Rob Lin, Avista Therapeutics CEO

Fol­low­ing Lux­tur­na and slate of eye dis­ease ap­provals, Roche adds an­oth­er oph­thal­mol­o­gy part­ner for AAV gene ther­a­pies

Roche’s oph­thal­mol­o­gy am­bi­tions have grown steadi­ly in re­cent years with two eye dis­ease ap­provals in the past year and the ac­qui­si­tion of Spark and its Lux­tur­na gene ther­a­py in 2019, and the Big Phar­ma is dou­bling down in the space with an­oth­er gene ther­a­py tie-up.

In­stead of Philadel­phia, Roche has head­ed to Pitts­burgh for this deal, link­ing arms with a Uni­ver­si­ty of Pitts­burgh Med­ical Cen­ter spin­out de­vel­op­ing new AAV gene ther­a­py vec­tors that the biotech’s CEO hopes will ad­dress lim­i­ta­tions of ex­ist­ing treat­ments, in­clud­ing Lux­tur­na. The deal al­so adds to Roche’s deep oph­thal­mol­o­gy pipeline, which in­cludes late-stage work at­tempt­ing to se­cure more in­di­ca­tions for re­cent­ly ap­proved Susvi­mo and Vabysmo.

While the up­front pay­ment is rel­a­tive­ly small at $7.5 mil­lion, the fledg­ling Avista Ther­a­peu­tics can se­cure more than $1 bil­lion in back-end pay­ments if the biotech eclipses cer­tain mile­stones and can get its gene ther­a­pies across the fin­ish line, the com­pa­nies an­nounced Tues­day morn­ing. The com­pa­ny has been seed­ed with $10 mil­lion from UPMC En­ter­pris­es to date and an­tic­i­pates go­ing to in­vestors in mid-2023 to raise a Se­ries A, CEO Rob Lin tells End­points News.

“Pret­ty good to be sit­ting out the fundrais­ing mar­ket at the present mo­ment,” Lin, who is al­so a VP at UPMC En­ter­pris­es, said. “Be­tween the up­front monies from the Roche part­ner­ship and the seed round, we’re pret­ty good for now.”

Leah Byrne

De­rived from avis­tar, which means “to catch sight of” in Span­ish and Por­tuguese, Avista is built up­on the work of UPMC re­searchers Leah Byrne, José-Alain Sa­hel and Paul Siev­ing, who pre­vi­ous­ly led the Na­tion­al Eye In­sti­tute. Byrne’s PhD work at UC-Berke­ley led to the cre­ation of vec­tor cap­sid AAV.7m8, which was li­censed to Ad­verum Biotech­nolo­gies, a biotech that has gone through a se­ries of hur­dles with its oc­u­lar dis­ease gene ther­a­pies. Sa­hel leads UPMC’s Eye Cen­ter and has co-found­ed a bevy of biotechs, in­clud­ing Spar­ingVi­sion, which ex­pects to file an IND on its first in­her­it­ed reti­nal dis­ease gene ther­a­py by the end of this year, said Lin, who is a board ob­serv­er.

Avista’s com­pu­ta­tion­al­ly-dri­ven in vi­vo plat­form, apt­ly dubbed scAAVen­gr, aims to cre­ate new cell-spe­cif­ic AAVs. In the Roche part­ner­ship, Avista will de­ploy the sin­gle-cell en­gi­neer­ing plat­form to cre­ate in­trav­it­re­al AAV cap­sids. The Big Phar­ma can then choose which ones it wants to li­cense and will be re­spon­si­ble for all pre­clin­i­cal, clin­i­cal and com­mer­cial­iza­tion work that might come out of the gene ther­a­py pro­grams us­ing those cap­sids. Avista’s in­ter­nal pipeline will re­main dis­tinct from the work with its Swiss part­ner.

The goal is to “open the win­dow of dis­eases” that gene ther­a­pies can treat, Lin said.

“One of the things we saw was that sub­reti­nal de­liv­ery in­volves stick­ing a hole in the reti­na and cre­at­ing a lit­tle bub­ble there to de­liv­er the gene ther­a­py prod­uct, and while it’s been shown to be ef­fec­tive in a prod­uct like Lux­tur­na, the sur­gi­cal com­pli­ca­tions of that make it lim­it­ing both in terms of how many peo­ple can be treat­ed with that but al­so with the types of dis­eases that per­haps maybe don’t want to stick a hole in the reti­na,” the CEO said.

The three-em­ploy­ee biotech will grow to about 10 em­ploy­ees next year as Avista search­es for ad­di­tion­al C-suite mem­bers and be­gins the hunt for ad­di­tion­al fi­nanc­ing, Lin said. Mean­while, he said, Avista is heads down on de­liv­er­ing on its Roche agree­ment, aim­ing to have “a num­ber of vec­tors” in the next 18 months that the phar­ma can opt in­to.

Bar­bara Lueck­el

“Our sci­en­tists were fa­mil­iar with the in­no­v­a­tive work by Leah Byrne, and when the spin-out of Avista was be­ing con­tem­plat­ed, we were ea­ger to be Avista’s first part­ner and de­vel­op en­abling cap­sids to ad­dress de­bil­i­tat­ing oc­u­lar dis­eases to­geth­er,” said Bar­bara Lueck­el, head of re­search tech at Roche Phar­ma Part­ner­ing, in a state­ment.

Avista is among a slate of gene ther­a­py play­ers look­ing at dis­eases in the eye. Along with Ad­verum and Spar­ingVi­sion, the field al­so in­cludes Coave Ther­a­peu­tics and Opus Ge­net­ics, found­ed by Lux­tur­na in­ven­tor Jean Ben­nett.

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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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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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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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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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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Patrick Lucy, Lykan Bioscience CEO

Two CD­MOs come to­geth­er as they look to ex­pand their reach in cell ther­a­py man­u­fac­tur­ing

As the cell therapy field has matured, the manufacturing of such therapies becomes a greater focus for companies small and large. Amid this growth, two CDMOs have come together in a merger to expand their reach.

Lykan Bioscience, based in Massachusetts, and RoslinCT, based in Edinburgh, UK, will become a single advanced therapies CDMO. The newly combined group will offer process development expertise as well as cGMP manufacturing for a broad range of autologous and allogeneic cell therapies, along with gene editing and induced pluripotent stem cell (iPSC) capabilities for cell therapy development. Financial terms of the deal were not disclosed.