Scott Got­tlieb gave a savvy re­view of what we can ex­pect at the FDA; When times got rough, a biotech CEO demon­strat­ed re­al in­tegri­ty

Smart, rea­son­able and ex­treme­ly well pre­pared: What did we learn from Scott Got­tlieb’s con­fir­ma­tion hear­ing?

On Wednes­day, when Scott Got­tlieb showed up for his con­fir­ma­tion hear­ing at the Sen­ate, it be­came quick­ly ap­par­ent that the prospec­tive FDA com­mis­sion­er was the best pre­pared per­son talk­ing that day. What the Dems had billed as a sharp at­tack on Got­tlieb’s in­dus­try ties quick­ly dis­in­te­grat­ed in­to a se­ries of ran­dom, dis­joint­ed com­ments di­rect­ed against large­ly un­re­lat­ed is­sues.

Com­mis­sion­er-des­ig­nate Scott Got­tlieb at his hear­ing Zach Gib­son/Get­ty Im­ages

The Re­pub­li­cans were there to pat him on the back, which they man­aged to do just fine. A few used the oc­ca­sion to preen to their vot­ers. It’s what law­mak­ers do. No mat­ter.

It was a non-event if you were look­ing for fire­works and con­fronta­tion. If you lis­tened care­ful­ly, though, there was some re­al meat to this ex­change as well, and some vi­tal­ly im­por­tant in­sights in­to how he sees his role in man­ag­ing re­form from the in­side rather than es­tab­lish­ing him­self as the agency’s biggest crit­ic.

It was a huge­ly im­por­tant mo­ment for the bio­phar­ma in­dus­try, which has had to fear the worst over the past three months.

Got­tlieb looked like he had been prep­ping for this mo­ment for the past 20 years, or more. He has a back­ground in the FDA and deep ties to the in­dus­try. Over the past decade he has amassed a line­up of con­nec­tions to in­vest­ment groups and bio­phar­ma com­pa­nies. He will not set him­self in op­po­si­tion to the in­dus­try, nor should he. More im­por­tant­ly, he’s pre­pared to cham­pi­on the R&D gold stan­dard on safe­ty and ef­fi­ca­cy.

Three cheers. But in ad­di­tion to all those jobs he pur­sued un­der the Oba­ma ad­min­is­tra­tion, there was one more po­si­tion he vol­un­teered for: FDA com­mis­sion­er-in-wait­ing.

Back be­fore his nom­i­na­tion, I took a close look at Got­tlieb. Based on the pub­lic record and peo­ple close to him, it was clear he had a spe­cif­ic agen­da in mind. And he ticked off most of those box­es to the sen­a­tors.

He was go­ing to go af­ter com­plex gener­ics, clear­ing the way for new com­pe­ti­tion to some very ex­ten­sive ther­a­pies. He would cel­e­brate Richard Paz­dur’s clear achieve­ments in ac­cel­er­at­ing can­cer drug re­views — a role that has over­hauled and speed­ed a mas­sive amount of prod­uct de­vel­op­ment in the field — and promised to get some lag­gards at the agency up to speed.

He likes adap­tive tri­al de­signs, look­ing for new and bet­ter ways to in­tel­li­gent­ly as­sess safe­ty/ef­fi­ca­cy.

Drugs for rare dis­eases al­so look like they’ll be a pri­or­i­ty. There’s a way for skilled FDA staff to over­see small, fo­cused stud­ies for new drugs that can avoid the mess that Sarep­ta cre­at­ed and still suf­fers from.

I would have liked to have heard more specifics, such as pub­lish­ing CRLs (or trans­paren­cy in gen­er­al), how he will over­haul the or­phan drug process and do­ing away with the need for some out­comes stud­ies ahead of ap­provals, when they can be done af­ter­wards.

(Though can you al­so hear pay­ers sharp­en­ing their knives for those drugs? When reg­u­la­to­ry risk de­clines, pay­er risk ris­es dis­pro­por­tion­ate­ly these days. And af­ter Gilead’s So­val­di primed the pump, it’s a brand new day in the pay­er uni­verse.)

Maybe we can get some of that ex­tra de­tail in the fi­nal Q&A round to come. Pub­lish­ing CRLs won’t over­ly dis­turb the re­spon­si­ble play­ers in the in­dus­try and Got­tlieb could add some much-need­ed trans­paren­cy as a way to dis­tin­guish his stint as an in­de­pen­dent com­mis­sion­er. It’s not all go­ing to be cozy and fun.

What I was par­tic­u­lar­ly pleased to hear is that Got­tlieb views re­cruit­ing tal­ent as his biggest chal­lenge, while look­ing for a “bot­tom up” ap­proach from the cen­ters to dri­ve re­form. Got­tlieb’s stint as FDA com­mis­sion­er would be long and fruit­less if he took a con­fronta­tion­al po­si­tion with staff.

