The com­pas­sion­ate use poll: A lop­sided vote fa­vors post­ing poli­cies, with plen­ty of fret­ting

We ran a snap poll yes­ter­day on com­pas­sion­ate use poli­cies, and there’s clear­ly plen­ty of pos­i­tive sen­ti­ment among our read­ers back­ing some sort of eas­i­ly ac­ces­si­ble, on­line state­ment ex­plain­ing each biotech’s poli­cies on ex­pand­ed ac­cess to ex­per­i­men­tal drugs.

The vote land­ed 70% for, 30% against post­ing a com­pas­sion­ate use pol­i­cy on­line among the 149 votes we col­lect­ed in the last 24 hours. But maybe that has a lot to do with the fact that a very sim­i­lar sized group al­so said that such pol­i­cy state­ments don’t ap­ply to their com­pa­nies. Among the 49 who said it did ap­ply, 33 said their com­pa­nies didn’t post a pol­i­cy, against 16 who said they did.

That two-to-one ra­tio against, though, is still a far cry from the neg­li­gi­ble 4% of small biotechs tracked by Avalere Health which post­ed such poli­cies.

No, said one poll re­spon­dent: “The de­ci­sion to try an ex­per­i­men­tal med­i­cine is very com­plex (sci­en­tif­i­cal­ly, eth­i­cal­ly and reg­u­la­to­ri­ly) and mak­ing it ‘easy’ is go­ing to open a Pan­do­ra’s box that we’re not ready for.”

The ‘no’ camp al­so ex­pressed con­cerns that a sin­gle ad­verse event from com­pas­sion­ate use could de­rail a pro­gram, as well as wor­ries about the cost of pro­vid­ing drugs for small com­pa­nies years away from mak­ing any mon­ey.

A lot of the com­ments for the “yes” vote echoed this sen­ti­ment:

“Post­ing com­pas­sion­ate use poli­cies on your web­site does not promise that your case will be ac­cept­ed, but at least it pro­vides trans­paren­cy on their pol­i­cy and of­fers the po­ten­tial to be treat­ed, ” said one.

Yes, but. “Com­pas­sion­ate Use should on­ly be of­fered for drugs suc­cess­ful­ly shown to be both ef­fi­ca­cious and safe in Phase IIb/III tri­als. Pri­or to that, it is not ad­vis­able. Re­mem­ber “first do no harm,” says an­oth­er.

In­deed, one read­er al­so steered me to Bio­Marin’s pol­i­cy, say­ing that it’s com­mon for such state­ments to re­strict any com­pas­sion­ate use to drugs which have fin­ished Phase III, when you have sol­id ef­fi­ca­cy and safe­ty da­ta, but be­fore the FDA has act­ed.

Art Ca­plan

That’s a nar­row win­dow. NYU’s not­ed bioethi­cist Art Ca­plan tells me he’s seen com­pas­sion­ate use re­quests for drugs in every­thing from an­i­mal-stage test­ing on up. Some small com­pa­nies will start en­ter­tain­ing re­quests as ear­ly as Phase I. And no biotech should ex­pect the pleas to be re­strict­ed for use in the des­ig­nat­ed dis­eases be­ing stud­ied. It could be for a dif­fer­ent dose as well as a sep­a­rate in­di­ca­tion, which is what caught Chimerix in the mid­dle of an on­line mob as the par­ents of one young boy sought ac­cess for its lead ther­a­py through a pub­lic lob­by­ing ef­fort that sud­den­ly went vi­ral.

Iron­i­cal­ly, I checked out Chimerix’s web site and couldn’t find any men­tion of a com­pas­sion­ate use pol­i­cy. The biotech, which has ex­pe­ri­enced a cou­ple of set­backs with the pro­gram, de­clined com­ment.

Any time you get press cov­er­age of a new drug, Ca­plan tells me, you raise the chances of com­pas­sion­ate use re­quests. And Ca­plan doesn’t sign on with the post-Phase III on­ly camp. The im­por­tant thing is mak­ing sure the drug has a well-es­tab­lished safe­ty pro­file and at least a hint of ef­fi­ca­cy. For Ca­plan, Phase IIb is where you hit the sweet spot for con­sid­er­ing these re­quests.

And he adds that he wasn’t in the least bit sur­prised to hear that few biotechs have clear­ly enun­ci­at­ed poli­cies spelled out on­line.

“If you have a com­pa­ny with one CEO and 20 peo­ple chained to a bench, web­site de­vel­op­ment is not a top pri­or­i­ty,” says Ca­plan. But that doesn’t mean they shouldn’t do it.

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

A new study points to $6.5B in pub­lic sup­port build­ing the sci­en­tif­ic foun­da­tion of Gilead­'s remde­sivir. Should that be re­flect­ed in the price?

