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.”

Con­quer­ing a silent killer: HDV and Eiger Bio­Phar­ma­ceu­ti­cals

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

Am­gen chops 172 more staffers in R&D, op­er­a­tions and sales amid neu­ro­science ex­it, rev­enue down­turn

Neuroscience wasn’t the only unit that’s being hit by a reorganization underway at Amgen. As well as axing 149 employees in its Cambridge office, the company has disclosed that 172 others nationwide, including some from its Thousand Oaks, CA headquarters, are being let go.

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Ahead of strate­gic up­date, new Sanofi CEO mulls op­tions for con­sumer health­care arm — re­ports

Big pharma has made moves to sharpen its focus on developing new medicines, while slow-growing consumer health divisions fall by the wayside. Looks like another large drugmaker is considering a similar move. On Thursday, reports citing sources indicated that Sanofi is reportedly mulling a joint venture, sale, or a public listing of its consumer health arm.

The French group is in discussions for options that could value the division at $30 billion, Bloomberg and Reuters reported, citing sources familiar with the matter.

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The triple crown in biotech: An all-or-noth­ing bet on an FDA ap­proval of 3 drugs over 16 months starts to­day

Bristol-Myers Squibb’s $74 billion Celgene deal closed as expected Wednesday evening. And now a new clock has begun to tick down for Celgene shareholders who came away from the deal with CVRs — contingent value rights — worth $9 or nothing. Those CVRs start trading today as $BMYRT.

The new deadline they have is the end of March 2021, a little more than 16 months from now, when Bristol-Myers will need to gain approvals on 3 late-stage drugs it’s picking up in the buyout: Ozanimod and liso-cel (JCAR017) are due up at the end of 2020, with bb2121 deadlined at the end of Q1 in 2021.

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Genap­sys fi­nal­ly un­veils vaunt­ed se­quencer, but can it dent Il­lu­mi­na?

Hesaam Esfandyarpour holds what looks like a mini-cooler up to the computer screen in his California office.

Esfandyarpour is in his late-30s, with crows feet creeping up against a youthful face. He wears a gray polo and the device in his hand — with its hard plastic-looking shell, blue-and-white pattern, and a white plastic paddle resembling a handle jutting out the front — might contain diced strawberries and peanut-butter sandwiches to meet mom and the kids at a SoCal park. Instead, Esfandyarpour tells me it’s going to change medicine and biopharma research.

UP­DAT­ED: Make that 2 ap­proved RNAi drugs at Al­ny­lam af­ter the FDA of­fers a speedy OK on ul­tra-rare dis­ease drug

Seventeen years into the game, Alnylam’s pivot into commercial operations is picking up speed.
The bellwether biotech $ALNY has nabbed their second FDA OK for an RNAi drug, this time for givosiran, the only therapy now approved for acute hepatic porphyria. This second approval came months ahead of the February deadline — even after winning priority review following their ‘breakthrough’ title earlier.
AHP is an extremely rare disease, with some 3,000 patients in Europe and the US, not all diagnosed, and analysts have projected peak revenue of $600 million to $700 million a year. The drug will be sold as Givlaari.

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David Ricks. Eli Lilly

Eli Lil­ly touts $400M man­u­fac­tur­ing ex­pan­sion, 100 new jobs to much fan­fare in In­di­anapo­lis — even though it's been chop­ping staff

Eli Lilly is pouring in $400 million to beef up manufacturing facilities at its home base of Indianapolis. The investment, which was lauded by the city’s mayor, is expected to create 100 new jobs.

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Brii Bio backs in­fec­tious dis­ease start­up while ink­ing deal for its lead TB drug, dou­bling down on an­tibi­otics

Almost two years after leaving GSK to launch Brii Bio with a whopping $260 million in funding, Zhi Hong is seeing the trans-Pacific infectious disease specialist he set out to build take shape.

“Our pipeline is coming together,” he told Endpoints News, with 12 partnered assets plus some internal programs.

As its latest partner, AN2 Therapeutics, comes into the limelight for the first time with a $12 million seed round, so is Brii’s plans in the antibiotics space. Brii has obtained China rights to AN2’s antibacterial targeting mycobacterium tuberculosis for multi-drug resistant TB, which it says is in the clinical stage.

No­var­tis, Bay­er, Long­wood back ge­nomics start­up to speed search for im­munother­a­py tar­gets

Nearly a century passed between the first proto-immunotherapy attempts in cancer — crude and obscure but nonetheless with some scientific basis — and Jim Allison’s first T cell paper. Thirty-plus years flipped between the discovery of CTLA-4 as an off-switch and the approval of Yervoy. Twenty-two rolled between PD-1’s isolation and Opdiva and Keytruda. 

Longwood co-founder Lea Hachigian is betting she can hasten that. It’s a bet on newly established single-cell genomic analysis tech and the ability to crunch endless troves of data at a rate few others can, and investors including Leaps by Bayer and Novartis Venture Fund just put $39 million behind it. They call it Immunitas.