Corporate Presentation June 2020 Abeona Therapeutics Corporate - - PowerPoint PPT Presentation

Abeona Therapeutics Corporate Presentation, June 2020

Corporate Presentation

June 2020

Abeona Therapeutics Corporate Presentation, June 2020

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 opportunities for all of the company’s products and product candidates, and the company’s goals and

• bjectives. These statements are subject to numerous risks and uncertainties, including but not limited to the risks detailed in

the Company’s Annual Report on Form 10-K for the year ended December 31, 2019, including risks associated with the ongoing coronavirus/COVID-19 pandemic (as further explained in the Company’s “Stakeholder Letter in Response to the COVID-19 Pandemic” dated March 27, 2020) and other reports filed by the company with the Securities and Exchange Commission. This presentation does not constitute an offer or invitation for the sale or purchase of securities or to engage in any other transaction with Abeona Therapeutics or its affiliates. The information in this presentation is not targeted at the residents of any particular country or jurisdiction and is not intended for distribution to, or use by, any person in any jurisdiction or country where such distribution or use would be contrary to local laws or regulations. The Company undertakes no obligations to make any revisions to the forward-looking statements contained in this presentation or to update them to reflect events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments or

• therwise.

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Abeona Therapeutics Corporate Presentation, June 2020

• Pivotal Phase III clinical trial in RDEB initiated 1Q20
• Ongoing Phase I/II clinical trials in MPS IIIA and MPS IIIB
• CLN program approaching the clinic
• Next generation AIM™ AAV capsid platform: in vivo proof of concept data in various disorders
• Fully-functional GMP facility validated and governed by comprehensive quality systems

⎼ Phase III manufacture for EB-101 ⎼ Capable of clinical and commercial production of AAV gene therapies

• $116M in cash, cash equivalents and marketable securities (March 31, 2020)

Abeona: A Fully-Integrated Gene & Cell Therapy Company

COMPREHENSIVE GENE & CELL THERAPY CAPABILITIES GENE & CELL THERAPY EXPERTISE AND MANUFACTURING CAPABILITIES LATE-STAGE FIRST-TO-MARKET OPPORTUNITIES ROBUST PIPELINE OF CLINICAL STAGE AND PRECLINICAL PROGRAMS AAV9 AND PROPRIETARY AAV (AIMTM) PROGRAMS BREAKTHROUGH THERAPY

WITH GENE CORRECTED

CELL THERAPY

3

Abeona Therapeutics Corporate Presentation, June 2020

Robust Pipeline

4

Abeona Therapeutics Corporate Presentation, June 2020

• RDEB Review
• EB-101 Clinical Program

ORPHAN DRUG DESIGNATION (FDA)

✓

ORPHAN DRUG DESIGNATION (EU)

✓

RARE PEDIATRIC DISEASE DESIGNATION (FDA)

✓

BREAKTHROUGH THERAPY DESIGNATION (FDA)

✓

REGENERATIVE MEDICINE ADVANCED THERAPY DESIGNATION (FDA)

✓

Abeona Therapeutics Corporate Presentation, June 2020

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:

• Severe pain and widespread scarring
• Numerous debilitating and life-threatening systemic complications
• Inflammation, infections, loss of heat - high metabolic rate and malnutrition
• 75-90% risk of developing squamous cell carcinoma (SCC)

EB: Devastating Inherited Connective Tissue Disorder

50%

• f generalized severe

patients die before 35

75%

die before 40

The lack of functioning anchoring fibrils in RDEB patients leads to skin blistering and tears with minor trauma normal skin affected skin

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Abeona Therapeutics Corporate Presentation, June 2020

Lifelong Burden with No Relief and No FDA Approved Treatments

• Current wound care is wrapping and bandaging to prevent trauma, promote healing, prevent

infection

• These treatments are inadequate and:

Do not address underlying disease cause Are time intensive and burdensome with daily changes Reliance on opioids due to pain associated with cleaning wounds Are costly with spend over $150,000 per year

