Aerovate Raises $72.6M to Develop Potential PAH Inhaled Therapy AV-101

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by Forest Ray PhD |

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Aerovate Therapeutics received $72.6 million in Series A funding to advance trials testing AV-101, an experimental inhaled version of imatinib for the treatment of pulmonary arterial hypertension (PAH).

AV-101 is an inhaled dry powder that delivers imatinib directly to a patient’s lungs.

Benjamin Dake, PhD, Aerovate founding president, said this financing will allow the company to advance the clinical development of AV-101, with a registrational trial enrolling PAH patients scheduled to begin during the first half of 2021. “Sufferers of PAH desperately need a disease-modifying therapy,” Dake said in a press release.

According to Aerovate, an oral version of imatinib showed promising results in a Phase 3 trial as a disease-modifying therapy for PAH. However, that version was poorly tolerated by patients because of widespread side effects.

The inhaled version is expected to reduce systemic, or whole-body, exposure to imatinib, while effectively increasing the dose of medicine that reaches lung tissues — where it is most needed.

“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 Maha Katabi, PhD, Aerovate’s board director and a general partner at Sofinnova Investments.

Sofinnova led the the Series A funding. Other investors include Atlas Venture, Cormorant Asset Management, Surveyor Capital (a Citadel company), and Osage University Partners.

Aerovate was founded by RA Capital Management and incubated by Carnot Pharma, a spinoff of RA Capital.

“It is exciting to secure financing from such a strong syndicate to progress AV-101, our lead program,” Dake said.

“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,” said Joshua Resnick, MD, RA Capital managing director and Aerovate board chair.

“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,” he added.

Aerovate expects AV-101 to be easier to use than other nebulized medications. It will be administered via a simple inhaler, unlike other medications that often involve prolonged dosing protocols that may take several minutes and must be performed multiple times per day. The complicated assembly and filling procedures involved in some nebulizers also may impact how consistently patients use them, thereby affecting their efficacy.

PAH affects an estimated 80,000 people worldwide, and to date there is no approved disease-modifying therapy — one that addresses a condition’s underlying cause — Aerovate noted.

“Imatinib addresses the underlying cause of the disease, unlike currently available vasodilator therapies,” said Hunter Gillies, MD, Aerovate’s chief medical officer. “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.”

The therapy has long been used for treating certain types of cancer.

“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,” Gillies concluded.

A Conversation With Rare Disease Advocates