(NYSE:PLX) $1.55 on January 10th, 2023
45,56 Million shares issued & outstanding
Market Cap = $71M;
Cash Runway into 3Q23
Convertible Notes: $28.7M due Sep. 2024
$1B+ in Milestone payments and 15-40% Royalties on PRX-102
Summary: Protalix is a biopharmaceutical company focused on the development and commercialization of proprietary recombinant therapeutic proteins expressed through its proprietary ProCellEx® plant cell-based protein expression system.
Using ProCellEx®, Protalix is developing a pipeline of novel proteins that target large, established pharmaceutical markets and that rely upon known biological mechanisms of action.
The Company’s ProCellEx® platform intends to solve key challenges associated with current enzyme replacement therapy (ERT),producing recombinant protein therapies with improved stability, reduced immunogenicity, and less frequent dosing.
· 1H23: Anticipated EU & US Commercial Launch of PRX-102
Why meet Protalix now?
· Clinically-Validated Platform with Scalable Production Capacity: Protalix’s platform technology ProCellEx® is a proprietary plant cell-based expression system for industrial scale manufacturing of recombinant therapeutic proteins.
Protalix is the only company to achieve FDA approval of a therapeutic protein produced through this method.
· Strong Strategic Partnerships to Drive Commercialization Strategy: Protalix has a committed financial and commercial partner in Chiesi Farmaceutici S.p.A., an international research-focused pharmaceuticals and healthcare group with demonstrated expertise in marketing ERTs.
· Pipeline Expansion Opportunities: Protalix is leveraging its unique platform to drive pipeline expansion into additional indications, with the ability to rapidly produce customized recombinant protein therapies that are difficult to produce in other systems.
· Revenue Generating: Protalix has a commercial product, Eleyso®, for the treatment of Gaucher disease. In FY20, Eleyso had $16M in sales from both PFE (the Company’s global partner ex-Brazil) and sales in Brazil.
Lead Candidate PRX-102 (Fabry Disease, Ph:3)
· Protalix has completed three ph:3 studies (BRIDGE, BRIGHT, balance) of PRX-102 for the treatment of Fabry Disease and recently reported positive Top-line data from the BALANCE trial, a 24-month, blinded Head to Head trial vs SoC Fabryzme. Collectively, these three trials represent the most comprehensive and robust current Ph:3 clinical program for the treatment of Fabry disease.
· In April, 2021 the Company received a CRL from the FDA regarding the BLA seeking accelerated approval of PRX–102.
The CRL did not report any concerns relating to the potential safety or efficacy of PRX-102 in the submitted data package, but noted an inspection is required before the FDA can approve the BLA.
Due to travel restrictions relating to the COVID-19 pandemic, the FDA was unable to conduct the required inspection during the review cycle.
The company plans to re-submit the BLA to the FDA for PRX-102 (Fabry Disease) in 2H22, which will include the three completed ph:3 studies.
· The company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in February, 2022 for the review of PRX 102 for the treatment of Fabry disease.
The submission included data from the phase III BALANCE trial 12-month interim analysis, the completed phase III BRIDGE trial (including the completed 12-month switch over), the completed phase 1/2 clinical trial in naive or untreated patients, and data from the extension studies.
· Fabry Disease is estimated to be a $2B+ market globally and growing at > 10% CAGR. Protalix has partnered with Chiesi Farmaceutici S.p.A. for the development and commercialization of pegunigalsidase alfa. Under the agreement, Protalix has potential for $800M+ in commercial and regulatory milestone payments, plus royalties of 15-40% from US sales and 15-35% for ROW sales.
· Eleyso® (taliglucerase alfa): approved by FDA in May 2012 for Gaucher disease and subsequently licensed global rights (ex-Brazil) to Pfizer Inc. Protalix retains full rights in Brazil.
· PRX-102 (pegunigalsidase alfa), a PEGylated recombinant human alpha-GAL-A protein for the treatment of Fabry disease.
· PRX-115 (Uricase) is a PEGylated recombinant Uricase, expressed via ProCellEx®, designed to lower uric acid levels in patients with refractory gout. Protalix plans to initiate toxicity studies in 2H22.
· PRX-119 (Long-acting DNase I) is a cutting edge PEGylated recombinant human DNase I, expressed via ProCellEx®, designed to digest DNA-rich Neutrophil extracellular traps (NETs) and reduce NET toxicity in NETs-related diseases.