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.

As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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The Advance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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No­var­tis jumps in­to Covid-19 vac­cine hunt, as Big Phar­ma and big biotech com­mit to bil­lions of dos­es

After spending most of the pandemic on the sidelines, Novartis is offering its aid in the race to develop a Covid-19 vaccine.

AveXis, the Swiss pharma’s gene therapy subsidiary, has agreed to manufacture the vaccine being developed by Massachusetts Eye and Ear and Massachusetts General Hospital. The biotech will begin manufacturing this month, while the vaccine undergoes further preclinical testing. They’ve agreed to provide the vaccine for free for clinical trials beginning in the second half of 2020, but have not disclosed financials for after.

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Bris­tol My­ers Squibb fi­nal­ly gets in the front­line NSCLC game dom­i­nat­ed by Mer­ck, adding a sec­ond Op­di­vo/Yer­voy-based op­tion

Bristol Myers Squibb may be trailing Merck and Roche in the checkpoint race to treat frontline cases of non-small cell lung cancer, but as it does, it makes sure to bring its best feet forward.

Just days after scoring a landmark NSCLC approval for Opdivo and Yervoy alone for PD-L1 positive patients, the company said the FDA has also OK’d using the two agents with a limited course of chemo regardless of the biomarker status.

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Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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