IND accepted and first subject dosed in Phase 1 single ascending dose (SAD) study of RGLS8429 for the treatment of autosomal dominant polycystic kidney disease (ADPKD)
RGLS8429 received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA)
Scientific leadership strengthened with the appointment of Amin KamelPh.D., as Vice President, Drug Metabolism and Pharmacokinetics (DMPK)
SAN DIEGO, August 11, 2022 /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, 2022 and provided a corporate update.
“This has been an exciting quarter, culminating in the achievement of multiple milestones. We continue to make steady progress with RGLS8429, demonstrated by the dosing of the first subject in our Phase 1 SAD study,” he commented. Jay Hagan, CEO of Regulus. “In addition, we are pleased to have Dr. Amin Kamel join Team Regulus at this crucial time. We are proud of the work our team is doing to advance our new therapeutic candidate for ADPKD patients, and we look forward to providing additional updates on progress in the coming quarters.”
Program updates
RGLS8429 for ADPKD: Early June 2022, the company announced dosing of the first subject in its Phase 1 SAD study of RGLS8429 for the treatment of ADPKD. The study will evaluate the safety, tolerability and pharmacokinetics of RGLS8429 in healthy volunteers. The company plans to subsequently start a Phase 1b multiple ascending dose (MAD) study to assess the safety, tolerability and pharmacokinetics of RGLS8429 in adult patients with ADPKD, and to evaluate the efficacy of RGLS8429 treatment at three different dose levels by measuring changes in polycystins, volume of the cystic kidney (htTKV) and the global kidney. function
In late June, the company announced that the FDA had granted ODD to RGLS8429 for the treatment of ADPKD. The FDA’s Office of Orphan Product Development grants orphan designation status to drugs and biological products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases, or conditions that affect fewer than 200,000 people to the United States. drug development for rare diseases and may provide several benefits to drug developers, including financial incentives to support clinical development and the potential for up to seven years of market exclusivity in the US following regulatory approval.
Corporate aspects
Expanded team: En June 2022the Company announced the appointment of Amin Kamel, Ph.D., as Vice President, DMPK. More recently, Dr. Kamel was the Scientific Director, DMPK at Takeda, where he also served as Scientific Advisor. Before Takeda, Dr. Kamel held positions at Biogen, Novartis and Pfizer.
Financial results
Cash, cash equivalents and marketable securities: from June 30, 2022had Regulus 47.5 million dollars in cash, cash equivalents and marketable securities.
Research and Development (R&D) Expenses: Research and development expenses were 4.7 million dollars i 8.4 million dollars during the past three and six months June 30, 2022respectively, compared to 4.2 million dollars i 7.5 million dollars for the same periods in 2021, respectively. These amounts reflect the internal and external costs associated with advancing our pipeline.
General and administrative expenses (G&A): General and administrative expenses were $2.5 millions and $5.4 million during the three and six months ended June 30, 2022respectively, compared to $2.5 millions and 5.0 million dollars for the same periods in 2021, respectively. These amounts reflect the company’s general operating and staff-related costs.
Net Loss: The net loss was 7.3 million dollarsor $0.50 per share (basic and diluted), i $14.0 millionor $0.96 per share (basic and diluted), for the three and six months ended June 30, 2022compared to 6.0 million dollarsor $0.78 per share (basic and diluted) and $12.0 million, or $1.62 per share (basic and diluted), for the same periods in 2021.
Conference and webcast information:
The company will host a conference call and live audio broadcast today at 5:00 pm Eastern Daylight Time to discuss its second quarter 2022 financial results and corporate update. To access the call, dial (866) 652-5200 (domestic) or (412) 317-6060 (international). To access the telephone replay of the call, dial (877) 344-7529 (domestic) or (412) 317-0088 (international) and refer to the incoming replay code 5578933. The webcast and telephone replay will be archived on the company website. at www.regulusrx.com following the call.
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 mainly in the kidneys and, to a lesser extent, in the liver and other organs. Excessive proliferation of renal cyst cells, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60 years. Approximately 160,000 people are diagnosed with the disease a 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 preferentially target the kidney. Administration of RGLS8429 has shown strong 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 the compound of first generation of Regulus. Regulus is currently conducting a phase 1 dose-escalation study in healthy volunteers to evaluate the safety, tolerability and pharmacokinetics of RGLS8429. In June 2022the FDA granted ODD for RGLS849 for the treatment of ADPKD.
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 expertise in oligonucleotide drug discovery and development to develop a pipeline complemented by a rich heritage of intellectual property 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, the expected time to commence a clinical phase 1. study, the expected timing of reporting of front-line data and the timing and future appearance of data relating to the Company’s preclinical programs. Because these statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by these forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “target,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based on Regulus’ current expectations and involve assumptions that may never materialize or may turn out to be incorrect. Actual results and the timing of events could differ materially from those anticipated in these forward-looking statements as a result of various risks and uncertainties, including, without limitation, risks associated with the process of discovering, developing and commercializing safe and secure medicines. effective for use as a human therapeutic and in the effort to build a business around these drugs, and the risk that additional toxicology data may be negative. 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 goals and the value and market of its common stock, 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 social and business closure requirements in the US and other countries, and the effectiveness of actions taken globally to contain and treat the disease. These and other risks are described in more detail in Regulus’ filings with the Securities and Exchange Commission, including under the heading “Risk Factors” in Regulus’ most recent quarterly report on Form 10-Q. All forward-looking statements contained in this press release speak only as of the date they are made. Regulus undertakes no obligation to update these statements to reflect events or circumstances occurring after the date they are made.
SOURCE Regulus Therapeutics Inc.
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