Mer­ck R&D chief Roger Perl­mut­ter of­fers some heavy­weight R&D glam to a lit­tle up­start biotech out to cure Duchenne MD

Back when Roger Perl­mut­ter jumped in­to a lead­ing role as R&D chief at Mer­ck, one of the first things he did was bow out of the round of board po­si­tions he had built up af­ter de­part­ing Am­gen.

It didn’t make any sense to di­vide his time, he tells me, un­til things qui­et­ed down at the phar­ma gi­ant, which has been spear­head­ing the rev­o­lu­tion in PD-1/L1 check­point in­hibitors with hun­dreds of on­go­ing stud­ies in­volv­ing Keytru­da.

Now, 5 years lat­er, he’s mak­ing an ul­tra rare ex­cep­tion, join­ing the board of a small, Cam­bridge, MA-based up­start that is work­ing on tak­ing the ad­vances that Er­ic Ol­son at UT South­west­ern made and push­ing in­to the clin­ic with a CRISPR/Cas9 ap­proach to gene edit­ing that could re­pair ex­on mu­ta­tions that trig­ger lethal cas­es of Duchenne MD.

“It looked like a re­al­ly great set­ting to use that gene edit­ing ap­proach,” Perl­mut­ter tells me about his new board role. “I nev­er thought [CRISPR/Cas9] could be ap­plied in a large scale clin­i­cal set­ting, so it’s not the sort of thing I would do here at Mer­ck.”

David Goed­del

Over the years, Perl­mut­ter has oc­ca­sion­al­ly fo­cused fa­vor­able at­ten­tion on cer­tain biotechs, of­ten based on his re­la­tion­ship with 1 or 2 of the peo­ple at the com­pa­ny and his in­ter­est in the tech­nol­o­gy they were work­ing with. In this case, Ex­on­ics chair­man and the man who just arranged a $40 mil­lion launch round, Dave Goed­del from The Col­umn Group — where Perl­mut­ter is a sci­ence part­ner — made the over­ture.

And Perl­mut­ter counts him­self as a long­time ad­mir­er of the prin­ci­pal sci­en­tist be­hind Ex­on­ics’ pre­clin­i­cal pro­gram.

“Er­ic Ol­son is just a ter­rif­ic mol­e­c­u­lar bi­ol­o­gist,” says Perl­mut­ter ef­fu­sive­ly.

“I must say that I had to sell many peo­ple over time on what I thought might be a chal­leng­ing de­ci­sion to make based on their busy sched­ules,” says Ex­on­ics CEO John Rip­ple. Perl­mut­ter, though, “was in­stant­ly in­ter­est­ed.”

John Rip­ple

It’s not lost on any­one at Ex­on­ics — one of this year’s End­points 11 biotechs — that Perl­mut­ter’s un­usu­al pres­ence on the board of­fers a high-pro­file val­i­da­tion of their ear­ly stage work. And that will at­tract more in­ter­est as they wind their way to the clin­ic — a twist­ing and dan­ger­ous path un­der the best of cir­cum­stances.

For Perl­mut­ter, it’s a chance to ap­ply some of his knowl­edge in trans­la­tion­al sci­ence, help­ing guide the com­pa­ny to the clin­ic and its first ren­dezvous with a pa­tient. And first up he’ll be help­ing out with iden­ti­fy­ing re­cruits for the team at Ex­on­ics.

“I think all com­pa­nies ir­re­spec­tive of size face the same chal­lenge of iden­ti­fy­ing places where very high qual­i­ty sci­en­tif­ic think­ing can be trans­lat­ed in­to med­i­cines that make a re­al dif­fer­ence,” says Perl­mut­ter. “It’s still the case. It all comes down to project cham­pi­ons and their sci­en­tif­ic in­sights, and that’s the same prob­lem every­where.”

“It comes down to pick­ing tar­gets and pur­su­ing them as­sid­u­ous­ly and with great clar­i­ty.

“That’s true for every­body.”


Im­age: Roger Perl­mut­ter. Mer­ck

Fol­low­ing news of job cuts in Eu­ro­pean R&D ops, Sanofi con­firms it’s of­fer­ing US work­ers an 'ear­ly ex­it'

Ear­li­er in the week we learned that Sanofi was bring­ing out the bud­get ax to trim 466 R&D jobs in Eu­rope, re­tool­ing its ap­proach to car­dio as re­search chief John Reed beefed up their work in can­cer and gene ther­a­pies. And we’re end­ing the week with news that the phar­ma gi­ant has al­so been qui­et­ly re­duc­ing staff in the US, tar­get­ing hun­dreds of jobs as the com­pa­ny push­es vol­un­tary buy­outs with a fo­cus on R&D sup­port ser­vices.

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.

Why would the FDA ap­prove an­oth­er con­tro­ver­sial drug to spur a woman’s li­bido with these da­ta? And why no ex­pert pan­el re­view?

AMAG Pharmaceuticals’ newly approved drug for spurring women’s sexual desire may never make much money, but it’s a big hit at sparking media attention.

The therapy — Vyleesi (bremelanotide) — got the green light from regulators on Friday evening, swiftly lighting up a range of stories around the world, from The New York Times to The Guardian. Several headlines inevitably referred to it as the “female Viagra,” invoking Pfizer’s old erectile dysfunction blockbuster.

But the two drugs have little in common.

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Which top 10 big phar­mas have the most to gain — or lose — over the next 5 years?

When Evaluate Pharma crunched the likely drug sales numbers for the big 10, 2 stood out. 

Takeda, with its big Shire buyout under its belt, is set to almost double its worldwide sales record for 2018 over 5 years, putting it in the big 10 — the 9th spot, to be exact — which is exactly where CEO Christophe Weber wants to be. 

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Martin Shkreli. AP Images

Af­ter years of claims and ac­cu­sa­tions, fraud­ster Mar­tin Shkre­li set­tles up with Retrophin

Two weeks af­ter fraud­ster Mar­tin Shkre­li filed a law­suit against one of the biotechs he had found­ed — and been kicked out of — it’s over.

A spokesman for Retrophin told CN­BC that they had re­solved “all out­stand­ing dis­putes be­tween them.” And that in­cludes the suit that Retrophin had brought against its for­mer CEO three years ago, claim­ing that he had breached his fidu­cia­ry re­spon­si­bil­i­ty to the com­pa­ny.

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.

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.

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.

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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