INNOVATIVE VACCINES FOR CANCER AND INFECTIOUS DISEASES

Transcription

1 INNOVATIVE VACCINES FOR CANCER AND INFECTIOUS DISEASES June

2 Vaccines for cancer and infectious diseases Founded in 1994 Two Phase 3 programs Strong IP position Listed on NASDAQ OMX Market cap: USD 315m 450+ employees in Denmark, Germany and USA 2

3 Our business Cancer Immunotherapy Infectious Diseases Pipeline in major cancers Lead product candidate in Phase 3 Encouraging survival results in Phase 2 Broadly applicable technology platform Pipeline in biodefense and commercial vaccines Lead product candidate in Phase 3 positive opinion received from EMA Long-standing partnership with the USG Profitable division with ongoing vaccine deliveries to the USG 3

4 Growing A Multi-Product Biotech Company Financial Strategy: Obtain profitability Growth Strategy: Move pipeline forward in both divisions Multiple products in cancer and infectious diseases Strategic partnerships Cancer Immunotherapy Infectious Diseases Objective: Approval of PROSTVAC (FDA/EMA/ROW) Broaden PROSTVAC indications Expanded product pipeline Objective: Maintain profitability Continue and expand biodefense portfolio Develop strong innovative vaccine portfolio with commercial potential 4

5 Recent Highlights Cancer Immunotherapy PROSPECT study: 120 sites in 11 countries now active Interim analysis plan for the Phase 3 PROSPECT trial agreed with the FDA New exciting data on CV-301 in colorectal cancer Infectious Diseases New $228m IMVAMUNE delivery contract with USG Positive opinion for IMVANEX (IMVAMUNE ) received from EMA The first of two Phase 3 studies of IMVAMUNE was initiated in the U.S. Consolidation of manufacturing activities has begun at the facility in Kvistgaard, Denmark 5

6 Cancer Immunotherapy CANCER IMMUNOTHERAPY Prostate cancer PROSTVAC Preclinical Phase 1 Phase 1/2 Phase 2 Phase 3 Market Colorectal cancer Breast cancer Lung cancer Ovarian cancer Prostate cancer Breast cancer CV-301 colon CV-301 breast CV-301 lung CV-301 ovarian MVA-BN PRO MVA-BN HER2 6

7 Prostate Cancer A Large Unmet Medical Need Common cause of death in men More than 900,000 cases/year 1 More than 250,000 deaths/year 1 Metastatic disease is incurable 1) Global Cancer Facts & Figures, 2 nd Edition, American Cancer Society 7

8 PROSTVAC - Asset with Solid Data Journal of Clinical Oncology March 1, 2010 vol. 28 no PROSTVAC has more clinical data from combination trials and trials in earlier disease stages than other prostate cancer immunotherapies 22 ongoing or completed clinical trials Completed (paper or abstract available) Ongoing (ongoing or planned) Phase Phase Overall Survival Analysis of a Phase II Randomized Controlled Trial of a Poxviral-Based PSA-Targeted Immunotherapy in Metastatic Castration- Resistant Prostate Cancer Phase 3-1 Total 17 5 Total patients ,400+ 8

9 PROSTVAC Off-the-shelf vial vaccine Sequentially dosed combination of two different Poxviruses Targets a unique cancer cell antigen (PSA) and encodes costimulatory molecules Subcutaneous injection Vaccinia-PSA TRICOM Fowlpox-PSA-TRICOM Multicenter Phase 2 1 Randomized, placebo-controlled Double-blind 125 mcrpc patients enrolled in 43 sites 83 received PROSTVAC + GM-CSF 41 received placebo Inclusion criteria: Bone scan+ or CT scan+ Rising PSA despite androgen suppression Low level testosterone Castration-resistant (CRPC) Gleason 7 or less Exclusion criteria: Metastasis to other sites Liver, lung, brain Narcotics for pain Prior chemotherapy 9 1) Kantoff et al., Journal of Clinical Oncology, January 2010

10 PROSTVAC Phase 2 Results survival (% of patients) months 25.1 months Significantly extended overall survival Δ 8.5 months N Deaths Median Control PROSTVAC Hazard ratio 0.56 (95% CI ) p= months Source: Kantoff et al., Journal of Clinical Oncology, January

11 PROSPECT A Randomized, Double-blind, Global Phase 3 Efficacy Trial of PROSTVAC in Metastatic Castration-Resistant Prostate Cancer 11 countries active, 120 sites US, Canada, Spain, France, UK, Iceland, Estonia, Belgium, Denmark, Israel & Russia as of May, 2013 Full enrollment anticipated in H1, study arms 1,200 patients PROSTVAC + GM-CSF PROSTVAC Interim Analysis agreed with FDA Pre-specified interim analyses of data that will be performed to evaluate whether the trial should continue as planned or potentially be stopped early for efficacy Placebo Primary endpoint is overall survival Either one or both of the treatment arms must be superior to placebo Each comparison requires 534 deaths for the final analysis Phase 2 results: Demonstrated hazard ratio 0.56 = 44% reduction in risk of death 11 SPA terms for Phase 3: Required hazard ratio 0.82 or less = 18% reduction in risk of death

