Protalix BioTherapeutics, Inc. (PLX) announced encouraging initial results from a phase I study on its pipeline candidate, PRX-112 an oral glucocerebrosidase (:GCD) being evaluated for treating patients suffering from Gaucher disease.
We note that Protalix has one approved product, Elelyso/Uplyso, for the treatment of Gaucher disease in its portfolio. Protalix has a worldwide commercialization partnership (except Brazil and Israel) with Pfizer Inc. (PFE) for the product.
Oral GCD was observed to be safe and well-tolerated in all the 12 patients enrolled in the open label safety and pharmacokinetic phase I study. The study examined the delivery of plant cell expressed recombinant GCD following administration of Oral GCD in Gaucher patients.
No serious side effects were observed in the study. Enzyme presence was also observed in patients’ blood circulation and the observed enzyme showed biological activity. Additionally thrombocytopenia patients who had low platelet count exhibited a significant improvement in platelet count.
The study has been extended to enroll additional Gaucher patients with low platelet count. The study is expected to be completed by year end.
We note that Protalix is making substantial efforts to develop its pipeline and the company raised funds in Sep 2013 for the same.
Apart from PRX-112, the company has other interesting pipeline candidates like PRX-102 (phase I/II - Fabry disease).
Furthermore, the company added three new compounds to its pipeline, PRX-106 (immune mediated disorders), PRX-110 (cystic fibrosis) and PRX-107 (emphysema) in Jun 2013.
Protalix carries a Zacks Rank #3 (Hold). Currently, companies which look attractive include Actelion Ltd. (ALIOF) and AMAG Pharmaceuticals, Inc. (AMAG) with a Zacks Rank #1 (Strong Buy) and a Zacks Rank #2 (Buy), respectively.