Foresee Pharmaceuticals has announced a strategic pivot in its clinical development program for mirivadelgat (FP-045), an ALDH2 activator, shifting primary focus from Fanconi anemia to pulmonary hypertension associated with interstitial lung disease (PH-ILD).
The decision comes after persistent challenges in patient recruitment for the Phase 2 Fanconi anemia trial, which began following FDA clearance in September 2020. Despite regulatory accommodations, including relaxed inclusion criteria and reduced enrollment requirements, the trial failed to recruit its first eligible patient, largely due to the ultra-rare nature of the disease and complications from the COVID-19 pandemic.
Strategic Redirection to PH-ILD
The company's development efforts will now concentrate on advancing mirivadelgat's Phase 2 clinical trial for PH-ILD. This program has already gained momentum, with positive feedback from both the U.S. FDA and Taiwan FDA in 2024. Clinical trials have commenced at several hospitals in Taiwan, marking a significant step forward in the drug's development pathway.
Evolution in Fanconi Anemia Research Approach
While stepping back from the original Phase 2 trial, Foresee Pharmaceuticals maintains its commitment to the Fanconi anemia community through a modified approach. The company is exploring an Investigator Sponsor Study (ISS) with a U.S. research site to evaluate mirivadelgat's safety profile and potential efficacy as a cancer prevention agent in adult Fanconi anemia patients.
This shift aligns with broader trends in the scientific community, where research focus has increasingly turned toward cancer prevention and early detection, driven by advances in precision medicine and novel therapeutic approaches.
Development Strategy and Resource Optimization
The strategic reallocation of resources reflects Foresee Pharmaceuticals' adaptive approach to drug development, balancing scientific opportunity with practical considerations. The company's decision to prioritize PH-ILD development while maintaining a presence in Fanconi anemia research through the ISS demonstrates a commitment to both efficient resource utilization and continued innovation in areas of high unmet medical need.
