Faraz Ali, Tenaya

San Fran­cis­co biotech has its heart in heart fail­ure — and $92M in the bank

When a sala­man­der’s heart suf­fers dam­age, the am­phib­ian’s cells are able to di­vide and re­pair the or­gan. Tenaya Ther­a­peu­tics wants to em­pow­er your heart with the same abil­i­ty.

The South San Fran­cis­co-based com­pa­ny, found­ed by sci­en­tists at the Glad­stone In­sti­tutes and Uni­ver­si­ty of Texas South­west­ern Med­ical Cen­ter in Oc­to­ber 2016, has a mul­ti-lay­ered ap­proach to ad­dress­ing trou­bles of the heart — cel­lu­lar re­gen­er­a­tion, gene ther­a­py, and pre­ci­sion med­i­cine.

“We’re all in…we have put our en­tire heart in­to heart fail­ure,” said chief Faraz Ali in an in­ter­view with End­points News.

Tenaya, which is named af­ter an alpine lake in Yosemite Na­tion­al Park, on Thurs­day raised $92 mil­lion Se­ries B fi­nanc­ing, led by Cas­din Cap­i­tal and the par­tic­i­pa­tion of GV, The Col­umn Group, and ad­di­tion­al in­vestors. The funds will be used to take its slate of undis­closed pre­clin­i­cal pro­grams in­to hu­man stud­ies in the com­ing years.

Over the last few decades, sci­en­tists have at­tempt­ed to ad­dress heart dam­age by push­ing cells from the out­side in­to the heart or by us­ing stem cells or oth­er prog­en­i­tor cells, but none of those ap­proach­es have worked, Ali not­ed. “Most of the ap­proved ther­a­pies just ad­dress the symp­toms of heart dis­ease, but they don’t ac­tu­al­ly tar­get the un­der­ly­ing cause.”

As part of its cel­lu­lar re­gen­er­a­tion plat­form, ini­tial­ly aimed at pa­tients who have suf­fered my­ocar­dial in­farc­tion, Tenaya has en­gi­neered the con­ver­sion of res­i­dent car­diac fi­brob­lasts — which rep­re­sent about 50% of the heart — in­to heart mus­cle cells (car­diomy­ocytes) by adding cer­tain fac­tors.

This process, in an­i­mal mod­els, has shown to trig­ger cell di­vi­sion and re­pro­duc­tion. “It sounds like sci­ence fic­tion. — but it ac­tu­al­ly works!” Ali said.

For its gene ther­a­py tech­nol­o­gy, Tenaya learned from the mis­takes of Cel­ladon, which saw its bid to de­vel­op a gene ther­a­py for heart fail­ure go up in smoke af­ter a spec­tac­u­lar late-stage set­back in 2015.

“They had not spent the time to look for a nov­el vec­tors, didn’t have high­er speci­fici­ty, or high­er trans­duc­tion for the rel­e­vant cell types in the heart,” Ali said. “That’s one thing that we’re do­ing dif­fer­ent­ly, where we have de­vel­oped our own nov­el vec­tors…that have the at­trib­ut­es that are su­pe­ri­or to the parental AAV vec­tors.”

Tenaya, and its team of 45, is not alone in its quest to de­vel­op a gene ther­a­py geared to­wards the heart. Philade­phia-based and No­var­tis-backed Ren­o­va­cor, which raised $11 mil­lion in a Se­ries A round in Au­gust, is fo­cus­ing on a mu­tant gene that is un­der­stood to cause a rare heart con­di­tion — di­lat­ed car­diomy­opa­thy.

In May, the head of the Cen­ter for Hu­man Ge­net­ic Re­search at Mass­a­chu­setts Gen­er­al Hos­pi­tal and the Broad’s Car­dio­vas­cu­lar Dis­ease Ini­tia­tive, Sekar Kathire­san, set up his own shop to tweak genes, such as APOC3 or ANGPTL3, which car­ry mu­ta­tions that can rapid­ly clear triglyc­eride-rich lipopro­teins — which raise in­di­vid­u­als’ risk of heart at­tack — from cir­cu­la­tion.

Tenaya is al­so strate­gi­cal­ly tar­get­ing younger pa­tients that are ge­net­i­cal­ly pre­dis­posed to heart dam­age, un­like oth­ers who have large­ly gone af­ter the geri­atric pop­u­la­tion — which has in part con­tributed to the fail­ures of the past, Ali said, adding that Tenaya is al­so be­ing pru­dent about keep­ing its man­u­fac­tur­ing heft in-house, de­spite be­ing years away from the clin­ic.

Oth­er drug­mak­ers have tak­en their re­spec­tive plat­forms and tried to ap­ply them across a range of ther­a­peu­tic in­di­ca­tions.

“We could take our gene ther­a­pies and our man­u­fac­tur­ing ca­pa­bil­i­ties and turn our at­ten­tion to oth­er ther­a­peu­tic ar­eas,” he added. “But in­stead of be­ing the sixth com­pa­ny to go af­ter he­mo­phil­ia, or the twen­ti­eth in­sti­tu­tion to go af­ter Duchenne mus­cu­lar dy­s­tro­phy, or to be that fifti­eth CAR -T pro­gram…we think that there’s just a tremen­dous op­por­tu­ni­ty to fo­cus on the heart.”

