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.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Jackie Fouse, Agios CEO

Agios scores its sec­ond pos­i­tive round of da­ta for its lead pipeline drug — but that won't an­swer the stub­born ques­tions that sur­round this pro­gram

Agios $AGIO bet the farm on its PKR activator drug mitapivat when it recently decided to sell off its pioneering cancer drug Tibsovo and go back to being a development-stage company — for what CEO Jackie Fouse hoped would be a short stretch before they got back into commercialization.

On Tuesday evening, the bellwether biotech flashed more positive topline data — this time from a small group of patients in a single-arm study. And the executive team plans to package this with its earlier positive results from a controlled study to make its case for a quick OK.

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Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

Af­ter 3 deaths rock the field, gene ther­a­py re­searchers con­tem­plate AAV's fu­ture

Nicole Paulk was scrolling through her phone in bed early one morning in June when an email from a colleague jolted her awake. It was an article: Two patients in an Audentes gene therapy trial had died, grinding the study to a halt.

Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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George Yancopoulos (L) and Len Schleifer (Regeneron)

Re­gen­eron touts pos­i­tive pre­lim­i­nary im­pact of its Covid an­ti­body cock­tail, pre­vent­ing symp­to­matic in­fec­tions in high-risk group

Regeneron flipped its cards on an interim analysis of the data being collected for its Covid-19 antibody cocktail used as a safeguard against exposure to the virus. And the results are distinctly positive.

The big biotech reported Tuesday morning that their casirivimab and imdevimab combo prevented any symptomatic infections from occurring in a group of 186 people exposed to the virus through a family connection, while the placebo arm saw 8 of 223 people experience symptomatic infection. Symptomatic combined with asymptomatic infections occurred in 23 people among the 223 placebo patients compared to 10 of the 186 subjects in the cocktail arm.

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Vir's CMO says he's sur­prised that a low dose of their he­pati­tis B drug ap­pears promis­ing in ear­ly slice of da­ta — shares soar

Initial topline data from a Phase I study of a new therapeutic for chronic hepatitis B virus was so promising that it surprised even the CMO of the company that produces it.

Vir Biotechnology on Tuesday announced that its VIR-3434 molecule reduced the level of virus surface antigens present in a blinded patient cohort after eight days of the trial with just a single 6 mg dose. Six of the eight patients in the cohort were given the molecule, and the other two a placebo—all six who received the molecule saw a mean antigen reduction of 1.3 log10 IU/mL, Vir said.

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David Marek, Myovant

My­ovant beefs up da­ta pack­age in NDA #3, boost­ing its case for longterm dos­ing of Pfiz­er-part­nered re­l­u­golix

When Pfizer handed over $650 million in cash to partner on Myovant’s relugolix, the pharma giant made clear that the deal — valued at $4.2 billion total — was just as much about the approved indication of prostate cancer as the two women’s health conditions the drug could treat.

A month later, the two companies are offering another glimpse of the therapy’s longterm potential in endometriosis.

Look­ing to win over some skep­ti­cal an­a­lysts, Rhythm beats the drum on in­ter­im da­ta in PhII bas­ket study for ad­di­tion­al in­di­ca­tions

Rhythm Pharmaceuticals has been working toward expanding the FDA approval they received just two months ago for three rare genetic disorders that result in obesity. In December, their Phase III cut of data saw mixed reactions from analysts, but new interim results released Tuesday may provide more excitement.

In an ongoing Phase II study for setmelanotide across individuals with one of three distinct rare genetic diseases of obesity, 65 patients had reached the Dec. 17 cutoff date for evaluation. Among patients who met the primary endpoint of at least 5% weight loss over three months, Rhythm saw an average reduction of no less than 7.1% in any of the groups.

Ron Cooper, Albireo CEO

Al­bireo just ad­vanced down to the 10-yard line at the FDA. And Ron Coop­er’s team is get­ting prepped for the next big play

When Albireo Pharma’s board $ALBO moved to bring in Ron Cooper as the CEO more than 5 years ago, the development-stage company went with an experienced commercial player who had a big-time position on his resume after running Bristol Myers’ commercial ops in Europe.

Now, after successfully navigating a pivotal study, putting them in a foot race with a rival toward an FDA OK, Cooper is getting a boost from regulators on the last drive back to an arena he understands completely.

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