We recently hosted a webinar featuring Dr. Tien Lee and Dr. Manasi Jaiman of Aardvark Therapeutics. They dove into the science and promise behind ARD‑101—an innovative oral medication aimed at curbing hyperphagia in individuals with Prader‑Willi syndrome (PWS).
A 30-minute recording of the session is now available. If you’re short on time, we’ve summarized key takeaways below—plus answered common questions about the HERO Phase 3 clinical trial, which is currently enrolling.
Differentiating Hunger vs. Appetite
Dr. Lee highlighted a crucial distinction:
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Appetite is driven by brain reward circuits (e.g., GLP-1 pathways).
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Hunger is driven by physical need, regulated by hormones such as cholecystokinin (CCK).
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How ARD‑101 Works
ARD‑101 is a gut-targeted therapy that binds bitter-taste receptors in the intestine. This action stimulates localized CCK release to restore gut-brain signals that signal fullness—without triggering reward-related eating.
Unlike GLP‑1 agonists, which affect appetite and metabolism system-wide, ARD‑101 uses a local, targeted mechanism that directly addresses hunger—a distinguishing and hopeful strategy.
Promising Phase 2 Results
In a Phase 2 open-label study:
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Out of 12 participants, 11 showed reduced hyperphagia, and 4 achieved near-complete symptom relief.
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ARD‑101 also showed anti-inflammatory benefits.
Moving into Phase 3: HERO Trial
Aardvark has launched the HERO Phase 3 trial to further evaluate ARD‑101—a vital step toward approval and real-world use. The study is now enrolling people with PWS ages 13 and older in the US, Europe, Australia, and South Korea. The enrollment goal is 90 participants.
Frequently Asked Questions
What are the requirements to participate in the HERO Trial?
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The participant must have a confirmed diagnosis of PWS, be at least 13 years old, have the same caregiver present at all site visits, and must complete all questionnaires and attend each visit.
Where can I go to learn more and/or sign up for the HERO Trial?
- You can learn more about the HERO Trial by visiting the trial website: HEROforPWS.com.
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To ensure someone is able to participate, go to the HERO website and connect with Leapcure. Take a look at the sites and see if there is a site close by. If not, discuss interest with the participant's endocrinologist.
What is Leapcure?
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Leapcure is a concierge service that helps with prescreening questions and connecting families to trial sites quickly. Participants can reach out to trial sites and their physicians directly or go through Leapcure. See the HERO website for more information.
Can families go to any site that is open, or do they need to stay within a certain region close to where they live?
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Participants can go to any site, but Aardvark encourages participants to go to the site closest to where they live to make traveling easier on families. The site is open to anybody.
Can you change your site location after the initial visit?
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That would be on an individual-by-individual basis based on what the timing would look like. We would work out the dynamics with the participant.
Are meals and other travel expenses covered?
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Yes. Aardvark wants this to be as easy on participants as possible.
If a person gets placebo during the double blind portion, will they have an opportunity to have the active drug afterwards?
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Yes. The trial is three months. After full participation, everyone is guaranteed to receive the drug.
Is there an upper limit to the age requirement?
- No. Anyone over the age of 13 is eligible.
What if my child is 12? Will they be able to participate when they turn 13?
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Aardvark is very encouraged by the community's vigor to participate, so we hope to complete enrollment for this study quickly. If you want to participate, please reach out to Leapcure when your child is 13.
How long will the open-label extension be available to participants of the double blind portion of the study?
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The goal is to have the open-label extension available until FDA approval is obtained. Theoretically, a participant could have access to this drug for years.
Can my child participate in this trial while taking VYKAT XR or any other drug for maintenance?
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Aardvark needs to have pure data on how ARD‑101 works, so your loved one cannot be on VYKAT XR while participating in the HERO study.
What countries with this study be available in?
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Study sites are available in the US, UK, Canada, Australia, and South Korea. Aardvark is still in the process of opening sites, so not every site is currently available. ClinicalTrials.gov and HEROforPWS.com both have full lists of current sites.
Do people need to be on a special diet while participating in this trial?
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No, a special diet is not required and is not recommended! Participants should stay on their current diet and limit changes while participating in the trial.
What were some of the side effects observed during the phase 2 study?
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One participant had a history of reflux, so they experienced a bitter taste in the mouth—but it resolved in a day or two, and they continued and completed the study. Another participant had diarrhea, which also resolved on its own.
Learn More or Enroll in the HERO Trial
Want to take the next step? Use the resources below to find out if your loved one qualifies, connect with trial sites, or stay updated:
FPWR and other advocacy groups will continue to share updates as they become available.
