Pfiz­er, Mer­ck KGaA score a land­mark FDA OK for check­point con­tender avelum­ab

Lu­ciano Ros­set­ti, Mer­ck Serono

Mer­ck KGaA and Pfiz­er have done it.

The bio­phar­ma part­ners came away with their first FDA ap­proval of the check­point in­hibitor avelum­ab, mak­ing it the fourth drug in this cat­e­go­ry to make it to the mar­ket. They beat As­traZeneca, which will now an­gle for its first ap­proval of dur­val­um­ab as the 5th check­point.

The FDA gave out its ap­proval for Merkel cell car­ci­no­ma un­der an ac­cel­er­at­ed ap­proval process. The ther­a­py, the first for a rare form of skin can­cer, has al­so been giv­en break­through drug sta­tus. The drug is cur­rent­ly in 30 clin­i­cal pro­grams as the two play­ers look to wedge their way in­to a multi­bil­lion-dol­lar mar­ket for drugs that are gain­ing wide use in treat­ing can­cer.

The ap­proval marks an­oth­er big ad­vance for Pfiz­er, which paid $850 mil­lion up­front to part­ner with Ger­many’s Mer­ck — a record sum. The ap­proval of this an­ti-PD-L1 IgG1 mon­o­clon­al an­ti­body al­so marks a ma­jor win for Mer­ck KGaA, which has suf­fered through more than a decade with­out a block­buster OK. Mer­ck KGaA al­so came away with a pack­age of reg­u­la­to­ry and com­mer­cial mile­stones on avelum­ab worth up to $2 bil­lion when it tied up with Pfiz­er in 2014.

The ther­a­py will be sold as Baven­cio. A spokesper­son for Pfiz­er said the drug will cost $13,000 a month whole­sale — ahead of pay­er dis­counts.

The new ap­proval marks just how quick­ly the FDA can move when it wants to. The OK is based on a sin­gle arm study in­clud­ing 88 pa­tients. Of the 88 pa­tients who re­ceived Baven­cio in the tri­al, says the FDA, 33% ex­pe­ri­enced com­plete or par­tial shrink­age of their tu­mors. The re­sponse last­ed for more than six months in 86% of re­spond­ing pa­tients and more than 12 months in 45% of re­spond­ing pa­tients.

Not on­ly was avelum­ab a big suc­cess as a check­point, the de­vel­op­ment pro­gram from start to first ap­proval was ex­e­cut­ed swift­ly.

“Three-and-a-half years af­ter the first-in-hu­man dose es­ca­la­tion stud­ies, we are get­ting our first ap­proval,” says Lu­ciano Ros­set­ti, the head of R&D at Mer­ck Serono. And don’t ex­pect any­one to start sit­ting on lau­rels. Check­point in­hi­bi­tion is a big field, and the Pfiz­er/Mer­ck KGaA team plan to make their mark as the pi­o­neers divvy up lead­er­ship roles in a wide ar­ray of can­cers.

As­traZeneca re­jigged its de­vel­op­ment cam­paign for the next check­point in the pipeline, re­ly­ing heav­i­ly on its work in com­bin­ing it with the CT­LA-4 check­point treme­li­mum­ab to try and leapfrog the fast mov­ing trio in the lead: Mer­ck, Bris­tol-My­ers Squibb and Roche/Genen­tech. They’ll be star­ing down some of the biggest ri­vals in the in­dus­try in a make-or-beak ef­fort to dis­tin­guish its R&D op­er­a­tions, which has had sev­er­al suc­cess­es in can­cer but con­tin­ues to be plagued by set­backs in the clin­ic and at the FDA.

You can add Mer­ck KGaA to the list now.

“You need to have a strong PD1/ PD-L1 to an­chor com­bi­na­tions for oth­er as­sets,” Ros­set­ti tells me. And now they have one ap­proved and en­ter­ing the mar­ket.

The FDA ac­cept­ed their ap­pli­ca­tion for urothe­lial car­ci­no­ma just a few weeks ago, and Ros­set­ti says they’re push­ing hard on ovar­i­an, gas­tric and lung can­cer work that could pave the way for near-term ap­provals.

“To be prac­ti­cal,” he adds, “the biggest op­por­tu­ni­ty is in non-small cell lung can­cer.” One of those big op­por­tu­ni­ties is demon­strat­ing an ef­fect in low PD-L1 ex­pressers with re­searchers al­so ex­plor­ing high in­ten­si­ty ther­a­py for high ex­pressers.

Ros­set­ti, though, is care­ful to of­fer no big promis­es on what the da­ta will show. He’s acute­ly aware that when they mod­i­fied the lung can­cer study, the com­pa­ny was gam­bling on a dis­ease that proved a dis­as­ter for Bris­tol-My­ers Squibb and a chance for Mer­ck to leap ahead of the pack.

The first thing that Ros­set­ti did when he was named R&D chief in 2014 was to fo­cus the pipeline, get­ting rid of 13 pro­grams so they could con­cen­trate on a few se­lect ef­forts like avelum­ab. Now he says the com­pa­ny is in­ter­est­ed in do­ing a few deals to help beef up its fo­cus on com­bi­na­tions and more. But they’ll be very strate­gic about launch­ing new tri­als.

“While skin can­cer is one of the most com­mon can­cers, pa­tients with a rare form called Merkel cell can­cer have not had an ap­proved treat­ment op­tion un­til now,” said Richard Paz­dur, MD, act­ing di­rec­tor of the Of­fice of Hema­tol­ogy and On­col­o­gy Prod­ucts in the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search and di­rec­tor of the FDA’s On­col­o­gy Cen­ter of Ex­cel­lence. “The sci­en­tif­ic com­mu­ni­ty con­tin­ues to make ad­vances tar­get­ing the body’s im­mune sys­tem mech­a­nisms for the treat­ment of var­i­ous types of can­cer. These ad­vance­ments are lead­ing to new ther­a­pies—even in rare forms of can­cer where treat­ment op­tions are lim­it­ed or non-ex­is­tent.”

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.

UP­DAT­ED: With loom­ing ‘apoc­a­lypse of drug re­sis­tance,’ Mer­ck’s com­bi­na­tion an­tibi­ot­ic scores FDA ap­proval on two fronts

Merck — one of the last large biopharmaceuticals companies in the beleaguered field of antibiotic drug development — on Wednesday said the FDA had sanctioned the approval of its combination antibacterial for the treatment of complicated urinary tract and intra-abdominal infections.

To curb the rise of drug-resistant bacteria and maintain the efficacy of the therapy, Recarbrio (and other antibacterials) — the drug must be used to treat or prevent infections that are proven or strongly suspected to be caused by susceptible gram-negative bacteria, Merck $MRK said.

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