Protalix and Chiesi Submit BLA to the FDA 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 α-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.”

About Fabry Disease

Fabry disease is an X-linked inherited disease that results from deficient activity of the lysosomal α‑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 α‑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 α-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.

About Chiesi Group

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 α‑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.

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