Multi-Modal BioInformatics Solution for Ovarian Cancer

Transcription

1 VERMILLION Multi-Modal BioInformatics Solution for Ovarian Cancer NASDAQ: VRML l April Vermillion Inc. All Rights Reserved 1

2 FORWARD-LOOKING STATEMENTS This presentation contains forward-looking statements, as defined in the Private Securities Litigation Reform Act of Words such as may, expects, intends, anticipates, believes, estimates, plans, seeks, could, should, continue, will, potential, projects and similar expressions are intended to identify such forward-looking statements. Readers are cautioned that these forward-looking statements speak only as of the date of this presentation, and the Company does not assume any obligation to update, amend or clarify them to reflect events, new information or circumstances after such date except as required by law. Company estimates set forth in this presentation are based on various sources of information and various assumptions and judgments made by the Company, which Company management believes are reasonable. However, the Company cannot assure you that Company estimates are correct, and actual data may materially differ from Company estimates. The forward-looking statements reflect the views of the Company as of the date of this presentation and are subject to certain risks, uncertainties and assumptions, including those described in the section entitled Risk Factors in the Company s Annual Report on Form 10-K for the year ended December 31, 2017 and quarterly report on Form 10-Q for the quarter ended September 30, This presentation is copyright 2019 by Vermillion, Inc. All Rights Reserved. 2

3 Investment Highlights Commercial stage company with ovarian cancer cancer risk assessment test Pipeline of compelling diagnostic bioinformatic solutions FDA approved and incorporated into clinical treatment guidelines Broad managed care coverage CMS reimbursement rate of $897 Intellectual property protecting methods and use Seasoned management team 3

4 Commercial Stage Company Save Lives Replace Standard of Care Payers * Guidelines Bioinformatic Tools + Current Standard Care Strong IP and FDA-Cleared Science Foundation in place for OVA 1 to become the new Standard of Care Payer Coverage: 5 out of 10 lives covered in U.S. 4

5 The Size of the Pelvic Pain and Mass Market TAM Cost $ 18.5M 500K- 1M $22B $0.8B Pelvic Masses + (Endo + PCOS+ Func. Cysts) Pelvic Masses (Benign masses, cancer, non-gyn mass) Technology Solutions Today None None VRML Portfolio POC Complete (DXA II) Watch + Wait (DXA1) 200K Pelvic Masses to Surgery Ova1+ (OVA1, Overa) Ova1+ (OVA1, Overa) 22K $5.2B Ovarian Cancer CA125 Recurrence Monitoring Portfolio Funnel Ovarian 15K Cancer Deaths Total: 20M $28B 5

6 Paradigm Shift- Single Modality vs. Multi-modality Approach Protein Biomarkers Cell Free DNA OVA 360 FDA Cleared Algorithms - Systemic Dx OVA360 = - Focused Dx - Family Hx / Genetics - Patient Reported Data (Symptom Index) Clinical Assessment + Symptom Index + Imaging Hereditary Breast and Ovarian Cancer (HBOC) Genetics 6

7 Ovarian Cancer: A Severe Clinical Problem - > 60% of Ovarian Cancers are diagnosed at late stage - > 50% mortality rate (only gender specific disease with greater than a 50% mortality rate) - Mortality has not changed in 30 years Presentation Stage and 5-Year Survival Rate Presentation Stage Incidence 5-year survival rate (Stage I) Localized 15% 93% 4 (Stage II) Regional 20% 73% (Stage III) Distant 60% 29% (Stage IV) Unstaged 6% 25% NEED A BETTER SOLUTION 7

8 The Why: Current Assessment Tools are Inadequate CLINICAL ASSESSMENT: PHYSICAL EXAM AND ULTRASOUND Analysis of results is subjective Results are specialist-dependent BLOOD TUMOR MARKER: OFF-LABEL USE OF CA-125 (1981) / ALTERNATIVE TECHNOLOGY ROMA (2011) Both approaches have high rate of false negatives Especially in premenopausal and early stage patients TISSUE ANALYSIS: PREOPERATIVE BIOPSY- NOT MEDICALLY APPROPRIATE Cannot biopsy pelvic cavity Possible spread of tumor intra-abdominally if biopsy ruptures mass 8

