Full Press Release Details
Protalix BioTherapeutics and Chiesi Global
Rare Diseases Announce Submission of Biologics License Application to U.S. Food and Drug Administration for Pegunigalsidase Alfa
for the Treatment of Fabry Disease
CARMIEL, Israel, May 28, 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 the submission on May 27,
2020 of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for pegunigalsidase alfa for
the proposed treatment of adult patients with Fabry disease via the FDA's Accelerated Approval pathway. Pegunigalsidase
alfa, or PRX-102, was granted Fast Track designation by the FDA in January 2018. Pegunigalsidase alfa is the Company's purposefully-designed,
long-acting recombinant, PEGylated, cross-linked -galactosidase-A investigational product candidate.
The 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. Upon the BLA approval, if approved, the Company will be eligible to receive a milestone
payment from Chiesi.
"We are grateful for the assistance
the FDA provided leading up to the submission of this BLA via the Accelerated Approval pathway, and we look forward, together with
Chiesi, to working with the FDA as we seek marketing approval for PRX-102," said Dror
Bashan, Protalix's President and Chief Executive Officer. "Together with Chiesi, we thank the investigators and study
participants who have made reaching this milestone possible and have supported Protalix in our commitment to bringing this new
treatment option to the Fabry patient community."
"The submission of this BLA to the FDA represents a significant
milestone for our Global Rare Diseases division that was established earlier this year to strengthen Chiesi's focus on making
a difference for patients living with rare diseases around the world," said Giacomo Chiesi, head of Chiesi Global Rare Diseases.
"Our partnership and active collaboration with Protalix are a great example showing how we can leverage Chiesi's global
reach and decades of experience in drug development to support patients and their families living with Fabry disease and many other
devastating rare diseases."
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.
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.
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 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; 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
in the currently anticipated timeline or at all and, if approved, whether PRX-102 will 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 not accept an application for accelerated approval of PRX-102
with the data generated to date or 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 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 related to our ability to maintain
and manage our relationship with Chiesi Farmaceutici and any other collaborator, distributor or partner; risks related to the amount
of our future revenues and expenditures; the risk that despite the FDA's grant of Fast Track designation for PRX-102, we
may not experience a faster development process, review or approval compared to applications considered for approval under conventional
FDA procedures; risks related to the FDA's ability to withdraw the Fast Track designation at any time; our dependence on
performance by third party providers of services and supplies, including without limitation, clinical trial services; delays in