Slide 1: Investor Presentation September 2015
Investor Presentation September 2015 1
Slide 2: Safe Harbor Statement
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, including as a result of certain risks a
Slide 3: Rexahn: Developing the Next Generation of Cancer Therapies
Rexahn: Developing the Next Generation of Cancer Therapies* 3 REXAHN’S PLATFORM FOR GROWTHNew Management Team – Proprietary Technologies – Robust Clinical & Pre-Clinical Pipeline Therapies target onlycancer cells Unique mechanismof action 3in clinicaltrials Novelproducts in pipeline Efficacy against
Slide 4: Agenda: The Company
AGENDANext Generation of Cancer Therapies The Company The Pipeline The Future 4
Slide 5: Rexahn: At a Glance
Rexahn: At a Glance Clinical stage biopharmaceutical company developing novel targeted cancer therapeuticsselectively destroy cancer cellsspare normal, healthy cellsHeadquartered in Rockville, MarylandNYSE MKT: RNNMarket cap: $105M7% owned by management/insidersCash and investments at June 30, 2015:
Slide 6: New, Experienced Leadership Team
New, Experienced Leadership Team – Built in Last 2 Years Reza Mazhari, Vice President, Translational Medicine Experienced pharmaceutical executive; success taking multiple compounds from concept to clinic Co-Founder of Cardioxyl Pharmaceuticals, VP, Drug Discovery and Development at Cerecor Ted Jeon
Slide 7: A Diversified Portfolio of Targeted Cancer Therapeutics
A Diversified Portfolio of Targeted Cancer Therapeutics DrugCandidate Mechanismof Action Preclinical Phase I Phase Ib/IIa Preliminary Data Clinical Proof of Concept SupinoxinTM (RX-5902) Phosphorylated p68 inhibitor Phase IQ3 2015 Initiate 2016 RX-3117 Cancer cell specific nucleoside analog Phase I
Slide 8: What Differentiates Rexahn's Programs
What Differentiates Rexahn’s Programs: Potential Advantages Over Existing and Emerging Therapies 8 Traditional Chemotherapy PD1 / CAR T-CellTherapies RexahnTherapies Selectively targets cancer cells Reduced adverse events Convenient oral dosing(Supinoxin™ and RX 3117) Active against toughest cancers
Slide 9: Agenda: The Pipeline
AGENDANext Generation of Cancer Therapies The Company The Pipeline The Future 9
Slide 10: Advancing Our Clinical-Stage Products
Advancing Our Clinical-Stage Products* 10 DRUGCANDIDATE POTENTIALINDICATION STATUS MARKETOPPORTUNITY Supinoxin™ Relapsed & Refractory Solid Tumors Phase I >$3B RX-3117 Gemcitabine Resistant Solid Tumors Phase Ib >$4B Archexin® Metastatic Renal Cell Carcinoma Phase IIa >$700M *Company estimates based
Slide 11: Supinoxin Overview
SUPINOXIN™ OVERVIEWPotential First-in-Class Inhibitor of a Unique Cancer Protein The CandidateOrally active, highly potent small molecule inhibitor of phosphorylated p68 (p-p68)Significant Unmet Medical NeedDemonstrated activity in >100 human cancer cell lines including: triple-negative breast, colo
Slide 12: Supinoxin Unique Mechanism of Action
SUPINOXIN™ UNIQUE MECHANISM OF ACTIONPotent, Well-Tolerated with Activity Against Difficult-to-Treat Cancers* 12 Decreases proliferation / growth of cancer cellsSynergy with existing anti-cancer drugsActive against hard-to-treat cancers Supinoxin™ MOA supports a biomarker strategy for patient select
Slide 13: Supinoxin Evidence of Success: Blocks Growth of Triple-Negative Breast Cancer Cells
