Mereo BioPharma provides the latest information on Investing.com’s leading clinical programs

The Orbit phase 3 study of cetrusumab in osteogenesis imperfecta continues with a planned second interim analysis, expected in mid-2025.

Alvelestat, for lung disease associated with alpha-1 antitrypsin deficiency, receives positive opinion from EMA on European application for orphan drug designation; The European Commission is expected to make a final decision in the first quarter of 2025

LONDON, Jan. 12, 2025 (GLOBE NEWSWIRE) — Mereo BioPharma Group plc (NASDAQ: MREO ) (Mereo or the Company), a clinical-stage biopharmaceutical company focused on rare diseases, today announced an update on its lead clinical program, setrusumab , a monoclonal antibody in Phase 3 clinical development for the treatment of Osteogenesis Imperfecta (OI) through a partnership with Ultragenyx Pharmaceutical (NASDAQ:), Inc. (Ultragenyx) and alvelestat, an oral neutrophil elastase inhibitor being studied for the treatment of lung disease associated with alpha-1 antitrypsin deficiency (AATD-LD). The company also reiterated its previous cash guidance that its current cash and cash equivalents are expected to fund operations in 2027, through multiple key turning points.

Based on very promising data from completed studies of cetrusumab in OI, including part of the phase 2 Orbit study, we remain confident in the potential of cetrusumab to become the standard of care in OI. We look forward to a second interim analysis expected in the middle of the year as we continue with launch preparation activities in key European markets, said Dr. Denise Scots-Knight, Chief Executive Officer of Mereo BioPharma. In addition, the EU Orphan Designation, which follows the granting of both Ophan Medicine and Fast Track Designation by the US FDA, is another important milestone in our ongoing partnership process and our efforts to bring alvelestat to patients worldwide, including patients in earlier stages who are currently ineligible for augmentation therapy in many countries. With our cash runway in 2027, we remain in a strong position to deliver on our key milestones through 2025.

Setrusumab (UX143)

As announced by the company’s partner, Ultragenyx, the Phase 3 Orbit Study of setrusumab in OI continues to dose patients and is progressing toward a planned second interim analysis expected in mid-2025, with a potential final analysis in the fourth quarter of 2025. Additionally, treatment continues in Cosmic, an open-label phase 3 study evaluating cetrusumab versus intravenous bisphosphonate therapy in patients aged 2 to <7 years. Data from the space study will be evaluated in parallel with Orbit's interim and final analyses.

Alvelestat (MPH-966)

The Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) issued a positive opinion on the Company’s request for orphan drug designation for alvelestat. The COMP recommendation has been submitted to the European Commission, which is expected to issue a final decision on Orphan Drug Designation in the first quarter of 2025. Alvelestat previously received Orphan Drug Designation and Fast Track Designation from the US FDA in 2021 and 2022, respectively.

The European Orphan Designation is awarded to therapeutic candidates targeting the treatment, prevention or diagnosis of life-threatening or chronically debilitating diseases with a prevalence of less than 5 per 10,000 people in the European Union that provide a significant advantage over available therapies or for which there are no approved therapies . Therapeutics that receive EU Orphan Designation are eligible for ten years of marketing exclusivity upon approval, as well as fee reductions for various centralized activities including the application for marketing authorization, inspections and protocol assistance. Individual EU Member States also provide specific incentives to support the development, review and availability of medicines for rare diseases at the time of HTA evaluation and price and reimbursement negotiations.

About Mereo BioPharma

Mereo BioPharma is a biopharmaceutical company focused on the development of innovative therapy for rare diseases. The company has two rare disease drug candidates: setrusumab for the treatment of osteogenesis imperfecta (OI); and alvelestat, primarily for the treatment of severe lung disease associated with alpha-1 antitrypsin deficiency (AATD-LD). The company’s partner, Ultragenyx Pharmaceutical (TADAWUL:), Inc., completed enrollment in the Phase 3 portion of a pivotal Phase 2/3 study in pediatrics and young adults (5 to 25 years) for cetrusumab in OI and in a Phase 3 study in pediatric patients ( 2 to <7 years) in the first half of 2024. The partnership with Ultragenyx includes potential additional payments of up to $245 million and royalties to Mere for commercial sales of territories of Ultragenyx. Mereo has retained commercial rights in the EU and UK and will pay Ultragenyx royalties for commercial sales in those territories. Setrusumab received orphan designation for osteogenesis imperfecta from the EMA and FDA, PRIME designation from the EMA, and Breakthrough Therapy designation and rare pediatric disease designation from the FDA. Alvelestat has received US Orphan Drug Designation for the treatment of AATD and Fast Track designation from the FDA. Following the results of ASTRAEUS and ATALANT in AATD-lung disease, the Company has aligned with the FDA and EMA on the primary endpoints for a pivotal Phase 3 study that, if successful, could lead to full approval in both the US and Europe . In addition to the rare disease program, Mereo has two oncology product candidates, etigilimab, an anti-TIGIT; and navicixizumab for the potential treatment of late-line ovarian cancer. Navicixizumab has partnered with Feng Biosciences, Inc. in a global licensing agreement that includes payments and royalties. Mereo also entered into an exclusive global license agreement with ReproNovo SA, a reproductive medicine company, to develop and commercialize leflutrozole, a non-steroidal aromatase inhibitor.

Forward-looking statements

This press release contains forward-looking statements that involve significant risks and uncertainties. All statements other than statements of historical fact contained herein are forward-looking statements within the meaning of Section 27A of the United States Securities Act of 1933, as amended, and Section 21E of the United States Securities Exchange Act of 1934, as amended. changes and additions. Forward-looking statements typically relate to future events and expected revenues, earnings, cash flows or other aspects of our business or operating results. Forward-looking statements are often identified by the words believe, expect, anticipate, plan, intend, predict, should, would, could, could, estimate, predict and similar expressions, including negative expressions. However, the lack of these words does not mean that the statements are not forward-looking. These forward-looking statements are based on the Company’s current expectations, beliefs and assumptions regarding future developments and business conditions and their potential impact on the Company. Although management believes that these forward-looking statements are reasonable as and when made, there can be no assurance that future developments affecting the Company will be as they anticipate.

All of the Company’s forward-looking statements involve known and unknown risks and uncertainties, some of which are significant or beyond control, and assumptions that could cause actual results to differ materially from the Company’s historical experience and its current expectations or projections. Such risks and uncertainties include, among others, uncertainties inherent in the clinical development process; the Company’s reliance on third parties to conduct and finance clinical trials; the Company’s dependence on patient enrollment in its clinical trials; and the Company’s dependence on its key executives. You should carefully consider the above factors and other risks and uncertainties affecting the Company’s business, including those described in the Risk Factors section of its Annual Report on Form 10-K, as well as the discussions of potential risks, uncertainties and other important factors in the Company’s subsequent filings Securities and Exchange Commission. The Company cautions you not to place undue reliance on any forward-looking statements that speak only as of the date hereof. The Company undertakes no obligation to publicly update or revise any of our forward-looking statements after the date they are made, whether as a result of new information, future events or otherwise, except to the extent required by law.

Source: Mereo BioPharma Group plc