Protalix Biotherapeutics (NYSE American: PLX; TASE: PLX) has reported positive results in a trial comparing its PRX-102 drug for treatment of Fabry Disease to Replagal, the second largest competitor in the Fabry treatment market, which is marketed by Shire. The trial tested 16 patients who previously used Replagal for at least two years, including a stable dose for the past six months, then switched to Protalix's product. The average kidney functioning in this group, which declined during the period in which Replagal was used, improved when the patients switched to PRX-102. Despite the small sample size, the difference was statistically significant.
Protalix's share price rose 16% on the NYSE American index last Friday, boosting the company's market cap to $107 million, and is currently up 14% on the TASE.
Fabry is a rare genetic disease in which harmful proteins accumulate in many organs in the body. This is the first trial comparing PRX-102 to a competing product. In its Phase I/II trial, Protalix's product was administered to new patients who had never been treated before with a drug. The results were superior to past results for the existing products in the market, but a comparison with past data is considered poorer evidence of a drug's superiority than a comparative trial on the same patients.
Protalix has only three competitors in the Fabry market. The leading product is Fabrazyme, made by Genzyme, which dominates the US market. Shire's Replagal, with which PRX-102 was compared in the recent trial, is marketed only outside the US; it shares the European market roughly equally with Genzyme. Amicus Therapeutics has developed an orally administered product for Fabry (the others are injected), but it is only suitable for some patients. The entire market current totals $1.4 billion, but earlier diagnosis and treatment of patients is increasing its size.
The most important trial that Protalix is currently conducting is a Phase III trial on 78 patients comparing PRX-102 to Genzyme's product. Final results from this trial are due in 2021, although Protalix is about to consult with the US Food and Drug Administration (FDA) about requesting a shorter track for its product.
Protalix has partnered with Italian company Chiesi Farmaceutici for the development and commercialization of PRX-102. The Italian company has exclusive marketing rights for the drug. If the drug is very successful, Protalix's revenue from it could amount to over $1 billion.
Direct comparison trials are especially important for Protalix because when current CEO Moshe Manor entered his position in 2014, he said that the company would bring only products to market if they were superior to the existing drugs. Protalix's first product, for treatment of Gaucher's Disease, was cheaper to produce and better protected from contamination in production than its competitors, but its clinical performance was similar to theirs, and therefore failed to obtain a substantial market share in most countries. It is therefore important for Protalix to show that its Fabry Disease product is clinically superior, because it is aimed at a market in which its competitors are well established.
Protalix has a few million dollars in quarterly revenue from its product for Gaucher's Disease. The company recently reached a debt arrangement that left it with a $40 million debt in convertible bonds. If its Phase III trials is successful, milestone payments from its agreement with Chiesi will probably cover its debt, and it is also possible that the bonds will be converted into shares. As of the end of the second quarter, Protalix had $28.3 million in cash.
Published by Globes [online], Israel business news - www.globes-online.com - on September 25, 2018
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