Al­ler­gan/Ed­i­tas fi­nal­ly kick off CRISPR tri­al — mark­ing first in vi­vo test of the gene-edit­ing tech­nol­o­gy

More three years af­ter Al­ler­gan $AGN dipped its toes in­to the world of CRISPR/Cas9 gene edit­ing by en­list­ing Ed­i­tas $ED­IT as a part­ner — the com­pa­nies are now off to the races with a tri­al in pa­tients with a rare eye dis­ease.

This tri­al marks the first in­stance of us­ing the tech­nol­o­gy to ed­it DNA with­in the body.

Mean­while, part­ners Ver­tex $VRTX and CRISPR Ther­a­peu­tics $CR­SP kick­start­ed a tri­al ear­li­er this year that em­ployed the tech­nol­o­gy to en­gi­neer a gene-edit­ed stem cell ther­a­py to treat pa­tients suf­fer­ing from se­vere he­mo­glo­binopathies, a group of blood dis­or­ders. Sang­amo Ther­a­peu­tics $SG­MO has test­ed its old­er zinc fin­ger tech­nol­o­gy in pa­tients with two lyso­so­mal stor­age dis­eases, but a set­back dif­fused en­thu­si­asm for the pro­gram — but the com­pa­ny bounced back with en­cour­ag­ing ear­ly da­ta on its he­mo­phil­ia pro­gram part­nered with Pfiz­er.

Al­ler­gan and Ed­i­tas had orig­i­nal­ly ex­pect­ed to file an ap­pli­ca­tion to test their ther­a­py in hu­mans by the end of 2017, but man­u­fac­tur­ing trou­ble pushed that time­line to Oc­to­ber 2018. To get the FDA en­dorse­ment, Ed­i­tas fought off a stiff patent chal­lenge — and like its peers — bat­ted back per­sis­tent ques­tions about off-tar­get ef­fects to ad­e­quate­ly con­vince the reg­u­la­tor it was armed and ready to make the jump from an­i­mal stud­ies to hu­mans.

The tri­al, chris­tened Bril­liance, will eval­u­ate their ex­per­i­men­tal ther­a­py AGN-151587 (ED­IT-101) for the treat­ment of Leber con­gen­i­tal amau­ro­sis 10 (LCA10), a rare, in­her­it­ed form of blind­ness caused by mu­ta­tions in the CEP290 gene.

LCA is a group of in­her­it­ed reti­nal de­gen­er­a­tive dis­or­ders caused by mu­ta­tions in at least 18 dif­fer­ent genes, and are con­sid­ered the most com­mon cause of in­her­it­ed child­hood blind­ness, oc­cur­ring in two to three per 100,000 live births glob­al­ly. The most com­mon form of the dis­ease, LCA10, ac­counts for ap­prox­i­mate­ly 20% to 30% of all LCA pa­tients.

Ka­trine Bosley Ed­i­tas

Spark Ther­a­peu­tics’ ap­proved gene ther­a­py Lux­tur­na, which de­liv­ered via a vi­ral vec­tor, is tai­lored to treat an­oth­er rare eye dis­ease due to mu­ta­tions in both copies of the RPE65 gene. The FDA nod sparked Roche’s $4.3 bil­lion takeover of the com­pa­ny, al­though the deal is yet to be con­sum­mat­ed.

The Bril­liance study is set to en­roll 18 pa­tients — and in­clude up to five co­horts and eval­u­ate three dos­es of the drug. Each pa­tient will re­ceive a sin­gle dose of AGN-151587 ad­min­is­tered via an eye in­jec­tion aimed at pho­tore­cep­tor cells in the reti­na.

Ed­i­tas’ se­nior team has seen up­heaval in the past year. In Jan­u­ary, CEO Ka­trine Bosley, who led the com­pa­ny for near­ly five years, an­nounced her ex­it and sev­ered ties with the board, with­out ex­pla­na­tion. Chief fi­nan­cial of­fi­cer An­drew Hack an­nounced his de­ci­sion to leave by March, back in De­cem­ber 2018, and chief med­ical of­fi­cer Ger­ald Cox jumped ship at the end of last year.

Be­set by set­backs, Al­ler­gan’s Brent Saun­ders made the best of a bad sit­u­a­tion by agree­ing to be swal­lowed by Ab­b­Vie $AB­BV in an eye-wa­ter­ing $63 bil­lion deal ear­li­er this year. The US drug­mak­er was swayed by the po­ten­tial of Al­ler­gan’s block­buster Botox fran­chise to sta­bi­lize its rev­enue as its $20 bil­lion cash cow Hu­mi­ra hur­tles to­wards a patent cliff in the Unit­ed States.

