RGLS Regulus Therapeutics Inc.

0.74
+0.02  (+2%)
Previous Close 0.72
Open 0.73
52 Week Low 0.45
52 Week High 2.32
Market Cap $64,192,247
Shares 87,040,335
Float 73,050,362
Enterprise Value $28,186,543
Volume 317,789
Av. Daily Volume 849,542
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Upcoming Catalysts

Drug Stage Catalyst Date
RGLS4326
Autosomal dominant polycystic kidney disease (ADPKD)
Phase 1b
Phase 1b
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Drug Pipeline

Drug Stage Notes
RG-012 - HERA
Alport Syndrome
Phase 2
Phase 2
Phase 2 enrolling.

Latest News

  1. SAN DIEGO, Sept. 9, 2021 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced that Jay Hagan, President and Chief Executive Officer of Regulus, will present at the H.C. Wainwright 23rd Annual Global Investment Conference: An on-demand webcast presentation will be available on Monday, September 13, 2021 at 7:00 a.m. ET.

    A webcast of the presentation will be available under "Events and Presentations" through the investor relations section of the Company's website at www.regulusrx.com. A replay of the webcast will be archived for 30 days following the presentation date.

    About Regulus

    Regulus Therapeutics Inc. (NASDAQ:RGLS) is a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a pipeline complemented by a rich intellectual property estate in the microRNA field. Regulus maintains its corporate headquarters in San Diego, CA.

    Forward-Looking Statements

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company's expected cash runway and statements associated with the Company's RGLS4326 program, including the potential sufficiency of the data required to recommence clinical studies for extended duration dosing, the timing of the Company's interactions with FDA regarding the clinical hold, the expected timing for reporting topline data, and the timing and future occurrence of other preclinical and clinical activities.  Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Regulus' current expectations and involve assumptions that may never materialize or may prove to be incorrect.  Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and in the endeavor of building a business around such drugs, the risk that feedback from the FDA may be negative or may not be provided when we expect, risks associated with our need for additional capital. In addition, while Regulus expects the COVID-19 pandemic to adversely affect its business operations and financial results, the extent of the impact on Regulus' ability to achieve its preclinical and clinical development objectives and the value of and market for its common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the U.S. and in other countries, and the effectiveness of actions taken globally to contain and treat the disease.  These and other risks are described in additional detail in Regulus' filings with the Securities and Exchange Commission, including under the "Risk Factors" heading of Regulus most recently quarterly report on Form 10-Q.  All forward-looking statements contained in this press release speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/regulus-therapeutics-to-present-at-the-hc-wainwright-23rd-annual-global-investment-conference-301372990.html

    SOURCE Regulus Therapeutics Inc.

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  2. SAN DIEGO, Aug. 10, 2021 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today reported financial results for the second quarter ended June 30, 2021 and provided a corporate update. 

    "The team at Regulus accomplished a lot over the last several months, including the achievement of several major milestones in our ADPKD program, the completion of meeting preparations with FDA and extending our cash runway into Q4 of next year," commented Jay Hagan, CEO of Regulus. "In addition, we are pleased to have completed enrollment of the second cohort of our Phase 1b trial of RGLS4326 for ADPKD, for which we expect to report topline data in the next several months. Additionally, we look forward to obtaining FDA feedback on our approach for addressing the remaining partial clinical hold requirements through our meeting request."

    Program Updates

    RGLS4326 for ADPKD: In June 2021, the Company presented additional data from the first cohort of patients in its Phase 1b clinical trial of RGLS4326 for the treatment of ADPKD, as well as new preclinical data from relevant animal models of the disease, at PKD Connect 2021. The additional data presented at PKD Connect demonstrated clinical proof of mechanism by showing statistically significant increases in polycystin levels in ADPKD patients by targeting miR-17 in the kidneys. Levels of PC1 and PC2 have previously been shown to be inversely correlated with disease severity. RGLS4326 was well-tolerated with no serious adverse events.

