Regulus Therapeutics Announces Q1 2024 Financial Results and Updates

Regulus Therapeutics Inc., a biopharmaceutical firm committed to developing therapies targeting microRNAs, has provided a financial and corporate update for the first quarter of 2024. The San Diego-based company, listed on Nasdaq as RGLS, has been making significant progress in their research, particularly in the treatment of autosomal dominant polycystic kidney disease (ADPKD).

Jay Hagan, CEO of Regulus, announced the commencement of the fourth and final cohort in their Phase 1b multiple-ascending dose (MAD) study of RGLS8429. This study aims to explore the efficacy of RGLS8429 in treating ADPKD. The completion of a $100 million private placement in March 2024 has fortified the company's financial position, enabling them to focus on achieving important milestones, including the release of topline data from the third cohort expected by mid-2024.

Regulus completed enrollment for the third cohort of the RGLS8429 MAD study in January. Patients in this group were administered 3 mg/kg of RGLS8429 or a placebo biweekly for three months. The latest development involves the fourth cohort, initiated on May 6, where patients will receive a fixed dose of 300 mg of RGLS8429 every other week for three months.

In March 2024, Regulus reported encouraging topline data from the second cohort of the Phase 1b MAD study, where 14 patients received either 2 mg/kg of RGLS8429 or a placebo biweekly for three months. The results indicated that RGLS8429 was well tolerated with no significant safety concerns. The treatment demonstrated greater biological activity at 2 mg/kg, as shown by higher urinary polycystin levels compared to lower doses and the placebo. Imaging-based biomarkers revealed promising results, with notable reductions in height-adjusted total kidney volume (htTKV) and total kidney cyst volume (TKCV) observed in patients with the highest increases in PC1 and PC2. These findings suggest that targeting miR-17 may positively influence htTKV and TKCV in ADPKD patients. Consequently, Regulus has expanded the sample size for the fourth cohort to up to 30 patients to further investigate these effects.

From a financial standpoint, Regulus ended the first quarter of 2024 with $107.7 million in cash, cash equivalents, and investments, projecting this runway to extend into the first half of 2026. Research and development expenses for the first quarter were $6.0 million, compared to $4.9 million in the previous year, reflecting the costs associated with advancing their clinical and preclinical pipeline. General and administrative expenses also saw an increase to $2.8 million from $2.4 million over the same period, attributed to personnel-related and general business operating costs. The net loss for the first quarter stood at $8.5 million, or $0.29 per share, an improvement from the $7.1 million loss, or $0.42 per share, in the same period of 2023.

ADPKD is a common monogenic disorder caused by mutations in the PKD1 or PKD2 genes, leading to the formation of numerous fluid-filled cysts in the kidneys and sometimes in other organs like the liver. This condition often results in end-stage renal disease in about half of the affected individuals by age 60. In the United States alone, approximately 160,000 people are diagnosed with ADPKD, with an estimated global prevalence ranging from 4 to 7 million.

RGLS8429, Regulus' next-generation oligonucleotide, is designed to inhibit miR-17 and preferentially target the kidney. Preclinical models have shown that RGLS8429 significantly improves kidney function and reduces disease severity. The Phase 1 SAD study completed in September 2022 confirmed the safety and pharmacokinetic profile of RGLS8429, with no serious adverse events reported. The ongoing Phase 1b MAD study continues to build on these findings, with promising results from the first and second cohorts and the final cohort now underway.

Regulus Therapeutics remains focused on the discovery and development of innovative treatments, leveraging their expertise in oligonucleotide drug discovery to address the underlying genetic causes of diseases like ADPKD.