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SAN DIEGO - Acadia Pharmaceuticals Inc. (NASDAQ:ACAD), a biopharmaceutical company with a market capitalization of $3.05 billion and strong financial health according to InvestingPro metrics, has announced the publication of a study in the journal Med, which supports the safety and tolerability of DAYBUE® (trofinetide) for treating Rett syndrome in children aged two to four. The DAFFODIL™ study, a long-term Phase 2/3 trial, showed that DAYBUE had a similar safety profile in this age group as observed in older individuals with Rett syndrome in previous studies. These findings played a role in the FDA’s approval of DAYBUE for patients two years and older. The company’s focus on breakthrough treatments has contributed to its impressive 31.85% revenue growth over the last twelve months.
Rett syndrome, a rare neurodevelopmental disorder, typically manifests in early childhood and leads to severe impairments in communication and motor skills. The DAFFODIL study results are particularly significant as they offer insights into the treatment of younger patients, who are closer to the onset of symptoms.
During the 78-week study, the most common treatment-emergent adverse events (TEAEs) were diarrhea and vomiting, with all cases being mild or moderate. The implementation of a diarrhea management plan resulted in only one discontinuation due to diarrhea. There were four serious TEAEs, but they were deemed unrelated to the treatment.
Exploratory efficacy endpoints suggested symptom improvement in participants. Scores on the Clinical Global Impression-Improvement (CGI-I) and Caregiver Global Impression-Improvement (CaGI-I) scales indicated progress over time. Additionally, the Impact of Childhood Neurologic Disability-Quality of Life (ICND-QoL) scale scores increased from baseline to the study’s end.
Caregiver feedback highlighted communication as a critical area of concern and improvement, with notable advances in verbal communication and eye contact.
Acadia Pharmaceuticals is recognized for its focus on neuroscience and has previously brought to market the first FDA-approved drugs for Parkinson’s disease psychosis and Rett syndrome. The company continues to develop treatments for other neuro-psychiatric and neuro-rare diseases. InvestingPro analysis reveals the company maintains a healthy balance sheet with more cash than debt and a strong current ratio of 2.38, providing financial flexibility for continued research and development. For detailed insights into ACAD’s valuation and growth prospects, investors can access the comprehensive Pro Research Report, available exclusively to InvestingPro subscribers.
DAYBUE, a synthetic analog of insulin-like growth factor 1, is designed to treat Rett syndrome, although its exact therapeutic mechanism is not fully understood. It is available as an oral solution and has associated risks, including diarrhea, vomiting, and weight loss, which healthcare providers are advised to monitor and manage.
This information is based on a press release statement from Acadia Pharmaceuticals Inc. The company’s solid financial performance, with earnings per share of $1.37 and a return on assets of 23.39%, suggests strong operational execution. Additional financial metrics and analysis are available through InvestingPro, which offers exclusive access to over 30 key financial indicators and expert insights.
In other recent news, Acadia Pharmaceuticals reported a 12% increase in its fourth-quarter 2024 revenue, reaching $259.6 million, contributing to a total annual revenue of $957.8 million—a 32% increase from the previous year. The company’s DAYBUE product was a significant driver of growth, with 2024 sales surging 97% to $348.4 million. Looking forward, Acadia forecasts 2025 revenue to be between $1.03 billion and $1.095 billion, with expectations for DAYBUE sales to reach between $380 million and $450 million. Meanwhile, TD Cowen has maintained a Buy rating on Acadia, setting a price target of $35, following the company’s earnings that exceeded expectations. Citizens JMP, however, adjusted its price target from $39 to $37 while retaining a Market Outperform rating, citing revised projections for DAYBUE. Acadia is also advancing its clinical pipeline, with a Phase 2 trial for ’204 in Lewy Body Dementia anticipated to begin in the third quarter of 2025. These developments highlight Acadia’s ongoing efforts to expand its market presence and strengthen its product offerings.
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