Hillary Clin­ton's War on Phar­ma II; A call for com­mon sense re­port­ing on Alzheimer's R&D

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

Hillary Clin­ton want­ed that war with phar­ma, bad­ly

For all the back­lash around Mar­tin Shkre­li’s brief, dis­as­trous turn as CEO of Tur­ing, the Tweet from Hillary Clin­ton con­demn­ing his price goug­ing stands out for the wide­spread dam­age it did to biotech stocks. There are lots of rea­sons why the biotech stock in­dex­es have tanked over the past year. And it’s easy to see why that may have been over­due. But hav­ing a pres­i­den­tial can­di­date use Shkre­li to de­clare a “war on pharma” sets the stage for the bat­tle to con­tin­ue in DC af­ter the votes are count­ed. Try­ing to score cheap points with vot­ers in a pri­ma­ry is a bad way to es­tab­lish pol­i­cy. Yet that is what we are look­ing at if Clin­ton is elect­ed. The US — and the bad­ly frac­tured health sys­tem that needs re­form­ing — de­serve bet­ter. And biotech has enough prob­lems right now with­out get­ting caught up in the war. Let’s urge Clin­ton to take a more re­spon­si­ble stand if she wins the big elec­tion Tues­day.

The SEC sends an im­por­tant mes­sage on who biotech ac­coun­tants are work­ing for

We re­port­ed this week that the SEC wants to sanc­tion the PwC ac­coun­tant who let Steven Bur­rill go his mer­ry way for years be­fore he was caught – by oth­er means – loot­ing the biotech fund he man­aged. The SEC has al­ready dealt with Bur­rill, who was forced to re­im­burse funds used for per­son­al ex­pens­es, but en­forcers want to send a mes­sage here to oth­er ac­coun­tants do­ing the books for biotechs and all com­pa­nies. You have a pro­fes­sion­al re­spon­si­bil­i­ty to check out why ex­ecs are, say, ad­vanc­ing big­ger and big­ger sums for work not yet done. Sums that may nev­er have been war­rant­ed un­der any cir­cum­stances. And you can’t be waved away by the same ex­ecs who may have some­thing to hide. Bur­rill’s in­vestors de­served bet­ter. What­ev­er the in­di­vid­ual mer­its of these charges, let’s hope that mes­sage comes through loud and clear.

New in­ter­nal FDA doc­u­ments un­der­score Wood­cock’s un­in­formed de­ci­sion to back Sarep­ta drug

As I’ve said be­fore, there’s no rea­son to ex­pect the Sarep­ta con­tro­ver­sy to go away any­time soon. The lat­est doc­u­ment drop from the FDA on the in­ter­nal war over Janet Wood­cock’s in­sis­tence on an ap­proval now in­cludes ac­cu­sa­tions that she ig­nored in­ter­nal as­sess­ments on its Duchenne drug Ex­ondys 51 from se­nior of­fi­cials. This was noth­ing more than a gut-lev­el de­ci­sion which clear­ly de­vi­at­ed from agency stan­dards for an ap­proval. And FDA Com­mis­sion­er Robert Califf un­wise­ly de­cid­ed to let her have her way, open­ing up a new and dan­ger­ous path for oth­ers to fol­low. Let’s all con­tin­ue to hope this drug is as safe and ef­fi­ca­cious as the com­pa­ny and pa­tients ad­vo­cates in­sist it is. But ap­prov­ing an ex­per­i­men­tal drug like this was worse than a mis­take. It was bad pol­i­cy mak­ing at its worse.

Let’s re­main pro­fes­sion­al­ly skep­ti­cal about Alzheimer’s drug re­search

Look­ing over all the ways main­stream me­dia fawns over ear­ly-stage Alzheimer’s drug re­search has be­come some­thing of a hob­by of mine. And this week we were treat­ed to the full smor­gas­bord of gulli­bil­i­ty with a string of un­re­served re­views for the ear­ly an­i­mal and hu­man da­ta that’s be­come avail­able for Mer­ck’s late-stage BACE drug. Some of the UK pa­pers (hel­lo Dai­ly Mail) have a ten­den­cy to jump from the slight­est in­di­ca­tion of ef­fi­ca­cy in small groups to won­der­ing when the new cure will be ap­proved. We saw the same thing re­cent­ly when Bio­gen tout­ed its ear­ly da­ta on ad­u­canum­ab. But it’s a cru­el hoax on the pa­tients and their fam­i­lies. There are good rea­sons why Alzheimer’s has been an R&D dis­as­ter zone for more than a decade. Let’s wait for some con­vinc­ing da­ta be­fore herald­ing the next big break­through. We’re a long way from a cure. And that’s a hard truth. Alzheimer’s de­vel­op­ment has a long, long way to go, un­der the best of cir­cum­stances.

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.