Gene-edit­ing up­start lays out a $100M IPO with a plan to quick­ly leapfrog the lead­ers in their field

CRISPR/Cas9. TAL­EN. Zinc fin­ger nu­cle­ase tech. The ARC nu­cle­ase.

You may have heard about those first 3 gene-edit­ing plat­forms. But what’s an ARC nu­cle­ase?

Matthew Kane

AR­CUS was put to­geth­er by a group of sci­en­tists in North Car­oli­na who have been mak­ing the pitch that they have a bet­ter way to ac­com­plish the DNA hack­ing pop­u­lar­ized 5 years ago by the orig­i­nal trio of star­tups: CRISPR Ther­a­peu­tics, Ed­i­tas, In­tel­lia. Those biotechs are just now get­ting in­to the clin­ic, with Pre­ci­sion Bio­Sciences com­ing in right be­hind with its own new­ly filed IND. They’re fo­cused on a gene-edit­ed al­lo­gene­ic (off the shelf) CAR-T cell pro­gram tar­get­ing CD19 (not for the first time) which they plan on launch­ing soon, with a Phase I/IIa clin­i­cal tri­al in pa­tients with acute lym­phoblas­tic leukemia and non-hodgkin lym­phoma. 

AR­CUS be­longs to the start­up Pre­ci­sion Bio­Sciences, which on Fri­day filed for an IPO, pen­cilling in $100 mil­lion as the tar­get.

Their claim to fame rests on a one-step en­gi­neer­ing process, which they are sell­ing as a sim­pler, more ef­fec­tive way of com­plet­ing the gene edit­ing process that will trans­late well to a less ex­pen­sive mass pro­duc­tion ap­proach. 

The sim­ple ex­pla­na­tion is that Pre­ci­sion Bio be­lieves it has a bet­ter sur­gi­cal tool — the ARC nu­cle­ase — for slic­ing in­to a spe­cif­ic DNA se­quence need­ed to cor­rect a dis­ease.

Jeff Smith

This ARC nu­cle­ase, they say, is “a ful­ly syn­thet­ic en­zyme sim­i­lar to a hom­ing en­donu­cle­ase but sig­nif­i­cant­ly im­proved to be the start­ing point for the genome-edit­ing plat­form.” It’s small, they claim, with “in­com­pa­ra­ble” speci­fici­ty that can be cus­tomized to hit the right tar­get in just the right way to im­prove po­ten­cy.

Every one of the pi­o­neers has a sim­i­lar claim to the best tech. CRISPR $CR­SP and In­tel­lia $NT­LA are Cas9 spe­cial­ists, pop­u­lar­iz­ing a new tool cre­at­ed by Jen­nifer Doud­na and Em­manuelle Char­p­en­tier that’s known for be­ing cheap and easy to use. This tech has spread like wild­fire in aca­d­e­m­ic labs. Ed­i­tas $ED­IT is us­ing a new-and-im­proved ver­sion of Cas9. Cel­lec­tis $CLLS CEO An­dré Chouli­ka is diplo­mat­ic about it, but he’s pas­sion­ate about TAL­EN, which he helped cre­ate. Sang­amo, which on­ly re­cent­ly of­fered its first hu­man da­ta, was off tar­get on the da­ta but hap­py about the ef­fect it was see­ing in hu­mans.

All the pi­o­neers have seen their shares beat up over the past year. But then, that’s true for a lot of pub­lic biotechs.

Derek Jantz

The whole field, which has at­tract­ed large in­vest­ments, is pri­mar­i­ly based on non-hu­man pri­mate da­ta. But it’s at a cross­roads, with much more hu­man da­ta on the near hori­zon. The win­ners will be rich­ly re­ward­ed. The losers will face the scrap heap.

The Durham, NC-based biotech with close con­nec­tions to Duke rolled out a $110 mil­lion mega round last sum­mer from a laun­dry list of back­ers that in­clud­ed Gilead. And as we said at the time, it had every ear­mark of a clas­sic crossover round point­ed straight at the $100 mil­lion IPO you’re read­ing about now.

David Thomp­son

Ar­row­Mark Part­ners led the deal and was joined by oth­er new in­vestors: Franklin Tem­ple­ton In­vest­ments, Cowen Health­care In­vest­ments, Brace Phar­ma Cap­i­tal, Pon­tif­ax AgTech, OCV Part­ners, Adage Cap­i­tal Man­age­ment, Cor­morant As­set Man­age­ment, Vi­vo Cap­i­tal, Alexan­dria Ven­ture In­vest­ments, Ridge­back Cap­i­tal, Agent Cap­i­tal, and en­ti­ties af­fil­i­at­ed with Leerink Part­ners. Ex­ist­ing in­vestors ven­Bio, F-Prime, RA Cap­i­tal Man­age­ment, Am­gen

Ven­tures, Os­age Uni­ver­si­ty Part­ners, DU­MAC, and the Longevi­ty Fund al­so par­tic­i­pat­ed in the fi­nanc­ing.

Gilead fol­lowed up with a $445 mil­lion pact with Pre­ci­sion in the fall, fo­cused on he­pati­tis B. And then gene edit­ing ex­perts at the Uni­ver­si­ty of Penn­syl­va­nia stepped up with a sci­en­tif­ic col­lab­o­ra­tion. They split off their ag ops just ahead of the new round last year.

