Aardvark Pauses Phase 3 HERO Trial for Prader-Willi Syndrome Over Cardiac Safety Concerns

Event summary

  • Aardvark Therapeutics voluntarily paused its Phase 3 HERO trial and open-label extension for Prader-Willi Syndrome due to reversible cardiac observations at above-target doses in a healthy volunteer study.
  • The pause follows routine safety monitoring, with the company conducting a comprehensive data review to determine next steps.
  • Topline data from the HERO trial, previously expected in Q3 2026, is now delayed, with updated guidance anticipated in Q2 2026.
  • ARD-101, the trial's investigational drug, is a gut-restricted small-molecule agonist targeting hyperphagia in Prader-Willi Syndrome.

The big picture

Aardvark's pause highlights the critical balance between accelerating rare disease treatments and ensuring patient safety. The delay in the HERO trial underscores the challenges in developing novel therapies for Prader-Willi Syndrome, where unmet medical needs are high but regulatory standards remain stringent. The company's ability to navigate this setback will be key to its long-term strategy in the metabolic disease space.

What we're watching

Regulatory Scrutiny
How the FDA's collaboration with Aardvark will shape the trial's future, given the cardiac safety signals observed.
Execution Risk
Whether Aardvark can mitigate the delay and maintain investor confidence while reevaluating ARD-101's therapeutic dosing.
Trial Dynamics
The pace at which Aardvark resumes the HERO trial and the potential impact on its timeline for announcing topline data.