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.

UP­DAT­ED: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.

(Image: Associated Press)

Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.

Federal Trade Commission commissioner Rohit Chopra testifies on Capitol Hill (AP Photo/Susan Walsh)

FTC clears Bris­tol-My­ers’ $74B deal to buy Cel­gene — but Dems sig­nal a po­ten­tial hard shift against Big Phar­ma M&A

Bristol-Myers Squibb’s record $74 billion takeover of Celgene is a done deal. And it will all be over — except for the lingering complaints from die-hard Celgene investors — on Wednesday.

Like much else that’s going on in Washington these days, the vote among the 5 FTC commissioners split along party lines, with the 3 Republicans voting to clear the way and the 2 Democrats steamed over what they see as a major M&A move that will lessen competition and innovation. And that split has big implications for the M&A side of the business if the Dems take the White House in 2020.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.

BeiGene CEO John Oyler at an Endpoints event in Shanghai, October 2018 (Credit: Endpoints News/PharmCube)

UP­DAT­ED: In a first, FDA green-lights use of a Chi­nese built can­cer ther­a­py — and more are com­ing

Weeks after Amgen took a $2.7 billion stake in BeiGene, the Beijing-based biotech has secured its first-ever FDA approval for zanubrutinib, a BTK inhibitor, months ahead of schedule.

BeiGene’s drug, branded as Brukinsa, has secured accelerated approval for adult patients with mantle cell lymphoma (MCL) — a typically aggressive, rare, form of blood cancer — who have received at least one prior therapy.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.

What does $62B buy you these days? A lot, says Take­da ex­ecs as the phar­ma play­er promis­es a block­buster R&D fu­ture

First comes the $62 billion buyout. Then comes the asset auction and reorganization to pay down debt. Now comes the detailed pledge of a bigger, brighter future in drug development.

That’s where Takeda finds itself on R&D day today, about 11 months after closing on their Shire acquisition. R&D chief Andy Plump is joining CEO Christophe Weber and other top members of the team to outline a new set of priorities in the greatly expanded pipeline at Takeda, which has jumped into the top ranks of the world’s pharma giants in the wake of the Shire deal.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.

GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.

Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.

Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.

(Image: Associated Press)

No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,700+ biopharma pros reading Endpoints daily — and it's free.