Nastech Pharmaceutical Company Inc. Company Presentation

Transcription

1 Nastech Pharmaceutical Company Inc. Company Presentation 2005 Nastech

2 Forward-Looking Statements Statements made in this presentation may be forward-looking statements within the meaning of Federal Securities laws that are subject to certain risks and uncertainties and involve factors that may cause actual results to differ materially from those projected or suggested. Factors that could cause actual results to differ materially from those in forward-looking statements include, but are not limited to: (i) the ability of the Company to obtain additional funding; (ii) the ability of the Company to attract and/or maintain manufacturing, research, development and commercialization partners; (iii) the Company's and/or a partner s ability to successfully complete product research and development, including pre-clinical and clinical studies and commercialization; (iv) the Company s and/or a partner s ability to obtain required governmental approvals; and (v) the Company's and/or a partner s ability to develop and commercialize products that can compete favorably with those of competitors. Additional factors that could cause actual results to differ materially from those projected or suggested in any forwardlooking statements are contained in the Company's most recent periodic reports on Form 10-K and Form 10-Q that are filed with the Securities and Exchange Commission. The Company assumes no obligation to update and supplement forward-looking statements that become untrue because of subsequent events Nastech 2

3 Nastech Highlights Financially Driven Business Strategy Feasibility Studies / in-house skunk works drive business opportunities Proprietary tight junction technology drives patent-based royalties State of the Art manufacturing capability drives profitable manufacturing supply agreements Nine Product Programs Addressing Large Markets 4 clinical products 5 pre-clinical programs Six Partnerships Merck for PYY Par for Calcitonin Feasibility study in Obesity Feasibility study in Type 2 Diabetes Feasibility study in Alzheimer s s Disease Feasibility study in Anemia 2005 Nastech 3

4 Nastech Business Strategy: Feasibility Studies to Collaborations to Revenues Feasibility Studies Pre-Commercial Post-Commercial Partner Revenue Nastech Post-Commercial Revenue Milestone Revenue Nastech Revenue Nastech Expense Manufacturing Revenue Nastech Revenue Royalty Revenue Note: Graph not to scale Nastech 4

5 Product Pipeline STAGE PARTNER PRE-CLINICAL PHASE I PHASE II PHASE III NDA / ANDA Peptide YY 3-36 Obesity Parathyroid Hormone (PTH 1-34 ) Osteoporosis Calcitonin Osteoporosis Morphine Gluconate Breakthrough Cancer Pain RNAi Inflammatory Diseases, Infections, Metabolic Diseases and Cancer Feasibility Studies Type II Diabetes Obesity Alzheimer s disease Anemia Pharma & Biotech 2005 Nastech 5

6 Nastech s Tight Junction Technology Enables Non-Invasive Delivery of Peptides What are Tight Junctions? Gate-like structures between cells Regulate passage of pharmaceuticals across the cell barrier Many drugs need to efficiently pass through these tissues to reach their site of action Why is Nastech focused on this area? Broadly applicable for drug delivery Important for non-injectable delivery of large molecule drugs Validated technology lab to clinic 2005 Nastech 6

7 Peptide YY 3-36 for Obesity Natural hormone released during eating New mechanism for obesity treatment Central acting satiety agent Increases metabolism Slows gastric emptying Nastech s PYY 3-36 Nasal Spray Demonstrated ability to regulate appetite control and reduce caloric intake Proprietary tight junction technology enables non-injectable formulation for peptide Multiple clinical programs completed Significant commercial partnership with Merck & Co Nastech 7

8 Obesity Market Opportunity Why is obesity an important target market? DO THE MATH! 127 million (65%) adult Americans are overweight 61 million (31%) adult Americans are obese Current therapy is about $3.00 per day or $1,000 per patient per year 1988 Obesity Trends Among U.S. Adults 2003 (BMI 30, or ~ 30 lbs overweight for 5 4 person) No Data <10% 10%-14% 15-19% 20-24% 25% Source: CDC, BRFSS, EASO, IOTF 2005 Nastech 8

9 Partnership to Fuel Commercialization- PYY Alliance Exclusive development, commercial and patent licensing agreement Merck will assume primary responsibility for pre-clinical studies, clinical studies and regulatory approvals Nastech responsible for all CMC development, clinical and commercial manufacturing Separate supply agreement for the commercial manufacturing of PYY nasal spray Merck will lead and fund world-wide commercialization Nastech to co-promote in the U.S. "Nastech's innovative research and development capabilities are reflected in its rapid advancement of PYY," said Peter S. Kim, Ph.D., President of Merck Research Laboratories 2005 Nastech 9

10 Financial Highlights $346 million in fees and milestone payments $5 million upfront license fee $131 million development and approval milestones $210 million sales milestones Significant patent-based royalties on product sales Profitable manufacturing supply agreement both during product development and post-approval commercialization Reimbursement for all ongoing Nastech development costs Reimbursement for Nastech co-promotion sales force 2005 Nastech 10

11 PTH 1-34 (Teriparatide) Spray for Osteoporosis Patient preferred alternative for large market Non-invasive delivery alternative to currently marketed injectable PTH product for osteoporosis (Eli Lilly s Forteo ) with sales of $239 million in 2004 and growing Limited development risk as efficacy and safety well-known Injectable PTH is the only FDA approved product resulting in bone growth PTH has shown unsurpassed increases in bone mineral density and reduction of fractures in postmenopausal women Significant patent estate developed with intranasal PTH 2005 Nastech 11

