CEO Matthew Kane (Precision BioSciences)

In an ap­par­ent R&D about-face, Eli Lil­ly part­ners with Pre­ci­sion Bio­Sciences on genome edit­ing in a deal worth up to near­ly $2.7B

As a large multi­na­tion­al cor­po­ra­tion, Eli Lil­ly has their hands in bound­less projects, from can­cer and im­muno-on­col­o­gy to di­a­betes, pso­ri­a­sis and Crohn’s dis­ease. But Fri­day they sig­naled a shift in their R&D fo­cus to­ward genome edit­ing, leap­ing in­to a cut­ting-edge field CEO Dave Ricks had shied away from as re­cent­ly as Jan­u­ary 2019.

The big phar­ma is pony­ing up $100 mil­lion up­front to part­ner with Pre­ci­sion Bio­Sciences, fo­cus­ing ini­tial­ly on Duchenne mus­cu­lar dy­s­tro­phy and two oth­er undis­closed in vi­vo tar­gets. Lil­ly is al­so ac­quir­ing $35 mil­lion worth of the biotech’s stock, and has the op­tion to de­vel­op three ad­di­tion­al in vi­vo ther­a­pies.

By of­fer­ing up to $420 mil­lion in R&D and com­mer­cial­iza­tion mile­stones per prod­uct, Lil­ly could end up pay­ing Pre­ci­sion as much as $2.655 bil­lion when all is said and done. On top of that, the biotech is el­i­gi­ble for sin­gle-dig­it to low-teen roy­al­ties on suc­cess­ful ther­a­pies.

Pre­ci­sion $DTIL in­vestors greet­ed the news warm­ly, send­ing shares up more than 12% in ear­ly trad­ing Fri­day.

“We feel like this is a strong state­ment from Lil­ly,” Pre­ci­sion CEO Matthew Kane told End­points News. “This is clear­ly a val­i­dat­ing event for the com­pa­ny, but im­por­tant­ly it un­locks the po­ten­tial for us to more ag­gres­sive­ly go af­ter some of these dis­eases.”

Derek Jantz

At the heart of the deal is Pre­ci­sion’s AR­CUS genome edit­ing plat­form, com­ing from a group of North Car­oli­na sci­en­tists — in­clud­ing CSO Derek Jantz — who claim they have a bet­ter way to ac­com­plish DNA hack­ing than the gene edit­ing pro­mot­ed by biotechs work­ing on CRISPR/Cas9 tech­nolo­gies like CRISPR Ther­a­peu­tics, In­tel­lia and Ed­i­tas.

AR­CUS deals with what’s known as the ARC nu­cle­ase, with the com­pa­ny say­ing it pro­vides a sim­pler, more ef­fec­tive way of com­plet­ing the gene edit­ing process and al­lows for low­er pro­duc­tion costs when pro­duc­tion even­tu­al­ly has to scale up. The en­zyme it­self is syn­thet­ic and comes from a hom­ing en­donu­cle­ase found in al­gae called I-CreI, with sci­en­tists re-en­gi­neer­ing its edit­ing abil­i­ties to knock in, knock out or re­pair cells as they see fit.

“We’ve spent the last 15 years get­ting good at mod­i­fy­ing this nat­ur­al en­zyme from al­gae and bend­ing it to our will, and mak­ing it have the abil­i­ty to ed­it DNA se­quences that we’re in­ter­est­ed in,” Jantz said.

He added that while Pre­ci­sion is look­ing at mul­ti­ple de­liv­ery op­tions, the biotech is “fond” of AAV tech­nol­o­gy be­cause of its long track record in the clin­ic.

Pre­ci­sion’s cur­rent lead pro­gram is an off-the-shelf CAR-T ther­a­py acute lym­phoblas­tic leukemia and non-Hodgkin lym­phoma, aim­ing to tar­get CD19, with Phase I da­ta ex­pect­ed no ear­li­er than the end of 2020. Such treat­ments and oth­er ex vi­vo pro­grams are not in­clud­ed in Fri­day’s part­ner­ship, how­ev­er, and Duchenne had not been one of the biotech’s pre­vi­ous pipeline tar­gets.

Kane said it’s too ear­ly to know when the DMD pro­gram could hit the clin­ic, but de­scribed the pro­gram as mov­ing “ag­gres­sive­ly.”

Dave Ricks

For Lil­ly, Ricks has stat­ed his wari­ness of gene ther­a­pies in the past, de­spite sev­er­al oth­er big com­pa­nies in­vest­ing heav­i­ly in the area. Though the col­lab­o­ra­tion doesn’t deal with the CAR-Ts Pre­ci­sion is de­vel­op­ing in­ter­nal­ly, Fri­day marks an ap­par­ent course cor­rec­tion. Lil­ly will be jump­ing in­to a high­ly com­pet­i­tive DMD field where there are al­ready mul­ti­ple pro­grams in the clin­ic, in­clud­ing those from Pfiz­er, Sol­id Bio­sciences, Ver­tex and Sarep­ta.

