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.

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Vas Narasimhan. Getty Images

UP­DAT­ED: Failed PhI­II fe­vip­iprant tri­als pour more cold wa­ter on No­var­tis' block­buster R&D en­gine — and briefly spread the chill to a high-pro­file biotech

Back in July, during an investor call where Novartis execs ran through an upbeat assessment of their Q2 performance, CEO Vas Narasimhan and development chief John Tsai were pressed to predict which of the two looming Phase III readouts — involving cardio drug Entresto and asthma therapy fevipiprant, respectively — had a higher likelihood of success. Tsai gave the PARAGON-HF study with Entresto minimally better odds, but Narasimhan emphasized that their strategy of giving fevipiprant to more severe patients gave them confidence.

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UP­DAT­ED: The FDA sets a reg­u­la­to­ry speed record, pro­vid­ing a snap OK for Ver­tex's break­through triplet for cys­tic fi­bro­sis

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IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

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Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
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Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

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UP­DAT­ED: Clay Sie­gall’s $614M wa­ger on tu­ca­tinib pays off with solid­ly pos­i­tive piv­otal da­ta and a date with the FDA

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David Liu, Liu Group

David Liu un­veils newest ad­vance­ment in CRISPR tech: Prime edit­ing

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Bhaskar Chaudhuri. Frazier Healthcare Partners

Fra­zier Health­care Part­ner­s' der­ma­tol­ogy up­start at­tracts a mar­quee syn­di­cate, $94M+ for 'in-be­tween' top­i­cal drug

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A new com­pa­ny en­ters the Tec­fidera fight, of­fer­ing to kill two birds

The remedy for the most common side effect for one of the most common multiple sclerosis drugs is simple: aspirin.

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