PACT Phar­ma says it's per­fect­ed the tech to se­lect neoanti­gens for per­son­al­ized ther­a­py — now on­to the clin­ic

At PACT Phar­ma, the lofty goal to un­leash a “tsuna­mi” of T cells per­son­al­ized for each pa­tient has hinged on the abil­i­ty to cor­rect­ly iden­ti­fy the neoanti­gens that form some­thing of a fin­ger­print for each tu­mor, and ex­tract the small group of T cells primed to at­tack the can­cer. It still has a long way to go test­ing a treat­ment in hu­mans, but the biotech says it has nailed that high­ly tech­ni­cal piece of the process.

Cristi­na Puig-Saus

It’s a key step in mak­ing im­muno-on­col­o­gy work for every­one be­cause sci­en­tists can now pin­point what ex­act­ly is dri­ving, say, pa­tient re­sponse to check­point in­hibitors, ac­cord­ing to the team be­hind the work — open­ing the door to elim­i­nat­ing sol­id tu­mors.

Work­ing with some im­mune cells cap­tured from a pa­tient who had a long-last­ing re­sponse to an­ti-PD-1 ther­a­py, re­searchers ran the sam­ple through their “ul­tra-sen­si­tive and high-through­put” iso­la­tion tech­nol­o­gy, dubbed im­PACT. Af­ter re­triev­ing the T cells that they be­lieve were re­spon­si­ble for the re­sponse, they en­gi­neered (with non-vi­ral gene edit­ing) oth­er T cells to ex­press those same re­cep­tors and demon­strat­ed the re­sult­ing T cells can kill the same per­son’s can­cer cells.

An­toni Ribas

With back­ing from the Park­er In­sti­tute for Can­cer Im­munother­a­py, Cristi­na Puig-Saus led the study at UCLA, where PACT co-founder An­toni Ribas di­rects the tu­mor im­munol­o­gy pro­gram at the Jon­s­son Com­pre­hen­sive Can­cer Cen­ter. Ribas is list­ed as a se­nior au­thor.

“We hope that a bet­ter un­der­stand­ing of the T cell re­spons­es that oc­cur af­ter im­mune check­point block­ade will guide the de­sign of per­son­al­ized adop­tive T cell ther­a­pies,” Puig-Saus said in a re­lease ac­com­pa­ny­ing their pre­sen­ta­tion at an AACR spe­cial con­fer­ence days ago.

Once PACT gets to clin­i­cal stud­ies — and it’s re­cruit­ing for a Phase I now — it would al­so have to show it can quick­ly ex­pand the pop­u­la­tion of those T cells and safe­ly re­in­fuse them in­to the pa­tients.

Alex Franzu­soff

“While it is ear­ly, the re­sults demon­strate the pos­si­bil­i­ty for PACT’s ap­proach to ig­nite a pa­tient’s im­mune re­sponse di­rect­ly against their unique tu­mor mu­ta­tion sig­na­ture, with­in a clin­i­cal­ly rel­e­vant time­frame, with po­ten­tial ap­plic­a­bil­i­ty to most can­cers and all eth­nic­i­ties across the globe,” CEO Alex Franzu­soff said in a state­ment.

The ap­proach puts a twist on CAR-T and next-gen TCR ther­a­pies, which rec­og­nize a fixed set of anti­gens shared among pa­tients. Can­cer vac­cines are of­ten too slow to catch up with tu­mor growth; tu­mor-in­fil­trat­ing lym­pho­cytes are hard to man­u­fac­ture in vast quan­ti­ties, the com­pa­ny wrote — prob­lems that oth­er star­tups such as Neon Ther­a­peu­tics and Io­vance are at­tempt­ing to ad­dress.

PACT, of course, will al­so face its own chal­lenges in ad­vanc­ing a tai­lor-made im­munother­a­py. From a pa­per quot­ed on its web­site:

Per­son­al­iz­ing the im­mune ef­fec­tors used and the can­cer anti­gens tar­get­ed will re­quire rec­on­cil­ing timescales of clin­i­cal need, on-de­mand man­u­fac­tur­ing, and reg­u­la­to­ry com­pli­ance.

With a star-stud­ded founder team — in­clud­ing Ribas, David Bal­ti­more, Jim Heath, Ter­ry Rosen and Juan Jaen — and a well-heeled syn­di­cate that’s poured in $126 mil­lion, PACT is ready to tack­le the chal­lenges head on.

