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Msg  40637 of 42560  at  12/7/2021 7:14:10 AM  by




Acadia Pharma Shares Jump 19% After Positive Results For Rett Syndrome Treatment

6:33 am ET December 7, 2021 (Dow Jones) Print

By Kimberly Chin


Acadia Pharmaceuticals Inc.'s shares rose 19% to $23 in premarket trade Tuesday after the company reported positive top-line results from its advanced Phase 3 trial of Trofinetide in girls and young women between the ages of 5 and 20 years with Rett syndrome.

Trofinetide showed significant improvement in core symptoms and conditions over the placebo, Acadia said Monday. The company also saw improvement in communication and behavior.

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

"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 pediatrics in the division of neurology, pharmacology and special education at Vanderbilt University Medical Center.

Mr. Neul said the positive results can support a potential treatment for Rett syndrome, a rare and debilitating neurological disorder that occurs mostly in females following normal development for the first six months of life. It affects about one of every 10,000 to 15,000 female births world-wide and about 6,000 to 9,000 people in the U.S., the company said.

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Msg # Subject Author Recs Date Posted
40640 Re: ACADRhetts, Las Vegas cm4cmc 3 12/7/2021 9:38:28 AM

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