Transformational Products in Soft Tissue Regeneration and Cartilage Repair

Transcription

1 Transformational Products in Soft Tissue Regeneration and Cartilage Repair

2 Company History Founded in 2000 Late stage clinical company Privately held, based in Waltham, MA 42 Employees Acquisition of ProChon BioTech, Ltd., May 2011 Merged cell therapy technology with tissue engineering technology Raised $49M Series A Round, June 2012 Drive flagship technology through Phase III study and secure CE Mark for second lead product Sofinnova Ventures and Split Rock Partners Seasoned Management team with extensive industry experience driving biologics and medical devices through the clinic, commercialization, and acquisition. Internal developments focused on articular cartilage tissue repair and regeneration implants in the Orthopedics/Sports Medicine market P. 2

3 Rich Portfolio of Proprietary Technologies 3-D Scaffolds Growth Factors 1. Type I Collagen 2. Fibrin & hyaluronic acid 3. Nanophase hydroxyapatit FGF-2v, FGF-4v, FGF-9v FGF is a potent angiogenesis growth factor Enhancement of cell proliferation CT3 Methylated collagen and PEG 10X stronger than fibrin-based adhesives No necrosis Strategic Applications: 1. Targeted delivery of cells, growth factors, and drugs to injured tissue 2. Osteoconductive environment for cellular in-growth, attachment, and proliferation Strategic Applications: 1. Therapeutic Applications 2. Reagent/Cell Expansion Applications Strategic Applications: Soft-Tissue adhesive and temporary approximation 91 issued patents and applications P. 3

4 Cartilage Injuries Cartilage is designed to absorb shocks and tension. It allows smooth movement of the joint. 60% of injuries are post-traumatic. Knee femoral condyle injury is most common. Articular cartilage is an avascular tissue structure, incapable of self-repair. As a result, patients may experience symptoms of pain and restricted mobility, which can progress to debilitating osteoarthritis over time. The current gold standard for treating patients is a microfracture procedure

5 Current Solutions Microfracture Gold Standard : Fibrous cartilage tissue regeneration leads to inconsistent long-term results and repeat procedures The Problem Current Microfracture Therapies repairs produce are Inadequate fibrocartilage clinical outcomes reported to deteriorate significantly 18 months post-op 1. Kreuz PC et al, Arthroscopy 22(11), (2006) 2. Frisbie DD et al, Clin Orthop. Relat Res (407), (2003). Mosaicplasty/Osteochondral Grafting: Harvest site morbidity Poor implant integration Deterioration of implant over time Current ACI Technologies: Heterogeneous cell population Mixed fibrous and hyaline cartilage regeneration Unsatisfactory long-term clinical outcomes No major improvement on rehabilitation time Expensive P. 5

6 Cartilage Repair & Regeneration Market U.S. & E.U. 2.4M Cartilage Defects in 2015 $6B Clear Potential Market opportunity Demographic and medical trends Patients years of age, privately insured Very active and strong desire to stay active during prime years Highly educated consumer, driven to avoid bridge burning invasive procedures Aging of the population - Baby boomers desire to stay active, Significant unmet need driving regenerative medicine Only 26% of defects are treated due to unsatisfactory procedures and products P. 6

7 Approximately 41 million children and teens in the U.S. participate in some form of competitive sports Musculoskeletal injuries comprise the largest portion of athletic injuries with 25% of athletes expecting to incur a musculoskeletal injury in any 12-month period. Knee injuries account for 22% of all musculoskeletal injuries. According to the NCAA, one major knee injury occurs per football team every year. Approximately one-third of ski injuries involve the knee. Cartilage Repair An escalating opportunity More than 200 million people play soccer worldwide and up to 40% of soccer injuries involve the knee. The emergence of the Adult Athlete - Approximately 150 million adults participate in some form of competitive sport. NHIS study data: 9.29% of the US population will be diagnosed with symptomatic knee OA by the age of 60. The A.A.O.S.M membership has increased from 209 to 3042 in the past 30 years doubling in the past ten years. Sports Medicine is the fastest growing segment of the ortho market 8%, $4.2B Market P. 7

