The land­mark can­cer drug ima­tinib has been on the mar­ket since 2001, first sold by No­var­tis as Gleevec and in re­cent years as a gener­ic. Now, a new Boston biotech is aim­ing to re­pur­pose the drug as a treat­ment for pul­monary ar­te­r­i­al hy­per­ten­sion.

Aerovate emerged from stealth Thurs­day and an­nounced a $72.6 mil­lion Se­ries A, which will be used to de­vel­op and run tri­als for its can­di­date AV-101 — a dry pow­der ver­sion of ima­tinib meant to be used with an in­haler. The com­pa­ny emerged from RA Cap­i­tal’s in­cu­ba­tor and fund­ing was led by Sofinno­va.

Ima­tinib had been test­ed oral­ly in PAH pa­tients be­fore, CEO Ben Dake tells me. Re­sults from a Phase III tri­al with the oral de­liv­ery ver­sion showed promis­ing ef­fi­ca­cy lev­els, but PAH pa­tients didn’t tol­er­ate the drug well.

“Tak­ing an an­ti-pro­lif­er­a­tive can­cer drug and try­ing to fig­ure out a way to make it work in a dif­fer­ent dis­ease made a lot of sense to me and to the team,” Dake told End­points News. “But even though it was dif­fi­cult for them to tol­er­ate, it did work well on ex­er­cise ca­pac­i­ty, which is the pri­ma­ry end­point for ap­proval for PAH drugs, as well as he­mo­dy­nam­ics.”

One of the key chal­lenges Aerovate faced in de­vel­op­ment was how to de­liv­er the drug ex­act­ly to where it need­ed to be in the lungs. The biotech set­tled on an in­haled method but, due to the na­ture of such ther­a­pies, still had to de­ter­mine pre­cise­ly the right size par­ti­cle and make sure the mol­e­cule doesn’t ab­sorb wa­ter.

Ul­ti­mate­ly the com­pa­ny set­tled on ima­tinib and craft­ed a prod­uct that they hope is eas­i­er to use than oth­er PAH treat­ments like a liq­uid neb­u­liz­er, in which pa­tients have to mix the so­lu­tions them­selves and car­ry around a large de­vice. Af­ter set­tling on ima­tinib and pri­or­i­tiz­ing ease of use, Aerovate is con­tent with where it land­ed.

“We had to do quite a bit of work to find a good, sta­ble, well-per­form­ing aerosol,” Dake said. “It’s not some­thing as sim­ple as grind­ing up a pill.”

Aerovate sees it­self as step­ping in PAH pa­tients whose con­di­tions have wors­ened de­spite oth­er ther­a­pies, Dake said. Gen­er­al­ly, some­one with PAH will start on a reg­i­men of PDE5 in­hibitors that di­late blood ves­sels and progress through two or three oth­er pills be­fore po­ten­tial­ly need­ing an IV or sub­cu­ta­neous pump.

At that point, there is no fur­ther treat­ment avail­able. AV-101 aims to fill that gap as Dake says it shifts the ther­a­py par­a­digm from stan­dard of care to dis­ease-mod­i­fy­ing treat­ment.

“We want­ed to be aware of where the ther­a­peu­tic fo­cus was go­ing and not with an old­er-style of drug, and there’s oth­er ad­van­tages to dry pow­der but the main one is for pa­tients in that it’s easy to use,” Dake said. “Where we would come in is where pa­tients are al­ready on a dou­ble ther­a­py or triple ther­a­py, they can add on our in­haler. And that is where the oral [ima­tinib] tri­al was run.”

Long-term, Aerovate has oth­er pro­grams it would like to de­vel­op but is fo­cused on bring­ing AV-101 to mar­ket. Josh Resnick, a man­ag­ing di­rec­tor at RA Cap­i­tal and mem­ber of Aerovate’s board, said the path to com­mer­cial­iza­tion is “rel­a­tive­ly straight­for­ward” giv­en that the FDA is al­ready fa­mil­iar with the ima­tinib com­pound.

“It’s a pa­tient pop­u­la­tion that’s well-un­der­stood, it’s a set of physi­cians that’s well-un­der­stood, so fun­da­men­tal­ly we an­tic­i­pate it to be a first-in-class ther­a­peu­tic in the space and get it in­to the hands of the pa­tients,” Resnick said.

Oth­er in­vestors join­ing Sofinno­va are At­las Ven­ture, Cor­morant As­set Man­age­ment, Sur­vey­or Cap­i­tal and Os­age Uni­ver­si­ty Part­ners.

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.