Abeona Therapeutics Inc. · NASDAQ:ABEO
Abeona Therapeutics — PlasmaTech Biopharmaceuticals — Investor Presentation (January 2015)
24 slides from Abeona Therapeutics's investor presentation, Jan 6, 2015
Slide 1: Investor Presentation January 2015
Investor Presentation January 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 with two proprietary platform technologies, addressing large unmet medical needs x Patented, disruptive fractionation process generating multiple biosimilar protein therapeutics • Plasma protein market is large (>$15 billion WW) and growing at 11% annuall
Slide 4: Product Pipeline Targeting >$17B Market Opportunity by 2017
SDF Process: Salt Diafiltration Process Complete 2014 2015 2017 SDF Alpha™ (Alpha-1) Process Scale UpRegulatory > $ 2.5B SDF Gamma™ (IVIG) Process Scale UpRegulatory > $11.5B PlasmaTech™ Ultra-Orphan Discovery TBD TBD > $ 1B Plasma Protein Addressable Markets ~ $15B MuGard ® 510(k) Europe: Norgine,
Slide 5: Significant Plasma Therapeutics Opportunity
Significant Plasma Therapeutics Opportunity x Large, Fast - Growing Market • Plasma protein therapeutics derived from blood plasma • WW market >$15 billion, growing at approximately 11% annually • Growth driven by greater awareness of therapeutic potential plasma proteins, better and earlier diagnos
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 x Significantly decreased adoption risk; reduced cost Benefits of PlasmaTech Process x Alpha - 1 yield increase ~10X x IV
Slide 7: PlasmaTech will be the only company focused on Alpha-First
PlasmaTech will be the only company focused on “ Alpha - 1 First ” x Alpha - 1 Antitrypsin – Serving Large Underserved Medical Need • Alpha - 1 Antitrypsin Deficiency (“AATD”): Genetic condition whereby insufficient AAT protein made in the liver x Driving Growth in Alpha - 1 Market • Focus on early
Slide 8: COPD is a large and under-treated disease
COPD is a large and under - treated disease x AATD is an under - diagnosed hereditary condition • Alpha - 1 Antitrypsin Deficiency (“AATD”): Genetic condition whereby insufficient AAT protein made in the liver • Alpha - 1 protects lungs from inflammation and damage caused by infection and inhaled ir
Slide 9: Alpha-1 market size and supply forecast
8 • Alpha - 1 CAGR: 35%, Other plasma protein CAGR: 11% • Global plasma processing capacity assumed growth: 3.2%/year, 24.5 million liters ww in 2005, with only 70% of total capacity app licable for AAT processing • Dose calculation: 5g/week “This is an underappreciated problem that will affect the
Slide 10: Why is Alpha-1 a lower priority for fractionators?
Why is Alpha - 1 a lower priority for fractionators? x IVIG represents significantly larger opportunity for Cohn fractionators • For above example of 150,000 L plasma processed, costing ~$21m • Cohn produces ~$9.8m Alpha - 1 vs. ~195m for IVIG IVIG represents a 20X larger opportunity • SDF produces
Slide 11: 351(a) BLA pathway follows previous AAT approval pathway
351(a) BLA pathway follows previous AAT approval pathway x Abbreviated Regulatory Pathway • BLA approval pathway 351(a) – estimate $ 5 to 9 million per protein to regulatory approval • 9 to 12 month scale - up and analytical method development • Replacement therapy efficacy and safety studies (50 ta
Slide 12: 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 13: PlasmaTech SDF Gamma Process produces IVIG
PlasmaTech 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 • ~50% of the total $15 b
Slide 14: Ultra Orphan Proteins
Ultra Orphan Proteins 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 Products • C - 1 - ester
Slide 15: PHT Products & Platform
PHT Products & Platform x Unique aqueous pseudoplastic liquid : beneficial in inhibition / treatment of mucositis & other oral cavity disorders x Patented mucoadhesive hydrogel delivery system enables extended delivery of drugs to mucosal tissue x System enables multiple products in patented deliver
Slide 16: MuGard for Oral Mucositis: unique and differentiated
Significant Medical Issue Often Unrecognized, Undiagnosed and Undertreated Well - Being x Pain x Problems with eating, drinking, speaking and swallowing x Weight loss x Impairment of quality of life (e.g. mood) Infection x Reduces infection barrier Compromised Treatment x Reduced dosage x Breaks or
Slide 17: MuGard for Oral Mucositis: Strong Progress
MuGard for Oral Mucositis: Strong Progress x MuGard for Oral Mucositis (“OM”) • Received FDA Marketing Clearance / launching now • No competing products with demonstrated clinical benefit x $1B+ Global Market Opportunity: • > 400K diagnosed OM cases: US alone 1 • 90% of radiation patients / 40% of c
Slide 18: MuGard clinical trial results in head and neck cancer
x Protocol – Multi - center, randomized, placebo controlled study in 120 head and neck cancer patients x Positive - MuGard was statistically significant with regard to : • Primary endpoint of reduction in mouth and throat soreness ( p= 0 . 034 ) • Reduction in OM disease severity by WHO score at end
Slide 19: ProctiGard for Radiation Proctitis
ProctiGard for Radiation Proctitis x Radiation Proctitis - Large Unmet Medical Need • Inflammation & damage to the lower portion of the colon post exposure to x - rays or ionizing radiation as part of radiation therapy. M ost common after treatments for cancer such as cervical, prostate & colon canc
Slide 20: Management & Board of Directors
Board of Directors Management Management & Board of Directors Scott Schorer - CEO ▪ 18+ years leadership - healthcare biologics, devices, healthcare IT ▪ President, CEO: CentriMed , IST ▪ President: Systagenix Wound Management Harrison Wehner - President & CFO ▪ 20+ years healthcare & biotech IB, fi
Slide 21: 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 22: 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 @ 12/4/14 Equity Value Enterprise Value (2) LTM 2014 2015 LTM 2014 2015 GRIFOLS SA - ADR GRFS $3
Slide 23: Capitalization Table
Recent events: PTBI recently completed a $14 million financing and up listing to NASDAQ No long term debt, no convertible preferred stock Capitalization Table January 1, 2015 Shares Outstanding WAEP Common shares out (primary) 20,683,248 - Warrants (PTBIW) 3,500,000 $5.00 Warrants (non - traded) 577
Slide 24: Investment Highlights
x Commercial stage biopharmaceutical company with two proprietary platform technologies, addressing large unmet medical needs x Patented, disruptive fractionation process generating multiple biosimilar protein therapeutics • Plasma protein market is large (>$15 billion WW) and growing at >10% annual
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