Enrollment remains on pace for a Q3 2026 topline readout in Aardvark Therapeutics’ Phase 3 HERO trial of ARD-101 in Prader-Willi syndrome, with first dosing completed in Australia, regulatory clearance in Canada and the UK, and all patients who have completed the blinded 12-week period to date opting into the open-label extension.

The core development is operational: HERO (NCT06828861) is now enrolling across the U.S. and Australia, with Canada and the UK cleared to start imminently. The company plans to enroll 90 patients with PWS-related hyperphagia into a randomized, double-blind, placebo-controlled study measuring change in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) from baseline to Week 12 as the primary endpoint, with caregiver and clinician global severity scales as secondaries. Notably, Aardvark now expects to meet enrollment targets without activating previously planned EU sites, trimming its site footprint while maintaining an international mix that includes North America and the Commonwealth markets. ARD-101, an oral, gut-restricted bitter taste receptor agonist designed to stimulate satiety signaling via enteroendocrine pathways, carries U.S. Orphan Drug and Rare Pediatric Disease designations.

Strategically, this is a focus-and-execute trial operations plan rather than a market-expansion play. Concentrating enrollment in the U.S., Australia, Canada, and the UK reduces startup friction, language validation complexity for PROs, and regulatory heterogeneity—variables that can drag timelines in rare disease trials built on caregiver-reported endpoints. It also reflects a competitive clock in PWS hyperphagia, where late-stage programs have elevated expectations for measurable improvements on standardized instruments. By leaning into a leaner, English-language-heavy site network and signaling that EU sites may be unnecessary, Aardvark is prioritizing speed, data consistency, and cost control over broader geographic generalizability ahead of a pivotal dataset.

For sites, HERO’s short blinded duration and clear caregiver engagement signal manageable visit burden and potentially strong retention, aided by an open-label extension that can minimize post-Week-12 attrition. The reliance on HQ-CT heightens the importance of rigorous rater training, consistent ePRO workflows, and tight data quality controls across countries to mitigate placebo and expectation effects common in behavioral endpoints. CROs will need to harmonize training and monitoring across regions while controlling variability that could dilute signal in a 90-patient study. For regulators, the study’s design aligns with existing PWS regulatory dialogue, but the 12-week primary window places pressure on early, clinically meaningful change, with caregiver- and clinician-reported outcomes needing clear, prespecified analyses and responder definitions. Vendors and technology partners should expect emphasis on centralized data review and instrument fidelity rather than heavy decentralization, given the specialized-site model typical in PWS.

The near-term watch list is operational: first-patient-in milestones in Canada and the UK, confirmation of whether South Korea joins as planned, and evidence that enrollment momentum obviates EU activation through mid-2026. Methodologically, expect scrutiny on HQ-CT handling across geographies, durability signals emerging from the extension, and any indications of additive benefit with background GLP-1 use, which could shape subsequent lifecycle planning. Key risks remain the well-known variability of caregiver-reported endpoints, short primary duration for a complex behavioral phenotype, and the compressed sample size in a competitive late-stage setting. If HERO maintains enrollment velocity and delivers a clean, consistent HQ-CT signal with supportive global impressions, Aardvark will have a credible pathway to regulatory engagement and could position ARD-101 as a gut-restricted, potentially combinable option in a PWS landscape that is increasingly demanding robust, patient- and caregiver-centered outcomes.

Source link: https://www.globenewswire.com/news-release/2025/12/10/3203500/0/en/Aardvark-Therapeutics-Announces-First-Patient-Dosed-in-Australia-in-HERO-Phase-3-Trial-for-Prader-Willi-Syndrome.html

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Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.