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.

Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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Scott Gottlieb, AP Images

Scott Got­tlieb has a new board po­si­tion to add to the re­sume — and this one is fo­cused on a fa­vorite sub­ject

Scott Gottlieb has another position to add to his lengthy roster of boards and advisory roles in the wake of his departure from the helm of the FDA.

He’ll be joining the advisory board of FasterCures, a think tank which former junk bond king Michael Milken set up to help drive more drugs to the market, looking to accelerate drug R&D. That’s a subject close to the heart of Gottlieb, who blazed a trail at the FDA focused on hustling up the process. That helped endear him to the industry, making him one of the most popular commissioners in FDA history.

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Karyopharm lines up $150 mil­lion cash in­jec­tion to back con­tro­ver­sial drug launch

Karyopharm has entered into a royalty agreement worth up to $150 million to back the launch of their multiple myeloma drug — recently approved by the FDA over the objections of a majority of the agency’s outside experts.

The deal with HealthCare Royalty Partners, worth $75 million now and $75 million once certain regulatory and commercial milestones have been reached, will fund the commercialization of Karyopharm’s oral SINE compound Xpovio (selinexor) for patients with multiple myeloma who have already had at least four prior therapies. The money will help Karyopharm as it markets its newly approved drug and pushes through clinical trials testing the drug on refractory multiple myeloma patients with one to three therapies and patients with treatment-resistant diffuse large B-cell lymphoma. It will give Karyopharm a cushion through mid-2021.

Af­ter a run of CT­LA-4 com­bo fail­ures, sci­en­tists spot­light a way to make it work — in se­lect pa­tients

CTLA-4/PD-(L)1 combinations have been one of the El Dorados of oncology, its promise forever behind that next hill but apparently unattainable after a series of pivotal clinical failures. But researchers at New York’s Memorial Sloan Kettering Cancer Center and the Technical University of Munich think they may know how to fix what’s wrong and boost the drive to next-gen cancer combos.

In a preclinical animal research program, researchers found that within a cell, checkpoints rely on a specific molecule — RNA-sensing molecule RIG-I — to work. If that sounds familiar, it’s because it has already been identified as a target for boosting immune responses and was subject to at least one Phase I/II trial. Pfizer in December allied itself with Kineta with $15 million upfront and $505 million in potential milestones to develop RIG-I immunotherapies, and three years ago Merck purchased German upstart Rigontec for $137 million upfront and over $400 million in potential milestones for the same purpose.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Pur­due Phar­ma files for bank­rupt­cy as first step in $10B opi­oid set­tle­ment

It’s settled. Purdue Pharma has filed for bankruptcy as part of a deal that would see the OxyContin maker hand over $10 billion in cash and other contributions to mitigate the opioid crisis — without acknowledging any wrongdoing in the protracted epidemic that’s resulted in hundreds of thousands of deaths.

The announcement came two weeks after news of a proposed settlement surfaced and largely confirm what’s already been reported.

Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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