Bris­tol My­ers Squibb turns in pri­or­i­ty re­view vouch­er for sup­ple­men­tal in­di­ca­tion on an old Cel­gene drug

Af­ter turn­ing around Cel­gene’s S1P mod­u­la­tor for a long-await­ed ap­proval in mul­ti­ple scle­ro­sis, Bris­tol My­ers Squibb is now trad­ing in an FDA wild­card to get a quick re­view in ul­cer­a­tive col­i­tis.

BMS has re­deemed a pri­or­i­ty re­view vouch­er for a speedy de­ci­sion on its sup­ple­men­tal NDA for Zeposia, which hit the mar­ket this past June for MS. The FDA set a PDU­FA date for May 30 — and if giv­en the thumbs up, BMS says it would have the first oral sphin­go­sine-1-phos­phate (S1P) re­cep­tor mod­u­la­tor for UC.

The sub­mis­sion was based on re­sults from the Phase III True North study, in which Zeposia met both pri­ma­ry end­points in adults with mod­er­ate­ly to se­vere­ly ac­tive UC: the pro­por­tion of pa­tients in clin­i­cal re­mis­sion based at week 10 in the in­duc­tion phase, and at week 52 for the main­te­nance phase. Zeposia’s ef­fect was high­ly sta­tis­ti­cal­ly sig­nif­i­cant (p-val­ue < 0.0001) the com­pa­ny said in June, adding that the drug al­so met key sec­ondary end­points.

The over­all safe­ty pro­file was con­sis­tent with that of the al­ready ap­proved in­di­ca­tion, BMS said in a state­ment.

UC is a chron­ic in­flam­ma­to­ry bow­el dis­ease char­ac­ter­ized by an ab­nor­mal, pro­longed im­mune re­sponse that cre­ates long-last­ing in­flam­ma­tion and ul­cers in the lin­ing of the large in­tes­tine. Zeposia works in MS by re­duc­ing the ca­pac­i­ty of lym­pho­cytes to ex­it lymph nodes, thus re­duc­ing the num­ber of cir­cu­lat­ing lym­pho­cytes in pe­riph­er­al blood. While the mech­a­nism of ac­tion in UC is still un­clear, BMS thinks it may in­volve the re­duc­tion of lym­pho­cyte mi­gra­tion in­to the in­flamed in­testi­nal mu­cosa, or lin­ing.

BMS snagged Zeposia when it bought out Cel­gene for $74 bil­lion back in 2019. The drug, chem­i­cal­ly known as ozan­i­mod, had been side­lined af­ter Cel­gene was flagged for lame con­duct in fil­ing a drug ap­pli­ca­tion. The FDA slapped Cel­gene with a refuse-to-file let­ter, con­clud­ing that the “non­clin­i­cal and clin­i­cal phar­ma­col­o­gy sec­tions in the NDA were in­suf­fi­cient to per­mit a com­plete re­view.” BMS flipped the drug around for an OK in March, mak­ing it the third oral S1P mod­u­la­tor ap­proved for MS.

The com­pa­ny held back on com­mer­cial­iza­tion un­til June, due to the Covid-19 pan­dem­ic. Now time is of the essence, as they race for mar­ket­ing ap­proval in UC.

Pri­or­i­ty re­view vouch­ers are, in some ways, like a reg­u­la­to­ry “I owe you”: They al­low com­pa­nies to have one of their drugs fast-tracked un­der the FDA’s pri­or­i­ty re­view sys­tem. The idea of us­ing a vouch­er sys­tem was first pro­posed as a way to in­cen­tivize the de­vel­op­ment of drugs for ne­glect­ed trop­i­cal dis­eases. The first ver­sion was in­cor­po­rat­ed in the Food and Drug Ad­min­is­tra­tion Amend­ments Act (FDAAA) of 2007. Since then, guid­ance has been is­sued cov­er­ing more in­di­ca­tions, and al­low­ing com­pa­nies to buy and sell vouch­ers.

Robert Bradway (Photographer: Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Am­gen snaps up can­cer drug play­er Five Prime, adding PhI­II-ready FGFR2b drug in $2B M&A play

Amgen is making a long-awaited move on the M&A side, buying South San Francisco-based Five Prime $FPRX for close to $2 billion and adding a slate of new cancer drugs to the pipeline.

