Aardvark Therapeutics has officially concluded a clinical trial that had been voluntarily paused for nearly three months due to safety concerns. The biotechnology firm announced its plans to disclose data from this trial to assess the future of its lead candidate for metabolic disorders, ARD-101, which is under clinical scrutiny by the FDA.
The complete clinical suspension, announced following Thursday’s market close, affects all studies involving ARD-101, which is in development for Prader-Willi syndrome. This rare genetic disorder results in hyperphagia, characterized by excessive and insatiable hunger regardless of food intake. The associated morbid obesity can lead to severe respiratory and cardiovascular complications, as well as increased risks of gastrointestinal rupture.
Aardvark had progressed its Prader-Willi medication into Phase 3 trials. In February, the company revealed that a cardiac safety signal had emerged during routine monitoring in a study involving healthy volunteers. Consequently, Aardvark voluntarily halted recruitment and dosing in the Phase 3 trial while investigating the cardiac issue, keeping the data undisclosed.
As of February 27, when Aardvark announced the voluntary trial pause, 68 patients had been dosed in the Phase 3 study and 19 in the open-label extension study. The company is now set to unblind the data from both trials. Through this evaluation of the drug’s efficacy and safety among participants, Aardvark aims to make an informed decision regarding the next steps in its program.
Aardvark is operating under tight timelines. As of the end of the first quarter, the company reported a cash position of $91.2 million, sufficient to sustain operations until mid-2027. However, it will need to gain clarity on ARD-101 well before this timeframe to engage with the FDA effectively. Investors are keen for regulatory certainty before committing additional funds for another critical study. Analyst Andy Hsieh from William Blair noted that unblinding the ARD-101 data could yield positive insights.
“We believe this could pave the way for early effectiveness signals, allowing Aardvark to better position itself for a second registration study,” he stated in a research note released Friday. “We anticipate a more comprehensive update, particularly regarding a path forward aligned with the FDA, later this year.”
ARD-101 is an oral small molecule designed to activate bitter taste receptors, known as TAS2 receptors, which trigger the secretion of intestinal peptide hormones that suppress hunger. The twice-daily pill is specifically formulated to target TAS2 receptors in the gut, avoiding receptors elsewhere in the body that could lead to toxic effects.
Aardvark distinguishes hunger, defined as discomfort from lack of food, from appetite, which is the desire for food as a reward. The company claims that these two sensations are regulated by distinct neuronal signaling pathways and that its appetite-suppressing drugs could complement the appetite suppression observed in other medications, such as GLP-1 receptor agonists.
Last year, Aardvark advanced its strategy targeting metabolic diseases through a $94 million initial public offering. The company is part of a small group conducting research on Prader-Willi syndrome, an indication that has experienced numerous failures in clinical trials.
The only available treatment for Prader-Willi syndrome is Vykat XR, a drug by Soleno Therapeutics that received FDA approval last year. Vykat is formulated as an oral small molecule administered once daily and is believed to work by activating potassium channels involved in regulating physiological processes, including appetite. Vykat’s commercialization efforts are set to continue under Neurocrine Biosciences, which announced a $2.9 billion acquisition deal with Soleno last month.
Photo: Streeter Lecka, Getty Images
