Laurent Fischer, Adverum CEO

Ad­verum lays off 38% of staff as it en­ters new tri­al for oc­u­lar gene ther­a­py af­ter last year's safe­ty woes

Ad­verum is mov­ing for­ward with its eye dis­ease gene ther­a­py that caused headaches in April 2021, but is do­ing so with few­er staff on board.

The biotech is lay­ing off 78 em­ploy­ees, or about 38% of its work­force, in or­der to con­serve cash and keep the lights on in­to 2025, ac­cord­ing to an SEC fil­ing.

Ad­verum CMO Richard Beck­man

The Red­wood City, CA, biotech said Wednes­day it has amend­ed the IND for a Phase II study of its gene ther­a­py in wet age-re­lat­ed mac­u­lar de­gen­er­a­tion (AMD). The tri­al of AD­VM-022 will kick off this quar­ter, CMO Richard Beck­man said in a state­ment.

Ad­verum’s stock $AD­VM rose near­ly 9% af­ter the open­ing bell Thurs­day. The re­struc­tur­ing will elim­i­nate about $15.3 mil­lion an­nu­al­ly in “di­rect la­bor cost,” ac­cord­ing to the biotech’s SEC fil­ing.

Now dubbed ixobero­gene soropar­vovec (Ixo-vec), an ear­li­er tri­al of the gene ther­a­py led to a loss of vi­sion in one pa­tient’s treat­ed eye last year. That pa­tient had di­a­bet­ic mac­u­lar ede­ma and re­ceived a high­er dose of the gene ther­a­py.

The safe­ty is­sues from the high dose end­ed up af­fect­ing five of 12 pa­tients with di­a­bet­ic mac­u­lar ede­ma in that Phase II tri­al, the com­pa­ny said last Ju­ly. It is now mov­ing for­ward with a low­er dose and is on­ly test­ing the gene ther­a­py in pa­tients with wet AMD in the Phase II LU­NA tri­al.

“We de­signed the LU­NA tri­al af­ter a thor­ough re­view of da­ta gen­er­at­ed in all 55 study par­tic­i­pants treat­ed to date with a sin­gle in­jec­tion of AD­VM-022, some of whom have now demon­strat­ed con­tin­u­ous and sta­ble afliber­cept ex­pres­sion for over three years. We have deep­ened our un­der­stand­ing of ade­no-as­so­ci­at­ed virus (AAV) me­di­at­ed oc­u­lar gene ther­a­py, in­clud­ing the in­flam­ma­to­ry re­sponse to AAV, and this is in­form­ing our strat­e­gy to mit­i­gate im­muno­genic­i­ty,” Beck­man, the med­ical chief, said in a state­ment.

CEO Lau­rent Fis­ch­er said the com­pa­ny has worked on its man­u­fac­tur­ing strat­e­gy to have a “scal­able ap­proach de­signed to pro­vide an am­ple sup­ply of Ixo-vec to sup­port our clin­i­cal de­vel­op­ment pro­gram through bi­o­log­ics li­cense ap­pli­ca­tion fil­ing.”

With the lay­offs and re­fined fo­cus on AD­VM-022, Ad­verum has enough cash to fund it­self be­yond one-year topline re­sults from the 72-pa­tient LU­NA tri­al, Fis­ch­er said in a state­ment. The biotech had $271.1 mil­lion in cash as of March 31.

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

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Ad­dress­ing the ‘Ca­pac­i­ty Crunch’ with a Scal­able Plat­form Process Ap­proach

The field of gene therapy has been diligently moving forward over the past several decades to bring potentially life-saving treatments to patients with genetic diseases. In addition to two approved adeno-associated viral (AAV) gene therapies, there are more than 250 AAV gene therapies in various clinical trial stages.1 AAV vectors remain the most frequently used vector for delivering therapeutic transgenes to target tissues due to their demonstrated and lasting clinical efficacy and extensive safety track record. As AAV therapies advance through clinical trials and into commercialization, many biotech companies are turning to contract development and manufacturing organizations (CDMOs) to prepare their programs for late-stage clinical and commercial scale manufacturing. Given the scope and scale of the manufacturing needs that will accompany regulatory approvals for these assets, CDMOs continue to expand their capacity to meet the needs of increasing prevalent patient populations. However, despite rapid growth, projected gene therapy manufacturing demands still outpace the collective capacity of the CDMO industry.

A $5B Pfiz­er buy­out? Am­gen, Gilead head­line M&A Thurs­day; Al­ny­lam's AT­TR sweep; An­drew Lo's rare dis­ease quest; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

One of the cool things about adding EndpointsPharma to the daily roster is that my colleagues can now dedicate time to tracking quarterly updates and tuning into calls with Big Pharma companies. Check out their dispatch from the Q2 earnings below.

