RP-G28: First in Class Rx for Treatment of Lactose Intolerance. March 2013

Transcription

1 RP-G28: First in Class Rx for Treatment of Lactose Intolerance March 2013

2 Value Proposition Specialty pharmaceutical company, focused on GI disease Core science based on colonic adaptation First company to announce the ability to induce change in the colonic microbiome First in class treatment for Lactose Intolerance One time, 30 day course of treatment Ability to expand to other GI indications $1.2 Billion market potential, reimbursement likely Positive phase 2 clinical data Company preparing late stage clinical development 2

3 The Market Opportunity - Lactose intolerance is a widespread condition affecting >15% of the U.S. adult population, 65 million adults in Europe and >90% of Asian population. - There is no prescription drug currently available for LI despite desire and need for a prescription treatment. Lactose Intolerance is a Global Issue (Lactose Intolerant Popula?on in Millions) >70% have Moderate to Severe Symptoms Severe 12% Don't Know 1% US Europe Japan Es:mated 1 Billion+ Lactose Intolerance Sufferers Worldwide Mild 27% Moderate 60% Sources: (1) Objective Insights, Market Research Analysis and Forecasts on Lactose Intolerance and RP-G28. June,

4 Billions $ are Spent on Unsatisfactory Options Dairy Avoidance Probio?cs Percent of Pa?ents Dissa?sfied with Current Physician Recommended LI Management Op?ons 70% 54% 65% 66% 29% 46% Dairy Subs?tutes Lactose- Free Products Lactase Supplements Dairy Avoidance Lactase Supplements Lactose/Dairy- Free Foods Physicians Pa:ent Sa:sfac:on 4

5 RP-G28: Structure and Key Properties > 95% Oligosaccharide Target claim: Treatment for symptoms of lactose intolerance Delivered to patient in sachet 30 day course treatment Maintenance product for subset of patients 5

6 Clinical Results: Key Outcomes 1) Drug was well tolerated, no SAEs reported 2) Demonstrated Mechanism of Action Positive changes in the microbiome of the digestive tact Increase in complexity & number of specific bacterial species Increased lactose metabolism 3) Indicated clinically meaningful benefit to patients Treated patients 6X more likely to be Lactose Tolerant (p=0.039) Durable reduction in abdominal pain day 36 & 66 (p=0.023) Abdominal pain reduction similar in magnitude to published IBS studies (p=.028) 6

7 Microbiome Only Shifts in Subjects Treated with RP-G28 Absolute OTU Counts* RP- G28 (n=35) Placebo (n=17) Day 0 Day 36 Day 66 OTU = Operational Transcription Unit by TRFLP analysis identifies bacteria Pyrosequencing: 82% of patients taking RP-G28 showed a shift in microbiome Abundance p=0.031 Diversity p=0.003 Fecal sample analysis performed in labs of: Todd R. Klaenhammer, Departments of Food, Bioprocessing and Nutrition Sciences, North Carolina State University M. Andrea Azcarate-Peril, Ph.D., Director, Microbiome Core Facility, University of North Carolina 7

8 Demonstrated Clinically Meaningful Benefit to Patients Reported tolerance to dairy foods Substan:al reduc:on in abdominal pain Abdominal pain defined as pa:ents that reported NO pain at both Day 36 & Day 66 :me points Percent of Subjects Global Assessment Placebo (n=18), RP-G28 (n=40) 70% 60% 50% 40% 30% 20% 10% P=0.039 Placebo No Abdominal Pain Placebo (n=19), RP-G28 (n=36) RP- G28 P= % Lactose Tolerant Day 66 No Pain Day 36 & 66 8

9 Development Plan: NDA 2016 Q2 13 Q3 13 Q4 13 Q1 14 Q2 14 Q3 14 Q4 14 Q1 15 Q2 15 Q3 15 Q4 15 Q1 16 Q2 16 Q3 16 Q4 16 Phase 3 Study Phase 2b EOP2 NDA Open Label Ext Phase 3 Study Open Label Extension Pediatric Toxicology Registration Batches 9

10 Intellectual Property Multiple PCT Applications 12/707,037, 13/096,711, 13/198,497 Methods for using RP-G28 to treat lactose intolerance & other GI diseases (received notice of allowance for key claim) IP excludes low GOS products from being promoted for treatment of lactose intolerance Claims cover broad range of oligosaccharides and purities ranges Specific formulations, regimens, product profile & application use Expiring 2029 in U.S. & R.O.W. Manufacturing Exclusive rights to PCT application for manufacturing process of high purity API IP Legal Counsel: Vern Norviel at Wilson Sonsini Goodrich & Rosati 10

11 Medical Advisory Board Dennis Savaiano, Ph.D. (Chair) Professor of Foods & Nutrition & Associate Provost at Purdue University. Has conducted over 30 years of research on lactose intolerance. Considered one of foremost LI experts in the world. W. Allan Walker, M.D. Director of the Division of Nutrition at Harvard Medical School Todd Klaenhammer, Ph.D. Byron L. Cryer, M.D. Warren Grundfest, M.D., FACS Harry Greene, M.D. Roger Clemens, Ph.D. John Sherman, M.D. Chris Landon, M.D. Director of the Southeast Dairy Foods Research Center & Professor of Food Science, Microbiology & Genetics at North Carolina State University. National Academy of Science Member. Professor of Medicine in Digestive & Liver Diseases and Associate Dean at the University of Texas Southwestern Medical Center at Dallas & the North Texas VA Health Care System Professor at University of California, Los Angeles Former Chief, Division of Pediatric Gastroenterology /Nutrition and Director of the NIH sponsored Clinical Nutrition Research Unit. Associate Director of the Regulatory Science Program at University of Southern California s School of Pharmacy Internal Medicine at Cedars Sinai Medical Center Director of the Landon Research Institute, Ventura CA 11

12 Leadership: Over 15 Years of LI Research Ira E. Ritter Chairman Andrew J. Ritter President & Chief Executive Officer Larry Good, M.D. Chief Medical Officer Bryan Jones, Ph.D. Chief Business Officer Howard Foyt, M.D., Ph.D. FACP Clinical Development Alene Campbell, MBA Operations & Corporate Development Randy Whitcomb, M.D. Clinical & Strategic Advisor Formerly President & Vice-Chairman of Quality King 40+ years serving on Executive Boards; Rockwood, Oak Media, RG Publishing Inventor of RP-G years of research in Lactose Intolerance Practicing board certified gastroenterologist for over 30 years Formerly served as the Chief Physician in Gastroenterology at South Nassau Communities Hospital and Lydia E. Hall Hospital in New York Over 25 years of BD & drug development experience ICOS, BMS, Amylin, Mt Cook, Sherrington Ph.D. in Genetics, University of Washington Over 19 years of key clinical development roles at Pfizer & Parke-Davis Former VP, Clinical Development & Chief Medical Officer at Metabasis Therapeutics Over 30 years of healthcare industry experience at Executive levels. Former CEO & COO of MicroPhage, VP Corp Dev. at Myogen/ Gilead, VP RPI-SiRNA / Merck Founder QuatRx Pharmaceuticals VP Clinical Research Parke-Davis Former faculty member at Harvard Medical School 12

13 RP-G28: Summary First in class treatment for lactose intolerance Large and growing global market Estimated U.S. sales of $1.2B Successfully completed Phase 2 trial Meeting with FDA Potential for GI indications beyond lactose intolerance IBS, IBD and Metabolic Syndrome Strong intellectual property portfolio NDA filing in 2016 Ritter seeks funding for clinical program and/or partnership opportunities 13

14 Thank You Ritter Pharmaceuticals, Inc.