    The Phase 1b study is an adaptive design, open-label, multiple dose study in up to three cohorts of patients with ADPKD evaluating the safety, pharmacokinetics, and effects on pharmacodynamic biomarkers of multiple doses of RGLS4326. In the first cohort of the study, nine patients were enrolled and received four doses of 1mg/kg of RGLS4326 administered every other week.

    The Company also presented new data from relevant preclinical models showing treatment with RGLS4326 resulted in increased gene and polycystin levels in vitro. Improvements in key disease markers including serum creatinine and BUN were demonstrated in the Pkd1(F/RC) mouse model that harbors a mutation in the Pkd1 gene equivalent to human ADPKD. Mutation to the Pkd1 gene is reported for 85% of diagnosed patients with ADPKD.

    In late July 2021, the Company completed enrollment of the second cohort of patients in its Phase 1b clinical study of RGLS4326. The patients in the second cohort are being administered 0.3mg/kg of RGLS4326 every other week for four doses. The dose of RGLS4326 for the third and final cohort will be chosen based on the results of this second cohort and enrollment is expected to commence thereafter. The Company expects to report topline data from the second cohort in the fourth quarter.

    The Company requested a Type A meeting with FDA to discuss the data supporting its approach to addressing the remaining partial clinical hold requirements. FDA typically responds to a sponsor's request with the meeting being scheduled within 30 days from the receipt of the meeting request.

    Corporate Highlights

    Raised $15.4 Million in Gross Proceeds Through its ATM Facility: A total of 12,007,546 shares were sold and settled for gross proceeds of $15.4 million at a weighted average price per share of $1.28 under the ATM facility during the three months ended June 30, 2021. The Company expects its existing cash will provide cash resources to fund planned activities into Q4 2022.

    Financial Results

    Cash Position: As of June 30, 2021, Regulus had $41.4 million in cash and cash equivalents.

    Research and Development (R&D) Expenses: Research and development expenses were $4.2 million and $7.5 million for the three and six months ended June 30, 2021, respectively, compared to $4.2 million and $7.4 million for the same periods in 2020, respectively. These amounts reflect internal and external costs associated with advancing our clinical and preclinical pipeline.

    General and Administrative (G&A) Expenses: General and administrative expenses were $2.5 million and $5.0 million for the three and six months ended June 30, 2021, respectively, compared to $2.3 million and $4.7 million for the same periods in 2020, respectively. These amounts reflect personnel-related and ongoing general business operating costs.

    Net Loss: Net loss was $6.0 million, or $0.08 per share (basic and diluted), and $12.0 million, or $0.16 per share (basic and diluted), for the three and six months ended June 30, 2021, compared to $6.9 million, or $0.23 per share (basic and diluted), and $12.9 million, or $0.48 per share (basic and diluted), for the same periods in 2020. 

    About ADPKD

    ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

    About RGLS4326

    RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of Pkd1 and Pkd2, reduction of cyst growth in human in vitro ADPKD models, and attenuation of cyst proliferation and improvement of kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a partial clinical hold for treatment of extended duration by FDA until the second set of requirements outlined by the agency have been satisfactorily addressed. The Company will use information from the Phase 1 clinical studies, including the first cohort of the Phase 1b together with information from the recently completed additional nonclinical studies generated in 2020, in its plan to address the second set of requirements outlined in the Partial Clinical Hold letter to support studies of extended duration. Regulus plans to discuss its approach to addressing the remaining Partial Clinical Hold requirements with FDA in the third quarter 2021.  RGLS4326 received orphan drug designation from FDA in July 2020.

    About Regulus

    Regulus Therapeutics Inc. (NASDAQ:RGLS) is a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a pipeline complemented by a rich intellectual property estate in the microRNA field. Regulus maintains its corporate headquarters in San Diego, CA. 

