Protalix BioTherapeutics(PLX) - 2025 Q1 - Quarterly Report

Product Development and Approval - The company has successfully developed two commercial products: Elfabrio® for Fabry disease and Elelyso® for Gaucher disease, with Elelyso approved in 23 markets including the US and Brazil[62] - Elfabrio has received marketing approval in multiple regions including the US, EU, and Russia, indicating a broad market acceptance[62] - The FDA determined that substantial evidence of effectiveness for Elfabrio was established through a well-controlled study, with the BALANCE study meeting its primary efficacy endpoint[86] - The company has licensed the rights to commercialize Elelyso worldwide (excluding Brazil) to Pfizer and in Brazil to Fiocruz[62] - The company is focused on developing treatments for rare and orphan diseases, leveraging its proprietary ProCellEx technology for new product candidates[66] - The company is currently in advanced stages of preparations for a Phase II clinical trial of PRX-115, expected to commence in the second half of 2025[64] - The company plans to submit a Variation Submission to the EMA to add a new dosing regimen of 2 mg/kg every four weeks for adult patients with Fabry disease[93] Market Analysis - The global market for Gaucher disease treatments was valued at $1.7 billion in 2024 and is forecasted to grow at a compound annual growth rate (CAGR) of approximately -0.46% from 2024 to 2030[83] - The global market for Fabry disease treatments is projected to reach approximately $2.3 billion by 2025, growing at a CAGR of 6.6% to about $3.1 billion by 2030[90] - Elelyso is the only alternative enzyme replacement therapy (ERT) for Gaucher disease besides Cerezyme and Vpriv, highlighting its unique market position[84] Clinical Trials and Efficacy - The clinical development program for PRX-102 included three pivotal studies: BALANCE, BRIDGE, and BRIGHT, focusing on safety and efficacy compared to existing treatments[95] - The BALANCE study demonstrated a favorable tolerability profile for PRX-102, with 90.4% of patients experiencing at least one treatment-emergent adverse event (TEAE) compared to 96.0% in the agalsidase beta arm[97] - In the BRIDGE study, the mean annualized eGFR slope improved from -5.90 mL/min/1.73m²/year on agalsidase alfa to -1.19 mL/min/1.73m²/year on PRX-102, indicating substantial renal function improvement[106] - The BRIGHT study evaluated the safety and efficacy of a 2 mg/kg dosage of PRX-102 administered every four weeks, although this regimen has not yet received regulatory approval[108] - Following the switch to PRX-102 in the BRIDGE study, there was a decrease in patients with progressing or fast-progressing kidney disease, aligning with therapeutic goals for Fabry disease[106] - The phase I/II study demonstrated a 90% reduction in lyso-Gb3 levels from baseline after 24 months[120] - The Mainz Severity Score Index (MSSI) showed an improvement of over 40% in disease severity across various parameters[120] - The BRIGHT study showed that 2 mg/kg of PRX-102 administered every four weeks was well tolerated, with no serious treatment-related adverse events reported[110] - Approximately 75% of study participants reported improvement or no change in average pain severity at Week 52[116] - Plasma lyso-Gb3 concentrations remained stable with a mean change of 3.01 nM from baseline to Week 52, indicating treatment stability[114] - Mean eGFR values were stable during the 52-week treatment period, with a mean change from baseline of -1.27 mL/min/1.73 m²[114] - Out of 30 patients, 29 completed the one-year study, with 28 receiving the intended regimen of 2 mg/kg every four weeks[111] Financial Performance - Revenues from selling goods increased to $10.0 million for the three months ended March 31, 2025, a rise of $6.3 million, or 170%, compared to $3.7 million for the same period in 2024[163] - Cost of goods sold rose to $8.2 million for the three months ended March 31, 2025, an increase of $5.6 million, or 215%, from $2.6 million for the same period in 2024[165] - Research and development expenses totaled approximately $3.5 million for the three months ended March 31, 2025, an increase of $0.6 million, or 21%, compared to $2.9 million for the same period in 2024[166][167] - Selling, general and administrative expenses decreased to $2.6 million for the three months ended March 31, 2025, a decrease of $0.5 million, or 16%, compared to $3.1 million for the same period in 2024[168] - Net cash used in operations for Q1 2025 was $5.1 million, with a net loss of $3.6 million[174] - Net cash provided by operations for Q1 2024 was $4.2 million, with a net loss of $4.6 million[175] - Significant research and development expenditures have been incurred without offsetting revenues, particularly for the products Elelyso and Elfabrio[176] - The company expects to continue incurring significant expenditures for research and development, with no anticipated revenues sufficient to cover these costs[178] Strategic Focus and Future Outlook - The corporate strategy includes developing treatments for rare diseases, focusing on early-stage product candidates with high unmet needs[136] - The company is exploring novel platform technologies and enhancing its ProCellEx capabilities to address high unmet needs in various diseases[66] - Future financing needs may arise from costs associated with clinical trials, employee salaries, and operational expenses[178] - The company has not engaged in hedging transactions to mitigate currency risk but may consider this in the future[184] - As of March 31, 2025, the company had $34.7 million in cash and cash equivalents and short-term bank deposits[171] - The company generated gross proceeds of approximately $3.0 million from the sale of 1,325,179 shares of Common Stock during the quarter ended March 31, 2025[172] - As of March 31, 2025, approximately $19.7 million in gross proceeds from Common Stock sales remain available under the Sales Agreement[179] - Approximately 44% of the company's costs are incurred in NIS, with potential inflation in Israel affecting U.S. dollar costs[184] - The average exchange rate of the U.S. dollar to NIS for Q1 2025 was 3.613, compared to 3.662 for Q1 2024[184] - The company has no off-balance sheet arrangements as of March 31, 2025[181]