Q2 2022 Regulus Therapeutics Inc Earnings Call

regular Therapeutics second quarter financial results conference call.

All participants will be in a listen-only mode. Should you need assistance, please signal a conference specialist by pressing the star key followed by zero. After today's presentation, there will be an opportunity to ask questions. To ask a question, you may press star then one on your touch-tone phone.

To withdraw your question, please press star then 2. Please note this event is being recorded.

I would now like to turn the conference over to Chris Calcata, Chief Financial Officer of Regulus. Please go ahead.

Thank you. Good afternoon, everyone, and thank you for joining us to discuss Regulus Therapeutics Second Quarter 2022 Financial Results and Corporate Highlights.

Joining me on today's call is Jay Hagan, President and Chief Executive Officer, and Dr. 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 regular therapeutic 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 Jane.

Thanks, Chris, and welcome everyone to our second quarter earnings call and business update.

I'll begin first with a general update on our ADP-ED program.

The second quarter was particularly eventful, beginning with the U.S. Food and Drug Administration's acceptance of our IND for RGLS8429 for 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 RGLSA 429 to assess the safety, tolerability, and pharmacogenetics of the drug in healthy volunteers.

Following this study, we plan to initiate a phase 1B multiple ascending dose study in adult patients with ADPKD.

to assess the safety, tolerability, and pharmacokinetics of RGLS A429 treatment.

as well as to evaluate the dose response.

of RGLSA 429.

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 first 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 A429 treated ADPKD patients this year.

in the first half of 2023.

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 8429 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 Dr. Amin Kamal as our new Vice President of DNPK, a key new hire for the Regulus team. We look forward to his contributions as we progress RGLS 8429 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? Thank you, Jay.

According to our financial results, as of June 30, 2022, our cash, cash equivalents and marketable securities totaled approximately $47.5 million. We expect our cash runway to extend into the fourth quarter of 2023.

Research and development expenses for the second quarter of 2022 totaled $4.7 million compared to $4.2 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 within our pipeline.

General and administrative expenses for the second quarter of both 2021 and 2022 totaled $2.5 million. These amounts reflect personnel related and ongoing general business operating costs.

Net loss for the second quarter of 2022 was $7.3 million compared to net loss of $6 million for the same period in 2021.

Basic and diluted net loss per share for the second quarter of 2022 was 50 cents per share, compared to basic and diluted net loss of 78 cents per share for the same period in 2021. Whatever is all it takes to get your

With that, I will turn the call back over to Jane.

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

Thank you.

We will now begin the question and answer session. To ask a question you may press star then one on your touch tone phone. If you are using a speakerphone please pick up your handset before pressing the keys.

To withdraw your question, please press star then two.

Your first question comes from Yai Chen with HC Wainwright. Please go ahead.

This is Yifar Este-Munright. Thank you for taking my question. I'm sorry if you mentioned on the call. Is there a plan for RG 01012 now that Sanofi has not really achieved success in the alpha syndrome trial? Is it still in the hand of Sanofi or Regulus could do something about it?

Sanofi notified us, which we shared in our 8K update, that they're looking for other opportunities for the compound without giving any specifics to us. But you may know that MIR21 is implicated 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 two to one, so that's 16 on active and eight on placebo, had completed...

one year of blinded treatment. And that's what the futility analysis was based upon.

Do you know whether Sanofi will make a decision as to whether they will proceed with another indication before the end of this year?

We don't know the exact timing. There's standard diligence clauses in an agreement of this type where they have to use commercially reasonable efforts to work on the program.

Regarding the SAD trial of 8429, we should have some initial data in the fourth quarter of this year, correct?

Yes, yes, we'll plan an update, Yee, 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. 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 it.

Is the company considering any other preclinical stage candidates?

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.

Thank you.

Thank you. You're welcome.

Once again, if you wish to ask a question, please press star then 1. We will now pause a moment to allow for any final questioners to register.

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

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.

We'll catch up soon. Thank you.

