Richard Pazdur (vis AACR)

FDA en­cour­ages in­clud­ing in­cur­able can­cer pa­tients in tri­als, re­gard­less of pri­or ther­a­pies

The FDA on Thurs­day called to in­clude those with in­cur­able can­cers (when there is no po­ten­tial for cure or for pro­longed/near nor­mal sur­vival) in ap­pro­pri­ate clin­i­cal tri­als, re­gard­less of whether they have re­ceived ex­ist­ing al­ter­na­tive treat­ments.

His­tor­i­cal­ly, many can­cer clin­i­cal tri­als have re­quired that par­tic­i­pat­ing pa­tients pre­vi­ous­ly re­ceived mul­ti­ple ther­a­pies, ac­cord­ing to Richard Paz­dur, di­rec­tor of the FDA’s On­col­o­gy Cen­ter of Ex­cel­lence.

But the new, 2-page draft guid­ance rec­om­mends ex­pand­ing the el­i­gi­bil­i­ty cri­te­ria “such that, with ap­pro­pri­ate in­formed con­sent, pa­tients may be el­i­gi­ble for in­clu­sion in tri­als of in­ves­ti­ga­tion­al drugs, in­clud­ing first-in-hu­man tri­als, re­gard­less of whether they have re­ceived avail­able ther­a­py in the non-cu­ra­tive set­ting.”

Vin­cent Ra­jku­mar

FDA al­so said the in­formed con­sent doc­u­ments that pa­tients sign be­fore par­tic­i­pat­ing in the tri­al should clear­ly state whether there are oth­er treat­ment op­tions known to con­fer clin­i­cal ben­e­fit, and should in­clude a dis­cus­sion of pos­si­ble ben­e­fits, risks, and un­cer­tain­ties.

Vin­cent Ra­jku­mar, can­cer doc­tor and pro­fes­sor at the Mayo Clin­ic, told End­points News that what the guid­ance is de­scrib­ing al­ready oc­curs in prac­tice, and that he ful­ly sup­ports it.

“We risk los­ing a lot of po­ten­tial­ly ac­tive drugs be­cause we are test­ing them too late af­ter ex­haust­ing all oth­er treat­ments by which time it’s pret­ty hard for any drug to work,” he said. “So try­ing new drugs ear­ly in dis­ease course with in­formed con­sent is some­thing I ful­ly sup­port.”

The guid­ance al­so calls on spon­sors to eval­u­ate pa­tients who have re­ceived avail­able ther­a­py(ies) and those who haven’t in sep­a­rate co­horts, “par­tic­u­lar­ly if in­ter­pre­ta­tion of ef­fi­ca­cy re­sults re­quires a ho­moge­nous pa­tient pop­u­la­tion.”

Gen­er­al­ly, FDA said, el­i­gi­bil­i­ty cri­te­ria should re­quire that pa­tients have re­ceived avail­able ther­a­py(ies) that could cure a sub­stan­tial pro­por­tion of pa­tients, such as with avail­able treat­ment op­tions for pe­di­atric acute lym­phoblas­tic leukemia, clas­sic Hodgkin lym­phoma, or tes­tic­u­lar can­cer, in clin­i­cal tri­als eval­u­at­ing in­ves­ti­ga­tion­al can­cer drugs.

“Al­ter­na­tive­ly, such avail­able ther­a­py should be ad­min­is­tered to all pa­tients in the tri­al, where the in­ves­ti­ga­tion­al drug is added to such ther­a­py (i.e., add-on tri­al),” FDA said. “How­ev­er, el­i­gi­bil­i­ty cri­te­ria in which pa­tients re­ceive an in­ves­ti­ga­tion­al drug(s) in lieu of avail­able ther­a­py is rea­son­able in the non-cu­ra­tive set­ting (i.e., when there is no po­ten­tial for cure or for pro­longed/near nor­mal sur­vival) when pa­tients have been pro­vid­ed ad­e­quate in­for­ma­tion to make an in­formed de­ci­sion on tri­al par­tic­i­pa­tion.”

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Matt Gline (L) and Pete Salzmann

UP­DAT­ED: Roivant bumps stake in Im­muno­vant with a $200M deal. But with M&A off the ta­ble, shares crater

Roivant has worked out a deal to pick up a chunk of stock in its majority-owned sub Immunovant $IMVT, but the stock buy falls far short of its much-discussed thoughts about buying out all of the 43% of shares it doesn’t already own.

Roivant, which recently inked a SPAC move to the market at a $7 billion-plus valuation, has forged a deal to boost its ownership in Immunovant by 6.3 points, ending with 63.8% of the biotech’s stock following a $200 million injection. That cash will bolster Immunovant’s cash reserves, giving it a $600 million war chest to fund a slate of late-stage studies for its big drug: the anti-FcRn antibody IMVT-1401.

