No, Pres­i­dent Trump, bio­phar­ma does not want you to dereg­u­late drug de­vel­op­ment

Nobody wants rad­i­cal changes at the FDA, ex­cept per­haps Pres­i­dent Trump and a few close as­so­ciates.

We’ve been track­ing the in­dus­try’s clear re­luc­tance to dis­card the FDA gold stan­dard in drug de­vel­op­ment. In our most re­cent sur­vey yes­ter­day, a plu­ral­i­ty of biotech ex­ecs vot­ed against sig­nif­i­cant changes at the agency, say­ing that re­forms over the past few years has al­ready sped up the process. And many of the ex­ecs that would like to see con­tin­ued change were clear­ly more in­ter­est­ed in specif­i­cal­ly tai­lored up­grades, not a whole­sale over­haul.

To­day, Reuters added a few more bio­phar­ma voic­es to that mix, most no­tably to me Roger Perl­mut­ter at Mer­ck.

Roger M. Perl­mut­ter, Mer­ck

“Peo­ple of­ten ar­gue that the FDA is too re­stric­tive,” Perl­mut­ter told the wire ser­vice. “We have the sense that the bal­ance is pret­ty right … you have to have a well-char­ac­ter­ized risk/ben­e­fit pro­file.”

Big Phar­ma’s big goal at the new ad­min­is­tra­tion has been tax re­form, and most of the CEOs have been no­to­ri­ous­ly qui­et in the face of Trump’s in­sis­tence that vast seg­ments of the reg­u­la­tions around drug de­vel­op­ment need to be in­cin­er­at­ed. So Perl­mut­ter’s po­si­tion here speaks loud­ly to the in­dus­try’s stand on dereg­u­la­tion.

Your run of the mill biotech fo­cused al­most ex­clu­sive­ly on pipeline de­vel­op­ment will have no prob­lem em­brac­ing Perl­mut­ter’s po­si­tion. And in­ter­est­ing­ly, even BIO is will­ing to sig­nal now — qui­et­ly — that it’s ad­vis­ing Trump against a rad­i­cal change in strat­e­gy at the FDA.

That’s why there’s been so much ac­tive sup­port for Scott Got­tlieb as the new com­mis­sion­er. Got­tlieb, a physi­cian, is seen as some­one who can help con­tin­ue stream­lin­ing the FDA with­out up­root­ing stan­dards that are es­sen­tial to do­ing drug R&D with in­tegri­ty.

Richard Paz­dur

A few years ago, I was im­pressed to hear Richard Paz­dur stand up at AS­CO and in no un­cer­tain terms em­brace the break­through ther­a­py des­ig­na­tion. Can any­one de­ny that his on­col­o­gy group has fol­lowed through, com­plete­ly over­haul­ing time­lines for new can­cer drugs?

Dif­fer­ent dis­eases call for dif­fer­ent stan­dards. Di­a­betes and heart dis­ease are not can­cer, and safe­ty con­cerns for can­cer drugs ini­tial­ly treat­ed in dy­ing pa­tients aren’t the same as safe­ty con­cerns for drugs de­signed to pre­vent an­oth­er heart at­tack or live with a chron­ic dis­ease. Still, the rest of the FDA can do more to fol­low Paz­dur’s lead.

Change is need­ed at the FDA. Just not the kind of bomb throw­ing that has been so loud­ly voiced by Trump. We hope he gets the mes­sage, soon.

George Yancopoulos (Regeneron)

Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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No­var­tis los­es biosim­i­lar ap­peal as court up­holds a 31-year mo­nop­oly by Am­gen's En­brel

A new court ruling has strengthened Amgen’s grip on the IP estate around Enbrel, keeping biosimilars of the autoimmune and inflammatory drug at bay until 2029.

Novartis, the patent challenger, isn’t throwing in the towel yet. In a statement noting the failed appeal, its generics division Sandoz noted its reviewing options, “including potential appeal to US Supreme Court.”

It’s been almost four years since the FDA approved Erelzi, Sandoz’s copycat version of Enbrel. While sales of the Pfizer-partnered drug in the US — the market Amgen is in charge of — have dipped slightly during that time, it remains a solid megablockbuster with 2019 revenue slightly above $5 billion.

Douglas Love, Annexon CEO (Annexon)

IPO bound? A Bay Area biotech grabs a mega-round on the road to a piv­otal neu­rode­gen­er­a­tion pro­gram

South San Francisco-based Annexon has added $100 million to its cash reserves, along with a new roster of marquee investors backing their play on the classical complement pathway involved in neurodegeneration. And that may well fit the profile for an IPO — though right now everything seems to be working on that score.

Eighteen months after Bain and their syndicate partners put up $75 million to fuel clinical work, Annexon is back at the trough. And this time they’re adding Redmile Group for the lead role, with supporting investments from these new arrivals: BlackRock, Deerfield Management Company, Eventide Asset Management, Farallon Capital Management, Janus Henderson Investors and Logos Capital.

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Joseph Kim, Inovio CEO (Andrew Harnik, AP Images)

Pos­i­tive Covid-19 vac­cine da­ta? New mouse study? OWS in­clu­sion? Yep, but some­how, the usu­al tid­bits from In­ovio back­fire

You don’t go more than 40 years in biotech without ever getting a product to market unless you can learn the art of writing a promotional press release. And Inovio captures the prize in baiting the hook.

Tuesday morning Inovio, which has been struggling to get its Covid-19 vaccine lined up for mass manufacturing, put out a release that touched on virtually every hot button in pandemic PR.

There was, first and foremost, an interim snapshot of efficacy from their Phase I program for INO-4800.

FDA ap­provals roundup: Zo­genix's Fin­tepla, Ul­tragenyx's Do­jolvi, and Genen­tech's Ph­es­go

A weekly update on new drug approvals and indications from the FDA:

New approvals

Fintepla gets the go-ahead for Dravet syndrome

Zogenix’s Fintepla (fenfluramine) has been approved for the treatment of seizures associated with Dravet syndrome in patients aged 2 years or older. The syndrome is a rare, life-threatening form of epilepsy.

The approval was based on findings from two clinical studies in 202 participants aged between 2 and 18 years, in which the change from baseline in frequency of convulsive seizures was measured. In both studies, children who received Fintepla had significantly greater reductions in the frequency of convulsive seizures, compared with those receiving placebo. The reductions were seen within 3-4 weeks of therapy initiation and remained generally consistent during the treatment periods of 14-15 weeks.

Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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