FDA waves Epizyme's $186K rare can­cer drug through to mar­ket — now get ready for the sec­ond act

Af­ter win­ning the hearts of the ex­pert pan­el con­vened by the FDA de­spite a bleak in-house re­view and a check­ered de­vel­op­ment his­to­ry, Robert Baze­more has steered Epizyme to its first-ever OK for a rare can­cer drug.

Robert Baze­more

The ap­proval in ep­ithe­lioid sar­co­ma sets tazeme­to­stat, now Tazverik, up nice­ly for a quick ex­pan­sion to fol­lic­u­lar lym­phoma — a much big­ger in­di­ca­tion for which the biotech has just sub­mit­ted an NDA.

With that and oth­er am­bi­tions in mind, Epizyme has priced the drug — an in­hibitor of the EZH2 en­zyme — at $15,500 per month, or $186,000 for a year. A field-based team of 19 is al­ready in place to push Tazverik to a spe­cial­ty dis­tri­b­u­tion net­work with­in 10 days, chief strat­e­gy & busi­ness of­fi­cer Matthew Ros said in a con­fer­ence call.

Ep­ithe­lioid sar­co­ma, or ES, is an ex­treme­ly rare sub­type of soft tis­sue sar­co­ma. Per Epizyme es­ti­mates around 800 are liv­ing with the con­di­tion in the US, many of them young adults. Tazverik’s la­bel cov­ers its use in pa­tients 16 or old­er with metasta­t­ic or lo­cal­ly ad­vanced dis­ease who are not el­i­gi­ble for the stan­dard treat­ment: com­plete re­sec­tion.

“There are 300 ES, INI- pa­tients in the U.S. who qual­i­fy for the la­bel,” Cowen an­a­lyst Yaron Wer­ber wrote in a note. “Our es­ti­mates pre­dict mar­ket share ris­ing from 8% in FY20 to 65% in FY25.”

Matthew Ros

Epizyme’s suc­cess here once again un­der­scores the FDA’s will­ing­ness to hand out ac­cel­er­at­ed ap­provals in the face of tough can­cers that leaves pa­tients with few to no op­tions. In the sin­gle-arm study that formed the ba­sis of ap­proval, the over­all re­sponse rate was 15% among 62 pa­tients who took 800 mg of Tazverik twice a day. That’s a to­tal of nine pa­tients. Mean­while, 37% had a se­ri­ous ad­verse event and 34% need­ed to sus­pend dos­ing due to tox­i­c­i­ty. And that’s af­ter Epizyme had to con­tend with a clin­i­cal hold and a de­ci­sion to scrap the monother­a­py  pro­gram in dif­fuse large B-cell lym­phoma.

But the FDA high­light­ed how 50% of ES pa­tients would have had metasta­t­ic dis­ease at the time of di­ag­no­sis, which would be life-threat­en­ing.

“Ep­ithe­lioid sar­co­ma ac­counts for less than one per­cent of all soft tis­sue sar­co­mas,” Richard Paz­dur, the pow­er­ful di­rec­tor of the FDA’s On­col­o­gy Cen­ter of Ex­cel­lence, re­marked. “Un­til to­day, there were no treat­ment op­tions specif­i­cal­ly for pa­tients with ep­ithe­lioid sar­co­ma.”

She­fali Agar­w­al

Fol­lic­u­lar lym­phoma (FL) is a much dif­fer­ent sto­ry, with mul­ti­ple play­ers equipped with new tech such as CAR-T and bis­pecifics vy­ing for the mar­ket. But Epizyme be­lieves it has got­ten a head start.

“This is a very ef­fi­cient NDA,” CMO She­fali Agar­w­al said re­gard­ing their sub­mis­sion for third-line fol­lic­u­lar lym­phoma. “It’s a small NDA be­cause we cross ref­er­enced most of the oth­er mod­ules like pre­clin­i­cal, CMC and clin­i­cal phar­ma­col­o­gy, so all that has been re­viewed with ES. Now all that’s left in the NDA is just the clin­i­cal as­pect.”

Michael Yee of Jef­feries has pre­dict­ed that Tazverik can bring in $500 mil­lion-plus in peak sales as an FL ther­a­py — while ES is not re­al­ly in the mod­el.

Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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Cel­lec­tis slammed af­ter pa­tient dies and FDA slaps a hold on their tri­al for an off-the-shelf CAR-T for mul­ti­ple myelo­ma

Cellectis was slammed after the market close on Monday as the biotech reported that the FDA demanded it hit the brakes on their MELANI-01 trial for their off-the-shelf cell therapy UCARTCS1A after one of the patients in the study died of treatment-related cardiac arrest.

