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.

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Joe Jimenez, Getty

Ex-No­var­tis CEO Joe Jimenez is tak­ing an­oth­er crack at open­ing a new chap­ter in his ca­reer — and that in­cludes a new board seat and a $250M start­up

Joe Jimenez is back.

The ex-CEO of Novartis has taken a board seat on Century Therapeutics, the Versant and Bayer-backed startup focused on coming up with a brand new twist on cell therapies for cancer — a field where Jimenez made his mark backing the first personalized CAR-T approved for use.

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Can we make the an­tibi­ot­ic mar­ket great again?

The standard for-profit model in drug development is straightforward. Spend millions, even billions, to develop a medicine from scratch. The return on investment (and ideally a tidy profit) comes via volume and/or price, depending on the disease. But the string of big pharma exits and slew of biotech bankruptcies indicate that the model is sorely flawed when it comes to antibiotics.

The industry players contributing to the arsenal of antimicrobials are fast dwindling, and the pipeline for new antibiotics is embarrassingly sparse, the WHO has warned. Drugmakers are enticed by greener pastures, compared to the long, arduous and expensive path to antibiotic approval that offers little financial gain as treatments are typically priced cheaply, and often lose potency over time as microbes grow resistant to them.

Top Har­vard chemist caught up in FBI’s 'T­hou­sand Tal­ents' drag­net, ac­cused of ly­ing about Chi­nese con­nec­tions, pay

The FBI’s probe into the alleged theft of R&D secrets by Chinese authorities has drawn Harvard’s top chemist into its net.

The agency accused Charles M. Lieber, who chairs the university’s chemistry and chemical biology department, with lying about his involvement in China’s Thousand Talents campaign, which was established as a way of drawing in innovators from around the world. And the scientist, 60, was charged with making false statements about his ties to China.

The US attorneys office in Boston also announced charges against two Chinese nationals for helping the Chinese government.

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Eye­ing a trio of tri­al ini­ti­a­tions, Jim Wilson's gene ther­a­py start­up woos Bruce Gold­smith from Deer­field as CEO

Passage Bio — Jim Wilson’s self-described “legacy company” — has wooed a seasoned biotech executive to steer the clinical entry of its first three gene therapy programs.

Bruce Goldsmith jumps to the helm of Passage after a brief CEO stint at Civetta, a cancer-focused startup he helped launch while a venture partner at Deerfield. He takes over from OrbiMed partner and interim chief Stephen Squinto, who will now lead the R&D team.

The FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. They plan to squash it — and per­ma­nent­ly ex­ile him

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition. And their lawsuit is asking that Shkreli — with several years left on his prison sentence — be banned permanently from the pharma industry.

UP­DAT­ED: Ac­celeron res­ur­rects block­buster hopes for so­tater­cept with pos­i­tive PhII — and shares rock­et up

Acceleron $XLRN says that its first major trial readout of 2020 is a success.

In a Phase II study of 106 patients with pulmonary arterial hypertension (PAH), Acceleron’s experimental drug sotatercept hit its primary endpoint: a significant reduction in pulmonary vascular resistance. The drug also met three different secondary endpoints, including the 6-minute walking test.

“We’re thrilled to report such positive topline results from the PULSAR trial,” Acceleron CEO Habib Dable said in a statement. The company said in a conference call they plan on discussing a Phase III trial design with regulators.

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Short at­tack­er Sahm Ad­ran­gi draws crosshairs over a fa­vorite of Sanofi’s new CEO — with PhII da­ta loom­ing

Sahm Adrang Kerrisdale

Kerrisdale chief Sahm Adrangi took a lengthy break from his series of biotech short attacks after his chief analyst in the field pulled up stakes and went solo. But he’s making a return to drug development this morning, drawing crosshairs over a company that’s one of new Sanofi CEO Paul Hudson’s favorite collaborators.

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Amber Saltzman (Ohana)

Flag­ship's first ven­ture of 2020 is out, and it's all about sperm

A couple years ago, Amber Salzman got a call as she was returning East full-time after a two-year stint running a gene therapy company in California.

It was from someone at Flagship Pioneering, the deep-pocketed biotech venture firm. They had a new company with a new way of thinking about sperm. It had been incubating for over a year, and now they wanted her to run it.

“It exactly fit,” Salzman told Endpoints News. “I just thought I had to do something.”