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Acadia Pharma (ACAD) up 17% on test trial results

By Daniel Tyson

13:52, 7 December 2021

Colorful pills falling out of a bottle
Acadia Pharma latest clinical trial successful - Photo: Shutterstock

Acadia Pharmaceuticals rose 17% overnight after the company posted positive results from its Phase 3 trial of Trofinetide, a treatment for Rett Syndrome.

Rett Syndrome is a genetic disorder primarily affecting brain development in girls.

In premarket trading Tuesday, the stock jumped to $23 from $19.40 at the closing bell Monday.  The company’s 52-week high was recorded on 21 December 2020 of $57.34. Its low was $15.68 on 14 September 2021.

Significant improvements

During the trials, patients using Trofinetide showed significant improvements in core symptoms and conditions over those administered a placebo. Patients included girls and young women between the ages of 5 and 20 years with Rett syndrome, the company said Monday in a press release.

The studies also showed improvement in communication and behaviour. 


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Trofinetide is a novel synthetic analogue of the amino-terminal tripeptide of an important growth hormone. It has the potential to reduce neuroinflammation and support synaptic function, Acadia said.

Core symptoms

 “Patients reported improvements in core symptoms, like being able to respond to a choice when asked by their parents or experiencing more freedom from the repetitive hand movements that create obstacles in other areas of their lives,” said Jeffrey Neul, a professor of paediatrics in the division of neurology, pharmacology and special education at Vanderbilt University Medical Center and a study investigator.

Rett Syndrome affects about one out of every 10,000 to 15,000 female births worldwide, the company said.

Acadia Pharmaceuticals focuses on the development and commercialisation of medicines to address unmet medical needs in central nervous system disorders, according to the company’s website.

Read more: IPO imminent for Phase 1 pharma company Nuvectis

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