Findings reinforce efficacy and tolerability profile in patients prescribed DAYBUE in the U.S. for as long as 12 months, consistent with the Phase 3 LAVENDER trial that supported FDA approval and open-label LILAC, LILAC-2 and DAFFODIL studies

Acadia Pharmaceuticals Inc. announced that the journal Developmental Medicine and Child Neurology published interim results from the caregiver-reported observational online, open-label, ongoing LOTUS study evaluating effectiveness and tolerability outcomes in patients with Rett syndrome who are prescribed DAYBUE  (trofinetide) under routine clinical care in the U.S. Findings included reported improvements in symptoms of Rett syndrome, with early insights into managing gastrointestinal (GI) symptoms in the real world among patients receiving up to 12 months of treatment with DAYBUE.

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“These findings from LOTUS help deepen our understanding of the potential outcomes associated with long-term treatment of DAYBUE in the real-world setting,” said Ponni Subbiah, M.D., M.P.H., Senior Vice President, Global Head of Medical Affairs and Chief Medical Officer. “The data are supportive of what we’ve seen in DAYBUE clinical trials and offer new scientific insights to clinicians that improvements in Rett syndrome signs and symptoms are possible among a broadly inclusive sample of Rett patients. It’s particularly gratifying to document improvements in adult patients, knowing that they may have waited decades without knowing if a treatment would become available within their lifetime.”

This interim analysis, which included 227 people living with Rett syndrome with an age range of one to 60 years old, showed:

  • 71–90% of those who filled out the Behavioral Improvement questionnaire (BIQ) reported at least one behavioral symptom improvement that were new or maintained when compared with before DAYBUE treatment. The BIQ was developed by Acadia specifically for this study to document caregiver observations.
  • Consistent with the previously reported six-month interim analysis, the most frequent caregiver-reported improvements were nonverbal communication (48–71%), alertness (44–70%), and social interaction/connectedness (33–58%), as measured by the BIQ.
  • Additionally, the median change from baseline in Quality-of-Life Inventory-Disability Questionnaire (QI-Disability) total score was 4.6 [IQR, -0.2 to 10.2] for those patients (n=33) who had both baseline and 12-month assessments.
  • The incidence of diarrhea reported by caregivers varied from weeks 1 to 12 (23–50%) and months 4 to 12 (26–38%). Most reports of diarrhea were contained inside the patient’s diaper, as measured by the Gastrointestinal (GI) Health Questionnaire, also developed by Acadia specifically for this study in consultation with Rett experts and caregivers. The most commonly reported diarrhea management strategies included avoiding constipation medications, increasing fluid intake, consuming supplementary fiber, and using antidiarrheal medication.
  • Caregivers reported normal stools over the last 3 days immediately prior to completing the GI assessment from weeks 1 to 12 (43−54%) and months 4 to 12 (55−63%).
  • The median dose reported at Week 1 was 36% of the target weight-banded FDA approved dose; by Week 10 onwards, the median dose was at least 80% of labeled dose.

The results of this 12-month follow-up are limited by the number of patients who had reached later time points, which resulted in the data being restricted to 12 months, lack of a placebo arm, missing data, lack of validation of BIQ and GI questionnaires, reliance solely on caregiver reports, the use of descriptive statistics and the online nature of this study. Participants are being enrolled in the LOTUS study for at least 12 months from initiation of DAYBUE treatment, with the option to extend participation for an additional 12 months.

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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. In the U.S., 6,000 to 9,000 patients are affected. 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.2 Rett syndrome is typically caused by a genetic mutation on the MECP2 gene. 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.

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

About DAYBUE

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.

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Source – businesswire