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.

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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What does $6.9B buy these days in on­col­o­gy R&D? As­traZeneca has a land­mark an­swer

Given the way the FDA has been whisking through new drug approvals months ahead of their PDUFA date, AstraZeneca and their partners Daiichi Sankyo may not have to wait until Q2 of next year to get a green light on trastuzumab deruxtecan (DS-8201).

The pharma giant this morning played their ace in the hole, showing off why they were willing to commit to a $6.9 billion deal — with $1.35 billion in a cash upfront — to partner on the drug.

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Large advertisements for the drug Vivitrol decorate the walls of Grand Central Station on June 15, 2017 in New York City. (Photo: Andrew Lichtenstein via Getty)

FDA slaps down Alk­er­mes for mis­lead­ing Viv­it­rol ads — don't for­get vul­ner­a­bil­i­ty to opi­oid over­dose

The ads piqued interest as soon as they started appearing in 2016: at Grand Central Station, on the Red Line in Cambridge, and on a billboard off the New Jersey Turnpike. All showed a young person, generally with his or her arms crossed, and the question, “what is Vivitrol?”

Vivitrol’s maker, Alkermes, was in the midst of a marketing and lobbying campaign to promote the anti-opioid addiction drug — a campaign that would face significant backlash for tarnishing competitors despite little evidence for Vivitrol’s superiority.

Paul Hudson, Sanofi

Paul Hud­son promis­es a bright new fu­ture at Sanofi, kick­ing loose me-too drugs and fo­cus­ing on land­mark ad­vances. But can he de­liv­er?

Paul Hudson was on a mission Tuesday morning as he stood up to address Sanofi’s new R&D and business strategy.

Still fresh into the job, the new CEO set out to convince his audience — including the legions of nervous staffers inevitably devoting much of their day to listening in — that the pharma giant is shedding the layers of bureaucracy that had held them back from making progress in the past, dropping the duds in the pipeline and reprioritizing a more narrow set of experimental drugs that were promised as first-in-class or best-in-class.  The company, he added, is now positioned to “go after other opportunities” that could offer a transformational approach to treating its core diseases.

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FDA in-house re­view spot­lights an is­sue with one of Hori­zon's end­points but notes ef­fi­ca­cy for lead drug

The FDA in-house review highlights a disagreement of investigators’ use of a key endpoint by Horizon Pharma in the late-stage trial for the top drug in its pipeline, but largely agreed that the antibody was effective.

Horizon submitted a BLA for thyroid eye disease (TED) drug teprotumumab in March, less than two years after they bought the drug (and the rest of a division) from Narrow River for $145 million upfront. With breakthrough status, priority review, orphan designation and in-house sales projections of up to $750 million, the one-time Roche reject became the marquee pipeline asset for a company that’s developed some of the world’s most expensive drugs.

Seat­tle Ge­net­ics de­tails pos­i­tive OS and PFS da­ta for tu­ca­tinib in breast can­cer

Seattle Genetics $SGEN is showing off more positive data around tucatinib, its pivotal-stage drug for HER2 positive breast cancer.

A month after hearing about solidly upbeat hazard ratios, we learned today that the estimated progression-free survival rate at one year was 33% in the tucatinib arm compared to 12% for patients taking trastuzumab and capecitabine alone.

Median PFS was 7.8 months (95% CI: 7.5, 9.6) in the tucatinib arm, compared to 5.6 months (95% CI: 4.2, 7.1) in the control arm.

Bat­tered, cash hun­gry In­tec feels the burn of No­var­tis re­jec­tion

It’s a case of some bad timing for Intec.

Just when a key trial testing the company’s Accordion drug delivery tech imploded in Parkinson’s disease, they handed Novartis data from a successful PK study of a custom Accordion pill engineered to deliver a Novartis compound to entice the Swiss drugmaker into signing a licensing agreement.

Novartis said thanks, but no thanks.

For the cash-strapped Israeli drug developer, the failure to clinch the deal marks a big blow. As of the third quarter, the company has $15.7 million in cash and equivalents, which HC Wainwright analysts estimate will keep the lights on into mid-2020.

Bris­tol-My­ers shows off a low-pro­file AML con­tender it gained from Cel­gene buy­out — and they’re tak­ing it straight to the FDA

Bristol-Myers Squibb reaped an enormous pipeline with its much-criticized $64 billion megadeal to buy Celgene. And it got a few hidden gems in the deal.

One of those gems was brought out for display on Tuesday, with a late-breaker at ASH on CC-486, which is now being prepped for regulatory filings at the FDA and elsewhere.

Celgene top-lined the positive results in a maintenance setting for acute myeloid leukemia a few months ago, but at ASH investigators pulled back the curtains on the all-important data they believe will give them an advantage in the commercial wars to come.

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