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.

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“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.

New Report Reveals National Trends Reshaping the Life Sciences Workforce

By Sheran Brown • June 24, 2025
The Life Sciences Workforce Collaborative (LSWC) has released the 2025 Life Sciences Workforce Trends Report—offering one of the most comprehensive pictures to date of the hiring, training, and talent dynamics shaping the future of the industry. Developed in partnership with TEConomy Partners, LLC , and supported by InnovATEBIO , this seventh edition of the biennial report is based on a rich set of national data: ● Survey responses from over 500 life sciences companies across 30 states and Puerto Rico ● Executive interviews with more than 200 industry leaders ● Analysis of 2.9 million unique job postings from the past four years Together, these inputs paint a nuanced picture of a sector in transition—where innovation is thriving, but economic pressures and rapid technological change are forcing organizations to rethink their workforce strategies. Slower Growth, But Not a Slowdown in Strategy The report finds that after several years of rapid post-pandemic hiring, the U.S. life sciences industry experienced a modest employment contraction of 0.3% in 2024. Hiring has become more strategic, and companies are shifting focus from volume to value—investing in automation, workforce upskilling, and future-forward technologies. Despite the hiring slowdown, long-term growth indicators are strong: more than $200 billion in U.S. life sciences manufacturing and R&D investments have been announced by major companies including Johnson & Johnson, Eli Lilly, Merck, Regeneron, and others. These expansions point to sustained demand for a skilled, resilient, and adaptable workforce. AI, Automation, and the Rise of Hybrid Talent One of the clearest trends in the report is the growing integration of AI, machine learning, and industrial automation across R&D, regulatory, and manufacturing operations. Larger companies are leading adoption, but small and mid-sized firms are quickly following. As these technologies become embedded in daily workflows, employers are seeking “bilingual” workers—those with both technical life sciences expertise and digital fluency. From AI-powered regulatory documentation to predictive maintenance in biomanufacturing, tomorrow’s workforce must be prepared to operate at the intersection of science and software. --- Upskilling, Not Just Hiring With lower turnover and cautious new hiring, many life sciences companies are turning inward—investing more heavily in their existing workforce. Employers report increasing reliance on internal training, microcredentialing, and competency-based assessments, particularly for technical production roles, quality assurance, and regulatory functions. Performance-based credentialing programs, like those offered by the Bioscience Core Skills Institute (BCSI), are helping bridge gaps between traditional education and job-readiness—especially for entry-level and mid-career talent. STEM Engagement Surging Nationwide The report also highlights a remarkable rise in employer involvement in STEM education. Compared to two years ago, more than twice as many companies report engaging with K–12 students through classroom visits, mentorship programs, science fairs, and summer camps. At the postsecondary level, internships, co-op programs, and apprenticeships are helping to build direct pathways into the workforce. Nearly 80% of companies surveyed are actively offering internships to students from four-year institutions, and nearly two-thirds to community college students. This surge in engagement reflects a broader industry focus on ensuring a future-ready talent pipeline—starting early and growing local. Why This Report Matters For employers, educators, and policymakers, the 2025 Life Sciences Workforce Trends Report is more than a snapshot—it’s a strategic tool. With workforce challenges affecting everything from drug development to supply chain resilience, coordinated, data-informed action is more urgent than ever. “This report illustrates a new era in workforce dynamics—where slower overall growth is paired with opportunity in key areas, growing emphasis on upskilling, and a shift toward technology-integrated roles,” said Liisa Bozinovic, Executive Director of Oregon Bio and Chair of LSWC. “At BIO, we are proud to support these efforts and to champion policies that strengthen our industry’s talent pipeline, from the classroom to the lab and beyond,” added John Crowley, President & CEO of the Biotechnology Innovation Organization (BIO). “As noted by the National Security Commission on Emerging Biotechnology, keeping pace with the rapidly evolving skills and talent needs of the biotechnology sector is essential to U.S. leadership in this industry,” said Linnea Fletcher, Principal Investigator at InnovATEBIO. “Our national network of advanced technical education programs is working nimbly to build the next generation of biotech talent—one that is diverse, industry-aligned, and ready to power the biotechnology revolution.” --- Access the Full Report The full 2025 Life Sciences Workforce Trends Report is now available to download: https://www.lifesciencesworkforce.org/national-workforce-trends To learn more about the Life Sciences Workforce Collaborative and how your organization can engage, visit: www.LifeSciencesWorkforce.org Follow LSWC on social media for highlights and updates: 🔹 Twitter/X: X: @LifeSciWork 🔹 LinkedIn: Life Sciences Workforce Collaborative 🔹 Instagram: @lifesciworkforce 🔹 YouTube: @lifesciworkforce

