Aerami Therapeutics Announces Presentation of Phase 1 Clinical Trial Data for Lead Program AER-901 at the 2023 European Respiratory Society (ERS) International Congress

DURHAM, N.C., Sept. 12, 2023 (GLOBE NEWSWIRE) -- Aerami Therapeutics (“Aerami”), a clinical stage biopharmaceutical company dedicated to breathing life into the treatment of serious and rare cardiopulmonary diseases, today announced the presentation of results from the randomized double-blind, placebo-controlled Phase 1 clinical trial (NCT04903730) of AER-901 at the 2023 European Respiratory Society (ERS) International Congress. The ERS International Congress is being held September 9-13, 2023, at the MiCo convention center in Milan, Italy. The oral presentation and e-poster are available through the conference portal and will be posted to the Aerami website following the meeting.

AER-901, a proprietary liquid formulation of imatinib for inhalation delivered by a breath activated, high-performance, handheld, smart nebulizer, is in development to address the unmet medical need for people with serious and rare forms of pulmonary hypertension, including the lead indication, pulmonary hypertension associated with interstitial lung disease (PH-ILD), as well as pulmonary arterial hypertension (PAH). In animal models of pulmonary hypertension, imatinib reversed the proliferative, fibrotic, and inflammatory pulmonary vascular remodeling process that is believed to drive symptom severity, disease progression, and outcomes in many forms of the disease. In a Phase 3 clinical trial sponsored by Novartis, oral imatinib demonstrated substantial efficacy when added to 2 or 3 background standard-of-care medications for PAH. However, the efficacious oral doses were poorly tolerated with an unacceptable safety profile. By targeting administration of AER-901 directly to the lung, nonclinical data suggest that an approximate 10-fold dose reduction relative to oral imatinib is appropriate.

Clinical data presented at the 2023 ERS International Congress include safety, tolerability, and pharmacokinetic results from the AER-901 Phase 1 trial. The trial enrolled 84 healthy volunteers and included single ascending dose and multiple ascending dose studies, as well as a cross-over study that compared AER-901 with oral imatinib. Once- and twice-daily dosing with an additional formulation were also evaluated.

Highlights from the presentation include:

• AER-901 was generally well tolerated with predominantly mild adverse events. Low rates of gastrointestinal and systemic adverse events were observed, likely due to low systemic exposures relative to oral imatinib.
• AER-901 was absorbed rapidly, consistent with expectations of the liquid formulation of imatinib and Aerami’s nonclinical data.
• AER-901 demonstrated a distinct absorption profile compared with oral imatinib, suggesting efficient lung deposition and little, if any inadvertent swallowing.
  

“The data from our Phase 1 clinical trial demonstrate that the AER-901 drug-device combination can efficiently deliver imatinib directly to the lung with a potentially improved systemic safety profile,” said Dr. Gary Burgess, Aerami’s Chief Medical Officer. “These data are consistent with our hypothesis that AER-901 has the potential to achieve efficacy with improved safety and tolerability relative to oral imatinib, and we believe that they strongly support the design of our planned platform Phase 2 trial, uniPHied, which will enroll people with either PH-ILD or PAH.”

About AER-901
AER-901 is a drug-device combination that is designed to efficiently deliver imatinib therapy deep into the diseased tissues of the lung. AER-901 is comprised of a proprietary, liquid formulation of imatinib for inhalation that is administered by a breath-activated, high-performance, handheld, smart nebulizer that controls flow rate and provides real-time feedback to help optimize lung deposition. AER-901 is currently in clinical development for people with PH-ILD and PAH, two rare and serious forms of pulmonary hypertension with high unmet medical need for improved treatment options.

About the uniPHied Trial
The uniPHied trial is a planned Phase 2, placebo-controlled, randomized, parallel group, proof-of-concept platform clinical trial that will evaluate AER-901 in addition to background standard of care therapy. The trial will enroll people with either PH-ILD or PAH into one of 2 cohorts. Participants will then be randomized to receive either AER-901 or placebo administered twice daily via the AER-901 inhalation device. The primary endpoint for each cohort will be the percentage reduction in pulmonary vascular resistance (PVR) following 24 weeks of treatment. Aerami plans to allow all participants who complete the 24-week treatment period to enter a long-term extension study.

About Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD) 
Interstitial lung disease (ILD) is an umbrella term for several conditions that cause inflammation and scarring (fibrosis) of the lung tissue. Pulmonary hypertension is a serious complication of ILD for more than 80,000 people in the United States, Europe, and Japan, and is characterized by high blood pressure in the arteries of the lungs that is believed to be associated with pulmonary vascular remodeling. Pulmonary vascular remodeling is a proliferative, fibrotic, and inflammatory process that leads to narrowing and obstruction of small pulmonary arteries. Over time, pulmonary vascular remodeling is believed to drive increased pulmonary arterial pressure, which causes the heart to work harder as it pumps blood through the lungs, eventually leading to right heart failure. There is only one FDA-approved treatment for PH-ILD, and estimated survival is less than 5 years.

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, which disproportionately impacts women and frequently during the middle part of their lives, affects approximately 70,000 people in the United States and Europe. As in PH-ILD, pulmonary vascular remodeling is believed to drive PAH symptoms, disease progression, and outcomes. Currently approved treatments primarily mediate vasodilation, and despite advances in therapy, median survival remains approximately 5-7 years.

About Aerami Therapeutics
Aerami is a clinical stage biopharmaceutical company dedicated to breathing life into the treatment of serious and rare cardiopulmonary conditions. Aerami’s mission-driven approach to product development seeks to help patients live longer and live better by combining precision medicines and advanced administration platforms to support ease-of-use and quality-of-life. For more information visit our website at www.aerami.com.

This press release contains “forward-looking statements” which include statements, other than statements of historical fact, regarding, among other things: the plans for, or progress, scope, cost, initiation, duration, enrollment, results or timing for availability of results of, development of AER-901 or any other Aerami product candidate or the development of Aerami’s current and planned clinical trials, Aerami’s business development efforts and its expectations regarding its prospects, the planned Phase 2 uniPHied trial for AER-901 in patients with either PH-ILD or PAH, the potential approval and commercialization of AER-901, discussions with the FDA or EMA regarding Aerami’s programs, the potential benefits or competitive position of AER-901, the period for which Aerami’s cash resources will be sufficient to fund its operating requirements (runway), or Aerami’s plans, expectations or future operations, financial position, revenues, costs or expenses. 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, uncertainties associated with the clinical development process, including, among other things, the timing, expense, and results of the planned Phase 2 uniPHied trial for AER-901 as well as other clinical trials and regulatory processes, whether favorable findings from the Phase 1 trial for AER-901 to date will be predictive of results from the Phase 2 uniPHied trial or any other clinical trials for AER-901, Aerami’s ability to financially support its drug-device product candidate clinical development programs, the timing and outcome of Aerami’s anticipated interactions with regulatory authorities, Aerami’s ability to obtain coverage, pricing or reimbursement for any approved products in the United States or Europe, delays or other challenges in the recruitment of patients for, or the conduct of, Aerami’s clinical trials, and Aerami’s ability to make required payments under its outstanding convertible notes or any other indebtedness as they come due and Aerami’s ability to obtain additional financing and raise capital as necessary should the product development process become more extended. 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 cautions you not to place undue reliance on any forward-looking statement.

Aerami Media Contact:
Chad Whitaker
Aerami Therapeutics
cwhitaker@aerami.com

Aerami Investor Contact:
Jeremy Feffer
LifeSci Advisors
212-915-2568
jfeffer@lifesciadvisors.com

Released September 12, 2023