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Abeona Therapeutics Corporate Presentation, June 2020

Recurrent Wounds Over Time (N=25)

Recurrent and Chronic 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

% Baseline Wound Area Weeks

Recurrent Wounds Over Time (N=25)

Weeks % Baseline Wound Area

Chronic Wounds Over Time (N=25)

Natural history of chronic wounds in patients with recessive dystrophic epidermolysis bullosa; Solis, D. et al.; Journal of Investigative Dermatology, Volume 137, Issue 5, S37

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Abeona Therapeutics Corporate Presentation, June 2020

Size Distribution According to Wound Types

Sequentially Photographed Wounds: N=25 patients, 62 wounds

Natural history of wounds in patients with recessive dystrophic epidermolysis bullosa; Teng et al., Abstract #251; Society of Investigational Dermatology Annual Meeting, 2019

9 p = 0.001 p = NS

% of Wounds

Recurrent

20-39 cm2 ≥40 cm2 ≤19 cm2 26 cm2

Mean Size

5 years

Mean Duration Chronic Open

118 cm2 7 years

Large wounds are the most urgent to treat as they carry the greatest burden, including Pain, Pruritis, and Risk of infection

70% 60% 50% 40% 30% 20% 10% 0%

64% 27% 21% 20% 15% 53%

Abeona Therapeutics Corporate Presentation, June 2020

Natural history of wounds in patients with recessive dystrophic epidermolysis bullosa; Teng et al., Abstract #251; Society of Investigational Dermatology Annual Meeting, 2019

Chronic Open Wounds Have Greater Pain and Itch

10

Sequentially Photographed Wounds: N=25 patients, 62 wounds

0.5 1 1.5 2 2.5 Itch 1 2 3 4 5 6 Pain

Pain Itch

Recurrent Chronic Open

Wong Baker FACES™ Pain Rating Scale

5.0 2.3

Itch Man Scale

2.4 1.5

Abeona Therapeutics Corporate Presentation, June 2020

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 – e.g. keratinocytes
• Durably restore COL7 function by correcting keratinocyte progenitors
• Address the most common and challenging patient wounds

⎼

Chronic wounds, which average ~ 118 cm2

⎼

Recurrent wounds, average ~30 cm2

11

Abeona Therapeutics Corporate Presentation, June 2020

EB-101: Ex-Vivo Autologous Gene Corrected Breakthrough Therapy

12

Keratin inocyte c cell lls expanded and p prepared t the f for n next t step. 10 10-12 d 12 days o s of continued c cell l maturatio ion a and growth. Kerati tinocy cyte te c cells e expanded a and harves ested ed as 5 5.5x7.5 .5cm s sheet ets. Functio ional T Type V VII c colla llagen transduce ced i into to k kerati tinocy cyte te cells ls; c cell m l maturatio ion l leads t to gene-correc ected ed t type V VII c collag agen en expres ession. .

GENE-CORRECTED CELL THERAPY THAT RESTORES NORMAL FUNCTIONAL COLLAGEN VII TO KERATINOCYTES AND THEIR PROGENITORS

EB EB-101 i 101 is transp nsplant nted o

• nto

patie ients’ w wounds i in as l little le a as 26 d 26 days. Two 8 8mm mm s skin b n biopsies s shi hipped t to Abeona, p a, produced ed i into 6 6-8 8 ~40 c 0 cm2

2

sheet eets. .

• PERSONALIZED TREATMENT
• BIOPSY TO PATIENT-READY IN ~26 DAYS
• 2 SKIN BIOPSIES = 6 SHEETS = 240 CM2

Abeona Therapeutics Corporate Presentation, June 2020

Study Description

A Phase III Clinical Trial of Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa (RDEB) using EB-101 for autologous tissue transplantation

Trial Design

• Multi-center, randomized trial led by Stanford University
• 10-15 RDEB patients, with approximately 30 chronic wound sites treated in total
• Follow-up visits at 1 month-12 months, then yearly in a long-term follow-up protocol

until year 5

Primary Endpoint

The proportion of wounds with healing at three months, comparing treated with untreated wound sites on the same patient

Secondary Endpoints

Patient’s global impression of change in pain from baseline as well as other patient reported outcomes assessing pain during dressing changes, pain impact and physical function.