“Al­most every­thing I am aware of is sin­gle gene ed­it de­fects, which ul­ti­mate­ly leads you to pret­ty ul­tra-rare con­di­tions, which are not our area of in­ter­est,” Ricks told Reuters in a Jan. 2019 in­ter­view, adding lat­er, “We don’t need new ar­eas to grow.”

Kane said that while he can’t speak for Lil­ly, he not­ed that genome edit­ing is dis­tinct from “tra­di­tion­al” gene ther­a­pies.

“When we think of tra­di­tion­al gene ther­a­py if you will, even though it’s still such a new and emerg­ing field, there we’re typ­i­cal­ly in­sert­ing in or adding a gene that’s miss­ing from the body, but we’re not ac­tu­al­ly im­pact­ing the pa­tient’s genome,” Kane said. “With gene edit­ing, we ac­tu­al­ly do that. We have an op­por­tu­ni­ty to make a per­ma­nent change to the pa­tient’s genome.”

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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Peter Greenleaf, Aurinia CEO

Af­ter pass­ing on Ac­celeron, Bris­tol My­ers eyes bolt-on ac­qui­si­tion of au­toim­mune spe­cial­ist — re­port

Bristol Myers Squibb is looking to beef up its autoimmune portfolio by scooping up Aurinia Pharmaceuticals, Bloomberg reported.

The recent overtures to Aurinia, relayed by anonymous insiders, came just as Bristol Myers turned down buyout talks with partners at Acceleron — which Merck ultimately struck a deal to acquire for $11.5 billion. Bristol Myers has reportedly decided to cash out on its minority stake, likely bagging $1.3 billion in the process, while keeping the royalty deals on two of Acceleron’s blood disorder drugs.

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Robert Califf (Pablo Martinez Monsivais, AP Images, File)

As buzz on Califf FDA nom heats up, in­dus­try and agency in­sid­ers of­fer a strong nod for the ‘per­fect’ choice

For once in this long, dramatic road to finding a new FDA commissioner, there’s been some continuity. Both CNN and Politico reported this weekend that Rob Califf met with President Biden to discuss the permanent commish role, following earlier news broken by the Washington Post that all signs point to Califf.

Although there may be a few Democrats who continue to grandstand about the dangers of COI (Califf has worked for Verily, sits on the board of Centessa Pharmaceuticals, and has other ties to industry research), with the pandemic ongoing and the need for some kind of continuity at FDA mounting, Califf is likely to meet the same fate as when he first won Senate confirmation in 2016, by a vote of 89-4 — Bernie Sanders and 6 others didn’t vote.

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AstraZeneca CEO Pascal Soriot (Raphael Lafargue/Abaca/Sipa USA)

A com­bo of As­traZeneca's Imfinzi and chemo wins where oth­ers have failed in piv­otal bil­iary tract test

Looking to run with the big dogs in the PD-(L)1 class, AstraZeneca’s Imfinzi has a tall hill to climb to compete in an increasingly bustling market. An aggressive combo strategy for the drug has paid off so far, and now AstraZeneca is adding another notch to its belt.

A combo of Imfinzi (durvalumab) and chemotherapy significantly extended the lives of first-line patients with advanced biliary tract cancer over chemo alone, according to topline results from the Phase III TOPAZ-1 study revealed Monday.

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Sean Ianchulev, Eyenovia CEO and CMO

Re­cent court de­ci­sion push­es FDA to re­ject and re­clas­si­fy drug-de­vice com­bo, crush­ing shares

Back in April, the FDA lost a crucial court case in which its broad discretion of regulating medical products that might satisfy the legal definitions of either “drug” and/or “medical device” was sharply curtailed.

In addition to the appeals court ruling that Genus Medical Technologies’ contrast agent barium sulfate (aka Vanilla SilQ) should not be considered a drug, as the FDA had initially ruled, but as a medical device, the agency also was forced to spell out which drugs would transition to devices as a result of the ruling.

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So — that pig-to-hu­man trans­plant; Po­ten­tial di­a­betes cure reach­es pa­tient; Ac­cused MIT sci­en­tist lash­es back; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

We’re incredibly excited to welcome Beth Bulik, seasoned pharma marketing reporter, to the team. You can find much of her work in our new Marketing channel — and in her weekly newsletter, Endpoints PharmaRx, which will launch in early November. Add it to your subscriptions here.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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