Ultragenyx Announces Three Abstracts Accepted for Poster Presentation at 18th Annual World Muscle Society Congress

Ultragenyx Announces Three Abstracts Accepted for Poster Presentation at 18th Annual World Muscle Society Congress

NOVATO, Calif., Sep 26, 2013 (GLOBE NEWSWIRE via COMTEX) — Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that three abstracts related to UX001 Sialic Acid for Hereditary Inclusion Body Myopathy, also known as GNE Myopathy, were accepted for poster presentation at the 18th Annual World Muscle Society Congress, October 1-5, 2013 in Asilomar, California.

Ultragenyx selected as 2013 Fierce 15 Company

FierceBiotech selected Ultragenyx Pharmaceutical as one of the 2013 Fierce 15 Companies.

What makes Ultragenyx fierce: The most recent addition to the pipeline is KRN23, an antibody designed to treat rare cases of X-linked hypophosphatemia that is now wrapping a Phase I/II study at Kyowa Hakko Kirin. At the time KRN23 showed up on Ultragenyx’s radar, it was the only rare-disease program at the Japanese pharma company, says CEO Emil Kakkis. When the deal was done just days ago, it was Ultragenyx’s fourth midstage therapy.

Ultragenyx Announces Positive Data from Retrospective Study of UX007 Triheptanoin in Patients with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

 
Treatment with triheptanoin appears to reduce the frequency and length of hospitalizations associated with LC-FAOD disease.

NOVATO, CA –September 16, 2013—Ultragenyx Pharmaceutical Inc., a biotechnology company, announced the release of positive data from a retrospective protocol-driven medical record review oftriheptanoin treatment of patients with long-chain fatty acid oxidation disorders(LC-FAOD). LC-FAODpatients have severe morbidity and mortality with frequent complications and hospitalizations. The study evaluated the impact of triheptanoin treatment on the rate and extent of hospitalizationsin 20 of 24 patients who have been treated with triheptanoin for up to 13 years as part of a compassionate use protocol and consented to be part of the retrospective study. The study compared the incident rate for the major medical events before and after triheptanoin treatment, including the total number of hospitalizations and hospital days per year due to all causes,muscle rupture (rhabdomyolysis), hypoglycemia, or cardiomyopathy.

Ultragenyx Announces Collaboration with Kyowa Hakko Kirin to Develop and Commercialize Phase 2-stage KRN23 for X-linked Hypophosphatemia

 
Exclusive partnership will advance global clinical development of
KRN23, an antibody for a rare bone disease

Novato, CA – September 3, 2013–Ultragenyx Pharmaceutical Inc.today announced it has entered into a collaboration and license agreement with Kyowa Hakko Kirin Co., Ltd. (KHK),to develop and commercialize KRN23. KRN23 is a recombinant fully human monoclonal IgG1 antibody intended to treat X-linked hypophosphatemia (XLH). KHK is currently completing a Phase 1/2 study in adults with XLH in the US and Canada. The two companies plan to initiate a pediatric XLH program in 2014.

Ultragenyx Receives Approval of Clinical Trial Application (CTA) for a Phase 1/2 Trial TestingUX003 in Mucopolysaccharidosis Type 7 (MPS 7)

 
Ultragenyx Receives Approval of Clinical Trial Application (CTA)
for a Phase 1/2 Trial TestingUX003 in Mucopolysaccharidosis Type 7 (MPS 7)

NOVATO, CA – August 14, 2013 – Ultragenyx Pharmaceutical Inc., a biotechnology company, received Clinical Trial Application (CTA) approval from the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. to conduct a Phase 1/2 clinical trial of UX003, recombinant human β-glucuronidase, in MPS 7, a rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the enzyme β-glucuronidase. MPS 7 is a severe multi-system disease resulting in cellular and organ dysfunction. There is no approved drug therapy.

Ultragenyx Initiates New Development Program Studying Triheptanoin (UX007) for the Treatment of Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)

 
Phase 2 Trial Initiation Planned for Year End

NOVATO, Calif., Aug 05, 2013 (GLOBE NEWSWIRE via COMTEX) — Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced its plan to develop triheptanoin (UX007) for the treatment of seizures in glucose transporter type-1 deficiency syndrome (Glut1 DS). Glut1 DS is a rare and severely debilitating disease caused by mutations in the SLC2A1 gene, which encodes for a protein called glucose transporter type-1 (Glut1). The disease is characterized by seizures, developmental delay and movement disorders. The Glut1 protein transports glucose from blood into the brain. Because glucose is the primary source of energy for the brain, this defect in the Glut1 transporter results in a chronic state of brain energy deficiency. The company is planning to initiate a Phase 2 trial studying triheptanoin in Glut1 DS by the end of 2013.

