With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

On the hunt for a bet­ter AAV cap­sid for gene ther­a­py, Er­ic Kel­sic’s Dyno Ther­a­peu­tics has set it­self apart with its fo­cus on ma­chine learn­ing to help speed dis­cov­ery. Now, Japan­ese drug­mak­er Astel­las — fresh off a slate of gene ther­a­py burns — is tak­ing a bet on Dyno as it looks to the fu­ture.

Astel­las and Dyno will work to­geth­er as part of an R&D pact to de­vel­op next-gen AAV vec­tors for gene ther­a­py us­ing Dyno’s Cap­sidMap plat­form di­rect­ed at skele­tal and car­diac mus­cle, the com­pa­nies said Wednes­day. Un­der the terms of the deal, Dyno will de­sign AAV cap­sids for gene ther­a­py, while Astel­las will be re­spon­si­ble for con­duct­ing pre­clin­i­cal, clin­i­cal and com­mer­cial­iza­tion ac­tiv­i­ties for gene ther­a­py prod­uct can­di­dates us­ing the cap­sids.

Dyno will get $18 mil­lion up­front, and is el­i­gi­ble for po­ten­tial down­stream pay­ments worth $235 mil­lion per prod­uct and $1.6 bil­lion in to­tal.

“This was al­ways a part of our long term strat­e­gy and we do re­al­ize that it’s es­sen­tial to our long term goal — as we look across the land­scape, Dyno stood out to us,” SVP of gene ther­a­py re­search and tech­ni­cal op­er­a­tions Math­ew Pletch­er said in a call with End­points News.

Cap­sidMap us­es in vi­vo ex­per­i­men­tal da­ta and ma­chine learn­ing to cre­ate new AAV cap­sids, which are the cell-tar­get­ing pro­tein shells of vi­ral vec­tors that are de­signed to op­ti­mize tis­sue tar­get­ing and im­mune-evad­ing prop­er­ties. Cap­sidMap can si­mul­ta­ne­ous­ly de­liv­er across mul­ti­ple or­gans, Dyno says.

The com­pa­ny spun out of George Church’s lab three years ago, and this year raised $100 mil­lion in its Se­ries A fundrais­ing round. Its fo­cus on ma­chine learn­ing, Dyno CEO Er­ic Kel­sic says, sets it apart from oth­ers in the gene ther­a­py space. With the prod­uct, comes a huge amount of da­ta sur­round­ing in vi­vo da­ta, which is rel­e­vant for im­prov­ing the per­for­mance of Cap­sidMap.

“In the past, (com­pa­nies have) tak­en a lot­tery tick­et ap­proach: buy a lot of tick­ets, and see what works, but chance of suc­cess is pret­ty low,” Kel­sic told End­points. “But now, we can learn a lot about the pro­tein fit­ness land­scape … and how do you use that to make a bet­ter cap­sid?”

The Dyno deal comes as Astel­las looks to turn the page on its ac­qui­si­tion of gene ther­a­py spe­cial­ist Au­dentes in 2019, as its lead pro­gram has run in­to a se­ries of se­ri­ous safe­ty is­sues in the clin­ic.

Less than a year af­ter Astel­las bought out Au­dentes for $3 bil­lion, that pro­gram was roiled by a pa­tient death in May 2020. Two ad­di­tion­al pa­tients in the study had died by Au­gust, lead­ing to a mod­i­fied tri­al for the drug, dubbed AT132. At the time, no pa­tients tak­ing the low­er dose had de­vel­oped any se­ri­ous side ef­fects. In Sep­tem­ber, Astel­las an­nounced a fourth boy had died in that tri­al.

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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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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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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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.

FDA slams door to piv­otal tri­al for bub­ble boy dis­ease gene ther­a­py as Mus­tang Bio runs in­to an­oth­er hold

Mustang Bio is in familiar territory, but that isn’t a place it necessarily wants to be.

The FDA has placed a hold on Mustang Bio’s pivitol trial for its gene therapy to treat patients with bubble boy disease, citing issues surrounding chemistry, manufacturing and controls clearance. It’s the second hold due to CMC issues the company has received in roughly 18 months.

An investigational new drug application was submitted in December 2021. If granted an IND, a Phase II study will then assess safety, tolerability and efficacy of MB-207. If approved by the FDA, the therapy would one day be eligible for a rare pediatric disease voucher.

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.

Joaquin Duato, J&J CEO (Photo by Charles Sykes/Invision/AP)

New J&J CEO Joaquin Du­a­to promis­es an ag­gres­sive M&A hunt in quest to grow phar­ma sales

Joaquin Duato stepped away from the sideline and directly into the spotlight on Tuesday, delivering his first quarterly review for J&J as its newly-tapped CEO after an 11-year run in senior posts. And he had some mixed financial news to deliver today while laying claim to a string of blockbuster drugs in the making and outlining an appetite for small and medium-sized M&A deals.

Duato also didn’t exactly shun large buyouts when asked about the future of the company’s medtech business — where they look to be in either the top or number 2 position in every segment they’re in — even though the bar for getting those deals done is so much higher.

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Amgen's Twitter campaign #DearAsthma inspired thousands of people to express struggles and frustrations with the disease

Am­gen’s #Dear­Asth­ma spon­sored tweet lands big on game day, spark­ing thou­sands to re­spond

Amgen wanted to know how people with asthma really felt about daily life with the disease. So it bought a promoted tweet on Twitter noting the not-so-simple realities of life with asthma and ended the post with a #DearAsthma hashtag, a megaphone emoji and a re-tweet button.

That was just over one week ago and the responses haven’t stopped. More than 7,000 posts so far on Twitter replied to #DearAsthma to detail struggles of daily life, expressing humor, frustration and sometimes anger. More than a few f-bombs have been typed or gif-ed in reply to communicate just how much many people “hate” the disease.