Abeona Therapeutics Inc. · NASDAQ:ABEO
Abeona Therapeutics — PlasmaTech Biopharmaceuticals — Investor Presentation (February 2015)
22 slides from Abeona Therapeutics's investor presentation, Feb 9, 2015
Slide 1: PlasmaTech Biopharmaceuticals Company Overview Q1 2015
(as. PLASMATECH BIOPHARMACEUTICALS Company Overview Q1 2015
Slide 2: Safe Harbor Statement
This presentation contains certain statements that may be forward - looking within the meaning of Section 27 a of the Securities Act of 1933 , as amended, including statements relating to the product portfolio and pipeline and clinical programs of the company, the market opportunities for the Plasma
Slide 3: Investment Highlights
x Commercial stage biopharmaceutical company driving innovation in plasma proteins x Patented, disruptive fractionation process • ~$21bn market, 11% CAGR • Alpha - 1 Proteinase Inhibitor (“A1PI”) shortage looming, poor Cohn yield • SDF Process increases yields and margins • A1PI, Immunoglobulin, Ult
Slide 4: Product Pipeline
Salt Diafiltration Process ("SDF Process") Complete 2014 2015 2017 SDF Alpha™ (Alpha-1) Process validation, Patent PTBI License, Manufacturer Qualification Pre-IND, IND Filing Clinical Study, BLA Filing > $ 2.5B 2016 Commercial 2017 Market Opportunity SDF Gamma™ (IVIG) Process Validation TBD TBD ~$
Slide 5: Significant Plasma Therapeutics Opportunity
Significant Plasma Therapeutics Opportunity x Large, Fast - Growing Market • WW market ~$21 billion, ~11% CAGR • Growth driven by greater awareness of therapeutic potential plasma proteins, better and earlier diagnostics x Supply Constraints Hampered by Cohn Process • Antiquated Cohn fractionation d
Slide 6: Highly Optimized PlasmaTech SDF Process
Highly Optimized PlasmaTech SDF ™ Process PlasmaTech SDF Process x Simple 2 - Stage Sodium Citrate Precipitation + Diafiltration x No ethanol or pH changes vs. Cohn Fractionation Benefits of PlasmaTech Process x Alpha - 1 yield increase ~10X x IVIG yield increase ~ 20% x Potential for multiple ultra
Slide 7: PlasmaTech will be the only company focused on First A1PI
PlasmaTech will be the only company focused on “ Alpha - 1 First ” x Alpha - 1 Proteinase Inhibitor – Serving Large Underserved Medical Need • 2 - 4% penetration x Driving Growth in Alpha - 1 Market in next 3 years • Early detection; newborn screening in NY and MA • Clinician awareness • New indicat
Slide 8: #1 Clinical Target: Inherited COPD
#1 Clinical Target: Inherited COPD AATD is an under - diagnosed hereditary condition • Alpha - 1 Antitrypsin Deficiency (“AATD”): Genetic condition: insufficient AAT protein produced by liver • Alpha - 1 protects lungs from inflammation and damage caused by infection and inhaled irritants • AATD may
Slide 9: Alpha-1 Market
Alpha - 1 Market Alpha - 1 Market • $900m: 2014 – CAGR: 20 - 35% • Pricing, US: – ’ 15: $400/g – ’ 16 : $ 430/ g – Price CAGR: 7% • Inherited COPD – > 300k patients worldwide – ~ 10k current – 2 - 4% penetration • Aralast, Aralast MP • Launched: 2003 • Prolastin, Prolastin C • Launched: 1988 • First
Slide 10: Looming Supply Shortage
• Alpha - 1 CAGR: 35%, Other plasma protein CAGR: 11% • Global plasma capacity assumed growth: 3.2%/year, 23.5 million liters ww in 2015, 70% of total capacity applicable for Alpha - 1 • Dose calculation: 5g/week “This is an underappreciated problem that will affect the entire Alpha - 1 community. T
Slide 11: SDF: Restructuring margins in target proteins
Large Cohn PlasmaTech SDF™ Difference Revenue GP, $M GM, % Revenue GP, $M GM, % 60 14 24% 12 (34) -277% 183 131 72% 125 73 59% 123 48% 113 336% Alpha-1 Alpha-1 + IgG SDF™: Restructuring margins in target proteins 11 x SDF Process radically improves yield & margin vs. Cohn x Illustrates why smaller c
Slide 12: Abbreviated Regulatory Pathway: 351(a) BLA
