InFlectis BioScience and Aix Marseille Université Expand IFB-088 Efficacy to Axonal Charcot-Marie-Tooth Disease
TL;DR
InFlectis BioScience's IFB-088 demonstrates efficacy in CMT-2A, positioning it as a potential first-in-class treatment for multiple rare neurodegenerative disease subtypes with no current therapies.
IFB-088 modulates the Integrated Stress Response and mitochondrial function, reversing pathological phenotypes in human iPS cell-derived motor neurons and preventing locomotor impairments in mouse models of CMT-2A.
This breakthrough offers hope for patients with rare CMT subtypes lacking treatments, potentially improving quality of life and advancing care for neurodegenerative diseases worldwide.
A French biotech collaboration reveals a compound that protects neurons by targeting cellular stress pathways, bridging academic research with patient-focused drug development for rare diseases.
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InFlectis BioScience, a clinical-stage biotechnology company developing treatments targeting the Integrated Stress Response, has achieved a significant breakthrough with its lead compound IFB-088, now showing efficacy in animal models of axonal Charcot-Marie-Tooth disease type 2A (CMT-2A). This development marks the first time IFB-088 has demonstrated therapeutic potential beyond demyelinating CMT forms, specifically addressing the most prevalent axonal variant of this inherited neurological disorder.
Researchers from Aix Marseille Université, collaborating with InFlectis BioScience, have demonstrated that IFB-088 reverses pathological cellular phenotypes in human CMT2A induced pluripotent stem cell-derived motor neurons carrying MFN2Arg94Gln and MFN2Arg707Trp mutations. Crucially, the compound also prevents locomotor impairments in mouse models of axonal CMT2A with MFN2Arg94Gln mutations. These findings, supported by NeuroSchool and funded through the France 2030 national investment plan, highlight the molecule's expanded potential to address multiple CMT subtypes by targeting both integrated stress response and mitochondrial dysfunction pathways.
The research collaboration represents a significant advancement for the CMT community, particularly for patients with rare subtypes such as CMT1B, CMT1E, and CMT2A, for which no approved treatments currently exist. Patient advocacy groups have been strong supporters of InFlectis and actively encourage the development of IFB-088 for these underserved patient populations.
Pierre Miniou, Chief Operating Officer of InFlectis, emphasized the importance of these findings, stating that with IFB-088 poised to enter Phase 2 clinical development for CMT, these compelling preclinical results alongside previously published CMT1A and CMT1B data could attract pharmaceutical partners or investors to finance upcoming clinical trials in Europe and the United States. The company's approach to restoring proteostasis and enhancing cellular resilience addresses fundamental mechanisms in neurodegenerative diseases with high unmet medical need.
The research was further strengthened by the involvement of Dr. Zeinab Hamze, who joined the InFlectis team through a postdoctoral fellowship fully funded by Aix Marseille Université. This initiative, backed by the France 2030 national investment plan and the Amidex university foundation, promotes industry-academic collaboration and supports the long-term employability of early-career neuroscientists while accelerating therapeutic development.
IFB-088 represents a first-in-class, multi-functional, brain-penetrant, orally-administered small molecule that selectively inhibits the dephosphorylation of eIF2α, amplifying the integrated stress response while also antagonizing NMDA receptors containing the GluN2B subunit. This dual mechanism addresses both endoplasmic reticulum stress and glutamate excitotoxicity, normalizing dysregulated calcium homeostasis and oxidative stress to provide neuroprotection across different disease contexts.
The expansion of IFB-088's therapeutic promise to axonal CMT forms represents a critical step toward addressing the complete spectrum of Charcot-Marie-Tooth disease, potentially offering the first disease-modifying treatment for patients suffering from these debilitating neurological conditions. The successful translation of these preclinical findings could significantly impact the treatment landscape for rare neurodegenerative diseases worldwide.
Curated from Reportable
