Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced the
initiation of the Phase 3 COMPASS PWS study evaluating the efficacy
and safety of carbetocin nasal spray (ACP-101) for the treatment of
hyperphagia in Prader-Willi syndrome (PWS). PWS is a rare,
neurobehavioral genetic disorder that is estimated to affect 8,000
to 10,000 patients in the United States.1-4 The most common symptom
is hyperphagia, which is an unrelenting lack of satiety. Other
defining features of PWS may include altered metabolism,
developmental delays, behavioral challenges and moderate cognitive
deficits.5
“Currently there is no FDA-approved treatment for hyperphagia in
PWS, which presents serious challenges for those living with this
condition and their families,” said Shawn McCandless, M.D., Chair,
Department of Genetics and Metabolism, Children’s Hospital Colorado
and COMPASS PWS study investigator. “Essentially all experience
hyperphagia, feeling a near-constant state of hunger, as though
their body is telling them that they are starving, despite being
actually well-nourished. As a result, individuals with PWS often
exhibit food-seeking behaviors that require constant supervision to
prevent life-threatening risks, including gastric rupture,
irregular swallowing and choking. I look forward to the outcome of
this Phase 3 clinical trial, and the potential of having a
treatment option to offer those living with PWS.”
COMPASS PWS is a 12-week, double-blind, randomized,
placebo-controlled global Phase 3 trial evaluating the efficacy and
safety of carbetocin nasal spray 3.2 mg three times daily (TID) in
approximately 170 children and adults aged five to 30 years with
PWS. The primary efficacy endpoint of the study is change from
baseline to week 12 on the hyperphagia questionnaire for clinical
trials (HQ-CT) score, a caregiver assessment for
hyperphagia-related behaviors. Participants who complete the Phase
3 study will be eligible to enroll in a long-term, open-label
extension study designed to investigate the safety and tolerability
of long-term treatment with ACP-101.
“The launch of the Phase 3 COMPASS PWS trial will build on
previous Phase 3 clinical trial experience, where carbetocin nasal
spray 3.2 mg was observed to reduce hyperphagia-related behaviors,”
said Ponni Subbiah, M.D., M.P.H., Senior Vice President, Global
Head of Medical Affairs and Chief Medical Officer. “We look forward
to working with the Prader-Willi community and clinical research
sites as we continue to advance the ACP-101 clinical development
program for those living with this debilitating syndrome and their
families.”
More information about the COMPASS PWS study is available at
www.CompassPWS.com.
About Prader-Willi Syndrome
Prader-Willi syndrome (PWS) is a rare neurobehavioral genetic
disorder that affects both males and females.6 PWS is estimated to
affect approximately 8,000 to 10,000 patients in the United
States.3,4 PWS affects the functioning of the hypothalamus and
other aspects of the brain with symptoms varying by individual.5,6
The most common symptom is hyperphagia, which is an unrelenting
lack of satiety, to which a deficiency in oxytocin is believed to
be contributory.5,7 Individuals living with PWS have fewer
oxytocin-producing neurons in the brain.7 Other defining features
of the syndrome may include altered metabolism, developmental
delays, behavioral challenges and moderate cognitive deficits.3
Patients may also experience high pain tolerance, sleep
disturbances, gastrointestinal issues, respiratory and temperature
regulation abnormalities.5,7-9 There is no FDA-approved treatment
for the hyperphagia associated with PWS.5
About Carbetocin Nasal Spray (ACP-101)
Carbetocin nasal spray is an investigational drug being
developed for the treatment of hyperphagia in Prader-Willi syndrome
(PWS). Carbetocin has improved drug qualities relative to oxytocin,
including an extended half-life and greater specificity for the
oxytocin receptor compared to vasopressin receptors which could
provide meaningful efficacy with an attractive safety profile in
patients with PWS.10 For the treatment of PWS 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. Acadia acquired Levo Therapeutics and
worldwide rights to carbetocin nasal spray in June 2022. Carbetocin
nasal spray has been granted Orphan Drug, Fast Track, and Rare
Pediatric Disease designations by the FDA.
About Acadia Pharmaceuticals
Acadia is advancing breakthroughs in neuroscience to elevate
life. For 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 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. 2 Cassidy SB, Schwartz S, Miller JL, et al.
Prader-Willi syndrome. Genetics in Medicine. 2012;14(1). 3
McCandless SE, Suh M, Yin D, et al. SUN-604 U.S. Prevalence &
Mortality of Prader-Willi Syndrome: A Population-Based Study of
Medical Claims. J Endocr Soc. 2020 May 8;4(Suppl 1):SUN-604. 4
Acadia Pharmaceuticals Inc. Data on File. PWS Prevalence. 2023. 5
Prader-Willi Syndrome Association. What Is Prader-Willi Syndrome?
Retrieved from
https://www.pwsausa.org/what-is-prader-willi-syndrome/. Accessed
October 24, 2023. 6 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. J Clin Endocrinol Metab. 1995;80(2):573-579. 7
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. 8 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. 9 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. 10
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.
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Media Contact: Acadia Pharmaceuticals Inc. Deb Kazenelson (818)
395-3043 media@acadia-pharm.com Investor Contact: Acadia
Pharmaceuticals Inc. Al Kildani (858) 261-2872 ir@acadia-pharm.com
Acadia Pharmaceuticals Inc. Jessica Tieszen (858) 261-2950
ir@acadia-pharm.com
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