Regulus Therapeutics Announces Q3 2024 Financial Results and Updates

Regulus Therapeutics Inc., a biopharmaceutical entity targeting microRNAs, reported significant progress and financial results for the third quarter ending September 30, 2024. The company has been focused on the development of RGLS8429, which is intended to treat autosomal dominant polycystic kidney disease (ADPKD).

Key developments include the completion of patient enrollment in the fourth cohort of the Phase 1b multiple-ascending dose (MAD) clinical trial of RGLS8429. Patients in this cohort are administered a 300 mg fixed dose of RGLS8429 every other week over three months. Regulus aims to present preliminary topline data from this group in early 2025. This follows earlier positive results from the first three cohorts, which demonstrated a mechanistic dose response and reductions in height-adjusted total kidney volume (htTKV), crucial metrics for evaluating the drug's effectiveness.

The company also presented data affiliated with RGLS8429 at two significant meetings. At the American Society of Nephrology (ASN) Kidney Week, a poster highlighted the clinical proof of RGLS8429’s dose-responsive activity on urinary polycystins PC1 and PC2. Additionally, at the Oligonucleotide Therapeutics Society Annual Meeting, Regulus shared insights on the pathway to developing RGLS8429, emphasizing its transition from an earlier molecule, RGLS4326, due to observed central nervous system-related toxicity. The new generation molecule, RGLS8429, has shown no such off-target interactions while maintaining its potency against miR-17.

Regulus also announced a Patent & Technology License Agreement with The University of Texas System, granting exclusive rights to Regulus for products targeting miR-17 for ADPKD treatment. This agreement involves an upfront payment plus reimbursement of certain expenses, along with specified milestone payments based on clinical and commercial achievements.

Financially, Regulus ended the third quarter of 2024 with $87.3 million in cash, cash equivalents, and investments, providing a cash runway into the first half of 2026. Research and development expenses for the three and nine months ended September 30, 2024, increased significantly compared to the same periods in 2023, reflecting accelerated efforts in clinical and preclinical advancements. General and administrative expenses also rose, indicating increased operational activities. Consequently, the net loss for the three and nine months ending September 30, 2024, was notably higher compared to the previous year.

ADPKD is caused by mutations in the PKD1 or PKD2 genes and leads to the development of multiple fluid-filled cysts primarily in the kidneys, progressing to end-stage renal disease in many patients by age 60. The disease affects around 160,000 individuals in the U.S., with a global prevalence estimated between 4 to 7 million.

RGLS8429, specifically designed to inhibit miR-17, is aimed at targeting the kidneys preferentially. Preclinical models have shown that RGLS8429 improves kidney function and reduces disease severity markers. The Phase 1 single ascending dose (SAD) study for RGLS8429, completed in September 2022, demonstrated favorable safety and pharmacokinetic profiles. No serious adverse events were reported, and the drug exposure was linear across the tested doses.

Overall, Regulus Therapeutics is optimistic about the future prospects of RGLS8429 as it continues to advance through clinical trials, aiming to provide a much-needed therapeutic option for ADPKD patients.