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 clin­i­cal­tri­als.gov 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.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

Overnight for­tunes are be­ing made in biotech these days — and it's both en­cour­ag­ing and more than a lit­tle bit scary

Just to complete the last leg of a running story I’ve been tracking for a few weeks, Olema $OLMA has come through its IPO from the Thursday night pricing at $19 a share with a market cap just north of $2 billion.

That leaves newly-named CEO Sean Bohen holding a batch of 1,110,896 shares with a strike price of $4.82. As of Tuesday morning, the stock is now trading at $53.40, giving him a portfolio value of $53.4 million. Not bad for someone who was hired in September.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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News brief­ing: Gilead part­ner Gala­pa­gos sells off CRO for $37M; Polyphor bags $3.3M from CF Foun­da­tion

Close Gilead ally Galapagos is selling off one of its contract research organizations to a Polish pharma company.

Galapagos has agreed to sell 100% of the outstanding shares in the CRO Fidelta to Selvita, in a deal worth roughly $37 million expected to close in the first week of January. The acquisition is expected to nearly double Selvita’s revenues, the company says, as well as expand its drug discovery efforts.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Gen­mab ax­es an ADC de­vel­op­ment pro­gram af­ter the da­ta fail to im­press

Genmab $GMAB has opted to ax one of its antibody-drug conjugates after watching it flop in the clinic.

The Danish biotech reported Tuesday that it decided to kill their program for enapotamab vedotin after the data gathered from expansion cohorts failed to measure up. According to the company:

While enapotamab vedotin has shown some evidence of clinical activity, this was not optimized by different dose schedules and/or predictive biomarkers. Accordingly, the data from the expansion cohorts did not meet Genmab’s stringent criteria for proof-of-concept.

Michelle Longmire, Medable CEO (Jeff Rumans)

Med­able gets $91M for vir­tu­al clin­i­cal tri­als, bring­ing to­tal raise to $136M

As biotechs look to get clinical studies back on track amid the pandemic, Medable returned to the venture well for the second time this year, bagging a $91 million Series C to build out its virtual trial platform.

The software provider recently launched three new apps for decentralizing clinical trials, and saw a 500% revenue spike this year. And it isn’t alone. Back in August, Science 37 secured a $40 million round for its virtual trial tech, with support from Novartis, Sanofi Ventures and Amgen. Patients and researchers are taking a liking to the online approach, suggesting regulators could allow it to become a new normal even after the pandemic is over.

PhRMA sues Trump gov­ern­ment over drug im­por­ta­tion rule — days be­fore it's set to be ef­fec­tive

Ever since President Donald Trump floated the idea of using state-sponsored importation to lower drug prices, PhRMA has made its opposition abundant. Not only is the proposal dangerous and futile,  but the trade group has also argued that it may even be illegal.

Now that the FDA has issued its final rule permitting states to bring certain drugs from Canada, PhRMA is taking the government to court — just a few days before the rule is slated to take effect.