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

what our financial results conference call. All participants will be in a listen only mode. Should you need assistance, please signal a conference specials by pressing the star key followed by zero. After today's presentation, there will be an opportunity to ask questions. To ask a question, you may press star then one on your touch-tone phone. To withdraw your question, please press star then two. Please note this event is being recorded. I would now like to turn the conference over to Chris Calcata, Chief Financial Officer of Regulus. Please go ahead. Thank you. Good afternoon, everyone. And thank you for joining us to discuss regular therapeutic second quarter, 2022 financial results and corporate highlights. Joining me on today's call is Jay Hagan, President and Chief Executive Officer and Dr. Dennis Drogens, 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 regular therapeutic 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. 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, Chris, and welcome everyone to our second quarter earnings call and business update. I'll begin first with the general update on our ADPKD program. The second quarter was particularly eventful, beginning with the US Food and Drug Administration's acceptance of our IND for RGLS 842-9 for the treatment of autosomal dominant polycystic kidney disease. This was followed by the dosing of the first subject in our phase one single ascending dose study of RGLS 842-9 to assess the safety, tolerability, and pharmacokinetics of the drug in healthy volunteers. Following the study, we plan to initiate a phase one B multiple ascending dose study in adult patients with ADPKD to assess the safety, tolerability, and pharmacokinetics of RGLS 842-9 treatment, as well as to evaluate the dose response of RGLS 842-9 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 first 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 842-9 treated ADPKD patients in the first half of 2023. 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-entracked for initiation shortly after completion of the SAD study. Additionally, RGLS 842-9 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 Dr. Meen Kamal, as our new vice president of DMPK, a key new hire for the regular scene. We look forward to his contributions as we progress RGLS 842-9 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? Thank you, Jay. Turning to our financial results, as June 30, 2022, our cash equivalent and marketable securities totaled approximately $47.5 million. We expect our cash runway to extend to the fourth quarter of 2023. Research and development expenses for the second quarter of 2022 totaled $4.7 million compared to $4.2 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 within our pipeline. General and administrative expenses for the second quarter of both 2021 and 2022 totaled $2.5 million. These amounts reflect personnel related and ongoing general business operating costs. Net loss for the second quarter of 2022 was $7.3 million compared to net loss of $6 million for the same period in 2021. Basic and deluded net loss per share for the second quarter of 2022 was $0.50 per share compared to basic and deluded net loss of $0.78 per share for the same period in 2021. With that, I will turn the call back over to Jay. Thanks, Chris. At this time, we're happy to take any questions you might have. Operator, could you please open the line? Thank you. We will now begin the question and answer session. To ask a question, you may press star then one on your touch-turn phone. If you're using a speaker phone, please pick up your handset before pressing the keys. To withdraw your question, please press star then two. Your first question comes from Yai Ken with HC WingWrite. Please go ahead. Thank you. This is Yai for HC WingWrite. Thank you for taking my question. Sorry if you mentioned on the call. So is there a plan for RG01, 012? No, that's not the, sorry, Seno-V has not going to, I mean, Seno-V didn't really achieve the success in the out pressing control trial. Is it shooting the hand-up Seno-V or regularist could do something about it? Yeah. Seno-V notified us, which we shared in our AK update, that they're looking for other opportunities for the compound without giving any specifics to us. But you may know that MIR21 is implicated in a number of different fibiotic 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 inter-analysis when 24 subjects randomized 2 to 1, so that's 16 on active and 8 on placebo, had completed one year of blinded treatment. And that's what the futility analysis was based upon. So do you know whether Seno-V will make a decision as to whether they will proceed with his mother education before the end of this year? We don't know the exact timing. There's standard diligence clauses and an agreement of this type where they have to use commercially reasonable efforts to work on the program. And regarding the SAV trial of 8429, we should have the pre-initiative data in the fourth quarter of this year, correct? Yes, yes. Well, we'll plan an update 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 it. Is the company considering any other pre-canico stage candidates? 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. Got it. Thank you. You're welcome. Thank you. Once again, if you wish to ask a question, please press star then one. We will now pause a moment to allow for any final questioners to register. There are no further questions at this time. I would now like to turn the conference back over to Jay Hagen for closing remarks. Well, thanks everyone for joining us today for this brief call. As I mentioned, the programs are all on track for 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. The conference is now concluded. Thank you for attending today's presentation. You may now disconnect. You