InFlectis BioScience and Aix Marseille Université Expand IFB-088's Therapeutic Potential to Axonal Charcot-Marie-Tooth Disease
InFlectis BioScience and Aix Marseille Université have demonstrated that lead compound IFB-088 shows efficacy in animal models of axonal Charcot-Marie-Tooth disease type 2A, expanding its potential to address multiple CMT subtypes with no approved treatments.
InFlectis BioScience, in collaboration with researchers from Aix Marseille Université, has achieved a significant breakthrough in the development of IFB-088, demonstrating its efficacy in an animal model of axonal Charcot-Marie-Tooth disease type 2A (CMT-2A). This marks the first time the compound has shown promise for axonal forms of CMT, having previously established proof of concept in demyelinating subtypes including CMT1A and CMT1B.
The research team showed that IFB-088 reverses the pathological cellular phenotype in two different types of human CMT2A iPS cell-derived motor neurons bearing MFN2Arg94Gln and MFN2Arg707Trp mutations. Crucially, the compound also prevents locomotor impairments in a mouse model of axonal CMT2A bearing MFN2Arg94Gln. These findings highlight the molecule's expanded potential to address multiple CMT subtypes by targeting integrated stress response and mitochondrial dysfunction.
Pierre Miniou, Chief Operating Officer of InFlectis, emphasized the importance of these results, stating that with IFB-088 poised to enter Phase 2 clinical development for CMT, these compelling preclinical findings alongside previously published CMT1A and CMT1B data should attract pharmaceutical partners or investors to finance upcoming clinical trials in Europe and the United States. He noted that in today's challenging economic climate, such partnerships are essential to ensure this promising therapeutic candidate reaches patients who need it most.
The research was supported by NeuroSchool, amU's Graduate School in Neuroscience, through a joint innovation program to foster young scientific talent. The university fully funded a one-year postdoctoral fellowship for company-based neuroscience research, backed by the France 2030 national investment plan and the Amidex university foundation. This initiative promotes industry-academic collaboration and supports the long-term employability of early-career neuroscientists.
Dr. Zeinab Hamze was selected for this postdoctoral research position and joined the InFlectis team through the MMG/U1251 Inserm unit at the Faculty of Medical and Paramedical Science in Marseille, contributing to further translational development of IFB-088. She described the project as bridging the frontier of neuroscience and drug development, noting the motivation of working on translational efforts with direct patient relevance.
Patient advocacy groups in the CMT community have been strong supporters of InFlectis and are actively encouraging the development of IFB-088, particularly for rare subtypes such as CMT1B, CMT1E, and CMT2A—for which no approved treatments currently exist. The expansion of IFB-088's therapeutic reach to axonal forms represents a significant advancement in addressing the unmet medical needs of CMT patients across multiple disease subtypes.