Corporate Overview NASDAQ: CLRB 5/3/18

Transcription

1 Corporate Overview NASDAQ: CLRB 5/3/18

2 Safe Harbor Statement This presentation contains forward-looking statements. Such statements are valid only as of today, and we disclaim any obligation to update this information. These statements are only estimates and predictions and are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. These statements are based on our current beliefs and expectations as to such future outcomes. Drug discovery and development involve a high degree of risk. Factors that might cause such a material difference include, among others, uncertainties related to the ability to raise additional capital required to complete the development programs described herein, the ability to attract and retain partners for our technologies, the identification of lead compounds, the successful pre-clinical development thereof, the completion of clinical trials, the FDA review process and other government regulation, our pharmaceutical collaborators ability to successfully develop and commercialize drug candidates, competition from other pharmaceutical companies, product pricing and third-party reimbursement. This presentation includes industry and market data that we obtained from industry publications and journals, third party studies and surveys, internal company studies and surveys, and other publicly available information. Industry publications and surveys generally state that the information contained therein has been obtained from sources believed to be reliable. Although we believe the industry and market data to be reliable as of the date of this presentation, this information could prove to be inaccurate. Industry and market data could be wrong because of the method by which sources obtained their data and because information cannot always be verified with complete certainty due to the limits on the availability and reliability of raw data, the voluntary nature of the data gathering process and other limitations and uncertainties. In addition, we do not know all of the assumptions that were used in preparing the forecasts from the sources relied upon or cited herein. A complete description of risks and uncertainties related to our business is contained in our periodic reports filed with the Securities and Exchange Commission including our Form 10-K for the year ended December 31, These forward looking statements are made only as of the date hereof, and we disclaim any obligation to update any such forward looking statements. 2

3 Improving Cancer Patient Outcomes Through Innovation Disruptive technology platform creating the next generation of targeted therapies Pipeline composed of projects with multi-billion dollar sales potential as well as ultra-orphan opportunities R&D includes 4 clinical programs for ~$10M 2018 cash burn Platform validated through multiple partnerships Management team with extensive and successful discovery through commercialization experience 3

4 Strong Proprietary Pipeline PDC 1 Indications Discovery Pre-IND Phase 1 Phase 2 Milestones CLR 131 CLR 1700 Multiple Myeloma B-Cell Lymphomas 2 Pediatric Head & Neck 3 Hematologic Tumors Phase 2 Readout 1H2019 Phase 1b: Cohort 5 Data Q32018 mos Update(s) 2018 Phase 2 Readout 1H2019 Phase 1 Initiation Q32018 Phase 1 Initiation 2H2018 CLR 1900 CLR 1800 CLR 2000 CLR 2100 Solid Tumors Solid Tumors Performance-based Solid Tumors Partnerships CLR 2200 Solid Tumors 1. Phospholipid Drug Conjugates 2. Partially funded by $2M NCI Fast Track Grant 3. Predominately funded by University of Wisconsin NCI SPORE Grant 4

5 Advancing Innovation in Targeted Therapies Precision Targeting Enhanced Entry Unique Linker Chemistry & Diversity of Payloads PDC Extracellular Space Lipid rafts region Intracellular (Cytoplasm) Phospholipid ethers (PLEs) provide specific targeting PLEs bind to specific membrane region (lipid rafts) rather than a single epitope Takes advantage of the tumors metabolic need Entry via lipid rafts and transmembrane flipping Delivery directly to cytosol PDCs will accumulate along the Golgi apparatus network and endoplasmic reticulum Custom designed linkers Allows for control of rate, mechanism, and localization of drug release Maximizes therapeutic benefit Based on World Class Research in Phospholipids, Tumor Cell Membranes and Cutting Edge Expertise in Protease Linker Design 5

