Regulus Therapeutics (RGLS): Alport Syndrome and Other microRNA Targets

Edward Kim  |

Alport syndrome is a life threatening condition driven by genetic mutation in the kidneys, leading to kidney disease, hearing loss and eye abnormalities. According to the National Organization for Rare Disorders, Alport syndrome is estimated to affect one in every 5,000 to 10,000 people in the US, or as many as 65,000 patients based on the latest census estimates. People with Alport syndrome experience progressive loss of kidney function, according to the National Institutes of Health, and many affected individuals have blood or high levels of protein in their urine. The kidneys become less able to function as this condition progresses and can result in end-stage renal disease. Alport syndrome is estimated to account for 3% of children with chronic kidney disease and 0.2% of adults with end-stage renal disease in the United States. There are no approved therapies.

We've been following a company called Regulus Therapeutics (Nasdaq: RGLS) that is developing a treatment for Alport syndrome based on the science of targeting microRNAs - small naturally occurring non-coding ribonucleic acids that are 22 to 25 nucleotides in length. This novel class of small non-coding RNAs has been shown to function as important regulators of gene expression and play a role in multiple cellular processes. [GIF source: Katharina Petsche]

Source: Regulus Therapeutics Corporate Presentation, March 2017

Regulus believes that its proprietary microRNA platform has several significant advantages over conventional drug targeting methods including:

The company's clinical program for Alport syndrome is being conducted in partnership with Sanofi Genzyme (NYSE: SNY).

Source: Regulus Therapeutics website

Regulus has been publicly traded since 2012, and the stock carried a market value in excess of $1 billion in 2014. The stock has fallen off dramatically since that peak, as the company has suffered a series of disappointments in the clinic, most recently with the announcement last month that it was discontinuing clinical development of its hepatitis C virus program and its cholestatic disease (an impairment of bile function or flow from the liver to the gall bladder) program. While the market has reacted accordingly to the disappointing news, the long view at current valuation may be that it's positive for a company at this stage to recognize when a program has reached its clinical utility and to decide to direct precious resources elsewhere.

The company just raised $46 million in gross proceeds in a follow-on offering priced last week at 91 cents (post-money valuation of $94 million), doubling the cash on its balance sheet, and the stock has traded up 30% since then. Given the lead program in Alport syndrome and the demonstrated underlying science of microRNA targeting, we'll be listening with interest to the company's Q2 update next week on Tuesday, Aug. 1st, at 5:00 pm ET. A live webcast of the call will be available online, or investors may dial 877-257-8599 (domestic) or 970-315-0459 (international). Conference ID is 57973022.

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