Idylla A fully automated, highly accurate, easy to use MDx platform: Too much to ask for?

Transcription

1 GOING MOLECULAR HAS NEVER BEEN EASIER Idylla A fully automated, highly accurate, easy to use MDx platform: Too much to ask for? October 18 th, 2017

2 NOTICES AND WARNINGS This presentation has been prepared by the management of Biocartis Group NV (the "Company"). It does not constitute or form part of, and should not be construed as, an offer, solicitation or invitation to subscribe for, underwrite or otherwise acquire, any securities of the Company or any member of its group nor should it or any part of it form the basis of, or be relied on in connection with, any contract to purchase or subscribe for any securities of the Company or any member of its group, nor shall it or any part of it form the basis of or be relied on in connection with any contract or commitment whatsoever. It is not a prospectus or offering memorandum. The information included in this presentation has been provided to you solely for your information and background and is subject to updating, completion, revision and amendment and such information may change materially. No person is under any obligation to update or keep current the information contained in this presentation and any opinions expressed in relation thereto are subject to change without notice. No representation or warranty, express or implied, is made as to the fairness, accuracy, reasonableness or completeness of the information contained herein. Neither the Company nor any other person accepts any liability for any loss howsoever arising, directly or indirectly, from this presentation or its contents. This presentation includes forward-looking statements that reflect the Company's intentions, beliefs or current expectations concerning, among other things, the Company's results, condition, performance, prospects, growth, strategies and the industry in which the Company operates. These forward-looking statements are subject to risks, uncertainties and assumptions and other factors that could cause the Company's actual results, condition, performance, prospects, growth or opportunities, as well as those of the markets it serves or intends to serve, to differ materially from those expressed in, or suggested by, these forward-looking statements. The Company cautions you that forward-looking statements are not guarantees of future performance and that its actual results and condition and the development of the industry in which the Company operates may differ materially from those made in or suggested by the forward-looking statements contained in this presentation. In addition, even if the Company's results, condition, and growth and the development of the industry in which the Company operates are consistent with the forward-looking statements contained in this presentation, those results or developments may not be indicative of results or developments in future periods. The Company and each of its directors, officers and employees expressly disclaim any obligation or undertaking to review, update or release any update of or revisions to any forward-looking statements in this presentation or any change in the Company's expectations or any change in events, conditions or circumstances on which these forward-looking statements are based, except as required by applicable law or regulation. This document and any materials distributed in connection with this document are not directed to, or intended for distribution to or use by, any person or entity that is a citizen or resident or located in any locality, state, country or other jurisdiction where such distribution, publication, availability or use would be contrary to law or regulation or which would require any registration or licensing within such jurisdiction. The distribution of this document in certain jurisdictions may be restricted by law and persons into whose possession this document comes should inform themselves about, and observe any such restrictions. The Company's securities have not been and will not be registered under the US Securities Act of 1933 (the "Securities Act") and may not be offered or sold in the United States absent registration under the Securities Act or exemption from the registration requirement thereof. 2

3 Biocartis: who we are First ever fully-automated MDx system Idylla with a fast growing menu primarily focused on oncology Headquartered in Mechelen (Belgium), employing > 350 people US commercialization partnership signed with Thermo Fisher in November 2016 Biocartis US Inc. founded in March 2017 Brussels Stock exchange (Euronext) listed since April

4 Continued expansion global commercial footprint* 2017/2018 Over 70 countries covered through three sales channels: Direct sales force covering Western European countries and the US 2 3 Distributor contracts in place covering approx. 58 countries including US Global pharma collaborations (e.g. Merck and Amgen) Direct sales Distributors Partnership under discussion 4 Close to 500 Idylla instruments installed per end H * Situation as per 30 June

5 Is biomarker TAT an issue? USA 3 Median 27 days Range: days UK days FRANCE 4, days 26% more than 4 weeks AUSTRALIA 1 Median 17 days 20% more than 4 weeks Source: 1 Scott et al. (2014) Asia Pac J Clin Oncol. 10(3):261-5; 2 Saunders et al. ESMO 2016, Abstract #; 3 Schwaederle et al. (2014) Oncologist 19, ; 4 INCa 2012 Survey, 4 Etude FLASH RAS 2014; 5 Mellert H et al. (2017) J Mol Pathol. 19(3):

