Mereo BioPharma (MREO) reported Thursday that it expects significant clinical and regulatory developments across its pipeline within the next three to four months, as the drug developer advanced talks with the FDA and prepared Phase II and Phase III trials for its lead programs.
Speaking at the 12th Annual Cantor Fitzgerald Global Healthcare Conference, CEO Dr. Denise Scots-Knight said the company’s key milestones are imminent. "It's all happening in the next three to four months," she said. "Hopefully we'll move the story on significantly."
Mereo disclosed that it holds sufficient cash to fund operations through late 2027, supported by upfront and R&D payments from partner Sentynl covering Phase III costs for alvelestat. Zydus Lifesciences is handling commercial manufacturing scale-up for the program, with Mereo retaining European and ex-U.S. rights. The company noted potential milestone payments of up to GBP 475 million for alvelestat and approximately GBP 1 billion in total across its portfolio, along with double-digit U.S. royalties on alvelestat sales.
Alvelestat, targeting alpha-1 antitrypsin deficiency and other neutrophilic-driven diseases, posted 90% inhibition of neutrophil elastase activity in Phase II ASTRAEUS and ATALANTa studies, with improvements observed in desmosine and Aα-val360 biomarkers. A pooled analysis showed roughly a 40% reduction in exacerbations. FDA discussions on Phase III endpoint design—balancing SGRQ, lung density by CT, FEV1 and exacerbation measures—are planned for the fourth quarter.
For setrusumab in osteogenesis imperfecta, Mereo acknowledged missed primary endpoints in Phase III ORBIT and COSMIC trials. The ORBIT study enrolled nearly 160 patients, including 135 pediatric subjects, while COSMIC enrolled more than 60 pediatric patients; over half of placebo patients recorded zero fractures during the trial period. However, post hoc analyses showed marked reductions in vertebral fractures and statistically significant pain improvements on the POSNA-PASI domain. Approximately 200 pediatric patients from both trials remain in an open-label extension, with crossover patients expected to complete 12 months by Q4 2024. FDA guidance on a BLA filing path is expected by year-end.
Vantictumab, developed with partner āshibio for autosomal dominant osteopetrosis type 2, was readied for Phase II initiation. The Wnt pathway antibody acts oppositely to setrusumab, blocking Wnt signaling to reduce bone formation and increase osteoclast activity. Preclinical work was conducted by Indiana University's Econs Laboratory.
Mereo's ADR traded around $0.293 on the day, and the company carried a "WEAK" financial health rating from InvestingPro, with analysts not projecting profitability this year.
CSO Dr. John Lewicki addressed the fracture-endpoint challenge in setrusumab's development. "It's tough to beat zero when you have over half of the patients with zero," he said. "Pain is the key issue—but the agencies are very focused on fractures."
