On September 29, 2021 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported that the first patient has been dosed in the dose confirmation study of SY-2101, a novel oral form of arsenic trioxide (ATO) (Press release, Syros Pharmaceuticals, SEP 29, 2021, View Source [SID1234590480]). The trial will evaluate the pharmacokinetics (PK), safety, and tolerability of SY-2101 to confirm the optimal dose to advance into a planned Phase 3 clinical trial in newly diagnosed acute promyelocytic leukemia (APL) patients.
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"The current standard of care cures most patients but is tremendously burdensome, requiring regular and lengthy infusions of an IV formulation of ATO over nearly a yearlong course of treatment," said Farhad Ravandi, M.D., Professor of Medicine, Chief of Section of Acute Myeloid Leukemia, Department of Leukemia at The University of Texas – MD Anderson Cancer Center. "An oral form of ATO that offers similar efficacy while dramatically reducing the treatment burden would represent a major advance for APL patients. The preliminary Phase 1 data for SY-2101 are very promising, and I look forward to its continued advancement in the current and future studies."
APL is a well-defined subtype of acute myeloid leukemia (AML), which accounts for about 10% of AML cases, with approximately 2,000 APL patients diagnosed annually in the United States and Europe. An intravenously administered formulation of ATO is approved for use in combination with All-Trans-Retinoic-Acid (ATRA) in newly diagnosed APL and, while curative in more than 80% of patients, its administration requires up to 140 infusions over the typical 10-month course of induction and consolidation treatment. In an earlier Phase 1 clinical trial, SY-2101, which is dosed once daily, demonstrated oral bioavailability, PK exposures similar to IV ATO, and a generally well-tolerated safety profile.
"We are thrilled to now be dosing patients in our dose confirmation study of SY-2101," said David A. Roth, M.D., Chief Medical Officer at Syros. "This milestone represents an important step toward delivering a new option for people with APL and a meaningful advance in our efforts to build a leading portfolio of targeted hematology therapies. We believe SY-2101 could quickly become the new standard of care for APL by offering patients similar efficacy with a substantially more accessible and convenient therapy. We plan to move swiftly from our dose confirmation study into a Phase 3 trial next year, with the goal of filing a New Drug Application (NDA) in 2024."
The dose confirmation study is expected to enroll up to 24 patients with newly diagnosed APL and will evaluate safety, tolerability, and PK as well as the effect of food on the absorption of SY-2101. Patients will be enrolled during the consolidation phase of their APL treatment and will receive single doses of SY-2101 in a fasted state, SY-2101 in a fed state, and IV ATO. Patients will then have the option to roll over into a multiple-dose cohort to receive either SY-2101 or IV ATO, which will provide additional data on the steady state PK and safety of SY-2101 in comparison to IV ATO. Syros expects to report data from this study in the first half of 2022.
Based on the results of the dose confirmation study, Syros expects to initiate a Phase 3 clinical trial in newly diagnosed APL patients. Based on input from the FDA, Syros believes molecular complete response rate and event-free survival in comparison to historical control data with IV ATO may support accelerated and full approval, respectively.