Lisa Deschamps, AviadoBio CEO

Ex-No­var­tis busi­ness head hops over to a gene ther­a­py start­up — and she's reeled in $80M for a dash to the clin­ic

Neu­rol­o­gist and King’s Col­lege Lon­don pro­fes­sor Christo­pher Shaw has been re­search­ing neu­rode­gen­er­a­tive dis­eases like ALS and col­lab­o­rat­ing with drug­mak­ers for the last 25 years in the hopes of push­ing new ther­a­pies for­ward. But un­for­tu­nate­ly, none of those ef­forts have come any­where close to fruition.

“So, you know, af­ter 20 years in the game, I said, ‘Let’s try and do it our­selves,’” he told End­points News. 

In 2019, he launched Avi­ado­Bio along with mol­e­c­u­lar neu­ro­bi­ol­o­gist Youn Bok Lee and vec­tor bi­ol­o­gist Do Young Lee to pur­sue a new take on gene ther­a­py for neu­ro dis­or­ders. And on Thurs­day, they un­wrapped an $80 mil­lion Se­ries A round led to bring their lead can­di­date for fron­totem­po­ral de­men­tia (FTD) in­to the clin­ic.

Christo­pher Shaw

“I was an in­stant be­liev­er,” said Lisa De­schamps, who left her role as CBO of No­var­tis Gene Ther­a­pies to take the helm at Avi­ado­Bio in Oc­to­ber.

The team is tar­get­ing dis­eases that have an ag­gres­sive ear­ly pro­gres­sion, Shaw said. While most of their com­peti­tors are putting vec­tors in­to the cere­brospinal flu­id — a liq­uid found with­in the tis­sue that sur­rounds the brain and spinal cord —  Avi­ado­Bio is look­ing to de­liv­er the vec­tors right to the brain.

“We get fan­tas­tic ex­pres­sion by do­ing that,” Shaw said. “It al­so al­lows us to mi­cro­dose — we’re giv­ing about a thou­sandth of the dose of our com­peti­tors.”

Their lead can­di­date, AVB-PGRN, and an in­trathal­a­m­i­cal­ly dosed ther­a­py de­signed to de­liv­er a func­tion­al copy of the pro­gran­ulin (GRN) gene. FTD is caused by a de­fi­cien­cy of pro­gran­ulin, which plays a role in lyso­so­mal func­tion, or the pro­tein re­cy­cling part of the cell.

“Neu­rons are in­cred­i­bly de­pen­dent on hav­ing good re­cy­cling be­cause the neu­rons you die with are the neu­rons you’re born with,” Shaw said. “So you know, they can’t be ado­les­cents. They can’t have a messy room. They’ve got to have a re­al­ly, re­al­ly crisp, tidy space … So what we’re try­ing to do is just re­turn back to phys­i­o­log­i­cal lev­els this re­al­ly im­por­tant re­cy­cling pro­tein.”

Avi­ado­Bio will com­pete with a hand­ful of play­ers work­ing on po­ten­tial treat­ments for FTD, in­clud­ing GSK-part­nered Alec­tor, which un­veiled da­ta this sum­mer show­ing its an­ti­body AL001 brought pro­gran­ulin lev­els back to near-nor­mal lev­els af­ter a half-a-year. And Ex­pan­sion Ther­a­peu­tics un­veiled an $80 mil­lion Se­ries B round just a cou­ple months ago to bring its small mol­e­cule RNA plat­form one step clos­er to the clin­ic – in­clud­ing a can­di­date for FTD.

De­schamps said Avi­ado­Bio’s can­di­date should en­ter the clin­ic by the end of next year, while an­oth­er pre­clin­i­cal can­di­date for ALS is a bit fur­ther be­hind. The Se­ries A round was led by New En­ter­prise As­so­ci­ates and Mono­graph Cap­i­tal. A hand­ful of big-name back­ers pitched in­to a $16.5 mil­lion seed round last year, in­clud­ing Ad­vent Life Sci­ences, De­men­tia Dis­cov­ery Fund (DDF), F-Prime Cap­i­tal, John­son & John­son In­no­va­tion (JJDC), and LifeArc.

De­spite Big Phar­ma’s re­treat from neu­ro a few years ago, the in­dus­try seems to be warm­ing back up to the field, De­schamps said — and new com­pa­nies are emerg­ing with gene ther­a­pies to take a shot at dif­fi­cult-to-treat con­di­tions. This past sum­mer, for ex­am­ple, Al­cy­one Ther­a­peu­tics emerged from stealth with $23 mil­lion and 12 gene ther­a­pies in tow, with a rare neu­rode­vel­op­men­tal con­di­tion called Rett syn­drome up first.

“I was lead­ing the glob­al neu­ro­science busi­ness at No­var­tis … one of the things we had talked about years ago was how many Big Phar­ma com­pa­nies were ex­it­ing the space. It is quite the op­po­site these days,” De­schamps said. “We see cer­tain­ly a resur­gence across big and small com­pa­nies, you know, com­ing back in­to the neu­ro­science space.”

Dominic Borie, Kyverna Therapeutics CEO

Well-con­nect­ed, Gilead-backed biotech gets an­oth­er stack of cash to pur­sue CAR-Ts for au­toim­mune dis­ease

Almost exactly two years after its debut at the 2020 JP Morgan confab — and on the heels of a new partnership with the gene editing experts at Intellia — a Gilead-backed, autoimmune disease-focused startup has returned to the well with a clearer outline of just what it plans to do with its CAR-T platform.

