SAN DIEGO, June 23, 2023 /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 Preston S. Klassen, M.D., President and Head of Research & Development, has been appointed to the Company's Board of Directors.

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"We are delighted to have Dr. Klassen join our board during this exciting time for Regulus, as we advance the clinical development of RGLS8429 for the treatment of ADPKD," said Stelios Papadopoulos, Ph.D., Chairman of the Board of Directors of Regulus. "His expertise in nephrology, along with his previous leadership and board experience in the industry, will make him an invaluable member, and we look forward to his contributions to Regulus and our science."

Preston Klassen, M.D., recently joined Regulus Therapeutics as President and Head of Research and Development. Dr. Klassen is a nephrologist by training who brings over 20 years of experience in pharmaceuticals, including positions in leadership, medical affairs, and research and development across multiple therapeutic areas. Most recently he served as the President and CEO of Metacrine. Prior to that, he was an Executive Vice President and Head of R&D at Arena Pharmaceuticals, CMO and President at SANIFIT, and Senior Vice President and Head of Global Development at Orexigen Therapeutics. Previously, while at Amgen, he held multiple leadership roles, including Executive Medical Director and Therapeutics Area Head for Nephrology. Dr. Klassen earned his M.D. from the University of Nebraska Medical Center and completed both his residency in internal medicine and fellowship in nephrology as well as his Master's in Health Sciences at Duke University Medical Center.

About ADPKD

Autosomal Dominant Polycystic Kidney Disease (ADPKD), caused by 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. Approximately 160,000 individuals are diagnosed with the disease in the United States alone, with an estimated global prevalence of 4 to 7 million.

About RGLS8429

RGLS8429 is a novel, next generation oligonucleotide for the treatment of ADPKD designed to inhibit miR-17 and to preferentially target the kidney. Administration of RGLS8429 has shown robust data in preclinical models, where clear improvements in kidney function, size, and other measures of disease severity have been demonstrated along with a superior pharmacologic profile in preclinical studies compared to Regulus' first-generation compound, RGLS4326. Regulus announced completion of the Phase 1 SAD study in September 2022.  The Phase 1 SAD study demonstrated that RGLS8429 has a favorable safety and PK profile.  RGLS8429 was well-tolerated with no serious adverse events reported and plasma exposure was approximately linear across the four doses tested and is similar to the PK data from the first-generation compound. In April, Regulus announced completion of enrollment for the first cohort of patients in the Phase 1b MAD study and with the recent review of all available safety data, is advancing to the second cohort where patients will receive 2 mg/kg of RGLS8429 or placebo every other week for three months.

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 presentation 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 Company's RGLS8429 program, including the expected timing for initiating clinical studies, the expected timing for reporting topline data, the safety of RGLS8429 potentially achieving therapeutic efficacy and clinical translation for ADPKD patients 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, the approach we are taking to discover and develop drugs is novel and may never lead to marketable products, preliminary or initial results may not be indicative of future results, preclinical and clinical studies may not be successful, risks related to regulatory review and approval, risks related to our reliance on third-party collaborators and other third parties, risks related to intellectual property,  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 additional toxicology data may be negative and the need for additional capital. 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 filed 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.

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