The Company is also planning to initiate a Phase 1b short-term dosing study in patients with ADPKD in the second half of 2020 to evaluate RGLS4326 for safety, pharmacokinetics, and biomarkers of pharmacodynamic activity. There-initiation of the MAD study was allowed following a satisfactory complete response provided to FDA in November 2019 to address the partial clinical hold placed on the clinical program in July 2019. A partial clinical hold on studies of RGLS4326 dosed for extended durations remains in effect until the second set of requirements outlined by FDA have been satisfactorily addressed. Information from the Phase 1 clinical studies, together with information from additional nonclinical studies, will be used to address the second set of requirements to support studies of extended duration.
Financial Results
Cash Position: As of December 31, 2019, Regulus had $34.1 million in cash and cash equivalents.
Research and Development (R&D) Expenses:R&D expenses were $2.1 million and $12.3 million for the quarter and year ended December 31, 2019, respectively, compared to $5.3 million and $34.0 million for the same periods in 2018. The decreases were largely driven by decreases in external development expenses, primarily attributable to the pause of the RGLS4326 MAD study in the third quarter of 2018 and transfer of theRG-012 program to Sanofi beginning in the fourth quarter of 2018. Additionally, the decreases were driven by reductions in personnel and internal expenses, primarily attributable to a reduction in costs subsequent to our corporate restructuring in the third quarter of 2018.
General and Administrative (G&A) Expenses:G&A expenses were $2.4 million and $11.3 million for the quarter and year ended December 31, 2019, respectively, compared to $2.7 million and $12.9 million for the same periods in 2018. The decreases were mostly driven by a reduction in costs subsequent to our corporate restructuring in the third quarter of 2018.
Revenue:Revenue was less than $0.1 million and $6.8 million for the quarter and year ended December 31, 2019, respectively, compared to less than $0.1 million and $0.1 million for the same periods in 2018. The increase for the year ended December 31, 2019 was attributable to revenue recognition of the upfront payments received under the 2018 Sanofi Amendment related to the transfer of theRG-012 program to Sanofi.
Net Loss:Net loss was $4.9 million and $18.6 million for the quarter and year ended December 31, 2019, respectively, compared to a net loss of $8.6 million and $48.7 million for the same periods in 2018. Basic and diluted net loss per share was $0.23 and $1.08 for the quarter and year ended December 31, 2019, respectively, compared to $0.98 and $5.59 for the same periods in 2018.
About ADPKD
ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause ofend-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 toend-stage renal disease in approximately 50% of ADPKD patients by age 60.
About RGLS4326
RGLS4326 is a novel oligonucleotide designed to inhibitmiR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of PKD1 and PKD2 in human ADPKD cyst cells, a reduction in kidney cyst formation, improved kidney weight/body weight ratio, decreased cyst cell proliferation, and preserved kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a Partial Clinical Hold for treatment of extended duration by the U.S. Food and Drug Administration.
2
