Immunity for Life TM. Sven Rohmann VP Business Development

Transcription

1 Immunity for Life TM Sven Rohmann VP Business Development

2 Disclaimer This Presentation includes and is based, inter alia, on forward-looking information and statements that are subject to risks and uncertainties that could cause actual results to differ. These statements and this Presentation are based on current expectations, estimates and projections, which generally are identifiable by statements containing words such as expects, believes, estimates or similar expressions. Important factors that could cause actual results to differ materially from those expectations include, among others, general economic and industry conditions in markets which are expected to be major markets for Biotec Pharmacon ASA s products, as well as risks and uncertainties related to product development, regulatory approvals, commercial partnerships, the outcome of intellectual property rights litigation and the competitive situation. Although Biotec Pharmacon ASA believes that its expectations and the Presentation are based upon reasonable assumptions, it can give no assurance that those expectations will be achieved or that the actual results will be as set out in the Presentation. Biotec Pharmacon ASA is making no representation or warranty, expressed or implied, as to the accuracy, reliability or completeness of the Presentation, and neither Biotec Pharmacon ASA nor any of its directors, officers or employees will have any liability to you or any other persons resulting from your use of the information contained herein. This presentation was prepared for BioPharm America 2009 in San Francisco on September September, 2009, and the information contained within will not be updated in this presentation. The following slides should be read and considered in connection with other information provided by the company. No shares of Biotec Pharmacon are being offered in connection with this presentation and no such shares have been registered under the U.S. Securities Act of 1933, as amended (the "Act"), and such shares may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Act. 2

3 Pharmaceutical concept and drug development pipeline

4 SBG - Clinical development portfolio Diabetic ulcers Oral mucositis Preclinical Phase I Phase II Phase III NDA Immunotherapy of cancer IBD Addressing unresolved medical problems in major disease areas No well established therapies, few new product candidates in development Commercial aspects of SBG: Innovative products - attractive pricing, Underdeveloped market - high growth potential, Hospital products - easier market access 4

5 Pharmaceutical concept SBG stimulates the immune system SBG (Soluble Beta Glucan) a unique, highly bioactive, soluble beta-1,3/1,6-glucan from cell walls of yeast alien to the human body How it works: stimulates the innate immune system enhances the efficacy of the adaptive immune system addresses a range of diseases 5

6 Mechanisms of action SBG binds to receptors on macrophages CR3 (CD11b/CD18)-receptor SBG Dectin-1 Human b-glucan receptor A B C Toll like receptor 2/6 6

7 TNF-a (pg/ml) In-vitro results SBG stimulates cytokine production in macrophages SBG in a mice study shows strong effect on TNF-alpha A close to linear increase in cytokine production (TNF-alpha) in peritoneal macrophages with increasing dose of SBG SBG Placebo Concentration of stimulant (mg/ml) Zykova

8 Proof of concept Ulcers and wounds SBG treatment of ulcers in diabetic mice Statistically significant difference in favor of SBG vs. non-treated control group at day 15 Methodology; Wounds established on the back of diabetic C57NI/KSdb/db mice under general anesthesia Wound treatment with topical application of SBG in active group 60% 50% 40% 30% 20% 10% 0% Wound-size reduction (%) 18% Non-treated control 47% SBG 8

9 Toxicology/safety in animals SBG in in-vitro and in-vivo animal studies Very favourable toxicity No potential clinical hazard identified for proposed human use, for either topical or oral administration No potential found for mutagenicity or genotoxicity Minimal lethal dose could not be found in toxicity studies in rodents or non-rodents NOEL dose of 200 mg/kg/day after oral administration for 28 days in rats, 300mg/kg/day in dogs Demonstrated good local tolerance after topical application 9

10 Proof of Concept Clinical phase II SBG in treatment of diabetic foot ulcers SBG Control 10

11 2 Pivotal Trials in Europe - Phase III Diabetic foot ulcers Clinical phase Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Phase III, Nottingham, UK Phase III, Europe/Eastern Europe Blue area = periods of patient inclusion, black areas = periods of study completion and reporting Patient populations First study; n=120, second study; n=130 Patient enrolment completed No changes made after interim analyses Primary endpoint Proportion of patients with target ulcers that heal within 8 weeks Secondary endpoints Proportion that heals within 12 weeks Time to healing of target ulcers % change in target ulcer area Recurrence within 12 weeks after healing First study Second study 11

