Immunity Pharma has reported positive topline results from a Phase IIa trial assessing its lead drug candidate IPL344 in patients with amyotrophic lateral sclerosis (ALS).

IPL344 is an anti-apoptosis stressin-1 peptide that activates the Akt pathway, a key signalling hub responsible for activation and maintenance of cell survival and cell repair mechanisms.

The experimental treatment received orphan drug designation from both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in February 2020.

Immunity Pharma CEO, Eran Ovadia, said that the Phase IIa data gives the company confidence to progress the candidate to Phase III.

The trial demonstrated that once-daily treatment with IPL344 for up to 36 months was well tolerated, with no major drug related serious adverse events (SAE). No participants discontinued treatment due to drug-related AEs.

IPL344 treated patients showed a mean slope of decline in ALSFRS-R of 48% slower disease progression. Adjustment for disease stage and rate-indicating covariates, indicated a 64% slower ALSFRS-R progression.

A statistically significant increase was also observed in the body weight of IPL344 treated patients. Median survival of patients in this study was 29 months vs. 19 months of placebo treated.

Biomarker data for plasma neurofilament light (NfL) levels were obtained for eight subjects. NfL concentrations were reduced during IPL344 treatment in all but one participant. In the six subjects that had blood sampling past the initial dose-escalating phase, plasma NfL concentrations were reduced by a mean of 20%, suggesting decrease of neuronal injury following IPL344 treatment.

The Phase IIa trial (NCT03755167) conducted in Jerusalem was an open-label study involving nine patients with probable or definite ALS. Patients were dosed with once-daily IPL344 treatment administered at home by IV for 36 months.

The listing on ClinicalTrials.gov says the investigators planned to enrol 15 patients on the trial, with the trial currently classified as suspended due to lack of IPL344.

Efficacy outcomes included the rate of progression on ALSFRS-R, slow vital capacity (SVC), weight, and survival compared with historical placebo from the PRO-ACT database and ceftriaxone study.

ALS landscape

ALS is a fatal type of motor neuron disease, characterised by progressive degeneration of nerve cells in the spinal cord and brain. The average survival time with ALS is two to five years, some people live five years, 10 years or even longer.

There are currently seven drugs approved by the FDA to treat ALS and its symptoms; Qalsody, Relyvrio, Radicava, Rilutek, Tiglutik, Exservan and Nuedexta.

GlobalData predicts the ALS market size in the US to be £780m in 2029.

GlobalData is the parent company of the Clinical Trials Arena.