Inflectis Bioscience's oral therapy IFB-088 (icerguastat) has demonstrated promising results in a Phase 2 clinical trial for bulbar-onset amyotrophic lateral sclerosis (ALS), meeting its primary safety endpoint while showing encouraging signs of efficacy in slowing disease progression.
The P288ALS TRIAL (NCT05508074) evaluated 51 patients with bulbar-onset ALS, a variant that initially affects muscles controlling speech and swallowing. Participants received either IFB-088 at 25 mg twice daily or placebo, alongside standard riluzole therapy, with 41 patients completing at least six months of treatment.
Safety and Efficacy Outcomes
The drug demonstrated a favorable safety profile, meeting its primary endpoint with no significant safety concerns reported. While efficacy was not the primary focus, the results revealed notable benefits in disease progression measures.
In the per protocol population of 40 patients who adhered to the treatment regimen, IFB-088 showed statistically significant advantages. Treated patients experienced a slower decline rate of 0.95 points per month on the ALS Functional Rating Scale Revised (ALSFRS-R), compared to 1.42 points in the placebo group. Additional measurements of lung function and ALS staging also suggested reduced disease progression.
"We are very encouraged by the results we observed with IFB-088," stated Pierre Miniou, CEO of Inflectis Bioscience. "These compelling findings position IFB-088 for forthcoming pivotal studies and pave the way for new treatment possibilities for ALS."
Mechanism of Action and Biomarker Validation
IFB-088's therapeutic approach targets multiple pathways implicated in ALS pathology. The drug activates the integrated stress response while reducing oxidative stress and excitotoxicity. Biomarker analysis from the trial confirmed these mechanisms, showing successful activation of stress response pathways and reduction in inflammation and nerve damage.
Dr. Giuseppe Lauria, principal investigator and professor of neurology at the University of Milan, emphasized the significance of these findings: "The positive results, supported by both clinical outcomes and biomarker validation, strengthen the therapeutic potential of targeting the integrated stress response and oxidative stress pathways in bulbar onset ALS."
Future Development Plans
Dr. Kuldip Dave, senior vice president for research at the ALS Association, highlighted the urgent need for new treatments: "New treatments are urgently needed to make ALS a livable disease, so we are thrilled to see IFB-088 hit its primary endpoint of safety and tolerability."
Inflectis Bioscience is now seeking global partners to advance IFB-088's development and registration for ALS treatment, with potential applications in other neurological conditions. The company's Chief Medical Officer, Dr. Anne Visbecq, confirmed that these results strongly support moving forward with pivotal studies.
