Clinical Trial Update

Biosight Launches A Phase 1/2 Clinical Trial Of Aspacytarabine In Combination With Venetoclax For First-Line AML Induction Therapy, Followed By Aspacytarabine Consolidation

November 24,2022 05:08 AM
- By Admin

BioSight Ltd. Ltd., a pharmaceutical development company developing innovative therapeutics for hematological malignancies and disorders, today announced enrollment of a first patient to a phase 1/2 clinical trial designed to evaluate the safety and efficacy of its lead asset aspacytarabine (BST-236) in combination with the BCL2 inhibitor venetoclax for induction therapy of newly-diagnosed Acute Myeloid Leukemia (AML), followed by aspacytarabine single-agent consolidation therapy. The trial, conducted in leading clinical centers in the United States, enrolls newly-diagnosed AML patients who are unfit for standard induction chemotherapy due to age or comorbidities.

"Encouraged by the safety and efficacy of aspacytarabine monotherapy in the treatment of over 120 AML and myelodysplastic syndrome (MDS) patients to date", said Dr. Ruth Ben Yakar, Biosight's CEO, "we believe that aspacytarabine may be positioned to serve as a superior novel backbone for AML and MDS therapy, either as a single agent or in combination with other therapies, including targeted therapy agents. We are therefore very excited to be launching this first combination therapy trial".

About Aspacytarabine (BST-236)

Aspacytarabine (BST-236) is a novel proprietary anti-metabolite. It is composed of cytarabine covalently bound to asparagine, acting as a pro-drug of cytarabine. Cytarabine serves as the backbone of Acute Myeloid Leukemia (AML) therapy for over 50 years due to its superior efficacy, however, it is associated with severe bone marrow, gastrointestinal, and neurological toxicities, which significantly limit its use, especially in older and medically compromised patients. Due to its unique pharmacokinetics and metabolism, aspacytarabine enables high-dose therapy with lower systemic exposure to free cytarabine and relative sparing of normal tissues. As such, aspacytarabine may serve as a superior therapy for AML and other hematological malignancies and disorders, including for older adults who are unfit for intensive therapy.

Aspacytarabine was granted FDA Fast Track Designation for first-line treatment of AML patients unfit for standard chemotherapy, and Orphan Drug designations from the FDA and EMA in AML, as well as Orphan Drug designation in myelodysplastic syndrome (MDS) from the FDA, which entitle Biosight to seven and ten years of market exclusivity upon aspacytarabine marketing approval for the treatment of AML and MDS in the US, and AML in Europe, respectively.

Results from a recently completed Phase 2b study evaluating aspacytarabine as a single-agent first-line AML therapy demonstrate safety and impressive single-agent activity. Additional studies are ongoing to evaluate aspacytarabine as a second line treatment for patients with relapsed or refractory MDS or AML