Regulus Therapeutics - Q1 2022

Transcript

Speaker 0

Please note this event is being recorded. I would now like to turn the conference over to Chris Calzada, Chief Financial Officer. Please go ahead.

Speaker 1

Thank you, operator. Good afternoon, everyone, and thank you for joining us to discuss Regulus Therapeutics' Q1 2022 financial results and corporate highlights. Joining me on today's call is Jay Hagen, President and Chief Executive Officer and Doctor. Dennis Dryden, 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.

Thanks,

Speaker 2

Chris, and welcome everyone to our Q1 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 RGLS-eight thousand four hundred and twenty nine for the treatment of ADPKD.

The study is a single is a Phase 1 single ascending dose study in healthy volunteers to assess safety, tolerability and pharmacokinetics of RGLS-eight thousand four hundred and twenty nine. Following this study, we plan to initiate a Phase 1b multiple ascending study in adult patients with ADPKD to assess the safety, tolerability and pharmacokinetics of RGLS-eight thousand four hundred and twenty nine treatment as well as evaluate the dose response of RGLS-eight thousand four hundred and twenty nine 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 effect were demonstrated with our 1st generation compound, Argelis 4,326. We anticipate top line data from the healthy volunteer portion of the study in the second half of twenty twenty two and top line biomarker data from the first cohort of RGLS-eight thousand four hundred and twenty nine treated patients in the first half of twenty twenty three. Now I'd like to shift gears and share the latest update regarding latimercin, our RG-twelve program for the treatment of Alport syndrome.

In February, we announced completion of enrollment by our partner Sanofi in the Phase 2 HERA clinical study evaluating ledamericin 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 twenty twenty three 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 $25,000,000 milestone. Moving on to our early stage research activities. In March, we announced the initiation of preclinical studies in ALS under collaboration agreement with the Brigham and Women's Hospital. The collaboration entails an exploration of the biologic of miR-one hundred and fifty five 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 targeted targeting dysregulated microRNA for other Grievous CNS diseases. With that, I'll turn the call back over to Kris?

Speaker 1

Thank you, Jay. Turning to our financial results. As of March 31, 2022, our cash and cash equivalents totaled approximately $53,900,000 We expect our cash runway to extend into the 4th quarter of 2023. This guidance does not include the potential Sanofi milestones that Jay discussed earlier. Research and development expenses for the Q1 of 2022 totaled $3,700,000 compared to $3,300,000 in the same period in 2021.

These amounts reflect the internal and external costs associated with advancing expenses for the Q1 of 2022 totaled $2,900,000 compared to $2,500,000 for the same period in 2021. These amounts reflect personnel related and ongoing general business operating costs. Net loss for the Q1 of 2022 was $6,700,000 compared to net loss of $6,000,000 for the same period in 2021. Basic and diluted net loss per share for the Q1 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 Jane.

Speaker 2

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

Speaker 0

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

Speaker 3

Thank you for taking my question. Could you comment on the overall expenses associated with the upcoming trial for 8429?

Speaker 1

Sure. So our 8,429 program since we have our history of 4,326 will start the SAD study, which will commence here in the Q2, 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 twenty twenty two.

Speaker 2

And the cost associated with it, Yi, if that's where you're going, they're very similar to the 4,326 costs, say, for inflationary environment we're in. And then the longer the Phase Ib study, obviously, we stopped the 4,326 study short by one 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 8,429 study. So instead of 9 patients per cohort, we have 12 that we did in the 1st 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 4,326 study.

Speaker 3

Okay, got it. And will there be any additional milestone associated with the data readout from Alport syndrome in the first half of twenty twenty three?

Speaker 2

The $25,000,000 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 founding agreement.

Speaker 3

Okay. Thank you.

Speaker 2

Thank you.

Speaker 0

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

Speaker 2

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.

Speaker 0

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