What's com­ing? Paz­dur, Marks and Wood­cock dis­cuss FDA changes

Three of the FDA’s di­rec­tors — Richard Paz­dur, Pe­ter Marks and Janet Wood­cock — took the stage Wednes­day at Pre­vi­sion Pol­i­cy’s Bio­phar­ma Con­gress to dis­cuss all things FDA: from why the agency is viewed more pos­i­tive­ly now than in the past to what needs to hap­pen in each of their cen­ters.

CDER di­rec­tor Wood­cock said that the dif­fer­ence be­tween years past, when FDA was viewed neg­a­tive­ly, and now is that there has been a sort of sci­en­tif­ic rev­o­lu­tion, with bi­ol­o­gy be­ing har­nessed in nov­el ways and in­vest­ments at NIH pay­ing off.

Richard Paz­dur

On­col­o­gy Cen­ter of Ex­cel­lence di­rec­tor Paz­dur al­so not­ed the tremen­dous growth in un­der­stand­ing dis­eases and in the im­munol­o­gy of can­cer.

“One part of the piece in on­col­o­gy is that our staff has em­braced these changes,” he said, not­ing they’re “not just bean coun­ters for sur­vival da­ta” but have got­ten in­volved in the sci­ence of on­col­o­gy. The staff are part­ners in the process rather than just tick­ing box­es, he said.

And Marks, di­rec­tor of the Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search, ex­plained how the idea of screen­ing li­braries of com­pounds has gone out the win­dow, while nowa­days it’s more about ra­tio­nal drug de­vel­op­ment – “and gene ther­a­py is the epit­o­me of that.”

On the top­ic of gene ther­a­py, Marks not­ed there are now 700 in­ves­ti­ga­tion­al new drug ap­pli­ca­tions be­fore FDA. But com­pare that with cell ther­a­pies, he said, not­ing a gray mar­ket of un­proven treat­ments, which in some cas­es are noth­ing bet­ter than bad­ly man­u­fac­tured snake-oil that don’t con­tain live cells. He al­so said the use of un­ap­proved stem cell ther­a­pies around the coun­try is a sur­ro­gate mark­er for how Right to Try may play out. FDA doesn’t usu­al­ly learn about what is go­ing on un­til some­thing re­al­ly bad hap­pens, Marks added.

On the prospects of what’s to come at FDA, Wood­cock not­ed the re­al-time re­view pi­lot in the On­col­o­gy Cen­ter of Ex­cel­lence, which re­views parts of a sub­mis­sion be­fore the en­tire ap­pli­ca­tion is sub­mit­ted, is “fore­shad­ow­ing some­thing big­ger.” But she al­so ex­plained how the speed of re­views does not re­al­ly in­flu­ence the re­sults, say­ing: “what has changed is the cost of these drugs, and it’s changed the de­bate be­cause of val­ue.”

Nowa­days and mov­ing in­to the fu­ture, there’s a so­ci­etal ben­e­fit and harm dis­cus­sion that im­plic­it­ly in­vokes “trade-offs in val­ue – that’s the new de­bate we’re hav­ing – too fast and too slow is re­al­ly not the is­sue.”

For Marks, the biggest prob­lem mov­ing for­ward for gene ther­a­pies is the quan­tum leap need­ed in man­u­fac­tur­ing and to ap­ply­ing the tech­niques be­yond just rare dis­eases. “We need to do the kind of trans­for­ma­tion Steve Jobs did for dig­i­tal,” with man­u­fac­tur­ing gene ther­a­pies. He al­so not­ed FDA is “very le­nient” about some of its man­u­fac­tur­ing stan­dards for get­ting gene ther­a­pies in­to Phase 1 tri­als, but scal­ing up the man­u­fac­tur­ing is what’s dif­fi­cult.

Mov­ing in­to the fu­ture leg­isla­tive­ly, Wood­cock said she’s in­ter­est­ed in leg­is­la­tion on gener­ic drug la­bel­ing. “We have to clean up the ar­ma­men­tar­i­um so it doesn’t get out of date,” she said. She fur­ther not­ed FDA is work­ing with Con­gress on over-the-counter  drug re­forms, which is the ba­sis for the Of­fice of New Drug mod­ern­iza­tion. “If we don’t get the OTC re­form, we’ll have to pull back on the OND re­forms,” she said.


Im­age: Janet Wood­cock

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Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

UP­DAT­ED: End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

(This piece was last updated on August 13. Endpoints News will continue to track the latest developments through the FDA’s marketing decisions.)

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

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Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

Qi­a­gen in­vestors spurn Ther­mo Fish­er’s takeover of­fer, de­rail­ing a $12B+ deal

Thermo Fisher Scientific had announced an $11.5 billion takeover of Dutch diagnostics company Qiagen back in March, but the deal apparently did not sit well with Qiagen investors.

After getting hammered by critics who contended that Qiagen $QGEN was worth a lot more than what Thermo Fisher wanted to spend, investors turned thumbs down on the offer — derailing the buyout even after Thermo Fisher increased its offer to $12.6 billion in July. Qiagen’s share price has been boosted considerably by Covid-19 as demand for its testing kits surged.

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Xuefeng Yu in Hong Kong, 2019 (Imaginechina via AP Images)

CanSi­no reaps $748M wind­fall from Shang­hai IPO — as it warns Covid-19 vac­cine won't be a huge mon­ey mak­er

CanSino began the year with a clear goal to secure a secondary listing on Shanghai’s STAR market. Then something more urgent came along: As a rising vaccine developer on a mission to bring global standard immunizations to China, it heeded the call to make a vaccine to protect against a virus that would paralyze the whole world.

Xuefeng Yu and his team managed to keep doing both.

More than a month after CanSino’s Covid-19 vaccine candidate is authorized for military use in China, the Hong Kong-listed company has made a roaring debut in Shanghai. It fetched $748 million (RMB$5.2 billion) by floating 24.8 million shares, and soared 88% on its first trading day.

James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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Giovanni Caforio, Bristol Myers Squibb CEO (Christopher Goodney/Bloomberg via Getty Images)

UP­DAT­ED: Bris­tol My­ers Squibb com­mits $300 mil­lion to com­bat racial dis­par­i­ties, but de­clines to re­lease own de­mo­graph­ic da­ta

After the police killing of George Floyd, a flurry of pharma and biotech companies, executives and investors jumped out to make statements, either expressing support for Black Lives Matter and the protests or condemning systemic racism.

Now, a Big Pharma company is publicly putting some teeth behind those statements. This morning, Bristol Myers Squibb announced they would spend $300 million on a broad effort to reduce racial health disparities, and diversify both their clinical trials and their own executive team and workforce.