9 Our Solution = OVA1 + OVERA (OVA1 +) For the detection of Ovarian Cancer Risk in Women with Pelvic Masses Launch 2010 OVA1 evaluates the levels of five ovarian cancer-associated markers in the blood Levels combined into a single ovarian cancer risk score. Multi-variate Index Assay (MIA) in ACOG Guidelines Positive NCCN and SGO position statements Launch Q Overa incorporates 2 new markers Global Platform Specificity + 9

10 Improved Specificity OVA1 + - Ova1/Overa Reflex Offering- Launched Q Low Risk ACOG Endorsed Intermediate Risk Reflex to: ACOG Endorsed Elevated Risk Premenopausal 2 : Low Risk < 5.0; Intermediate ; Elevated Risk > 7.0 Postmenopausal 2 : Low Risk < 4.4; Intermediate ; Elevated Risk > 6.5 OVA1 1 (95%CI) Overa 1 (95% CI) OVA1 + (95% CI) % Diff OVA1 vs OVA1+ Sensitivity 92% 91% 88% -4% Specificity 54% 69% > 30% improvement in specificity 1. Coleman RL, Herzog TJ, Chan DW, et al. Validation of a second-generation multivariate index assay for malignancy risk of adnexal masses. Am J Obstet Gynecol 2016;215:82.e Reference Ranges established by ASPiRA Labs, Austin Tx. 72% 33% 10

11 Improved Early Stage Detection: OVA1 Plus vs Standard of Care in Stage I + II Patients 94% Improvement in Rate of Cancer Detected with OVA1 11

12 Non-white women, and African-American women in particular, display significantly lower CA125 values compared to Caucasian women This disparity is found in healthy women, women at high risk for ovarian cancer, and women with ovarian cancer (Caucasian) 1645 (Non-white) 582 (Caucasian) 35 (Non-white) (Caucasian) 89 (African-American) 1-4: 1. Pauler, D., et al. Factors Influencing Serum CA125II Levels in Healthy Postmenopausal Women. Cancer Epidemiology, Biomarkers & Prevention, 10: , 2001, 2. Skates, S., at al. Large Prospective Study of Ovarian Cancer Screening in High-risk Women: CA125 Cut-point Defined by Menopausal Status. Cancer Prevention Research, 4(9), , 2011, 3. Cramer, D., et al. Correlates of the pre-operative level of CA125 at presentation of ovarian cancer. Gynecologic Oncology, 119(3), , 2010., 4. Babic, A., at al. Predictors of pretreatment CA125 at ovarian cancer diagnosis: a pooled analysis in the Ovarian Cancer Association Consortium. Cancer Causes & Control : CCC, 28(5), , (Premenopausal, non-african-american) 68 (Premenopausal, African American) 1604 (Postmenopausal, non-african-american) 52 (Postmenopausal, African-American) 12

13 High Level Internal Data from ASPiRA LABS OVA1 Superiority over CA125 in African American Women OVA1 CA125 Sens (%) Spec (%) PPV NPV Caucasian (N=873) African American (N=156) Caucasian (N=873) African American (N=156) CA125 has an unacceptable sensitivity for cancer detection in African American women Abstract accepted and presented at Mid Atlantic Gynecologic Oncology Society (MAGOS) in October 2018 Abstract accepted at American Association for Cancer Research (AACR) Manuscript submitted for publication- pending acceptance (OVA1 shows an acceptable sensitivity for cancer detection in African American women, cutoff adjustment is in process for pre and post menopausal women, to achieve the 90% sensitivity obtained for Caucasian women 5) 5: ASPiRA Labs Data on File, Combined OVA1 and OVA500 studies Vermillion Inc. All Rights Reserved 13

14 Summary of OVA1 + Sensitivity Across All Stages* CA125 Stage I X Stage II X Stage III Stage IV Sensitivity Across Menopausal Status* Pre-menopausal X Post-menopausal Sensitivity Across Histological Subtypes* Epithelial ovarian cancer Non-epithelial cancer X Low malignant potential X Metastatic Other gyn cancer X Sensitivity Across All Ethnicities* Caucasian and African American X Ovarian Cancer still remains a diagnostic dilemma for GYN and Primary Care physicians making OVA1 + more clinically relevant For High Risk: o Early detection and appropriate referral to Gyn Oncologists give patients a survival benefit and is proven to be cost-effective For Low Risk: o The NPV of OVA1 + will ensure the correct treatment plan, and reassure both the patient and the Gyn provider when planning surgery OVA1 + truly changes how providers treat patients Vermillion Inc. All Rights Reserved 14

