Protalix BioTherapeutics(US:PLX)2025-11-13 11:58Product Development and Commercialization - The company has successfully developed two commercial products: Elfabrio® (pegunigalsidase alfa) for Fabry disease and Elelyso® (taliglucerase alfa) for Gaucher disease, with Elelyso approved in 23 markets and Elfabrio approved in multiple regions including the US and EU[74]. - The company submitted an Investigational New Drug (IND) application for PRX-115, which has become effective, and plans to initiate a phase 2 clinical trial in Q4 2025[70][79]. - The company has licensed the rights to commercialize Elelyso worldwide (excluding Brazil) to Pfizer and has partnered with Chiesi for the development and commercialization of Elfabrio[75][77]. - The company is developing additional products, including PRX-119 for NETs-related diseases, leveraging its ProCellEx technology for future innovations[79][84]. - The company is focused on expanding its product pipeline to address high unmet needs in rare and orphan diseases, including renal diseases[84]. - The company is currently recruiting for the Pediatric FLY Study to assess the safety and efficacy of pegunigalsidase alfa in patients aged 2 to less than 18 years with confirmed Fabry disease[136]. Clinical Trials and Efficacy - The BALANCE study for PRX-102 showed a favorable tolerability profile, with 90.4% of patients in the PRX-102 arm experiencing at least one treatment-emergent adverse event compared to 96.0% in the agalsidase beta arm[109]. - In the BALANCE study, the number of infusion-related reactions adjusted to 100 infusions was 0.5 for the PRX-102 arm versus 3.9 for the agalsidase beta arm, indicating a better safety profile for PRX-102[111]. - The phase 3 clinical program for PRX-102 included three studies, with the BALANCE study demonstrating comparability in treatment effects to agalsidase beta for primary efficacy renal endpoints[115]. - The FDA determined that substantial evidence of effectiveness for Elfabrio was established through a phase 1/2 clinical trial and the BALANCE study[98]. - The BRIDGE study showed a significant improvement in renal function, with the mean annualized eGFR slope improving from -5.90 mL/min/1.73m²/year on agalsidase alfa to -1.19 mL/min/1.73m²/year on PRX-102[118]. - In the BRIDGE study, male patients' eGFR improved from -6.36 mL/min/1.73m²/year to -1.73 mL/min/1.73m²/year, while female patients improved from -5.03 mL/min/1.73m²/year to -0.21 mL/min/1.73m²/year[118]. - The overall mean eGFR slope at the end of the BRIGHT study was -2.92 mL/min/1.73m²/year, indicating stability in renal function[125]. - Following the transition to PRX-102, none of the patients without ADAs at screening developed treatment-induced ADAs[122]. Financial Performance - Revenues from selling goods for the three months ended September 30, 2025, were $17.7 million, a decrease of 1% from $17.8 million in the same period in 2024, primarily due to a $3.6 million decrease in sales to Chiesi[174]. - Research and development expenses for the three months ended September 30, 2025, were approximately $4.5 million, an increase from approximately $3.0 million in the same period in 2024[177]. - Revenues from selling goods reached $43.1 million for the nine months ended September 30, 2025, an increase of $8.3 million, or 24%, compared to $34.8 million for the same period in 2024[183]. - Selling, general and administrative expenses were $2.9 million for the three months ended September 30, 2025, an increase of $0.3 million, or 12%, compared to $2.6 million for the same period in 2024[180]. - The company had $29.4 million in cash and cash equivalents and short-term bank deposits as of September 30, 2025[192]. - Net cash used in operations was $14.0 million for the nine months ended September 30, 2025, with a net loss of $1.1 million[195]. - The company sold 2,775,215 shares of Common Stock under the Sales Agreement, generating gross proceeds of approximately $7.0 million during the nine-month period ended September 30, 2025[193]. Market Insights - The global market for Gaucher disease treatments is projected to be $1.7 billion in 2025, with a CAGR of approximately -1.82% from 2024 to 2030[96]. - The global market for Fabry disease treatments is forecasted to reach approximately $2.2 billion in 2025, growing at a CAGR of 8.19% from 2024 to 2030, potentially reaching $3.4 billion by 2030[102]. - The prevalence of Gaucher disease ranges from 0.70 to 1.75 per 100,000 in the general population, with higher estimates in individuals of Ashkenazi Jewish heritage[95]. - Market research indicates that approximately 25% of the gout population in the US and Western Europe do not have their condition controlled[163]. Research and Development - The company utilizes its proprietary ProCellEx® platform for the production of recombinant therapeutic proteins, being the first to gain FDA approval for a protein produced through plant cell-based expression[72][85]. - The company is committed to enhancing its ProCellEx technology to improve the development and manufacturing of therapeutic proteins[92]. - The company expects to continue incurring significant research and development expenses as it advances its product candidates[188]. - Total research and development expenses for the nine months ended September 30, 2025, were approximately $13.9 million, an increase of $5.1 million, or 58%, compared to $8.8 million for the same period in 2024[186][187]. - The phase 1 clinical trial of PRX-115 demonstrated that 25% of subjects reported drug-related adverse events, with the majority being mild to moderate[159]. - PRX-115 showed a dose-dependent reduction in plasma urate levels, remaining below 6.0 mg/dL for up to 12 weeks at the highest doses[154]. Partnerships and Agreements - The company has received $50 million in upfront payments and $45 million in development cost reimbursements from Chiesi, with potential milestone payments exceeding $1 billion[141]. - Under the Chiesi Ex-US Agreement, the company granted an exclusive license for PRX-102 commercialization outside the U.S., with an upfront payment of $25 million[142]. - The Fill/Finish Agreement with Chiesi includes a 10-year initial term for commercial fill/finish services for PRX-102, with potential extension options[146]. - Protalix Ltd. received an upfront payment of $25.0 million from Chiesi for the exclusive license to develop PRX-102, with potential additional payments of up to $760.0 million in milestone payments[144].