More Sen­a­tors ac­cuse Marathon CEO Aronin of ex­ploit­ing Duchenne MD pa­tients as well as FDA’s or­phan in­cen­tives

A group of US Sen­a­tors is tight­en­ing the screws on Marathon Phar­ma­ceu­ti­cals in the wake of the lat­est drug pric­ing scan­dal.

Jef­frey Aronin, Marathon

Just weeks af­ter Marathon CEO Jeff Aronin man­aged to out­rage a host of Duchenne mus­cu­lar dy­s­tro­phy fam­i­lies and Ver­mont Sen­a­tor Bernie Sanders with his de­ci­sion to price a cheap, gener­ic steroid at $89,000 a year af­ter fi­nagling an FDA ap­proval and some lu­cra­tive in­cen­tives specif­i­cal­ly tai­lored to the dis­ease, a group of US Sen­a­tors has come to­geth­er to step up de­mands for an­swers from the com­pa­ny about what it ac­tu­al­ly spent to prep this ther­a­py for an ap­proval.

“Marathon ex­ec­u­tives have de­fend­ed the price of the drug,” the sen­a­tors wrote, “ar­gu­ing that oth­er treat­ments for “or­phan dis­eases” — those that af­fect few­er than 200,000 na­tion­wide — cost more than $300,000. Marathon’s Chief Fi­nan­cial Of­fi­cer has stat­ed that Em­flaza is “mod­est­ly priced for an or­phan drug.” While this is true, or­phan drugs for rare dis­eases are gen­er­al­ly not old com­pounds that have been used to treat such rare dis­ease for decades.

They added: “We are con­cerned that Marathon’s pric­ing un­fair­ly ex­ploits the DMD pa­tient pop­u­la­tion and the FDA’s or­phan drug in­cen­tives.”

This group in­cludes sev­en De­moc­rats, such as Pat­ty Mur­ray, Al Franken and Corey Book­er, along with in­de­pen­dent An­gus King. And they fol­lowed up with a de­tailed list of ques­tions about how much Marathon spent to de­vel­op de­flaza­cort for the US mar­ket.

As I re­port­ed with­in days of the ap­proval, Marathon’s boast about its ex­ten­sive R&D ef­fort — the “heavy lift­ing” need­ed to win over the FDA which would take years of mar­ket­ing to cov­er — was thin cam­ou­flage for a rel­a­tive­ly mi­nor af­fair in­volv­ing a group of pre­clin­i­cal and clin­i­cal stud­ies that could be eas­i­ly cov­ered with the sale of the drug to a small part of the pa­tient pop­u­la­tion for the past year.

The furor over de­flaza­cort has fo­cused new at­ten­tion on how easy it is for bio­phar­ma com­pa­nies to game the FDA in com­plete­ly le­gal ways. In this case, Marathon took a steroid that’s been sold for around a thou­sand dol­lars a year to pa­tients in coun­tries around the world — as well as a num­ber of pa­tients and fam­i­lies in the US — and ma­neu­vered it to an OK as an or­phan drug, win­ning reg­u­la­tors’ ap­proval and snag­ging a pri­or­i­ty re­view vouch­er.

The last PRV was sold for $125 mil­lion just days ago and one fetched $350 mil­lion.

Pinned by crit­ics as the lat­est prof­i­teer to gouge pa­tients, Aronin sud­den­ly de­cid­ed to “pause” the launch of de­flaza­cort as he claimed that it was time to re­flect on the price and con­sult with stake­hold­ers. At the same time, the CEO went un­der­ground, re­fus­ing in­ter­views and pub­lic ap­pear­ances with Duchenne fam­i­lies.

The con­tro­ver­sy ar­rived at pre­cise­ly the worst pos­si­ble time for the trade group PhRMA, which has been try­ing to dis­tin­guish its mem­bers from price gougers like the no­to­ri­ous Mar­tin Shkre­li, who out­raged mil­lions by jack­ing up the price of gener­ic Dara­prim by more than 5000%. But that’s ex­act­ly the same for­mu­la that Aronin used, and he’s cur­rent­ly a board mem­ber of PhRMA, which is spend­ing tens of mil­lions of dol­lars in a PR cam­paign aimed at win­ning over the pub­lic at a time that drug prices have come un­der in­tense scruti­ny.

Stephen Ubl

PhRMA CEO Stephen Ubl says the group is now re­view­ing terms for mem­ber­ship, and has sug­gest­ed that Aronin may not be on the board for long.

The con­tro­ver­sy around Marathon shows no signs of sim­mer­ing down any­time soon, even while Aronin hun­kers down in the face of a hur­ri­cane of crit­i­cism in Con­gress.

The CEO has re­fused mul­ti­ple re­quests from me for an in­ter­view. I’ll leave it as a stand­ing in­vi­ta­tion, but I’ll be sur­prised if he ever re­sponds di­rect­ly.

Norbert Bischofberger. Kronos

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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 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.

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→ 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.

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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

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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.