Q1 2022 Regulus Therapeutics Inc Earnings Call

Good day and welcome to the Regulus Therapeutics incorporated first quarter 2022 conference call.

Operator: Welcome to the Regulus Therapeutics Incorporated First Quarter 2022 conference call. All participants will be in a listen-only mode.

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Operator: 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 Cris Calsada, Chief Financial Officer. Please go ahead.

Please note this event is being recorded.

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

Thank you operator good.

Good afternoon, everyone and thank you for joining us to discuss Regulus Therapeutics first quarter 2022 financial results and corporate highlights John .

Cris 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. 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. However, actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors.

Joining me on today's call is Jay Hagan, President and Chief Executive Officer, and Doctor dentist drives and Chief Scientific Officer, Jay will provide opening remarks and share progress on our ADP could be another program.

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.

Cris Calsada: 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.

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.

Jay Hagan: I'll now turn the call over to Jane. Thanks, Chris. And welcome everyone to our first quarter earnings call and business update. I'll begin 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 8429 for the treatment of ADPKD. The study is a phase one single ascending dose study in healthy volunteers to assess safety, tolerability, and pharmacokinetics of RGLS8429.

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

Jay Hagan: 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 RGLS8429 treatment, as well as evaluate the dose response of RGLS8429 on ADPKD biomarkers, including polycystins, cystic kidney volume, and overall kidney function.

I'll begin first with a general update our ADP <unk> program.

We're proud to share with you that earlier this week the U S food and drug administration accepted our R&D for our G. L. S. Eight four to nine for the treatment of ADP J D.

The study is a single is a phase one single ascending dose study in healthy volunteers to assess safety Tolerability and pharmacokinetics of RG lessee for two nine.

Following this study we plan to initiate a phase one b multiple ascending study in adult patients with 80 P. J D to assess the safety Tolerability and pharmacokinetics of RG, let's say four to nine treatment as well as evaluate the dose response of RG, let's say four to nine on ADP Katie Biomarkers include.

Probably systems cystic kidney volume and overall kidney function.

Recall that our objective in this study is to establish a dose response around the dose levels were robust clinical biomarkers effects were demonstrated with our first generation compound RG list for three to six.

Jay Hagan: Recall that our objective in this study is to establish a dose response around the dose levels where robust clinical biomarker 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 Latimersin, our RG012 program for the treatment of Alport Syndrome.

We anticipate top line data from the healthy volunteer portion of the study in the second half of 2022 and topline biomarker data from the first cohort of <unk> 8429 treated patients in the first half of 2023.

Now I'd like to shift gears and share the latest update regarding latter medicine, our RG 012 program for the treatment of output syndrome.

In February we announced completion of enrollment by our partner Sanofi in the phase two HERA clinical study evaluating let a medicine for the treatment of adult patients with outboard syndrome under our collaboration and license agreement with Sanofi.

Jay Hagan: In February, we announced completion of enrollment by our partner Sanofi in the Phase 2 HERA clinical study evaluating ledimersin 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.

Final data is expected in the first half of 2023.

And if successful would further validate the potential of our platform technology.

Jay Hagan: And if it's successful, it 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 biological 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.

Vince 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.

Jay Hagan: 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 Chris for an update on our financial results.

In AOS under 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 weakened 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 Dysregulation microrna for other grievous CNS diseases.

With that I'll turn the call back over to Chris for an update on our financial results, Chris. Thank you J T.

Cris 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.

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 the Jay discussed earlier.

Cris Calsada: Research and development expenses for the first quarter of 2022 totaled $3.7 million, compared to $3.3 million in the same period of 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.

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 ADP <unk> program.

Other research efforts and our pipeline Jeff.

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.

Cris Calsada: Net loss for the first quarter of 2022 was $6.7 million compared to a 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 a basic and diluted net loss of $0.08 per share for the same period in 2021.

Basic and diluted net loss per share for the first quarter of 2022 was <unk> <unk> per share compared to basic and diluted net loss of eight cents per share for the same period in 2021.

