Biosight Completes Enrollment in Phase 2b Study for First-Line Acute Myeloid Leukemia Therapy

Biosight Ltd. recently announced it has completed enrollment in the company’s ongoing Phase 2b trial evaluating aspacytarabine (BST-236) as a single-agent first-line acute myeloid leukemia (AML) therapy for patients unfit for standard intensive chemotherapy.

“Completing enrollment in our first-line AML Phase 2b study is an important milestone in the development of aspacytarabine,” said Dr. Ruth Ben Yakar, Chief Executive Officer of Biosight. “The data reported to date provide compelling evidence that aspacytarabine, with a differentiated mechanism that enables high-dose chemotherapy with reduced toxicity, has the potential to transform standard of care for AML patients. We are grateful to the investigators and patients who have enabled this accomplishment, and are dedicated to continue our progress with aspacytarabine as we work to provide new treatment options for patients with the greatest need and limited standard of care options, including patients unfit for intensive chemotherapy, and patients with poor-prognosis baseline characteristics.”

Dr. Liat Flaishon, MD, PhD, VP of Medical Affairs at Biosight, added “We are highly encouraged by the responsiveness of the investigators and patients who have chosen to participate in the study, particularly in light of the challenges associated with the ongoing COVID-19 pandemic. We look forward to presenting updated interim data from this trial at ASCO and to expanding the potential of aspacytarabine via the advancement of additional trials and indications, including in relapsed/refractory myelodysplastic syndrome (MDS) and AML.”

Jessica K. Altman, MD, Professor, Medicine, Feinberg School of Medicine of Northwestern University and Lead Study Investigator, said “Aspacytarabine offers a much needed potential new treatment for older adults with AML. The limited duration of the treatment and the indicated compelling efficacy have the potential to provide key treatment advantages which may address unmet needs in poor-prognosis patient populations.”

Aspacytarabine 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 AML therapy for over 45 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, which entitles Biosight to seven and ten years of market exclusivity upon aspacytarabine marketing approval for the treatment of AML in the US and Europe, respectively.

Interim results from an ongoing Phase 2b study evaluating aspacytarabine as a single-agent first-line AML therapy demonstrate safety and single-agent activity, and additional studies are launched to evaluate aspacytarabine as a second line treatment for patients with relapsed or refractory myelodysplastic syndrome (MDS) or AML. For more information regarding the Phase 2b clinical study of BST-236, please visit www.clinicaltrials.gov.

Biosight is a private Phase 2 clinical-stage biotech company developing innovative therapeutics for hematological malignancies and disorders. Biosight’s lead product, aspacytarabine (BST-236), is an innovative proprietary anti-metabolite which addresses unmet medical needs by enabling high-dose chemotherapy with reduced systemic toxicity. Aspacytarabine is currently being investigated as a single agent in a Phase 2b for first-line treatment of acute myeloid leukemia (AML), following completion of a Phase 1/2a study which demonstrated tolerability with promising efficacy in the challenging population of AML patients unfit for standard of care chemotherapy. Additional Phase 2 studies in relapsed/refractory AML and MDS, including a study in collaboration with the European cooperative group, GFM. For additional information, visit www.biosight-pharma.com.