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.

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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Adam Russell, ARPA-H's incoming acting deputy director

NI­H's new, in­de­pen­dent break­through drug ac­cel­er­a­tor ARPA-H gets its first em­ploy­ee

Despite the controversy of housing it in NIH, HHS Secretary Xavier Becerra on Wednesday afternoon formally announced the establishment of the Advanced Research Project Agency for Health (ARPA-H) as an independent entity within the NIH, as HHS had previously stipulated that “NIH may not subject ARPA-H to NIH policies.”

Becerra also announced the appointment of ARPA-H’s inaugural employee, Adam Russell, who will serve as acting deputy director.

Katrine Bosley (AP Photo/Mark Lennihan)

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London-based early-stage investor Advent Life Sciences announced Thursday that Bosley is joining the firm as venture partner. It’s also adding two general partners to the team: Dominic Schmidt, formerly of Syncona, will be in the UK; and Satish Jindal, most recently the CEO of investment fund BioMotiv, will be based in Boston, just like Bosley.

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LA biotech founder ar­rest­ed, charged in mur­der-for-hire scheme be­hind 2018 death

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ProFound Therapeutics founding team

Flag­ship's lat­est biotech could turn some of the thou­sands of new pro­teins it dis­cov­ered in­to ther­a­pies — and it has $75M to start

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Like other drug developers that have turned proteins into therapeutics (think insulin for diabetes), Flagship’s latest creation, ProFound Therapeutics, wants to tap into this new trove of proteins as part of its mission to treat indications ranging from rare diseases to cancer to immunological diseases.

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This time around, Lyri­ca's in­ven­tor is de­vel­op­ing his North­west­ern dis­cov­er­ies at his own biotech

Richard Silverman was left in the dark for the last five years of clinical development of the drug he discovered. The Northwestern University professor found out about the first approval of Lyrica, in the last few days of 2004, like most other people: in the newspaper.

What became one of Pfizer’s top-selling meds, at $5 billion in 2017 global sales before losing patent protection in 2019, started slipping out of his hands when Northwestern licensed it out to Parke-Davis, one of two biotechs that showed interest in developing the drug in the pre-email days, when the university’s two-person tech transfer team had to ship out letters to garner industry appetite.

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Michael Corbo, Pfizer CDO of inflammation & immunology

UP­DAT­ED: Plan­ning ahead for crowd­ed ul­cer­a­tive col­i­tis mar­ket, Pfiz­er spells out PhI­II da­ta on $6.7B Are­na drug

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Pivotal data from the ELEVATE program in ulcerative colitis — which consists of two Phase III trials, one lasting 52 weeks and the other just 12 weeks — illustrate an “encouraging balance of efficacy and safety,” according to Michael Corbo, chief development officer of inflammation & immunology at Pfizer. The company is presenting the results as a late breaker at Digestive Disease Week.

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David Ricks, Eli Lilly CEO (David Paul Morris/Bloomberg via Getty Images)

Eli Lil­ly set to in­vest $2.1B in home state man­u­fac­tur­ing boost

Eli Lilly is looking to expand its footprint in its home Hoosier State by making a major investment in manufacturing.

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The two new facilities will expand Lilly’s manufacturing network for active ingredients and new therapeutic modalities, including genetic medicines, according to a press release.

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Up­dat­ed: US sees spike in Paxlovid us­age as Mer­ck­'s mol­nupi­ravir and As­traZeneca's Evusheld are slow­er off the shelf

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