Abeona Therapeutics Inc. · NASDAQ:ABEO
Abeona Therapeutics — Technical Presentation (November 2022)
26 slides from Abeona Therapeutics's technical presentation, Nov 3, 2022
Slide 1: Topline results from VIITAL, EB-101
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Slide 2: Note regarding forward-looking statements
Note regarding forward-looking statements This presentation contains certain statements that may be forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, including statements relating to the pr
Slide 3: Introduction
Introduction Vish Seshadri, Chief Executive Officer Recessive dystrophic epidermolysis bullosa (RDEB) and EB-101 Vish Seshadri, Chief Executive Officer VIITAL study topline results Igor Grachev, M.D., Ph.D., Head of Clinical Development Takeaways for EB-101 and next steps Vish Seshadri, Chief Execut
Slide 4: EB-101 is the only investigative therapy targeting large chronic wounds
EB-101 is the only investigative therapy RDEB wounds are the targeting large chronic most painful, hard to ° wounds, demonstrating treat wounds that inflict unique value wound healing and pain the greatest burden on proposition reduction with multiple patients & their families years of durability af
Slide 5: Positive VIITAL results: EB-101 delivers clinically meaningful wound healing and pain reduction
Positive VIITAL results: EB-101 delivers clinically meaningful wound healing and pain reduction in large chronic RDEB wounds Statistically significant improvement vs. control at 6 months: wound healing rate (co-primary endpoint) Pain reduction (co-primary endpoint) 275% wound healing rate (explorato
Slide 6: Recessive dystrophic epidermolysis bullosa (RDEB) and EB-101
Recessive dystrophic epidermolysis bullosa (RDEB) and EB-101 Vish Seshadri Chief Executive Officer
Slide 7: RDEB: disease with lifelong burden afflicting thousands of U.S. patients
Recessive dystrophic epidermoly: disease with lifelong burden afflicting thousands of U.S. patients * Inherited connective tissue disorder with debilitating pain and systemic complications leading to early death + Primarily characterized by skin blisters and erosions * Caused by mutations in COL7A1
Slide 8: EB-101 restores functional collagen VII to patient's own cells
EB-101 restores functional collagen VII to patient's own cells HEALTHY SKIN RDEB SKIN WITH OPEN WOUND EB-101 TREATED SKIN rls <olagen Vil Fibroblasts 5. TRANSPLANTATION 1. BIOESY Sor ne teeny cenecorreced BEEN a ss epidermal set EES ° 2. SKIN CELL ISOLATION 4, MATURATION Skin cells are expanded and
Slide 9: Phase VIITAL study topline results
' \. a Fig Abeona Phase 3 VIITAL study topline results Igor Grachev, M.D., Ph.D. Head of Clinical Development
Slide 10: Phase VIITAL study evaluated EB-101 using intra-patient randomization of wounds
Phase 3 VIITAL study evaluated EB-101 for wound healing and pain reduction using intra-patient randomization of wounds Vi ITA L FDA-aligned endpoints include 250% wound healing PHASE 3 STUDY and mean pain reduction after 6 months Co-Primary Endpoints: Target Enrollment: Randomized + 250% wound heali
Slide 11: VIITAL study baseline characteristics
VIITAL study baseline characteristics # patients treated * 11 patients (every patient biopsied received EB-101 treatment) * 43 treated wounds vs. 43 paired untreated wounds (randomized) lores chuonkewounds + 14non-randomized treated wounds Age (years) * Mean: 22.5; Range: 6 to 40 Body surface area (
Slide 12: Handling of missing data for primary analysis
Handling of missing data for primary analysis 250% wound healing rate Pain reduction analysis * Wounds with missing wound healing data * Wound pairs with missing pain data at are considered as “not healed” for the baseline are excluded from the primary primary analysis analysis * Four randomized wou
Slide 13: Significantly more wounds achieved ≥50% healing and showed significant pain reduction with EB-101
Significantly more wounds achieved =50% healing and showed significant pain reduction with EB-101 % Wounds with 250% Healing Mean Pain Reduction* at six months vs. baseline from baseline at 6 months EB-101 Control n=43 wound pairs 9 < = 81% p-value: <0.0001 O60 16% The mean pairwise difference acros
