Dr. Christopher M. Starr, the CEO of Raptor Pharmaceutical Corp. (RPTP), Interviews with The Wall Street Transcript

Wall Street Transcript



Companies include: Raptor Pharmaceutical Corp. (RPTP) and many more.

In the following excerpt from the Biotechnology and Pharmaceuticals Report, the CEO of Raptor Pharmaceutical Corp. (RPTP) discusses company strategy and the outlook for this vital industry:

TWST: Tell us about PROCYSBI. What is the science behind this product, and is there anything else like it?

Dr. Starr: It's actually an extremely interesting story. Cystinosis is an inherited disorder in which patients are born with a defect in their ability to clear a certain aminoacid called cystine from their body. When this amino acid accumulates in cells in the body, it becomes toxic and results in tissue and organ failure. Before any therapy was available, these kids would die within the first decade of life. Even after kidney transplants came about and patients with cystinosis could get them, patients would ultimately die of other problems with the disease.

Like many orphan disease programs, the PROCYSBI story is entrenched in some basic science research in medical centers and the passionate families of patients that drove the research. Immediate-release cysteamine bitartrate has been available as a treatment for cystinosis since 1994, and when used as directed it can be effective. However, most patients are unable to adhere to the treatment regimen, despite the fact that every missed dose leads to irreversible and progressive damage to organs in the body.

Immediate-release cysteamine has to be taken every six hours, and so you're talking about waking kids up in the middle of the night every single night to give them the drug, and the kids must take one dose while they are at school. It's a difficult drug to take too, often causing severe nausea and vomiting in kids. In addition, the drug causes halitosis and a body odor, which many patients found to be embarrassing. This causes social issues for the patients - sometimes other kids make fun of them or bully them in school.

This is where the cystinosis patient community really got involved and pushed researchers to work on ways of potentially making a better drug for patients. The Cystinosis Research Foundation funded some early research at UC San Diego primarily around some really elegant pioneering work in reformulating cysteamine. They were able to show in a proof-of-concept study that...


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