Ultragenyx Gains Worldwide Rights for Triheptanoin (UX007)

 
Option Exercised for Ex-North American Territories

Novato, CA –July 11, 2013–Ultragenyx announced that it has expanded its exclusive license from Baylor Research Institute (BRI)in Dallas, Texas,to develop and commercialize triheptanoin outside of North America. The global license includes rights to patents, patent applications and other intellectual property related to the composition and formulation of UX007 as well as its use in treating a number of diseases including fatty acid oxidation disorders (FAOD), the lead indication being developed by the company.

Ultragenyx Announces a Positive Signal in Interim Data from Phase 2 Study of UX001 in Hereditary Inclusion Body Myopathy

 
Study to continue to 48 weeks, followed by extension study testing higher dosage

Novato, CA—July 3, 2013—Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, announced interim 24-week data from a 48-week Phase 2 clinical study of UX001 in 47 patients with hereditary inclusion body myopathy (HIBM), a progressive muscle-wasting disease. The study compared treatment with a total daily dose of 6 grams or 3 grams of UX001 with placebo. UX001, an oral sialic acid extended-release (SA-ER) tablet, is designed to replace the deficient sialic acid substrate in patients with HIBM.

Ultragenyx Advances Clinical Development of UX003 for the Treatment of Mucopolysaccharidosis Type 7 (MPS 7)

 
NOVATO, CA – May 15, 2013 – Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced a Phase 1/2 study of UX003 for mucopolysaccharidosis type 7 (MPS 7, or Sly Syndrome). UX003 is a recombinant human β-glucuronidase intended as an enzyme replacement therapy (ERT) for the treatment of MPS 7, an extremely rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the lysosomal enzyme β-glucuronidase and a severe multi-system disease. MPS 7 has no approved therapies.

Ultragenyx Appoints Eric Yuen, MD as Chief Medical Officer and Senior Vice President

NOVATO, CA –May 1, 2013 – Ultragenyx Pharmaceutical Inc., a biotechnology company focused on the development of treatments for rare and ultra-rare genetic disorders, today announced it has appointed Eric Yuen, MD as its Chief Medical Officer and Senior Vice President.  In this new role, Dr. Yuen will provide leadership and direction to Clinical Operations, Clinical Affairs, Clinical Sciences, Data Management/Biometrics, and Drug Safety.  He will report to Dr. Emil Kakkis, CEO, and will serve on the Senior Management Team.

Dr. Yuen brings over 19 years of clinical development experience in academia and industry.   In 2004, Dr. Yuen joined Johnson & Johnson as Senior Director and Neurology Franchise Medical Leader.   During his tenure at J&J, he also held several executive positions including Vice President, Therapeutic Area Head, CNS in 2006 and Vice President, Scientific Licensing from 2008 to 2009.  Dr. Yuen joins Ultragenyx from Janssen Alzheimer Immunotherapy R&D, LLC, a J&J affiliate, where he was the Head of Clinical Development since 2009.  Prior to J&J, Dr. Yuen also served as Director of Clinical Research, Department of Clinical Neuroscience at the Merck Research Laboratories. 

“As a board-certified neurologist, Dr. Yuen’s addition to the management team at Ultragenyx will provide critical support in advancing our clinical programs,” said Dr. Kakkis.  “His clinical vision and proven leadership, as well as his extensive medical and research experience, will strengthen our clinical development efforts and support the progress of our product pipeline.”

Dr. Yuen commented, “I am excited about being a part of a dedicated team focused on developing novel treatments for rare disease patients who have no approved treatments and applying my experiences in clinical development and neuromuscular diseases to help solve the devastating medical problems of these metabolic genetic diseases.” 

Dr. Yuen received his undergraduate training at Stanford University and his M.D. from the University of Chicago, Pritzker School of Medicine.  He completed his residency in Neurology and post-doctoral fellowships at the University of California, San Francisco.  Before joining industry in 2000, Dr. Yuen was a faculty member at the University of Washington, serving as a clinical investigator, studying treatments for ALS, neuropathy and multiple sclerosis. 

About Ultragenyx

Ultragenyx is a privately held, development-stage biotechnology company committed to bringing to market life-transforming therapeutics for patients with rare and ultra-rare metabolic genetic diseases.  The company focuses on diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no effective treatments. 

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics.  Ultragenyx’ strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. 

For more information on Ultragenyx, please visit the company’s website at www.ultragenyx.com.