With new $106M crossover round, Tenaya preps heart fail­ure pro­grams for the clin­ic — and maybe an IPO

Less than a year and a half since pulling in its last round of fund­ing, Tenaya Ther­a­peu­tics is back with a crossover round that could set it up for an IPO down the road.

Pri­vate and pub­lic in­vestors have now jumped aboard the Tenaya train to the tune of a $106 mil­lion Se­ries C, the com­pa­ny an­nounced Mon­day morn­ing, with the hopes of ad­vanc­ing its lead gene ther­a­py pro­gram for heart fail­ure in­to the clin­ic. Tenaya al­so aims to fund its oth­er heart fail­ure plat­forms, hav­ing made much progress in cel­lu­lar re­gen­er­a­tion and pre­ci­sion med­i­cine as well, CEO Faraz Ali told End­points News.

Faraz Ali

“For us it’s not about op­por­tunis­ti­cal­ly try­ing to catch an IPO win­dow,” Ali said, “but feel­ing that our sci­ence has ar­rived at a point on mul­ti­ple fronts that we have a rea­son to be­lieve that each of these plat­forms can de­liv­er prod­ucts that could be mean­ing­ful in hu­mans.”

Ali added that Tenaya hasn’t made any con­crete plans for an IPO just yet, but they’re “ab­solute­ly think­ing about” go­ing pub­lic, giv­en the mar­ket con­di­tions that have led to a biotech boom over the last 12 months.

The South San Fran­cis­co-based com­pa­ny got start­ed way back in De­cem­ber 2016 with a $50 mil­lion Se­ries A look at how fresh sources of re­gen­er­a­tive cells could po­ten­tial­ly re­pair a dam­aged heart. It had been an area that pre­vi­ous­ly drove a va­ri­ety of large­ly un­suc­cess­ful re­search ef­forts aimed at help­ing pa­tients who have suf­fered from heart at­tacks and strokes.

But Tenaya has grown to about 80 staffers over the last sev­er­al years and built out its three dif­fer­ent plat­forms. Aside from the gene ther­a­py pro­grams, Tenaya is work­ing on a sin­gle AAV vec­tor-based cell ther­a­py and a small mol­e­cule treat­ment for dif­fer­ent heart fail­ure pop­u­la­tions.

In Oc­to­ber 2019, Ali led a $92 mil­lion Se­ries B, and Tenaya hadn’t yet dis­closed many of its tar­gets. Now, how­ev­er, they’re ready to dish on the de­tails. Their lead can­di­date is tar­get­ing ge­net­ic hy­per­trophic car­diomy­opa­thy due to mu­ta­tions in the MYBPC3 gene, with an AAV gene ther­a­py, in both adults and chil­dren.

The dis­ease it­self has a broad range of symp­toms, with some adults ex­pe­ri­enc­ing no symp­toms for years while oth­ers can die with­in a few weeks of be­ing born. It es­sen­tial­ly caus­es sick­en­ing of the heart ven­tri­cles, Ali said, caus­ing re­duc­tions in ejec­tion frac­tion. There’s cur­rent­ly no tar­get­ed ther­a­py for the dis­ease, and Tenaya is aim­ing to be the first to de­liv­er a healthy copy of the gene.

They’re con­sid­er­ing an IV in­fu­sion for­mu­la­tion or a pos­si­ble de­liv­ery clos­er to the heart. To ac­com­plish this and low­er man­u­fac­tur­ing costs, Tenaya has al­so in­vest­ed in its own catheters.

Be­yond that, the com­pa­ny is re­search­ing an­oth­er gene ther­a­py in­volv­ing DWORF, a mi­cro-pep­tide act­ing on the SER­CA path­way. There’s al­so a pro­gram fo­cused on car­diac re­gen­er­a­tion, us­ing a sin­gle AAV vec­tor to de­liv­er com­bi­na­tions of mul­ti­ple genes that dri­ve in vi­vo re­pro­gram­ming of car­diac fi­brob­lasts to cre­ate new heart mus­cle cells, as well as a small mol­e­cule go­ing af­ter a still-un­named tar­get to treat ge­net­ic di­lat­ed car­diomy­opa­thy.

And as Tenaya pre­pares to po­ten­tial­ly go pub­lic, they’re look­ing at a tra­di­tion­al IPO rather than a SPAC, which has al­so seen sky­rock­et­ing amounts of cap­i­tal re­cent­ly.

“The heart fail­ure field had re­al­ly fall­en out of fa­vor for a pe­ri­od of time,” Ali said. “Tra­di­tion­al small mol­e­cule pro­grams against some of the usu­al tar­gets … had sort of pe­tered out, and there were sev­er­al fail­ures in large out­come stud­ies. So some­thing had to change.”

