Puma wins a big round in fight for ner­a­tinib OK as ex­perts of­fer a cru­cial thumbs up

Puma Biotech­nol­o­gy $PBYI won a cru­cial vote in fa­vor of its ex­per­i­men­tal breast can­cer drug ner­a­tinib, with a ma­jor­i­ty of out­side ex­perts of­fer­ing the FDA a thumbs-up vote in fa­vor of ap­proval as an ex­tend­ed ad­ju­vant treat­ment.

On the key ques­tion of whether the risk/ben­e­fit pro­file of ner­a­tinib war­rant­ed an ap­proval, the pan­el vot­ed twelve to four in fa­vor of the drug. But the sup­port came with plen­ty of caveats about the nar­row ben­e­fit the da­ta demon­strat­ed and ques­tions about the drug’s of­ten harsh im­pact on pa­tients. Sev­er­al pan­el mem­bers called on the FDA to nar­row the ap­proval to a small­er set of pa­tients most like­ly to ben­e­fit.

“There’s no ex­cla­ma­tion point af­ter my ‘yes’,” said An­drew Sei­d­man, an on­col­o­gist at Memo­r­i­al Sloan Ket­ter­ing. “It’s just a yes.” He added that he was hap­py that the drug looks durable and the sta­tis­ti­cal analy­ses ap­plied to the da­ta giv­en the sig­nif­i­cant changes to the study de­sign. “I do think that physi­cians will se­lect pa­tients very se­lec­tive­ly for us­ing this,” he not­ed, echo­ing a con­cern that the ap­proval the com­pa­ny was seek­ing was too broad giv­en the da­ta avail­able.

If it makes it to the mar­ket, those com­ments may come back to haunt Puma, to some ex­tent. But to­day, Puma ex­ecs had plen­ty to cel­e­brate. Its shares im­me­di­ate­ly shot up more than 25% af­ter trad­ing re­sumed in the ear­ly af­ter­noon, adding to the big spike that oc­curred when the FDA’s writ­ten re­view hit on Mon­day.

Puma has been at the cen­ter of a rov­ing street fight be­tween in­vestors go­ing long and short on the stock. But there were none of the fire­works pre­dict­ed dur­ing the re­view on Wednes­day.

The bears’ ar­gu­ment that the drug trig­gered such se­ri­ous ad­verse events that it would be spurned by the FDA ran in­to the ex­perts’ sup­port for the mar­gin­al ben­e­fit the drug demon­strat­ed, im­prov­ing dis­ease-free sur­vival in HER2 pos­i­tive pa­tients by 2.3%. About 94.2% of pa­tients in the ner­a­tinib arm were alive with­out their dis­ease pro­gress­ing at two years, com­pared with 91.9% in the con­trol arm.

One of the biggest is­sues high­light­ed in the FDA’s writ­ten re­view of the drug was that agency of­fi­cials had ad­vised Puma not to file based on the da­ta from their piv­otal tri­al. That didn’t seem to weigh heav­i­ly on the pan­el, though Puma won’t know for sure if it’s out of the woods un­til the FDA pro­vides a for­mal de­ci­sion.

Pan­el votes are not bind­ing.

There were se­ri­ous ad­verse events in the study, pri­mar­i­ly re­flect­ed in high lev­els of se­ri­ous, though not life threat­en­ing, cas­es of di­ar­rhea. The agency’s re­view, though, did not raise ma­jor red flags for Puma.

“Most tox­i­c­i­ties are non­se­ri­ous and re­versible,” said the FDA’s Aman­da Walk­er, “but the true mag­ni­tude of ben­e­fit re­mains un­cer­tain.”

The ben­e­fit was un­cer­tain be­cause of the changes Puma made to the reg­is­tra­tional study af­ter tak­ing over from Pfiz­er. In­ves­ti­ga­tors for Puma went back to re­con­sent pa­tients for a longer pe­ri­od of re­view and did that with 75% of the pa­tients, but da­ta were still miss­ing on 754 pa­tients.

But why would a physi­cian choose this drug com­pared to what’s al­ready avail­able, asked one of the pan­elists.

Puma con­sul­tant Jose Bisel­ga not­ed the “rel­a­tive risk re­duc­tion of 34% com­pared to what is avail­able cur­rent­ly.” Would I want the op­tion to use it? “To this I would an­swer ‘yes.’ I would go a long way to get this done.”

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.

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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Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

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.

Den­mark's Gen­mab hits the jack­pot with $500M+ US IPO as small­er biotechs rake in a com­bined $147M

Danish drugmaker Genmab A/S is off to the races with perhaps one of the biggest biotech public listings in decades, having reaped over $500 million on the Nasdaq, as it positions itself as a bonafide player in antibody-based cancer therapies.

The company, which has long served as J&J’s $JNJ key partner on the blockbuster multiple myeloma therapy Darzalex, has asserted it has been looking to launch its own proprietary product — one it owns at least half of — by 2025.

FDA over­rides ad­comm opin­ions a fifth of the time, study finds — but why?

For drugmakers, FDA advisory panels are often an apprehended barometer of regulators’ final decisions. While the experts’ endorsement or criticism often translate directly to final outcomes, the FDA sometimes stun observers by diverging from recommendations.

A new paper out of Milbank Quarterly put a number on that trend by analyzing 376 voting meetings and subsequent actions from 2008 through 2015, confirming the general impression that regulators tend to agree with the adcomms most of the time — with discordances in only 22% of the cases.