Am­i­cus chief John Crow­ley is snubbed at the FDA on his lat­est pitch for quick drug re­view. He'll be back, but an­a­lyst sees big de­lay

John Crow­ley

Click on the im­age to see the full-sized ver­sion

Am­i­cus CEO John Crow­ley’s lat­est pitch to put a new drug on a path­way to ac­cel­er­at­ed ap­proval has run in­to a hur­dle at the FDA. Crow­ley ac­knowl­edged in a state­ment out ear­ly Mon­day that a meet­ing with reg­u­la­tors failed to gen­er­ate much en­thu­si­asm for a quick re­view of AT-GAA (ATB200/AT2221) based on what they’ve seen so far in a Phase I/II tri­al.

But Crow­ley $FOLD, who re­cent­ly over­came the odds in win­ning a re­ver­sal of the FDA’s de­ci­sion to ini­tial­ly re­ject Galafold, isn’t about to stop now. The com­pa­ny says it plans to keep press­ing, just as it did ear­li­er this sum­mer af­ter the EMA al­so held up a stop sign to a con­di­tion­al ap­proval.

Am­i­cus Ther­a­peu­tics in­tends to con­tin­ue to gen­er­ate da­ta to sup­port fur­ther dis­cus­sions on a po­ten­tial path­way for ac­cel­er­at­ed cp­proval with the FDA in 2019, in­clud­ing:

  • Da­ta from up to 10 ad­di­tion­al ERT-switch pa­tients in a new Co­hort 4 as part of the on­go­ing Phase 1/2 study (da­ta ex­pect­ed in 2019)
  • Pre­sen­ta­tion of longer-term clin­i­cal da­ta out to 18-months for the 19 orig­i­nal Phase 1/2 pa­tients (da­ta ex­pect­ed in 2H 2018)
  • Com­ple­tion of a ret­ro­spec­tive nat­ur­al his­to­ry study in ap­prox­i­mate­ly 100 ERT-treat­ed Pompe pa­tients (da­ta ex­pect­ed in 2H 2018).

That’s the same game plan Am­i­cus out­lined when the EMA ear­li­er closed the door on their quest to gain a con­di­tion­al ap­proval in Eu­rope.

There have been wan­ing ex­pec­ta­tions that the FDA would come through here on lit­tle more than bio­mark­er da­ta. But with the new Trump ad­min­is­tra­tion — rep­re­sent­ed by FDA com­mis­sion­er Scott Got­tlieb — cel­e­brat­ing fast new OKs while ham­mer­ing any prospec­tive price in­creas­es, hope lin­gered. And Crow­ley isn’t known for tak­ing no as an an­swer.

Leerink’s Joseph Schwartz, who led the sell side cheer­lead­ers, has been keep­ing the flame burn­ing. He not­ed re­cent­ly:

Galafold ap­proval could re­flect a more flex­i­ble FDA that is will­ing to ac­cel­er­ate the reg­u­la­to­ry process in the US.

Not this time. Or at least, not yet. And Schwartz this morn­ing says the snub will like­ly lead to a lengthy de­lay for this drug ahead of any ap­proval.

We had pre­vi­ous­ly as­signed 50% prob­a­bil­i­ty of a 2019 mkt. en­try but we are now push­ing out the com­mer­cial launch to 2022E, with a 75% PoS.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.

FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.

Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the biotech.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.

Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.

Scoop: ARCH’s Bob Nelsen is back­ing an mR­NA up­start that promis­es to up­end the en­tire man­u­fac­tur­ing side of the glob­al busi­ness

For the past 2 years, serial entrepreneur Igor Khandros relied on a small network of friends and close insiders to supply the first millions he needed to fund a secretive project to master a new approach to manufacturing mRNA therapies.

Right now, he says, he has a working “GMP-in-a-box” prototype for a new company he’s building — after launching 3 public companies — which plans to spread this contained, precise manufacturing tech around the world with a set of partners. He’s raised $60 million, recruited some prominent experts. And not coincidentally, he’s going semi-public with this just as a small group of pioneers appears to be on the threshold of ushering in the world’s first mRNA vaccines to fight a worldwide pandemic.

Endpoints Premium

Premium subscription required

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

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.