12 Broadly Applicable Technology Platform PROSTVAC Prostate cancer CV-301 Breast, Lung, Colorectal, Ovarian, Gastric, Bladder, Liver Esophageal and Renal cancer PSA CEA MUC-1 TRICOM TRIad of CO-stimulatory Molecules LFA-3 ICAM-1 B7.1 VF Prime-boost: Vaccinia + Fowlpox GM-CSF can be used as adjuvant in both PROSTVAC and CV

13 CV-301 colorectal cancer NCI-sponsored Ph2 study at Duke University in metastatic colorectal cancer (n=74) Surgical resection of metastatic colorectal cancer followed by chemotherapy and CV-301 (± dendritic cells) RANDOMIZE Arm A: CV-301 s.c. + GM-CSF Arm B: CV-301 infected dendritic cells 13

14 Survival probability CV-301 colorectal cancer study results 161 concurrent, matched control patients OS superior (p < ) PFS not different Duke controls CV-301 Months from resection Morse MA et al, Ann Surg 2013

15 Cancer Immunotherapy Short/Mid-Term Objectives Complete enrolment in the PROSTVAC Phase 3 trial (PROSPECT) Initiate Phase 2 combination trials of PROSTVAC and enzalutamide Report data from NCI-sponsored clinical trials of PROSTVAC Determine future development strategy for CV-301 Prepare the Kvistgaard facility for commercial manufacturing of PROSTVAC 15

16 Infectious Diseases BIODEFENSE Smallpox IMVAMUNE Preclinical Phase 1 Phase 1/2 Phase 2 Phase 3 Market Smallpox Anthrax Filoviruses Foot-and-mouth disease COMMERCIAL RSV IMVAMUNE freeze-dried MVA-BN Anthrax MVA-BN Filo MVA-BN FMDV MVA-BN RSV Sold to government stockpiles under national emergency rules. Positive opinion received from EMA 16

17 IMVAMUNE Advantages Traditional smallpox vaccines Currently stockpiled vaccines are based on a replicating vaccinia virus: DryVax, ACAM2000, LC16m8, Elstree-BN All have been shown to produce some/all of following side effects: Encephalitis, generalized vaccinia, inadvertent infection skin and eyes/self and contacts, myo-pericarditis 25% of the population cannot receive current smallpox vaccines Young, elderly, people with a history of atopic disorders (e.g. eczema), HIV or AIDS, cancer patients IMVAMUNE Based on a non-replicating virus More than 4,300 individuals have been vaccinated Well tolerated even in immune-compromised No product-related severe adverse events reported 17 *Pre-clinical data Induces faster protective immune response Protects already after 3-4 days vs days with the traditional vaccines*

18 U.S. Government Contracts Year Contracts with the U.S. Government Value Early clinical and technical development of IMVAMUNE 500,000 doses of IMVAMUNE delivered Clinical studies to support Emergency Use million doses of IMVAMUNE Licensing for at-risk individuals Development for immune compromised 2009 Development of freeze-dried version of IMVAMUNE Validation of production process Preclinical and clinical studies to support advanced development 2012 Marburg vaccine Advanced development of an MVA-BN -based vaccine against Marburg virus 2012 Foot-and-mouth disease Development of a veterinary vaccine against foot-and-mouth disease virus million doses of IMVAMUNE Deliveries through 2014 USD 130m USD 549m USD 95m USD 18m USD 1m USD 228m Total value of contracts awarded to-date USD 1,021m 18

19 IMVAMUNE - Anticipated Developments and on Deliveries, base Deliveries, new contract Maintenance orders Freeze-dried Phase 2 trial of freeze-dried version to support emergency use/stockpiling Phase 3 lot consistency trial Phase 3 non-inferiority trial BLA Decision * Market opportunity Decision Market opportunity * Positive opinion received from EMA 19

20 Infectious Diseases Short/Mid-Term Objectives Deliver 7 million doses of IMVAMUNE to the U.S. Strategic National Stockpile in 2013 Ensure sustainable and growing profitability in division Complete enrolment in the IMVAMUNE Phase 3 lot consistency trial Initiate Phase 3 non-inferiority trial of IMVAMUNE Final marketing authorization for IMVANEX (IMVAMUNE ) in the EU Potential marketing authorization for IMVAMUNE in Canada 20

21 Financial Outlook Revenue Income before tax Cash preparedness at year-end 2013 US$ 193m US$ 0m US$ 105m Assumptions: Deliver and revenue recognize 7 million doses of IMVAMUNE to the U.S. Strategic National Stockpile R&D costs - GROUP Infectious Disease Division, EBIT * US$ 81m US$ 63m Cancer Vaccines Division, EBIT US$ -57m All numbers are approximate * R&D costs include additional approximately DKK 110 million in contract expenses (stated under production costs in the profit and loss statement). USD/DKK =

22 This presentation includes "forward-looking statements" that involve risks, uncertainties and other factors, many of which are outside of our control, that could cause actual results to differ materially from the results discussed in the forward-looking statements. Forward-looking statements include statements concerning our plans, objectives, goals, future events, performance and/or other information that is not historical information. We undertake 22 no obligation to publicly update or revise forward-looking statements to reflect subsequent events or circumstances after the date made, except as required by law.