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.”

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Roivant par­lays a $450M chunk of eq­ui­ty in biotech buy­out, grab­bing a com­pu­ta­tion­al group to dri­ve dis­cov­ery work

New Roivant CEO Matt Gline has crafted an all-equity upfront deal to buy out a Boston-based biotech that has been toiling for several years now at building a supercomputing-based computational platform to design new drugs. And he’s adding it to the Erector set of science operations that are being built up to support their network of biotech subsidiaries with an eye to growing the pipeline in a play to create a new kind of pharma company.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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Roche and Genen­tech re­searchers plot $53M dis­cov­ery quest aimed at spark­ing a 'Holy moly' piv­ot in neu­ro R&D

Roche and Genentech have committed $53 million to back a 10-year quest aimed at going back to the drawing board to use new technology and fresh scientific insights to generate a pipeline of drugs for neurological diseases.

Researchers from both Roche and its big South San Francisco hub — mixing teams from gRED and pRED this time — will mix it up with the scientists drawn together for the Weill Neurohub — formed in 2019 as a joint research partnership involving UCSF, Berkeley and the University of Washington — in an exploration of the field to develop new therapies for some of the toughest diseases in drug R&D: Alzheimer’s, Parkinson’s, Huntington’s, ALS and autism.

With dust set­tled on ac­tivist at­tack, Lau­rence Coop­er leaves Zio­pharm to a new board

Laurence Cooper has done his part.

In the five years since he left a tenured position at Houston’s MD Anderson Cancer Center to become CEO of Boston-based Ziopharm, he’s steered the small-cap immunotherapy player through patient deaths in trials, clinical holds, short attacks and, most recently, an activist attack on the board.

So when the company has “fantastic news” like an IND clearance for a TCR T cell therapy program, he’s ready to pass on the baton.

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Doug Ingram (file photo)

Why not? Sarep­ta’s third Duchenne MD drug sails to ac­cel­er­at­ed ap­proval

Sarepta may be running into some trouble with its next-gen gene therapy approach to Duchenne muscular dystrophy. But when it comes to antisense oligonucleotides, the well-trodden regulatory path is still leading straight to an accelerated approval for casimersen, now christened Amondys 45.

We just have to wait until 2024 to find out if it works.

Amondys 45’s approval was unceremonious, compared to its two older siblings. There was no controversy within the FDA over approving a drug based on a biomarker rather than clinical benefit, setting up a powerful precedent that still haunts acting FDA commissioner Janet Woodcock as biotech insiders weighed her potential permanent appointment; no drama like the FDA issuing a stunning rejection only to reverse its decision and hand out an OK four months later, which got more complicated after the scathing complete response letter was published; no anxious tea leaf reading or heated arguments from drug developers and patient advocates who were tired of having corticosteroids as their loved ones’ only (sometimes expensive) option.

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J&J ad­comm live blog: Com­mit­tee votes 22-0 to rec­om­mend an FDA OK for the J&J vac­cine, set­ting up 3rd US Covid-19 jab

The US could have a third authorized Covid-19 vaccine within hours.

The FDA’s advisory committee voted unanimously — 22-0 — to recommend the agency issue an emergency use authorization for J&J’s vaccine. If they follow the precedent of the Pfizer and Moderna vaccine,  the FDA will likely authorize the vaccine by Saturday, immediately adding a few million doses to the US supply and adding a 100 million by June. An authorization would give the world its first single-dose vaccine, a major weapon in the effort to vaccinate the world and bring the virus to heel, particularly in rural and developing areas.