Regulus Therapeutics - Q2 2022

Transcript

Speaker 0

Please note this event is being recorded. I would now like to turn the conference over to Chris Calcutta, Chief Financial Officer of Regulus.

Please go ahead.

Speaker 1

Thank you. Good afternoon, everyone, and thank you for joining us to discuss Regulus Therapeutics' Q2 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 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 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.

Speaker 2

Thanks, Chris, and welcome everyone to our Q2 earnings call and business update. I'll begin first with a general update on our ADPKD program. The Q2 was particularly eventful beginning with the U. S. Food and Drug Administration's acceptance of our IND for RGLS-eight thousand four hundred and twenty nine the treatment of autosomal dominant polycystic kidney disease.

This was followed by the dosing of the first subject in our Phase 1 single ascending dose study of RGLS A129 to assess the safety, tolerability and pharmacokinetics of the drug in healthy volunteers. Following this study, we plan to initiate a Phase Ib multiple ascending dose 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 to 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. Our objective is to establish a dose response around the dose level where robust clinical biomarker effects were demonstrated with our 1st generation compound. We anticipate top line data from the healthy volunteer portion of the study later this year. And top line biomarker data from the first cohort of RGLS-eight thousand four hundred and twenty nine treated ADPKD patients in the first half of twenty twenty three.

The single ascending dose study is nearing completion consistent with our planned execution and timeline and the multiple ascending dose portion of the study is well on track for initiation shortly after completion of the SAD study. Additionally, RGLS-eight thousand four hundred and twenty nine was granted orphan drug designation from the FDA. Orphan drug designation or ODD identifies drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the United States. And with that helps facilitate drug development by providing potential financial and regulatory incentives. Finally, we're thrilled to welcome Doctor.

Amin Kamal as our new Vice President of DMPK, a key new hire for the Regulus team. We look forward to his contributions as we progress ARGLS-eight thousand four hundred and twenty nine as well as the rest of the pipeline. With that, I'll now turn the call back over to Chris for an update on our financial results. Chris?

Speaker 1

Thank you, Jay. Turning to our financial results. As of June 30, 2022, our cash, cash equivalents and marketable securities totaled approximately $47,500,000 We expect our cash runway to extend into the Q4 of 2023. Research and development expenses for the Q2 of 2022 totaled $4,700,000 compared to $4,200,000 in the same period in 2021. These amounts reflect the internal and external costs associated with advancing our ADPKD program and other research efforts within our pipeline.

General and administrative expenses for the Q2 of both 2021 and 2022 totaled $2,500,000 These amounts reflect personnel related and ongoing general business operating costs. Net loss for the Q2 of 2022 was $7,300,000 compared to net loss of $6,000,000 for the same period in 2021. Basic and diluted net loss per share for the Q2 of 2022 was $0.50 per share compared to basic and diluted net loss of $0.78 per share for the same period in 2021. With that, I will turn the call back over to James.

Speaker 2

Thanks, Chris. At this time, we're happy to take any questions you might have. Operator, could you please open the lines?

Speaker 1

Thank you. We will

Speaker 0

now begin the question and answer session. Your first question comes from Yi Chen with H. C. Wainwright. Please go ahead.

Speaker 3

Thank you. This is Yi for H. C. Wainwright. Thank you for taking my question.

I'm sorry if you mentioned on the call, so is there a plan for 0112 now that Sanofi sorry, Sanofi has not going to I mean Sanofi didn't really achieve the success in the Alport syndrome trial. Is it still in the hand of Sanofi or Regulus could do something about it?

Speaker 2

Yes. Sanofi notified us, which we shared in our 8 ks update that they're looking for other opportunities for the compound without giving any specifics to us. But you may know that in a number of different fibrotic diseases. And additionally, you may ask around any specifics with respect to the data. We have not, per the terms of the agreement, have had access yet to the data.

What we do know is that this was a planned pre specified interim analysis when 24 subjects randomized 2 to 1, so that's 16 on active and 8 on placebo had completed 1 year of blinded treatment. And that's what the futility analysis was based upon.

Speaker 3

So do you know whether Sanofi will make a decision as to whether they will proceed with another indication

Speaker 2

agreement of this type where they have to use commercially reasonable efforts to work on the program. Got it.

Speaker 3

Okay. And regarding the SAD trial of 8,429, we should have the initial data in the Q4 of this year, correct?

Speaker 2

Yes. Yes. We'll plan an update, Yi, because we'll obviously be announcing when we initiate the MAD. The initiation of the MAD comes after review of all the safety data from the SAD. And so once we've completed that and announced that we're moving into the MAD, we'll plan to provide an update to investors at that time.

Got

Speaker 3

it. Is the company considering any other preclinical stage candidates?

Speaker 2

Yes, we actively are. We don't talk about them a lot, but we have quite an interesting early stage effort ongoing in a number of different CNS diseases and we look forward to providing an update as those programs move through several in vivo proof of concept studies. But the team is actively working on those and we look forward to providing that update sometime likely early next year.

Speaker 3

Got

Speaker 2

it. Thank you. You're welcome.

Speaker 0

Thank

Speaker 1

you.

Speaker 0

There are no further questions at this time. I would now like to turn the conference back over to Jay Hargan for closing remarks.

Speaker 2

Well, thanks everyone for joining us today for this brief call. As I mentioned, the programs are all on track per our timelines and we look forward to providing updates on them as they advance and reach key milestones. We appreciate your support and interest in Regulus and we'll catch up soon. Thank you.