Nantes, France – July 2nd, 2025 – InFlectis BioScience, a clinical-stage biotechnology company focused on developing therapies for neurodegenerative diseases, is proud to announce the acceptance of its latest research article titled “Sephin1 reduces TDP-43 cytoplasmic mislocalization and improves motor neuron survival in ALS models” for publication in the peer-reviewed journal Life Science Alliance, effective July 1, 2025.
This study, conducted in collaboration with leading academic and clinical research institutions across Europe, provides compelling preclinical evidence that IFB-088 (also known as Sephin1 or Icerguastat), a modulator of the Unfolded Protein Response (UPR), significantly reduces the cytoplasmic mislocalization of TDP-43 - a pathological hallmark in 97% of amyotrophic lateral sclerosis (ALS) cases – and decreases abnormal RNA splicing associated with TDP-43 nuclear loss of function. The research demonstrates that IFB-088 enhances motor neuron survival and function in multiple ALS models, including SOD1G93A mice and TDP-43 transgenic zebrafish.
“Our findings highlight the potential of IFB-088 to address TDP-43 proteinopathy, a major unmet need in ALS therapy,” said Dr. Emmanuelle Abgueguen, lead author and Senior Principal Scientist at InFlectis BioScience. “Unlike antisense oligonucleotide approaches that target the mis-splicing of a single gene, IFB-088 acts upstream by modulating the cellular stress response, thereby reducing abnormal RNA splicing across multiple targets. This broader mechanism of action could offer wider therapeutic benefits to ALS patients”.
The study shows that IFB-088:
- Reduces mitochondrial oxidative stress and TDP-43 cytoplasmic accumulation in motor neurons
- Improves motor neuron survival in both in vitro and in vivo ALS models
- Decreases abnormal RNA splicing associated with TDP-43 nuclear loss of function
- Enhances motor function and survival in TDP-43 transgenic zebrafish
These results build on the company’s ongoing clinical efforts, including a recently completed Phase II trial of IFB-088 in bulbar-onset ALS patients (NCT05508074).
“This publication represents a major milestone for InFlectis and further strengthens our commitment to advancing disease-modifying therapies for ALS. The presented data strongly support the continued clinical development of IFB-088 and its potential application in ALS patients”, said Dr. Pierre Miniou, co-author and Chief Operating Officer at InFlectis BioScience. “We sincerely hope that these compelling preclinical results will help us secure the support of a pharmaceutical partner or investor to co-finance our upcoming Phase 2B clinical trial in Europe and the United States. In the current challenging economic environment, such partnerships are crucial—without them, this promising drug candidate may never reach the patients who need it most.”
The full article will be available online in Life Science Alliance and via the journal’s website.
