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Ray Takigiku, PhD - Chief Executive Officer, Bexion Pharmaceuticals Timothy J. Schroeder - Chief Executive Officer, CTI Clinical Trial & Consulting Services May 6, 2016 Regionalism in Life Sciences
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Developing Cures for Cancer Bexion Pharmaceuticals Series A investment Bexion wins partnership with Nanotechnology Characterization Lab of NCI (NCL), and Phase II SBIR for GBM Phase II SBIR Bridge Award for GBM 20092010201120122013201420152016 Phase I SBIR grants for: Prostate Cancer Pancreatic Cancer Glioblastoma Multiforme (GBM) Pre-IND Meeting with FDA Tibbet’s Award FDA granted Orphan Drug Status for GBM IND and Phase 1 clinical trial The Bexion Story 2 Activity across multiple solid tumor types, including pancreatic cancer and GBM
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Developing Cures for Cancer Bexion Pharmaceuticals BXQ-350 (“SapC-DOPS”) + 1,2-Dioleoyl-sn-Glycero-3-Phospho- L-Serine (Sodium Salt) (DOPS) Lipid (aminophospholipid) C 18 BXQ-350 Protein/Lipid nanovesicle Vesicle diameter, ca. 60 nm = Protein Sphingolipid Activator Protein, Saposin C (SapC) 5,300+ vials of Phase 1 material ready to enter clinic
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Developing Cures for Cancer Bexion Pharmaceuticals YearQuarterActivity 2015Q4 2016Q1Complete 2 nd GLP rat study Q2Complete IND Q3Initiate Phase 1a – PIs for 4 sites are awaiting IND Q4Initiate 2 nd GMP campaign 2017Initiate Phase 1b MTD Activities 2015 through 2017 Phase 1a Phase 1b
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Developing Cures for Cancer Bexion Pharmaceuticals Targets unique and ubiquitous tumor target (phosphatidylserine) Bypasses blood-brain barrier to reach GBM Preclinical efficacy with a broad range of tumors Market potential $3-4B Clinical trials starting 2016 Seeking Series B of $25M SUMMARY
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6 CTI History 17 year old privately held multi-national company providing services to 18 of the top 20 pharmaceutical and biotechnology companies 95% which is higher than the industry average 95% employee retention rate A 17 year track record of steady growth by leading more than 2,700 successful, innovative and cost-effective projects Currently working on behalf of more than 120 sponsors more than 100 approvals Successfully been a part of
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7 100+ Drug Approvals Ambulatory Monitoring Cardiac Arrhythmia COPD Depression Diabetes Type I & II Excessive Sleepiness Fibromyalgia and RLS GvHD H1N1 and H5N1 Influenza Hematology Hepatitis C Hypertension Insomnia Major Depressive Disorder Meningococcal Meningitis Migraine Migraine and Pain Next Day Sleepiness OA Pain Oncology Pain Management/Relief Postherpetic Neuralgia and Restless Leg Syndrome Psychiatrics RA Rare Diseases Restless Leg Syndrome Shingles & HPV Short Bowel Syndrome Sleep - Wake Disorder Sleep Aid Submental Fat Toxic plasma Methotrexate concentrations Transplantation Weight Management Wound Management Indications include:
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8 2015 Drug Approvals Asthma Binge Eating Disorder Hypoactive Sexual Desire Glycemic Control LAL Deficiency/ MPS III Lymphangiol- eiomyoma-tosis – 1 st approved treatment Major Depressive Disorder Oncology – 1 st biosimilar approved in US Oncology/ Drug Toxicity- 1 st /only antidote for emergency treatment Osteoarthritis pain Submental fullness Type II Diabetes Weight Management 2015 Drug Approval Therapeutic Areas CTI was a part of 16 approvals in 2015 alone, comprising nearly 20% of the FDA and EMA approvals for the year
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9 CTI Continued Success
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10 Development & Strategy Lifecycle Support Health Outcomes Clinical Services Clinical Research Center CTI Service Offerings Regulatory Strategy Clinical Trial Planning & Design Protocol & Briefing Documents eCTD Submissions KOL/Site Identification Contracts & Budgets Regulatory Affairs Biostatistics Clinical Monitoring Data Management Trial Management Medical Monitoring Quality Assurance Global Drug Safety Phase I-IV 2 locations 60-bed inpatient unit located on hospital campus > 750 studies completed Late Phase & Expanded Access Programs Training/Medical Education Market Assessment Advisory Board Systematic Literature Review & Meta-Analysis Large Database Analytics Simulation Modeling Data Visualization
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11 Emerging Companies Early Stage Obstacles
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Developing Cures for Cancer Bexion Pharmaceuticals Drug R&D Target ID & Validation IND Pre-clinical Clinical Phase II-IV Sales & Marketing Basic Research Library Synthesis Assay Development HTS Basic Research Custom Synthesis Efficacy/Tox/ADME Screens In vivo Efficacy Library Synthesis API Preparation Formulation, Analytical, CMC Safety & Genetic Toxicology Bioanalytical, metabolism Pharmacokinetics & ADME IND Preparation and Submission Clinical Trial Material Manufacturing Cell Line Generation and Banking Fermentation University PIs CRO Drug Company Medical Centers Go it alone or License Partner Drug Discovery Clinical Phase I VALLEY OF DEATH University PIs VALLEY OF DEATH CRO Drug Company Medical Centers Go it alone or License Partner
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BioOhio represents nearly 400 members that employ over 100,000 Ohioans – from Ohio’s largest employers to emerging start-ups, schools & universities, research institutions, students, and individuals. BioOhio is the lead organization for the bioscience community and the Ohio affiliate for global bioscience associations AdvaMed, BIO, MDMA and PhRMA. With a focus on using advocacy as a tool to create a more favorable business environment, The Kentucky Life Sciences Council (KLSC) works with industry and government leaders to promote effective legislation on behalf of the industry. BioCrossroads advances Indiana’s signature strengths in the life sciences by connecting with corporate, academic and philanthropic partners; facilitating investments in promising start ups and building new enterprises; and educating through conferences, reports and market development knowledge.
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Developing Cures for Cancer Bexion Pharmaceuticals Indiana AIT Ohio Cinti Children’s Hospital University of Cincinnati The Ohio State University CTI Ricerca Kentucky University of KY Coldstream Labs Wyse Innovations bioLOGIC LBR SBIR Matching Funds (KSTC) Private Investors KY as Investor Tri-Ed Other Arizona New Jersey India Global Private Investors Cultivating a Strong Bioscience and Innovation Ecosystem: The Bexion Story
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