Pipeline
Mirna’s pipeline consists of six lead candidates, all of which are directed toward oncology. Extensive experimental testing demonstrated robust anti-tumor activity in at least two of three cancers. Many of the candidates show promising efficacy in multiple cancer models, suggesting that these miRNAs are key tumor suppressors with broad therapeutic potential. Mirna’s primary focus is on solid cancers, including non-small cell lung cancer and advanced prostate cancer, all of which represent areas of large, unmet clinical need. Mirna anticipates filing its first IND in 2011. Among the lead candidates are let-7 and miR-34. Please click here to learn more about these programs (let-7). Mirna’s platform also contains a second-generation of oncology-directed therapies, as well as therapies for other disease areas.
About non-small cell lung cancer:
Lung cancer is the most common and the most deadly cancer in the world, accounting for 28% of total cancer mortality. Over 219,000 new cases of lung cancer and 159,000 deaths from lung cancer are predicted to occur in the United States during 2009. The number of lung cancer deaths is still increasing. Survival of lung cancer patients is strongly correlated with the stage of disease. Cure rates after surgical resection in patients without distant or loco-regional tumor spread is approximately 70%. Unfortunately 85% of lung cancer patients have advanced disease at presentation. For patients with advanced disease, the prognosis is dismal because the available treatment regimens (chemotherapy and radiation therapy) are essentially non-curative and primarily serve to prolong survival. Several oncogenes have been associated with lung cancer including p53, RAS, and MYC, but drugs specifically targeting these genes have yet to be commercialized.
Lung cancer has two primary presentations. Small cell lung cancer (SCLC) affects 15% of patients and non-small cell lung cancer (NSCLC) affects approximately 84%. NSCLC consists of three major types: adenocarcinoma, squamous cell carcinoma, and large cell carcinoma, with lung adenocarcinomas and squamous cell carcinomas accounting for the vast majority of all lung cancers. Academic and industry labs have developed aerosol-based methods for delivering anti-sense oligonucleotides, plasmids, and siRNAs to the lungs of mice and rats. The promise of using this route of delivery and the paucity of alternative therapeutic treatments for lung cancer make this disease indication an attractive target for miRNA-based therapies. The observation that several miRNAs play key roles in lung cancer, the relative ease of miRNA drug delivery to lung and the large number of people with the disease makes lung cancer an ideal target for a miRNA based drug development program.
Among Mirna’s drug candidates in the treatment of non-small cell lung cancer is let-7. Therapeutic delivery of let-7 leads to growth inhibition of cultured lung cancer cells and tumor growth in the animal. To learn more about this program, click here.
About metastatic prostate cancer:
In the US, carcinoma of the prostate is the second most common cause of cancer-related death in men following lung cancer. In the US alone, 1 in 6 men will develop prostate cancer in their lifetime; 1 in 30 men will die of this disease. Prostate cancer generally occurs in men 50 and older, with the median age at diagnosis of 72. The American Cancer Society estimates that more than 192,000 new cases will be diagnosed in 2009 and more than 27,000 men will die of the disease. According to autopsy data, histologically-apparent cancer may be found in the prostates of as many as 42% men over 50 years of age who die of other causes. While surgery has proven to be effective in treating patients with localized disease, the survival rates for patients who have metastatic prostate cancer are less than 20%.
Mirna’s miRNA expression studies in tumor and normal prostate tissues show that the expression levels of more than ten miRNAs are consistently altered in prostate cancer. Many of these miRNAs are further altered in the most tumorigenic cells in prostate tumors, suggesting that the altered expression of the miRNAs contributes to the aggressiveness of the disease. miR-34 is one of the candidates that Mirna is developing as a therapeutic for prostate cancer.