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.

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Tom Barnes, Orna Therapeutics CEO

UP­DAT­ED: 'We have failed to fail': Mer­ck gam­bles $250M cash on a next-gen ap­proach to mR­NA — af­ter punt­ing its big al­liance with Mod­er­na

Merck went in deep on its collaboration with Moderna on new mRNA programs, and dropped them all over time, including their RSV partnership. But after writing off what turned out as one of the most successful infectious disease players in the business, Merck is coming in this morning with a new preclinical alliance — this time embracing a biotech that hopes to eventually outdo the famously successful mRNA in a new run at vaccines and therapeutics.

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Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”

FDA ap­proves one of the prici­est new treat­ments of all time — blue­bird's gene ther­a­py for be­ta tha­lassemia

The FDA on Wednesday approved the first gene therapy for a chronic condition — bluebird bio’s new Zynteglo (beti-cel) as a potentially curative treatment for those with transfusion-dependent thalassemia.

The thumbs up from the FDA follows a unanimous adcomm vote in June, with outside experts pointing to extraordinary efficacy, with 89% of subjects with TDT who received beti-cel having achieved transfusion independence.

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Joel Dudley, new partner at Innovation Endeavors (Tempus Labs)

For­mer Google CEO’s VC is mak­ing a big­ger push in­to the biotech world, hir­ing promi­nent Ther­a­nos skep­tic

Venture capital firm Innovation Endeavors has mainly had its focus on investments across the tech space, but it has been slowly turning its attention to the biotech world. Now, a new partner is coming into the fold showing that its interest in biotech is likely to grow further.

The Silicon Valley-based company, which is headed up by former Google CEO Eric Schmidt, has brought on Joel Dudley as a partner. According to Dudley’s LinkedIn page, he is joining Innovation Endeavors after serving as the chief science officer of biotech startup Tempus Labs from 2020.

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Paul Perreault, CSL Behring CEO

CSL CEO Paul Per­reault de­ter­mined to grow plas­ma col­lec­tion af­ter full-year sales dip

As the ink dries on CSL’s $11.7 billion Vifor buyout, the company posted a dip in profits, due in part to a drop in plasma donations amid the pandemic.

However, CEO Paul Perreault assured investors and analysts on the full-year call that the team has left “no stone unturned” when assessing options to grow plasma volumes. The chief executive also spelled out positive results for the company’s monoclonal antibody garadacimab in hereditary angioedema (HAE), though he isn’t revealing the exact numbers just yet.

Blaise Coleman, Endo International CEO

En­do files for Chap­ter 11 as it looks to fin­ish off its opi­oid lit­i­ga­tion

Irish drugmaker Endo International is entering into bankruptcy as it faces the weight of serious litigation related to its involvement in the opioid epidemic in the US.

The company has filed Chapter 11 proceedings in the US Bankruptcy Court for the Southern District of New York, with the company expected to file recognition proceedings in Canada, the UK and Australia. The company’s bankruptcy filing showed the company had assets and liabilities in the range of $1 billion to $10 billion.