A 'high-risk, high-re­ward' ef­fort on NGF pain med fas­inum­ab by Re­gen­eron, Te­va clears a re­vised PhI­II -- se­ri­ous ques­tions linger

Just a few months af­ter Re­gen­eron $REGN and its part­ners at Te­va $TE­VA were forced to drop the two high-dose arms of their Phase III study of the NGF pain med fas­inum­ab, re­searchers say the re­main­ing two low-dose arms cleared a late-stage hur­dle, open­ing the way to more Phase III tri­als as they hunt an elu­sive ap­proval for a brand new class of pain ther­a­pies.

This one isn’t easy.

Just a few months ago the top team at Re­gen­eron un­easi­ly ac­knowl­edged in a Q1 call with an­a­lysts that the in­de­pen­dent mon­i­tor­ing board had told them to shelve the two high­est dos­es in the study, cit­ing con­cerns about the risk/ben­e­fit pro­file. Once a dar­ling in Big Phar­ma cir­cles, NGF drugs were tied to se­vere ad­verse events that forced a lull in tri­al work from 2012 to 2015, un­til re­searchers could per­suade reg­u­la­tors that they could test it with­out threat­en­ing pa­tients. And Re­gen­eron and Te­va have al­ready en­dured a clin­i­cal hold for their pro­gram, which came right af­ter Te­va paid $250 mil­lion to part­ner on the ther­a­py.

George Yan­copou­los

The re­cent set­back on fas­inum­ab stirred some se­ri­ous con­cerns that those old prob­lems had once again be­come an is­sue — while a ri­val late-stage pro­gram at Pfiz­er $PFE and Eli Lil­ly $LLY for tanezum­ab has al­so stirred lin­ger­ing con­cerns. Just a month ago the part­ners said that their drug had al­so cleared a Phase III, but not­ed that the rate of “rapid­ly pro­gres­sive os­teoarthri­tis was ob­served in tanezum­ab-treat­ed pa­tients at a fre­quen­cy of less than 1.5%, and was not ob­served in the place­bo arm.”

“(T)his is a high-risk, high-re­ward pro­gram as we’ve de­scribed in the past,” R&D chief George Yan­copou­los told an­a­lysts in May. “It’s pret­ty well-demon­strat­ed that the mol­e­cule has ac­tiv­i­ty, but it al­so has cer­tain side ef­fects. It’s not os­teonecro­sis, it’s more de­fined as rapid pro­gres­sion of the os­teoarthri­tis in some pa­tients. And this is some­thing that ob­vi­ous­ly has been seen with this class and with our mol­e­cule be­fore. And so what the in­de­pen­dent da­ta mon­i­tor­ing com­mit­tee did was they ob­vi­ous­ly took an analy­sis to look at the ben­e­fit and the risk that is the ther­a­peu­tic ben­e­fit com­pared to their analy­sis of the risk com­ing from these rapid­ly pro­gres­sive os­teoarthri­tis events and they de­cid­ed that we should ter­mi­nate the up­per two dos­es and con­tin­ue with the two low­er dos­es.”

Those two low­er dos­es came through, though. In their re­lease Thurs­day the part­ners not­ed high­ly sig­nif­i­cant p val­ues for the 1 mg dose every 4 and 8 weeks for both pain and phys­i­cal func­tion. They added that the drug al­so hit goals for “key” sec­ondary end­points. Us­ing ra­di­ograph­ic mon­i­tor­ing of their joints, re­searchers pegged the place­bo-ad­just­ed rate of ad­ju­di­cat­ed arthropathies at “ap­prox­i­mate­ly 2%.”

The ma­jor­i­ty of pa­tients suf­fered from os­teoarthri­tis of the knee.

That safe­ty is­sue, says Ever­core ISI an­a­lyst Uber Raf­fat, pret­ty much elim­i­nat­ed any per­ceived val­ue in this drug. And Raf­fat re­mains on high alert re­gard­ing the safe­ty pro­file.

He not­ed Thurs­day morn­ing:

As it stands now, the place­bo-ad­just­ed in­crease in RPOA is 2%  (again, this is the IN­CREASE – we don’t know the ab­solute rates in this tri­al). We did NOT get a clear state­ment in PR for whether there has been a Type 2 RPOA (rapid pro­gres­sive OA type).

If these NGF drugs can get through Phase III in­tact, some an­a­lysts still be­lieve that there’s a big mar­ket wait­ing for a nono­pi­oid pain med. But there’s still a long way to go in Phase III, with this cur­rent read­out cen­tered on a pre­lim­i­nary sub-study. They’re re­cruit­ing pa­tients for three more Phase III stud­ies while this tri­al con­tin­ues on, with a 52-week mark ahead and a fur­ther 72-week safe­ty as­sess­ment.

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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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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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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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.