SAN DIEGO - Acadia Pharmaceuticals Inc. (NASDAQ: ACAD) has shared results from two extension studies, LILAC-1 and LILAC-2, indicating that DAYBUE, a treatment for Rett syndrome, showed sustained symptom improvement over time.
The findings, published in the journal Med, suggest that patients who continued DAYBUE treatment experienced better outcomes in Rett syndrome behavior, as measured by the Rett Syndrome Behaviour Questionnaire (RSBQ).
In the LILAC-1 study, 154 female patients aged five to 21 received DAYBUE for 40 weeks after a 12-week double-blind period. The study reported a mean change in RSBQ scores indicating improvements regardless of whether patients were initially treated with DAYBUE or a placebo.
Similarly, LILAC-2, a 32-month study of 77 participants from LILAC-1, showed sustained RSBQ score improvements, with a significant percentage of participants experiencing a decrease of at least 10% at week 104.
Caregivers also noted improvements in engagement, hand use, and eye gaze, with some participants developing new sounds or words. Despite these positive outcomes, the most common side effects included diarrhea and vomiting, which were mostly mild or moderate in severity.
These long-term studies support the safety and efficacy of DAYBUE, mirroring the safety profile from the initial LAVENDER trial. The consistency of these findings could be significant for the estimated 5,000 diagnosed patients with Rett syndrome in the U.S., a neurodevelopmental disorder characterized by a regression in communication skills and purposeful hand use, among other symptoms.
DAYBUE, whose therapeutic mechanism in Rett syndrome remains unknown, is a synthetic analog of a peptide derived from insulin-like growth factor 1. While the treatment has shown promise in increasing synaptic plasticity signals in animal studies, its effect in humans is being closely monitored.
Acadia Pharmaceuticals, focusing on breakthroughs in neuroscience, has emphasized that these studies add to the robust data portfolio for DAYBUE's role in treating Rett syndrome. This announcement is based on a press release statement and does not constitute an endorsement of DAYBUE's efficacy or safety.
In other recent news, Acadia Pharmaceuticals has been the subject of several analyst adjustments following the presentation of Phase 4 LOTUS and LILAC-2 data, as well as the company's Q1 2024 earnings report.
TD Cowen maintained a Buy rating on the company, signaling confidence in the therapeutic prospects of Acadia's drug Daybue, which has shown potential benefits in patient care. However, H.C. Wainwright and BofA Securities lowered their price targets for Acadia, citing revised expectations for the market penetration of Daybue and the potential of ACP-101.
Acadia reported Q1 2024 revenues of $205.8 million, with sales of Daybue reaching $75.9 million, slightly below consensus projections. Despite this, the company remains confident in its growth trajectory. The LOTUS study, examining the efficacy of Daybue in treating Rett syndrome, has shown improvements in more than two-thirds of the patients, offering hope for improvements in patient care.
The company's ACP-101, an investigational therapy currently in clinical trials, is also seen as a potential growth driver. These are recent developments in Acadia Pharmaceuticals' operations and market performance.
InvestingPro Insights
As Acadia Pharmaceuticals Inc. (NASDAQ: ACAD) continues to report promising results for its Rett syndrome treatment, DAYBUE, investors and stakeholders may find additional context in the company's financial health and market performance. Here are key insights from InvestingPro that could provide a broader understanding of Acadia's position in the market:
InvestingPro Data shows Acadia's Market Cap stands at $3.03 billion, reflecting the company's size and investor valuation within the pharmaceutical industry. Despite not being profitable over the last twelve months, the company's Revenue Growth was impressive, with a 56.43% increase during the last twelve months as of Q1 2024 and an even more remarkable quarterly surge of 73.75% in Q1 2024. Acadia's Gross Profit Margin at 50.22% indicates a solid capability to control costs relative to revenue—a critical factor for potential long-term sustainability.
An InvestingPro Tip that stands out is the company's strong return over the last month, with a 23.4% price total return, which could signal growing investor confidence following positive news from the clinical trials. Moreover, Acadia's liquid assets surpass short-term obligations, suggesting the company is well-positioned to manage its immediate financial commitments.
For interested parties looking to dive deeper into Acadia's financial metrics and strategic outlook, there are additional InvestingPro Tips available. These include insights on the company's cash position, expected net income growth, and analyst profitability predictions for the year.
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