BI-1584862: An Investigational Oral Small Molecule for Geographic Atrophy Secondary to AMD - Development Status and Clinical Program Overview

Executive Summary

BI-1584862 is an investigational small molecule drug candidate, classified as a new molecular entity, under development by Boehringer Ingelheim for the treatment of Geographic Atrophy (GA) secondary to age-related macular degeneration (AMD). Notably, it is being developed as an oral formulation, administered as tablets twice daily in its current Phase II trial. This contrasts with existing approved GA therapies, which require intravitreal injections. The specific biological target and mechanism of action for BI-1584862 remain undisclosed in the available information. Following the successful completion of several Phase I studies in healthy volunteers that assessed safety, tolerability, pharmacokinetics, food effects, and potential drug interactions, BI-1584862 has advanced into a Phase II clinical trial (NCT06769048, JADE). This ongoing, randomized, double-masked, placebo-controlled, dose-finding study aims to evaluate the efficacy and safety of multiple doses of BI-1584862 over one year in approximately 138 adults with non-foveal GA exhibiting signs of active progression. The primary objectives are to assess the drug's impact on GA progression and visual parameters and to identify an optimal dose for potential future development. While no specific regulatory designations (e.g., FDA Fast Track) have been reported, the program aligns with Boehringer Ingelheim's strategic focus on retinal diseases. The unknown mechanism of action represents a significant information gap, making the outcomes of the JADE trial critical for understanding the drug's potential therapeutic value and differentiating features, particularly its novel oral route of administration, within the evolving GA treatment landscape.

Introduction to BI-1584862