Al­lied with Penn, Tmu­ni­ty’s cell ther­a­py pi­o­neers bag $100M mega-round to back a break­through quest on CAR-T, CRISPR

No­var­tis vet Us­man “Oz” Azam and Penn re­searcher Carl June are two of the ul­ti­mate in­sid­ers in the CAR-T rev­o­lu­tion. They each played promi­nent roles in ad­vanc­ing No­var­tis’ break­through ther­a­py Kym­ri­ah to an his­toric first-ever ap­proval. They are now al­lied in an at­tempt to do it all much, much bet­ter with next-gen tech which just at­tract­ed a $100 mil­lion mega-round from an un­con­ven­tion­al group of glob­al glo­biotech in­vestors.

And af­ter stay­ing qui­et for more than a year, they’re al­ready in the clin­ic with two open pro­grams, play­ing a key role co-fund­ing a cut­ting-edge cell ther­a­py study at Penn us­ing CRISPR to mod­i­fy im­mune cells.

Azam, you may re­call, head­ed up No­var­tis’ cell and gene ther­a­py unit be­fore its spec­tac­u­lar breakup a year and a half ago, as the phar­ma gi­ant man­aged the un­like­ly task of slash­ing its costs while main­tain­ing the lead on get­ting the first CAR-T ther­a­py ap­proved. As the re­struc­tur­ing went in place in the sum­mer of 2016, Azam left and soon af­ter jumped to the Penn spin­out, re­main­ing re­lent­less­ly un­der the radar — un­til to­day.

So what, giv­en the bil­lions of dol­lars be­ing in­vest­ed in I/O, makes Tmu­ni­ty dif­fer­ent from the rest of the grow­ing crowd?

Azam quick­ly ze­roes in on June and the net­work of sci­en­tists at Penn en­list­ed in the ex­clu­sive col­lab­o­ra­tion deal they have to work to­geth­er.

It’s the “biotech-like struc­ture that Carl found­ed, that can trans­late these ther­a­pies through the reg­u­la­to­ry hur­dles and in­to clin­i­cal tri­al set­tings” that make one key dif­fer­ence, he says.

Azam of­fered that Tmu­ni­ty along with the Park­er In­sti­tute for Can­cer Im­munother­a­py are fund­ing a new­ly opened — and close­ly watched — tri­al at the Uni­ver­si­ty of Penn­syl­va­nia us­ing CRISPR gene edit­ing tech to mod­i­fy can­cer cells. And he adds there’s al­so a clin­i­cal tri­al un­der­way in prostate can­cer they’re back­ing as well, with more pro­grams in the pipeline out of Penn.

I asked Azam if there was one key thing he had learned about the field at No­var­tis. His re­ply:

“The big learn­ing I had was link­ing and bring­ing to­geth­er trans­la­tion­al med­i­cine and man­u­fac­tur­ing as soon as pos­si­ble.”

Jiang Zhang

The jour­ney from proof-of-con­cept to com­mer­cial­iza­tion was just 7 years, he adds. “That’s pret­ty breath­tak­ing.” And do­ing it re­quired a quick leap from ear­ly clin­i­cal de­vel­op­ment in trans­la­tion­al med­i­cine and “em­brac­ing man­u­fac­tur­ing and tech­ni­cal com­pe­ten­cies ear­ly on. That’s been the biggest chal­lenge for the field. Not just aca­d­e­m­ic set­ting suc­cess, but mak­ing prod­ucts.”

The in­vestors in­clude:

— Gilead, the new own­er of CAR-T play­er Kite, which has vowed to stay in the fore­front of new tech in the space.

— The Park­er In­sti­tute, the non­prof­it re­search group backed by Face­Book bil­lion­aire Sean Park­er, out to spark new brush­fires of in­no­va­tion.

— Ping An Ven­tures, the ven­ture arm of the Glob­al For­tune 500 Chi­nese in­sur­ance out­fit, which al­so backed Shang­hai-based In­novent Bi­o­log­ics.

— And Be The Match Bio­Ther­a­pies, with the seed round in­vestors at the Uni­ver­si­ty of Penn­syl­va­nia and Lil­ly Asia Ven­tures.

“We were al­so at­tract­ed to the glob­al po­ten­tial of the pipeline, es­pe­cial­ly the T cell ther­a­pies in on­col­o­gy in Chi­na, as well as the scope be­yond on­col­o­gy in­to au­toim­mune and in­fec­tious dis­eases, as we be­gin to ex­pand our in­vest­ment port­fo­lio,” said Jiang Zhang, man­ag­ing part­ner of Ping An Ven­tures.

Michael Polan­sky

I asked the Park­er In­sti­tute why they de­cid­ed to help bankroll the com­pa­ny, and got a re­sponse from Michael Polan­sky, pres­i­dent of the Park­er Group and mem­ber of the Park­er In­sti­tute for Can­cer Im­munother­a­py Board of Di­rec­tors:

As you know Carl June at the Uni­ver­si­ty of Penn­syl­va­nia is such a gi­ant in the field of cell ther­a­py and CAR-T, such a pi­o­neer, which is why we want­ed to bring him on as a di­rec­tor at the Park­er In­sti­tute. His lead­er­ship and in­sight in­to the field of cell ther­a­py are in­valu­able.

With him as sci­en­tif­ic founder of Tmu­ni­ty, that gave us a lot of con­fi­dence that the tech­nol­o­gy and the sci­ence will be top tier. And from what we’ve seen there’s no ques­tion that they have a sol­id plan for de­vel­op­ing the next wave of suc­cess­ful cell and gene ther­a­pies, re­al­ly trans­lat­ing those the dis­cov­er­ies so they can be de­vel­oped in­to ther­a­pies at an ac­cel­er­at­ed pace to help more can­cer pa­tients faster.

So far, the biotech has made all of two an­nounce­ments, start­ing with Azam’s ar­rival and end­ing with Mike Chris­tiano’s move to the BD side of the busi­ness af­ter play­ing a sim­i­lar role in No­var­tis.

Now that the A round is in, Azam says he can start re­cruit­ing, build­ing up the small team as they con­tin­ue to work with top in­ves­ti­ga­tors at Penn on the re­search, tech­nol­o­gy and clin­i­cal tri­als.

“It’s a great space to be in now,” he says, “and will be for decades to come.”

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His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; 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.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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Seagen interim CEO Roger Dansey and Daiichi Sankyo CEO Sunao Manabe

Paving the way for Mer­ck­'s buy­out, Seagen los­es ar­bi­tra­tion dis­pute with Dai­ichi over ADC tech

As Seagen awaits a final buyout offer from Merck that could be in the territory of $40 billion, Seagen revealed Friday afternoon that it lost an arbitration dispute with Daiichi Sankyo relating to the companies’ 2008 collaboration around the use of antibody-drug conjugate (ADC) technology.

But that loss likely won’t matter much when it comes to Merck’s deal.

After breaking off its pact with Daiichi in mid-2015, the two companies battled over “linker” tech — a chemical bridge between an ADC’s antibody component and the cytotoxic payload — that Seagen claims Daiichi would improve upon and implement in its current generation of ADCs.

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House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

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