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.

Athens Bioscience Names New Leadership Team to Drive Commercial Growth

June 18, 2026
June 18, 2026 - Athens Bioscience, Inc., a US manufacturer of native human and animal proteins, today announced a change in leadership. Benjamin Newland, the company’s Executive Chairman, has become Chairman and Chief Executive Officer, effective today. John Mitchell, who has led the company as CEO for five years, will continue as special advisor. Karson Durie rejoins Athens as Chief Operating Officer, effective June 8, 2026. Dee Athwal, a biotechnology executive and antibody engineer, will join the board of directors on July 1, 2026. Newland becomes chief executive four decades after his father, Dr. Hillary Newland, co-founded the company that became Athens Bioscience in 1986 at the University of Georgia. Newland has served as Chairman and majority shareholder since March 2024 and is relocating from Spain to Athens, Georgia, to take the role. “For forty years Athens has made native proteins in-house, lot after lot — proteins that researchers and diagnostics labs build their own work on top of,” said Benjamin Newland, Chairman and CEO of Athens Bioscience. “What we want to do now is deepen relationships with our core customers and broaden our distribution.We also intend to develop our custom and contract manufacturing line of business. John was instrumental in building internal systems and processes and now we are ready to scale.” Mitchell led Athens for five years. As special advisor, he will support the transition and continue to advise on customer and commercial matters. “Athens made great proteins long before I showed up. What it needed was a tighter operation behind them – steadier planning, cleaner production, shipments you can count on,” said John Mitchell. “That’s built now. The next thing is getting Athens in front of more of the world and I look forward to supporting Benjamin as he moves forward with that.” Durie returns to Athens as Chief Operating Officer, having most recently served as Director of Product Development at Danimer Scientific. She previously served as Lab Director at Athens. She holds a PhD in polymer chemistry and an MBA in finance from the University of Georgia and is a Project Management Professional (PMP) and a registered patent agent. “I know this facility and the people in it,” said Karson Durie, Chief Operating Officer of Athens Bioscience. “Returning as COO is a chance to scale what already works — consistent lots, tight quality control, reliable supply — as demand grows across diagnostics and cell culture.” Athwal will join the board on July 1. He trained as a biophysicist and established the antibody engineering group at Celltech, where he is named as an inventor on foundational antibody-engineering patents. He has founded or co-founded five biotechnology companies and held C-level roles across the UK, US, Europe, Asia, and Latin America, including building Complement Therapeutics as CEO and leading the biologics strategy at Kelix Bio through its acquisition by Mubadala. His work in antibodies, immune proteins, and complement biology maps directly to Athens’s largest product lines. About Athens Bioscience  Athens Bioscience, Inc. (formerly Athens Research & Technology) manufactures native human and animal proteins for research, cell culture media, and in vitro diagnostics. Founded in 1985 at the University of Georgia, the company purifies more than 170 native proteins in-house at its ISO 9001:2015-certified facility in Athens, Georgia. Athens proteins have been cited in more than 2,500 peer-reviewed publications and reach customers across the US, Europe, Asia, the Middle East, and Africa. Athens does not resell or broker. It manufactures. Media Contact Christie DeMasi Athens Bioscience, Inc. christie@athensbioscience.com +1.706.546.0207

Showcase Your Startup at the 2026 Georgia Life Sciences Summit

June 17, 2026
As part of the Georgia Life Sciences Summit (August 25–26), applications are now open for the Startup Showcase , sponsored by Johnson & Johnson , highlighting early-stage innovation across MedTech and Therapeutics . The Showcase will feature some of Georgia’s most innovative life science startups, giving emerging companies the opportunity to present groundbreaking technologies, products, and solutions to a distinguished audience of industry leaders, investors, researchers, and partners. Selected startups will present during the August 26 luncheon program and participate in a live investor Q&A session with John Gutierrez (Ascenta Capital), Emma Heckenberg, Ph.D. (Solas BioVentures), Patrick Jordan (NovaQuest Capital Management), and Emily Dinu (Numinous Capital) Members of the Johnson & Johnson external innovation team will also be onsite for the program and select companies will be scheduled for a 1:1 meeting to discuss strategic alignment and potential collaboration opportunities. If you or companies in your network are building in MedTech or Therapeutics, this is a strong opportunity to gain visibility, receive feedback on commercialization and growth strategy, and connect with key stakeholders. All applicants receive a complimentary Summit registration, and one selected company will also be invited to participate in the NewYorkBIO / New York Stock Exchange Life Sciences Showcase on December 10. If this is relevant to your work, it would be a strong opportunity to consider applying. If not, it may be worth sharing with companies in your network who are building in this space.

Georgia Life Sciences Educator Connection | June Newsletter

June 15, 2026
Workforce & Education Impact: Building Georgia's Future  Life Sciences Workforce - One Teacher at a Time June 16, 2026 - As Georgia Life Sciences concludes the 2025-2026 cycle of the Biotech Teacher Training Initiative (BTTI), the results reinforce the critical role educators play in building the state's future life sciences workforce. Since July of 2025, BTTI engaged 98 educators representing 40 schools across 23 school systems, reaching an estimated 5,375 students through hands-on biotechnology instruction, career-connected learning, and industry-relevant classroom experiences. The Georgia Life Sciences Equipment Depot further expanded the program's impact by supporting 238 teachers with access to laboratory equipment and materials that make authentic life sciences learning possible. Georgia Life Sciences is also pleased to report that funding for BTTI was maintained in the Georgia House version of the FY 2027 budget approved earlier this year. The upcoming 2026-2027 program cycle will mark an important evolution for BTTI. Building on feedback from industry partners and workforce trends across the life sciences sector, Georgia Life Sciences will expand the program's scope to better reflect the skills and competencies employers increasingly need. In addition to foundational biotechnology concepts, future programming will be organized around key industry domains, including Quality Assurance and Quality Control (QA/QC), Research & Development (R&D), MedTech, Artificial Intelligence, Automation, and Advanced Manufacturing. This expanded approach will provide educators with greater insight into the breadth of career opportunities available across Georgia's life sciences ecosystem while helping students develop awareness of the technologies and disciplines shaping the industry's future. The impact extends far beyond individual workshops, creating lasting connections between Georgia classrooms and the state's growing life sciences ecosystem while helping build the talent pipeline that will power Georgia's future innovation economy. "Before BTTI, I was hesitant to do biotech labs because they seemed too complex and expensive," shared Josephine Jeganathan of Stockbridge High School. "The program showed me how simplified it can be and provided the equipment and materials needed to successfully implement the Central Dogma Lab with all my classes." Teachers are also seeing increased student engagement through hands-on learning experiences. Tonie Curry of North Clayton High School used a chromatography lab to connect environmental science concepts to water pollution and sustainability. "Students were highly interested in seeing how substances separated and made strong connections to water pollution and environmental sustainability," Curry noted. "The hands-on nature of the lab encouraged curiosity and deeper understanding." For many educators, one of the most valuable aspects of the program is the connection between classroom learning and real-world careers. As Marshai Waiters of Marietta Middle School reflected: "Exposure is key. There are so many avenues to work in STEM, and they are all accessible with opportunity and knowledge. The insights gained from industry speakers will inform my teaching and create new opportunities for student exposure." When teachers are trained, equipped, and connected to industry, students gain more than a science lesson—they gain a window into Georgia's life sciences future.
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