Sanofi and Al­ny­lam plunge in­to PhI­II with a ri­val to Roche’s top he­mo­phil­ia drug

In­ves­ti­ga­tors won’t pull back the cov­ers from the lat­est mid-stage da­ta on fi­tusir­an for a few days yet, but Al­ny­lam $AL­NY and its Big Phar­ma part­ners at Sanofi Gen­zyme $SNY have fired the starter pis­tol on an am­bi­tious Phase III pro­gram for the an­tithrom­bin he­mo­phil­ia drug that will look to field a ri­val to Roche’s close­ly watched emi­cizum­ab (ACE910).

The com­pa­nies out­lined their plans for the three stud­ies in the piv­otal ef­fort, which will col­lec­tive­ly re­quire 250 pa­tients with top-line da­ta due in mid- to late-2019.

Akin Ak­inc

“I think we’re go­ing to hope to file by the end of 2019, that would be the goal,” Al­ny­lam’s Akin Ak­inc, the GM of the fi­tusir­an pro­gram, tells me. “We’re now in a time where there are lots of new ap­proach­es mov­ing for­ward.”

That time­line leaves the two part­ners well be­hind Roche, which has been field­ing im­pres­sive da­ta on emi­cizum­ab, a drug that has demon­strat­ed some strong po­ten­tial in slash­ing the an­nu­al­ized bleed­ing rate for high-risk pa­tients who have de­vel­oped in­hibitors to stan­dard ther­a­py. But Roche has al­so had to con­tend with throm­boem­bol­ic events — blood clots — in its Phase III, which have left lin­ger­ing ques­tions about the drug’s safe­ty pro­file.

“These re­sults are at least as good as what’s been pre­sent­ed for emi­cizum­ab,” Al­ny­lam CEO John Maraganore told Bloomberg late last year.

Up to now, Al­ny­lam’s up­dates on its RNAi ap­proach have high­light­ed some cas­es of el­e­vat­ed liv­er en­zymes, which in­ves­ti­ga­tors say may be the re­sult of he­pati­tis C. In any case, they say they have been able to con­tin­ue treat­ment, with great ef­fect. In the lat­est up­date on the mid-stage pro­gram last De­cem­ber, re­searchers not­ed that the an­nu­al­ized bleed rate for pa­tients was ze­ro.

Ac­cord­ing to Ak­inc, Sanofi and Al­ny­lam will be split­ting the cost of the Phase III as part of their part­ner­ship agree­ment. Sanofi agreed to buy a mi­nor­i­ty stake in the biotech for $700 mil­lion when they mapped out their part­ner­ship. And Sanofi is com­mit­ted to pro­vid­ing $75 mil­lion in de­vel­op­ment and reg­u­la­to­ry mile­stones fol­low­ing a $25 mil­lion up­front due when Phase III starts.

Fi­tusir­an’s move in­to the clin­ic comes as Al­ny­lam con­tin­ues to shift fo­cus to­ward its late-stage pipeline, a cru­cial time in which the biotech will be ex­pect­ed to meet some high ex­pec­ta­tions on per­for­mance, par­tic­u­lar­ly af­ter Al­ny­lam was forced to scrap re­vusir­an last fall af­ter safe­ty prob­lems scut­tled the work.

Fifty he­mo­phil­ia A and B pa­tients with in­hibitors to cur­rent stan­dard of care re­ceiv­ing pri­or on-de­mand ther­a­py will be en­rolled for one study. An­oth­er 100 pa­tients with­out in­hibitors will be in a sep­a­rate study. And the third is re­cruit­ing 100 pa­tients with he­mo­phil­ia A or B with or with­out in­hibitors re­ceiv­ing pro­phy­lax­is ther­a­py as pri­or stan­dard of care. In this study, pa­tients will re­ceive stan­dard of care pro­phy­lax­is for six months and then tran­si­tion to fi­tusir­an treat­ment for sev­en months. The an­nu­al­ized bleed­ing rate – the clas­sic pri­ma­ry end­point for these stud­ies — will be prospec­tive­ly mea­sured in both pe­ri­ods.

The move by Al­ny­lam and Sanofi comes one day af­ter Shire filed an IND for their he­mo­phil­ia gene ther­a­py pro­gram. Gene ther­a­py could one day pro­vide a once-and-done ap­proach to fix­ing he­mo­phil­ia and that prospect has at­tract­ed sev­er­al play­ers in­to the field.

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.