In a recent development, biopharmaceutical company Aerovate Therapeutics has announced the cessation of the Phase 3 portion of its IMPAHCT (Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial) for AV-101, an investigational drug for the treatment of pulmonary arterial hypertension (PAH). The decision was made following the review of data from the Phase 2b segment, which did not indicate significant improvements in several secondary endpoints.

The study's independent study advisory committee, in agreement with Aerovate, recommended halting enrollment for both the Phase 3 trial and the long-term extension study. The unexpected and disappointing results from the Phase 2b portion of IMPAHCT prompted the company to prioritize transparently sharing these findings with investigators, patients, and the PAH community. CEO Tim Noyes expressed gratitude to trial participants, investigators, and site teams for their commitment to advancing therapeutic options for PAH.

Aerovate intends to release the full data from the Phase 2b portion of IMPAHCT at a later date, but the timing has yet to be determined. As of June 15, 2024, the company reports having approximately $100 million in cash, cash equivalents, and short-term investments.

AV-101, a proprietary dry powder inhaled formulation of the antiproliferative drug imatinib, specifically targets cellular hyperproliferation and resistance to apoptosis in the distal pulmonary arteries. The drug aims to improve the lives of patients with PAH by delivering therapeutic benefits directly into the lungs while minimizing systemic adverse effects.

IMPAHCT is a multinational, placebo-controlled Phase 2b/Phase 3 trial that enrolled adult patients with PAH. The Phase 2b segment evaluated different doses of AV-101 over 24 weeks, comparing them to a placebo, to determine the optimal dose based on primary endpoints, including the change in pulmonary vascular resistance (PVR), safety, tolerability, and other clinical measures.

Aerovate Therapeutics is a clinical-stage biopharmaceutical company focused on addressing the needs of patients with rare cardiopulmonary diseases. Its primary focus has been on advancing AV-101 as a treatment for PAH.

As of now, there is no further information regarding the continuation of the AV-101 development pipeline. The company plans to engage closely with the IMPAHCT study advisory committee and the PAH community to thoroughly evaluate the data and its implications before making any additional decisions.

While forward-looking statements have not been provided in this news article, Aerovate reminds investors and the public to exercise caution when interpreting any previous statements, as they are subject to risks, uncertainties, and important factors that may cause actual results to differ from expectations. These factors include the regulatory landscape, public health crises, and the outcome of ongoing clinical trials.

Aerovate will continue to disclose material information through various channels, including filings with the U.S. Securities and Exchange Commission (SEC) and press releases, to ensure broad distribution and compliance with disclosure obligations.

Stay tuned for further updates on Aerovate's future plans for AV-101 as more information becomes available.