Protalix Drug in Phase IIa Study for Gaucher Disease - Analyst Blog

Protalix BioTherapeutics, Inc. PLX announced that it has enrolled the first patient in a 28-day open-label, sequential dose escalation phase IIa study on PRX-112 (oral glucocerebrosidase − GCD) for the treatment of Gaucher disease.

The study will evaluate the safety of PRX-112 and assess its dose dependant pharmacokinetics in 10 treatment-naive patients suffering from Gaucher disease. Protalix expects to complete the study in the third quarter of 2014.

Gaucher disease is a serious lysosomal storage disorder with severe and debilitating symptoms that affects 1 in every 20,000 live births in the U.S.

We note that Protalix already has an approved Gaucher disease product in its portfolio − Elelyso. Elelyso, an enzyme replacement therapy, eas approved in May 2012, by the FDA for the long-term treatment of adults diagnosed with type I Gaucher disease. Protalix is currently seeking pediatric approval for Elelyso in the U.S.

Another drug approved for the treatment of Gaucher disease is Shire's SHPG Vpriv. Vpriv has orphan drug status in the EU. Sanofi's SNY Cerdelga is under priority review in the U.S. for the same indication with a response expected in the second half of 2014.

Meanwhile, Protalix is working on strengthening its pipeline. PRX-102 is in a phase I/II study for the treatment of Fabry disease. The company intends to complete patient enrolment by year end. Final results from the study should be out in the first half of 2015.

Protalix carries a Zacks Rank #4 (Sell). A better-ranked stock in the biotech sector is Gilead Sciences Inc. GILD, carrying a Zacks Rank #1 (Strong Buy).