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.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

An­gion's or­gan dam­age drug strikes out again, this time in high-risk kid­ney trans­plant pa­tients

After flopping a test in Covid-19 earlier this year, Angion’s lead organ damage drug has now hit the skids again in kidney transplant patients.

Angion and partner Vifor Pharma’s ANG-3777 failed to beat out placebo in terms of improving eGFR, a measure of kidney function, in patients who had received a deceased donor kidney transplant and were at high risk of developing what is known as delayed graft function, according to Phase III results released Tuesday.

An image of Alzheimer's brain tissue. The red show gingipains, a protein from P. gingivalis, intermixing with neurons (yellow) and glial cells (green)

An Alzheimer's dark­horse fails its first big tri­al, but of­fers hope for a long-over­looked hy­poth­e­sis

Three years ago, Cortexyme emerged out of obscurity with some big-name backers and an unorthodox approach to treating Alzheimer’s.

They moved their drug into a pivotal study the next year, offering one of the first major tests for a hypothesis that has fluttered on the outskirts of Alzheimer’s research for decades: that, in many cases, the disease is driven by infectious agents — the havoc they wreak in the brain and the inflammation the body uses to try to fend them off. And that quashing the infection could slow patients’ cognitive decline.

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No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

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Peter Nell, Mammoth Biosciences CBO

UP­DAT­ED: Jen­nifer Doud­na spin­out inks a Mam­moth CRISPR deal with Ver­tex worth near­ly $700M

When a company gets its start in gene editing pioneer Jennifer Doudna’s lab, it’s bound to make headlines. But three years in, the fanfare still hasn’t died down for Mammoth Biosciences. Now, the Brisbane, CA-based company is cheering on its first major R&D pact.

Mammoth unveiled a nearly $700 million deal with Vertex on Tuesday morning, good for the development of in vivo gene therapies for two mystery diseases. The stars of the show are Mammoth’s ultra-small CRISPR systems, including two Cas enzymes licensed from Doudna’s lab over the past couple years, Cas14 and Casɸ.

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FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.