Dive Brief:
- San Diego-based biotechnology company Acadia Pharmaceuticals on Tuesday announced plans to start a Phase 3 trial of a drug for the rare genetic disease Prader-Willi Syndrome that the Food and Drug Administration rejected last year.
- The experimental treatment was previously developed by Ferring Pharmaceuticals, Inserm and most recently privately held startup Levo Therapeutics, which brought the drug to a regulatory review. But the FDA rejected Levo’s application in January 2022 and asked the company to run another trial.
- Five months later, Acadia acquired Levo for $10 million in cash upfront. It has since met with the FDA on the design of a new trial meant to address the agency’s concerns. That study will begin in the fourth quarter.
Dive Insight:
The decision adds what Stifel analyst Paul Matteis called in a Tuesday evening client note a “low-cost, albeit high-risk” option to Acadia’s pipeline.
The drug is an intranasal form of carbetocin, which is used to treat excessive bleeding after childbirth. Levo had been developing it for Prader-Willi as its effects appear to be stronger than the hormone oxytocin, levels of which are low in the brains of people with the disease. Oxytocin deficiency is believed to be associated with the constant sense of hunger, or hyperhagia, that characterizes Prader-Willi, a condition with no approved treatments.
Levo had hoped its drug, called ACP-101, would be the first. While a Phase 3 study didn’t meet its primary goal, It filed for approval based on data showing that a low dose appeared to provide some benefit. An FDA advisory panel disagreed, voting 12-1 against an approval in late 2021. The FDA to issued a complete response letter shortly thereafter and recommended the company run another trial.
Acadia will now take on that task. The company is planning a new trial at that lower dose. It’s also making design tweaks, such as changing the length and expanding the size of the study, which will have only one main goal rather than two.
The effort isn’t the first time Acadia has tried to bring a rejected drug back to the FDA. The biotech tried and failed to get its drug pimavanserin cleared as a treatment for hallucinations and delusions associated with Alzheimer’s disease.
On the other side of the ledger, earlier this year Acadia won FDA clearance of a drug for Rett syndrome that it had acquired in a prior licensing deal.
However, analysts are skeptical Acadia can repeat that kind of business development success.
“We are not so sure the company can catch lightning in a bottle twice,” wrote Raymond James analyst Danielle Brill, referring to Acadia’s success in Rett syndrome. “While PWS is an indication with a high unmet need, the FDA already [rejected] this asset last year, so it’s possible that the agency will be less flexible this time around."
Stifel analyst Matteis estimated the company has a less than 50% chance of success given the drug’s history, but the cost of the acquisition as well as the future trial “are modest enough” that it’s a “reasonable investment,” he said.