cytr corp mar 2015

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Corporate Overview March 2015 NASDAQ: CYTR

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Corporate Overview of Cytrx

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Page 1: Cytr Corp Mar 2015

Corporate Overview

March 2015

NASDAQ: CYTR

Page 2: Cytr Corp Mar 2015

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CytRx Safe Harbor Statement

THIS PRESENTATION CONTAINS FORWARD-LOOKING STATEMENTS THATINVOLVE CERTAIN RISKS AND UNCERTAINTIES ASSOCIATED WITH ADEVELOPMENT-STAGE COMPANY. ACTUAL RESULTS COULD DIFFERMATERIALLY FROM THOSE PROJECTED IN THE FORWARD-LOOKINGSTATEMENTS AS A RESULT OF THE RISK FACTORS DISCUSSED IN CYTRXREPORTS ON FILE WITH THE U.S. SECURITIES AND EXCHANGECOMMISSION INCLUDING, BUT NOT LIMITED TO, THE REPORT ON FORM10-K FOR THE YEAR ENDED DECEMBER 31, 2013, AND FORM 10-Q FORTHE QUARTER ENDED SEPTEMBER 30,2014.

Page 3: Cytr Corp Mar 2015

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CytRx Investment Opportunity

Late-stage, technology-validating lead program Pivotal Ph 3 (SPA) on-going in 2nd-line soft tissue sarcoma (STS) Potential for first FDA approval in 2017 Fast follow on indication: 2nd-line small cell lung cancer Ph 2b underway

Proprietary Oncology Delivery Platform Can be used with a wide variety of oncology compounds Optimized delivery concentrates drug release at the tumor, minimizing

systemic exposure and allowing for higher doses and additional cycles

Risk-mitigated strategy Creates potential blockbuster oncology therapies from proven cancer drugs,

substantially reducing both development and regulatory risk

Page 4: Cytr Corp Mar 2015

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Widely used for many solid and hematological tumors

Class limited by cumulative dose-related cardiotoxicity

No clinically significant cardiotoxicity seen with aldoroxorubicin

Aldoxorubicin: A Novel, Targeted Anthracycline

1960s ’87 ’90 ’95 ‘96 ‘98 ‘99

Doxorubicin

Idarubicin

Doxil

DaunoXome

Epirubicin

Daunorubicin

Aldoxorubicin:Potential 1st

anthracycline NCEin >16 years

Valrubicin

Page 5: Cytr Corp Mar 2015

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Aldoxorubicin: A Best-in-Class, Targeted Anthracycline

Superior efficacy to doxorubicin in comparative trial Ability to administer 3.5x more doxorubicin equivalents at each cycle Potential to treat beyond doxorubicin’s black box dose limit

No clinically significant cardiotoxicity Patients have received up to 8x more cumulative anthracycline with

aldoxorubicin than possible with doxorubicin

Improved quality of life Alopecia less severe and not as frequent as doxorubicin Very well tolerated at lower doses; few Grade 3/4 adverse events

Orphan drug status granted USA: STS, small cell lung, glioblastoma, pancreatic & ovarian cancers Europe: STS

Page 6: Cytr Corp Mar 2015

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Beyond Aldoxorubicin: CytRx R&D

Creation of a continuous pipeline

Lab located in Freiburg, Germany

Design and optimization of new albumin-binding drug conjugates

Rapidly move novel drug conjugates from the bench to the clinic

Led by Felix Kratz, PhD, renowned chemist and inventor of aldoxorubicin, with more than 260 publications on conjugation chemistry and developing novel linkers for improved delivery of chemotherapy

Page 7: Cytr Corp Mar 2015

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Seasoned R&D Team

Felix Kratz, PhD Andre Warnecke, PhD

Dan Levitt, MD, PhD5 oncology drug approvals

Formerly at Roche, Sandoz, Protein Design Labs, Affymax, Dynavax, Geron, Cerimon

Inventor of

aldoxorubicin

Steven Kriegsman

Page 8: Cytr Corp Mar 2015

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Aldoxorubicin Clinical Development Program

Indication Preclinical Phase 1 Phase 2 Phase 3

2nd‐Line Soft Tissue Sarcoma (STS)

1st‐Line Soft Tissue Sarcoma

2nd‐Line Small Cell Lung Cancer

Glioblastoma  Multiforme (GBM)

Kaposi’s Sarcoma

Pharmacokinetic Study

Combo with gemcitabine

Combo with ifosfamide

Phase 2b  on‐goingPhase 2b  on‐going

Phase 2b  CompletedPhase 2b  Completed

Phase 3 Pivotal Trial on‐goingPhase 3 Pivotal Trial on‐going

Phase 1 completed             Phase 1 completed             

Phase 2  on‐goingPhase 2  on‐going

Phase 2 on‐goingPhase 2 on‐going

Ph 1b on‐goingPh 1b on‐going

Ph 1b  on‐goingPh 1b  on‐going

Data Reported

Page 9: Cytr Corp Mar 2015

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Soft Tissue Sarcoma (STS)

Cancer of the soft, connective tissue Affects muscle, fat, blood vessels, nerves, tendons and the lining of

joints (synovial tissues) More than 50 different sub-types

Growing, underserved target market Approximately 40,000 new cases in the

U.S. and Europe annually More than 13,000 deaths annually

Significant unmet medical need Doxorubicin is the standard of care All patients progress on existing therapies

Page 10: Cytr Corp Mar 2015

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Why Soft Tissue Sarcomas?