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Bob Bradway, Amgen CEO (Justin Kase Conder/AP Images for Amgen)

UP­DAT­ED: Am­gen chief Brad­way nabs a rare dis­ease play­er in $4B buy­out as the M&A tem­po ac­cel­er­ates

Amgen CEO Bob Bradway is bellying up to the M&A table today, scooping up the newly anointed commercial biotech ChemoCentryx $CCXI and its recently approved rare disease drug for $3.7 billion out of the cash stockpile. The deal comes in at $52 a share — a hefty increase over the $24.11 close yesterday.

Bradway and the Amgen team get a drug called Tavneos (avacopan) in the deal, a complement factor C5a inhibitor OK’d to treat anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis, an autoimmune disease which can be lethal.

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Albert Bourla, Pfizer CEO (Laurent Gillieron/Keystone via AP)

Break­ing: Pfiz­er in hot pur­suit of a $5B buy­out of Glob­al Blood Ther­a­peu­tics — re­port

Pfizer CEO Albert Bourla has vowed to leave no stone unturned in the search for new biotech deals, and the BD team is not letting him down.

The Wall Street Journal reported today that Pfizer is in the final stages of acquiring Global Blood Therapeutics for $5 billion. According to the Journal report, though, Pfizer is not the only buyer at the deal table and while the pharma giant may be close to clinching it, there are no guarantees it will continue.

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Al Sandrock, Voyager Therapeutics CEO

Al San­drock prunes his post-Bio­gen voy­age with sim­i­lar fo­cus on ALS

Al Sandrock is narrowing the focus of Voyager Therapeutics, concentrating on CNS diseases that were the hallmark of his time leading R&D at Biogen, including an emphasis on a familial form of ALS for which his former employer is getting a speedy review at the FDA.

Less than six months into his journey as CEO at Voyager, Sandrock is focusing the preclinical pipeline on Alzheimer’s disease, GBA1 Parkinson’s disease and SOD1 amyotrophic lateral sclerosis, the rare form of ALS for which the FDA will decide whether to approve Biogen’s tofersen by Jan. 25, 2023.

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George Yancopoulos, Regeneron president and CSO (Brendan McDermid/Reuters/Alamy)

George Yan­copou­los says he's on the trail of the holy grail: ‘This could rep­re­sent the next break­through for im­munother­a­py’

Two of the most outspoken — and successful — drug developers in biotech say they’ve collected early-stage clinical data that are pointing them down the trail to the holy grail in cancer immunotherapy R&D.

While analysts largely busied themselves today with chronicling the ongoing success of Regeneron’s two big cash cows — Dupixent and Eylea — chief scientist George Yancopoulos and CEO Len Schleifer used the Q2 call to spotlight their early success with a combination of the “homegrown” PSMAxCD28 costimulatory bispecific antibody REGN5678 in combination with their PD-1 checkpoint Libtayo. The presentation comes just weeks after Regeneron completed a deal to gather all rights to the PD-1 that had been in Sanofi’s hands. And the two top execs are unstinting in their praise of the potential of a whole set of costimulatory pipeline projects which they say may finally deliver the long-awaited next-level approach to broadening the immunotherapy field of drugs.

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David Hallal (L) and George Daley (Hallal photo: Bertrand Guay/AFP via Getty Images)

David Hal­lal's El­e­vate­Bio launch­es new com­pa­ny to 'dis­rup­t' off-the-shelf cell ther­a­py, but pro­vides few oth­er de­tails

David Hallal’s ElevateBio is launching a new company Thursday as it looks to continue making its mark in the cell and gene therapy spaces. But Hallal is also keeping his cards close to the vest, preferring to toe the line between bombast and mystique rather than going all-in in one direction.

The new company comes out of a partnership with Boston Children’s Hospital and research from George Daley, the dean of Harvard Medical School. The triumvirate claims to have found a way to design better off-the-shelf cell therapies using new methods discovered in Daley’s Boston Children’s Hospital lab (Harvard is not involved in the collaboration).

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(AP Photo/Richard Vogel, File)

US de­clares mon­key­pox a na­tion­al health emer­gency, as new drug­mak­ers con­sid­er en­ter­ing vac­cine race

Rising monkeypox cases have put the US on high alert as it announces a national health emergency, which grants the government more power in its response.

The news comes as Bavarian Nordic continues to fill orders for its Jynneos vaccine and other companies – including Moderna – consider jumping into the vaccine race. Meanwhile, the New York Times reports that the US has allowed around 20 million doses of smallpox vaccine in its stockpile to expire.