RiboMed Biotechnologies, Inc.

Transcription

1 RiboMed Biotechnologies, Inc. Ribomed Clinical Services Lab San Diego Science Center 3030 Bunker Hill, Suite 117 San Diego, CA CLIA# O5D For more information contact: Michelle Hanna, Ph. D. Founder and CEO Business Offices & Manufacturing 3469 Kurtz St. San Diego, CA Ph:

2 RiboMed Overview Privately held commercialization stage diagnostic company Developing liquid biopsy tests for cancer detection, subtyping, therapy selection & monitoring Proprietary platform detection technologies o Abscription & CAPS (Coupled Abscription PCR Signaling) o 30 issued or pending patents on Abscription based biomarker detection, including DNA, DNA methylation, RNA, Proteins Offer products & services for detecting genetic & epigenetic biomarkers o o Proprietary RUO products based on Abscription MethylMagnet, MethylMeter, CAPS Proprietary Abscription based LDTs Offered in CLIA/CAP accredited Ribomed Clinical Services Lab Use Ribomed s RUO products 2

3 Peak Area Abscription : One Technology for Multiple Biomarker Types Detection of Viral RNA DNA Methylation RNA Abscription DNA Duplex Detection of CEA & SEB SEB ng/ml: 0 CEA ng/ml: SEB CEA Duplex Assay (5 min) AAU (SEB) AAG (CEA) Detection of Botulinum toxin from raw milk Pfp and Pfn << 10-6 Proteins Detection of Salmonella DNA Funded by $9M from NIH, DOD & DHS

4 The Need: Noninvasive Diagnostics aka Liquid Biopsies Ribomed is utilizing its proprietary detection technology to develop sensitive, multivariate diagnostic tests using both epigenetic & genetic biomarkers for disease detection & stratification from bodily fluids. Tests in development Liquid Biopsies o Urine based tests/blood backup o DNA methylation profiles plus other clinically relevant biomarkers o Focus on genitourinary cancer (Initial funding by NCI SBIR) Screening test for differential diagnosis of Prostate, Urinary/Bladder, & Kidney Cancer (PUK) Prostate cancer multivariate diagnostic/prognostic test for post high PSA Bladder cancer post-treatment monitoring 4 Commercialized tests (CLIA/CAP) FFPE Tumor Analysis o GliomaSTRAT : Brain cancer (Glioma, GBM) LDTs o DNA methylation profiles plus glioma-genic mutation

5 Why Epigenetic Biomarkers/ DNA Methylation? 5 6 CH CCCGTCAGATGCGATGAG 3 GGGCAGTCTACGCTACTC DNMT CH I 3 CH I 3 CCCGTCAGATGCGATGAG GGGCAGTCTACGCTACTC C I H3 C I H3 5-H-C 5-Me-C Gene ON Gene OFF 5 Epigenetic changes in DNA methylation regulate genes and dysregulation occurs early in disease o Diagnostics: early detection & screening o Prognostics: disease staging & subtyping o Theranostics: predict treatment response Changes in DNA methylation can be reversed o Good therapeutic targets; FDA approved DNMT inhibitors Methylated DNA is a stable biomarker o Retrospective studies archived samples o Reproducibility with surgical samples

6 Ribomed s Competitive Advantage: CAPS Why have so few DNA methylation based tests been translated to LDTs? It has been a technology problem. Methylated dc is not directly detectable by PCR assays based on sequence differences because the CH 3 (Me) group is not replicated and is lost after the 1st PCR round. Current detection methods (bisulfite MS-PCR, MSRE-PCR) are good for biomarker discovery but have translated poorly to the clinical lab because of sensitivity and target size issues. 573 FFPE tumor blocks selected for bisulfite analysis 266 (46%) Rejected: too little DNA for bisulfite 307 analyzed by MS- PCR 101 (33%) Gave no results 206 produced results (36% success) 6 Ribomed s solution: CAPS - Based MethylMeter o times more sensitive than qpcr based detection o Uses less patient DNA than either other methods o Sensitivity compatible with liquid biopsies o Low failure rate o Works with saliva, urine, plasma & FFPE tissues o Can detect short, degraded DNA ( 40 bp) often found in urine, blood and FFPE samples

7 Current Standard for Prostate Cancer Diagnosis & Grading TRUS Better tests needed here Before the biopsy (Transrectal Ultrasound Guided 12 Core Biopsy) 43.6 M At Risk in US (age 50-74) 20 M PSA Tests 23% 4.6 M Abnormal (PSA >4.0 ng/ml, biopsy recommended)? Molecular Testing to confirm abnormal PSA 28% 1.3 M TRUS Biopsies 18.5% 241,000 positive for PCa? Molecular Testing for Aggressiveness Treatment Invasive: 12 core biopsies - less than 1% probed Poor Sensitivity: >25% False Negative Rate (FNR) High Repeat Rate (to verify negative): 43% undergo a 2nd biopsy 7

