Abeona Therapeutics Inc. · NASDAQ:ABEO
Abeona Therapeutics — Investor Presentation (February 2017)
41 slides from Abeona Therapeutics's investor presentation, Feb 17, 2017
Slide 1: Abeona Therapeutics
NASDAQ: ABEO www.abeonatherapeutics.com
Slide 2: Safe Harbor Statement
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 opport
Slide 3: Investment Highlights: A World Leader in Rare Disease Therapies
Investment Highlights A World Leader in Rare Disease Therapies Strategic Focus on Rare Diseases • Substantial unmet medical need to treat life threatening diseases caused by a defect in a single gene • Most advanced programs in neurological / lysosomal storage diseases where there are no treatment o
Slide 4: Rare Disease Pipeline
Rare Disease Pipeline Gene Therapies Research Prectinial Phase it Marketed Metabolic 290102 anh ES 000 /ABO-201 (scAAV-CLN3) [SESE SPIT Fi Dermatology £B-101 (LZRSE-Col7A1) [J STORY eZN)) Epidermolysis bullosa (EB) essive dystrophic epidermolysis bullosa (RDEB) Hematology Plasma Therapies SDF ALPHA
Slide 5: Focus on Gene Therapy
Focus on Gene Therapy What is Gene Therapy? • The transplantation of normal genes into cells in place of missing or defective ones in order to correct genetic disorders • Excitement in gene therapy sector due to recent European approvals of two gene therapies, impressive clinical data and anticipate
Slide 6: AAV Based Gene Therapy Platform
AAV Based Gene Therapy Platform Vector crosses the Blood Brain Barrier • Expressed in multiple nervous system cell types • Supra - physiological enzyme expression in brain for over a year observed in animals Self - complementary AAV (scAAV) persists as a stable episome in non - dividing cells with t
Slide 7: AAV Serotypes into the CNS: Serotype makes a difference
AAV 1, 2, 5, 8, 9, 10 into the CNS: Serotype makes a difference Brain - directed injection of AAV vectors encoding green fluorescent protein gene (AAV/GFP). Approximately 2.0 x 10 10 vg of AAV/GFP vectors (serotypes 1, 2, 5, 8, 9, and 10) were injected into the right striatum over a period of 5 min.
Slide 8: Single Stranded vs. Self-Complementary AAV
Single Stranded vs. Self - Complementary AAV Source: Miyaki et al., Medicine » Medical Genetics » "Gene Therapy - Principles and Challenges" , book edited by Doaa Hashad, ISBN 978 - ### - ## - #### - 0, Published: November 26, 2015 After 7.0 x 10 12 vg of AAV9/GFP vectors were injected via tail vein
Slide 9: Global Foundation Support
9 Global Foundation Support Investors and ongoing supporters of Abeona Original founding foundations
Slide 10: Clinical-Stage Programs
Clinical-Stage Programs GR Abeone
Slide 11: Sanfilippo Syndromes (MPS IIIA and IIIB)
Sanfilippo Syndromes (MPS IIIA and IIIB) Rare lysosomal storage disease affecting children Sanfilippo syndrome (MPS IIIA & IIIB) is an inherited monogenic disorder that causes lysosomal enzyme deficiency • Set of four different disease states, each categorized by a missing or deficient enzyme - MPS
Slide 12: Natural History Study of Sanfilippo Patients
Natural History Study 25 - patients with Sanfilippo syndrome assessed Enrollment complete : 25 subjects, 15 MPS IIIA & 10 MPS IIIB Study visits – assessments at 0 , 6 , and 12 months : • Neurocognitive (Leiter) and parental rating assessments (ABAS II) • Timed functional motor tests • Standard labor
Slide 13: Phase 1/2 ABO-102 Clinical Trial for MPS IIIA
Phase 1/2 ABO - 102 ( scAAV - SGSH) Clinical T rial for MPS IIIA Global open - label, dose - escalation gene transfer trial Phase 1/2 open - label, dose - escalation clinical trials • Low dose: n = 3 patients; high dose: n = 3 - 6 patients • Three sites: United States, Spain and Australia Two dose c
Slide 14: No Serious Adverse Events Through 644 Days
No subjects to date have had a serious adverse event (SAE ) through 644 days cumulative post - injection • Upper respiratory infection most common AE (n=3, judged unrelated) • Irritability (n=2) attributed to prednisolone dosing • IFN - γ ELISpot stable through 3 patients 0 20 40 60 80 100 120 MPS-I
Slide 15: Serum Transaminases Decline and Remain Stable
