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.

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

Up­dat­ed: Hit by an­oth­er PhI­II flop, Sanofi culls breast can­cer drug — sound­ing alarm for the class

Sanofi is officially giving up on its oral SERD.

The French drugmaker put out word Wednesday morning that it will discontinue the global development program of amcenestrant, the selective estrogen receptor degrader once billed as a top late-stage prospect. Having already failed a Phase II monotherapy test earlier this year, a combo with the drug also missed the bar in a second trial for breast cancer, triggering the decision to drop the whole program.

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Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Joe Jonas (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

So­lo Jonas broth­er car­ries Merz's new tune in Botox ri­val cam­paign

As the lyrics of his band’s 2019 pop-rock single suggest, Joe Jonas is only human — and that means even he gets frown lines. The 33-year-old singer-songwriter is Merz’s newest celebrity brand partner for its Botox rival Xeomin, as medical aesthetics brands target a younger audience.

Merz kicked off its “Beauty on Your Terms” campaign on Tuesday, featuring the Jonas brother in a video ad for its double-filtered anti-wrinkle injection Xeomin.

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Tom Barnes, Orna Therapeutics CEO

UP­DAT­ED: 'We have failed to fail': Mer­ck gam­bles $250M cash on a next-gen ap­proach to mR­NA — af­ter punt­ing its big al­liance with Mod­er­na

Merck went in deep on its collaboration with Moderna on new mRNA programs, and dropped them all over time, including their RSV partnership. But after writing off what turned out as one of the most successful infectious disease players in the business, Merck is coming in this morning with a new preclinical alliance — this time embracing a biotech that hopes to eventually outdo the famously successful mRNA in a new run at vaccines and therapeutics.

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Marisol Peron, Genmab SVP of communications and corporate affairs

Gen­mab launch­es cor­po­rate cam­paign am­pli­fy­ing its ‘knock your socks off’ an­ti­bod­ies

Genmab often talks about its “knock-your-socks-off” antibodies — and now the term is getting its own logo and corporate campaign.

The teal and purple logo for the acronym KYSO — Genmab pronounces it “ky-so” — debuts on Wednesday and comes on the heels of Genmab’s newly announced 2030 vision. That aspiration aims to expand Genmab’s drug development beyond oncology to include other serious diseases, while also doubling down on its own drug development.

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President Joe Biden signs the Democrats' landmark climate change and health care bill. From L-R: Sen. Joe Manchin (D-WV), Senate Majority Leader Chuck Schumer (D-NY), House Majority Whip James Clyburn (D-SC), Rep. Frank Pallone (D-NJ) and Rep. Kathy Castor (D-FL). (Susan Walsh/AP Images)

Pres­i­dent Biden signs ma­jor drug pric­ing re­forms in­to law: What's com­ing for bio­phar­ma?

President Joe Biden yesterday afternoon signed into law historic, decades-in-the-making new drug pricing reforms as part of a wider reconciliation bill that will likely take a chunk out of biopharma companies’ profits for some blockbusters just prior to generic or biosimilar competition.

The partisan bill (all Democrats in the House and Senate voted for it, and all Republicans voted against it) includes not only Medicare price negotiations — which won’t kick off until 2026, leaving ample time for a legal challenge — but mandatory inflation-related rebates, and a $2,000 annual cap on what seniors’ pay for their prescription drugs.

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Paul Perreault, CSL Behring CEO

CSL CEO Paul Per­reault de­ter­mined to grow plas­ma col­lec­tion af­ter full-year sales dip

As the ink dries on CSL’s $11.7 billion Vifor buyout, the company posted a dip in profits, due in part to a drop in plasma donations amid the pandemic.

However, CEO Paul Perreault assured investors and analysts on the full-year call that the team has left “no stone unturned” when assessing options to grow plasma volumes. The chief executive also spelled out positive results for the company’s monoclonal antibody garadacimab in hereditary angioedema (HAE), though he isn’t revealing the exact numbers just yet.