Updated: Acadia wins first FDA approval for Rett syndrome drug

The FDA on Friday approved Acadia Pharmaceuticals’ treatment for Rett syndrome, a rare neurodevelopmental disorder, which will be sold under the brand name Daybue (trofinetide), the company announced.

The average list price for the drug will be about $575,000 to $595,000 a year, CEO Steve Davis said on a conference call Monday morning. That comes from a $21.10 per milliliter price that’s used for weight-based dosing. The company’s “net realized cost” will be approximately $375,000, he said.

Wall Street analysts have been awaiting word on how the company would price the drug, and  Mizuho analyst Uy Ear said over the weekend that the price of the drug will be a major factor in its commercial success.

“At current level, we believe the stock’s value incorporates a list price of ~$400K per year at launch,” Ear said in a note to clients.

Shares of $ACAD fell 7.2% Monday morning after the call.

There are about 4,500 diagnosed Rett syndrome patients in the US, according to Acadia. Caused by a genetic mutation, the patients — typically girls — develop normally in infancy and then begin to deteriorate, losing their speech, motor control of their hands and other functions.

Acadia added that it believes there are between six and nine thousand patients in the US with the disease.

The company said Daybue will be available for those two years old and older by the end of April. It’s the first approved US treatment for the disease, and Acadia will get a priority review voucher from the FDA, which could bring the company about $100 million if it decides to sell the voucher.

Mark Schneyer

CFO Mark Schneyer said on Monday’s call that it has to pay milestones to Neuren Pharmaceuticals, the company from which Acadia in-licensed Daybue in 2018 to get US rights. Neuren retains all rights to Daybue outside of the US.

Acadia owes Neuren $40 million within 60 days of Daybue’s first commercial sale. If the company uses or sells its priority review voucher, Neuren gets one-third of the voucher’s value, Schneyer said — on top of royalties and sales milestones.

Acadia’s chief commercial officer Brendan Teehan told Endpoints News the sales force will consist of 50 people who will be spread out among centers of excellence, “high-volume institutions,” and then out in the community.

Brendan Teehan

“We just got the label on Friday. So we will train the field force, but they will be out in full effect in mid-April,” Teehan said, noting that the drug will be available before the end of April.

As for Acadia’s plans to reach the undiagnosed population, Teehan said that introducing a therapeutic option could drive people to get a diagnosis.

“If you didn’t think you had therapeutic options, then knowing more about a disease isn’t terribly helpful. But if you do know that there are therapeutic options, people tend to go and get a confirmatory diagnosis, which we think is likely to occur,” the exec added.

Kathie Bishop

Davis, alongside Acadia’s head of rare disease Kathie Bishop, referenced a collaboration with Stoke Therapeutics on the conference call, which was cemented last year in a $900 million plus deal for SYNGAP1 syndrome, followed by Rett syndrome (MECP2) and another undisclosed program.

“We believe this will be the beginning of additional therapies coming in due course. And we want to be at the forefront of that,” Davis said.

“Those programs are still preclinical, so we’re not talking too much about them yet,” Bishop added — noting that it represents Acadia’s emphasis on rare diseases and Rett in particular.

The drug was approved based on a Phase III trial of 187 female patients showing that they improved on two scoring systems used to assess people with the disease. Common side effects were diarrhea and vomiting.

The approval is a welcome dose of good news for Acadia, which has been going back and forth with the FDA over its drug Nuplazid, its main source of revenue, for use in Alzheimer’s-related psychosis. The agency rejected it for that use last year, recommending another trial.

Editor’s note: This story has been updated with context from Acadia’s conference call Monday morning, alongside an interview with Acadia’s chief commercial officer Brendan Teehan.

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