Mereo BioPharma provides update on pipeline progress, corporate developments

Mereo BioPharma Group (MREO) provided an update on its pipeline programs as well as an update on recent corporate developments. Recent Pipeline Progress: Setrusumab: Enrollment in the Phase 3 portion of the Global Phase 2/3 Orbit study led by Mereo’s partner, Ultragenyx Pharmaceutical, was initiated in mid-2023 and the study is expected to be fully enrolled around the end of the first quarter of 2024. Recent data reported in October 2023, from the 24 patients enrolled in the Phase 2 portion of the Orbit study with at least 6 months of treatment, showed that treatment with setrusumab reduced the annualized fracture rate by 67%. If approved by the U.S. Food and Drug Administration and the European Medicines Agency, setrusumab would be the first FDA and EMA-approved treatment for Osteogenesis Imperfecta. Alvelestat: The Company has continued its interactions with the Division of Pulmonology, Allergy and Critical Care and the Division of Clinical Outcome Assessment of the FDA regarding the use of a Patient Reported Outcome instrument as a potential primary endpoint in its planned Phase 3 study of alvelestat for the treatment of Alpha-1 Antitrypsin Deficiency-associated Lung Disease. Data from both the ATALANTa and ASTRAEUS studies support the use of the SGRQ Total Score as the primary endpoint in the proposed Phase 3 study based on benefits observed in patients with the severe Pi*ZZ phenotype, including those who are in the early stages of the lung disease. In some countries, these patients are currently not eligible for, or not treated with, augmentation therapy. Following additional FDA interactions in the fourth quarter of 2023, subsequent to the DCOA meeting, the Company has aligned on a Phase 3 study design using SGRQ Total Score as the primary endpoint with a functional assessment as a key secondary endpoint which, if the study is successful, is expected to support submissions for full regulatory approval in the U.S. Inclusion of patients with earlier and later stage lung disease progression in the planned registrational study could increase the addressable patient population for alvelestat. The Company remains engaged with multiple potential partners for the development and potential commercialization of alvelestat and expects to provide further details on these efforts in 2024. Leflutrozole: The Company recently entered into an exclusive global license agreement with ReproNovo SA for the development and commercialization of leflutrozole, a non-steroidal aromatase inhibitor. Under the terms of the License Agreement, ReproNovo, a reproductive medicine company, is responsible for all future development and commercialization of leflutrozole.