FDA Grants Mereo Fast Track Designation for Alvelestat for Treatment AATD-associated Lung Disease

The U.S. Food and Drug Administration granted Mereo BioPharma Group Fast Track designation for alvelestat, its experimental therapy to treat a rare, genetic lung disease.

Photo: Denise Scots-Knight, CEO of Mereo

Mereo said it intends to have an End-of-Phase 2 meeting with the FDA to discuss the design of a registration-enabling study for alvelestat as a treatment for AATD-associated lung disease, including the potential opportunity for an accelerated approval pathway, around the end of the year.

Alpha-1 Antitrypsin Deficiency (AATD) results in a deficiency of a protein that protects the lungs against damaging enzymes that the body releases during inflammation. AATD can cause pulmonary emphysema, a progressive, life-threatening lung disease, recurring chest infections and permanent enlargement of parts of the lungs’ airways. There are an estimated 50,000 people in North America and 60,000 in Europe with severe AATD.

Alvelestat is an oral drug that is being developed to treat AATD lung disease. Lung disease is the most common impact of AATD in adults. Alpha-1 antitrypsin deficiency is thought to be the cause of 1% of emphysema, but often the diagnosis is overlooked. Alvelestat acts to inhibit the neutrophil elastase enzyme and Mereo believes that it has the potential to protect AATD patients from further lung damage. Alvelestat is not expected to impact the liver disease, however, other companies are researching in this area.

Fast Track designation is designed to facilitate development and expedite the review of therapies with the potential to treat serious or life-threatening conditions where there is a major unmet medical need. Investigational products that receive Fast Track designation may benefit from early and frequent communication with the FDA and are eligible for rolling submission and review of the marketing application. Additionally, this designation provides potential pathways for accelerated regulatory approval.

In May 2022, Mereo reported positive top-line safety and efficacy data from the ASTRAEUS Phase 2 study of alvelestat in severe AATD-associated emphysema. The company plans to provide a further update on the program on October 31, 2022.

“In the ASTRAEUS study, we demonstrated alvelestat’s ability to inhibit multiple specific biomarkers relevant to the disease pathway based on neutrophil elastase inhibition,” said Denise Scots-Knight, CEO of Mereo.

The investigator-led ATALANTa trial, which is studying alvelestat in a broader range of AATD patient populations, including those on augmentation therapy, is expected to read out in mid-2023. In addition, alvelestat is being studied in Bronchiolitis Obliterans Syndrome (BOS), a rare condition where excessive inflammation causes thickening of the airways, severely limiting lung function.

Author: Rare Daily Staff