SUPINOXIN™ EVIDENCE OF SUCCESSBlocks the Growth of Human Triple-Negative Breast Cancer Cells 13 Large opportunity: Triple Negative Breast Cancer represents 20% of breast cancer diagnoses with limited treatment options; potential rapid path to market Human Triple-Negative Breast Cancer (MDA-MB-231) X
Slide 14: Supinoxin Evidence of Success: Survival Benefit in Animal Models
SUPINOXIN™ EVIDENCE OF SUCCESSSurvival Benefit in Human Triple Negative Breast Cancer Animal Models 14 SupinoxinTM cessation Human Triple Negative Breast Cancer (MDA-MB-231) Xenograft Model Supinoxin™ (600 mg/kg, po, 5 qwk x3) Abraxane (5mg/kg, iv, biwk x 3) Vehicle Supinoxin™ (320 mg/kg, po, 5 qwk
Slide 15: Supinoxin Trial Status
SUPINOXIN™ TRIAL STATUSOngoing Phase I Dose-Escalation Trial 15 Primary Endpoints MaximumTolerated Dose(MTD) 25, 50, 100, 150, 225, 300 , 425, 575, and 775 mg dose cycles completePatient enrollment and dosing ongoingMaximum tolerated dose (MTD) not yet achieved Dose Limiting Toxicities Not yet deter
Slide 16: Supinoxin Next Steps
SUPINOXIN™ NEXT STEPSClinical Plan – Determine Clinical Activity Prior to Initiating Pivotal Phase Ib/IIa Clinical Trial 16 DrugCandidate 2015 H1 2016 H2 2016 / 2017 Indication SupinoxinTM(RX-5902) Phase Ib/IIa Phase Ib/IIa Phase Ib/IIa Phase Ib Safety + MTD Study New Study 3 arms Triple-negativebre
Slide 17: Advancing Our Clinical-Stage Products
Advancing Our Clinical-Stage Products* 17 DRUGCANDIDATE POTENTIALINDICATION STATUS MARKET OPPORTUNITY Supinoxin™ Relapsed & Refractory Solid Tumors Phase I >$3B RX-3117 Gemcitabine Resistant Solid Tumors Phase Ib >$4B Archexin® Metastatic Renal Cell Carcinoma Phase IIa >$700M *Company estimates base
Slide 18: RX-3117 Overview
RX-3117 OVERVIEWNovel Next Generation Nucleoside Compound The CandidateCancer cell specific small molecule nucleoside analogue that inhibits DNA and RNA synthesis causing cell death Prodrug activated by UCK2 which is only present in cancer cellsActive following oral administrationSignificant Unmet M
Slide 19: RX-3117 Unique Mechanism of Action
Anti-tumor activities against a broad spectrum of cancersAnti-cancer effect on gemcitabine resistant cancers RX-3117 UNIQUE MECHANISM OF ACTIONWell Tolerated with Tumor-Specific Activity in Drug-Resistant Cancers* 19 RX-3117 MOA supports a biomarker strategy for patient selection ADVANTAGES *Based o
Slide 20: RX-3117 Evidence of Success: Efficacy Across Human Cancer Cell Types
RX-3117 EVIDENCE OF SUCCESSEfficacy Against Broad Range of Human Cancer Cell Types 20 % Tumor InhibitionAnimalXenograftModel Colon Lung Panc Renal Ovary Cervical TUMOR TYPE More effective than Gemcitabine across broad range of human tumor types RX-3117 Gemcitabine
Slide 21: RX-3117 Evidence of Success: Effective Against Gemcitabine Resistant Cancers
Tumor Growth Inhibition RX-3117 EVIDENCE OF SUCCESSEffective Against Gemcitabine Resistant Cancers – Key Advantage 21 25%-40% of cancer patients receiving gemcitabinerapidly become resistant to chemotherapy RX-3117resulted ingreater tumorgrowth inhibitionthan seen withgemcitabine Patient derived xen
Slide 22: RX-3117 Trial Status
RX-3117 TRIAL STATUSOngoing Phase Ib Dose-Escalation Trial Primary Endpoints MaximumTolerated Dose(MTD) 30, 60, 100, 150, 200, 500, 1000, and 1500 mg dose cycles completePatient enrollment and dosing ongoingMaximum tolerated dose (MTD) not yet achieved Dose Limiting Toxicities Not yet determined Saf
