Corporate Overview February 2018 NASDAQ: CYTR
CytRx Safe Harbor Statement THIS PRESENTATION CONTAINS FORWARD-LOOKING STATEMENTS THAT INVOLVE CERTAIN RISKS AND UNCERTAINTIES. ACTUAL RESULTS COULD DIFFER MATERIALLY FROM THOSE PROJECTED IN THE FORWARD- LOOKING STATEMENTS AS A RESULT OF THE RISK FACTORS DISCUSSED IN CYTRX REPORTS ON FILE WITH THE U.S. SECURITIES AND EXCHANGE COMMISSION INCLUDING, BUT NOT LIMITED TO, THE REPORTS ON FORM 10-K FOR THE YEAR ENDED DECEMBER 31, 2016 AND ON FORM 10-Q FOR THE QUARTER ENDED SEPTEMBER 30, 2017. 1
CytRx Investment Highlights Proprietary LADR TM Drug Generation Platform LADR TM (Linker-Activated Drug Release) Technology concentrates drug release at the tumor, potentially minimizing systemic exposure Create novel oncology therapies utilizing LADR TM technology with Ultra High Potency drug payloads Late-stage, technology-validating lead drug candidate Entered into exclusive worldwide license with NantCell Inc. for aldoxorubicin, CytRx s lead anti-cancer drug conjugate Strategic investment and milestones of up to $356 million in addition to royalties Aldoxorubicin, an albumin-binding drug conjugate of doxorubicin, has been tested in over 600 cancer patients 2
CytRx Pipeline Preclinical Phase 1 Phase 2 Phase 3 LADR High Potency Conjugate LADR High Potency Conjugate Plan IND in 2H18 Plan IND in 2H18 NantCell Partnered Product - Aldoxorubicin 2 nd -Line Soft Tissue Sarcoma 2 nd -Line Small Cell Lung Cancer Combo with ifosfamide - STS Ph 3 Completed; NantCell has IND Ph 2 Fully enrolled; NantCell has IND Ph 1b/2 NantCell has IND Combination Trials with Immunotherapy Pancreatic Cancer Squamous Cell Carcinoma Triple-Negative Breast Cancer Ph 1b/2 On-going Ph 1b/2 On-going Planned by NantCell 3
LADR TM Technology: A Potential Breakthrough in Cancer Drugs Targeting Ability Active and passive tumor targeting strategies Cleavable Linker Chemistries for controlled extra- or intra-cellular release of drug Drug Payload Ultra-high potency cytotoxic agents Cancer Drug Shortcomings LADR TM Technology Advantages Limited therapeutic index Prolonged drug exposure Off-target toxic effects Allows drug to accumulate in the tumor Linker reduces release in healthy cells Limited efficacy Ability to deliver drug payloads that are 10-1000x more potent than standard anti-cancer agents Drug resistance LADR TM conjugates can evade traditional drug resistance mechanism 4
Albumin as a Drug Delivery Vehicle Albumin Most abundant protein in human blood plasma Transport molecule Long half-life (20 days) Major source of essential amino acids ( fuel ) for cancer cells Localizes at tumor 5
Mechanism of LADR Conjugates Cytotoxic Agent Linker Albumin 2 1 Cytotoxic Agent Linker Rapid and specific binding to circulating albumin 3 Drug-linker conjugate is infused 4 Tumor cells Linker dissolves in the acidic (low ph) environment, releasing the drug payload Albumin transports drug to the tumor and surrounding microenvironment 6
Why Albumin-Binding LADR Conjugates are Superior to Antibody-Drug Conjugates Chemotherapy Albumin-Binding LADR Conjugates Antibody-Drug Conjugates Potential Indications: Broad Not targeted Potential Indications: Broad Targeted: Using albumin, not receptor-based Potential Indications: Limited by target expression Targeted: Specific receptor required Low Potency High Potency High Potency Immune response to therapy: not likely Immune response to therapy: not likely Immune response to therapy: more likely 7
Antibody Drug Conjugate Limitation: Which Target? >30 different targets being pursued for solid tumors Target selection is critical. Needs to have greater expression on cancer cells versus healthy cells. Most targets require internalization. Historically only 1-2% of ADC is internalized by the tumor. Br J Cancer. 2016 Feb 16; 114(4): 362 367. LADR conjugates bind to a single target: cysteine-34 on serum albumin. Albumin then transports the drug to the tumor resulting in advantageous broader use than a specific ADC. 8
