Thumbs Up/Thumbs Down: The FDA dam­aged it­self bad­ly with the Sarep­ta OK

End­points as­sess­es the big bio­phar­ma R&D sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

  Califf’s de­ci­sion to bow to Janet Wood­cock on Sarep­ta hurts the en­tire biotech in­dus­try.

Janet Wood­cock’s de­ci­sion to over­ride the caus­tic in­ter­nal crit­i­cism of Sarep­ta’s weak and at times mis­lead­ing case for its Duchenne drug eteplirsen has dam­aged the FDA in ways that will leave a mark for years to come. Over­rul­ing her col­leagues – in­clud­ing se­nior FDA of­fi­cials — to pro­vide an ac­cel­er­at­ed ap­proval this week for this ap­pli­ca­tion sends a clear mes­sage to biotech: If you can muster enough pub­lic sup­port for a cam­paign – which in­cludes an er­ro­neous study for bait — you should pro­ceed full steam ahead. To his cred­it, FDA com­mis­sion­er Robert Califf post­ed his own re­view of the case as well as the heat­ed ob­jec­tions of two top reg­u­la­tors, in­clud­ing the cur­rent act­ing chief sci­en­tist. Not to his cred­it, Califf chose to stand down in “def­er­ence” to Wood­cock’s in­tran­si­gence, but not be­fore chid­ing Sarep­ta for pro­mot­ing a study that raised false hopes in the pa­tient com­mu­ni­ty, which swal­lowed it all hook, line and sinker. He was al­so none too hap­py that Sarep­ta stayed fo­cused on its lob­by­ing cam­paign rather than the clin­i­cal work that need­ed to be done. I sin­cere­ly hope that eteplirsen does every­thing that ad­vo­cates de­vout­ly be­lieve it does, with­out pre­sent­ing the dan­gers cit­ed for a “sci­en­tif­i­cal­ly el­e­gant place­bo.” And let’s hope pa­tients aren’t left to cov­er any of the $300,000 an­nu­al cost. Pay­ers, and ul­ti­mate­ly so­ci­ety, can and should do that now. And what hap­pens next? Does the FDA now slap back the next com­pa­ny to use sim­i­lar tac­tics, to try and reestab­lish old bound­aries? Or does it low­er its stan­dards per­ma­nent­ly, en­cour­ag­ing more large demon­stra­tions and dodgy da­ta to back weak ap­pli­ca­tions? That’s a po­si­tion the FDA should nev­er have been put in. And that is on Janet Wood­cock and Robert Califf.

 The im­muno-on­col­o­gy rev­o­lu­tion will cause lots of pain, but it will be worth it.

We are liv­ing at the start of the im­muno-on­col­o­gy rev­o­lu­tion, and it’s been a re­al priv­i­lege cov­er­ing the ex­plo­sion of deals and col­lab­o­ra­tions that are be­ing struck now. Every day it seems brings one, or two, new I/O pacts. This week there was iOmx’s start­up round to fund the hunt for new check­point in­hibitors. Genen­tech struck a po­ten­tial­ly ground­break­ing part­ner­ship with BioN­Tech on us­ing mes­sen­ger RNA to de­vel­op per­son­al­ized can­cer vac­cines. With the three lead­ing check­points on the mar­ket and more com­ing, the po­ten­tial to part­ner on new com­bos is dri­ving bil­lions of dol­lars in fresh in­vest­ments. True, any­thing this hot will breed an ex­cess of al­ter­na­tives. In­vestors will get burned. The shake­out will have to oc­cur even­tu­al­ly and not every­one will suc­ceed. Many, most, will fail or fall by the way­side. But it’s been fun watch­ing the spot­light grow big­ger and more in­tense every week. I was chat­ting with an­oth­er CEO at a US start­up in this field ear­li­er in the week, and he be­lieves that we’re still just at the be­gin­ning of this i/o era. This will go on for an­oth­er 20 years, he says. And he’s like­ly right. What a great time to be in biotech.

 Set­backs hap­pen. It’s time for the big com­pa­nies to be more trans­par­ent about them.

