RARE Daily

Aerovate Emerges with $72.6 Million to Develop PAH Therapy

August 7, 2020

Rare Daily Staff

Aerovate Therapeutics wants to repurpose the cancer drug imatinib (better known as the 20-year old drug Gleevec), to treat pulmonary arterial hypertension and has raised $72.6 million in a series A financing to do that.

RA Capital Management founded and incubated Aerovate. Sofinnova Investments led the series A financing with participation by new investors Atlas Venture, Cormorant Asset Management, Surveyor Capital, and Osage University Partners.

Pulmonary arterial hypertension (PAH) is a rare proliferative disorder of the pulmonary vasculature that progresses rapidly and afflicts approximately 80,000 people worldwide and for which there are, to date, no approved disease-modifying therapies. PAH is characterized by remodeling, constriction, and occlusion of the small pulmonary arteries resulting in elevated blood pressure in the pulmonary circulation. PAH causes significant strain on the heart, leading to limited physical activity, heart failure, and reduced life expectancy.

Vasodilator therapies, the current standard of care, dilate pulmonary vessels but do not directly modify the disease. PAH often progresses at a fast pace. The five-year survival rate is only 57 percent. Aerovate estimates that based on $5 billion annual sales from the currently approved vasodilator drugs, the market for disease modifying PAH therapies is significant.

An oral version of imatinib showed significant efficacy in a phase 3 trial as a disease-modifying therapy for PAH, but it was poorly tolerated by PAH patients because of systemic side effects. Aerovate’s AV-101is a proprietary inhaled, dry powder aerosol version of imatinib to treat pulmonary arterial hypertension.

The financing will take AV-101 through a clinical efficacy read-out, with a registrational trial with PAH patients scheduled to begin during the first half of 2021 with PAH patients.

Imatinib addresses the underlying cause of the disease, unlike currently available vasodilator therapies. In multiple trials, imatinib showed strong clinical benefit for PAH patients that lasted for many months; they experienced improved exercise capacity, lower pulmonary vascular resistance, decreased pulmonary artery pressure and increased cardiac output.

Aerovate’s AV-101 is an easy-to-use, proprietary dry powder formulation of imatinib that delivers the medicine directly to the lungs. This approach should decrease the systemic adverse effects that hampered the oral version in PAH trials by reducing systemic exposure to the drug while simultaneously increasing the dose of medicine delivered directly to diseased lung tissues. AV-101 is administered with a simple inhaler, providing a convenient solution for patients compared to more cumbersome nebulized drugs currently approved in PAH. Those often require prolonged dosing protocols lasting many minutes, multiple times a day, including complicated device assembly and filling – likely reducing patient adherence and impacting real-world efficacy.

“Building on imatinib’s established efficacy, our research gives us strong confidence that AV-101, through targeted delivery to the lungs, will minimize systemic adverse effects, provide good tolerability and potentially be even better at treating the underlying disease than the oral version,” said Hunter Gillies, chief medical officer of Aerovate.

Aerovate emerged out of RA Capital’s seed and incubation program. The concept and proprietary drug product underlying AV-101 were co-invented by the RA Capital venture group and supported by its incubator, Carnot Pharma.

Photo: Hunter Gillies, chief medical officer of Aerovate

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