See how you can guide the path her cancer takes

Transcription

1 See how you can guide the path her cancer takes 1

2 Idylla : easy, rapid and accurate molecular medicine for every patient Paola Valente Strategic Marketing Biocartis 6th meeting on external quality assessment in molecular pathology May 12-13, 2017 Naples 2

3 Biocartis: highlights MDx company founded in 2007, headquartered in Mechelen (Belgium), employing ~350 people Listed on Euronext (Brussels) Proprietary MDx system Idylla and diagnostic tests for oncology and infectious diseases Biocartis aims to provide direct access to personalized medicine for patients worldwide by developing fully integrated and broadly applicable molecular diagnostics 3

4 Today, there are barriers for adoption of precision medicine 4

5 Molecular diagnostics complexity Blood/plasma Batch based processes Manual labour intensive Tissue (FFPE) Swabs Range of clinical specimen types Require high complexity lab infrastructure Require highly trained personnel Sputum Complexity of biomatrix Urine In oncology: sample volumes are small FNA s Limited Number of Labs and Increased TAT* to Physician and Patient Stool/feces * TaT: Turnaround Time 5

6 How can we improve access to high precision diagnostics? 6

7 Idylla features Accurate results at right sensitivity Console Fully automated sample-to-result Any clinical sample type* High levels of multiplexing Short turnaround time Modular and scalable Data connectivity Instrument *validated on FFPE and nasal swabs Disposable cartridge 7

8 Limitation of erroneous results due to standardized cartridge Any sample type* No sample pre-treatment All reagents on board No PCR lab infrastructure No cold chain Stable at room temperature *validated on FFPE and nasal swabs 8

9 High precision diagnostics for high precision medicine Biocartis ambition THERAPY SELECTION PATIENT MONITORING EARLY DIAGNOSIS Idylla Treatment guidance Companion diagnostics Monitoring of treatment progress Early detection of relapse Rapid diagnosis High sensitivity Comprehensive panels First time right molecular diagnostic system Combining advantages of point of care testing with quality of lab reference testing 9

10 Idylla FFPE tests on the market Oncology BRAF Test Melanoma Solid biopsy 1 FFPE tumor slice CE-IVD 7 mutations Sensitivity of 1% TaT* approx. 90 min KRAS Test Colorectal (CRC) Solid biopsy 1 FFPE tumor slice CE-IVD 21 mutations Sensitivity of 5% TaT* approx. 2 hours NRAS-BRAF Test Colorectal (CRC) Solid biopsy 1 FFPE tumor slice CE-IVD 23 mutations Sensitivity 5% TaT* approx. 2 hours EGFR Assay NSCLC Solid biopsy 1 FFPE tumor slice Research Use Only (RUO) 52 mutations Sensitivity of 1-5% TaT* approx. 2.5 hours Offering complete CRC mutation analysis o Follows most recent clinical guidelines o Opens routes towards faster treatment selection * TaT: Turnaround time RUO assays are not for sale in the USA Launch first test lung cancer menu with Idylla EGFR Mutation Assay RUO CE-IVD available in

11 Idylla ctdna tests on the market and in development Oncology ctbraf Assay Research Use Only (RUO) 7 mutations Sensitivity of < 1% TaT* approx. 85 min ctkras Assay Research Use Only (RUO) 21 mutations Sensitivity of 1-5% TaT* approx. 130 min ctnras-braf- S492R Assay Research Use Only (RUO) 23 mutations Sensitivity 5% TaT* 110 min ctegfr Assay Research Use Only (RUO) From DNA extracted from plasma Available end 2017 ctkras and ctnras First assay under Merck KGaA collaboration launched: Idylla ctkras and ctnras Assay 11

12 High performance in comparative studies Publication update 16 publications in key journals Key takeaways Superior sensitivity compared to competing NGS and qpcr technologies Unrivalled ease of use 5x KRAS 6x BRAF Shorter turnaround times By test 1x EGFR 4x ctbraf Flexibility towards different sample types* Suitable for both solid and liquid biopsies *validated on FFPE and nasal swabs * Janku et al. BRAF Mutation Testing in Cell-Free DNA from the Plasma of Patients with Advanced Cancers Using a Rapid, Automated Molecular Diagnostics System. Mol Cancer Ther (2016) 15(6): 1 8; Schreuer et al. Quantitative assessment of BRAF V600 mutant cellfree tumor DNA from plasma as a diagnostic and therapeutic biomarker in patients with BRAF V600 mutant melanoma. ASCO 2015; De Biase et al. Fully Automated PCR detection of KRAS Mutations on Pancreatic Endoscopic Ultrasound Fine Needle Aspirates. J Clin Pathol 2016; Reijans et al. ESMO 2016, published on 6 October 2016; De Luca et al., J Clin Pathol 2016; J.L. Sherwood et al., KRAS ESMO Abstract 91 P: Implications of key differences across 12 KRAS mutation detection technologies and their relevance in clinical practice ; Ellen Vercauteren et al., NRAS ESMO Abstract 1175P: Ultra-rapid, sensitive, and fully automated extended RAS testing for metastatic colorectal cancer evaluation of an NRAS/BRAF/EGFR492 module ; Preliminary Performance Study based on Research data. Martin Reijans et al., EGFR ESMO Abstract 1173P: Fully automated and sensitive detection of EGFR exon 18, 19, 20 and 21 mutational status in less than 2.5 hours from a single FFPE slice ; Jérôme Solassol et al., Multi-Center Evaluation of the Fully Automated PCR-Based Idylla KRAS Mutation Assay for Rapid KRAS Mutation Status Determination on Formalin-Fixed Paraffin-Embedded Tissue of Human Colorectal Cancer 12

