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.

RWE chal­lenges for to­day's bio­phar­ma

The rapid development of technology — and the resulting avalanche of data — are catalysts for significant change in the biopharmaceutical industry. This translates into urgent pressures for today’s biopharma, including a need to quickly and affordably develop products with proven therapeutic efficacy and value. This urgency is expedited by the growth of value-based contracting, where access to reimbursement and profit depends on these abilities.

UP­DAT­ED: In a stun­ning turn­around, Bio­gen says that ad­u­canum­ab does work for Alzheimer's — but da­ta min­ing in­cites con­tro­ver­sy and ques­tions

Biogen has confounded the biotech world one more time.

In a stunning about-face, the company and its partners at Eisai say that a new analysis of a larger dataset on aducanumab has restored its faith in the drug as a game-changer for Alzheimer’s and, after talking it over with the FDA, they’ll now be filing for an approval of a drug that had been given up for dead.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 63,100+ biopharma pros reading Endpoints daily — and it's free.

As shares suf­fer from a lin­ger­ing slump, a bruised Alk­er­mes slash­es 160 jobs in R&D re­struc­tur­ing

With its share price in a deep slump after suffering through a regulatory debacle over their depression drug ALKS 5461, Alkermes CEO Richard Pops is taking the ax to its R&D organization in a restructuring aimed at cutting costs ahead of its next attempt at a rollout in a tough field.

Richard Pops, Endpoints via Youtube

Click on the image to see the full-sized version

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 63,100+ biopharma pros reading Endpoints daily — and it's free.

Acor­da's Ron Co­hen brings the ax back out as new drug sales on­ly trick­le in while cash cow is led to the slaugh­ter

With its new drug earning meager sums and its one-time cash cow reduced to a bony shadow of its former self, Acorda Therapeutics today is rolling out a new restructuring aimed at slashing the staff and cutting costs to get through the hard times ahead.

The biotech is chopping a quarter of its staff today, carving back R&D as well as SG&A expenses. And CEO Ron Cohen is cutting deep.

Under the new austerity budget, Acorda’s R&D expenses for the full year 2019 are expected to be $55 – $60 million, reduced from $70 – $80 million. SG&A expenses for the full year 2019 are expected to be $185 – $190 million, reduced from $200 – $210 million. R&D expenses for the full year 2020 are expected to be $20 – $25 million and SG&A
expenses for the full year 2020 are expected to be $160 – $165 million.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 63,100+ biopharma pros reading Endpoints daily — and it's free.

RAPT Ther­a­peu­tics re­turns to Wall Street to re­vive IPO bid

On May 24, FLX Bio, a small cancer and inflammation biotech with backing from GV, changed its name to RAPT Therapeutics and filed confidentially for an IPO. On July 5th, they filed to raise up to $86 million. On July 22, they announced the IPO with a $75 million goal.  And on August 1, they abruptly and without explanation called it all off.

Now, without explanation, they’re reviving the bid, filing again for a $75 million IPO, this time with a new bookrunner and a new drug candidate in the clinic. The terms will be the same: 5 million shares at $14-$16 per share. It would give them a diluted market value of $351 million.

EY vet set to re­place re­tir­ing Am­gen CFO Meline

Ahead of its third-quarter results next week, Amgen on Tuesday disclosed the planned retirement of David Meline, who has served as the company’s chief financial officer since 2014.

Meline will be replaced by Ernst & Young vet, Peter Griffith, as CFO come January 1, 2020 — but until then Griffith will serve as executive vice president, finance.

“Over the last 5 years at Amgen, Meline instituted many major changes that led to operational efficiencies and margin expansion while successfully returning cash to shareholders. Now that Amgen is on solid footing, it was a good time to step away,” Cowen’s Yaron Werber wrote in a note. “We do not anticipate any major changes to strategy or operations immediately due to this transition as Amgen is on solid footing.”

Eli Lil­ly’s USA, di­a­betes chief En­rique Con­ter­no is head­ing out af­ter 27 years, and he’s be­ing re­placed by a com­pa­ny in­sid­er

Close to 3 years after Eli Lilly CEO Dave Ricks added the title of president of the US operations to Enrique Conterno’s resume, which included his helmsmanship of the diabetes franchise, the Peruvian born exec is set to retire after a 27-year run at the pharma giant.

Lilly put out the news just as it was posting Q3 results, with a mix of upbeat and downbeat results in the latest set of numbers from Lilly.
Conterno — a grizzled, deeply experienced and sometimes gruff veteran of the pharma world — was a high-profile figure at Lilly, stepping up to expanded duties as the company was forced to deal with intense pricing pressure on the diabetes side of the business. He had replaced outgoing US president Alex Azar, who later popped up as head of Health and Human Services in the Trump administration.
As head of the diabetes unit, Conterno had to deal with an extraordinarily competitive field as payers demanded bigger discounts. Trulicity’s success helped generate new revenue for the company, but Q3’s miss on revenue had a lot to do with the need for discounting the drug ahead of Novo Nordisk’s rival therapy, Rybelsus, which was priced on the wholesale level at an almost identical rate.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 63,100+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis hands off $80M in cash to part­ner up with a top biotech play­er in the fi­bro­sis sec­tor

Never underestimate the power of a good showing at a scientific conference.
In a presentation late last year, the researchers at Pliant Therapeutics launched a series of discussions about the preclinical data they were pulling together around their work on their small-molecule integrin inhibitor aimed at transforming growth factor beta, or TGF-β, a key pathway involved in fibrosis.
And they got some serious attention for the work.
“We got interest from pharma partners and at the end Novartis basically made it,” says Pliant CEO Bernard Coulie.

Is there a recipe for M&A suc­cess? The best and worst buy­out deals in the past decade of­fer some keys to suc­cess — and fail­ure

It’s not easy achieving a solid win in M&A in this industry. But if you follow a few simple guidelines, you may be able to increase your odds of success.
Geoffrey Porges and the team at SVB Leerink went about the “notoriously difficult” task of scoring the biopharma buyout of 2009 to 2019. Sizing up current and expected revenue from the products that were gained, they came up with the 5 winners:
Merck/Schering Plough
Bristol/Medarex
Gilead/Pharmasset
Sanofi/Genzyme
AstraZeneca/Acerta
It says a lot about the field that it’s much easier sorting out the 5 worst deals, though there’s also a lot more competition for that title, notes Porges. As picked by the analysts:
J&J/Actelion
Merck/Cubist
Alexion/Synageva
AbbVie/Stemcentrx
Gilead/Kite

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 63,100+ biopharma pros reading Endpoints daily — and it's free.