By drug R&D standards, Gilead’s move to repurpose remdesivir for Covid-19 and grab an emergency use authorization was a remarkably easy, low-cost layup that required modest efficacy and a clean safety profile from just a small group of patients.

The drug OK also arrived after Gilead had paid much of the freight on getting it positioned to move fast.

In a study by Fred Ledley, director of the Center for Integration of Science and Industry at Bentley University in Waltham, MA, researchers concluded that the NIH had invested only $46.5 million in the research devoted to the drug ahead of the pandemic, a small sum compared to the more than $1 billion Gilead expected to spend getting it out this year, all on top of what it had already cost in R&D expenses.

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FDA bars the door — for now — against Mer­ck’s star can­cer drug af­ter Roche beat them to the punch

Merck has been handed a rare setback at the FDA.

After filing for the accelerated approval of a combination of their star PD-1 drug Keytruda with Eisai’s Lenvima as a first-line treatment for unresectable hepatocellular carcinoma, the FDA nixed the move, handing out a CRL because Roche beat them to the punch on the same indication by a matter of weeks.

According to Merck:

Ahead of the Prescription Drug User Fee Act action dates of Merck’s and Eisai’s applications, another combination therapy was approved based on a randomized, controlled trial that demonstrated overall survival. Consequently, the CRL stated that Merck’s and Eisai’s applications do not provide evidence that Keytruda in combination with Lenvima represents a meaningful advantage over available therapies for the treatment of unresectable or metastatic HCC with no prior systemic therapy for advanced disease. Since the applications for KEYNOTE-524/Study 116 no longer meet the criteria for accelerated approval, both companies plan to work with the FDA to take appropriate next steps, which include conducting a well-controlled clinical trial that demonstrates substantial evidence of effectiveness and the clinical benefit of the combination.

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Covid-19 roundup: Mod­er­na sticks to Ju­ly for its Phase III as ru­mors swirl; Fol­low­ing US lead, EU buys up Covid-19 treat­ments

The Phase III might be delayed from its original early July goal, but Moderna says it will still kick off the pivotal study for what could ultimately be the first Covid-19 vaccine before the end of the month.

A day after Reuters reported that squabbling between the Cambridge biotech and government regulators had held up the trial by about two weeks, Moderna released a statement saying that they had completed enrollment of their 650-person Phase II trial and were on track to begin Phase III by the end of the month. The protocol for that study, which is meant to prove whether or not the vaccine can prevent people from becoming sick, has been finalized, they said.

Stephen Hahn, AP

Trump and Navar­ro press again for hy­drox­y­chloro­quine. Can the FDA stay in­de­pen­dent?

Tuesday morning, economist and Trump advisor Peter Navarro walked onto the White House driveway and promptly brought a political cloud back onto the FDA.

Speaking to a White House pool reporter, Navarro said that four Detroit doctors were, based on a single disputed study, filing for the FDA to again issue an emergency authorization for hydroxychloroquine, the anti-malarial pill that President Trump hyped for months as a Covid-19 treatment over the objections of his own scientists. Then, while avoiding directly calling for the FDA to OK the drug, blasted the agency. He said its decision to pull an earlier authorization “was based on bad science” and “had a tremendously negative effect” on doctors and patients.

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Noubar Afeyan, Flagship CEO and Tessera chairman (Victor Boyko/Getty Images)

Flag­ship ex­ecs take a les­son from na­ture to mas­ter ‘gene writ­ing,’ launch­ing a star-stud­ded biotech with big am­bi­tions to cure dis­ease

Flagship Pioneering has opened up its deep pockets to fund a biotech upstart out to revolutionize the whole gene therapy/gene editing field — before gene editing has even made it to the market. And they’ve surrounded themselves with some marquee scientists and execs who have crowded around to help shepherd the technology ahead.

The lead player here is Flagship general partner Geoff von Maltzahn, an MIT-trained synthetic biologist who set out in 2018 to do CRISPR — a widely used gene editing tool — and other rival technologies one or two better. Von Maltzahn has been working with Sana co-founder Jake Rubens, another synthetic biology player out of MIT who he describes as his “superstar,” who’s taken the CSO role.

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Covid-19 roundup: Squab­bles with gov­ern­ment de­lay Mod­er­na’s PhI­II — re­ports; No­vavax se­cures largest Warp Speed deal yet: $1.6B

A much-anticipated Phase III trial for Moderna’s Covid-19 vaccine is being held up as the company delayed submitting trial protocols and sparred with government scientists on how to run the study and even what the benchmark for success should be, Reuters reported.

Moderna, the first US company to put their vaccine into human testing, was supposed to enter a 30,000-person study this month in partnership with the NIH to determine whether it can prevent infection. STAT reported last week that the trial was facing delays over the protocol, but that a July start was still possible. Neither the NIH nor Moderna ever disclosed a specific date the trial should start, but Reuters reported that the agency had hoped to begin on July 10.

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