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Acadia's DAYBUE wins first Canada nod for Rett syndrome treatment

Published 10/17/2024, 04:14 AM
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SAN DIEGO - Acadia Pharmaceuticals Inc. (NASDAQ:ACAD) announced today that Health Canada has approved DAYBUE™ (trofinetide) for treating Rett syndrome in adults and children aged two and older. This marks the first authorized therapy for Rett syndrome in Canada, a rare neurodevelopmental disorder predominantly affecting females.

Rett syndrome is characterized by severe impairments following an initial period of normal development within the first two years of life. Symptoms include communication difficulties, loss of motor skills, and repetitive hand movements. The disorder impacts an estimated 600 to 900 individuals across Canada.

The approval of DAYBUE was expedited under Health Canada's Priority Review process, reflecting the urgent need for a specific treatment for Rett syndrome, which has historically been managed only symptomatically. The authorization was based on results from the Phase 3 LAVENDER™ study, which demonstrated significant improvements in behavior and clinical global impressions over a 12-week period compared to placebo.

Catherine Owen Adams, CEO of Acadia Pharmaceuticals, expressed the company's dedication to making DAYBUE available to Canadian patients as quickly as possible. The drug's approval is a significant advancement for the Rett syndrome community, offering a new therapeutic option beyond symptomatic care.

Healthcare professionals, including Dr. Anita Datta, Pediatric Neurologist, highlighted the variability of Rett syndrome's presentation and the lack of specific treatments prior to DAYBUE's approval. The Ontario Rett Syndrome Association also welcomed the news, emphasizing the potential impact on the quality of life for individuals and families affected by the condition.

DAYBUE is indicated for Rett syndrome patients two years and older weighing at least 9 kg. Its therapeutic mechanism in Rett syndrome is not fully understood, but it is a synthetic analog of a protein associated with growth and development.

Acadia Pharmaceuticals, known for its neuroscience breakthroughs, has previously developed treatments for Parkinson's disease psychosis and is actively researching therapies for other central nervous system disorders.

This news article is based on a press release statement from Acadia Pharmaceuticals Inc.

In other recent news, Acadia Pharmaceuticals reported significant growth in Q2 2024 earnings, with revenues reaching $242 million, primarily driven by its commercial products, NUPLAZID and DAYBUE. The company updated its annual sales guidance for NUPLAZID, noting an 11% increase in net product sales, while DAYBUE also saw an 11% sales increase. However, Acadia has revised its total revenue guidance for 2024, now expecting between $930 million and $980 million.

On the leadership front, Catherine Owen Adams has been appointed as the new CEO of Acadia Pharmaceuticals, succeeding Stephen R. Davis. Adams brings over 25 years of experience in the pharmaceutical industry and is expected to guide the company through its next phase of growth.

In analyst notes, Needham has adjusted its price target for Acadia, reducing it to $28 from the previous $30, while maintaining a Buy rating on the stock. This adjustment follows a shortfall in Daybue sales revealed in Acadia's second-quarter earnings. Other developments include BMO Capital Markets maintaining its optimistic stance on Acadia Pharmaceuticals, reiterating an Outperform rating with a steady price target of $31.00. The focus is on the potential approval of KarXT (Cobenfy) for treating psychosis in schizophrenia and its implications for Acadia's pipeline.

InvestingPro Insights

As Acadia Pharmaceuticals (NASDAQ:ACAD) celebrates this significant milestone with the approval of DAYBUE in Canada, it's worth examining the company's financial health and market position. According to InvestingPro data, Acadia's market capitalization stands at $2.5 billion, reflecting its position as a mid-cap biopharmaceutical company.

The approval of DAYBUE in Canada aligns with an InvestingPro Tip indicating that Acadia's net income is expected to grow this year. This growth projection is further supported by the company's impressive revenue growth of 61.65% over the last twelve months, with quarterly revenue growth of 46.44% in Q2 2024. These figures suggest that Acadia's innovative therapies, including DAYBUE, are gaining traction in the market.

Another relevant InvestingPro Tip highlights that Acadia holds more cash than debt on its balance sheet. This strong financial position could provide the company with the flexibility needed to support the commercialization of DAYBUE in Canada and potentially fund further research and development initiatives.

It's worth noting that Acadia's P/E ratio stands at 81.46, which may seem high at first glance. However, when considering the company's growth prospects and the potential market for DAYBUE, this valuation could be justified. The PEG ratio of 0.64 for the last twelve months as of Q2 2024 suggests that the stock might be undervalued relative to its earnings growth potential.

For investors seeking a more comprehensive analysis, InvestingPro offers 11 additional tips for Acadia Pharmaceuticals, providing a deeper understanding of the company's financial health and market position.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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