6 Cellectar s Proprietary Pipeline Projects 1 CLR CLR CLR

7 Corporate Strategy and CLR 131 Overview Multi-faceted development strategy Cellectar to drive ultra-orphan diseases to commercialization LPL 1, MCL 1, pediatric cancers (neuroblastoma, high grade glioma) Reduced clinical development expense with accelerated time to market Premium pricing with significant revenue Manageable and synergistic commercial infrastructure for highly scalable market Attract partners for larger market opportunities and future development Leverage proof of concept study data; relapsed/refractory (RR) multiple myeloma (MM), diffuse large B-cell lymphoma and head and neck cancer Novel targeted radiotherapeutic First in-class PDC currently in a Phase 1 & 2 for multiple tumor types Payload: Iodine-131 (I-131) conjugated to PLE delivery platform I-131 demonstrated to be a tolerated and efficacious radio-isotope Rapid 30 minute IV infusion Orphan drug designation granted for RRMM & neuroblastoma 1. LPL, lymphoplasmacytic lymphoma; MCL, mantle cell lymphoma 7

8 MONTHS CLR 131: Significant Market Need Remain Patients & physicians need new treatments for relapsed or refractory MM Key Unmet Need: Improved Survival Rates and Tolerability Most currently approved drugs for RRMM in third line or later average 11.9 months of survival, including several recent additions Median Overall Survival (mos) in Third Line 18.6 Months Darzalex for treatment of third line patients averages 18.6 months for survival Most treatments are now given in combinations for use in earlier lines of therapy; most frequent is triplet combination More patient-friendly dosing regimens required, fewer infusions, less pills Common adverse events include peripheral neuropathy, infection, deep vein thrombosis, severe cytopenia, fatigue Months 11.9 Months 4.9 Months 2 3 All Drugs Carfilzomib Daratumumab Pomal. (+dex) 4 O N G O I N G 11.9 Months CLR 131 Can Capture Significant Market Share in Third Line or Later Based on an Improved Efficacy, Safety and Tolerability Product Profile 1 Bortezomib 2 Jurczyszyn et al (2014). New drugs in multiple myeloma role of carfilzomib and pomalidomide. Contemp Oncol. 3 Usmani, et al (2016). Clinical efficacy of daratumumab monotherapy in patients with heavily pretreated relapsed or refractory multiple myeloma. Blood Journal. 4 Dimopolous et al (2016). Safety and efficacy of pomalidomide plus low-dose dexamethasone in STRATUS (MM-010): a phase 3b study in refractory multiple myeloma. Blood Rev. 8

9 CLR 131: Strong Multiple Myeloma Preclinical Data Preclinical Results Significant multiple myeloma cell uptake observed in vitro with fresh patient samples Animal models showed tumor volume reduction and survival benefit with single doses Similar to previous in vivo experiments, fractionated doses significantly increase tumor volume reduction and survival benefit Multiple Myeloma Patient Cell Uptake 120% 100% 80% 60% 40% 20% Survival in MM Mouse Model CLR 131 Fractionated Dosing vs. CLR 131 Single Dose and Bortezomib 0% Vehicle CLR uCi 1 CLR uCi Multidose CLR uCi 1 Bortezomib Fractionated Doses of CLR 131 Provide a Statistically Significant Survival Benefit and Tumor Reduction vs. a Single Dose in Preclinical Studies 9

10 CLR 131: Phase 1 R/R Multiple Myeloma Study Overview Primary study endpoints are safety, tolerability and determination of maximum tolerated dose One 30 Minute Infusion Two 30 Minute Infusions Cohort 1 Cohort 2 Cohort 3 Cohort 4 Cohort mci/m mci/m mci/m mci/m mci/m 2 x 2 4 of 4 Stable Disease Metric 4 of 4 Stable Disease Cohort 1 (12.5 mci/m 2 ) 4 of 4 Stable Disease Patient Demographics Cohort 2 (18.75 mci/m 2 ) 2 of 3 Stable Disease, 1 of 3 PR Cohort 3 (25 mci/m 2 ) Cohort 4 (31.25 mci/m 2 ) Average Age Prior # of Treatment Lines Tumor Burden Triple Combination Treatment 4/4 4/4 4/4 3/3 Stem Cell Transplant 1/4 3/4 4/4 2/3 Patients are Stage II or III and Heavily Pre-treated 1. Median Overall Survival 2. Progression Free Survival 3. Overall Response Rate 4. Baseline B2 Microglobulin 10