13 Outperforming reference testing Approx. 5% more patients could have benefited from targeted BRAF therapy BRAF Mutation Test KRAS Mutation Test STUDY/ TRIAL LOCATION # OF SAMPLES OVERALL CON- CORDANCE* OVERALL CONCORDANCE AFTER DISCORDANCE TEST** STUDY/ TRIAL LOCATION # OF SAMPLES OVERALL CON- CORDANCE + OVERALL CONCORDANCE AFTER DISCORDANCE TEST ++ Validation study Beta trial Research study Trial (off-label) Trial (off-label) Charité Berlin, UZA Antwerp 6 regional hospitals*** % 99.6% % 100% MD Anderson % 100% Medical University of Vienna Oxford University Hospitals NHS Trust % 100% % 100% Mini-performance evaluation (NGS) Verification study (Ultra Deep Seq) Verification study (alpha trial) Validation study Beta trials In-house % 100% In-house % 99.1% Commercial reference lab University Hospital Antwerp 12 european sites % 99.1% % 100% % 98.9% Outperforms reference testing in studies with 821 clinical samples (of which ~532 melanoma samples) * Reference methods: Roche cobas, CLIA laboratory PCR-based sequencing, Sequenom MassARRAY and Pyrosequencing ** Ultra deep sequencing and digital droplet PCR *** Rigshospital Copenhagen, Imelda Bonheiden, Jan Yperman Ieper, Hospital del Mar Barcelona, Karlsruhe, LabPON Hengelo Note: number samples tested with discordance test varies between study/trial Outperforms reference testing in studies with 850 clinical samples + Reference methods: Roche Cobas and NGS ++ Ultra deep sequencing and digital droplet PCR +++ Out of a subset of the discordant samples, insufficient sample was available to perform a discordance test 13

14 AstraZeneca study confirms high performance of Idylla KRAS Background Conclusions Comparative study organized by AstraZeneca Technology Overall sensivity Comparison of 12 different KRAS mutation detecting technologies: 5x NGS 3x qpcr 2x mass spec. 1x ddpcr 1x Sanger sequencing Focused on detection of KRAS mutations in lung cancer based on blinded samples Sensitivity Ease-of-use TaT** Idylla KRAS 96% Other qpcr (cobas/therascreen) 46-52% Mass-spectrometry 58-92% NGS % ddpcr 56% Sanger sequencing 0% Highest score for Idylla KRAS technology: o Lowest number of manual handling steps in sample preparation (1 to 2 steps versus 3 to > 20 steps) o Requires lowest level of expertise (1 versus 2-4 for others * ) Highest score for Idylla KRAS technology on total turnaround time (2 to 4 hours versus 1 day to 3 weeks) Source: poster by James L. Sherwood et al., presented at 2016 ESMO conference Copenhagen (Denmark) * One being the lowest level of expertise and four the highest ** TaT = total turnaround time 14

15 Idylla UK Neqas Scheme Idylla reported 100% correct results on FFPE samples in round 1 and 2 for BRAF in melanoma (CE), KRAS in colorectal cancer (CE), NRAS in colorectal cancer (RUO) and EGFR in lung cancer (RUO) 15

16 A Phase II clinical trial on the combination of dabrafenib plus trametinib for BRAF (+MEK)-inhibitor pretreated patients with advanced BRAF V600 mutant melanoma Schreuer et al. Publication Lancet Oncology and Poster presented at ESMO Copenhagen 2016 AIM: Rechallenge BRAF V600mut melanoma patients who previously progressed on BRAF-inhibitors and immunotherapy with dabrafenib + trametinib (BRAF and MEK inhibitor) and this after treatment interruption. 25 patients (stage IV melanoma) PFS (progression free survival) 4,9 months 8 pts PR (32%) 10 pts SD (40%) (partial response) (stable disease) No mutant V600 ctbraf present 2 weeks after start rechallenge treatment Better PFS ctbraf levels could be an early indicator of outcome 16

17 Rapidly expanding test menu 17

18 Our Idylla menu Onco Solid Biopsy Onco Liquid Biopsy On market assay Idylla TM Retrieve CDx Infectious Area On market end Focus as from 2019 (indicative) Oncology Colorectal Lung Melanoma Other KRAS CE NRAS-BRAF CE NRAS/BRAF/EGFR492 RUO ctkras RUO EGFR RUO BRAF CE ctbraf RUO ctnras/braf/ EGFR492 RUO NRAS CE ctkras CE ctnras-braf CE EGFR CE ctegfr RUO MSI ctegfr CE GeneFusion Panel NGS Hotspot Panel Additional assays to be launched for CRC and lung cancer menus Expansion into major oncology areas: o Breast o Urology Colorectal o Immunotherapy o DNA repair* Additional NGS Prep Panels to be launched for number of pan-tumor indications CDx CDx signed Additional CDx programs to be added Infectious diseases IFV-RSV Panel CE + Ebola EUA IFV-RSV Panel 510k + Syndromic panels through partnership CE = CE-marked tests. RUO = Research Use Only. EUA = Emergency Use Authorization label + JnJ test Note: overview is subject to changes in prioritization of test development driven by several factors such as commercial and operational considerations. Overview excludes regional expansion, life cycle management and potential partner tests. 18

19 Unlocking the Power of High Precision Medicine for Every Patient