6 Is insufficient sample affecting the testing? NSCLC Colorectal Cancer Lung Cancer Mutation Consortium s prospective multiinstitutional study of genomic profiling 1 From a total of 1536 adenocarcinoma samples, 28% were not eligible for genomic testing. The primary reason for ineligibility was the lack of tumor tissue for genomic testing. Retrospective analysis of KRAS testing rates in a community-based setting 3 From >1350 mcrc samples, 10% could not be tested due to insufficient tumor tissue In a study with 814 NSCLC samples, 10% could not be tested because of insufficient sample quality or quantity 2 >60% of mcrc samples provided for genomic testing contain a high amount of necrotic material 4 Source: 1 Kris MG et al. (2014). JAMA 311: , ; 2 Gutierrez et a. (2017) Clinical Lung Cancer; 3 Carter GC et al. (2015) J Exp Clin Cancer Res 34(1): 29; 4 personal communication MD Anderson Pathology 6

7 Instruments Best-in-class molecular testing Idylla System Highlights Fully automated walk-away system Less than 2 minutes hands-on-time Fast assay turn around times Between minutes Console Minimal amount of sample needed for a reduced invalid rate Only 1 slice of tissue or 1 ml of plasma per assay High accuracy and reproducibility Highly standardized technology and a contamination controlled - design Cartridge Modular instrument Ready to accommodate your lab s throughput Closed ready-to-use desktop system Small footprint and low consumable costs 7

8 Rapid molecular testing with Idylla Traditional way of molecular testing The Idylla way Where: IN HOUSE OR SEND-OUT Specialized labs only Molecular infrastructure needed Experienced lab technicians needed OR Sending out samples How: Batching of samples Increased hands-on time due to manual workflows 1/week or biweekly VS Where: IN-HOUSE All labs Any laboratory setting All lab technicians In-house testing How: Individual or batched analysis of samples < 2 min hands-on time due to automated workflow On demand 8

9 Idylla - Minimal handling steps and hands-on time Number of potentially contaminating sample handling steps* (pipetting, mixing, etc.) Average hands-on time per sample* (minutes, including controls) >200 >110 >20 1 >20 2 NGS PCR Idylla NGS PCR Idylla Data interpretation Molecular test setup and analysis Sample preparation * Based on workflow exercise (2015), batches of 16 samples, NGS technology: Illumina, PCR technology: Therascreen. Data validated in several university hospitals 9

10 Idylla - Less HOT compared to other PCR-based methods Therascreen Cobas % hands-on time 10

11 Idylla Less investment, reduced consumable cost Other RT-PCR NGS Instruments Instruments Instruments Consumables Consumables Consumables Lab infrastructure (# of rooms) 1 Lab infrastructure (# of rooms) 3 Lab infrastructure (# of rooms) 4 11

12 FDA 510K Exempt 510(k) exempt status for Idylla Instrument and Console. Since July 12 th, 2017 Biocartis Idylla Instrument and Idylla Console are listed as class II exempt from FDA 510(k) notification with FDA-cleared or -approved assays. 12

13 Idylla tissue specimen requirements KRAS NRAS-BRAF* 1 x 5µm FFPE glass mounted tissue section mm² 1 x 10µm FFPE tissue section mm² Neoplastic cell content 10% If < 10%, macrodissection needed BRAF 1 x 5µm FFPE glass mounted tissue section mm² 1 x 10µm FFPE tissue section mm² Neoplastic cell content 50% If < 50%, macrodissection needed To be determined by the pathologist EGFR 1 x 5µm FFPE tissue section 1 mm² Neoplastic cell content 10% If < 10%, macrodissection needed 13 *Included in the NRAS-BRAF-EGFR S492R product

14 Idylla plasma specimen requirements ctkras ctnras-braf* ctbraf Assay requires only 1 ml plasma per cartridge Two validated tube types: 1 2 K 2 EDTA tubes process within 4 hours Streck Cell-free DNA BCTs process within 3 days 14 *Included in the NRAS-BRAF-EGFR S492R product

15 Our Idylla menu today Relevant gene content - selected from relevant industry guidelines, key opinion leaders, and pharmaceutical research KRAS NRAS-BRAF* 21 mutations in KRAS exon 2, 3, and 4 23 mutations in NRAS exon 2, 3, 4 and BRAF exon 15 BRAF 7 mutations in BRAF exon 15 NRAS-BRAF* 23 mutations in NRAS exon 2, 3, 4 and BRAF exon 15 EGFR KRAS BRAF 51 mutations in EGFR exon 18, 19, 20 and mutations in KRAS exon 2, 3, and 4 7 mutations in BRAF exon *Included in the NRAS-BRAF-EGFR S492R product Assay contains 2 additional EGFR 492 mutations

