39th Annual Cowen Healthcare Conference March 12, 2019 Exhibit 99.1
These slides and the accompanying oral presentation contain forward-looking statements and information. The use of words such as “may,” “might,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward-looking statements. For example, all statements we make regarding the initiation, timing, progress and results of our preclinical and clinical studies and our research and development programs, our ability to advance product candidates into, and successfully complete, clinical studies, and the timing or likelihood of regulatory filings and approvals are forward looking. All forward-looking statements are based on estimates and assumptions by our management that, although we believe to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that we expected. These statements are also subject to a number of material risks and uncertainties that are described in our most recent quarterly report on Form 10-Q, as well as our subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. Forward-Looking Statements
RADICAL CARE We care in a way that’s intense and truly sets us apart. WE RECODE FOR LIFE THIS IS PERSONAL Gene therapy is about saving lives one person at a time. And we are, each of us, personally all in. PIONEERS WITH PURPOSE We’re exploring new frontiers for the sake of patients.
T H E G E N E T H E R A P Y P R O D U C T S C O M P A N Y LentiGlobin SCD 2022 Potential Filing/Approval LentiGlobin TDT 2019 EU Potential Approval 2020 U.S. Potential Approval Lenti-D CALD 2021 Potential Approval bb2121 Multiple Myeloma 2020 Potential Approval Products on the Market 4 INDs Per Year Beginning 2020 1-2 Clinical Programs 5+ Patient Impact ∞ Our 2022 Vision -- Just Got BOLDER
SCD: Potential Global LAUNCH MM: Earlier Line APPROVALS & LAUNCHES 2023+ SCD: U.S. & EU FILING/APPROVALS CALD: Global EXPANSION MM: Earlier Line Studies 2022 TDT: Label EXPANSION CALD: U.S./EU APPROVAL MM: Global EXPANSION in Relapsed/Refractory Earlier Line Studies 2021 TDT: EU Country EXPANSION U.S. APPROVAL & LAUNCH Label EXPANSION MM: APPROVAL in Relapsed/Refractory 2020 TDT: EU APPROVAL & 1st LAUNCH 2019 UNPRECEDENTED OPPORTUNITY Anticipated research, development, regulatory and commercial milestones 1-2 INDs 1-2 INDs 1-2 INDs 1-2 Clinical Starts 5+ New Clinical Programs
RECODE THE SCIENCE: R&D with SOUL WHAT YOU SEE WHAT YOU DON’T SEE Internal Research Tools & Technology P13K Inhibition Enhanced Mftg TGF-B Flip CBL-B Edit DARIC Controls GAMMA DELTA Partners for Success T Cell megaTALs PRODUCT CANDIDATES PROGRAM AREA PRECLINICAL PHASE 1/2 PHASE 2/3 RIGHTS/PARTNER Severe Genetic Diseases Lenti-D™ Drug Product Worldwide LentiGlobin™ Drug Product Worldwide BCL11a shRNA (miR)* Worldwide Cancer bb2121 Celgene bb21217 Celgene Multiple Myeloma Third Line** Cerebral Adrenoleukodystrophy Transfusion-Dependent β-Thalassemia β0/β0 Multiple Myeloma Fourth Line Sickle Cell Disease Multiple Myeloma First Line** Multiple Myeloma Second Line** *Development is led by Dana-Farber/Boston Children's Cancer and Blood Disorders Center **Planned studies ** Multiple Myeloma Fourth Line Transfusion-Dependent β-Thalassemia Non-β0/β0 Sickle Cell Disease
RECODING TRADITIONAL R&D 1:Many Target Strategy INTEGRATE ITERATE INTERNAL EXTERNAL Platform Tools & Technology R&D Enablers Clinical Insights MM - bb21217 SCD - BCL11a Liquid Tumor Programs Solid Tumor Programs SGD Programs TDT – LentiGlobin SCD – LentiGlobin MM – bb2121 CALD – Lenti-D Anti-Pure Play Principles - What Do We Mean?
Virus Manufacturing Drug Product SCALABLE Clinical Data Real World Evidence to Support BLUE Style: Field Team Supply Chain Collecting Cells, Preparing, Shipping, Receiving Cells DELIVERY PLATFOM Platform Is Gearing Up for Launch TDT MAKE IT SEAMLESS
Lifetime cost-time effectiveness timeframe Base value only on patient QOL and Life Extension OBJECTIVE STRATEGIC APPROACH Approach – VALUE-BASED PAYMENT Over Time Based on OUTCOME FAIR VALUE RECOGNITION SHARED RISK HEALTH SYSTEM AFFORDABILITY PER PATIENT AFFORDABILITY Pay ONLY IF the treatment works Put UP TO 80% of the price at risk based on success Spread payments over UP TO A FIVE YEAR period NO PRICE INCREASES above CPI NO COST after payment period (vs. for life) 1 2 3 4
Potential 2019 Catalysts LentiGlobin TDT EU Approval & First Launch Potential U.S. Filing Northstar-2 and Northstar-3 Data Update LentiGlobin SCD HGB-210 Study Start HGB-206 Group C Data Update bb2121 MM CRB-401 Data Update* KarMMa-1 Data* LentiGlobin TDT Northstar-2 (HGB-207) & Northstar-3 (HGB-212) Data Update LentiGlobin SCD HGB-206 Group C Data Update bb2121 MM KarMMa-2 & KarMMa-3 Study Start* Pipeline Analyst Day *Driven by Celgene/BMS bb21217 MM CRB-402 Data Update By End of Year By Mid Year Cash Position as of December 31, 2018: $1.9B CASH RUNWAY INTO 2022
