Ryvu Therapeutics receives Orphan Drug Designation from FDA for SEL120 to treat Acute Myeloid Leukemia

Krakow, Poland – 28 March 2020 – Ryvu Therapeutics (WSE: RVU), a clinical-stage biopharmaceutical company developing novel small molecule therapies that address emerging targets in oncology, today announced that the U.S. Food and Drug Administration (FDA) has granted an orphan drug designation (ODD) to Ryvu’s SEL120, for the treatment of patients with acute myeloid leukemia (AML).

SEL120 is an oral, selective inhibitor of CDK8 kinase which is implicated in the development of hematological malignancies and solid tumors. A clinical phase 1b study of SEL120 is currently enrolling in 6 investigational sites in USA, investigating safety and preliminary efficacy of SEL120 in treatment of patients with relapsed or refractory AML or high-risk myelodysplastic syndrome (HR-MDS).

“The FDA’s granting orphan drug designation to SEL120, is a significant encouragement for advancement of our clinical strategic plan addressing the unmet medical needs in the area of AML treatment, a disease where patients still face poor prognosis” said Setareh Shamsili, MD, PhD, Chief Medical Officer and Executive VP at Ryvu Therapeutics.  “SEL120 has shown strong proof of concept in the preclinical studies and has received a strategic support from The Leukemia & Lymphoma Society (LLS) through its Therapy Acceleration Program® (TAP). SEL120 may have the potential to offer an important therapeutic benefit in AML and in particular to those refractory/relapsed AML patients with the poorest prognosis, worldwide. “

Orphan drug status (ODD) is intended to advance drug development for rare diseases. The FDA provides ODD to drugs and biologics that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions that affect fewer than 200,000 people in the U.S. The FDA’s orphan drug designation allows the drug for the designated indication to be eligible for requesting a seven-year period of U.S. marketing exclusivity upon approval of the drug, as well as potential of other development assistance and financial incentives: https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=733220.

About SEL120

SEL120 is an oral, selective inhibitor of CDK8, a kinase which is a part of the mediator complex and is essential for the activity of super-enhancers important to the regulation of RNA transcription. CDK8 kinase is implicated in the development of hematological malignancies and solid tumors. SEL120 was discovered with the Ryvu Therapeutics discovery engine platform and is currently in Phase 1b study in adult patients with AML or HR-MDS who are refractory to treatment or have relapsed after previous therapies. Patients are enrolled in the study independent of specific tumor mutational burden. The study will also assess pharmacokinetic and pharmacodynamic parameters of SEL120.

SEL120 has received support from The Leukemia & Lymphoma Society Therapy Acceleration Program® (TAP), a strategic initiative to partner directly with innovative biotechnology companies and leading research institutions to accelerate the development of promising new therapies for blood cancers. More information about TAP program is available at: https://www.lls.org/therapy-acceleration-program

About Acute Myeloid Leukemia

Acute myelogenous leukemia or acute myeloid leukemia (AML) is a heterogenous hematological malignancy involving the clonal expansion of myeloid blasts in the bone marrow and peripheral blood with possible spread to liver and spleen. It is estimated that there are around 90 000 cases of AML worldwide each year, and American Cancer Society estimates that approximately 20 000 of these in U.S. only. The median age at diagnosis is 66 years, with 54% patients over 65 years, and 33% over 75 years. Of those diagnosed at a later age, the diagnosis is often associated with underlying myelodysplastic syndromes (MDS), sometimes linked to cancer chemotherapy and radiotherapy exposure.