Marathon CEO tries to call a time out on con­tro­ver­sy as law­mak­ers rip in­to $89K de­flaza­cort price

Sen­a­tor Bernie Sanders and Con­gress­man Eli­jah Cum­mings have found their new poster boy for phar­ma price goug­ing. His name is Jeff Aronin, the CEO of Marathon Phar­ma­ceu­ti­cals.

In a joint let­ter ad­dressed to Aronin, the two law­mak­ers slammed the com­pa­ny’s “out­ra­geous plans” to set a list price of $89,000 on their new­ly ap­proved de­flaza­cort, a cheap steroid that’s been avail­able for decades in oth­er coun­tries around the world. And they launched an in­ves­ti­ga­tion in­to Marathon’s plans, de­mand­ing doc­u­ments re­lat­ed to the ac­tu­al cost of the pro­gram while in­sist­ing that Marathon “sig­nif­i­cant­ly low­er the price” for re­leas­ing the drug.

The let­ter cites sto­ries from End­points News, The Wash­ing­ton Post, Stat and oth­ers.

Al­most si­mul­ta­ne­ous­ly, Marathon ex­e­cut­ed a strate­gic re­treat, telling a group of rep­re­sen­ta­tives from var­i­ous Duchenne groups at a pol­i­cy meet­ing in Wash­ing­ton DC that they were call­ing a “pause on the launch,” ac­cord­ing to one of the at­ten­dees, who asked not to be iden­ti­fied.

Var­i­ous Marathon rep­re­sen­ta­tives were at the meet­ing, in­clud­ing mar­ket­ing chief Er­ic Mesnner and de­vel­op­ment chief Tim Cun­niff, and a state­ment from Aronin was read out as­sur­ing the Duchenne com­mu­ni­ty that they would still be able to or­der de­flaza­cort from Mas­ters Glob­al in the UK “un­til we fig­ure this out.”

In his state­ment, Aronin re­peat­ed his ear­li­er, wide­ly dis­put­ed po­si­tion that the com­pa­ny’s pri­ma­ry in­ter­est was in bring­ing the steroid to pa­tients who cur­rent­ly can’t get it. He pledged:

“We will meet with care­givers and ex­plain our com­mer­cial­iza­tion plans, re­view their con­cerns, dis­cuss all op­tions, and move for­ward with com­mer­cial­iza­tion based on an agreed plan of ac­tion.”

That will re­quire some quick foot­work with law­mak­ers.

“Marathon did not de­vel­op de­flaza­cort,” Sanders and Cum­mings wrote. “Rather Marathon ac­quired the rights to his­tor­i­cal clin­i­cal tri­al da­ta from the 1990s and com­plet­ed some ad­di­tion­al analy­ses to gain ap­proval from the Food and Drug Ad­min­is­tra­tion to sell the drug in the Unit­ed States.

“We be­lieve that Marathon is abus­ing our na­tion’s or­phan drug pro­gram, which grants com­pa­nies sev­en years of mar­ket ex­clu­siv­i­ty to en­cour­age re­search in­to new treat­ments for rare dis­eases, not to pro­vide com­pa­nies like Marathon with lu­cra­tive mar­ket ex­clu­siv­i­ty rights for drugs that have been avail­able for decades.”

As we re­port­ed this morn­ing, Marathon has made much out of its R&D ef­forts, say­ing the price was jus­ti­fied by the amount that was spent to gain an ap­proval. Tri­al ex­perts, though, say it could have been done for any­thing from less than $10 mil­lion to a high of $75 mil­lion, a sum that could be eas­i­ly cov­ered just by the sale of the pri­or­i­ty re­view vouch­er that it ob­tained from the ap­proval.

The lat­est pric­ing con­tro­ver­sy wasn’t over­looked by pay­ers and drug ben­e­fit man­agers.

“As you might ex­pect,” not­ed a spokesper­son for Ex­press Scripts in a mes­sage to me, “we be­lieve this is an­oth­er ex­am­ple of egre­gious pric­ing for an old drug that is avail­able else­where for much less.”

Marathon has con­sis­tent­ly re­fused to re­spond to my re­peat­ed re­quests for an in­ter­view with Aronin, whose broth­er, J&J vet Greg Aronin, works as a lob­by­ist for the com­pa­ny.

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Chris Garabedian. Xontogeny

Per­cep­tive teams up with Chris Garabe­di­an to open up a new, $210M biotech fund fo­cused on A rounds

Perceptive Advisors is one of those prolific biotech investor groups which has traditionally enjoyed zeroing in on clinical-stage investments and crossover rounds, a group that prefers more established drug development players with near-term payoff potential.

