INFUSE Bone Graft Oral Maxillofacial Bone Grafting Procedures



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INFUSE Bone Graft Oral Maxillofacial Bone Grafting Procedures Dental Products Advisory Panel Committee Ed Chin, DPh Group Director, Regulatory Affairs Medtronic Spinal and Biologics 1

INFUSE Bone Graft FDA Panel Presentations Agenda Speaker Welcome & Introduction Clinical Need Clinical Evidence Sinus Augmentation Clinical Evidence Extraction Socket and Overall Safety Conclusions Ed Chin, DPh Robert Marx, DDS Robert Marx, DDS David Cochran, DDS, PhD Ed Chin, DPh 2

Additional Resources Leon Assael, DMD Philip Boyne, DMD, MS, DSc James T. Mellonig, DDS, MS Myron Nevins, DDS Daniel B. Spagnoli, DDS, MS, PhD R. Gilbert Triplett, DDS, PhD Pirkka Nummikoski, DDS Steve Cook, PhD Hal Mathews, MD Mildred Christian, PhD Scott Kern, MD Douglas Hawkins, PhD Barbara Boyan, PhD Jeffrey Toth, PhD Medtronic Staff Wyeth Staff Alquest Staff 3

BMP History 1965 Urist discovers demineralized bone induces new bone 1971 Urist coins the term BMP 1977 BMP extracted from bone is inductive 1988 First recombinant human BMP produced (Wozney) BMP is destined to bring osteogenesis under the control of surgeons Urist MR, J NIH Res,1997 4

Recombinant rhbmp-2 Manufacture Cell Culture Purification Formulation Thaw Gene 5

INFUSE Bone Graft (rhbmp-2/acs) recombinant human Bone Morphogenetic Protein 2 (rhbmp-2) 4.2 or 12 mg vials 1.5 mg/ml concentration Absorbable Collagen Sponge (ACS) Carrier for rhbmp-2 Type I bovine collagen sponge 1 X 2 or 3 X 4 size Over 20 years of clinical use as Helistat Same product used in OMF pivotal study 6

INFUSE Bone Graft (rhbmp-2/acs) Two PMAs proved safety and efficacy: 2002: Single level spinal fusion procedures 2004: Open tibia fractures 437 patients received rhbmp-2/acs in IDE clinical trials which supported these PMAs In addition, over 1,200 patients received INFUSE Bone Graft or rhbmp-2 with other carriers in clinical studies 7

Overall Program Aim INFUSE Bone Graft Bone Growth Patient Therapeutic Benefit Spine Fusion: 2002 Tooth Replacement Before After Acute Tibia Fracture: 2004 Before After Before After 8

Clinical Oral Indications INFUSE Bone Graft Dental Implants Tooth Replacement Models Examined Sinus Augmentation IDE Extraction Socket IDE 3 Studies 2 Studies 9

BMP Scientific Research 6000 5000 4000 3000 2000 1000 5000 Cumulative Published Literature 31 Regulated rhbmp-2 Clinical Studies 35 30 25 20 15 10 5 0 1975 1977 1979 1981 1983 1985 1987 1989 1991 1993 1995 1997 1999 2001 2003 2005 0 10

Oral Maxillofacial IDE Clinical and Pre-Clinical Evidence Human Experience 5 IDE Studies Levels of Proof Non-human Primate Canine Caprine Feline Lapine 8 Studies 16 Studies 2 Studies 1 Study 1 Study General Safety 32 Studies 11

Proposed Indication For Use Statement "INFUSE Bone Graft is indicated as an alternative to autogenous bone graft for sinus augmentations and localized alveolar ridge augmentations for defects associated with extraction sockets." 12

INFUSE Bone Graft Program Objectives Regenerates bone in these oral indications Supports dental implant placement Supports long term functional loading 13

The AAOMS Journal Editorial Board 2005 Daniel M. Laskin Award 14

The American Academy of Periodontology Foundation 2005 Tarrson Research Award in Oral Plastic Surgery 15

Robert E. Marx, DDS Professor of Surgery Chief, Oral and Maxillofacial Surgery University of Miami Miller School of Medicine 16

Clinical Need for Maxillofacial Bone Grafting Regenerate bone lost due to disease, trauma or developmental defects: Provide bone support to replace missing teeth Restore structure and function Improve patient s appearance (esthetics and self image) 17

Maxilla Mandible

Current Standard - Autogenous Bone Graft Advantages Patient s own bone Proven effectiveness Disadvantages Donor site morbidity Extended surgical and anesthesia time Limited availability 19

Bone Graft Harvest Morbidity Hematoma Edema Erythema Exudate Blood Loss = 232 cc Hematoma = 3% Sensory loss = 8% (5% permanent) Chronic pain = 2% Infection Wound Dehiscence Blood loss Sensitive and painful scar 20

