Steve Davis, Acadia CEO

In $900M+ deal, rare dis­ease biotechs team up on RNA ther­a­pies for CNS de­vel­op­men­tal dis­or­ders

Two biotechs that have built their rep­u­ta­tions in rare dis­ease re­search signed a new col­lab­o­ra­tion Mon­day morn­ing aim­ing to de­vel­op three new RNA-based ther­a­pies. And the deal could be worth near­ly $1 bil­lion in biobucks if every mile­stone is met.

Aca­dia Phar­ma­ceu­ti­cals and Stoke Ther­a­peu­tics will team up on the treat­ments in rare ge­net­ic neu­rode­vel­op­men­tal dis­eases of the CNS, the com­pa­nies an­nounced. Up first will be the re­cent­ly dis­cov­ered SYN­GAP1 syn­drome, fol­lowed by Rett syn­drome (MECP2) and an undis­closed tar­get the biotechs say is of mu­tu­al in­ter­est.

“Com­bin­ing Stoke’s ca­pa­bil­i­ties with Aca­dia’s ex­ten­sive ex­per­tise in neu­ro­science drug de­vel­op­ment and com­mer­cial­iza­tion en­ables us to push hard­er and faster in ex­plor­ing some of the new fron­tiers in rare cen­tral ner­vous sys­tem dis­or­ders,” Aca­dia CEO Steve Davis said in a state­ment.

Stoke will net a $60 mil­lion up­front pay­ment from Aca­dia to kick things off and is el­i­gi­ble to re­ceive up to $907 mil­lion in mile­stones.

The com­pa­nies will split R&D costs and prof­its even­ly for the SYN­GAP1 can­di­date, ac­cord­ing to the press re­lease. Stoke will take the lead in pre­clin­i­cal de­vel­op­ment for the oth­er two pro­grams — ef­forts that will be “ful­ly fund­ed” by Aca­dia — af­ter which Aca­dia will head up clin­i­cal stud­ies and com­mer­cial­iza­tion.

SYN­GAP1 syn­drome was first iden­ti­fied in 2009, the com­pa­nies say, and can be char­ac­ter­ized by de­vel­op­men­tal de­lay or in­tel­lec­tu­al dis­abil­i­ty, gen­er­al­ized epilep­sy and autism spec­trum dis­or­der, among oth­er things, pre­sent­ing in ear­ly child­hood. The con­di­tion is caused by mu­ta­tions in the SYN­GAP1 gene and is said to ac­count for “1% to 2%” of all in­tel­lec­tu­al dis­abil­i­ty cas­es.

Rett syn­drome, mean­while, typ­i­cal­ly oc­curs in young girls due to a mu­ta­tion of a gene on the X chro­mo­some. It’s a con­di­tion in which Aca­dia is work­ing on an­oth­er pro­gram, hav­ing re­vealed pos­i­tive topline da­ta in a Phase III study last month show­ing the can­di­date, known as trofine­tide, beat place­bo on two co-pri­ma­ry end­points.

Though many col­lab­o­ra­tions in the space are signed be­tween a Big Phar­ma com­pa­ny and a small­er biotech, Mon­day’s deal comes from two biotechs with his­to­ries of de­vel­op­ing rare dis­ease and CNS ther­a­pies. Aca­dia’s Nu­plazid was the first drug ap­proved to treat hal­lu­ci­na­tions and psy­chosis re­lat­ed to Parkin­son’s dis­ease, while Stoke re­vealed the first clin­i­cal da­ta for its lead Dravet syn­drome pro­gram last month.

Aca­dia has run in­to some trou­ble more re­cent­ly in try­ing to ex­pand Nu­plazid’s in­di­ca­tions, how­ev­er, as the FDA in April is­sued a CRL for de­men­tia-re­lat­ed psy­chosis. The biotech said a few weeks ago it’s plan­ning to re­sub­mit the sN­DA with a fo­cus on Alzheimer’s in­duced psy­chosis some­time this quar­ter.

Alexander Lefterov/Endpoints News

A new can­cer im­munother­a­py brings cau­tious hope for a field long await­ing the next big break­through

Bob Seibert sat silent across from his daughter at their favorite Spanish restaurant near his home in Charleston County, SC, their paella growing cold as he read through all the places in his body doctors found tumors.

He had texted his wife, a pediatric intensive care nurse, when he got the alert that his online chart was ready. Although he saw immediately it was bad, many of the terms — peritoneal, right iliac — were inscrutable. But she was five hours downstate, at a loud group dinner the night before another daughter’s cheer competition.

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In a set­back, FDA or­ders Gilead to hit the brakes on their late-stage, $5B can­cer play

Gilead’s $5 billion drug magrolimab has run into a serious setback.

