Corporate Overview NASDAQ: CLRB

Transcription

1 Corporate Overview NASDAQ: CLRB

2 Safe Harbor Statement 2 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.

3 Investment Highlights 3 Platform creating the next generation of targeted therapies Multi-asset product portfolio for treatment of various cancers CLR 131 median overall survival exceeding approved drugs Platform validated through multiple partnerships Portfolio of intellectual property supporting assets & platform New management team with extensive healthcare leadership

4 The Challenge Today 4 Off-target drug delivery increasing toxicity and impacting outcomes Targeted therapy objective - improve toxicity and outcomes Antibody drug conjugates (ADCs) Specific pathway inhibitors (SPIs) Reality neither has delivered on the promise Significant adverse events Limitations on efficacy Significant need remains Tumor cell targeting Efficacy and adverse event profile Quality of life

5 Our Platform Technology 5 Precision targeting of tumor cells Validated in vitro, in vivo, and human clinical trials Phospholipid Drug Conjugates TM (PDCs) Delivers a diverse collection of industry-leading anti-cancer payloads Successfully delivering various classes of compounds to tumors Unique linker chemistry used to provide versatility and further specificity Innovative cleavage systems and attachment methods to payloads

6 Integrated Strategic Growth Drivers 6 Platform Best in class targeting Greater specificity Delivery directly to the cytosol Increased payload diversity Targeting unique validated pathways Operational Excellence Accelerated screening paradigm Improved manufacturing processes Reducing time to commercialization Capital efficient development model Development and Market Focus Accelerated regulatory pathway Rare/orphan cancers Limited treatment options Unmet medical need Strategic Collaborations Access to proprietary payloads Accelerate drug development Defray R&D costs Generate non-dilutive capital

7 1. Partially funded by $2M NCI Fast Track Grant 2. Predominately funded by NCI SPORE 3. Cost sharing with University of Wisconsin PDC Proprietary Product Pipeline 7 PDC Program Payload / Mechanism of Action Indication Discovery Pre-IND Phase 1 Phase 2 Collaboration Multiple Myeloma Phase 1 Multiple Myeloma 1 Phase 2 CLR 131 Iodine-131 / DNA Damage Hematologic Tumors 1 Phase 2 Head & Neck 2 Pre-IND Pediatric 3 IND CTX CLR 1700 Series Proprietary / BTK Inhibitor Hematologic Tumors Pre-IND CTX CLR 1900 Series Proprietary / Cell Cycle Arrest Solid Tumors Discovery

8 PDC Partnered Product Pipeline 8 PDC Program Payload / Mechanism of Action Indication Discovery Pre-IND Phase 1 Phase 2 Collaboration CTX CLR 1800 Series Proprietary / Translation Inhibition Solid Tumors Pre-IND CTX CLR 2000 Series Proprietary / Cytoskeleton Disruption Performancebased Discovery CTX CLR 2100 Series Proprietary / Ribosomal Inhibitor Solid Tumors Discovery CTX CLR 2200 Series Proprietary / Tubulin Disruption Solid Tumors Discovery

9 Advancing Innovation in Targeted Therapies 9 Mechanism #1 Targeting Mechanism #2 Entry Mechanism #3 Release 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

10 CLR 131 Targeted Radiotherapeutic Lead PDC Molecule in Two Clinical Trials

11 CLR 131: Phase 1 R/R Multiple Myeloma Study Overview 11 Phase 1 multicenter, open-label, dose-escalation trial Primary objective Characterize safety & tolerability Secondary objectives Assess therapeutic activity Establish Phase 2 dose Single-dose infusion demonstrates safety, tolerability & efficacy Cohort 1 Cohort 2 Cohort 3 Cohort 4 Cohort mci/m mci/m mci/m mci/m mci/m 2 x 2 2 Doses of CLR 131 Provide a Statistically Significant Survival Benefit vs. a Single Dose in Preclinical Studies

12 CLR 131: Phase 1 Patient Characteristics & Adverse Events 12 Metric Cohort 1 (12.5 mci/m 2 ) Cohort 2 (18.75 mci/m 2 ) 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 Adverse Events Avg. Number/Patient Avg. Grade/Patient Median Grade Cohort 1 (12.5) Cohort 2 (18.75) Cohort 3 (25) Cohort 4 (31.25) Baseline B2 Microglobulin

13 CLR 131: Phase 1 Median Overall Survival 13 As of 11/3/17 #1 (12.5 mci/m 2 ) Initiation Date 4/ Months ONGOING C O H O Initiation Date 2/2016 #2 (18.75 mci/m 2 ) 15.4 Months ONGOING R T S Initiation Date 10/2016 #3 (25 mci/m 2 ) 10 Months ONGOING TIME (MONTHS) 25

14 CLR 131: Phase 2 Study in R/R B-Cell Malignancies 14 Patients screened Supported with a $2,000,000 NCI SBIR 1 Grant 10 MM 2 10 CLL/SLL, MZL, LPL 2 10 MCL 2 Repeat all efficacy assessments Optional second dose (Day ) Interim efficacy assessments for each cohort MM CLL/SLL, MZL, LPL MCL 2 Repeat all efficacy assessments Optional second dose (Day ) Final efficacy assessments 10 DLBCL 2 Follow-up ( 1 y after last dose) DLBCL 2 Follow-up ( 1 y after last dose) Dose 1 (25 mci/m 2 ) Expand cohorts Total enrolled ~ 80 Dose 1 (25 mci/m 2 ) 1. NCI SBIR - National Cancer Institute Small Business Innovation Research 2. MM, multiple myeloma; 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.

