ACADIA Pharmaceuticals Inc. ( ACAD Quick Quote ACAD - Free Report) announced positive top-line data from the pivotal phase III Lavender study evaluating the safety and efficacy of its pipeline candidate, trofinetide, for the treatment of Rett syndrome, a rare neurological disorder for girls aged between five and 20 years.
The study met its co-primary endpoints by demonstrating statistically significant improvement over placebo in the Rett syndrome behavior questionnaire and the clinical global impression of progress. Also, treatment with trofinetide led to statistically significant improvement versus placebo in CSBS-DP-IT–Social, caregiver scale of ability to communicate – a key secondary endpoint.
The company is planning to hold a pre-NDA meeting with the FDA in the first quarter of 2022 and expects to file a new drug application or NDA for trofinetide to treat Rett syndrome around mid-2022.
Shares of ACADIA were up 3.3% in after-hours trading on Monday following the announcement of the news. The stock was also up in pre-market trading on Tuesday. ACADIA's stock has plunged 63.7% so far this year compared with the
industry’s decline of 21.7%. Image Source: Zacks Investment Research
We note that, in 2018, ACADIA entered into a license agreement with Australian biopharmaceutical company, Neuren Pharmaceuticals Limited, and obtained exclusive North American rights to develop and commercialize trofinetide for Rett syndrome and other indications
The FDA has granted a Rare Pediatric Disease designation to trofinetide for the treatment of Rett syndrome. Currently, there is no approved medicine for the given indication.
If successfully developed and upon potential approval, trofinetide can lend a significant boost to the company and lower its sole dependence on Nuplazid for revenues.
We remind investors that, presently, ACADIA’s top line solely comprises sales of its only marketed drug, Nuplazid (pimavanserin), in the United States. The drug is approved for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis.
Several additional studies on pimavanserin targeting different neurological and psychiatric disorders are currently ongoing.
However, competition looms large on ACADIA as many companies are developing treatments to address various central nervous system (“CNS”) disorders.
Axsome Therapeutics ( AXSM Quick Quote AXSM - Free Report) is developing its pipeline candidates AXS-05, AXS-07 and several others — for multiple CNS indications. —
Axsome has submitted an NDA for AXS-05 to treat major depressive disorder. Also, a similar filing for AXS-07 as an acute treatment of migraine is under review, with a decision expected in April 2022.
Zacks Rank & Stocks to Consider
ACADIA currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the biotech sector include
Sarepta Therapeutics, Inc. ( SRPT Quick Quote SRPT - Free Report) and Editas Medicine, Inc. ( EDIT Quick Quote EDIT - Free Report) , both sporting a Zacks Rank #2 (Buy) at present. You can see . the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here
Sarepta Therapeutics’ loss per share estimates have narrowed 31.3% for 2021 and 26% for 2022 over the past 60 days.
Earnings of Sarepta Therapeutics have surpassed estimates in two of the trailing four quarters and missed the same on the other two occasions.
Editas Medicine’s loss per share estimates have narrowed 11.2% for 2021 and 4.6% for 2022 over the past 60 days.
Editas Medicine’s earnings have surpassed estimates in two of the trailing four quarters and missed the same on the other two occasions.