FDA launch­es pi­lot pro­gram in sup­port of com­plex tri­al de­signs for drugs and bi­o­log­ics

The FDA an­nounced a new pi­lot pro­gram Wednes­day aimed at en­cour­ag­ing the use of com­plex in­no­v­a­tive tri­al de­signs in the de­vel­op­ment of drugs and bi­o­log­ics.

Spon­sors se­lect­ed by the FDA based on the cri­te­ria for par­tic­i­pa­tion in the “Com­plex In­no­v­a­tive De­signs Pi­lot Meet­ing Pro­gram” will be able to en­gage staff in ear­ly dis­cus­sions on ap­proach­es to com­plex de­signs.

Scott Got­tlieb

The pi­lot will re­main op­er­a­tional through 30 Sep­tem­ber 2022, but spon­sors have un­til 30 June 2022 to sub­mit meet­ing re­quests, ac­cord­ing to Wednes­day’s no­tice. Meet­ings un­der the pi­lot will be con­duct­ed by FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search or its Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search.

Tri­als de­signed as part of the pi­lot, which ful­fills a com­mit­ment un­der the sixth it­er­a­tion of the Pre­scrip­tion Drug User Fee Act, will be used by the FDA to de­vel­op case stud­ies. Up to two meet­ing re­quests will be ac­cept­ed on a quar­ter­ly ba­sis each year through­out the pi­lot’s du­ra­tion.

Par­tic­i­pa­tion in­volves an ini­tial and a fol­low-up meet­ing on a de­sign and prod­uct with­in about 120 days, and the se­lect­ed spon­sors will re­ceive a sum­ma­ry “with­in 60 days of each meet­ing,” the no­tice adds.

The no­tice al­so ex­plains the process for sub­mit­ting meet­ing re­quests and the rec­om­mend­ed for­mat­ting of the in­for­ma­tion pack­age se­lect­ed spon­sors are re­quired to sub­mit elec­tron­i­cal­ly to the FDA.

Plans to launch the pi­lot were first dis­cussed dur­ing a pub­lic meet­ing in March, though cer­tain con­cerns were raised by stake­hold­ers such as Genen­tech and Bio­gen re­gard­ing flex­i­bil­i­ty and scope.

“Through in­ter­ac­tion be­tween FDA and pi­lot par­tic­i­pants, this pro­gram will help ac­cel­er­ate the de­vel­op­ment of prod­ucts in ar­eas of un­met need, and en­sure knowl­edge ex­change to help ad­vance the sci­en­tif­ic com­mu­ni­ty’s ap­proach to de­sign­ing clin­i­cal tri­als over­all…,” FDA Com­mis­sion­er Scott Got­tlieb said in a Wednes­day post. He al­so point­ed to oth­er re­cent ef­forts aimed at achiev­ing the pi­lot’s goals.

The re­lat­ed ef­forts in­clude new draft guid­ance doc­u­ments on seam­less clin­i­cal tri­als for ex­pe­dit­ed drug de­vel­op­ment and on the use of place­bo-con­trolled tri­al de­signs, both of which were is­sued this month.

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. 

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Dif­fu­sion to hand Nas­daq spot to EIP Phar­ma for PhI­Ib de­men­tia study of ex-Ver­tex drug

One of the more than a dozen bidders for Diffusion Pharmaceuticals’ spot on Nasdaq has prevailed.

Boston biotech EIP Pharma will merge with Diffusion in an all-stock deal, with plans to start a Phase IIb clinical trial in the coming months in a common form of dementia with no approved treatments. The combined company will be renamed CervoMed.

The nine-year-old privately-held EIP is working on a former Vertex drug that it will test in a 160-person Phase IIb in patients with dementia with Lewy bodies, or DLB. The National Institute on Aging is expected to fund that trial with a $21 million grant. With the reverse merger, slated for closing in the middle of this year, EIP will be funded through that readout in the second half of 2024. EIP’s equity and debt holders will own about 77.25% of the combined company.

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CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

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