    Forward-Looking Statements

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company's expected cash runway and statements associated with the Company's RGLS4326 program, including the potential sufficiency of the data required to recommence clinical studies for extended duration dosing, the timing of the Company's interactions with FDA regarding the clinical hold, the expected timing for reporting topline data, and the timing and future occurrence of other preclinical and clinical activities.  Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Regulus' current expectations and involve assumptions that may never materialize or may prove to be incorrect.  Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and in the endeavor of building a business around such drugs, the risk that feedback from the FDA may be negative or may not be provided when we expect, risks associated with our need for additional capital. In addition, while Regulus expects the COVID-19 pandemic to adversely affect its business operations and financial results, the extent of the impact on Regulus' ability to achieve its preclinical and clinical development objectives and the value of and market for its common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the U.S. and in other countries, and the effectiveness of actions taken globally to contain and treat the disease.  These and other risks are described in additional detail in Regulus' filings with the Securities and Exchange Commission, including under the "Risk Factors" heading of Regulus most recently quarterly report on Form 10-Q.  All forward-looking statements contained in this press release speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

     

    Regulus Therapeutics Inc.



    Selected Financial Information

    Condensed Statement of Operations

    (In thousands, except share and per share data)







    Three months ended

    June 30,



    Six months ended

    June 30,





    2021



    2020



    2021



    2020







    Revenues:

























    Revenue under strategic alliances                              



    $

    -



    $

    -



    $

    -



    $

    6

    Operating expenses:

















    Research and development



    4,150



    4,242



    7,470



    7,360

    General and administrative



    2,488



    2,254



    4,967



    4,676

    Total operating expenses



    6,638



    6,496



    12,437



    12,036

    Loss from operations



    (6,638)



    (6,496)



    (12,437)



    (12,030)

    Other income (expense), net



    605



    (451)



    391



    (862)

    Loss before income taxes



    (6,033)



    (6,947)



    (12,046)



    (12,892)

    Income tax (expense) benefit





    (1)





    -



    (1)



    8

    Net loss



    $

    (6,034)



    $

    (6,947)



    $

    (12,047)



    $

    (12,884)



























    Net loss per share, basic and diluted



    $

    (0.08)



    $

    (0.23)



    $

    (0.16)



    $

    (0.48)

    Weighted average shares used to compute basic and diluted net loss per

         share:





    77,175,831





    29,801,974





    74,249,631





    26,933,173

     





    June 30,

    2021



    December 31,

    2020







    Cash and cash equivalents



    $

    41,440



    $

    31,087

    Total assets



    49,516



    37,604

    Term loan, less debt issuance costs



    4,662



    4,652

    Stockholders' equity





    36,828





    26,026

     

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/regulus-therapeutics-reports-second-quarter-2021-financial-results-and-recent-updates-301352493.html

    SOURCE Regulus Therapeutics Inc.

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  3. SAN DIEGO, Aug, 3, 2021 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced that it will report financial results and highlights for the quarter ended June 30, 2021 on Tuesday, August 10, 2021, after the U.S. financial markets close. 

    The Company will host a conference call and live audio webcast on Tuesday, August 10, 2021 at 5:00 p.m. Eastern Daylight Time to report its second quarter 2021 financial results and provide a corporate update.  To access the call, please dial (877) 257-8599 (domestic) or (970) 315-0459 (international) and refer to conference ID 2825907.  To access the telephone replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international), passcode ID 2825907.  The webcast and telephone replay will be archived on the Company's website at www.regulusrx.com following the call.

    Wedbush PacGrow Healthcare Conference

    Additionally, the Company announced that Jay Hagan, President and Chief Executive Officer, will participate in a panel at the 2021 Wedbush PacGrow Healthcare Conference on Wednesday, August 11, 2021, at 4:05 p.m. E.T.

    A live webcast of the panel will be available on the investor relations section of the Company's website at www.regulusrx.com. A replay of the webcast will be archived for 30 days following the presentation date.

    About Regulus

    Regulus Therapeutics Inc. (NASDAQ:RGLS) is a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a pipeline complemented by a rich intellectual property estate in the microRNA field. Regulus maintains its corporate headquarters in San Diego, CA. 

