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

First pub­lished here. reg­u­la­to­ry fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email news@raps.org for more in­for­ma­tion.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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Stephen Isaacs, Aduro president and CEO (Aduro)

Once a high fly­er, a stag­ger­ing Aduro is auc­tion­ing off most of the pipeline as CEO Stephen Isaacs hands off the shell to new own­ers

After a drumbeat of failure, setbacks and reorganizations over the last few years, Aduro CEO Stephen Isaacs is handing over his largely gutted-out shell of a public company to another biotech company and putting up some questionable assets in a going-out-of-business sale.

Isaacs —who forged a string of high-profile Big Pharma deals along the way — has wrapped a 13-year run at the biotech with one program for kidney disease going to the new owners at Chinook Therapeutics. A host of once-heralded assets like their STING agonist program partnered with Novartis (which dumped their work on ADU-S100 after looking over weak clinical results), the Lilly-allied cGAS-STING inhibitor program and the anti-CD27 program out-licensed to Merck will all be posted for auction under a strategic review process.

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Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Bris­tol My­ers Squib­b's just-launched MS drug Zeposia makes the cut in key ul­cer­a­tive col­i­tis tri­al

In March, Zeposia became the third oral S1P modulator to secure US approval for multiple sclerosis. Now, the drug has succeeded in a key ulcerative colitis study.

The immunomodulator, akin to others in its class, controls lymphocyte trafficking by limiting the white blood cells to the lymphatic system, in the lymph nodes, and thwarting their ability to jam up lymph nodes — precluding their ability to penetrate the bloodstream and the central nervous system.

Hill­house re­casts spot­light on Chi­na's biotech scene with $160M round for Shang­hai-based an­ti­body mak­er

Almost two years after first buying into Genor Biopharma’s pipeline of cancer and autoimmune therapies, Hillhouse Capital has led a $160 million cash injection to push the late-stage assets over the finish line while continuing to fund both internal R&D and dealmaking.

The Series B has landed right around the time Genor would have listed on the Hong Kong stock exchange, according to plans reported by Bloomberg late last year. Insiders had said that the company was looking to raise about $200 million.

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Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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Novus Ther­a­peu­tics plunges deep in­to pen­ny stock ter­ri­to­ry af­ter failed ear tri­al

After a more than 15-year run, a California-based biotech is exploring options, including a sale, after its lead experimental therapy failed an exploratory mid-stage study in patients with middle ear infections characterized by a build-up of fluid behind the eardrum.

The company, initially called Tokai Pharmaceuticals but which subsequently changed its name to Novus Therapeutics in 2017, saw its shares more than halve on Monday after the drug — OP0201— did not pass muster as an adjunct therapy to oral antibiotics in infants and children aged 6 to 24 months with acute otitis media (OM).

Federico Mingozzi (Spark)

Spark touts an­i­mal da­ta for a so­lu­tion to AAV gene ther­a­py's an­ti­body prob­lem

Among all the limitations of using an adeno-associated virus as a vector to deliver a gene — still the most established modality in gene therapy given years of trial and error and finally success — the presence of neutralizing antibodies, whether pre-existing or induced, looms large.

“When I think about the immune responses in AAV, I try to sort of layer them,” Federico Mingozzi, the CSO at Spark Therapeutics, told Endpoints News. “The antibody is the first layer. It’s the first block that you find when you’re trying to do gene transfer.”