Rasna Therapeutics Advances Development Of A Novel Treatment For NPM1-Mutated Acute Myeloid Leukemia

NEW YORK--(BUSINESS WIRE)--Rasna Therapeutics, Inc. (OTCQX:RASP), a clinical stage biotechnology company focused on the development of disease-modifying drugs for hematological malignancies, today provided an update from further studies towards the development of formulated RASP-101, a novel modality for treatment of NPM1-mutated acute myeloid leukemia (AML).

NPM1-mutated AML is a specific genetic variation of leukemia that accounts for approximately one third cases of AML in adults. A phase II clinical trial which was initiated in 2014 and completed in two and a half years, involved patients with refractory or relapsed NPM1-mutated AML treated with cycles of dactinomycin at a dose of 15µg per kilogram per day for 5 consecutive days (EudraCT number 2014-000693-18). As earlier published, intravenous treatment of refractory or relapsed AML patients with dactinomycin (12.5µg or 15µg per day) for 5 consecutive days had produced hematological complete response in specific patients (Falini et al., N Eng J Med. 373: 12, 2015).

“We are pleased to report an update to these initial promising findings, which are now corroborated by the follow up of a phase II clinical study which achieved complete response in 40% of patients. Although further studies are warranted to understand the mechanism of action of dactinomycin in NPM1-mutated AML, we are delighted to move forward with development of a formulated RASP-101 for treatment of AML patients,” said Dr. Brunangelo Falini, a member of the scientific advisory board of Rasna Therapeutics, Inc.

“We believe that our approach is a breakthrough and a first-in-class treatment for AML patients. We are determined to rapidly develop formulation of RASP-101 to enable multi-center clinical studies in NPM1-mutated AML. Patient stratification, based on specific AML gene mutations, is part of Rasna’s strategy to improve clinical outcome for this unmet clinical need,” commented Alessandro Padova, Chairman of Rasna.

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About Dr. Brunangelo Falini, M.D.

Brunangelo Falini is the head of the Institute of Hematology and Hemopoietic Stem Cell Transplantation at the University of Perugia, Perugia, Italy. His research activity has mainly focused on the genetic characterization of lymphomas and leukemias using monoclonal antibodies and, more recently, NGS technologies. He led the research group who discovered NPM1 mutations in AML in 2005 and the BRAF-V600E mutation in hairy cell leukemia in 2011. Both of these seminal discoveries have translated into a better diagnosis and therapy of patients affected by these hematological malignancies. Dr. Falini is the recipient of numerous prestigious prizes, including the “Josè Carreras Award” from EHA (Barcelona, 2010) and the “Leopold Griffuel Prize” from ARC (Paris, 2015).

About Rasna Therapeutics, Inc.

Rasna Therapeutics, Inc. is a clinical stage biotechnology company focused primarily on the development of drug candidates for leukemia and lymphoma. Abnormal epigenetic modification is recognized to play an important role in the pathogenesis of acute myeloid leukemia (AML), leading to silencing of genes involved in tumor suppression and cellular reproduction. Rasna has a balanced portfolio targeting NPM1-mutated AML through direct and indirect disease-modifying approaches. Rasna is also exploiting inhibition of lysine specific demethylase-1 (LSD1), an enzyme involved in epigenetic control, as a promising and novel approach against AML. Rasna is entering preclinical development with a novel class of potent and selective reversible LSD1 regulators that have shown efficacy and LSD1 target modulation in in-vitro and in IND-enabling pre-clinical studies.

Forward-Looking Statements

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