Immunity Pharma Reports Positive Phase 2a Results for IPL344 in ALS

In a recent announcement, Immunity Pharma, a biopharmaceutical company focusing on neurology and neurodegenerative diseases, reported promising outcomes from its Phase 2a trial involving IPL344 for amyotrophic lateral sclerosis (ALS). The results indicated notable efficacy and tolerability of the drug.

Conducted in Jerusalem, this open-label study lasted up to 36 months with daily intravenous administration of IPL344. The primary measure of efficacy was the ALS Functional Rating Scale-Revised (ALSFRS-R). According to the findings, the participants experienced a 48% reduction in the progression of the disease, as reflected by a change in the ALSFRS-R slope (p=0.028). When adjusted for disease stage and rate, this reduction was 64% (p=0.034). Additionally, patients showed significant weight gain (p=0.02) and had improved median survival rates, showing 29 months compared to 19 months for those on placebo in a comparative ceftriaxone study. Although the trend in respiratory capacity preservation was not statistically significant, it indicated a potential benefit.

This study included neurofilament light (NfL) plasma level evaluations for eight subjects. A reduction in NfL levels was observed in almost all participants, with a mean decrease of 20% post the initial dose-escalating phase, suggesting less neuronal damage.

Dr. Ilana Cohen, Immunity Pharma's VP of R&D, noted the encouraging signs of efficacy in the treatment, emphasizing the potential for results greater than those achieved with existing ALS treatments. The company plans to progress IPL344 to a Phase 3 trial to confirm these findings.

The Phase 1/2a trial, registered under NCT03652805 and NCT03755167, was executed at Hadassah Medical Center and involved nine participants with probable or definitive ALS who showed a progression rate of over 0.55 points monthly on the ALSFRS-R. The statistical analysis incorporated methods to adjust for inter-study and inter-center variability.

IPL344 functions as a biologically active peptide and stimulates therapeutic cell-signaling pathways, notably the Akt pathway, which typically diminishes in neurodegenerative conditions. The peptide was developed in Professor Irun Cohen's Laboratory at the Weizmann Institute of Science in Israel and holds orphan drug designations from both the FDA and EMA, ensuring market exclusivity for a minimum of seven years.

Immunity Pharma, a privately-held company, focuses on developing treatments for neurodegenerative disorders such as ALS and Parkinson's disease through the use of biologically active peptides that foster intra-cellular survival-supporting mechanisms, aimed to slow disease progression.

These initial results advocate for further research and trials to explore IPL344's therapeutic potential fully. The advancement to more extensive studies is anticipated, potentially making significant strides in the fight against ALS.