Pre­ci­sion Bio wants to show there's a bet­ter way to ed­it genes — and now it has $126M in IPO cash to prove it

Af­ter spend­ing the sec­ond half of last year lay­ing the car­pet for an IPO — com­plete with a crossover round and a big part­ner­ship with Gilead — Pre­ci­sion Bio­Sciences has raised $126 mil­lion in its pub­lic de­but.

The go­ing price was $16 each for 7.9 mil­lion shares $DTIL, smack in the mid­dle of its pro­posed range but land­ing above Pre­ci­sion’s orig­i­nal pen­cilled-in goal of $100 mil­lion.

The Durham, NC-based gene edit­ing up­start is pitch­ing its ARC nu­cle­ase ap­proach as a bet­ter al­ter­na­tive to the promi­nent trio of CRISPR, TAL­EN and zinc fin­ger nu­cle­ase. Dubbed AR­CUS, the tech de­rives its name from a hom­ing en­donu­cle­ase found in al­gae called I-Crel, re­pur­pos­ing its nat­ur­al genome edit­ing abil­i­ties to cut and paste on­to cells as need­ed.

Be­cause the syn­thet­ic en­zyme can rec­og­nize long se­quences of DNA while stay­ing rel­a­tive­ly small, Pre­ci­sion claims, AR­CUS is more spe­cif­ic and flex­i­ble than oth­er gene edit­ing meth­ods out there. It is set to be test­ed in hu­mans soon as part of a tri­al for the com­pa­ny’s off-the-shelf CAR-T pro­gram, just as the CRISPR pi­o­neers — CRISPR Ther­a­peu­tics, In­tel­lia and Ed­i­tas — are get­ting in­to the clin­ic.

Specif­i­cal­ly, Pre­ci­sion’s first CAR-T ther­a­py will aim to treat acute lym­phoblas­tic leukemia and non-Hodgkin lym­phoma by en­gi­neer­ing the donor T cells to tar­get CD19.

Next up in its pipeline is a he­pati­tis B pro­gram that Gilead has al­ready bought in­to, rep­re­sent­ing the “in vi­vo gene cor­rec­tion” half of its med­i­cine port­fo­lio. An IND for that po­ten­tial hep B cure is ex­pect­ed in 2020. Mean­while, oth­er dis­eases in that bas­ket in­clude he­mo­phil­ia A, fa­mil­ial amy­loid polyneu­ropa­thy, lipopro­tein li­pase de­fi­cien­cy and fa­mil­ial hy­per­c­ho­les­terolemia, from which Pre­ci­sion will choose one to el­e­vate out of dis­cov­ery stage.

Aside from clin­i­cal spend­ing, the biotech has al­lo­cat­ed $12 mil­lion to $14 mil­lion for a man­u­fac­tur­ing fa­cil­i­ty be­ing built in Durham.

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

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I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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Re­gen­eron's Evkeeza shows promise in curb­ing high triglyc­erides, but will ge­net­ic dis­par­i­ties lim­it use?

When Regeneron scored an early approval for lipid lowering antibody Evkeeza back in February, the drugmaker cracked open a new pathway to lower abnormally high cholesterol levels. Now, Regeneron is chasing high triglycerides as well with some promising mid-stage data — but will genetic restrictions limit the drug’s use?

Regeneron’s Evkeeza (evinacumab) cut median triglyceride levels by more than 800 mg/dL (57%) in patients with a rare disorder causing abnormally high triglyceride levels compared with an overall increase of 50 mg/dL (1.8%) in participants on placebo, according to Phase II data presented Sunday at the virtual American College of Cardiology meeting.

As­traZeneca's Farx­i­ga missed big on Covid-19 study, but it's tak­ing SGLT2 safe­ty da­ta as a sil­ver lin­ing

AstraZeneca hasn’t seen many setbacks in recent months for SGLT2 inhibitor Farxiga, which broke ground in heart failure and kidney disease regardless of diabetes diagnosis. But the British drugmaker had to admit defeat in taking Farxiga into Covid-19, but follow-up results add a bit of a silver lining to that trial’s safety data.

Of hospitalized Covid-19 patients dosed with AstraZeneca’s Farxiga, 11.2% experienced an organ failure or died after 30 days of therapy compared with 13.8% of those given placebo, according to follow-up data from the DARE-19 study revealed Sunday at the virtual American College of Cardiology meeting.

Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Pfiz­er, Bris­tol My­er­s' Eliquis flops in post-heart surgery pa­tients, spurring an 'un­ex­plained sig­nal' in cer­tain deaths

Pfizer and Bristol Myers Squibb’s non-warfarin blood thinner Eliquis has raced out to become the most prescribed drug of its class on the market — even overtaking warfarin’s long-time lead. But in tricky-to-treat patients after a valve replacement, an investigator-sponsored study couldn’t turn up benefit and raised a troubling safety signal.

Eliquis failed to show benefit over standard of care in preventing serious clinical outcomes after a transaortic valve replacement (TAVR) and was linked to an “unexplained signal” in a subset of populations with a higher rate of non-CV deaths who did not need blood thinners apart from the surgery, according to data presented Saturday at the virtual American College of Cardiology meeting.

Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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Abbie Celniker (L) and Rob Sims (Flare)

A Third Rock-backed play­er charts a new course against tran­scrip­tion fac­tors. Do 'switch sites' hold the mag­ic sauce?

Long known for their role in guiding gene expression but considered “undruggable,” DNA binding transcription factors have long been a Holy Grail for drug developers. Now, a new startup from Third Rock Ventures thinks it could have the juice to get after transcription factors once and for all — and it all started with a “flare” of inspiration from an article out of an Oxford lab.

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