Aerovate Therapeutics Announces 24-Week Topline Results from the Phase 2b Portion of IMPAHCT Evaluating AV-101 for the Treatment of Pulmonary Arterial Hypertension
Aerovate Therapeutics announced 24-week topline results from the Phase 2b portion of the IMPAHCT trial evaluating AV-101 for pulmonary arterial hypertension (PAH). The trial did not meet its primary endpoint of reducing pulmonary vascular resistance (PVR) or secondary endpoint of improving six-minute walk distance (6MWD) for any studied dose. AV-101 was well-tolerated at all doses but failed to show meaningful benefits in additional secondary endpoints as well. Consequently, Aerovate will halt the Phase 3 portion and the long-term extension study of IMPAHCT. The company aims to engage with the PAH community to discuss the results and plans to release full data later. As of June 15, 2024, Aerovate holds approximately $100 million in cash and equivalents.
- AV-101 was well-tolerated across all dose groups, indicating a favorable safety profile.
- Aerovate holds approximately $100 million in cash and equivalents as of June 15, 2024, providing financial stability.
- AV-101 did not meet the primary endpoint of reducing pulmonary vascular resistance (PVR) for any studied dose.
- No meaningful improvements were observed in the secondary endpoint of six-minute walk distance (6MWD).
- Additional secondary endpoints also failed to show meaningful improvements.
- Enrollment and further stages of the IMPAHCT trial will be halted, impacting future research and development.
- The failure to meet primary and secondary endpoints could negatively affect investor sentiment and stock performance.
Insights
The topline results from Aerovate's Phase 2b clinical trial for AV-101 are indeed disappointing. Not meeting the primary endpoint of improving pulmonary vascular resistance (PVR) is a significant setback. In clinical trials, achieving the primary endpoint is important as it serves as the main measure of the drug's efficacy. Additionally, the failure to show meaningful improvement in the secondary endpoint, such as the six-minute walk distance (6MWD), further questions the potential of AV-101 for treating pulmonary arterial hypertension (PAH). PAH is a severe condition characterized by high blood pressure in the lungs' arteries, making effective treatment options critical. The decision to halt the Phase 3 portion and the long-term extension study is a prudent step given these results. It allows Aerovate to conserve resources and avoid further investment in a drug that does not appear to yield the desired benefits.
From an investment perspective, the news of AV-101 not meeting its primary endpoint is likely to have a negative impact on Aerovate's stock price in the short term. Investors typically view the failure to achieve primary and secondary endpoints in a Phase 2b trial as a significant risk, especially when the trial was expected to pave the way for a Phase 3 study. The halt in further development of AV-101 signifies a potential loss in projected future revenues. However, Aerovate's decision to halt the trial also demonstrates fiscal responsibility, possibly preserving their cash reserves of approximately
AV-101 did not meet the primary endpoint of change in pulmonary vascular resistance (PVR) for any of the studied doses
WALTHAM, Mass., June 17, 2024 (GLOBE NEWSWIRE) -- Aerovate Therapeutics, Inc. (Nasdaq: AVTE) today announced topline results from the Phase 2b portion of the Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial (IMPAHCT), a Phase 2b/Phase 3, randomized, double-blind, placebo-controlled, multi-national trial of AV-101, a novel dry powder inhaled formulation of imatinib, in adults with pulmonary arterial hypertension (PAH).
The objective of the Phase 2b portion of IMPAHCT was to assess the efficacy, safety and tolerability of three different doses of AV-101 compared to placebo. The primary endpoint for the Phase 2b portion of IMPAHCT is change in PVR compared with placebo. Results showed that, while AV-101 was well tolerated across all dose groups, the study did not meet its primary endpoint for improvement in PVR compared to placebo for any of the studied doses or show meaningful improvements in the secondary endpoint of change in six minute walk distance (6MWD).
Primary Endpoint – ITT analysis of PVR (dynes*sec/cm^5)
| Dose | Least-squares mean difference as compared with placebo ( | P value |
| 10mg BID (N=50) | 42.8 (-80.57 to 166.09) | 0.4968 |
| 35mg BID (N=49) | -5.5 (-129.16 to 118.18) | 0.9306 |
| 70mg BID (N=51) | -57.0 (-181.14 to 67.20) | 0.3685 |
Secondary Endpoint – ITT analysis of 6MWD (meters)
| Dose | Least-squares mean difference as compared with placebo ( |
| 10mg BID (N=50) | -11.7 (-34.75 to 11.26) |
| 35mg BID (N=49) | -4.2 (-27.74 to 19.37) |
| 70mg BID (N=51) | +1.3 (-22.09 to 24.60) |
The Company has also reviewed data from several additional secondary endpoints of the Phase 2b portion of IMPAHCT, which also failed to show meaningful improvements. Based upon these results, Aerovate, in agreement with the independent study advisory committee, is halting enrollment and shutting down the Phase 3 portion of IMPAHCT as well as the long-term extension study.
"The results of the Phase 2b portion of IMPAHCT were unexpected and disappointing. Our immediate focus is on transparently sharing these findings with investigators, patients and the PAH community. In the coming weeks, we will engage closely with the IMPAHCT study advisory committee and the PAH community to thoroughly discuss these data and their implications,” said Tim Noyes, Chief Executive Officer of Aerovate. “We extend our heartfelt gratitude to all trial participants, investigators, and site teams for their dedication to advancing therapeutic options for the treatment of pulmonary arterial hypertension.”
Aerovate plans to release full data from the Phase 2b portion of IMPAHCT at a later date, the timing of which is to be determined.
As of June 15, 2024, Aerovate has approximately
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. 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.
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 from Phase 2b to Phase 3. The Phase 2b portion 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 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 U.S. Securities and Exchange Commission (SEC), press releases, public conference calls, webcasts, the investor relations section of the Company website at ir.aerovatetx.com, and the Company’s X (formerly known as Twitter) account @AerovateTx in order to achieve broad, non-exclusionary distribution of information to the public and for complying with its disclosure obligations under Regulation FD.
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,” 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 Phase 2b/Phase 3 IMPAHCT, including the future release of full clinical data and the Company’s plan to halt the Phase 3 portion of the IMPAHCT trial.
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 the identification and activation of clinical sites, patient enrollment, initiation, delivery of drug supply and continuation of our Phase 2b/Phase 3 trial of AV-101 in PAH patients; the impact of public health crises on our business, 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 the United States and foreign countries; as well as those risks and uncertainties set forth more fully under the caption “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 any forward-looking statements, which speak only as of the date they are made. We disclaim any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that 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 its views as of any subsequent date.
Media Contact
Marites Coulter
marites.coulter@vergescientific.com
Investor Contact
IR@Aerovatetx.com
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