Kyverna brought in $85 million in its oversubscribed Series B, the company announced Wednesday. Northpond Ventures led the round, and Westlake Village BioPartners, Vida Ventures, Gilead and Intellia all contributed as well.

Alexander Lefterov/Endpoints News

A new can­cer im­munother­a­py brings cau­tious hope for a field long await­ing the next big break­through

Bob Seibert sat silent across from his daughter at their favorite Spanish restaurant near his home in Charleston County, SC, their paella growing cold as he read through all the places in his body doctors found tumors.

He had texted his wife, a pediatric intensive care nurse, when he got the alert that his online chart was ready. Although he saw immediately it was bad, many of the terms — peritoneal, right iliac — were inscrutable. But she was five hours downstate, at a loud group dinner the night before another daughter’s cheer competition.

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Bahija Jallal, Immunocore CEO

BREAK­ING: FDA hur­ries up a quick ap­proval for the world's first TCR -- af­ter a 14-year R&D trek

Over the 14 years since Immunocore was spun out of MediGene in a quest to develop a gamechanging cancer med, the biotech has raised record sums and undergone a major shakeup on a long roller coaster ride of valuations for investors. But they survived and thrived and today they’re popping the champagne corks to celebrate an FDA approval of their first TCR drug.

Immunocore flagged the FDA’s green light for tebentafusp Wednesday morning by highlighting a series of firsts.

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Days af­ter Gilead yanks PI3K drug, In­cyte with­draws NDA for its own PI3K — say­ing con­fir­ma­to­ry tri­als would take too long

The FDA’s intensifying scrutiny on accelerated approvals isn’t just putting pressure on drugmakers with marketed products. It is also subtly reshaping the regulatory dynamics.

Case in point: Incyte announced late Tuesday that it has made the “business decision” to withdraw an NDA for parsaclisib, its oral PI3Kδ inhibitor, after deciding that running the confirmatory studies the agency was asking for to support an accelerated approval wouldn’t be worth it.

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Dan O'Day (Getty Images)

In a set­back, FDA or­ders Gilead to hit the brakes on their late-stage, $5B can­cer play

Gilead’s $5 billion drug magrolimab has run into a serious setback.

The FDA ordered Gilead to halt enrollment on their studies of the drug in combination with azacitidine after investigators reports revealed an “apparent imbalance” in the suspected unexpected serious adverse reactions between study arms. And the halt is raising questions about Gilead’s plans for a quick pitch to regulators.

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Graphic: Alexander Lefterov for Endpoints News

Small biotechs with big drug am­bi­tions threat­en to up­end the tra­di­tion­al drug launch play­book

Of the countless decisions Vlad Coric had to make as Biohaven’s CEO over the past seven years, there was one that felt particularly nerve-wracking: Instead of selling to a Big Pharma, the company decided it would commercialize its migraine drug itself.

“I remember some investors yelling and pounding on the table like, you can’t do this. What are you thinking? You’re going to get crushed by AbbVie,” he recalled.

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Mar­ket­ingRx roundup: Pfiz­er de­buts Pre­vnar 20 TV ads; Lil­ly gets first FDA 2022 pro­mo slap down let­ter

Pfizer debuted its first TV ad for its Prevnar 20 next-generation pneumococcal pneumonia vaccine. In the 60-second spot, several people (actor portrayals) with their ages listed as 65 or older are shown walking into a clinic as they turn to say they’re getting vaccinated with Prevnar 20 because they’re at risk.

The update to Pfizer’s blockbuster Prevnar 13 vaccine was approved in June, and as its name suggests is a vaccine for 20 serotypes — the original 13 plus seven more that cause pneumococcal disease. Pfizer used to spend heavily on TV ads to promote Prevnar 13 in 2018 and 2019 but cut back its TV budgets in the past two fall and winter seasonal spending cycles. Prevnar had been Pfizer’s top-selling drug, notching sales of just under $6 billion in 2020, and was the world’s top-selling vaccine before the Covid-19 vaccines came to market last year.

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FDA slams door to piv­otal tri­al for bub­ble boy dis­ease gene ther­a­py as Mus­tang Bio runs in­to an­oth­er hold

Mustang Bio is in familiar territory, but that isn’t a place it necessarily wants to be.

The FDA has placed a hold on Mustang Bio’s pivitol trial for its gene therapy to treat patients with bubble boy disease, citing issues surrounding chemistry, manufacturing and controls clearance. It’s the second hold due to CMC issues the company has received in roughly 18 months.

An investigational new drug application was submitted in December 2021. If granted an IND, a Phase II study will then assess safety, tolerability and efficacy of MB-207. If approved by the FDA, the therapy would one day be eligible for a rare pediatric disease voucher.

Brian Thomas, Metagenomi CEO

Gen 2: Berke­ley spin­out lands $175M megaround to keep it on the cut­ting edge of the boom­ing gene-edit­ing field

The big bucks keep pumping into the gene-editing field.

This morning Metagenomi, allied with one of the biggest names in the mRNA field with a company DNA that includes the ubiquitous Jennifer Doudna, is showing off a $175 million B round that will pay for a rapid swelling of its staff in pursuit of some of the cutting-edge tech that keeps this field in the spotlight. And they’re aligning themselves with some major industry players with an eye on the clinic while getting behind some startups to help expand the work into new fields.

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