Abid Ansari

The top 3 ex­ecs haven’t ex­act­ly short­changed them­selves on in­come. CEO Matthew Kane took home a com­pen­sa­tion pack­age worth $1.6 mil­lion for last year. CFO Abid Ansari snagged $1.4 mil­lion and David Thomp­son, the chief de­vel­op­ment of­fi­cer, got $1.8 mil­lion — all big mon­ey in the start­up world. They al­so got rais­es for their base salary, now at $523,000 for Kane, who al­so has 5.6% of the stock, which will be worth mil­lions if the IPO comes in as they hope.

Jeff Smith — a co-founder and CTO out of Duke Uni­ver­si­ty — has a wedge of 10% of the eq­ui­ty, which puts him up with the two top in­vestors: ven­Bio at 11% and F-Prime at 9.7%. The oth­er sci­en­tif­ic co-founder is Derek Jantz, whose bio in­cludes a ci­ta­tion for ear­ly work de­vel­op­ing the zinc fin­ger tech.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

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The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Two wild weeks for Grail end in $8B Il­lu­mi­na buy­out

Grail’s whirlwind two weeks have ended in the wealthy arms of its former founder and benefactors.

Illumina has shelled out $8 billion to reacquire the closely-watched liquid biopsy startup they spun out just 5 years ago and sold off much of its shares just 3 years ago. The deal comes nearly two weeks after the well-heeled startup filed for a potentially massive IPO — one that was disrupted just a week later when Bloomberg reported that Illumina was in talks to buy their former spinout for up to $8 billion.

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#ES­MO20: 'As good as any PD-1 out there': Re­gen­eron flash­es PD-(L)1 lung can­cer da­ta to ri­val Mer­ck

Regeneron entered the PD-(L)1 game late, so they devised a two-pronged strategy to catch up with Big Pharma rivals: They would push it into cancers where PD-1s had yet been tested, and they would prove that it’s as powerful in the big indications as any other on the market.

They cleared a hurdle on the first goal Friday, showing a 31% response in patients with the rare skin cancer basal cell carcinoma. And with the data they’re rolling out Monday, Regeneron cancer chief Israel Lowy is ready to declare success on the second.

Roche vaults to the front of the NL­RP3 clin­i­cal race, pay­ing $448M up­front to bag In­fla­zome

Roche is going all in on NLRP3.

The pharma giant is putting down $448 million (€380 million) upfront to snatch Novartis-backed Inflazome, which makes it a clinical player in the space overnight.

Dublin and Cambridge, UK-based Inflazome is the second NLRP3-focused biotech Roche has acquired in less than two years, and although no numbers were disclosed in the Jecure buyout, this is almost certainly a much larger deal.

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As­traZeneca pub­lish­es Covid-19 vac­cine PhI­II pro­to­cols in lock­step with Mod­er­na and Pfiz­er. How are they dif­fer­ent?

Following in the steps of Moderna and Pfizer, the other two American drugmakers currently in Phase III trials for their Covid-19 vaccines, AstraZeneca posted its own study protocols over the weekend. The move is the latest in a series of rare peeks behind the curtain, as such blueprints are typically shared once such trials are completed.

“Given the unprecedented global impact of the Coronavirus pandemic and the need for public information, AstraZeneca has published the detailed protocol and design of our AZD1222 clinical trial. As with most clinical development, protocols are not typically shared publicly due to the importance of maintaining confidentiality and integrity of trials. AstraZeneca continues to work with industry peers to ensure a consistent approach to sharing timely clinical trial information,” the company said in a statement.

Eli Lilly CSO Dan Skovronsky (file photo)

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The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

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Sebastian Nijman (file photo)

Roche looks to ge­net­ic mod­i­fiers for new drug tar­gets, team­ing up with Dutch biotech in $375M deal

Roche is gambling on a new way of discovering drug targets and, ultimately, promising to infuse more than $375 million into a small biotech if all goes well.

A spinout of the Netherlands Cancer Institute and Oxford University, Scenic Biotech set out to pioneer a field that’s gaining some traction among top VCs in the US: to harness the natural protecting powers of genetic modifiers — specific genes that suppress a disease phenotype.

Greg Friberg (File photo)

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The first time I sat down with Amgen’s Greg Friberg to talk about the pharma giant’s KRAS G12C program for sotorasib (AMG 510) at ASCO a little more than a year ago, there was high excitement about the first glimpse of efficacy from their Phase I study, with 5 of 10 evaluable non-small cell lung cancer patients demonstrating a response to the drug.

After decades of failure targeting KRAS, sotorasib offered the first positive look at a new approach that promised to open a door to a whole new approach by targeting a particular mutation to a big target that had remained “undruggable” for decades.

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#ES­MO20: Out to beat Tagris­so, J&J touts 100% ORR for EGFR bis­pe­cif­ic/TKI com­bo — fu­el­ing a quick leap to PhI­II

J&J’s one-two punch on EGFR-mutant non-small cell lung cancer has turned up some promising — although decidedly early — results, fueling the idea that there’s yet room to one up on third-generation tyrosine kinase inhibitors.

Twenty out of 20 advanced NSCLC patients had a response after taking a combination of an in-house TKI dubbed lazertinib and amivantamab, a bispecific antibody targeting both EGFR and cMET engineered on partner Genmab’s platform, J&J reported at ESMO. All were treatment-naïve, and none has seen their cancer progress at a median follow-up of seven months.