Cel­gene part­ners on Phase 0 can­cer drug stud­ies; FDA lifts clin­i­cal hold on Xen­cor's bis­pe­cif­ic

→  Cel­gene is ty­ing up with Seat­tle-based Presage Bio­sciences to test its ear­ly-stage can­cer drugs on pa­tients in Phase 0 stud­ies. In Phase 0, pa­tients are giv­en mi­cro­dos­es of a drug to test pa­tient re­spons­es. It’s one way to help in­crease the odds of suc­cess, and help over­come the in­her­ent lim­i­ta­tions of an­i­mal stud­ies.

Hide­ho Oka­da

Tmu­ni­ty and the Uni­ver­si­ty of Cal­i­for­nia San Fran­cis­co will en­ter a li­cens­ing and re­search col­lab­o­ra­tion to ad­vance TCR ther­a­pies for cur­rent­ly in­cur­able pe­di­atric can­cers with high mor­tal­i­ty rates. The com­pa­ny says that ini­tial re­search will fo­cus on dif­fuse in­trin­sic pon­tine glioma (DIPG), a rare and high­ly ag­gres­sive brain stem tu­mor, which af­fects around 300 chil­dren in the US each year. Hide­ho Oka­da, a pro­fes­sor of neu­ro­log­i­cal surgery at UCSF who de­vised the TCR at the cen­ter of the pact, will work with Tmu­ni­ty through IND-en­abling stud­ies.

“This col­lab­o­ra­tion with UCSF em­bod­ies our com­mit­ment to ad­vance nov­el T cell ther­a­pies in­to the clin­ic faster. Sad­ly, chil­dren who are di­ag­nosed with DIPG cur­rent­ly have very few ther­a­peu­tic op­tions and there has been lit­tle progress in im­prov­ing treat­ments and over­all sur­vival rates for DIPG over the last few decades,” said CEO Us­man “Oz” Azam, adding that their new ther­a­py is al­so a po­ten­tial break­through for oth­er gliomas.

→ More than two months af­ter the FDA or­dered a par­tial hold on a Phase I study of Xen­cor’s CD123 x CD3 bis­pe­cif­ic, XmAb14045, the biotech says it can now re­sume re­cruit­ment of pa­tients. Reg­u­la­tors lift­ed the hold, ap­par­ent­ly sat­is­fied with an amend­ed pro­to­col that now fea­tures guid­ance on the mon­i­tor­ing and clin­i­cal man­age­ment of cy­tokine re­lease syn­drome — which caused one of the two deaths that in­ves­ti­ga­tors tracked in the study. The oth­er pa­tient died from acute pul­monary ede­ma.

→ Swiss biotech AMAL Ther­a­peu­tics is test­ing its can­cer vac­cine with Boehringer In­gel­heim’s PD-1, BI754091, in a Phase Ib tri­al that will re­cruit Mi­crosatel­lite Sta­ble (MSS) pa­tients with stage IV col­orec­tal can­cer. The idea is to see how ATP128 — which com­pris­es a cell pen­e­trat­ing pep­tide for anti­gen de­liv­ery, a TLR pep­tide ag­o­nist and a mul­ti­ple anti­genic do­main — per­forms both as a sin­gle agent and in com­bo. Boehringer is pro­vid­ing its drug for joint own­er­ship of the re­sults.

→ Sin­ga­pore’s tech­nol­o­gy agency has en­ticed a Hong Kong-based phar­ma­ceu­ti­cal group called Ap­to­rum to es­tab­lish a ven­ture fund catered to health care star­tups in Sin­ga­pore. To­geth­er, they will raise $90 mil­lion to fund up to 20 star­tups, some of which have al­ready been iden­ti­fied as com­pa­nies work­ing on 2D and 3D mag­net­ic res­o­nance imag­ing and sur­gi­cal ro­bot­ics. Ae­neas Cap­i­tal, which is al­so con­trolled by Ap­to­rum co-founder Ian Huen, will al­so chip in. 

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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Gala­pa­gos posts a safe­ty win for fil­go­tinib, but is it too lit­tle, too late?; Bio-Techne inks $320M mol­e­c­u­lar di­ag­nos­tics buy­out

Once a promising $725 million play in immunology, Gilead’s big bet on filgotinib effectively disintegrated in December when the drugmaker reworked its partnership with Galapagos. Now, Galapagos is sporting safety data that will come as a relief — but will it make a difference on filgotinib’s chances in the US?

In a study designed to compare filgotinib’s effect on sperm count with placebo, Galapagos’ JAK inhibitor saw fewer patients post a 50% or more reduction in sperm concentration after 13 weeks of treatment, according to data from the MANTA and MANTA-RAy studies unveiled Thursday.

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.

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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Hal Barron, Endpoints UKBIO19

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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