Aerovate Therapeutics Closes on $72.6 million Series A Funding to Advance Repurposed Cancer Drug for PAH

Aug 13, 2020 | Investor Watch, News

Aerovate Therapeutics Closes on $72.6 million Series A Funding to Advance Repurposed Cancer Drug for PAH

Aerovate Therapeutics has closed on $72.6 million in Series A funding to advance trials of AV-101, an inhaled, dry powder aerosol version of imatinib to treat pulmonary arterial hypertension (PAH). The Series A for Aerovate, which was founded and incubated by RA Capital Management, is led by Sofinnova Investments, a new investor in the company. Atlas Venture, Cormorant Asset Management, Surveyor Capital (a Citadel company), and Osage University Partners are the other new investors in the round. 

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

Novartis previously developed an oral version of imatinib (Gleevec), which showed significant efficacy in a phase 3 trial as a disease-modifying therapy for PAH, but it was poorly tolerated due to systemic side effects. Aerovate believes that its inhaled formulation should decrease the adverse effects the oral product revealed by reducing systemic exposure to the drug while simultaneously increasing the dose of medicine delivered directly to diseased lung tissues.

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

“With the support of the high caliber syndicate around the table, I believe Aerovate is poised to move this important treatment into the clinic immediately. This financing keeps the company on a rapid path towards offering this potentially transformational therapeutic to patients with real need,” said RA Capital Managing Director and Aerovate Board Chair Joshua Resnick, MD. “AV-101 strives to provide PAH patients with the benefits of a disease-modifying drug – in an easy-to-use format, free from the inconvenience of nebulized products – ­without the cancer-drug-like side effects of the oral version. This is extremely encouraging news for sufferers of this disease,” said Resnick.

“We are excited about AV-101’s potential because the core molecule, imatinib, has shown real efficacy for this use, including cases of hemodynamic remission in severely ill PAH patients. Aerovate’s team and their deep experience with PAH and aerosol development suggest that AV-101 may progress quickly and successfully through trials,” said Sofinnova General Partner and Aerovate Board Director Maha Katabi, PhD.

PAH is a proliferative disorder of the pulmonary vasculature 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. Even with the current standard of care vasodilator therapies, which dilate pulmonary vessels but do not directly modify the disease, PAH often progresses at a fast pace. Aerovate estimates that, based on $5 billion annual sales from the currently approved vasodilator drugs, the market for disease-modifying PAH therapies is significant.


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