PTC shares rock­et up as Eu­ro­pean reg­u­la­tors of­fer a thumbs-up on Duchenne drug mar­ket­ing

PTC Ther­a­peu­tics CEO Stu­art Peltz

A few days af­ter warn­ing in­vestors of the ris­ing un­cer­tain­ty it faced in the on­go­ing Eu­ro­pean re­view of its con­di­tion­al ap­proval for Translar­na, shares of PTC Ther­a­peu­tics $PTCT sky­rock­et­ed this morn­ing af­ter reg­u­la­tors gave it a thumbs up on keep­ing the con­tro­ver­sial Duchenne drug on the mar­ket.

There is a con­di­tion at­tached to the rec­om­men­da­tion from the Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use. The CHMP wants PTC to con­duct a place­bo-con­trolled, 18-month study of their drug fol­lowed by an 18-month ex­ten­sion study, with da­ta ex­pect­ed in ear­ly 2021. That would give them a clear mar­ket­ing run­way of close to 5 more years, if the EMA for­mer­ly ac­cepts the rec­om­men­da­tion.

PTC’s shares, beat­en down by the skep­ti­cism is faced, shot up more than 100% on the news.

PTC skep­tics abound. Its drug has re­peat­ed­ly failed clin­i­cal tri­als and a num­ber of an­a­lysts have spec­u­lat­ed that EU reg­u­la­tors would jerk the drug, which was giv­en a con­di­tion­al ap­proval back in 2014, ahead of a failed Phase III study. The FDA re­cent­ly re­fused to even con­sid­er its ap­pli­ca­tion, say­ing PTC had too weak a case to mer­it a thor­ough re­view.

PTC is ap­peal­ing that de­ci­sion, look­ing to get its drug and its pa­tient ad­vo­cates in front of the same reg­u­la­tors that gave Sarep­ta an ap­proval for Ex­ondys 51, even though in­sid­ers at the agency waged a hard fought cam­paign in­sist­ing that the drug had nev­er come close to meet­ing the FDA’s stan­dards for an ap­proval.

But Duchenne mus­cu­lar dy­s­tro­phy is a field un­to it­self, where the hard log­ic and high reg­u­la­to­ry bar that of­ten trig­gers drug re­jec­tions have been blunt­ed by pa­tient ad­vo­cates des­per­ate to have some drug avail­able that might slow a dis­ease that grad­u­al­ly kills its young vic­tims. That de­sire to have some­thing, any­thing, avail­able has been a boon to PTC and Sarep­ta, even as crit­ics shake their heads at the idea of ex­per­i­men­tal drugs with lit­tle ev­i­dence of ef­fi­ca­cy be­ing sold to pa­tients for six-fig­ure prices.

Bar­clays fol­lowed up with a dis­cus­sion with PTC ex­ecs and came away with this:

In speak­ing with man­age­ment, it is clear that there was sig­nif­i­cant dis­cus­sion and en­gage­ment with EMA over the past year. CHMP de­ter­mined that the drug does slow pro­gres­sion and has a fa­vor­able risk ben­e­fit pro­file. The com­pa­ny has not yet dis­closed oth­er as­pects of the tri­al, name­ly end­points and in­clu­sion/ ex­clu­sion cri­te­ria, but not­ed that the tri­al will be sim­i­lar in size to ACT DMD (230 en­roll­ment). Man­age­ment em­pha­sized that this up­date on­ly per­tains to EMA, as FDA dis­cus­sions are still on­go­ing. While the com­pa­ny would like to align tri­al ef­forts be­tween the EU and US, they not­ed that they hope that Ata­lau­ren would be avail­able in the US well be­fore the 2021 read out of this new con­fir­ma­to­ry tri­al. Giv­en the high lev­el of un­cer­tain­ty around nmD­MD in the US we con­tin­ue to as­sign no val­ue in our NPV analy­sis.

“We are pleased with this out­come which took in­to ac­count all avail­able da­ta for Translar­na,” said Stu­art W. Peltz, PhD, Chief Ex­ec­u­tive Of­fi­cer, PTC Ther­a­peu­tics, Inc. “This de­ci­sion re­flects the ben­e­fit that Translar­na is hav­ing for pa­tients suf­fer­ing from non­sense mu­ta­tion Duchenne mus­cu­lar dy­s­tro­phy.”

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.