Committee for Medicinal Products for Human Use (CHMP) issues a positive opinion following re-examination, which will be reviewed by the European Commission (EC), with a decision anticipated by March 2026 If approved by the EC, this dosing regimen would reduce the burden to eligible adult patients, their families and the broader healthcare system due to the current requirement to visit infusion centres or have home infusions every-2-weeks for treatment PARMA, Italy and CARMIEL, Israel, Jan. 30, 2026 (GLOBE N ...

Core Insights - Protalix BioTherapeutics and Secarna Pharmaceuticals have entered into a collaboration and option agreement to develop antisense oligonucleotide therapies for rare renal diseases, leveraging Protalix's expertise in rare diseases and Secarna's AI-powered OligoCreator platform [1][2][3] Company Overview - Protalix BioTherapeutics focuses on innovative therapeutics for rare diseases and has developed two enzyme replacement therapies available in multiple markets, utilizing a proprietary plant cell-based expression system called ProCellEx [4][5] - Secarna Pharmaceuticals specializes in oligonucleotide therapeutics and employs its OligoCreator platform to rapidly generate high-quality antisense candidates, aiming to transform untreatable conditions into treatable ones [7] Collaboration Details - The collaboration involves Protalix selecting pharmaceutical targets for rare renal indications, while Secarna will use its OligoCreator platform to design and profile ASO candidates against these targets [2] - Protalix has the option for an exclusive, worldwide license to further develop and commercialize the therapeutic programs resulting from this collaboration [2][3] Strategic Goals - The partnership aims to advance programs from preclinical stages to clinical trials, addressing significant unmet medical needs in the rare kidney disease space [2][3] - Both companies express excitement about the collaboration, highlighting the combination of Secarna's rapid candidate generation and Protalix's experience in bringing therapies to market [3]

Core Insights - Chiesi Global Rare Diseases and Protalix BioTherapeutics received a negative opinion from the CHMP of the EMA regarding the approval of a new dosing regimen for Elfabrio (pegunigalsidase alfa) [1][2] - The proposed regimen of 2 mg/kg every 4 weeks was not deemed to have sufficient efficacy compared to the currently approved regimen of 1 mg/kg every 2 weeks [1][2] Company Overview - Chiesi Global Rare Diseases is a unit of the Chiesi Group focused on innovative therapies for rare diseases [1][16] - Protalix BioTherapeutics specializes in developing recombinant therapeutic proteins using its proprietary ProCellEx® plant cell-based expression system [1][19] - Both companies are committed to reducing the treatment burden for patients with Fabry disease [2][20] Clinical Trial Insights - The CHMP review was based on data from the BRIGHT trial and its extension study, which had a median exposure of almost six years [2] - The data from these studies, along with modeling and exposure-response analyses from prior trials, were insufficient to demonstrate similar efficacy for the new dosing regimen [2] Fabry Disease Context - Fabry disease is a rare lysosomal storage disorder caused by mutations in the GLA gene, leading to serious health complications [11][12] - Early detection and access to treatment are critical for managing symptoms and slowing disease progression [12] Safety Information - Elfabrio is indicated for adults with confirmed Fabry disease and has been associated with hypersensitivity reactions, including anaphylaxis [3][4] - In clinical trials, 14% of patients experienced hypersensitivity reactions, with 3% experiencing anaphylaxis [6][9]

Core Viewpoint - Protalix BioTherapeutics, Inc. will release its financial results for the quarter ended June 30, 2025, and provide a business and clinical update on August 14, 2025 [1] Group 1: Financial Results and Conference Call - The management will host a conference call to discuss the financial results and provide updates on recent corporate and regulatory developments [2] - The conference call is scheduled for August 14, 2025, at 8:30 a.m. EDT [3] Group 2: Webcast and Participation Details - The conference will be webcast live from the Protalix website, and participants are encouraged to access the call at least 15 minutes early [4] - A replay of the call will be available for two weeks on the Events Calendar of the Company's website [4] Group 3: Company Overview - Protalix is focused on the development and commercialization of recombinant therapeutic proteins using its proprietary ProCellEx plant cell-based expression system [5] - The company has FDA approval for its first product, taliglucerase alfa, for the treatment of Gaucher disease, and its second product, Elfabrio®, was approved in May 2023 [5][6] - Protalix has partnered with Chiesi Farmaceutici S.p.A. for the global development and commercialization of Elfabrio [6]