Regulus provides pipeline updates

Regulus provided an update on advancements to its pipeline programs. In Hepatitis B virus programs, the company has identified several microRNA targets that serve as host factors for the virus. The company believes that targeting a host factor in the liver represents a unique mechanism of action for treatment of the virus compared to other programs in development and holds the potential for achieving a functional cure. The company currently expects to file an IND for the HBV program in 2H19, with the potential of achieving human proof-of-concept in a Phase 1 study. In the Glioblastoma multiforme program, its lead anti-miR candidate targeting microRNA-10b demonstrated statistically significant improvements in survival as both a monotherapy as well as in combination with temozolamide in an orthotopic GBM animal model. In combination with TMZ, the addition of a single dose of anti-mir-10b led to a more than two-fold improvement in survival compared to TMZ alone. The company expects to nominate a clinical candidate by year-end and plans to seek a partner to further advance its development. In its Non-Alcoholic Steatohepatitis program, its lead candidate has demonstrated improvement in key endpoints with significant improvement of liver fibrosis and hyperglycemia compared to control-treated animals. The company plans to seek a partner to further advance its development. In its Autosomal Dominant Polycystic Kidney Disease program, and in consultation with the FDA, the company has initiated a new 27-week chronic mouse toxicity study with several changes believed to address the unexpected findings in the earlier terminated chronic mouse toxicity study. Data from the new study are anticipated in 1Q19.
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