ACADIA Pharmaceuticals (NASDAQ:ACAD) highlighted its growing commercial portfolio, upcoming clinical catalysts, and cash position during a presentation at the Citizens Life Sciences Conference, with remarks from CFO Mark Schneyer and Head of Clinical Development Srdjan Stankovic.
Commercial portfolio surpasses $1 billion in net sales
Schneyer said the company’s two commercial-stage products, NUPLAZID (for Parkinson’s disease psychosis) and DAYBUE (for Rett syndrome), have combined for more than $1 billion in net sales for the first time in company history. He added that ACADIA’s guidance at the midpoint is about $1.25 billion for the year.
Remlifanserin Alzheimer’s disease psychosis data expected in late summer/early fall
On the pipeline, Schneyer and Stankovic pointed to remlifanserin as the company’s most advanced investigational asset. It is being studied in Alzheimer’s disease psychosis and Lewy body dementia psychosis. Schneyer said the phase 2 Alzheimer’s disease psychosis trial is expected to read out in the August to October timeframe.
Stankovic described remlifanserin as a “new 5-HT2A inverse agonist” and said the mechanism has been validated through ACADIA’s experience with pimavanserin (NUPLAZID). He said prior studies in Alzheimer’s disease psychosis and in dementia-related psychosis provided proof-of-concept signals that inform the company’s confidence in the approach.
Stankovic outlined several lessons learned from prior work with pimavanserin that ACADIA is applying in the remlifanserin program:
- Targeting patients with more severe psychosis, where the company has observed stronger effect sizes.
- Using a more sensitive outcome measure, stating that SAPS-H+D was more sensitive than NPI-NH in prior Alzheimer’s disease work.
- Recognizing that patients achieving higher blood levels showed better efficacy, supporting the goal of a program where dosing can be “pushed.”
- Running a dedicated Alzheimer’s disease program with appropriate replication and exposure.
Addressing placebo response, Stankovic said steep placebo improvement can attenuate drug-placebo separation, and that the company is taking steps intended to reduce noise, including enrolling a severe and durable psychosis population, confirming diagnosis with blood-based biomarkers, and implementing stringent rater training and monitoring with third-party oversight to manage rater drift.
When asked how ACADIA will define success beyond statistical significance, Stankovic said success would be viewed within the broader target product profile, including once-daily dosing (with or without food) and a safety and tolerability profile “similar to pimavanserin,” which he said does not adversely affect motor symptoms or cognition. He also emphasized the unmet need in Alzheimer’s disease psychosis, noting that “nothing is approved” and that off-label treatments often bring significant adverse effects and, in his view, do not work reliably.
NUPLAZID growth tied to renewed DTC investment and expanded reach
Turning to NUPLAZID, Schneyer attributed recent growth to the company’s renewed direct-to-consumer spending over the last year and a half, including an unbranded disease awareness campaign with Ryan Reynolds and a branded NUPLAZID campaign. He said the efforts increased awareness among caregivers and led to more patient and caregiver discussions with physicians, supporting prescription growth.
Schneyer said that despite NUPLAZID being on the market for close to 10 years, the company achieved 9% volume growth last year and expects to continue investing with expectations for growth this year as well. He added that an expansion of commercial-facing personnel was hired, trained, and deployed in the first quarter, but said it was too early to quantify impact, noting it can take six to nine months to see meaningful effects.
On the patient mix, Schneyer said roughly three-quarters of NUPLAZID patients are in the community living at home, while about 25% are in long-term care facilities. He said growth has been seen in both segments, though DTC activity tends to be more effective in the community setting.
Discussing the company’s outlook, Schneyer said guidance for both brands is primarily volume-based, with a modest price benefit he characterized as around 2%.
DAYBUE: growth investments, new STIX formulation, and Europe re-examination
On DAYBUE, Schneyer said ACADIA is approaching the product’s three-year anniversary on the market. He described an early strong launch followed by a plateau typical of rare disease launches, as early adopters are treated quickly and later growth requires additional education. He said ACADIA increased the DAYBUE field force by about 30% in the second quarter of last year to improve reach and frequency with community physicians treating Rett syndrome and said the company has seen success from that effort.
Schneyer highlighted approval of a new formulation, DAYBUE STIX, announced in late December. He said the STIX format is designed to address formulation-related barriers for some patients, including large liquid volume and taste concerns. He said STIX can be mixed into a non-dairy beverage of choice, can be taken in a smaller volume than the prior liquid formulation, has a natural strawberry flavor, does not require refrigeration, and avoids certain excipients present in the liquid (including Red Dye 40). Schneyer said ACADIA believes STIX may provide access to an incremental 400 patients who either never tried DAYBUE liquid or discontinued due to formulation issues.
Schneyer estimated there are about 6,000 diagnosed and treated Rett patients in the U.S. based on medical records and claims databases, while prevalent population estimates range from 6,000 to 9,000. He said in the fourth quarter the company treated roughly 1,000 patients on DAYBUE and that about 2,000 patients have tried the therapy to date. On persistency, Schneyer said about 55% of patients remain on therapy by month 12 and over 45% remain at 18 months, with persistence tending to plateau thereafter. He added that more than 70% of current patients have been on therapy for 12 months or longer.
Outside the U.S., the presenters discussed the European regulatory path for DAYBUE (trofinetide). They said the company received negative feedback from the EMA’s CHMP and has requested re-examination. Stankovic said CHMP’s position was that the primary endpoints used were not adequate; he said ACADIA disagrees, pointing to two co-primary endpoints in the trial: the Rett syndrome behavioral questionnaire (45 items) and the clinician’s Clinical Global Impressions assessment. He noted re-examination allows new analyses and patient and caregiver perspectives but not new data, and he cited an estimated 20% to 30% success rate for such re-examinations.
Early-stage pipeline includes ACP-211, essential tremor program, and next Rett candidate
Beyond remlifanserin, Stankovic discussed ACP-211, describing it as a deuterated major metabolite of ketamine. He said ACADIA hopes to demonstrate antidepressant efficacy comparable to Spravato or IV ketamine with improved patient experience and lower supervision needs, and he stated that phase 1 data so far suggest ACP-211 lacks anesthetic properties and has contained psychotomimetic and dissociative effects. He said the program is still in early stages and the company will disclose timing when it has a better sense of recruitment and readout.
Stankovic also described an essential tremor program targeting GABA alpha-3, which he said appears distinct from other GABA agents studied for the condition. He said ACADIA anticipates beginning a phase 2 program toward the end of the year or early next year.
Finally, Stankovic briefly mentioned ACP-2591, describing it as a next-generation program that reaches higher brain levels than trofinetide. He said ACADIA is preparing to test it in Rett syndrome first and will share progress as it approaches further development milestones.
In closing, Schneyer said the company’s cash flow profile supports funding its commercial initiatives and pipeline, and he added that ACADIA expects to continue evaluating business development opportunities to broaden its portfolio.
About ACADIA Pharmaceuticals (NASDAQ:ACAD)
ACADIA Pharmaceuticals Inc is a biopharmaceutical company focused on the development and commercialization of innovative therapies for central nervous system (CNS) disorders. Established in 1993 and headquartered in San Diego, California, ACADIA’s research centers concentrate on conditions with significant unmet medical needs, including Parkinson’s disease psychosis, Alzheimer’s disease psychosis, and schizophrenia. The company utilizes a range of scientific platforms, including selective receptor modulation and precision-targeted compounds, to advance its portfolio of small-molecule therapeutics.
The company’s flagship product, NUPLAZID® (pimavanserin), received U.S.
