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In one of the riski­est in­dus­tries — Biotech — a sure­fire way to neu­tral­ize some of that risk emerges
top stories
1. CRISPR Therapeutics' gene editing therapy reduces bad cholesterol and triglycerides by as much as 80%
2. Theriva’s oncolytic virus succeeds in pancreatic cancer study, but stock falls
3. Charles River launches ‘strategic review’ alongside activist investor Elliott
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8. Marea heads to Phase 2b after updated biomarker data show CV drug’s promise
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Jaimy Lee
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Join us next week for the US Pharma and Biotech Summit in New York. There will be a wide range of panel discusions, including conversations with Bristol Myers Squibb CEO Chris Boehrner and RFK Jr. advisor Calley Means. Register here!

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Jaimy Lee
Deputy Editor, Endpoints News
Credit: Amanda Sabga for Endpoints News
1
by Ryan Cross

CRISPR Ther­a­peu­tics has re­vealed promis­ing but ear­ly da­ta from its first clin­i­cal study of a gene edit­ing ther­a­py to low­er the risk of heart dis­ease, set­ting the stage for com­pe­ti­tion with oth­er drug­mak­ers work­ing on sim­i­lar treat­ments.

The ther­a­py, dubbed CTX310, us­es lipid nanopar­ti­cles (LNPs) to de­liv­er the CRISPR ther­a­py in­to the liv­er, where it cuts a gene called ANGPTL3. The ap­proach aims to mim­ic the ef­fects of nat­ur­al mu­ta­tions that cause some peo­ple to have un­usu­al­ly low lev­els of cho­les­terol and triglyc­erides.

The Phase 1 tri­al test­ed four dose lev­els in 10 peo­ple, but the most dra­mat­ic re­sults came from the sole pa­tient who got the high­est dose, which re­duced their triglyc­eride lev­els by 82% and low-den­si­ty lipopro­tein (LDL) — of­ten called “bad" cho­les­terol — by 65% a month af­ter the in­fu­sion.

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2
by Lei Lei Wu

Theri­va Bi­o­log­ic­s' ex­per­i­men­tal on­colyt­ic virus treat­ment and chemother­a­py helped pa­tients in a mid-stage study stay alive longer than chemother­a­py alone.

The com­pa­ny said Wednes­day that the Phase 2b study in­clud­ed 96 pa­tients with new­ly di­ag­nosed metasta­t­ic pan­cre­at­ic can­cer. Half re­ceived Theri­va’s VCN-01 plus chemother­a­py, while the oth­er half re­ceived just chemother­a­py.

Those who re­ceived Theri­va’s drug and chemother­a­py lived for a me­di­an of 10.8 months, while those who re­ceived chemother­a­py alone sur­vived for a me­di­an of 8.6 months. The p-val­ue was 0.0546, which was sta­tis­ti­cal­ly sig­nif­i­cant since the study was de­signed with a sta­tis­ti­cal sig­nif­i­cance thresh­old of 0.1. Manel Cas­cal­ló, Theri­va’s EU sub­sidiary gen­er­al di­rec­tor, told End­points News that’s be­cause Phase 2 tri­als in­clude a small­er num­ber of pa­tients com­pared to Phase 3 stud­ies.

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3
by Anna Brown

Charles Riv­er Lab­o­ra­to­ries is set to start a strate­gic re­view and add four new board mem­bers af­ter it set­tled on a “co­op­er­a­tion agree­ment” with El­liott In­vest­ment Man­age­ment.

The Wednes­day an­nounce­ment comes as the FDA said last month it was mov­ing away from us­ing an­i­mal mod­els in drug de­vel­op­ment, which makes up around 20% of Charles Riv­er’s to­tal rev­enue.

“We be­lieve that Charles Riv­er’s cur­rent val­ue is sig­nif­i­cant­ly dis­con­nect­ed from its un­der­ly­ing po­ten­tial, and as Charles Riv­er’s largest in­vestor, we see a sub­stan­tial op­por­tu­ni­ty to un­lock that val­ue,” El­liott part­ner Marc Stein­berg said in the re­lease.

The ac­tivist in­vestor has tak­en a stake in nu­mer­ous phar­ma and