderate. However, a cardiac serious adverse event was reported in the HERO trial at the 800 mg twice daily dose in a participant also taking pitolisant. The U.S. Food and Drug Administration (FDA) determined this event was related to a drug-drug interaction. Aardvark plans to resume dosing at a lower dose pending FDA discussion.
The FDA lifted the clinical hold on the Investigational New Drug application for ARD-101 on October 22, 2026. Aardvark intends to request a meeting with the FDA to align on a new Phase 3 trial design and expects to resume clinical activities in 2027.
