Ophthalmology Innovation SummitAAO 2016

October 13, 2016

Forward Looking Statement

This presentation contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These forward looking statements are made only as the date thereof, and Ohr undertakes no obligation to update or revise the forward looking statement whether as a result of new information, future events or otherwise. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of various factors and uncertainties, including the future success of our scientific studies, our ability to successfully develop products, rapid technological change in our markets, changes in demand for our future products, legislative, regulatory and competitive developments, the financial resources available to us, and general economic conditions. For example, there can be no assurance that Ohr will be able to sustain operations for expected periods. Shareholders and prospective investors are cautioned that no assurance of the efficacy of pharmaceutical products can be claimed or assured until final testing; and no assurance or warranty can be made that the FDA will approve final testing or marketing of any pharmaceutical product. Ohr's most recent Annual Report and subsequent Quarterly Reports discuss some of the important risk factors that may affect our business, results of operations and financial condition. We disclaim any intent to revise or update publicly any forward-looking statements for any reason.

Ohr Pharmaceutical Overview

• Ophthalmology-focused company with compelling early and late stage pipeline– Utilize innovative drug development strategies to meet patient needs in ocular disease

• Lead program – Squalamine lactate ophthalmic solution – Phase 3 program in wet AMD actively enrolling – Marked visual acuity benefit of combination therapy using topical Squalamine and

anti-VEGF injections in phase 2 study in wet AMD

• Additional indications for Squalamine – Retinal vascular occlusive disease and diabetic retinopathy– Visual acuity benefit seen in investigator sponsored trial in retinal vein occlusion

• Sustained release platform technology – Four active internal programs

• Strong management team with deep expertise in ophthalmology drug development

Unmet Need: A Topical Therapy That Improves and Maintains VA Gains

Increase Initial Visual Acuity Gains

Maintain Long Term Visual Acuity Gains

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Squalamine in the Wet AMD Armamentarium

VEGF-R1

Lucentis®Genentech/Novartis

Avastin®Genentech/Roche

RTH258Alcon/Novartis

VEGF

VEGF-R2

Eylea®Regeneron/Bayer

PDGF Fovista®Ophthotech/Novartis

REG-2176Regeneron

Squalamine

PDGF-R bFGF-R

Extracellular

Intracellular

DARPinAllergan

Prevents downstream signaling of multiple angiogenic factors

Vascular Biology of CNV

12%

39%

49%

Incidence

Mixed Classic/Occult CNV

Occult CNV

Classic CNV

AJO 2014; 158: 769-779

Responsive to anti-VEGF therapy • Reduction in leakage and at least partial vascular involution • Located between the retina and retinal pigment epithelium (RPE) • Vessels tend to be very rapidly growing and frequently manifest

symptoms early with leakage and bleeding which causes vision loss • Mostly immature vessels with little pericyte coverage in the initial stages

of development

Classic Neovascularization

Primary target for combination therapy • More resistant to anti-VEGF therapy – reduction in leakage but little

effect on anatomy • Located beneath the RPE, just above the normal choroidal vascular

layers • Vessels grow slowly and (as much as 30%) may be asymptomatic and

only later develop exudation and become clinically evident• More mature vasculature and more pericyte coverage

Occult Neovascularization

Phase 2 Study in Wet AMD

Mandated Lucentis retreatment SD-OCT evidence of:• Any Retinal Cystic Changes• Any Retinal Fluid• Any Subretinal Fluid• Meaningful RPE Elevation

Treatment-naïve patients with exudative AMD• All lesion compositions (classic, mixed and occult only)• Up to 12 disc areas in size• VA 20/40 to 20/320

Lucentis® is a registered trademark of Genentech Inc.

Positive Visual Outcomes in Squalamine Combination Therapy Driven by Occult CNV Size

• Occult CNV size at baseline directly correlates with week 36 visual acuity outcomes in Squalamine combination therapy

• This was independent of the presence/absence of classic CNV • This relationship was not observed with ranibizumab

monotherapy treatment.• Consistent positive visual acuity benefit with Squalamine

combination therapy in occult CNV < 10mm2 (~ 4 Disc Areas)

Occult CNV < 10mm2 Occult CNV > 10mm2

Positive Visual Outcomes in Occult CNV < 10mm2

Efficacy Consistent Through Month 9

Mean Change in Visual Acuity (Letters)

+5.3 letters P = .038

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Exploratory p-value

Positive Visual Outcomes in Occult CNV < 10mm2

Efficacy Consistent Through Month 9

Proportion with > 3 line VA Gain

54% Benefit

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Improved Functional Vision OutcomeBetter Final VA with Squalamine Combination Therapy

*One additional subject did not have a VA assessment at week 36

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Phase 3 Study Design

Treatment-naïve patients with exudative AMD• All lesion compositions (classic, mixed and occult only)• Occult CNV (if present) <10 mm2

• VA 20/40 to 20/320

Primary Endpoint: Visual acuity gain at month 9

Squalamine Lactate Ophthalmic SolutionSummary• Squalamine has the potential to be a convenient and cost-effective

therapy in retinal disease

• Indication in Exudative AMD– Unmet need for improved vision beyond anti-VEGF monotherapy– Strong phase 2 data supporting role of squalamine combination therapy in providing

clinically meaningful vision gains

• Use with Multiple Anti-VEGF Agents– Combination effect with all current and future anti-VEGF agents– Topical delivery adaptable for use with any anti-VEGF treatment regimen/frequency

• Multiple back-of-the-eye indications– Initial data in investigator led clinical trial evaluating retinal vein occlusion indicate

improved vision outcomes– Suggests potential role in retinal vascular disease (RVO and DME)

Ohr Pharmaceutical Overview

• Ophthalmology-focused company with compelling early and late stage pipeline– Utilize innovative drug development strategies to meet patient needs in ocular disease

• Lead program – Squalamine lactate ophthalmic solution – Phase 3 program in wet AMD actively enrolling – Marked visual acuity benefit of combination therapy using topical Squalamine and

anti-VEGF injections in phase 2 study in wet AMD

• Additional indications for Squalamine – Retinal vascular occlusive disease and diabetic retinopathy– Visual acuity benefit seen in investigator sponsored trial in retinal vein occlusion

• Sustained release platform technology – Four active internal programs

• Strong management team with deep expertise in ophthalmology drug development

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