Product Candidates - Systemic Lupus Eyrthematosus

	Autoimmune Disease
	Riquent® for Lupus
	SSAO Inflammation Program

Product Candidates :: Riquent® for Lupus

Lead Candidate Riquent® (abetimus sodium) Progresses as Potential Therapy for Lupus

La Jolla Pharmaceutical's lead drug candidate, Riquent® (abetimus sodium), previously referred to as LJP 394, is currently being developed as a novel treatment for systemic lupus erythematosus (SLE). Lupus, which affects about one million people in the U.S. and Europe, is a chronic, potentially life-threatening autoimmune disorder in which patients' diseased B cells produce antibodies to double-stranded DNA (dsDNA). These antibodies are believed to cause lupus kidney disease that can lead to kidney failure, dialysis, kidney transplantation, and death.

Riquent was developed based on the Company's patented Tolerance Technology® and is comprised of a lupus disease specific epitope attached to a carrier platform. The family of molecules based on this technology are called Toleragens®. The design of Riquent is based on scientific evidence of the role of antibodies to dsDNA in lupus. Riquent was designed to suppress the production of antibodies to dsDNA in lupus patients without suppressing the normal function of the immune system.

Published studies of lupus patients indicate that a rise in the level of antibodies to dsDNA may be predictive of renal flares in lupus patients with renal involvement, and that reducing antibodies to dsDNA by treating with corticosteroids can prevent relapse. Furthermore, based on published data from our own Phase 2/3 and Phase 3 trials, a reduction in the levels of antibodies to dsDNA significantly correlated with a reduced risk of renal flare and improved health-related quality of life. Based on these same published data, a rise in antibodies to dsDNA significantly correlated with an increased risk of renal flare and no change or deterioration in health-related quality of life.

Orphan Drug Status

The FDA granted the Company orphan drug designation for Riquent for the treatment of lupus nephritis in 2000. The Orphan Drug Act potentially enables the Company to obtain research funding, tax credits for certain research expenses and a waiver of the application user fees. In addition, the Orphan Drug Act allows for seven years of exclusive marketing rights to a specific drug for a specific orphan indication. The European Commission granted the Company orphan medicinal product designation in the European Union for Riquent for the treatment of lupus nephritis in 2001. Orphan designation in Europe provides for 10 years of marketing exclusivity in the European Union and enables us to receive significant fee reductions for scientific advice from the Committee for Orphan Medicinal Products, marketing authorization and inspections.

FDA Fast Track Designation for Riquent

In 2005, the FDA granted the Company fast track designation for Riquent for the treatment of lupus renal disease. The FDA’s fast track program is designed to facilitate the development and to expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address an unmet medical need.

GMP Manufacturing Facility in Place

The Company has developed a proprietary, scalable manufacturing process for Riquent that meets current Good Manufacturing Practice quality standards and is capable of multi-kilogram production levels. Manufacturing capacity may be expanded by working with contract manufacturers.