Ultragenyx Initiates Novel Disease Monitoring Program for Hereditary Inclusion Body Myopathy

Ultragenyx Initiates Novel Disease Monitoring Program for Hereditary Inclusion Body Myopathy

 

First patient enrolled in integrated program designed to improve knowledge of rare disease

NOVATO, CA –April 8, 2013 -Ultragenyx Pharmaceutical Inc. today announced the launch of a unique Disease Monitoring Program (DMP) for Hereditary Inclusion Body Myopathy (HIBM), also known as GNE Myopathy. The goal of the HIBM-DMP is to improve the body of knowledge about this rare disease and its typical course. This novel program is being conducted in partnership with the University of Newcastle’s TREATNMD organization, a global neuromuscular physician network in Newcastle, England.The main objectives of the HIBM-DMP are to expand knowledge of the clinical presentation, progression and variation of HIBM patients; identify and qualify biomarkers and other efficacy measures; inform the design and interpretation of clinical studies of investigational products for HIBM and eventually to optimize patient management.

Ultragenyx Initiates Novel Disease Monitoring Program for Hereditary Inclusion Body Myopathy

First patient enrolled in integrated program designed to improve knowledge of rare disease

NOVATO, CA –April 8, 2013 -Ultragenyx Pharmaceutical Inc. today announced the launch of a unique Disease Monitoring Program (DMP) for Hereditary Inclusion Body Myopathy (HIBM), also known as GNE Myopathy. The goal of the HIBM-DMP is to improve the body of knowledge about this rare disease and its typical course. This novel program is being conducted in partnership with the University of Newcastle’s TREATNMD organization, a global neuromuscular physician network in Newcastle, England.The main objectives of the HIBM-DMP are to expand knowledge of the clinical presentation, progression and variation of HIBM patients; identify and qualify biomarkers and other efficacy measures; inform the design and interpretation of clinical studies of investigational products for HIBM and eventually to optimize patient management. 

The HIBM-DMP is designed to assist the HIBM communities in several ways, including:

– integration of an online registry capturing patient-reported information, a fully monitored physician-driven natural history study, and potentially any post-approval patient follow-up into a single cohesive program.

– knowledge-sharing gained from the program about HIBM with investigators, physicians and patients in a manner compliant with patient data protection regulations.

Man with a plan: Emil Kakkis aims to lure big-market drugs toward rare diseases

 

February 26, 2013

Emil Kakkis is making another run at rare diseases.

The president and CEO of Ultragenyx Pharmaceutical Inc. and former chief medical officer at BioMarin Pharmaceutical Inc. is laying out a new strategy Tuesday to get more drugs into children with rare diseases. Under the banner of the EveryLife Foundation for Rare Diseases, which Kakkis founded and has largely bankrolled, he is proposing that companies with already-approved, big-market drugs be rewarded with a two-month extension of that drug’s patent life if it wins a new Food and Drug Administration-approved label in an orphan disease.

Man with a plan: Emil Kakkis aims to lure big-market drugs toward rare diseases

Emil Kakkis is making another run at rare diseases.

The president and CEO of Ultragenyx Pharmaceutical Inc. and former chief medical officer at BioMarin Pharmaceutical Inc. is laying out a new strategy Tuesday to get more drugs into children with rare diseases. Under the banner of the EveryLife Foundation for Rare Diseases, which Kakkis founded and has largely bankrolled, he is proposing that companies with already-approved, big-market drugs be rewarded with a two-month extension of that drug’s patent life if it wins a new Food and Drug Administration-approved label in an orphan disease.

Read the full story here.

Ultragenyx Announces In-Licensing of Clinical-Stage Product Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorders

NOVATO, CA –January 10, 2013— Ultragenyx Pharmaceutical Inc., a biotechnology company focused on the development of treatments for rare and ultra-rare genetic disorders, announced that it has in-licensed rights for triheptanoin,  a promising treatment for long-chain fatty acid oxidation disorders (FAOD), from Baylor Research Institute in Dallas, TX. Triheptanoin is intended as a substrate replacement therapy to restore deficient intermediates in the mitochondria and to enable energy metabolism in patients with FAOD and potentially other genetic metabolic disorders.

Ultragenyx Raises $75 Million in Oversubscribed Crossover Financing

NOVATO, CA – December 20, 2012 – Ultragenyx Pharmaceutical Inc., a biotechnology company focused on the development of treatments for rare and ultra-rare genetic disorders, today announced the successful completion of a $75 million Series B round financing led by Adage Capital Partners, LP.  Joining Adage as new investors in this financing are mutual funds and separate account clients advised by T. Rowe Price Associates, Inc., Jennison Associates LLC (on behalf of clients), funds and accounts under management by subsidiaries of BlackRock, Inc., Sanofi-Genzyme BioVentures 1, Shire plc and additional blue chip public market funds.  Existing investors, TPG Biotech, Fidelity Biosciences, HealthCap and Pappas Ventures also participated in the transaction.

Ultragenyx Press Release – Ultragenyx Raises $75 Million in Oversubscribed Crossover Financing

NOVATO, CA – December 20, 2012 – Ultragenyx Pharmaceutical Inc., a biotechnology company focused on the development of treatments for rare and ultra-rare genetic disorders, today announced the successful completion of a $75 million Series B round financing led by Adage Capital Partners, LP.  Joining Adage as new investors in this financing are mutual funds and separate account clients advised by T. Rowe Price Associates, Inc., Jennison Associates LLC (on behalf of clients), funds and accounts under management by subsidiaries of BlackRock, Inc., Sanofi-Genzyme BioVentures 1, Shire plc and additional blue chip public market funds.  Existing investors, TPG Biotech, Fidelity Biosciences, HealthCap and Pappas Ventures also participated in the transaction.

Ultragenyx Initiates Phase 2 Study of UX001 in Hereditary Inclusion Body Myopathy

 

NOVATO, Calif., July 5, 2012 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced the dosing of the first two patients in a Phase 2 study of UX001 for hereditary inclusion body myopathy (HIBM). HIBM is a rare, severe, neuromuscular disease caused by sialic acid deficiency. UX001 is an extended-release oral tablet formulation of sialic acid (SA-ER) intended as a substrate replacement therapy for HIBM.

Ultragenyx Initiates Phase 2 Study of UX001 in Hereditary Inclusion Body Myopathy

NOVATO, Calif., July 5, 2012 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced the dosing of the first two patients in a Phase 2 study of UX001 for hereditary inclusion body myopathy (HIBM). HIBM is a rare, severe, neuromuscular disease caused by sialic acid deficiency. UX001 is an extended-release oral tablet formulation of sialic acid (SA-ER) intended as a substrate replacement therapy for HIBM.

Read more here.

Ultragenyx Announces Phase 1 Results of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease

NOVATO, Calif., May 1, 2012 /PRNewswire/ Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced results from a first-in-human, multi-center, Phase 1 study of UX001 in patients with hereditary inclusion body myopathy (HIBM) showing that UX001 was well-tolerated with an expected extended release profile on absorption after oral administration. UX001 is an extended release formulation of sialic acid intended as a substrate replacement therapy for HIBM, a severe, neuromuscular disease caused by sialic acid deficiency. Based on the Phase 1 results, Ultragenyx plans to initiate an international, multi-center, randomized, double-blind, placebo-controlled, parallel group Phase 2 study of UX001 in HIBM patients in the second quarter of this year.