So what do we get?

We get a com­mis­sion­er who’s pledged to speed things up some more, re­spon­si­bly. He’s go­ing to bring in new, bad­ly need­ed tal­ent. And we hope he clear­ly choos­es trans­paren­cy over the long and un­ac­cept­able black box that the agency has long been re­quired to work in — though that has yet to be seen.

I’ll take it.


In­tegri­ty mat­ters more than ever in bio­phar­ma

I’ve been writ­ing about bio­phar­ma on a dai­ly ba­sis for close to 14 years now. And I’m more than fa­mil­iar with the re­struc­tur­ing drill.

Com­pa­ny X runs in­to some trou­ble. The biotech boat be­comes a life raft, or a log, and staffers have to go. There’s a press re­lease, a canned quote from the CEO say­ing how grate­ful they were to the new­ly job­less, and on to the next thing.

So I was more than a lit­tle shocked when CEO Ron Co­hen reached out to talk to me about the lay­offs and down­siz­ing at Acor­da this week fol­low­ing a set­back on his patent front for Ampyra. That nev­er hap­pens. But he spelled out what the cuts meant, where the ax would fall and how they could mon­e­tize as­sets.

Co­hen has a Plan B. He hasn’t been stand­ing on the tracks, wait­ing for the train to come. It’s ex­tra­or­di­nar­i­ly high risk, and Plan B hasn’t al­ways gone ac­cord­ing to plan over the years — which is par for this unique course. And now fail­ure is not an op­tion.

That’s not a great place to be. No one en­vies his po­si­tion. But in­stead of duck­ing and tak­ing the easy way out, Co­hen demon­strat­ed re­al in­tegri­ty in stand­ing up and tak­ing it all on, di­rect­ly.

Sure, you could throw a cheap shot or two in his di­rec­tion. But in this in­dus­try in­tegri­ty mat­ters more than ever. And I re­spect it more than ever. We all should.

Albert Bourla 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

UP­DAT­ED: Pfiz­er CEO Al­bert Bourla is back in the M&A game, but why is he pay­ing $11.4B for Ar­ray?

Pfiz­er $PFE has cut short its time on the side­lines of bio­phar­ma M&A.

Mon­day morn­ing the phar­ma gi­ant un­veiled an $11.4 bil­lion deal to ac­quire Ar­ray Bio­Phar­ma, beef­ing up its on­col­o­gy work and adding a new re­search hub in Boul­der, Col­orado to its glob­al op­er­a­tions.

At $48 a share, Ar­ray $AR­RY in­vestors will be get­ting a 62% pre­mi­um off the Fri­day close of $29.59.

Pfiz­er, which has strug­gled to gain all the up­side promised in past buy­outs like Medi­va­tion, high­light­ed the ac­qui­si­tion of 2 ap­proved drugs in the deal — Braftovi (en­co­rafenib) and Mek­tovi (binime­tinib).

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Gene ther­a­py R&D deals turn red hot as Big Phar­ma steps up to play

This September will mark the 20th anniversary of the death of Jesse Gelsinger, a young man suffering from X-linked genetic disease of the liver. He was killed in a gene therapy study conducted by Penn’s James Wilson, and the entire field endured a lengthy deep freeze as the field grappled with the safety issues inherent in the work.

Some thought gene therapy R&D would never survive. But it did. And this year marked a landmark approval for Zolgensma, a new gene therapy for spinal muscular atrophy Novartis priced at $2.1 million.

“Gene therapy is the hottest item on the block now. But there was a time when we first got into this trial, where there wasn’t a person in the world who believed that gene therapy would work. We have to remember that,” noted gene therapy investigator Jerry Mendell told SMA News Today.

We’re still right on the pioneering frontier when it comes to getting approvals for gene therapies and launching marketing campaigns with the European green light for bluebird's leading program last Friday underscoring the nascent nature of the field. But gene therapy R&D is booming, and has been for several years now.

The rapid growth of gene therapy clinical development is well known, but we decided to put some numbers on it, to quantify what’s going on. DealForma chief Chris Dokomajilar took a lot over the past 10 years, as the number of deals, R&D partnerships and buyouts steadily gained steam, spiking last year and on track to maintain the surge in 2019.

The upfronts and totals for the dollars on deals so far in 2019 is already close to the 2018 mark, underscoring a new phase of negotiations as the major players step up to gain a piece of the late-stage and commercial action.

Once again, we’re looking at an “overnight” biotech success story, decades in the making.

At some point, that may start to brake the numbers we’re seeing. But for now, as rivals line up to compete for frontline prominence across a range of diseases, the arrows are all pointed north.

