Kazia Therapeutics Limited American Depositary Shares
Kazia Therapeutics Limited, together with its subsidiaries, operates as an oncology-focused biotechnology company in South Korea. Its lead development candidate is paxalisib, a small molecule, brain-penetrant inhibitor of the phosphoinositide-3-kinase (PI3K)/AKT/mammalian target of rapamycin (mTOR) pathway, which is in Phase 3 clinical trial, for the treatment of glioblastoma, isocitrate dehydrogenase-mutant glioma, diffuse intrinsic pontine glioma, atypical teratoid/rhabdoid tumor, brain metastases, primary central nervous system (CNS) lymphoma, triple negative breast cancer, and ovarian cancer. The company also develops EVT801, a small-molecule selective inhibitor of vascular endothelial growth factor receptor 3, which is in Phase 1 clinical trial, to treat advanced solid tumors. It has collaborations with the Australian and New Zealand Children's Haematology/Oncology Group, OIMR Berghofer Medical Research Institute, Genentech Inc., Global Coalition for Adaptive Research, Vivesto AB, Simcere Pharmaceutical Group Ltd, Evotec SE, and Sovargen Co., Ltd. The company was formerly known as Novogen Limited and changed its name to Kazia Therapeutics Limited in November 2017. Kazia Therapeutics Limited was incorporated in 1994 and is based in Sydney, Australia.
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Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.
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