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.


Covid-19 roundup: Eu­rope pur­chas­es 80M dos­es of Mod­er­na's vac­cine; CO­V­AXX se­cures $2.8B in emerg­ing mar­ket pre-or­ders

With the announcement of its vaccine efficacy data last week, Moderna is starting to line up customers for its Covid-19 mRNA jabs.

The Massachusetts-based biotech announced Wednesday it has agreed to sell an initial round of 80 million doses to the European Commission, with the option to double the amount to 160 million. Once the member states rubber stamp the approval, the deal will be finalized.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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Feng Tian, Ambrx CEO (Ambrx)

Af­ter 5 qui­et years, a for­mer Scripps spin­out rais­es $200M and an­nounces plans to try again at an IPO

The first time San Diego biotech Ambrx tried to go public in 2014, they failed and the company’s board switched to a radically different strategy: They sold themselves for an undisclosed amount to a syndicate of Chinese investors and pharma companies.

Now, after 5 quiet years, that syndicate has raised a mountain of cash and indicated they’ll soon make another bid to go public.

Earlier this month, Ambrx raised $200 million in what they billed as a crossover round financed by Fidelity, BlackRock, Cormorant Asset Management, HBM Healthcare Investments, Invus, Adage Capital Partners and Suvretta Capital Management. It’s the largest amount they’ve ever raised and, according to Crunchbase figures, more than doubles the total amount of VC capital collected since their launch 17 years ago.

FDA hands Liq­uidia and Re­vance a CRL and de­fer­ral, re­spec­tive­ly, as Covid-19 cre­ates in­spec­tion chal­lenge

Two biotechs said they got turned away by the FDA on Wednesday, in part due to pandemic-related travel restrictions.

North Carolina-based Liquidia Technologies was handed a CRL for its lead pulmonary arterial hypertension drug, citing the need for more CMC data and on-site pre-approval inspections, which the FDA hasn’t been able to conduct due to travel restrictions. The agency also deferred its decision on Revance Therapeutics’ BLA for its frown line treatment, because it needs to inspect the company’s northern California manufacturing facility. The action, Revance emphasized, was not a CRL.

News brief­ing: FDA re­quests new tri­al for Reata's Friedre­ich's atax­ia pro­gram; J&J's Trem­fya picks up ex­pand­ed la­bel in Eu­rope

Three months after Reata Pharmaceuticals suggested its Friedreich’s ataxia program omaveloxolone could be delayed, the company revealed that is indeed going to be the case.

Reata $RETA shares took a nosedive Wednesday after the biotech revealed that the FDA said supplemental data for its pivotal trial did not strengthen the case for approval. As a result, the drug is likely to need another study before the FDA takes up the case.

Jef­frey Hat­field takes over from Diego Mi­ralles as CEO of Vi­vid­ion; Drag­on­fly scores a new ex­ec with COO Alex Lu­gov­skoy

→ San Diego protein degradation startup Vividion Therapeutics has made a change at the top with Jeffrey Hatfield taking the helm as CEO, replacing Diego Miralles six months after Roche forked over $135 million to collaborate with Vividion on their small molecule degraders. Hatfield is chairman of the board at miRagen Therapeutics and previously held the CEO job at Zafgen and Vitae Pharmaceuticals. He also had a series of leadership roles at Bristol Myers Squibb from 1996-2004, including SVP, immunology and virology divisions.

Chi­na opens the door for biotech in­vestors in Hong Kong to buy Shang­hai stocks, and vice ver­sa

When Shanghai’s STAR board began opening its doors to biotech, it was considered not just a rival to Nasdaq but also the stock exchange in Hong Kong. Those perceptions may take an amicable turn as China expands a mutual access program with the city.

The changes mean investors in mainland China will be able to own Hong Kong biotech chapter stocks, while those in Hong Kong — a much more internationally connected group — would have access to those listed on STAR. In effect, it turns the Shanghai market into a globally accessible exchange overnight while also broadening a key source of revenue for HKEX.