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.

John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

In­vestor day prep at Mer­ck in­cludes a new strat­e­gy to pick up the pace on M&A — re­port

Mer­ck’s re­cent deals to buy up two bolt-on biotechs — Ti­los and Pelo­ton — weren’t an aber­ra­tion. In­stead, both ac­qui­si­tions mark a new strat­e­gy to beef up its dom­i­nant can­cer drug op­er­a­tions cen­tered on Keytru­da while look­ing to ad­dress grow­ing con­cerns that too many of its eggs are in the one I/O bas­ket for their PD-1 pro­gram. And Mer­ck is go­ing af­ter more small- and mid-sized buy­outs to calm those fears.

Dave Barrett, Brian Chee, Amir Nashat, Amy Schulman. Polaris

Bob Langer's first port of call — Po­laris Part­ners — maps $400M for ninth fund

Health and tech ven­ture group Po­laris Part­ners, which counts Alec­tor, Al­ny­lam and Ed­i­tas Med­i­cine as part of its port­fo­lio, is set­ting up its ninth fund, rough­ly two years af­ter it closed Po­laris VI­II with $435 mil­lion in the bank, sur­pass­ing its tar­get by $35 mil­lion.

The Boston-based firm, in an SEC fil­ing, said it in­tends to raise $400 mil­lion for the fund. Po­laris — which rou­tine­ly backs com­pa­nies mold­ed out of the work done in the lab of pro­lif­ic sci­en­tist Bob Langer of MIT  — typ­i­cal­ly in­vests ear­ly, and sticks around till com­pa­nies are in the green. Like its peers at Flag­ship and Third Rock, Po­laris is all about cham­pi­oning the lo­cal biotech scene with a steady flow of start­up cash.

The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

Partners Innovation Fund

David de Graaf now has his $28.5M launch round in place, build­ing a coen­zyme A plat­form in his lat­est start­up

Long­time biotech ex­ec David de Graaf has the cash he needs to set up the pre­clin­i­cal foun­da­tion for his coen­zyme A me­tab­o­lism com­pa­ny Comet. A few high-pro­file in­vestors joined the ven­ture syn­di­cate to sup­ply Comet with $28.5 mil­lion in launch mon­ey — enough to get it two years in­to the plat­form-build­ing game, with­in knock­ing dis­tance of the clin­ic.

Canaan jumped in along­side ex­ist­ing in­vestor Sofinno­va Part­ners to co-lead the round, with par­tic­i­pa­tion by ex­ist­ing in­vestor INKEF Cap­i­tal and new in­vestor BioIn­no­va­tion Cap­i­tal.

Right back at you, Pfiz­er: BeiGene and a Pfiz­er spin­out launch a new­co to de­vel­op a MEK/BRAF in­hibitor that could ri­val $11.4B com­bo

A day af­ter Pfiz­er bought Ar­ray and its ap­proved can­cer com­bo, BeiGene and Pfiz­er spin­out Spring­Works have part­nered in launch­ing a new biotech that has an eye on the very same mar­ket the phar­ma gi­ant just paid bil­lions for. And they’re plan­ning on us­ing an ex-Pfiz­er drug to do it.

In a nut­shell, Chi­na’s BeiGene is toss­ing in a pre­clin­i­cal BRAF in­hibitor — BGB-3245, which cov­ers both V600 and non-V600 BRAF mu­ta­tions — for a big stake in a new, joint­ly con­trolled biotech called Map­Kure with Bain-backed Spring­Works.

Step­ping on Roche's toes, Mer­ck cuts in­to SCLC niche with third-line Keytru­da OK

In the in­creas­ing­ly crowd­ed check­point race, small cell lung can­cer has been a rare area where Roche, a sec­ond run­ner-up, has a lead over the en­trenched lead­ers Mer­ck and Bris­tol-My­ers Squibb. But Mer­ck is fi­nal­ly mak­ing some head­way in that di­rec­tion with the lat­est ap­proval for its PD-1 star.

The lat­est green light en­dors­es Keytru­da in the third-line treat­ment of metasta­t­ic SCLC, where it would be giv­en to pa­tients whose dis­ease ei­ther don’t re­spond to or re­lapse af­ter chemother­a­py, which would have fol­lowed at least one pri­or line of ther­a­py.

Sanofi aligns it­self with Google to stream­line drug de­vel­op­ment

Tech­nol­o­gy is bleed­ing in­to health­care, and big phar­ma is rid­ing the wave. Sanofi $SNY ap­point­ed its first chief dig­i­tal of­fi­cer this Feb­ru­ary, fol­low­ing the foot­steps of its peers. By May, the French drug­mak­er and some of its big phar­ma com­pa­tri­ots joined forces with Google par­ent Al­pha­bet’s Ver­i­ly unit to aug­ment clin­i­cal tri­al re­search. On Tues­day, the Parisian com­pa­ny tied up with Google to ac­cess its cloud com­put­ing and ar­ti­fi­cial in­tel­li­gence tech to spur the de­vel­op­ment of new ther­a­pies.

Af­ter watch­ing its share price soar on a Bloomberg re­port and heat­ed ru­mors, Bio­haven stock takes a bil­lion-dol­lar bath

Back in April, Biohaven Pharmaceutical became one hot biotech stock $BHVN based on a report in Bloomberg that some “potential bidders” had been kicking the tires at the biotech, which has a lead drug for migraines. Then the rumor mill really started to smoke when execs canceled a presentation at an investor conference a little more than a week ago.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.