Abeona Therapeutics Inc. · NASDAQ:ABEO
Abeona Therapeutics — Investor Presentation (December 2015)
40 slides from Abeona Therapeutics's investor presentation, Dec 1, 2015
Slide 1: Abeona Therapeutics
NASDAQ: ABEO www.abeonatherapeutics.com 1
Slide 2: Safe Harbor Statement
Safe Harbor Statement This presentation contains certain statements that may be forward - looking within the meaning of Section 27a 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 opportun
Slide 3: Abeona Therapeutics: Developing Therapies for Children with Rare Genetic Diseases
Abeona Therapeutics : Developing Therapies for Children with Rare Genetic Diseases 3 • Pronunciation : a y - bee - oh - n uh • Origin : Roman Goddess said to be the protector of children as they start out on their journey • Focus : Abeona Therapeutics develops and delivers gene therapy and plasma -
Slide 4: Abeona Therapeutics - Pipeline
Abeona Therapeutics - Pipeline 4
Slide 5: Gene Therapy
GENE THERAPY 5
Slide 6: Lysosomes and lysosomal storage disorders
Lysosomes : an organelle in the cytoplasm of animal cells containing degradative enzymes enclosed in a membrane 6 Normal cell Cell with lysosome deficiency • Lysosomes serve as cell recycling center • Uses enzymes to break down sugars • Genetic defects can cause enzymes to not function properly • Th
Slide 7: Potential Routes of Delivery for AAV Gene Therapy
Potential Routes of Delivery for AAV Gene Therapy 7 Intravenous: Injection into a blood vessel • Whole body delivery for treatment of multiple organs systems, including brain • Strong patient adoption, low safety risk • Abeona’s approach utilizes AAV9 for treating lysosomal storage diseases Intracer
Slide 8: Intravenous AAV9 delivery crosses the blood-brain barrier
Delivery of AAV9 by intravenous delivery crosses the blood - brain barrier with broad coverage • Single, intravenous AAV9 injection • Expressed in multiple nervous system cell types • Supra - physiological enzyme expression in brain for over a year observed in animals • Self - complementary AAV9 (sc
Slide 9: Sanfilippo Syndrome (MPS IIIB & MPS IIIA)
Sanfilippo Syndrome ( MPS IIIB & MPS IIIA) 9
Slide 10: Sanfilippo Syndrome (MPS III)
Sanfilippo Syndrome (MPS III) • Sanfilippo syndrome ( mucopolysaccharidosis (MPS) type III ) is an inherited monogenic disorder that causes lysosomal enzyme deficiency – Divided into four types (A to D) based on specific enzyme defect • IIIA - N - sulfoglucosamine sulfohydrolase (SGSH) • IIIB - α -
Slide 11: Abeona's Gene Therapy: Whole body treatment by intravenous injection
Abeona’s Gene Therapy: Whole body treatment by intravenous injection 11 AAV9 virus Intravenous injection Whole - body correction • Increased enzyme activity • Normalized GAG content • Neuromuscular correction • Cognitive improvements • 100% improved survival 4 – 6 week old MPS III animals 4 – 6 mont
Slide 12: IV Delivery Induces Clearance of Lysosomal GAG Storage in the CNS
IV Delivery of ABO - 101 Induces Clearance of Lysosomal GAG Storage in the CNS and Somatic Tissues rAAV9 - mediated rNAGLU expression in MPS IIIB mouse brain • NAGLU expression at or above heterozygote (and often above wild - type) 12 16 - 27 mo pi
Slide 13: IV Delivery Clears GAG Storage in CNS and Somatic Tissues
IV Delivery of ABO - 101 Induces Clearance of Lysosomal GAG Storage in the CNS and Somatic Tissues Lower bars are better. Demonstrates that treated IIIB mice have reduced GAG content, similar to unaffected animals, in multiple tissues compared to untreated IIIB mice (with exception of kidney). 13 So
