Pharma Focus Europe

Aptorum Group Shares Latest Progress on Clinical Trial for Neuroblastoma Using Repurposed Small Molecule Drug SACT-1: Phase 1b/2a Updates

Friday, June 23, 2023

Aptorum Group Limited (Nasdaq: APM) is a clinical stage biopharmaceutical company that focuses on developing novel technologies for the treatment of oncological diseases, including neuroblastoma. Recently, Aptorum announced that it has submitted the Phase 1b/2a clinical trial protocol for its drug candidate, SACT-1, to the US Food and Drug Administration (FDA). SACT-1 is an orally administered repurposed small molecule drug that is being evaluated in combination with chemotherapy for the treatment of high-risk neuroblastoma in patients experiencing first relapse or refractory cases.

Darren Lui, the Chief Executive Officer of Aptorum Group, expressed his satisfaction with the progress made and stated that the company is ready to proceed with the Phase 1b/2a trials for SACT-1 in neuroblastoma patients, pending recruitment of suitable participants. He emphasized the significance of this milestone and the potential of SACT-1 to effectively target neuroblastoma, which is a solid tumor primarily affecting pediatric patients and known for its aggressive nature. Lui also highlighted the importance of addressing the unmet medical needs in this field.

The objectives of the Phase 1b part of the study are to determine the recommended phase 2 dose (RP2D) based on safety, pharmacokinetics, and efficacy. The Phase 2a part of the study will focus on assessing the preliminary efficacy of SACT-1 in neuroblastoma patients. Aptorum Group is committed to working closely with regulatory agencies to gain approval for SACT-1 and make a positive impact on the treatment of neuroblastoma.

SACT-1 is an orally administered repurposed small molecule drug specifically designed to target neuroblastoma. Preclinical studies have shown that SACT-1 enhances tumor cell death and suppresses MYCN expression, which is commonly observed in high-risk or relapsed neuroblastoma cases with MYCN amplification. The drug is intended to be used in combination with standard-of-care chemotherapy to improve treatment outcomes for patients with neuroblastoma.

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