Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top ca­reer staff at the FDA has vowed not to let pol­i­tics over­rule sci­ence when look­ing at vac­cine da­ta this fall. But Alex Azar, who hap­pens to be their boss’s boss, ap­par­ent­ly won’t even give them a chance to stand in the way.

In a new mem­o­ran­dum is­sued Tues­day last week, the HHS chief stripped the FDA and oth­er health agen­cies un­der his purview of their rule mak­ing abil­i­ty, as­sert­ing all such pow­er “is re­served to the Sec­re­tary.” Sheila Ka­plan of the New York Times first ob­tained and re­port­ed the de­tails of the Sep­tem­ber 15 bul­letin.

Azar’s sig­na­ture is now re­quired on all new rule­mak­ing, which are typ­i­cal­ly signed by agency lawyers and bu­reau­crats.

“This was sim­ply push­ing a re­set but­ton,” claimed Azar’s top aide Bri­an Har­ri­son to the Times. “This is good gov­er­nance and should have no op­er­a­tional im­pact.”

It’s not im­me­di­ate­ly clear what the im­pact on this or­der will have on the agency’s day-to-day op­er­a­tions — es­pe­cial­ly on rou­tine mat­ters where it has long en­joyed po­lit­i­cal in­de­pen­dence. An agency of­fi­cial not au­tho­rized to speak with the me­dia told the Times they’re still “de­ci­pher­ing what Mr. Azar’s memo would mean for their work.”

The move comes just days af­ter a group of high-pro­file FDA ca­reer ser­vants — in­clud­ing Pe­ter Marks, Jeff Shuren, and Janet Wood­cock — wrote in a USA To­day op-ed that “we will work with agency lead­er­ship to main­tain FDA’s stead­fast com­mit­ment to en­sur­ing our de­ci­sions will con­tin­ue to be guid­ed by the best sci­ence.”

By tak­ing au­thor­i­ty that was once the clear do­main of pro­fes­sion­al staff, the new memo is in line with the cen­tral­iza­tion of pow­er across the fed­er­al gov­ern­ment in the hands of Trump loy­al­ists. And crit­ics note that it blazes a path where the FDA could be side-stepped for an emer­gency use au­tho­riza­tion on an ear­ly coro­n­avirus vac­cine. Er­ic Topol, di­rec­tor at the Scripps Re­search In­sti­tute, spelled out on Twit­ter a po­ten­tial sce­nario with Pfiz­er’s can­di­date:

Low­ell Schiller

The pow­er grab by Azar has ev­i­dent­ly dri­ven at least one es­tab­lished ca­reer ser­vant to the ex­its. On Thurs­day, Politi­co re­port­ed that the FDA’s prin­ci­pal as­so­ciate com­mis­sion­er for pol­i­cy, Low­ell Schiller, left the agency last week. Schiller, a Scott Got­tlieb hire, signed near­ly every FDA no­tice on the fed­er­al reg­is­ter and played a cen­tral role in writ­ing the FDA’s Covid-19 guid­ance. (Up­date: Politi­co now re­ports the de­par­ture was un­re­lat­ed to the memo.)

Af­ter the Times pub­lished its sto­ry, an HHS spokesper­son took to Twit­ter to again re­fute any sug­ges­tion that the FDA’s work would be slowed down but did not ad­dress con­cerns that the new memo could be used to over­rule de­ci­sions by FDA staff. “The memo ma­ligned in this sto­ry is a good gov­er­nance, house­keep­ing ac­tion, which will not slow down the im­por­tant work of the FDA or any HHS di­vi­sion,” the tweet said. Lat­er on Sun­day, the agency put out a FAQ-style re­sponse to news sto­ries on the memo but did not pro­vide the memo’s ac­tu­al text.

The HHS memo is one of the most far-reach­ing ac­tions in a decades-old quest by hard-right Amer­i­can con­ser­v­a­tives to crip­ple the reg­u­la­to­ry pow­ers of pro­fes­sion­als in ex­ec­u­tive-branch agen­cies. De­spite be­ing a con­gres­sion­al­ly del­e­gat­ed au­thor­i­ty, Pres­i­dent Trump and his po­lit­i­cal al­lies rou­tine­ly frame the is­sue of as one of fed­er­al em­ploy­ees act­ing like “un­ac­count­able bu­reau­crats.” Last year, while Trump was sign­ing ex­ec­u­tive or­ders aimed at pair­ing back rule­mak­ing across sev­er­al agen­cies, his pre­pared re­marks stat­ed that “all too of­ten guid­ance doc­u­ments are a back door for reg­u­la­tors to ef­fec­tive­ly change the laws and vast­ly ex­pand their scope and reach.”

