Marina Biotech Reports That Partner Mirna Therapeutics Presented Interim Phase 1 Data on Its First-in-Class microRNA-34 Mimic - Seite 2

About Mirna Therapeutic's Phase 1 Trial
The Phase 1 MRX34 study design consists of an initial dose-escalation phase, followed by an expansion phase in one or more focused cancer types. In the study, MRX34 is administered intravenously (IV) to patients in one of two dosing schedules, either twice a week for three weeks with one week off, during 28-day cycles or daily for five days with two weeks off, in 21-day cycles, until disease progression or intolerance. Dose escalation in the daily times five dosing schedule is currently ongoing. The primary objectives of the clinical trial are to establish the maximum tolerated dose and the recommended Phase 2 dose for future clinical trials. The secondary objectives are to assess the safety, tolerability and pharmacokinetic profile of MRX34 after IV dosing as well as to assess pharmacodynamics and clinical activity of MRX34. Clinical activity is assessed by tumor response using RECIST, modified RECIST (primary liver cancer), or other cancer-specific criteria (hematologic malignancies) and evaluated by Computed Tomography/Magnetic Resonance Imaging (CT/MRI), Positron Emission Tomography/ Computed Tomography (PET/CT), or other standard methods every six to eight weeks. The study is being conducted at leading cancer research centers in the U.S. and Korea.

Data from 52 patients are included in the interim analysis, which showed that treatment emergent adverse events primarily consisted of infusion reactions such as fever, chills, nausea, vomiting, back and flank pain. The addition of dexamethasone, a corticosteroid, as premedication was found to ameliorate infusion reactions. Other treatment emergent adverse events included fatigue, diarrhea, headache, dehydration, elevation of liver enzymes, decreased albumin, hyponatremia, lymphopenia, thrombocytopenia, and neutropenia.

About MRX34
MRX34 is a double-stranded microRNA "mimic" of the naturally occurring tumor suppressor miR-34, which inhibits cell cycle progression and induces cancer cell death. Mirna filed its first Investigational New Drug (IND) Application with the U.S. Food and Drug Administration (FDA) for MRX34 in early 2013 and initiated the ongoing Phase 1 clinical trial in April 2013, making MRX34 the first microRNA replacement therapy product candidate to enter a clinical trial in cancer. MRX34 is delivered using the SMARTICLES^® liposomal delivery formulation, in-licensed from Marina Biotech.