In December 2024, Regulus met with the FDA for an End-of-Phase 1 meeting where they reached alignment on the acceptability of the program’s CMC, non-clinical and clinical pharmacology plans and key components of a
Phase 3 trial design as a single pivotal study. Key components agreed upon in the meeting include a single active dose and placebo administered every other week in a 2:1 randomization scheme, a 12-month htTKV
endpoint for potential Accelerated Approval and a 24-month eGFR endpoint for potential Full Approval and an acceptable safety database size.
Financial Results
Cash, Cash Equivalents and
Short-term Investments: As of December 31, 2024, Regulus had $75.8 million in cash, cash equivalents and short-term investments. The Company expects its cash runway to extend into early 2026.
Research and Development (R&D) Expenses: Research and development expenses were $9.7 million and $35.4 million for the fourth quarter and
year ended December 31, 2024, respectively, compared to $5.8 million and $21.2 million for the same periods in 2023, respectively.
General and Administrative (G&A) Expenses: General and administrative expenses were $4.1 million and $14.7 million for the fourth quarter
and year ended December 31, 2024, respectively, compared to $2.5 million and $10.0 million for the same periods in 2023, respectively.
Net Loss: Net loss was $12.8 million, or $0.20 per share (basic and diluted), and $46.4 million, or $0.82 per share (basic and diluted),
for the fourth quarter and year ended December 31, 2024, compared to $8.1 million, or $0.40 per share (basic and diluted), and $30.0 million, or $1.58 per share (basic and diluted), for the same periods in
2023.
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 Farabursen (RGLS8429)
Farabursen is a novel, next generation oligonucleotide for the treatment of ADPKD designed to inhibit miR-17 and to
preferentially target the kidney. Administration of farabursen has shown clear improvements in kidney function, size, and other measures of disease severity in preclinical models. Regulus announced completion of the Phase 1 SAD study
in September 2022. The Phase 1 SAD study demonstrated that farabursen has a favorable safety and PK profile. Farabursen was well-tolerated with no serious adverse events reported and plasma exposure was approximately linear across the four
doses tested. In the Phase 1b MAD study, Regulus announced topline data from the first cohort of patients in September 2023, from the second cohort of patients in March 2024, from the third cohort of patients in June 2024,
and from the first 14 patients from the fourth cohort in January 2025. Patients in the fourth cohort received an open-label 300 mg fixed dose of farabursen which was administered every other week for three months. Review of complete safety data
from all cohorts demonstrated that farabursen was well tolerated.
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 oligonucleotide drug discovery and development expertise to develop a pipeline complemented by a rich intellectual property estate in the microRNA field. Regulus maintains its corporate headquarters in San Diego, CA.