Aerovate Therapeutics Announces 24-Week Topline Results from the Phase 2b Portion of IMPAHCT Evaluating AV-101 for the Treatment of Pulmonary Arterial Hypertension AV-101 did not meet the primary endpoint of change in pu

Aerovate Therapeutics

Announces 24-Week Topline Results from the Phase 2b Portion of

IMPAHCT Evaluating AV-101 for the Treatment of Pulmonary

Arterial Hypertension

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 -- 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).

Endpoint - ITT analysis of PVR (dynes*sec/cm^5)

Secondary Endpoint - ITT analysis of 6MWD (meters)

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 $100 million of

cash, cash equivalents and short-term investments.

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.

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.

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.

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