No­var­tis wins a key OK for Kym­ri­ah, but con­tin­u­ing man­u­fac­tur­ing woes hob­ble roll­out — ri­val Gilead CAR-T breaks in­to Eu­rope

No­var­tis CAR-T man­u­fac­tur­ing woes are con­tin­u­ing to put a crimp in its abil­i­ty to pro­vide ther­a­py to all po­ten­tial pa­tients.

Vas Narasimhan

The phar­ma gi­ant re­port­ed this morn­ing that the EC has stamped its ap­proval on mar­ket­ing Kym­ri­ah in Eu­rope for B-cell acute lym­phoblas­tic leukemia as well as treat­ment-re­sis­tant adults suf­fer­ing from dif­fuse large B-cell lym­phoma. But No­var­tis can on­ly make it avail­able to chil­dren with ALL now — adults will have to wait.

No­var­tis’s top ex­ecs have worked hard to smooth things over with an­a­lysts, but it’s been be­dev­iled by man­u­fac­tur­ing is­sues through­out the de­vel­op­ment stage and con­tin­ues to be af­fect­ed as it goes toe-to-toe with Gilead’s Kite, ap­proved for large B-cell lym­phoma. And Gilead is still com­ing on strong, not­ing this morn­ing that they re­ceived a Eu­ro­pean OK to mar­ket Yescar­ta for dif­fuse and pri­ma­ry me­di­asti­nal large B-cell lym­phoma.

A month ago No­var­tis CEO Vas Narasimhan not­ed dur­ing the Q2 call that:

I think it’s ear­ly days and we’ve al­ways said this is go­ing to be a five-year jour­ney with Kym­ri­ah to re­al­ly get us to be the glob­al­ly suc­cess­ful brand we want it to be.

On man­u­fac­tur­ing, we have seen some vari­abil­i­ty in our prod­uct spec­i­fi­ca­tions. This is some­thing we’re look­ing at now in DL­B­CL to make sure that we can con­tin­ue to ramp-up the de­mand. We feel con­fi­dent in the over­all longer-term out­look for Kym­ri­ah.

Liz Bar­rett

Liz Bar­rett, who runs No­var­tis on­col­o­gy, had this to say:

I think the most im­por­tant thing to let you know is that the vari­abil­i­ty that we’ve seen in the com­mer­cial spec­i­fi­ca­tions, which isn’t un­usu­al with a new ther­a­py as you launch in­to a new tar­get pa­tient pop­u­la­tion, is that we have still been able to de­liv­er fi­nal prod­uct to the ma­jor­i­ty of pa­tients.

As we’ve re­port­ed, though, the cell man­u­fac­tur­ing chief’s of­fice has been out­fit­ted with a re­volv­ing door at No­var­tis in re­cent years. Karen Walk­er left that post in ear­ly 2017, fol­lowed last spring by Spencer Fisk as he jumped ship to join the No­var­tis ex­ecs at Ru­bius. Those de­par­tures came on top of a ma­jor re­vamp for the cell and gene ther­a­py unit in 2016, which raised ques­tions at the time about No­var­tis’ abil­i­ty to keep up with Kite — though the phar­ma gi­ant wound up with the pi­o­neer­ing first OK. And No­var­tis’ is­sues with man­u­fac­tur­ing cell ther­a­pies ex­tend back to the sna­fus it ex­pe­ri­enced with late de­liv­ery to a small but sig­nif­i­cant group of pa­tients en­rolled in clin­i­cal tri­als.

At one stage in the de­vel­op­ment process, No­var­tis had a 6% fail­ure rate on the man­u­fac­tur­ing process in a key clin­i­cal tri­al, which they lat­er said was large­ly re­solved and brought down to 3%.

This morn­ing No­var­tis is fol­low­ing up with plans to in­vest €78.8 mil­lion in­to their cell and gene ther­a­py man­u­fac­tur­ing op­er­a­tions in Switzer­land in their lat­est stab at fix­ing the prob­lems.

