Acadia Pharmaceuticals Announces Phase 3 Development Candidate ACP-101 (intranasal carbetocin) for Prader-Willi Syndrome

— Acadia acquired Levo Therapeutics and worldwide rights to ACP-101 in June 2022

— ACP-101 (intranasal carbetocin) is a selective oxytocin-receptor agonist for the treatment of hyperphagia in Prader-Willi syndrome

— Company recently completed a meeting with the FDA and plans to initiate a Phase 3 study in the fourth quarter of 2023

— Webcast to be held today at 5:00 p.m. Eastern Time

SAN DIEGO–( BUSINESS WIRE )– Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced the addition of a new Phase 3 development candidate to its rare disease portfolio, ACP-101 (intranasal carbetocin), for the treatment of hyperphagia (a false and unrelenting state of starvation) in Prader-Willi syndrome (PWS). Acadia acquired worldwide rights to develop and commercialize ACP-101 with the acquisition of Levo Therapeutics in June 2022.

ACADIA logo

“Acadia’s acquisition of ACP-101 demonstrates our commitment to acquiring and developing novel drug candidates that address significant unmet needs in central nervous system disorders. The addition of this drug candidate to our rare disease portfolio is an important next step in the execution of our business development strategy,” said Steve Davis, Acadia’s President and Chief Executive Officer. “Prader-Willi syndrome is a rare genetic disorder with no approved treatments, characterized by life-threatening hyperphagia, in addition to a broad range of severe metabolic issues and behavioral challenges. We look forward to working with the Prader-Willi community and clinical experts as we continue to advance development of this program.”

Prior to Acadia’s acquisition, Levo conducted a Phase 3 multi-center, randomized, double-blind, 8-week placebo-controlled study evaluating two doses of ACP-101, 3.2 mg and 9.6 mg, versus placebo three times daily with an even randomization (1:1:1). Top-line results showed that ACP-101 was safe and well-tolerated and demonstrated nominally statistically significant efficacy at the 3.2 mg dose.

“We recently met with the FDA and reached alignment to further evaluate the 3.2 milligram dose of ACP-101 in a pivotal Phase 3 study,” said Doug Williamson, Acadia’s Executive Vice President, Head of Research and Development. “If positive, we plan to promptly submit a new drug application for the treatment of hyperphagia in PWS to the FDA.”

Prader-Willi syndrome is a rare, neurobehavioral genetic disorder characterized by severe and life-threatening hyperphagia, metabolic issues, intellectual deficits and other behavioral problems that is estimated to affect 8,000 to 10,000 patients in the United States. 1,2 There is no FDA approved medicine to treat hyperphagia in PWS. 5

Conference Call and Webcast Information

Acadia will discuss the ACP-101 program via conference call and webcast today at 5:00 p.m. Eastern Time. The conference call will be available on Acadia’s website, www.acadia.com under the investors section and will be archived there until July 13, 2023. The conference call may also be accessed by registering for the call here. Once registered, participants will receive an email with the dial-in number and unique PIN number to use for accessing the call.

About ACP-101 (intranasal carbetocin)

ACP-101 is an investigational drug in the form of an intranasal formulation of carbetocin being developed for the treatment of hyperphagia in Prader-Willi syndrome (PWS). Carbetocin has improved drug qualities relative to oxytocin, including an extended duration of action and greater specificity for the oxytocin receptors compared to vasopressin receptors which could provide meaningful efficacy with an attractive safety profile in patients with PWS. 8 For the treatment of Prader-Willi syndrome specifically, a central nervous system disorder, an intranasal formulation of carbetocin was developed, which provides direct delivery of the drug to the brain, greatly reducing systemic exposure and the potential for side effects. ACP-101 has been granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations by the FDA.

