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NOVATO, Calif., Jan. 7, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced a license agreement with Inserm Transfert SA and Institut du Cerveau et de la Moelle Epiniere (ICM) for intellectual property related to the treatment of Huntington's disease with triheptanoin (UX007).

"Huntington's is a severe and lethal rare genetic disease," commented Emil D. Kakkis, Ph.D., M.D., Chief Executive Officer and President of Ultragenyx. "Energy deficiency is believed to play a role in the pathophysiology of Huntington's disease, and we are encouraged by data from a small pilot study suggesting improvement in brain energy metabolism after treatment with triheptanoin."

NOVATO, Calif., Jan. 6, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, announced the initiation of a new development program for the human monoclonal anti-FGF23 antibody KRN23 (UX023) in tumor-induced osteomalacia (TIO). TIO results from typically benign tumors that produce excess levels of fibroblast growth factor 23 (FGF23), which can lead to severe osteomalacia, fractures, bone and muscle pain, and muscle weakness. Ultragenyx intends to initiate a Phase 2 study of KRN23 in six adult TIO patients in the first half of 2015.

KRN23 is being developed under a license and collaboration agreement between Ultragenyx and Kyowa Hakko Kirin Co., Ltd. KRN23 is being evaluated in a separate Phase 2 clinical study for pediatric patients with X-linked hypophosphatemia (XLH) and has completed multiple Phase 1/2 studies in adults with XLH.

Agreement Reached With Both FDA and EMA on Pivotal Trial Design
NOVATO, Calif., Dec. 15, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the dosing of the first patient in the pivotal Phase 3 study of recombinant human beta-glucuronidase (rhGUS, UX003), an investigational therapy for the treatment of Mucopolysaccharidosis 7 (MPS 7, Sly syndrome).

"We are pleased to have reached alignment with both the FDA and EMA on our single pivotal study design for this devastating disease," commented Sunil Agarwal, M.D., Chief Medical Officer of Ultragenyx. "This is an important step forward for these patients who have no approved treatment options. It is also an important milestone for Ultragenyx as it is the start of our first Phase 3 program."

The Phase 3 global, randomized, placebo-controlled, blind-start clinical study will assess the efficacy and safety of rhGUS in 12 patients between 5 and 35 years of age. Patients will be randomized to one of four groups. One cohort begins rhGUS therapy immediately, while the other three start on placebo and cross over to rhGUS at different predefined time points in a blinded manner. This novel trial design generates treatment data from all 12 patients, improving the statistical power of the study relative to a traditional parallel-group design. Based on data from the Phase 1/2 study, patients will be dosed with 4 mg/kg of rhGUS every other week for up to a total of 48 weeks, and all groups will receive a minimum of 24 weeks of treatment with rhGUS.

All Patients Continued to Demonstrate Increases in Serum Phosphorus and the Majority Maintained Levels in the Normal Range

NOVATO, Calif., Sept. 15, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the presentation of results from a long-term Phase 1/2 extension study, conducted by Kyowa Hakko Kirin Pharma, Inc., of the investigational fully human anti-FGF23 monoclonal antibody KRN23 (UX023) in adult patients with X-linked hypophosphatemia (XLH). The cumulative 16-month data, combining the four-month dose escalation period data from INT-001 and the 12-month extension data from INT-002, were presented at the American Society of Bone and Mineral Research (ASBMR) Annual Meeting in Houston.

New Patent Also Issued With Composition Claims for Triheptanoin

NOVATO, Calif., Aug. 5, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced a license agreement with UniQuest Pty Limited for intellectual property related to the treatment of refractory epilepsy and other seizure-related and neurologic disorders with triheptanoin (UX007). The intellectual property originated from research on epilepsy and other neurologic models conducted at The University of Queensland.

Upper Extremity Muscle Strength Preserved Over 48 Weeks in HIBM Patients on 6 Grams
NOVATO, Calif., April 30, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the presentation of detailed results from a 48-week Phase 2 clinical study of sialic acid extended-release (SA-ER, UX001) tablets in 47 patients with hereditary inclusion body myopathy (HIBM; also known by its new name as GNE myopathy), a rare, progressive muscle-wasting disease. SA-ER is designed to replace the deficient sialic acid substrate in patients with HIBM.