    Forward-Looking Statements

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the clinical activities concerning the RGLS4326 program, including the preliminary biomarker, pharmacokinetic and safety data resulting from the first cohort of patients from the ongoing clinical study, the sufficiency of the data required to recommence clinical studies for extended duration dosing, the timing of the Company's interactions with FDA regarding the clinical hold, the timing and of other preclinical and clinical activities and the Company's expected cash runway.  Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Regulus' current expectations and involve assumptions that may never materialize or may prove to be incorrect.  Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and in the endeavor of building a business around such drugs, and feedback from the FDA. In addition, while Regulus expects the COVID-19 pandemic to adversely affect its business operations and financial results, the extent of the impact on Regulus' ability to achieve its preclinical and clinical development objectives and the value of and market for its common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the U.S. and in other countries, and the effectiveness of actions taken globally to contain and treat the disease.   These and other risks are described in additional detail in Regulus' filings with the Securities and Exchange Commission.  All forward-looking statements contained in this press release speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/regulus-therapeutics-announces-timing-for-second-quarter-2021-financial-results-webcast-and-conference-call-301347375.html

    SOURCE Regulus Therapeutics Inc.

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  4. SAN DIEGO, June 25, 2021 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced the presentation of additional data from the first cohort of patients in the Company's Phase 1b clinical trial of RGLS4326 for the treatment of autosomal dominant polycystic kidney disease (ADPKD), as well as new preclinical data from relevant animal models of the disease. The E-poster presentation, titled "Preclinical Evaluation and Results from the First Cohort of Phase 1b Clinical Trial of RGLS4326 for the Treatment of Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD),"  is available to…

    SAN DIEGO, June 25, 2021 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced the presentation of additional data from the first cohort of patients in the Company's Phase 1b clinical trial of RGLS4326 for the treatment of autosomal dominant polycystic kidney disease (ADPKD), as well as new preclinical data from relevant animal models of the disease. The E-poster presentation, titled "Preclinical Evaluation and Results from the First Cohort of Phase 1b Clinical Trial of RGLS4326 for the Treatment of Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD),"  is available to attendees of the PKD Connect Conference 2021, on Friday, June 25, from 2-3 p.m. CDT.

    The poster presents results from the Company's ongoing Phase 1b study evaluating the safety, pharmacokinetics, and effects on pharmacodynamic biomarkers of multiple doses of RGLS4326 in patients with ADPKD. The data demonstrate clinical proof of mechanism by showing target engagement in the kidneys through a statistically significant increase in urinary biomarkers PC1 and PC2, validating miR-17 as a target for ADPKD treatment. Levels of PC1 and PC2 have previously been shown to be inversely correlated with disease severity. RGLS4326 was well-tolerated with no serious adverse events. The poster also describes new data from relevant preclinical models showing treatment with RGLS4326 results in increased gene and polycystin levels in vitro. In addition, improvements in key disease markers including serum creatinine and BUN were demonstrated in the Pkd1(F/RC) mouse model that harbors Pkd1 mutation equivalent to human ADPKD.

    "These exciting results lend further support to the therapeutic potential of RGLS4326 in ADPKD and also bring us a step closer to our mission to improve the lives of patients suffering from this disease," commented Jay Hagan, President and Chief Executive Officer of Regulus. "The data from the first cohort of patients in this clinical study suggest that further increases in polycystin levels may be achievable with longer term and, potentially, less frequent, dosing and reinforce our belief that RGLS4326 represents an innovative approach to the treatment of underlying genetic drivers of ADPKD."

    Clinical study details:

    RGLS4326 is currently being evaluated in a Phase 1b, multicenter, open-label, adaptive design dose-ranging study to evaluate its safety, tolerability, pharmacokinetics and pharmacodynamics in patients with ADPKD. In the first cohort of the Phase 1b study, nine patients were enrolled and received 4 doses of 1 mg/kg of RGLS4326 administered every other week.

    Clinical study results:

    • The data demonstrate clinical proof of mechanism by showing target engagement in the kidneys through statistically significant increases in urinary biomarkers, PC1 and PC2, at study completion compared to baseline.
    • In addition, one patient with pre-study levels uNGAL almost twice the upper limit of normal saw their uNGAL levels drop into the normal range during the course of the study.
    • Administration of RGLS4326 was well-tolerated with no serious adverse events.

