Epizyme hits the ground run­ning with sec­ond — and cru­cial — OK for Tazverik, in fol­lic­u­lar lym­phoma

When Epizyme scored the first-ever ap­proval for tazeme­to­stat, in ep­ithe­lioid sar­co­ma, ex­ecs made it clear it was on­ly an ap­pe­tiz­er, some­thing to whet the ap­petites of reg­u­la­tors, in­vestors and physi­cians to the po­ten­tial of the methyl­trans­ferase in­hibitor. Five months lat­er, they are ready to serve the main course.

She­fali Agar­w­al

The biotech got all the help from the FDA to ex­pand the la­bel for Tazverik, go­ing through pri­or­i­ty re­view to land an ac­cel­er­at­ed ap­proval in two in­di­ca­tions of fol­lic­u­lar lym­phoma:

  • Adult pa­tients with re­lapsed or re­frac­to­ry FL whose tu­mors are pos­i­tive for an EZH2 mu­ta­tion as de­tect­ed by an FDA-ap­proved test and who have re­ceived at least two pri­or sys­temic ther­a­pies
  • Adult pa­tients with re­lapsed or re­frac­to­ry FL who have no sat­is­fac­to­ry al­ter­na­tive treat­ment op­tions

The OK was an­chored on over­all re­sponse rate and du­ra­tion of re­sponse in a Phase II, which en­com­passed both mu­tat­ed and wild-type EZH2.

“As a re­minder, in the EZH2 MT co­hort, the re­sponse rate was 69%, while in the EZH2 WT co­hort, the re­sponse rate was rough­ly half, at 35%,” SVB Leerink an­a­lyst An­drew Berens wrote at the news of the pri­or­i­ty re­view. “While we think that the ben­e­fit in the wild-type pa­tients is still com­pelling enough for some pa­tients to con­sid­er ther­a­py, it is less com­pelling than that seen in the EZH2m co­hort.”

Robert Baze­more

That could mean that in prac­tice, clin­i­cians will want a EZH2 test be­fore pre­scrib­ing the drug, re­duc­ing the sig­nif­i­cance of the broad la­bel.

As such, he mod­els $17.5 mil­lion in FL sales and $5.6 mil­lion of ES sales for 2020.

CMO She­fali Agar­w­al, though, is clear­ly pleased with what they achieved with the “very ef­fi­cient” NDA, lever­ag­ing da­ta that have al­ready been sub­mit­ted with the ES pack­age. Es­pe­cial­ly con­sid­er­ing that FL, a sub­type of non-Hodgkin lym­phoma that ac­counts for 20% to 30% of cas­es, is of­ten con­sid­ered a chron­ic dis­ease.

“In our view, there re­mains no clear stan­dard of care in the re­lapsed and/or re­frac­to­ry FL pop­u­la­tion as not all pa­tients ben­e­fit from to­day’s avail­able ther­a­pies,” she said in a state­ment. “Based on this la­bel, physi­cians will have the abil­i­ty to use their clin­i­cal dis­cre­tion to pre­scribe TAZVERIK for their re­lapsed or re­frac­to­ry pa­tients re­gard­less of EZH2 mu­ta­tion­al sta­tus and with­out re­gard to a spe­cif­ic line of treat­ment where oth­er op­tions are not sat­is­fac­to­ry.”

CEO Robert Baze­more added that Epizyme has al­ready “seam­less­ly” ex­pand­ed the com­mer­cial team, armed with what they be­lieve is a no­table safe­ty pro­file and com­pelling op­tion for oral, at-home ad­min­is­tra­tion.

A full ap­proval will hinge up­on a 500-pa­tient con­fir­ma­to­ry tri­al Epizyme is con­duct­ing that pairs Tazverik with Revlim­id and rit­ux­imab (Rit­ux­an) for FL pa­tients in the sec­ond or lat­er lines.

A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Su­per-se­cre­tive an­ti-ag­ing biotech Cal­i­co tees up the first vis­i­ble clin­i­cal tri­al of an ex­per­i­men­tal drug. And it’s for can­cer?

Over the past 7 years, Calico has been so much more than your average, run-of-the-mill secretive biotech players. It’s a riddle, wrapped in a mystery, inside an enigma, to repurpose an old Winston Churchill line dating from the time he confronted the Iron Curtain surrounding Stalin’s thoughts.