15 Hematologic Market Opportunity 15 Multiple Myeloma Lymphoma CLL 18% Other 50% DLBCL 22% <0.4 <1.0 <1.8 <2.1 <5.7 LPL 2% Mantel Cell 2% Marginal Zone 6% CLL DLBCL Mantel Cell Marginal Zone LPL Other CLR 131 potential to extend overall survival ~ 37.5B MM market size in 2024 >59,000 patients diagnosed annually Expected to nearly double by 2030 Incurable disease All patients will relapse or become refractory Outcomes significantly reduced after 3 rd line CLR 131 potential to address unmet need for better 2nd line or maintenance therapy 5 select lymphomas could generate > $500M in revenue CLR 131 targeted to ~50% of lymphomas >145,000 lymphoma patients diagnosed annually US represents largest portion of lymphoma patients

16 Expanding CLR 131 Utilization into Solid Tumors 16 Head & Neck Cancer Pediatric Tumors Distribution of Childhood Cancer CLR 131 potential to improve patient outcomes and reduce toxicities Statistically significant reduction in tumor growth (preclinically) Reduces the amount of external beam radiation required for treatment Annual head & neck cancer prevalence ~ 62,000 patients per year Nearly 50% experience reoccurrence CLR 131 potential best-in-class radiotherapy for pediatric patients Statistically significant increase in survival based on preclinical studies Statistically significant reduction in tumor growth based on preclinical studies Potential to provide a new & unique therapy to ~50% of pediatric patients ~10,000 new pediatric cancer cases a year

17 PDC Platform Targeted Chemotherapeutics Next-Generation Targeted Delivery

18 Proprietary Chemotherapeutic PDC Programs 18 CLR 1700 Mechanism of Action CLR 1900 Mechanism of Action CLR 1900 Inhibits CLR 1700 payload inhibits Burton s tyrosine kinase (BTK) BTK inhibitors work only in hematologic cancers B-cell malignancies Induces tumor cell apoptosis Currently approved BTK inhibitors generate revenue of ~$4B annually CLR 1900 payload inhibits mitosis (cell division) Targets a key pathway required to inhibit rapidly dividing cells Validated pathway that results in apoptosis of tumor cells

19 Recent Accomplishments and Near-term R&D Milestones 19 Achievements Successfully repositioned company to therapeutic focus Improved efficiencies and reduced operating expenses Expanded I.P. Portfolio CLR 131 development 2016/ Advanced Phase 1 Study Initiated Phase 2 study and achieved MM efficacy hurdle Additional lymphoma indications Submitted Pediatric tumor IND Acquired non-dilutive funding Established multiple corporate partnerships Initiated 2 new PDC programs Key R&D Deliverables CLR 131 development Phase 1 multiple myeloma data readout Phase 2 multiple myeloma data readout Phase 2 Lymphoma data readouts Initiate Phase 1 pediatric tumor study Initiate Phase 1 head & neck study Initiate IND enabling studies CLR 1700 CLR 1800 CLR 1900 Key R&D Deliverables CLR 131 development Initiate Phase 3 study Final readout from all Phase 2 cohorts Phase 1 pediatric tumor study readout Initiate Phase 1 Studies CLR 1700 CLR 1800 CLR 1900 Initiate IND enabling studies CLR 2000

20 Financial Summary 20 Capitalization as of December 26, 2017 Common Stock Outstanding 17,041,446 Reserved for issuance: Warrants 11,835,074 Convertible Preferred Stock 1 962,845 Employee Options 531,729 Fully Diluted 30,371,094 Pro Forma Cash (9/30/17 balance plus October 2017 financing) 2 ~$12.7 million shares of Preferred Stock issued on October 12, 2017, convertible into an aggregate of 962,845 shares of common stock remain outstanding. 2. We had approximately $5.7 million of cash on September 30, 2017 and we raised approximately $7.0 million, net, in the October 2017 financing.

21 Company Leadership 21 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 John Hamill Interim Chief Financial Officer NephroGenex CEO & CFO; Savient Pharmaceuticals Co-President & CFO; PharmaNet Development Group, Inc.- EVP & CFO Jarrod Longcor Chief Business Officer Avillion LLP CBO Melinta Therapeutics, Inc. (formerly Rib- X Pharmaceuticals, Inc). VP Corp Dev and Operations Executive Team with ~90 Years of Healthcare Leadership

22 Investment Highlights 22 Platform creating the next generation of targeted therapies Multi-asset product portfolio for treatment of various cancers CLR 131 median overall survival exceeding approved drugs Platform validated through multiple partnerships Portfolio of intellectual property supporting assets & platform New management team with extensive healthcare leadership