#AS­CO17: Epizyme will hunt a quick OK for tazeme­to­stat, de­spite dis­ap­point­ing an­a­lysts with new da­ta

Robert Baze­more, CEO Epizyme

As the dust be­gan to set­tle from the AS­CO ab­stract drop last night, sev­er­al an­a­lysts weighed in not­ing their cau­tious lack of op­ti­mism for Epizyme’s lat­est look at the da­ta for tazeme­to­stat, its EZH2 in­hibitor now in Phase II for ep­ithe­lioid sar­co­ma.

RBC’s Matthew Eck­ler not­ed the lat­est da­ta were “be­low our ex­pec­ta­tions.”

For Ge­of­frey Porges at Leerink: “These re­sults squeaked over the tri­al’s hur­dle for pro­ceed­ing to co­hort ex­pan­sion and the 13% ORR is be­low our men­tal thresh­old of a 20-30% re­sponse rate.”

But ear­ly Thurs­day Epizyme $EPZM was up and run­ning with an en­thu­si­as­tic as­sess­ment of its chances for gain­ing an ac­cel­er­at­ed FDA OK af­ter fil­ing for an ap­proval next year on the Phase II.

The biotech’s shares were down 4% in pre-mar­ket trad­ing Thurs­day.

Not­ed the Cam­bridge, MA-based biotech:

Based on dis­cus­sions with the FDA, the Com­pa­ny has iden­ti­fied a path to sub­mis­sion for ac­cel­er­at­ed ap­proval of tazeme­to­stat based on the 60-pa­tient co­hort from its Phase II study, and will tar­get a New Drug Ap­pli­ca­tion sub­mis­sion in 2018.

The an­a­lysts aren’t rul­ing out a suc­cess here. But sev­er­al are plain­ly dis­ap­point­ed with where Epizyme ap­pears to be right now.

“We had pre­vi­ous­ly writ­ten that we were pos­i­tive in­to this da­ta re­lease, with the ex­pec­ta­tion that the ob­jec­tive re­sponse rate would ex­ceed 20% in this co­hort,” Eck­ler wrote. “Bar­ring im­prove­ments in fu­ture da­ta pre­sen­ta­tions, we were ob­vi­ous­ly wrong. Tazeme­to­stat showed a 13% ORR (0 CRs, 4 PRs) in 31 pa­tients en­rolled in the ep­ithe­lioid sar­co­ma co­hort (ab­stract # 11058), be­low pre­vi­ous­ly re­port­ed Phase I da­ta….”

Where an­a­lysts were see­ing is­sues, though, Epizyme de­ci­phered new op­por­tu­ni­ties.

“Ep­ithe­lioid sar­co­ma is a dif­fi­cult can­cer for sar­co­ma on­col­o­gists like me to treat due to there be­ing few avail­able ther­a­peu­tic op­tions, which are as­so­ci­at­ed with lim­it­ed ben­e­fit and chal­leng­ing side ef­fects for pa­tients,” said Dr. Mri­nal M. Gounder, lead in­ves­ti­ga­tor, in a pre­pared state­ment. “INI1 loss is a defin­ing fea­ture of ep­ithe­lioid sar­co­ma and the mech­a­nism of tazeme­to­stat makes this a com­pelling agent. These da­ta show en­cour­ag­ing ac­tiv­i­ty of tazeme­to­stat as char­ac­ter­ized by ob­jec­tive re­spons­es, du­ra­tion of re­spons­es and pro­longed dis­ease sta­bi­liza­tion, and I look for­ward to its con­tin­ued de­vel­op­ment.”

“Bring­ing tazeme­to­stat to pa­tients is our num­ber one pri­or­i­ty,” said Robert Baze­more, pres­i­dent and chief ex­ec­u­tive of­fi­cer of Epizyme.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Roche nabs front­line OK for Avastin/Tecen­triq in com­mon liv­er can­cer, best­ing an old Bay­er drug

For the first time in 12 years, the FDA has approved a new frontline treatment for the most common form of liver cancer.

The agency okayed a combination of Roche’s anti-VEGF antibody Avastin and their immunotherapy Tecentriq for patients with unresectable or metastatic hepatocellular carcinoma (HCC). The approval comes two weeks after Roche and their big biotech sub Genentech published Phase III results showing the combo improved both progression-free survival and, crucially, helped patients live longer than the long-running standard-of-care, Bayer’s Nexavar.

Ver­sant-backed, Bris­tol My­ers-stamped Re­pare Ther­a­peu­tics guns for $100M IPO

With a Bristol Myers Squibb endorsement in tow, Versant-backed cancer drug developer Repare Therapeutics has set its sights on a Nasdaq debut.

On Friday, the Montreal-based company with operations in Cambridge, Massachusetts that is yet to enter the clinic, unveiled plans for a $100 million IPO, banking on its “synthetic lethality” platform.

The basic idea is to target the genetic basis of tumors, a common idea across precision oncology medicines. But instead of targeting the perpetrator mutation directly, the compound is designed to go after the other gene in the gene pair. The rationale is based on the decades-old genetic principle that indicates two mutations are lethal only when combined together.

Jean-Jacques Bienaime, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

Lynn Seely, Myovant CEO

My­ovant’s re­l­u­golix wins a piv­otal prostate can­cer show­down with an old stan­dard — com­ing down to the wire on ap­provals

Myovant $MYOV has rounded the final turn in its development race to get relugolix down to the regulatory wire at the FDA. And the biotech joined the virtual crowd at ASCO with the kind of data needed to keep the investor crowd’s attention.

Much of the attention on the drug has been focused on uterine fibroids, where AbbVie just scored a regulatory win for their rival drug Oriahnn (elagolix) as the biotech posted results in prostate cancer at the ASCO meeting.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

File photo (Endpoints News)

In a sting­ing set­back, Pfiz­er’s can­cer block­buster Ibrance flops in key ad­ju­vant set­ting

One of Pfizer’s top, long-running R&D catalysts has gone up in smoke, and it took an $11 billion bite of their market cap in the process.

The monitoring committee determined that Pfizer’s adjuvant study using Ibrance combined with standard endocrine therapy in an adjuvant setting for early-stage breast cancer has officially failed to make the cut. The combo failed to beat the standard alone, tripping over the futility analysis. And the Pfizer team will now wrap the study early after pumping up hopes that their blockbuster cancer therapy could find billions more by proving its efficacy for disease-free survival in a major area — something AstraZeneca just accomplished with Tagrisso to great fanfare.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.