On August 11, 2022 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported financial results for the second quarter ended June 30, 2022 and provided a corporate update (Press release, Regulus, AUG 11, 2022, View Source [SID1234618179]).
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"This has been an exciting quarter, which culminated in the achievement of multiple milestones. We continue to make steady progress with RGLS8429, evidenced by the dosing of the first subject in our Phase 1 SAD study," commented Jay Hagan, CEO of Regulus. "Moreover, we are pleased to have Dr. Amin Kamel join the Regulus team at this pivotal moment. We are proud of the work our team is doing to advance our novel therapeutic candidate for ADPKD patients, and we look forward to providing additional updates on progress in the coming quarters."
Program Updates
RGLS8429 for ADPKD: In early June 2022, the Company announced the dosing of the first subject in its Phase 1 SAD study of RGLS8429 for the treatment of ADPKD. The study will assess the safety, tolerability, and pharmacokinetics of RGLS8429 in healthy volunteers. The Company subsequently plans to initiate a Phase 1b multiple ascending dose (MAD) study to assess safety, tolerability, and pharmacokinetics of RGLS8429 in adult patients with ADPKD, and evaluate the efficacy of RGLS8429 treatment across three different dose levels measuring changes in polycystins, cystic kidney volume (htTKV), and overall kidney function.
In late June, the Company announced that FDA had granted ODD to RGLS8429 for the treatment of ADPKD. The FDA’s Office of Orphan Products Development grants orphan designation status to 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 U.S. Orphan designation status is intended to facilitate 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 U.S. upon regulatory approval.
Corporate Highlights
Expanded Team: In June 2022, the Company announced the appointment of Amin Kamel, Ph.D., as Vice President, DMPK. Most recently, Dr. Kamel was the Scientific Director, DMPK at Takeda, where he also served as a scientific advisor. Prior to Takeda, Dr. Kamel held positions at Biogen, Novartis, and Pfizer.
Financial Results
Cash, Cash Equivalents and Marketable Securities: As of June 30, 2022, Regulus had $47.5 million in cash, cash equivalents and marketable securities.
Research and Development (R&D) Expenses: Research and development expenses were $4.7 million and $8.4 million for the three and six months ended June 30, 2022, respectively, compared to $4.2 million and $7.5 million for the same periods in 2021, respectively. These amounts reflect internal and external costs associated with advancing our pipeline.
General and Administrative (G&A) Expenses: General and administrative expenses were $2.5 million and $5.4 million for the three and six months ended June 30, 2022, respectively, compared to $2.5 million and $5.0 million for the same periods in 2021, respectively. These amounts reflect personnel-related and ongoing general business operating costs.
Net Loss: Net loss was $7.3 million, or $0.50 per share (basic and diluted), and $14.0 million, or $0.96 per share (basic and diluted), for the three and six months ended June 30, 2022, compared to $6.0 million, or $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 Call and Webcast Information:
The Company will host a conference call and live audio webcast today at 5:00 p.m. Eastern Daylight Time to discuss its second quarter 2022 financial results and corporate update. To access the call, please 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 entry replay code 5578933. The webcast and telephone replay will be archived on the Company’s 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 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. Regulus is currently conducting a Phase 1 single-ascending dose study in healthy volunteers to assess safety, tolerability, and pharmacokinetics of RGLS8429. In June 2022, FDA granted ODD for RGLS849 for the treatment of ADPKD.