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Dicerna (DRNA) Reports Second Quarter 2017 Financial And Operating Results And Provides Corporate Update

8/11/2017 6:40:09 AM

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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today reported financial and operating results for the second quarter ended June 30, 2017.

We are pleased to have reported a number of significant events during this quarter that support the execution of our business strategy,” stated Douglas Fambrough, president and chief executive officer of Dicerna. “Specifically, the completion of our $70 million convertible preferred stock financing and the key addition of Dr. Ralf Rosskamp as chief medical officer, have served to further solidify our financial and leadership capacity. As a result, we are in a strong position to continue to advance DCR-PHXC, our lead GalXC™-based product candidate, into Phase 1 clinical studies for primary hyperoxaluria (PH) early next year and to continue to pursue Investigational New Drug (IND) application-enabling activities for our undisclosed rare disease program as well as for DCR-HBVS. More recently, during the 12th International Workshop on Primary Hyperoxaluria held this past July, we presented new preclinical data for DCR-PHXC demonstrating how inhibition of the lactate dehydrogenase A (LDHA) gene reduced oxalate production in multiple animal models of PH. These data highlight the role of LDHA as an optimal therapeutic target and the potential utility of DCR-PHXC to treat all forms of the disease. These findings indicate that this novel target may offer the ability to treat an expanded population of patients who currently have no other effective options.”

GalXC™ Program Update

  • During the second quarter of 2017, Dicerna continued to progress preclinical activities for its four core therapeutic programs, including DCR-PHXC for PH, an undisclosed rare disease program, DCR-HBVS for hepatitis B virus, and DCR-PCSK9 for hypercholesterolemia. Dicerna’s development activities are focused in the areas of rare diseases, chronic liver diseases, cardiovascular diseases, and viral infectious diseases.
    • Primary Hyperoxaluria: On July 15, 2017, in a series of presentations at the 12th International Workshop on Primary Hyperoxaluria, Dicerna presented new preclinical data showing DCR-PHXC’s ability to inhibit LDHA resulting in consistent and significant reduction in urinary oxalate levels in animal models of PH type 1 (PH1), PH type 2 (PH2) and idiopathic PH. PH is a family of severe, rare, genetic liver disorders characterized by overproduction of oxalate that often results in kidney failure.

Research from multiple animal models of PH demonstrated how DCR-PHXC inhibits LDHA, which the Company has identified as potentially being an optimal therapeutic target in patients with the disease. The data highlights included:

  • LDHA inhibition reduces oxalate to normal or near-normal levels in PH types 1, 2, and ethylene glycol-induced hyperoxaluria (a model for idiopathic PH).
  • LDHA reduction has a near-linear correlation with oxalate reduction and offers a minimal metabolic intervention. These benefits of LDHA inhibition may translate into consistent therapeutic activity even in the event of a missed dose. There are numerous case reports of LDHA deficiency naturally occurring in healthy humans, with no reported adverse effects due to deficiency in the liver.
  • DCR-PHXC appeared to be well tolerated in these animal studies, with no adverse effects in the liver. Formal animal toxicology studies are ongoing.

During the workshop, Dicerna also reported data from its Primary HYperoxaluria Observational Study (PHYOS), an international, multicenter, observational study in patients with a genetically confirmed diagnosis of PH1. PHYOS is collecting data on key biochemical parameters, including changes in oxalate, glycolate, and other metabolites, implicated in the pathogenesis of the disease. Dicerna continues to advance PHYOS to facilitate DCR-PHXC development and hopes to use the data to better understand the baseline PH1 disease state, which will help guide long-term drug development plans.

  • Twenty (20) patients were enrolled in the study, with a median age at screening of 21 years (range 12-61 years). The patients had been diagnosed at a median age of 7 years (range 1-59 years), and 14 patients (74%) had a medical history of renal stones.
  • Over the six-month observation period, the variability (coefficient of variation) between 24-hour urine measurements of oxalate at different time points was 28%.
  • These data will be used by Dicerna’s clinical team in the design of future clinical studies using 24-hour urinary oxalate excretion as a surrogate marker for clinical benefit.

Dicerna is on track to file a clinical trial application (CTA) in Europe for DCR-PHXC in late 2017 and to commence human clinical trials in the first quarter of 2018. During the workshop, Dicerna disclosed that the DCR-PHXC clinical trial will be conducted at multiple sites in Europe and will include both healthy volunteer and patient cohorts. The Company anticipates that study participants will receive a single ascending dose of DCR-PHXC via subcutaneous injection, transitioning, as appropriate, to multiple ascending doses. The primary endpoints will include safety and tolerability, urine and plasma biomarkers, and pharmacokinetics.

  • Undisclosed Rare Disease Involving the Liver: Dicerna advanced IND application-enabling activities for a second GalXC-based clinical candidate targeting an undisclosed rare disease. For competitive reasons, the Company has not yet publicly disclosed the target gene or disease. Dicerna is on track to file an IND application in the U.S. and/or CTA in Europe for this program in the second quarter of 2018.
  • Chronic Hepatitis B Virus (HBV): Dicerna continued to progress its DCR-HBVS program, which targets HBV directly, and has initiated formal IND-enabling activities. Current therapies for HBV rarely lead to a long-term immunological cure as measured by the clearance of HBV surface antigen (HBsAg) and sustained HBV deoxyribonucleic acid suppression. Based on findings from its preclinical studies, Dicerna is evaluating whether its GalXC RNAi platform can produce an experimental HBV-targeted therapy that significantly reduces HBsAg expression in affected patients and that has the potential to be delivered in a subcutaneous dosing paradigm. The Company expects to file an IND application in the U.S. or CTA in Europe for this program at approximately the end of 2018.
  • Hypercholesterolemia: 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 commercial properties, such as small subcutaneous injection volumes and less frequent dosing.

Financing Update

  • As previously reported, on April 11, 2017, Dicerna closed a stock purchase transaction for the sale of redeemable convertible preferred stock (Preferred Stock) to a syndicate of current and new investors led by Bain Capital Life Sciences, under which the Company received gross proceeds of $70.0 million (Private Placement). At the closing, Dicerna issued 700,000 shares of Preferred Stock, which are convertible into common shares at an initial conversion price of $3.19 per share. In addition to the lead investor, other participants in the Private Placement included Cormorant Asset Management, Domain Associates, EcoR1 Capital, RA Capital and Skyline Ventures, among others. Under the terms of the Preferred Stock purchase agreement, Adam M. Koppel, M.D., Ph.D., a managing director of Bain Capital Life Sciences, joined Dicerna's board of directors, which has been expanded to nine seats.

Corporate Update

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