Car­dio­vas­cu­lar dis­ease is the lead­ing cause of death glob­al­ly — al­though can­cer is catch­ing up. About 17.9 mil­lion peo­ple died from car­dio­vas­cu­lar dis­ease in 2016, rep­re­sent­ing about a third of all glob­al deaths, es­ti­mates the WHO.

So far, Tenaya has raised $142 mil­lion, in­clud­ing a $50 mil­lion Se­ries A round in 2016.

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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Noubar Afeyan, Flagship CEO and Tessera chairman (Victor Boyko/Getty Images)

Flag­ship ex­ecs take a les­son from na­ture to mas­ter ‘gene writ­ing,’ launch­ing a star-stud­ded biotech with big am­bi­tions to cure dis­ease

Flagship Pioneering has opened up its deep pockets to fund a biotech upstart out to revolutionize the whole gene therapy/gene editing field — before gene editing has even made it to the market. And they’ve surrounded themselves with some marquee scientists and execs who have crowded around to help shepherd the technology ahead.

The lead player here is Flagship general partner Geoff von Maltzahn, an MIT-trained synthetic biologist who set out in 2018 to do CRISPR — a widely used gene editing tool — and other rival technologies one or two better. Von Maltzahn has been working with Sana co-founder Jake Rubens, another synthetic biology player out of MIT who he describes as his “superstar,” who’s taken the CSO role.

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Bill Haney, Dragonfly CEO (Dave Pedley/Getty Images for SXSW)

A boom­ing Drag­on­fly is tak­ing its TriN­KETs to Copen­hagen as the lat­est Bris­tol My­ers pact spurs ex­pan­sion plans — out­side the US

Bristol Myers Squibb is making a habit out of collaborating with the crew at Dragonfly, adding their 3rd deal in a series that now will take them into newly charted R&D territory. And the fast-growing team at the Cambridge-based biotech is adding a facility in Copenhagen for its next growth spurt, where the government is making it easy to recruit scientists internationally as the U.S. throttles back.

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RA Cap­i­tal dou­bles down on Sid­dhartha Mukher­jee's vi­sion for a new cell en­gi­neer­ing ap­proach, lead­ing Vor's $110M Se­ries B

Vor Biopharma is muscling up.

CEO Robert Ang, who was reluctant to divulge the headcount when discussing his move from Neon Therapeutics to Vor last August, readily offered that the team has grown from 6 to 50 in less than a year. The biotech is moving to a larger office on Cambridge Parkway Drive in weeks, giving it more space to complete the IND-enabling work and manufacturing scale-up — conducted by a CDMO partner — in preparation for clinical trials planned for the first half of 2021.

Covid-19 roundup: Squab­bles with gov­ern­ment de­lay Mod­er­na’s PhI­II — re­ports; No­vavax se­cures largest Warp Speed deal yet: $1.6B

A much-anticipated Phase III trial for Moderna’s Covid-19 vaccine is being held up as the company delayed submitting trial protocols and sparred with government scientists on how to run the study and even what the benchmark for success should be, Reuters reported.

Moderna, the first US company to put their vaccine into human testing, was supposed to enter a 30,000-person study this month in partnership with the NIH to determine whether it can prevent infection. STAT reported last week that the trial was facing delays over the protocol, but that a July start was still possible. Neither the NIH nor Moderna ever disclosed a specific date the trial should start, but Reuters reported that the agency had hoped to begin on July 10.

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Boehringer In­gel­heim ties the knot with Num­ab on new an­ti­bod­ies; Ca­balet­ta inks pact with Ar­ti­san

→ Switzerland’s Numab Therapeutics has added Boehringer Ingelheim to its roster of collaborators. And they will start with two projects aiming at developing new drugs for difficult-to-treat lung and gastrointestinal cancers and patients with geographic atrophy. “Numab’s technology platform fits well with our internal antibody discovery and engineering capabilities and will enhance our efforts to deliver transformative antibody-based therapeutics to patients,” said Paige Mahaney, an SVP at Boehringer Ingelheim.

Cel­lec­tis slammed af­ter pa­tient dies and FDA slaps a hold on their tri­al for an off-the-shelf CAR-T for mul­ti­ple myelo­ma

Cellectis was slammed after the market close on Monday as the biotech reported that the FDA demanded it hit the brakes on their MELANI-01 trial for their off-the-shelf cell therapy UCARTCS1A after one of the patients in the study died of treatment-related cardiac arrest.

The multiple myeloma patient had previously been treated unsuccessfully with various therapies, noted the biotech, and had been given dose level two (DL2) of their allogeneic CAR-T.

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Shoshanna Shendelman, Applied Therapeutics CEO (Applied Therapeutics)

A lit­tle biotech slaps back at a 'crim­i­nal' short at­tack, vow­ing to pur­sue a pros­e­cu­tion of their case

As short attacks go, Biotech Research Partners’ assault on Applied Therapeutics’ “cherry picked” data and a variety of so-called red flags didn’t cause a whole lot of damage. Ahead of the July 4 holiday, its shares $APLT were dinged and showed signs of quick recovery.

But that didn’t stop an incendiary response, as the biotech swung into action bright and early Monday morning.

Applied Therapeutics accused the authors of the short report of manipulating graphs and figures, misrepresenting data and included factual misrepresentations — all of which added up, in their view, to fraud.

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