WALTHAM, Mass., June 17, 2024 (GLOBE NEWSWIRE) — Aerovate Therapeutics, Inc. (Nasdaq: AVTE) today announced topline results from the Phase 2b portion of the II inhaledMr.atinib P.pulmonary Amaterial Hhypertension VSline Trial (IMPAHCT), a multinational phase 2b/phase 3, randomized, double-blind, placebo-controlled trial of AV-101, a novel inhaled dry powder formulation of imatinib, in adults with pulmonary arterial hypertension (PAH).
The objective of the Phase 2b portion of IMPAHCT was to evaluate the efficacy, safety, and tolerability of three different doses of AV-101 compared to placebo. The primary endpoint of the phase 2b part of IMPAHCT is change in PVR compared to placebo. Results showed that although AV-101 was well tolerated in all dose groups, the study did not meet its primary endpoint of improvement in PVR compared to placebo for any doses studied nor showed significant improvements in the secondary endpoint of six-minute change. walking distance (6MWD).
Primary Endpoint – ITT Analysis of PVR (dynes*sec/cm^5)
| Dose | Least squares mean difference from placebo (95% CI) | P-value |
| 10 mg twice daily (N=50) | 42.8 (-80.57 to 166.09) | 0.4968 |
| 35 mg twice daily (N=49) | -5.5 (-129.16 to 118.18) | 0.9306 |
| 70 mg twice daily (N=51) | -57.0 (-181.14 to 67.20) | 0.3685 |
Secondary endpoint – ITT analysis of 6MWD (meters)
| Dose | Least squares mean difference from placebo (95% CI) |
| 10 mg twice daily (N=50) | -11.7 (-34.75 to 11.26) |
| 35 mg twice daily (N=49) | -4.2 (-27.74 to 19.37) |
| 70 mg twice daily (N=51) | +1.3 (-22.09 to 24.60) |
The Company also reviewed data from several additional secondary endpoints from the Phase 2b portion of IMPAHCT, which also did not show significant improvements. Based on these results, Aerovate, in agreement with the study’s independent advisory committee, is halting recruitment and stopping the phase 3 portion of IMPAHCT as well as the long-term extension study.
“The results from phase 2b of the IMPAHCT study were unexpected and disappointing. Our immediate goal is to share these results transparently with researchers, patients and the PAH community. In the coming weeks, we will work closely with the IMPAHCT Study Advisory Committee and the PAH community to discuss these data and their implications in depth,” said Tim Noyes, CEO of Aerovate. “We express our sincere gratitude to all trial participants, investigators and site teams for their dedication to advancing therapeutic options for the treatment of lung diseases. high blood pressure.”
Aerovate plans to release the full data from the phase 2b portion of IMPAHCT at a later date, the timing of which remains to be determined.
As of June 15, 2024, Aerovate had approximately $100 million in cash, cash equivalents and short-term investments.
About the AV-101
AV-101 is an investigational, proprietary inhaled dry powder formulation of the antiproliferative drug imatinib. Developed specifically for pulmonary arterial hypertension (PAH), AV-101 targets cellular hyperproliferation and resistance to apoptosis, caused by inappropriate signaling in cells of the distal pulmonary arteries. AV-101 is designed to be delivered by an easy-to-use dry powder inhaler directly to the lungs to maximize potential clinical benefits and limit systemic adverse effects.
About the IMPAHCT Trial
IMPAHCT (Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial) is a multinational phase 2b/phase 3 placebo-controlled trial in adults with PAH that enrolled patients continuously from phase 2b to phase 3. The phase 2b part of the trial evaluated 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 . More information about this trial is available at https://clinicaltrials.gov/ct2/show/NCT05036135.
About Aerovate Therapeutics, Inc.
Aerovate is a clinical-stage biopharmaceutical company focused on developing medicines that significantly improve the lives of patients with rare cardiopulmonary diseases. Aerovate’s initial goal is to advance AV-101, its proprietary inhaled dry powder formulation of the drug imatinib for the treatment of patients with PAH. Learn more at aerovatetx.com or follow the company on X (formerly Twitter) and LinkedIn.
Information available
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Caution 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”, “objective”, “have the intention to”, “anticipate”. “can”, “plan”, “potential”, “predict”, “project”, “seek”, “strategy”, “should”, “target”, “will”, “would” and similar expressions regarding the future . periods. These forward-looking statements include, but are not limited to, statements regarding the Phase 2b/Phase 3 IMPAHCT study, including the future release of full clinical data and the Company’s plan to discontinue the Phase 3 portion of the IMPAHCT trial.
Any forward-looking statements contained 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 differ materially from those expressed or implied by any forward-looking forecast. statements contained in this press release, including, without limitation, risks and uncertainties relating to the therapeutic potential and clinical benefits of AV-101; the timeline associated with the identification and activation of clinical sites, patient recruitment, initiation, delivery of drug supply and continuation of our Phase 2b/Phase 3 trial on the ‘AV-101 in patients with PAH; the impact of public health crises on our business, clinical trials, operations and objectives; positive results from a clinical study are not necessarily predictive of results from future or ongoing clinical studies; regulatory developments in the United States and foreign countries; as well as the risks and uncertainties discussed in more detail under the heading “Risk Factors” in our most recent Annual Report on Form 10-Q filed with the SEC and subsequent filings with the SEC. We caution you not to place undue reliance on forward-looking statements, which speak only as of the date they are made. We undertake no obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which such statements may be based, or which may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent our views only as of the date hereof and should not be relied upon as representing our views as of any subsequent date.
Media contact
Marites Coulter
marites.coulter@vergescientific.com
Investor contact
IR@Aerovatetx.com
