Ultragenyx - Page 4 - Pappas Capital

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.

Ultragenyx Pharmaceutical - 02/26/2013

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 Pharmaceutical - 01/10/2013

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 Pharmaceutical - 12/20/2012

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.

Ultragenyx Pharmaceutical - 05/01/2012

Ultragenyx Granted Orphan Drug Designation for UX003 for the Treatment of Mucopolysaccharidosis Type 7 MPS 7

March 14, 2012

Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, announced that the FDA Office of Orphan Products Development has granted orphan drug designation for UX003 for the treatment of MPS 7. MPS 7 is an extremely rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the lysosomal enzyme Beta-glucuronidase, required for the degradation of the glycosaminoglycans dermatan sulfate (DS) and heparan sulfate (HS). UX003 is a recombinant human Beta-glucuronidase intended as an enzyme replacement therapy for the treatment of MPS 7. MPS 7 was originally described in 1973 by William Sly, MD, St. Louis University School of Medicine, and is also known as Sly Syndrome. Ultragenyx in-licensed the MPS 7 program from St. Louis University.

Ultragenyx Pharmaceutical - 03/14/2012

Ultragenyx Announces the Completion of the Phase 1 Clinical Study of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease

NOVATO, Calif., March 8, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the Phase 1 study of UX001 for hereditary inclusion body myopathy (HIBM) has been completed. 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. UX001 is the first program from the company's pipeline to enter the clinic since its founding in 2010. The FDA Office of Orphan Products Development has granted orphan drug designation for UX001 for the treatment of HIBM.

Ultragenyx Pharmaceutical - 03/09/2012

FDA grants orphan drug designation to Ultragenyx UX003 for treatment of MPS 7

February 28, 2012

Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the FDA Office of Orphan Products Development has granted orphan drug designation for UX003 for the treatment of MPS 7. MPS 7 is an extremely rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the lysosomal enzyme Beta-glucuronidase, required for the degradation of the glycosaminoglycans dermatan sulfate (DS) and heparan sulfate (HS).

UX003 is a recombinant human Beta-glucuronidase intended as an enzyme replacement therapy for the treatment of MPS 7. MPS 7 was originally described in 1973 by William Sly, MD, St. Louis University School of Medicine, and is also known as Sly Syndrome. Ultragenyx in-licensed the MPS 7 program from St. Louis University.

Ultragenyx Pharmaceutical - 02/28/2012

Ultragenyx Announces Series A Financing

$45 million to support development of rare disease therapeutics

NOVATO, Calif., June 20, 2011--(EON: Enhanced Online News)--Ultragenyx Pharmaceutical Inc. today announced the closing of a $45 million Series A financing to support the development of rare disease therapeutics. The co-lead investors are TPG Biotech and Fidelity Biosciences, joined by European investor HealthCap, and Pappas Ventures. This funding will advance multiple rare disease product programs in the pipeline, as well as the development of new product candidates and partnerships. The lead product, UX-001, a first-in-class therapy for treatment of Hereditary Inclusion Body Myopathy (HIBM), is expected to enter the clinic in 2011.

Ultragenyx Pharmaceutical - 06/20/2011