But af­ter record­ing on­ly $16 mil­lion in sales for Q2, the big ques­tion loom­ing for No­var­tis is why pa­tients would want to take a risk with their ther­a­py if an­oth­er provider comes in­to the mar­ket which can do bet­ter on a com­plex task: ex­tract­ing T cells from pa­tients, mod­i­fy­ing them and then in­fus­ing pa­tients with a ther­a­peu­tic punch?

You can be sure that the top crew at Gilead and Kite — work­ing with a be­spoke man­u­fac­tur­ing site — are pay­ing close at­ten­tion.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Ken Frazier, AP Images

Why Mer­ck wait­ed, and what they now bring to the Covid-19 fight

Nicholas Kartsonis had been running clinical infectious disease research at Merck for almost 2 years when, in mid-January, he got a new assignment: searching the pharma giant’s vast libraries for something that could treat the novel coronavirus.

The outbreak was barely two weeks old when Kartsonis and a few dozen others got to work, first in small teams and then in a larger task force that sucked in more and more parts of the sprawling company as Covid-19 infected more and more of the globe. By late February, the group began formally searching for vaccine and antiviral candidates to license. Still, while other companies jumped out to announce their programs and, eventually and sometimes controversially, early glimpses at human data, Merck remained silent. They made only a brief announcement about a data collection partnership in April and mentioned vaguely a vaccine and antiviral search in their April 28 earnings call.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Mark Genovese (Stanford via Twitter)

Gilead woos fil­go­tinib clin­i­cal in­ves­ti­ga­tor from Stan­ford to lead the charge on NASH, in­flam­ma­to­ry dis­eases

With an FDA OK for the use of filgotinib in rheumatoid arthritis expected to drop any day now, Gilead has recruited a new leader from academia to lead its foray into inflammatory diseases.

Mark Genovese — a longtime Stanford professor and most recently the clinical chief in the division of immunology and rheumatology — was the principal investigator in FINCH 2, one of three studies that supported Gilead’s NDA filing. In his new role as SVP, inflammation, he will oversee the clinical development of the entire portfolio.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Stephen Isaacs, Aduro president and CEO (Aduro)

Once a high fly­er, a stag­ger­ing Aduro is auc­tion­ing off most of the pipeline as CEO Stephen Isaacs hands off the shell to new own­ers

After a drumbeat of failure, setbacks and reorganizations over the last few years, Aduro CEO Stephen Isaacs is handing over his largely gutted-out shell of a public company to another biotech company and putting up some questionable assets in a going-out-of-business sale.

Isaacs —who forged a string of high-profile Big Pharma deals along the way — has wrapped a 13-year run at the biotech with one program for kidney disease going to the new owners at Chinook Therapeutics. A host of once-heralded assets like their STING agonist program partnered with Novartis (which dumped their work on ADU-S100 after looking over weak clinical results), the Lilly-allied cGAS-STING inhibitor program and the anti-CD27 program out-licensed to Merck will all be posted for auction under a strategic review process.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Hill­house re­casts spot­light on Chi­na's biotech scene with $160M round for Shang­hai-based an­ti­body mak­er

Almost two years after first buying into Genor Biopharma’s pipeline of cancer and autoimmune therapies, Hillhouse Capital has led a $160 million cash injection to push the late-stage assets over the finish line while continuing to fund both internal R&D and dealmaking.

The Series B has landed right around the time Genor would have listed on the Hong Kong stock exchange, according to plans reported by Bloomberg late last year. Insiders had said that the company was looking to raise about $200 million.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,100+ biopharma pros reading Endpoints daily — and it's free.

Federico Mingozzi (Spark)

Spark touts an­i­mal da­ta for a so­lu­tion to AAV gene ther­a­py's an­ti­body prob­lem

Among all the limitations of using an adeno-associated virus as a vector to deliver a gene — still the most established modality in gene therapy given years of trial and error and finally success — the presence of neutralizing antibodies, whether pre-existing or induced, looms large.

“When I think about the immune responses in AAV, I try to sort of layer them,” Federico Mingozzi, the CSO at Spark Therapeutics, told Endpoints News. “The antibody is the first layer. It’s the first block that you find when you’re trying to do gene transfer.”