But now they’re partnering with Xontogeny chief and longtime biotech entrepreneur Chris Garabedian on a $210 million fund — with money contributed by institutional investors and family funds — to go into the launch space with their first early-stage VC fund. Dubbed the Perceptive Xontogeny Venture Fund, LP, or just PXV Fund, they plan to favor upstarts that Garabedian is fostering in his incubator. But they’ll also plan to reach outside that inner circle for more A rounds to back, with plans to dominate initial funding with $10 million to $20 million per newborn biotech.

Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

This is the second biotech buyout pact today, marking a brisk tempo of M&A deals in the lead-up to the big JP Morgan gathering in mid-January. It’s no surprise the acquisitions are both for cancer drugs, where Sanofi will try to make its mark while Merck beefs up a stellar oncology franchise. And bolt-ons are all the rage at the major pharma players, which you could also see in Novartis’ recent $9.7 billion MedCo buyout.

ArQule — which comes out on top after their original lead drug foundered in Phase III — highlighted early data on ‘531 at EHA from a group of 6 chronic lymphocytic leukemia patients who got the 65 mg dose. Four of them experienced a partial response — a big advance for a company that failed with earlier attempts.

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US biosim­i­lar launch­es about to turn a cor­ner

The US biosimilar industry has lingered in the shadow of the European market since the US pathway for approvals was initiated in 2009.

Ten years later (or less than five years since the first FDA approval of a biosimilar), and just 42% (11 out of 26) of FDA-approved biosimilars have launched. But in the next three months (see chart below), a clutch of new biosimilars will hit the market, including new ones in oncology, hinting at a wave of uptake.

Left top to right: Mark Timney, Alex Denner, Vas Narasimhan. (The Medicines Company, Getty, AP/Endpoints News)

In a play-by-play of the $9.7B Med­Co buy­out, No­var­tis ad­mits it over­paid while of­fer­ing a huge wind­fall to ex­ecs

A month into his tenure at The Medicines Company, new CEO Mark Timney reached out to then-Novartis pharma chief Paul Hudson: Any interest in a partnership?

No, Hudson told him. Not now, at least.

Ten months later, Hudson had left to run Sanofi and Novartis CEO Vas Narasimhan was paying $9.7 billion for the one-drug biotech – the largest in the string of acquisitions Narasimhan has signed since his 2017 appointment.

The deal was the product of an activist investor and his controversial partner working through nearly a year of cat-and-mouse negotiations to secure a deal with Big Pharma’s most expansionist executive. It represented a huge bet in a cardiovascular field that already saw two major busts in recent years and brought massive returns for two of the industry’s most eye-raising names.

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Paul Hudson. Sanofi

New Sanofi CEO Hud­son adds next-gen can­cer drug tech to the R&D quest, buy­ing Syn­thorx for $2.5B

When Paul Hudson lays out his R&D vision for Sanofi tomorrow, he will have a new slate of interleukin therapies and a synthetic biology platform to boast about.

The French pharma giant announced early Monday that it is snagging San Diego biotech Synthorx in a $2.5 billion deal. That marks an affordable bolt-on for Sanofi but a considerable return for Synthorx backers, including Avalon, RA Capital and OrbiMed: At $68 per share, the price represents a 172% premium to Friday’s closing.

Synthorx’s take on alternative IL-2 drugs for both cancer and autoimmune disorders — enabled by a synthetic DNA base pair pioneered by Scripps professor Floyd Romesberg — “fits perfectly” with the kind of innovation that he wants at Sanofi, Hudson said.

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Game on: Re­gen­eron's BC­MA bis­pe­cif­ic makes clin­i­cal da­ta de­but, kick­ing off mul­ti­ple myelo­ma matchup with Bris­tol-My­ers

As J&J attempts to jostle past Bristol-Myers Squibb and bluebird for a landmark approval of its anti-BCMA CAR-T — and while GlaxoSmithKline maps a quick path to the FDA riding on its own BCMA-targeting antibody-drug conjugates — the bispecifics are arriving on the scene to stake a claim for a market that could cross $10 billion per year.

The main rivalry in multiple myeloma is shaping up to be one between Regeneron and Bristol-Myers, which picked up a bispecific antibody to BCMA through its recently closed $74 billion takeover of Celgene. Both presented promising first-in-human data at the ASH 2019 meeting.

FDA lifts hold on Abeon­a's but­ter­fly dis­ease ther­a­py, paving way for piv­otal study

It’s been a difficult few years for gene and cell therapy startup Abeona Therapeutics. Its newly crowned chief Carsten Thiel was forced out last year following accusations of unspecified “personal misconduct,” and this September, the FDA imposed a clinical hold on its therapy for a form of “butterfly” disease. But things are beginning to perk up. On Monday, the company said the regulator had lifted its hold and the experimental therapy is now set to be evaluated in a late-stage study.