Areteia Therapeutics AnnouncesPositive Topline Results from the First Phase III Study of Oral Dexpramipexole in Eosinophilic Asthma

In the EXHALE-4 study, dexpramipexole met the primary endpoint with statistically significant improvement in lung function compared with placeboDexpramipexole demonstrated statistically significant reduction in blood absolute eosinophil count (AEC) compared with placeboFull results will be presented at an upcoming medical meeting; Areteia continues to enroll the EXHALE-2 and EXHALE-3 Phase III exacerbation studies of dexpramipexole in eosinophilic asthma

Areteia Therapeutics, Inc. (“Areteia”) today announced positive results from the Phase III EXHALE-4 efficacy and safety study of dexpramipexole as an add-on oral therapy in participants with eosinophilic asthma.

“More than half of people with asthma have the eosinophilic subtype, yet there remains a profound need for easily administered oral treatment options that can help address their symptoms, which are often severe,” said Ian Pavord, MA, DM, Professor of Respiratory Medicine at the University of Oxford, UK and a member of Areteia’s Scientific Advisory Board. “Initial results from the Phase III EXHALE-4 study are extremely promising, particularly the statistically significant improvement in lung function and the reduction in eosinophils observed with dexpramipexole versus placebo, which show the potential for dexpramipexole to become the first oral treatment approved for eosinophilic asthma.”

“We are pleased to report positive topline results for the EXHALE-4 Phase III study, which demonstrate dexpramipexole’s ability to address the unmet needs of people with eosinophilic asthma by improving their lung function. Dexpramipexole has the potential to be the first oral treatment for this indication,” said Jorge Bartolome, President and Chief Executive Officer of Areteia. “We look forward to presenting the full EXHALE-4 results at an upcoming medical meeting as we also advance the EXHALE-2 and EXHALE-3 studies and work to transform the patient journey in eosinophilic asthma.”

Dexpramipexole significantly improved lung function averaged over Weeks 20 and 24, as measured by the change from baseline in pre-bronchodilator forced expiratory volume (pre-BD FEV₁),in the dexpramipexole 150 mg twice daily (BID) group compared with placebo. Significant improvements in lung function were observed as early as Week 4.

Dexpramipexole 150 mg BID and 75mg BID significantly reduced blood AEC averaged over Weeks 20 and 24, as measured by the change from baseline in geometric mean AEC values compared with placebo.

In this study, dexpramipexole was well tolerated, with a safety profile similar to previous studies.

Full study results will be presented at an upcoming medical meeting. Dexpramipexole is an investigational drug and is not licensed for use in eosinophilic asthma.

About the EXHALE-4 Study (NCT05748600)
EXHALE-4 is a randomized, double-blind, placebo-controlled Phase III study in participants (N=600) aged ≥12 years with inadequately controlled moderate-to-severe asthma and blood AEC ≥300 cells/μL. Participants continued their usual asthma medications and added either dexpramipexole or placebo. Participants were randomly assignedto placebo BID, 150 mg BID, or 75 mg BID dexpramipexole groups in a 5:5:1 ratio respectively. The primary endpoint of the study is pre-BD FEV₁, absolute change from baseline, averaged over Weeks 20 and 24. The primary comparison was conducted on the dexpramipexole 150 mg BID dose and placebo. Dexpramipexole 75mg BID was also included to assess eosinophil lowering effects in blood as well as pharmacokinetic/pharmacodynamic relationship.

About Dexpramipexole
Dexpramipexole, an investigational drug, is an oral small molecule that has been shown to lower eosinophil levels in blood and tissue and is currently in Phase III development for treatment of adults with moderate-to-severe eosinophilic asthma. The proposed mechanism of action is that dexpramipexole inhibits the maturation of eosinophils in the bone marrow, based on evidence from cell cultures and human biopsies, thereby lowering peripheral blood and tissue eosinophil levels. In a Phase II study in participants with moderate-to-severe eosinophilic asthma, treatment with dexpramipexole resulted in a significant, dose-dependent reduction in blood AEC at all doses tested (37.5 mg, 75 mg, or 150 mg BID) compared with placebo. In addition, dose-dependent improvements in lung function were observed (Siddiqui et al. JACI. 2023). Oral dexpramipexole was well tolerated, with adverse events balanced across treatment and placebo groups.

Dexpramipexole is formulated as dexpramipexole dihydrochloride (75 mg and 150 mg dexpramipexole dihydrochloride are equivalent to 56 mg and 112 mg dexpramipexole, respectively). The doses administered in the dexpramipexole treatment groups mentioned throughout this document refer to dexpramipexole dihydrochloride.

About Eosinophilic Asthma
Asthma disrupts the lives of more than a quarter of a billion people worldwide. More than half of people with asthma have eosinophilic asthma which is driven by an elevated number of white blood cells called eosinophils, in the blood, tissue, and sputum. By inhibiting the maturation of eosinophils, we believe that oral administration of the investigational drug dexpramipexole acts to lower eosinophils. Currently approved injectable anti-IL-5/5R biologic therapies provide clinical benefit through eosinophil lowering. The global asthma biologic market is experiencing robust growth, having doubled in the last three years, and is valued today at approximately $10 billion USD. If approved as a first-to-market oral therapy, dexpramipexole could provide an alternative to injectable biologics.

About Areteia
Areteia is a clinical-stage biotechnology company engaged in the development and delivery of a potential first-in-class oral therapy for inflammatory airway diseases, with an initial focus on severe eosinophilic asthma and eosinophilic chronic obstructive pulmonary disease. Areteia has advanced its investigational drug, dexpramipexole, into three separate Phase III clinical trials in eosinophilic asthma, including two 52-week global exacerbation trials (EXHALE-2 and EXHALE-3) and one 24-week lung function trial (EXHALE-4). Areteia was created by Population Health Partners and Knopp Biosciences. A syndicate of leading life sciences and strategic investors has committed to invest up to $425 million USD in Series A financing to establish Areteia and advance dexpramipexole. To learn more, please visit www.areteiatx.com.

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