About Prader-Willi Syndrome

Prader-Willi syndrome is a rare neurobehavioral genetic disorder that affects both males and females. 1 Prevalence estimates range from 1 in 15,000 to 1 in 25,000 live births worldwide translating to an estimated 8,000 to 10,000 patients in the United States. 2 PWS affects the functioning of the hypothalamus and other aspects of the brain with symptoms varying by individual. 1,5 The condition is typically characterized by hyperphagia which is an insatiable appetite and lack of satiety, to which a deficiency in oxytocin is believed to be contributory. Oxytocin is a natural hormone that regulates several functions in the body, including hunger, anxiety, social behavior, and bonding. 1 Individuals living with PWS have fewer neurons that produce oxytocin in the brain. 6 Other defining features of the syndrome may include reduced resting energy expenditure, developmental delays and behavioral challenges including anxiety and depression. Patients may also experience bone disorders, high pain tolerance leading to unsuspected issues such as fractures and gastrointestinal issues, respiratory and temperature regulation abnormalities. 3-5,7 There is no FDA approved treatment for the hyperphagia associated with PWS. 5

About Acadia Pharmaceuticals

Acadia is advancing breakthroughs in neuroscience to elevate life. For almost 30 years we have been working at the forefront of healthcare to bring vital solutions to people who need them most. We developed and commercialized the first and only approved therapies for hallucinations and delusions associated with Parkinson’s disease psychosis and for the treatment of Rett syndrome. Our clinical-stage development efforts are focused on treating the negative symptoms of schizophrenia, Prader-Willi syndrome, Alzheimer’s disease psychosis and neuropsychiatric symptoms in central nervous system disorders. For more information, visit us at www.acadia.com and follow us on LinkedIn and Twitter.

Forward-Looking Statements

Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements include but are not limited to statements regarding the timing of future events. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks and uncertainties inherent in drug development, approval, and commercialization. For a discussion of these and other factors, please refer to Acadia’s annual report on Form 10-K for the year ended December 31, 2022, as well as Acadia’s subsequent filings with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All forward-looking statements are qualified in their entirety by this cautionary statement and Acadia undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof, except as required by law.

References

1 Swaab DF, Purba JS, and Hofman MA. Alterations in the hypothalamic paraventricular nucleus and its oxytocin neurons (putative satiety cells) in Prader-Willi syndrome: a study of five cases.” The Journal of Clinical Endocrinology & Metabolism. 1995; 80 (2): 573-579.
2 Burd L, Vesely B, Martsolf J, et. al. Prevalence study of Prader-Willi syndrome in North Dakota. Am J Med Genet. 1990; 37: 97-9.
3 Kayadjanian N, Vrana-Diaz C, Bohonowych J, et al. Characteristics and relationship between hyperphagia, anxiety, behavioral challenges and caregiver burden in Prader-Willi syndrome. PloS ONE. 2021; 16 (3): e0248739.
4 Einfeld SL, Kavanagh SJ, Smith A, et al. Mortality in Prader-Willi syndrome. Am J Ment Retard. 2006; 111 (3): 193-8.
5 Prader-Willi Syndrome Association. What Is Prader-Willi Syndrome? Retrieved from https://www.pwsausa.org/what-is-prader-willi-syndrome/. Accessed June 1, 2023.
6 Miller JL, Tamura R, Butler MG, et al. Oxytocin treatment in children with Prader–Willi syndrome: A double-blind, placebo-controlled, crossover study. Am J Med Genet A. 2017; 173 (5): 1243-1250.
7 Butler MG, Theodoro MF, Bittel DC, et al. Energy Expenditure and Physical Activity in Prader–Willi Syndrome. Am J Med Genet A. 2007; 143A (5): 449-459.
8 Engstrom T, Barth T, Villhardt M. Oxytocin receptor binding and uterotonic activity of carbetocin and its metabolites following enzymatic degradation. Eur J Pharmacol. 1998; 355 (2-3): 203-210.

Contacts

Media Contact:
Acadia Pharmaceuticals Inc.
Deb Kazenelson
(818) 395-3043
media@acadia-pharm.com

Investor Contact:
Acadia Pharmaceuticals Inc.
Mark Johnson, CFA
(858) 261-2771
ir@acadia-pharm.com

Source: Acadia Pharmaceuticals Inc.