It is a Model Tumor to establish aldoxorubicin’s POC Single-agent doxorubicin is approved as 1st-line therapy for STS 1st-line STS provided setting for head-to-head Phase 2b trial

comparing aldoxorubicin to doxorubicin Established aldoxorubicin’s clinical superiority to doxorubicin

Why 2nd-line STS for the Phase 3 Pivotal Trial? Significant unmet medical need: no standard of care for patients PFS is acceptable primary endpoint Clinical data to support aldoxorubicin activity in 2nd-line STS patients 1st-line STS had competing trials

Page 11: Cytr Corp Mar 2015

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Phase 3 Pivotal Trial in 2nd-Line STS

Randomized, Comparative Trial Design with SPA−Special Protocol Assessment (SPA) granted by FDA−SPA allows for dosing until disease progression

Patient Population−400 STS patients that have progressed following treatment

with chemotherapy−Up to 5 prior cycles or 375mg/m2 of doxorubicin or liposomal

doxorubicin equivalents allowed Endpoints−Primary: Progression Free Survival−Secondary: Overall survival, response rates, safety, etc.

Page 12: Cytr Corp Mar 2015

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Phase 3 Pivotal Trial in 2nd-Line STS

Trial is currently on-going−Up to 85 sites worldwide−Sites located in the USA, Canada, Denmark, France, Hungary,

Italy, The Netherlands, Poland, Russia, Spain, Sweden, Bulgaria, Australia, Chile, Israel and South Korea

Timing−Enrollment to be completed by year-end 2015−PFS data to be available mid-2016−Projected NDA filing at end of 2016−Market launch in 2017

Page 13: Cytr Corp Mar 2015

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Phase 2b STS Trial: PFS Results

All SubjectsIntent-to-treat

P Value

Scans Read by Investigator

Aldoxorubicin 8.4 monthsP=0.0004

Doxorubicin 4.7 monthsImprovement over dox 3.7 mos. (79%)

Hazard ratio 0.419 (0.25-0.69) P=0.0007

Scans Read by Central Lab

Aldoxorubicin 5.7 monthsP=0.014

Doxorubicin 2.8 monthsImprovement over dox 2.9 mos. (104%)

Hazard ratio 0.584 (0.37-0.93) P=0.024

Page 14: Cytr Corp Mar 2015

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Secondary Endpoint Results

Aldoxorubicin Doxorubicin

PFS at 6 months

Scans Read by Investigator 68.1% 36.6%

Scans Read by Central Lab 45.7% 22.9%

Overall Response Rate

Scans Read by Investigator 21.7% 5.0%

Scans Read by Central Lab 23.8% 0%

Overall Survival

Median Survival months [95% CI] 16 [13.1-Not Reached] 14.4 [8.7-20.9]

Hazard Ratio [95%CI] 0.73 [0.44-1.20]

% of patients surviving ≥2 years 41% 20%

Page 15: Cytr Corp Mar 2015

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Phase 2b: Safety Data

Adverse events were consistent with known doxorubicin toxicities Grade 3 or 4 neutropenia, mucositis and nausea/vomiting are

higher in aldoxorubicin-treated subjects but are not treatment limiting Aldoxorubicin treated subjects had no evidence of clinically

relevant decreased left ventricular ejection fraction (LVEF) ~9% of doxorubicin patients had clinically significant

cardiotoxicity

Page 16: Cytr Corp Mar 2015

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Alopecia: A hallmark side effect of doxorubicin

92% of doxorubicin treated patients experience hair loss1

Typically seen during 1-2 cycles (75mg/m2)

Aldoxorubicin treated patients reported fewer cases of alopecia with less severity Emerges later in treatment beyond cycle 4

Patient from Phase 1 combo trial with gemcitabine No hair loss through cycle 5 Treatment: 170mg/m2 aldoxorubicin

(125mg/m2 dox equivalents) and 1,800mg/m2 gemcitabine

Cumulative dox equivalents of 625mg/m2

1 Source: doxorubicin package insert

Page 17: Cytr Corp Mar 2015

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Small Cell Lung Cancer

~15% of all lung cancers and usually advanced at diagnosis

Almost all cases associated with heavy smoking

Only approved 2nd-line chemotherapy is topotecan Significant toxicities and patients can rarely finish a course of

therapy

Severe need for better treatment options for 2nd-line SCLC

SCLC outcomes with current treatment following first relapsePFS ~3 monthsOS ~7 monthsResponse rate 5-20%

