Curated News
By: NewsRamp Editorial Staff
September 03, 2025
InFlectis BioScience Breakthrough: IFB-088 Shows Efficacy in Axonal CMT-2A
TLDR
- 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.
Impact - Why it Matters
This development matters because Charcot-Marie-Tooth disease affects approximately 1 in 2,500 people worldwide, making it one of the most common inherited neurological disorders. For patients with CMT2A and other rare subtypes, there are currently no approved treatments available, leaving them with progressive nerve damage, muscle weakness, and disability. The expansion of IFB-088's efficacy to axonal forms represents hope for thousands of patients who previously had no therapeutic options. Additionally, the successful industry-academic collaboration model demonstrates how strategic partnerships can accelerate drug development for rare diseases, potentially creating a blueprint for treating other neurodegenerative conditions. The economic implications are significant too—successful development could not only improve patients' quality of life but also reduce long-term healthcare costs associated with progressive disability.
Summary
In a groundbreaking development, InFlectis BioScience has achieved a major breakthrough with its lead compound IFB-088, demonstrating efficacy in treating axonal Charcot-Marie-Tooth disease (CMT-2A) for the first time. The pioneering biotech company collaborated with researchers from Aix Marseille Université to show that IFB-088 reverses pathological cellular phenotypes in human CMT2A iPS cell-derived motor neurons and prevents locomotor impairments in mouse models. This represents a significant expansion of the molecule's potential, as it had previously shown promise only in demyelinating CMT subtypes like CMT1A and CMT1B, but now demonstrates effectiveness against axonal forms through targeting mitochondrial dysfunction and the Integrated Stress Response.
The research partnership exemplifies successful industry-academic collaboration, supported by NeuroSchool and France 2030 funding initiatives that foster young scientific talent. Dr. Zeinab Hamze, selected for a postdoctoral fellowship through this program, joined the InFlectis team to contribute to the translational development of IFB-088. Company executives, including Chief Operating Officer Pierre Miniou, emphasize that these compelling preclinical findings position IFB-088 for Phase 2 clinical development and highlight the urgent need for pharmaceutical partners or investors to finance upcoming clinical trials in Europe and the United States, especially given the challenging economic climate that threatens to delay access to potentially life-changing treatments.
Patient advocacy groups within the CMT community have been strong supporters of InFlectis BioScience, actively encouraging the development of IFB-088 for rare subtypes including CMT1B, CMT1E, and CMT2A—conditions for which no approved treatments currently exist. The compound represents a first-in-class, multi-functional, orally-administered small molecule that selectively inhibits eIF2α dephosphorylation and antagonizes specific NMDA receptors, providing neuroprotection by normalizing calcium homeostasis and reducing oxidative stress. This approach holds promise for designing disease-modifying therapeutics against intractable neurodegenerative diseases beyond CMT, including ALS, where IFB-088 has already demonstrated favorable safety and efficacy in clinical trials.
Source Statement
This curated news summary relied on content disributed by Reportable. Read the original source here, InFlectis BioScience Breakthrough: IFB-088 Shows Efficacy in Axonal CMT-2A
