Rare neu­ro­log­i­cal dis­or­ders have long proved dif­fi­cult for drug de­vel­op­ers, with huge R&D bud­gets and com­mer­cial plans of­ten go­ing bust at the very last minute. Now, Aca­dia Phar­ma is tout­ing a win for its late-stage can­di­date in Rett syn­drome, which could help juice its com­peti­tors’ ef­forts as well.

Aca­dia’s trofine­tide hit its co-pri­ma­ry end­points in Phase III study in 187 girls and young women with the rare neu­ro­log­i­cal dis­ease Rett syn­drome, which im­pacts be­tween 6,000 and 9,000 US pa­tients and is of­ten mis­di­ag­nosed as autism, cere­bral pal­sy or non-spe­cif­ic de­vel­op­men­tal de­lay, the com­pa­ny said Tues­day.

The drug best­ed place­bo on two met­rics judged by a pa­tient’s care­giv­er and physi­cian, re­spec­tive­ly, called the Rett Syn­drome Be­hav­iour Ques­tion­naire and the Clin­i­cal Glob­al Im­pres­sion–Im­prove­ment (CGI-I) as­sess­ment. Both as­sess­ments de­ter­mined im­prove­ments in pa­tients’ symp­toms over the 12-week du­ra­tion of the study.

On the RS­BQ as­sess­ment, pa­tients’ scores dropped an av­er­age of 5.1 points ver­sus a 1.7-point de­crease in the con­trol (p=0.0175). Mean­while, the CGI-I test showed a 3.5-point change for pa­tients in the trofine­tide arm ver­sus 3.8 for place­bo (p=0.003).

Tak­ing home a late-stage win in Rett syn­drome is a good look for Aca­dia, and in­vestors cheered the news with the com­pa­ny’s stock $ACAD trad­ing at just above $20 ear­ly Tues­day, up rough­ly 5% from Mon­day’s clos­ing price.

Aca­dia now plans to take its da­ta in front of the FDA by the mid­dle of next year, with a pre-NDA meet­ing sched­uled for the first quar­ter, it said. Trofine­tide has re­ceived fast track des­ig­na­tion from the agency and would be el­i­gi­ble for pri­or­i­ty re­view.

Aca­dia is one of a group of drug­mak­ers look­ing to se­cure the first-ap­proved ther­a­py for Rett syn­drome in the US. The dis­ease is one of a suite of rare neu­ro­log­i­cal con­di­tions that has stumped drug de­vel­op­ers for years, but this late-stage win — and a po­ten­tial pos­i­tive re­view — could put some wind in the sails of Aca­dia’s com­peti­tors in this space.

Those play­ers in­clude Anavex, which al­so has a fast track des­ig­na­tion for its blar­came­sine, and gene ther­a­py start­up Al­cy­one, which launched this sum­mer, among oth­ers. But the field has al­so been ripe with fail­ure. In 2020, Newron Phar­ma dropped its own late-stage Rett pro­gram sari­zotan af­ter the drug flopped a Phase II/III tri­al.

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Novartis’ sickle cell drug, approved in 2019 and branded as Adakveo, has failed an ongoing Phase III, according to preliminary results.

The Swiss pharma giant unveiled early data from the ongoing STAND Phase III study on Friday, saying that crizanlizumab showed no statistically significant difference between the drug at two different dose levels compared to placebo in annualized rates of vaso-occlusive crises that lead to a healthcare visit over the first year since being randomized into the trial.

Merck’s blockbuster cancer treatment Keytruda has been handed another indication by the FDA.

The US regulator announced on Thursday that it has approved Keytruda to serve as an adjuvant treatment for non-small cell lung cancer (NSCLC), which is its fifth indication in NSCLC and 34th indication overall.

According to a Merck release, the approval is based on data from a Phase III trial, dubbed Keynote-091, which measured disease-free survival in patients who received chemotherapy following surgery. The data from Merck displayed that Keytruda cut down on the risk of disease recurrence or death by 27% versus placebo.

J&J and Legend Biotech’s next step in turning their CAR-T therapy Carvykti into a potential megablockbuster has succeeded, the companies said Friday.

Carvykti achieved the primary endpoint — progression-free survival — in an open-label Phase III study testing the treatment in second- to fourth-line multiple myeloma patients. The CARTITUDE-4 trial, for which there aren’t any hard data yet, represents the biggest development for Carvykti’s ability to compete with Bristol Myers Squibb’s Abecma since its approval last February.