Oryzon Genomics has received EMA approval for a Phase 1b trial of iadademstat in sickle cell disease (SCD). This marks the first clinical investigation of iadademstat for a non-malignant hematological indication.
The RESTORE trial will enroll 40 adult SCD patients across multiple Spanish sites. The primary objectives are to evaluate iadademstat’s safety, tolerability, and establish a recommended Phase 2 dose. Secondary objectives include measuring the drug’s ability to induce fetal hemoglobin (HbF).
This move by Oryzon reflects a strategic expansion beyond its current focus on oncology, leveraging iadademstat’s potential in a new therapeutic area with significant unmet need. Given the limited treatment options and the commercial success of previously approved (and subsequently withdrawn) SCD therapies like Oxbryta, the SCD market presents a compelling growth opportunity. Iadademstat’s mechanism, involving LSD1 inhibition and HbF induction, offers a differentiated approach compared to existing and emerging gene therapies, potentially addressing accessibility and cost challenges.
The trial’s design and location suggest a focus on generating early clinical signals and validating the drug’s mechanism in humans, building on preclinical data from baboons. Choosing Spain as the initial trial location may reflect operational advantages and access to specialized SCD patient populations. This approach allows Oryzon to gather preliminary data on efficacy and safety before committing to larger, more complex multinational trials.
The success of this Phase 1b trial will hinge on demonstrating a clear HbF induction response coupled with a manageable safety profile. Oryzon will need to navigate potential challenges such as patient recruitment and retention, particularly given the chronic and complex nature of SCD. Positive results could pave the way for accelerated development, including discussions with regulatory agencies about potential expedited pathways. Looking ahead, Oryzon must also consider long-term manufacturing and commercialization strategies, especially if iadademstat aims to compete with established and emerging gene therapies in the evolving SCD landscape. The trial’s outcome will not only determine the future of iadademstat in SCD but also offer broader insights into the viability of LSD1 inhibition as a therapeutic strategy for non-malignant hematological disorders.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
