William Cao (Bloomberg via Getty)

Nas­daq rings in its first biotech IPOs of 2021: a CAR-T spe­cial­ist from Chi­na, a can­cer port­fo­lio play, and a SPAC

There was once a time when stel­lar CAR-T da­ta from Chi­na would come — in a sense — out of nowhere and be greet­ed with sur­prise, or even out­right skep­ti­cism.

It wasn’t that long ago, but those days are now gone. This is a time a CAR-T ther­a­py orig­i­nat­ing from Chi­na can be de­vel­oped in the pub­lic eye, down to every cut of da­ta, by a com­pa­ny bound by Amer­i­can rules of dis­clo­sure.

Case in point: Gra­cell Biotech­nolo­gies, a Shang­hai-based out­fit that’s grab­bing Nas­daq’s first biotech IPO list­ing along­side Cam­bridge, MA-based Cul­li­nan On­col­o­gy.

“We know val­u­a­tion at Nas­daq is prob­a­bly the low­est (com­pared to Hong Kong or Shang­hai), but the rea­son for us to go for Nas­daq was — is still — very clear,” co-founder and CEO William Cao told End­points News. “We’re a tech­nol­o­gy play com­pa­ny; in­no­va­tion is our soul and spir­it.”

By to­day’s stan­dards, Gra­cell’s raise of $209 mil­lion is some­what mod­est. But with the back­ing of mar­quee in­vestors like Lil­ly Asia Ven­tures, Morn­ing­side, Or­biMed, Temasek, Vi­vo Cap­i­tal and Welling­ton, Cao is less con­cerned about the mon­ey than gain­ing pres­ence in the US, where he is plan­ning an R&D cen­ter and mul­ti­ple tri­als.

The break­through suc­cess­es of No­var­tis, Kite and — per­haps to a less­er ex­tent — Juno back in 2016 and 2017 did much more than in­spire the next gen­er­a­tion of US and Eu­ro­pean star­tups bran­dish­ing new tech­nolo­gies to make more durable cell ther­a­pies, faster, Cao re­flect­ed. Af­ter re­tir­ing from the helm of CB­MG, the com­pa­ny he co-found­ed which in 2018 be­came No­var­tis’ man­u­fac­tur­ing part­ner in Chi­na, he took a stint as a ven­ture part­ner at 6 Di­men­sions, look­ing for start­up ideas to in­vest in.

Mar­ti­na Ser­sch

The fan­cy new ideas and great sci­en­tists teams he en­coun­tered, though, lacked the busi­ness acu­men to solve what he termed in­dus­tri­al chal­lenges — bot­tle­necks such as lengthy pro­duc­tion times and a “clum­sy” process were much more press­ing to him.

“I’m less in­ter­est­ed in ad­vanc­ing very fu­tur­is­tic CAR-T de­sign or TCR de­sign and I’m more in­ter­est­ed in how to solve the prac­ti­cal is­sues,” he said.

He soon got to work set­ting up his own shop, con­fer­ence-hop­ping around the US and Chi­na to ac­quaint him­self with the lat­est sci­ence as well as top tal­ent that he lat­er hand­picked for his team. Gra­cell was built on two tech­nolo­gies: FasT­CAR, which promis­es to short­en man­u­fac­tur­ing turn­around time to 22-36 hours; and Tru­U­CAR, its take on al­lo­gene­ic CAR-T. It has its own pro­duc­tion site in Suzhou to keep the key process­es in-house.

The C-suite now fea­tures Kevin Xie, the for­mer pres­i­dent at Fo­s­un Health­care, as CFO and Mar­ti­na Ser­sch as CMO.

“So I saw the mul­ti­ple myelo­ma da­ta,” said Ser­sch, who helped Am­gen de­vel­op its mul­ti­ple myelo­ma strat­e­gy be­fore jump­ing to Mus­tang Bio. “And at the time, I re­mem­ber William was not con­vinced of the da­ta. But when I saw the da­ta I said, wow, this looks very promis­ing. If this is hold­ing through and true through a cou­ple of more months for fol­lowup, you may have some­thing re­al­ly mean­ing­ful.”

Kevin Xie

In its F-1 fil­ing, Gra­cell re­port­ed that GC012F, its au­tol­o­gous CAR-T ther­a­py tar­get­ing both BC­MA and CD19, has treat­ed 16 pa­tients with re­lapsed/re­frac­to­ry MM and that 15 of them achieved and main­tained a re­sponse. The high­est dose co­hort record­ed a 100% strin­gent com­plete re­sponse rate for the six evalu­able pa­tients.

“Most pa­tients ex­pe­ri­enced Grade 1 or Grade 2 CRS, on­ly two pa­tients ex­pe­ri­enced Grade 3, and no pa­tient ex­pe­ri­enced Grade 4 or Grade 5 CRS or ICANS of any grade,” the com­pa­ny wrote.

It will be no easy task com­pet­ing with ei­ther the well-fund­ed US play­ers from Al­lo­gene to Lyell and Sana, or the ti­tan-backed joint ven­tures like JW, Fo­s­un/Kite and Al­lo­gene/Over­land. But Gra­cell is plung­ing full speed ahead with a reg­is­tra­tional tri­al in Chi­na that it just got cleared for and plans to start US tri­als in 2021.

