No­var­tis’ CAR-T chief Azam is leav­ing, but phar­ma gi­ant de­nies any ‘re­treat’ is un­der­way


No­var­tis re­spond­ed to a re­port in End­points News this morn­ing which out­lines the com­pa­ny’s de­ci­sion to purge most of the ex­ec­u­tive team in­volved in its pi­o­neer­ing cell and gene ther­a­py unit. Ac­cord­ing to the com­pa­ny, the de­ci­sion to cut up to 120 staffers as it re­de­ployed most of the 400 staffers back in­to its on­col­o­gy di­vi­sion does not sig­ni­fy any kind of a “re­treat.”

No­var­tis is still on track to nab the first FDA OK for a CAR-T to treat pe­di­atric acute lym­phoblas­tic leukemia, as­serts a com­pa­ny state­ment. In ad­di­tion, the com­pa­ny said that Us­man “Oz” Azam, the glob­al chief for this unit, has de­cid­ed to leave the com­pa­ny, while oth­er team ex­ecs in­volved in the cut­back still have a chance to land new jobs at the phar­ma gi­ant.

Ear­li­er this week End­points broke the sto­ry that No­var­tis is dis­band­ing the unit, which had been charged with de­vel­op­ing No­var­tis’s CAR-T pipeline, backed with con­sid­er­able hoopla two years ago in a cov­er sto­ry on CEO Joe Jimenez in Forbes.

This morn­ing, we fol­lowed up with a new re­port af­ter gain­ing ac­cess to the full memo that Azam had writ­ten. In it, he not­ed:

Un­for­tu­nate­ly a num­ber of col­leagues will be im­pact­ed by this change as many po­si­tions are be­ing elim­i­nat­ed. Im­pact­ed US-based as­so­ciates are be­ing no­ti­fied in meet­ings to­day. As­so­ci­ates based in Basel will learn more about their in­di­vid­ual cir­cum­stances on Thurs­day. The ma­jor­i­ty of the CG­TU Lead­er­ship Team mem­bers, who are among the best I have worked with, are al­so im­pact­ed.

The com­pa­ny’s re­sponse, though, is not like­ly to change the grow­ing per­cep­tion that the com­pa­ny is pulling back from the field just as the first CAR-T drugs are be­ing prepped for the FDA. Kite is now the wide­ly rec­og­nized leader af­ter a re­cent set­back at Juno, with plans to hus­tle an ap­pli­ca­tion to the FDA lat­er this year. And a num­ber of oth­er com­pa­nies are in the hunt to fol­low up with new drugs of their own.

Here’s the full re­sponse:

No­var­tis is seek­ing to clar­i­fy mis­in­for­ma­tion in the lat­est End­points re­port on the re-in­te­gra­tion of the Cell & Gene Ther­a­pies Unit (CG­TU) in­to the broad­er No­var­tis or­ga­ni­za­tion.

Con­trary to your head­line that we are “re­treat­ing from CAR-T,” our com­mit­ment to CAR-T and CRISPR tech­nolo­gies re­mains strong.  As we have pre­vi­ous­ly stat­ed, CTL019 is on track to be filed with the FDA ear­ly next year and with the EMA lat­er in 2017.  In fact, we ex­pect CTL019 will be the first chimeric anti­gen re­cep­tor T-cell (CAR T) ther­a­py to be ap­proved for pe­di­atric acute lym­phoblas­tic leukemia.

Dr. Us­man Azam along with his lead­er­ship team helped to de­sign the en­tire re-in­te­gra­tion plan. Ac­tions like re-in­te­gra­tion in­to oth­er parts of busi­ness­es can cause re­dun­dan­cies in roles, e.g. HR, fi­nance, le­gal, and that is why some peo­ple will leave our or­ga­ni­za­tion.  Dr. Azam de­cid­ed that as part of the re-in­te­gra­tion he would like to use this tran­si­tion to look at new op­por­tu­ni­ties out­side of the com­pa­ny. The CG­TU lead­er­ship team, along with ap­prox­i­mate­ly 120 oth­er as­so­ciates who will be im­pact­ed, have the op­por­tu­ni­ty to stay at No­var­tis if there is a role that is avail­able or suit­able for them.  It is im­por­tant to note that most of the CG­TU em­ploy­ees will be re­de­ployed to oth­er parts of the com­pa­ny.

At No­var­tis, we are trans­form­ing the com­pa­ny to be more fo­cused with a less-com­plex, more ag­ile struc­ture. While the CG­TU struc­ture was crit­i­cal to ramp up our ca­pa­bil­i­ties in cer­tain ar­eas like man­u­fac­tur­ing, clin­i­cal tri­als and com­mer­cial­iza­tion for a com­plete­ly new way of treat­ing can­cer, now that the com­pa­ny has re-or­ga­nized un­der a new op­er­at­ing mod­el, the re-in­te­gra­tion of CG­TU is the log­i­cal next step in our evo­lu­tion.

The No­var­tis en­ter­prise has all of the ca­pa­bil­i­ties we need to ef­fi­cient­ly re-in­te­grate CG­TU in­to the broad­er or­ga­ni­za­tion: lead­er­ship in on­col­o­gy and hema­tol­ogy specif­i­cal­ly, a glob­al com­mer­cial and de­vel­op­ment foot­print, a strong en­ter­prise man­u­fac­tur­ing or­ga­ni­za­tion, as well as sup­port func­tions to en­able a suc­cess­ful tran­si­tion.

No­var­tis is proud that we have played a lead­er­ship role in ad­vanc­ing the sci­en­tif­ic con­cept of CAR-T to the point where this com­plex way of treat­ing can­cer can be­come a re­al­i­ty for pa­tients. We look for­ward to con­tin­u­ing to ad­vance the sci­ence and our ca­pa­bil­i­ties in this ex­cit­ing area of med­i­cine.

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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UP­DAT­ED: New play­ers are jump­ing in­to the scram­ble to de­vel­op a vac­cine as pan­dem­ic pan­ic spreads fast

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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Mer­ck KGaA spin­out gets first fund­ing to bring dual-act­ing can­cer mol­e­cules in­to the clin­ic

Two and a half years after launch, Merck KGaA spinout iOnctura is getting its first major round of funding.

The oncology startup raised €15 million ($16.6 million) to put its lead drug into the clinic and get its second drug past IND-enabling tests. INKEF Capital and VI Partners co-led the round and were joined by the biotech’s longtime backer M Ventures, an arm of Merck KGaA, and Schroder Adveq.

Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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Am­gen aug­ments Asia foothold by tak­ing over Astel­las joint ven­ture in Japan

California-based Amgen, which does the bulk of its business in the United States, made its ambition to reinvigorate its growth prospects by expanding its presence in Asia clear at the sidelines of the JP Morgan healthcare conference in San Francisco earlier this month.

The Thousand Oaks-based company on Thursday executed its plan to dissolve the joint venture with Astellas — created in 2013 — to operate the unit independently in Japan. With its rapidly aging population, the region represents an appealing market for Amgen’s osteoporosis treatments Prolia and Evenity as well as a cholesterol-lowering injection Repatha.

Daphne Zohar (PureTech)

PureTech bags $200M from sale of Karuna shares — still siz­zling from promis­ing schiz­o­phre­nia da­ta

Cashing in on the exuberance around Karuna Therapeutics and its potential blockbuster CNS drug, PureTech has sold a chunk of the biotech’s shares to Goldman Sachs for $200 million.

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