Marathon Phar­ma­ceu­ti­cals sig­nals it will wind down af­ter stir­ring a hor­net’s nest with $89,000 Duchenne treat­ment


Marathon Phar­ma­ceu­ti­cals has been qui­et­ly dis­cussing plans to shut­ter its op­er­a­tions af­ter kick­ing up a storm of con­tro­ver­sy over an ef­fort to mar­ket a cheap over­seas steroid to the Duchenne mus­cu­lar dy­s­tro­phy com­mu­ni­ty in the US at a list price of $89,000.

In an email out to a con­tact in the Duchenne mus­cu­lar dy­s­tro­phy com­mu­ni­ty in late March, which I ob­tained from some­one fa­mil­iar with the sit­u­a­tion, R&D di­rec­tor Tim Cun­niff not­ed:

With the di­vesti­ture of de­flaza­cort to PTC, the com­pa­ny is wind­ing down and prob­a­bly won’t ex­ist much past May 1.

That drop-dead date may have sub­se­quent­ly been pushed back a bit as the com­pa­ny con­tin­ues ef­forts to sell its pri­or­i­ty re­view vouch­er, ob­tained with the FDA’s ap­proval of de­flaza­cort.

I con­tact­ed the com­pa­ny about its plans and re­ceived this state­ment:

With the wind down of our Em­flaza busi­ness fol­low­ing the close of the PTC trans­ac­tion on April 20, we will con­tin­ue to man­age the lega­cy mat­ters of Marathon Phar­ma­ceu­ti­cals.

Just days ago, North­brook, IL-based Marathon bowed out of PhRMA, which had said it was re­view­ing its mem­ber­ship goals with a plan to re­strict the group to com­pa­nies in­vest­ing heav­i­ly in R&D.

Marathon’s mar­ket­ing plans drew heavy flak from mem­bers of the Duchenne dis­ease com­mu­ni­ty, who had been buy­ing it for a lit­tle more than $1,000 a year from a UK sup­pli­er. Sen­a­tor Bernie Sanders and Rep. Eli­jah Cum­mings fol­lowed up, de­mand­ing doc­u­ments from the com­pa­ny to prove its claim that it had in­vest­ed heav­i­ly in the R&D pro­gram, some­thing that biotech ex­ecs doubt­ed giv­en the com­pa­ny’s out­line of its re­search ef­forts.

The bulk of the ef­fi­ca­cy da­ta was decades old, ob­tained from the orig­i­nal in­vestors for on­ly $350,000 — ac­cord­ing to a re­port in the Wall Street Jour­nal — the equiv­a­lent for four pre­scrip­tions at the full list price.

Caught in the spot­light, Marathon swift­ly put its launch plans on hold and then sold de­flaza­cort — or Em­flaza — to PTC Ther­a­peu­tics, which has been try­ing to gain an FDA ap­proval for ataluren, a drug that has failed the past three stud­ies. PTC agreed to pay $140 mil­lion for the drug, plus an­oth­er $50 mil­lion for a mile­stone. PTC is work­ing to for­mal­ly close the deal and has yet to an­nounce its own price, which is like­ly to gar­ner fresh head­lines.

PTC is al­so like­ly to face a harsh kick­back from pay­ers, who have been pay­ing pen­nies a pill for the ri­val gener­ic steroid, pred­nisone.

Marathon’s web­site says it has drugs for or­phan dis­eases in the pipeline, but doesn’t list any­thing. One of the biggest out­stand­ing is­sues is what hap­pens to its pri­or­i­ty re­view vouch­er, an in­cen­tive won from the FDA for its or­phan pro­gram. The last sold for $125 mil­lion and have been known to fetch as much as $350 mil­lion.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.


ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology


ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development


CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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UP­DAT­ED: Pay­back? An­a­lysts say Sarep­ta was blind­sided by an FDA re­jec­tion dri­ven by reg­u­la­to­ry re­venge

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
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FDA de­ci­sion on Ver­tex's CF triple will come just ahead of planned CEO shake­up

Vertex has clinched a priority review for the all-important cystic fibrosis triple that will blaze the trail for treating a large group of patients unhelped by its current drugs.

FDA regulators have set a PDUFA date of March 19, 2020, just a year after the Boston biotech posted positive Phase III results showing that people with two F508del mutations experienced statistically significant improvements in lung function after a 4-week regimen of VX-445, tezacaftor and ivacaftor. After reviewing 24-week data among patients with one F508del mutation and one minimal function mutation — and thoroughly comparing the VX-445 triple with another combo featuring VX-659 on scores like safety, drug-drug interactions, and photosensitivity — Vertex ultimately went with VX-445.

An MIT spin­out kills one of its ‘liv­ing ther­a­peu­tics’ af­ter flunk­ing an ear­ly-stage study — shares rout­ed

Just a few weeks after bagging $80 million in a deal to collaborate with Gingko Bioworks on its special blend of engineered bacteria used for “living therapeutics,” little Synlogic in Boston $SYBX is tossing one of its two clinical programs after watching an early-stage study go down in defeat.

Their Phase Ib/IIa study for SYNB1020 to counter the accumulation of ammonia in the body, a condition called hyperammonemia or urea cycle disorder, floundered at the interim readout, forcing the biotech to kill it and reserve its cash for pipeline therapies with greater potential.

Elan­co to buy Bay­er's an­i­mal health busi­ness for $7.6B, as deal­mak­ing gath­ers steam in the sec­tor

Last week, Elanco explicitly dodged answering questions about its rumored interest in Bayer’s animal health business in its post-earnings call. On Tuesday, the Eli Lilly spinoff disclosed it was purchasing the German drug maker’s veterinary unit in a cash-and-stock deal worth $7.6 billion. 

Elanco $ELAN has been busy on the deal-making front. In April, it laid out plans to swallow its partner, Kansas-based pet therapeutics company Aratana $PETX. A July report by Reuters suggested a potential Bayer deal was being explored, and Bloomberg last week said the deal was imminent, citing sources.