Am­i­cus maps out a 3-year de­lay at the FDA for lead drug, send­ing shares in­to a tail­spin

CEO John Crow­ley

More than a year af­ter Am­i­cus Ther­a­peu­tics was forced to do a sud­den about-face and walk back promis­es of a quick sub­mis­sion for its lead drug mi­gala­s­tat, the biotech has laid out plans for a new tri­al that will be need­ed to win an FDA ap­proval — push­ing back any prospect of an OK for treat­ing Fab­ry dis­ease by at least three more years.

The Cran­bury, NJ-based biotech’s shares $FOLD cratered on the news, drop­ping 28%.

The biotech says it will now need to mount a spe­cial tri­al to ex­plore the drug’s im­pact on GI symp­toms. It will ex­e­cute a 12-month, ran­dom­ized study with 35 pa­tients, with da­ta ar­riv­ing in 2019.

The lat­est set­back for a com­pa­ny that has been on a roller coast­er ride for years dashed hopes of in­vestors that the biotech would be able to line up for an ac­cel­er­at­ed ap­proval of the drug — al­ready OK’d in Eu­rope — with the da­ta al­ready in hand. In­ves­ti­ga­tors had pinned down ev­i­dence of mi­gala­s­tat’s suc­cess in re­duc­ing lev­els of GL-3 that ac­cu­mu­lates in the kid­ney cells of pa­tients with the dis­ease. The drug is de­signed to work among pa­tients with amenable mu­ta­tions cov­er­ing about half of the pop­u­la­tion, rough­ly 25% of whom are in the US.

Gen­zyme and Shire mar­ket in­fused en­zyme re­place­ment ther­a­pies for the con­di­tion, which Am­i­cus wants to re­place with their pill.

Go­ing all the way back to the fall of 2015, Am­i­cus ex­ecs had ini­tial­ly ex­pressed en­thu­si­asm for their sit-down with reg­u­la­tors at the FDA. Then they took a sec­ond look at the min­utes of their meet­ing and rad­i­cal­ly changed course, rais­ing the prospect of a new tri­al.

CEO John Crow­ley put the de­lay in the best light pos­si­ble.

“While we be­lieve that the to­tal­i­ty of the da­ta from our stud­ies with mi­gala­s­tat sup­port the sub­mis­sion of a new drug ap­pli­ca­tion to­day, we ac­knowl­edge the FDA’s po­si­tion that ac­cel­er­at­ed ap­proval based on kid­ney GL-3 re­duc­tion is not cur­rent­ly an op­tion. We have thus de­fined a plan to col­lect ad­di­tion­al GI da­ta to sup­port full ap­proval for mi­gala­s­tat that we be­lieve is fea­si­ble in a rea­son­able amount of time and with a high like­li­hood of suc­cess based on pos­i­tive GI da­ta gen­er­at­ed in our pre­vi­ous Phase III Study 011. FDA has been flex­i­ble in al­low­ing a crossover de­sign and in our use of es­tab­lished GI end­points to mea­sure clin­i­cal ben­e­fit in Fab­ry pa­tients. We are ful­ly com­mit­ted to the ad­di­tion­al work nec­es­sary to move mi­gala­s­tat to­ward ap­proval in the Unit­ed States.”

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.

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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.

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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.

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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.

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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.

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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.

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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.

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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.