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. 

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.

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.

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.

Read more here.

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 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.

The Phase 1 clinical trial was a multi-center, sequential dose-escalation study designed to characterize the safety, tolerability and pharmacokinetics of UX001 Sialic Acid-Extended Release (SA-ER) tablets in patients with HIBM disease. Twenty-six subjects received SA-ER tablets orally at one of five (5) dose levels in the single-dose phase and one of four (4) dose levels in the repeat-dose phase. Preliminary data showed that SA-ER was well-tolerated at the doses evaluated, with no serious adverse events reported. The adverse event profile was unremarkable and showed no pattern or dose dependent relationship. Pharmacokinetic analysis from the study showed that single doses of SA-ER are absorbed and provide significant drug levels over a 12-16 hour period. On repeated 3 times per day dosing, serum free sialic acid concentrations reached relatively steady levels over a 24 hour cycle.

“We are encouraged by the results of this early stage clinical trial which suggest SA-ER in single and repeated oral dosing is well-tolerated and has the potential to be a treatment for patients suffering from HIBM,” said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. “Based on these favorable Phase 1 results, we look forward to moving quickly to initiate a Phase 2 study of UX001.”

Ultragenyx plans to present the Phase 1 data at the 17th Annual International World Muscle Society Congress in Perth, Australia later this year.

About HIBM

HIBM is also known as GNE myopathy, distal myopathy with rimmed vacuoles (DMRV) and Nonaka disease. HIBM is a severe, adult-onset, progressive, genetic neuromuscular disease caused by a deficiency of an enzyme in the first step of sialic acid biosynthesis needed for the modification of proteins and fats. Patients with HIBM typically begin to have weakness and abnormal walking at 18 to 30 years of age. Over the ensuing 10 to 20 years, many patients progressively lose significant functional ability and become wheelchair-bound. There are no current treatments for this disease.

About Ultragenyx

Ultragenyx is a privately held, developmental stage biotechnology company committed to bringing life-enhancing therapeutics for patients with rare and ultra-rare genetic diseases, also known as orphan and ultra-orphan diseases, to market. The company focuses on rare metabolic diseases that affect small numbers of patients, but for which the unmet medical need is high and there are no effective treatments. Ultragenyx intends to build a sustainable pipeline of safe and effective therapies to address these underserved diseases. Ultragenyx’ lead program, UX001, is being evaluated as a potential treatment for hereditary inclusion body myopathy (HIBM), also known as GNE myopathy.

The company is led by an experienced management team in rare disease therapeutics. Ultragenyx is striving toward an improved model for successful rare disease drug development which has the potential to increase efficiency while maintaining appropriate safety and efficacy standards. The company believes that it can deliver significant value to patients by building a high quality pipeline of rare disease therapeutics and efficiently transforming good science into great medicine.

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