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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'

 

Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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FDA ad­comm votes unan­i­mous­ly in sup­port of a J&J Covid-19 boost­er two months af­ter one-dose shot

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Friday voted 19-0 in favor of authorizing a second shot of J&J’s Covid-19 vaccine to follow at least two months after the initial dose.

Regulators don’t have to follow VRBPAC’s recommendation, but they almost always do. Considering that the CDC’s advisory committee has already been set to review the expanded EUA, VRBPAC’s recommendation is likely to be adopted.

In­cyte yanks EU ap­pli­ca­tion for PD-1 drug, cit­ing in­abil­i­ty to an­swer reg­u­la­tors' 'ma­jor con­cern­s'

Hoping to wedge its way into a crowded PD-(L)1 field, Incyte faced a huge setback when the FDA sent back its application for an initial indication earlier this year. Now, the drugmaker is giving up hope in Europe as well.

Incyte has withdrawn its EU application for PD-1 drug Zynyz, or retifanlimab, after saying it couldn’t adequately address the CHMP’s questions over survival data underpinning its market hopes in squamous cell carcinoma of the anal cavity, the EMA revealed.

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Tillman Gerngross, Adagio CEO

Q&A: Till­man Gern­gross ex­plains why his Covid mAb will have an edge over an al­ready crowd­ed field

If anyone knows about monoclonal antibodies, it’s serial entrepreneur, Adimab CEO, and Dartmouth professor of bioengineering Tillman Gerngross.

Even the name of Gerngross’ new antibody startup Adagio Therapeutics is meant to reflect his vision behind the development of his Covid-19 mAb: slowly, he said, explaining that “everyone else, whether it’s Regeneron, Lilly, or AstraZeneca, Vir, they all valued speed over everything.”

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