De­fy­ing the odds, Pox­el plots a PhI­II course for its Type 2 di­a­betes med and shares surge on new da­ta

Thomas Kuhn, Pox­el

Pox­el is try­ing to mas­ter one of the tough­est acts in biotech: De­vel­op a new drug for Type 2 di­a­betes and win a reg­u­la­to­ry ap­proval — on its own.

It’s not easy, in the way that nav­i­gat­ing a mine field isn’t easy. But they are mov­ing ahead and say they have a very man­age­able game plan for get­ting through Phase III in Japan while look­ing for part­ners that can do much of the heavy lift­ing in Eu­rope and the US.

About 10 months ago, France’s Pox­el hit the brakes on a planned move to Nas­daq, un­able to gen­er­ate much en­thu­si­asm in a tough mar­ket. In­stead, ex­ecs raised some ad­di­tion­al cash from its pub­lic list­ing in Eu­rope to fund a Phase IIb tri­al of its lead ther­a­py for Type 2 di­a­betes that would hope­ful­ly pave the way to a launch in Japan.

To­day, the com­pa­ny says it scored the pri­ma­ry and at least one sec­ondary end­point for imeglim­in, track­ing he­mo­glo­bin A1c re­duc­tions of 0.52%, 0.94% and 1.00% for the 500 mg, 1000 mg and 1500 mg dose twice-dai­ly. Sev­en per­cent or less is the key num­ber for glycemic con­trol. The drug al­so trig­gered a sta­tis­ti­cal­ly sig­nif­i­cant drop in fast­ing plas­ma glu­cose. And now the com­pa­ny plans to set the stage for a piv­otal pro­gram in Japan that can launch lat­er this year — pro­vid­ed Japan­ese reg­u­la­tors sign off.

That was wel­come news to its in­vestors, who bid up shares {$POX­EL: PA} by more than 35%. But the biotech has a long way to go be­fore it can de­clare vic­to­ry.

Late-stage test­ing for a di­a­betes drug, which would be steer­ing in­to an in­tense­ly com­pet­i­tive mar­ket among a hand­ful of gi­ants in the field, is nei­ther easy nor cheap. Big Phase III bud­gets need­ed to suf­fi­cient­ly test a mass mar­ket drug have scut­tled ear­li­er con­tenders like Phe­nomix (RIP: 2010). MannKind strug­gled its way through the clin­ic to end up with an in­haled in­sulin that di­a­bet­ics — and af­ter Sanofi washed its hands of a deal, the rest of the play­ers — have shunned. And none of that has made di­a­betes par­tic­u­lar­ly pop­u­lar with VCs or gen­er­al­ist in­vestors.

But Pox­el ex­ecs say they have found a path that can work.

“For a biotech com­pa­ny like this ac­cess­ing this mar­ket (Japan) is much eas­i­er,” CEO Thomas Kuhn tells me. While most glob­al bio­phar­ma com­pa­nies pre­fer to hand off their drugs to Japan­ese part­ners bet­ter equipped to work with the coun­try’s reg­u­la­tors, Pox­el cre­at­ed their own lo­cal team in the Asian coun­try, and Kuhn says the coun­try low­ered reg­u­la­to­ry bar­ri­ers to out­side com­pa­nies a cou­ple of years ago, mak­ing it pos­si­ble for Pox­el to pi­o­neer this on their own.

But there are even more im­por­tant as­pects that make Japan one of the few places on the plan­et Pox­el could ex­pect to do this on their own. One is that reg­u­la­tors are open to new drugs in the field, Kuhn ar­gues, sen­si­tive to the is­sues Japan­ese pa­tients have with met­formin. Pox­el could get an ap­proval on a slate of three stud­ies in­volv­ing about 1,000 di­a­betes pa­tients in Japan, says the CEO, while it would ul­ti­mate­ly take some 7,000 pa­tients in the US. Japan­ese pa­tients are al­so much lean­er than the av­er­age US pa­tient, avoid­ing the ne­ces­si­ty of a car­dio out­comes study in ad­vance of an ap­proval. And fi­nal­ly, he notes, as a Mer­ck Serono spin­out, they have the kind of ex­per­tise nec­es­sary to make it all hap­pen.

Pox­el still says it would need a Japan­ese part­ner to com­mer­cial­ize the drug, but that’s some­thing that Kuhn feels is doable, giv­en the num­ber of glob­al and lo­cal com­pa­nies that work in the mar­ket — which he says is the sec­ond largest in the world.

The biotech has al­so com­plet­ed Phase II stud­ies in the EU and the US for the drug, which tar­gets mi­to­chon­dr­i­al dys­func­tion. So the com­pa­ny can con­tin­ue to par­lay with Japan­ese reg­u­la­tors as they work with con­tacts in the US and Eu­rope for those big­ger mar­kets. And a Nas­daq IPO re­mains an op­tion, when the time is right.

That may not be easy. But the way Kuhn says it, it doesn’t sound im­pos­si­ble ei­ther.

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.