Protalix Biotherapeutics

Gene Therapy Landscape Expands for Fabry Disease: Multiple Clinical Trials Show Promise in 2024

5 months ago

Multiple gene therapy trials for Fabry disease demonstrated significant progress in 2024, with AMT-191, 4D-310, and ST-920 showing promising safety and efficacy data. These therapies aim to address the underlying genetic cause of Fabry disease through different approaches, potentially offering long-term treatment options for patients.

Protalix BioTherapeutics Advances Pipeline and Receives EMA Validation for Fabry Disease Treatment

5 months ago

• Protalix BioTherapeutics announced the validation by the EMA of Chiesi's variation submission for pegunigalsidase alfa, aiming for less frequent dosing for Fabry disease. • A Phase I clinical trial of PRX-115, a recombinant PEGylated uricase for uncontrolled gout, showed promising safety and efficacy results with potential for wide dosing intervals. • Protalix is refining its R&D strategy, focusing on plant-based drug delivery systems for renal rare diseases, leveraging its ProCellEx platform. • The company reported satisfaction with sales growth of Elfabrio to Chiesi, Elelyso to Pfizer, and Uplyso to Brazil, and has fully repaid its outstanding convertible notes.

EMA Validates Application for Less Frequent Elfabrio Dosing in Fabry Disease

5 months ago

• The EMA has validated an application for a less frequent Elfabrio (pegunigalsidase alfa) regimen for Fabry disease, potentially reducing treatment burden. • The proposed regimen involves administering Elfabrio every four weeks at 2 mg/kg, compared to the current bi-weekly regimen of 1 mg/kg. • The application is supported by data from the Phase 3 BRIGHT trial and its ongoing open-label extension study, demonstrating safety and efficacy. • If approved, this new regimen could offer Fabry patients in the EU a beneficial, alternative dosing option.

EMA Validates Less Frequent Dosing Regimen for Elfabrio in Fabry Disease

6 months ago

• The EMA has validated a variation submission for Elfabrio, proposing a less frequent dosing regimen for adult Fabry disease patients. • The new regimen suggests 2 mg/kg every four weeks, an alternative to the current 1 mg/kg bi-weekly dose. • Validation is supported by Phase 3 BRIGHT trial data, showing potential benefit for patients previously treated with other therapies. • This milestone aims to reduce treatment burden and improve the quality of life for Fabry disease patients.

Protalix's PRX-115 Demonstrates Dose-Dependent Urate-Lowering in Phase 1 Gout Study

6 months ago

• Protalix BioTherapeutics' PRX-115, a PEGylated uricase, shows promising dose-dependent urate-lowering effects in a Phase 1 study for individuals with gout. • The first-in-human study demonstrated that PRX-115 was well-tolerated, with most adverse events being mild to moderate and transient. • Single doses of PRX-115 rapidly reduced plasma urate levels, with the highest doses maintaining levels below 6.0 mg/dL for up to 12 weeks. • Protalix is actively planning a Phase 2 clinical trial of PRX-115 in gout patients, expected to begin in the second half of 2025.

Advancements in Clinical Trials for Fabry Disease, C. difficile Infections, and Mydriasis

6 months ago

• Clinical trials are actively progressing for Fabry Disease, with companies like Idorsia and Sanofi Genzyme developing novel therapies. • Emerging treatments like Venglustat and Pegunigalsidase alfa are expected to significantly impact the Fabry Disease market in the coming years. • Research and development efforts are also focused on Clostridium Difficile Infections, with Vedanta Biosciences leading Phase 3 trials for VE303. • Mydriasis treatments are advancing, with companies like Eyenovia exploring solutions like MYDCOMBI for pupil dilation.

Protalix's PRX-115 Demonstrates Promising Results in Phase I Gout Trial

6 months ago

• Protalix Biotherapeutics' PRX-115 shows dose-dependent reduction in plasma urate levels in Phase I trial for severe gout. • The Phase I trial demonstrated that PRX-115 was well-tolerated, with most adverse events being mild and resolving completely. • PRX-115 exhibited prolonged presence in plasma, up to 12 weeks, suggesting potential for less frequent dosing compared to existing treatments. • Protalix is planning a Phase II trial for PRX-115, with the trial design expected in the coming quarters and a launch anticipated in the second half of 2025.

Protalix BioTherapeutics Announces Positive Phase 1 Results for PRX-115 in Uncontrolled Gout

6 months ago

• Protalix BioTherapeutics reports completion of Phase 1 trial for PRX-115, a recombinant uricase candidate for uncontrolled gout, with encouraging preliminary results. • The Phase 1 study demonstrated dose-dependent increases in PRX-115 exposure and rapid reduction of plasma uric acid levels, with levels remaining below 6.0 mg/dL for up to 12 weeks at the highest doses. • PRX-115 was well-tolerated, with mostly mild to moderate adverse events; Protalix is actively planning a Phase 2 clinical trial in gout patients for the second half of 2025. • Protalix reported a net income of $3.2 million for the third quarter of 2024, driven by increased revenues from sales of goods, particularly to Chiesi Farmaceutici and Pfizer.

Less Frequent Elfabrio Dosing Shows Promise in Fabry Disease

7 months ago

• A Phase 3 clinical study suggests that less frequent dosing of Elfabrio (pegunigalsidase alfa) is safe and effective for adults with stable Fabry disease. • The BRIGHT study evaluated Elfabrio administered every four weeks at 2mg/kg, compared to the approved bi-weekly regimen of 1mg/kg. • Results indicated that Elfabrio maintained effective bloodstream levels, with mostly mild to moderate side effects and no new immune reactions. • Further research with a larger patient population is needed to confirm these findings and explore less-frequent dosing as a treatment option.

Sangamo Therapeutics' Fabry Disease Candidate ST-920 Gains Accelerated Approval Pathway

7 months ago

• Sangamo Therapeutics' shares surged after the FDA agreed to a regulatory pathway for accelerated approval of isaralgagene civaparvovec (ST-920) for Fabry disease. • The FDA will consider data from the Phase I/II STAAR trial, using the rate of decline in eGFR at 52 weeks as the primary basis for approval. • Sangamo plans to submit a BLA in the second half of 2025, three years ahead of previous estimates, potentially bringing the treatment to patients sooner. • Septerna, focusing on GPCR therapies, raised $288 million in an upsized IPO, highlighting renewed interest in biotech IPOs.

Protalix Bio Presents Positive Phase 1 Data for PRX-115 in Uncontrolled Gout

7 months ago

• Protalix BioTherapeutics announced Phase 1 clinical trial data for PRX-115, a novel PEGylated uricase, showing promise in lowering urate levels. • The study evaluated PRX-115 in participants with elevated urate, demonstrating prolonged plasma urate-lowering effects after a single intravenous dose. • These findings were presented at the American College of Rheumatology (ACR) Convergence 2024, highlighting the potential of PRX-115 for uncontrolled gout. • Protalix plans to make the poster presentation available on their website, offering further insights into the study's results and methodology.