miRagen Gets Orphan-Drug Designation From FDA

miRagen Therapeutics (NASDAQ:MGEN) received an orphan-drug designation for its MRG-106 candidate against mycosis fungoides, from the US Food and Drug Administration.
As quoted in the press release:

Mycosis fungoides is the most common form of a type of blood cancer called cutaneous T-cell lymphoma (“CTCL”). CTCL occurs when certain types of T-cells become cancerous and cause debilitating tumors in the skin and in other parts of the body.
Paul Rubin, M.D., miRagen’s Executive Vice President of Research and Development, said, “We see the FDA’s granting of orphan-drug designation for MRG-106 as underscoring the medical need for novel therapies in the treatment of mycosis fungoides, and we believe it should facilitate our ability to continue developing this product candidate for potential use in these patients.”
“This designation is an important step forward for our MRG-106 program as we continue to advance our clinical and regulatory strategy in areas of high unmet medical need,” added William S. Marshall, Ph.D., President and CEO of miRagen. “Patients suffering from mycosis fungoides have few treatment options available, and MRG-106 could be an alternative approach to battling the disease.”
About Miragen Therapeutics, Inc.
Miragen Therapeutics, Inc. is a clinical-stage biopharmaceutical company discovering and developing proprietary RNA-targeted therapeutics with a specific focus on microRNAs and their role in diseases where there is a high unmet medical need. microRNAs are short RNA molecules, or oligonucleotides, that regulate gene expression or activity and play a vital role in influencing the pathways responsible for many disease processes. miRagen believes its experience in microRNA biology and chemistry, drug discovery, bioinformatics, and translational medicine provide it with a potential competitive advantage to identify and develop microRNA-targeted drugs designed to regulate gene pathways to result in disease modification. miRagen uses its expertise in systems biology and oligonucleotide chemistry to discover and develop a pipeline of product candidates. miRagen’s two lead product candidates, MRG-106 and MRG-201, are currently in Phase 1 clinical trials. miRagen’s clinical product candidate for the treatment of certain cancers, MRG-106, is an inhibitor of microRNA-155, which is found at abnormally high levels in several blood cancers. miRagen’s clinical product candidate for the treatment of pathological fibrosis, MRG-201, is a replacement for miR-29, which is found at abnormally low levels in a number of pathological fibrotic conditions, including cardiac, renal, hepatic, and pulmonary fibrosis, as well as systemic sclerosis. In addition to miRagen’s clinical programs, it continues to discover and develop a pipeline of pre-clinical product candidates. The goal of miRagen’s translational medicine strategy is to progress rapidly to first in human studies once it has established the pharmacokinetics (the movement of drug into, through, and out of the body), pharmacodynamics (the effect and mechanism of action of a drug), safety and manufacturability of the product candidate in preclinical studies.  For more information, please visit www.miragentherapeutics.com.
For information on clinical trials please visit www.clinicaltrials.gov.

Click here to read the full press release.

Source: www.miragentherapeutics.com