la jolla pharmaceutical co (LJPC) Key Developments
La Jolla Pharmaceutical Company Receives Orphan Drug Designation for Two Novel Compounds for Fibrodysplasia Ossificans Progressiva
Aug 18 15
La Jolla Pharmaceutical Company announced that the U.S. Food and Drug Administration(FDA) Office of Orphan Products Development has granted La Jolla orphan drug designation for two novel compounds for fibrodysplasia ossificans progressiva (FOP). The compounds that received orphan drug designation are small-molecule kinase inhibitors designed to selectively block a specific member of the bone morphogenetic protein (BMP) type-I receptor family, ALK2. FOP is a rare genetic disorder where the body turns muscle into bone. FOP is caused by a genetic mutation in ALK2 that results in excessive signaling of this pathway. In early childhood, afflicted individuals develop soft tissue swellings that transform into bone. Development of such lesions is exacerbated by trauma, and surgical intervention leads to dramatic and explosive new bone growth.
La Jolla Pharmaceutical Company Announces Exclusive Research and License Agreement Covering Vanderbilt's Research Program and Intellectual Property Rights
Aug 18 15
La Jolla Pharmaceutical Company announced an exclusive, worldwide research and license agreement covering Vanderbilt's research program and intellectual property rights relating to small-molecule kinase inhibitors designed to selectively block specific members of the bone morphogenetic protein (BMP) type-I receptor family. The seven members of the BMP type-I receptor family, activin receptor-like kinase (ALK) 1-7, play critical roles in human development and physiology. In turn, the improper activation of these receptor pathways is responsible for a wide range of disease conditions. For example, fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder where the body turns muscle into bone, is caused by a genetic mutation in ALK2 that results in excessive signaling of this pathway. Members of the BMP type-I receptor family also are involved in other diseases such as acquired heterotopic ossification (formation of bone in soft tissue caused by injury or trauma), muscular dystrophies including Duchenne muscular dystrophy, anemia of chronic disease (decrease of red blood cells or hemoglobin from chronic infection, chronic immune activation and malignancy), cancer, cardiovascular diseases and inflammatory bowel disease.
La Jolla Pharmaceutical Company Announces FDA Acceptance of IND for LJPC-401
Aug 11 15
La Jolla Pharmaceutical announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s investigational new drug application (IND) for LJPC-401, La Jolla’s novel formulation of hepcidin. La Jolla expects to release preliminary results from a Phase 1 clinical trial of LJPC-401 by the end of 2015. Hepcidin is a naturally occurring regulator of iron absorption and distribution. By regulating the absorption and distribution of iron, hepcidin prevents excessive iron accumulation in tissues, such as the liver and heart, where it can cause significant damage and even result in death. La Jolla is developing LJPC-401 for the treatment of iron overload, which occurs as a result of diseases such as hereditary hemochromatosis (HH) and beta thalassemia. HH is a disease caused by a genetic deficiency in hepcidin that results in excessive iron accumulation. HH is the most common genetic disease in Caucasians and causes liver cirrhosis, liver cancer, heart disease and/or failure, dementia and diabetes. LJPC-401 has been shown to be effective in reducing serum iron in preclinical testing. Specifically, La Jolla has completed animal toxicology studies that demonstrated a dose-dependent reduction in serum iron levels in all species tested.
La Jolla Pharmaceutical Co. Reports Unaudited Consolidated Earnings Results for the Second Quarter and Six Months Ended June 30, 2015
Aug 7 15
La Jolla Pharmaceutical Co. reported unaudited consolidated earnings results for the second quarter and six months ended June 30, 2015. For the quarter, the company reported loss from operations of $10,658,000 and net loss and comprehensive loss of $10,650,000 or $0.70 per basic and diluted share against loss from operations of $4,286,000 and net loss and comprehensive loss of $4,284,000 or $0.53 per basic and diluted share reported for the same period a year ago. Net cash used for operating activities was $5.7 million against $2.0 million a year ago.
For the six months, the company reported loss from operations of $19,625,000 and net loss and comprehensive loss of $19,605,000 or $1.29 per basic and diluted share against loss from operations of $9,416,000 and net loss and comprehensive loss of $9,412,000 or $1.38 per basic and diluted share reported for the same period a year ago. Net cash used for operating activities was $11.2 million compared to net cash used for operating activities $4.7 million a year ago. The increases in net cash used for operating activities and net loss in 2015 as compared to 2014 were primarily due to increased clinical development costs associated with the initiation of the ATHOS 3 trial of LJPC-501 in CRH, the Phase 2b clinical trial of GCS-100 in advanced chronic kidney disease, the continuing Phase ½ clinical trial of LJPC-501 in hepatorenal syndrome and preclinical costs associated with LJPC-401.
La Jolla Pharmaceutical Company Announces License Agreements Covering LJPC-30Sa and LJPC-30Sb
Aug 5 15
La Jolla Pharmaceutical Company announced that it has signed two exclusive worldwide license agreements for the intellectual property rights covering La Jolla’s next-generation gentamicin derivatives, LJPC-30Sa and LJPC-30Sb. The first license agreement is with the Indiana University Research and Technology Corporation (IURTC), and the second is with the IURTC and the University of Alabama at Birmingham (UAB). The license agreements, which follow from the previously announced option agreements, cover the use of LJPC-30Sa and LJPC-30Sb as antimicrobial agents and for the potential treatment of rare genetic disorders. La Jolla plans to pursue a dual development strategy with its next-generation gentamicin derivative program.