No­var­tis gets a speedy re­view for its CAR-T pi­o­neer as FDA lines up a mar­ket­ing de­ci­sion

Vas­ant Narasimhan, No­var­tis

No­var­tis $NVS has pulled in­to the in­side track at the FDA with its CAR-T CTL019, pick­ing up a pri­or­i­ty re­view from the agency that will land a mar­ket­ing de­ci­sion for re­lapsed and re­frac­to­ry (r/r) pe­di­atric and young adult pa­tients with B-cell acute lym­phoblas­tic leukemia.

The pi­o­neer­ing ap­pli­ca­tion comes as we wait for Kite to make its move at the agency af­ter be­ing forced to wait for 6-month da­ta on its ri­val CAR-T. Kite $KITE has promised to fin­ish its rolling ap­pli­ca­tion by the end of Q1, now just days away. And a spokesper­son for Kite tells me now that the biotech is on track to wrap the sub­mis­sion by Fri­day.

The pri­or­i­ty re­view leaves No­var­tis in shoot­ing range of the world’s first ap­proval for a CAR-T, which takes T-cells from pa­tients and then en­gi­neers them to at­tack can­cer cells. Blood can­cers proved to be the best, ear­ly tar­gets in the field, which has seen a line­up of com­pa­nies jump in on the promise of dra­mat­ic re­spons­es for some pa­tients.

No­var­tis pushed ahead in CAR-T af­ter stun­ning the in­dus­try with its de­ci­sion last sum­mer to dis­solve its cell and gene ther­a­py unit, a sto­ry we broke just af­ter launch­ing End­points News. The phar­ma gi­ant in­sist­ed that the sud­den sharp turn, which prompt­ed the unit’s chief, Us­man “Oz” Azam, to join an ex­o­dus of ex­ecs out of No­var­tis over the past year, wouldn’t side­track its pro­grams.

But it’s def­i­nite­ly in a tight race for the fin­ish line.

The progress of these ear­ly lead­ers hasn’t come eas­i­ly. Juno was once in the lead pack, un­til its fron­trun­ner was side­tracked twice by pa­tient deaths which it nev­er ful­ly ex­plained. Now that lead pro­gram has been shelved as Juno switch­es fo­cus to the next ther­a­py in the pipeline, hand­ing the lead to No­var­tis and Kite.

No­var­tis says it’s prep­ping an EMA ap­pli­ca­tion and plans ad­di­tion­al fil­ings for CTL019 in the US and EU mar­kets lat­er this year, in­clud­ing a BLA with the FDA for treat­ment of adults with r/r dif­fuse large B-cell lym­phoma (DL­B­CL) and ap­pli­ca­tions for mar­ket­ing au­tho­riza­tion with the EMA in r/r B-cell ALL and r/r DL­B­CL.

Wed­bush’s David Nieren­garten re­cent­ly spec­u­lat­ed that Kite’s da­ta up­date put it in line for a late 2017 ap­proval, shift­ing the fo­cus to pric­ing. He not­ed:

“(W)e note to jus­ti­fy the cur­rent mar­ket cap­i­tal­iza­tion, we would re­quire ei­ther a price point of $500,000 per course or up to 50% mar­ket share (or some com­bi­na­tion), both of which, while pos­si­ble, we be­lieve is less like­ly.”

But some an­a­lysts start this bid­ding at $250,000.

Who­ev­er gets on­to the mar­ket first will have a big edge in set­ting the price for these first wave drugs.

“With CTL019, No­var­tis is at the fore­front of the sci­ence and de­vel­op­ment of im­muno­cel­lu­lar ther­a­py as a po­ten­tial new in­no­v­a­tive ap­proach to treat­ing cer­tain can­cers where there are lim­it­ed op­tions,” said Vas Narasimhan, Glob­al Head of Drug De­vel­op­ment and Chief Med­ical Of­fi­cer, No­var­tis. “The pri­or­i­ty re­view and file ac­cep­tance of CTL019 by the FDA brings us one step clos­er to de­liv­er­ing this nov­el treat­ment op­tion to chil­dren and young adults with r/r B-cell ALL in the Unit­ed States.”

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.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

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.

Bahija Jallal (file photo)

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

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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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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.

News brief­ing: Gilead part­ner Gala­pa­gos sells off CRO for $37M; Polyphor bags $3.3M from CF Foun­da­tion

Close Gilead ally Galapagos is selling off one of its contract research organizations to a Polish pharma company.

Galapagos has agreed to sell 100% of the outstanding shares in the CRO Fidelta to Selvita, in a deal worth roughly $37 million expected to close in the first week of January. The acquisition is expected to nearly double Selvita’s revenues, the company says, as well as expand its drug discovery efforts.