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Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) today announced that its licensee, Onyx Pharmaceuticals (Nasdaq: ONXX), received accelerated approval from the U.S. Food and Drug Administration (FDA) for Kyprolis™ (carfilzomib) for Injection, a proteasome inhibitor indicated for the treatment of patients with multiple myeloma who have received at least two prior therapies, including bortezomib and an immunomodulatory agent, and have demonstrated disease progression on or within 60 days of completion of the last therapy. The indication for Kyprolis is based on response rate. Currently, no data are available for Kyprolis that demonstrate an improvement in progression-free survival or overall survival.
Kyprolis is formulated with Ligand’s Captisol
®, which improves drug solubility and enables a reduced drug load. Ligand is entitled to receive a $600,000 milestone payment from Onyx Pharmaceuticals; Ligand also is required to make a payment of $3.5 million toward the former stockholders of CyDex Pharmaceuticals, which Ligand acquired in 2011.
“We are very pleased with the FDA’s decision and congratulate Onyx on this significant achievement,” said John Higgins, President and Chief Executive Officer of Ligand Pharmaceuticals. “Our Captisol license agreement with Onyx is a highly valuable asset for Ligand, and we look forward to the near-term launch of the product. The approval of Kyprolis further demonstrates the clinical advantages and commercial potential of Captisol.”
The approval of Kyprolis was based on the results of the Phase 2b 003-A1 study, a single-arm, multicenter clinical trial that enrolled 266 patients with multiple myeloma, who had received a median of five prior anti-myeloma regimens. The primary efficacy endpoint was overall response (ORR) and determined by an Independent Review Committee using the International Myeloma Working Group (IMWG) criteria. ORR was 22.9% and median response duration was 7.8 months.
Safety data were evaluated in 526 patients with relapsed and/or refractory multiple myeloma who received single-agent carfilzomib. There were 37 deaths on study, or 7% of patients. The most common causes of death, other than disease progression, were cardiac (5 patients), end-organ failure (4 patients), and infection (4 patients). Important warnings and precautions include cardiac arrest, congestive heart failure, myocardial ischemia; pulmonary hypertension, pulmonary complications, infusion reactions, infusion reactions, tumor lysis syndrome, thrombocytopenia, hepatic toxicity and embryo-fetal toxicity. The most common serious adverse reactions were pneumonia, acute renal failure, pyrexia, and congestive heart failure. The most common adverse reactions (incidence of 30% or greater) observed in clinical trials of patients with multiple myeloma were fatigue, anemia, nausea, thrombocytopenia, dyspnea, diarrhea, and pyrexia. Serious adverse reactions were reported in 45% of patients.