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.”

In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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It is 'kind of a proven tech­nol­o­gy': Hep B vac­cine mak­er joins glob­al hunt for coro­n­avirus vac­cine

Using lab-grown proteins that are engineered to mimic the architecture of viruses to induce an immune response, VBI Vaccines is joining the hunt for a coronavirus vaccine — harnessing technology that has initially been proved safe in early trials as a prophylactic for cytomegalovirus (CMV) infection.

Unlike the raft of the companies in the Covid-19 vaccine race — including Moderna, CureVac and J&J — VBI is taking a pan-coronavirus approach, by developing a vaccine that will encompass Covid-19, severe acute respiratory syndrome (SARS), and Middle East respiratory syndrome (MERS).

Can a pair of top AveX­is alum­ni steer a new gene ther­a­py up­start to R&D glo­ry? 3 VCs bet $60M on it

VCs love few things more than a proven executive team when it comes to launching a new company. And now a group of A-listers has turned to a pair of top execs out of AveXis to steer the latest gene therapy player into the clinic.

The biotech is Waltham, MA-based Affinia and the two execs are Sean Nolan and Rick Modi — the former CEO and CBO respectively of AveXis, the gene therapy pioneer that fetched $8.7 billion in a sale to Novartis. Nolan has now taken the chairman’s role at Affinia while Modi moves up to the CEO post at the company.

Un­de­terred by a pan­dem­ic, Gilde Health­care rais­es their largest fund yet

When Pieter van der Meer started raising the capital for Gilde Healthcare’s fifth fund in the waning months of 2019, he had his eyes on a different chain of events that could change the healthcare system and perhaps even play to his firm’s advantage: The US presidential election.

Since raising their third fund in 2011, the 34-year-old Dutch firm had focused on value-based care. They chose late-stage biotechs that came up with new devices and delivery systems for de-risked established compounds, and when they chose preclinical biotechs, they spoke with potential pharma partners, payers and regulators to ask where and at what prices the drug made sense. As the Democratic primary became a contest over how to lower healthcare costs, it looked like a strategy that could pay off.

Daniel O'Day (AP Images)

Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

In addition to trying to reassure patients that they will once again have an avenue to pursue access, O’Day also reassured some analysts who had been fretting that China’s quick comeback from the coronavirus outbreak could derail its ultra-fast schedule for testing the drug in patients. The data are still expected in a few weeks, he says in the letter, putting the readout in April.

O’Day emphasizes that Gilead intends to pursue a pricing approach that will make this drug widely available — if it proves effective and safe. But no one is quite sure just what the longterm value would be, given the work being done on a variety of vaccines that may be rolled out as early as this fall — at least to the most heavily threatened groups.

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Covid-19 roundup: J&J, BAR­DA set ear­ly 2021 fin­ish line for $1B vac­cine race; FDA al­lows emer­gency drug use, ahead of piv­otal da­ta

J&J has zeroed in on a Covid-19 vaccine candidate that it hopes to begin testing in humans by September this year — with the extraordinary goal of getting it ready for emergency use in early 2021. And together with BARDA, it’s committing $1 billion to make it happen.

That kind of accelerated timeline would fall on the fast side of NIAID director Anthony Fauci’s well-publicized prediction that it would be another 12 to 18 months before a vaccine can be available for public use. A Phase I trial of Moderna’s mRNA vaccine began two weeks ago, and both the biotech and fellow mRNA player CureVac have discussed similar, if not even faster, timelines for emergency use among healthcare workers.