Cit­ing lin­ger­ing con­cerns, FDA stops Evoke at the 10-yard line a month be­fore PDU­FA date

It took Evoke Phar­ma two years and a cou­ple rounds of fundrais­ing to slice and pull the da­ta for an NDA of its on­ly drug out of a Phase III wreck­age. But just a month be­fore the FDA was slat­ed to give its ver­dict, the biotech re­port­ed that the ef­fort might not have been enough af­ter all.

Dave Gony­er

The agency has seen de­fi­cien­cies dur­ing the ini­tial stage of its re­view, Evoke said on Mon­day, in three crit­i­cal sec­tions of the NDA. Al­though the com­ments out­lined in the mul­ti-dis­ci­pli­nary re­view are pre­lim­i­nary, the com­pa­ny con­ced­ed that “ap­proval of Gi­moti by the PDU­FA date of April 1, 2019 is un­cer­tain giv­en the let­ter.”

In­vestors of the mi­cro­cap biotech $EVOK were alarmed, trig­ger­ing a 57% land­slide in share price through­out the day, end­ing at $1.35.

An in­tranasal spray for­mu­la­tion of the FDA-ap­proved meto­clo­pramide, Gi­moti (EVK-001) was de­signed to over­come the lim­i­ta­tions of oral drug de­liv­ery in di­a­bet­ic gas­tro­pare­sis, giv­en that pa­tients of­ten ex­pe­ri­ence symp­toms — such as vom­it­ing and gas­tric emp­ty­ing — that hin­der ab­sorp­tion of the oral med­ica­tion. The drug al­so specif­i­cal­ly tar­gets women, who make up the ma­jor­i­ty of the pa­tient pop­u­la­tion.

Af­ter the drug failed a Phase III study in 2016, ham­mer­ing the stock, Evoke — too low on cash to con­duct an­oth­er late-stage tri­al — in­stead ran a se­ries of post-hoc analysas and con­clud­ed that Gi­moti was ef­fec­tive among those with mod­er­ate to se­vere symp­toms like nau­sea and up­per ab­dom­i­nal pain. The San Diego-based com­pa­ny al­so rolled out new phar­ma­co­ki­net­ic da­ta to show there are “sta­tis­ti­cal­ly sig­nif­i­cant dif­fer­ences in ex­po­sure be­tween men and women giv­en the same meto­clo­pramide dose.”

That was enough to earn them a place in the queue. But in its en­tire­ty, the FDA found the NDA’s de­scrip­tions on chem­istry, clin­i­cal and phar­ma­col­o­gy all want­i­ng:

  • Chem­istry: com­bi­na­tion prod­uct qual­i­ty con­trol and re­pro­ducibil­i­ty spe­cif­ic to the com­mer­cial­ly avail­able sprayer de­vice used with Gi­moti;
  • Clin­i­cal: lack of ad­e­quate in­for­ma­tion to sup­port sex-based ef­fi­ca­cy dif­fer­ences;
  • Clin­i­cal Phar­ma­col­o­gy: max­i­mum con­cen­tra­tion (Cmax) not with­in the pa­ra­me­ters for bioe­quiv­a­lence for ab­bre­vi­at­ed NDAs

The Evoke team is work­ing to eval­u­ate and ad­dress the FDA’s con­cerns, with a plan to re­spond be­fore the PDU­FA of April 1, CEO Dave Gony­er said.

Roger Perlmutter. Merck via webcast

'Our lega­cy mat­ter­s': Mer­ck maps out Keytru­da king­dom while spot­light­ing ad­vances in vac­cines, hos­pi­tal care

“You can for the mo­ment stop tak­ing notes. You can put down your pens and your pad. I have no slides. I have no sub­stan­tive da­ta. I have no pitch.”

So be­gan Roger Perl­mut­ter’s brief ap­pear­ance on­stage at Mer­ck’s first in­vestor day in five years, where he dived in­to the com­pa­ny’s his­to­ry dat­ing back to 1933. The first em­ploy­ees at Mer­ck Re­search Lab­o­ra­to­ries, hand­picked by founder George W. Mer­ck, were crit­i­cal to Mer­ck’s abil­i­ty to achieve clin­i­cal and com­mer­cial suc­cess.

