Abeona Therapeutics Inc. · NASDAQ:ABEO
Abeona Therapeutics — Investor Presentation (August 2019)
38 slides from Abeona Therapeutics's investor presentation, Aug 14, 2019
Slide 1: Living with RDEB Corporate Presentation August 2019
MONSIE Living with RDEB Corporate Presentation August 2019
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: Abeona: A Fully-Integrated Gene & Cell Therapy Company
• Pivotal Phase 3 preparation underway in severe form of Epidermolysis Bullosa (EB) • Ongoing Phase 1/2 clinical trials in MPS IIIA and MPS IIIB • Additional programs approaching the clinic in Batten disease (CLN1 and CLN3) • Next generation AIM™ AAV vector platform with in vivo proof of concept dat
Slide 4: Robust Pipeline
Robust Pipeline Bottosa coca) ans ABO-10: cerspers searco renrvotmevaeone sees EE ABO.101 SSS SS SRerro smbrone rire ———— === Se => > ABO-202 INPANTICE SATTEN DISEASE CLND) ABO-201 ‘oven BATTEN orsease LNs) ABO-401 verte Fienosis (cr) ABO-50X ‘RETINAL OISeASES AIM” VECTORS
Slide 5: RDEB Clinical Program EB-101
RDEB Clinical Program EB - 101 Orphan Drug Designation (FDA) ✓ Orphan Drug Designation (EU) ✓ Rare Pediatric Disease Designation (FDA) ✓ Breakthrough Therapy Designation (FDA) ✓ Regenerative Medicine Advanced Therapy Designation (FDA) ✓
Slide 6: EB: Devastating Inherited Connective Tissue Disorder
Recessive Dystrophic Epidermolysis Bullosa (RDEB): • Primarily characterized by skin blisters and erosions • Caused by mutations in COL7A1 gene, which encodes type VII collagen • Estimated 2,500 patients in the U.S. Patients suffer from having up to 80% of their body covered in wounds, leading to: •
Slide 7: Lifelong Burden with No Relief and No FDA Approved Treatments
Lifelong Burden with No Relief and No FDA Approved Treatments 7 CHRONIC WOUNDS Average size: 118 cm 2 Average duration of open wound: 7 years RECURRENT WOUNDS Average size: 26 cm 2 Average duration of open wound: 5 years Up to 80% of patients’ body surface can be covered in wounds with two main type
Slide 8: Chronic Open and Recurrent Wounds Have Distinct Time Courses
Recurrent Wounds Over Time (N=25) Chronic Open and Recurrent Wounds Have Distinct Time Courses Time to heal (6 weeks) Time to re - blister (3 weeks) baseline 2 months 5 months baseline 2 months 3 months Chronic Recurrent Recurrent Wounds Over Time (N=25) Chronic Wounds Over Time (N=25) 8 Natural his
Slide 9: Size Distribution According to Wound Types
Size Distribution According to Wound Types 9 Large wounds are the most urgent to treat as they carry the greatest burden, including Pain, Pruritis, and Risk of infection Recurrent Chronic p - value Mean Size (cm) 26 118 0.001 Mean Duration ( yr ) 5 7 NS Sequentially Photographed Wounds: N=25 patient
Slide 10: Relative Sizes of Recurrent and Chronic Wounds
Relative Sizes of Recurrent and Chronic Wounds 10cm 2 19 cm 2 2.5in / 6.4cm 1.4in / 3.6cm Common Size of Watch Face (36mm) 73% of chronic wounds are larger than 19 cm 2 26 cm 2 2.5in / 6.4cm Average size of recurrent wounds 40 cm 2 More than half of chronic wounds are greater than 40 cm 2 118 cm 2 1
Slide 11: Chronic Open Wounds Have Greater Pain and Itch
Natural history of wounds in patients with recessive dystrophic epidermolysis bullosa; Teng et al., Abstract #251; Society of In vestigational Dermatology Annual Meeting, 2019 Sequentially Photographed Wounds (N=25 Patient, 62 Wounds) 0 1 2 3 4 5 6 Pain Chronic Recurrent Wong Baker FACES™ Pain Ratin
Slide 12: Developing an Ideal RDEB Treatment
Developing an Ideal RDEB Treatment The ideal RDEB treatment will: • Target the underlying genetic cause of disease • Correct the genetic defect in the appropriate cells – keratinocytes, the natural producer of COL7 in healthy skin • Durably restore COL7 function by correcting keratinocyte progenitor
Slide 13: EB-101: Ex-Vivo Autologous Gene-Corrected Cell Therapy
EB - 101: Ex - Vivo Autologous Gene - Corrected Cell Therapy 13 EB - 101 is a personalized treatment that addresses RDEB patient needs Manufacturing process goes from biopsy to patient ready graft in as little as 26 days Two 8mm biopsies produces 6 - 8 sheets that are ~40 cm2 and keratinocyte stem c
