FDA re­vis­es guid­ance on post­mar­ket­ing stud­ies to re­flect ARIA sys­tem, SUP­PORT act

The FDA on Thurs­day re­vised its draft guid­ance on post­mar­ket­ing stud­ies and clin­i­cal tri­als un­der Sec­tion 505(o)(3) of the Fed­er­al Food, Drug, and Cos­met­ic Act (FD&C Act).

The FDA says the draft guid­ance, first is­sued for com­ment in 2011, is be­ing re­vised to de­tail the fac­tors the agency con­sid­ers when de­ter­min­ing whether a post­mar­ket­ing study or clin­i­cal tri­al will be re­quired for a drug or bi­o­log­ic or whether post­mar­ket­ing re­ports and the agency’s ac­tive post­mar­ket risk iden­ti­fi­ca­tion and analy­sis (ARIA) sys­tem are suf­fi­cient to as­sess a prod­uct’s risks in the post­mar­ket set­ting.

The FDA ex­plains that at the time the draft guid­ance was first is­sued, “the ARIA sys­tem was still in ear­ly de­vel­op­ment” and now that it is ful­ly op­er­a­tional, it must be con­sid­ered when de­ter­min­ing if a post­mar­ket­ing study or clin­i­cal tri­al is re­quired.

The guid­ance is al­so be­ing up­dat­ed to re­flect a pro­vi­sion of the 2018 Sub­stance Use-Dis­or­der Pre­ven­tion that Pro­motes Opi­oid Re­cov­ery and Treat­ment for Pa­tients and Com­mu­ni­ties Act (SUP­PORT Act) that en­ables the FDA to re­quire post­mar­ket­ing stud­ies un­der sec­tion 505(o)(3) to as­sess the po­ten­tial re­duc­tion in ef­fi­ca­cy of a drug.

While the SUP­PORT Act fo­cus­es on opi­oids and oth­er con­trolled sub­stances, the FDA notes that “at this time, the agency does not in­tend to treat con­trolled sub­stances dif­fer­ent­ly than oth­er pre­scrip­tion drugs.”

The re­vised draft guid­ance pro­vides a list of four ex­am­ples of clin­i­cal tri­als to as­sess risks re­lat­ed to re­duced ef­fec­tive­ness; though, in line with the agency’s state­ment on not dif­fer­en­ti­at­ing be­tween con­trolled sub­stances and oth­er drugs, does not in­clude any ex­am­ples of tri­als in­volv­ing opi­oids or oth­er con­trolled sub­stances.


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The 20 un­der 40: In­side the next gen­er­a­tion of bio­phar­ma lead­ers

“Each generation needs a new music,” Francis Crick wrote in 1988, reflecting back on his landmark discovery. Crick was 35, then, in 1953, when he began working with a 23-year-old named James Watson, and 37 when the pair unveiled the double helix. Rosalind Franklin, whose diffraction work undergirded their metal model, was 32.

The model would become the score for a new era in biology, one devoted to cracking the basic structures turning inside life. Subsequent years would bring new conductors and new rhythms: Robert Swanson, 29 when he convinced a 39-year-old Herb Boyer to build a company off his work and call it Genentech; Phillip Sharp, 29 when he discovered RNA splicing and 34 when he co-founded Biogen; Frances Arnold, 36 when she pioneered directed evolution; Feng Zhang, 31 when he published his CRISPR paper.

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FDA Commissioner Stephen Hahn and President Donald Trump at a press briefing on March 19, 2020. (AP Images)

Biotech ex­ecs warn that the FDA is fum­bling their re­sponse to the Covid-19 open-door promise, de­lay­ing progress

A few days ago the FDA touted a procedure for Covid-19 meds that committed the agency to immediate action for developers, formalizing a high-speed response that’s been promised for weeks.

Bioregnum Opinion Column by John Carroll

Decisions that once required months would be measured in hours under the Coronavirus Treatment Acceleration Program. “In many cases” trial protocols could be hammered out in less than a single day. If you had a potential solution to the crisis, the appropriate staffer would be in touch “to get studies underway quickly.”

It would be the ultimate high-speed regulatory pathway from Phase I to approval. Red tape was banished.

But it’s clear that for some — and quite likely many — biopharma execs, the actual agency response has not measured up to the promise. Beyond the front ranks of advanced companies in the field, like Gilead, or for drugs endorsed by President Trump, it may not even come close.

“The first response is this form letter everyone gets,” says one biotech CEO who’s reached out to the FDA on Covid-19. And when you try to cut through that, the ball gets dropped as it is passed from top officials to the frontline staff actually charged with getting things done.

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GSK's Hal Bar­ron buys a $250M stake in George Scan­gos' Vir and makes a bee­line to the clin­ic with Covid-19 an­ti­bod­ies

GlaxoSmithKline is diving straight into the swirling waters of Covid-19 R&D work, and investing $250 million to grab a chunk of equity in one of the emerging stars in infectious disease research to make it official.

GSK put out word this morning that it is partnering with Vir Biotechnology $VIR, the infectious disease startup founded in the Bay Area by former Biogen CEO George Scangos. They’re planning a leap into Phase II studies for 2 preclinical antibody candidates — VIR-7831 and VIR-7832 — that have been engineered to target the SARS-CoV-2 spike protein.