The multiple myeloma patient had previously been treated unsuccessfully with various therapies, noted the biotech, and had been given dose level two (DL2) of their allogeneic CAR-T.

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Shoshanna Shendelman, Applied Therapeutics CEO (Applied Therapeutics)

A lit­tle biotech slaps back at a 'crim­i­nal' short at­tack, vow­ing to pur­sue a pros­e­cu­tion of their case

As short attacks go, Biotech Research Partners’ assault on Applied Therapeutics’ “cherry picked” data and a variety of so-called red flags didn’t cause a whole lot of damage. Ahead of the July 4 holiday, its shares $APLT were dinged and showed signs of quick recovery.

But that didn’t stop an incendiary response, as the biotech swung into action bright and early Monday morning.

Applied Therapeutics accused the authors of the short report of manipulating graphs and figures, misrepresenting data and included factual misrepresentations — all of which added up, in their view, to fraud.

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UP­DAT­ED: Im­munomedics spells out PFS ben­e­fit of Trodelvy in mTNBC, hunt­ing a full OK just weeks af­ter ac­cel­er­at­ed ap­proval

By the time the FDA finally granted an accelerated OK for Immunomedics’ Trodelvy, we already got a very strong hint that their confirmatory Phase III study in metastatic triple-negative breast cancer was a success.

That’s because the independent data safety monitoring committee recommended that the trial be stopped early. But just what pointed them to the conclusion was still unclear.

“We do not know the totality of their decision other than it’s pretty evident that the primary endpoint was met; otherwise they could not request to halt the study,” Behzad Aghazadeh, the executive chairman, told Endpoints News at the time.

FDA lifts par­tial hold on ADC, clear­ing way for a 2021 read­out and two po­ten­tial near-term ap­provals

In throwing $267 million and a multi-billion dollar valuation at ADC Therapeutics for their May IPO, investors were betting that the partial hold the FDA had just placed on their second lead drug would prove immaterial. Time, it appears, has proven them right.

The FDA has lifted the partial hold, ADC said this morning. The move clears the way for an anticipated 2021 readout from their second pivotal study, a Phase II trial testing their experimental antibody drug conjugate camidanlumab tesirine, or Cami, in Hodgkin’s lymphoma.

Bill Haney, Dragonfly CEO (Dave Pedley/Getty Images for SXSW)

A boom­ing Drag­on­fly is tak­ing its TriN­KETs to Copen­hagen as the lat­est Bris­tol My­ers pact spurs ex­pan­sion plans — out­side the US

Bristol Myers Squibb is making a habit out of collaborating with the crew at Dragonfly, adding their 3rd deal in a series that now will take them into newly charted R&D territory. And the fast-growing team at the Cambridge-based biotech is adding a facility in Copenhagen for its next growth spurt, where the government is making it easy to recruit scientists internationally as the U.S. throttles back.

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Covid-19 roundup: Teamed up with NIH, Re­gen­eron launch­es PhI­II pre­ven­tion tri­al for an­ti­body cock­tail

As Regeneron moves its antibody cocktail into Phase II/III trials testing REGN-COV2 as a treatment for both hospitalized and non-hospitalized patients with Covid-19, the biotech is also starting a Phase III in the prevention setting.

The National Institute of Allergy and Infectious Diseases — which orchestrated the large, randomized study for remdesivir that produced positive results — will jointly run the study.

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Douglas Love, Annexon CEO (Annexon)

IPO bound? Ac­tu­al­ly, An­nex­on was al­ready prepped and primed to toss its S-1 to Wall Street as in­vestors ral­lied

The Wall Street IPO shuffle generally calls for a little distance between the crossover ante and the Wall Street double, but with the window on the street wide open and biotech sizzling hot, who’s waiting?

The crew at Annexon didn’t leave anyone in suspense for long about their IPO plans. A day after the Bay Area biotech with clinical plans to target neurodegeneration quietly unveiled a $100 million raise, they were back with an S-1 outlining a pitch to double that — or more.

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Jean-Paul Clozel, Idorsia CEO (Patrick Straub/Keystone via AP Images)

Sec­ond PhI­II study for Idor­si­a's sleep drug re­turns pos­i­tive re­sults, but al­so rais­es new ques­tions

Following a successful Phase III study in April showcasing the safety and potential of its new sleep drug, Idorsia posted some mixed news in the second Phase III study, but that won’t stop a planned filing aimed at regulatory approval.

The drug, a dual orexin receptor antagonist (DORA) called daridorexant, was found to significantly improve sleep maintenance and subjective total sleep time in 25 mg doses, replicating results from the first Phase III study. However, improvements in sleep onset and daytime functioning narrowly missed statistical significance, despite numerical consistency with the April study.