Full Press Release Details
Protalix BioTherapeutics Announces Preliminary Top-Line Positive Data from taliglucerase alfa
CARMIEL, Israel, November 2, 2010 (PR NEWSWIRE) Protalix BioTherapeutics, Inc. (NYSE-AMEX: PLX,
TASE:PLX), announced today positive preliminary data from the first 15 patients that completed the
Company s nine month, worldwide, multi-center, open-label, switchover trial of taliglucerase alfa
for the treatment of Gaucher disease under a protocol cleared by the U.S. Food and Drug Administration (FDA). The data indicate that
patients can safely be switched to taliglucerase alfa from imiglucerase (Cerezyme ).
Patients enrolled in the trial were switched from imiglucerase (doses ranging from 10-60 U/kg every
other week) to an equivalent dose using the same number of units of taliglucerase alfa. The data
from the first 15 patients demonstrate that maintenance of efficacy was achieved over a nine month
period with no increased safety concerns. Patients hemoglobin and platelet counts remained stable
demonstrating hematological stability. As measured by MRI, mean spleen volume and liver volume
also remained stable. There was no evidence of increased safety concerns in patients switched from
Cerezyme to taliglucerase alfa and there were no drug related serious adverse events.
Hypersensitivity reactions were not reported in this patient group. One patient developed
non-neutralizing IgG antibodies to taliglucerase at the end of the study. Detailed data will be
presented at an upcoming medical meeting.
The switchover trial was originally designed and cleared by FDA to enroll 15 patients, however, it
was expanded to recruit a total of 30 patients as a result of the shortage of enzyme replacement
therapy for Gaucher patients. Adult enrollment in the study has closed; pediatric enrollment
We are pleased with the interim results of the switchover trial to taliglucerase alfa, said Dr.
David Aviezer, the Company s President and Chief Executive Officer. Through our Phase III pivotal
and extension trial, pediatric study in na ve patients, switch over trial, expanded access and
named patient programs, ATU program in France and supply agreement in Brazil, we are generating a
robust clinical database for taliglucerase alfa.
Taliglucerase alfa is under review by the U.S. Food and Drug Administration with a Prescription
Drug User Fee Action (PDUFA) date scheduled for February 25, 2011.
Protalix is a biopharmaceutical company focused on the development and commercialization of
recombinant therapeutic proteins expressed through its proprietary plant cell based expression
system. Protalix s ProCellEx presents a proprietary method for the expression of recombinant
proteins that Protalix believes will allow for the cost-effective, industrial-scale production of
recombinant therapeutic proteins in an environment free of mammalian components and viruses.
Protalix is also advancing additional recombinant biopharmaceutical drug development programs.
Taliglucerase alfa is an enzyme replacement therapy in development under a Special Protocol
Assessment with the FDA for Gaucher disease. The Company s new drug application
taliglucerase alfa has been accepted by the U.S. Food and Drug
Administration (FDA) and
granted a Prescription Drug User Fee ACT (PDUFA) action date of February 25, 2011.
Safe Harbor Statement
To the extent that statements in this press release are not strictly historical, all such
statements are forward-looking, and are made pursuant to the safe-harbor provisions of the Private
Securities Litigation Reform Act of 1995. These forward-looking statements are subject to known and
unknown risks and uncertainties that may cause actual future experience and results to differ
materially from the statements made. These statements are based on our current beliefs and
expectations as to such future outcomes. Drug discovery and development involve a high degree of
risk. Factors that might cause material differences include, among others, risks relating to: the
successful preclinical development of our product candidates; the completion of our clinical
trials; the review process of the FDA, the EMEA, other foreign regulatory bodies and other
governmental regulatory bodies, including the FDA s and the EMEA s review of any filings we make in
connection with the treatment protocol for taliglucerase alfa and including the risk that
regulatory authorities may find that the data from our clinical trials and other studies is
insufficient for regulatory approval; delays in the FDA s, the EMEA s or other health regulatory
authorities approval of any applications we file or refusals to approve such filings, including
the NDA we filed with the FDA for taliglucerase alfa for the treatment of Gaucher disease; refusals
by such regulatory authorities to approve the marketing and sale of a drug product even after
acceptance of an application we file for any such drug product; and other factors described in our
filings with the Securities and Exchange Commission. Companies in the pharmaceutical and
biotechnology industries have suffered significant setbacks in advanced or late-stage clinical
trials, even after obtaining promising earlier trial results or in preliminary findings for such
clinical trials. Further, even if favorable testing data is generated by clinical trials of drug
products, the FDA, EMEA or any other foreign regulatory authority may not accept or approve an NDA
filed by a pharmaceutical or biotechnology company for such drug product. Failure to obtain
approval from the FDA, EMEA or any other foreign regulatory authority of any of our drug candidates
in a timely manner, if at all, will severely undermine our business and results of operations by
reducing our potential marketable products and our ability to generate corresponding product
revenues. The statements in this release are valid only as of the date hereof and we disclaim any
obligation to update this information.
The Trout Group, LLC
Telephone: 646-378-2927
Email: mnanus@troutgroup.com