Ed­i­tas los­es its lead in the biotech race to launch the first CRISPR/Cas9 hu­man study

Ka­trine Bosley, Ed­i­tas

Ed­i­tas was the first of three gene edit­ing biotechs to make a splash on Nas­daq and up un­til this week it al­so looked like it was in the lead in the race to be the first in the group to make it in­to a ground­break­ing hu­man study.

No more.

On Mon­day Ed­i­tas $ED­IT CEO Ka­trine Bosley said that a “man­u­fac­tur­ing de­lay re­lat­ed to pro­duc­tion of in­put ma­te­ri­als for AAV man­u­fac­tur­ing” forced the com­pa­ny to de­lay their planned IND fil­ing to the mid­dle of next year. As ex­ecs ex­plained it, one of the in­put ma­te­ri­als used in mak­ing their de­liv­ery ve­hi­cle failed a qual­i­ty con­trol in­spec­tion, and the de­lay in fix­ing that caused them to lose their time slot with the CMO.

CRISPR Ther­a­peu­tics CEO Rodger No­vak

It’s a sig­nif­i­cant de­lay. Ed­i­tas, which saw its stock drop 5% in pre-mar­ket trad­ing Tues­day, had planned to file late this year. And in the process of slip­ping be­hind, the biotech lost its lead to CRISPR Ther­a­peu­tics. In CRISPR’s Q1 up­date $CR­SP a few days ago CEO Rodger No­vak not­ed that his group plans to file its CTA — clin­i­cal tri­al au­tho­riza­tion in Eu­rope — in late 2017 for its lead ther­a­peu­tic prod­uct to treat be­ta-tha­lassemia.

In­tel­lia $NT­LA, mean­while, is still fo­cused more on an­i­mal stud­ies, with IND en­abling stud­ies for its AT­TR pro­gram slat­ed to run through the sec­ond half of 2017 and the first half of 2018.

These three biotechs have been slug­ging it out over con­trol of the patents be­hind one of the most dra­mat­ic new open­ings in drug R&D of the decade. By us­ing CRISPR/Cas9 tech to ed­it ge­nom­ic DNA, these com­pa­nies be­lieve that they are start­ing down a path that will lead to once-and-done cures for a le­gion of ail­ments. And the sci­en­tists be­hind the launch of CRISPR Ther­a­peu­tics and In­tel­lia — Em­manuelle Char­p­en­tier and Jen­nifer Doud­na — have worked to get Ed­i­tas’ patents from the Broad thrown out, say­ing they in­vent­ed the tech­nique. So far, though, they have failed at that.

How­ev­er the patent fight turns out, they’re all years away from prov­ing who can take the lead in com­mer­cial­iz­ing the tech­nol­o­gy. But these ear­ly plays are al­ready high­light­ing how sig­nif­i­cant changes in ini­tial clin­i­cal time­lines can ul­ti­mate­ly af­fect the out­come of the big­ger late-stage race to come.

None of the biotechs can claim to be the first to try it in hu­mans. That dis­tinc­tion al­ready fell to a group in Chi­na that pulled off an ear­ly ex­plorato­ry study. And Sean Park­er has agreed to fund a hu­man study led by Carl June at the Uni­ver­si­ty of Penn­syl­va­nia that could be the first to get start­ed in the U.S.

There are bound to be plen­ty of big sur­pris­es ahead.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Ryan Watts, Denali CEO

Bio­gen hands De­nali $1B-plus in cash, $1B-plus in mile­stones to part­ner on late-stage Parkin­son’s drug

Biogen is handing over more than a billion dollars cash to partner with the up-and-coming neurosciences crew at Denali on a new therapy for Parkinson’s. And the big biotech is ready to pile on more than a billion dollars more in milestones — if the alliance is a success.

For Biogen $BIIB, the move on Denali’s small molecule inhibitors of LRRK2 puts them in line to collaborate on a late-stage program for DNL151, which is scheduled to start next year.

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Levi Garraway, Roche CMO (Source: Genentech)

FDA hands out a quick OK for po­ten­tial SMA block­buster ris­diplam, giv­ing Genen­tech and Roche a chance to chal­lenge ri­vals on the price

US regulators handed Roche and Genentech a big win Friday afternoon, one that has market-shaping potential for its high-priced rivals from Novartis and Biogen.