How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

Eli Casdin, Casdin Capital

Eli Cas­din backs Codex­is' plat­form tech with $50M eq­ui­ty buy

About a month af­ter Codex­is notched a deal with No­var­tis $NVS, the Cal­i­for­nia com­pa­ny $CDXS on Thurs­day said long-time in­vestor Cas­din Cap­i­tal is putting up $50 mil­lion in a pri­vate place­ment, which puts the New York-based in­vest­ment firm in con­trol of more than 5% of the pro­tein en­gi­neer­ing play­er’s stock.

Eli Cas­din start­ed his epony­mous in­vest­ment firm in 2012 and dates his re­la­tion­ship with Codex­is back to at least a decade. About three years ago, Cas­din Cap­i­tal be­gan in­vest­ing in the in­dus­tri­al biotech com­pa­ny, af­ter it piv­ot­ed its fo­cus to the life sci­ences — un­der the aus­pices of new chief John Nicols — away from the en­er­gy in­dus­try.

In starved an­tibi­ot­ic field, Melin­ta soars as FDA grants speedy drug re­view

Such is the state of af­fairs in an­tibi­ot­ic land that the FDA agree­ing to pri­or­i­ty re­view an ap­pli­ca­tion to ex­pand the use of an an­tibi­ot­ic can rock­et up a stock more than two-fold.

On Wednes­day, Melin­ta Ther­a­peu­tics said its ap­proved an­tibi­ot­ic Baxdela had been grant­ed pri­or­i­ty re­view for use in com­mu­ni­ty-ac­quired bac­te­r­i­al pneu­mo­nia (CAPB). The FDA is ex­pect­ed to make its de­ci­sion by Oc­to­ber 24. Shares of the Con­necti­cut drug­mak­er $ML­NT cat­a­pult­ed, clos­ing up near­ly 224% at $6.41.

Brent Saunders at an Endpoints News event in 2017 — File photo

An­a­lyst call with Al­ler­gan ex­ecs stokes an­tic­i­pa­tion of a plan to split the com­pa­ny in ‘a month or two’

So what’s up at Al­ler­gan?

Ear­li­er this week the ubiq­ui­tous Ever­core ISI an­a­lyst Umer Raf­fat was on the line with com­pa­ny ex­ec­u­tives to probe in­to the lat­est on the num­bers as well as CEO Brent Saun­ders’ re­cent de­c­la­ra­tion that he’d be do­ing some­thing de­fin­i­tive to help long-suf­fer­ing in­vestors who have watched their shares dwin­dle in val­ue.

He came away with the im­pres­sion that a sig­nif­i­cant com­pa­ny split is on the way. And not on some dis­tant time hori­zon.

In­vestors pony up $476M for the lat­est round of biotech IPOs to hit the Street

Three biotechs — and a genome se­quenc­ing play­er — have caught the lat­est tide to the Gold Coast of IPOs, round­ing out the first half of 2019 with 23 new drug de­vel­op­ers mak­ing it on Nas­daq.

Most of these com­pa­nies filed their IPOs al­most si­mul­ta­ne­ous­ly, though we’re still wait­ing on word of fel­low class­mate Bridge­Bio’s pric­ing af­ter CEO Neil Ku­mar set the terms at $14 to $16 a share on Mon­day in search of a $240 mil­lion (or so) wind­fall. If he’s suc­cess­ful, that would take the one-week haul past the $700 mil­lion mark, a fresh sign that in­vestors’ en­thu­si­asm for new­ly coined pub­lic biotechs hasn’t cooled.

John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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Robert Forrester, Verastem

Ve­rastem CEO For­rester steps to the ex­it as the board hunts com­mer­cial-savvy ex­ec for the be­lea­guered biotech

Robert For­rester is step­ping down as CEO of Ve­rastem On­col­o­gy $VSTM just 8 months af­ter the com­pa­ny nabbed an ap­proval for du­velis­ib, a PI3K drug with a sto­ried past — and what ap­pears as not much of a fu­ture.

The biotech put out word this morn­ing that For­rester will take an ad­vi­so­ry role with Ve­rastem while COO Dan Pa­ter­son steps up to take charge of the lead­er­ship team and the board looks around for a new CEO.