On November 1, 2016 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, reported financial results for the three and nine months ended September 30, 2016 and provided a summary of corporate highlights (Press release, Regulus, NOV 1, 2016, View Source [SID1234516322]).

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"Our focus in the third quarter included the advancement of the RG-012 development program and addressing the clinical hold for RG-101 while expanding the pre-clinical pipeline," said Paul Grint, M.D., President and Chief Executive Officer of Regulus. "We continue to be excited with the progress of our research programs, and look forward to sharing an update at our first R&D day on December 6th."

Financial Results

Revenue: Revenue was $0.2 million and $1.2 million for the three and nine months ended September 30, 2016, respectively, compared to $1.9 million and $9.9 million for the same periods in 2015. Revenue for the three and nine months ended September 30, 2016 and 2015 consisted of amortization of up-front payments from Regulus’ strategic alliances and collaborations. Revenue for the three and nine months ended September 30, 2015 included $0.9 million and $4.1 million, respectively, for research services under Regulus’ strategic alliances and collaborations. Preclinical milestones earned under Regulus’ strategic alliances and collaborations were $0.3 million and $3.2 million for the three and nine months ended September 30, 2015, respectively.

Research and Development (R&D) Expenses: R&D expenses were $14.6 million and $49.3 million for the three and nine months ended September 30, 2016, respectively, compared to $11.0 million and $43.6 million for the same periods in 2015. The increases in R&D expenses were primarily driven by the advancement of our clinical programs and increased investment in our preclinical pipeline.

General and Administrative (G&A) Expenses: G&A expenses were $4.8 million and $13.6 million for the three and nine months ended September 30, 2016, respectively, compared to $4.2 million and $13.7 million for the same periods in 2015.

Net Loss: Net loss was $19.5 million, or $0.37 per share, and $61.8 million, or $1.17 per share, for the three and nine months ended September 30, 2016, respectively, compared to a net loss of $13.0 million, or $0.25 per share, and $48.5 million, or $0.95 per share, for the same periods in 2015.

Cash Position: Cash, cash equivalents, and short-term investments were $91.7 million at September 30, 2016, compared with $108.0 million at June 30, 2016 and $115.3 million at December 31, 2015.

Recent Events

In October, Dr. Timothy Wright joined Regulus as its Chief R&D Officer.
In September, Regulus initiated the HERA study, an international randomized, double-blind, placebo-controlled, multi-center Phase 2 clinical trial designed to evaluate the safety, pharmacodynamics, pharmacokinetics, dose selection, and preliminary efficacy of weekly RG-012 injections in approximately 30 patients with Alport syndrome. In order to address study design comments from European regulators, a multiple-ascending dose (MAD) study in healthy volunteers will be implemented (4-week repeat dosing) prior to expanding to Alport patients. Regulus anticipates the MAD study will be completed in the first half of 2017. Based on predicted enrollment rates, Regulus anticipates interim results from HERA in the first half of 2018.
In July, as anticipated, Regulus received written communication from the U.S. Food and Drug Administration (FDA) outlining information required to resolve the clinical hold for its Investigational New Drug (IND) for RG-101, which was announced on June 27, 2016. Based on the completion of additional mechanistic pre-clinical studies, Regulus expects a response to its submission from the FDA in the first quarter of 2017.
Upcoming Events

On November 13, 2016, Regulus will present three posters at American Association for the Study of Liver Disease (AASLD) in Boston.
On November 15, 2016 at 4:30 pm Eastern Time, Regulus will present a corporate overview at the Stifel 2016 Healthcare Conference in New York.
On November 18 and 19, 2016, Regulus will present two posters at American Society of Nephrology (ASN) Kidney Week in Chicago.
On December 6, 2016, Regulus will host its first R&D Day.
On December 13, 2016, Regulus will participate in the 4th Annual Boston Healthcare Conference.
On December 14, 2016, Regulus will participate in the BMO Capital Markets Prescriptions for Success Healthcare Conference.