Slide 23: RX-3117 Next Steps
RX-3117 NEXT STEPSClinical Plan – Determine Clinical Activity Prior to Initiating a Pivotal Phase Ib/IIa Clinical Trial 23 DrugCandidate 2015 H1 2016 H2 2016 / 2017 Indication RX-3117 Phase Ib/IIa Phase Ib/IIa Phase Ib/IIa Phase Ib Safety + MTD Study New Study 3 arms Pancreatic cancer Bladder cancer
Slide 24: Advancing Our Clinical-Stage Products
Advancing Our Clinical-Stage Products* DRUGCANDIDATE POTENTIAL INDICATION STATUS MARKET OPPORTUNITY Supinoxin™ Relapsed & Refractory Solid Tumors Phase I >$3B RX-3117 Gemcitabine resistant Solid Tumors Phase Ib >$4B Archexin® Metastatic Renal Cell Carcinoma Phase IIa >$700M 24 *Company estimates bas
Slide 25: Archexin Overview
ARCHEXIN® OVERVIEWPotential Best-in-Class AKT-1 Inhibitor The CandidateNovel inhibitor of cancer cell signaling protein, Akt-1, increasing cancer cell deathTargets clinically validated cancer pathwayAlso inhibits drug resistance; synergistic with approved drugsSignificant Unmet Medical NeedCurrently
Slide 26: Archexin Unique Mechanism of Action
ARCHEXIN® UNIQUE MECHANISM OF ACTIONArchexin® Targets A Clinically Validated Cancer Pathway* PI3K/AKT-1/mTOR pathway involved in cancer cell growth and proliferationAKT-1 inhibitionBlocks the development of resistance to mTOR and TKI inhibitorsBlocks the growth/proliferation of cancer cells Decrease
Slide 27: Archexin: A Selective Inhibitor of AKT-1
Archexin®: A Selective Inhibitor of AKT-1 27 Xenograft model using luciferase-expressinghuman pancreatic cancer cells Control +Archexin (1 week) +Archexin (2 weeks) Anti-cancer activity against multiple solid cancer tumorsSynergistic with mTOR and tyrosine kinase inhibitorsPrevents the development o
Slide 28: Archexin Trial Status
ARCHEXIN® TRIAL STATUSCompleted Phase I and Phase IIa Trials 28 Primary Endpoints MaximumTolerated Dose(MTD) 250 mg/m2/d in patients with an advanced cancer after up to two cycles of treatment Dose Limiting Toxicities Grade 3 fatigue; no significant hematological abnormalities Phase I (Cancer Patien
Slide 29: Archexin Status and Next Steps
29 Phase IIa StudyDesign Metastatic renal cell carcinoma (mRCC)Second line therapyAdministered in combination with everolimus (Affinitor®)Part A: Identify maximum tolerated dose in combination with everolimus Part B: Determine safety and efficacy in 30 additional mRCC patients ARCHEXIN® STATUS AND N
Slide 30: Agenda: The Future
AGENDANext Generation of Cancer Therapies The Company The Pipeline The Future 30
Slide 31: Robust Pipeline Targeting Multiple Cancer Indications
Robust Pipeline Targeting Multiple Cancer Indications 31 DrugCandidate 2015 H1 2016 H2 2016 / 2017 Proposed Indications Supinoxin™(RX-5902) Triple-negative breast cancer Colon cancer Ovarian cancer RX-3117 Pancreatic cancer Bladder cancer NSCL cancer Archexin® Metastatic renal cell cancer Phase Ib/I
Slide 32: Advance cancer therapies through proof-of-concept clinical development
Advance cancer therapies through proof-of-concept clinical development Establish partnerships with pharmaceutical companies; focus on maximizing shareholder value Advance pre-clinical oncology programs to address significant unmet needs 32
Slide 33: Investor Presentation September 2015 33
Investor Presentation September 2015 33