Regulus Therapeutics Inc.

Ladies and gentlemen, thank you for standing by. Welcome to the Regulus Therapeutics Second Quarter 2021 Financial Results Conference call. At this time, all participants' lines are in listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press Star 1 on your telephone. Please be advised that today's conference is being recorded. If you require any further assistance, please press Star 0. I would now like to hand the conference over to Chris Calzada, Chief Financial Officer of Regulus Therapeutics. Thank you. Please go ahead.

Thank you. Good afternoon, and thank you for joining us to discuss Regulus Therapeutics' second quarter 2021 financial results and corporate highlights. Joining me on today's call is Jay Hagan, President and Chief Executive Officer, and Dennis Drygen, Chief Scientific Officer. Jay will provide opening remarks and share progress on our ADPKD program, and I will review the financial results before we open the line for questions. Before we begin, I'd like to remind you that this call will contain forward-looking statements concerning Regulus Therapeutics' future expectations, plans, prospects, corporate strategy, and performance, which constitute forward-looking statements for the purposes of the Safe Harbor provision under the Private Security 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 our filings with the SEC. Any forward-looking statements represent our views only as of the date of this webcast and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligations to update such statements. I will now turn the call over to Jay.

Thanks, Chris, and welcome everyone to our second quarter earnings call and business update. The team at Regulus had a very productive second quarter, continuing through the last several weeks, which I'm pleased to share with you today. We just completed our preparations and associated documents and have requested a Type A meeting with FDA. The objective for this meeting is to get feedback on our overall approach to addressing the remaining hold requirements and the data generated to date that forms the basis of the modeling requirements laid out by FDA. When a Type A meeting is requested, FDA typically grants a meeting or provides feedback within 30 days from receipt of the meeting request. We look forward to sharing updates on that progress. This past June at PKD Connect, we announced additional data from the first cohort of patients in our Phase 1B clinical trial of RGLS4326 in patients with ADPKD. The additional data demonstrated clinical proof of mechanism by showing that short-term treatment with RGLS4326, led to target engagement in the kidneys and consequently led to statistically significant increases in both polycystin 1 and polycystin 2. The overall trend in these protein changes show increasing levels of both PC1 and PC2 over time with a sustained effect one month after completion of dosing, which suggests to us that less frequent dosing could be utilized. Measured levels of PC1 and PC2 have previously been shown to be inversely correlated with disease severity. Thus, these data serve to further validate MIR-17 as a target for ADPKD treatment. Also at PKD Connect in June, we presented new data generated by our collaborator at University of Texas Southwestern, Dr. Vishal Patel. These new data were from a certain preclinical model that is very analogous to the human disease and showed that treatment with RGLS4326 results in increased gene and polycystin levels in vitro, as well as significant improvements in total kidney size, cyst count, and overall kidney health. The model is relevant in that it harbors a PKD1 mutation equivalent to human ADPKD. With regards to safety, as previously reported, RGLS4326 was well-tolerated by all nine patients in this first cohort, with no serious adverse events reported. And all reported AEs or adverse events were mild and generally transient in nature. Returning to our ongoing Phase 1B clinical study of RGLS4326, we're also pleased to announce today that we've recently completed enrollment of the second cohort of patients. Now, these patients are being administered 0.3 milligrams per kilogram of RGLS4326 every other week for four doses. The dose of RGS4326 for the third and final cohort will be chosen based on the results of this second cohort in relation to what we've been able to demonstrate already at one milligram per kilogram. And enrollment in that third and final cohort would be expected to commence sometime in the fourth quarter this year. We anticipate top line data from the second cohort in the fourth quarter as well. And finally, after completing what we believe is a robust and extensive non-GLP tox assessment of our next-generation compound targeting MIR17, we have formally nominated the molecule as a clinical candidate named RGLSA429. GMP manufacturing for GLP tox and Phase I clinical studies is underway, with an IND anticipated in the first quarter next year. Recall this molecule was designed to have an overall improved product profile compared to RGLS4326. By retaining all of the key beneficial aspects, including its potency for the target, MIR17, as well as preferential kidney distribution, while also dialing out activity towards the putative receptor underlying the clinical signs associated at the dose-limiting tox level of RGLS4326. And finally, a team back in our labs at Regulus continues to advance our understanding of the role of microRNA in important disease areas. I'll now turn the call back over to Chris for a discussion on our financial results before we get to your questions. Chris.