Aptorum Group Limited Has Received IND Clearance From the US FDA to Initiate Clinical Trials for Repurposed Small Molecule Drug SACT-1 for the Treatment of Neuroblastoma

NEW YORK & LONDON & PARIS – Aptorum Group Limited (Nasdaq: APM, Euronext Paris: APM) (“Aptorum Group” or “Aptorum”), a clinical stage biopharmaceutical company focused on novel technologies including the targeting of oncological diseases, announced that the group has received clearance from the US FDA regarding the IND application to initiate clinical trials of SACT-1, an orally administered repurposed small molecule drug for the treatment of neuroblastoma.

Dr. Clark Cheng, Chief Medical Officer and Executive Director of Aptorum Group, commented: “Further to our current ALS-4 clinical trial program, we are pleased to announce the clearance from the US FDA regarding our IND application to initiate clinical trials for SACT-1. This represents another key milestone for the company and one of the targeted strategic goals for the year of 2021. This milestone supports the focus of Aptorum Group in the United States and reflects the potential of our scientific rigor and novel approach of our products. Neuroblastoma is a highly unmet solid tumor arising in the nervous system outside of the brain predominantly in pediatric patients. The clinical behavior of neuroblastoma is highly variable with majority cases being highly aggressive. We believe that SACT-1 has the potential to effectively target this disease and address the unmet demands of such.”

Based on the prior recommendations provided by the US FDA in our Pre-IND meeting, the IND-opening clinical trial, a bioavailability/food effect study which is believed to last for approximately four months, will be conducted in the United States, followed by a planned Phase 1b/2a trial in pediatric patients suffering from relapsed or refractory high-risk neuroblastoma, subject to further clearance by the US FDA. The objective of the bioavailability/food effect study is to compare the relative bioavailability of the newly developed SACT-1 pediatric formulation in healthy adult subjects.

About SACT-1

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.

About Aptorum Group

Aptorum Group Limited (Nasdaq: APM, Euronext Paris: APM) 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 (i) 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 (ii) 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.

For more information about Aptorum Group, please visit www.aptorumgroup.com.

Contacts:

Aptorum Group Limited
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+44 20 80929299

Redchip – Financial Communications United States
Investor relations
Dave Gentry
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+1 407 491 4498

Actifin – Financial Communications Europe
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Ghislaine Gasparetto
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