Knobias Clip Report (10-23-2007)

Submitted By Knobias ClipReport

FOLD: Sees 52-Week High on Tuesday After Positive Ph I Study for Pompe Disease

Shares of Amicus Therapeutics, Inc. (FOLD) hit a 52-week high on Tuesday after it announced positive results from two completed Phase 1 clinical studies of AT2220 (1-deoxynojirimycin HCl) for Pompe disease. The Phase 1 results showed that AT2220 was well-tolerated. The Company expects to initiate a Phase 2 clinical trial of AT2220 for Pompe disease in early 2008. The FDA has also granted orphan drug designation for AT2220 in the United States.

Pompe disease is a rare inherited metabolic muscle disorder that affects about 1 in 40,000 people in the U.S. It is caused by the buildup of a complex sugar called glycogen in the body's cells. The accumulation of glycogen in certain tissues, especially muscles, impairs their ability to function normally. The early onset form of the disease is the most severe, progresses most rapidly, and is characterized by musculoskeletal, pulmonary, gastrointestinal, and cardiac symptoms that usually lead to death for patients between 1 and 2 years of age. The late onset form of the disease begins between childhood and adulthood and has a slower rate of progression that is characterized by musculoskeletal and pulmonary symptoms that usually lead to progressive weakness and respiratory insufficiency.
There is currently no approved cure for Pompe disease. Genzyme Corp.'s (GENZ) Myozyme(R) was granted FDA approval for the treatment of Pompe disease in 2006. It has been shown to improve ventilator-free survival in patients with infantile-onset Pompe disease as compared to an untreated historical control, whereas use of Myozyme in patients with other forms of Pompe disease has not been adequately studied to assure safety and efficacy.

Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genetic diseases with unmet medical need. Amigal is currently in Phase 2 clinical trials for Fabry disease and Phase 2 clinical trials of Plicera for Gaucher disease.

Fabry disease is a genetically inherited disease that is caused by the lack of or faulty enzyme needed to metabolize lipids, fat-like substances that include oils, waxes, and fatty acids. A mutation in the gene that controls this enzyme causes insufficient breakdown of lipids, which build up to harmful levels in the eyes, kidneys, autonomic nervous system, and cardiovascular system. Males tend to experience the most severe clinical symptoms, while females vary from virtually no symptoms to those as serious as males.

Gaucher's disease is the most common of the lysosomal storage diseases. It is caused by a deficiency of the enzyme glucocerebrosidase, leading to an accumulation of its substrate, the fatty substance glucocerebroside. Fatty material can collect in the spleen, liver, kidneys, lungs, brain and bone marrow. Symptoms may include enlarged spleen and liver, liver malfunction, skeletal disorders and bone lesions that may cause pain, severe neurologic complications, swelling of lymph nodes and (occasionally) adjacent joints, distended abdomen, a brownish tint to the skin, anemia, low blood platelets and yellow fatty deposits on the sclera.

Genzyme, the early pioneer in this area, has developed Fabrazyme(R) (agalsidase beta) to replace the missing enzyme in patients with Fabry disease. It is available in over 30 countries, including the United States and Europe. Genzyme's Cerezyme(R) is indicated for Gaucher's disease.

All of the conditions that the Company is currently developing products for are quite rare, and the enzyme drugs are among the most expensive in the world. Cerezyme, for instance, costs $200,000 a year. Sales of the drug last year were $1 billion. The demand and lack of competition in this area could provide significant opportunities for Amicus.


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