Es­pe­ri­on’s cho­les­terol drug clears the last big safe­ty hur­dle, turn­ing the cor­ner to the FDA — now the big chal­lenge looms

Es­pe­ri­on $ES­PR looks set to make the fi­nal step in its long jour­ney to­ward a mar­ket­ing ap­pli­ca­tion at the FDA, with good odds of suc­cess next year. But the lat­est batch of pos­i­tive safe­ty and ef­fi­ca­cy da­ta won’t dis­pel the per­sis­tent ques­tions over its sales strat­e­gy.

Tim Mayleben

That’s go­ing to take some hard dol­lar num­bers.

First, the tri­al da­ta.

The drug arm saw a mod­er­ate drop in LDL of 18% with a 19% cut in high-sen­si­tiv­i­ty C-re­ac­tive pro­tein. That fits in well with what we’ve seen be­fore in a string of Es­pe­ri­on stud­ies, with the ther­a­py fit­ting neat­ly be­tween the gener­ics that dom­i­nate the mar­ket and the PC­SK9s that have had trou­ble find­ing trac­tion, spurring some deep dis­count­ing that could spell trou­ble for Es­pe­ri­on.

It was safe­ty where Es­pe­ri­on faced the steep­est chal­lenge, as ear­li­er ques­tions fo­cused on some con­tro­ver­sial mor­tal­i­ty is­sues that at least tem­porar­i­ly desta­bi­lized the stock. An­a­lysts want­ed these 52-week re­sults to get a much bet­ter idea of any risks pre­sent­ed by this drug. But as Jef­feries’ Michael Yee not­ed, Es­pe­ri­on’s be­mpe­doic acid came through with a clean bill of health.

In the pri­ma­ry safe­ty analy­sis for this study, the two con­cerns ap­pear al­le­vi­at­ed here with no ma­jor is­sues: (1) CV deaths were bal­anced at 0.8% drug vs 0.8% pbo, (2) there were no fa­tal AEs due to neo­plasms/ can­cer. Oth­er SAEs and fa­tal AEs were gen­er­al­ly bal­anced (e.g. SAEs of 20% vs 19% pbo and fa­tal AEs of 1.1% vs 0.8% pbo), and none of the fa­tal AEs were de­ter­mined to be re­lat­ed to study med­ica­tion. The fa­tal AEs would be bal­anced at 0.8% vs 0.8% pbo as well, ex­cept for two one-off cas­es ob­vi­ous­ly not even re­lat­ed to drug – so we be­lieve the fa­tal AEs are bal­anced.

The com­pa­ny is al­so mak­ing much out of a trend to­ward a ben­e­fit on ma­jor car­dio events, or MACE, but they’ll have to nail down that one with an out­comes study, which will take years to play out, with a read­out planned for 2022.

Es­pe­ri­on’s ex­ec­u­tive team plans to sub­mit their US ap­pli­ca­tion in ear­ly 2019, with an EMA pitch com­ing soon af­ter.

That all sound­ed good to in­vestors, who pushed up Es­pe­ri­on’s share price by 10% in pre-mar­ket trad­ing Mon­day.

As­sum­ing there are no nasty sur­pris­es to come in the reg­u­la­to­ry process, what can we ex­pect in a mar­ket­ing show­down be­tween lit­tle Es­pe­ri­on and the gi­ants at Re­gen­eron/Sanofi and Am­gen which have been slash­ing their prices on the PC­SK9 — break­throughs that have demon­strat­ed a mod­er­ate but sig­nif­i­cant ben­e­fit in car­dio risk re­duc­tion?

The three big play­ers have been steadi­ly ax­ing away at their orig­i­nal prices af­ter pay­ers es­sen­tial­ly blocked ac­cess to their drugs to a ma­jor pop­u­la­tion. Es­pe­ri­on CEO Tim Mayleben — who is aim­ing at a sig­nif­i­cant por­tion of the mar­ket that doesn’t get what they need from statins and don’t need what they can get from PC­SK9s — has told me re­peat­ed­ly that they still ex­pect to price in un­der the PC­SK9 over­lords, but his abil­i­ty to main­tain a price ad­van­tage will be at the dis­cre­tion of the big 3.

Amarin’s re­cent out­comes da­ta, which wowed every­one on its (sep­a­rate) in­dus­tri­al-strength fish oil ap­proach with triglyc­erides, al­so un­der­scores just how change­able the mar­ket has been.

You can al­so count Ethan Weiss, a car­di­ol­o­gist at UC San Fran­cis­co, as one of the skep­ti­cal ob­servers. In a Tweet out af­ter the da­ta ar­rived, he not­ed:

I see the most like­ly case as be­mpe­doic acid as be­ing a more $$ eze­tim­ibe. Less like­ly: un­ex­pect­ed ben­e­fit due to in­flam­ma­tion or oth­er ef­fects. Al­so pos­si­ble it has no CVOT ben­e­fit or stud­ies not pow­ered ad­e­quate­ly

The big take­away, though, is that Es­pe­ri­on ap­pears poised to chal­lenge the ma­jors, set­ting up a fur­ther dis­rup­tion that pay­ers should be more than hap­py to see. 

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.

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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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