Phase 2 Data from “ASTRAEUS” Trial of Mereo BioPharma’s Alvelestat in Alpha-1 Antitrypsin Deficiency-associated Lung Disease Presented at the 2023 American Thoracic Society International Conference - Seite 2

Findings from the biomarker-SGRQ post-hoc analysis showed an association between the extent of reduction in biomarkers and degree of improvement in the SGRQ-Activity domain in alvelestat-treated subjects. By week 12, there was an observed difference in improvement in SGRQ-Activity in biomarker responders compared to non-responders, with a mean improvement of 4.4 in those showing a >0% biomarker decrease and 6.1 in those with >5% biomarker decrease (P=0.05 and p=0.02 respectively), compared to those on alvelestat without biomarker decrease. This association was not observed in the patients who received placebo. These data support a potential association between the effect of alvelestat on the NE pathway and improvement in how a patient feels and functions based on SGRQ score. Data from ASTRAEUS and other research support the hypothesis that longer term treatment is expected to lead to a deepening of the biomarker and associated clinical response.

Alvelestat has been generally safe, with no safety signals of concern observed to date. Adverse events leading to study drug discontinuation (one liver function and one prolonged QTc) resolved on study drug cessation. Three treatment-related SAEs of headache were reported in the alvelestat arms. Headache is a known adverse event associated with alvelestat and is being addressed through dose-escalation during initiation of alvelestat treatment.  

“We are very pleased to have these data presented to the scientific community for the first time at ATS2023. These findings have informed our recent and ongoing discussions with both the FDA and EMA, and validate the proposed design for our planned Phase 3 trial, which we believe will be the first registrational study in AATD-LD to use both a PRO approach and an objective clinical outcome measure as independent primary endpoints,” said Dr. Denise Scots-Knight, CEO of Mereo BioPharma. "We are excited by the potential of alvelestat to become the first-in-class oral neutrophil elastase inhibitor for the treatment of AATD-LD and look forward to continued collaboration with the regulatory authorities, scientific and patient communities as we further refine our development plans ahead of the pivotal study. We look forward to sharing further updates on the progress of the alvelestat program, including the results of the investigator-led Phase 2 ATALANTA study evaluating alvelestat in combination with augmentation therapy, which is expected to read out in Q3 2023.”