Scoop: No­var­tis dis­bands its pi­o­neer­ing cell and gene ther­a­py unit

No­var­tis is dis­solv­ing its high-pro­file cell and gene ther­a­py unit op­er­at­ing un­der the guid­ing hand of Us­man “Oz” Azam, End­points has learned.

Us­man “Oz” Azam

Azam has is­sued a note to the 400 staffers in the group, which was ob­tained by End­points, that says:

“The risk of em­bark­ing on a new ad­ven­ture in un­chart­ed ter­ri­to­ry is that things don’t al­ways work out as en­vi­sioned. To­day, I have the un­for­tu­nate task of an­nounc­ing that we are dis­solv­ing the Cell and Gene Ther­a­pies Unit.”

No­var­tis con­firmed that the 400-mem­ber group is be­ing dis­band­ed, with most be­ing “re­de­ployed” to new po­si­tions while 120 could be left with­out a job.

That move is a stun­ner for many in the CAR-T field, es­pe­cial­ly af­ter all the hoopla at No­var­tis around its work.

Azam and his team have played point for one of No­var­tis’s biggest pipeline moves, jump­ing in ear­ly on CAR-T ther­a­pies that reengi­neer a pa­tient’s T cells in­to at­tack ve­hi­cles point­ed at can­cer cells. Work­ing through a part­ner­ship at the Uni­ver­si­ty of Penn­syl­va­nia, the group has ad­vanced one of the lead­ing CAR-T ther­a­pies now in the pipeline, as it races against Kite $KITE and Juno $JUNO to be in the first wave of new drugs to hit the mar­ket.

The field has been mov­ing fast, but not al­ways for­ward. Juno ex­pe­ri­enced a brief in­ter­rup­tion for its lead pro­gram when the FDA im­posed a clin­i­cal hold for a few days that ul­ti­mate­ly de­railed its de­vel­op­ment sched­ule and de­layed any ap­pli­ca­tion un­til 2018. That leaves Kite and No­var­tis rac­ing to the FDA, with Kite plan­ning a sub­mis­sion lat­er this year.

At one point, No­var­tis CEO Joe Jimenez told re­porters that he had made out a blank check for its CAR-T work, de­ter­mined to pay what was nec­es­sary to get these drugs to the mar­ket as fast as pos­si­ble. In a re­cent let­ter to share­hold­ers, Jimenez high­light­ed the CAR-T work as a key part of its strat­e­gy for its on­col­o­gy R&D ef­fort. But in the midst of a heat­ed Phase III ri­val­ry, the phar­ma gi­ant be­lieves that an iso­lat­ed unit no longer makes sense. And that is like­ly to add to the im­pres­sion that No­var­tis is falling be­hind.

The news hit Kite and Juno as well, though. Shares of Juno dropped 5% Wednes­day morn­ing, with Kite down 4%.

In re­sponse to a query from End­points, a spokesper­son for No­var­tis is­sued the fol­low­ing state­ment:

“We have made the de­ci­sion to re-in­te­grate ac­tiv­i­ties con­duct­ed by the Cell & Gene Ther­a­pies Unit in­to the larg­er No­var­tis or­ga­ni­za­tion, as part of a nat­ur­al evo­lu­tion of our in­ter­nal or­ga­ni­za­tion­al de­sign. An iso­lat­ed Unit worked well un­der our pri­or Phar­ma Di­vi­sion struc­ture, but with a new in­te­grat­ed de­vel­op­ment mod­el, we can ef­fi­cient­ly ad­vance our work on CART as part of our fo­cus in im­muno-on­col­o­gy by rein­te­grat­ing the func­tions.

This or­ga­ni­za­tion­al change will not im­pact the plan to file CTL019 in pe­di­atric r/r ALL with the US FDA in ear­ly 2017 and with EMA lat­er in 2017.

Most as­so­ciates who were pre­vi­ous­ly ded­i­cat­ed to cell & gene ther­a­pies will now be re­de­ployed to ar­eas where they will share their knowl­edge and im­prove ex­e­cu­tion of nov­el ther­a­peu­tics in the im­munother­a­py space to bet­ter de­liv­er on our mis­sion to im­prove and ex­tend peo­ple’s lives.

These or­ga­ni­za­tion­al changes will im­pact ap­prox­i­mate­ly 120 po­si­tions across sev­er­al dif­fer­ent No­var­tis af­fil­i­ates.  No­var­tis is com­mit­ted to as­sist­ing in the place­ment of im­pact­ed as­so­ciates wher­ev­er ap­pro­pri­ate in­ter­nal op­por­tu­ni­ties may ex­ist and in treat­ing dis­placed as­so­ciates fair­ly and with dig­ni­ty.”

In a fol­lowup, No­var­tis added that the move to dis­solve the unit will not af­fect its col­lab­o­ra­tion with Penn.

No­var­tis and Penn have an ex­clu­sive glob­al col­lab­o­ra­tion to re­search, de­vel­op and com­mer­cial­ize CAR T cell ther­a­pies for the in­ves­ti­ga­tion­al treat­ment of can­cers. We con­tin­ue to work with Penn un­der the terms of our agree­ment and look for­ward to ad­vanc­ing CART ther­a­pies.

In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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A top drug pro­gram at Bay­er clears a high bar for CKD — open­ing the door to an FDA pitch

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

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Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Adrian Gottschalk, Foghorn CEO (Foghorn)

Foghorn hits Nas­daq in $120M de­but as the biotech IPO boom shows no sign of slow­ing

It’s been a record year for biotech IPOs, and the execs at Nasdaq would like nothing better than to see that momentum continue into the first half of next year.

Since January, 72 biotech and biopharma companies have hit Wall Street, according to Nasdaq head of healthcare listings Jordan Saxe, together raising $13.2 billion.

The latest is Flagship’s Foghorn Therapeutics, which priced its shares last night at $16 apiece, the midpoint of a $15 to $17 range. The Cambridge, MA-based biotech — which initially filed for a $100 million raise on Oct. 2 — is netting $120 million from a 7.5 million-share offering. The proceeds will go right into its gene traffic control platform, including two lead preclinical oncology candidates.

News brief­ing: Gilead com­pletes $21B buy­out of Im­munomedics; In­nate re­ceives $50M mile­stone pay­ment from As­traZeneca

Gilead’s $21 billion mega-acquisition of Immunomedics is now officially complete, the companies announced Friday morning.

The full merger process took a little over a month, with Gilead and Immunomedics signing an agreement on Sept. 13. Gilead acquired all outstanding stock of Immunomedics for $88 per share, a 108% premium on the previous day’s closing price.

Gilead’s big prize was Trodelvy, approved in July for the treatment of metastatic triple-negative breast cancer. The drug also impressed at last month’s ESMO conference, reducing the risk of death by 52% in a Phase III study.