Announcing the Launch of the Life Sciences Workforce Collaborative — and a Preview of What’s Ahead

By Sheran Brown • June 17, 2025
This week marks an important milestone for workforce development in the U.S. life sciences sector: the official launch of the Life Sciences Workforce Collaborative (LSWC) . Formerly known as the Coalition of State Bioscience Institutes (CSBI) , LSWC premiered at the 2025 BIO International Convention as a new national nonprofit—building on over a decade of state and regional leadership to expand industry-aligned solutions for talent development. As the life sciences industry confronts evolving technologies, shifting economic headwinds, and persistent workforce challenges - including specialized talent needs, unmet demand for skilled biomanufacturers, positioning for future growth —LSWC offers a unified platform to scale solutions, support workforce readiness, and strengthen public-private partnerships across the country. Just out of the gate, the new organization is pleased to announce a major insight-driven release later this month: the upcoming 2025 National Life Sciences Workforce Trends Report , produced in partnership with TEConomy Partners and the NSF-funded InnovATEBIO National Biotech Education Center . Comprised of data from over 700 life science companies and 2.9 million job postings, the report will go live on June 24, 2025. Why This Matters The newly launched LSWC builds on more than a decade of trusted collaboration among 50+ state, regional and national life science organizations and workforce partners. Its mission: to align, advise, and accelerate talent development efforts across the entire life sciences talent pipeline—from middle school to mid-career--to build a competitive life sciences workforce. Next week, the LSWC will launch the 2025 National Workforce Trends Report, the organization’s flagship effort. Now in its seventh edition, this biennial report draws on: Responses from over 500 companies across 30 states and Puerto Rico Interviews with over 200 life science executives Analysis of 2.9 million unique job postings across the U.S. from the last four years The report is the definitive source for understanding the real-world trends shaping hiring, upskilling, and STEM education engagement across the life sciences ecosystem. More to come next week. What Comes Next In the weeks ahead, we’ll be working with our partners to roll out media toolkits, schedule events, and activate coordinated campaigns to bring visibility to both the LSWC launch and the report. We are deeply grateful to our investor-level supporters—AZBio/AZ Advances, BioNJ, BioUtah, California Life Sciences, Georgia Life Sciences/Institute, Colorado Bioscience Association/Institute, MichBio, NewYorkBIO/Institute, Ohio Life Sciences, Oregon Bioscience Association, SCbio, and Southern California Biomedical Council (SoCalBio)—LSWC partners and connectors, and to every organization that has shaped this initiative from the ground up. Together, we are building a stronger, competitive, and future-ready workforce—one that ensures the U.S. remains the global leader in biotechnology, biomanufacturing, and life sciences innovation. Stay tuned and learn how you can partner with us to advance the life sciences workforce. Learn more at: www.LifeSciencesWorkforce.org For media inquiries: connect@lifesciencesworkforce.org

Georgia Life Sciences Accepted into ARPA‑H Customer Experience Hub

By Sheran Brown • June 16, 2025
GLS has been named a new Spoke Member of the ARPA‑H Customer Experience Hub—ARPA‑H’s patient‑centric network dedicated to embedding real-world user insights and representation into health innovation. As part of the nationwide ARPANET‑H hub‑and‑spoke initiative (with hubs in Dallas, Boston, and D.C.), Georgia Life Sciences will help prioritize inclusive design, usability testing, and equitable trial participation in next-gen therapies.
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