XML 16 R6.htm IDEA: XBRL DOCUMENT v3.5.0.2
The Company and Basis of Presentation
9 Months Ended
Sep. 30, 2016
The Company and Basis of Presentation  
The Company and Basis of Presentation

 

1. The Company and Basis of Presentation

 

The Company is a pharmaceutical company focused on developing and commercializing oral therapies for the treatment of patients with elevated low density lipoprotein cholesterol (“LDL-C”). Bempedoic acid, the Company’s lead product candidate, is a first-in-class ATP Citrate Lyase (“ACL”) inhibitor that reduces cholesterol biosynthesis and lowers elevated levels of LDL-C by up-regulating the LDL receptor, but with reduced potential for muscle-related side effects. In June 2016, the Company provided a clinical development and regulatory update for bempedoic acid and the clinical program, known as Cholesterol Lowering via BEmpedoic Acid, an ACL-inhibiting Regimen (“CLEAR”). The CLEAR program has two major components: 1) the global pivotal Phase 3 LDL-C lowering program in patients with hypercholesterolemia who are on maximally tolerated background lipid-modifying therapy, including patients who are only able to tolerate less than the lowest approved daily starting dose of a statin (considered “statin intolerant”), and 2) the global cardiovascular outcomes trial (“CLEAR Outcomes”), in patients with hypercholesterolemia who are at high risk for cardiovascular disease and who can be considered statin intolerant.  In October 2016, the Company announced that the CLEAR LDL-C lowering program will include patients with hypercholesterolemia on maximally tolerated background lipid-modifying therapy, including on any statin at any dose, based on positive top-line results from the Phase 2 pharmacokinetics and pharmacodynamics study (1002-035) of bempedoic acid added to atorvastatin 80 mg and the Phase 1 pharmacokinetics study (1002-037) to assess the safety and tolerability of bempedoic acid and its effects on the pharmacokinetics of single doses of four high-dose statins, and the previously completed Phase 1 and Phase 2 studies. Prior to the end of 2016, the Company expects to initiate the three global pivotal Phase 3 CLEAR LDL-C lowering efficacy studies — 1002-046, 1002-047, and 1002-048—  and the CLEAR Outcomes study. The Company owns the exclusive worldwide rights to bempedoic acid.

 

The Company’s primary activities since incorporation have been conducting research and development activities, including nonclinical, preclinical and clinical testing, performing business and financial planning, recruiting personnel, and raising capital. Accordingly, the Company has not commenced principal operations and is subject to risks and uncertainties which include the need to research, develop, and clinically test potential therapeutic products; obtain regulatory approvals for its products and commercialize them, if approved; expand its management and scientific staff; and finance its operations with an ultimate goal of achieving profitable operations.

 

The Company has sustained operating losses since inception and expects such losses to continue over the foreseeable future. Management plans to continue to fund operations through public or private equity or debt financings or through other sources, which may include collaborations with third parties. If adequate funds are not available, the Company may not be able to continue the development of its current or future product candidates, or to commercialize its current or future product candidates, if approved.

 

Basis of Presentation

 

The accompanying condensed financial statements are unaudited and were prepared by the Company in accordance with generally accepted accounting principles in the United States of America (“GAAP”). In the opinion of management, the Company has made all adjustments, which include only normal recurring adjustments necessary for a fair statement of the Company’s financial position and results of operations for the interim periods presented. Certain information and disclosures normally included in the annual financial statements prepared in accordance with GAAP have been condensed or omitted. These condensed interim financial statements should be read in conjunction with the audited financial statements as of and for the year ended December 31, 2015, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2015. The results of operations for the interim periods are not necessarily indicative of the results to be expected for a full year, any other interim periods or any future year or period.