Acadia Pharmaceuticals Announces Trofinetide New Drug Submission for Treatment of Rett Syndrome Has Been Accepted for Filing and Priority Review by Health Canada - Seite 2

About Rett Syndrome

Rett syndrome is a rare genetic neurodevelopmental disorder that occurs primarily in females following a near normal development in the first two years of life.^1,2 It is caused by mutations on the X chromosome on a gene called MECP2.³ Rett syndrome is a complex and multisystem disorder that causes profound impairment to central nervous system (CNS) function, including loss of communication skills, purposeful hand use, gait abnormalities, and stereotypic hand movements such as hand wringing/squeezing, clapping/tapping, mouthing and washing/rubbing automatisms.²

Rett syndrome occurs worldwide in approximately one of every 10,000 to 15,000 female births.⁴ In Canada, prevalence of Rett syndrome is estimated to be 600 to 900 patients.⁵ Children with Rett syndrome experience a period of developmental regression between 18-30 months of age, which is typically followed by a plateau period lasting years to decades.^1-3 Rett syndrome is diagnosed based on clinical evaluation, typically by about three years of age.^2,6

About Acadia Pharmaceuticals Inc.

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 in the United States the first and only FDA-approved drug to treat hallucinations and delusions associated with Parkinson’s disease psychosis and the first and only FDA-approved drug for the treatment of Rett syndrome. Our clinical-stage development efforts are focused on Prader-Willi syndrome, Alzheimer’s disease psychosis and multiple other programs targeting neuropsychiatric symptoms in central nervous system disorders. For more information, visit us at Acadia.com and follow us on LinkedIn and Twitter.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements other than statements of historical fact and can be identified by terms such as “intends,” “may,” “will,” “should,” “can,” “could,” “would,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “projects,” “predicts,” “potential” and similar expressions (including the negative thereof) intended to identify forward-looking statements. Forward-looking statements contained in this press release, include, but are not limited to, statements about: (i) the potential approval of trofinetide as the first option in Canada for the treatment of Rett Syndrome, (ii) the efficacy and safety profile of trofinetide for patients with Rett Syndrome, (iii) market acceptance in Canada, including the importance of trofinetide for the treatment of Rett Syndrome, if approved, for Rett patients or families with patients with Rett Syndrome and (iv) the ability for trofinetide to address the unmet medical needs of Canadians living with Rett syndrome. Forward-looking statements are subject to known and unknown risks, uncertainties, assumptions, and other factors that may cause our actual results, performance or achievements to differ materially and adversely from those anticipated or implied by our forward-looking statements. Such risks, uncertainties, assumptions and other factors include, but are not limited to: the ability for trofinetide to deliver efficacious and safe results to patients, if approved, our dependency on the continued successful commercialization of Daybue in the United States, where it is approved, our ability to obtain regulatory approval of trofinetide in Canada and other jurisdictions outside the United States, our ability to protect and enhance our intellectual property; and our ability to continue to stay in compliance with applicable laws and regulations. Given the risks and uncertainties, you should not place undue reliance on these forward-looking statements. For a discussion of these and other risks, uncertainties, assumptions, and other factors that may cause our actual results, performance or achievements to differ, please refer to our annual report on Form 10-K for the year ended December 31, 2023, filed with the Securities and Exchange Commission on February 28, 2024, as well as our subsequent filings with the Securities and Exchange Commission from time to time. The forward-looking statements contained herein are made as of the date hereof, and we undertake no obligation to update them after this date, except as required by law.