Mereo BioPharma Provides Update on Pipeline Progress and Corporate Developments

Phase 3 portion of Orbit Study and Phase 3 Cosmic Study of setrusumab for treatment of Osteogenesis Imperfecta (OI) conducted by partner Ultragenyx expected to complete enrollment around the end of 1Q 2024 and 1H 2024, respectively; Additional Phase 2 data expected in 2024

Alignment on design for single, global Phase 3 study of alvelestat in Alpha-1 Antitrypsin Deficiency-associated Lung Disease (AATD-LD) following additional interactions with U.S. FDA in 2H 2023

Signs global licensing deal for development and commercialization of leflutrozole

Reiterates previous cash runway guidance; current cash expected to fund operations into 2026

LONDON, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) (“Mereo” or the “Company”), a clinical-stage biopharmaceutical company focused on rare diseases, today provided an update on its pipeline programs as well as an update on recent corporate developments.

“2023 was a year of tremendous progress for Mereo. Key milestones in the development of setrusumab for the treatment of OI included positive data from the Phase 2 portion of the Orbit study, and initiation of the Phase 3 portion of the Orbit study and Phase 3 Cosmic study by our partner, Ultragenyx,” said Dr. Denise Scots-Knight, Chief Executive Officer of Mereo. “In addition, we significantly advanced the development of alvelestat, gaining valuable clarity on the regulatory path with both the FDA and EMA. If the proposed Phase 3 study is successful, it could support submissions for full regulatory approvals for this first-in-class therapy addressing a major unmet medical need in Alpha-1 Antitrypsin Deficiency-associated Lung Disease. These developments are expected to further support our ongoing partnering activities for alvelestat. We look forward to providing further updates on both setrusumab and alvelestat during the remainder of the year. With a cash runway into 2026 and several potential important value inflection points on the horizon in 2024, we believe that Mereo remains well positioned for long-term growth and success.”

Recent Pipeline Progress

Setrusumab (UX143)

Enrollment in the Phase 3 portion of the Global Phase 2/3 Orbit study led by Mereo’s partner, Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), was initiated in mid-2023 and the study is expected to be fully enrolled around the end of the first quarter of 2024. The pivotal Phase 3 portion of the Orbit study will include up to 195 patients aged 5 to <26 years randomized 2:1 to receive setrusumab or placebo, with a primary efficacy endpoint of annualized clinical fracture rate excluding fingers, toes, skull and face. Additionally, Cosmic, a Global Phase 3 open-label, randomized, active-controlled study in approximately 65 patients aged 2 to <7 years evaluating setrusumab compared to intravenous bisphosphonates on reduction in total fracture rate, including morphometric vertebral fractures, was initiated in the second half of 2023 and is anticipated to be fully enrolled in the first half of 2024.