Annual Meeting June 7, 2011

Transcription

1 Annual Meeting June 7,

2 Safe Harbor This presentation includes forward looking statements and predictions, including statements about potential revenue bearing transactions, the market potential of CBLI s technologies and product candidates, and the potential value of pipeline products. These statements represent the Company s judgment as of the date of this presentation and are subject to risks and uncertainties that could cause actual results of events to differ materially from those expressed in such forward looking statements. In particular, CBLI faces risks and uncertainties that it may not be able to sustain its business model, that revenues may be lower or expenses higher than projected, that product sales may not increase, that development of product candidates in the Company s pipeline may not succeed or that commercial transactions may not go forward as planned. 2

3 Mission CBLI develops drugs against major unmet biodefense and medical needs Countermeasures against lethal radiation exposure Supportive care drugs against side effects of radiotherapy and chemotherapy of cancer Novel anticancer therapies 3 3

4 Strategic Advantages Unique drug lead & technology portfolio Paradigm shifting innovative drug development and treatment concepts developed by CBLI s founder Prof. Andrei Gudkov Advanced leads of Protectan and Curaxin classes Accelerated drug development path Radiation biodefense countermeasures FDA animal efficacy rule Advanced stage of development Oncology market Alliance with leading partners 4 4

5 Leading Biodefense Drug CBLB502 Landscape Increasingly high risk of nuclear disaster Terrorist attack 10KT nuclear device estimated to cause up to 400,000 casualties if exploded in Manhattan Nuclear stations Fukushima: nuclear industry forced to recognize a need for drugs dealing with consequences of nuclear accidents Lack of approved countermeasures Growing pressure on governments Need for CBLB502 today is higher than yesterday CBLB502 uniquely positioned to become a critical element in defense against such accidents 5

6 Leading Biodefense Drug CBLB502 Competitive Advantages CBLB502 remains strongest radiation countermeasure Lack of reports on comparably powerful drugs Growing information on outstanding efficacy in primates Furthest advancement along the Animal Rule path Established large scale production line Outstanding stability Streamlined accelerated FDA process Recognition of advantages by government agencies Rapid development process facilitated by top quality regulatory team Clear shortlist of remaining steps 6

7 From June 2010 to June 2011 Critical steps forward Accelerated regulatory approval process FDA s Fast Track drug status for CBLB502 FDA s Orphan Drug status for CBLB502 Reduced number of remaining tasks in preclinical program Completion of reproductive toxicity studies Completion of second human trial for CBLB502 in radioprotection 100 subjects Material for strengthening biomarkers Strong foundation for dose conversion Strengthened regulatory and clinical development team EVP Regulatory Affairs and Quality Assurance (Dr. Ann Hards) with outstanding credentials Strategic Regulatory Consultant (Dr. Ray Lipicky) with 21 year history at FDA Strategic Regulatory Consultant (Dr. Thomas Fleming) FDA and NIH advisor on aspects of animal rule and clinical trial design 7 7

8 From June 2010 to June 2011 Opening additional market opportunities for CBLB502 Discovery of direct anticancer action of CBLB502: from supporting care only drug to combined supporting care and anticancer drug Approval of Phase I/II trial of CBLB502 as single agent by Scientific Review Committee of Roswell Park Demonstration of CBLB502 efficacy in preclinical model of local irradiation Approval of Phase I/II trial of CBLB502 as supportive care in head and neck cancer patients by Scientific Review Committee of Roswell Park 8 8

9 From June 2010 to June 2011 Strengthening IP Protection New US patents granted Composition of matter for CBLB502 and CBLB612 CBLB502/612 patents granted in major markets European Patent Organization (EPO), Eurasian Patent Organization (EAPO), China, Japan, New Zealand, Australia, and others New applications covering fresh discoveries 9 9

10 From June 2010 to June 2011 Funding Critical Path $45 million development and conditional purchase contract from Dept. of Defense for CBLB502 recognition of leading position $1.6 million grant from DTRA for CBLB502 Extension of Grand Opportunities grant from NIH: second tranche of $5.3M total 10 10

