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.

What’s Biohaven without its CGRP portfolio? That’s what CEO Vlad Coric is tasked with deciding as he maps out the new Biohaven post-Pfizer takeover.

Pfizer officially scooped up Biohaven’s CGRP assets on Monday, including blockbuster migraine drug Nurtec and the investigational zavegepant, for $11.6 billion. As a result, Coric spun the broader pipeline into an independent company on Tuesday — with the same R&D team behind Nurtec but about 1,000 fewer staffers and a renewed focus on neuroscience and rare disease.

There are some odd-looking purple creatures lurking around the halls of AstraZenca lately. The “Phil” character cutouts are purple, personified eosinophils with big buggy eyes and wide mouths, and they’re a part of AZ’s newest awareness effort to help people understand eosinophilic asthma.

The “Asthma Behaving Badly” characters aren’t only on the walls at AZ to show the new campaign to employees, however. The “Phils” are also showing up online on the campaign website, and in digital and social ads and posts on Facebook and Instagram.

As Leo Pharma aims to take on heavyweight champ Dupixent in atopic dermatitis, the company is launching “AD Days Around the World,” an awareness campaign documenting real patient stories across Europe.

The project, unveiled on Monday, spotlights four patients: Marjolaine, Laura, Julia and África from France, Italy, Germany and Spain, respectively, in short video clips on the challenges of living with AD, the most common form of eczema.

In response to a question on manufacturing scale at Legend Biotech’s R&D day yesterday, the company’s top exec said its partnership with Johnson & Johnson will be doubling its investment in its New Jersey manufacturing center and will be investing a total of $500 million.

With an eye on their BCMA-directed CAR-T therapy Carvykti (cilta-cel), approved in February as a fifth-line treatment for multiple myeloma, Legend CEO Ying Huang said that the ramp-up in production and the decision to manufacture its own lentiviral vectors — currently in shortage worldwide — means they won’t have to deal with that shortage.