Page 18: Cytr Corp Mar 2015

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Phase 2b Trial in 2nd-Line SCLC

Randomized, Comparative Trial Design−40 clinical sites in the USA, Spain, Italy and Hungary−Treatment until disease progression with lower dose than STS

Patient Population−132 SCLC patients that have progressed or relapsed following

treatment with chemotherapy−Randomized 1:1 to receive either aldoxorubicin or topotecan

Endpoints−Primary: Progression Free Survival−Secondary: Overall survival, response rates, safety, etc.

Timing:−PFS data expected in mid-2016

Page 19: Cytr Corp Mar 2015

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Aldox significantly improves survival in vivo

Glioblastoma Multiforme (GBM)

GBM is one of the most frequent adult primary brain tumors

Median survival is 14 months

5 year survival: ~4%

Phase 2 trial enrolling Evaluating aldoxorubicin as a

treatment in late-stage glioblastoma following surgery, radiation and TMZ

28 patients to be enrolled Patients to be treated until disease

progression

Dox: dashed greenVehicle: dashed red

Aldox treated

P<0.0001

Preclinical GBM results presented at ESMO and AACR Meetings

Orthotopic xenograft mouse model with intracranial implanted human GBM cells

Page 20: Cytr Corp Mar 2015

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Aldoxorubicin enters the brain tumor

Doxorubicin does not enter the brain

Dox+ Aldox+

Tumor

Tumor

Healthy brain tissue

Healthy brain tissue

Aldoxorubicin enters brain and delivers

dox to tumor

Page 21: Cytr Corp Mar 2015

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Preliminary Ph 2 GBM Results

Clinical activity including tumor shrinkage and prolonged stable disease seen in several of the first 12 patients

One patient had a no microscopic evidence of tumor when the tissue was examined after surgery, representing a complete response

Results suggest that aldoxorubicin can cross the blood-brain barrier in humans

Data submitted for presentation at ASCO

Albumin-binding drug conjugates represent a new approach treating GBM and other brain cancers

Page 22: Cytr Corp Mar 2015

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AIDS-Related Kaposi’s Sarcoma

Cancer of the cells that line lymph and blood vessels− Found in immune compromised subjects (late stage AIDS patients)− Manifests as skin +/- internal lesions− Doxil® is the standard of care

Phase 2 trial on-going− Up to 30 patients to receive 50, 100 or 150mg/m2 every 3 weeks until

disease progression, unacceptable toxicity or withdrawal

Positive interim trial results from first 9 patients− Aldoxorubicin can be detected in all tumor biopsies 24 hours after

administration− All 9 patients had a decrease in skin lesions and number of cancer cells

expressing KS virus DNA− 4 of 6 patients with lung tumors had a complete or partial response and no

patient had disease progression

Page 23: Cytr Corp Mar 2015

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Combination Trials

Aldoxorubicin + ifosfamide Doxorubicin + ifosfamide is regularly used to treat STS Phase 1b in patients with advanced soft tissue sarcomas Trial on-going; data expected 2H 2015

Aldoxorubicin + gemcitabine Gemcitabine is routinely administered for many tumor types Phase 1b in patients with advanced solid tumors Trial on-going; data expected 2H 2015 Potential to pursue larger Orphan indications Platinum-resistant ovarian cancer Pancreatic cancer

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1H15: Preliminary Phase 2 GBM results expected

1H15: Preliminary results from Phase 2 Kaposi’s sarcoma trial

1H15: OS data expected from Phase 2b 1st‐line STS trial 

1H15:  Present GBM and KS clinical results at a medical meeting

2H15: Complete enrollment in Phase 3 STS trial

2H15: Phase 1 combo trials results expected

2H15: Initiate Phase 1 for new drug‐conjugate

20152015

Expected Upcoming Catalysts

20162016

1H16: Phase 3 STS PFS data

2H16: NDA filing in STS

2H16: Phase 2b 2nd‐line SCLC data expected

2017: NDA approval for aldoxorubicin in STS

2017: Market launch of  aldoxorubicin for soft tissue sarcoma

2017

Page 25: Cytr Corp Mar 2015

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Financial Summary

Balance sheet (as of 9/30/14)−Cash: $90.0M−Debt: $ 0.0M

Shares Outstanding 55.7M

Options‒ Average strike price: $3.19 6.7M

Warrants‒ Average strike price: $4.80 7.9M

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Conclusion

Aldoxorubicin demonstrates superiority to doxorubicin

Novel delivery platform with broad applicability

Late-stage pipeline with near-term clinical catalysts

Risk-mitigated strategy