LADR TM Drug Discovery Progress >75 rationally-designed LADR conjugates synthesized and evaluated in 2017 4 lead candidates from two distinct classes of compounds entered toxicology studies One or more clinical candidates to be selected in Q1 2018 9
Albumin Binding LADR Conjugates: Value Proposition Highly Potent Anti-Cancer Activity Minimized Systemic Toxicity Targeted Delivery and Release of Cancer Killing Compounds 10
License with NantCell, Inc. Exclusive worldwide license for aldoxorubicin in all indications NantCell plans to initiate multiple trials with aldoxorubicin in combination with immunotherapy and cell based therapies NantCell is responsible for future development, manufacturing and commercialization $13 M up-front as a strategic investment in CytRx at $6.60 per share split-adjusted, representing a 92% premium to the then current stock price Up to $343 M in potential milestones for regulatory approvals and commercial milestones Increasing double-digit royalties for soft tissue sarcomas Increasing single-digit royalties for all other indications 11
Aldoxorubicin Expanded Development NantCell is expanding aldoxorubicin s use by combining it with immunotherapies and cell based treatments Jan. 18: Commenced Ph. 1b/2 clinical trial in metastatic pancreatic cancer Feb. 18: Initiated Ph. 1b/2 clinical trial in advanced squamous cell carcinoma of the head and neck or non-small cell lung Triple negative breast cancer clinical trial planned to begin in 2018 Pancreatic Cancer Squamous Cell Carcinoma Triple-Negative Breast Cancer 3 rd leading cause of cancer related deaths Only 8% of patients will survive more than five years SCC represents 25-30% of all lung cancers Head & Neck SCC is the 7 th most common cancer globally 15-20% of all breast cancers are triplenegative Approximately every 30 minutes, a woman in the USA is diagnosed with TNBC 12
Recent and Upcoming Catalysts 2017 2018 1Q17: Met with the FDA for aldoxorubicin as a treatment for STS 2Q17: Oral presentation of aldoxorubicin Phase 3 STS data at ASCO 2Q17: Present updated Phase 1b/2 results from combination trial of aldoxorubicin and ifosfamide in advanced sarcomas at ASCO 3Q17: Announced global strategic alliance for aldoxorubicin with NantCell 4Q17: Regain Nasdaq listing compliance 4Q17: File patent applications for LADR TM drug candidates 4Q17: Initiate activities for GMP manufacturing of LADR TM linkers 1Q18: NantCell initiated Ph 1b/2 clinical trials in metastatic pancreatic cancer and advanced squamous cell carcinoma 1Q18: Nominate one or more ultra-high potency LADR TM conjugates for clinical development 1H18: Begin partnership discussions for high potency LADR TM conjugates 1H18: Present data on LADR TM conjugates at major scientific meeting 1H18: Request pre-ind meeting with FDA to gain agreement on development program 2H18: File IND for first-in-human study with LADR TM drug conjugate 13
Financial Summary Cash Position (9/30/17) Cash: Debt (principal, interest & fees): $46.0 M $13.7 M Shares Outstanding (11/1/17) 27.6M Options Weighted-average strike price: $14.28 2.5M Warrants Weighted-average strike price: $4.26 4.0M Including: Strike price $3.033, expires July 2018 2.8M NantCell warrant at $6.60; expires January 2019 0.5M Fully-Diluted Share Count 34.1M Could bring in up to ~$16.6M in additional capital 14
Conclusion Novel LADR TM technology platform with broad applicability for potential partnerships Next generation of LADR TM derived Ultra-High Potency conjugates in development First IND submission for LADR TM conjugate planned for 2018 Global strategic alliance with NantCell for aldoxorubicin in all indications NantCell began testing of aldoxorubicin in combination clinical trials for pancreatic and squamous cell cancers 15