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.

Jean-Paul Clozel, Idorsia CEO (Patrick Straub/Keystone via AP Images)

Idor­si­a's brain bleed drug flunks PhI­II tri­al, a decade af­ter pre­vi­ous flop

Idorsia’s long journey with clazosentan came to an abrupt “unexpected result” Monday morning with a Phase III flop.

The Swiss biopharma said the drug did not meet the main goal of the late-stage REACT study, conducted in the US, Canada and Europe since early 2019.

The 409-patient trial tested the intravenous drug’s ability to prevent complications due to delayed cerebral ischemia following aneurysmal subarachnoid hemorrhage (aSAH), in which blood vessels in the brain narrow and blood accumulates around the brain’s surface, which then dials up the pressure on the brain.

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Kenji Yasukawa, Astellas Pharma CEO (Photographer: Akio Kon/Bloomberg via Getty Images)

Astel­las taps chief strat­e­gy of­fi­cer as next CEO to 'go on the ag­gres­sive'

Five years into its big R&D revamp, Astellas says it’s time for a changing of the guard.

Kenji Yasukawa, who took over as president and CEO in 2018, will step down to become chairman of the board in April, making room for Naoki Okamura to take over. Okamura joined the company in 1986 and has served in a variety of finance, business and strategy roles, including most recently as chief strategy officer.

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Clin­i­cal tri­al di­ver­si­ty da­ta show mis­match be­tween en­roll­ment and dis­ease preva­lence, GSK says

A lack of diversity in clinical trials has persisted despite decades of initiatives to try to turn the tide.

In a recent review of 17 years of clinical trials, drugmaker GSK found that there were some mismatches between the demographics of its US-based trials and how prevalent diseases were in those populations.

The results, the company says, will help GSK and others design studies that better represent epidemiological rates within races and ethnicities.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; 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.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Uğur Şahin, BioNTech CEO (Andreas Arnold/picture-alliance/dpa/AP Images)

BioN­Tech opens new plas­mid DNA man­u­fac­tur­ing fa­cil­i­ty in Ger­many

German mRNA player BioNTech opened the doors to a new manufacturing facility on Thursday, this one just about 75 miles north of its headquarters in Mainz, Germany.

BioNTech announced on Thursday that it has completed the construction of its first plasmid DNA manufacturing facility in Marburg, Germany. The facility will produce materials for mRNA-based vaccines and therapies along with cell therapies.

Goldfinch Bio CEO Tony Johnson (L) and Karuna Therapeutics CEO Bill Meury

Karuna li­cens­es Goldfinch as­sets to com­pete with Boehringer In­gel­heim in neu­ro­science

Karuna Therapeutics is looking to compete with Boehringer Ingelheim on depression and anxiety with a new license to Goldfinch Bio’s assets, starting with $15 million to the shuttered biotech.

Karuna steps into an arena already being tested by Boehringer in multiple Phase II studies — the two are targeting transient receptor potential canonical 4 and 5, or TRPC4/5, which is thought to have a role in neuroscience indications. Goldfinch’s asset went through a Phase II in kidney diseases, but Karuna’s sights are set on mood and anxiety disorders for now.

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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David Kirn, 4D Molecular Therapeutics CEO (via website)

FDA places hold on 4D Mol­e­c­u­lar’s Fab­ry gene ther­a­py

4D Molecular Therapeutics quietly tucked an FDA clinical hold on its Fabry gene therapy into an SEC filing.

Meanwhile, the biotech issued a press release the same day after the closing bell on Thursday touting an IND for another asset, in diabetic macular edema.

The California biotech had paused enrollment of patients in its two trials of the Fabry gene therapy (4D-310) last month after three patients experienced kidney issues, all of which were resolved within four weeks. At the time, 4DMT said it would wait until the second half of this year to look at 12-month clinical data on six patients in the Phase I/II trials, one in the US and one in Taiwan and Australia.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.