FDA OKs CSL’s new HAE drug, al­ready em­broiled in a block­buster fight with Shire

CSL Behring’s Bern, Switzer­land man­u­fac­tur­ing cam­pus


CSL Behring has picked up an FDA OK to start mar­ket­ing its new drug for rare cas­es of hered­i­tary an­gioede­ma, which is al­ready em­broiled in a court fight trig­gered by its HAE ri­vals at Shire. And in do­ing so, the FDA has now crossed the line for all its new drug ap­provals of 2016, still just shy of the halfway mark to the year.

The agency’s ap­proval for the C1 es­terase in­hibitor Hae­gar­da came through Thurs­day evening, two months af­ter Shire fired a pre­emp­tive le­gal shot in US Dis­trict Court in Delaware claim­ing that CSL’s drug vi­o­lates its new patent for its own C1 drug Cin­ryze. Shire — which saw its shares $SH­PG drop 1.3% Fri­day morn­ing — al­so mar­kets Fi­razyr and Kalbitor for HAE, a fran­chise that CSL hopes to carve up with a drug that can be self-ad­min­is­tered.

It comes with a clear en­dorse­ment from the FDA.

“The ap­proval of Hae­gar­da pro­vides a new treat­ment op­tion for ado­les­cents and adults with hered­i­tary an­gioede­ma,” said Pe­ter Marks, di­rec­tor of FDA’s Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search. “The sub­cu­ta­neous for­mu­la­tion al­lows pa­tients to ad­min­is­ter the prod­uct at home to help pre­vent at­tacks.”

The drug was green light­ed by reg­u­la­tors af­ter in­ves­ti­ga­tors put it through a piv­otal tri­al with 70 pa­tients, demon­strat­ing its su­pe­ri­or­i­ty over a place­bo in pre­vent­ing at­tacks. It will now go in­to a small mar­ket with some 6,000 to 10,000 pa­tients in the US.

Flem­ming Orn­skov

CSL is an ex­pe­ri­enced play­er in the rare dis­ease field, which Shire CEO Flem­ming Orn­skov has col­ored in as its own pri­ma­ry fo­cus. CSL mar­kets Idelvion with a $500,000 whole­sale price. Idelvion — one of the top 10 most ex­pen­sive drugs on the plan­et — is a long-act­ing he­mo­phil­ia B drug that is the first in its class to in­clude the blood pro­tein al­bu­min.

Shire is fight­ing for time as it hus­tles along an FDA ap­pli­ca­tion for its new HAE drug lanadelum­ab af­ter bag­ging stel­lar Phase III da­ta just last month. Shire’s drug — ob­tained in a $6.5 bil­lion deal to ac­quire Dyax — has al­so been des­ig­nat­ed a break­through drug. Some an­a­lysts be­lieve it could be worth $2 bil­lion in peak sales, un­less CSL man­ages to make the most of its first mover ad­van­tage.

CSL’s drug re­duced HAE at­tacks by 89% and 95% while Shire’s new drug came in right along­side with an 87% re­duc­tion. These are not head-to-head re­sults, though, re­flect­ing dif­fer­ent ways to mea­sure ef­fi­ca­cy. Those fine points can make a big dif­fer­ence.

Law360 not­ed that Shire filed a law­suit against CSL the same day it ob­tained the patent for Cin­ryze.

These patent fights have be­come the or­der of the day in bio­phar­ma. Am­gen has been par­tic­u­lar­ly ag­gres­sive in the courts, push­ing an ad­van­tage it has over Re­gen­eron and Sanofi in a scuf­fle over its PC­SK9 drug patents. And Juno just had its case against Kite thrown out af­ter the judge de­cid­ed that the court didn’t have ju­ris­dic­tion af­ter de­ter­min­ing there was no way to guar­an­tee that Kite’s CAR-T would soon be ap­proved, even though it faces a loom­ing PDU­FA date.

Now at the halfway mark in the year, the FDA’s ac­tion marks the 23rd new drug ap­proved so far in 2017, 1 more than for all of 2016.

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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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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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).

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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