Acadia Pharmaceuticals Submits Marketing Authorization Application to the European Medicines Agency for Trofinetide for the Treatment of Rett Syndrome

-- Filing marks company’s first Marketing Authorization Application in Europe

SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the company has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for trofinetide for the treatment of Rett syndrome in adults and pediatric patients two years of age and older. If granted marketing authorization, trofinetide will be the first and only approved therapy for Rett syndrome in the European Union (EU). Trofinetide is already approved in the United States and Canada for Rett syndrome in adults and pediatric patients two years of age and older.

“This application underscores our continued dedication to the Rett community, and our commitment to making a meaningful impact for people living with Rett syndrome and their caregivers in the EU, who currently have no approved treatment options specifically for this condition,” said Catherine Owen Adams, Acadia’s Chief Executive Officer. “We look forward to working with the EMA to address this unmet need in the hopes of potentially bringing this therapy to families who are impacted by Rett syndrome.”

The MAA is supported by positive results from the pivotal Phase 3 LAVENDER™ study evaluating the efficacy and safety of trofinetide versus placebo in 187 girls and young women five to 20 years of age with Rett syndrome. The co-primary endpoints were change from baseline in the Rett Syndrome Behaviour Questionnaire (RSBQ) total score, a caregiver assessment, and Clinical Global Impression–Improvement (CGI-I) scale score, clinician perspective, at week 12; both were statistically significant. The RSBQ is a 45-item rating scale completed by the caregiver that assesses a range of symptoms of Rett syndrome (breathing, hand movements or stereotypies, repetitive behaviors, night-time behaviors, vocalizations, facial expressions, eye gaze, and mood). The key secondary endpoint measuring the change from baseline to week 12 in the Communication and Symbolic Behavior Scales Development Profile™ Infant-Toddler Checklist–Social composite score (CSBS-DP-IT–Social) was also statistically significant when compared to placebo.1

“Rett syndrome is a severe and complex neurodevelopmental disorder that manifests differently in each affected person, leading to a wide range of symptoms over the course of their lives,” said Becky Jenner, President of Rett Syndrome Europe. “The Rett syndrome community in Europe is excited by the prospect of having an available treatment option. Rett syndrome has a significant impact on people living with this condition, and also impacts caregivers and families who often have to navigate challenging symptoms that impact their daily lives.”

About Rett Syndrome

Rett syndrome is a rare, complex, neurodevelopmental disorder that may occur over four stages and occurs in approximately one of every 10,000 to 15,000 female births worldwide.2-4 A child with Rett syndrome exhibits an early period of apparently normal development until six to 18 months, when their skills seem to slow down or stagnate. This is typically followed by a duration of regression when the child loses acquired communication skills and purposeful hand use. The child may then experience a plateau period in which they show mild recovery in cognitive interests, but body movements remain severely diminished. As they age, those living with Rett may continue to experience a stage of motor deterioration which can last the rest of the patient’s life.3 Rett syndrome is typically caused by a genetic mutation on the MECP2 gene.5 In preclinical studies, deficiency in MeCP2function is thought to lead to impairment in synaptic communication, and the deficits in synaptic function may be associated with Rett manifestations.5-7

Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities.8 Most people living with Rett syndrome typically live into adulthood and require round-the-clock care.2,9

About Trofinetide

Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1 (IGF-1). The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown. In animal studies, trofinetide has been shown to increase branching of dendrites and synaptic plasticity signals.10

About Acadia Pharmaceuticals Inc.

Acadia is advancing breakthroughs in neuroscience to elevate life. Since our founding 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 FDA-approved drug to treat hallucinations and delusions associated with Parkinson’s disease psychosis and the first and only approved drug in the United States and Canada 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 X.

Contacts

Investor Contact: Acadia Pharmaceuticals Inc. Al Kildani (858) 261-2872 [email protected]

Media Contact: Acadia Pharmaceuticals Inc. Deb Kazenelson (818) 395-3043 [email protected]