16 Oncology Rapidly expanding Idylla TM test menu Onco Solid Biopsy Onco Liquid Biopsy On market assay Idylla TM Retrieve CDx Infectious Area On market end Focus as from 2019 (indicative) Colorectal Lung KRAS CE NRAS-BRAF CE NRAS/BRAF/EGFR492 RUO ctkras RUO EGFR RUO ctnras/braf/ EGFR492 RUO NRAS CE ctkras CE ctnras-braf CE EGFR CE ctegfr RUO MSI ctegfr CE GeneFusion Panel Expansion of existing assay menus: CRC Colorectal Lung cancer Melanoma Expansion into major oncology areas: Immunotherapy (MSI to be first test) Urology Melanoma BRAF CE ctbraf RUO DNA repair Breast Therapy selection Oncotype DX +++ Resistance monitoring To be further expanded Other MSI (immunotherapy) NGS Hotspot Panel Additional NGS Prep Panels to be launched Infectious diseases IFV-RSV Panel CE + Ebola EUA IFV-RSV Panel 510k+ Syndromic panels (initial assay Respiratory MP ++ ) and bloodstream infections (including sepsis) 16 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. CE = CE-marked tests. RUO = Research Use Only. EUA = Emergency Use Authorization label; + JnJ test; ++ Fast-track Diagnostics development; +++ The Oncotype Breast Recurrence Score test under development by Genomic Health

17 Strategic collaboration with Background collaboration Oncotype Breast Recurrence Score test Focused on exclusive test development of proprietary Genomic Health tests on the Idylla platform Aimed at accelerating adoption and market access around the world of Genomic Health s tests 17 First test to be developed on Idylla is the Oncotype DX Breast Recurrence Score test Background Genomic Health Leading provider of genomic-based diagnostic tests in cancer with revenues of USD 328m in 2016 Based in California (US) and listed on NASDAQ (GHDX) with a market cap of approx. USD 1bn On-market tests for breast, prostate and colon cancer, currently offered through own service laboratories Source: company website and financial reporting Genomic Health Provides personalized information for tailoring treatment of breast cancer patients based on the biology of their individual disease Predicts the likelihood of chemotherapy benefit as well as the chance of cancer recurrence in early-stage breast cancer patients Included in all major cancer guidelines worldwide and considered as standard of care for women with earlystage breast cancer

18 Initiated breast cancer menu development with partners Test Description Partner Partnership structure Resistance monitoring test Liquid biopsy test Monitoring of metastatic breast cancer patients for resistance to hormone therapy UK based medical research charity 1 Development multiple Idylla tests LifeArc acts as development contractor Biocartis responsible for commercialization under own label Therapy selection test Solid biopsy test Supporting optimal therapy selection decisions for breast cancer patients Singapore s Agency for Science, Technology and Research 2 Parties will co-invest in development of selected Idylla tests A*STAR acts as development partner Biocartis responsible for commercialization under own label Oncotype Dx Breast Recurrence Score test Solid biopsy test Tailoring treatment of breast cancer patients based on the biology of their individual disease US based provider of genomic-based diagnostic tests in cancer Genomic Health to develop Idylla TM versions of proprietary Genomic Health tests Genomic Health responsible for commercialization under own label Biocartis acts as supplier of tests On 15 June 2017 MRC Technology changed its name in LifeArc. LifeArc has been involved in helping deliver a number of therapies including Keytruda (pembrolizumab, marketed by MSD) which is an important immunotherapy treatment for various cancers. 2. Partnership is with ETPL, the commercialization arm of A*STAR.

19 TaT** Ease-of-use Sensitivity AstraZeneca study confirms best-in-class status Idylla Background Conclusions Comparative study organized by AstraZeneca 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 Technology Idylla KRAS 96% Other qpcr (cobas/therascreen) 46-52% Mass-spectrometry 58-92% NGS % ddpcr 56% Sanger sequencing 0% Overall sensivity 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) 19 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

20 Idylla MSI Test Some key characteristics Exclusively licensed novel set of biomarkers for MSI Proposed Intended Use The Idylla MSI Test will qualitatively detect a novel panel of about 7-8 MSI biomarkers in formalin-fixed, paraffin-embedded (FFPE) tissue sections from human colorectal cancer (CRC) tissue Novel MSI biomarker panel The novel biomarkers are different from the Bethesda markers Excellent concordance between the Promega panel and the novel panel was presented at ESMO 150 minutes total turnaround time Less than 2 minutes hands-on time Directly on FFPE tissue sections 20 The MSI Test is currently in development. Product characteristics mentioned are anticipated but not yet validated.

