BioCancell Therapeutics

Transcription

1 BioCancell Therapeutics An Innovative Cancer Treatment Approach June, 2009 Avi Barak, CEO BioCancell Therapeutics, Inc.

2 This presentation contains "forward-looking" statements, including statements with respect to the further development and potential safety and efficacy of BioCancell s products and development strategy. Any statements contained in this presentation that are not statements of historical fact may be deemed to be forward-looking statements. There are a number of important factors that could cause the results of BioCancell to differ materially from those indicated by these forward-looking statements, including, among others, the risk that the U.S. Food and Drug Administration may require changes to the protocols and informed consents for clinical trials of BC-819, which changes may have a material adverse effect on the timing of, and BioCancell's ability to conduct, those clinical trials, risks related to the clinical advancement of its BC-819 plasmid, including, but not limited, to the risk that clinical trials for this product candidate may not demonstrate safety and efficacy sufficient to obtain the requisite regulatory approvals or to result in a marketable product and risks related to the potential for others to develop products containing or based on BC-819. BioCancell does not undertake any obligation to update forward-looking statements.

3 Overview Incorporated in Delaware, July 2004 Fundraising of $14 million, including IPO on TASE (TASE:BICL) CSO ($1.5M) and BIRDF ($0.95M) grants S1 prospectus was filed to SEC

4 Overview H19 Gene as a treatment platform for Targeted treatment: H19 is the big differentiator - expressed in the embryo, repressed post-natally and only re-expressed within cancer cells. A common denominator in 40 solid tumors. Win-Win Technology NO SIDE EFFECTS, superior efficacy Targeted indications to date- bladder, ovary, pancreas, liver Proven Clinical Data: Lead product BC-819 has successfully completed a Phase I/IIa clinical trial for treatment of superficial bladder cancer, and has commenced Phase IIb, as well as a Phase I/IIa trial of ovarian cancer.

5 Pipeline Bladder cancer: Phase I/IIa clinical trial completed (Q3/07) Phase II IND clinical trial commenced (Q1/08) Pancreatic cancer Preclinical development - concluded Phase I/IIa clinical trial commencing (Q2/09) Ovarian cancer: Phase I/IIa clinical trial commenced (Q2/09) Metastatic liver cancer: Phase I/IIa clinical trial IND submission (Q3/09)

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8 The Drug - BC-819 Plasmid

9 Targeted Therapy - H19 gene drives the synthesis of Diphtheria toxin only inside cancer cells

10 The Advantages of BC-819 Strong safety profile No side effects Targeting - yields superior success rate Low cost manufacturing Expected faster regulatory pathway (Fast Track) Same drug for 40 different cancer types Reduces Multi-Drug Resistance (MDR)

11 The Use of BC-819 (H19-DTA) in Human Cancer Indications Sought Bladder Cancer Ovarian Cancer Pancreatic Cancer Metastatic Liver Cancer Breast Cancer Hepatocellular Cancer Brain Cancer Head & Neck Cancer 30 additional cancer indications

12 Frequency of H19 Expression in Cancer Indication New Cases* Deaths* Expression (%) Bladder** 70,980 14, % Ovary 21,550 14, % Pancreas 42,470 35,240 70% Breast*** 194,280 40, % Colorectal*** 146,970 49,920 70% Hepatocellular 22,620 18,160 75% Total 498, ,860 * Data: American Cancer Society, estimated ** Bladder cancer prevalence in the U.S. is estimated at 600,000 *** Major source of metastatic liver cancer - average H19 expression 70%. Data refers to U.S. only and represents 40% of cases in developed countries

13 Therapeutic Use of BC-819

14 Bladder Cancer

15 Clinical Study Phase I/IIa using BC-819 for Treatment of Bladder Cancer

16 Phase I/IIa - Analysis No. of Patients Percentage (%) Responders Non-responders Non-recurrence Recurrence Non-progressive Progressive LM Response LM Non-response No Serious Adverse Events (SAEs) related to the plasmid treatment have been detected

17 Overview of Phase IIb Bladder Cancer using BC-819 Phase IIb multi-center, two-stage-design clinical study to assess the efficacy and safety of BC-819 in patients with intermediate-risk superficial bladder carcinoma. The study is being performed at seven medical centers in Israel and one in the U.S.A. Inclusion criteria - patients with TCC (recurrent stages Ta, T1, low grade) that is positive for H19, who have failed treatment with BCG or chemotherapy. Eligible patients will receive a six-week induction course of onceweekly intravesical administrations of 20 mg BC-819. Patients with a CR will continue to be treated with three maintenance courses of the plasmid. 33 patients will participate: At least 5 of first 18 evaluable patients must have a CR to meet the criteria to continue accrual of 15 additional evaluable patients.

18 Ovarian Cancer

19 BC-819 Compassionate Use Treatment for Ovarian Cancer - Background A 69 year-old female patient was first diagnosed as suffering from spread epithelial ovarian cancer in The patient underwent ovarectomy, hysterectomy, omentectomy and colon sigmoidectomy to remove most of the tumor. The patient received 6 courses of combined chemotherapy with carboplatin and palitexal. In December 2004, a recurrence of the disease was diagnosed. Combined chemotherapy of carboplatin and palitexal was administered. In January 2007, disease recurrence was observed by PET/CT analysis. The patient was treated by systemic chemotherapy with gemcitabine, doxil and lastly with VP16.

20 BC-819 Compassionate Use Treatment for Ovarian Cancer - Description First Trial The patient was given 10 courses of BC-819 (intraperitoneally, for the first time) in doses ranging from 80mg to 120mg. No SAEs were reported, tumor growth was arrested, the levels of cancer marker CA-125 dropped by approx. 50%, the number of cancerous cells in the patient s ascitic fluid dropped, and the ascitic fluid level itself was reduced to nothing. The patient and her doctors reported significant clinical improvement. Second Trial The patient was given a combination of BC-819 and Taxotere (a derivative of chematherapeutic drug Taxol). Results showed an additional drop in the levels of CA-125.

