John Theo, Amarin

The FDA de­cides to hold an ex­pert pan­el re­view of Vas­cepa af­ter all — and Amarin shares tank on un­ex­pect­ed de­lay

Mark your cal­en­dars for No­vem­ber 14.

Af­ter slow­ly and cau­tious­ly rais­ing its hopes to the point where com­pa­ny ex­ecs be­lieved an Ad­Com on its sup­ple­men­tal la­bel for Vas­cepa was “un­like­ly,” the biotech put out word late Thurs­day that the FDA has pen­cilled in an ex­pert pan­el meet­ing for them in mid-No­vem­ber.

And in­vestors — who were grow­ing in­creas­ing­ly warm to the idea that the com­pa­ny could be on a cake­walk to a cru­cial, and po­ten­tial­ly block­buster, ap­proval on the Sep­tem­ber 28 PDU­FA date — pan­icked. Shares $AM­RN plunged 22% on the news.

That PDU­FA date next month?

You can prob­a­bly scrap that as well.

Amarin does not ex­pect the FDA to take ac­tion on the sN­DA by the pre­vi­ous­ly an­nounced Sep­tem­ber 28, 2019 Pre­scrip­tion Drug User Fee Act (PDU­FA) goal date. Amarin did not re­ceive no­tice from the FDA of a PDU­FA date ex­ten­sion. In light of the ten­ta­tive Ad­Com date, Amarin an­tic­i­pates that the PDU­FA date will be ex­tend­ed, as­sum­ing a typ­i­cal three-month ex­ten­sion, to a date in late De­cem­ber 2019. If so, this an­tic­i­pat­ed re­vised PDU­FA date tim­ing would off­set three of the four months that were ex­pect­ed to be gained from FDA’s ear­li­er de­ter­mi­na­tion to con­duct a pri­or­i­ty re­view of the RE­DUCE-IT sN­DA.

An Ad­Com on Vas­cepa isn’t nec­es­sar­i­ly the end of the world, by any means, for Amarin boost­ers. But it will be an op­por­tu­ni­ty for FDA in­ves­ti­ga­tors to air every ques­tion they have about the da­ta and in­vite their ex­perts to scru­ti­nize any weak point in their ap­pli­ca­tion.

As it stands, the com­pa­ny has had every op­por­tu­ni­ty to boast of the sig­nif­i­cant car­dio ben­e­fits their in­ves­ti­ga­tors re­port­ed on RE­DUCE-IT, and any word to the con­trary could ding its prospects among pay­ers. On the oth­er hand, a clear vote in its fa­vor would mark a big en­dorse­ment for the drug as Amarin beefs up its staff in prepa­ra­tion for a great­ly ex­pand­ed mar­ket­ing cam­paign.

FDA chief Stephen Hahn on Capitol Hill earlier this week (Getty Images)

As FDA’s work­load buck­les un­der the strain, Trump again ac­cus­es the agency of a po­lit­i­cal hit job

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

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

President Trump walks past HHS secretary Alex Azar (Getty Images)

Azar falls in line un­der Trump again. Ex­perts say he's re­in­forc­ing a dark sig­nal sent to the FDA

In the latest incident where Alex Azar has steadfastly taken the side of President Donald Trump over that of the FDA, the HHS secretary was noncommittal this morning when asked if he supports the attempt by his subordinates at the FDA to strengthen guidelines for a vaccine EUA.

Appearing on NBC’s Today Show, the HHS secretary muddied the waters, stating that the guidance that matters is the one that is “actually already out there.”

CDC’s Robert Redfield, NIAID’s Anthony Fauci, Admiral Brett Giroir at HHS, and FDA’s Stephen Hahn prepare to testify at a House hearing on June 23 (Getty)

'Ex­treme­ly po­lit­i­cal' — Trump neuters FDA's at­tempt to strength­en vac­cine EUA

Stephen Hahn went before a Senate committee Wednesday and declared he’s fighting. “Every one of the decisions we have reached has been made by career FDA scientists based on science and data, not politics,” he exclaimed, adding that “FDA will not permit any pressure from anyone to change that. I will fight for science.”

A few hours later, he was undermined by President Donald Trump when a reporter asked if he was okay with stricter vaccine guidelines that the FDA was said to be cooking up. “That has to be approved by the White House. We may or may not approve it. That sounds like a political move,” he decided.

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.

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Vas Narasimhan (AP Images)

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.

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President Donald Trump and FDA Commissioner Stephen Hahn (AP Images)

FDA is­sues fi­nal rule al­low­ing im­por­ta­tion of drugs from Cana­da — but al­so keeps the pow­er to re­voke it

Just over a month away from the presidential election, the FDA has issued a final regulation fulfilling President Trump’s promise to let states import certain prescription drugs from Canada.

On Thursday, Trump told a crowd in North Carolina that the new rule goes into effect “today.” But the published regulation states that it won’t take effect for 60 days. And even then, it could be a while before cheaper drugs make it across the border.

The win­dow is wide open as four more biotechs join the go-go IPO class of 2020

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

J&J of­fers PhI/IIa da­ta show­ing its sin­gle-dose vac­cine can stir up suf­fi­cient im­mune re­sponse

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.