Nantes, France – September 2nd, 2025 – InFlectis BioScience, a pioneering biotech company advancing treatments that modulate the Integrated Stress Response (ISR), announces a major milestone in the development of its lead compound IFB-088, now demonstrating efficacy in an animal model of the most prevalent form of axonal Charcot-Marie-Tooth disease, CMT-2A.

Until now, InFlectis had established proof of concepts for IFB-088 in animal models of demyelinating CMTs, including CMT1A and CMT1B. For the first time, researchers of Aix Marseille Université (amU), in collaboration with InFlectis, have shown that IFB-088 is reverting the pathological cellular phenotype of two different types of human CMT2A iPS cell-derived motor neurons bearing MFN2Arg94Gln and MFN2Arg707Trp mutations. Importantly, IFB-088 is also preventing locomotor impairments in a mouse model of axonal CMT2A bearing MFN2Arg94Gln. These new findings underscore the molecule expanded potential to address multiple CMT subtypes by targeting ISR and mitochondrial dysfunction.

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. 

Pierre Miniou, Chief Operating Officer of InFlectis, declared: “We are greatly encouraged by the promising results observed with IFB-088 and deeply value the collaboration with the MMG/U1251 team at Aix Marseille Université. With IFB-088 now poised to enter Phase 2 clinical development for CMT, we sincerely hope these compelling preclinical findings—alongside previously published CMT1A and CMT1B preclinical data—will attract the interest of a pharmaceutical partner or investor to finance an upcoming clinical trial in Europe and the United States. In today’s challenging economic climate, such partnerships are not just beneficial — they are essential. Without them, this promising therapeutic candidate may never reach the patients who need it most”.

This landmark research was supported by NeuroSchool, amU’s Graduate School in Neuroscience. As part of a joint innovation program to foster young scientific talent, amU has fully funded a one-year postdoctoral fellowship for company-based neuroscience research. 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.

Dr. Zeinab Hamze, PhD, 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.

"This project bridges the frontier of neuroscience and drug development. Being part of a translational effort with direct patient relevance is incredibly motivating, and I'm proud to contribute to expanding the therapeutic reach of IFB-088 to axonal forms of CMT” said Dr. Zeinab Hamze, Postdoctoral Researcher at amU and InFlectis BioScience.

Dr. Nathalie Bernard-Marissal, MMG/U1251 Inserm unit Researcher & Program Coordinator, declared: "Our partnership with InFlectis exemplifies the mission of amU and NeuroSchool: to empower young researchers while advancing innovative solutions for neurological disorders. Supporting partnerships within industry accelerates both knowledge transfer and therapeutic impact."

About amU and the NeuroSchool Initiative

Aix Marseille Université hosts 18 interdisciplinary institutes that aim to strengthen the research-training link, as well as interaction with the economic and cultural world, interdisciplinarity and internationalization around strategic fields of research. Their core unifying role: training a new generation of academics to meet the scientific and societal challenges of our time. The NeuroMarseille Institute federates basic and clinical research in Neuroscience at amU, the second largest national site for brain research. 

Within the NeuroMarseille Institute, NeuroSchool*, graduate school in neuroscience, prepares the next generation of neuroscientists through advanced training for BSc, MSc and PhD students, tailored to their professional goals. Thanks to funding from the French National Agency for Research (ANR, France 2030 program) and the Amidex university foundation, NeuroSchool offers each year to fully fund a one-year post-doctoral salary for a research project carried out in a company by one of its alumni. The aim of this post-doctoral year in a company is to encourage young PhDs to enter the world of business. With this program, 5 postdoc researchers have been funded to work in a biotech company.

(*This project has received funding from the French government under the “France 2030” investment plan managed by the French National Research Agency (ref. ANR-16-CONV000X / ANR-17-EURE-0029) and from Excellence Initiative of Aix Marseille University - AMIDEX (AMX-19-IET-004).)

About InFlectis BioScienceInFlectis BioScience is a private, clinical-stage biotechnology company pioneering a new class of small molecule therapies that target protein misfolding and oxidative stress. By restoring proteostasis and enhancing cellular resilience, the company aims to treat rare neurodegenerative diseases with high unmet medical need. In February 2025, InFlectis successfully completed an exploratory Phase 2 clinical trial of its lead compound, IFB-088, in 51 patients with bulbar-onset Amyotrophic Lateral Sclerosis (ALS). The study demonstrated a favorable safety profile, clinical benefits across validated endpoints, engagement of the drug’s biological pathways, and improvements in key biomarkers—validating the therapeutic hypothesis. 

About IFB-088 IFB-088 (also named sephin1 or icerguastat) is a first-in-class, multi-functional, brain-penetrant, orally-administered small molecule that selectively inhibits the dephosphorylation of eIF2α. By doing so, IFB-088 amplifies the integrated stress response (ISR), acting as a formidable shield against various cellular stresses, including endoplasmic reticulum (ER) stress which is a hallmark of neurodegeneration. IFB-088 also selectively antagonizes NMDA receptors containing the GluN2B subunit, which are involved in glutamate excitotoxicity that triggers calcium influx, mitochondrial dysfunction, and reactive oxygen species (ROS) production, and ultimately contributes to neurodegeneration. IFB-088 also reduces mitochondrial ROS production in an NMDAR-independent manner. Hence, IFB-088 normalizes dysregulated calcium homeostasis and oxidative stress to provide neuroprotection in different diseases context. This approach holds promise for designing disease-modifying therapeutics to fight intractable diseases such as ALS and CMT.

This contant was orignally distributed by Reportable. Blockchain Registration, Verification & Enhancement provided by NewsRamp™. The source URL for this press release is Aix Marseille Université (amU) and InFlectis BioScience Expand Therapeutic Promise of IFB-088 to Axonal Forms of Charcot-Marie-Tooth Disease.