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.