Protalix Plans Human Clinical Tests of Medication for Rare Fabry's Disease

Protalix BioTherapeutics Ltd., an Israeli biotechnology company, plans to start clinical trials of a drug treating Fabry disease this year and a medication against rheumatoid arthritis in early 2012.

Protalix aims to bring the two products to market by 2016, Chief Executive Officer David Aviezer said. The Carmiel-based company is also targeting full U.S. regulatory approval in 2011 for the only medicine it already sells, taliglucerase alfa, after temporary clearance to supply the Gaucher-disease treatment to make up for a shortage of a competing product.

The market for drugs to treat Fabry disease, an illness that leads to fat buildups affecting kidney or heart functions, may reach about $850 million by 2019, said Steven Tepper, an analyst at Harel Finance Ltd. Sales of drugs such as the rheumatoid-arthritis treatment, which seeks to block a protein called tumor necrosis factor, may total $6.2 billion by then, he said.

“We continue to advance other drugs we have in our pipeline at full force,” Aviezer said in an interview at Protalix’s office in the Tel Aviv suburb of Ramat Gan. “We believe the Fabry disease and anti-TNF rheumatoid arthritis drugs will be in clinical development in humans already in the year to come, ready for sale within the next five years.”

The Fabry drug, PRX-102, would compete with Genzyme Corp. (GENZ)’s Fabrazyme and Shire Plc’s Replagal. PRX-106, the rheumatoid arthritis drug, would be a biological copy of Amgen Inc. (AMGN)’s Enbrel, Protalix has said.

Fat Buildup

Taliglucerase alfa, Protalix’s drug for Gaucher disease, provides an enzyme that prevents fat buildups in the liver, spleen, bone marrow and nervous system. Like Fabry disease, Gaucher is a rare inherited illness in which patients lack the enzymes to prevent the accumulation of fat. Protalix in 2009 sold a global marketing license for taliglucerase alfa to New York-based Pfizer Inc. (PFE), the world’s largest drugmaker, giving the U.S. company 60 percent of the drug’s revenue.

Protalix has had provisional Food and Drug Administration permission to supply the drug to the U.S. after a virus contamination in 2009 at a Genzyme factory in Boston cut the production of its Gaucher treatment, Cerezyme. The Israeli company failed last month to win full marketing approval when the FDA asked for more data from two studies and information on testing specifications.

FDA approval probably will come this year, Aviezer said in the interview March 22. The drug also would compete with Shire Plc (SHP)’s Vpriv.

Lower Rating

The FDA decision prompted Harel analyst Tepper to reduce his recommendation on Protalix to “market perform” from “buy,” and cut his share-price estimate to $8 from $12.

“The drugs in the pipeline add some value to the company, but it is still mainly driven by the product for treating Gaucher disease,” Tepper said in a phone interview. “Although the chances Protalix will receive the FDA final approval for marketing its Gaucher disease drug are high, it is more realistic to assume approval will come only next year.”

Protalix rose 1.9 percent to 21.47 shekels at the 4:30 p.m. close in Tel Aviv, giving the company a market value of 1.74 billion shekels ($490 million).

The company’s U.S. shares have dropped about 13 percent in the past year, compared with a 3.1 percent increase in the Bloomberg World Biotech Index. The stock has lost 35 percent since failing to win the FDA approval.

Protalix sold 4 million shares at $5.50 each to finance research including clinical trials, the company said March 18.

To contact the reporter on this story: Ronit Goodman in Tel Aviv at rgoodman9@bloomberg.net

To contact the editor responsible for this story: Claudia Maedler at cmaedler@bloomberg.net

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