    The study is continuing to enroll patients with ADPKD in additional cohorts to evaluate different doses of RGLS4326. The second cohort of patients are receiving a dose of 0.3mg/kg administered every other week and is nearing completion of enrollment. 

    Preclinical study details:

    Pkd1(RC/-) cells were treated with either vehicle, control or RGL4326 for 2 days prior to measuring changes in Pkd1 and Pkd2 genes, and for 3 days prior to measuring changes in PC1 and PC2 proteins. In addition, Pkd1(F/RC) mice were dosed with either a control or RGLS4326 for four doses prior to assessment of efficacy.

    Preclinical study results:

    • RGLS4326 treatment led to increased Pkd1 and Pkd2 gene expression (>100% increase) and increased levels of their encoded proteins PC1 and PC2 (~50% increase) in Pkd1(RC/-) cells in vitro.
    • RGLS4326 treatment demonstrated improvement in key efficacy parameters including kidney-weight-to-body-weight ratio (~75% decrease), serum creatinine (~50% decrease) and BUN (~60% decrease) in Pkd1(F/RC) mice compared to control.

    About RGLS4326 Phase 1b

    The Phase 1b is an adaptive design, open-label, multiple dose study in up to three cohorts of patients with ADPKD.  The study is designed to evaluate the safety, pharmacokinetics, and changes in levels of PC1 and PC2 in patients with ADPKD administered RGLS4326 every other week for a total of four doses.  To characterize the effect of RGLS4326 within each cohort, biomarker values at the end of study are compared to baseline values using a two-sided paired t-test. P-values less than 0.05 are considered statistically significant with no adjustment for multiplicity. The dose level for the first cohort is 1mg/kg of RGLS4326 and the dose level for the second cohort is 0.3mg/kg.  The third and final cohort will be dosed at a level to be determined based on the results of the first two cohorts. 

    For more information about the clinical trial design, please visit www.clinicaltrials.gov (NCT04536688).

    About RGLS4326

    RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of Pkd1 and Pkd2, reduction of cyst growth in human in vitro ADPKD models, and attenuation of cyst proliferation and improvement of kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a partial clinical hold for treatment of extended duration by FDA until the second set of requirements outlined by the agency have been satisfactorily addressed. The Company will use information from the Phase 1 clinical studies, including the first cohort of the Phase 1b study together with information from the recently completed additional nonclinical studies generated in 2020, in its plan to address the second set of requirements outlined in the partial clinical hold letter to support studies of extended duration. Regulus plans to discuss its approach to addressing the remaining partial clinical hold requirements with FDA in mid-2021.  RGLS4326 has received orphan drug designation from FDA in July 2020.

    About ADPKD

    ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

    About Regulus

    Regulus Therapeutics Inc. (NASDAQ:RGLS) is a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a pipeline complemented by a rich intellectual property estate in the microRNA field. Regulus maintains its corporate headquarters in San Diego, CA. 

    Forward-Looking Statements

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the clinical activities concerning the RGLS4326 program, including the preliminary biomarker, pharmacokinetic and safety data resulting from the first cohort of patients from the ongoing clinical study, the sufficiency of the data required to recommence clinical studies for extended duration dosing, the timing of the Company's interactions with FDA regarding the clinical hold and the timing and of other preclinical and clinical activities.  Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Regulus' current expectations and involve assumptions that may never materialize or may prove to be incorrect.  Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and in the endeavor of building a business around such drugs, and feedback from the FDA. In addition, while Regulus expects the COVID-19 pandemic to adversely affect its business operations and financial results, the extent of the impact on Regulus' ability to achieve its preclinical and clinical development objectives and the value of and market for its common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the U.S. and in other countries, and the effectiveness of actions taken globally to contain and treat the disease.   These and other risks are described in additional detail in Regulus' filings with the Securities and Exchange Commission.  All forward-looking statements contained in this press release speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

     

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/regulus-therapeutics-presents-additional-data-from-its-autosomal-dominant-polycystic-kidney-disease-adpkd-program-at-pkd-connect-2021-301320096.html

    SOURCE Regulus Therapeutics Inc.

