Iain McGill, Quell CEO

Eu­ro­pean in­vestors pour $156M to beat Blue­stone, Third Rock and RA Cap­i­tal in multi­bil­lion-dol­lar race to the clin­ic

Amid bur­geon­ing ef­forts to cre­ate a new type of cell ther­a­py out of reg­u­la­to­ry T cells — whether by chan­nel­ing or block­ing their im­muno­sup­pres­sive pow­er — Quell Ther­a­peu­tics wants to shoot for a first.

If every­thing goes well, the Syn­cona-backed biotech will be in the clin­ic ear­ly next year, mark­ing what it calls the his­toric feat of dos­ing a pa­tient with a CAR-Treg with mul­ti­ple edit­ed genes.

Hav­ing shied away from the spot­light since clos­ing a mod­est Se­ries A two years ago, Quell is now loud and clear about those next steps with $156 mil­lion in Se­ries B cash.

“This is the round where peo­ple sit up and take no­tice,” was how CEO Iain McGill put it to End­points News.

The megaround is just part of it — and like­ly a small part at that. With­in the past year, mul­ti­ple out­fits from Jef­frey Blue­stone’s Sono­ma to RA Cap­i­tal-backed Gen­tiBio to Third Rock’s Aba­ta Ther­a­peu­tics have each bagged im­pres­sive rais­es to fund their re­spec­tive ap­proach­es, and that’s not to men­tion Mozart, Egle, TRex Bio and oth­ers who are all promis­ing to gen­er­ate po­tent, sta­ble, safe and durable cells that can tamp ex­ces­sive au­toim­mune or in­flam­ma­to­ry re­ac­tions.

But come 2022, Quell reck­ons it will be in the ex­clu­sive club of clin­i­cal-stage com­pa­nies de­vel­op­ing a CAR-Treg. Sang­amo is the on­ly oth­er play­er it sees there for now.

The com­pa­ny al­so be­lieves that its plat­form, based on sci­ence out of King’s Col­lege Lon­don, Uni­ver­si­ty Col­lege Lon­don and Han­nover Med­ical School, has picked out de­sign com­po­nents that make its Tregs even stronger.

Tregs, McGill ex­plained, are a dou­ble-edged sword: They can flip from a sup­pres­sor phe­no­type in­to an at­tack­ing, ef­fec­tor phe­no­type. In a worst case sce­nario, this change can lead not on­ly to lack of ef­fi­ca­cy but a safe­ty con­cern where­by the CAR-Tregs turn out to de­stroy the very tis­sue they were in­tend­ed to pro­tect.

To pre­vent that, Quell trans­duces the cells with mul­ti­ple copies of FOXP3, which McGill called the “mas­ter tran­scrip­tion fac­tor” for a Treg.

“By hard­wiring the cells with mul­ti­ple copies of FOXP3, you sort of lock the cells in that phe­no­typ­ic sup­pres­sion mode,” he said. “So you tran­scrip­tion­al­ly lock them as a Treg. And that gives them sta­bil­i­ty. What we found when we start­ed do­ing that, be­cause in ad­di­tion to giv­ing the cells phe­no­typ­ic sta­bil­i­ty, it al­so in­creased their sup­pres­sive po­ten­cy.”

The first pro­gram, which is de­signed to wean pa­tients off im­muno­sup­pres­sants fol­low­ing liv­er trans­plants, knocks in three ad­di­tion­al genes to “re­set the me­chan­ics of re­jec­tion.” The UK has cleared a clin­i­cal tri­al and Quell is on track to re­cruit its first pa­tient by the end of this year.

Be­hind that it has lined up two pro­grams in dif­fer­ent di­rec­tions, one aimed at ALS and the oth­er for type 1 di­a­betes.

“Here, we ex­pect ex­e­cu­tion to be a con­tin­u­ing, fo­cus­ing chal­lenge and a key dif­fer­en­tia­tor for suc­cess,” said Rachel Mears, part­ner at Jeito Cap­i­tal, which co-led the round along­side Ridge­back Cap­i­tal In­vest­ments, SV Health In­vestors and Fi­deli­ty Man­age­ment & Re­search Com­pa­ny.

Mears is join­ing the board of di­rec­tors along­side Ridge­back man­ag­ing di­rec­tor Jef­frey Long-McGie and SV man­ag­ing part­ner Houman Ashrafi­an.

The round will al­so help fund the build out of man­u­fac­tur­ing ca­pa­bil­i­ties, which McGill not­ed is sim­i­lar to tra­di­tion­al CAR-T but dif­fer­ent in mean­ing­ful ways. For in­stance, the pa­tients they’re look­ing to treat are dif­fer­ent from the very sick can­cer pa­tients seek­ing au­tol­o­gous cell ther­a­pies, for whom vein-to-vein time is a life-or-death mat­ter.

Quell, on the oth­er hand, is work­ing with sta­ble pa­tients, such as those be­tween one and five years post-trans­plant:

We don’t have to in­ter­vene im­me­di­ate­ly for each of these pa­tients, which means that we can ware­house pa­tients, we can se­quence pa­tients, we can bring them in­to our man­u­fac­tur­ing cen­ter in a way that suits the sched­ul­ing of man­u­fac­tur­ing, rather than hav­ing to or­ga­nize man­u­fac­tur­ing around pro­vi­sion of the leuka­phere­sis to man­u­fac­ture a prod­uct, which is ex­act­ly what you have to do in on­col­o­gy. So we can use the fa­cil­i­ty in a more or­dered and struc­tured process, which I think brings great ben­e­fit in terms of sta­bil­i­ty and ro­bust­ness of the man­u­fac­tur­ing process go­ing for­ward.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 129,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 129,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 129,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Brian Thomas, Metagenomi CEO

Gen 2: Berke­ley spin­out lands $175M megaround to keep it on the cut­ting edge of the boom­ing gene-edit­ing field

The big bucks keep pumping into the gene-editing field.

This morning Metagenomi, allied with one of the biggest names in the mRNA field with a company DNA that includes the ubiquitous Jennifer Doudna, is showing off a $175 million B round that will pay for a rapid swelling of its staff in pursuit of some of the cutting-edge tech that keeps this field in the spotlight. And they’re aligning themselves with some major industry players with an eye on the clinic while getting behind some startups to help expand the work into new fields.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 129,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 129,600+ biopharma pros reading Endpoints daily — and it's free.