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ACAD Seeks EU Nod for Rett Syndrome Drug, Outlines 2025 Pipeline Goals (Revised)
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Acadia Pharmaceuticals (ACAD - Free Report) announced that it has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) seeking approval for trofinetide to treat Rett syndrome in adults and pediatric patients aged two years and above.
An approval in the EU is expected in the first quarter of 2026.
If approved in the European Union, trofinetide will become the first and only approved therapy for Rett syndrome in the region. The MAA also marks ACAD’s first regulatory filing for a product in Europe.
Trofinetide is already approved in the United States under the trade name Daybue for treating Rett syndrome in adult and pediatric patients aged two years and older. Daybue was launched in the United States in April 2023 and is the first and only drug to be approved by the FDA for the given indication.
In the past year, shares of Acadia have plunged 41.6% compared with the industry’s decline of 14.8%.
Image Source: Zacks Investment Research
ACAD's MAA Based on the LAVENDER Study
The MAA for trofinetide in the EU was based on positive data from the phase III LAVENDER study.
The LAVENDER study evaluated the safety and efficacy of trofinetide in 187 female patients aged between five to 20 years with Rett syndrome against treatment with a placebo.
The study met both its co-primary endpoints, showing statistically significant improvements in treatment with Daybue against placebo, as measured by the change from baseline in the Rett Syndrome Behaviour Questionnaire (RSBQ) total score and the Clinical Global Impression-Improvement (CGI-I) scale score at week 12.
The RSBQ is a caregiver assessment that evaluates a range of symptoms of Rett syndrome.
The key secondary endpoint of the study, which measured the change from baseline to week 12 in the Communication and Symbolic Behavior Scales Development Profile Infant-Toddler Checklist–Social composite score (CSBS-DP-IT–Social), was also statistically significant versus placebo.
A potential approval and launch of trofinetide in the EU are likely to boost ACAD’s growth prospects.
ACAD's Other Pipeline Goals for 2025-2026
We note that Acadia’s first drug, Nuplazid (pimavanserin), is approved in the United States for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis.
In a separate press release, the company announced that it expects to generate more than $1 billion in combined net sales for Nuplazid and Daybue in 2025.
Acadia is evaluating its pipeline candidate, ACP-101, in the phase III COMPASS PWS study for the treatment of Prader-Willi Syndrome (PWS). Enrollment of the last patient in the study is likely to be completed in the fourth quarter of 2025, while top-line data from the same is expected in the first half of 2026.
Acadia is developing another pipeline candidate, ACP-204, in the phase II RADIANT study for treating Alzheimer’s disease psychosis. Enrollment of the last patient in the study is likely to be completed in the first quarter of 2026, with top-line data from the same expected in mid-2026.
The company also plans to initiate a phase II study on ACP-204 for a second indication – Lewy Body Dementia – in the third quarter of 2025.
In the past 60 days, estimates for Voyager Therapeutics’ loss per share have narrowed from $1.72 to $1.48 for 2025. In the past year, shares of VYGR have plunged 33%.
VYGR’s earnings beat estimates in each of the trailing four quarters, the average surprise being 120.87%.
In the past 60 days, estimates for CytomX Therapeutics’ loss per share have narrowed from 46 cents to 35 cents for 2025. In the past year, shares of CTMX have plunged 49.5%.
CTMX’s earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average surprise being 115.70%.
In the past 60 days, estimates for Castle Biosciences’ loss per share have narrowed from $1.88 to $1.84 for 2025. In the past year, shares of CSTL have surged 31.5%.
CSTL’s earnings beat estimates in each of the trailing four quarters, the average surprise being 172.72%.
(We are reissuing this article to correct a mistake. The original article, issued on January 15, 2025, should no longer be relied upon.)
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ACAD Seeks EU Nod for Rett Syndrome Drug, Outlines 2025 Pipeline Goals (Revised)
Acadia Pharmaceuticals (ACAD - Free Report) announced that it has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) seeking approval for trofinetide to treat Rett syndrome in adults and pediatric patients aged two years and above.
An approval in the EU is expected in the first quarter of 2026.
If approved in the European Union, trofinetide will become the first and only approved therapy for Rett syndrome in the region. The MAA also marks ACAD’s first regulatory filing for a product in Europe.
Trofinetide is already approved in the United States under the trade name Daybue for treating Rett syndrome in adult and pediatric patients aged two years and older. Daybue was launched in the United States in April 2023 and is the first and only drug to be approved by the FDA for the given indication.
In the past year, shares of Acadia have plunged 41.6% compared with the industry’s decline of 14.8%.
Image Source: Zacks Investment Research
ACAD's MAA Based on the LAVENDER Study
The MAA for trofinetide in the EU was based on positive data from the phase III LAVENDER study.
The LAVENDER study evaluated the safety and efficacy of trofinetide in 187 female patients aged between five to 20 years with Rett syndrome against treatment with a placebo.
The study met both its co-primary endpoints, showing statistically significant improvements in treatment with Daybue against placebo, as measured by the change from baseline in the Rett Syndrome Behaviour Questionnaire (RSBQ) total score and the Clinical Global Impression-Improvement (CGI-I) scale score at week 12.
The RSBQ is a caregiver assessment that evaluates a range of symptoms of Rett syndrome.
The key secondary endpoint of the study, which measured the change from baseline to week 12 in the Communication and Symbolic Behavior Scales Development Profile Infant-Toddler Checklist–Social composite score (CSBS-DP-IT–Social), was also statistically significant versus placebo.
A potential approval and launch of trofinetide in the EU are likely to boost ACAD’s growth prospects.
ACAD's Other Pipeline Goals for 2025-2026
We note that Acadia’s first drug, Nuplazid (pimavanserin), is approved in the United States for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis.
In a separate press release, the company announced that it expects to generate more than $1 billion in combined net sales for Nuplazid and Daybue in 2025.
Acadia is evaluating its pipeline candidate, ACP-101, in the phase III COMPASS PWS study for the treatment of Prader-Willi Syndrome (PWS). Enrollment of the last patient in the study is likely to be completed in the fourth quarter of 2025, while top-line data from the same is expected in the first half of 2026.
Acadia is developing another pipeline candidate, ACP-204, in the phase II RADIANT study for treating Alzheimer’s disease psychosis. Enrollment of the last patient in the study is likely to be completed in the first quarter of 2026, with top-line data from the same expected in mid-2026.
The company also plans to initiate a phase II study on ACP-204 for a second indication – Lewy Body Dementia – in the third quarter of 2025.
ACAD's Zacks Rank & Stocks to Consider
Acadia currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Voyager Therapeutics, Inc. (VYGR - Free Report) , CytomX Therapeutics, Inc. (CTMX - Free Report) and Castle Biosciences, Inc. (CSTL - Free Report) , each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for Voyager Therapeutics’ loss per share have narrowed from $1.72 to $1.48 for 2025. In the past year, shares of VYGR have plunged 33%.
VYGR’s earnings beat estimates in each of the trailing four quarters, the average surprise being 120.87%.
In the past 60 days, estimates for CytomX Therapeutics’ loss per share have narrowed from 46 cents to 35 cents for 2025. In the past year, shares of CTMX have plunged 49.5%.
CTMX’s earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average surprise being 115.70%.
In the past 60 days, estimates for Castle Biosciences’ loss per share have narrowed from $1.88 to $1.84 for 2025. In the past year, shares of CSTL have surged 31.5%.
CSTL’s earnings beat estimates in each of the trailing four quarters, the average surprise being 172.72%.
(We are reissuing this article to correct a mistake. The original article, issued on January 15, 2025, should no longer be relied upon.)