Good day, ladies and gentlemen, and welcome to the Dicerna Pharmaceuticals' Second Quarter 2016 Earnings Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow at that time. [Operator Instructions] As a reminder, this conference call is being recorded. I'd now like to turn the conference over to your host for today, Steve Silver. Sir, you may begin.

Thank you, Operator. Good afternoon, and welcome to Dicerna's conference call to discuss the company's 2016 second quarter results. For anyone who has not had the chance to review our results, we issued a press release after close of market today, which is available under the Investor tab on our Web site at www.dicerna.com. You may also listen to this conference call via webcast on our Web site, which will be archived just 30 days beginning approximately two hours after the call is completed. I'd like to remind listeners that management will be making forward-looking statements on today's call, related to the company's future expectations, plans and prospects that constitute forward-looking statements for purposes of the Safe Harbor provision under the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in the Risk Factor section of our Form 10-Q filed with the SEC today. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views of any subsequent date. While we may elect to update these forward-looking statements at some point in the future, we specifically disclaim any obligation to do so if our views change. Now, I'd like to turn the call over to Dr. Douglas Fambrough, Dicerna's President and Chief Executive Officer.

Thank you, Steve. Good afternoon, and thanks to all of you who have joined today's call. With me today are Bob Brown, our Chief Scientific Officer and SVP of Research; and Jack Green, our Chief Financial Officer. The second quarter of 2016 was an exciting period for Dicerna as we formally rolled out our GalXC technology platform as a fully optimized and enabled drug discovery engine. By that, I mean that our efforts over the last few years have created the capability to fully silence disease-associated genes in the liver using a single subcutaneous injection delivered once monthly or even less frequently, and that we can do this reproducibly and consistently across any liver expressed gene across multiple therapeutic areas. During our Investor Day in June, we illustrated these capabilities with examples of GalXC molecules silencing 12 different disease-associated genes, including six demonstrations in non-human primates. These demonstrations include inhibitors of disease targets that will populate our pipeline going forward, and others that we expect offer significant partnering opportunities to Dicerna. The GalXC platform supports our long-term strategy to retain a full or substantial ownership stake and invest internally for indications with focused patient populations, such as certain rare diseases, and we see such indications representing opportunities that carry high probabilities of success, have easily identifiable patient populations, and a limited number of centers of excellence to facilitate reaching these patients. For more complex diseases with multiple gene dysfunctions and larger patient populations, we plan to pursue partnerships that can provide the enhanced scale, resources, and commercial infrastructure required to maximize these prospects. We continue to engage in such discussions and believe that the data we have generated to-date and that which we shared at our Investor Day strongly positions Dicerna to attract interest, not just from any potential partner,…

Thank you, Doug. It was gratifying to be able to present what we view as robust and compelling dataset at our Investor Day in June. That event marked the culmination of the process that saw Dicerna make significant advances in optimizing the GalXC platform, and arriving at a point where we believe the platform is fully enabled and will allow the execution of our mission to deliver novel RNAi therapies to patients with diseases that need new treatment options. We believe the key features of the GalXC platform are its ability to potently silence targeted genes with long durations of action and to do so with the strong safety and tolerability profile. We are encouraged by our progress on all fronts. As described during our Investor Day presentation, our GalXC molecules consist of two strands of RNAi. One strand has been extended, a unique property of our Dicer substrate RNAi structures that provides additional space on the RNAi duplexes for elements that provide enhanced pharmaceutical functions. This extended strand is the origin of the X in GalXC and the C represents the Conjugation to GalNAc with the Gal of course referring to the GalNAc amino sugars that direct GalXC molecules to the liver. In one of our GalXC configurations, the conjugation to GalNAc occurs on the extended region of the RNAi on a folded motive known as a Tetraloop. Tetraloop serve as the stabilizing structure that are ideally suited to provide multiple independent conjugation points for the GalNAc sugars and for potentially conjugating targeting agents for delivery to different tissue types in the future. Tetraloop's are evolutionarily conserve sequences present in many natural RNAis that have very high melting temperatures and high resistance to nucleuses and other degradative activities found inside cells. In addition, the Tetraloop sequence in our GalXC…

Thanks Bob. As I previously mentioned, we plan to retain rights and invest internally in rare diseases as we believe that rare diseases offer an ideal opportunity to leverage our RNAi technology. In rare diseases, and particularly monogenic disorders, that is diseases caused by a single gene acromegaly. The patient population is usually easily-identifiable, and are often associated with centers of excellence for treatment facilitating clinical development. In addition, many of these patients have supported advocacy organizations that can help in drug development efforts. Since in many cases the disease ideology is clear, one can have a higher confidence that a given treatment will provide benefit. The clinical program is often more focused and more rapid than average and also less expensive. All of these characteristics make rare diseases particularly attractive to Dicerna. Our initial focus in rare diseases is primary Hyperoxaluria Type 1, which has the beneficial characteristics I just described. As a brief reminder primary Hyperoxaluria Type 1 or PH1 is a severe rare generic disease where a single gene error in liver metabolism causes patients to have high levels of oxylate in the urine resulting in progressive and severe damage to the kidneys and other organs. As the existing standard of care does not halt disease progression, most PH1 patients experience complete renal failure by their early 20s. Aside from combined liver kidney transplant there are no FDA approved therapies or effective treatments from most PH1 patients. Dicerna's first experimental treatment for PH1 dogged DCR PH1 is a lipid nanoparticle encapsulated DsiRNA-EX-Molecule targeting the HAO1 gene that is administrated via IV infusion. To-date, the compound has shown a profound ability to reduce urinary oxylate levels in the mouse genetic model of PH1 and is now the subject of two clinical trials. The first is an abbreviated dose…

