Aardvark Therapeutics is planning to unblind data from its phase 3 Prader-Willi syndrome (PWS) program after the FDA put a full clinical hold on its studies.
San Diego-based Aardvark voluntarily paused a phase 3 trial of ARD-101 as a treatment for hyperphagia, the medical term for a feeling of extreme, insatiable hunger, in PWS in March. A cardiac safety signal in a healthy volunteer study triggered the pause. Later that month, the biotech expanded the pause to cover trials of ARD-201, a phase 2 candidate that combines ARD-101 with a DPP-4 inhibitor.
Up to this point, Aardvark’s actions have been voluntary, with the biotech saying it initially paused work on the gut-targeting taste receptor agonist ARD-101 “out of an abundance of caution.” However, the FDA has now imposed a full clinical hold on all studies of ARD-101, including the phase 3 PWS trial and an associated open-label extension.
Before pausing the studies in March, Aardvark expected to deliver topline data in the third quarter. The timing was important. With Aardvark’s cash runway forecast to fund operations into the second quarter of 2027, the biotech had a narrow window in which to post the data and use the findings to raise money or land a partner to secure its future.
Aardvark’s cash runway now extends to mid-2027. But with the program already paused for 11 weeks and now under full clinical hold, the situation is squeezing the time the biotech has to complete the trial, share the data and secure its future.
Facing those pressures, Aardvark plans to unblind data from the phase 3 trial and open-label extension. The biotech had dosed 68 patients in the randomized phase 3 trial and 19 people in the extension when it paused the studies.
Unblinding data on the patients will allow Aardvark “to assess the totality of available efficacy and safety data and to support an informed determination of next steps for the ARD-101 program,” the company said in a statement. Aardvark’s share price fell 16% to $5.65 in after-hours trading. The stock closed at $12.49 before news of the voluntary pause broke in February.
Aardvark previously presented evidence that the phase 3 dose may be unlikely to drive the safety events that caused the pause. The biotech saw cardiac QRS prolongation of greater than 25%—the significance threshold—in two of eight healthy volunteers who received twice the phase 3 dose without titration. A later study of the phase 3 dose saw one case of cardiac QRS prolongation of less than 25% in 23 people.