BIO Statement on the Section 232 Pharmaceutical Proclamation

April 4, 2026
April 2, 2026- WASHINGTON, D.C. – John F. Crowley, President and CEO of the Biotechnology Innovation Organization (BIO) , released the following statement on Section 232 Pharmaceutical Proclamation. “A thriving American biotechnology ecosystem is essential to growing the U.S. economy, strengthening national security, and improving the health and well‑being of everyday Americans. While we appreciate the Administration’s recognition of the need for tariff exemptions for certain critical biotech products, the reality is that any tariffs on America’s medicines will raise costs, impede domestic manufacturing, and delay the development of new treatments - all while doing nothing to enhance our national security. “U.S. biotech companies have been eager to expand investments here at home, but tariffs, along with an uncertain policy environment and efforts to force “most‑favored nation” schemes, work directly against that goal. The risks are especially acute for small and mid‑size biotech companies, which develop more than half of all FDA‑approved medicines yet often lack the capital to build dedicated manufacturing facilities as they weather an industry defined by high costs, long development timelines, and significant risk. “The fact is: tariffs divert scarce resources away from research and development, weaken American biotech against China’s rising industry, and ultimately, harm health and economic wellbeing of Americans. “We stand ready to work with the Administration on a long‑term strategy that encourages biotechnology investment, reduces the time, cost, and uncertainty of developing new medicines, expands U.S. biomanufacturing capacity, and ensures American innovation is fairly valued overseas. Tariffs and MFN are not the answer." Source - https://www.bio.org/press-release/bio-statement-section-232-pharmaceutical-proclamation

Georgia Life Sciences Announces 2026 Georgia BioGENEius Challenge Winner

April 1, 2026
Atlanta, GA (April 1, 2026) – Georgia Life Sciences (GLS) is proud to announce that Saisurya Lakkimsetti, a junior at Lakeside High School in Columbia County, has been named the winner of the 2026 Georgia BioGENEius Challenge. The Georgia BioGENEius Challenge took place, as part of the statewide Georgia Science and Engineering Fair (GSEF) at the Classic Center in Athens, Georgia. Forty-seven students from across Georgia competed for this year’s title and cash prize. Jaehyeon Lee, an 11th-grade student from Walton High School, was named runner-up in this year’s competition. The Georgia BioGENEius Challenge recognizes outstanding high school students who are conducting innovative biotechnology research with real-world applications. This year’s top projects, presented in the Global Healthcare Challenge track, demonstrated exceptional scientific rigor and forward-thinking potential in addressing critical healthcare challenges. Saisurya’s research focuses on identifying potential inhibitors for Endocan, a protein known to play a role in glioblastoma tumor growth. Using advanced computational modeling techniques—including AlphaFold and molecular docking tools—she screened thousands of small molecules to identify compounds that may block tumor-promoting signaling pathways. Her work identified several promising candidates that could serve as a foundation for future drug development targeting glioblastoma. Jaehyeon’s project investigates how varying glucose concentrations affect regeneration in planaria, modeling impaired wound healing in diabetic conditions. By testing graded glucose environments and measuring regeneration indicators such as growth and differentiation, Jaehyeon demonstrated that lower glucose levels enhance regeneration while higher levels inhibit healing. The study establishes a model to better understand hyperglycemia’s impact on diabetic wound healing. “The work by these students is a powerful example of the innovation and determination we see in Georgia’s next generation of life sciences leaders,” said Maria Thacker Goethe, President and CEO of Georgia Life Sciences. “The BioGENEius Challenge is critical because it provides students with a platform to apply cutting-edge science to real-world problems, while also strengthening the future workforce that will drive breakthroughs in healthcare, biotechnology, and beyond.” The Georgia BioGENEius Challenge is part of Georgia Life Sciences’ broader commitment to advancing workforce development and fostering innovation across the state’s rapidly growing life sciences ecosystem. Judging the 2026 Georgia BioGENEius Challenge: Ian Biggs; Ralph Cordell, CDC; Alex Harvey, ViaMune; Jamie Graham, Smith Gambrell Russell; and Evan Scullin, LuminiSci.

Georgia Life Science's Feature in Very First Atlanta Edition of Inside Medicine

March 24, 2026
Georgia Life Sciences is thrilled to be featured in the very first Atlanta edition of Inside Medicine . This inaugural issue represents something truly special. Atlanta’s healthcare and life sciences community is driven by innovation, collaboration, and outstanding leadership—and we’re honored to be part of this exciting launch. Also in the issue, GLS's Kennedy Dumas is featured, sharing her journey on how observation and research evolved into a powerful practice of journaling. As the founder of Stationery Black, she creates notebooks designed to showcase, uplift, and inspire people of color. Read the full article here.
MORE POSTS