Aerami Therapeutics Announces Completion of Dosing in Phase 1 Trial of AER-901 (inhaled imatinib), a Critical Milestone Toward the Planned Initiation of a Phase 2 Trial in Pulmonary Arterial Hypertension (PAH)

• AER-901 (inhaled imatinib), a potential reverse-remodeling therapy, is being developed to address unmet need for PAH treatments that target pulmonary vascular remodeling while supporting ease-of-use for patients.
• PAH is a rare and progressive disease with no cure; pulmonary vascular remodeling is believed to drive disease progression and poor outcomes, however, all currently approved therapies primarily mediate vasodilation.
• Aerami plans to advance AER-901 into a Phase 2 trial during the first half of 2023, following an amendment to the open AER-901 IND application with the U.S. Food and Drug Administration (FDA).

DURHAM, N.C., Dec. 21, 2022 (GLOBE NEWSWIRE) -- Aerami Therapeutics Holdings, Inc (“Aerami”), a clinical stage biopharmaceutical company dedicated to breathing life into the treatment of serious cardiopulmonary and cardiometabolic conditions, today announced completion of dosing in a Phase 1 study of AER-901, a drug-device combination comprised of imatinib for inhalation and the FOX^® nebulizer.

The Phase 1 study (NCT04903730) of AER-901 was designed to assess safety, tolerability, and pharmacokinetics (PK) versus placebo after single and multiple ascending doses in healthy volunteers. In addition, alternative AER-901 formulations and once-daily and twice-daily administration were evaluated; a single dose of AER-901 was compared to a single dose of oral imatinib in a crossover cohort. AER-901 doses evaluated in the Phase 1 study were 5-to-80-fold lower than the oral imatinib doses evaluated in IMPRES.

Imatinib demonstrated clinically meaningful and statistically significant improvement in pulmonary hemodynamics and six-minute walk distance (6MWD) in the Phase 3 IMPRES study when delivered orally. Nevertheless, poor tolerability and systemic adverse events were observed in the trial, potentially due to use of high oral doses (200 to 400 mg once-daily), and imatinib was never approved in PAH. Inhaled administration of medications, such as imatinib with AER-901, may allow high drug concentrations to be delivered to the lung, potentially achieving similar efficacy at a fraction of the oral dose.

“Completion of dosing in our Phase 1 trial of AER-901 marks an important milestone toward our goal of realizing the potential of imatinib in PAH through targeted delivery to the deep lung,” said Lisa Yañez, chief executive officer of Aerami. “We look forward to progressing AER-901 through clinical development and continuing on our path towards obtaining regulatory approval of AER-901 for the treatment of PAH.”

Aerami plans to amend the open Investigational New Drug (IND) application with the FDA to initiate a Phase 2 proof-of-concept clinical trial in the first half of 2023.

About AER-901
AER-901 is a drug-device combination that is designed to deliver potentially reverse-remodeling therapy with imatinib deeply and efficiently throughout the diseased tissue of the lung via the FOX^® nebulizer, a handheld device which uses a mechanical flow regulator to control the patient’s inhalation flow. AER-901 is in development under an exclusive global license from Vectura Group plc and the FOX^® nebulizer is both 510(k) cleared and CE marked. A version of the FOX^® nebulizer is currently in use in Europe as part of another drug-device combination for the treatment of PAH.

About Pulmonary Arterial Hypertension (PAH)
PAH is a rare and progressive form of pulmonary hypertension characterized by high blood pressure in the arteries of the lungs due to their narrowing or a blockage. PAH affects approximately 70,000 patients in the United States and Europe. Pulmonary vascular remodeling is thought to be the major driver of disease progression, leading to right heart failure, lung transplantation or death. Pulmonary vascular remodeling leads to narrowing and obstruction of small pulmonary arteries that increases pulmonary arterial pressure, which makes the heart work harder as it pumps blood through the lungs, eventually leading to right heart failure and, ultimately, death. Currently approved therapies primarily mediate vasodilation and lung or heart-lung transplantation is the only definitive treatment. Median survival remains approximately 5-7 years.

About Aerami Therapeutics Holdings, Inc.
Aerami is a clinical stage biopharmaceutical company dedicated to breathing life into the treatment of serious cardiopulmonary and cardiometabolic conditions. In addition to AER-901, in development for PAH, the company’s cardiometabolic portfolio includes drug-device combination product candidates in Types 1 and 2 diabetes, which leverage Aerami’s proprietary AFINA^TM inhaler technology. Our product candidates have not been approved by any regulatory authority, and their safety, efficacy or other desirable attributes have not been established. For more information visit http://www.aerami.com.

This press release contains “forward-looking statements” concerning the development and commercialization of Aerami’s product candidates, timing of clinical trials, the company’s business development efforts and its expectations regarding its prospects, including, but not limited to, the timing and outcome of current and planned clinical trials. Forward-looking statements are subject to risks, assumptions and uncertainties that could cause actual future events or results to differ materially from such statements, including, but not limited to, risks associated with the clinical development process and the ability to obtain funding to support planned clinical activities, such as the completion of Aerami’s planned Phase 2 trial for AER-901. These statements are made as of the date of this press release. Actual results may vary. Aerami undertakes no obligation to update any forward-looking statements for any reason.

Aerami Investor and Media Contact:
Joshua Ziel, PhD
VP, Program Development
jziel@aerami.com