Sean Park­er finds a big role to play in can­cer R&D: The dis­rup­tive bil­lion­aire

In biotech, where every­thing is un­cer­tain, ex­ecs like to be as pre­dictable as pos­si­ble. Scripts are main­tained, nasty sur­pris­es are avoid­ed when­ev­er pos­si­ble and speed is fine, so long as you stick to your sched­ule and don’t look like you’re in too much of a hur­ry.

Forbes’ Matthew Her­p­er with Sean Park­er and Dr. Carl June

And then there’s Sean Park­er. Park­er had an easy role to play as the bil­lion­aire founder of a new can­cer R&D in­sti­tute. The trail­blaz­ers could get a fresh source of cash and a few years from now he could claim progress on the ever-pop­u­lar no­tion of find­ing a cure for can­cer.

Park­er, a cen­tral fig­ure in this gen­er­a­tion’s so­cial me­dia rev­o­lu­tion, clear­ly had a more dis­rup­tive plan in mind.

On Mon­day evening, the Uni­ver­si­ty of Penn­syl­va­nia and the Park­er In­sti­tute con­firmed the news that An­to­nio Re­gal­a­do at MIT Tech­nol­o­gy Re­view had bro­ken ear­li­er in the day: Park­er is bankrolling a sur­prise move by Penn’s leg­endary Carl June and some of his col­leagues to do the first gene edit­ing ex­per­i­ment in hu­mans us­ing CRISPR-Cas9, a new tech­nol­o­gy that has been her­ald­ed as a sim­ple, ef­fec­tive tool for slic­ing and dic­ing DNA.

They’re look­ing to use it on im­prov­ing adop­tive cell ther­a­pies for can­cer, one of the hottest fields in im­muno-on­col­o­gy re­search.

“The study, an open la­bel, Phase I tri­al which will ex­am­ine safe­ty and man­u­fac­tur­ing fea­si­bil­i­ty of au­tol­o­gous T cells en­gi­neered to ex­press NY-ESO-1 TCR and gene edit­ed to elim­i­nate en­doge­nous TCR and PD-1, will build on Penn’s progress in­ves­ti­gat­ing oth­er types of per­son­al­ized T cell ther­a­pies for can­cer, in­clud­ing both chimeric anti­gen re­cep­tor (CAR) T cells and NY-ESO-1 trans­genic T cells,” Penn said in a state­ment to END­POINTS. “The study is de­signed to test the hy­pothe­ses that check­point re­sis­tant T cells may be safe and have im­proved an­ti­tu­mor ac­tiv­i­ty. The pi­lot tri­al will en­roll pa­tients with mul­ti­ple myelo­ma, melanoma and sar­co­ma at Penn’s Abram­son Can­cer Cen­ter and two oth­er aca­d­e­m­ic cen­ters. Fund­ing for the tri­al has been pro­vid­ed by the Park­er In­sti­tute for Can­cer Im­munother­a­py.”

Part of the pur­pose of the study, not­ed Park­er’s in­sti­tute in a fol­lowup mes­sage, is to un­leash “the pow­er of the T-cell while si­mul­ta­ne­ous­ly re­duc­ing the chance for au­toim­mu­ni­ty.”

Penn’s team pre­sent­ed their study to an NIH safe­ty board Tues­day morn­ing, out­lin­ing plans to re­cruit 18 pa­tients: 6 pa­tients with myelo­ma, 6 with sar­co­ma and 6 with melanoma.

These pa­tients will be se­lect­ed by their ex­pres­sion of the anti­gen NY-ESO, with re­searchers at Penn, MD An­der­son and UC San Fran­cis­co all par­tic­i­pat­ing. Us­ing CRISPR, they plan to dis­rupt ex­pres­sion of en­doge­nous TCR and PD-1, which may in­crease the per­sis­tence of these cells, in­creas­ing the safe­ty and ef­fi­ca­cy of the treat­ment. Tar­get­ing PD-1, they be­lieve, should cre­ate “check­point” re­sis­tant T cells, im­prov­ing pro­lif­er­a­tion of the cells.

Ini­tial­ly, they ex­pect to try this on three pa­tients over 4 weeks, then check and see if there are any un­ex­pect­ed safe­ty is­sues. If there aren’t any, then they’ll go ahead with the rest of the pa­tients.

One of the great­est risks as­so­ci­at­ed with CAR-Ts has been cy­tokine re­lease syn­drome, or cy­tokine storm. The in­ves­ti­ga­tors not­ed that there’s a well-es­tab­lished pro­to­col for track­ing this threat and re­cruit­ing pa­tients that are less like­ly to be killed by it. And the re­searchers al­so want to see just how fea­si­ble it will be to man­u­fac­ture this CRISPR-edit­ed ther­a­py.

Pan­el mem­ber Lau­rie Zoloth pressed June and his col­leagues about the fi­nan­cial im­pli­ca­tions of the study. No­var­tis has played a ma­jor role in fund­ing Penn’s CAR-T work, adapt­ing cells with a chimeric anti­gen re­cep­tor to tar­get them against can­cer cells. But June not­ed that this isn’t a CAR, it’s a gene edit­ed cell which “has no re­la­tion­ship with No­var­tis.”

Pan­el mem­bers al­so ad­vised the Penn team to high­light the pi­o­neer­ing as­pect of the tri­al as the first ever use of CRISPR in hu­mans to any pa­tients be­fore they sign on. And at about noon they vot­ed unan­i­mous­ly – with one ab­sten­tion – to ap­prove the tri­al.

Com­ing up with a bet­ter, safer way to spur a T cell at­tack on can­cer has be­come one of the Holy Grails of biotech. But it wasn’t sup­posed to play out this way in gene edit­ing.

There are sev­er­al biotechs look­ing to use gene edit­ing tech in can­cer. Ed­i­tas, which took an ear­ly lead on CRISPR-Cas9, was al­so wide­ly billed as the one that would like­ly be the first to use it in hu­man stud­ies. And Cel­lec­tis has been us­ing TAL­ENS in an ef­fort to prove that its pre­co­cious CEO An­dre Chouli­ka has a bet­ter tech­nol­o­gy for the job.

Park­er, though, seems in­tent on tear­ing up a care­ful­ly arranged stage. From his start with Nap­ster, which got him in trou­ble with the feds even as it point­ed him down the road to Face­book fame and for­tune, Park­er en­joyed the dis­rup­tion — chang­ing the mu­sic in­dus­try and the way peo­ple com­mu­ni­cate in ever grow­ing net­works.

Now he’s found a way to adopt a sim­i­lar role in can­cer R&D. And every­one will be watch­ing to see what he comes up with next.

Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors. 

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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