No­var­tis is keep­ing JULI­ET in hid­ing, but the FDA takes a peek and hands over a ‘break­through’ ti­tle

Vas­ant Narasimhan

There’s been con­sid­er­able spec­u­la­tion of late about the da­ta No­var­tis has col­lect­ed from its JULI­ET study in­volv­ing its close­ly-watched CAR-T drug. The phar­ma gi­ant isn’t the best at com­mu­ni­cat­ing with the out­side world and won’t say what it has seen so far or when ex­act­ly it will be put on dis­play. But they have qui­et­ly giv­en the FDA a peek, and reg­u­la­tors seem im­pressed.

In fact, the FDA has now hand­ed over their break­through ther­a­py des­ig­na­tion for CTL019 as a new ther­a­py for dif­fuse large B-cell lym­phoma.

What’s the big deal?

BTDs are in­creas­ing­ly com­mon, but this one is a bit dif­fer­ent than your run-of-the-mill open door promise at the agency. While No­var­tis and Kite have been run­ning neck and neck to get the first pi­o­neer­ing ap­proval for a CAR-T, No­var­tis is tak­ing its first shot at chil­dren with acute lym­phoblas­tic leukemia while Kite shoots for DL­B­CL. Kite has put its DL­B­CL da­ta up for the world to see, at 3 and 6 months, and now CAR-T ob­servers want to see how they match up in the com­ing ri­val­ry.

The FDA’s BTD in­di­cates that No­var­tis has a good shot at it, but a tac­it en­dorse­ment like this is no guar­an­tee that CTL019 is any bet­ter than KTE-C19, al­so known as axi-cel. Af­ter all, the FDA al­ready hand­ed out break­through sta­tus to Kite’s drug for DL­B­CL, trans­formed fol­lic­u­lar lym­phoma and pri­ma­ry me­di­asti­nal b-cell lym­phoma.

We’ll have to wait for an un­spec­i­fied sci­en­tif­ic con­fer­ence to find out what No­var­tis has been keep­ing se­cret.

In the mean­time Juno has been re­or­ga­niz­ing its CAR-T R&D group, a sto­ry End­points News broke on Sun­day evening, with the hire of Sunil Agar­w­al as the new head of re­search. Juno had to shelve its lead pro­gram af­ter it killed 5 pa­tients in clin­i­cal tri­als. The biotech has fall­en more than a year be­hind the two lead­ers in the field.

Forbes‘ Matt Her­p­er (left) with Sean Park­er and Dr. Carl June (right) – screen­shot

“We are en­cour­aged by the FDA’s recog­ni­tion in the po­ten­tial of CTL019 for this in­di­ca­tion, which fol­lows our promis­ing stud­ies of this ther­a­py for ALL and the FDA fil­ing by No­var­tis in pe­di­atric and young adult ALL that re­ceived pri­or­i­ty re­view,” said Penn’s Carl June, di­rec­tor of the Cen­ter for Cel­lu­lar Im­munother­a­pies at the uni­ver­si­ty’s Perel­man School of Med­i­cine.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

Vas Narasimhan's 'Wild Card' drugs: No­var­tis CEO high­lights po­ten­tial jack­pots, as well as late-stage stars, in R&D pre­sen­ta­tion

Novartis is always one of the industry’s biggest R&D spenders. As they often do toward the end of each year, company execs are highlighting the drugs they expect will most likely be winners in 2021.

And they’re also dreaming about some potential big-time lottery tickets.

As part of its annual investor presentation Tuesday, where the company allows investors and analysts to virtually schmooze with the bigwigs, Novartis CEO Vas Narasimhan will outline what he thinks are the pharma’s “Wild Cards.” The slate of five experimental drugs are those that Novartis hopes can be high-risk, high-reward entrants into the market over the next half-decade or so, and cover a wide range of indications.

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UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Gen­mab ax­es an ADC de­vel­op­ment pro­gram af­ter the da­ta fail to im­press

Genmab $GMAB has opted to ax one of its antibody-drug conjugates after watching it flop in the clinic.

The Danish biotech reported Tuesday that it decided to kill their program for enapotamab vedotin after the data gathered from expansion cohorts failed to measure up. According to the company:

While enapotamab vedotin has shown some evidence of clinical activity, this was not optimized by different dose schedules and/or predictive biomarkers. Accordingly, the data from the expansion cohorts did not meet Genmab’s stringent criteria for proof-of-concept.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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Pur­due Phar­ma pleads guilty in fed­er­al Oxy­Con­tin probe, for­mal­ly rec­og­niz­ing it played a part in the opi­oid cri­sis

Purdue Pharma, the producer of the prescription painkiller OxyContin, admitted Tuesday that, yes, it did contribute to America’s opioid epidemic.

The drugmaker formally pleaded guilty to three criminal charges, the AP reported, including getting in the way of the DEA’s efforts to combat the crisis, failing to prevent the painkillers from ending up on the black market and encouraging doctors to write more painkiller prescriptions through two methods: paying them in a speakers program and directing a medical records company to send them certain patient information. Purdue’s plea deal calls for $8.3 billion in criminal fines and penalties, but the company is only liable for a fraction of that total — $225 million.

John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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Am­gen sev­ers 14-year Cy­to­ki­net­ics part­ner­ship, bail­ing on ome­cam­tiv af­ter mixed PhI­II re­sults

Amgen is shrugging off a 14-year development alliance and the tens of millions of dollars spent to develop a new heart drug at Cytokinetics after a Phase III trial turned up weak data — leaving Cytokinetics to soldier on alone.

Omecamtiv mecarbil technically worked, meeting the primary composite endpoint in the Phase III GALACTIC-HF study. But it missed a key secondary endpoint, which analysts had been following as a key marker for success — reduction of cardiovascular (CV) death. While Cytokinetics celebrated the results, its stock tanked 43% upon the news, and analysts warned of an uncertain path ahead. Now, Amgen wants out.