21 Promising MSI test to be launched in 2018 Background Microsatellite instability (MSI) is the consequence of errors in the body s so-called DNA mismatch repair system, resulting in potential tumor growth Initial target markets for MSI testing: o Recommended in several guidelines 1 for CRC (present in several other tumor types as well, such as gastric cancer) o Could be the sole independent factor to predict a patient s response to certain immunotherapies 2 for oncology Biocartis MSI test: o Is based on exclusively licensed biomarkers from the VIB 3 o Does not require sample control; only 1 FFPE slice per patient required In CRC samples 4 In gastric samples 5 Performance data licensed MSI Biomarkers 3 (Reference method ( RM ) is Promega MSI analysis) Included 870 samples 94% overall agreement with RM (discordance testing showed that MSI Biomarkers detected 6% more MSIhigh status) 12% of the tests performed with RM failed, even after repeat testing, compared to 4% with MSI Biomarkers Included 150 samples (study in collaboration with Merck KGaA) 100% overall agreement with RM for valid results 11% of samples tested with RM failed, even after repeat testing, MSI Biomarkers generated a result in 100% of the tests NCCN Guidelines Colon Cancer version ; and, Van Cutsem et al. (2016) ESMO Consensus Guidelines for the management of patients with mcrc. Annals of Oncology 27, Recent data have shown that advanced CRC patients with an MSI-high status respond particularly well to certain immunotherapies (Xiao Y et al. (2015) 3. Exclusive license agreement with the Flemish Institute for Biotechnology (VIB) for rt-pcr compatible MSI markers (the MSI Biomarkers ) 4. Maertens et al., Detection of microsatellite instability (MSI) in colorectal cancer samples with the automated Idylla MSI Test, 2017, to be presented as ESMO, 8-12 September 2017, Madrid, Spain 5. De Craene et al., Detection of microsatellite instability (MSI) with a novel panel of biomarkers in gastric cancer samples, 2017, to be presented as ESMO, 8-12 September 2017, Madrid, Spain

22 Idylla tissue-based assay performance Mutations Detected 21 mutations across 6 codons 18 NRAS mutations across 6 codons 5 BRAF V600 mutations 2 EGFR S492 mutations 7 mutations at codon V mutations in exons TAT 130 minutes 120 minutes 90 minutes 150 minutes LOD 5% 1-15% 1% 5% Concordance >95.9% (362 samples, Roche Cobas, HRM, pyrosequencing, Sanger, Therascreen, NGS) >99.6% (271 samples, NGS, ddpcr, pyrosequencing) >96.0% (Roche Cobas, NGS, SOC MD Anderson) >99.2% (261 samples, NGS) Values reported have not been validated in clinical studies 22 NRAS-BRAF is included in the NRAS-BRAF-EGFR S492R product FOR RESEARCH USE ONLY. NOT FOR USE IN DIAGNOSTIC PROCEDURES.

23 Idylla plasma-based assay performance Mutations Detected 21 mutations across 6 codons 18 NRAS mutations across 6 codons 5 BRAF V600 mutations 2 EGFR S492 mutations 7 mutations at codon V600 TAT 120 minutes 110 minutes 90 minutes LOD 0.1-2% (depending on mutation) 0.1% -3% (depending on mutation) 0.06%-0.2% (depending on mutation) Concordance Sensitivity: 98% Specificity: 95% Concordance: 96% Sensitivity: 100% (BRAF) Specificity: 99.3% (BRAF) Concordance: >99% Concordance: 88% Discordant analysis showed 100% agreement between Idylla, ddpcr, and BEAMing Values reported have not been validated in clinical studies 23 ctnras-braf is included in the ctnras-braf-egfr S492R product FOR RESEARCH USE ONLY. NOT FOR USE IN DIAGNOSTIC PROCEDURES.

24 Flexible pricing Capital Equipment and Cartridge Sales Reagent Rental Programs 24

25 Support and service Dedicated product specialists for installations, training and support Idylla average time between services is 18 months Rapid instrument swapout if service required 25

26 MULTIPLEXING 1 Tissue Slice 1ml Plasma The Idylla way High Reproducibility Fully HIGH ACCURACY Standardization FAST Closed System Automated Small Footprint Modular Contamination Control TESTING IN-HOUSE ANY LAB Clinically Relevant Mutations 26

27 Thank You Any Questions? GOING MOLECULAR HAS NEVER BEEN EASIER