With that I will turn the call back over to Jay.

Jay Hagan: With that, I will turn the call back over to Jane. Thanks, Chris. At this time, we're happy to take any questions. Operator, you can open the lines.

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

Okay.

We will now begin the question and answer session.

Operator: We will now begin the question and answer session. To ask a question, you may press star, then 1 on your touchtone phone. If you are using a speakerphone, please pick up your handset before pressing the keys.

To ask a question you May Press Star then one on your Touchtone phone.

If youre using a speakerphone please pick up your handset before pressing the keys.

If at any time. Your question has been addressed and you would like to withdraw your question. Please press Star then two.

Operator: If at any time your question has been addressed and you would like to withdraw your question, please press star, then two. At this time, we will pause momentarily to assemble our wrap. Once again, to ask a question, that is star, then one. The first question comes from Yi Chen with HC Wainwright, please go ahead.

At this time, we will pause momentarily to assemble Iraq.

Okay.

Yeah.

Once again to ask a question that is star then one.

The first question comes from <unk>, Chen with H C. Wainwright. Please go ahead.

Thank you for taking my question could you come the overall.

Yi Chen: Thank you for taking my question. Could you comment on the overall expenses associated with the upcoming trial for AD421? Sure.

Expenses associated with the upcoming.

Ah trial for 80 for 20 months.

Sure.

Jay Hagan: So, you know, our 84-9 program, since we have our history of 4-3-2-6, we'll start the SAD study, which will commence here in the second quarter with 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. And the cost associated with it, Yi, if that's where you're going, you know, they're very similar to the 4.326 cost, say, for the inflationary environment we're in, and then the longer, the Phase 1B study, you know, obviously we stopped the 4.326 study short by one cohort, saving some money for us, and now can redirect those into the 8.429 program.

84, <unk> nine program since we have a history for three to six well.

Start the <unk>.

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.

And the costs associated with it.

If that's where you're going.

Very similar to the $43 six cost say for inflationary environment. We're in and then the the longer the phase <unk> study.

Obviously, we stopped the for three to six study.

Short by one cohort saving some money for us and now can redirect those into the 849 program.

Jay Hagan: But we are doing a placebo-controlled arm in the 8.429 study. So instead of nine 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 of patients will be, you know, probably approximately 50% higher than the cost for the 4.326 study.

But we are doing.

A placebo controlled.

Arms in the in the April two nine studies, so instead of nine patients per cohort, we have <unk> 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 for the MA portion of patients will be.

Probably approximately 50% higher than the cost for the fourth of 206 study.

Okay.

Got it.

And will.

Jay Hagan: Okay. And will there be any additional milestones associated with the data readout from Alport Syndrome in the first half of 2023? The $25 million is the last and final milestone from Tel Aviv, and they're responsible for the royalties that we would have owed to IONIS and El Nino, per our founding agreement.

Will there be any additional milestone associated with the data.

Data readout from outsourcing Jerome and the first half of 2023.

The $25 million is the <unk>.

Last and final milestone from promoting healthy and they're responsible for the royalties that we would have one or two I honestly don't island.

Per our foundry agreement.

Okay. Thank you.

Jay Hagan: Okay, thank you. Thank you. Once again, if you would like to ask a question, please press star then 1 to join the queue. This concludes our question and answer session. I would like to turn the conference back over to Jay Hagan for any closing remarks. Thanks, Operator, and thanks, everyone, for joining us today for our brief update on our progress, and we appreciate your support of Regulus. We look forward to providing future updates. Thank you. The conference has now concluded. Thank you for attending today's presentation. You may now disconnect. BF-WATCH TV 2021

Yeah.

Thank you.

Once again, if you'd like to ask a question. Please press <unk>.

Then wanted to join the queue.

Yeah.

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

Thanks, operator, and thanks, everyone for joining us today on our brief update on our progress.

Get your support Regulus, we look forward to providing future updates. Thank you.

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

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