Regulus Therapeutics Inc.
Q1 2022 Earnings Call Transcript

Published: 12 May 2022

[Operator:]

Good day, and welcome to the Regulus Therapeutics Inc. First Quarter 2022 Conference Call. . Please note, this event is being recorded. I would now like to turn the conference over to Cris Calsada, Chief Financial Officer. Please go ahead.

[Crispina Calsada:]

Thank you, operator. Good afternoon, everyone, and thank you for joining us to discuss Regulus Therapeutics First Quarter 2022 Financial Results and Corporate Highlights. Joining me on today's call is Jay Hagan, President and Chief Executive Officer; and Dr. Denis Drygin, Chief Scientific Officer. Jay will provide opening remarks and share progress on our ADPKD and other programs, 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 purpose 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 our filings with the SEC. In addition, 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'll now turn the call over to Jay.

[Joseph Hagan:]

Thanks, Cris, and welcome, everyone, to our first quarter earnings call and business update. I'll begin first with a general update on our ADPKD program. We're proud to share with you that earlier this week, the U.S. Food and Drug Administration accepted our IND for RGLS8429 for the treatment of ADPKD. The study is a Phase I single ascending dose study in healthy volunteers to assess safety, tolerability and pharmacokinetics of RGLS8429. Following this study, we plan to initiate a Phase Ib multiple ascending study in adult patients with ADPKD to assess the safety, tolerability and pharmacokinetics of RGLS8429 treatment, as well as evaluate the dose response of RGLS8429 on ADPKD biomarkers, including polycystins, cystic kidney volume and overall kidney function. Recall that our objective in this study is to establish a dose response around the dose levels where robust clinical biomarkers effects were demonstrated with our first-generation compound, RGLS4326. We anticipate top line data from the healthy volunteer portion of the study in the second half of 2022 and top line biomarker data from the first cohort of RGLS8429 treated patients in the first half of 2023. Now I'd like to shift gears and share the latest update regarding Lademirsen, our RG-012 program for the treatment of Alport syndrome. In February, we announced completion of enrollment by our partner, Sanofi, in the Phase II HERA clinical study evaluating Lademirsen for the treatment of adult patients with Alport syndrome under our collaboration and license agreement with Sanofi. Final data is expected in the first half of 2023, and if successful, would further validate the potential of our platform technology, advance our understanding of genetic kidney diseases and strengthen the financial position of the company with a $25 million milestone. Moving on to our early-stage research activities. In March, we announced the initiation of preclinical studies in ALS under a collaboration agreement with the Brigham and Women's Hospital. The collaboration entails an exploration of the biologic effects of miR-155 inhibitors in both in vitro and in vivo models of ALS, a devastating nervous system disorder that weakens muscles and impacts physical function. Results from these initial studies will be used to guide the design of future experiments in additional animal models of the disease and characterize their properties, and we look forward to providing future updates on these preclinical studies. This work dovetails nicely with our internal work looking at targeting dysregulated microRNA for other grievous CNS diseases. With that, I'll turn the call back over to Cris for an update on our financial results. Cris?

[Crispina Calsada:]

Thank you, Jay. Turning to our financial results. As of March 31, 2022, our cash and cash equivalents totaled approximately $53.9 million. We expect our cash runway to extend into the fourth quarter of 2023. This guidance does not include the potential Sanofi milestone that Jay discussed earlier. Research and development expenses for the first quarter of 2022 totaled $3.7 million compared to $3.3 million in the same period in 2021. These amounts reflect the internal and external costs associated with advancing our ADPKD program and other research efforts in our pipeline. General and administrative expenses for the first quarter of 2022 totaled $2.9 million compared to $2.5 million for the same period in 2021. These amounts reflect personnel related and ongoing general business operating costs. Net loss for the first quarter of 2022 was $6.7 million compared to net loss of $6 million for the same period in 2021. Basic and diluted net loss per share for the first quarter of 2022 was $0.05 per share compared to basic and diluted net loss of $0.08 per share for the same period in 2021. With that, I will turn the call back over to Jay.

[Joseph Hagan:]

Thanks, Cris. At this time, we're happy to take any questions. Operator, you can open the lines.

[Operator:]

. The first question comes from Yi Chen with H.C. Wainwright.

[Yi Chen:]

Could you comment on the overall expenses associated with the upcoming trial for 8429?

[Crispina Calsada:]

Sure. So our 8429 program since we have our history of 4326 will start the SAD study, which will commence here in the second quarter with the data readout in the second half of this year. And then our plan is to, immediately after that, move into commencing our MAD study in the second half of 2022.

[Joseph Hagan:]

And the costs associated with it, Yi, et cetera, where you're going, they're very similar to the 4326 costs, say, for inflationary environment we're in. And then the longer -- the Phase Ib study, obviously, we stopped the 4326 study short by 1 cohort, saving some money for us and now can redirect those into the 8429 program. But we are doing a placebo-controlled arms in the 8429 study. So instead of 9 patients per cohort, we have 12 that we did in the first generation compound. We have 12 patients per cohort, and we're dosing for twice as long. So the cost for the MAD portion in patients will be probably approximately 50% higher than the cost for the 4326 study.

[Yi Chen:]

Okay. Got it. And will there be any additional milestone associated with the data readout from Alport syndrome in the first half of 2023?

[Joseph Hagan:]

The $25 million is the last and final milestone from Sanofi, and they're responsible for the royalties that we would have owed to Ionis and Alnylam for our funding agreement.

[Operator:]

. This concludes our question-and-answer session. I would like to turn the conference back over to Jay Hagan for any closing remarks.

[Joseph Hagan:]

Thanks, operator, and thanks, everyone, for joining us today on our brief update on our progress, and we appreciate your support of Regulus. We look forward to providing future updates. Thank you.

[Operator:]

The conference has now concluded. Thank you for attending today's presentation. You may now disconnect.