The Phase III VIITALTM Study: A Pivotal Clinical Evaluation of EB-101 for RDEB

First patient treated in Q1 2020; additional 10 patients prescreened

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Abeona Therapeutics Corporate Presentation, June 2020

Phase I/IIa trial addressed wounds of increasing severity and complexity

• Study participants had challenging wounds representative of those most troublesome for the RDEB population
• Learnings from program de-risked Phase III, providing essential guidance for future wound treatment

Abeona believes most wounds, regardless of size or duration, can be addressed with EB-101

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 1-12 months post treatment; yearly thereafter until year 5

EB-101 Phase I/IIa Trial – Addressed Complex and Challenging Wounds

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Abeona Therapeutics Corporate Presentation, June 2020

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% healed < 50% healed

Baseline 3 months 6 months 12 months

EB-101 Restored Collagen 7 that Forms Functional Anchoring Fibrils

Anti-COl7A1 NC2 Mab

Hoechst 33342 15

Abeona Therapeutics Corporate Presentation, June 2020

EB-101 Phase I/IIa Results Demonstrated Durable Efficacy

• Addressing wounds not treated by
• ther gene therapies in

development

• Wounds up to 400 cm2 and open

up to 20 years

16

Abeona Therapeutics Corporate Presentation, June 2020

EB-101 Phase I/IIa: Proportion of Wounds with ≥ 50% & ≥75% Healing

Average wound area healed at 3 and 6 months was 130 cm2 and 120 cm2 (range 130- 157 cm2), respectively per patient

(Note: healed area was calculated based on minimum % healing per wound site, e.g. 50% used for wound sites that healed ≥ 50%)

Pre-Grafting 3 Months 6 Months 9 Months 12 Months

Pain at wound site

58% 0% 17% 20% 0%

Itch at wound site

67% 5% 17% 50% 25%

Lack of durability at wound site

90% 0% 0% 0% 0%

Ease of blistering at wound site

83% 0% 0% 0% 0%

Data Shows Significant Improvement in Patient-ReportedOutcomes

(% reported yes)

17

Abeona Therapeutics Corporate Presentation, June 2020

EB-101: ready for patients

Fully Integrated, Independent and Scalable Manufacturing

Large-scale cGMP capacity and deep expertise

• 26,000 sq. ft facility in Cleveland
• State-of-the-art laboratories to support CMC development for process

and analytics

Control of supply chain, including timelines and cost Internal Abeona quality systems and highly-trained staff Commercial readiness of Abeona’s facility

• Capacity for 120 patients per year at launch
• Potential capacity to support up to 500 patients within a year of launch

18

Abeona Therapeutics Corporate Presentation, June 2020

EB-101 Summary and Anticipated Next Steps

VIITAL™ Phase III Trial

• First patient treated in Q1 2020
• Majority of potential subjects have been pre-screened
• Enrollment completion expected in late-2020, depending on restart time post-COVID-19

Successful Phase I/II

• 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 manufacturing capability at Abeona

• Phase III manufacture in place
• Scalable capacity to support commercial launch

19

Abeona Therapeutics Corporate Presentation, June 2020

• MPS III Review
• ABO-102* and ABO-101 Clinical Programs

ORPHAN DRUG DESIGNATION (FDA)

✓

ORPHAN DRUG DESIGNATION (EU)

✓

RARE PEDIATRIC DISEASE DESIGNATION (FDA)

✓

FAST TRACK DESIGNATION (FDA)