11 CLR 131: Showing Improvement in Tolerability & Overall Survival (OS) Key Results To Date 1 All cohorts determined to be safe and tolerated by independent DMC No patients experiencing peripheral neuropathy, deep vein thrombosis, cardiotoxicities, embolisms, or GI toxicities Cytopenias most common adverse events All viewed as predictable & manageable Grade 3 fatigue and fever = 7% No change in liver enzymes or renal function Adverse Events Avg. Number 2 Avg. Grade 2 Median Grade Cohort 1 (12.50) Cohort 2 (18.75) Cohort 3 (25.00) Cohort 4 (31.25) Pooled Phase 1 Study: Overall Survival (n=15) 15.0 Months ONGOING Median overall survival not reached to date TIME (MONTHS) 1. Study ongoing n=15 - Final results may differ from data presented 2. Per patient As of 02/20/18 11

12 CLR 131: Phase 2 Study in R/R Multiple Myeloma Patients screened 10 MM Dose 1 (25mCi/m 2 ) Multiple efficacy & safety assessments Interim efficacy assessments for each cohort Expand cohorts MM Dose 1 (25mCi/m 2 ) Multiple efficacy & safety assessments Final efficacy assessments Follow-up ( 1 y after last dose) All patient have potential for second dose days post initial dose with all efficacy and safety assessments repeated for additional 64 days Proposed Phase 3 Pivotal Study Design Program Timing 1 Granted orphan drug designation Phase 3 Pivotal randomized, open-label, multi-center, global study comparing CLR Best Supportive Care (BSC) vs. BSC (N= ) Primary endpoint: Overall response rate or overall survival Secondary endpoints: Clinical benefit rate and duration of response 1. Estimated 2. NCI SBIR - National Cancer Institute Small Business Innovation Research Phase 2 complete 1H2019 Phase 3 initiation 2020 NDA submission 2025 Supported with an NCI SBIR 2 Grant Clinical Costs 1 (USD) Phase 2 $2,500,000 Phase 3 $25,000,000 - $35,000,000 12

13 CLR 131: B-Cell Lymphoma Market Select niche 1 B-cell lymphomas provide significant market opportunity Newly Diagnosed Patients Per Year Relapse/Refractory (R/R) Patients Per Year CLL/SLL ~47,000 ~10,000 Select Lymphoma Market Overview Orphan drug designations MZL ~15,700 ~1,200 Few approved treatments for LPL, MCL and MZL Limited clinical trial competition for LPL, MCL and MZL LPL ~5,000 ~1,000 Positive data in CLL and DLBCL may attract larger partner MCL ~6,200 ~1,600 R/R LPL, MZL and MCL represent ~$800M market opportunity DLBCL and CLL represent multibillion dollar market opportunity DLBCL ~57,000 ~10,000 LPL, MZL, and MCL Offer Potential for Smaller Scale Studies and Accelerated Route to Market 1. CLL, chronic lymphocytic leukemia; DLBCL, diffuse large b-cell lymphoma; LPL, lymphoplasmacytic lymphoma; MCL, mantle cell lymphoma; MZL, marginal zone lymphoma; SLL, small lymphocytic lymphoma 13

14 CLR 131: Phase 2 Study in Select R/R B-Cell Lymphomas 10 CLL/SLL, MZL, LPL CLL/SLL, MZL, LPL Patients screened 10 MCL Interim efficacy assessments for each cohort MCL Final efficacy assessments 10 DLBCL DLBCL Follow-up ( 1 y after last dose) Dose 1 (25 mci/m 2 ) Expand cohorts Dose 1 (25 mci/m 2 ) All patients eligible for a second 25 mci/m 2 dose (day after first dose) Primary endpoint is efficacy as determined by clinical benefit rate (can occur on either dose) Secondary endpoints include overall response rate, overall survival and safety All cohorts currently enrolling; Expect to complete study in 1H19 Upon study completion, individual cohorts may advance to a pivotal trial Supported with an NCI SBIR Grant 1. NCI SBIR - National Cancer Institute Small Business Innovation Research 2. CLL, chronic lymphocytic leukemia; DLBCL, diffuse large b-cell lymphoma; LPL, lymphoplasmacytic lymphoma; MCL, mantle cell lymphoma; MZL, marginal zone lymphoma; SLL, small lymphocytic lymphoma. 14

