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.

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

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CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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US gov­ern­ment re­port­ed­ly be­gins prepar­ing for Covid-19 chal­lenge tri­als. Are they eth­i­cal?

Controversial human challenge trials for potential Covid-19 vaccines reportedly have a new booster — the US government.

Scientists working for the government have begun manufacturing a strain of the novel coronavirus that could be used in such studies, Reuters reported Friday morning. The trials would enroll healthy volunteers to be vaccinated and then intentionally infected with a weakened coronavirus.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

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Eye­ing in-vi­vo edit­ing, Mam­moth li­cens­es Jen­nifer Doud­na’s new CRISPR en­zyme

Last month, Jennifer Doudna revealed in Science a new, “hyper-compact” CRISPR enzyme that was half the size of traditional CRISPR enzymes and could, she suspected, offer a new, more versatile tool for gene editing.

Now, the University of California-Berkeley has licensed that enzyme, known as Casφ, exclusively to a biotech startup she and two former students set up three years ago: Mammoth Biosciences. It’s the second new CRISPR protein Mammoth has licensed from Doudna’s lab, after they licensed Cas14 in 2019.

Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).