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UP­DAT­ED: Watch out Glax­o­SmithK­line: As­traZeneca's once-failed lu­pus drug is now ap­proved

Capping a roller coaster journey, AstraZeneca has steered its lupus drug anifrolumab across the finish line.

Saphnelo, as the antibody will be marketed, is the only treatment that’s been approved for systemic lupus erythematosus since GlaxoSmithKline’s Benlysta clinched an OK in 2011. The British drugmaker notes it’s also the first to target the type I interferon receptor.

Mirroring the population that the drug was tested on in late-stage trials, regulators sanctioned it for patients with moderate to severe cases who are already receiving standard therapy — setting up a launch planned for the end of August, according to Ruud Dobber, who’s in charge of AstraZeneca’s biopharmaceuticals business unit.

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Sanofi preps a multi­bil­lion-dol­lar buy­out of an mR­NA pi­o­neer af­ter falling be­hind in the race for a Covid-19 jab — re­port

It looks like Sanofi CEO Paul Hudson is dead serious about his intention to vault directly into contention for the future of mRNA vaccines.

A year after paying Translate Bio a whopping $425 million in an upfront and equity payment to help guide the pharma giant to the promised land of mRNA vaccines for Covid-19, Sanofi is reportedly ready to close the deal with a buyout.

Translate’s stock $TBIO soared 78% after the market closed Monday. A spokesperson for Sanofi declined to comment on the report, telling Endpoints News that the company doesn’t comment on market rumors.

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Rick Pazdur (via AACR)

FDA's on­col­o­gy head Rick Paz­dur de­fends the ac­cel­er­at­ed ap­proval path­way, claim­ing it is 'un­der at­tack'

The FDA is sounding the alarm over its accelerated approval pathway as backlash continues over the recent nod in favor of Biogen’s Alzheimer’s drug Aduhelm, and an ODAC meeting on six such approvals that could potentially be pulled from the market — two of which already have.

“Do you think accelerated approval is under attack? I do,” Rick Pazdur, head of FDA’s Oncology Center of Excellence, said at a Friends of Cancer Research webinar on Thursday.

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Alan Hirzel, Abcam

Drug sup­pli­er Ab­cam brings a long­time col­lab­o­ra­tor in house as part of $340M buy­out pact

BioVision has supplied Abcam with research tools since 1999, and now the two are making it official as part of a merger unveiled Monday.

Abcam will buyout BioVision as part of a $340 million acquisition deal to bring aboard the supplier’s biochemical and cell-based assays for biological research, as well as recombinant proteins, antibodies and enzymes.

The deal will give Abcam control of BioVision’s portfolio and allow for both the expansion of research existing areas of focus such as oncology, neuroscience and epigenetics and preparation to expand into new products. As a part of the deal, Abcam will develop and supply products and services to NKY, the previous owner of BioVision and receive support for ongoing development and commercialization of in vitro diagnostic products.

Tib­so­vo clears an­oth­er hur­dle for Servi­er, but can it make Agios' old drug prof­itable?

When European regulators saw the data Agios used to win US approval for their AML drug Tibsovo, they sent the more than decade-old biotech back to the drawing board. A single, single-armed trial was not going to cut it.

On Monday, though, the drug’s new owners announced it had cleared a more rigorous study. In a randomized, Phase III trial of certain newly diagnosed patients, those who received a combination of Tibsovo and chemotherapy lived longer than those who received a combination of placebo and chemotherapy. Those patients also had higher response rates and complete remission rates.

Not all mR­NA vac­cines are cre­at­ed equal. Does it mat­ter?; Neu­ro is back; Pri­vate M&A af­fair; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As part of our broader and deeper drive, Endpoints has been pairing webinars with our special reports to cover more angles on a given topic. In conjunction with Max Gelman’s neuroscience feature, Kyle Blankenship moderated an insightful panel to discuss where the field is headed. You can register to watch it on demand here.

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Bris­tol My­ers pulls lym­phoma in­di­ca­tion for Is­to­dax af­ter con­fir­ma­to­ry tri­al falls flat

Amid an industrywide review of cancer drugs with accelerated approval, Bristol Myers Squibb had to make the tough call last month to yank an approval for leading I/O drug Opdivo after flopping a confirmatory study. Now, a second Bristol Myers drug is on the chopping block.

Bristol Myers has pulled aging HDAC inhibitor Istodax’s indication in peripheral T cell lymphoma after a Phase III confirmatory study for the drug flopped on its progression-free survival endpoint, the drugmaker said Monday.