Diagnostics for the early detection and prevention of colon cancer. Publication of DeeP-C Study Data in New England Journal of Medicine March 2014

Transcription

1 Diagnostics for the early detection and prevention of colon cancer Publication of DeeP-C Study Data in New England Journal of Medicine March 2014

2 Safe Harbor Statement Certain statements made in this presentation contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities and Exchange Act of 1934, as amended, that are intended to be covered by the safe harbor created by those sections. Forward-looking statements, which are based on certain assumptions and describe our future plans, strategies and expectations, can generally be identified by the use of forward-looking terms such as believe, expect, may, will, should, could, seek, intend, plan, estimate, anticipate or other comparable terms. Forward-looking statements in this presentation may address the following subjects among others: statements regarding the sufficiency of our capital resources, expected operating losses, anticipated results and timing of our pivotal clinical trial, expectations concerning our ability to secure FDA approval of our Cologuard test, expected license fee revenues, expected research and development expenses, expected general and administrative expenses and our expectations concerning our business strategy. Forward-looking statements involve inherent risks and uncertainties which could cause actual results to differ materially from those in the forward-looking statements, as a result of various factors including those risks and uncertainties described in the Risk Factors and in Management s Discussion and Analysis of Financial Condition and Results of Operations sections of our most recently filed Annual Report on Form 10-K and our subsequently filed Quarterly Reports on Form 10-Q. We urge you to consider those risks and uncertainties in evaluating our forwardlooking statements. We caution readers not to place undue reliance upon any such forward-looking statements, which speak only as of the date made. Except as otherwise required by the federal securities laws, we disclaim any obligation or undertaking to publicly release any updates or revisions to any forward-looking statement contained herein (or elsewhere) to reflect any change in our expectations with regard thereto or any change in events, conditions or circumstances on which any such statement is based. We have filed a registration statement (File No ), including a prospectus, with the U.S. Securities and Exchange Commission (the SEC ) for the offering to which this communication relates. Before you invest, you should read the prospectus in that registration statement and other documents the issuer has filed with the SEC for more complete information about the issuer and this offering. You may get these documents for free by visiting EDGAR on the SEC website at Alternatively, the issuer, any underwriter, or any dealer participating in the offering will arrange to send you the prospectus if you request it by calling or For Investigational Use Only. The performance characteristics of this product have not been established.

3 Study Design Primary Objective Determine sensitivity and specificity of Cologuard for colorectal cancer Secondary Objective Compare sensitivity and specificity of Cologuard to FIT for colorectal cancer and advanced adenoma Prospective, multicenter study 90 sites enrolled >10,000 subjects All subjects completed Cologuard, FIT, and colonoscopy (reference method) Designed with input from national experts, FDA and CMS 2

4 Primary Endpoints Endpoint Success Criteria CRC Sensitivity Colonoscopy as reference method 65% One-sided 95% lower bound Specificity Colonoscopy as reference method 85% One-sided 95% lower bound 3

5 Secondary Endpoints Endpoint CRC Sensitivity Colonoscopy as reference method AA Sensitivity Colonoscopy as reference method Success Criteria Non-inferiority to FIT Performance difference no more than 5% (using 95% CI lower bound) Superiority to FIT McNemar s comparison test, one-sided p-value <0.025 Superiority to FIT McNemar s comparison test, one-sided p-value <

6 90 Enrollment Sites 5

7 Eligibility Criteria Adults between the ages of 50 and 84 years (inclusive) At average risk for colorectal cancer No history of CRC or adenoma, aerodigestive tract cancer or high risk conditions for CRC No family history of FAP or HNPCC No positive fecal occult blood test or FIT in the previous 6 months No prior colorectal resection for any reason other than sigmoid diverticular disease No overt rectal bleeding in the previous 30 days No colonoscopy in past 9 years or barium x-ray, virtual colonoscopy or flexible sigmoidoscopy in the past 5 years 6

8 Primary enrollment = 10,023 Gender Race Ethnicity DeeP-C Study U.S. Average (50-84) 1 Men 46.3% 47.1% Women 53.7% 52.9% White 84.1% 83.0% Black/African American 10.7% 10.7% Other 5.2% 6.3% Hispanic 9.9% 9.3% Non-Hispanic 90.1% 90.7% Age Average (years) US Census Bureau 2012 estimate

9 Cologuard Outperforms FIT in Deep-C Study: Results are Statistically Significant Cologuard FIT P Value Cancer 92.3% (60/65) 73.8% (48/65) 18.5% Advanced Adenomas 42.4% (321/757) 23.8% (180/757) 18.6% <

10 In a theoretical 10,000-patient cohort Cologuard would detect 12 more cancers than FIT Cancer F I T Cologuard Cologuard would detect 141 more pre-cancers than FIT Pre- Cancer F I T Cologuard

11 Cologuard Cancer Sensitivity by Stage Cologuard FIT 89.7% 100.0% 90.0% 90.0% 93.3% 65.5% 76.2% 75.0% 75.0% 73.3% Stage I Stage II Stage III Stage IV Stages (I III) (N = 29) (N = 21) (N = 10) (N = 4) (N = 60) 10

12 Cologuard Cancer & Pre-cancer Detection by Location Cologuard FIT Cancer 90.0% 94.3% 80.0% Pre-cancer 54.5% 66.7% 33.2% 34.8% 15.5% Right-Sided Cancer Left-Sided Cancer (N = 30) (N = 35) 11 Right-Sided Pre- Cancer (N = 431) Left-Sided Pre- Cancer (N = 325)

13 Pre-cancer Detection by Polyp Size Cologuard FIT 64.6% 68.4% 30.3% 15.2% 39.0% 20.9% 43.0% 42.1% <1.0cm cm cm 3.0cm (N = 66) (N = 574) (N = 79) (N = 38) 12

14 Pre-cancer Detection by Polyp Type 69.2% Cologuard FIT 46.2% 42.4% 5.1% High Grade Dysplasia Sessile Serrated lesions 1.0cm (N = 39) (N = 99) 13

15 Cologuard Specificity Per Study Design With Negative Colonoscopy 86.6% 89.8% Includes non-advanced adenomas as a negative finding Includes hyperplastic polyps as a negative finding No excision or biopsy sent to pathology Matches 90% specificity performance in company s cut-off validation study 14

16 Presentation Summary Publication in New England Journal of Medicine highlights Cologuard s performance In DeeP-C study, Cologuard detected significant numbers of cancers and pre-cancers that FIT missed Cologuard s performed well Stage of cancer Size and type of pre-cancer Location of cancer or pre-cancer 15

17 16 Appendix

18 DeeP-C Exclusion Waterfall Primary reason for exclusion (in order) Total Enrollment 12,776 1) Withdrew Consent 464 2) No Colonoscopy 1,168 3) Colonoscopy Not Useable 304 4) No Stool Sample 128 5) Stool Sample Unusable 474 6) No Cologuard result 213 7) Missing Samples 2 8) No FIT with no Cologuard result 333/689 had no FIT result In total, 689 with no Cologuard compares to 367 with no FIT result Included in analysis 9,989 17