The safety and effectiveness of JUXTAPID have not been established in patients with hypercholesterolemia who do not have HoFH. The effect of JUXTAPID on cardiovascular morbidity and mortality has not been determined. The safety and effectiveness of JUXTAPID have not been established in pediatric patients.
Expanding the Clinical Development Program of JUXTAPID
During the fourth quarter of 2012, Aegerion initiated enrollment of Japanese subjects into a Phase I bridging study of the pharmacokinetic and pharmacodynamic properties of JUXTAPID. Following the outcome of this study, Aegerion plans to conduct a small therapeutic study of JUXTAPID in Japanese HoFH patients in support of a planned filing for marketing authorization in Japan.
As previously disclosed, the FDA has established a post-marketing requirement for Aegerion to conduct a juvenile toxicology study in rodents. The study will seek to ascertain the impact, if any, of JUXTAPID on growth and development prior to initiating a clinical study of JUXTAPID in pediatric patients. Following the completion of the nonclinical study, the company expects to begin a clinical trial in pediatric patients in 2014.Advancing Regulatory Activities for JUXTAPID During 2012, Aegerion submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA), requesting approval to market lomitapide as an adjunct to a low-fat diet and other lipid-lowering therapies, with or without apheresis, to reduce LDL-C, total cholesterol, apolipoprotein B, and triglycerides in adults with HoFH. Aegerion continues to anticipate a European Medicines Agency (EMA) decision in mid-2013. If the application is approved, the company will pursue reimbursement on a country by country basis and anticipates commencing commercial activity in Europe by the end of 2013. Financial Guidance Aegerion ended FY 2012 with approximately $78 million to $83 million in cash and cash equivalents. In addition, the Company also announced the following financial guidance:
- Aegerion expects global net revenues of $15 million to $25 million for FY 2013 with 250 to 300 patients on JUXTAPID therapy by year-end 2013.
- 18 months post approval of JUXTAPID in the EU, if approved, the Company expects to:
Webcast Aegerion will webcast its corporate presentation at the 31st Annual J.P. Morgan Healthcare Conference on Tuesday, January 8, 2013 at 2:00 p.m. PST (5:00 p.m. EST). A link to the live webcast will be available and may be accessed for up to 14 days following the conference in the "Investors" section of Aegerion's website, www.aegerion.com . Important Safety Information, including BOXED WARNING which states: WARNING: RISK OF HEPATOTOXICITY JUXTAPID can cause elevations in transaminases. In the JUXTAPID clinical trial, 10 (34%) of the 29 patients treated with JUXTAPID had at least one elevation in alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3x upper limit of normal (ULN). There were no concomitant clinically meaningful elevations of total bilirubin, international normalized ratio (INR), or alkaline phosphatase. JUXTAPID also increases hepatic fat, with or without concomitant increases in transaminases. The median absolute increase in hepatic fat was 6% after both 26 and 78 weeks of treatment, from 1% at baseline, measured by magnetic resonance spectroscopy. Hepatic steatosis associated with JUXTAPID treatment may be a risk factor for progressive liver disease, including steatohepatitis and cirrhosis.
- generate global net revenue at a $100 million annualized run rate; and
- achieve cash flow breakeven from operations.