✓

REGENERATIVE MEDICINE ADVANCED THERAPY DESIGNATION* (FDA)

✓

PRIORITY MEDICINES DESIGNATION* (EMA)

✓

Abeona Therapeutics Corporate Presentation, June 2020

Inherited monogenic disorders causing lysosomal enzyme deficiency

• Global incidence varies by regions and it is estimated 0.17-2.35 per 100,000 births1
• Two most common forms categorized by deficient enzymes:

– MPS IIIA (SGSH), MPS IIIB (NAGLU)

• Abnormal accumulation of glycosaminoglycans (GAGs; heparan sulfate (HS))
• Progressive language and cognitive decline, behavioral abnormalities, seizures, sleep

disturbances

• Most children with MPS III have clear dementia by age 3 years
• 70% of children with MPS III do not reach age 18 years

No approved treatments available Ongoing global clinical trials

Cell with lysosome deficiency Normal cell

Sanfilippo Syndrome (MPS III)

• 1. Zelei et al. 2018. Orphanet Journal of Rare Diseases

21

Abeona Therapeutics Corporate Presentation, June 2020

MPS IIIA Natural History: Cognitive & Developmental Assessments

Chronological Age (mo) Cognitive Age Equivalent (mo)

Shapiro et al. 2016

3 6 9 1 2 1 5 1 8 2 1 2 4 2 7 3 3 3 3 6 3 9 4 2 4 5 4 8 5 1 5 4 5 7 6 6 3 6 6 6 9 7 2 7 5 7 8 8 1 8 4 8 7 9 9 3 9 6 9 9 1 2 1 5 1 8 1 1 1 1 1 4 3 6 9 12 15 18 21 24 27 30 33 36 39 42 45

Chronological Age (Month) p g ( ) DQ 100

Chronological Age (mo) Developmental Age (mo)

Truxal, K.V. et al. 2016

36m age equivalent 36m age equivalent

22

Abeona Therapeutics Corporate Presentation, June 2020

A Phase I/II Clinical Trial (ABT-001) for MPS IIIA with AAV9-SGSH

Intravenous Dosing

• Cohort 1: 5 x 1012 vg/kg (n=3 subjects) completed
• Cohort 2: 1 x 1013 vg/kg (n=3 subjects) completed
• Cohort 3: 3 x 1013 vg/kg (n=9-16 subjects) 9 patients treated

Primary Endpoint

• Change from baseline in age equivalent developmental score
• Safety

Secondary Endpoints

• Cerebrospinal Fluid (CSF) and/or urinary HS and/or GAGs
• CSF and serum SGSH enzyme activity
• Liver, spleen and brain volume by MRI
• Neurocognitive function as measured by Leiter International Performance Scale and the Mullen

Scales of Early Learning

• Adaptive functioning, by Vineland Adaptive Behavior Scale (caregiver report)

2-year, open-label, dose-escalation clinical trial

ClinicalTrials.gov: NCT02716246; Study Sponsor Abeona Therapeutics Inc

23

Abeona Therapeutics Corporate Presentation, June 2020

Reduction in CSF ganglioside (GM2 and GM3) Levels Post Treatment

24

* Data from the first three subjects analyzed (A and B from Cohort 1 and C from Cohort 2)

5 10 15 20 25 30 35 40 45 50

Post injections Visits (Months) GM2 (pmol/mL)

001-001 001-002

6 12

001-005

24 48 5 10 15 20 25

Post injections Visits (Months) GM3 (pmol/mL)

001-001 001-002

6 12

001-005

24 48

GM3 GM2

Cohort 1 A* Cohort 1 B Cohort 2 C Cohort 1 A Cohort 1 B Cohort 2 C

Abeona Therapeutics Corporate Presentation, June 2020

Age (Months) Typical developmental pattern for children with MPS IIIA according to Natural History Data** 95% credible interval, incorporating variability from patient-to-patient differences and measurement error. Expected development for children without disease

