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Duchenne Muscular Dystrophy: Business Developments (07.28.13)

July 28, 2013

Duchenne Muscular Dystrophy (DMD) has attracted a lot of interest from investors and the media recently :

•    Sarepta Therapeutics’ announcement that the company is submitting a New Drug Application (NDA) to the FDA for orphan drug Eteplirsen in the first half of 2014
•   GlaxoSmithKline/Prosensa’s orphan drug Drisapersen receives FDA’s Breakthrough Therapy Designation
•   DART Therapeutics start of new clinical studies for HT-100
•   Prosensa IPO
•   PTC Therapeutics IPO.

This is the first Blog Post this week that discusses  and reviews recent business developments for DMD.

I – DART Therapeutics

DART Therapeutics, a Cambridge-based biotechnology company in Massachusetts, specializes in rare pediatric neuromuscular diseases.  The company is financed and owned by patient advocacy foundations. It is an example of a successful model for research and drug development centered on the needs of families affected by a specific rare disease – DMD. While the company is patient-funded and founded, it has a professional staff and business processes like any other biotechnology company. DART stands for “Disease Action Research Therapy”.  DART Therapeutics is created in 2010 by DART Therapeutics’ CEO Gene Williams, a former Genzyme executive  and two DMD foundations :

  1. Charley’s Fund
  2.  Nash Avery Foundation.

DART Therapeutis announces on July 15, 2013, that a Phase Ib/IIa study is initiated to determine the safety and tolerability of lead drug candidate HT-100 (delayed-release Halofuginone):

“DART Therapeutics’ study will also evaluate a new endpoint that could make DMD studies faster, more precise, less expensive, and inclusive of a larger group of boys. Presently, the six-minute walk (6MW) is the standard endpoint for DMD studies. However, the 6MW has shortcomings … The proposed endpoint, electrical impedance myography (EIM), is a simple, non-invasive technique that can measure the health of a muscle and track its changes over time. As a validated endpoint for DMD, EIM would allow researchers to include a wide range of boys in studies and more effectively and rapidly understand how well a treatment is working to halt disease progression.”

The HT-100 has orphan drug designation in both the US and the EU.

II – US Biotest

US Biotest, a private biological research company in San Luis Obispo, California, receives on July 25, 2013, FDA Orphan Drug Designation for angiotensin (1-7)[A(1-7)] for the treatment of DMD.

III – Prosensa/GlaxoSmithKline

Prosensa announces on July 8, 2013, the closing of its previously announced Initial Public Offering (IPO) of 6.9 million of its ordinary shares at a price of $13.00/share – including an additional 900,000 ordinary shares pursuant to the exercise of the over-allotment option by the underwriters. Prosensa raises $78 million from their IPO. The ordinary shares began trading on NASDAQ on June 28, 2013, under the symbol “RNA”.

Prosensa is partnering with GlaxoSmithKling (GSK) to develop Drisapersen, a Phase III Exon 51 Skipping drug candidate for DMD.  Drispersen recently receives FDA Breakthrough Therapy Designation.

Please Note: “AZT” by John Crawford (Photographer); NIH, US Federal Gov’t [Public Domain] | Wikimedia Commons.

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

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