FDA Grants Fast Track Designation to Aspacytarabine to Treat Acute Myeloid Leukemia


Biosight Ltd announced that the FDA granted fast track designation to aspacytarabine for the treatment of adult patients aged 75 years or older with acute myeloid leukemia.

The FDA granted fast track designation to aspacytarabine (BST-236) to treat adult patients 75 years or older with acute myeloid leukemia (AML), or who have comorbidities that preclude the use of intensive induction chemotherapy.1

Biosight Ltd, the pharmaceutical developmental company for whom the designation was granted, is currently enrolling patients in a phase 2b study to evaluate aspacytarabine as a first-line therapy for patients unfit for standard chemotherapy.

“Receiving Fast Track designation from the FDA is an important recognition of the potential of BST-236 to address the significant unmet need in the population of AML patients who are medically unfit to receive intensive chemotherapy, and to improve the outcomes for these patients,” said Dr. Ruth Ben Yakar, Chief Executive Officer of Biosight, in a press release.

The company also announced release a planned collaboration with the European cooperative group, GFM, to initiate a Phase 2 trial evaluating aspacytarabine in relapsed/refractory myelodysplastic syndrome (MDS) and AML patients, beginning later this year.

The company’s leading product candidate, aspacytarabine, is a novel proprietary anti-metabolite designed to provide the benefits of intensive chemotherapy without many of the toxicities. The drug is composed of cytarabine covalently bound to asparagine.

The FDA previously granted orphan drug designation to aspacytarabine, allowing Biosight Ltd to have 7 years of market exclusivity upon marketing approval for the treatment of AML.

“The compelling safety and efficacy data from both a completed Phase 1/2a and ongoing Phase 2b studies of BST-236, may establish it as a new intensive therapy backbone of AML and may, for the first time, allow older adults deemed unfit for standard chemotherapy, to benefit from an intensive treatment,” said Yakar in a press release.

The FDA grants the fast track designation to “facilitate the development and expedite the review of drugs to treat serious conditions to fulfill an unmet medical need.” Typically, this allows a drug to be eligible for frequent interactions and communication with the FDA on eligibility for accelerated approval and priority review designations.

According to the American Cancer Society, AML is a rare form of leukemia, accounting for only 1% of all cancers in adults. Estimates for 2020 suggest about 19,940 new cases of AML will emerge in the United States, with about 11,180 deaths from AML. Almost all cases and deaths will be in adults.2


1. Biosight Granted U.S. FDA Fast Track Designation for BST-236 for the Treatment of Acute Myeloid Leukemia [news release]. Airport City, Israel. Published August 4, 2020. https://www.globenewswire.com/news-release/2020/08/04/2072449/0/en/Biosight-Granted-U-S-FDA-Fast-Track-Designation-for-BST-236-for-the-Treatment-of-Acute-Myeloid-Leukemia.html. Accessed August 4, 2020.

2. Key Statistics for Acute Myeloid Leukemia (AML). American Cancer Society. https://www.cancer.org/cancer/acute-myeloid-leukemia/about/key-statistics.html. Accessed August 4, 2020.

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