15 Commercialization Strategy Direct to Gynecologists - Centralized Testing 20 Sales Territories Calling on Gynecologists to drive volume Calling on and Gynecologic Oncologists to increase awareness Tests performed in our Lab Entrepreneurial Commercial Force Sales team has average of 15 years experience in genetics, diagnostic lab and women s health space Strong relationships with large Ob Gyn practices/physicians in target markets Top performers in companies with disruptive technology successful growing volume from ground level >75% of hires were involved in the initial rollout of NIPT 15

16 Commercial Investment S A L E S T E A M Payers Investment in Commercial 2.5X year on year PAMA Rate Evicore Live Launch: Decentralized Platform OVA1+ 16

17 Commercialization Strategy- US + Ex US Decentralized- Performed in Local Setting Testing Performed in Hospital Systems/Large Gyn Super Group Increase POC (Point of Care) Test performed locally with access to Vermillion risk assessment software via web service OVA1 performed on existing Platform Roche Cobas installed base of over 10K units globally Algorithm Warehouse OvaCalc Cloud based bioinformatics platform Technology Transfer Customer Vermillion Data Repository 17

18 International - Commercial Strategy Long Term Both OVA1/Overa have CE Mark Philippines Large prospective study in process International via Platform/Web Service Israel Q Coverage received in Israel by CLALIT 2 nd largest integrated delivery network in the world CLALIT (#1 Payer, 50% pop) Study in process to validate Overa & OVA1 on local population 18

19 Market Access Covered Lives Cigna added in January 2019 Coverage: Patient Lives In Millions 167M Cigna added Ova1 to its National Preferred Coverage List in January 2019 Goal: Targeted Growth with Positive Medical Policy Decisions 51% of the Population Now Under Positive Coverage 1/

20 Reimbursement Rates have increased steadily Weighted Average Ova1 Price Per Test TARGET GOAL PAMA Price set for OVA1 PAMA Rate = $897 CPT Code

21 Historical Cash Utilization Q Disciplined capital deployment PayersCash to support commercialization plans Q1 '17 Q2 '17 Q3 '17 Q4 '17 Q1 '18 Q2 '18 Q3 '18 Q4 '18 Beginning Cash Balance $5,242 $7,897 $6,028 $7,700 $5,539 $3,100 $14,134 $11,760 Financing Activities $5,255 ($129) $3,581 ($5) $0 $13,529 $0 $0 Cash Utilization ($2,600) ($1,740) ($1,909) ($2,156) ($2,439) ($2,495) ($2,374) ($2,400) Ending Cash Balance $7,897 $6,028 $7,700 $5,539 $3,100 $14,134 $11,760 $9,360 Additional existing sources of financing: State of CT Forgivable Loan $2M (Contingent on achieving milestones) Callable Warrants $5M (Contingent on stock price performance) 21

22 Portfolio Opportunity Size US + Ex-US Overall Total: $2.2B - $4.4B US $90M-$269M EX-US $100M-$300M US $440M-$880M EX-US $500M-$1.0B US $1.2B-$2.4B EX-US $1.0B-2.0B US K TAM EX-US 1M-3M TAM Guided referral US 250K-1M TAM EX-US 2.5M-10M TAM Rule out; watchful waiting, surgical choice, disease progression, high risk BRCA, recurrence monitoring US>1M TAM EX-US > 10M TAM Endometriosis, polycystic ovary syndrome 22

23 2019 Key Catalysts Payer Expansion Bio-informatics Platform- Beyond Proteins Portfolio Expansion- Watch and Wait + ( M) OVA1+ vs Standard of Care Publications/Tools Ca125 Disparity Data Published/ Symptom Index Tool Live Commercial Team Expansion Complete Decentralized Partnership Expansion Pelvic Mass Portfolio Expansion 23

24 Strategy: Built For Growth Strategic Strengths and Capabilities MYGN GHDX EXAS VRML FDA-cleared product X HBOC X Unique Category 1 CPT code (AMA) Medicare Coverage/Gap Fill Pricing IVD Kit X X X CLIA lab for LDTs Multi-modal Predictive predictive Analytics analysis X X X Proprietary Disease Registry/Samples Leading Peer-reviewed Publications Strong Outreach: KOLs/Advocacy 24

25 Investment Summary Commercial stage company with ovarian cancer risk assessment test Recently Expanded Sales Team Broad managed care coverage CMS reimbursement rate of $897 Pipeline of compelling diagnostic bioinformatic solutions Seasoned management team 25

