Rebiotix: Business, Regulatory and Clinical Challenges in Commercializing a Microbiota Based Drug. May 2016

Transcription

1 Rebiotix: Business, Regulatory and Clinical Challenges in Commercializing a Microbiota Based Drug May 2016

2 Rebiotix Pioneer in Microbiome Therapeutics Privately held clinical stage biotechnology company developing a new class of biologic drugs for the treatment of debilitating diseases. Our product platform, Microbiota Restoration Therapy (MRT), is based on live human derived microbes. 2

3 Rebiotix Development Highlights First company to take live microbiota based drug through the FDA Lead product RBX2660 has FDA Fast Track, Orphan Drug, and Breakthrough Therapy designation Demonstrated 87% clinical cure in Phase 2 trial with lead product RBX2660 for recurrent Clostridium difficile Completed interim analysis of Phase 2B multicenter, double-blind, randomized, placebo controlled trial first study of its kind under IND. First standardized, stabilized, patient deliverable drug based on live human-derived microbes. 3

4 Challenging Landscape for MRT Drug Development No precedent in regulatory agencies Tissue transplant or drug? Rebiotix first to file IND No real clinical proof Mostly anecdotal data Lack of safety data No standardized product Lack of manufacturing/quality system predicate No test methods/potency assays No donor screening methods Fresh stool only/no preservation techniques 4

5 US Regulatory Question Asked Tissue transplant or drug? We asked the FDA in You asked the Tissue Reference Group (TRG) to recommend whether this product meets the criteria for a human cell, tissue, or cellular or tissue based product (HCT/P) regulated solely under 21 CFR part 1271 and section 361 of the Public Health Service Act. 5

6 US Regulatory Question Answered The FDA answered: Drug, not tissue transplant We recommend that your GI microbiota product does not consist of human cells or tissues and therefore does not meet the definition of an HCT/P regulated under section 361 of the PHS Act. This product appears to be the type of product regulated by CBER s Office of Vaccine Research and Review (OVVR). 6

7 Why Did That Matter? Tissue products can be sold immediately in US Drug products require: clinical trial program strict quality system Lots of $$$$$ 7

8 New Drug Commercialization Strategy: RBX2660 for Recurrent CDI Strategy: Comprehensive clinical trial plan to produce medical based scientific evidence (no hype) Product Objective: Readily available standardized, quality controlled, FDA approved product Lead Product: RBX2660 Broad spectrum microbial composition Cryopreserved Enhanced donor screening Simple off-the-shelf enema delivery system No patient anesthesia or bowel prep* 8 *Currently only product under IND investigation in US without bowel prep

9 FDA Caused Havoc First, in May 2013, FDA announced fecal microbiota transplants (FMT) were to be regulated as a drug Needed an Investigational New Drug (IND) application to use Then, FDA issued a guidance document regarding FMT: Enforcement discretion for FMT for recurrent C diff 9

10 Now Everyone is Confused Companies began and continue to sell unauthorized FMT drug products Patients demanded this new treatment with no clinical proof - Putting pressure on doctors FDA issued a new guidance to clarify the old one but took no other action Clinical trials became difficult to conduct Enrollment rate went down by 2/3 because of stool banks $$$$ went up 10

11 11 Hype: Fecal Transplant Cures All

12 12 Rebiotix Answered the Challenges for MRT Drug Development Established regulatory precedent Rebiotix first to file IND Generated real clinical proof Treated >250 pts in rigorous clinical trials Generated safety and efficacy data Shared results through publications Standardized product Implemented manufacturing/quality system Developed and validated test methods/potency assays Created robust donor screening methods Standardized, stabilized, patient deliverable product

13 May 2013 Nov 2013 Fast Track status granted Continued Rebiotix Regulatory Progress Mar 2014 Dec 2014 Orphan Drug designation granted Phase 2 clinical enrollment completed Health Canada clinical No Objections letter Oct 2015 Phase 2B enrollment completed Oct 2015 Breakthrough Therapy Designation granted RBX2660 Regulatory Milestones 13

14 Robust Clinical Program PUNCH CD PUNCH CD 2 PUNCH CD 3 Multi-center, open-label; min 30 pts followed for 6 months Primary Objective: Assess the safety of RBX2660 Secondary Objective: Efficacy, no CDI recurrence at 8 wks. Enrollment completed Nov 2013 Multi-center, randomized, double-blind, placebo controlled; 133 pts followed for 24 months Primary Objective: Efficacy of RBX2660 Secondary Objective: Safety of RBX2660 Enrollment completed Oct 2015 Confirmatory Trial Multi-center, prospective efficacy and safety study Randomized, controlled Supportive for BLA application 14

15 Manufacturing: Consistent, Controlled, Scalable Robust Donor Program FDA supported safety screening 3yr successful donor recruitment commercially scalable Multiple doses per donation Proven Drug Manufacturing Produces consistent 150 ml doses of cryopreserved RBX2660 Doses packaged in simple, ready to ship enema delivery system Long term inventory shelf life/attractive cost of goods Chemistry/Mfg/Controls BLA readiness assessment conducted by outside group Validated analytical methods and manufacturing processes GMP quality system implemented 15

16 16 Rebiotix is Committed Rebiotix supports FDA new drug regulations and will continue to pursue FDA BLA approval Products are early with innovations to come Safety and efficacy are a promise to the patient; good oversite and practices will lead to safety and efficacy Systematic collection and publication of data will build a strong foundation for this new industry We are heavily invested based on early FDA decisions 16

17 Rebiotix is Harnessing the Power of the Gut Microbiome Pioneering new microbiota based drugs to solve urgent unmet medical needs Providing wide access to potentially life saving drugs through FDA approval process Changing the therapeutic landscape 17 Rebiotix Facility

18 Thank you Additional information, contact: Lee Jones