Acadia to stop development of rare disease therapy after trial failure

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By Sneha S K

(Reuters) -Acadia Pharmaceuticals said on Wednesday it will discontinue development of its intranasal therapy to treat a rare genetic condition after it failed to show a benefit in patients in a late-stage trial.

The company’s shares were down 11.7% at $20.83 in premarket trading.

Acadia was testing an intranasal formulation of carbetocin, or ACP-101, as a potential treatment for hyperphagia, or feelings of intense and persistent hunger, in patients with a genetic disorder called Prader-Willi syndrome.

ACP-101 did not show a statistically significant improvement over placebo on the study’s main goal that measured changes in hyperphagia in patients. The therapy also did not meet any of the secondary goals, the company said.

“Given these results, we do not intend to investigate intranasal carbetocin any further,” Elizabeth Thompson, Acadia’s head of research and development said.

Analysts said that investors were skeptical ahead of the results as previous late-stage trial data showed a higher dose of carbetocin was also not statistically significant.

“While expectations were likely somewhat lower for success given the lack of a dose response, success in PWS would have meant meaningful upside for Acadia and could have set the company up for commercialization of their third product,” BMO Capital Markets analyst Evan Seigerman said.

The 12-week study was evaluating the efficacy and safety of a 3.2 milligram dose of ACP-101, when taken thrice daily in 175 children and adults with Prader-Willi syndrome aged between five to 30. The safety and tolerability of intranasal carbetocin was consistent with previous clinical trials, Acadia said.

The debilitating condition, which affects about 50,000 people in the U.S., impacts everyday aspects of life, such as eating, behavior and mood.

Shares of rival Soleno Therapeutics rose 14% premarket, as it currently has the only approved treatment for hyperphagia, a once-daily oral pill called Vykat XR.

(Reporting by Sneha S K in Bengaluru; Editing by Sahal Muhammed and Shailesh Kuber)