So­cial im­age: Shut­ter­stock

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

Talk about your bad timing.

A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

And then, just days later, one of the cancer drugs he bet big on as a top prospect — bintrafusp, partnered with Merck KGaA — failed its first pivotal test in non-small cell lung cancer.

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Fast on Glax­o­SmithK­line's heels, Au­rinia wins OK to steer a sec­ond lu­pus nephri­tis drug straight to the mar­ket

GlaxoSmithKline’s Benlysta isn’t alone in the small circle of approved lupus nephritis drugs anymore.

Little Aurinia Pharmaceuticals has gotten the green light from the FDA to start marketing its first and only program, voclosporin, under the brand name Lupkynis — something CEO Peter Greenleaf says it’s been ready to do since December.

Regulators went right down to the wire on the decision, keeping the company and the entire salesforce it’s already assembled on its toes.

Eli Lil­ly's an­ti­body cuts risk of Covid-19 by up to 80% among the most vul­ner­a­ble — but will it have a place next to vac­cines?

Eli Lilly says bamlanivimab lowered the risk of contracting symptomatic Covid-19 in a first-of-its-kind trial involving nursing home residents and staff, paving the way for a new option to protect against the virus.

But how big of an impact it might have, and what role it will play, at a time vaccines are being rolled out to the exact population it is targeting still remains unclear.

Among 965 participants in the study — all of whom tested negative for the coronavirus at baseline — the number of symptomatic cases reported in the bamlanivimab arm was 57% lower than that in the placebo arm (odds ratio 0.43, p=0.00021). In addition to that primary endpoint, all secondary endpoints reached statistical significance.

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Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

It’s official: Janet Woodcock is now the acting chief of the FDA.

And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed.

To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come.

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What’s next for End­points — and how to sup­port our in­de­pen­dent bio­phar­ma news mis­sion

The firehose of biopharma news is gushing these days.

That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.

Charlie Fuchs, Roche and Genentech global head of product development for oncology and hematology (Yale Cancer Center)

Yale can­cer spe­cial­ist Char­lie Fuchs tapped as new glob­al de­vel­op­ment chief for Roche/Genen­tech

Roche and their big sub Genentech have just recruited a top cancer specialist at Yale to head up global product development in oncology and hematology.

I just got word that the pharma giant, which leads one of the most active cancer research operations in the world, recruited Charlie Fuchs, director of the Yale Cancer Center and physician-in-chief of Smilow Cancer Hospital. He’ll join the global operation March 1 and will be based in South San Francisco, where Genentech is based.

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Jonathan Weissman (MIT)

Can a new CRISPR tech­nique un­lock the se­crets of how can­cer spreads?

Jonathan Weissman’s team watched the cancer cells spread across the doomed mouse. Engineered with a bioluminescent enzyme, they appeared in scans first as a small navy blue diamond lodged near the heart; a week later, as a triangle splayed across the mouse’s upper body, with streaks of green and two distinct bright red hubs of activity. By day 54, the mouse resembled a lava lamp.

The images would have been familiar to any cancer biologist, but they didn’t actually tell you much about what was going on: why the cancer was metastasizing or which cells were responsible. For that, Weissman’s team had designed a new tool. Inside the original navy blue diamond, they had engineered the microbiological equivalent of an airplane’s black box — a “molecular recorder” that, after the mouse’s death, could allow them to extract the cells and wind back intimate footage of a single cancer’s ascent.

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Glax­o­SmithK­line scraps a LAG3 study, mark­ing an­oth­er fail­ure for the pipeline af­ter a crit­i­cal set­back

Another gap has appeared in GlaxoSmithKline’s pipeline.

Friday morning the Australian biotech Immutep put out word that Hal Barron’s R&D group at GSK had decided to scrap a Phase II proof-of-concept study in ulcerative colitis for their anti-LAG3 therapy GSK2831781. According to the biotech, the program didn’t survive an interim review.

The trial was stopped by GSK based on the assessment of clinical data as part of a planned interim analysis conducted in consultation with the trial’s Data Review Committee. GSK is conducting further reporting, assessment and analyses of the efficacy and safety data and evaluating the biology to determine next steps for the GSK2831781 development program.

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