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Covid-19 roundup: Trump push­es untest­ed malar­ia drugs again; Covid-19-pos­i­tive British PM goes to hos­pi­tal

In a White House briefing on Sunday, President Trump once again issued an impassioned endorsement of malaria drugs chloroquine and hydroxychloroquine as treatments for Covid-19, as the virus behind the disease ravages the United States.

“I want people to live, and I’m seeing people dying. I’ve seen people dying without it! They should do it (use the drugs), what really do we have to lose?” he said. “This medicine’s been tested for many years, for malaria and for lupus and its been out there, so it’s out there and it doesn’t kill people. We have some very good results and some very good tests — but we don’t have time to say gee, let’s take a couple of years and test it out, and let’s go and test with the test tubes and the laboratories.”

Ax­some cel­e­brates mi­graine PhI­II win, shift­ing eyes to­ward a pair of Q4 sub­mis­sions

One week after reporting a late-stage failure for its lead drug, Axsome is back with positive news: In the 302-person Phase III INTERCEPT trial, its experimental migraine drug hit both primary endpoints: Stopping migraine pain entirely and preventing pain from progressing beyond ”mild intensity.”

The trial win sets up a big fourth quarter for the 8-year-old company and keeps them on track to file for approval for the migraine drug, known as AXS-05. And despite the late-stage failure, they plan to file for approval on their lead depression drug, known as AXS-07, citing secondary endpoints and the high placebo rates that have long clouded depression trial results.

Bris­tol My­ers Squibb, Ac­celeron rack up sec­ond OK for Re­blozyl, on­to block­buster sta­tus

Reblozyl, the anemia drug Celgene paid a modest $25 million upfront to partner on 9 years ago, brought Bristol Myers Squibb one of only two new drug approvals in 2019. Merely five months later, it’s taking home another supplemental OK expected to catapult sales into blockbuster territory.

The FDA has approved Reblozyl (luspatercept) as a treatment for anemia in a subset of adults with lower-risk myelodysplastic syndromes. Specifically, it’s limited to patients who have failed an erythropoiesis stimulating agent and require 2 or more red blood cell units over 8 weeks. These MDS patients are further characterized by ring sideroblasts, where iron accumulates in the mitochondria of red blood cells, rendering them dysfunctional.

Gilead CEO Daniel O'Day attends a meeting with the President and other biopharma leaders at the White House on March 2, 2020 (AP Photo)

Ramp­ing up glob­al pro­duc­tion of remde­sivir, Gilead CEO Dan O’Day de­tails plans to dis­trib­ute 1.5M dos­es to fight Covid-19 — for free

Gilead is still some days away from turning the card on its first round of data on remdesivir’s ability to fight severe cases of Covid-19, but the big biotech is ramping up an emergency supply of a million courses of therapy as it starts free distribution of the drug to tens of thousands of patients under their compassionate use and expanded access program as well as clinical trials.

In his latest open letter posted over the weekend, Gilead CEO Dan O’Day outlined how the company has been successful in cutting production time on remdesivir while repurposing some of their own facilities and turning to contract manufacturers to build a near-term supply of 1.5 million doses. They are still working on efficacy and dosing, but that supply could cover 140,000 courses of treatment. That supply, he added, would be more widely available following a potential approval.

UP­DAT­ED: A small, ob­scure biotech just won big with their IPO. In this mar­ket. Are you kid­ding me?

How could a small, largely unknown biotech that emerged from stealth mode just months ago with early-stage cancer programs jump onto Wall Street in the middle of a Category 6 financial hurricane and sail through with a $165 million IPO?

And what does that mean for the rest of the industry waiting to see just how much damage global lockdowns will wreak on clinical development?

The biotech is a company called Zentalis. The crew there nabbed an $85 million crossover round late last year — notably waiting 5 years before waving the numbers around to attract attention, according to my read of a FierceBiotech story. Perceptive joined in, but the syndicate was not in general the kind of marquee affair that gets tongues wagging.

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Bob Nelsen at the Milken Institute Global Conference on April 29, 2019 in Beverly Hills, California. (Photo by Michael Kovac/Getty Images)

ARCH chief Bob Nelsen has $1.5B to prove 2 sim­ple points: ‘We’re in the most in­no­v­a­tive time ever’ and in­vestors are stay­ing

ARCH co-founder and managing director Bob Nelsen has a well known yen for the home run swing, betting big on potentially transformative meds and tech and the biotech teams he helps bring together. He thrives and bleeds on the cutting edge. And now Nelsen and the ARCH group have debuted 2 big funds to prove that this is the time for the best of biotech to shine — deadly pandemic be damned.

Two new funds, ARCH Venture Fund X and ARCH Venture Fund X Overage, gathered a combined $1.46 billion. And that’s a record. ARCH Venture Fund IX and ARCH Venture Fund IX Overage closed in 2016 with a combined $1.1 billion. ARCH Venture Fund VIII and ARCH Venture Fund VIII Overage closed in 2014 with a combined $560 million.

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