11 Continuous Successful Pursuit of Non dilutive Funding GRANT/CONTRACT TITLE AMOUNT DATES DoD /DTRA, Med. Chem. & Biol. Defense Res. Program DoD/CBMS-JPEO Chemical Biological Medical Systems Joint Project Mgt. Radioprotective Mechanisms of CBLB502 BAA Advanced Development of a Medical Radiation Countermeasure $1,300,000 3/07-3/10 $10,340,000 3/08-10/09 NIAID (NIH) BioShield Program BARDA (HHS) BioShield Program CBLB502 mitigation of radiation induced thrombocytopenia BAA Development of CBLB502 of mitigation of HP syndrome $1,230,000 9/08-3/10 $15,800,000 9/08-10/10 NIH/NIAID Grand Opportunities (GO) Grant Protectan CBLB502 $5,300,000 9/09-9/11 DoD/CBMS-JPEO Chemical Biological Medical Systems Joint Project Mgt. RFP W9113M-09-R-0010 Advanced Development of a Medical Radiation Countermeasure $45,000,000 (15,000, ,000,000) 9/10-9/13 DoD /DTRA, Med. Chem. & Biol. Defense Res. Program Radioprotective Mechanisms of CBLB502 Several pending proposals >$50,000,000 $1,589,106 1/11-4/12 Expected

12 Major Funding Opportunity for CBLB502 Defense BARDA History of partnership: September 2008: BARDA awarded CBLI a contract "Therapies for Hematopoietic Syndrome, Bone Marrow Stromal Cell Loss, and Vascular Injury Resulting from Acute Exposure to Ionizing Radiation" covering: Performing selected preclinical experiments with non human primates Performing stability studies of the GMP grade CBLB502 Conducting 50 subject healthy volunteer trial Planning, initiating and overseeing 100 subjects dose validation trial on healthy volunteers and drafting and finalizing the clinical report and submitting such reports to the BARDA and the FDA Starting total contract value was $13.3M September 2009: BARDA increased the total contract value adding $2.3 million, the maximum allowed for non competitive increase CBLI successfully completed all tasks related to this contract by February 15, 2011, 6 months ahead of schedule Since 2010, CBLI and BARDA have been negotiating a new contract to fund the remaining steps of the developmental program May 2010: BARDA requested coordination of development program with FDA as a condition for further contract negotiations 12

13 Major Funding Opportunity for CBLB502 Defense BARDA Path to Contract: Meet with FDA to receive input on development path Update BARDA proposal and resubmit BARDA review and award determination 13 13

14 CBLB502 Biodefense on Path to Approval Remaining Tasks Completed GMP process developed and tested, drug suitable for clinical trials released Data from ~1,000 primates demonstrates dramatic survival benefits and accelerated recovery CMC Efficacy Remaining steps Additional consistency runs Pivotal animal studies Human safety Two trials: 50 subject dose escalation and 100 subject study completed Open IND, Fast Track Status, Orphan Drug Status FDA process Definitive safety study Coordinating study protocols, BLA submission 14

15 CBLB502 Scientific Update & Medical Applications 15 15

16 Scientific and Clinical Program Goals Support Animal Rule driven development of CBLB502 for biodefense applications Mechanism of action studies: rational choice of biomarkers Defining CBLB502 efficacy range Determination and validation of human dose Moving CBLB502 to oncology clinic Radiotherapy adjuvant (local irradiation models) Chemotherapy adjuvant Effect on tumors 16

17 Mechanism of Action Survival Mechanism is manifold and sufficiently understood to select efficacy biomarkers and predict potential adverse effects in humans all essential for human dose selection 17

18 Mitigating efficacy of CBLB502 against GI manifestation of acute radiation syndrome in primates (Rhesus macaques) that received extremely high radiation doses Small intestine, day 7 after 11 Gy TBI CBLB502 vehicle 18

19 Human Trial Program Extension A randomized, open label, Phase Ib study in healthy subjects Total of 150 human volunteers received a range of doses of CBLB502 Dose limiting toxicity defined; adverse event profile is predictable and related to known pharmacology of CBLB502 Methodology established to determine projected human efficacious dose (based on biomarkers) All biomarkers project similar human dose 19