15 Randomization CLR 131: B-Cell Lymphoma Clinical Development Strategy Proposed Phase 2/3 Adaptive Design Pivotal Study (designed for LPL, MZL, or MCL) Phase 2b Portion Cohort 1 Single dose Cohort 2 Multi-dose Phase 3 Pivotal Portion Optimal dosing N = Interim N = Proposed Phase 2/3 Pivotal Study Design Program Timing 1 Relapsed/refractory niche lymphoma indication Phase 2b enrollment of ~20 patients Phase 3 Pivotal: Single arm Primary endpoint: Overall Response Rate (ORR) Secondary endpoints: Overall Survival, Progression Free Survival (PFS) Assessment Phase 2a to complete 1H2019 Phase 2b/3 initiation 1H2020 NDA submission 2023 Phase 2b $4 - $8M Clinical Costs 1 (USD) Phase 3 Pivotal trial ~$15 - $20M 1. Estimated 15

16 CLR 131: Pediatric Tumor Opportunity U.S. Market Neuroblastoma ~ 700 new cases/year incidence median age 19 months Represents 15% of pediatric cancer deaths Second line treatment is MIBG-I131 High Grade Glioma ~3,600 new cases/year Rhabdomyosarcoma and Ewing s Sarcoma ~1,500 new cases/year Osteosarcoma ~500 new cases/year The majority of these diseases are treated in ~30 centers across the U.S. Cancers in Children 0-14 Years Old Leukemia CNS Lymphomas Soft Tissue Neuroblastoma Renal Bone Tumors Carcinomas Germ Cell Retinoblastoma % 25.4% 10.0% 6.6% 6.1% 4.7% 4.4% 4% 3.4% 2.5% Hepatic Other 1.5% 0.3% Approval in Any Indication May Provide Priority Review Voucher & Potential for NCCN Compendium Listing for Other Tumor Types 1. Also includes Melanoma 16

17 CLR 131: Efficacy in Pediatric Preclinical Models Preclinical Results Various mouse models demonstrate significant uptake of CLR 131 Neuroblastoma, Rhabdomyosarcoma, Ewing's Sarcoma, Osteosarcoma Uptake correlated into reduction in tumor volume and ~50% slowing of tumor growth Minimal adverse effects were seen on hematologic parameters 17

18 Solid Tumors/ Lymphomas Malignant Brain Tumors CLR 131: Pediatric Clinical Development Strategy FDA Agreement on Phase 1 Study Design Level 1 Level 2 Level 3 Add l levels 15 mci/m 2 30 mci/m 2 45 mci/m mci/m 2 N = 1 N = 3 N = 3 N = 3 Level 1 Level 2 Level 3 Add l levels 15 mci/m 2 30 mci/m 2 45 mci/m mci/m 2 Phase 1 Study Primary objective is to determine the safety and tolerability of CLR 131 Accelerated dose escalation design with up to 30 patients Proposed Phase 2/3 Pivotal Study Design Program Timing 5 Granted ODD 1 and RPDD 2 R/R 3 high risk neuroblastoma patient ages 2 to 21 Initial enrollment of patients to confirm dose; upon appropriate efficacy expand into Phase 3 Phase 3 Pivotal Trial single arm, ~65 patients Primary endpoint: Event Free Survival Secondary endpoints: CBR 4, PFS, ORR Phase 1 to complete 4Q2019 Phase 2/3 Pivotal initiation end of 2Q2020 NDA submission 2023 Phase 1 ~$4M Clinical Costs 5 (USD) Phase 2/3 pivotal trial ~$15M 1. Orphan Drug Designation 2. Rare Pediatric Disease Designation 3. Relapsed/Refractory 4. Clinical benefit response rate 5. Estimated 18

