Oct 7, 2021

Zachary Brennan Senior Editor The FDA’s an­timi­cro­bial drugs ad­vi­so­ry com­mit­tee on Thurs­day vot­ed unan­i­mous­ly in fa­vor of FDA ap­prov­ing Take­da’s an­tivi­ral for post-trans­plant cy­tomegalovirus. The ad­comm vot­ed 17-0 on the ques­tion of whether the over­all ben­e­fit-risk pro­file is fa­vor­able for the use of Take­da’s marib­avir for the treat­ment of trans­plant re­cip­i­ents with CMV in­fec­tion and dis­ease re­frac­to­ry to treat­ment and with geno­typ­ic re­sis­tance to four oth­er an­tivi­rals — gan­ci­clovir, val­gan­ci­clovir, fos­car­net or cid­o­fovir. On whether the drug, which took about two decades to de­vel­op, should be ap­proved for the same pop­u­la­tion but those with­out geno­typ­ic re­sis­tance to the four an­tivi­rals, the com­mit­tee vot­ed 17-0. FDA’s brief­ing doc­u­ments re­leased ahead of the meet­ing showed how marib­avir proved safe across mul­ti­ple stud­ies and sta­tis­ti­cal­ly su­pe­ri­or to in­ves­ti­ga­tor-as­signed treat­ment (IAT) in a Phase III tri­al, which Take­da an­nounced ear­li­er this year. While sev­er­al mem­bers of the ad­comm said dur­ing the dis­cus­sion that they don’t want to let per­fect be the en­e­my of the good in terms of the da­ta around marib­avir, sev­er­al oth­ers ques­tioned the end­point used in that tri­al, and why the tri­al didn’t in­clude a more di­verse pop­u­la­tion. Ad­comm chair Lind­sey Baden of the Dana-Far­ber Can­cer In­sti­tute, who vot­ed in fa­vor of the ap­proval, al­so called the da­ta “messy,” but said the to­tal­i­ty of da­ta is per­sua­sive. He al­so not­ed the ad­van­tages of an oral drug in this in­di­ca­tion. Com­mit­tee mem­ber Nan­cy Bridges, chief of the trans­plan­ta­tion branch at the NIH’s Na­tion­al In­sti­tute of Al­ler­gy and In­fec­tious Dis­eases, who vot­ed in fa­vor of ap­proval al­so said that over­all, “the da­ta are very per­sua­sive.” But she not­ed that the par­tic­i­pants in the Phase III tri­al “were over­whelm­ing­ly white,” even though this con­di­tion has a high­er preva­lence among African Amer­i­cans. “I’d like to see some da­ta from the com­pa­ny on ef­fi­ca­cy in African Amer­i­cans, which we don’t have,” Bridges said. George Siber­ry, a med­ical of­fi­cer at the US Agency for In­ter­na­tion­al De­vel­op­ment, al­so said it was dis­ap­point­ing that no ado­les­cents were in­clud­ed in the piv­otal tri­al. Pe­ter Weina, di­rec­tor of the De­fense Health Agency, said that even if the drug is ap­proved for a nar­row pop­u­la­tion, it’ll be used for all dif­fer­ent in­di­ca­tions off-la­bel. “I’m torn that the dif­fer­ence be­tween geno­typ­ic re­sis­tance and no geno­typ­ic re­sis­tance is tru­ly a moot point here,” Weina said. An ap­proval from FDA would cap a long and rocky his­to­ry for the drug, which has changed hands sev­er­al times over the last two decades fol­low­ing both pos­i­tive and neg­a­tive tri­als. Glax­o­SmithK­line first syn­the­sized the drug about 20 years ago and did some ear­ly clin­i­cal work be­fore li­cens­ing it to the rare dis­ease and in­fec­tious dis­ease-fo­cused biotech Vi­roPhar­ma. Vi­roPhar­ma then missed a pri­ma­ry end­point in a Phase III study in 2009. Marib­avir failed to pre­vent CMV in­fec­tions bet­ter than place­bo in pa­tients re­ceiv­ing bone mar­row trans­plants. Vi­roPhar­ma sug­gest­ed a high­er dose might lead to a bet­ter re­sponse, but end­ed up pass­ing the can­di­date to Shire as part of a $4.2 bil­lion buy­out in 2013. Shire upped the dose in var­i­ous phar­ma­co­ki­net­ic and ef­fi­ca­cy stud­ies, and in 2016, a Phase II study showed that the drug helped clear in­fec­tions. The FDA com­mend­ed the re­sults with a break­through des­ig­na­tion in 2018. And the fol­low­ing year, Take­da com­plet­ed its ac­qui­si­tion of Shire for $62 bil­lion. AUTHOR John Carroll Editor & Founder The FDA has slapped a clinical hold on Allogene’s clinical trials after a patient in one of their early-stage studies for the off-the-shelf cell therapy ALLO-501A developed a “chromosomal abnormality” in their CAR-T cells. The biotech $ALLO reported after the market close on Thursday that executives plan to follow up in the coming weeks with regulators as the FDA considers the biotech’s plan to take this drug into a pivotal trial. Keep reading Endpoints