Scott Got­tlieb

Scott Got­tlieb, a con­ser­v­a­tive who won wide praise across the po­lit­i­cal spec­trum for his tenure as Trump’s first FDA com­mis­sion­er, fought the en­croach­ment on the agency’s rule­mak­ing abil­i­ty through­out his en­tire tenure. He used the pow­ers as a cud­gel against to­bac­co and va­p­ing in­dus­tries, and ac­cord­ing to the Times re­port, even signed one of the last or­ders on it, hop­ing that his sig­na­ture, rather than a low­er-rank­ing staffer’s, would give his mea­sure stay­ing pow­er on the books.

Azar, who has now as­sert­ed him­self as the ul­ti­mate rule mak­er at the FDA, is Trump’s sec­ond HHS sec­re­tary, tak­ing of­fice in 2018. He was named pres­i­dent of the US di­vi­sion of Eli Lil­ly in 2012 and served on BIO’s board. He de­part­ed Lil­ly in 2017 when CEO David Ricks re­vamped the org chart.

Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining.

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Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

CRISPR Ther­a­peu­tics gets a snap­shot of off-the-shelf CAR-T suc­cess in B-cell ma­lig­nan­cies — marred by the death of a pa­tient

Just days after scientific founder Emmanuelle Charpentier shared the Nobel prize for her work on CRISPR/Cas9, CRISPR Therapeutics $CRSP is showing off a snapshot of success in their early-stage study for an off-the-shelf CAR-T approach to CD19+ B cell malignancies — a snapshot marred by the death of a patient who had been given a high dose of the treatment.

Using their gene editing tech, researchers for CRISPR engineered cells from healthy donors into an attack vehicle aimed at cancer, something that has been achieved with great success using patients’ own cells — the autologous approach. But autologous CAR-T is hampered by the more complex vein-to-vein requirement that delays treatment, and now CRISPR Therapeutics along with other players like Allogene are determined to replace the pioneers with CAR-T 2.0.

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CEO Grace Colón (InCarda)

Look­ing to re­pur­pose an old drug to treat ir­reg­u­lar heart­beats, In­Car­da rais­es $30M in first Se­ries C close

A little less than two years after completing its $42 million Series B round, InCarda has returned to the venture well.

The San Francisco-based biotech announced the first portion of its Series C on Wednesday, pulling in $30 million in new funding. Most of the money will give enough runway for InCarda’s InRhythm program, an inhaled therapeutic aiming to treat sudden episodes of irregular heartbeats, through its Phase II trials and prepare it for Phase III.

Covid-19 roundup: As­traZeneca could soon re­sume US vac­cine tri­als; Pfiz­er's dis­tri­b­u­tion plan in­volves spe­cial ship­ping box­es, no whole­salers 

AstraZeneca’s Covid-19 vaccine trial could resume in the US later this week, according to a Reuters report.

Experimental dosing of the candidate came screeching to a halt in September, when a participant in the UK suffered what’s believed to be the spinal inflammatory condition transverse myelitis. Trials have already resumed in the UK, Brazil, India, Japan and South Africa — and four anonymous sources told Reuters that a green light from the US could come as early as this week.

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Roche finds a home for a new, $500M man­u­fac­tur­ing lo­gis­tics hub, promis­ing 500 jobs

Roche is pouring $500 million into its Canadian headquarters in Mississauga, Ontario to set up a new hub that will coordinate logistics for its global supply chain.

Over the 5-year investment, the Swiss pharma giant expects to add 200 jobs over next year and another 300 by the end of 2023.

Introduced as a $190 million global pharmaceutical development site in 2011, the campus currently houses Roche’s Canadian commercial unit as well as product development, global procurement and pharma informatics. The new expansion will see it organize manufacturing across 13 plants and 11 sites, according to FiercePharma.

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Un­fazed by PhII miss, Roche ush­ers Prothena's Parkin­son's drug in­to late-stage tri­al — a $60M move

Prothena’s prasinezumab may not have met the primary endpoint in Phase II, but its partners at Roche are seeing enough to move it into a late-stage trial for Parkinson’s disease.

The Phase IIb will build on the Phase II PASADENA study, adding a subgroup of early Parkinson’s patients on stable levodopa therapy to the population.

It’s a significant milestone for a $600 million deal that dates back to 2013, as dosing of the first patient — expected next year — will trigger a $60 million milestone payment to Prothena.

Steve Chen, Cellis Therapeutics president and CMO (Cellics)

UC San Diego spin­out award­ed up to $15M for nanosponge de­signed to soak up sep­sis-caus­ing tox­ins

CARB-X, a global partnership looking to spur the development of new antibacterial drugs, is awarding Cellics Therapeutics $3.94 million to do what president and CMO Steve Chen calls “looking at traditional drug development upside down.”

Instead of going after a target directly — in this case bacterial toxins and inflammatory cytokines that cause sepsis — Cellics researchers “flip it around” to examine the host cells being attacked. The UC San Diego spinout then creates what it calls “nanosponges” — nanoparticles cloaked in the fragments of macrophage cell membranes. Chen says the “sponges” are designed to trap the sepsis-causing endotoxins and cytokines on their cell membranes, neutralizing them.