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A uni­corn stalks Wall Street in search of IPO cash; CASI Phar­ma in-li­cens­es CD19 ther­a­py from Chi­na’s Ju­ven­tas

→ A herd of up­start biotechs will look to Wall Street for some ma­jor wind­falls this week as a burst of new of­fer­ings con­tin­ues to feed cash in­to the R&D sys­tem. To­day we learned that Bridge­Bio will look to raise in the neigh­bor­hood of $225 mil­lion by of­fer­ing 15 mil­lion shares for $14 to $16 each. And they have a string of joint bookrun­ners: J.P. Mor­gan, Gold­man Sachs, Jef­feries, SVB Leerink, KKR, Piper Jaf­fray, Mizuho Se­cu­ri­ties, BMO Cap­i­tal Mar­kets and Ray­mond James. If suc­cess­ful, Bridge­Bio will emerge with a mar­ket cap of around $1.7 bil­lion. There are 5 biotechs look­ing to IPO this week, in­clud­ing Akero and Pre­vail.

UP­DAT­ED: Sanofi Gen­zyme deserts gene ther­a­py de­vel­op­er Voy­ager Ther­a­peu­tics

While gene ther­a­py com­pa­nies re­joice as the sec­tor gains trac­tion with ap­provals and a flur­ry of M&A ac­tiv­i­ty, one play­er is feel­ing the heat.

Back in 2015, Voy­ager Ther­a­peu­tics joined forces with Sanofi Gen­zyme in a deal worth up to $845 mil­lion ($100 mil­lion up­front + a po­ten­tial $745 mil­lion in mile­stones) to co-de­vel­op gene ther­a­pies for se­vere cen­tral ner­vous sys­tem dis­or­ders. But two years lat­er, the French drug­mak­er re­treat­ed, elect­ing to not pick up the op­tion to work on Voy­ager’s Parkin­son’s dis­ease pro­gram. (Last year, the FDA dis­ap­point­ed Voy­ager, telling the com­pa­ny that it was not open to an ac­cel­er­at­ed fil­ing on the Parkin­son’s drug on the ba­sis of Phase II da­ta — in­stead of re­quir­ing an ad­di­tion­al piv­otal study.)

In­vestors fret as VBI's hep B vac­cine fails key sec­ondary PhI­II study goal

Sobered by mount­ing costs, Dy­navax $DVAX last month made the de­ci­sion to fo­cus all its re­sources on its 2017-ap­proved he­pati­tis B vac­cine Hep­lisav-B, which ri­vals and su­per­sedes the ef­fi­ca­cy and con­ve­nience pro­file of GSK’s $GSK es­tab­lished En­ger­ix-B. The Cal­i­for­nia-based com­pa­ny will be on the look­out for an­oth­er com­peti­tor — VBI Vac­cines, which on Mon­day un­veiled late-stage da­ta on its hep B vac­cine: Sci-B-Vac.

John Oyler, Founder & CEO of BeiGene, at the US-China Biopharma Innovation and Investment Summit in Shanghai on October 23, 2018; Credit: Endpoints News, PharmCube

UP­DAT­ED: As Bris­tol-My­ers/Cel­gene tie up loose ends, BeiGene pock­ets $150M from PD-1 breakup

As soon as Bristol-Myers Squibb announced its $74 billion buyout for Celgene, BeiGene emerged as a prominent example of a player whose pact with the big biotech could sour, as its PD-1 candidate seems to overlap with Opdivo. After six months of suspense, the partners say they are finally bringing the 2-year-old deal to an amicable end.

BeiGene $BGNE gets $150 million for the termination in addition to full global rights to tislelizumab. In 2017 Celgene had paid $263 million in upfront license fees to develop the PD-1 inhibitor for solid cancers in the US, Europe, Japan and the rest of the world outside Asia. It also threw in a $150 million equity investment in exchange for BeiGene handling its commercial operations — think Abraxane, Revlimid and Vidaza — in China.

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Exterior of the 1 million square foot Discovery Labs in Upper Merion, PA (PR Newswire)

Philadel­phia cham­pi­ons life sci­ences 'co-work­ing,' re­viv­ing for­mer GSK cam­pus in $500M makeover

In a boost to Philadel­phia’s thriv­ing life sci­ences scene, a for­mer Glax­o­SmithK­line cam­pus and a near­by site has been turned in­to what its de­vel­op­er calls “the largest cowork­ing ecosys­tem” for health­care com­pa­nies in the coun­try.

The Dis­cov­ery Labs, a com­pa­ny spawned by MLP Ven­tures, has se­lect­ed two lo­ca­tions in the King of Prus­sia area as the $500 mil­lion test case for its strat­e­gy of ac­quir­ing and con­vert­ing old phar­ma­ceu­ti­cal R&D fa­cil­i­ties world­wide. The sites add up to 1.64 mil­lion square feet.

Ted Love. HAVERFORD COLLEGE

Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.