Ultragenyx (RARE) began trading on the NASDAQ on 31 January 2014

NEW YORK (AP) -- Shares of Ultragenyx have more than doubled in their trading debut after the drug developer's initial public offering raised $121 million.

The Novato, Calif., company is developing several possible treatments for rare or extremely rare diseases, including Sly Syndrome, a cellular and organ dysfunction that typically leads to death by early adulthood.

Ultragenyx Pharmaceutical Inc. sold 5.8 million shares at $21 each, well above its mid-January forecast of 4.8 million shares going for $14 to $17.

The stock climbed $24 to $45 per share in midday trading Friday.

Two other drug developers debuted on the public market Friday: Trevena Inc., which raised about $64.8 million, and Cara Therapeutics Inc. in a $55 million IPO.

3 weeks after IPO

Address

60 Leveroni Court
NOVATO, CA 94949
United States 

Website 

http://www.ultragenyx.com

Ultragenyx Pharmaceutical Inc., a development-stage biotechnology company, focuses on the identification, acquisition, development, and commercialization of various products for the treatment of rare and ultra-rare diseases in the United States. It develops various biologics product candidates, including KRN23, a human monoclonal antibody to bind and reduce the biological activity of fibroblast growth factor to enhance abnormally low phosphate levels in patients with X-linked hypophosphatemia; recombinant human beta-glucuronidase, an intravenous enzyme replacement therapy for the treatment of mucopolysaccharidosis 7; and recombinant human protective protein cathepsin-A, an enzyme replacement therapy for galactosialidosis. The company is also developing a range of small-molecule product candidates comprising triheptanoin, a substrate replacement therapy for patients with fatty acid oxidation disorders, as well as for patients with glucose transporter type-1 deficiency syndrome; and oral formulation of sialic acid to treat hereditary inclusion body myopathy. It has license agreements with AAI Pharma Services Corp. and HIBM Research Group; and a collaboration and license agreement with Nobelpharma Co., Ltd.
The company was founded in 2010 and is headquartered in Novato, California.

Orphan drug maker Ultragenyx ups IPO price to $19-$20

Ultragenyx Pharmaceutical Inc. upped its initial public offering price to $19 to $20 per share, hoping to raise as much as $98 million from the sale of 4.8 million shares this week.

The Novato-based rare drug developer, which would be traded on the NASDAQ exchange as "RARE," previously priced the IPO at $14 to $17 per share.

Based on a midpoint of $19.50 per share, Ultragenyx would net about $84.7 million, the company said in a Securities and Exchange Commission filing, but if underwriters exercise all their options, proceeds could climb to $97.9 million.

The IPO is being closely watched as one of the first biotech offerings of 2014, but also a test of CEO Emil Kakkis' vision and the drug-development industry's focus in recent years on so-called orphan diseases.
Generally, drugs to treat rare diseases cost more and insurance companies have not balked at covering those costs for patients.

Ultragenyx has four drugs in clinical trials. Its lead program, UX-001, is an extended-release form of sialic acid aimed at hereditary inclusion body myopathy. Another drug, UX-023, is a monoclonal antibody that targets X-linked hypophosphatemia, a bone disease that causes bowed legs, shorter height and muscle weakness.

In the Bay Area, KineMed Inc. of Emeryville, Revance Therapeutics Inc. of Newark, Asterias Biotherapeutics Inc. of Menlo Park and CardioDx Inc. of Palo Alto are planning IPOs after about a half-dozen life sciences IPOs last year.