with a free subscription Unlock this story instantly and join 119,200+ biopharma pros reading Endpoints daily — and it's free. SIGN UP Jason Mast Editor ARCH Venture is doubling — and tripling and quadrupling — down on the belief that the 2020s will be neuroscience’s decade. The VC firm, known for periodically launching massive companies from scratch, announced on Thursday the creation of Neumora, a biotech ARCH’s partners believe can finally crack a set of well-known neurodegenerative and psychiatric diseases that have long eluded drugmakers. The company, whose existence was first reported by Endpoints News in August, emerges from stealth with $500 million in the bank — $400 million from ARCH and other VCs and $100 million equity investment as part of a collaboration with Amgen. Keep reading Endpoints with a free subscription Unlock this story instantly and join 119,200+ biopharma pros reading Endpoints daily — and it's free. SIGN UP Zachary Brennan Senior Editor President Biden told reporters yesterday that he’s close to nominating someone to be the next FDA commissioner, but it still remains unclear who he might select. With a little more than a month to make his nomination, experts and Politico pointed yesterday to Dana-Farber Cancer Institute CEO Laurie Glimcher as a top candidate, given her leadership position and drug development experience. But Glimcher, an immunologist by training who also serves on GlaxoSmithKline’s board, told Endpoints News via email on Thursday: “I am committed to Dana-Farber Cancer institute and our ongoing progress against cancer, and I am not a candidate to be the next commissioner of the FDA.” Keep reading Endpoints with a free subscription Unlock this story instantly and join 119,200+ biopharma pros reading Endpoints daily — and it's free. SIGN UP GlaxoSmithKline is radically downsizing — and the move has nothing to do with their head count. Spurred by the pandemic-inspired reduction in the need for office space, the pharma giant is giving up its iconic facilities in Philadelphia and Research Triangle Park, and replacing the space with new digs that require only a fraction of what they once used. Starting early next year, GlaxoSmithKline will be shrinking its office space in Philly by 75% as the pharma leaves their bespoke building in the Navy Yard — moving to the FMC Tower in Philly’s University City district. The company is also moving its offices from RTP to downtown Durham, NC by June, the company said — a downsize of close to 90%. Keep reading Endpoints with a free subscription Unlock this story instantly and join 119,200+ biopharma pros reading Endpoints daily — and it's free. SIGN UP Josh Sullivan Associate Editor In 2019, High Purity New England, a biopharma solutions and equipment supplier and distributor based out of Smithfield, Rhode Island, had just 60 employees. Thanks to a push in demand, largely fueled by the pandemic, it hopes it can end the year 2022 with 350. The hiring push is all part of the company’s plan to open a new facility in December, a $10 million project that would triple its manufacturing output and bring its total square footage to more than 100,000. All three buildings will be located on the same street, and the new site will be used to create a sixth clean room for manufacturing, a distribution center, a lab and more office space. Keep reading Endpoints with a free subscription Unlock this story instantly and join 119,200+ biopharma pros reading Endpoints daily — and it's free. SIGN UP Kyle Blankenship Managing Editor Bristol Myers Squibb’s deucravacitinib, seeking to become the first TYK2 drug approved, flopped a key Phase II test in ulcerative colitis, failing to spur clinical remission at three months and missing key secondary endpoints at an interim check-in, the drug giant admitted Thursday. Details were slim in a press release, but Bristol Myers noted it still has plans to pursue deucravacitinib in ulcerative colitis — another Phase II test is currently ongoing — as well as a host of other indications for what it still sees as a $4 billion-per-year peak seller by the end of the decade. Keep reading Endpoints with a free subscription Unlock this story instantly and join 119,200+ biopharma pros reading Endpoints daily — and it's free. SIGN UP LOG IN