Biosight Receives Orphan Medicinal Product Designation from the European Medicines Agency for ...
AIRPORT CITY, Israel, Dec. 17, 2020 (GLOBE NEWSWIRE) -- Biosight Ltd., a pharmaceutical development company developing innovative therapeutics for hematological malignancies and disorders, today announced the European Medicines Agency (EMA) has granted Orphan Medicinal Product Designation to aspacytarabine (BST-236) for the treatment of acute myeloid leukemia (AML). Aspacytarabine, Biosight’s lead product candidate, is a novel antimetabolite designed to provide the benefit of intensive chemotherapy while reducing the associated systemic toxicity. This new EMA designation, in combination with the Orphan Drug Designation granted last year by the U.S. Food and Drug Administration (FDA), provide Biosight with 7 and 10 years of market exclusivity in the U.S. and Europe, respectively.
“Obtaining Orphan Medicinal Product Designation from the EMA is an additional, important recognition of aspacytarabine’s potential to provide a significant benefit to AML patients, including to older adults who are unfit for standard chemotherapy, by addressing the major need for effective and well tolerated treatment options,” said Ruth Ben Yakar, Ph.D., CEO of Biosight. “We are encouraged by our progress, recently presenting updated data from our ongoing Phase 2b study at the 62nd American Society of Hematology (ASH) Annual Meeting that demonstrate efficacy across key measures including encouraging complete remission and MRD (-) rates, duration of response and overall survival. These positive data, in combination with our recently announced Series C financing, leave us well positioned to continue our rapid development of aspacytarabine, which we are expanding to additional Phase 2 trials, including in relapsed/refractory myelodysplastic syndrome and AML.”
Orphan Medicinal Product Designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. To qualify, an investigational medicine must be intended to treat a seriously debilitating or life-threatening condition that affects fewer than five in 10,000 people in the EU, and there must be sufficient non-clinical or clinical data to suggest the investigational medicine may produce clinically relevant outcomes. EMA orphan drug designation provides companies with certain benefits and incentives, including 10 years of market exclusivity upon marketing authorization, clinical protocol assistance, access to a centralized marketing authorization procedure valid in all EU member states and reduced regulatory fees.
About Aspacytarabine (BST-236)
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 40 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 treatment of AML patients unfit for standard chemotherapy, and FDA and EMA Orphan Drug Designations, which entitle Biosight to seven and ten years of market exclusivity in the U.S. and Europe, respectively, upon aspacytarabine marketing approval for the treatment of AML in each territory.
A Phase 2b study is ongoing to confirm the promising results obtained in a Phase 1/2a study of aspacytarabine as a single-agent first-line AML therapy. For more information regarding the Phase 2b clinical study of BST-236, please visit www.clinicaltrials.gov.
About Biosight Ltd.
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 to be initiated 2021 include a study in relapsed/refractory AML and myelodysplastic syndrome (MDS) under a collaboration agreement recently signed with the European cooperative group, GFM. For additional information, please visit www.biosight-pharma.com.
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