Skip to content

Rare Diseases: New IPO

November 9, 2013

According to Renaissance Capital (Greenwich, Connecticut), the following is a snapshot of the United States 3Q 2013 United States IPO Market :

•   60 IPOs completed in the 3Q 2013, compared to 26 IPOs in the 3Q 2012

•   $11.3 billion raised in the 3Q 2013, compared to $6.5 billion in the 3Q 2012

•   27 VC-backed deals in the 3Q 2013, compared to 9 VC-backed deals in the 3Q 2012

•   $2.5 billion VC-backed proceeds in the 3Q 2013, compared to $0.9 billion in the 3Q 2012.

Ultragenyx Pharmaceutical, a California biotechnology company bringing new therapies for rare and ultra-rare metabolic genetic diseases, announces on November 8 the filing of a registration statement on Form S-1, with the United States Securities and Exchange Commission (SEC), relating to the proposed Initial Public Offering (IPO) of its common stock. The company plans to raise $86.25 million. What is quite interesting and is a great marketing tool, is the plan to list on the NASDAQ under the symbol “RARE”. J.P. Morgan and Morgan Stanley are the joint bookrunners on the deal.

Ultragenyx Pharmaceutical, is started in 2010 by Emil Kakkis, former Chief Medical Officer at rare disease drug developer BioMarin Pharmaceutical. During his tenure at BioMarin Pharmaceutial, three rare disease products receive FDA approval :

•   Aldurazyme (Laronidase) for Mucopolysaccharidosis I (MPS I)

•   Naglazyme (Galsulfase) for Mucopolysaccharidosis VI (MPS VI)

•   Kuvan (Sapropterin Dihydrochloride) for Phenylketonuria (PKU).

In 2009, Dr. Kakkis starts the EveryLife Foundation for Rare Diseases with the mission of “accelerating biotech innovation for rare disease treatments through science-driven public policy”.

Ultragenyx Pharmaceutical’s lead program, UX-001 (Sialic Acid), receives FDA Orphan Drug Designation (ODD) in September 2011, for the treatment of Hereditary Inclusion Body Myopathy (HIBM). The oral drug UX-001 uses an extended-release formulation of sialic acid. HIBM is a rare, progressive neuromuscular disease caused by a deficiency in sialic acid that results in muscles slowly wasting away. Many patients require a wheelchair within 10 – 15 years of disease onset.There is no approved treatment for HIBM. There are approximately 1,200 – 2,000 patients in the developed world with the disease. A Phase II randomized, double-blind, placebo-controlled study is being conducted in 47 HIBM patients, to test UX-001’s safety, efficacy, and pharmacodynamics.

In May  2013, Ultragenyx Pharmaceutical announces a Phase I/II study of UX-003 for Mucopolysaccharidosis Type VII (MPS VII or Sly Syndrome). The company licenses exclusive worldwide rights from Saint Louis University in November 2010. UX-003 is an Enzyme Replacement Therapy (ERT) for the treatment of patients with MPS VII. MPS VII is a rare autosomal recessive lysosomal storage disorder with a deficiency of the  lysosomal enzyme β-glucuronidase. MPS VII currently has no approved therapies.

In September 2013, Ultragenyx Pharmaceutical announces that another experimental therapy, UX-023, is being co-developed and commercialized in a collaboration and license agreement with Japan’s Kyowa Hakko Kirin. UX-023 is for the potential treatment of a rare bone-disease, X-Linked Hypophosphatemia (XLH). XLH is caused by poor mineralization of bone that can result in short stature, muscle weakness, and other problems. There is currently no drug therapy for this rare disease. There are approximately 12,000 patients in the United States.

Ultragenyx Pharmaceutical joins other rare disease and orphan drug biotechnology companies in 2013 riding the wave of the IPO trend :

•   Acceleron Pharma

•   Bluebird bio

•   PTC Therapeutics.


Ultragenyx Pharmaceutical Pipeline.

Please Note: “Icon Announcer” by Orion 8 [Public domain] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

Leave a Comment

Leave a Reply

Fill in your details below or click an icon to log in: Logo

You are commenting using your account. Log Out / Change )

Twitter picture

You are commenting using your Twitter account. Log Out / Change )

Facebook photo

You are commenting using your Facebook account. Log Out / Change )

Google+ photo

You are commenting using your Google+ account. Log Out / Change )

Connecting to %s

%d bloggers like this: