In October 2023, the Company announced that, after a review of all available safety data in cohort two, it
advanced to the third cohort in the Phase 1b MAD study. Shortly after, in November 2023, the Company announced dosing of the first patient in the third cohort. Patients in the third cohort are being dosed at 3mg/kg of RGLS8429 or placebo every other
week for three months.
Financial Results
Cash,
Cash Equivalents and Marketable Securities: As of September 30, 2023, the Company had $30.8 million in cash, cash equivalents and short-term investments.
Research and Development (R&D) Expenses: Research and development expenses were $5.5 million and $15.4 million for the three and nine
months ended September 30, 2023, respectively, compared to $5.3 million and $13.7 million for the same periods in 2022, respectively. These amounts reflect internal and external costs associated with advancing our pipeline.
General and Administrative (G&A) Expenses: General and administrative expenses were $2.6 million and $7.4 million for the three and nine
months ended September 30, 2023, respectively, compared to $2.3 million and $7.6 million for the same periods in 2022, respectively. These amounts reflect personnel-related and ongoing general business operating costs.
Net Loss: Net loss was $7.8 million, or $0.40 per share (basic and diluted), and $22.0 million, or $1.19 per share (basic and diluted),
for the three and nine months ended September 30, 2023, respectively, compared to $7.6 million, or $0.50 per share (basic and diluted), and $21.5 million, or $1.46 per share (basic and diluted), for the same
periods in 2022.
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, RGLS4326. Regulus
announced completion of the Phase 1 SAD study in September 2022. The Phase 1 SAD study demonstrated that RGLS8429 has a favorable safety and PK profile. RGLS8429 was well-tolerated with no serious adverse events reported and plasma exposure was
approximately linear across the four doses tested and is similar to the PK data from the first-generation compound. In the Phase 1b MAD study Regulus announced both top line data from the first cohort of patients, and completion of enrollment for
the second cohort of patients in September 2023. After review of all available safety data, Regulus has advanced to the third cohort where dosing has begun, and patients will receive 3 mg/kg of RGLS8429 or placebo every other week for three months.