Item 8.01 Other Events.
On January 4, 2019, Regulus Therapeutics Inc. (the “Company”) announced the preliminary results of a planned interim data analysis from the new mouse chronic toxicity study of RGLS4326 in development for the treatment of Autosomal Dominant Polycystic Kidney Disease (“ADPKD”).
As previously reported by the Company in July 2018, and in consultation with the U.S. Food and Drug Administration (“FDA”), the Company voluntarily paused its ongoing Phase 1 Multiple Ascending Dose (“MAD”) study of RGLS4326 in healthy volunteers due to unexpected observations in the27-week mouse chronic toxicity study. The Company terminated that mouse study prematurely at week 14. The study was run in parallel to the Phase 1 program to enable initiation of the Phase 2 program in ADPKD patients upon completion of the Phase 1 MAD study. The observations from the mouse chronic toxicity study were unexpected given the favorable safety profile of RGLS4326 in previous7-weeknon-GLP and GLP toxicity studies in both mice andnon-human primates required for Phase 1 testing, which had no significant findings across similar dose levels and frequencies. Based upon the Company’s investigation and the results announced today, the Company believes the unexpected observations from the previously terminated study were likely a result of technical issues at the contract research organization (“CRO”).
In September 2018, the Company announced the initiation of a new27-week mouse chronic toxicity study, incorporating several changes intended to address the unexpected observations in the previous mouse chronic toxicity study. Certain key changes included the use of a different CRO to conduct the study and the use of a new batch of RGLS4326.
The planned interim analysis of this study after13-weeks of dosing has shown no adverse or other significant findings across the range of doses tested and is intended to supportre-initiation of the Phase 1 MAD study after consultation with FDA. RGLS4326 has also been generally well-tolerated in the Phase 1 Single Ascending Dose (“SAD”) and MAD studies in human subjects to date.
The Company plans to submit a comprehensive data package for RGLS4326 to FDA that will include the results from the planned13-week interim analysis of the ongoing repeat mouse chronic toxicity study, as well as results from additional investigations, analytical testing, additional data from the previously terminated mouse chronic toxicity study, data from the completed Phase 1 SAD study and data from the first cohort of the Phase 1 MAD study. The Company anticipates engagement with FDA in the coming weeks to discuss the resolution of the voluntary pause in human dosing and the plan to resume the Phase 1 MAD study.
Forward-Looking Statements
Statements contained in this report regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of the Company to undertake certain activities and accomplish certain goals (including with respect to development and other activities related to RGLS4326 and its ability to recommence human clinical trials), the projected timeline of clinical development activities, the anticipated engagement with FDA regarding the Company’s RGLS4326 program and the timing thereof, and expectations regarding future therapeutic and commercial potential of the Company’s business plans, technologies and intellectual property related to microRNA therapeutics and biomarkers being discovered and developed by the Company. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking
