FDA Lifts Hold on ARD-101 as Aardvark Reports Exploratory Trial Data
The FDA lifted the clinical hold on Aardvark Therapeutics’ ARD-101 program for hyperphagia in Prader-Willi syndrome on October 22, 2026. Aardvark reported exploratory results from its terminated Phase 3 HERO trial, which randomized 67 of 90 planned participants, with HQ-CT scores falling during treatment but returning near baseline after discontinuation. The company plans to seek FDA input on a new Phase 3 design and resume clinical activities in 2027, while noting one cardiac serious adverse event at 800 mg twice daily was linked to a drug-drug interaction.
Why it matters
The lifted hold permits Aardvark to pursue further clinical development, but the early termination means the available efficacy data are exploratory and do not establish effectiveness. The company expects to resume PWS clinical activities in 2027, pending FDA discussions and a new trial design.
Key facts
- 1The HERO trial ended early after 67 of 90 planned participants had been randomized, so all efficacy findings were classified as exploratory. investing.com
- 2The FDA lifted the clinical hold on Aardvark’s Investigational New Drug application for ARD-101 on October 22, 2026. investing.com
- 3Participants who switched from placebo to ARD-101 in the open-label extension showed an average reduction of 6 points in HQ-CT scores by Day 28. stocktitan.net
- 4One cardiac serious adverse event occurred at 800 mg twice daily in a participant also taking pitolisant, and the FDA attributed it to a drug-drug interaction. investing.com
- 5Aardvark plans to request an FDA meeting on a new Phase 3 design and intends to resume PWS clinical activities in 2027. rttnews.com
Open questions
- One material reports no cardiac events at tested doses, while other materials report one cardiac serious adverse event linked to a drug-drug interaction.
- The available efficacy data are exploratory because the trial ended early, so the treatment’s effectiveness remains unconfirmed.
Summary written by AlphAI AI Desk from 6 of 6 sources. Not investment advice. Figures are as stated by the linked sources.