Product Development and Commercialization - The company has successfully developed two commercial products: Elfabrio® for Fabry disease and Elelyso® for Gaucher disease, with Elfabrio generating 29.3millioninsalesin2024[42].−Elelysoisapprovedin23marketsglobally,with12.6 million generated from sales to Pfizer and 11.0millionfromsalesinBrazilin2024[41].−TheFDAapprovedElfabrioonMay9,2023,foradultpatientswithFabrydisease,coveringadosageof1mg/kgeverytwoweeks[22].−ThecompanyhaslicensedElelyso′scommercializationrightsworldwidetoPfizer,excludingBrazil,whereitismarketedasBioManguinhosalfataliglicerase[18].−Thecompany’sfirstcommercialproduct,Elelyso,isnowapprovedin23marketsincludingtheUnitedStates,Brazil,andIsrael[51].−Thecompany’ssecondcommercialproduct,Elfabrio,wasapprovedformarketingintheEUandtheUnitedStatesinMay2023foradultpatientswithFabrydisease[55].ClinicalTrialsandResearch−ThecompanyisdevelopingPRX−115,aPEGylateduricaseforuncontrolledgout,withanestimated251.7 billion in 2024 and is forecasted to grow at a compound annual growth rate (CAGR) of approximately -0.46% from 2024-2030[53]. - The global market for Fabry disease is forecasted to be approximately 2.3billionin2025,growingataCAGRof6.63.1 billion in annual sales by 2030[61]. - In September 2024, the company fully repaid all outstanding principal and interest under its 2024 Notes, financed entirely with available cash[49]. Regulatory and Compliance - The Biologics License Application for Elfabrio was resubmitted to the FDA on November 9, 2022, after an initial Complete Response Letter[21]. - The FDA requires substantial time and financial resources for regulatory approvals, with potential sanctions for non-compliance, including product recalls and clinical holds[160]. - The IND application must include preclinical test results, manufacturing information, and analytical data, becoming effective 30 days after submission unless placed on clinical hold by the FDA[161]. - The FDA review process for a BLA or NDA typically takes one to three years, with potential delays if additional data is required[172]. - The FDA may withdraw product approval if regulatory compliance is not maintained or if new problems arise post-approval[188]. Intellectual Property and Licensing - The company holds a robust patent portfolio of approximately 75 patents globally, with about 50 pending applications[125]. - New patents have been granted in the U.S., Australia, Chile, and Mexico for the treatment of Fabry disease using stabilized alpha-galactosidase[128]. - Protalix Ltd. has entered into two exclusive global licensing agreements with Chiesi for PRX-102, receiving 50millioninupfrontpaymentsanddevelopmentcostreimbursementsof45 million, with potential milestone payments exceeding 1billion[146].−UndertheChiesiUSAgreement,ProtalixLtd.iseligibleforupto760 million in regulatory and commercial milestone payments, with tiered payments of 15% to 40% of net sales depending on annual sales[148]. Manufacturing and Supply Chain - The manufacturing facility in Carmiel, Israel, has approximately 14,700 sq/ft of clean rooms and has been approved by the FDA as a multi-product facility since 2017, capable of meeting all current and expected commercial and clinical needs[152][153]. - Protalix Ltd. relies on a single approved supplier for certain materials but has identified additional suppliers for most raw materials required for production[155]. - The company is committed to identifying alternative approved suppliers to ensure an uninterrupted supply of necessary raw materials for drug development[156]. Taxation and Financial Incentives - The corporate tax rate in Israel is 23% for income not derived from "Approved Enterprises" as of 2018[205]. - Under the Investment Law, an Approved Enterprise may be exempt from corporate tax for a period of 2 to 10 years depending on its geographic location[209]. - The company has an Approved Enterprise program, allowing for tax benefits for a 10-year period if it qualifies as a foreign investors' company[210]. - A foreign investors' company with over 25% foreign ownership can benefit from a reduced corporate tax rate ranging from 10% to 23%[211].
Protalix BioTherapeutics(PLX)2025-03-17 11:18