26 Appendix 26

27 Majority of Pelvic Masses Are Benign A Low False Negative Rate Is Critical For Patient Care Early Stage Sensitivity (%) Specificity (%) Early Stage False Negative Rate(%) False Positive Rate(%) Clinical Assessment (CA) Standalone Risk Stratification Ultrasound alone CA125 alone ROMA (Ca125 & HE4) OVA1 alone Overa alone OVA1 +* Demonstration of Improvement - Reducing False Negatives by Over 50% 1. Longoria, TC et al. AJOG Jan 2014, 210(1,): 78.e1-78.e9 2. Pavlik EJ, van Nagell JR Jr. Womens Health (Lond) Jan;9(1): Partheen K, Kristjansdottir B, Sundfeldt K. J Gynecol Oncol. 2011;22(4): Chudecka-Glaz, A et al. J Mol Biomark Diagn. 2013, S4: Bristow, RE et al. Gynecol Cncol. 2013, 128: Coleman RL, Herzog TJ, Chan DW, et al. Am J Obstet Gynecol 2016;215:82.e ASPiRA Labs Data on File, Combined OVA1 and OVA500 studies. *Internal data on file Average of 4 trials in study

28 Better Science & Demonstrated Lower Cost OVA1 +: better science than CA-125 Use of OVA1 + vs CA-125 lowers total costs while improving care and addressing racial disparity Our study findings suggest that OVA1 testing due to its increased sensitivity and NPV may result in savings for payers of $0.05 PMPM by enabling physicians to better manage patients in the appropriate setting of care 1 24-month average reimbursement for early- and late-stage cancer Cancer Stage Commercial Insurance Reimbursement Premenopausal Women Postmenopausal Women Early-stage Cancer $35,754 $37,195 Late-stage Cancer $224,922 $197,757 * Reimbursement rates are static between current and future states If we could just make sure that women get to the people who are trained to take care of them, the impact would be much greater than that of any new chemotherapy drug or biological agent, Dr. Robert E. Bristow, Director of Gynecologic Oncology, University of California 1 Brodsky B.S., Owens G.M., Scotti, D.J., et al. AHDB. 2017:10(7): Vermillion Inc. All Rights Reserved 28

29 Trusted Solution: Care Pathway Guidelines Publications Drive Standard of Care Published Evidence OVAI (MIA) Guidelines / Position Statements * Ueland, et al Obstetrics and Gynecology, 2011 Bristow, et al. Gynecologic Oncology, 2013 Am J Gynecol, 2013 ACOG Practice Bulletin Number 174, November 2016, page 10 Longoria, et al. Am J Obstet Gynecol, 2013 Goodrich, et al. Am J Obstet Gynecol, 2015 Forde, et al. Curr Med Res Opin, 2015 National Comprehensive Cancer Network Guidelines, Version 5, 2017 Updated Feb 2, 2018 Coleman, et al. Am J Obstet Gynecol, 2016 Eskander, et al. Am J Obstet Gynecol, 2016 Urban, et al. Int. J Gynecol Cancer, 2017 Brodsky, et al. Am Health & Drug Benefits, 2017 Society of Gynecologic Oncology Position Statements Issued 2011 Updated 2013 * In 100% of all Key Guidelines 29

30 Care Pathway Guidelines Converted to the Gyn Practice Best Care pathway- LEVEL A + LEVEL B ACOG Pelvic Mass Detected on Ultrasound (ACOG Level A) Clearly Benign Simple appearance, smooth, thin walls, absence of solid components or septations, lack of internal blood flow on color Doppler. Generally <10cm, but even above 10cm if cyst is simple risk of malignancy is 1%. Watchful waiting management of symptoms Not Clear Everything else (3-10cm) not thin walled, > 1 septation, small nodules. With OVA1 you get a clear picture when managing adnexal masses Elevated Risk Of Malignancy Refer GynOnc GynOnc Consults ACOG Level B Low Risk Of Malignancy OB / GYN Treats No Further Imaging Included in ACOG Adnexal Mass Management Guidelines Clearly Malignant Cyst. 10cm, papillary or solid components, irregularity, presence of ascites high color Doppler flow. Get CA-125 and refer to Gyn Onc immediately 30

31 Protected Solutions: Strong IP Intellectual Property: Strong Portfolio Granted Pending Family USA Ex US Total USA Ex US Total Issued patents covering various ovarian cancer biomarkers Pending patent applications covering OVA1 and Overa products Algorithm kept as trade secret 31