Slide 14: EB-101 showed greatest pain reduction benefit in wounds with severe baseline pain
EB-101 showed greatest pain reduction benefit in wounds with severe baseline pain Mean Pain Reduction in EB-101 Treated Wounds (incl Randomized and Non-randomized) from baseline at 6 months All treated wounds All treated wounds with baseline pain 26 “ Abeona
Slide 15: Greater wound healing is associated with greater magnitude in pain reduction
Greater wound healing is associated with greater magnitude in pain reduction Mean Pain Reduction from baseline at 6 months <50% healing 250% healing 275% healing Complete healing n=8 n=28 1.75 3.86 45 Abeona
Slide 16: EB-101 significantly improved wound healing vs. control across all levels of healing
EB-101 significantly improved wound healing vs. control across all levels of healing % Wounds that Met or Exceeded Healing Threshold Indicated at six months vs. baseline (n=43) 81% p-value: <0.0001 @ EB-101 65% p-value: Control <0.0001 p-value: 0.0160 250% Healing 275% Healing Complete Healing* Comp
Slide 17: Stringent criteria applied to score wounds as completely healed
Stringent criteria applied to score wounds as completely healed e Complete re-epithelialization with no drainage or erosion e No major crusting as adjudged by investigator (subjective) e In VIITAL, with any crusting, inability to verify underlying epithelial formation led to wound scored as not havi
Slide 18: Example of ≥75% healed after EB-101 treatment
Example of 275% healed after EB-101 treatment (upper left thigh) Baseline Tattooed wounds scored as >75% healed but not complete wound healing at Week 24 18 Source: VIITAL patient Abeona
Slide 19: Example of ≥75% healed after EB-101 treatment
Example of 275% healed after EB-101 treatment (right medial and lateral scapula) Baseline Surgery Week 24 ree B3 (treated wound) B3 scored as >75% healed but not B4 (untreated control) complete wound healing at Week 24 w Source: VIITAL patient Abeona
Slide 20: Examples of 75% and complete wound healing after EB-101 treatment
Examples of 75% and complete wound healing after EB-101 treatment (upper trunk) Baseline E = B4 (treated wound) B4 scored as >75% healed at Week 24 E9 (treated wound) E9 scored as complete wound healing at Week 24 20 Source: VIITAL patient Abeona
Slide 21: EB-101 was shown to be well tolerated in VIITAL
EB-101 was shown to be well tolerated in VIITAL, consistent with past clinical trial experience e There were no treatment-related serious adverse events (SAEs) reported and no safety signal observed in the VIITAL study nor in the duration of the clinical development program. Two subjects (2/11, 18.2
Slide 22: Takeaways for EB-101 and next steps
7 whabeets Takeaways for EB-101 and next steps Vish Seshadri Chief Executive Officer
Slide 23: Positive VIITAL results reinforce EB-101 value proposition
Positive VIITAL results reinforce EB-101 value proposition v4 Statistically significant and clinically meaningful results across endpoints in VIITAL — Wound healing by investigator assessment at all levels vs. control - Pain reduction reported by patient vs. control vf More pronounced pain reduction
Slide 24: Phase 1/2a data complements VIITAL with evidence of multi-year wound healing
Phase 1/2a data complements VIITAL with evidence of multi-year wound healing and pain reduction after EB-101 % of Wounds with 250% Healing Overall Wound Pain: Relief Associated with EB-101 (20/38) % Painful Wounds (n/N) 6-month timepoint agreed with FDA for efficacy primary endpoints 93 a 6 12 24 36
Slide 25: EB-101 Anticipated Next Steps
Regulatory + BLA filing in 2Q 2023 EB-101 _ a ; " A oA * Application for Priority Review Voucher at time of BLA filing Anticipated * Potential BLA approval in 1Q 2024 Next Steps Commercial Launch * Initiate launch preparation activities in 1Q 2023 while continuing to explore partnership opportunitie
Slide 26: Abeona Therapeutics
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