Event - Protalix to add further data to the Gaucher's story

Amicus Therapeutics is not the only company gearing up to announce important data on a new treatment for Gaucher’s disease; Protalix BioTherapeutics is also set to reveal results from a pivotal trial with its enzyme replacement candidate, prGCD, next month (Event – Amicus set for major boost from Gaucher's data, September 22, 2009).

The data are expected to be positive and will complete the rolling submission that is already being sent to the FDA for approval. If this is ultimately granted prGCD, which will be branded Uplyso, would be Protalix’s first product on the market. A positive regulatory review, meanwhile, would provide crucial validation of the company’s novel platform technology. Therefore, positive data is required next month to set this important chain of events into motion.

Green fingers

Protalix announced last week that the trial in 31 patients had completed, adding that no serious adverse events had occurred. This is important, because the method used to produce the enzyme that Gaucher’s patients lack is entirely novel.

The Israeli group has developed a technology platform called ProCellEx, a plant cell system which generates recombinant therapeutic proteins. The proteins are very similar to their naturally-produced human counterparts, in terms of structure and biological activity, hence their therapeutic applicability.

Importantly, the company believes this plant-based system can produce high quantities of proteins at a fraction of the cost and complexity of mammalian cell culture systems, which are employed to produce therapies such as Genzyme’s blockbuster Gaucher’s treatment Cerezyme, production of which has been seriously curtailed by contamination at a bioreactor; another issue that plant cell-based systems should avoid.

High hopes

Instead Uplyso is a plant-expressed recombinant form of glucocerebrosidase, the enzyme lacked by Gaucher’s sufferers. Admittedly, not much clinical data exists on the product because regulators allowed the company to progress straight into phase III trials, after successful phase I research. However, early data on enzyme therapies tends to be predictive, therefore hopes for success are high.

Naturally the safety profile will be a key focus, as will any signs of immunogenicity, however few complications are anticipated.

As such, Protalix shares are currently trading at a two-year high of $7.80, and have quadrupled in value so far this year. Anything other than stellar efficacy and a whistle clean safety profile, and some investors might be disappointed.

With the company thought to be looking for marketing partners, a fair amount of deal anticipation is also reflected in the share price.

Taking advantage

The Gaucher’s story is multi-faceted at the moment, with Shire also at a similar stage with its enzyme replacement product, velaglucerase. Both are hoping to quickly capitalise on Genzyme’s current woes and the two will naturally be compared, whilst a set back for either company would be a boon for the other (Therapeutic focus - Spotlight falls on new Gaucher treatments amid Genzyme's woes, August 6, 2009).

Still, both should be able to carve out if a niche. Analysts at Canaccord Adams, who today started coverage of Protalix with a buy recommendation and an $11 price target, said they believe Uplyso should be able to capture 15% of Cerezyme’s $1.25bn market. The company is expected to compete heavily on price.

Of course, Uplyso is only part of the Protalix story. As EP Vantagehas previously discussed, investors are also excited about Protalix’s potential to generate biosimilar proteins, at the fraction of the cost of the original products. Its novel production technique would essentially bypass many traditional protein drug patents. (Protalix's potential in biosimilars is becoming clearer, July 6, 2009)

The upcoming Uplyso data will help evaluate this broader potential, and help investors decide whether recent share price gains are warranted.

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