8 Addressing Large, Unmet Stage in the Treatment Continuum Physical Stretching Exercise Message Accupuncture Injections Corticosteroids Viscosupplementation Hyaluronic acid Invasive Surgery Resurfacing implants Total joint replacement Least Invasive Most Invasive Behavioral Cognitive Pacing Relaxation Meditation Hypnosis Medications Topical NSAIDS Oral Acetaminophen NSAIDS COX-2 inhibitors Opioids Minimally invasive surgery Debridement Microfracture Mosaicplasty Autologous Chondrocyte Implantation

9 Products: NeoCart Autologous Neo-Cartilage Implant Next-generation Cartilage Regeneration System Target market: ICRS stage III/IV defects of the knee Phase II demonstrated superiority to microfracture 4-yr data to be presented at the A.A.O.S conference, yr data presented at the I.C.R.S conference, yr data published in JBJS, 2012 Phase III IND study under Special Protocol Assessment (SPA) Safety: No severe adverse events related to NeoCart in Phase I or Phase II studies. Efficacy: Superiority to microfracture. NeoCart Autologous cartilage regeneration implant Global, debilitating, quality-of-life problem (market) currently treated by unsatisfactory procedures or products Patient's own cartilage infused into a patented 3D scaffold to create a neocartilage implant Tissue contains both cells and true cartilage tissues. Opportunity tomultiplying replace the current gold standard Readily integrates with surrounding healthy cartilage with a first-line therapy Easy to handle, can treat large cartilage defects Suture-less implant o Proprietary bioadhesive microfracture procedure Strong and secure - allows for early weight-bearing and acceleration of rehabilitation process NeoCart cartilage regeneration implantation procedure P. 9

10 Image of NeoCart post-implantation NeoCart Implant at 3 Months Post-implantation Smooth integration of hyaline cartilage implant * Image of Phase I subject who suffered a meniscal tear 2 weeks after the implant that required a second arthroscopy at 3 months; Injury was unrelated to NeoCart implant Hyaline-like fill predicts high likelihood of long-term functional success Efficacy data confirms validity of product attributes

11 Clinical & Regulatory NeoCart Cartilage Regeneration Phase III IND Clinical Study 245 patient multi-center randomized, superiority to microfracture CBER Special Protocol Assessment (SPA) from FDA Significantly De-risked regulatory path Primary Endpoint: The responder rate of the NeoCart arm is compared to the responder rate of the Microfracture procedure arm at 12 months post-implantation. A responder is defined as a subject that shows clinical improvement of at least 20 points in the IKDC score and at least 12 points in the KOOS pain score. Currently enrolling at nation s top orthopedic sports medicine clinical centers. The 25 sites include UCSF, Duke, Cleveland Clinic, Hospital for Special Surgery, UPenn, UConn, OHSU, UPMC, Oschner Clinic, Harvard, and Brigham & Womens P. 11

12 Phase II Data Indicates Confidence in Achieving Phase III Superiority Achieved clinical significance at the 6, 12, and 24 month follow up time points A 54% point NeoCart responder rate difference at the 1-year primary endpoint indicates superiority to Microfracture 100% 80% % Responders 60% Robust study design, clinical superiority and SPA 40% = High MFX 20% degree of confidence in achieving FDA approval NeoCart 0% 6 mos 12 mos 24 mos Time post treatment n = 30 at 6-12 mos, n=27 at 24 mos. P. 12

13 Target market: ICRS stage II/III/IV defects 3cm 2 or less of the knee Augmentation of microfracture procedures to improve clinical outcome Products: VeriCart Single Step Cell-free Cartilage Repair Scaffold Off-the-shelf lyophilized, double structured collagen scaffold, easily cut to lesion size VeriCart Single Step Cartilage Repair Scaffold Suture-less implant Proprietary bioadhesive to secure placement and position of implant Attracts mesenchymal stem cells from surrounding tissue Microscopic Images of Scaffold Structure Strong and secure implant - early weight-bearing and acceleration of rehabilitation process Pursue CE Mark P. 13

14 Patrick O'Donnell President and CEO Histogenics Corporation 830 Winter Street Waltham, MA (781) Thank You