Research & Development
Aptorum Group Completes Phase I Clinical Trials for ALS-4 and SACT-1, Small Molecule Drugs Targeted for Infections Caused by Staphylococcus Aureus and Neuroblastoma
11 January 2022 - - UK-based biopharmaceutical company Aptorum Group Ltd. (NASDAQ: APM) (Euronext Paris: APM) has completed the Phase I clinical trial for ALS-4 (a first in-class anti-virulence based small molecule drug targeting infections caused by Staphylococcus aureus, including, but not limited to Methicillin Resistant Staphylococcus Aureus) and the Phase I clinical trial for assessing relative bioavailability and food effect of SACT-1 (a repurposed small molecule drug targeting Neuroblastoma and potentially other cancer types), the company said.

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 clinical evaluations of the Single Ascending Dose studies and Multiple Ascending Dose studies have now been completed for a total of 72 healthy subjects and Aptorum is pleased to announce that no subjects were dropped from the studies.

There were no Serious Adverse Events observed and no relevant clinical changes in respect of vital signs; ECG, clinical laboratory test results and physical examinations were observed compared to the relevant baseline in both SAD (25-200mg) and MAD (50-100mg).

The safety data of the last SAD cohort (300mg) and MAD cohort (200mg twice a day for 14 days) are pending.

With the encouraging safety data in our Phase 1 trial, we are on track to submit an IND application to the US FDA this year seeking to initiate a Phase 2 clinical study to assess the efficacy of ALS-4 in patients.

SACT-1's first in-human clinical trial is a Phase 1, Open-label Randomized, Single Cross Over Bioavailability and Food Effect Study of SACT-1 in healthy adult volunteers. Aptorum is pleased to announce the successful completion of the trial, during which no SAE were observed.

With the encouraging data in our trial so far, we are on track to submit an IND application to the US FDA this year seeking to initiate our planned Phase 1b/2a trial for SACT-1.

As part of Aptorum Group's Acticule infectious disease platform, ALS-4 is a novel first-in-class orally administered small molecule drug based on an anti-virulence approach targeting staphylococcus aureus including MRSA.

ALS-4 targets the antimicrobial resistant properties of the bacteria and is believed to render the bacteria highly susceptible to the host's immune clearance. ALS-4 is targeted for potential administration on a standalone or on a combination basis with other existing antibiotics such as vancomycin.

SACT-1 is an orally administered repurposed small molecule drug to target neuroblastoma. SACT-1's mechanism has been investigated in our preclinical studies to enhance tumor cell death and suppress MYCN expression (a common clinical diagnosis in high-risk or relapsed neuroblastoma patients where an amplification of MYCN is usually observed).

SACT-1 is designed to be used especially in combination with standard-of-care chemotherapy.

Aptorum Group is a clinical stage biopharmaceutical company dedicated to the discovery, development and commercialization of therapeutic assets to treat diseases with unmet medical needs, particularly in oncology (including orphan oncology indications) and infectious diseases.

The pipeline of Aptorum is also enriched through the establishment of drug discovery platforms that enable the discovery of new therapeutics assets through, e.g. systematic screening of existing approved drug molecules, and microbiome-based research platform for treatments of metabolic diseases; and the co-development of a novel molecular-based rapid pathogen identification and detection diagnostics technology with Accelerate Technologies Pte Ltd, commercialization arm of the Singapore's Agency for Science, Technology and Research.
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