Slide 14: Normalization of Cognitive and Motor Function in MPS IIIB Mice
AB0 - 101 Demonstrates Normalization of Cognitive and Motor Function in 4 - 6 Week Old MPS IIIB Mice Hidden task in Morris water maze Latency to fall from a rotarod 14 Source: Fu et al (2011) Correction of Neurological Disease of Mucopolysaccharidosis IIIB in adult mice by rAAv9 Trans - Blood - Brai
Slide 15: Single IV Infusion Normalized Survival in MPS IIIB Mice
Single iv Infusion of AB0 - 101 at 4 – 6 Weeks of Age Normalized The Survival in MPS IIIB Mice 15 Survival (rAAV9 - CMV - hNAGLU) Source: Fu et al (2011) Correction of Neurological Disease of Mucopolysaccharidosis IIIB in adult mice by rAAv9 Trans - Blood - Brain Barrier Gene Delivery , Mol. Ther 19
Slide 16: Persistence of NAGLU Activity in CNS in Non-Human Primates
Persistence of NAGLU (MPS IIIB enzyme) Activity in CNS 6 Months post - intravenous injection in Non - Human Primates Brain and somatic NAGLU activity is significantly increased over normal enzyme levels 6 months post - injection despite modest decreases in peripheral serum NAGLU levels Source: Murre
Slide 17: Clinical Trial Readiness: MPS IIIA and MPS IIIB Natural History Study
Clinical Trial Readiness: MPS IIIA and MPS IIIB Natural History Study • FDA requested Natural History study to support clinical endpoints • Enrollment complete: 25 subjects, 15 MPS IIIA and 10 MPS IIIB • Study visits – assessments at 0, 6, and 12 months: - Neurocognitive ( Leiter ) and parental rati
Slide 18: Phase 1/2 clinical trial of ABO-101 for MPS IIIB
Phase 1/2 open - label, dose - escalation clinical trial of ABO - 101 (AAV9 NAGLU) for MPS IIIB • Two dose cohorts of n=6 - 9 pts via intravenous injection • Primary objective is determination of safety, with secondary objectives: • CSF and serum NAGLU enzyme activity levels. • Improved cognitive ab
Slide 19: Juvenile Batten Disease (JBD)
Juvenile Batten Disease (JBD) 19
Slide 20: Juvenile Batten Disease
Juvenile Batten Disease (Juvenile Neuronal Ceroid Lipofuscinosis ) • Autosomal recessive (inherited) mutation in the CLN3 gene; estimated incidence of 1:100,000 births • Lysosomal storage disease characterized by the accumulation of autofluorescent ceroid lipopigments and proteins • Initially presen
Slide 21: ABO-201: IV Delivery results in expression in CLN3 mouse brain
ABO - 201: IV Delivery results in expression in the in CLN3 Δex7/8 mouse brain 21 Courtesy Tammy Kielian , Unpublished data
Slide 22: Motor behavior normalized in CLN3 mice treated with ABO-201
Motor behavior is normalized in CLN3 mice treated with intravenous injection of ABO - 201 3 months post - injection Courtesy Tammy Kielian , Unpublished data
Slide 23: CRISPR Cas9 Gene Editing for Rare Blood Diseases
CRISPR Cas9 Gene Editing for Rare Blood Diseases • CRISPRs (clustered regularly interspaced short palindromic repeats) are segments of DNA that contain short repeating base pairs • Cas9 is an enzyme specialized for cutting DNA, enabling gene editing to insert, replace or remove genetic information •
Slide 24: Fanconi Anemia (FA)
Fanconi Anemia (FA) 24
Slide 25: ABO-301 (AAV LK19 FANCC) for Fanconi Anemia
ABO - 301 (AAV LK19 FANCC) for Fanconi Anemia (FA) • 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 av
Slide 26: Gene Therapy and Rare Disease Comparables
Gene Therapy and Rare Disease Comparables 26 resourced from YahooFinance.com as of 11.11.15
Slide 27: Plasma Therapy
PLASMA THERAPY 27
Slide 28: Alpha-Antitrypsin (AAT) Deficiency
