Pfizer Program in DMD. Beth Belluscio, MD-Ph.D. Pfizer Rare Disease September 9, 2017

Transcription

1 Pfizer Program in DMD Beth Belluscio, MD-Ph.D. Pfizer Rare Disease September 9, 2017

2 Myostatin Inhibitor for the Potential Treatment of Duchenne Muscular Dystrophy

3 Disclaimer This presentation includes forward-looking statements about, among other things, development of Pfizer s Rare Disease products and product candidates, including domagrozumab, including their potential benefits, that are subject to substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Additional information regarding these factors can be found in Pfizer s Annual Report on Form 10-K for the fiscal year ended December 31, 2016 and in our subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information and Factors that May Affect Future Results, as well as in our subsequent reports on Form 8-K, all of which are filed with the US Securities and Exchange Commission (SEC) and available at and The forward-looking statements in this presentation speak only as of the original date of this presentation, and we undertake no obligation to update or revise any of these statements Property of Pfizer Inc., Not for distribution 2

4 Domagrozumab (PF ): Phase II Development in Duchenne Muscular Dystrophy (#NCT ) Period 1 (48W) Period 2 (48W) Sequence 1 Low dose Medium dose High dose High dose High dose High dose Sequence 2 Low dose Medium dose High dose Placebo Placebo Placebo Sequence 3 Placebo Placebo Placebo Low dose Medium dose High dose 6-<16 year old boys with DMD Ambulant on steroid Monthly 2hr IV infusion Dose escalation N=35 Randomized per sequence (Total 105) Enrollment completed Primary Analysis 4 Stair Climb time NSTAR, 6MWT, Strength MRI and DXA imaging Safety biomarkers External Data Monitoring Committee Secondary Analysis 3

5 Gene Therapy for the Potential Treatment of Duchenne Muscular Dystrophy 1

6 What is Gene Therapy? Therapeutic Gene Therapeutic Protein Modified viral particle Vector Muscular Dystrophy Association Intravenous injection of vectors Therapeutic Gene Muscular Dystrophy Association 1. Certain viral genes are replaced with therapeutic genes to produce a vector. 2. Vectors are injected into the bloodstream where they find their way to specific tissues or organs. 3. The vector delivers the therapeutic gene to a cell where it is used as a blueprint to make the missing protein. 6 Property of Pfizer Inc., Not for distribution

7 Towards a Gene Therapy for Duchenne Muscular Dystrophy Mini-dystrophin delivered by gene therapy is produced throughout the skeletal muscles and hearts of rats and dogs who lack dystrophin Mini-dystrophin expressed for up to 8 years in dog that lacks dystrophin Functional improvements seen in animal models Preclinical studies to select a dose for a human trial are complete Jelly at age 8 still produced minidystrophin in her muscles Orphan Drug status achieved in US and Europe Rare Pediatric Disease designation received from FDA Phase I/II human trial planned within the next 12 months (Based on the pioneering work of Dr. Samulski and Dr. Xiao) 7 Property of Pfizer Inc., Not for distribution

8 Gene Therapy for Duchenne: The Knowns and Unknowns Knowns: Unknowns: Gene therapy cannot be undone once administered Gene therapy eligibility is not determined by having a particular mutation Not everyone will be eligible: Those with pre-existing antibodies to the virus AAV9 will not be eligible, at least initially Will the effect be permanent? Can gene therapy be readministered? Will clinical trials for other types of drugs exclude boys who have previously participated in a gene therapy study? Will the problem of pre-existing antibodies be overcome to make gene therapy an option for more people? 8 Property of Pfizer Inc., Not for distribution