The FDA ordered Gilead to halt enrollment on their studies of the drug in combination with azacitidine after investigators reports revealed an “apparent imbalance” in the suspected unexpected serious adverse reactions between study arms. And the halt is raising questions about Gilead’s plans for a quick pitch to regulators.

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Graphic: Alexander Lefterov for Endpoints News

Small biotechs with big drug am­bi­tions threat­en to up­end the tra­di­tion­al drug launch play­book

Of the countless decisions Vlad Coric had to make as Biohaven’s CEO over the past seven years, there was one that felt particularly nerve-wracking: Instead of selling to a Big Pharma, the company decided it would commercialize its migraine drug itself.

“I remember some investors yelling and pounding on the table like, you can’t do this. What are you thinking? You’re going to get crushed by AbbVie,” he recalled.

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Mar­ket­ingRx roundup: Pfiz­er de­buts Pre­vnar 20 TV ads; Lil­ly gets first FDA 2022 pro­mo slap down let­ter

Pfizer debuted its first TV ad for its Prevnar 20 next-generation pneumococcal pneumonia vaccine. In the 60-second spot, several people (actor portrayals) with their ages listed as 65 or older are shown walking into a clinic as they turn to say they’re getting vaccinated with Prevnar 20 because they’re at risk.

The update to Pfizer’s blockbuster Prevnar 13 vaccine was approved in June, and as its name suggests is a vaccine for 20 serotypes — the original 13 plus seven more that cause pneumococcal disease. Pfizer used to spend heavily on TV ads to promote Prevnar 13 in 2018 and 2019 but cut back its TV budgets in the past two fall and winter seasonal spending cycles. Prevnar had been Pfizer’s top-selling drug, notching sales of just under $6 billion in 2020, and was the world’s top-selling vaccine before the Covid-19 vaccines came to market last year.

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Roy Baynes, Merck

FDA bats back Mer­ck’s ‘pipeline in a prod­uct,’ de­mands more ef­fi­ca­cy da­ta

Despite some heavy blowback from analysts, Merck execs maintained an upbeat attitude about the market potential of its chronic cough drug gefapixant. But the confidence may be fading somewhat today as Merck puts out news that the FDA is handing back its application with a CRL.

Dubbed by Merck’s development chief Roy Baynes as a “pipeline in a product” with a variety of potential uses, Merck had fielded positive late-stage data demonstrating the drug’s ability to combat chronic cough. The drug dramatically reduced chronic cough in Phase III, but so did placebo, leaving Merck’s research team with a marginal success on the p-value side of the equation.

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Albert Bourla (Photo by Steven Ferdman/Getty Images)

UP­DAT­ED: Pfiz­er fields a CRL for a $295M rare dis­ease play, giv­ing ri­val a big head start

Pfizer won’t be adding a new rare disease drug to the franchise club — for now, anyway.

The pharma giant put out word that their FDA application for the growth hormone therapy somatrogon got the regulatory heave-ho, though they didn’t even hint at a reason for the CRL. Following standard operating procedure, Pfizer said in a terse missive that they would be working with regulators on a followup.

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Michael Egholm, Standard BioTools president and CEO (IsoPlexis)

Eli Cas­din co-leads $250M in­fu­sion in­to mi­croflu­idics play­er that land­ed NIH fund­ing for Covid-19 test­ing

In about 17 months, Fluidigm has gone from working with sharks to Vikings.

The South San Francisco-based company, which landed NIH money in a Shark Tank-style program for Covid-19 testing, announced that it will take on an investment worth $250 million from Casdin Capital and Viking Global Investors. It will also rebrand, and call itself Standard BioTools. The investment will help the company focus on the highest growth areas of discovery and development and expand its CRO and CMO service providers. Right now, the company’s customer reach is limited to basic research, it said.

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Florida Gov. Ron DeSantis (AP Photo/Wilfredo Lee, File)

Opin­ion: Flori­da is so mAb crazy, Ron De­San­tis wants to use mAbs that don't work

Florida Gov. Ron DeSantis is trying so hard to politicize the FDA and demonize the federal government that he entered into an alternate universe on Monday evening in describing a recent FDA action to restrict the use of two monoclonal antibody, or mAb, treatments for Covid-19 that don’t work against Omicron.

Without further ado, let’s break down his statement from last night, line by line, adjective by adjective.

Not cheap­er by the dozen: Bris­tol My­ers be­comes the 12th phar­ma com­pa­ny to re­strict 340B sales

Bristol Myers Squibb recently joined 11 of its peer pharma companies in limiting how many contract pharmacies can access certain drugs discounted by a federal program known as 340B.

Bristol Myers is just the latest in a series of high-profile pharma companies moving in their own direction as the Biden administration’s Health Resources and Services Administration struggles to rein in the drug discount program for the neediest Americans.