BioMed News Bytes: Protalix (AMEX: PLX), Soligenix (OTC: SNGX.OB) Print E-mail
Thursday, 15 October 2009 09:39

On 10/15/09, Protalix BioTherapeutics (AMEX: PLX) announced positive top-line results of a pivotal Phase 3 study of its lead drug candidate, UPLYSO (Taliglucerase Alfa) in treatment naïve patients diagnosed with Gaucher disease.  In the study, UPLYSO met its primary endpoint of mean reduction in spleen volume compared to baseline after nine months in both treatment groups (60 U/kg and 30 U/kg doses). 

Statistically significant improvements compared with baselines were observed in hemoglobin level and liver size and significant nominal elevation in platelet count in the lower dose of 30 U/kg. The primary endpoint was stipulated in a Special Protocol Assessment (SPA) agreed on by Protalix and the FDA earlier this year.

Protalix is currently making available UPLYSO to Gaucher patients in the US and other countries through the FDA's Expanded Access Program due to the current shortage of Genzyme's Cerezyme, which was caused by the closing of a manufacturing plant. The safety analysis for both doses showed that UPLYSO was well tolerated and no serious adverse events were reported.  Only 6% of patients in the trial developed antibodies to UPLYSO during the study.  None of the patients in the trial developed neutralizing antibodies to UPLYSO.  Only 6% of the patients in the trial experienced hypersensitivity. Most adverse events were mild in intensity and not drug related and were transient in nature. Protalix will present more comprehensive results in the near future and at upcoming medical meetings while it expects to complete the ongoing rolling New Drug Application (NDA) submission for marketing clearance with FDA before the end of 2009.

On 10/15/09, Soligenix (OTC: SNGX.OB) announced that it has initiated enrollment in its confirmatory Phase 3 randomized, double-blind, placebo-controlled, multicenter clinical trial evaluating orBec for the treatment of acute gastrointestinal Graft-versus-Host disease (GI-GVHD). The initiation of enrollment in this trial also triggered the payment and receipt by Soligenix of a $1 million milestone pursuant to the Company's collaboration agreement with Sigma-Tau Pharma. This Phase 3 clinical trial, also referred to as the SUPPORTS protocol (Sparing Unnecessary Prednisone Phase 3 orBec Randomized Treatment Study), will enroll an estimated 166 patients to confirm the clinically meaningful endpoints observed in previous Phase 2 and Phase 3 clinical studies.

The primary endpoint is the treatment failure rate at Study Day 80. This endpoint was successfully measured as a secondary endpoint (p-value 0.005) in the Company's previous Phase 3 study as a key measure of durability following a 50-day course of treatment with orBec(®) (i.e., 30 days following cessation of treatment). The SUPPORTS trial will be conducted at major transplant centers throughout the US and is expected to complete with clinical data during 1H11. The SUPPORTS trial will be conducted pursuant to a Special Protocol Assessment (SPA) agreement that the Company had reached with FDA. The European Medicines Agency (EMEA) also agreed that should the new confirmatory Phase 3 study produce positive results, the data would be sufficient to support a marketing authorization approval in all 27 European Union (EU) member states.

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