FDA slaps down No­var­tis' block­buster pitch for canakinum­ab — so what went wrong?

In a ma­jor set­back, No­var­tis used its quar­ter­ly earn­ings re­port to flag an FDA slap­down on their ap­pli­ca­tion to mar­ket canakinum­ab for car­dio risk re­duc­tion.

Vas Narasimhan

Tout­ed as a block­buster in­di­ca­tion, the phar­ma gi­ant steered this drug through a big late-stage de­vel­op­ment pro­gram, even pulling out da­ta on a large sub­group of pa­tients that could ben­e­fit from the drug. The then de­vel­op­ment chief Vas Narasimhan was pro­mot­ed to CEO in part on the suc­cess he had with this drug — on­ly to get hit with a com­plete re­sponse let­ter.

A spokesper­son for the com­pa­ny of­fered an ex­pla­na­tion for what went wrong at the FDA:

Based on the cor­re­spon­dence from the FDA, the CAN­TOS da­ta would not sup­port la­bel­ing for the use of canakinum­ab as a tar­get­ed ther­a­py for those pa­tients with car­dio­vas­cu­lar dis­ease who achieved a re­duc­tion of hsCRP be­low the 2 mg/L tar­get. At this time, we are eval­u­at­ing the feed­back pro­vid­ed and plans fur­ther dis­cus­sions with the FDA.

That said, we are con­tin­u­ing to study canakinum­ab to treat non-small cell lung can­cer in sev­er­al Phase 3 stud­ies, with the first tri­al com­ple­tion ex­pect­ed in 2022.

The re­jec­tion is a par­tic­u­lar­ly bit­ter pill for No­var­tis to swal­low now, af­ter tout­ing the drug fol­low­ing the col­lapse of its ef­fort to get their oth­er car­dio drug sere­lax­in ap­proved.

Tim An­der­son

The an­ti-in­flam­ma­to­ry drug, sold as Ilaris for a niche mar­ket, earned $380 mil­lion last year. And con­sid­er­ing da­ta on an added risk of in­fec­tion as well as some ex­perts fret­ting about the some­what fuzzy clar­i­ty of the ben­e­fit ini­tial­ly on dis­play, Tim An­der­son pegged the peak sales at a con­sen­sus of $800 mil­lion, while not­ing that patent pro­tec­tion should run out in on­ly 7 years.

Oth­er an­a­lysts were much more up­beat, with Bank Von­to­bel of­fer­ing good odds of $2 bil­lion in peak sales — mon­ey No­var­tis needs to spur rev­enue growth.

Jef­feries’ Michael Yee not­ed:

Al­though over­all the drug showed a 15% CV ben­e­fit on the pri­ma­ry end­point, based on pri­or com­men­tary, it is like­ly NVS was seek­ing to in­clude the pre-spec­i­fied sub-group of pts who are able to get to >1.7-2.0mg of hsCRP re­duc­tion which led to much larg­er 25-27% CV ben­e­fits. NVS may feel this is the pop­u­la­tion that has sig­nif­i­cant ben­e­fit and would dri­ve suf­fi­cient com­mer­cial op­por­tu­ni­ty rather than the more mod­est 15% CV ben­e­fit in the over­all en­rolled pop­u­la­tion. While doc com­men­tary has sug­gest­ed that NVS would tar­get per­haps a seg­ment (ie 30-40%) of over­all high CV risk mar­ket (pts who had pri­or MI too) and not re­al­ly com­pete much for the pts tak­ing AMGN PC­SK9, it would be keen to watch this as a 25-27% ben­e­fit would be com­pelling for pts in the mar­ket…As a re­minder, ES­PR has al­so shown a sig­nif­i­cant 40% re­duc­tion in hsCRP as well in its stud­ies which may help add con­fi­dence to over­all treat­ment ef­fect in their CVOT study (we look to up­com­ing im­por­tant Phase III safe­ty da­ta com­ing H2 Oc­to­ber…if clean and no ma­jor im­bal­ances, this could bode well for ES­PR).

The treat­ment is de­signed to in­hib­it IL-1ß, a key cy­tokine, for a pro­longed pe­ri­od, tamp­ing down on in­flam­ma­tion to low­er risk to pa­tients.

Re­searchers al­so pulled out pos­i­tive sub­group da­ta on gout from the CAN­TOS study, which tracked more than 10,000 pa­tients for more than 6 years — one of the biggest stud­ies in the phar­ma gi­ant’s his­to­ry.

There is a side ben­e­fit to this drug that No­var­tis has been puz­zling out. The drug pro­duced a re­duc­tion in lung can­cer mor­tal­i­ty of 77%, un­der­scor­ing their the­o­ry that in­hibit­ing IL-1ß could pro­duce promis­ing da­ta in on­col­o­gy.

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.