Israeli biotech firm Protalix Biotherapeutics Ltd. (PLX) is only months away from announcing the late-stage study results of its lead product candidate prGCD for the treatment of Gaucher (pronounced Go-shay) disease.
Gaucher disease is an inherited genetic disorder, in which patients lack the normal form of the glucocerebrosidase, or GCD enzyme that breaks down specific fat molecules. The lack of this enzyme results in the accumulation of fat in liver, spleen and bone marrow.
The pivotal phase III clinical trial of prGCD, which is currently underway, is a multi-center, randomized, double-blind, parallel group, dose-ranging trial to assess the safety and efficacy of prGCD in 30 naive patients suffering from Gaucher disease. In the trial, patients are selected randomly for one of two dosing arms and receive IV infusions every two weeks for nine months. The primary endpoint of the study is the percent change in spleen volume from baseline, as measured by MRI (Magnetic Resonance Imaging). According to the company, no serious adverse events have been reported in the study. The company is anticipated to announce the phase III trial results of prGCD in the second-half of 2009.
The current standard of care for Gaucher patients is enzyme replacement therapy and Genzyme Inc.'s (GENZ) Cerezyme is currently the only approved enzyme replacement therapy for Gaucher disease.
Though Gaucher is a rare disease, the market for Gaucher drugs is huge due to severity of the symptoms and the chronic nature of the disease. The disease affects fewer than 200,000 people in the United States at any given time. Last year, the drug fetched in sales of $1.2 billion for Genzyme. On Tuesday, Genzyme announced that it is temporarily shutting down one of its manufacturing plants in the U.S. until the end of July to clean up viral contamination.
Protalix's prGCD, which is also an enzyme replacement therapy, is a plant cell expressed version of the GCD enzyme, developed through the company's ProCellEx protein expression system. According to the company, plant cells do not carry the risk of infection by human or other animal viruses and as a result the risk of contamination of its products is eliminated.
Unlike prGCD, the existing enzyme replacement therapies like Cerezyme are produced using mammalian cell-based expression systems.
The company believes that prGCD may prove more cost-effective than the currently marketed alternative due to the cost benefits of expression through its ProCellEx protein expression system.
Protalix anticipates submitting a New Drug Application for prGCD to the FDA and other comparable regulatory agencies in other countries in the fourth quarter of 2009.
As a reminder, Protalix became public through a reverse merger with Orthodontix Inc., and the transaction became effective on December 31, 2006. Protalix trades on the American Stock Exchange.
In addition to prGCD, Protalix is developing therapeutic protein candidates for the treatment of Fabry disease, a rare, genetic lysosomal disorder in humans, an acetylcholinesterase enzyme- based therapy for biodefense and intoxication treatments and an additional undisclosed therapeutic protein, all of which are currently being evaluated in animal studies. The company plans to file an investigational new drug application with the FDA with respect to acetylcholinesterase enzyme-based therapy for biodefense applications during this year and to initiate human clinical studies immediately thereafter.
The company has incurred significant losses since its inception in December 1993 and at March 31, 2009, had an accumulated deficit of $80.2 million. During the first-quarter ended March 31, 2009 net loss was $5.18 million or $0.07 per share, compared to $5.11 million or $0.07 per share in the year-ago period. The company has yet to bring a product to the market and does not generate revenue.
Protalix has no debt on its balance sheet and cash and cash equivalents at the end of March 31, 2009 totaled nearly $35 million.
PLX, which has been trading in the range of $0.96-$4.35 in the last twelve months, closed Wednesday's trading at $4.16, up 4.52% on an above-average volume of 210 thousand shares.
With the company approaching the end of the pivotal phase III clinical trial for its most promising drug, it is worth keeping an eye on PLX.
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