Penn spin­out preps for hu­man stud­ies of a new-mod­el CAAR, aim­ing at a one-time cure for au­toim­mune dis­eases

Michael Milone

Michael Milone learned about en­gi­neer­ing T cells for can­cer at the bench of the mas­ter, work­ing as a post doc in Carl June’s lab at the Uni­ver­si­ty of Penn­syl­va­nia from 2004 to 2007. He went on to play a key role in the sci­en­tif­ic lead­er­ship team de­vel­op­ing No­var­tis’ break­through CAR-T drug Kym­ri­ah at Penn. And now, at the head of his own team of trans­la­tion­al re­searchers, he and Penn’s Aimee Payne and Steven Nicht­berg­er are co-found­ing a biotech that will take what they’ve learned about T cell en­gi­neer­ing in­to au­toim­mune dis­eases, in search of a break­through, once-and-done ap­proach.

The up­start — Ca­balet­ta Bio, to be helmed by Nicht­berg­er — has grabbed a $38 mil­lion round from a group of in­vestors with an eye to go­ing in­to the clin­ic with the work they’ve done on mu­cos­al pem­phi­gus vul­garis, a rare skin blis­ter­ing dis­ease, re­cent­ly pub­lished in Sci­ence.

Aimee Payne

So what’s the big idea here? The team at Penn be­lieves they have con­struct­ed a next-gen CAAR — with Chimeric AutoAnti­body Recep­tors. These CAARs have shown a pre­clin­i­cal abil­i­ty to iden­ti­fy and elim­i­nate the spe­cif­ic B cells that me­di­ate a wide va­ri­ety of au­toim­mune con­di­tions, while leav­ing the nor­mal B cells alone. And they’re bet­ting that the years of clin­i­cal work on B cell me­di­at­ed can­cers — which has had to grap­ple with the lethal cy­tokine storms that can en­gulf and ex­tin­guish pa­tients — has sig­nif­i­cant­ly de-risked the safe­ty as­pects of their work.

It was Payne, the lead physi­cian in the Au­toim­mune Blis­ter­ing Clin­ic at Penn, who did the an­i­mal stud­ies on their lead drug DSG3-CAART, pro­vid­ing the pre­clin­i­cal proof of con­cept they need­ed to set up the clin­i­cal stud­ies. And they’ll have the full back­ing of Penn now that they’re shift­ing to hu­man stud­ies.

Steven Nicht­berg­er

“Dur­ing the past 18 months, Ca­balet­ta has ad­vanced a se­ries of CAAR T cell ther­a­py prod­ucts, li­censed foun­da­tion­al in­tel­lec­tu­al prop­er­ty and as­sem­bled lead­ing sci­en­tists, clin­i­cians and ex­perts in the dis­cov­ery, de­vel­op­ment, man­u­fac­tur­ing and reg­u­la­to­ry ap­proval of cell ther­a­py prod­ucts to ac­cel­er­ate de­vel­op­ment of high­ly spe­cif­ic CAAR T cell ther­a­pies that may of­fer a po­ten­tial one-time cure for cer­tain B cell-me­di­at­ed au­toim­mune dis­eases,” said Nicht­berg­er.

Their A round came in last month, led by 5AM Ven­tures, with par­tic­i­pa­tion from the founders: Adage Cap­i­tal Man­age­ment, a “sec­ond large U.S.-based, health­care-fo­cused in­vest­ment fund” and the Uni­ver­si­ty of Penn­syl­va­nia.


Im­age: PENN TO­DAY

Paul Hudson, Sanofi CEO (Getty Images)

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After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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The Advance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

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Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

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No­var­tis jumps in­to Covid-19 vac­cine hunt, as Big Phar­ma and big biotech com­mit to bil­lions of dos­es

After spending most of the pandemic on the sidelines, Novartis is offering its aid in the race to develop a Covid-19 vaccine.

AveXis, the Swiss pharma’s gene therapy subsidiary, has agreed to manufacture the vaccine being developed by Massachusetts Eye and Ear and Massachusetts General Hospital. The biotech will begin manufacturing this month, while the vaccine undergoes further preclinical testing. They’ve agreed to provide the vaccine for free for clinical trials beginning in the second half of 2020, but have not disclosed financials for after.

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Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

Stymied by the pan­dem­ic, Im­munomedic­s' new CEO bows out, tak­ing a mil­lion bucks plus perks as he heads out the vir­tu­al ex­it

Just a little more than a month since taking over as the latest CEO to helm Immunomedics, $IMMU Harout Semerjian is exiting the company after being confronted by “logistical” obstacles thrown up by the pandemic that made it impossible for him to move from London to carry out the job. And he’s getting a little over a million dollars in cash plus perks to grease the skids on the way out.

Word of the changeup arrived right after the market closed Wednesday.

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Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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David Hoey (Vaxxas)

In for the long vac­cine game, Mer­ck buys in­to patch de­liv­ery tech with pan­dem­ic po­ten­tial

When Merck dived into the R&D fray for a Covid-19 vaccine earlier this week, execs made it clear that they’re not necessarily looking to be first — with CEO Ken Frazier throwing cold water on the hotly-discussed 12- to 18-month timelines. But when it does emerge from behind, the pharma giant clearly expects to play a significant part.

Part of that will depend on next-generation delivery technology that reshapes the world’s imagination of a vaccine.