Ben Dake (Source: Aerovate)

RA Cap­i­tal-backed Aerovate launch­es with $72.6M to treat PAH with a re­pur­posed can­cer med

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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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Bet­ter Ther­a­peu­tics cuts 35% of staff while await­ing dig­i­tal ther­a­peu­tic ap­proval

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.

FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

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.

Steven James, Pionyr Immunotherapeutics CEO

Gilead pass­es on ful­ly ac­quir­ing Pi­o­nyr, as eyes now turn to Tizona, a fel­low sum­mer 2020 buy­out op­tion

Gilead and Pionyr Immunotherapeutics, a biotech trying to follow up on the first generation of checkpoint inhibitors, have “mutually agreed” on a rewrite to their 2020 terms, with Gilead deciding not to buy out the company.

The California biopharma waived its option to acquire the remaining 50.1% of Pionyr, which would have triggered a $315 million upfront payment and up to $1.15 billion down the road. Had Gilead waited to decide, the drugmaker would have had a potential payment to make in the near term under their agreement, a spokesperson said in an email to Endpoints News.

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Zhi Hong, Brii Biosciences CEO

Brii Bio­sciences stops man­u­fac­tur­ing Covid-19 an­ti­body com­bo, plans to with­draw EUA re­quest

Brii Biosciences said it will stop manufacturing its Covid-19 antibody combination, sold in China, and is working to withdraw its emergency use authorization request in the US, which it started in October 2021.

The Beijing and North Carolina biotech commercially launched the treatment in China last July but is now axing the work and reverting resources to other “high-priority programs,” per a Friday update. The focus now is namely hepatitis B viral infection, postpartum depression and major depressive disorders.

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