Why do so many Big Phar­ma com­pa­nies have such a hard time talk­ing about a set­back? In re­cent times Am­gen nev­er missed a beat when it came to tout­ing their kid­ney dis­ease drug Parsabiv (etel­cal­ce­tide), and then the FDA un­ex­pect­ed­ly re­ject­ed it and the big biotech shut down, adopt­ing the tur­tle de­fense in re­fus­ing to even at­tempt to ex­plain it. No­var­tis likes to slip bad news in­to quar­ter­ly re­ports, in­clud­ing a line item on the re­cent re­jec­tion of one of its biosim­i­lars. And this week J&J could on­ly ac­knowl­edge, af­ter I con­tact­ed them, that the FDA had placed a hold on one of its can­cer drugs. They de­clined to ex­plain the se­ri­ous ad­verse events cit­ed on clin­i­cal­tri­ that forced reg­u­la­tors’ hands. I’ve said it be­fore and no doubt will have plen­ty of oc­ca­sions to do it again, but com­pa­nies run­ning drug stud­ies have a re­spon­si­bil­i­ty to ex­plain these sna­fus in some de­tail. They owe it to their in­vestors, the peo­ple who sign up for all of their clin­i­cal stud­ies and the sci­en­tists try­ing to make safe progress in the same field. There’s far too lit­tle trans­paren­cy in drug de­vel­op­ment. At a time when the in­dus­try is look­ing for some un­der­stand­ing from the pub­lic on the cost of in­no­va­tion, big play­ers should al­so rec­og­nize that they can be lead­ers in own­ing up to what can go wrong in de­vel­op­ing a new drug. It won’t hurt as much as they ev­i­dent­ly think it will.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

Bio­gen touts new ev­i­dence from the gene ther­a­py com­pa­ny it wa­gered $800M on

A year ago, Biogen made a big bet on a small gene therapy company. Now they have new evidence one of their therapies could work.

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Anthony Fauci (AP Images)

UP­DAT­ED: NIH-part­nered Mod­er­na ships off its PhI-ready coro­n­avirus vac­cine can­di­date to a sea of un­cer­tain­ty

Off it goes.

Moderna has shipped the first batch of its mRNA vaccine against SARS-CoV-2 from its manufacturing facility in Norwood, Massachusetts, to the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland, for a pioneering Phase I study.

It’s a hectic race against time. In the 42 days since Moderna selected the sequence they would use to develop their vaccine — a record time, no less — the number of confirmed cases around the world has surged astronomically from a few dozen to over 80,000, per WHO and Johns Hopkins estimates.

The candidate that they came up with, mRNA-1273, encodes for a prefusion stabilized form of the spike protein, which gives the virus its crown shape and plays a key role in transmission. The Coalition for Epidemic Preparedness Innovations, the Oslo-based group better known as CEPI, funded the manufacture of this batch.

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In fi­nal re­port, ICER ap­pears to have a change of heart on new acute mi­graine ther­a­pies

ICER appears to have reversed course on the fresh crop of acute migraine therapies.

The cost-effectiveness watchdog in November issued a draft report suggesting that existing generic medicines are more effective and cheaper than Allergan’s December-approved CGRP ubrogepant, Biohaven rival molecule, rimegepant (which is under FDA review), and Lilly’s October-sanctioned lasmiditan, which binds to 5-HT1F receptors.

Bi­cy­cle Ther­a­peu­tics takes Roche's Genen­tech on an up to $2B im­muno-on­col­o­gy ride

Bicycle Therapeutics — which is developing a new class of chemically synthesized drugs designed to be pharmacologically as active as biologics, yet manufactured as small molecules —  has scored another big partner: Roche’s Genentech.

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When drug val­ue as­sess­ment meets re­al-world ev­i­dence: ICER en­lists Ae­tion in pric­ing eval­u­a­tion

In a union of two of the hottest trends in the US biopharma world, ICER is teaming up with a high-profile company to integrate real-world evidence in their assessment of treatment value.

The drug pricing watchdog — formally the Institute for Clinical and Economic Review — said it will utilize Aetion’s evidence platform in “select upcoming assessments” and their new 24-month re-evaluations of drugs granted accelerated approval by the FDA.

Anthony Fauci, AP Images

First US Covid-19 tri­als set to get un­der­way in Ne­bras­ka and Wash­ing­ton, backed by NIH

The first US clinical trials on the novel coronavirus are scheduled to get underway next month at the University of Nebraska Medical Center, where American passengers were taken after being evacuated from the Diamond Princess cruise ship, and at the Kaiser Permanente Washington Health Research Institute. Both trials are sponsored by the NIH’s National Institute for Allergy and Infectious Diseases, which has led the US’s medical response to the outbreak.

UP­DAT­ED: NGM Bio takes leap for­ward in crowd­ed NASH field

South San Francisco-based NGM Bio may have underwhelmed with its interim analysis of a key cohort from a mid-stage NASH study last fall — but stellar topline data unveiled on Monday showed the compound induced significant signs of antifibrotic activity, NASH resolution and liver fat reduction, sending the company’s stock soaring.

There are an estimated 50+ companies focused on developing drugs for non-alcoholic steatohepatitis, or NASH, a common liver disease that has long flummoxed researchers. The first wave of NASH drug developers struggled with efficacy as well as safety — and companies big and small have crashed and burned.

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