Let's re­think Clin­ton's war on phar­ma; The FDA can't or­der the ge­nie back in­to the mag­ic lamp

End­points as­sess­es the big bio­phar­ma R&D sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

 Sur­vey says!

Ever since I first start­ed think­ing about part­ner­ing up with Ar­salan Arif on End­points News, I want­ed to do a quar­ter­ly sur­vey of a sub­stan­tial num­ber of biotech ex­ecs to keep my thumb on the in­dus­try’s pulse. Now, 4 months from our launch, we did the first E100 sur­vey that found the pulse is quite strong. Yes, there is some flut­ter­ing ev­i­dent when you bring up top­ics like fu­ture fi­nanc­ings, IPOs, ac­cess to tal­ent, and so on. But by and large, biotechs are ex­pand­ing their teams and plan­ning to go in­to 2017 with am­bi­tious plans. We’ll fol­low up with a spe­cial 2017 fore­cast in Jan­u­ary. No one knows this in­dus­try bet­ter than the E100 com­bined. And you won’t want to miss what they have to say in the fu­ture.

 Hillary Clin­ton’s war on phar­ma was no chance event.

The lat­est batch of emails from Wik­iLeaks in­cludes the back­ground dis­cus­sion that oc­curred among Hillary Clin­ton’s cam­paign staff around her at­tack of Mar­tin Shkre­li, who still wears the black hat in biotech af­ter he de­cid­ed to jack up the price of Dara­prim by more than 5,000%. Clin­ton called it price goug­ing in a Tweet, trig­ger­ing a rout in biotech shares as the im­pli­ca­tions over some kind of price con­trols sank in. “FYI—We have start­ed the war with Phar­ma!!” ex­claimed se­nior ad­vis­er Ann O’Leary. “Great!” replied Mandy Grun­wald. There are bet­ter ways to have this dis­cus­sion than sin­gling out one bad ac­tor to slime an in­dus­try. Biotech is not Mar­tin Shkre­li, and the price of re­al in­no­va­tion in drugs will not be cheap. But that’s not price goug­ing. It’s time we sep­a­rat­ed drug de­vel­op­ment and the price of new drugs from the no­to­ri­ous prac­tice of rou­tine price hikes — or overnight prof­i­teer­ing — on old ther­a­pies. Maybe Clin­ton could Tweet about it some time. There’s no ques­tion now that she’s the odds-on fa­vorite to win this elec­tion.

 Kite is fly­ing high as its pi­o­neer­ing bi­o­log­ics ap­pli­ca­tion heads to the FDA

When Kite Phar­ma’s Arie Bellde­grun took to the stage in New York ear­li­er this week to spear­head a dis­cus­sion on the com­pa­ny’s am­bi­tious plans to launch the world’s first CAR-T, you could hear plen­ty of grum­bling on the side­lines. The da­ta are ear­ly. We all know that 3 months of re­spons­es in their piv­otal study is a quick read. Some want to wait for 6 months. We’ll see how that dis­cus­sion plays out. But while the com­pe­ti­tion at Juno and No­var­tis has fall­en well be­hind, Kite con­tin­ues to ag­gres­sive­ly press for­ward, not will­ing to give an inch un­less reg­u­la­tors re­quire it. Win or lose, you have to ad­mire the spir­it and com­pe­tence be­hind that.

 The FDA can’t make the Sarep­ta fall­out just dis­ap­pear

Now that the FDA has ac­tu­al­ly ap­proved Sarep­ta’s eteplirsen for Duchenne mus­cu­lar dy­s­tro­phy, the agency is try­ing to blunt the fall­out. Step one, for se­nior FDA of­fi­cial John Jenk­ins, was to spell out all the bad things that the biotech had done and then es­sen­tial­ly in­sist that no oth­er biotech will win an OK un­der sim­i­lar cir­cum­stances. Whip up ad­vo­cates to the ex­tent they at­tack FDA re­view­ers? Bad idea, says Jenk­ins. Use sus­pect da­ta from a woe­ful­ly in­ad­e­quate study to in­sist on an ap­proval? Uh-uh. The list goes on and on. What­ev­er the FDA says now, though, will do noth­ing to dis­pel the lessons to be learned from eteplirsen. And the next time a biotech comes along with an an­gry mob of sup­port­ers be­hind it, the FDA – and Janet Wood­cock – will have no one to blame but them­selves. This sto­ry is just be­gin­ning.

CRISPR: Los­ing some of the glit­ter

It was in­evitable. The third big IPO in gene edit­ing didn’t play out all that well, for­mal­ly end­ing gene edit­ing’s rep as the hottest thing in biotech. Noth­ing, of course, stays hot. But CRISPR Ther­a­peu­tics had to be dis­ap­point­ed this week that its tim­ing was off. Ed­i­tas and In­tel­lia had both made it out in big IPOs, on­ly to see their shares fade over the year. Gene edit­ing is still a pre­clin­i­cal field, with lots of changes in the tech to come. De­vel­op­ment ef­forts will be slow and painstak­ing, and it’s ask­ing a lot of in­vestors to stick through every­thing that has to hap­pen be­fore we see any ac­tu­al prod­ucts come out of it. Rev­o­lu­tions nev­er come fast or easy in biotech. Now comes the hard part. But it’s al­so the most valu­able stage for what comes next.

Gilead miss­es, again.

Gilead has plen­ty of re­sources to do R&D right. So it was kind of odd­ly fun­ny to see its messy da­ta from a tiny mid-stage pro­gram on a slate of in­di­ca­tions for GS-4997, in­clud­ing a pos­i­tive snap­shot for NASH. The prob­lem, though, was that the pic­ture proved too fuzzy to make out any ex­act ideas about its po­ten­tial. Gilead needs to prove now that it can do some­thing dra­mat­i­cal­ly great. This lit­tle stum­ble comes at a par­tic­u­lar­ly bad time.

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.

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.

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.

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.

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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Ken Frazier appears before the Senate Committee on Finance for a hearing on prescription drug pricing on Capitol Hill in Washington, DC, February 26, 2019. Chris Kleponis for CNP via AP Images

Who’s next in line to suc­ceed Ken Fra­zier as CEO of the Keytru­da-blessed Mer­ck?

When Merck waved off a looming forced retirement for Ken Frazier last September, the board cited flexibility in CEO transition as a key factor in the decision. Having Frazier — who’s also chairman of the company — around beyond his 65th birthday in 2019 would ensure they install the best person at the best time, they said.

The board has evidently begun that process with a clear preference for internal candidates, sources told Bloomberg. CFO Robert Davis, chief marketing officer Michael Nally, and chief commercial officer Frank Clyburn are all in the running, according to an insider.

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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.