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Patient Advocacy: Forming of Biopharmaceutical Companies

May 8, 2014

This is the first Blog Post in a series that examines the influence and power of patient advocacy groups, foundations, and alliances in helping and influencing the different stages of drug development in the rare disease and orphan drug space. Patient advocacy organizations provide capital investment in preclinical and clinical research programs with the goal of providing new treatment options. Patient advocacy organizations are also involved with the creation of start-up companies that are usually focused on finding a treatment or cure for a specific rare disease.

This Blog Post will look at two start-up companies, one created by a patient’s family and the other created by patient advocacy groups.

I – Dart Therapeutics

DART Therapeutics, a Cambridge, Massachusetts-based start-up biotechnology company, is an example of a rare disease startup company that uses the power of patient foundations and other stakeholders to drive the orphan drug development process. DART Therapeutics is a company financed and owned by patient 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, Duchenne Muscular Dystrophy (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 two DMD foundations and DART Therapeutics’ former CEO, Gene Williams. The two foundations are started by parents of boys with DMD :

•   Charley’s Fund is a 501(c)(3) tax-exempt public charity in Great Barrington, Massachusetts, with the mission to “direct money into the hands of researchers who have the best shot at developing a treatment or cure for DMD”
•   Nash Avery Foundation in Minnesota.

In March 2011, the company sets up an affiliate, Halo Therapeutics, to license in and develop the IND-stage DMD drug candidate HT-100, Halofuginone Hydrobromide. HT-100 aims to stop or slow down the formation of fibrosis and fatty tissue, which contributes to the death of DMD patients. HT-100 has orphan drug designation in both the US and the EU.  In October 2012, the company announces the completion of a study to evaluate a new non-invasive biomarker for DMD that measures muscle wasting in patients. The FDA has put HT-100 clinical trials on hold.

Dart Therapeutics’ other DMD product being developed is DT-200, which “aims to build muscle without the negative side effects caused by steroids”. DT-200 could soon begin human clinical studies.

II – Solid Ventures

Solid Ventures, is a biotechnology company founded by a father, Ilan Ganot, after his son, Eytani, is diagnosed with DMD. Mr. Ganot quit his job in London, moved to the Boston area, raised $17 million, and founded Solid Ventures. Eytani’s name translated from Hebrew to English is “solid”, and thus the company’s name.

With the financial support from JPMorgan (Mr. Ganot’s former employer) and other life sciences venture capitalists, Solid Ventures plans to acquire or license promising projects from other companies or academic laboratories, “move them through the earliest stages of testing with the help of contract research organizations, and ultimately partner or sell them to bigger pharmaceutical companies that can take them to market.”

Please Note: “Kid Hugging a Rainbow” by Marendo Müller, artwork (Own work) [Public domain] | Wikimedia Commons.

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

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