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January 2014 Revolutionizing the Treatment of Cancer 2 Safe Harbor Statement The statements that follow (including projections and business trends) are forward-looking statements. Rexahn's actual results may differ mater

Key Takeaway: Revolutionizing the Treatment of Cancer Safe Harbor Statement The statements that follow (including projections and business trends) are forward-looking statements. Rexahn's actual results may differ materially from anticipated results, and expectations expressed in these for

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Revolutionizing the Treatment of Cancer
Safe Harbor Statement
The statements that follow (including projections and business trends) are forward-looking
statements. Rexahn's actual results may differ materially from anticipated results, and
expectations expressed in these forward-looking statements, as a result of certain risks and
uncertainties, including Rexahn's lack of profitability, the need for additional capital to
operate its business to develop its product candidates; the risk that Rexahn's development
efforts relating to its product candidates may not be successful; the possibility of being unable
to obtain regulatory approval of Rexahn's product candidates; the risk that the results of
clinical trials may not be completed on time or support Rexahn's claims; demand for and
market acceptance of Rexahn's drug candidates; Rexahn's reliance on third party researchers
and manufacturers to develop its product candidates; Rexahn's ability to develop and obtain
protection of its intellectual property; and other risk factors set forth from time to time in our
filings with the Securities and Exchange Commission. Rexahn assumes no obligation to update
these forward-looking statements.
Rexahn: Revolutionizing the Treatment of Cancer
Identify novel drug targets which are specific to cancer cells:
Increased efficacy, reduced toxicity
Efficacy against multiple drug resistant cancer cells
Synergism with existing cytotoxic compounds
Develop in-licensed targeted drug delivery platforms:
Nano-Polymer-Drug Conjugate System (NPDCS) combines existing
anticancer agents with a polymer/signaling moiety which directs the
drug directly to the tumor
Lipid-Coated Albumin Nanoparticle (LCAN) to enhance delivery of
Deep Oncology Pipeline
Supinoxin TM (RX-5902) p68 RNA Helicase Inhibitor
RX-3117 Cancer Cell Specific nucleoside analog
Archexin Akt1 Inhibitor
RX-21101 RX-0201-nano Docetaxel Conjugate Akt1 inhibitor
Targeted Drug Delivery Platform
- Anticipated progress during 2014
SupinoxinTM(RX-5902)
Supinoxin: Best-in-Class p68 Helicase Inhibitor
n Inhibition of phosphorylated p68 RNA helicase
n Blocks upregulation of cancer related genes
n Solid tumors: pancreas, NSCLC, colon, renal and other solid
n Anti-proliferative effects
n Synergistic with cytotoxic agents
n Efficacy against drug resistant cancer cells
n Orally bioavailable
n New chemical entity with a strong patent position
n Phase I clinical trial in cancer patients initiated August 2013
n Initial data expected in Q1 2014
Clinical Development
Supinoxin: Mechanism of action
n Phosphorylated p68 is highly
expressed in cancer cells but not in
normal cells, and upregulates
cancer-related genes
n Supinoxin selectively inhibits
phosphorylated p68 RNA Helicase
Decreased proliferation/growth
Synergism with cytotoxic agents
Activity against drug resistant
Cancer cell Proliferation/Tumor growth
Supinoxin: Increased Survival in Human Renal Cell
Carcinoma and Pancreatic Cancer Xenograft Models
Treatment with Supinoxin on days 1
to 20 produced a survival benefit
RX-3117: Novel DNA synthesis inhibitor
n Cancer cell specific nucleoside compound that inhibits DNA
n Activated by UCK1 & UCK2
n Solid tumors: pancreas, NSCLC, colon, renal and other solid
n Effective against gemcitabine-resistant human cancer cell
n Orally administered
n Specifically targeted against cancer cells; reduced adverse
n New chemical entity with a strong patent position
n Completed exploratory Phase I clinical trial in cancer patients
Confirmed oral bioavailability and safety
n Phase Ib clinical trial in cancer patients initiated Dec 2013
Clinical Development
RX-3117: Compelling Efficacy in Animal Models
RX-3117 has shown robust anti-tumor effects across a broad variety
of tumor types in animal models (Colon, Non-Small Cell Lung, Small
Cell Lung, Pancreatic, Renal, Ovarian and Cervical)
RX-3117: Compelling Efficacy in Animal Models
RX-3117 offers significant benefits based on overall-survival via oral
administration in nude mice
RX-3117: Efficacy in Gemcitabine-Resistant Cell Lines
The efficacy of RX-3117 was examined in 12 different human tumor
(Colon, Non-Small Cell Lung, Small Cell Lung, Pancreatic, Renal,
Ovarian and Cervical)
RX-3117: Exploratory Phase I clinical Trial (Completed)
n Exploratory Phase I clinical trial in cancer patients was conducted in Europe in 2012
Evaluate oral bioavailability and pharmacokinetics
Assess safety and tolerability
n Drug administration cohorts:
Nine subjects, ages 47 to 67 years, were enrolled
RX-3117 was orally bioavailable with Tmax of 2-3 hours, T1/2 of 14-21 hours, and
oral bioavailability of 33 to 56%
RX-3117 was well tolerated with no post-dose adverse events, laboratory
abnormalities, or ECG changes emerging through 7 days of follow-up
RX-3117: Phase Ib Study Design (ongoing)
n Initiated December 2013
n Cancer patients with solid tumors
n Up to 30 patients and three clinical sites
n Treatment cycle is 28 days
Dosing 3 times a week for 3 weeks followed by 1 week off
n Dose Finding Study Design
Escalation decisions based on safety, dosing, PK, laboratory, etc
Patients may receive up to 8 cycles of treatment
Anti-tumor activity secondary endpoint
Last updated: Jan 14, 2014