Georgia Erbez, CFO of Raptor Pharmaceutical Corp. (RPTP), said the company has initiated an 18-month Huntington disease trial. She spoke at the Canaccord Genuity 34th Annual Growth Conference at the Intercontinental Hotel in Boston, Massachusetts.
Raptor Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of therapeutics that treat rare, debilitating and often fatal diseases. Among the areas being explored are nephropathic cystinosis, Huntington’s disease (HD), nonalcoholic steatohepatitis (NASH), Leigh syndrome and other mitochondrial diseases.
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The company’s Procysbi (cysteamine bitartrate) is on the market and used for the management of nephropathic cystinosis in children and adults. Net product sales for Procysbi for the second quarter were $16.3 million, representing a 34% increase over the first quarter of 2014, and $28.4 million for the six months ended June 30, 2014. Procysbi became commercially available in the U.S. in June 2013 and in Germany in April of this year.
Cystinosis is a rare genetic condition that affects an estimated 3,000 patients worldwide. It is fatal if not treated in early childhood and causes a protein building block called cystine to build up in every cell of the body. The buildup of cystine causes kidney problems, which can cause the body to lose too much sugar, proteins and salts through the urine.
Cystinosis may lead to slow body growth and small stature, weak bones and developing and worsening kidney failure. The product received orphan designation and holds that exclusively in the U.S. and EU.
“We have substantial commercial and pipeline growth,” said Erbez. “We intend to build a company that is capable of sustained, profitable growth.” Erbez added, “global growth is vital to our strategy. We intend to expand our commercial opportunity throughout the U.S. and EU.”
The company is engaged in a genetic screening program partnership with access to 4200 patients with end-stage renal disease, Erbez said. Through the study, they proved their hypothesis that there is an adult-onset form of cystinosis. They anticipate a publication of their finding next year.
The company will continue exploring additional therapeutic uses of its products, and intends to retain global rights to them, Erbez said. Raptor has a market cap of $753.7 million.
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