#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.

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But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

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Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

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The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.