Amgen is paying $38 a share, putting the deal value at $1.9 billion. The stock closed at $21.26 last night, giving investors a 78% premium.

The jewel in the crown of this deal is bemarituzumab, which Amgen describes as a first-in-class, Phase III-ready anti-FGFR2b antibody. Amgen was drawn to the bargaining table by Five Prime’s mid-stage data on gastric cancer, satisfied by PFS and OS data helping to validate FGFR2b as a target. Amgen researchers will now expand on the R&D program in other epithelial cancers, including lung, breast, ovarian and other cancers.

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David Liu (Casey Atkins Photography courtesy Broad Institute)

David Liu has a new big idea: pro­teome edit­ing. It could one day shred tau, RAS and some of the worst dis­ease-caus­ing pro­teins

Before David Liu became famous for inventing new forms of gene editing, he was known around academia in part for a more obscure innovation: a Rube Goldberg-esque system that uses bacteria-infecting viruses to take one protein and turn it into another.

Since 2011, Liu’s lab has used the system, called PACE, to dream up fantastical new proteins: DNA base editors far more powerful than the original; more versatile forms of the gene editor Cas9; insecticides that kill insecticide-resistant bugs; enzymes that slide synthetic amino acids into living organisms. But they struggled throughout to master one of the most common and powerful proteins in the biological world: proteases, a set of Swiss army knife enzymes that cut, cleave or shred other proteins in everything from viruses to humans.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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Pfiz­er's Xalko­ri fol­low-up, which smoked the old­er drug in 1st-line pa­tients, scores dou­ble win at the FDA

Despite being the first to market in ALK-positive non-small cell lung cancer, Pfizer’s Xalkori languished on the shelf as other competitors outpaced it. Now, after Pfizer’s follow-up drug absolutely smoked Xalkori in first-line patients, the FDA is taking notice.

In one fell swoop, the agency approved Lorbrena as a first-line treatment for patients with anaplastic lymphoma kinase (ALK)-positive NSCLC and extended the drug’s 2018 accelerated approval to a full approval, Pfizer announced on Wednesday.

In the lat­est big in­vest­ment in gene ther­a­py man­u­fac­tur­ing, Bio­gen com­mits $200M to a ma­jor new fa­cil­i­ty in NC

You’d be forgiven for thinking that the only R&D effort of any consequence at Biogen belongs to aducanumab, its controversial Alzheimer’s drug. But behind the uproar around that drug, the big biotech has a full scale pipeline in play that includes a growing focus on developing gene therapies.

Now Biogen plans to build up the kind of manufacturing muscle that will give it an advantage in gaining FDA approvals — where CMC is always key — and then marketing them around the world.

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Eli Lil­ly claims a TKO in its long-run­ning ti­tle fight with No­vo Nordisk for the block­buster di­a­betes mar­ket — but there’s a hitch

Eli Lilly isn’t just gunning for a better diabetes drug in tirzepatide. They want to cut ahead of Novo Nordisk’s blockbuster rival Ozempic (semaglutide) on the obesity front as well. But a newly-claimed win in a head-to-head Phase III showdown over reducing A1C while shedding pounds — complete with clear evidence of superiority over the approved rival — could prove a tough sell right now.

Let’s start with the latest data from Lilly.

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In­tro­duc­ing End­points FDA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.

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GSK, Vir's hopes for a Covid-19 an­ti­body fall flat in NIH 'mas­ter pro­to­col' with no ben­e­fit in hos­pi­tal­ized pa­tients

GlaxoSmithKline and Vir Biotechnology were hopeful that one of their partnered antibodies would carve out a win after getting the invite to a major NIH study in hospitalized Covid-19 patients. But just like Eli Lilly, the pair’s drug couldn’t hit the mark, and now they’ll be left to take a hard look at the game plan.

The NIH has shut down enrollment for GSK and Vir’s antibody VIR-7831 in its late-stage ACTIV-3 trial after the drug showed negligible effect in achieving sustained recovery in hospitalized Covid-19 patients, the partners said Wednesday.

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