Phase 1 Clinical Trial of Anticancer Drug MRX34 Underway
announced today that it has initiated a Phase 1 clinical study of MRX34, the first miRNA to advance into a human clinical trial for cancer. The Phase 1 trial is being conducted in patients with unresectable primary liver cancer or metastatic cancer with liver involvement.
“The initiation of this clinical trial is a landmark event for cancer drug development,” said Paul Lammers, M.D., President and Chief Executive Officer of Mirna Therapeutics. “Scientists at Mirna were among the first to elucidate the promise of tumor suppressor miRNAs as new therapeutic candidates. The preclinical profile of MRX34 across a range of tumors strongly suggests that miRNA-based therapeutics may represent a potent, new class of anticancer drugs working through a mechanism that affects multiple oncogenic pathways simultaneously.”
“Results from this initial clinical trial will be used to evaluate the safety of MRX34, and help us evaluate the potential of this compound for further clinical study,” said Andrew Brenner, M.D., Ph.D., medical oncologist and assistant professor in the School of Medicine at The University of Texas Health Science Center at San Antonio, and principal investigator on the study being conducted at the Cancer Therapy & Research Center at the Health Science Center. “ We are excited to be the first to bring this new and innovative therapeutic approach to our patients. We look forward to reviewing the clinical data and to furthering the study of miRNA-based cancertherapeutics.”
The Phase 1 MRX34 study will follow a standard oncology study design, consisting of an initial dose-escalation phase followed by an enrichment phase, and is expected to enroll up to 48 patients in total. MRX34 is a miRNA “mimic” of tumor suppressor miR-34 delivered using a liposomal delivery formulation in-licensed from Marina Biotech. Mirna filed its first Investigational New Drug Application with the U.S. Food and Drug Administration for MRX3 4 earlier this year. Additional information on the study and enrollment can be found at clinicaltrials.gov (http://clinicaltrials.gov/ct2/show/NCT01829971).
This project is funded in part by a Cancer Prevention and Research Institute of Texas (CPRIT) Commercialization grant.
miRNAs are approximately 20-25 nucleotides long and affect gene expression by interacting with messenger RNAs. Unlike short interfering RNAs (siRNAs), miRNAs are encoded in the human genome and are used as natural regulators of global gene expression. More than 1,500 miRNAs are encoded in the human genome and are thought to control expression of approximately 30% of all genes. Since each miRNA appears to regulate the expression of tens to hundreds of different genes, miRNAs can function as “master-switches,” efficiently regulating and coordinating multiple cellular pathways and processes. Misregulation of miRNAs appears to play a fundamental role in the occurrence, growth and dissemination of many cancers, and replacement of down regulated miRNAs in tumor cells results in a positive therapeutic response.
About Mirna Therapeutics
Mirna Therapeutics, Inc. (Mirna) is a biotechnology company focused on the development and commercialization of miRNA therapeutics. The Company has a foundational intellectual property portfolio on the therapeutic use of miRNAs developed by its own scientists as well as in-licensed from other institutions. Mirna’s IP portfolio contains more than 300 miRNAs with applications in oncology and other diseases. Oncology-directed miRNAs include those that are key tumor suppressors in cancer, such as miR-34 and let-7, which have been shown to block tumor growth in a number of different preclinical animal studies. The Company, founded in 2007 and located in Austin, Texas, has received significant funding from Sofinnova Ventures, New Enterprise Associates, Pfizer Venture Investments and other private investors, as well as the State of Texas, both through the State’s Emerging Technology Fund and from the Cancer Prevention and Research Institute of Texas (CPRIT). For more information, visit www.mirnarx.com.
Mirna Therapeutics, Inc.
Paul Lammers, M.D., M.Sc.
President and CEO