- Topline data expected in
June 2024 from Phase 2b portion of the IMPAHCT global Phase 2b/Phase 3 clinical trial of AV-101 for pulmonary arterial hypertension (PAH)
- Completed enrollment in the Phase 2b portion and enrolled first patient into the Phase 3 portion of the IMPAHCT trial of AV-101 in
November 2023
- Expanded intellectual property portfolio with two issued patents in 2023
- Board of Directors expanded in 2023 with the addition of
Donald Santel andHabib Dable , each a former CEO with PAH experience and significant product approval and launch expertise
- Cash runway into 2026, based on our current operating plan
“Aerovate reached important milestones in 2023 with the achievement of full enrollment of the Phase 2b portion and the first patient enrolled in the Phase 3 portion of our IMPAHCT trial evaluating AV-101 for patients with PAH,” said
2023 Highlights
Enrollment Complete and Topline Data Expected in
First Patient in Phase 3 Portion of IMPAHCT Enrolled in
Trial Data Published and Presented at Scientific Conferences. We presented at the
Expanded Intellectual Property Portfolio. We continued expanding our intellectual property portfolio in 2023 with the issuance of two additional patents bringing the total issued patents to six. In 2023, the United States Patent and Trademark Office issued Patents 11,806,349 and 11,813,263 to us, and we continue prosecuting multiple additional applications to cover our product globally.
Expanded Board of Directors with Experience in PAH. We appointed
2023 Financial Results
Cash, cash equivalents and short-term investments totaled
R&D expenses: Research and development (R&D) expenses for the year ended
G&A expenses: General and administrative (G&A) expenses for the year ended
Net loss: Net loss for the year ended
Financial guidance: We expect that our cash, cash equivalents and available-for-sale investments will be sufficient to fund our operations into 2026, based on our current operating plan.
About AV-101
AV-101 is an investigational, proprietary dry powder inhaled formulation of the antiproliferative drug imatinib. Developed specifically for pulmonary arterial hypertension (PAH), AV-101 targets cellular hyperproliferation and resistance to apoptosis, driven by improper signaling in cells of the distal pulmonary arteries. By targeting the proliferation and accumulation of cells in the arteries of the lungs, we believe AV-101 has the potential to provide meaningful improvements for patients beyond the capabilities of currently approved therapies. AV-101 is designed for delivery by an easy-to-use dry powder inhaler, directly into the lungs to maximize potential clinical benefit and limit systemic adverse effects. Phase 1 results published in
About the IMPAHCT Trial
IMPAHCT (Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial) is a multi-national, placebo-controlled Phase 2b/Phase 3 trial in adults with PAH that continuously enrolled patients as the study progresses from Phase 2b to Phase 3. The Phase 2b portion of the trial will evaluate three doses of AV-101 over 24 weeks, compared to placebo, to identify an optimal dose based on the primary endpoint, change in pulmonary vascular resistance (PVR), and safety, tolerability, and other clinical measures. The Phase 3 portion of the trial will compare patients taking the optimal dose of AV-101, selected from the Phase 2b data, to placebo. The primary endpoint of the Phase 3 portion of the trial will be change in six-minute walk distance (6MWD) over 24 weeks versus placebo. More information about this trial is available at https://clinicaltrials.gov/ct2/show/NCT05036135https://clinicaltrials.gov/ct2/show/NCT05036135.
About
Aerovate is a clinical-stage biopharmaceutical company focused on developing drugs that meaningfully improve the lives of patients with rare cardiopulmonary disease. Aerovate’s initial focus is on advancing AV-101, its proprietary dry powder inhaled formulation of the drug imatinib for the treatment of patients with PAH. Learn more at aerovatetx.com or follow the company on X (formerly known as Twitter) and LinkedIn.
Available Information
Aerovate announces material information to the public about the Company, its products and services, and other matters through a variety of means, including filings with the
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements can be identified by words such as “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “future,” “goal,” “intend,” “look forward to,” “may,” “plan,” “potential,” “predict,” “project,” “potential,” seek,” “strategy,” “should,” “target,” “will,” “would” and similar expressions regarding future periods. These forward-looking statements include, but are not limited to, statements regarding the therapeutic potential and clinical benefits of AV-101 in PAH; our anticipated timing for the release of topline data from the Phase 2b portion of the IMPAHCT trial; our expectations regarding continuing patient enrollment for the Phase 3 portion of the IMPAHCT trial; our belief that we will have capital to fund Aerovate into 2026; our expectations regarding the strength of our intellectual property portfolio globally; our business plans and objectives for AV-101, including potential regulatory submissions and approvals; the anticipated contribution of the members of our board of directors and management team to our operations and progress; and our growth and goals as a company.
Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those risks and uncertainties related to the therapeutic potential and clinical benefits of AV-101; the timing associated with patient enrollment, initiation, delivery of drug supply and continuation of our Phase 2b/Phase 3 trial of AV-101 in PAH patients; clinical trials, operations and goals; positive results from a clinical study may not necessarily be predictive of the results of future or ongoing clinical studies; regulatory developments in
Condensed Consolidated Balance Sheets | ||||||||
(Unaudited) | ||||||||
(in thousands) | ||||||||
2023 | 2022 | |||||||
Assets | ||||||||
Cash, cash equivalents and available-for-sale securities | $ | 122,439 | $ | 129,220 | ||||
Other assets | 4,979 | 6,081 | ||||||
Total assets | 127,418 | 135,301 | ||||||
Liabilities and Stockholders' Equity | ||||||||
Accounts payable and accrued and other current liabilities | $ | 17,161 | $ | 7,397 | ||||
Other liabilities | 745 | 1,161 | ||||||
Total liabilities | 17,906 | 8,558 | ||||||
Total stockholders’ equity | 109,512 | 126,743 | ||||||
Total liabilities and stockholders' equity | $ | 127,418 | $ | 135,301 |
Condensed Consolidated Statements of Operations and Comprehensive Loss | ||||||||||||||||
(Unaudited) | ||||||||||||||||
(in thousands, except share and per share amounts) | ||||||||||||||||
Three Months Ended | Year Ended | |||||||||||||||
2023 | 2022 | 2023 | 2022 | |||||||||||||
Operating expenses: | ||||||||||||||||
Research and development | $ | 17,813 | $ | 12,221 | $ | 64,219 | $ | 38,622 | ||||||||
General and administrative | 4,253 | 3,631 | 17,190 | 14,615 | ||||||||||||
Total operating expenses (1) | 22,066 | 15,852 | 81,409 | 53,237 | ||||||||||||
Loss from operations | (22,066 | ) | (15,852 | ) | (81,409 | ) | (53,237 | ) | ||||||||
Total other income | 1,709 | 856 | 5,944 | 1,751 | ||||||||||||
Provision for income taxes | 56 | 25 | 56 | 25 | ||||||||||||
Net loss | $ | (20,413 | ) | $ | (15,021 | ) | $ | (75,521 | ) | $ | (51,511 | ) | ||||
Net loss per share, basic and diluted | $ | (0.74 | ) | $ | (0.61 | ) | $ | (2.87 | ) | $ | (2.10 | ) | ||||
Weighted-average shares of common stock outstanding, basic and diluted | 27,695,184 | 24,610,723 | 26,331,630 | 24,472,104 | ||||||||||||
(1) Non-cash charges were | ||||||||||||||||
Media Contact
info@aerovatetx.com
Investor Contact
ir@aerovatetx.com
Source:
2024 GlobeNewswire, Inc., source