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  5. SAN DIEGO, June 21, 2021 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced that it will present new preclinical data and additional clinical data from the first cohort of patients in the Company's Phase 1b clinical trial of RGLS4326 for the treatment of ADPKD at the PKD Connect Conference 2021, being held virtually Friday, June 25.

    Presentation Details:

    • Title: Preclinical Evaluation and Results from the First Cohort of Phase 1b Clinical Trial of RGLS4326 for the Treatment of Patients with Autosomal Dominant Polycystic Kidney Disease
    • Date: June 25, 2021, 2:00– 3:00 p.m. CDT
    • Free registration for the conference is available through June 23, 2021 at pkdcure.org/conference/

    "We are excited to present the latest data from our RGLS4326 program for the treatment of ADPKD," commented Jay Hagan, President and Chief Executive Officer of Regulus. "This year's conference is an opportune occasion to share with the PKD community our additional data that further validate miR-17 as a target for ADPKD. We look forward to an engaging and productive event."

    About RGLS4326 Phase 1b

    The Phase 1b is an adaptive design, open-label, multiple dose study in up to three cohorts of patients with ADPKD.  The study is designed to evaluate the safety, pharmacokinetics, and changes in levels of PC1 and PC2 in patients with ADPKD administered RGLS4326 every other week for a total of four doses.  To characterize the effect of RGLS4326 within each cohort, biomarker values at the end of study are compared to baseline values using a two-sided paired t-test. P-values less than 0.05 are considered statistically significant with no adjustment for multiplicity. The dose level for the first cohort is 1mg/kg of RGLS4326 and the dose level for the second cohort is 0.3mg/kg.  The third and final cohort will be dosed at a level to be determined based on the results of the first two cohorts.

    For more information about the clinical trial design, please visit www.clinicaltrials.gov (NCT04536688).

    About RGLS4326

    RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of Pkd1 and Pkd2, reduction of cyst growth in human in vitro ADPKD models, and attenuation of cyst proliferation and improvement of kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a partial clinical hold for treatment of extended duration by FDA until the second set of requirements outlined by the agency have been satisfactorily addressed. The Company will use information from the Phase 1 clinical studies, including the first cohort of the Phase 1b study together with information from the recently completed additional nonclinical studies generated in 2020, in its plan to address the second set of requirements outlined in the partial clinical hold letter to support studies of extended duration. Regulus plans to discuss its approach to addressing the remaining partial clinical hold requirements with FDA in mid-2021.  RGLS4326 has received orphan drug designation from FDA in July 2020.

    About ADPKD

    ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

    About Regulus

    Regulus Therapeutics Inc. (NASDAQ:RGLS) is a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a pipeline complemented by a rich intellectual property estate in the microRNA field. Regulus maintains its corporate headquarters in San Diego, CA. 

    Forward-Looking Statements

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the clinical activities concerning the RGLS4326 program, including the preliminary biomarker, pharmacokinetic and safety data resulting from the first cohort of patients from the ongoing clinical study, the sufficiency of the data required to recommence clinical studies for extended duration dosing, the timing of the Company's interactions with FDA regarding the clinical hold and the timing and of other preclinical and clinical activities.  Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Regulus' current expectations and involve assumptions that may never materialize or may prove to be incorrect.  Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and in the endeavor of building a business around such drugs, and feedback from the FDA. In addition, while Regulus expects the COVID-19 pandemic to adversely affect its business operations and financial results, the extent of the impact on Regulus' ability to achieve its preclinical and clinical development objectives and the value of and market for its common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the U.S. and in other countries, and the effectiveness of actions taken globally to contain and treat the disease.   These and other risks are described in additional detail in Regulus' filings with the Securities and Exchange Commission.  All forward-looking statements contained in this press release speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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    SOURCE Regulus Therapeutics Inc.

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