Acadia to Move Experimental Pediatric Rett Syndrome Drug to Phase 3

Xconomy San Diego — 

Acadia Pharmaceuticals said it plans to advance a drug it licensed last year from Australia’s Neuren Pharmaceuticals into a late-stage trial following promising Phase 2 results of the potential Rett syndrome treatment.

San Diego-based Acadia (NASDAQ: ACAD) announced in August that it had licensed the drug, trofinetide, for development and commercialization in North America as a treatment for Rett syndrome and other indications, including Fragile X syndrome. Terms of the deal included a $10 million upfront payment to Neuren, up to $455 million more in potential milestone payments, and “double-digit percentage” royalties on any net sales.

The results of the trial in Rett syndrome, conducted by Neuren, were published Wednesday in Neurology, the American Academy of Neurology’s medical journal.

Rett syndrome is a neurological disorder typically recognized in infancy after about six months of apparently normally development. It almost exclusively affects girls. The resulting problems in brain function can impair the ability to speak, eat, and even breathe comfortably. According to the National Institutes of Health, the condition is estimated to affect between one in 10,000 to 22,000 girls in the US. While it’s a genetic disease, the mutation that causes Rett syndrome typically occurs randomly, not as a result of inheritance.

The study evaluated the effect of the drug in 82 girls age 5 to 15 with the disease, at three dose levels. At the highest dose tested, the drug appeared to reduce symptoms, including social communication deficits, anxiety-like behavior, and mood dysregulation. The drug was well-tolerated at all dose levels.

Acadia said it plans to start a Phase 3 trial, which will evaluate the drug and a placebo in about 180 girls and women age 5 to 20, beginning in the second half of this year.

About three years ago, the FDA approved Acadia’s first commercialized drug, pimavanserin (Nuplazid), for hallucinations and delusions associated with Parkinson’s disease psychosis. Revenue from product sales in 2018 totaled $223.8 million, up nearly 80 percent compared to the year prior.

The company reported a net loss of about $245.3 million in 2018, about 15 percent less than its loss in 2017, according to its annual report. Acadia is headed by CEO Stephen Davis (pictured), who joined the company in 2014 as executive vice president, chief financial officer, and chief business officer, and was named chief executive in 2015.