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.”

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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Joe Wiley, Amryt CEO

A biotech with a yen for pricey rare dis­ease drugs — and bar­gain base­ment shop­ping — adopts an­oth­er or­phan in lat­est M&A pact

After making it through a long, painful haul to get past a CRL and on to an FDA approval last summer, little Chiasma has found a buyer.

Amryt $AMYT, a company known for its appetite for acquiring expensive drugs for rare diseases at bargain prices, snagged Chiasma and its acromegaly drug Mycapssa (octreotide) capsules in an all-stock deal — with an exchange of 0.396 shares of Amryt for every share of Chiasma.

Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Paul Hastings, Nkarta CEO

With no up­front pay­ment or mile­stones on the line, Nkar­ta and CRISPR join forces on CAR-NK search

Most deals in biotech come with hefty upfront payments attached, and the promise of big biobucks if a program works out. Not this one.

Nkarta has struck what CEO Paul Hastings calls a “real collaboration” with CRISPR Therapeutics to co-develop and commercialize two CAR-NK therapies, in addition to an NK+T program. The duo will split all R&D costs — and any worldwide profits — 50/50, Hastings said.

Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

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Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.