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.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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Sanofi takes a $260M hit to ex­tri­cate it­self from a dis­as­trous al­liance with Lex­i­con

Sanofi spent $300 million in cash to get into a $1.7 billion alliance with Lexicon on their SGLT1/2 diabetes drug sotagliflozin. And now that the drug has been spurned by the FDA after burning through a program that provided mixed late-stage data and a late shot at a last-place finish, the French pharma giant is forking over another $260 million to get out of the deal.

Sanofi’s unhappiness was already apparent when the company — now under new CEO Paul Hudson — posted a statement back in July that they were dropping the deal. But it wasn’t that simple. 

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.