With just over 100 days as CEO now under her belt, Catherine Owen Adams is looking to cruise rare disease-focused Acadia Pharmaceuticals into its first $1 billion sales year.
Achieving the milestone will require the company’s two marketed products, Parkinson’s disease-related psychosis med Nuplazid and newer Rett syndrome treatment Daybue, to continue on their current growth trajectories. As it stands, both products are in line to grow sales within their approved indications, Acadia emphasized during its presentation at this year’s JP Morgan Healthcare Conference.
After Acadia generated $726 million in 2023, the company forecast its 2024 sales would reach between $940 million and $960. The company has yet to report full-year 2024 financial results.
Still, a $1 billion year would mark an important milestone for the 31-year-old biopharma firm.
Acadia's Nuplazid (pimavanserin) was approved in 2016 and took center stage in a safety and marketing controversy two years later. While nothing came of a DOJ investigation that kicked off in 2019, the FDA added another blow when it repeatedly snubbed the company’s efforts to move the drug into other patient populations, including those with dementia-related psychosis and those with Alzheimer’s disease-related psychosis.
Finally in 2024 after failing to meet the mark in a phase 3 study testing Nuplazid as a treatment for negative symptoms of schizophrenia, the company called it quits on label expansion efforts for the drug.
Despite the hard stop on further studies, there’s still room to grow in the drug’s currently approved patient population, Acadia’s new CEO Owen Adams told Fierce Pharma on the sidelines of the conference. Around 50% of the 1 million Parkinson’s disease patients in the U.S. are at risk of developing hallucinations or delusions over the course of their disease, but company research shows that fewer than 10% of caregivers and patients are aware of the symptoms.
Owen Adams joined Acadia at the end of September, leaving her role as Bristol Myers Squibb’s general manager of U.S. operations for the job. Before her five-year stint at BMS, she served in various positions at Johnson & Johnson for 25 years.
Outside of her attraction to Acadia’s commercial portfolio and pipeline, the new CEO felt a personal pull to the company and its focus in neuropsychology. She herself is a caregiver to her parents, both of whom have Alzheimer’s disease.
Daybue launch efforts
A significant focus of Owen Adams' first full year as CEO will be on Rett syndrome treatment Daybue. The drug won its FDA approval in 2023 and continues to grow at a rapid clip, generating $91 million in the third quarter of 2024.
An approval in Europe could drive further growth, as there are about 9,000 to 12,000 patients with the disease in the region, according to the company.
Having a product that’s “clearly available for our patients” globally is a key part of the company’s future, Owen Adams said. Acadia is already preparing for Daybue’s European rollout with an EU launch team, and it's anticipating initial sales from its ongoing launch in Canada this year.
In the U.S., Acadia's goal is to continue increasing the company’s field sales force to better support patients and physicians through the early months of treatment.
After a strong launch right out the gate, the team at Acadia noticed that a “significant amount” of patients were stopping treatment in the first two to three months due to gastrointestinal-related adverse effects, Owen Adams said.
However undesirable, sticking with the twice-daily treatment through the early side effects is crucial, she stressed. Daybue is the first treatment for Rett syndrome, a genetic neurodevelopmental disorder that hinders verbal and nonverbal communication, leaving patients “trapped in their own body,” Acadia’s former head of rare diseases Kathie Bishop, Ph.D., previously told Fierce Pharma.
With Daybue, however, patients' families are seeing their loved ones “socially interact differently," Owen Adams said.
"They're seeing eye contact differently, they're seeing motor function improved, they're seeing all sorts of improvements," she explained.
Now, amid Acadia's increased launch efforts, 66% of active patients have been on treatment for more than 10 months. About 1,600 patients have been treated to date, according to Acadia figures.
Elsewhere, Acadia’s pipeline features candidates for other rare diseases with no approved treatments such as hyperphagia (extreme hunger) in rare neurobehavioral disease Prader-Willi syndrome and Lewy body dementia with psychosis. The ability to be first-in-class, or best-in-class, is a priority when exploring new assets, according to Owen Adams.
As it stands, the company is sitting on more than $700 million ready to invest in potential business development deals. In November, Acadia flexed its dealmaking muscles with a deal worth up to $582 million for Danish biotech Saniona’s essential tremor candidate.
In the licensing agreement, the company committed to provide funds for Saniona’s ongoing phase 1 study and take responsibility for future clinical development, regulatory submissions and commercialization.