Regulus Therapeutics Q2 2024 Financial Results and Updates

SAN DIEGO, Aug. 8, 2024 -- Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on developing innovative medicines targeting microRNAs, reported its financial results for the second quarter ending June 30, 2024, and provided a corporate update.

Jay Hagan, CEO of Regulus, expressed satisfaction with data from the ongoing Phase 1b MAD study of RGLS8429 in treating autosomal dominant polycystic kidney disease (ADPKD). The data from the third cohort demonstrated continued improvements in both polycystin levels and height-adjusted total kidney volume (htTKV) after three months of treatment. This mirrored the results from the second cohort but with enhanced mechanistic activity in polycystin changes and a higher number of patients showing kidney volume reduction. This positive outcome has heightened the company's optimism about RGLS8429's potential as a treatment for ADPKD, a disease with significant unmet needs and limited treatment options. Regulus plans to meet with the FDA by the end of the year.

Regulus has advanced to the fourth cohort in the Phase 1b MAD study of RGLS8429, enrolling patients to receive 300 mg of the drug every other week for three months. Enrollment is progressing well, with topline data expected in early 2025. The third cohort showed no safety concerns, with a mechanistic dose response observed based on urinary PC1 and PC2 levels, and 70% of patients exhibited reductions in htTKV. Additional exploratory analyses indicated correlations between increases in PC1, PC2, and reductions in htTKV. Further data presented in July 2024 indicated that RGLS8429 does not adversely affect kidney function, supporting its potential for further clinical development.

Corporate highlights include the promotion of Rekha Garg, M.D., M.S., to Chief Medical Officer. Dr. Garg brings extensive experience in kidney disease and other therapeutic areas from her roles at Sanifit Therapeutics, Infinity Pharmaceuticals, Amgen, and Eli Lilly. Additionally, Regulus has joined the Russell 3000® and Russell 2000® Indexes, ensuring its membership for one year and inclusion in the large-cap Russell 1000® Index or small-cap Russell 2000® Index, alongside the appropriate Growth and Value Style indexes.

Financially, Regulus ended the second quarter of 2024 with $95.9 million in cash, cash equivalents, and investments, providing a cash runway into the first half of 2026. Research and development expenses were $8.3 million for the quarter and $14.3 million for the first half of 2024, reflecting investments in advancing the clinical and preclinical pipeline. General and administrative expenses rose to $4.0 million for the quarter and $6.7 million for the first half of the year, due to personnel-related and general business operation costs. The net loss for the quarter was $11.0 million, or $0.17 per share, and $19.5 million, or $0.41 per share, for the first half of 2024.

ADPKD is a common genetic disorder caused by mutations in the PKD1 or PKD2 genes, leading to the development of multiple cysts in the kidneys and other organs, which can result in end-stage renal disease. Approximately 160,000 individuals in the United States are diagnosed with ADPKD, with a global prevalence estimated between 4 to 7 million.

RGLS8429 is a next-generation oligonucleotide designed to inhibit miR-17 and target the kidney, showing improvements in kidney function and size in preclinical models. Following the positive results from the Phase 1 SAD study completed in September 2022 and subsequent cohorts in the Phase 1b MAD study, Regulus plans to discuss the potential for a pivotal Phase 2/3 study with the FDA under an Accelerated Approval pathway later this year.

Regulus Therapeutics Inc. continues to leverage its expertise in oligonucleotide drug discovery and development to advance its pipeline, supported by a rich intellectual property estate in the microRNA field. The company is headquartered in San Diego, CA.

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