21 Ultrasound Scans Pre- and Post-Treatment before treatments After treatment #8

22 Overview of Phase I/IIa Clinical Trial using BC-819 for treating Ovarian Cancer Phase 1/2a, Dose-Escalation, Safety, Pharmacokinetic, and Preliminary Efficacy Study of Intraperitoneal Administration of BC-819 in Patients with Ovarian Cancer. The study is being conducted in four medical centers, the NCIdesignated Massey Cancer Center and the University of Pennsylvania Medical Center in the U.S.A. and Hadassah and Wolfson Medical Centers in Israel The participation of 12 patients with ovarian cancer will be required. The primary objective is to determine the MTD of intraperitoneal BC- 819 and identify DLTs. The secondary objectives include determining the adverse events (AEs) profile, effects on clinical laboratory analytes, effects on cancer markers, vital signs, pharmacokinetics (PK), quality of life and clinical improvement.

23 Pancreatic Cancer

24 Summary A group of hamsters with pancreatic cancer (orthotopic model) was divided into two, with half receiving two courses of BC-819 intratumorally, and half serving as a control group. The tumor growth progression (TGP) was significantly reduced in the BC-819 treated group as compared with the control group. Primary pancreatic tumor ex vivo volume in hamsters treated with BC-819 was significantly reduced (50%) compared to the control group. 75% of the animals in the control group presented metastases, while NO metastases were found in the group treated with the BC-819 vector.

25 Overview of Planned Phase I/IIa using BC-819 for treating Pancreatic Cancer Phase 1/2a, Dose-Escalation, Safety, Pharmacokinetic, and Preliminary Efficacy Study of Intratumoral Administration of BC-819 in Patients with Unresectable Pancreatic Cancer. The study will be conducted in at least four medical centers, the NCIdesignated University of Maryland Medical Center and Massey Cancer Center in the U.S.A. and Hadassah and Sheba Medical Centers in Israel The participation of 9 patients with unresectable pancreatic cancer will be required. The primary objective is to determine the MTD of intratumoral BC-819 and identify DLTs. The secondary objectives include determining the adverse events (AEs) profile, effects on clinical laboratory analytes, vital signs, pharmacokinetics (PK), quality of life and tumor response.

26 Kidney (TCC) Cancer

27 Human Case Study 78 year old who underwent nephrectomy in 2000 due to high grade TCC of the right kidney. TCC recurrence in left ureter in 2004, and in left kidney in Tumor recurred despite conventional therapies. With no other solution, patient was candidate for second nephrectomy and permanent dialysis. The patient received 6 weekly courses of BC-819 Following end of treatment, patient s kidney was found to be tumor-free and continues to maintain a normal lifestyle. The treating physicians highly recommended this treatment for similar cases

28 Intellectual Property A patent for Methods and compositions for inducing tumor-specific cytotoxicity was submitted on October Granted: USA, Europe, China, Korea, Russia, Singapore, the Czech Republic, Canada, Israel, Mexico and Australia. Pending: Japan, India, Brazil, Hungary and Norway. Total: 8 patent families

29 * Indications with Fast-Track Potential Development Timeline Ovarian* Pancreas* Bladder IND Submission Phase I/IIa Phase IIb FDA Meeting Phase III NDA Submission & Review US Launch Compassionate Patient IND Submission Phase I/IIa Phase IIb FDA Meeting Phase III NDA Submission & review US Launch Phase IIb FDA Meeting Phase III NDA Submission & Review US Launch

30 What's next? In 2010, BioCancell plans to conduct: Phase III clinical trial - for bladder cancer Phase II clinical trial - for ovarian cancer Phase II clinical trial - for pancreatic cancer Expanding additional pipeline indications (Brain, Lung and Head & Neck cancers)

31 What's next? OTC/ NASDAQ listing Fundraising of $10-15M Establishing US presence International alliances

32 Consultants & Contractors Regulatory Consultant and CRA: Fast-Track Drugs & Biologics LLC, MD, USA Clinical PIs: George Coukos, M.D., Ph.D., University of Pennsylvania, PA, USA Cecilia H. Boardman, M.D., Massey Cancer Center, VA, USA Nader N. Hanna, M.D., Maryland Medical Center, MD, USA John D. Roberts, M.D., Massey Cancer Center, VA, USA Donald Lamm, M.D., BCG Oncology PC, AZ, USA Plasmid Manufacturing: Althea Technologies Inc., San Diego, CA, USA PEI Manufacturing: ChemCon GmbH, Germany The Sage Group (Wayne Pambianchi)

33 SAB Members Yechezkel Barenholz, Ph.D., Hebrew University of Jerusalem, Inventor of DOXIL Aaron Ciechanover, M.D., Ph.D., Nobel Prize Laureate, 2004 Roger D. Kornberg, Ph.D., Nobel Prize Laureate, 2006 Yaakov Naparstek, M.D., Chairman of Medicine at Hadassah University Hospital Hermona Soreq, Ph.D., Dean of the Faculty of Science at the Hebrew University of Jerusalem, Israel Mark L. Tykocinski, M.D., Dean of Jefferson Medical College and Senior Vice President of Thomas Jefferson University

34 Blockbuster Potential The Opportunity Clear Regulatory Path Active INDs for multiple indications Potential Fast Track (orphan drug) with FDA H19 - A well characterized target Precise toxin synthesis - only in cancer cells Outstanding therapeutic index - effectiveness with no side effects to date Strong IP position - world wide filing