Full Switch Plan Initiated and Published by Brazilian Ministry of Health for Gaucher Patients in
Brazil Yielding Significant Revenue Stream
Fabry Phase III Clinical Trial Underway with Interim Results Expected in 2018
Phase II Clinical Trial for Cystic Fibrosis Positive Interim Data Reported
with Full Results Expected During First Quarter of 2017
Phase II Clinical Trial of Oral anti TNF for Ulcerative Colitis Initiated
with Results Expected in Second Half of 2017
New Pipeline Candidates for Indications for which there are Currently No Approved Drugs or that
address Significant Unmet Medical Needs anticipated to be announced publicly and to progress into Clinical
Development
CARMIEL, Israel, Jan. 09, 2017 (GLOBE NEWSWIRE) -- Protalix BioTherapeutics, Inc. (NYSE MKT:PLX) (TASE:PLX) today announced a
review of the Company’s clinical and financial highlights for 2016, as well as an outlook for anticipated future strategic
milestones.
“2016 was a building year for Protalix. We aggressively pushed our clinical pipeline programs forward, which included high
level regulatory agency discussions and the initiation of clinical trials for all three of our disclosed leading assets,” said
Moshe Manor, Protalix’s President and Chief Executive Officer. “Given our recent financing and the projected revenue stream
from sales of alfataliglicerase in Brazil, we are well capitalized to deliver on our anticipated, value building milestones.
Over the course of 2017 and into 2018, we expect to announce data from our phase III clinical trial on Fabry disease, final results
from our phase II clinical trial of alidornase alfa for the treatment of Cystic Fibrosis and results from our phase II clinical
trial of OPRX-106 for the treatment of ulcerative colitis. We expect that 2017 will be an extremely important, inflection
year for us, with a number of significant commercial and clinical milestones that should bring considerable value to our
stockholders.”
2016 and Recent Clinical Highlights
- Received purchase order from the Brazilian Ministry of Health for purchase of alfataliglicerase to treat Gaucher
patients in Brazil. The order consists of a number of shipments during 2017, in increasing volumes, for a total of
approximately $24.3 million. The size of the final shipment of the order represents annual revenues of
approximately $42.0 million. The Brazilian Ministry of Health’s order was published in the December publication of
Brazil’s Official Diary of the Union (Diário Oficial).
- Reported positive interim results from the Company’s phase II clinical trial of alidornase alfa (PRX-110) for the treatment
of Cystic Fibrosis, which results include a clinically meaningful increase in percent predicted forced expiratory volume in one
second (ppFEV1).
- Initiated patient enrollment in the Company’s phase II clinical trial of OPRX-106 for the treatment of ulcerative
colitis. The Company is developing OPRX-106 to be the first ever oral protein treatment, as currently there are no other
oral protein treatments available.
- Received regulatory approval of alfataliglicerase in Brazil for children four years old and above with a confirmed diagnosis
of Type I Gaucher disease.
- Worked closely with the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) to determine the phase
III clinical trial program for pegunigalsidase alfa (PRX-102) for the treatment of Fabry disease:
- Initiated and enrolled patients in a global phase III trial of pegunigalsidase alfa versus Fabrazyme® to support a BLA
filing in the United States, with a 12-month non-inferiority interim analysis to support an MAA filing with the EMA.
- Determined a 12-month switch-over study from Replagal® will also be conducted to support FDA and EMA filings.
- Presented positive 6- and 12-month interim clinical data on pegunigalsidase alfa at the 12th Annual WORLD Symposium™ in March
2016.
- Exchanged $54.1 million principal amount of the Company’s $69.0 million 4.50% Senior Convertible Notes due 2018
for $40.2 million principal amount of newly issued 7.50% Senior Secured Convertible Notes due 2021 and approximately
23.8 million shares of common stock. Concurrently, the Company sold, in a private placement, $22.5
million principal amount of 2021 Notes.
- The Company had a strong cash balance of approximately $63.0 million as of December 31, 2016, which is currently projected to
fund operations into late 2019.
2017 Outlook and Projected Future
Milestones
- Full results for the phase II efficacy and safety study of alidornase alfa expected to be announced during the first quarter
of 2017.
- Full results for the phase II efficacy and safety study of OPRX-106 expected to be announced during the second half of
2017.
- Completion of enrollment in the phase III trial of pegunigalsidase alfa for the treatment of Fabry disease expected in 2017;
interim data analysis anticipated in 2018 to support EMA and other regulatory filings outside of the United States.