20 CBLB502 in Preclinical Model of Local Irradiation Head and neck irradiation model in mice Goal: Justification of use of CBLB502 as a supporting care radioprotection adjuvant Results: CBLB502 is efficacious against radiation induced mucositis and dermatitis Significance: Strong preclinical support of use of CBLB502 as radiotherapy adjuvant Justification of new application (protection from radiation induced dermatitis) Toll like Receptor 5 Agonist Protects Mice from Dermatitis and Oral Mucositis Caused by Local Radiation: Implications for Head and Neck Cancer Radiotherapy. (Int. J. Rad. Onc. Biol. Phys., in press) Approval of CBLB502 as supportive care trial protocol in head and neck cancer patients by Scientific Review Committee of Roswell Park 20

21 Extending Indications of CBLB502 Mitigation of chemotherapy side effects and direct anticancer action Irinotecan and CBLB502 against Wart colon tumors in Fisher rats CBLB502 displays both supportive care and direct antitumor activities in rat model of colon cancer

22 Histological Atlas of CBLB502 Activity Identification of Target Organs Liver is the primary target organ of CBLB502 22

23 Direct Anti tumor Effects of CBLB502 Identification of target tissues enables rational choice of indications and regimens Phase I/II CBLB502 as a single agent trial protocol was approved by Scientific Review Committee of Roswell Park 23

24 Prospective Clinical Trials of CBLB502 in Cancer Patients Reducing severity of mucositis and enhancing efficacy of radiotherapy of H&N cancer Reducing severity of bowel toxicity and enhancing efficacy of radiotherapy of pancreatic cancer Reducing severity of diarrhea in colon cancer patients treated with Irinotecan Treating primary hepatocellular carcinoma (liver cancer) Treating liver metastasis of colon cancer Treating liver metastasis of breast cancer Pre operational treatment of prostate cancer Many of these trials enable assessment of both supportive care and direct anti tumor activity of CBLB502 24

25 Major Scientific and Clinical Updates Demonstration of CBLB502 efficacy in preclinical model of local irradiation Approval of CBLB502 as supportive care trial in head and neck cancer patients by Scientific Review Committee of Roswell Park Discovery of direct anticancer action of CBLB502: from supportive care only drug to combined supportive care and anticancer drug Approval of Phase I/II CBLB502 as a single agent trial by Scientific Review Committee of Roswell Park Demonstration of radiomitigating efficacy of CBLB502 against GI manifestation of acute radiation syndrome in primates that received extremely high radiation doses Critical result for justifying extended indications of CBLB502 Building of Histological Atlas of CBLB502 activity, identification of target organs Path to optimal indications and regimens Completion of Phase Ib human dose validation trial 25

26 Curaxins Anticancer drugs 26 26

27 Curaxins Synthetic small molecules with proprietary structure Unique mechanism of action: simultaneously affect multiple molecular targets in cancer cell Efficacious in a broad spectrum of preclinical tumor models Mechanism of action enables additional clinical indications beyond cancer treatment (anti inflammatory, anti infective) First generation Curaxin CBLC102 is in clinical trial in patients with liver metastases New generation Curaxin CBLC137 is at advanced stage of preclinical development 27 27

28 Incuron JV for Curaxin Development 50/50 joint venture with Bioprocess Ventures, Moscow ~$18M to reach Phase II for new generation of Curaxins in US and Russia and conduct human trials in liver cancer in Russia for CBLC102 CBLI oversees mechanistic studies and formal development Phase Ib trial for CBLC102 in liver started October 2010 IND enabling studies for new generation of Curaxins on track for IND Demonstrates feasibility of model combining advantages of US and Russian development platforms 28 28

29 Milestones Start of pivotal animal efficacy studies for CBLB502 defense Start of definitive safety/dose validation trial in healthy volunteers for CBLB502 defense Start of CBLB502 Phase I/II trial in head and neck cancer patients for supportive care indication Start of CBLB502 Phase I/II trial in advanced liver metastases patients for safety/antitumor effect Completion of CBLC102 trial in liver cancer patients in Russia Filing of IND for studies of new generation curaxins Top level peer reviewed publications 29 29