Mon­day’s round was led by RTW In­vest­ments, and new in­vestors RA Cap­i­tal, Fi­deli­ty Man­age­ment & Re­search Com­pa­ny and funds and ac­counts ad­vised by T. Rowe Price. Tenaya saw ad­di­tion­al par­tic­i­pa­tion from all ex­ist­ing in­vestors in­clud­ing The Col­umn Group, Cas­din Cap­i­tal, GV and oth­ers.

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Chris Gibson (Photo By Vaughn Ridley/Sportsfile for Web Summit via Getty Images)

Re­cur­sion founders gin for­tunes as IPO back­ers show­er $436M on one of the biggest boasts in AI -- based on some very small deals

In the AI drug development world, boasting often comes with the territory. Yet few can rival Recursion when it comes to claiming the lead role in what company execs like to call the industrialization of drug development, with promises of continued exponential growth in the number of drugs it has in the pipeline.

On Friday, the Salt Lake City-based biotech translated its unicorn-sized boasts into a killer IPO, pricing more than 24 million shares at the high end of its range and bringing in $436 million — with a large chunk of that promised by some deep-pocket backers.

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Covid-19 vac­cine halt drags on, an FDA ap­point­ment at long last, the great CRO con­sol­i­da­tion, 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.

Conference season is upon us, and while we’d much prefer to be wandering down the hallways and presentation rooms in person, the team is ready to cover the most consequential data coming out of these scientific meetings. Get in touch early if you have news to share.

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Eli Lil­ly asks FDA to re­voke EUA for Covid-19 treat­ment

Eli Lilly on Friday requested that the FDA revoke the emergency authorization for its Covid-19 drug bamlanivimab, which is no longer as effective as a combo therapy because of a rise in coronavirus variants across the US.

“With the growing prevalence of variants in the U.S. that bamlanivimab alone may not fully neutralize, and with sufficient supply of etesevimab, we believe now is the right time to complete our planned transition and focus on the administration of these two neutralizing antibodies together,” Daniel Skovronsky, Lilly’s CSO, said in a statement.

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Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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Osman Kibar (Samumed, now Biosplice)

Os­man Kibar lays down his hand at Sa­mumed, step­ping away from CEO role as his once-her­ald­ed an­ti-ag­ing biotech re­brands

Samumed made quite the entrance back in 2016, when it launched with some anti-aging programs and a whopping $12 billion valuation. That level of fanfare was nowhere to be found on Thursday, when the company added another $120 million to its coffers and quietly changed its name to Biosplice Therapeutics.

Why the sudden rebrand?

“We did that for obvious reasons,” CFO and CBO Erich Horsley told Endpoints News. “The name Biosplice echoes our science much more than Samumed does.”

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Ex­clu­sive in­ter­view: Pe­ter Marks on why full Covid-19 vac­cine ap­provals could be just months away

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, took time out of his busy schedule last Friday to discuss with Endpoints News all things related to his work regulating vaccines and the pandemic.

Marks, who quietly coined the name “Operation Warp Speed” before deciding to stick with his work regulating vaccines at the FDA rather than join the Trump-era program, has been the face of vaccine regulation for the FDA throughout the pandemic, and is usually spotted in Zoom meetings seated in front of his wife’s paintings.

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J&J faces CDC ad­vi­so­ry com­mit­tee again next week to weigh Covid-19 vac­cine risks

The CDC’s Advisory Committee on Immunization Practices punted earlier this week on deciding whether or not to recommend lifting a pause on the administration of J&J’s Covid-19 vaccine, but the committee will meet again in an emergency session next Friday to discuss the safety issues further.

The timing of the meeting likely means that the J&J vaccine will not return to the US market before the end of next week as the FDA looks to work hand-in-hand with the CDC to ensure the benefits of the vaccine still outweigh the risks for all age groups.

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Pascal Soriot (AstraZeneca via YouTube)

Af­ter be­ing goad­ed to sell the com­pa­ny, Alex­ion's CEO set some am­bi­tious new goals for in­vestors. Then Pas­cal So­ri­ot came call­ing

Back in the spring of 2020, Alexion $ALXN CEO Ludwig Hantson was under considerable pressure to perform and had been for months. Elliott Advisers had been applying some high public heat on the biotech’s numbers. And in reaching out to some major stockholders, one thread of advice came through loud and clear: Sell the company or do something dramatic to change the narrative.

In the words of the rather dry SEC filing that offers a detailed backgrounder on the buyout deal, Alexion stated: ‘During the summer and fall of 2020, Alexion also continued to engage with its stockholders, and in these interactions, several stockholders encouraged the company to explore strategic alternatives.’

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