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

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.
In 2020, The National Cancer Institute estimated about 1.8 million new cases of cancer diagnosed in the United States, while the costs associated with treatment therapies continued to escalate. Given the current legislative climate on drug pricing, it’s never been more important to look at the evolution of drug pricing globally and control concerns of sustainable and affordable treatments in oncology.
Awash in cash from its Covid-19 vaccine and leaner than ever, Pfizer has pick of the litter in biotech for deals set to shape its portfolio for the next 10 to 15 years. Gene editing has a ton of momentum right now, and Pfizer is biting in a big way on a buzzy upstart working on “CRISPR 2.0.”
Pfizer will pay $300 million upfront and up to $1.05 billion in additional downstream milestones to Beam Therapeutics as part of a four-year research collaboration to develop base editors for a trio of therapeutic targets including the liver, CNS and muscle, the partners said Monday.
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Last to the game in the current generation of CAR-T cell therapies, Bristol Myers Squibb has wasted no time trying to challenge its closest rivals’ head start while keeping an eye to the future. On the latter front, the drug giant has now signed a deal with a small next-gen cell therapy player to get a leg up.
Bristol Myers will pay $100 million in upfront cash and make an additional $50 million equity investment in Century Therapeutics for access to up to four off-the-shelf engineered cell therapies derived from donor stem cells, the partners said Monday.
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Bristol Myers Squibb has loaded up plenty of ammunition for M&A and other types of deals over the next two years — even if the right targets aren’t always easy to come by.
Presenting at the virtual JP Morgan Healthcare Conference, the pharma giant revealed that it expects to have around $45 billion to $50 billion in free cash flow between 2022 and 2024, which it plans to spend on a “balanced capital allocation strategy, prioritizing business development and returning cash to shareholders” through dividend and share repurchase programs.
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Less than 3 months after Takeda scooped up GammaDelta in a bid to expand its work on next-gen cell therapies, the pharma giant is back — starting off JP Morgan week with another acquisition in the field.
This time, the buyout target is Adaptate Biotherapeutics and its preclinical work on an antibody-based γδ T cell engager platform, a spinout from GammaDelta that Takeda believes gives them a shot at leading the pack in cell therapies.
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German drug giant Bayer has looked to reinvent itself in recent years, moving on from its past as a primarily consumer health brand into one built around next-gen therapies. Now, Bayer is taking a flyer on one of the children of CRISPR maven Jennifer Doudna’s hallowed lab working on tiny versions of the gene editing tech.
Bayer will pay $40 million upfront and more than $1 billion in potential downstream milestones for up to five in vivo gene editing candidates from Mammoth Biosciences, a Doudna lab spinout developing ultra-small enzymes for easier packaging and delivery into cells, the partners said Monday.
Drug R&D has for years had an abysmal track record of success, with the vast majority of drug candidates never making it to market. The promise of AI to shorten the discovery time for new drugs and up their chances of success has more big drugmakers buying in — and Sanofi is the latest.
Sanofi will pay $100 million upfront with a potential $5.2 billion in downstream milestones for access to up to 15 small molecule drugs from Exscientia, a red-hot UK deep learning company at the forefront of the so-called “AI-discovered” drug R&D movement, the partners said Friday.
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Since coming into a massive windfall with its Covid-19 vaccines, Moderna has been looking for ways to expand its mRNA technologies into other promising areas. On Monday, the biotech took its next step toward that goal.
Moderna is partnering with Carisma Therapeutics on a huge slate of “CAR-M” programs to treat cancer, the companies announced Monday morning, promising $45 million upfront and $35 million in equity. Though the amount of milestones was not specified, the biotechs noted Moderna has the option to nominate up to 12 targets for development.
Ever since Biogen’s controversial Alzheimer’s drug Aduhelm won an accelerated approval in June, despite scant evidence of clinical benefit, the country has turned its attention to who might use the drug and who would pay for it.
So far, not too many have done either, with Biogen hauling in just $300,000 in early sales, and insurers still waiting to see what CMS will do with its national coverage decision, or NCD. The NCD will serve as the basis for how insurers decide to cover Aduhelm. The decision will provide CMS’ thinking behind whether Aduhelm and other potential amyloid-targeted drugs either are or are not “reasonable and necessary” for the treatment of Alzheimer’s disease.
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