Slide 14: EB-101: Ready for Patients
EB - 101: ready for patients Fully Integrated, Independent and Scalable Manufacturing 14 Commercial readiness of Abeona’s facility • Conversion of GMP to commercial scalability Large - scale cGMP capacity and deep expertise • 26,000 sq. ft facility in Cleveland • State - of - the - art laboratories
Slide 15: EB-101 Phase I/IIa Trial Study Description
Study Description A Phase I/IIa Single Center Trial of Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa (RDEB) using EB - 101 for autologous tissue transplantation Trial Design • Open - label, interventional study • Seven patients with RDEB (ages 18 to 45 years) • Follow - up visits at 1
Slide 16: Gene Therapy Skin Grafts Restore Collagen 7
Site Location Descript Estimated duration 3m 6m 12m 24m A R lateral hand Erosion 3 - 5 yrs B R medial hand Scar tissue 3 - 5 yrs C L ventral foot Erosion and scar 3 - 5 yrs D L hand Scar tissue 3 - 5 yrs E R foot Erosion and scar 3 - 5 yrs Z L ventral foot Induced wound New 75% healed 50 - 70 % he
Slide 17: EB-101: Phase 1/2a Results Demonstrate Durable Efficacy
EB - 101: Phase 1/2a Results Demonstrate Durable Efficacy RDEB Wound Healing >75% >50%-75% <50% 17 • Addressing wounds not treated by other gene therapies in development • Wounds up to 400 cm 2 and open up to 20 years
Slide 18: Proportion of Sites with ≥50% & ≥75% Healing
EB - 101 Phase I/IIa: Proportion of Sites with ≥ 50% & ≥75% Healing Average wound area healed at 3 and 6 months was 130 cm 2 and 120 cm 2 (range 130 - 157 cm 2 ), respectively per patient (Note: healed area was calculated based on minimum % healing per wound site, e.g. 50% used for wound sites that
Slide 19: EB-101 Summary and Anticipated Next Steps
EB - 101 Summary and Anticipated Next Steps Successful Phase 1/2 • Favorable safety profile with no product - related SAEs to date • Significant and durable wound healing, with up to 5 years of follow - up • Continuous type VII collagen expression 2+ years post treatment Established GMP manufacturin
Slide 20: MPS III Clinical Programs
MPS III Clinical Programs ABO - 102* ABO - 101 Orphan Drug Designation (FDA) ✓ Orphan Drug Designation (EU) ✓ Rare Pediatric Disease Designation (FDA) ✓ Fast Track Designation (FDA) ✓ Regenerative Medicine Advanced Therapy Designation* (FDA) ✓
Slide 21: Ongoing Phase 1/2 Clinical Trials for Sanfilippo Syndrome (MPS III)
Inherited monogenic disorders causing lysosomal enzyme deficiency • Two most common forms categorized by deficient enzymes: – MPS IIIA (SGSH), MPS IIIB (NAGLU) • Abnormal accumulation of glycosaminoglycans (GAGs; heparan sulfate (HS)) • Loss of speech/vision, cognitive decline, behavioral abnormalit
Slide 22: MPS IIIA Natural History: Cognitive & Developmental Assessments
MPS IIIA Natural History: Cognitive & Developmental Assessments 22 Chronological Age (mo) Cognitive Age Equivalent (mo) Shapiro et al. 2016 0 3 6 9 1 2 1 5 1 8 2 1 2 4 2 7 3 0 3 3 3 6 3 9 4 2 4 5 4 8 5 1 5 4 5 7 6 0 6 3 6 6 6 9 7 2 7 5 7 8 8 1 8 4 8 7 9 0 9 3 9 6 9 9 1 0 2 1 0 5 1 0 8 1 1 1 1 1 4 1
Slide 23: Phase 1/2 Clinical Trial (ABT-001) for MPS IIIA with AAV9-SGSH
Intravenous Dosing • Cohort 1 : 5 x 10 12 vg/kg (n=3) • Cohort 2 : 1 x 10 13 vg/kg (n=3) • Cohort 3 : 3 x 10 13 vg/kg (9 - 12) 8 subjects treated Primary Endpoint • Safety Secondary Endpoints • Cerebrospinal Fluid (CSF) and/or urinary HS and/or GAGs • CSF and serum SGSH enzyme activity • Liver, sple
Slide 24: Reduction in CSF Heparan Sulfate Post Treatment
Reduction in CSF Heparan Sulfate Post Treatment 24 0 3 6 9 12 15 18 21 24 27 30 Post Injection Visits (Months) 0 50 100 150 200 250 300 350 400 450 M e d i a n o f H S O L I G O S A C C H A R I D E S ( n m o l / L ) Cohort 3 Cohort 2 Cohort 1 Lower Limit Quantitation
Slide 25: Reduction in Liver Volume Post Treatment
Reduction in Liver Volume Post Treatment (N=13) 25 Truxal et. al., 2016, Mol. Genet. Metab .