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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Noubar Afeyan, Flagship CEO and Tessera chairman (Victor Boyko/Getty Images)

Flag­ship ex­ecs take a les­son from na­ture to mas­ter ‘gene writ­ing,’ launch­ing a star-stud­ded biotech with big am­bi­tions to cure dis­ease

Flagship Pioneering has opened up its deep pockets to fund a biotech upstart out to revolutionize the whole gene therapy/gene editing field — before gene editing has even made it to the market. And they’ve surrounded themselves with some marquee scientists and execs who have crowded around to help shepherd the technology ahead.

The lead player here is Flagship general partner Geoff von Maltzahn, an MIT-trained synthetic biologist who set out in 2018 to do CRISPR — a widely used gene editing tool — and other rival technologies one or two better. Von Maltzahn has been working with Sana co-founder Jake Rubens, another synthetic biology player out of MIT who he describes as his “superstar,” who’s taken the CSO role.

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Bill Haney, Dragonfly CEO (Dave Pedley/Getty Images for SXSW)

A boom­ing Drag­on­fly is tak­ing its TriN­KETs to Copen­hagen as the lat­est Bris­tol My­ers pact spurs ex­pan­sion plans — out­side the US

Bristol Myers Squibb is making a habit out of collaborating with the crew at Dragonfly, adding their 3rd deal in a series that now will take them into newly charted R&D territory. And the fast-growing team at the Cambridge-based biotech is adding a facility in Copenhagen for its next growth spurt, where the government is making it easy to recruit scientists internationally as the U.S. throttles back.

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RA Cap­i­tal dou­bles down on Sid­dhartha Mukher­jee's vi­sion for a new cell en­gi­neer­ing ap­proach, lead­ing Vor's $110M Se­ries B

Vor Biopharma is muscling up.

CEO Robert Ang, who was reluctant to divulge the headcount when discussing his move from Neon Therapeutics to Vor last August, readily offered that the team has grown from 6 to 50 in less than a year. The biotech is moving to a larger office on Cambridge Parkway Drive in weeks, giving it more space to complete the IND-enabling work and manufacturing scale-up — conducted by a CDMO partner — in preparation for clinical trials planned for the first half of 2021.

Covid-19 roundup: Squab­bles with gov­ern­ment de­lay Mod­er­na’s PhI­II — re­ports; No­vavax se­cures largest Warp Speed deal yet: $1.6B

A much-anticipated Phase III trial for Moderna’s Covid-19 vaccine is being held up as the company delayed submitting trial protocols and sparred with government scientists on how to run the study and even what the benchmark for success should be, Reuters reported.

Moderna, the first US company to put their vaccine into human testing, was supposed to enter a 30,000-person study this month in partnership with the NIH to determine whether it can prevent infection. STAT reported last week that the trial was facing delays over the protocol, but that a July start was still possible. Neither the NIH nor Moderna ever disclosed a specific date the trial should start, but Reuters reported that the agency had hoped to begin on July 10.

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Cel­lec­tis slammed af­ter pa­tient dies and FDA slaps a hold on their tri­al for an off-the-shelf CAR-T for mul­ti­ple myelo­ma

Cellectis was slammed after the market close on Monday as the biotech reported that the FDA demanded it hit the brakes on their MELANI-01 trial for their off-the-shelf cell therapy UCARTCS1A after one of the patients in the study died of treatment-related cardiac arrest.

The multiple myeloma patient had previously been treated unsuccessfully with various therapies, noted the biotech, and had been given dose level two (DL2) of their allogeneic CAR-T.

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Shoshanna Shendelman, Applied Therapeutics CEO (Applied Therapeutics)

A lit­tle biotech slaps back at a 'crim­i­nal' short at­tack, vow­ing to pur­sue a pros­e­cu­tion of their case

As short attacks go, Biotech Research Partners’ assault on Applied Therapeutics’ “cherry picked” data and a variety of so-called red flags didn’t cause a whole lot of damage. Ahead of the July 4 holiday, its shares $APLT were dinged and showed signs of quick recovery.

But that didn’t stop an incendiary response, as the biotech swung into action bright and early Monday morning.

Applied Therapeutics accused the authors of the short report of manipulating graphs and figures, misrepresenting data and included factual misrepresentations — all of which added up, in their view, to fraud.

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Covid-19 roundup: Teamed up with NIH, Re­gen­eron launch­es PhI­II pre­ven­tion tri­al for an­ti­body cock­tail

As Regeneron moves its antibody cocktail into Phase II/III trials testing REGN-COV2 as a treatment for both hospitalized and non-hospitalized patients with Covid-19, the biotech is also starting a Phase III in the prevention setting.

The National Institute of Allergy and Infectious Diseases — which orchestrated the large, randomized study for remdesivir that produced positive results — will jointly run the study.

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