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]