12 Rapid PTH 1-34 Clinical Development Program FDA has agreed that 505(b)2 NDA pathway is acceptable for approval Three part program: Pharmacokinetic studies in two populations, normal and elderly Single animal study focused on nasal irritation No lengthy systemic toxicology, reproductive toxicology, and carcinogenicity studies are required Single Phase 3 study of bone mineral density (6 months) No fracture studies are required Fracture studies can take 1000 s of patients and 2-3 years to complete Registration study planned to start in Nastech 12

13 Calcitonin Alliance Milestones Revenue Manufacturing Revenue Profit Split Distribution Fees Nastech Par Nastech Par Generic of Novartis Miacalcin with $260 M US sales U.S. distribution and marketing rights to Par Nastech responsible for regulatory approval, manufacture and supply Upfront, regulatory and post-approval milestone payments Transfer payments for final manufactured product Profit sharing following commercial launch Near-term Nastech product revenue opportunity ANDA accepted 1Q 2004 and launch anticipated on approval 2005 Nastech 13

14 Intranasal Spray for Type II Diabetes Feasibility collaboration is progressing on schedule Diabetes affects more than 18 million in the United States Approximately 90 to 95 percent of those affected have type 2 diabetes a condition where the body does not produce enough insulin and/or the cells in the body do not respond normally to insulin Diabetes is the fifth leading cause of death by disease in the United States Diabetes medical costs are approximately $132 billion per year 2005 Nastech 14

15 Intranasal Spray for Alzheimer s Disease Feasibility collaboration progressing on schedule Intranasal formulations have the potential to deliver higher levels of medication and minimize the side effects associated with oral delivery 4.5 million Americans have Alzheimer s disease direct and indirect costs exceed $60 billion annually Currently, there is no adequate treatment 2005 Nastech 15

16 Intranasal Spray for Obesity Today Feasibility collaboration progressing on schedule This is a therapeutic target distinct from the Y2 receptor and PYY A proprietary molecule which has demonstrated reduced food intake and weight loss in acute and sub-acute animal studies, including established models of diabetes and obesity 2005 Nastech 16

17 Intranasal Spray for Erythropoietin (EPO) Receptor Agonist collaboration Today Feasibility collaboration progressing on schedule Intranasal formulation of Erythropoietin (EPO) receptor agonist directed towards the treatment of severe anemia Used to treat anemia in patients with: chronic renal failure that are on dialysis anemia caused by certain medications, including some types of chemotherapy and zidovudine for treatment of HIV Used before and after some types of surgery to prevent blood loss Current injectable EPO market includes products such as Amgen s Epogen and Aranesp and Johnson & Johnson s Procrit and are estimated to have sales in excess of $10 billion 2005 Nastech 17

18 RNAi Therapeutics: A Solution to the Delivery Challenge RNAi holds immense promise, but delivery of sirna is the primary hurdle: Passing through cell membrane barrier Maintaining molecule stability Delivery into cells in effective quantities Nastech s novel carrier molecules enable intracellular delivery Nastech is the first to demonstrate successful in vivo systemic delivery of an RNAi therapeutic, and clinical effect, in rheumatoid arthritis 2005 Nastech 18

19 RNAi for Rheumatoid Arthritis h-tnf-α is over expressed in rheumatoid arthritis (RA) and plays key role in disease pathology RA is a multi-billion dollar product market including Amgen s Enbrel, J&J s Remicade and Abbott s Humira Current therapies work by binding to or stabilizing h-tnf-α or its receptor May results in increase circulating TNF-α concentrations and up regulate TNF-α production potentially leading to rebound issues Nastech s RNAi is different in that it is designed to reduce or eliminate TNF-a which may provide a more effective treatment option TNF-a (pg/ml) TNF-a Concentrations: infliximab vs. NSTK RNAi Day Post Treatment Date (Days) infliximab NSTK sirna 2005 Nastech 19

20 RNAi Inflammation Reduction Comparable to Infliximab (Remicade ) Transgenic mice with human TNF-α gene overexpress TNF-α and have clinical signs of joint disease and early mortality Three treatment groups: saline infliximab Nastech s formulation Nastech s formulation performed significantly better than saline and slightly better than infliximab Publication available at Paw Inflamm ation Score ( baseline adju sted) Age (week) saline infliximab NSTK sirna 2005 Nastech 20

21 Validated Nasal Spray Manufacturing Facilities State-of-the-art testing and manufacturing equipment FDA Inspected and Compliant Passed due diligence inspections by Merck, Par and other significant partners Clinical & Commercial-scale supply Capacity of 60+ million units per year Large scale QC testing capacity including spray characterization Generated CMC sections from both NDA s and ANDA s Two geographically separated facilities Bothell, WA 2005 Nastech 21

22 Financials Q ($ in millions) Total Revenues $3.3 Total Expenses ($9.4) Net Loss ($6.1) Q1 shares outstanding 17.8 million Cash $ Nastech 22

23 5 Year Historical Market Cap Growth: <$50 MM to >$260 MM $300.0 $250.0 $200.0 $150.0 $100.0 $50.0 $- Sep-00 Dec-00 Mar-01 Jun-01 Sep-01 Dec-01 Mar-02 Jun-02 Sep-02 Dec-02 Mar-03 Jun-03 Sep-03 Dec-03 Mar-04 Jun-04 Sep-04 Dec-04 Mar-05 Jun Nastech 23

24 Upcoming Milestones & Events Potential for significant near-term cash flows: Merck PYY development: funded development, clinical mfg. and development milestone revenues Par Calcitonin approval: milestones, profit-split and mfg. revenues Feasibility partnerships: R&D service revenue Advancement of PTH clinical program RNAi research program progress Analysts George Fulop & Mark Monane, Needham Robert Hazlett III, SunTrust Robinson Humphrey Quynh Pham, Delafield Hambrecht 2005 Nastech 24

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