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Pharma startup Raptor hopeful about therapies

ONE TREATS ALCOHOL DISEASE, SECOND ATTACKS RARE CHILDHOOD DISORDER

NOVATO – Raptor Pharmaceuticals Corp. has a strategy to soar above the challenges to small, preclinical drug developers, namely funding.

Although Raptor's portfolio contains potentially life-saving therapies and a drug delivery system that could increase the efficacy of many existing drugs, investors are reluctant to take a chance on therapies that haven't yet entered the clinical trials that may lead to FDA approval, said co-founder and CEO Christopher Starr, Ph.D.

"Investors like to see drug candidates entering into pivotal clinical trials and meeting clinical and regulatory milestones," said Dr. Starr.

Dr. Starr was co-founder and senior vice president of publicly traded BioMarin Pharmaceutical Inc., which has successfully taken several enzyme therapies from early development to market approval and commercialization.

Wanting to advance its pipeline, Raptor (OTCBB: RPTP) looked around for clinical-stage molecules, keeping in mind the factors that may help speed up the FDA approval process. Last year the company found two compounds, Convivia and Delayed-Release (DR) Cysteamine.

Ted Daley, president of Raptor's clinical division, developed Convivia, a reformation of an already approved antidote to certain toxins. Already in human trials, the drug shows promise to address a metabolic disorder in largely Asian populations that is activated by the consumption of alcohol.

"The results we've seen in our first studies – performed in Hawaii – show that flushing, palpitations and carcinogenic buildup in tissues are lessened, and alcohol can be better tolerated," said Mr. Daley.

The drug has quickly advanced to a Phase II trial.

Raptor will partner with Asian companies for the longer clinical development, he said.

DR Cysteamine is a new formulation of a drug to treat the genetic disease Nephropathic Cystinosis.

Researchers at the University of California at San Diego took the formulation through its early stages; Raptor plans to take it to market under an exclusive licensing agreement.

Because Cystinosis is rare – only 500 patients in the U.S. and a comparable number in Europe suffer from the condition – the new formulation has been granted orphan-product designation by the FDA.

"The need is compelling. Children with Cystinosis have to take nausea-inducing drugs every six hours. We expect our formulation will lessen the required dosage frequency and the side effects considerably," said Mr. Daley.

In addition to Convivia and DR Cysteamine, Raptor is moving forward with preclinical testing of human proteins manufactured to target the receptors of certain molecules in the brain and liver.

Receptor-associated protein – the "RAP" in Raptor -– have the potential to move across the blood-brain barrier into designated brain tissue. Raptor, which purchased the patent on the delivery technology from BioMarin, is studying its possibilities in association with researchers at Stanford University.

Called "chaperones," RAP and its variables also have the potential to carry therapeutics to targeted tissue in the liver.

"We're focusing on two types of liver disease, hepatitis and primary liver cancer, for which there is no current treatment," said Dr. Starr.

The preclinical therapies and RAP technologies demonstrate Raptor's intention to work with drugs that have multiple therapeutic indications.

"If you have a single drug, the biggest expense in early clinical development is often manufacturing it," said Dr. Starr. "But if you can make a compound and then test it for various clinical indications, you can avoid duplication of effort."

Raptor is small: About a dozen consultants work with the seven-member team in its testing lab and offices in Novato. But Dr. Starr hopes to grow the staff as the Raptor products move through the FDA approval process.

In June Raptor closed a $10 million private placement of its stock, netting the company $9.3 million, which should take Raptor to the second quarter of 2009, according to Kim Tsuchimoto, Raptor's CFO.

For more information, visit www.raptorpharma.com.