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.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

UP­DAT­ED: NGM Bio takes leap for­ward in crowd­ed NASH field

South San Francisco-based NGM Bio may have underwhelmed with its interim analysis of a key cohort from a mid-stage NASH study last fall — but stellar topline data unveiled on Monday showed the compound induced significant signs of antifibrotic activity, NASH resolution and liver fat reduction, sending the company’s stock soaring.

There are an estimated 50+ companies focused on developing drugs for non-alcoholic steatohepatitis, or NASH, a common liver disease that has long flummoxed researchers. The first wave of NASH drug developers struggled with efficacy as well as safety — and companies big and small have crashed and burned.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 72,900+ biopharma pros reading Endpoints daily — and it's free.

Mickey Kertesz, KidsandArtOrg via YouTube

Soft­Bank's newest, $165M biotech in­vest­ment looks for in­fec­tious traces in the blood

SoftBank has found its newest biotech investment.

The Japanese bank has invested $165 million into Karius, a company that uses blood tests to diagnose infectious diseases, as part of its new Vision Fund 2. The full scope of the new fund has yet to be announced, but the first and newly-beleaguered Vision Fund poured $100 billion into technology companies, including the biotechs Vir Biotechnology and Roivant and the sequencing company 10x Genomics.

Methicillin-resistant Staph aureus (Shutterstock)

FDA grants ‘break­through’ sta­tus to an­tibi­ot­ic al­ter­na­tive as Con­tra­Fect rush­es to join fight against su­per­bug

An experimental drug that promises to be the first anti-infective agent to prove superior to vancomycin — an antibiotic approved in 1958 — has notched the FDA’s “breakthrough” status.

ContraFect said the designation was based on Phase II data in which exebacase was tested against a superbug known as methicillin-resistant Staph aureus, or MRSA. In a subgroup analysis, the clinical responder rate at day 14 was 42.8% higher than that among those treated with standard of care, the company said (p=0.010).

Zhong Nanshan, CGTN via YouTube

Har­vard joins coro­n­avirus fight with $115 mil­lion and a high-pro­file Chi­nese part­ner

For two months, as the novel coronavirus swelled from a few early cases tied to a Wuhan market to a global epidemic, most of the world’s focus and dollars have flowed toward emergency initiatives: building vaccines at a record pace, plucking experimental antivirals out of freezers to see what sticks and immunizing mice for new antibodies.

Now a new and well-funded collaboration between Harvard and a top Chinese research institute will play the long game. In a 5-year, $115 million initiative backed by China Evergrande Group, researchers from the Harvard Medical School, Harvard T.H. Chan School of Public Health and Guangzhou Institute for Respiratory Health will study the virus in an effort to develop therapies against infections by the novel coronavirus, known as SARS–CoV-2, and to prevent new ones.

No­var­tis gets a boost in block­buster mul­ti­ple scle­ro­sis race with Roche

In the first step of what’s likely to be a long and uphill battle for the drugmaker, the FDA has accepted Novartis’s BLA submission for a new multiple sclerosis drug and given it priority review. The PDUFA date for the potential blockbuster drug is in June.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 72,900+ biopharma pros reading Endpoints daily — and it's free.

Juergen Horn

An­i­mal health vet Juer­gen Horn makes new an­ti­body play for pets, rak­ing $15M in Se­ries A haul

Zoetis forked over $85 million in 2017 to acquire Nexvet Biopharma and its pipeline of monoclonal antibodies. Juergen Horn, Nexvet’s former chief product development officer, has now secured $15 million for his own biologic company for animals: Invetx.

Buoyed by emerging advances in gene therapies for humans, scientists have started looking at harnessing the technology for animals setting up companies such as Penn-partnered Scout Bio and George Church-founded Rejuvenate Bio. But akin to Nexvet, Invetx is working on leveraging the time-tested science of monoclonal antibodies to treat chronic diseases that afflict man’s best friend.

As coro­n­avirus out­break reach­es 'tip­ping point,' GSK lends ad­ju­vant tech to Chi­nese part­ner armed with pre­clin­i­cal vac­cine

As the coronavirus originating out of Wuhan spreads to South Korea, Italy and Iran, stoking already intense fears of a pandemic, GlaxoSmithKline has found another pair of trusted hands to place its adjuvant system. China’s Clover Biopharmaceuticals will add the adjuvant to its preclinical, protein-based vaccine candidate against SARS-CoV-2.

Clover, which is based in the inland city of Chengdu, boasts of a platform dubbed Trimer-Tag that produces covalently-trimerized fusion proteins. Its candidate, COVID-19 S-Trimer, resembles the viral spike (S)-protein found in the virus.