Launched by industry legend Art Levinson of Genentech fame, with the infinitely deep pockets of Google for support, one of the few big headlines the anti-aging biotech has sparked focused on a major alliance with AbbVie — a giant outfit that conversely likes to show off its drug prospects whenever it can. Together, they’ve been focused on diseases that limit life span — quite an arc of ailments.

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RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

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Joe Biden (Carolyn Kaster, AP Images)

What about the Ger­man ne­go­ti­a­tion mod­el? Biden steers drug pric­ing de­bate to a show­down

From an ill-fated proposal to ban rebates for pharmacy benefit managers to an executive order demanding a “most-favored-nation price” for Medicare, if nothing else President Donald Trump has introduced Americans to a flurry of ideas to rein in pharma, an industry he once accused of “getting away with murder.” And now we’re getting the first glimpse of what a Joe Biden presidency might mean for prescription drug pricing.

Covid-19 roundup: Pars­ing Bourla, a top an­a­lyst sees im­proved chances for Pfiz­er vac­cine; Fau­ci: No sur­prise that Trump was hit by Covid-19

With a medley of adverse events hobbling the late-stage development of vaccines and drugs, Pfizer CEO Albert Bourla’s latest — extended — timeline for the mRNA approach they’re working on with BioNTech is giving some top analysts added confidence that the pharma giant can come up with the regulatory goods next month.

Parsing Bourla’s language in his comments last week, SVB Leerink’s Geoffrey Porges notes that Bourla’s decision to say they “may” be able to nail down the positive efficacy of their vaccine in a matter of days — a big change from his earlier certainty — may also indicate a delay on that to early November.

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IN8bio CEO William Ho (IN8bio)

Bring­ing their ge­net­i­cal­ly mod­i­fied gam­ma delta T cells to Nas­daq, IN8bio files for $86M IPO

The biotech IPO parade continues marching forward as 2020 turns toward the fourth quarter.

IN8bio, a New York-based company focused on genetically modified gamma delta T cell therapies, filed to go public Friday seeking an $86 million raise. The company has two clinical-stage candidates being studied in glioblastoma and leukemia, respectively.

By any stretch of the imagination, 2020 has already been a huge year for biotech, and nowhere does it appear more obvious than the vast amounts of companies hitting the public market.

News brief­ing: Ab­b­Vie and Roche's Ven­clex­ta scores an­oth­er FDA OK; Im­muno­Gen nabs Chi­na deal with $40M cash

AbbVie and Roche’s Venclexta has gotten a new FDA thumbs up.

The pair announced Monday that regulators have approved the drug in combination with azacitidine or low-dose cytarabine for newly-diagnosed acute myeloid leukemia in adults who are 75 or older or those who can’t undergo intensive chemotherapy. This follows the drug’s accelerated approval in 2018 and positive data from two Phase III confirmatory trials.

James Sabry (Roche)

Roche's James Sabry inks his sec­ond AI deal in back-to-back pacts — this time part­ner­ing Genen­tech with Stan­ford spin­out Gen­e­sis Ther­a­peu­tics

Less than a week after Roche joined forces with Dyno Therapeutics to develop gene therapies using artificial intelligence, its giant subsidiary Genentech is hopping on the AI bandwagon with a different player.

Genentech has inked a deal with Stanford spinout Genesis Therapeutics to harness its AI power for drug development and discovery. Genesis is getting an upfront payment and milestones, but the companies are keeping the details under wraps for now. The Burlingame, CA-based biotech also stands to earn future royalties on any approved Genentech drugs that come from the deal.

En­do pays $658M in a fur­ther bet on col­la­gen-based med­i­cines, buy­ing out long­time bio­phar­ma part­ner

A little less than two years after Endo Pharmaceuticals’ deal to purchase Somerset Therapeutics fell through, the Irish drugmaker is returning to the well with a much bigger acquisition.

Endo has agreed to buy BioSpecifics Technologies for a whopping $658 million, the two companies announced Monday, in the culmination of a research agreement signed all the way back in 2004. Endo will purchase all of BioSpecifics’ outstanding stock for about $540 million, valuing the company at $88.50 per share — a 45% premium on the $61.02 share price at which the company closed on Friday.