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.

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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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.

Gold­man Sachs jumps aboard Bain-backed 503(b) com­pound­ing phar­ma­cy with a $275M debt loan to sup­ply hos­pi­tals

Long the bane of the FDA’s existence, compounding pharmacies have seen a minor resurgence in the past year as short-term saviors for hospital drug shortages. Now, a 503(b) company specializing in hospital meds has earned a big backer to keep expanding its 200-drug strong portfolio.

Goldman Sachs and Owl Rock Capital Partners have doled out a $275 million debt loan to QuVa Pharma, a 503(b)-certified outsourcing facility providing compounded drugs to hospitals, the company said Thursday.

Bill Lis, Jasper Therapeutics

Jasper and its stem cell con­di­tion­ing an­ti­body earn a tick­et to Nas­daq in lat­est SPAC re­verse merg­er

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

Another biotech SPAC deal has landed as the glut of blank-check companies continues to make waves in the industry.

Thursday’s winner is Jasper Therapeutics, joining forces with Amplitude Healthcare Acquisition Corp. in a $100 million reverse-merger, Jasper announced. The deal also comes with a PIPE financing of an additional $100 million, setting Jasper up with a $490 million market cap once the merger closes in the third quarter.

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.