PTC's ataluren flops in an­oth­er PhI­II and an an­a­lyst says it's clear­ly a dud; kill it

PTC Ther­a­peu­tics CEO Stu­art Peltz

PTC Ther­a­peu­tics has whiffed once again on Translar­na (ataluren).

The biotech re­port­ed that ataluren, a re­peat flop in Duchenne mus­cu­lar dy­s­tro­phy, failed a Phase III for non­sense mu­ta­tion cys­tic fi­bro­sis on both the pri­ma­ry and sec­ondary end­points.

In­ves­ti­ga­tors tracked a slight but in­signif­i­cant ad­van­tage for pa­tients in the drug arm of the  CF study, not­ing a bet­ter num­ber for lung ca­pac­i­ty and the rate of pul­monary ex­ac­er­ba­tions. But none of it stacked up in a way worth tak­ing to reg­u­la­tors, and the biotech says it will scrap its work in CF and jerk its ap­pli­ca­tion for ap­proval in Eu­rope.

The com­pa­ny’s stock $PTCT dropped 20% on the news.

PTC has a long track record of fail­ure in the clin­ic when it comes to ataluren. A flop in Phase IIb as well as Phase III for DMD, the FDA re­fused to ac­cept its ap­pli­ca­tion for re­view in ear­ly 2016, say­ing the biotech’s pitch failed to qual­i­fy for a re­view — a rare slap­down from reg­u­la­tors. PTC is now ap­peal­ing that de­ci­sion for the sec­ond time.

De­spite that, though, Eu­ro­pean of­fi­cials have al­lowed the sale of Translar­na for Duchenne mus­cu­lar dy­s­tro­phy. The Eu­ro­pean Com­mis­sion rat­i­fied an ex­ten­sion of PTC’s mar­ket­ing ap­proval in ear­ly Jan­u­ary as PTC pur­sues an­oth­er study to keep its mar­ket­ing au­tho­riza­tion in place. And the biotech ex­pects to earn more than $100 mil­lion this year on sales out­side the US, backed by a num­ber of pa­tient ad­vo­cates who be­lieve the drug works, de­spite the da­ta.

RBC’s Simos Sime­oni­dis said this af­ter­noon that the string of tri­al fail­ures demon­strate that the drug is clear­ly a dud and ought to be pulled.

Fol­low­ing this com­pound’s fail­ure in the ACT DMD tri­al, which was sup­posed to be the con­fir­ma­to­ry tri­al (which based on post-hoc analy­ses from a Phase II tri­al in DMD that had failed to meet its pri­ma­ry end­point), we no longer be­lieved this com­pound worked in CF, and had thus as­signed 0% prob­a­bil­i­ty of suc­cess for it in ACT CF. We now be­lieve there is am­ple clin­i­cal ev­i­dence from mul­ti­ple ran­dom­ized clin­i­cal tri­als that this is an in­ac­tive com­pound.

“We are dis­ap­point­ed with the out­come of this tri­al as there are no treat­ments that tar­get the un­der­ly­ing cause of non­sense mu­ta­tion cys­tic fi­bro­sis, one of the most dif­fi­cult forms to treat,” said Stu­art W. Peltz, PhD, chief ex­ec­u­tive of­fi­cer of PTC Ther­a­peu­tics in pre­pared text. “We are par­tic­u­lar­ly grate­ful to pa­tients and in­ves­ti­ga­tors who par­tic­i­pat­ed in our tri­als. We re­main com­mit­ted to pa­tients re­ceiv­ing ataluren in oth­er in­di­ca­tions.”

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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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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Emma Walmsley, GlaxoSmithKline CEO (Kevin Dietsch/Pool via CNP/Alamy)

Glax­o­SmithK­line hus­tles the 7th PD-1 past the fin­ish line with Jem­per­li. But how big will up­take be?

Everything came up sevens for GlaxoSmithKline on Thursday as the pharma notched the seventh PD-1 approval seven years after the first such drugs were OK’ed in Keytruda and Opdivo. But will it bring GSK good fortune?

The FDA granted accelerated approval to dostarlimab, to be branded Jemperli, to treat recurrent or advanced endometrial cancer in a specific subset of patients following platinum-based chemo. It’s a drug that came to GSK through its buyout of Tesaro, which it snapped up for $5.1 billion back in December 2018.

JP Gabriel, Ocugen

JP Gabriel watched from the bleach­ers as the pan­dem­ic raged. Now head of sup­ply chain at Ocu­gen, he's ready to bat

The world was in the middle of the most pressing public health risk his generation had ever seen, and JP Gabriel felt like he was sitting on the sidelines. As a VP of biologics and mRNA manufacturing at Ultragenyx, Gabriel watched from the sidelines as players like Pfizer/BioNTech and Moderna used mRNA tech to chase their own Covid-19 vaccines.

This month, Gabriel got the chance to get his hands dirty against the pandemic — but it won’t be with mRNA.

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Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

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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