The FDA has green-lit the companies’ spinal muscular atrophy drug risdiplam, which will be marketed as Evrysdi in the US, for use in patients two months and older. It’s the first SMA drug that can be taken orally, as Biogen’s Spinraza is injected into the spine while Novartis’ Zolgensma is a gene therapy.

Moncef Slaoui, Getty Images

When will it end? Big Phar­ma's top vac­cine ex­pert at OWS of­fers a speedy time­line for a Covid-19 vac­cine — ei­ther be­fore or right af­ter the elec­tion

Moncef Slaoui hasn’t started making plans for his summer vacation next year. But he offers high odds that all Americans will be able to do that in the not too distant future.

In an interview with a pair of sympathetic podcasters at the conservative American Enterprise Institute, Slaoui provides an education to listeners on how any drug or vaccine can be sped through trials. And he leaves the door wide open to the notion that the leading vaccine developers can demonstrate efficacy and safety in a compelling fashion as early as October — or as late as the end of this year.

Per­cep­tive fields SPAC #3 as an­oth­er group of biotechs scoops up $364M in lat­est Nas­daq romp

There’s no sign that the windfall of cash dropping biotech’s way on Wall Street is abating. Three more biopharmas priced IPOs on Thursday and Friday morning, riding a historic boom with a $364 million payoff.

London-based biotech Freeline Therapeutics took home the lion’s share of the cash with $159 million after pricing 8,823,529 shares at $18 a pop. Checkmate Pharmaceuticals, of Cambridge, MA, raised $75 million with an offer of 5 million shares at $15 — right at the midpoint of its range. And Arya Sciences Acquisition Corp III, the third in a series from Perceptive, priced 13,000,000 shares at $10 per share.

Covid-19 roundup: Gates Foun­da­tion pours $150M in­to In­dia’s Serum In­sti­tute; Pfiz­er teams with Gilead on remde­sivir

By CEO and scion Adar Poonawalla’s estimation, the Serum Institute in India has already poured hundreds of millions of dollars into scaling up the unproven Covid-19 vaccine being developed by AstraZeneca and Oxford for use in low and middle income countries. It’s meant taking on a risk that other companies, including AstraZeneca, have mitigated with huge amounts of government funding.

Now, for the first time, Poonawalla is getting some outside help. The Gates Foundation has agreed to pay the institute $150 million to supply 100 million vaccines to India and other emerging economies next year, Reuters reported. That includes both the vaccine being developed by AstraZeneca and the one being developed by Novavax. Those vaccines will be available in 92 countries and be priced at $3 per dose.

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In sur­pris­ing set­back, com­bo of Roche’s Tecen­triq and chemo fails to help pa­tients with triple-neg­a­tive breast can­cer

Roche broke ground last year when they secured the first FDA approval for a checkpoint therapy in triple-negative breast cancer, a notoriously difficult-to-treat indication that has been passed over by the wave of targeted therapies.

Now, though, doctors are puzzling over why a combination of drugs meant to make that therapy more potent instead appeared to make it less effective.

Roche said Thursday that in a Phase III trial, combining their PD-1/L1 checkpoint therapy Tecentriq with the chemotherapy paclitaxel, did not significantly improve progression-free survival for patients with locally advanced or metastatic triple-negative breast cancer over giving those patients chemotherapy alone. In fact, patients on the Tecentriq-chemo arm had lower overall survival than patients on chemo, although the drugmaker cautioned that the trial was not powered for that endpoint and the data were immature.

Douglas Fambrough, Dicerna CEO (Boehringer Ingelheim via YouTube)

Roche-backed Dicer­na push­es in­to the pack rac­ing to­ward the block­buster hep B goal line, armed with PhI da­ta

Dicerna has lined up a set of proof-of-concept data from a small cohort of hepatitis B patients in a match-up against some heavyweight rivals which got out in front of this race. And right in the front row you’ll find a team from Roche, which paid $200 million in cash and offered another $1.5 billion in milestones to partner with Dicerna $DRNA on their RNAi program for hep B.

Right now it’s looking competitive, with lots of big challenges ahead.

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Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

When will it end? Gold­man econ­o­mist gives late-stage vac­cines a good shot at tar­get­ing 'large shares' of the US by mid-2021 — but the down­side is daunt­ing

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can create herd immunity in the US by the middle of next year, with Europe following a few months later.

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