Mullen’s Cognitive Age Equivalent Post Treatment vs. Natural-History Disease Progression Model

Ages of 3 youngest patients in cohort 3 at treatment: 27 months 19 months 12.5 months

Neurocognitive development of youngest patients preserved 18-24 months post treatment

DQ= Developmental Quotient DQ100 Approximate typical development trajectory (Espen et al. BMC Pediatrics 2013)

DQ100

* *

25

Age Equivalent

Truxal et al, Mol Genet Metab, 2016. Berman et al, J Inherit Metab Dis 2014. Shapiro et al, J Pediatrics, 2016. Wijburg et al, WORLD Symposium, 2018. Data on File. Abeona Therapeutics Inc.

**

Abeona Therapeutics Corporate Presentation, June 2020

Summary of MPS IIIA ABO-102 Phase I/II Study Data

26

Well-tolerated with no treatment-related SAEs and no clinically significant AEs at 18-48 months

• f post-treatment follow-up
• Cohort 1 (n=3; 45-48 months), Cohort 2 (n=3; 37-39 months), Cohort 3 (n=8; 18-33 months)

Evidence of clinical benefit

• Preservation of neurocognitive development in the three young patients treated before 30 months of

age in cohort 3 (18-24 months of follow-up)

• Rapid and sustained, dose-related reduction in disease-specific biomarkers

⎼ CSF levels of heparan sulfate reduced to lower limit of quantitation

• HS levels in the CSF provide evidence of CNS enzyme activity following ABO-102 administration (HS does not cross the blood-

brain barrier)

⎼ CSF gangliosides (GM2 and GM3) reduced significantly, within normal range in the case of GM2

• Sustained decrease in liver volume, with up to 24 months of follow-up in Cohorts 1, 2 and 3

Abeona Therapeutics Corporate Presentation, June 2020

*Clinical study protocol states 3 subjects in Cohort 1; however, due to exceptional circumstances and following robust safety profile and positive review from DSMB, trial was cleared in Europe to advance to Cohort 2 dose

Intravenous Dosing

• Cohort 1: 2 x 1013 vg/kg (n=2 subjects*) completed
• Cohort 2: 5 x 1013 vg/kg (n=5 subjects) completed
• Cohort 3:

: 1 x 1014 vg/kg (n=up to 5 subjects) 2 patients treated Primary Endpoint

• Change from baseline in the Age Equivalent Developmental score
• Safety

Secondary Endpoints

• CSF and/or urinary HS and/or GAG
• CSF and serum NAGLU enzyme activity levels
• Liver, spleen and brain volume (MRI)
• Neurocognitive function as measured by Leiter International Performance Scale and the Mullen

Scales of Early Learning

• Adaptive functioning, by Vineland Adaptive Behavior Scale (caregiver report)

A Phase I/II Clinical Trial (ABT-002) for MPS IIIB with AAV9-NAGLU

2-year, open-label, dose-escalation clinical trial

27

Abeona Therapeutics Corporate Presentation, June 2020

Summary of MPS IIIB ABO-101 Phase I/II Study Data

Well-tolerated with no treatment-related SAEs and no clinically significant AEs or laboratory abnormalities at 1-29 months of post-treatment follow-up

• Cohort 1 (n=2; 15 to 29 months); Cohort 2 (n=5; 5 to 12 months); Cohort 3 (n=2; 1 to 3 months)

Clear biologic effect post treatment

• Decreased CSF HS levels (maintained up to 12 months)
• Reduction in plasma and urine HS and GAGs
• Reduction in liver volume
• Additional assessment of neurological outcomes pending longer follow-up

28

Abeona Therapeutics Corporate Presentation, June 2020

• CLN1 (Infantile Batten) Disease Review
• ABO-202 Preclinical Program

ORPHAN DRUG DESIGNATION (FDA)

✓

ORPHAN DRUG DESIGNATION (EU)

