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.

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, just two weeks after submitting a supplemental BLA. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.

Rare diseases often have more than just physical effects on patients — especially when it comes to chronic conditions. In the case of the rare slow-growing blood cancer chronic lymphocytic leukemia (CLL), AbbVie wanted to try to assess the mental and emotional toll on patients.

So it surveyed more than 300 CLL patients, caregivers and physicians. While each group differed in how they felt — caregivers overwhelmingly (81%) felt positive about their role, for instance — patients described a “roller coaster” of emotions traversing diagnosis to treatment to remission and even relapse for some.