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Neuphoria Therapeutics Inc. Common Stock and Ultragenyx Pharmaceutical Inc.

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Neuphoria Therapeutics Inc. Common Stock

Neuphoria Therapeutics Inc., a clinical stage biopharmaceutical company, discovers and develops novel allosteric ion channel modulators for the treatment of central nervous system disorders in Australia. Its lead product candidate includes BNC210, a selective negative allosteric modulator of the a7 for the treatment of social anxiety disorder; and is phase 2b to treat post-traumatic stress disorder. The company also develops BNC101, a monoclonal antibody that has completed phase 1 clinical trial that targets cancer stem cells; and BNC105 for the treatment of cancer. In addition, it develops NextGen, which is in preclinical trial for the treatment of multiple central nervous system indications; and Kv3.1/3.2 agonist, which is in preclinical trial to treat potential cognitive deficits and negative symptoms/social withdrawal in schizophrenia and autism spectrum disorders. The company has a collaboration with Merck & Co., Inc. to develop a7 receptor positive allosteric modulators targeting cognitive dysfunction associated with alzheimer's disease and other central nervous system conditions. The company was formerly known as Bionomics Limited and changed its name Neuphoria Therapeutics Inc. in December 2024. Neuphoria Therapeutics Inc. was incorporated in 1996 and is based in Burlington, Massachusetts.

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Ultragenyx Pharmaceutical Inc.

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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