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SAN DIEGO - Acadia Pharmaceuticals Inc. (NASDAQ:ACAD), a $3.96 billion pharmaceutical company with strong financial health according to InvestingPro analysis, on Thursday reported interim results from its observational LOTUS study showing that patients with Rett syndrome experienced symptom improvements when treated with DAYBUE (trofinetide) for up to 12 months in real-world clinical settings.
The findings, published in the journal Developmental Medicine and Child Neurology, included data from 227 patients aged 1 to 60 years who were prescribed DAYBUE under routine clinical care in the U.S. The company, which has achieved impressive revenue growth of 14.41% over the last twelve months, continues to expand its market presence.
According to the interim analysis, 71-90% of caregivers reported at least one behavioral symptom improvement compared to pre-treatment baseline. The most frequently reported improvements were in nonverbal communication (48-71%), alertness (44-70%), and social interaction (33-58%), as measured by the Behavioral Improvement questionnaire.
The study also provided insights into managing gastrointestinal symptoms, a common side effect of DAYBUE treatment. The incidence of diarrhea varied from 23-50% during weeks 1-12 and 26-38% during months 4-12. Most diarrhea cases were contained inside patients’ diapers, and caregivers reported normal stools in 43-54% of patients during weeks 1-12, increasing to 55-63% during months 4-12.
Patients typically started at 36% of the target FDA-approved dose in week 1, gradually increasing to at least 80% of the labeled dose by week 10 and beyond.
The LOTUS study has limitations, including the lack of a placebo arm, missing data, reliance on caregiver reports, and the use of non-validated questionnaires developed specifically for this study.
DAYBUE was approved by the FDA for the treatment of Rett syndrome in adults and pediatric patients 2 years and older. Rett syndrome is a rare genetic neurodevelopmental disorder affecting approximately 6,000 to 9,000 patients in the U.S.
The company noted that these real-world findings are consistent with results from the Phase 3 LAVENDER trial that supported FDA approval and other open-label studies of the medication.
In other recent news, Acadia Pharmaceuticals has seen several updates regarding its financial outlook and management. The company reported second-quarter Nuplazid sales of $168.5 million, aligning with consensus expectations of $159.7 million. Additionally, Daybue sales surpassed estimates, reaching $96.1 million compared to the anticipated $93.8 million. Following these earnings results, Mizuho adjusted its price target on Acadia to $24, maintaining a Neutral rating, while Stifel also raised its target to $24 from $18, citing solid execution of the company’s products. Conversely, Mizuho had previously lowered its price target to $22 from $32, noting that second-quarter results fell short of expectations when excluding certain impacts. TD Cowen has increased its price target to $39, maintaining a Buy rating, ahead of anticipated data for ACP-101 in Prader-Willi Syndrome. In leadership news, Acadia appointed Konstantina "Tina" Katcheves as Senior Vice President, Chief Business and Strategy Officer. These developments highlight the company’s ongoing strategic and financial activities.
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