12 Diabetic foot ulcers Milestone schedule YTD Completed patient inclusion in both studies Q Completion of treatment and follow-up programs Data collection and analysis Q Final results expected to be ready from both studies July 2010 Filing for market approval in July

13 Proof of Concept clinical phase II SBG for prevention and treatment of oral mucositis Patients developing severe Oral Mucositis (%) Placebo SBG Source: Sook Bin Woo, emedicine, Chemotherapy-induced Oral Mucositis Duration of therapy (days) n=36 13

14 Pivotal Trial in Europe - Phase III Oral Mucositis Clinical phase Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Phase III, Europe Phase III, Eastern Europe 2 nd study put on hold awaiting results from the 1 st study Blue area = periods of patient inclusion, black areas = periods of study completion and reporting Patient population Number of patients; n= patients enrolled No changes made after sample size reassessment Primary endpoint Incidence of OM CTCgrade 3/4 at any time of commencement of radiotherapy up to 5 days after radiotherapy ceases. Secondary endpoints Time from commencement of Radiotherapy to onset of OM CTC grad 3/4 ; Duration of OM CTC grade 3/4 Overall incidence of OM CTC grade 2, 3 and 4 14 Eligible for orphan drug designation in Europe.

15 Proof of concept Cancer SBG and monoclonal antibodies in cancer therapy Significant (p<0.05) effect of mab+sbg versus mab alone Methodology: Inoculation of mice with human neuroblastoma cancer cells, leading to development of tumors Treatment with the mab 3F8 in active and control group SBG in addition to mab (3F8) in the active group Primary end point: Tumor size (% increase) mab alone mab + SBG 15

16 Proof of Concept clinical phase Ib/IIa SBG + mab for cancer therapy Memorial Sloan-Kettering Cancer Center open label study with SBG+3F8* for neuroblastoma in children Well tolerated even at very high dosage levels Promising efficacy data; objective response in ~40% of the patients Full results awaited in first half 2009 Marked decrease in neuroblastoma disease burden Before After 123I-MIBG scan of patient treated with one cycle of 3F8+SBG (80mg/kg/day) * 3F8 is a monoclonal antibody (mab) 16

17 Safety Clinical Phase I/II Clinical phase Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Ullevål: SBG+Herceptin for breast cancer Rikshospitalet: SBG+Rituximab for non-hodgkin s lymphoma Blue area = periods of patient inclusion, black areas = periods of study completion and reporting Patient populations Study 1; n=10, Study 2; n=12 Patient enrolment completed in both studies Primary endpoint Study 1: Assess the safety of oral SBG in combination with Herceptin given to breast cancer patients undergoing standard chemo- and antibody treatment. Study 2: Assess the safety of oral SBG in combination with MabThera (Rituximab) given to non-hodgkin s lymphoma patients undergoing standard chemo- and antibody treatment. Secondary endpoints Both studies: Assess the anti-tumor response of the combination treatment 17

18 Proof of concept IBD SBG in the treatment of ulcerative colitis SBG protects against DSS-induced colitis, and has effect on epithelial proliferation and intestinal restitution Oral SBG treatment stimulates expansion of Peyer s patches and mesenteric lymph nodes (not shown) *P<0.05, **P<0.01, ***P<0.001, 2-way ANOVA Sandvik et al submitted

19 Partnering opportunities and Summary

20 SBG Partnering Opportunities Diabetic ulcers Oral mucositis Preclinical Phase I Phase II Phase III NDA Immunotherapy of cancer IBD Open for partnering discussions with both global and regional partners Open for partnering opportunities for SBG for all disease indications 20

21 Summary SBG works Established Proof of Concept in four indications; Diabetic foot ulcer, oral mucositis, immunotherapy of cancer, and IBD Addressing serious unresolved medical problems in major market segments Well advanced clinical program Filing for marketing authorisation in Europe in July 2010 for diabetic foot ulcer Initiated partnering discussions Open to commercial partnerships for all indications in all markets 21

22 Contact: Sven Rohmann, MD, PhD Mobile: Biotec Pharmacon ASA, Strandgata 3 N-9008 Tromso, Norway, or Biotec Pharmacon ASA, Drammensveien 149 N-0277 Oslo, Norway 22