Protalix Biotherapeutics, Inc. (de) · AMEX:PLX
Protalix Biotherapeutics, Inc. (de) — Corporate Update (January 2019)
27 slides from Protalix Biotherapeutics, Inc. (de)'s corporate update, Jan 4, 2019
Slide 1: Protalix BioTherapeutics Corporate Update January 2019
Protalix BioTherapeutics Corporate Update January 2019
Slide 2: Forward-looking statements
This presentation contains forward - looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The forward - looking statements, including, among others, statements regarding e xpectations as to
Slide 3: Company highlights
Clinically differentiated and improved recombinant therapeutic proteins • FDA approved ProCellEx® plant cell culture based expression system • FDA multi - product and EMA approved manufacturing facility in Israel Elelyso® (taliglucerase alfa) approved and commercialized for Gaucher disease; Protalix
Slide 4: Protalix pipeline overview
BALANCE Phase III study alidornase alfa (PRX - 110 ) Cystic Fibrosis Oral anti - TNF - α (OPRX - 106) Ulcerative Colitis \ Inflammatory Bowel Disease Phase IIa completed BRIDGE Phase III study BRIGHT Phase III study (once monthly) Phase IIa completed * positive results * positive results (PRX - 102
Slide 5: Pegunigalsidase alfa for Fabry Disease
PROTALDX Biotherapeutics pequilgacidsseatia 1mg/kg/2 weeks BM 2mg/kg/4 weeks for Fabry Disease
Slide 6: Fabry disease remains a high unmet need
Fabry disease remains a high unmet need 6 Rare genetic lysosomal storage disorder caused by enzyme deficiency; potentially life threatening disease characterized by progressive kidney disease, cardiovascular and cerebrovascular complications and severe pain ~$1.4B growing market (CAGR ~10 %); ~5,000
Slide 7: Higher levels of active available enzyme
* Fabrazyme ® ( agalsidase beta) – USPI Higher levels of active available enzyme potentially more efficacious Substantially greater enzyme exposure than current ERTs A chemically modified plant cell derived PEGylated covalently bound homodimer active and stable enzyme throughout infusion inter
Slide 8: Higher uptake and prolonged activity in target organs
8 Higher uptake and prolonged activity in target organs µg/gr FW = amount of enzyme [µg] per gr of tissue fresh weight [FW], assessed by activity Kizhner et al (2015), Molecular Genetics and Metabolism 114: 259 – 267 Fabry mice model Higher levels of active enzyme in target organs potentially more
Slide 9: PK modeling shows greater AUC; once-monthly administration
9 PK modeling shows greater AUC in a single infusion of 2 mg/kg pegunigalsidase alfa vs. two bi - weekly infusions of Fabrazyme® over a 4 - week time frame. First - in - class, once - monthly administration Greater AUC potential for clinical stability with half the infusions
Slide 10: Positive results from completed phase I/II study
10 Positive results from completed phase I/II study *Fabrazyme prescribing information Positive impact on kidney function Stable cardiac parameters Improvement in gastro - intestinal symptoms Excellent safety and tolerability profile throughout ~35 patient years » Patients currently in their 4 th ye
Slide 11: Robust phase III pivotal clinical program
11 1mg/kg 2 weeks Head - to - Head vs. Fabrazyme® in Switch Patients 2mg/kg 4weeks Switch - over from Fabrazyme® and Replagal ® 1mg/kg 2 weeks Switch - over from Replagal® FDA* 24 mos Superiority 12 mos Safety and efficacy 12 mos Safety and efficacy EMA Rest of World 12 mos Comparability (potential
Slide 12: Twice the enzyme activity for antibody positive Fabrazyme patients
Twice the enzyme activity for antibody positive Fabrazyme patients if switched to pegunigalsidase alfa Analysis of baseline characteristics of the BALANCE study : Ex - vivo immunogenicity testing of blood samples: 12 Substantially less inhibited by Fabrazyme neutralizing antibodies ( nAbs ) Dr. Davi
Slide 13: Preliminary results of BRIDGE study: improved kidney function
Preliminary results of BRIDGE study: Improved kidney function following switch to pegunigalsidase alfa Based on available historical serum creatinine for approximately 2 years eGFR mL/min/ 1.73 m 2 is calculated using CKD - EPI formula eGFR Slope - mL/min/ 1.73 m 2 /year -30.00 -20.00 -10.00 0.00 10
Slide 14: Evidence for potential superiority
