Last $41.52 USD
Change Today +0.85 / 2.09%
Volume 110.7K
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As of 5:20 PM 07/29/14 All times are local (Market data is delayed by at least 15 minutes).

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ultragenyx pharmaceutical in (RARE) Details

Ultragenyx Pharmaceutical Inc., a development-stage biotechnology company, focuses on the identification, acquisition, development, and commercialization of various products for the treatment of rare and ultra-rare diseases in the United States. It develops various biologics product candidates, including KRN23, a human monoclonal antibody in Phase 1/2 clinical trial to bind and reduce the biological activity of fibroblast growth factor to enhance abnormally low phosphate levels in patients with X-linked hypophosphatemia; recombinant human beta-glucuronidase, an intravenous enzyme replacement therapy in Phase 1/2 clinical trial for the treatment of mucopolysaccharidosis 7; and recombinant human protective protein cathepsin-A, an enzyme replacement therapy in preclinical development for galactosialidosis. The company is also developing a range of small-molecule product candidates comprising triheptanoin, a substrate replacement therapy in a Phase 2 clinical trial for patients with fatty acid oxidation disorders, as well as for patients with glucose transporter type-1 deficiency syndrome; and oral formulation of sialic acid in a Phase 2 extension study to treat hereditary inclusion body myopathy. It has license agreements with AAI Pharma Services Corp. and HIBM Research Group; and a collaboration and license agreement with Nobelpharma Co., Ltd. The company was founded in 2010 and is headquartered in Novato, California.

69 Employees
Last Reported Date: 05/12/14
Founded in 2010

ultragenyx pharmaceutical in (RARE) Top Compensated Officers

Chief Executive Officer, President and Direct...
Total Annual Compensation: $306.0K
Chief Financial Officer and Senior Vice Presi...
Total Annual Compensation: $308.4K
Chief Business Officer and Senior Vice Presid...
Total Annual Compensation: $289.4K
Compensation as of Fiscal Year 2013.

ultragenyx pharmaceutical in (RARE) Key Developments

Ultragenyx Pharmaceutical Inc. Appoints Sunil Agarwal as Chief Medical Officer and Senior Vice President, Effective August 25, 2014

Ultragenyx Pharmaceutical Inc. announced appointment of Sunil Agarwal, M.D. as its Chief Medical Officer and Senior Vice President, effective August 25, 2014. In this role, Dr. Agarwal will provide strategic leadership and direction to Clinical Operations, Clinical Development, Regulatory Affairs, Biometrics, Pharmacovigilance, and Non-Clinical Research. He will report to Emil D. Kakkis, M.D., Ph.D., Ultragenyx's CEO, and will serve on the Senior Management Team. Dr. Agarwal most recently served as Senior Vice President and Global Head of Clinical Development for Immunology and Infectious Diseases, Metabolism, Neuroscience, and Ophthalmology at Genentech. Dr. Agarwal will join the senior medical team at Ultragenyx which includes Dr. Kakkis and Dr. Javier San Martin, Vice President, Clinical Development, a key leader for the KRN23 development program for X-linked hypophosphatemia (XLH). Prior to Ultragenyx, Dr. San Martin worked at Amgen as the Global Development Leader for the sclerostin antibody program and Prolia (denosumab) and at Eli Lilly as medical advisor on the Forteo (teriparatide) Product Team.

Ultragenyx Announces Initiation of a Phase 2 Study of KRN23 for Pediatric X-Linked Hypophosphatemia in the US and EU

Ultragenyx Pharmaceutical Inc. announced the first patient screened and enrolled in the Phase 2 study of the human monoclonal anti-FGF23 antibody KRN23 (UX023) in pediatric patients with X-linked hypophosphatemia (XLH). XLH is an inherited metabolic bone disease characterized by short stature, skeletal deformities, bone pain, fractures, and muscle weakness. The multi-center, randomized, open-label, dose-finding Phase 2 clinical study will evaluate safety and efficacy in approximately 30 prepubertal pediatric XLH patients. The primary objectives of the study are to identify a dose and dosing regimen and to establish the safety profile of treatment with KRN23 in pediatric XLH patients. Preliminary clinical effects of KRN23 treatment on bone health and deformity as measured by radiographic assessments, growth, muscle strength, and motor function will also be assessed, as well as markers of bone health and patient-reported outcomes of pain, disability, and quality of life. The study has been evaluated and accepted for conduct by the United States Food and Drug Administration (FDA), the United Kingdom Medicinal and Health Regulatory Authority (MHRA), and the Dutch Medicines Evaluation Board (CBG-MEB). The study will consist of a 16-week individual dose-titration period followed by a 48-week treatment period. The goal of the dose-titration period is to identify the individualized dose of KRN23 required to achieve stable serum phosphorus levels in the target range. Patients will be divided into three cohorts of escalating starting dose levels of KRN23 with either monthly or biweekly dosing regimens. At the end of the 16-week dose-titration period, patients will receive their individually-optimized dose of KRN23 on a monthly or biweekly basis for the 48-week treatment period. An interim analysis of safety and pharmacodynamic data will be conducted after 24 weeks of the treatment period.

Ultragenyx Pharmaceutical Mulls Acquisitions

Ultragenyx Pharmaceutical Inc. (NasdaqGS:RARE) is looking for acquisitions. Ultragenyx Pharmaceutical Inc. has filed a follow-on equity offering in the amount of $100 million and will use the proceeds to in-license, acquire, or invest in additional businesses, technologies, products, or assets, among others.


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