19 Cellectar s Proprietary Pipeline Projects 1 CLR CLR CLR

20 Flux per mass (photons/sec/g) Proprietary Chemotherapeutic PDC Program: CLR 1700 CLR 1700 Mechanism of Action CLR 1700 payload inhibits Burton s Tyrosine Kinase (BTK) BTK inhibitors work only in hematologic cancers Induces tumor cell apoptosis Currently approved BTK inhibitors generate revenue of ~$4B annually Program is currently in lead optimization 3.51E E+11 Biodistribution 2.51E E E E hr (n=5) 96 hr (n=8) 5.05E E+08 Liver Tumor Muscle Fat Blood Tissues 20

21 Cellectar s Proprietary Pipeline Projects 1 CLR CLR CLR

22 Proprietary Chemotherapeutic PDC Program: CLR 1900 CLR 1900 Mechanism of Action CLR 1900 payload inhibits mitosis (cell division) Targets a key element in the pathway required for mitosis Payload represents a novel class of molecules and a novel target Pathway inhibition has been validated with other classes of molecules; results in apoptosis of tumor cells Select solid tumor focus CLR 1900 Inhibits PDC Demonstrates Improved Therapeutic Index vs. Parent 22

23 Key Program Development Milestones Program CLR 131 Multiple Myeloma H 2H 1H 2H 1H 2H 1H Phase 1b Fractionated Dose Readout Phase 3 Interim Assessment Phase 1 mos 1 Phase 2 (MM) 2 Initiate Phase 3 Phase 1b Fractionated Dose Readout CLR 131 B-Cell Lymphoma Initiate Phase 2 (DLBCL) Phase 2 (CLL/SLL) 2 Initiate Phase 2/3 Phase 2 Final Readout Phase 2 (DLBCL) 2 Phase 2 (MZL) 2 CLR 131 Head & Neck Initiate Phase 1 Phase 1 Readout Initiate Phase 2 CLR 131 Pediatric Initiate Phase 1 Phase 1 Readout Pivotal Interim Assessment Initiate Phase 2/3 Pivotal CLR 1700 Initiate IND Enabling Studies Select Candidate Initiate Phase 1 CLR 1900 Select Candidate Select Candidate Initiate IND Enabling Studies Initiate Phase 1 1. Median overall survival 2. Topline data Initiations Data 23

24 Financial Summary Capitalization as of March 2, 2018 Common Stock Outstanding 17,388,344 Reserved for issuance: Warrants 11,787,470 Convertible Preferred Stock 1 615,947 Employee Options 517,618 Fully Diluted 30,309,379 Cash balance as of December 31, 2017 ~$10.0 million shares of Preferred Stock issued on October 12, 2017, convertible into an aggregate of 615,947 shares of common stock remain outstanding. 26

25 Strong and Experienced Management Team Management Jim Caruso President, CEO and Director HIP Innovation Technology - EVP & COO; Allos Therapeutics - EVP & CCO; BCI, Novartis, BASF, Bristol-Myers Squibb John Friend, MD Chief Medical Officer Helsinn Therapeutics - SVP & Head of R&D; Akros Pharma, Actavis, Alpharma, Hospira, Abbott Jarrod Longcor Chief Business Officer Avillion LLP - CBO Melinta Therapeutics, Inc. (formerly Rib-X Pharmaceuticals, Inc). - VP Corp Dev and Operations Brian Posner Chief Financial Officer Alliqua BioMedical, Ocean Power Technologies, Power Medical Interventions, Pharmacopeia - CFO; Executive Team with ~100 Years of Healthcare Leadership and Proven Track Record of Development and Commercialization 25

26 Improving Cancer Patient Outcomes Through Innovation Disruptive technology platform creating the next generation of targeted therapies Pipeline composed of projects with multi-billion dollar sales potential as well as ultra-orphan opportunities R&D includes 4 clinical programs for ~$10M 2018 cash burn Platform validated through multiple partnerships Management team with extensive and successful discovery through commercialization experience 26