Mereo BioPharma announced that alvelestat has been granted Fast Track designation by the US Food and Drug Administration (FDA) for the treatment of alpha-1 antitrypsin deficiency (AATD)-associated lung disease.
The designation, announced in a news release, includes the possibility of an accelerated approval pathway sometime around the end of 2022.
“AATD is a devastating inherited condition which severely affects patients’ quality of life. 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 Dr. Denise Scots-Knight, chief executive officer of Mereo.
Alvelestat is an investigational oral neutrophil elastase inhibitor that was already granted Orphan Drug designation by the FDA. The treatment is currently being assessed in a phase 2 proof-of-concept study called ASTRAEUS, as well as in ATALANTa, an investigator-led study on patients with severe AATD.
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The ASTRAEUS study has evaluated 98 patients who were given either a high or low dose of alvelestat over a 12-week period. At the high dose, alvelestat administration led to improvements in all three primary biomarker endpoints: blood neutrophil elastase activity, Aα-val360, and the elastin breakdown product, desmosine. These results were reported in May 2022 and a further update is expected on October 31, 2022.
During the remainder of 2022, Mereo will analyze the data on secondary and exploratory endpoints and then work with the FDA and European regulators on the design of a pivotal trial of alvelestat. The ATALANTa trial is currently evaluating alvelestat in a variety of patient populations, including those on augmentation therapy and those with other genotypes. The trial is expected to be completed in 2023.
AATD is a rare genetic condition that leads to low or no levels of AAT, which is a protein that protects the lungs from certain damaging enzymes that are released as a result of inflammation. The lack of this protein can lead to life-threatening lung disease.
Mereo BioPharma receives FDA Fast Track designation for Alvelestat for treatment of alpha-1 antitrypsin deficiency (AATD)-associated lung disease. News release. Mereo BioPharma; October 17, 2022.