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.

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Covid-19 man­u­fac­tur­ing roundup: Mary­land looks to grow biotech ca­pac­i­ty with $400M check; Rus­sia lands sec­ond Sput­nik V part­ner this week

A Maryland real estate project has added three new biotech-focused manufacturing and research buildings to an office park to keep up with demand created by the pandemic, the Washington Business Journal reported.

The Milestone Business Park — located off of I-270 in Germantown, MD — will see the new buildings and a total of 532,000 square feet as the campus rebrands to Milestone Innovation Park.

Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Bay­er plots a ma­jor facelift at Berke­ley cam­pus, un­cork­ing a 30-year, $1.2B plan to dri­ve cell and gene ther­a­pies

Bayer first set roots in Berkeley back in 1974, when it was still operating as Miles Labs. The site has pumped out three hemophilia A treatments for distribution worldwide; but now, as the pharma continues its cell and gene therapy push, it has something bigger in mind.

Bayer is planning a 30-year revamp at the campus, which includes 918,000 square feet in new buildings and double the jobs, according to a report by the Bay Area Council Economic Institute.

LLS backs 5 new can­cer drug projects with up to $50M; Trodelvy con­tin­ues to im­press with more TNBC da­ta

The Leukemia and Lymphoma Society has tapped 5 new early-stage projects to back with up to $10 million each in fresh investments. The 5 biotechs are:

— Caribou, headed by Rachel Haurwitz and co-founded by Jennifer Doudna, is working on next-gen, off-the-shelf CAR-Ts to replace the patient-derived cells now in use.

— The LLS supported NexImmune’s IPO, helping fund its work on nanoparticles that can gin up an immune response directed at cancer cells. The biotech has 2 projects now in Phase I trials.

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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