Business
Rasna Therapeutics, Inc., Announces Follow Up Phase II Clinical Data Confirming Efficacy of Actinomycin D in Patients with NPM1-Mutated Acute Myeloid Leukemia
Rasna Therapeutics, Inc., Announces Follow Up Phase II Clinical Data Confirming Efficacy of Actinomycin D in Patients with NPM1-Mutated Acute Myeloid Leukemia.
About this update from Actavia Life Sciences, Inc.
[{"type":"text","content":"\n \n Rasna Therapeutics, Inc. (OTCQX: RASP), a clinical stage biotechnology \n company focused on the development of disease-modifying drugs for \n hematological malignancies, today announced follow up Phase II clinical \n data showing that 4 out of 9 (44%) evaluable AML patients carrying the \n NPM1 gene mutation treated with actinomycin D (“Act D”), achieved \n complete remission (CR). Treatment with Act D was well tolerated, except \n that patients experienced oral mucositis as the major toxicity. Rasna \n Therapeutics has developed a proprietary nanoparticle based formulation \n of Act D (RASP-101), which is anticipated to maximize efficacy while \n minimizing oral mucositis. RASP-101 could potentially be a \n first-in-class modality for treatment of NPM1-mutated acute myeloid \n leukemia (AML).\n \n \n NPM1-mutated AML is a specific genetic leukemia entity that accounts for \n approximately one-third cases of AML in adults. As we reported \n previously, intravenous treatment of refractory or relapsed NPM1-mutated \n AML patients with Act D (12.5 µg or 15 µg/day) for 5 consecutive days \n produced hematological complete response in some of them (Falini et al., N \n Eng J Med. 373: 12, 2015).\n \n \n In a follow-up phase II clinical study (ActD-AML-PG01, EudraCT \n 2014-000693-18) in refractory/relapse (R/R) AML patients carrying NPM1 \n gene mutation, treatment with Act D at 15 µg/kg/day for 5 days every 28 \n days induced CR in 4 out of the 9 evaluable patients (44.4%) with only 1 \n or 2 cycles of therapy. Three out of the 4 (75%) patients who obtained \n CR relapsed after 3, 5 and 7 months, respectively. One patient underwent \n haploidentical allogeneic PBSC transplantation at 3 months after CR \n achievement and is alive in molecular CR (MRD-negative) after 24 months.\n \n \n “The precise mechanism of action of Act D in NPM1-mutated AML is still \n not clearly understood. Follow up data confirms our earlier findings and \n further support the use of Act D to induce complete hematological \n remissions with possible long-term molecular responses in NPM1-mutated \n AML patients. To note, our first previously reported NPM1-mutated AML \n patient (resistant to hypomethylating therapy) treated with Act D \n remains in molecular remission at over 3 years since CR achievement,” \n said D...