Serum transaminases initially decline, and remain largely stable through 6 months post - injection 0 25 50 75 100 125 150 0 25 50 75 100 125 150 MPS-IIIA-01 Day U / L D o s i n g S c r e e n i n g 7 1 4 3 0 4 5 6 0 7 5 B e g a n t a p e r 9 0 1 1 3 M o n t h 6 ALT AST 1 6 4 L a s t s t e r o i d d o
Slide 16: ABO-102 Reduced Heparan Sulfate and Total Urinary GAG
ABO - 102 Reduced Heparan Sulfate (HS) and Total Urinary GAG 0.0% - 35.2% - 51.5% - 30.7% - 34.4% -70% -60% -50% -40% -30% -20% -10% 0% Screening (n=3) Day 30 (n=3) Day 60 (n=3) Day 90 (n=3) Day 180 (n=2) % Reduction TOTAL GAG Urinary TOTAL GAG 0.0% - 57.6% - 57.6% - 44.4% -70% -60% -50% -40% -30% -
Slide 17: Reduction of CNS HS - Evidence of Biopotency
Reduction of CNS HS - Evidence of Biopotency CSF HS Fragments Decrease After Treatment 17 • Reduction of CSF HS fragment indicates ABO - 102 crosses the blood - brain barrier • No CSF GAG reduction was reported in a separate MPS IIIA Gene Therapy trial that has been discontinued • Continuous, sustai
Slide 18: ABO-102 Reduced Liver and Spleen Volumes
ABO - 102 Reduced Liver and Spleen Volumes 30 (n=3) and 180 d ays (n=2) post - injection a ssessed by MRI Source: : Weinreb NJ, et al. Am J Med. 2002;113:112 - 119 http:// www.fda.gov /downloads/drugs/ developmentapprovalprocess / howdrugsaredevel opedandapproved / approvalapplicationstherapeuticbio
Slide 19: Evidence of Biopotency & Clinical Benefit: Leiter-3
Evidence of Biopotency & Clinical Benefit Natural History Study ABO - 102 Clinical Study Leiter - 3 Nonverbal IQ Scale • The Leiter International Performance Scale Third Edition (Leiter - 3) is a test of nonverbal intellectual ability for ages 3 and above. • The Leiter – 3 does not require any trans
Slide 20: Evidence of Biopotency & Clinical Benefit: Vineland Score
Evidence of Biopotency & Clinical Benefit Natural History Study ABO - 102 Clinical Study Vineland Composite Score (MPSIIIA) Vineland Score with ABO - 102 Vineland Score with ABO - 102 • The Vineland Adaptive Behavior Scales, Second Edition (Vineland - II) measures : • A daptive behaviors • M aladapt
Slide 21: Mullen Scale Scores at Six Months Post-Treatment
Evidence of Biopotency & Clinical Benefit Mullen Scale Scores at Six Months Post - Treatment (N=2) 21 The Mullen Scales of Early Learning, a five domain ability assessment ( Gross Motor , Fine Motor, Visual Reception, Receptive Language, Expressive Language) assesses modality performance and identif
Slide 22: Summary of ABO-102 Clinical Data
Summary of ABO - 102 Clinical Data 22 ABO - 102 for MPS IIIA is Well Tolerated, and Demonstrates Biopotency and Early Signals of Neurocognitive and Behavioral Improvements • ABO - 102 is well tolerated to date, with no serious adverse events (N=4 through 650 days follow up) • All low dose subjects (
Slide 23: ABO-101 for MPS IIIB
ABO - 101 for MPS IIIB Second FDA allowed IND; clinical trial to commence shortly Pre - clinical data • ABO – 101 Single iv infusion of ABO - 101 at 4 - 6 weeks of age normalized the survival in MPS IIIB mice • Single IV Delivery of ABO - 101 induced clearance of lysosomal GAG storage in the CNS and
Slide 24: Epidermolysis Bullosa (EB)
Epidermolysis Bullosa (EB) Rare and devastating skin disease Group of devastating inherited connective tissue diseases; characterized by skin blisters and erosions that can also affect internal organs • There are four forms of EB; EB - 101 targets Recessive Dystrophic Epidermolysis Bullosa (RDEB), a
Slide 25: Abeona's EB-101 Approach
Abeona’s EB - 101 Approach Genetically corrected autologous skin grafts EB - 101: Ex - vivo gene therapy Gene therapy correction of patient cells To replace COL7A1 cDNA , followed by grafting EB - 101 – Gene therapy corrected skin sheets EB - 101 – Skin graft ready for application 25
Slide 26: EB-101 Clinical Program Update
EB - 101 Clinical Program Update Unprecedented wound healing in this population Phase 1 clinical trial in with Recessive Dystrophic Epidermolysis Bullosa (RDEB) - completed using genetically corrected autologous skin grafts • 5 adult patients treated with six grafts each • To date, positive 12 month
Slide 27: EB-101 Phase 1 Clinical Trial Results
