Cel­gene is squeez­ing the trig­ger on an ear­ly FDA fil­ing for key Agios can­cer drug

Agios CEO David Schenkein

Six years af­ter Cel­gene helped get the R&D ball rolling at Agios with a world­wide pact that cov­ered the glob­al rights for its promis­ing AG-221, the big biotech is tee­ing up a straight shot at an ac­cel­er­at­ed ap­proval for acute myeloid leukemia based af­ter plan­ning an ear­ly look at ef­fi­ca­cy da­ta.

In an SEC fil­ing out this morn­ing, Agios re­ports that lat­er to­day Cel­gene will re­view a plan to file for an ear­ly ap­proval on AML be­fore the end of this year. And Agios it­self is prep­ping a plan to hus­tle an NDA to the FDA next year for a quick OK on AG-120, a close­ly re­lat­ed IDH1 mu­ta­tion ther­a­py which Cel­gene re­cent­ly hand­ed back in a deal to re­work their long­stand­ing col­lab­o­ra­tion.

The news spurred a 20% spike in Agios’ share price on Wednes­day.

Ac­cord­ing to an 8-K, Agios re­ports that:

“The (Cel­gene) NDA will be based on da­ta from the on­go­ing phase 1/2 study of AG-221 in pa­tients with ad­vanced hema­to­log­ic ma­lig­nan­cies with an IDH2 mu­ta­tion. The NDA sub­mis­sion is ex­pect­ed to oc­cur by year-end 2016. Cel­gene will be dis­cussing the planned enasi­denib NDA sub­mis­sion at the Citi 11th An­nu­al Biotech Con­fer­ence in Boston in a we­b­cast event on Wednes­day, Sep­tem­ber 7, 2016 at 12:00 pm ET.”

Cel­gene was drawn to the part­ner­ship by Agios’ fo­cus on drugs that in­hib­it an en­zyme need­ed to me­tab­o­lize nu­tri­ents. IDH1 and IDH2 are mu­tat­ed in can­cer, dri­ving pro­lif­er­a­tion of the can­cer.

Back in the spring, Cel­gene hand­ed back its ex-US rights to AG-120, which has per­formed well for hema­to­log­i­cal ma­lig­nan­cies but not so well in sol­id tu­mors. But the big biotech al­so hand­ed over $200 mil­lion up front to add to its pack­age of mol­e­cules while car­ry­ing on with AG-221.

In March, Cel­gene and Agios set out to en­roll 150 AML pa­tients with a plan to treat them with a com­bi­na­tion of ei­ther AG-221 or AG-120 with Vi­daza or Vi­daza alone. Cel­gene holds the world­wide rights to AG-221, while Agios is still in line for a rich set of mile­stones for their de­vel­op­ment pro­gram.

The move at Cel­gene to shift their fo­cus to a po­ten­tial ap­proval at this stage marks a key turn­ing point for a biotech which Agios CEO David Schenkein is fond of re­call­ing start­ed out with lit­tle more than a blank sheet of pa­per. Since then, the pages at Agios have been filled with high-pro­file part­ner­ships an IPO for its po­ten­tial cut­ting-edge work and mixed progress in the clin­ic.

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Vas Narasimhan. Getty Images

UP­DAT­ED: Failed PhI­II fe­vip­iprant tri­als pour more cold wa­ter on No­var­tis' block­buster R&D en­gine — and briefly spread the chill to a high-pro­file biotech

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UP­DAT­ED: The FDA sets a reg­u­la­to­ry speed record, pro­vid­ing a snap OK for Ver­tex's break­through triplet for cys­tic fi­bro­sis

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David Liu, Liu Group

David Liu un­veils newest ad­vance­ment in CRISPR tech: Prime edit­ing

The researcher behind base-editing is out with what some scientists are hailing as the biggest advancement in CRISPR technology since that 2016 breakthrough: “prime editing.” The new molecular gadget is capable of erasing any base pair and stenciling in another and cutting or adding long segments of DNA without breaking both strands of the helix.

David Liu, base editing pioneer and founder of Beam Therapeutics, published the findings in Nature alongside Andrew Anzalone. They estimated that the breakthrough “in principle” puts 89% of human diseases in purview — although experts cautioned that human therapies were a long way off.

Bhaskar Chaudhuri. Frazier Healthcare Partners

Fra­zier Health­care Part­ner­s' der­ma­tol­ogy up­start at­tracts a mar­quee syn­di­cate, $94M+ for 'in-be­tween' top­i­cal drug

For the past three years Frazier Healthcare Partners’ Bhaskar Chaudhuri has been carefully and quietly grooming Arcutis Therapeutics, a new dermatology play he co-founded to deliver topical formulations of well-known drugs. Now that the biotech is poised to enter Phase III, he’s being joined by a marquee syndicate for its $94.5 million Series C.

HBM Healthcare Investments, Vivo Capital, BlackRock, Omega Funds, Pivotal BioVentures, and Goldman Sachs jumped on board, joining Bain Capital Life Sciences, OrbiMed and RA Capital Management in backing Arcutis’ lead topical cream for plaque psoriasis.

A new com­pa­ny en­ters the Tec­fidera fight, of­fer­ing to kill two birds

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Take­da tees up $420M deal for celi­ac an­ti­dote, con­tin­u­ing R&D re­fo­cus

Sometime in the 1st century AD, a patient presented to Arataeus looking like a varicose ghost. He was “emaciated and atrophied, pale, feeble and incapable of performing any of his accustomed works,” the Greek physician wrote, with hollow temples and huge veins running all over his body.

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IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

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