Safe Harbor Statement

Transcription

1 Active Biotech AB

2 Safe Harbor Statement Certain statements made during the course of this presentation are forward-looking statements. Such forward-looking statements involve known and unknown risks, uncertainties and other important factors that could cause the actual results, performance or achievements of the company, or industry results, to differ materially from any future results, performance or achievement implied by such forward-looking statements. Statements made during the course of this presentation that are forward-looking are based on the company s current beliefs regarding a large number of factors affecting its business. There can be no assurance that (i) the company has correctly measured or identified all of the factors affecting its business or the extent of their likely impact, (ii) the available information with respect to these factors on which the Company s analysis is based is complete or accurate, (iii) the company s analysis is correct or (iv) the Company s strategy, which is based in part on this analysis, will be successful. All forward-looking statements speak only as of the date of this presentation or, in the case of any document incorporated by reference, the date of that document. All subsequent written and oral forward-looking statements attributable to the company or any person acting on the company's behalf are qualified by this cautionary statement. The company does not undertake any obligation to update or publicly release any revisions to forward-looking statements to reflect events, circumstances or changes in expectations after the date of this presentation. 2

3 Company overview Swedish Biotechnology Company Core competence in Autoimmune/Inflammatory diseases and Cancer Spin out from Pharmacia 1998 Listed company NASDAQ OMX Nordic: ACTI Share price SEK (5.85 USD), market cap SEK 2.9 B (438 MUSD) as of February 24, 2014 A total of 59 employees 3

4 Active Biotech Pipeline 4

5 MS pathology and disease progression Relapsing-Remitting Secondary Progression Clinical Disability Clinical Threshold Brain Volume Inflammation Axonal Loss Frequent inflammation, demyelination, axonal transection, plasticity, and remyelination Continuing inflammation, persistent demyelination Infrequent inflammation, chronic axonal degeneration, gliosis Ref: Compston A, Coles A. Lancet. 2002;359:

6 Current Multiple Sclerosis Treatment Landscape Drugs on the market and in Phase III development Immunosuppressant Injectable Lemtrada Daclizumab (PIII) Tysabri Oral Gilenya Aubagio (Teriflunomide) Immunomodulator Copaxone Interferons Plegridy (PEG-interferon) Nerventra (laquinimod, PIII) Tecfidera (BG-12) PIII: In Phase III development 6

7 Laquinimod for the treatment of MS Oral, once-daily CNS-active disease-modifying treatment for MS Delaying disability progression (34.2%*), reducing flares (21.4%*) and brain atrophy (30%*) Good safety profile in a large patient population - 7,490 patient years - Some patients exposed for more than seven years A committed and established partner in Teva An ongoing clinical development program EU market application (MAA) for the treatment of RRMS: negative opinion from the CHMP January 2014; re-examination requested * Data from an integrated analysis of two Phase III studies (Allegro and Bravo). 7

8 Laquinimod clinical development Relapsing Remitting Multiple Sclerosis (RRMS) CONCERTO - a third Phase III study ongoing under a SPA - Two doses (0.6mg and 1.2mg) in approx. 1,800 RRMS patients for up to 24 months will be evaluated - Primary endpoint confirmed disability progression as measured by the Expanded Disability Status Scale (EDSS) Other indications Clinical studies in Huntington s disease and Primary Progressive Multiple Sclerosis (PPMS) to be initiated 2014 Further clinical development in Crohn s disease on hold until a clearer clinical strategy has been defined 8

9 Laquinimod Commercial Development and Marketing Agreement Teva Pharmaceutical Industries Teva has global exclusive rights to develop, register, manufacture and commercialize laquinimod since 2004 Teva conducts and funds further clinical development of drug Expected to generate USD 92 million in overall milestones whereof USD 22 million received so far Active Biotech to receive tiered double digit royalties on future sales - 15 year royalty period on country-by-country basis - Higher royalty level in the Nordic/Baltic territory 9