For Cao, Chi­na’s in­fra­struc­ture for in­ves­ti­ga­tor-ini­ti­at­ed tri­als is Gra­cell’s se­cret trans­la­tion­al weapon, al­low­ing for quick first-in-hu­man tests of prod­uct de­sign.

“To a cer­tain ex­tent, that’s the most im­por­tant fac­tor we’ve been en­joy­ing that re­al­ly en­abled us. I don’t think every­body — all the peo­ple re­al­ize how pow­er­ful it is for im­mune cell ther­a­py,” Cao said.

Patrick Baeuer­le’s hub-and-spoke mod­el earns Cul­li­nan $249M pub­lic de­but

In chip­ping in­to Cul­li­nan On­col­o­gy’s up­sized $249.9 mil­lion raise, in­vestors are nei­ther buy­ing in­to a sin­gle drug nor a plat­form. Rather, they are lav­ish­ing on a taster menu of some of the hottest ideas on fight­ing can­cer.

Patrick Baeuer­le

The ethos of the com­pa­ny, which was found­ed by biotech vet and MPM part­ner Patrick Baeuer­le, is per­haps summed up in this line from the prospec­tus: “(W)e think about cap­i­tal al­lo­ca­tion and risk as much as we think about drug de­vel­op­ment.”

That means cen­tral­iz­ing R&D, BD and ad­min­is­tra­tive work in one hold­ing com­pa­ny while bet­ting on as many dif­fer­ent ap­proach­es as pos­si­ble — and cut­ting any weak­lings loose mer­ci­less­ly. The 17 full-time staffers (along­side one part-timer and two con­sul­tants) are de­ployed to the sev­en pro­grams as need­ed.

CLN-081 is the first drug in the clin­ic and the on­ly tar­get­ed ther­a­py in the mix, tar­get­ing NSCLC with EGFR ex­on 20 in­ser­tion mu­ta­tions. Oth­er tech ap­proach­es rep­re­sent­ed in­clude bis­pecifics, NK cell-en­gag­ing an­ti­body, cy­tokine fu­sion pro­tein, as well as TCR-based ther­a­py.

Cul­li­nan filed for its IPO short­ly af­ter pre­sent­ing what they called ini­tial clin­i­cal da­ta on it, and just on the heels of an­nounc­ing a $131 mil­lion Se­ries C.

With $153M SPAC, Lo­cust Walk grabs a seat the head of the deal ta­ble

Hav­ing long billed it­self as a life sci­ence trans­ac­tion firm, Lo­cust Walk is ready to strike a deal of its own.

Chris Ehrlich

Its blank check com­pa­ny, Lo­cust Walk Ac­qui­si­tion Corp, is the first SPAC to gain a list­ing on Nas­daq through an IPO that brought in $153 mil­lion. The mon­ey will now go to­ward snap­ping up a promis­ing pri­vate com­pa­ny to be merged with the new­ly pub­lic shell.

As with oth­er deals of this kind, the peo­ple are re­al­ly what in­vestors are putting their mon­ey be­hind. In this case, Chris Ehrlich, Lo­cust Walk’s se­nior man­ag­ing di­rec­tor and glob­al head of strate­gic trans­ac­tions, is grab­bing the CEO post and tak­ing the lead on the quest to find a suit­able ac­qui­si­tion tar­get.

He will be joined by Daniel Gef­fken, the founder and man­ag­ing di­rec­tor at Dan­forth Ad­vi­sors, as CFO; Brain At­wood of Ver­sant as chair­man; as well as a hand­ful of di­rec­tors in­clud­ing Bar­bara Kosacz, Kro­nos’ COO and Eliz­a­beth Bhatt, the chief busi­ness and strat­e­gy of­fi­cer of Ap­plied Mol­e­c­u­lar Trans­port.

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Jason Kelly (Photographer: Kyle Grillot/Bloomberg via Getty Images)

Gink­go nabs $DNA, biotech's most sought af­ter tick­er, for free in sweet­en­er from NYSE

When Ginkgo went comparison shopping for a financial market to list their now $15 billion company, the New York Stock Exchange had a back-pocket sweetener the Nasdaq couldn’t offer: The most sought-after ticker in biotech, $DNA.

DNA — the most famous three letters in biology and the ticker for the world’s first biotech, Genentech, from 1999 until it was bought out by Roche for $48 billion in 2009 — will now be the ticker for Ginkgo, a 12-year-old synthetic biology startup with grand ambitions to change not only how drugs, but also everyday products like meat and perfumes, are made.

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Lark­spur Health Ac­qui­si­tion files to go pub­lic as this year's SPAC flood surges over $14B

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

Another day, another SPAC vying for a spot on Nasdaq.

On Wednesday, OncoSec Medical CEO Daniel O’Connor filed the S-1 paperwork for a new blank-check company he’s leading called Larkspur Health Acquisition. The former Advaxis chief penciled in a $75 million raise, with plans to offer 7.5 million shares at $10 apiece.

BAR­DA slows its $9B en­gine for new Covid-19 ther­a­peu­tics

The Biomedical Advanced Research and Development Authority is cooling its jets in looking for new, potential Covid-19 treatments, at least in the near term.

An HHS spokesperson told Endpoints News via email, “to date, BARDA has obligated more than $9 billion for the development and/or purchase of 13 therapeutics, beginning in February 2020 with support to develop Regeneron’s monoclonal antibody therapeutic. Therapeutics are an important element of the COVID-19 response, and we are focused on the programs currently underway and/or in negotiation using the funds available to us.”

Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.