Clinical-stage biopharmaceutical company Spinogenix Inc on Tuesday reported positive topline results from its completed Phase 2a study for SPG302 in patients with Amyotrophic Lateral Sclerosis (ALS).

SPG302, a once-a-day pill, is being developed as a regenerative treatment with the potential to restore synapses, the key connections between neurons that allow people to think, plan, remember and control movement.

The randomised, double-blind, placebo-controlled Phase 2a study of SPG302 evaluated safety, tolerability, and pharmacodynamic biomarkers in 23 ALS participants. The trial commenced with a double-blind placebo-controlled period of 28 days, after which all participants received active treatment for an additional 140 days. The goal of this first in ALS study was to bridge the gap in the translation from robust preclinical activity to humans in a small study to help with the planning of larger human trials.

Topline results from the trial, presented as a poster at the recent Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) Annual Meeting, revealed that SPG302 was well tolerated with no treatment-related serious adverse events over six months of oral daily dosing of 300mg. Quantitative and objective biomarker evidence helped in dose selection and a drug impact on brain areas affected by ALS. EEG recordings of brain activity revealed improvements in ALS-associated patterns, supporting apparent benefits observed in the rate of functional decline, and 82% of patients treated with SPG302 exhibited a stable or improved rate of decline at the end of treatment, as evaluated with the ALS Functional Rating Scale - Revised (ALSFRS-R). When compared to historical controls from the PRO-ACT database, these patients showed on average a 76% slower rate of decline through six months.

Dr. Stella Sarraf, Spinogenix CEO and founder, said: "We are very encouraged with the positive Phase 2a trial results for SPG302, in our initial proof of concept ALS study, demonstrating the potential of this new regenerative therapy. As we plan our larger registrational directed ALS trial, we also remain committed to making this investigation therapy available to patients who do not qualify for clinical trials through our FDA-cleared Expanded Access Program."