10 Prostate cancer Increasing disease progression 2-4 years Median survival months Diagnosis Surgery/radiation Hormonal treatment (approx. 50% of patients) Wait for progression Pre-chemo Taxotere + Pred. Post-chemo Local PC Metastatic CRPC Targeting AR Injectable Oral Zytiga Xtandi Orteronel (PIII) Over 400,000 new prostate cancer patients diagnosed annually Prostate cancer market 2013E; 7 BUSD 1) Non-AR Prostvac (PIII) Yervoy (PIII) Provenge Custirsen (PIII) Jevtana Xofigo (alpharadin) Tasquinimod (PIII) Cabozantinib (PIII) 1) Credit Suisse Oct

11 Tasquinimod - a new first-in-class oral anti-cancer therapy A unique MoA targeting the tumor s microenvironment, mainly by binding the S100A9 protein Tasquinimod, potentially a new therapeutic option for chemo naïve patients with metastatic CRPC (1) Adapted from Raymond et al, Cancer Chemother Pharmacol, 2013

12 Tasquinimod: Treating castrate resistant prostate cancer (CRPC) Phase II Disease progression: bone scan or CT scan, cancer pain, pathological event 206 asymptomatic metastatic CRPC patients in the US, Canada and Sweden 2:1 randomization 1.0 mg tasquinimod vs placebo Results 69 % of tasquinimod patients vs 34 % of placebo patients had not progressed at 6 months (p<0.0001) 1). Median Progression Free Survival 7.6 vs 3.3 months (p=0.0042) OS data 2) supports that the observed increase in PFS will translate into an increased OS in a trial with sufficient statistical power Placebo switch on tasquinimod 1) Pili et al, JCO Oct 20, 2011: ) Armstrong et al, Clin Cancer Res

13 Ongoing Tasquinimod Phase III Trial Diagnosis Surgery/radiation Hormonal treatment (approx. 50% of patients) Wait for progression Pre-chemo Taxotere + Pred. Post-chemo Local PC Metastatic CRPC Indication: Primary endpoint: Metastatic castrate resistant prostate cancer (mcrpc) pre-chemo Radiographic Progression Free Survival (rpfs) Key secondary endpoint: Overall survival (OS) Study design: Regions: Randomized, double-blind placebo-controlled Global including US/Canada, Europe & China No. of patients: 1,245 Study status: Patient recruitment completed Dec 2012 Estimated filing: 2015 Coordinating investigator: Michael A. Carducci, Johns Hopkins University, Baltimore, US 13

14 Tasquinimod Phase III Analysis plan Primary radiographic progression-free survival (rpfs) analysis at the same time as the first interim overall survival (OS) analysis Time point for the OS interim analysis will be driven by the number of OS events 14

15 Tasquinimod Further Clinical Development Ph III Diagnosis Surgery/radiation Hormonal treatment (approx. 50% of patients) Wait for progression Pre-chemo Taxotere + Pred. Post-chemo Local PC Metastatic CRPC Prostate cancer Ph II Phase II proof of concept clinical study ( Switch maintenance ) of tasquinimod as maintenance therapy in patients with mcrpc who have not progressed after a first line docetaxel based chemotherapy (Ipsen) Phase I investigator sponsored clinical trial, led by Dr. Andrew Armstrong at Duke University Hospital, US ( Tasquinimod and Jevtana) Ph I Other cancers Phase IIa study with tasquinimod in metastatic renal, hepatic, ovarian and gastric cancer (Ipsen) 15

16 Tasquinimod Co-development with Ipsen Partnership announced April 18, 2011 Up to 200 M, including 25 M upfront and 32 M milestones received so far, and additional payments (upon achievement of clinical, regulatory and commercial milestones) Ipsen will pay Active Biotech progressive double-digit royalties on its net sales Active Biotech will be responsible for and finance the ongoing phase III trial Active Biotech granted Ipsen exclusive rights to commercialize tasquinimod worldwide, except for North and South America and Japan where Active Biotech retains all commercial and marketing rights 16