Thank you, Doug. I'd like to briefly summarize the key financial results for the quarter ended June 30, 2016, and direct you to 10-Q filing for additional details. In the second quarter of 2016 the company had a net loss of $15.6 million or $0.75 per share, compared to a net loss of $16.2 million or $0.86 per share for the same period of 2015. Research and development expenses were $11 million for the second quarter of 2016, compared to $11.9 million for the same period of 2015. The year-to-year decrease was primarily due to the timing of manufacturing and preclinical activities related to our PH1 IND filing. And the decrease in platform related expenses due to lower spending in discovery and early development. These decreases were partially offset by an overall increase in clinical activities from initiating additional clinical sites, and patient enrollment in our clinical studies, as well as increased employee-related expenses primarily due to additional hiring. General and administrative expenses for the second quarter of 2016 totaled $4.7 million as compared to $4.5 million for the same period in 2015. The increase was primarily due to an increase in employee-related expenses, including an increase in headcount to support our operations. As of June 30, 2016, we had $69.2 million in cash, cash equivalents, and held-to-maturity investments, as compared to $94.6 million as of December 31, 2015. In addition, we also had $1.1 million of restricted cash on the balance sheet, which reflects collaterals securing our facility lease obligation. Finally, based on our current cash position and operating plan, we continue to expect that we have sufficient cash to fund operations through at least the next 12 months, excluding any additional partnership funding, debt or equity financings. More detail of the financial information and analysis may be found in the company's quarterly report on Form 10-Q, which we filed with the SEC today. I'd now like to turn the call back to Doug.

Thanks, Jack. To summarize, we are enthusiastic about our progress during the second quarter, and believe that our fully-enabled GalXC platform positions Dicerna to build one of the most dynamic pipelines in the biotech space over the coming years. We remain focused on moving forward with programs of high value, which we plan to develop both internally and with collaborators. We think the well of opportunities is very deep. And we believe we have the right team in place to execute on this strategy. We look forward to updating you on future calls, and thank you for your interest in Dicerna, and for your attendance today. We will now open the call to questions. Operator?

Thank you. [Operator Instructions] And our first question comes from Stephen Willey from Stifel. Your lines are open.

Hi, this is Philomena Kamya in for Stephen Willey. Thanks for taking our questions, and congratulations on the progress made this far. With respect to the DCR mixed trial, how many patients have been enrolled in the PNET expansion trial, and is there a target enrollment number in mind?

So, as you noticed, we didn't disclose the number of patients. There's quite a few patients in the trial. I don't think we have the exact number for you. It's an actively enrolling trial. We do have a soft target of 20, but it is -- will be in part a function of what's observed during the conduct of the trial. We are enrolling through the end of this month, and been pleased with the enrollment rate.

Thank you. If I could just ask one more question. With respect to the DCR-PH1 trial, you said that you would be, if I understood you correction, you'd be pursuing both the GalXC and the DCR in parallel, and if you wouldn't mind providing the rationale for evaluating both of them in parallel?

We are actively engaged with the key investigators in the PH1 space in multiple countries, U.S., Netherlands, U.K., France, Germany. And in a rare disease like PH1 there is not a long track record of exactly how trials should be conducted, how data should be evaluated. And based on the history of this particular disease indication, it's clear one needs to be very careful in the conduct of how these studies are conducted. Thus, what we're learning with DCR-PH1 is really critical in understanding how to effectively operate in this space. We believe DCR-PH1 could be an effective treatment for PH1. And between what we're learning while we do it, and the fact that we believe it to be effective, that provides a rationale for continuing to work even though the GalXC-based molecule may ultimately provide a more convenient patient solution for PH1 patients. We'll continue to assess whether it makes sense to develop both programs in parallel or whether it makes sense to consolidate our efforts around one of those programs. And we'll communicate our decisions as we make them.

Okay, sounds great. Thank you so much.

Thank you. And this does conclude our question-and-answer session. I would now like to turn the conference back over to Dr. Fambrough for any further remarks.

I want to thank everyone for your participation today. It was an exciting quarter for Dicerna to be able to formally rollout GalXC, which will underlie much of the future pipeline of the company, and I think provides a profile of a therapeutic platform technology that is about as good as you can get in this industry, so very pleased with it. Thanks very much.

Ladies and gentlemen, thank you for participating in today's conference. This concludes today's program. You may all disconnect. Everyone have a great day.