FPWR Invests in Aardvark, Gut-targeting PWS Therapy ARD-101

The Foundation for Prader-Willi Research (FPWR) announced its support for ARD-101, an investigational, gut-targeting Prader-Willi syndrome (PWS) therapy, by investing in Aardvark Therapeutics, the treatment’s developer.

“FPWR is committed to advancing the development of innovative treatments through multiple channels,” John Walter, CEO of FPWR, said in a press release.

“Aardvark’s drug targets multiple pathways in the gut potentially relevant to PWS and we look forward to confirming the benefits this potential therapeutic may have for people with PWS,” Walter added.

ARD-101 is an oral small molecule that stimulates bitter taste receptors, according to a video on the company’s website.

“Besides conveyance of flavor, bitter taste receptors exist beyond the mouth, to recognize potential toxic compounds,” Aardvark explains in the video.

Inside the gut, ARD-101 is intended to prompt the release of peptide hormones, which may have bodywide therapeutic benefits. In preclinical studies, the potential therapy reduced appetite and promoted weight loss, and showed anti-inflammatory activity in multiple models, according to Aardvark.

ARD-101 is now being evaluated for safety and tolerability in a Phase 1 clinical study involving healthy volunteers. The company is currently analyzing the data from that trial.

“The Phase 1 clinical trial of Aardvark’s lead drug ARD-101 successfully completed enrollment,” Dvorit Samid, PhD, Aardvark’s head of medical affairs, said in a phone call with Prader-Willi News.

“While the data is still being analyzed,” she continued, “we are encouraged by the excellent safety profile of all dose levels tested as well as preliminary signals of effect identified in the placebo-controlled Phase 1 trial. Our postulated drug mechanism of action indicates a potential for regulating appetite, metabolism, and inflammation.”

Aardvark now plans to evaluate the effect that ARD-101 has on hyperphagia, the uncontrollable hunger characteristic of PWS, in children with the disorder. That study also will examine the therapy’s effect on weight loss and other symptoms. Additionally, the company will conduct a separate trial to assess ARD-101’s efficacy in people with obesity, according to Samid.

“We look forward to evaluating ARD-101’s therapeutic potential in several Phase 2 clinical trials, including a Prader-Willi syndrome trial later this year,” Samid said.

Tien Lee, MD, Aardvark’s CEO, thanked the foundation, in the press release, for partnering with the company. Terms of the investment were not released.

“Our team at Aardvark has tremendous respect for FPWR’s commitment and innovative programs in support of children and adults with PWS,” Lee said. “Sharing in this mission, we are most grateful for our ongoing collaboration and look forward to initiating later this year a phase 2 clinical trial evaluating Aardvark’s novel lead drug, ARD-101, in people with PWS.”

The medication’s anticipated safety profile and new mechanism of action open the possibility of using it in combination with other therapies in diverse metabolic and inflammatory diseases, according to Aardvark. The investment from FPWR will aid in those efforts.

“Working closely together,” Lee said, “we hope to develop a safe and effective new treatment that will improve the health and well-being of those with PWS.”

Past investments by FPWR include a grant given to Essentials, now Soleno Therapeutics, in 2014. That grant supported the initial clinical trial of diazoxide choline controlled release (DCCR) as a PWS therapy. That medication has been showing promise in Phase 3 testing, although the next steps in its clinical development in the U.S. are currently a topic of debate.