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.

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

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Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

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Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

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Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

Lina Khan, FTC chair (Graeme Jennings/Pool via AP Images)

Pile-on over PBMs con­tin­ues with FTC com­ments and a new bi­par­ti­san Sen­ate bill

More than 500 stakeholders sent comments to the FTC on whether the commission should look further into pharma middlemen, known as PBMs, with many of the commenters calling for more federal oversight.

Similar to the critical open comment period in a deadlocked FTC session last February, pharmacies and pharmacy groups are continuing to call out the lack of transparency among the top 3 PBMs, which control about 80% of the market.

Pharma brands are losing their shine with US consumers who are now thinking about the economy and inflation instead of Covid. (Credit: Shutterstock)

Phar­ma brands fade in an­nu­al Har­ris con­sumer vis­i­bil­i­ty poll: Mod­er­na drops off and Pfiz­er dips

As Covid-19 concerns are fading in the US, so is biopharma visibility. The annual Axios Harris Poll survey to determine and rank the 100 most top-of-mind brands in the US finds Moderna, which was No. 3 last year, not on the list at all for 2022, and Pfizer sinking 37 spots.

However, it’s not that Moderna or Pfizer did anything wrong, it’s just that Americans have moved on to other worries beyond Covid.

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HHS Secretary Xavier Becerra (Jacquelyn Martin/AP Images)

HHS fin­ish­es off Trump-era rule that would've erased ba­sic FDA regs with­out fre­quent re­views

HHS on Thursday finalized its decision to withdraw a rule, proposed just before former President Donald Trump left office, that would’ve caused thousands of HHS and FDA regulations to automatically expire if they weren’t reviewed within two years, and every 10 years thereafter.

The decision follows the filing of a lawsuit last March, in which several nonprofits alleged that the outgoing administration planted “a ticking timebomb” for HHS, essentially forcing it to devote an enormous amount of resources to the unprecedented and infeasible task of reviewing thousands of regulations regularly.

Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

Vi­iV Health­care looks to make long-act­ing HIV pre­ven­tion shot ac­ces­si­ble in low- and mid­dle-in­come coun­tries

The Joint United Nations Programme on HIV and AIDS set a lofty goal back in 2019 to end the HIV epidemic by 2030. But according to the World Health Organization, infection rates are not falling rapidly enough to meet that target.

GSK’s ViiV Healthcare thinks it can help change that.

On Friday, ViiV announced that it’s in talks with the UN-backed Medicines Patent Pool (MPP) for patent rights to its cabotegravir long-acting HIV injectable for pre-exposure prophylaxis (PrEP) in low- and middle-income countries.