≥75% healed 50 - 70% healed < 50% healed B as e li n e 3 months 6 months 12 months EB 101 Phase 1 Clinical Trial Results Subject wounds pre - and post grafting Site Location Description Estimated duration 3m 6m 12m A R lateral hand Erosion 3 - 5 yrs B R medial hand Scar tissue 3 - 5 yrs C L ventral
Slide 28: EB-101 Phase 1 Results: Sustained C7 Levels
Current as of 10/26/15 EB - 101 Phase 1 Clinical Trial Results Induces sustained C7 levels at follow up Biopsies of grafts at follow up visits indicate high levels of C7 expressions Anti - COl7A1 NC2 Mab Hoechst 33342 Anti - COl7A1 NC2 Mab Hoechst 33342 Anti - COl7A1 NC2 Mab Hoechst 33342 Anti - COl
Slide 29: Pre-Clinical Programs
Pre-Clinical Programs S.. Abeona 28
Slide 30: Gene Therapies
Gene Therapies S.. Abeona 30
Slide 31: ABO-201 for Juvenile Batten Disease (CLN3)
ABO – 201 for Juvenile Batten Disease – CLN3 pre - IND meeting completed, clinical trial to commence in 2017 Source: Moving towards therapies for Juvenile batten disease, Experimental Neurology, 2008 JBD - Autosomal recessive (inherited) mutation in the CLN 3 gene • Initially presents as blindness,
Slide 32: ABO-202 for Infantile Batten Disease (CLN1)
ABO – 202 for Infantile Batten Disease – CLN1 Autosomal recessive (inherited) mutation in the CLN1 gene Diagnosed between ages 6 months and 2 years – with early death before age 5 • Affected children fail to thrive and have microcephaly; also typical are short, sharp muscle contradictions called myo
Slide 33: ABO-301 for Fanconi Anemia
ABO - 301 – for Fanconi Anemia Fanconi anemia (FA) is a rare (1: 160,000) pediatric, autosomal recessive (inherited) disease Characterized by multiple physical abnormalities, organ defects, bone marrow failure, and a higher than normal risk of cancer — with 20 to 30 year average lifespan • The major
Slide 34: AIM Next Generation AAV Vector Platform
AIM™ - Next Generation AAV Vector Platform For Future Programs AIM™ Vector Platform • Abeona has a next generation AAV viral vector platform to target CNS and other tissues with increased efficiency and greater tissue specificity • The next generation AIM™ Vector is a designer vector engineered by d
Slide 35: Manufacturing Strategy
Manufacturing Strategy Parallel paths enable clinical trial and commercial long - term sustainable supply A combination of third - party and internal manufacturing sources to ensure long - term continuous supply for domestic and international clinical trials as well as commercial demand . Third Part
Slide 36: Plasma Therapy
Plasma Therapy S.. Ye Abeona 36
Slide 37: Abeona Proprietary SDF Process for AATD
Abeona Proprietary ™ SDF Process for AATD Inherited autosomal disorder that results in insufficient AAT protein production by liver • AATD may lead to liver disease • Alpha - 1 protects lungs from inflammation and damage caused by infection and inhaled irritants Prevalence – need to diagnose and tre
Slide 38: Financial Overview
Financial Overview Cash (Q3 2016): $31.2 million* Cash used in operations ( Q1 - Q3 2016 ): $9.6 million No debt Primary Common S hares outstanding: 33.5 million ** 38 * Cash, cash equivalents, and short - term investments, does not include cash from public offering of $42M, closed November 1, 2016.
Slide 39: Investment Highlights: A World Leader in Rare Disease Therapies
Investment Highlights A World Leader in Rare Disease Therapies Strategic Focus on Rare Diseases • Substantial unmet medical need to treat life threatening diseases caused by a defect in a single gene • Most advanced programs in neurological / lysosomal storage diseases where there are no treatment o
Slide 40: Management & Boards
Management & Boards Management • Tim Miller, Ph.D. - President and Chief Executive Officer & Director • Jeffrey B. Davis - Chief Operating Officer & Director • Harrison G. Wehner - Chief Financial Officer • David P. Nowotnik, Ph.D. - Senior Vice President, Research & Development • Kaye Spratt, Ph.D.
Slide 41: Contacts
Contacts 41 Investor Contact: Christine Berni - Silverstein Vice - President, Investor Relations Phone: 212.786.6212 Email: csilverstein@abeonatherapeutics.com Media Contact: Andre’a Lucca Vice - President, Communications & Operations Phone: 212.786.6208 Email: alucca@abeonatherapeutics.com
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