Steve Davis, Acadia CEO

Aca­dia touts late-stage win for its Rett syn­drome drug, edg­ing out a lead against its com­peti­tors

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.

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

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' ap­proved sick­le cell dis­ease drug fails to beat place­bo in PhI­II

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.

Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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FDA ap­proves an­oth­er in­di­ca­tion for Keytru­da, this time in the ad­ju­vant NSCLC set­ting

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.

Ying Huang, Legend CEO

J&J, Leg­end say Carvyk­ti beat stan­dard ther­a­py in ear­li­er-line blood can­cer

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.

Dutch biotech starts liq­ui­da­tion af­ter end­ing PhI­II in GVHD

A 13-year-old Dutch biotech is going through a liquidation process after an unexpected end to its Phase III trial testing whether its combination of two monoclonal antibodies was superior to Incyte’s Jakafi.

Xenikos had hoped to prove its investigational therapy, named T-Guard, was better than Jakafi at garnering a complete response in patients experiencing life-threatening complications in which new cells from a hematopoietic stem cell transplant begin to fight the body. Jakafi was approved for the indication, steroid-refractory acute graft-versus-host disease, in May 2019.

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Eliot Forster, F-star CEO (Rachel Kiki for Endpoints News)

F-star gets down to the wire with $161M sale to Chi­nese buy­er as na­tion­al se­cu­ri­ty con­cerns linger

With the clock ticking on F-star Therapeutics’ takeover by a Chinese buyer, the companies are still scrambling to remove a hold on the deal from the US government’s Committee on Foreign Investment in the United States.

F-star and invoX Pharma said they are “actively negotiating” with CFIUS “about the terms of a mitigation agreement to address CFIUS’s concerns regarding potential national security risks posed by the transaction.”

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