8 Prostate Cancer Diagnostic Testing Competition Current tests each detect only 1 type of biomarker and are limited by the detection technology used. CAPS tests detect multiple biomarker types, both genetic and epigenetic. A urine based multivariate biomarker test (best of each type) including additional diagnostic & prognostic biomarkers BEFORE the 1 st biopsy would: Company Test Method Sample type Protein RNA DNA Screening PSA Ab 1 FDA $25 Diagnostic + Urine $500 RIBOMED CAPS TBD Prognostic Serum target Secondary screening Post Abnormal PSA Negative 1st Biopsy- Decision for 2nd biopsy Positive 1st Biopsy - Prognostic Tests Beckman Coulter Prostate Health Index (Phi) Decrease unnecessary initial and repeat biopsies performed (1 M annually) Provide information on aggressiveness before biopsy guide treatment Me- DNA Status Price Ab Serum 3 FDA $499 Opko 4KScore Ab Serum 4 LDT $395 Hologic MDx Heath Genomic Health Progensa PCA3 Score ConfirmMDx Prostate TMA, HPA Bisulfite MS-PCR Urine 2 FDA $385 Tissue 3 LDT $3,300 OncotypeDx RT-PCR Tissue LDT $3,800 Myriad Prolaris RT-PCR Tissue LDT $3,600 Rosetta Genomics RosettaGx Prostate Biomarker Target(s) FISH Tissue 2 LDT

9 9 The Markets & Commercialization Strategies RUO reagents & kits o Global RUO market for epigenetic products $1.3 B o MethylMagnet distributed OEM by Millipore/Sigma o MethylMeter paper published June 2016 Epigenomics o Direct sales & adding new distributors Current Brain Cancer LDTs - GliomaSTRAT o Offered in Ribomed s CLIA/CAP lab o TAM US = $20M; Probable best market = $5M o New WHO guidelines (6/16) should increase test demand o Add additional Pharma clinical trial customers o Marketing at Society of Neuro Oncologists Nov 2016 Proprietary Technologies (Abscription, CAPS) o Out-license to other CLIA labs Royalty model Liquid Biopsy Tests o Potential US market for pre-biopsy urine test at $500 = $2.3 B o Develop as RUO test o Validate & launch initially as LDT o Future funding for CE/IVD approval

10 Capital Requirements & Milestones Offering: $6 M convertible Series A Preferred stock $2.4 M invested $ 3.6 M remains Activity/Milestone Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 CLIA Lab Complete 1st LDT (MGMT) CLIA Certification Begin patient sample testing CAP Accreditation Expand GliomaSTRAT Marketing GliomaSTRAT Epigenetic RUO Products Scale up Manufacturing/QC Secure additional distributors Establish ecommerce website Develop Multiplexed Assay Liquid Biopsy Tests - PUK Prostate Cancer Detection Biomarker Identification Assay Validation LDT Launch Assay Development RUO Launch Bladder Cancer Detection Biomarker Identification Validation Assay Development RUO Kidney Cancer Detection Biomarker Identification Development

11 11 The Ribomed Team Michelle M. Hanna, Ph.D. Founder, CEO and Scientific Director Founder & CEO of Ribomed. Inventor of Abscription. Associate Member, U of A Cancer Center; Ph.D. Chemistry, UC Davis; Postdoctoral fellow Biochemistry, Berkeley; Former Adjunct Professor of Chemistry, Arizona State University; Associate Professor of Biochemistry; Assistant Professor of Medical Biochemistry, University of California Irvine College of Medicine; American Cancer Society & Beckman Foundation Young Investigator Awards; Principle Investigator on over $10 M in grants & contracts. Author of multiple articles & issued or pending patents. David McCarthy, Ph.D. - Chief Science Officer Ph.D. Molecular Biology, University of Arizona Health Sciences Center; Postdoctoral training in Molecular Biology, Brookhaven National Laboratory; Former Associate Professor of Microbiology, University of Oklahoma. Co-inventor of DNA methylation technologies & products. Author of multiple articles & issued or pending patents. R. Harper Summers, M.D. Medical Director Medical Director, Ribomed Clinical Services Laboratory; Laboratory Director & Staff Pathologist, Alvarado Hospital Medical Center, San Diego; American Board of Pathology Certification in Anatomic & Clinical Pathology Philip Cotter, Ph.D., FACMG, FFSc(RCPA) Board of Directors PhD. Human Genetics, Mt Sinai School of Medicine. Assoc. Clinical Professor of Pediatrics, UCSF; Principal, ResearchDx; Director Pacific Diagnostics Clinical Lab; Former: Lab Director Biocept, Director Molecular Cytogenetics, Mount Sinai School of Medicine & Director of Advanced Molecular Diagnostics, US Labs Max Gottesman, M.D., Ph.D. Board of Directors and Chair Scientific Advisory Board M.D., Ph. D. Pharmacology, Yale; Director Institute of Cancer Research & Revson Professor of Biochemistry & Molecular Biophysics, College of Physicians & Surgeons, Columbia University; Editor, J. Molecular Biology. David L. Alberts, M.D. Board of Directors, Scientific Advisory Board Director Emeritus, University of AZ Cancer Center; Regents Professor of Medicine, Author 700 articles, senior editor 8 medical texts, 20 patents involvement in 5 spin-off companies.