In an­oth­er CRISPR first, Penn re­searchers dose US can­cer pa­tients with gene-edit­ed T cell ther­a­py

CRISPR has been test­ed in US pa­tients for the first time — but per­haps not in the man­ner you imag­ined.

The Uni­ver­si­ty of Penn­syl­va­nia has led the way dos­ing two pa­tients with re­lapsed can­cers — one with mul­ti­ple myelo­ma and one with sar­co­ma — with a T cell ther­a­py in which the T cell re­cep­tor and PD-1 pro­tein are edit­ed out.

Us­man “Oz” Azam

Fund­ing for the tri­al came part­ly from Tmu­ni­ty, a biotech co-found­ed by Penn’s CAR-T vi­sion­ary Carl June and helmed by No­var­tis vet Us­man “Oz” Azam. Park­er In­sti­tute for Can­cer Im­munother­a­py, an ear­ly in­vestor in Tmu­ni­ty, is al­so bankrolling the tri­al. A Penn spokesper­son con­firmed the news, which was first re­port­ed by NPR.

The ba­sic idea here is not un­like CAR-T, ex­cept with dif­fer­ent el­e­ments and tech­nolo­gies: Take a pa­tient’s T cells, en­gi­neer it to ex­press NY-ESO-1 TCR, while elim­i­nat­ing TCR and PD-1 orig­i­nal­ly on the sur­face us­ing CRISPR.

A group of UCLA and UCSF sci­en­tists backed by the Park­er In­sti­tute has been study­ing new tech­niques to get new genes in­to T cells — not just elim­i­nat­ing them — with CRISPR.

That’s not the most straight­for­ward way of ap­ply­ing the gene edit­ing tech, which has en­ticed oth­er drug de­vel­op­ers with the po­ten­tial to el­e­gant­ly fix ge­net­ic er­rors lead­ing to dis­ease. But it does speak to the ver­sa­til­i­ty of CRISPR, now a pop­u­lar­ized tool that’s spawned up­starts rang­ing from di­ag­nos­tics to tar­get­ed bac­te­ria erad­i­ca­tion.

Carl June

Nev­er­the­less, the trio of CRISPR pi­o­neers as­so­ci­at­ed — Ed­i­tas Med­i­cines, In­tel­lia Ther­a­peu­tics and CRISPR Ther­a­peu­tics — re­main the ones to watch for first-in-hu­man test­ing. In fact, just two months ago CRISPR Ther­a­peu­tics dosed the first pa­tient with its Ver­tex-part­nered be­ta tha­lassemia drug. It is al­so plan­ning to treat a sick­le cell dis­ease pa­tient with the same drug in mid-2019 for a sep­a­rate tri­al.

Of course, that’s as­sum­ing we’re talk­ing out­side of Chi­na, where in­fu­sions of CRISPR-edit­ed T cells in can­cer pa­tients have been tak­ing place for more than a year. In a stun­ner, Chi­nese sci­en­tist Jiankui He claimed the birth of ba­by twins who were ge­net­i­cal­ly mod­i­fied as fe­tus­es, stir­ring up wide­spread con­dem­na­tion and soul search­ing in the sci­en­tif­ic com­mu­ni­ty.

“2019 is the year when the train­ing wheels come off and the world gets to see what CRISPR can re­al­ly do for the world in the most pos­i­tive sense,” Fy­o­dor Urnov, a gene-edit­ing sci­en­tist at the Al­tius In­sti­tute for Bio­med­ical Sci­ences in Seat­tle and the Uni­ver­si­ty of Cal­i­for­nia, Berke­ley, told NPR.

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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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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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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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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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.

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.

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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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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