Alpha - 1 Antitrypsin (AAT) Deficiency is a recessive (inherited) disease in which a deficiency of AAT produces COPD and liver disease from retained misfolded AAT • AAT deficiency is a protein folding disease • Protein folding is the process by which an unfolded polypeptide chain folds in to a speci
Slide 29: AAT augmentation treatment improves morbidity and mortality
Sources: AJRCCM 1998; 158:49; Worldwide racial and ethnic distribution of alpha(1) - antitrypsin deficiency. Chest 2002;122:1818 AAT a ugmentation t reatment i mproves morbidity and mortality • AAT deficiency predisposes those affected to COPD and liver disease • AAT treatment i mproves morbidity (
Slide 30: Proprietary SDF Manufacturing process vs Cohn process
Proprietary SDF Manufacturing process delivers significant advantages over current industry standard Cohn process 30 SDF Delivers… Kinder, gentler x 2 - stage sodium citrate precipitation + diafiltration x Compared with Cohn method: • No ethanol or harmful pH changes • Less denaturing of select prot
Slide 31: PTB-101 (SDF Alpha) Registration Program
PTB - 101 (SDF Alpha ™ ) Registration Program • Seeking FDA concurrence for registration studies to determine safety and bio - equivalency comparison of PTB - 101 SDF Alpha TM versus approved API in individuals with AATD • Endpoints with weekly 60 mg/kg dosing: • To demonstrate that the pharmacokine
Slide 32: Leadership & Scientific Advisory Board
Leadership & Scientific Advisory Board 32
Slide 33: Leadership Team
Leadership Team • Steven H. Rouhandeh - Chairman of the Board and Executive Chairman • Tim Miller, Ph.D . - President and Chief Executive Officer, Director • Jeffrey B. Davis - Chief Operating Officer, Director • Mark J. Ahn, Ph.D . - Vice Chairman, Director • Mark J. Alvino - Director • Stephen B.
Slide 34: Rare Disease Scientific Advisory Board
Rare Disease Scientific Advisory Board (SAB) Gene Therapy • Barry Byrne, MD, University of Florida, Powell Gene Therapy Center • Carsten Bonnemann , MD, Children’s Hospital of Philadelphia • Pol Boudes , MD, Cymabay Therapeutics • Brian Kaspar , Ph.D , Nationwide Children’s Hospital and Ohio State U
Slide 35: Financial Summary Capitalization
Financial Summary Capitalization Issued and Outstanding Shares WAEP (Weighted Average Exercise Price) EQUITY Outstanding common shares (ABEO) 32,732,783 - Warrants (ABEOW, fully traded) 2,572,881 $5.00 Warrants issued since Dec 2014 782,500 $9.40 Options 1 1,994,000 $6.90 Primary Total 32,732,783 Fu
Slide 36: Milestones
Milestones x Q2 15: Completed Abeona Therapeutics acquisition with lead gene therapy products ABO - 101 (AAV9 NAGLU) for Sanfilippo syndrome (MPS IIIB); and ABO - 102 (scAAV9 SGSH) for MPS IIIA x Q2 15 : Orphan Drug and Rare Pediatric Disease designations for Sanfilippo syndrome (MPS III) x Q2 15: W
Slide 37: Summary
Summary • Abeona Therapeutics develops and delivers gene therapy and plasma - based products for severe and life - threatening rare diseases • Innovative, validated and scalable technology platforms for gene therapy and plasma - based products • High value - added rare disease product pipeline: – AB
Slide 38: Contacts
Tim Miller, Ph.D President & CEO Email : tmiller@abeonatherapeutics.com Jeff Davis Chief Operating Officer Email : jdavis@abeonatherapeutics.com Andre’a Lucca Vice - President of Communications Phone : 212.786.6208 Email : alucca @abeonatherapeutics.com Magdalena Tyrpien Director of Investor Relatio
Slide 39: Abeona: Working together to find a cure
“BAveona Working together to find a cure.
Slide 40: Abeona: Working together to find a cure
“BAveona Working together to find a cure.
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