Pipeline

Our pipeline is focused on diseases of high unmet need where the latest discoveries in microRNA biology are challenging traditional drug development paradigms and creating opportunities for novel therapeutic approaches. Our team of microRNA experts and seasoned drug developers understands the unique needs of microRNA-based therapeutics development: identifying valid, ‘druggable’ targets and applying pharmacology and translational biology to optimize therapies with the greatest chance of clinical success.

Program
Indication
  • Phase Ph1
  • Ph2
  • Ph3
COBOMARSEN (MRG-106) Blood Cancers

COBOMARSEN (MRG-106) Blood Cancers

Hematological malignancies are cancers that affect blood, bone marrow, and lymph nodes, and include leukemias and lymphomas. They are also referred to as blood cancers or liquid tumors. Hematological malignancies result from improper production, function, and proliferation of blood cell including lymphocytes. Depending on the specific disease and its severity, treatment of hematological malignancies may involve chemotherapy, radiotherapy, or bone marrow transplant.

miRagen’s lead program targets miR-155, a microRNA that has key roles in the differentiation, function and proliferation of blood and lymph cells. Therapeutic inhibition of miR-155 in lymphoma cells is believed to restore normal function and reduce the aberrant cell proliferation that is characteristic of cancerous cells. We are developing MRG-106, to treat patients who suffer from the mycosis fungicides (MF) form of cutaneous T-cell lymphoma (CTCL). MF is the most common form of CTCL. It generally affects the skin, often causing rash and sometimes disfiguring skin lesions and tumors that can progress internally over time.

In addition to MF, elevation of miR-155 has been associated with several other blood cancers and certain solid tumors. We believe there is a potential opportunity to develop a companion diagnostic that could detect and quantify levels of miR-155 in circulating blood or malignant cells. We believe this approach may then allow for the selection of patients with elevated miR-155 levels who may be more likely to benefit from MRG-106 treatment and allow the drug to be used selectively in multiple cancers. There are several types of cancer in which high levels of miR-155 have been discovered, including subsets of diffuse large B-cell lymphoma, acute myeloid leukemia, certain virally induced lymphomas such as HTLV-1 associated lymphoma and Burkitt’s Lymphoma, Down Syndrome-associated acute lymphocytic leukemia, and other types of cancer. We plan to evaluate additional types of lymphoma and leukemia in Phase 1 clinical trials and intend to explore other potential applications for MRG-106 through additional clinical studies in other tumor types.

Cutaneous T-Cell Lymphoma (CTCL)
  • Ph1
Adult T-Cell Lymphoma/Leukemia
  • Ph1
Diffuse Large-B Cell Lymphoma
  • Ph1
Chronic Lymphocytic Leukemia
  • Ph1
MRG-201 Pathologic Fibrosis

MRG-201 Pathologic Fibrosis

Tissues and organs in the body respond to injury through a wound-healing response involving formation of fibrous scar tissue which is characterized by deposition of extracellular matrix proteins like collagen and elastin. In the setting of chronic stress however, progressive accumulation of fibrotic tissue impairs the function of vital organs like the heart, lungs, liver and kidney and is a major (and largely untreatable) contributor to morbidity and mortality.

We believe that miR-29 is a powerful regulator of extracellular matrix production and is an attractive therapeutic target for the treatment of cutaneous and pathological fibrosis.

Cutaneous Fibrosis
  • Ph1
Idiopathic Pulminary Fibrosis
  • Preclin
Other Fibrotic Indications
  • Preclin
MRG-107 Neurodegeneration

MRG-107 Neurodegeneration

Our Neuro-Inflammation program is initially focused on Amyotrophic Lateral Sclerosis (ALS), or Lou Gehrig’s disease. ALS is a neurodegenerative disease characterized by rapidly progressive muscle weakness and wasting throughout the body. Patients have trouble moving, speaking, swallowing and breathing. ALS is a severe disease and most patients ultimately succumb to breathing problems, typically within 3-5 years of initial symptoms. Improvements in survival with existing drug treatments are modest and new therapeutic approaches are urgently needed.

miRagen is targeting microRNA miR-155 as a novel therapy for ALS. miR-155 is inappropriately increased in the spinal cord of patients with ALS. We believe it plays key roles in the inflammation and immune mechanisms implicated in ALS pathology and that inhibition of miR-155 has the potential to alleviate symptoms and extend survival.

ALS
  • IND Enabling
MRG-110* Ischemia

MRG-110* Ischemia

miRagen’s program targets miR-92a, a microRNA linked to the regulation of blood vessel growth. Increasing blood vessel growth is an attractive revascularization approach for the treatment for chronic ischemic disorders. antimiR-92a offers a potential therapeutic to accelerate the healing process.

Heart Failure
  • Ph1
Incisional Complications
  • Ph1

We are pursuing a diverse set of disease targets identified as having both high unmet medical need and scientific evidence of benefit from microRNA modulation. Within that context, we prioritize programs on our ability to rapidly translate the pre-clinical science into a mechanistic proof-of-concept trial in patients. We emphasize target validation in human-derived in vitro systems and industry standard models of disease.

Programs that progress into human trials are designed to be accompanied by a validated set of PD biomarkers that allow us to stratify and enrich the study population. Through this approach, we seek to progressively reduce the risk of the programs by quantifying target engagement and identifying the efficacious dose prior to progression to Phase 2 trials.