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Lysosomal Storage Disorders: Start-up And IPO

February 7, 2014

This is the third Blog Post in a series over the next week that will examine Lysosomal Storage Disorders (LSDs) in the rare disease and orphan drug space. This Blog Post reviews a start-up company and a recent biopharmaceutical company’s Initial Public Offerings (IPO). Both are involved with LSDs.

I – Abeona Therapeutics

Abeona Therapeutics, a Cleveland-based biotechnology start-up, is created in March 2013 as a spinoff of Nationwide Children’s Hospital, with the goal of developing therapies for patients with the rare LSD, Sanfilippo (SF) Syndrome or Mucopolysaccharidosis III (MPS III). Abeona Therapeutics, who in partnership with scientists at Nationwide Children’s Hospital, and with financial support from multiple international patient advocacy groups, are collaborating to develop gene therapies for children with SF Types A and B.

In December 2013, Abeona Therapeutics closes $750,000 in seed financing to complete preclinical development of therapies for children with SF. “Preclinical studies demonstrate the therapy’s significant benefits in treating the disease, and the company will file for orphan drug status prior to its two Phase I/II clinical trials which are anticipated to begin in mid 2014.”

Collaborating scientists in Abeona Therapeutics’ Sanfilippo Consortium, receive in 2013 the Champion of Hope Award for Collaboration in Advocacy, from the Global Genes | RARE Project.

II – Ultragenyx Pharmaceutial

Ultragenyx Pharmaceutical, a California biotechnology company bringing new therapies for rare and ultra-rare metabolic genetic diseases, announces on February 5, the closing of its IPO of approximately 6.62 million shares of common stock at an IPO price of $21/share. The company estimates net proceeds from the offering to be approximately $126.4 million. What is quite interesting and is a great marketing tool, is the plan to list on the NASDAQ Global Select Market under the symbol “RARE”. J.P. Morgan and Morgan Stanley are the joint bookrunners on the deal.

Ultragenyx Pharmaceutical’s LSD treatment is UX003, an Enzyme Replacement Therapy (ERT) for the treatment of patients with Mucopolysaccharidosis Type VII (MPS VII or Sly Syndrome). The company licenses exclusive worldwide rights from Saint Louis University in November 2010. MPS VII is a rare autosomal recessive LSD with a deficiency of the  enzyme β-glucuronidase. MPS VII currently has no approved therapies.

The company announces in December 2013, the dosing of the first patient in a Phase I/II study of UX003 (recombinant human Beta-Glucuronidase or rhGUS) for MPS VII in the UK. The Phase I/II is an open-label clinical trial that will check the safety and efficacy of UX003 in a 12-week primary analysis, followed by dose-exploration and long-term extension. Five patients between 5 – 30 years of age will be enrolled and administered UX003 every other week using an intravenous infusion. Interim data is expected in 2014.

Please Note: “First Pigment Analysis (5893349712)” by NASA ICE (First Pigment Analysis  Uploaded by russavia) [CC-BY-2.0] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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