Health Systems Manager/Senior Manager

Acadia Pharmaceuticals announces broad U.S. Availability of DAYBUE STIX for the treatment of Rett Syndrome. Acadia Pharmaceuticals expands U.S. Access to DAYBUE STIX for patients with Rett Syndrome. Acadia Pharmaceuticals has announced that DAYBUE(R) STIX (trofinetide) for oral solution - a newly developed powder formulation of its Rett syndrome therapy - is now broadly available across the United States. This latest offering represents an important advancement in treatment flexibility for patients living with Rett syndrome, a complex genetic condition that primarily affects girls and is associated with severe cognitive, motor, and communication impairments. The DAYBUE STIX formulation, which received approval from the U.S. Food and Drug Administration in December 2025, is designed as a dye-free and preservative-free powder that can be mixed into liquids for oral administration. Importantly, the new formulation has been shown to be bioequivalent to the original DAYBUE oral solution, meaning it delivers the same level of efficacy and safety while offering enhanced convenience and adaptability. This ensures that patients can continue to benefit from the therapeutic effects of trofinetide while gaining additional options tailored to their individual preferences and needs. Trofinetide, the active ingredient in DAYBUE, is a synthetic analog of a naturally occurring peptide derived from insulin-like growth factor 1 (IGF-1). It is designed to address core symptoms of Rett syndrome by reducing neuroinflammation and supporting synaptic function. Since its initial approval in 2023 as an oral solution, DAYBUE has become a foundational therapy in the management of Rett syndrome, marking one of the first disease-specific treatments available for this condition. The introduction of DAYBUE STIX builds on this foundation by offering a more flexible mode of administration. Unlike the ready-to-use liquid formulation, the STIX version is provided in single-use packets containing powder that can be mixed with a variety of water-based liquids, including juice, tea, lemonade, limeade, or hydration beverages. This allows caregivers to tailor the taste and volume of each dose to suit the preferences and tolerability of the patient, which can be particularly important for individuals with sensory sensitivities or feeding challenges - a common issue in Rett syndrome. Additionally, the packet-based design enhances portability, making it easier for families to administer treatment outside the home, such as during travel or school activities. This added convenience is expected to improve adherence to therapy, which is critical for maintaining consistent therapeutic benefits over time. Early feedback from caregivers has been encouraging. According to Tom Garner, Chief Commercial Officer at Acadia Pharmaceuticals, initial responses from a limited launch group indicated high levels of satisfaction with the new formulation. More than 80% of early users reported positive experiences, particularly noting the increased flexibility and portability offered by DAYBUE STIX. Garner emphasized that these real-world insights are invaluable as the company continues to refine its approach to supporting patients and caregivers managing Rett syndrome. He also highlighted that the ability to customize dosing in real-world settings may lead to more personalized care, aligning with broader trends in precision medicine and patient-centered healthcare. Acadia remains committed to gathering ongoing feedback from the Rett syndrome community to better understand how its therapies can meet evolving patient needs. The importance of flexible and individualized treatment approaches has also been underscored by recent expert consensus recommendations. A steering committee composed of specialists affiliated with centers designated by the International Rett Syndrome Foundation has recognized trofinetide oral solution as part of the standard of care for individuals with Rett syndrome. These experts emphasized the value of early initiation of therapy and the importance of sustained treatment to achieve optimal outcomes. The consensus recommendations also highlight the need for personalized decision-making in clinical practice, taking into account the unique characteristics and circumstances of each patient. This includes considerations such as age, symptom severity, comorbidities, and caregiver preferences. The availability of multiple formulations of trofinetide, including DAYBUE STIX, supports this individualized approach by providing clinicians and families with greater flexibility in how treatment is administered. Clinical experts in the field have welcomed the introduction of the new formulation. Arthur Beisang, MD, from the Department of Pediatrics at Gillette Children's Specialty Healthcare, noted that DAYBUE STIX offers an additional option that can be tailored to the specific needs of patients and caregivers. He emphasized that this patient-centered approach is consistent with current best practices in Rett syndrome management, which prioritize comprehensive and individualized care strategies. Dr. Beisang also pointed out that the ability to customize treatment delivery may help address practical challenges faced by families, such as difficulties with medication administration or patient resistance to certain tastes or textures. By providing more options, healthcare providers can work more closely with families to identify the most suitable approach for each patient, potentially improving both adherence and overall quality of life. The approval of DAYBUE STIX was supported by a bioequivalence study demonstrating that the powder formulation provides comparable systemic exposure to the original oral solution. This ensures that patients transitioning between formulations can expect consistent therapeutic effects without compromising safety or efficacy. The foundational clinical evidence for trofinetide's benefits comes from the pivotal Phase 3 LAVENDER(TM) study, which evaluated the original oral solution in patients with Rett syndrome and demonstrated statistically significant improvements in key clinical endpoints. Acadia Pharmaceuticals has also emphasized its commitment to supporting patients and caregivers throughout the treatment journey. Through its Acadia Connect(R) program, the company offers a range of services, including financial assistance, insurance navigation, and prescription support. This comprehensive support system is designed to reduce barriers to access and ensure that patients can benefit from therapy without undue burden. Families interested in exploring DAYBUE STIX are encouraged to consult with their healthcare providers to determine whether the new formulation is appropriate for their specific situation. Importantly, the original DAYBUE oral solution remains available, giving patients and caregivers the option to choose the formulation that best meets their needs. In summary, the nationwide availability of DAYBUE STIX marks a meaningful advancement in the treatment of Rett syndrome. By combining proven efficacy with enhanced flexibility, portability, and patient-centered design, the new formulation has the potential to improve the treatment experience for both patients and caregivers. As Acadia Pharmaceuticals continues to expand its portfolio and deepen its engagement with the Rett syndrome community, innovations like DAYBUE STIX underscore the company's commitment to addressing unmet needs and improving outcomes in rare neurological disorders. 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. [5-7] In the US, 6,000 to 9,000 patients are affected. [8] 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. [6] Rett syndrome is typically caused by a genetic mutation on the MECP2 gene. [9] In preclinical studies, deficiency in MeCP2 function is thought to lead to impairment in synaptic communication, and the deficits in synaptic function may be associated with Rett manifestations. [9-11] Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities. [12] Most Rett patients typically live into adulthood and require round-the-clock care. [5.13] About DAYBUE (R)(trofinetide) and DAYBUE (R) STIX (trofinetide) Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor-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. [14]

ACADIA Pharmaceuticals reported over $1 billion in combined net sales from its commercial products NUPLAZID and DAYBUE during a presentation at the Citizens Life Sciences Conference. CFO Mark Schneyer said the company expects approximately $1.25 billion in sales for the year and maintains a strong balance sheet with over $800 million in cash, no debt, and positive cash flow. The company's lead investigational asset, remlifanserin, is being studied for Alzheimer's disease psychosis and Lewy body dementia psychosis. Phase 2 Alzheimer's disease psychosis trial results are expected between August and October. Head of Clinical Development Srdjan Stankovic said the programme incorporates lessons from prior work, including targeting patients with severe psychosis, using more sensitive outcome measures, and implementing blood-based biomarkers to confirm diagnosis.