17 Renal Cell Carcinoma (RCC) 180,000 new cases annually 1) RCC market 2011 USD 2.7 billion 2) Approx. 50% cured Diagnosis Surgery Metastases approx. 50% of patients 5-year survival 5-15 % Therapy Disease progression 1) Cowen Therapeutic Categories Outlook March ) EvaluatePharma March

18 ANYARA Therapeutic principle: Selective drug retention in tumor tissue Activation and targeting of effector T cells Direct and indirect tumor cell killing ANYARA Direct killing by CTL SEA/E-120 C H V H 5T4(V14) T lymphocyte ANYARA 5T4 Tumor cell C L V L 5T4(V18) TCR Superantigen Anti-5T4 Fab TNF-α IFN-γ 18

19 ANYARA: Phase II/III trial in RCC Number of patients 513 Randomization Countries Primary endpoint IFNα vs ANYARA+IFNα UK, Ru, Uk, Bu, Ro (50 sites) Overall survival From Phase I to Phase II/III powered for OS analysis in RCC Primary endpoint not reached Subgroup analysis demonstrated proof of concept Doubling of PFS and OS in 25% of patients Good safety profile, in line with previous observations; most common adverse events were grade 1-2 fever, nausea or vomiting 19

20 Phase II/III Subgroup Analysis The 25 % of patients with low/normal levels of the biomarker IL-6 at baseline and expected anti-superantigen antibody levels, showed a statistically significant treatment advantage on both OS and PFS Baseline IL-6 Median IL-6 normal/low Anti-SAg expected Median Baseline Anti-SAg OS for the ANYARA group 63.3 months vs 31.1 months for interferon-α, (p=0.02, HR=0.59) PFS 13.7 vs 5.8 months (p=0.016, HR=0.62) The subgroup accounts for 40-50% of the total number of advanced RCC patients in North America and Western Europe Future development strategies discussed with regulatory authorities Next step a pivotal trial to treat a biomarker-defined group of renal cancer patients in second-line therapy to start only when an out-licensing agreement has been reached with a partner 20

21 Systemic sclerosis (SSc) Orphan indication - 1 to 3 per 10,000 in the US & EU Chronic autoimmune disease of unknown cause Fibrosis and vascular abnormalities affecting the skin, subcutaneous tissue, muscles and internal organs such as gastrointestinal tract, lungs, heart and kidneys Clinically heterogeneous; autoantibody production, fibrosis and vascular damage Current treatment aimed at controlling symptoms and preventing complications Paquinimod (57-57): Oral Treatment of SSc Quinoline molecule previously in clinical development for treatment of SLE Orphan Medicinal Product status for the treatment of SSc granted in the EU Exploratory clinical study in SSc concluded: - 9 patients - safety - evaluate effects on disease related biomarkers Next step a Phase II study in systemic sclerosis - will commence only when an outlicensing agreement has been reached with a partner 21

22 The ISI ( Inhibition of S100 Interactions ) Project Background Quinolines bind S100A9 (PLoS Biology April 2009) S100A9 interacts with the pro-inflammatory RAGE and TLR4 receptors Inhibition of S100A9/TLR4 interactions delays tumor growth (PLoS ONE March 2012) ON OFF = RAGE = TLR4 = S100 The ISI Project First indication oncology Development of NCE s that target S100 protein interactions Patent applications filed First CD selection Q = compound 22

23 Company information

24 Financials January December 2013 MSEK MUSD Jan - Dec Jan - Dec Net sales 116,0 227,9 17,8 35,1 Operating loss -209,0-163,2-32,2-25,1 Loss after tax -212,1-175,0-32,6-26,9 Cash on hand at December 31, 2013: MSEK 376,2 (57,9 MUSD) SEK/USD: 6,50 24

25 Share information Active Biotech market cap (Feb 24, 2014) SEK 2.9 billion USD 438 million Major shareholders (January 31, 2014) Number of shares: 74.9 million MGA Holding % Nordstjernan % Investor % 3rd AP fund % Avanza Pension % EFG Bank % JPM Chase % All other % Total % Number of shareholders whereof 10 largest 60.6% whereof 20 largest 68.0% Ownership categories Swedish owners 83.9% Foreign owners 16.1% 25

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