Dicerna Pharmaceuticals (DRNA): Clinical Progress and Full Coffers

Edward Kim  |

We highlighted Dicerna Pharmaceuticals (NASDAQ: DRNA) in this space two months ago when the company was presenting at the Cowen Health Care Conference. The company provided a clinical update along with its Q1 results yesterday, and we continue to believe that this fallen biotech angel is one worth watching.

Dicerna is developing therapeutics based on the science of ribonucleic acid interference (RNAi). RNA serves several critical cellular functions, acting as a messenger between deoxyribonucleic acid (DNA) and the ribosomes where proteins are made, forming vital portions of the ribosomes themselves and acting as a carrier for amino acids to be used in the synthesis of proteins. By interfering with a particular RNA molecule's message, the synthesis of that protein and potentially the progress of a particular disease may be interrupted.

Source: Dicerna Pharmaceuticals Corporate Overview, April 7, 2017

Progress in the Clinic

Dicerna's proprietary technology platform - GalXC - is the basis for the development of next-generation RNAi-based therapies designed to silence disease-driving genes in the liver. Compounds produced via GalXC are intended to be broadly applicable across multiple therapeutic areas, including rare diseases, chronic liver diseases, cardiovascular diseases and viral infectious diseases.

The company has prioritized four therapeutic programs based on GalXC:

  • Primary Hyperoxaluria Type 1 (PH1): Dicerna continued to progress IND-enabling studies for its lead product candidate, DCR-PHXC, which is being evaluated for the treatment of patients with PH1, a rare, inborn error of metabolism in which the liver produces excessive levels of oxalate, which in turn causes damage to the kidneys and other tissues in the body. The company plans to announce additional details about its DCR-PHXC program at the Oxalosis and Hyperoxaluria Foundation’s 12th International Workshop on Primary Hyperoxaluria in Spain in July 2017, and is on track to file an investigational new drug (IND) submission with the FDA or a clinical trial application (CTA) with Health Canada later this year. Dicerna plans to commence Phase 1 clinical trials in Q1 2018.
  • Undisclosed Rare Liver Disease: In Q1 2017, Dicerna initiated IND-enabling activities for a second GalXC clinical candidate targeting an undisclosed rare disease. The Company expects to file an IND application or CTA for this program in Q2 2018.
  • Chronic Hepatitis B Virus: Dicerna has recently initiated formal IND-enabling studies for DCR-HBV, which targets HBV directly. Based on findings from its preclinical studies, the company is evaluating whether its GalXC RNAi platform can produce an experimental HBV-targeted therapy that significantly reduces hepatitis B surface antigen (HBsAg) expression in affected patients and that has the potential to be delivered in a subcutaneous dosing paradigm. Dicerna expects to file an IND or CTA for this program around Q4 2018.
  • Cardiovascular Disease: Dicerna continued to develop its DCR-PCSK9 program, which targets the PCSK9 gene and will be evaluated for the treatment of statin-refractory patients with hypercholesterolemia. The company is positioned to advance DCR-PCSK9 into formal preclinical development. Based on preclinical studies, Dicerna believes that its GalXC RNAi platform has the potential to produce a PCSK9-targeted therapy with attractive properties such as small subcutaneous injection volumes and relatively infrequent dosing.

$70 Million Convertible

Dicerna closed on $70 million of convertible preferred stock financing last month, led by Bain Capital Life Sciences and a syndicate of current and new investors. Combined with cash on hand, the company expects to be able to advance its first three development programs while progressing DCR-PCSK9 toward formal preclinical development, which would take Dicerna into 2019.

In our March 2017 article, we stated that we thought the stock had bottomed in February, and we reiterate that belief. While we won't predict whether or not it will ever see the lofty levels of two years ago, we note that Dicerna has an ambitious platform technology that, if successfully developed, could be the foundation of a range of therapeutics for several rare and not-so-rare diseases.

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