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.

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.