Fi­bro­Gen, As­traZeneca score Chi­na ap­proval for ane­mia drug ahead of piv­otal US da­ta

It may be the first time a multi­na­tion­al phar­ma­ceu­ti­cal com­pa­ny As­traZeneca $AZN could be­gin sell­ing a po­ten­tial block­buster med­i­cine in Chi­na, be­fore the Unit­ed States or Eu­rope. Fi­bro­Gen $FGEN and the British drug­mak­er have se­cured Chi­na ap­proval for their oral drug, rox­adu­s­tat, for ane­mic pa­tients with chron­ic kid­ney dis­ease who are de­pen­dent on dial­y­sis.

The drug, which stim­u­lates the pro­duc­tion of red blood cells by mim­ic­k­ing the ef­fect of high al­ti­tude in hu­mans, will be launched in the sec­ond half of next year un­der the Chi­nese brand name: ài ruì zhuó 爱瑞卓. While Fi­bro­Gen will take care of man­u­fac­tur­ing, As­traZeneca is in charge of com­mer­cial­iza­tion. Ap­proval for a larg­er pool of pa­tients — who are not de­pen­dent on dial­y­sis — is ex­pect­ed some­time in 2019, af­ter Chi­nese reg­u­la­tors com­plete an in­spec­tion of tri­al sites, the San Fran­cis­co-based com­pa­ny said on Mon­day.

The ap­proval could open up an es­ti­mat­ed $300-500 mil­lion sales op­por­tu­ni­ty, not­ed Jef­feries’ Michael Yee. “For Chi­na, FGEN has re­ceived $43 mil­lion from AZN al­ready and is en­ti­tled to up to $334 mil­lion of ad­di­tion­al pay­ments, much of which could be paid to FGEN this quar­ter,” he wrote. Over­all, Fi­bro­Gen has rough­ly $900 mil­lion in glob­al reg­u­la­to­ry mile­stones out­stand­ing, the ma­jor­i­ty of which should be re­al­ized over the next 12-18 months, not­ed Leerink’s Ge­of­frey Porges.

Over the past few years, Chi­na’s ver­sion of the FDA in­tro­duced a slate of re­forms to en­tice drug de­vel­op­ers, in­clud­ing over­turn­ing a rule that re­quired drug­mak­ers to con­duct sep­a­rate tri­als in Chi­na be­fore ap­provals were sanc­tioned. How­ev­er, Rox­adu­s­tat — which could even­tu­al­ly be used to treat mil­lions af­flict­ed with CKD in Chi­na — was giv­en pri­or­i­ty re­view by Chi­nese health reg­u­la­tors, who agreed to eval­u­ate the mar­ket­ing ap­pli­ca­tion on a rolling ba­sis, af­ter the drug per­formed well in two piv­otal stud­ies in Chi­na.

Porges es­ti­mat­ed rox­adusat will gen­er­ate sales of $9 mil­lion in Chi­na next year – but ex­pects that num­ber will climb to a hefty $1 bil­lion by 2025.

In the Unit­ed States, the drug is ex­pect­ed to be sub­mit­ted for re­view in the first half of next year, af­ter piv­otal tri­als are com­plet­ed. Top-line ef­fi­ca­cy da­ta from a US tri­al are ex­pect­ed in the com­ing weeks, fol­lowed by key MACE safe­ty da­ta in March/April 2019, Yee not­ed.

Fi­bro­Gen, which orig­i­nal­ly de­vel­oped the drug, has part­nered with As­traZeneca since 2013 to de­vel­op and sell the treat­ment in re­gions in­clud­ing the Unit­ed States, Chi­na, Aus­tralia, New Zealand and South­east Asia. Astel­las is work­ing with Fi­bro­Gen to do the same in ar­eas such as Japan, Eu­rope, the Mid­dle East and South Africa. An ap­pli­ca­tion to mar­ket rox­adu­s­tat in Japan was sub­mit­ted in Oc­to­ber.

Fi­bro­Gen and As­traZeneca are locked in a race with Ake­bia $AK­BA, whose ex­per­i­men­tal drug vadadu­s­tat has a sim­i­lar mech­a­nism of ac­tion. But piv­otal vadadu­s­tat da­ta are not ex­pect­ed un­til 2020. The dom­i­nant drugs for anaemia in the Unit­ed States are red-blood-cell boost­ing ery­thro­poiesis-stim­u­lat­ing agents (ESA) from Am­gen $AMGN.

Healthy kid­neys pro­duce a hor­mone called ery­thro­poi­etin (EPO), which prompts the bone mar­row to make red blood cells that car­ry oxy­gen through­out the body. If dis­eased or dam­aged, kid­neys are un­able to make enough EPO, re­sult­ing in few­er blood cells, and even­tu­al­ly anaemia.

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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