On Fri., Oct. 7, the company announced it would sell common stock and warrants to selected institutional and other accredited investors for gross proceeds to the company of approximately $66 million. The financing is intended to fund the continued development of Riquent (abetimus sodium) in a Phase 3 trial. The transaction is expected to close in Dec.
The company said it plans to expand the Phase 3 trial of Riquent to countries in Europe and Asia. Prior to reactivating enrollment, the company expects to seek approval from the Food and Drug Administration, or FDA, for amendments to the current Special Protocol Assessment, or SPA. Several proposed amendments, which are detailed below, were discussed with the FDA in July 2005. It is estimated that it will take approximately one year to complete enrollment once enrollment is reactivated and then another year for the last patient enrolled to complete the study.
La Jolla Pharmaceutical said it is continuing regulatory discussions with the FDA. In May 2005, the company received Fast Track designation for Riquent, which is intended to facilitate the FDA's ability to respond to questions and to review additional data. In July 2005, the company met with the cardio-renal division of the FDA to discuss further refinements to the trial design including selection of the higher dose, addition of an interim analysis to measure antibody levels and addition of a potential interim analysis for efficacy.
Based on the discussions with the FDA, the company said it intends to propose several revisions to the current SPA, including designating the 900 mg per week as the highest dose to be used in the study. The company also expects to assess the dose response of the treatment with Riquent on antibodies to dsDNA by conducting an interim analysis in the Phase 3 study to evaluate the effect of higher doses on the reduction of these antibodies. The antibody assessment is currently expected to occur approximately one year after reinitiating patient enrollment. The FDA has indicated that no statistical penalty would be needed as a result of conducting this analysis.
The company also expects to be able to conduct an interim analysis for efficacy at a point in time when approximately 70 percent of the projected number of renal flares has been observed. This formal interim analysis, if conducted, would result in an adjustment in the measure of statistical significance for the primary endpoint required for approval. The company is working to complete its review of the protocol and expects to submit the amended protocol to the FDA in the next few months.
La Jolla Pharmaceutical said it has several key goals for the next twelve months. First, the company is focused on completing the recently announced financing in December 2005. Second, the company expects to restart enrollment in the United States for Phase 3 trial early in 2006, after a final review of the revised protocol by the FDA. After the review, the company expects to expand the study to Europe and Asia. Third, the company plans to file the MAA application for Europe in the first quarter of 2006, to receive feedback and questions from the EMEA in the third quarter of 2006, and finally, to obtain data on the ability of higher doses of Riquent to reduce further the levels of antibodies to dsDNA late next year.
"There continues to be a significant unmet need in the treatment of lupus and we remain dedicated to providing a potentially safer treatment for lupus patients," said Steven Engle, chairman and CEO of La Jolla Pharmaceutical. "We are working hard to complete the financing announced on October 7. We look forward to moving forward with the Riquent Phase 3 trial."
Stock in La Jolla Pharmaceuticals rose 4 cents to 74 cents a share in morning trading.