✓

RARE PEDIATRIC DISEASE DESIGNATION (FDA)

✓

FAST TRACK DESIGNATION (FDA)

✓

Abeona Therapeutics Corporate Presentation, June 2020

Infantile Batten Disease (CLN1) and ABO-202 (scAAV9 PPT1) Program

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)

Onset of symptoms between 6 to 24 months of age

• Progressive visual failure, cognitive decline, loss of fine and gross motor skills develop by 2-3 years
• By 5 years of age there is loss of light perception, complete loss of motor skills and social interaction; myoclonus and

seizures can appear eventually

• Death usually occurs by 7 years of age

Therapeutic approach is a scAAV9 vector with codon-optimized CLN1 transgene

• Exclusive, worldwide license secured for AAV9 for treatment of CLN1

30

Abeona Therapeutics Corporate Presentation, June 2020

IND-enabling toxicology and efficacy studies completed

• Strong safety, with no significant toxicology findings in the combination dose escalation study
• Data demonstrated normalized survival, motor function and cognition

Potential benefits of IT/IV dosing include:

• Overcoming IT dose limitations due to volume constraints and bolsters IV doses, especially in older patients
• “Double exposure” to the CNS enables broad and distributed coverage

In-house manufacturing process developed Historical control supported by large, multi-year Natural History Study World-leading investigators and clinical sites for CLN1

• University of Rochester and University of Hamburg

ABO-202: Phase I/II Clinical Trial Readiness

31

Abeona Therapeutics Corporate Presentation, June 2020

Novel AIMTM AAV Platform

Abeona Therapeutics Corporate Presentation, June 2020

AIM™: Next Generation AAV Capsid Platform

AIM™ Capsid Platform

• AAV viral capsids 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 generations have increased tissue tropisms
• Over 100 capsids under evaluation

Potential for redosing previously treated AAV subjects Cystic Fibrosis and Ocular programs demonstrate proof-of-concept to support pre-clinical studies

DNASAL FRAGMENTATION ASSEMBLY AND AMPLIFICATION CHIMERIC CAPSID LIBRARY WILD TYPE AAVS

33

Abeona Therapeutics Corporate Presentation, June 2020

AAV GMP Manufacturing

Abeona Therapeutics Corporate Presentation, June 2020

AAV Gene Therapy Manufacturing and Quality Capabilities

• Experienced and trained staff in Quality, Validation, Process Development, and Assay

Development

• 8,000 sq. ft. of laboratory space
• GMP upstream capacity is 160L moving to 200L 1H2020
• AAV processing GMP facility supportive of clinical translation

⎼

Separate Upstream and Downstream Suites

⎼

Capable of Clinical and Commercial Production

Allegro PallSTR200

35

Abeona Therapeutics Corporate Presentation, June 2020

• Pivotal Phase III clinical trial in RDEB initiated 1Q20
• Ongoing Phase I/II clinical trials in MPS IIIA and MPS IIIB
• CLN program approaching the clinic
• Next generation AIM™ AAV capsid platform: in vivo proof of concept data in various disorders
• Fully-functional GMP facility validated and governed by comprehensive quality systems

⎼ Phase III manufacture for EB-101 ⎼ Capable of clinical and commercial production of AAV gene therapies

• $116M in cash, cash equivalents and marketable securities (March 31, 2020)

Abeona: A Fully-Integrated Gene & Cell Therapy Company

COMPREHENSIVE GENE & CELL THERAPY CAPABILITIES GENE & CELL THERAPY EXPERTISE AND MANUFACTURING CAPABILITIES LATE-STAGE FIRST-TO-MARKET OPPORTUNITIES ROBUST PIPELINE OF CLINICAL STAGE AND PRECLINICAL PROGRAMS AAV9 AND PROPRIETARY AAV (AIMTM) PROGRAMS BREAKTHROUGH THERAPY

WITH GENE CORRECTED

CELL THERAPY

36