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.”

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Tony Coles, Cerevel CEO

Cerev­el takes the pub­lic of­fer­ing route, with a twist — rais­ing big mon­ey thanks to ri­val da­ta

As public biotechs seek to climb out of the bear market, a popular strategy to raise cash has been through public offerings on the heels of positive data. But one proposed raise Wednesday appeared to take advantage not of a company’s own data, but those from a competitor.

Cerevel Therapeutics plans to raise $250 million in a public offering and another $250 million in debt, the biotech announced Wednesday afternoon, even though it did not report any news on its pipeline. However, the move comes days after rival Karuna Therapeutics touted positive Phase III data in schizophrenia, a field where Cerevel is pursuing a similar program.

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Who are the women blaz­ing trails in bio­phar­ma R&D? Nom­i­nate them for End­points' 2022 spe­cial re­port

Over the past three years, Endpoints News has spotlighted 60 women who have blazed trails and supercharged R&D across the biopharma world. And judging from the response we’ve received, to both our special reports and live events, telling their stories — including any obstacles they may have had to overcome — has inspired our readers in many different ways.

But change takes time, and the fact remains that women are still underrepresented at the upper ranks of the drug-making world.

Up­dat­ed: Amid mas­sive re­struc­tur­ing, Bio­gen looks to re­duce phys­i­cal pres­ence in Boston

Biogen is putting a sizable chunk of office and research space in Kendall Square and Weston, MA up for sublease, marking another big change as the biotech grapples with the aftershock of a disastrous and controversial rollout for its Alzheimer’s drug.

The subbleases are “part of Biogen’s overall implementation of the ‘Future of Work,’ which is allowing us to optimize our footprint and reduce the amount of space we occupy, taking into consideration new elements such as the hybrid work model,” Biogen spokesperson Ashleigh Koss wrote in a statement to Endpoints News, adding that the company has had subleases across several buildings for years.

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Bernat Olle, Vedanta Biosciences CEO

Cit­ing 'chal­leng­ing eco­nom­ic en­vi­ron­ment,' PhI­II-ready mi­cro­bio­me biotech lays off 20% of staffers

The market downturn isn’t just sweeping up public biotechs.

Vedanta Biosciences, a developer of oral drugs derived from the human microbiome, is laying off about 20% of its staff — an unfortunately common occurrence these days. But CEO Bernat Olle took the unusual step of sharing the decision on LinkedIn and offering to connect the employees being let go with any company that’s hiring in their areas.

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Hervé Affagard, MaaT Pharma CEO

One year in­to clin­i­cal hold, FDA has more ques­tions about 'pooled' mi­cro­bio­me ther­a­py

The FDA is still wary about a trial testing a microbiome therapy in patients with steroid-resistant acute graft-versus-host disease (aGVHD).

A year after MaaT Pharma’s IND application in the US was first met with a clinical hold, the French biotech said the agency is maintaining the hold. The crux of the matter, MaaT suggested, has to do with the way it puts together its drug candidate, which is administered as an enema (i.e. an injection of fluid into the bowel).

Lorenz Mayr, Vector BioPharma CEO (PhoreMost)

Up­dat­ed: 'Close to the mid­dle': Ver­sant launch­es Vec­tor with 'gut­less ade­n­ovirus' de­liv­ery method

Most of the gene therapy world centers around viral vectors, but a few biotechs have sprung up in recent years to try and avoid the safety issues that can come with adeno-associated viruses.

A new biotech has emerged to straddle somewhere “close to the middle,” in the words of Alex Mayweg, Versant managing director and board member of the incubator’s latest startup, Vector BioPharma.

Pfiz­er launch­es re­bate pro­gram for rare dis­ease pa­tients who have to stop tak­ing Panzy­ga

Pfizer is launching its second-ever rebate program, this time for Panzyga, its treatment for a rare neurological disease of the peripheral nerves.

The program began last month, according to STAT which first reported the news, and offers a refund of out-of-pocket costs for patients who must discontinue their course before the fifth treatment for “clinical reasons.”

Panzyga was approved back in 2018 to treat primary immunodeficiency (PI) in patients two years and older and chronic immune thrombocytopenia (cITP) in adults. It has since picked up an indication in chronic inflammatory demyelinating polyneuropathy (CIDP), a condition that’s characterized by weakness of the arms or legs, tingling or numbness, and a loss of deep tendon reflexes, according to the NIH.

Horizon's back-to-school campaign for children with cystinosis includes an all about me poster as part of a care package box.

Hori­zon read­ies kids and fam­i­lies for back to school with week­long ac­tiv­i­ties around rare dis­ease cysti­nosis

Going back to school is usually a bumpy readjustment from summer freedom for all kids, but especially for kids with chronic health conditions. Horizon Therapeutics is hoping to help smooth the way for some who have the rare disease cystinosis. Cystinosis is a genetic disease that causes the amino acid cystine to build up in different tissues and organs.

The “Gear Up” for school campaign is running all week with different online and at-home events and activities for families and children with cystinosis. Each family who signed up receives a care package mailed to their home including an activity coloring book “Michael’s Show-and-Tell.” The book tells Michael’s story about living with cystinosis while offering kids matching, coloring and finding object games along with information.

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