More than thirty years since its 1993 founding, Catherine Owen Adams and Elizabeth Thompson—the R&D combo that has led Acadia since last year—are managing two products on the market and a pipeline estimated to be worth an additional $12 billion in sales.
After 32 years in business, Acadia Pharmaceuticals recently held its first-ever R&D Day, forecasting a potential $12 billion in annual sales stemming from its investigational pipeline.
“We’re a small biotech now, but we have aspirations to be a biotech powerhouse,” CEO Catherine Owen Adams told BioSpace. When Owen Adams joined Acadia last September, her first priority was to “stabilize” the commercial business, which she said has now been done. Her second priority was “redefining [Acadia’s] strategy moving forward. Where are we going? How big could Acadia be?”
With two products on the market—Nuplazid for Parkinson’s disease psychosis and Daybue for Rett syndrome—and seven experimental assets in clinical and preclinical studies, the company believes the entire package could be worth $14 billion in annual revenue. This highly touted pipeline also has a new leader. Elizabeth (Liz) Thompson joined the company as head of research and development in April 2024 after a stint as Amgen’s EVP of R&D, rare disease
“Previously, there’s been a little bit of reticence about showing too much of the pipeline,” Owen Adams said, “and I think Liz and I really challenged that and wanted to show some of the more exciting nearer-term opportunities and also some of our new thinking about how we’re going to partner, how we’re going to focus.”
Owen Adams defines Acadia as a neurological and rare disease company—notably, one largely targeting symptomatic treatments for neurodegenerative diseases. The pipeline includes two assets in mid- to late-stage studies for hyperphagia in Prader-Willi syndrome, Alzheimer’s disease psychosis and lewy body dementia with psychosis. This focus places Acadia squarely at the center of an emerging trend that also includes Bristol Myers Squibb, AbbVie and Neumora Therapeutics: the holistic treatment of Alzheimer’s disease.
“Whilst memory loss is upsetting and difficult, the psychosis and . . . agitation . . . they’re really, really disturbing for families, and it means that caring for that patient becomes incredibly hard at home,” Owen Adams said. “One of the main reasons that patients are admitted to long-term care facilities is not actually their . . . memory loss, it’s their psychosis or agitation, so I think there will continue to be a lot of focus.”
First approved in 2016, Acadia’s Nuplazid (pimavanserin) was the first—and remains the only—FDA-approved treatment for hallucinations and delusions associated with Parkinson’s disease psychosis. ACP-204, currently in a Phase II trial for Alzheimer’s disease psychosis, is a follow-on compound to that drug, Owen Adams said.
At its marketed dose, pimavanserin is not associated with the heart rhythm disorder long QT syndrome, she explained. However, once that dose is raised, a QT signal is identified, “which in the elderly, is not great.” This has prevented Acadia from dosing pimavanserin at the higher levels that would be required to optimally treat these other indications, Owen Adams said. “So we redesigned ACP-204 to have little or no effect on QT prolongation,” which has enabled the company to double the dose of currently marketed pimavanserin to achieve a stronger response in clinical trials.
BMO Capital Markets analysts were bullish on ACP-204, saying in a June 25 note to investors that it could “provide key improvements” on pimavanserin’s profile by mitigating concerns over QT prolongation, enabling higher dosing and allowing for a faster time of onset.
This is one of the compounds Owen Adams is most excited about—partly for personal reasons: each of her parents is afflicted by one of these two diseases. “Just from a personal perspective, I understand the devastation it causes for families,” she said.
But like any proud parent, Owen Adams didn’t want to pick just one high-potential asset. She also highlighted ACP-101 (carbetocin nasal spray), currently in a Phase III study for hyperphagia in Prader-Willi syndrome, a rare, neurobehavioral genetic disorder characterized by severe, life-threatening hyperphagia—an abnormally great desire for food—metabolic issues and intellectual deficits, among other behavioral issues.
Acadia picked up ACP-101 in its $10 million acquisition of Levo Therapeutics in 2022. In a Phase III trial conducted prior to the transaction, 3.2 mg of the drug demonstrated “nominally statistically significant efficacy,” and was safe and well-tolerated, according to a June 2023 press release from Acadia. Still, the FDA rejected Levo’s application for the drug in January 2022, recommending another trial to confirm benefit.
ACP-101’s history is part of the draw for Owen Adams. “Just because it came from a failed Phase III study, a $10 million outlay [and] really a lot of skepticism around the science and the focus,” she said.
Acadia accelerated recruitment for the Phase III trial and pulled forward the timeline for topline results from 2026 to the fourth quarter of this year. “I’m really excited for that one because I think it could be a real game changer,” Owen Adams said.
BMO had a different selection, however. In their June 25 note, the analysts said they “are most optimistic about the potential of ACP-211 (being developed for treatment-resistant depression and major depressive disorder) given validated success of [J&J’s] Spravato [esketamine] in MDD.” ACP-211, an orally administered, deuterated form of R-Norketamine, is currently in Phase I, with a Phase II study expected to begin in the fourth quarter.
Back on Track
While Owen Adams expressed optimism for Acadia’s future, she acknowledged the path hasn’t always been a smooth one.
In March 2023, Acadia’s Daybue won FDA approval as the first therapy for Rett Syndrome, a rare, neurodevelopmental disorder primarily affecting girls. Then, after about nine months, the company began to hear reports of gastrointestinal side effects on the community. “We got off to a great start, strong support from our rep community,” Owen Adams said. Then “we had a pretty difficult 2024 with flat to declining patients.”
At the same time, Acadia learned more about how to leverage a titration approach to better manage those GI side effects and has since undertaken an educational campaign with patient families. “And now we’re back to growing patients again,” Owen Adams said.
Acadia’s focus on diseases that affect small numbers of people isn’t the biotech’s only rarity. The company is also led by women in key positions. “I think Liz and I are actually the only CEO/head of R&D all female combo,” Owen Adams said. She and Thompson are joined by Chief Legal Officer Jennifer Rhodes and Chief Medical Officer Ponni Subbiah.
“I think what it does give us is that diversity of thought and experience and energy,” Owen Adams said. She also pointed to the fact that she and Thompson are new to the Acadia team. “I think that energy is important in biotech, because you come in and you really want to make a difference, and you really want to ensure that we’re on the right track, and I think that is definitely evident right now.”
