Cataba­sis Phar­ma­ceu­ti­cals snaps up HAE an­ti­body in buy­out; Glad­stone, UCSF sci­en­tist spear­head launch of neu­ro re­search cen­ter

A few months af­ter fi­nal­ly ad­mit­ting de­feat on their failed Duchenne MD pro­gram, Cataba­sis Phar­ma­ceu­ti­cals is head­ing in a new di­rec­tion with the pur­chase of Quel­lis Bio­sciences and their lead an­ti­body for hered­i­tary an­gioede­ma.

Cataba­sis shared the news on Fri­day that they’re buy­ing out Quel­lis, a rare dis­ease-fo­cused biotech that was un­veiled over a year ago by Chris Garabe­di­an and Per­cep­tive Ad­vi­sors’ PXV Fund. With it, Cataba­sis is snap­ping up QLS-215, a mon­o­clon­al an­ti­body in­hibitor of plas­ma kallikrein. The can­di­date is cur­rent­ly in pre­clin­i­cal de­vel­op­ment — but won’t be for long, ac­cord­ing to Cataba­sis.

In ad­di­tion to the buy­out, the Boston-based biotech closed a $110 mil­lion pri­vate place­ment to fund the com­ple­tion of IND-en­abling, Phase Ia and Phase Ib/II stud­ies.

Cataba­sis and Quel­lis are keep­ing the terms of their deal un­der wraps. But af­ter the ac­qui­si­tion and pri­vate place­ment, Cataba­sis ex­pects to have a cash run­way through 2023.

The com­pa­ny plans on fil­ing an IND ap­pli­ca­tion for QLS-215 in the first half of 2022, and read­ing out ini­tial Phase I re­sults by the end of 2022. If all goes well, they hope to launch a Phase Ib/II tri­al in 2023, with ini­tial re­sults by the end of that year.

Back in Oc­to­ber, Cataba­sis ac­knowl­edged a fi­nal fail­ure for its Duchenne MD drug edasa­lonex­ent. Three years ear­li­er, CEO Jill Milne and the crew in­sist­ed they found rea­son for hope in da­ta from a failed study, and that it jus­ti­fied a try in Phase III. Af­ter the pri­ma­ry, change in base­line on the North Star Am­bu­la­to­ry As­sess­ment, and the sec­ondary on timed func­tion tests both came up short of sta­tis­ti­cal sig­nif­i­cance, Cataba­sis killed the ef­fort — leav­ing them with one oth­er drug in the pipeline, a pre­clin­i­cal pro­gram on au­tophagy.

Glad­stone, UCSF sci­en­tist spear­head launch of neu­ro re­search cen­ter

Drug hunters and de­vel­op­ers work­ing in neu­ro­log­i­cal dis­eases will now have a new place to turn to for in­sights.

The Glad­stone-UCSF Cen­ter for Neu­rovas­cu­lar Brain Im­munol­o­gy is launch­ing with a $2.5 mil­lion do­na­tion and neu­roim­mu­nol­o­gist Ka­te­ri­na Akas­soglou as the found­ing di­rec­tor.

“His­tor­i­cal­ly, neu­ro­log­i­cal dis­eases have been clas­si­fied as be­ing on­ly de­gen­er­a­tive, in­flam­ma­to­ry, or vas­cu­lar,” she said in a state­ment. “But we now know, giv­en re­cent in­sights from clin­i­cal re­search and the fail­ure of many clin­i­cal tri­als, that this clas­si­fi­ca­tion can­not ex­plain how dis­eases start and progress, nor has it been able to iden­ti­fy the best po­ten­tial drug can­di­dates.”

By in­te­grat­ing ba­sic and clin­i­cal re­search on vas­cu­lar bi­ol­o­gy, im­munol­o­gy and neu­ro­science, sci­en­tists at the new cen­ter aim to shed light on over­lap­ping mech­a­nisms be­hind dis­eases like mul­ti­ple scle­ro­sis, Alzheimer’s dis­ease and trau­mat­ic brain in­jury — and have im­pli­ca­tions ex­tend­ing as far as neona­tal brain in­jury, epilep­sy, in­fec­tious dis­eases and psy­chi­atric dis­or­ders.

Add it all to­geth­er, Akas­soglou said, and their ap­proach­es in imag­ing and drug dis­cov­ery may just “trans­form bio­med­i­cine.”

To un­cov­er the role of vas­cu­lar ab­nor­mal­i­ties in the brain, they will bring to­geth­er ba­sic and clin­i­cal ex­perts and de­vel­op new mol­e­c­u­lar di­ag­nos­tics, while test­ing new ther­a­pies and bio­mark­ers.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,700+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,700+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,700+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,700+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,700+ biopharma pros reading Endpoints daily — and it's free.