- If superiority to Fabrazyme is not yet achieved at that time, as shown in the interim data, the trial will continue for
an additional year before reporting two-year data to support a U.S. regulatory filing.
- Present, via two key opinion leader (KOL) presentations and a poster, pegunigalsidase alfa clinical data at the 13th Annual
WORLD Symposium™ to be held in February 2017.
- Present at the 5th Update on Fabry Nephropathy: Biomarkers, Progression and Treatment Opportunities in April 2017.
- Present, at the 40th European Cystic Fibrosis Conference, full data from the Company’s phase II efficacy and safety study of
alidornase alfa, in June 2017, and at the North American Cystic Fibrosis Conference in November 2017.
- Potential partnership for alidornase alfa.
- Potential collaboration for alidornase alfa with the Cystic Fibrosis Foundation.
- Potential partnership for oral anti-TNF.
A copy of the Company’s January 2017 Corporate Update will be posted in the Presentations page of the Investors tab of its
corporate website.
About Protalix BioTherapeutics, Inc.
Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins
expressed through its proprietary plant cell-based expression system, ProCellEx®. Protalix’s unique expression system
presents a proprietary method for developing recombinant proteins in a cost-effective, industrial-scale manner. Protalix’s
first product manufactured by ProCellEx, taliglucerase alfa, was approved for marketing by the U.S. Food and Drug Administration
(FDA) in May 2012 and, subsequently, by the regulatory authorities of other countries. Protalix has licensed to Pfizer Inc.
the worldwide development and commercialization rights for taliglucerase alfa, excluding Brazil, where Protalix retains full
rights. Protalix’s development pipeline includes the following product candidates: pegunigalsidase alfa, a modified version
of the recombinant human alpha-GAL-A protein for the treatment of Fabry disease; OPRX-106, an orally-delivered anti-inflammatory
treatment; alidornase alfa for the treatment of Cystic Fibrosis; and others.
Forward-Looking Statements
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. The terms “expect,”
“anticipate, “believe,” “estimate,” “project,” “plan,” “should” and “intend” and other words or phrases of similar import are
intended to identify forward-looking statements. 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 related to the
ultimate purchase by Fundação Oswaldo Cruz of alfataliglicerase pursuant to the stated purchase intentions of the Brazilian
Ministry of Health of the stated amounts, if at all; risks related to the successful conclusion of our negotiations with the
Brazilian Ministry of Health regarding the purchase of alfataliglicerase generally; risks related to our commercialization efforts
for alfataliglicerase in Brazil; risks relating to the compliance by Fundação Oswaldo Cruz with its purchase obligations and
related milestones under our supply and technology transfer agreement; risks related to the amount and sufficiency of our cash and
cash equivalents; risks related to the amount of our future revenues, operations and expenditures; failure or delay in the
commencement or completion of our preclinical and clinical trials which may be caused by several factors, including: slower than
expected rates of patient recruitment; unforeseen safety issues; determination of dosing issues; lack of effectiveness during
clinical trials; inability to monitor patients adequately during or after treatment; inability or unwillingness of medical
investigators and institutional review boards to follow our clinical protocols; and lack of sufficient funding to finance clinical
trials; the risk that the results of the clinical trials of our product candidates will not support our claims of safety or
efficacy, that our product candidates will not have the desired effects or will be associated with undesirable side effects or
other unexpected characteristics; risks relating to our ability to make scheduled payments of the principal of, to pay interest on
or to refinance our outstanding notes or any other indebtedness; our dependence on performance by third party providers of services
and supplies, including without limitation, clinical trial services; delays in our preparation and filing of applications for
regulatory approval; delays in the approval or potential rejection of any applications we file with the FDA or other health
regulatory authorities, and other risks relating to the review process; our ability to identify suitable product candidates and to
complete preclinical studies of such product candidates; the inherent risks and uncertainties in developing drug platforms and
products of the type we are developing; the impact of development of competing therapies and/or technologies by other companies and
institutions; potential product liability risks, and risks of securing adequate levels of product liability and other necessary
insurance coverage; and other factors described in our filings with the U.S. Securities and Exchange Commission. The
statements in this press release are valid only as of the date hereof and we disclaim any obligation to update this information,
except as may be required by law.
Investor Contact Marcy Nanus The Trout Group, LLC 646-378-2927 mnanus@troutgroup.com
