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.”

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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San Diego cou­ple charged with steal­ing trade se­crets, open­ing Chi­nese biotech as DOJ crack­down con­tin­ues

A San Diego couple has been charged with stealing trade secrets from a US hospital and opening a business based off those secrets in China as the controversial industry-wide crackdown on alleged corporate espionage continues. On the same day, the Department of Justice announced they had arrested Beijing representative Zhongsan Liu for allegedly trying to obtain research visas for government recruiters.

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Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

UP­DAT­ED: Bio­gen pulls the plug on prized IPF drug from $562M+ Stromedix buy­out

One of Biogen’s attempts to branch out has flopped as the biotech scraps a mid-stage program for idiopathic pulmonary fibrosis.

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Warts for the win: Aclar­is' lead drug clears piv­otal study

Aclaris Therapeutics has found a way to get rid of the warts and all.

The company — which earlier this month decided to focus on its arsenal of kinase inhibitors — on Monday unveiled positive data from a pivotal study testing its lead experimental drug for use in common warts.

The drug, A-101, was tested in a 502-patient study called THWART-2 — patients enrolled had one to six warts before qualifying for the trial. Patients either self-administered A-101 topical solution or a vehicle twice a week over a two-month period. A higher proportion of patients on the drug (a potent hydrogen peroxide topical solution) saw their warts disappear at day 60, versus the vehicle (p<0.0001) — meeting the main goal of the study.  Each secondary endpoint also emerged in favor of A-101, the company said.

Charles Nichols, LSU School of Medicine

Could psy­che­delics tack­le the obe­si­ty cri­sis? A long­time re­searcher in the field says his lat­est mouse study sug­gests po­ten­tial

Psychedelics have experienced a renaissance in recent years amid a torrent of preclinical and clinical research suggesting it might provide a path to treat mood disorders conventional remedies have only scraped at. Now a preclinical trial from a young biotech suggests at least one psychedelic compound has effects beyond the mind, and — if you believe the still very, very early hype — could provide the first single remedy for some of the main complications of obesity.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Ac­celeron drops a de­vel­op­ment pro­gram as #2 drug fails to spark func­tion­al ben­e­fits in pa­tients with a rare neu­ro­mus­cu­lar ail­ment

Acceleron is scrapping a muscular dystrophy development program underway for its number 2 drug in the pipeline after pouring over some failed mid-stage secondary data.

Gone is the ACE-083 project in patients with facioscapulohumeral muscular dystrophy. Their drug hit the primary endpoint on building muscle but flopped on key secondaries for functional improvements in patients, which execs felt was vital to the drug’s success.