351(a) BLA pathway follows previous A1PI approval pathway x Abbreviated Regulatory Pathway • BLA approval pathway 351(a) – estimate $ 5 to 9 million • 12 month scale - up & analytical method development • Replacement therapy efficacy and safety studies (50 target patients to 135 patients theoretical
Slide 13: SDF-Gamma Process produces IVIG
SDF - Gamma™ Process produces IVIG: a high value protein x PlasmaTech IVIG – increases IVIG yield by ~20% • Intravenous immunoglobulin (IVIG) is the high value plasma protein with multiple uses in patients with decreased or abolished antibody production capabilities • ~44% of the total ~21bn plasma
Slide 14: Ultra Orphan Protein Discovery Platform
Ultra Orphan Protein Discovery Platform x Multiple High Value Ultra - Orphan Protein Products • >200 additional proteins in human plasma with therapeutic potential • Denaturing impact of ethanol in Cohn process could create multiple product opportunities for PlasmaTech x Other Possible Plasma Produc
Slide 15: Scientific Advisory Board
Scientific Advisory Board Leaders in Alpha - 1 and Mucositis Eugene Zurlo, BS, MS Pharmacy, Chairman ▪ 56 years experience ▪ Founder/Chairman/ Inventor Plasma Technologies, LLC (Licensor) ▪ Baxter Hyland, Millipore, NY Blood Center, Alpine Biologics, Ayerst Laboratories ▪ Launched first commercial A
Slide 16: Hydrogel Products & Platform
Hydrogel Products & Platform x Unique aqueous pseudoplastic liquid x Patented mucoadhesive hydrogel delivery system enables extended delivery of drugs to mucosal tissue x Broad patent coverage and claims (six patents granted, multiple pending) . x Patent extension opportunity for generic active drug
Slide 17: MuGard Oral Mucositis Clinical Study
x Protocol – Multi - center, randomized, placebo controlled study in 120 head and neck cancer patients x Positive – MuGard statistically significant : • Primary endpoint of reduction in mouth and throat soreness ( p= 0 . 034 ) • Reduction in OM disease severity by WHO score at end of radiation treat
Slide 18: Management & Board of Directors
Board of Directors Management Management & Board of Directors Scott Schorer - CEO ▪ 19 years leadership - healthcare biologics, devices, healthcare IT ▪ President, CEO: CentriMed , IST ▪ President: Systagenix Wound Management Harrison Wehner - President & CFO ▪ 21 years healthcare & biotech IB, fina
Slide 19: Numerous Valuable Near-Term Milestones
Numerous Valuable Near - Term Milestones Events & Milestones Estimated Timing Global roll - out of MuGard for oral mucositis - ongoing x FDA 510(K) marketing approval: ProctiGard™ x MuGard ® Partnerships: Norgine ( Europe), Hanmi (Korea) x SDF License Executed, New Management Team x Norgine/Hanmi Mu
Slide 20: Valuation Comparables
Valuation Comparables Relative Valuations of Drug Delivery Companies / Related Companies Market Analysis (figures in millions, except per share data) Price (1) Revenue Ent. Value / Revenue Company Ticker @ 1/31/15 Equity Value Enterprise Value (2) LTM 2014 2015 LTM 2014 2015 GRIFOLS SA - ADR GRFS $3
Slide 21: Capitalization Table
Recent events: PTBI recently completed a ~$14 million financing and uplisting to NASDAQ No long term debt, no convertible preferred stock Capitalization Table January 1, 2015 Shares Outstanding WAEP Outstanding common shares (PTBI) 20,683,248 - Warrants (PTBIW, fully traded) 3,500,000 $5.00 Warrants
Slide 22: Investment Highlights
x Commercial stage biopharmaceutical company driving innovation in plasma proteins x Patented, disruptive fractionation process • ~$21bn market, 11% CAGR • Alpha - 1 Proteinase Inhibitor (“A1PI”) shortage looming, poor Cohn yield • SDF Process increases yields and margins • A1PI, Immunoglobulin, Ult
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