Newron's Mer­ck KGaA-in li­censed com­pound fails Rett Syn­drome study, shares wilt

Mu­ta­tions in a gene called MECP2 were iden­ti­fied as the cause of Rett Syn­drome in 1999 in­side a lab­o­ra­to­ry at the Bay­lor Col­lege of Med­i­cine — the same year, Ital­ian drug de­vel­op­er Newron Phar­ma­ceu­ti­cals was born. On Mon­day, the com­pa­ny’s ex­per­i­men­tal drug, sari­zotan, failed a Phase II/III study in pa­tients with the se­vere neu­rode­vel­op­men­tal dis­or­der that has no ap­proved ther­a­pies.

In-li­censed from Mer­ck KGaA, the com­pound is de­signed to mod­u­late the ac­tiv­i­ty of neu­ro­trans­mit­ters and was aban­doned by the Ger­man drug­mak­er in 2006 af­ter a pair of failed piv­otal stud­ies in Parkin­son’s dis­ease. Now, Newron is al­so ter­mi­nat­ing its de­vel­op­men­tal pro­gram in Rett syn­drome.

The tri­al test­ed two dos­es of sari­zotan (10 mg and 20 mg) against a place­bo in 129 pa­tients with the dis­ease that al­most ex­clu­sive­ly af­fects fe­males, and is char­ac­ter­ized by a loss of ac­quired fine and gross mo­tor skills as well as neu­ro­log­i­cal, cog­ni­tive and au­to­nom­ic dys­func­tion, which leads to loss of abil­i­ty to con­duct dai­ly life ac­tiv­i­ties, walk or com­mu­ni­cate.

The main goal of the study — to in­duce a re­duc­tion in episodes of ap­nea dur­ing wak­ing time com­pared with place­bo — was not met, nei­ther were any sec­ondary goals. De­tailed da­ta will be pre­sent­ed at a lat­er date, Newron said.

Ravi Anand, Newron

“The re­sults of this well de­signed and ex­e­cut­ed study, based on high­ly promis­ing da­ta from a ge­net­ic mod­el of Rett syn­drome in mice, in­di­cate the dif­fi­cul­ties in­her­ent in trans­lat­ing ef­fects in an­i­mal mod­els to hu­man clin­i­cal stud­ies,” the com­pa­ny’s CMO Ravi Anand said in a state­ment.

The com­pa­ny’s shares cratered more than 68% on the Swiss stock ex­change to CHF 1.98.

“This high-risk pro­gramme was a key fo­cus for the stock, with sari­zotan un­der­pin­ning c.70% of our CHF40/share PT (price tar­get),” Jef­feries an­a­lysts wrote in a note.

New York biotech Anavex is an­oth­er com­pa­ny eval­u­at­ing its lead ex­per­i­men­tal drug, blar­came­sine, in pa­tients with Rett Syn­drome — in Feb­ru­ary, the FDA grant­ed the drug fast track sta­tus for the in­di­ca­tion. Aca­dia Phar­ma­ceu­ti­cals, the mak­er of the Parkin­son’s dis­ease drug Nu­plazid, has an ex­per­i­men­tal Rett syn­drome drug in late-stage de­vel­op­ment. GW Phar­ma, the first-ever com­pa­ny to get a nat­ur­al cannabis-de­rived ther­a­py ap­proved by the FDA, al­so has a CBD drug-in-de­vel­op­ment for Rett, while the cat tran­quil­iz­er ke­t­a­mine is al­so be­ing eval­u­at­ed by an aca­d­e­m­ic group for the con­di­tion.

Newron, mean­while, is now left with two eggs in its bas­ket. It gets roy­al­ty in­come from its Parkin­son’s dis­ease drug, Xada­go, which is sold by its part­ners in dif­fer­ent parts of the world. The oth­er drug in the com­pa­ny’s pipeline, eve­namide, is be­ing de­vel­oped for schiz­o­phre­nia, al­though Covid-19 has de­layed late-stage en­abling tri­als.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,600+ biopharma pros reading Endpoints daily — and it's free.

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.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,600+ biopharma pros reading Endpoints daily — and it's free.