Aptorum Group Limited (Nasdaq: APM, Euronext Paris: APM) (“Aptorum Group” or “Aptorum”), a clinical-stage biopharmaceutical company, announces positive progress in respect of: (i) its ongoing Phase I clinical trial for ALS-4 (a first in-class anti-virulence approach based small molecule targeting Staphylococcus aureus, including, but not limited to Methicillin Resistant Staphylococcus Aureus (“MRSA”)), under which two initial cohorts of the single dose ascending dose (SAD) portion of the trial in healthy male and female adult subjects have been completed with no serious adverse events observed (with in total 6 cohorts for SAD and 3 cohorts for multiple ascending dose (MAD) have been planned); and (ii) its ongoing Pre-IND preparation for SACT-1, a repurposed small molecule targeting neuroblastoma, under which the Pre-IND meeting with the US FDA has been completed.
ALS-4’s first-in-human Phase I trial is a randomized, double-blinded, placebo-controlled, single and multiple ascending dose study designed to evaluate safety, tolerability, and pharmacokinetics of orally administered ALS-4 in healthy male and female adult volunteers. Dosing and safety reviews of Cohort A (25mg) and Cohort B (50mg) have been completed and eight subjects (6 received ALS-4 and 2 received placebo) were dosed in each cohort. We are pleased to announce that no human subjects were dropped out of the studies and there were no Serious Adverse Events (SAE) observed. In addition, no relevant clinical changes in respect of vital signs, ECG, clinical laboratory test results and physical examinations were observed compared to the relevant baseline. On this basis, the remaining ALS-4 Phase I study will continue to progress and as of this date, Cohort C (100mg) studies have been initiated.
SACT-1, a repurposed drug candidate for the treatment of neuroblastoma, has completed its Pre-IND meeting with the US FDA. With the guidance provided by the US FDA, SACT-1 is on track to open an IND to commence clinical studies in quarter 3 of 2021.
Full PR available here