Af­ter be­ing spurned at the FDA, PTC buys a con­tro­ver­sial Duchenne MD ther­a­py from Marathon

Af­ter be­ing stung by a fierce back­lash over its plans to sell a cheap, old steroid for Duchenne mus­cu­lar dy­s­tro­phy for $89,000 a year, Marathon Phar­ma­ceu­ti­cals is get­ting out. The com­pa­ny an­nounced this morn­ing that it is sell­ing de­flaza­cort (Em­flaza) to Duchenne MD play­er PTC Ther­a­peu­tics $PTCT, which has been strug­gling to get the FDA to pro­vide a se­ri­ous re­view for its own failed drug.

Marathon is get­ting $140 mil­lion in cash and stock in the deal, along with a shot at a one-time $50 mil­lion sales bonus on the ta­ble and a roy­al­ty stream that will ac­count for a low-to-mid 20s per­cent­age of the rev­enue.

PTC Ther­a­peu­tics CEO Stu­art Peltz

PTC CEO Stu­art Peltz doesn’t say in the re­lease how much he plans to charge for de­flaza­cort. The com­pa­ny’s stock slid 13% af­ter the news hit.

Marathon trig­gered a storm of crit­i­cism with its plans, which is con­tin­u­ing un­abat­ed now with a new fo­cus on the FDA’s role in the ap­proval. A large num­ber of Duchenne par­ents were able to buy gener­ic de­flaza­cort over­seas for around $1,000 a year, and crit­ics saw Marathon’s move as an­oth­er in a se­ries of price goug­ing scan­dals.

That scan­dal is now PTC’s to deal with.

“With our near­ly 20-year com­mit­ment to the Duchenne com­mu­ni­ty, it is deeply mean­ing­ful for us to bring this crit­i­cal ther­a­py to U.S. pa­tients,” said Peltz in a state­ment. “We be­lieve Em­flaza is a dis­ease-mod­i­fy­ing ther­a­py that has been shown to slow dis­ease pro­gres­sion. In keep­ing with PTC’s mis­sion, we are ex­cit­ed to work with the com­mu­ni­ty to raise the stan­dard of care for DMD pa­tients.”

Claim­ing that de­flaza­cort is a dis­ease mod­i­fy­ing ther­a­py will sur­prise many in the Duchenne com­mu­ni­ty. Like any steroid, it strength­ens pa­tients at a cost. Many of the par­ents came to pre­fer de­flazafort over pred­nisone be­cause it is as­so­ci­at­ed with less weight gain.

In ac­quir­ing the old steroid to be sold as a brand­ed ther­a­py, PTC is al­so adopt­ing Marathon’s claim that the ap­proval makes it pos­si­ble to ex­pand ac­cess — now “lim­it­ed to a small num­ber of pa­tients,” Peltz said in a call with an­a­lysts to­day — to all pa­tients over the age of 5.

Mark Rather, PTC CCO

“We plan to re­ex­am­ine the price of Em­flaza,” added Mark Rothera, PTC’s chief com­mer­cial of­fi­cer. “This is a clas­sic or­phan drug launch that we’re fac­ing in the Unit­ed States.”

“It re­al­ly wasn’t avail­able to pa­tients,” Peltz told an­a­lysts, es­ti­mat­ing that few­er than 10% of the pa­tients had ac­cess to the steroid.

Ac­cord­ing to Duchenne ad­vo­cate Chris­tine Mc­Sh­er­ry, though, quite a few fam­i­lies had no prob­lem get­ting over­seas sup­plies of the steroid. Mc­Sh­er­ry ini­tial­ly es­ti­mat­ed that 40% to 50% of the DMD kids are al­ready on de­flaza­cort and will now be forced to switch to the high­er priced US sup­pli­er, then ad­just­ed that down to a con­ser­v­a­tive 25%.

Law­mak­ers, in­clud­ing Sen­a­tor Bernie Sanders, have blast­ed Marathon’s claims that it need­ed to charge a high price to even­tu­al­ly re­gain the cost of de­vel­op­ment.

Peltz has tried and failed to prove that PTC’s drug ataluren — which failed back-to-back stud­ies and re­cent­ly flopped for cys­tic fi­bro­sis — could ben­e­fit Duchenne par­ents. He con­tin­ues to in­sist that the “to­tal­i­ty” of the da­ta proves its worth, but the FDA re­fused to even file their ap­pli­ca­tion, say­ing they didn’t have the da­ta need­ed for a re­view. PTC, though, re­cent­ly lever­aged the reg­u­la­tions to force a PDU­FA date for their drug. And ex­ecs styled this new deal as an open­ing to start US com­mer­cial­iza­tion ef­forts as it pre­pares to push ataluren in­to the mar­ket.

Eu­ro­pean reg­u­la­tors, mean­while, did give the drug a con­di­tion­al ap­proval, ex­tend­ing that with a new re­quire­ment to com­plete an ad­di­tion­al study in the next five years.

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

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CEO Pascal Soriot via Getty Images

As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.