Slide 26: Change in Mullen Developmental Age vs. Natural History
Change in Mullen Developmental Age Post Treatment vs. Natural History 26 Gray lines indicate natural history study patients ( Truxal et. al., 2016, Mol. Genet. Metab . ) All 3 youngest patients in cohort 3 continue to track within normal age equivalent development 12 - 18 months post treatment Ages
Slide 27: Summary of MPS IIIA ABO-102 Phase 1/2 Study Data
Summary of MPS IIIA ABO - 102 Phase 1/2 Study Data • N=14 as of July 2019 - Length of follow up in cohort 3: 6 months (N=8 of 8 total), 12 months (N=7 of 8 total), 18 months (N=4 of 8 total) • Clear dose - response, and sustained improvement in biomarkers (HS CSF, Liver Volume) • Positive neurocogni
Slide 28: Phase 1/2 Clinical Trial (ABT-002) for MPS IIIB with AAV9-NAGLU
* Clinical study protocol states 3 subjects in Cohort 1; however, due to exceptional circumstances and following robust safety profile and positive rev iew from DSMB, trial was cleared in Europe to advance to Cohort 2 dose Intravenous Dosing • Cohort 1 : 2 x 10 13 vg/kg (n=2 subjects*) – 2 patients
Slide 29: Reduction in Liver Volume, CSF and Urine Heparan Sulfate
1. Truxal et. al., 2016, Mol. Genet. Metab Liver Volume CSF and Urine HS Reduction in Liver Volume, CSF and Urine Heparan Sulfate Post Treatment vs. Natural History 29 *Gray lines indicate natural history study patients 1
Slide 30: Change in Mullen Developmental Age vs. Natural History
Change in Mullen Developmental Age Post Treatment vs. Natural History 30 *Gray lines indicate natural history study patients 1 1. Whitley et al., 2018
Slide 31: CLN1 (Infantile Batten) Disease ABO-202
CLN1 (Infantile Batten) Disease ABO - 202 Orphan Drug Designation (FDA) ✓ Orphan Drug Designation (EU) ✓ Rare Pediatric Disease Designation (FDA) ✓ Fast Track Designation (FDA) ✓
Slide 32: ABO-202: scAAV9 for Treatment of Infantile Batten Disease – CLN1
• Infantile Neuronal Ceroid Lipofuscinosis (INCL): severe neurodegenerative lysosomal storage disease, currently with no approved treatment - Caused by mutations in the CLN1 gene, encoding the soluble lysosomal enzyme palmitoyl - protein thioesterase - 1 (PPT1) • Exclusive, worldwide license secured
Slide 33: ABO-202: Phase 1/2 Clinical Trial Readiness
• IND - enabling toxicology and efficacy studies completed ⎻ Strong safety, with no significant toxicology findings in the combination dose escalation study ⎻ Data demonstrated normalized survival, muscle function and cognition • FDA IND clearance received Q2 2019 • Potential Benefits of IT/IV dosin
Slide 34: Next Generation AIM AAV Platform
Next Generation AIM TM AAV Platform
Slide 35: AIM: Next Generation AAV Vector Platform
AIM™: Next Generation AAV Vector Platform • AIM™ Vector Platform: AAV viral vector platform selected to target CNS, lung, skin, muscle, liver and other tissues • Key Advantages of AIM™ ⎼ First generation demonstrated increased gene delivery efficiency to specific tissues ⎼ Second and third generatio
Slide 36: AAV GMP Manufacturing
AAV GMP MANUFACTURING
Slide 37: Abeona's Gene Therapy Manufacturing and Quality Capabilities
Abeona’s Gene Therapy Manufacturing and Quality Capabilities • FDA Division of Manufacturing and Product Quality (DMPQ) supports clinical or single use commercial facility • 46 highly trained staff members in instrumentation and equipment Quality, technical operations, process development, assay dev
Slide 38: Management Team and Scientific Advisors
João Siffert, M.D. Chief Executive Officer, Head of R&D, Chief Medical Officer Timothy J. Miller, Ph.D. President, Chief Scientific Officer Christine Silverstein Chief Financial Officer Management Team Max Colao Chief Commercial Officer, Head of Business Development Edward Carr Chief Accounting Offi
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