Full Press Release Details
Protalix BioTherapeutics and Chiesi Global
Rare Diseases Announce
U.S. Food and Drug Administration Acceptance of Biologics License
Application (BLA) for Pegunigalsidase Alfa for the Proposed Treatment
Fabry Disease and Grants Priority Review
CARMIEL, Israel, August 11,
2020 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the
development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx
plant cell-based protein expression system, or the Company, together with its development and commercialization partner Chiesi
Global Rare Diseases, a unit of Chiesi, an international research-focused healthcare group, today announced that the U.S. Food
and Drug Administration (FDA) has accepted the Biologics License Application (BLA) and granted Priority Review designation for
pegunigalsidase alfa for the proposed treatment of adult patients with Fabry disease. The BLA was submitted via the FDA's
Accelerated Approval pathway. Pegunigalsidase alfa is the Company's purposefully designed, long-acting recombinant, PEGylated,
cross-linked -galactosidase-A investigational product candidate. The FDA set an action date of January 27, 2021, under the
Prescription Drug User Fee Act (PDUFA). The FDA also indicated in the BLA filing communication letter that it is not currently
planning to hold an advisory committee meeting to discuss the application.
Review is granted to therapies that the FDA determines have the potential to provide significant improvements in the treatment,
diagnosis or prevention of serious conditions. This designation shortens the FDA review period following the acceptance of the
BLA to six months compared to 10 months under standard review. Pegunigalsidase alfa was granted Fast Track designation by
the FDA in January 2018.
BLA submission includes a comprehensive set of preclinical, clinical
and manufacturing data compiled from the Company's completed Phase I/II clinical
trial of pegunigalsidase alfa, including the related extension study succeeding the Phase I/II
clinical trial, interim clinical data from the
Phase III BRIDGE switch-over
study and safety data from the Company's on-going clinical studies of PRX 102 in patients receiving 1 mg/kg
"The FDA's acceptance of
the BLA and grant of priority review for PRX-102 are significant achievements for Protalix and Chiesi, and represent a crucial
step forward as we look to establish a new treatment option to the Fabry patient community," said Dror Bashan, Protalix's
President and Chief Executive Officer. "Based on the encouraging results for PRX-102 we have seen to date, we are eager to
continue discussions with the FDA and to continue our other development efforts for PRX-102, as marketing approval of PRX-102 is
"PRX-102 represents an
important advance in research with the potential to deliver significant advantages to patients with Fabry disease,"
said Giacomo Chiesi, Head of Global Rare Diseases. "We are very encouraged by the strong interest in this therapy among
both patients and clinicians and we look forward to the prospect of making it available to patients around the world who can
benefit from treatment."
Fabry disease is an X-linked inherited disease that results
from deficient activity of the lysosomal Galactosidase A enzyme resulting in progressive accumulation of abnormal
deposits of a fatty substance called globotriaosylceramide (Gb3) in blood vessel walls throughout a person's body.
Fabry disease occurs in one person per 40,000 to 60,000. Fabry patients inherit a deficiency of the Galactosidase A
enzyme, which is normally responsible for the breakdown of Gb3. The abnormal storage of Gb3 increases with
time and, accordingly, Gb3 accumulates, primarily in the blood and in the blood vessel walls. The ultimate consequences
of Gb3 deposition range from episodes of pain and impaired peripheral sensation to end-organ failure - particularly
of the kidneys, but also of the heart and the cerebrovascular system.
About Pegunigalsidase Alfa
Pegunigalsidase alfa (PRX 102)
is an investigational, plant cell culture-expressed, and chemically modified stabilized version of the recombinant -Galactosidase-A
enzyme. Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with
unique pharmacokinetic parameters. In clinical studies, PRX 102 has been observed to have
a circulatory half-life of approximately 80 hours. The Company designed PRX 102 to potentially address the continued unmet
clinical need in Fabry patients.
About Chiesi Global Rare Diseases
Chiesi Global Rare Diseases is a business unit of the Chiesi
Group established in February 2020 and focused on research and development of treatments for rare and ultra-rare disorders. The
Global Rare Diseases unit works in collaboration with Chiesi Group to harness the full resources and capabilities of our global
network to bring innovative new treatment options to people living with rare diseases, many of whom have limited or no treatments
available. The unit is also a dedicated partner with global leaders in patient advocacy, research and patient care. For more information
Based in Parma, Italy, Chiesi Farmaceutici is an international
research-focused healthcare group with 85 years of experience in the pharmaceutical industry and a global presence in 29 countries.
Chiesi researches, develops, and markets innovative drugs in the respiratory therapeutics, specialist medicine, and rare disease
areas. Its R&D organization is headquartered in Parma (Italy), and is integrated with R&D groups in France, the USA, the
UK, and Sweden to advance Chiesi's pre-clinical, clinical, and registration programs. Chiesi employs nearly 6,000 people. Chiesi
Group is a certified Benefit Corporation. For more information www.chiesi.com.
About Protalix BioTherapeutics, Inc.
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 was the first company to gain U.S. Food
and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. Protalix's
unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner.
Protalix's first product manufactured by ProCellEx, taliglucerase
alfa, was approved for marketing by the 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 consists of proprietary
versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates:
pegunigalsidase alfa, a modified version of the recombinant human Galactosidase A protein for the proposed treatment
of Fabry disease; OPRX 106, an orally-delivered anti-inflammatory treatment; alidornase
alfa for the treatment of Cystic Fibrosis; PRX 115, a plant cell-expressed recombinant PEGylated Uricase for the treatment
of gout; and others. Protalix has partnered with Chiesi Farmaceutici S.p.A., both in the United States and outside the United States,
for the development and commercialization of pegunigalsidase alfa.
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 and the final results of a clinical
trial may be different than the preliminary findings for the clinical trial. Factors that might cause material differences
include, among others: that the FDA might not grant marketing approval for PRX 102 by
the PDUFA date or at all and, if approved, whether PRX 102 will may have significant
limitations on its use or be commercially successful; failure or delay in the commencement or completion of our preclinical
and clinical trials which may be caused by several factors, including: risks that the FDA will request additional data or
other conditions of our submission of any application for Accelerated Approval of PRX 102;
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; and inability or
unwillingness of medical investigators and institutional review boards to follow our clinical protocols; risks associated
with the novel coronavirus disease (COVID 19) outbreak, which may adversely impact
our business, preclinical studies and clinical trials; the risk that the results of the clinical trials of our product