14 Evidence for potential superiority pegunigalsidase alfa Fabrazyme® eGFR slope - 2.2 1 - 3.8 Half - life ~80 hours 2 hours Active enzyme >14 days ½ day Antibodies (ADA) 0% 3 74% 2 1. N=7 classic Fabry patients, 24 months 2. Fabrazyme prescribing information 3. 19% formation of anti - drug antibodi
Slide 15: Unique proposition for addressing significant unmet needs
15 Unique proposition for addressing significant unmet needs Two dosing regimens: potential for better efficacy and lower treatment burden x Treatment flexibility for patients x Two independent paths for product superiority 1mg/kg/2weeks 2mg/kg/4weeks Superior ERT for patients with progressing impai
Slide 16: Global partnership with Chiesi Farmaceutici S.p.A
16 Global partnership with Chiesi Farmaceutici S.p.A Ex - US partnership now expanded to include exclusive rights in the United States The combined two agreements reflect: Investment of $ 95M in upfront payments and development cost reimbursement Up to $ 1 billion in potential milestone payments Tie
Slide 17: Oral anti-TNF-α OPRX-106 for Inflammatory Bowel Disease
Oral anti - TNF - α OPRX - 106 for Inflammatory Bowel Disease
Slide 18: IBD – High unmet need despite available treatments
IBD are autoimmune inflammatory diseases of the digestive tract including Ulcerative Colitis (UC ) and Crohn’s Disease ( CD); debilitating disease characterized by severe diarrhea, abdominal pain, fatigue and weight loss >$ 12B market; >2.5 million patients across US and Europe 18 Inflammatory Bowel
Slide 19: OPRX-106: plant cell expressed oral anti-TNF-α
Developed via Protalix’s platform for orally delivered proteins whereby the plant cell wall protects the protein and serves as a natural oral administration vehicle Administered orally, OPRX - 106 is biologically active in the gut OPRX - 106 completed two clinical trials in Ulcerative Colitis: • Pha
Slide 20: Positive clinical results in phase IIa OPRX-106
20 Positive clinical results in phase IIa OPRX - 106 1. Reduced Mayo score of >3 points and decrease in the rectal bleeding sub - score of >1 point from baseline, or a rectal bleeding sub - score of 0 or 1 2. Mayo score of ≤ 2 with no sub - score reaching >1 point 3. Mayo endoscopy score of 0 or 1 8
Slide 21: OPRX-106 advantages
Inflammatory Bowel Disease market >$12B High clinical response and remission rates and meaningful mucosal healing in Ulcerative Colitis patients with moderate disease OPRX - 106 Advantages Anti - TNF alfa mechanism – known and established first line treatment for steroid refractory and lack of respo
Slide 22: alidornase alfa (PRX-110) for Cystic Fibrosis
alidornase alfa (PRX - 110) for Cystic Fibrosis
Slide 23: Cystic Fibrosis and alidornase alfa
23 Cystic Fibrosis (CF) is a rare genetic disease characterized by a highly viscous mucus most prominently leading to severe lung damage and loss of respiratory function; over 70,000 CF patients world - wide Leading product, Pulmozyme®, DNase enzyme, ~$700M annual sales with significant growth poten
Slide 24: Phase II trial demonstrates lung function improvement
24 Mean absolute change in ppFEV1 • Pulmozyme treated patients following 14 - day washout • 28 - day alidornase alfa treatment • 14 - day follow - up without treatment Phase II trial demonstrates clinically meaningful lung function improvement alidornase alfa ( PRX - 110 ) N=16 Extraordinary reducti
Slide 25: Financial Overview
Financial Overview 25 • ~ 148M shares outstanding as of December 31, 2018 • Dual listed on NYSE American and TASE • Cash position: ~$ 37.5M as of December 31, 2018 • Cash level projected to fund operations through read outs of all Fabry trials and potential major regulatory milestones • ~$58M conver
Slide 26: Protalix had significant achievements to date
Protalix had significant achievements to date 26 x Strong positive two - year data for pegunigalsidase alfa and positive interim data from the BRIDGE study (Phase III) reversing eGFR slope after switch to pegunigalsidase alfa x Global partnership for pegunigalsidase alfa with significant upfront, R&
Slide 27: Moshe Manor, President and CEO
Moshe Manor President and CEO Protalix BioTherapeutics moshe.manor@protalix.com
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