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Raptor Reports Promising Results From Phase IIa ALDH2 Deficiency Study

November 13, 2008

Raptor Pharmaceuticals, a development-stage biotechnology company, has announced positive results in its Phase IIa study of oral 4-methylpyrazole in subjects with aldehyde dehydrogenase deficiency, or ethanol intolerance, as the initial development stage of its Convivia program.

Convivia is Raptor’s proprietary oral formulation of 4-methylpyrazole (4-MP) designed to reduce systemic acetaldehyde exposure and related symptoms in aldehyde dehydrogenase (ALDH2) deficient persons following alcohol consumption.

Conducted in Hawaii, the blinded, intra-subject controlled, single dose, dose escalation study enrolled 32 subjects of Japanese descent with a history of symptoms of inborn altered ethanol metabolism with concomitant ethanol exposure. At the time of enrollment, study subjects also submitted to alcohol dehydrogenase (ADH) and ALDH2 genotyping.

The objectives of the study were to investigate the safety and efficacy of 4-MP as a potential treatment of symptoms resulting from acetaldehyde toxicity in ALDH2 deficient subjects who drink alcohol. The trial also sought to provide information leading to the most efficacious dose and dosage timing range.

The study results demonstrated that the active ingredient in Convivia significantly reduced heart palpitations (tachycardia), which are commonly experienced by ALDH2 deficient people who drink, at all dose levels tested.

The study also found that the 4-MP significantly reduced peak acetaldehyde levels and total acetaldehyde exposure in a subset of the study participants who possess specific genetic variants of the liver ADH and ALDH2 enzymes. This subset represents approximately one-third of the ALDH2 deficient adult population, the company said.

Ted Daley, president of clinical division at Raptor, said: “We are encouraged by these results from our Phase IIa study for Convivia. Currently there are no approved treatments for ALDH2 deficiency, and people with this disorder could potentially benefit by lessening their exposure to acetaldehyde, a known carcinogen, and mitigating the unpleasant reactions to drinking. We plan to seek partners with clinical and commercial operations in Asian countries, to continue the development of this product candidate.”




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