Oral Maxillofacial Human Clinical Trial Objectives Demonstrate that rhbmp-2/acs regenerates or grows normal physiologic bone provides bone for dental restorations or placement of dental implants produces stable bone under functional loading To demonstrate safety in oral maxillofacial indications 21

Clinical Models Sinus Augmentation Extraction Socket 22

Sinus Augmentation Studies Methods and Definition Prospective, multi-center (21), controlled human clinical trials with high level of evidence Pilot: 0.43 mg/ml Dosing (randomized): 0.75 and 1.50 mg/ml Pivotal (randomized): 1.5 mg/ml Pooled Data - Dosing and Pivotal Studies (1.5 mg/ml) Efficacy Endpoint Implant-borne restoration after 6 months of functional loading Target Success Rate - Greater than 73% Safety Endpoints 23

Effectiveness Sinus Augmentation Studies Primary Objectives Induce bone to successfully support implant-borne restoration after 6 months of functional loading Safety Evaluate the safety of rhbmp-2/acs and autogenous bone graft in two-stage maxillary sinus floor augmentation procedures 24

Sinus Augmentation Studies Secondary Objectives Evaluate new bone radiographically Evaluate histology of the new bone Functional loading: INFUSE Bone Graft compared to autogenous bone graft 25

Sinus Augmentation Studies Baseline rhbmp-2/acs Graft Bone Formation Dental Implant and Osseointegration Functional Loading T=0 4-6 mos 6-12 mos 12-24 mos 24-36 mos CT Scan CT Scan Biopsy CT Scan Periapical X-ray Periapical X-ray Safety Evaluation Throughout the Study 26

Study Design Schematic Sinus Augmentation Dosing Study N=48 Acute Safety Established Cohort 2 --1.5 1.5 mg/ml RANDOMIZATION Cohort 1 - Cohort 1-0.75 mg/ml Cohort 1-0.75 mg/ml RANDOMIZATION rhbmp-2/acs (16 patients) Bone Graft (8 patients) rhbmp-2/acs (16 patients) Bone Graft (8 patients) 27

Study Design Schematic Sinus Augmentation Pivotal Study N=160 Cohort --1.5 1.5 mg/ml RANDOMIZATION rhbmp-2/acs (80 patients) Cohort 1-0.75 mg/ml Bone Graft (80 patients) 28

Prospective, Controlled Sinus Augmentation IDE Studies (N=220) Sinus Augmentation N Pivotal Study rhbmp-2/acs (1.5 mg/ml) 82 Bone Graft 78 Dosing Study rhbmp-2/acs (1.5 mg/ml) 17 Bone Graft 13 rhbmp-2/acs (0.75 mg/ml) 18 rhbmp-2/acs Effectiveness N= 99 Pilot Study rhbmp-2/acs (0.43 mg/ml) 12 29

CT Scan of de novo Bone Induced by rhbmp-2/acs Pre-op 16-weeks Post rhbmp-2/acs Placement 30

CT Scan of de novo Bone Pre-Op (3.9 mm Baseline) 6 months Post-Op (16.0 mm) 31

Sinus Augmentation Bone Growth Treated with rhbmp-2/acs 1.5mg/ml (N=98) Bone Height Change by Subject mean ± SD = 8.2 ± 3.8 mm Change in Bone Height (mm) 0 5 10 15 32

Primary Objective INFUSE Bone Graft Functional Loading Success Time Period Dental implants without augmentation Received prosthesis (functionally loaded) 6 months Sinus Augmentation Studies 82.8% (82/99) 79.8% (79/99) 79.6%* (78/98) *Target Success Rate 73% 33

Secondary Objective Functional Loading by Patient 100% 90% 80% 70% 60% 50% 40% 30% 20% 10% 0% Bone Graft rhbmp-2/acs (1.50 mg/ml) DIP w/o addt l augmentation Received prosthesis 6 months 12 months 18 months 24 months P Value 0.0683 0.1117 0.0667 0.0286 34

Secondary Objective Functional Loading by Implant 100% 90% 80% 70% 60% 50% 40% 30% Bone Graft rhbmp-2/acs (1.50 mg/ml) 20% 10% 0% Received prosthesis 6 months 12 months 18 months 24 months P Value 0.8218 0.6645 0.4579 0.3643 0.2112 35

Bone Density from CT Scan Sinus Augmentation Dosing Study Density at 4 months Post Grafting Density at 4 months Post Grafting Density at 6 months Post Prosthesis Density at 6 months Post Prosthesis Density (mg/cc) 0 100 200 300 400 500 Density (mg/cc) 0 100 200 300 400 500 Bone Graft 1.50 mg/ml rhbmp-2/acs Bone Graft 1.50 mg/ml rhbmp-2/acs Bone Graft 1.50 mg/ml rhbmp-2/acs Bone Graft 1.50 mg/ml rhbmp-2/acs 36

Sinus Augmentation Histology Demonstrates Normal Physiologic Bone rhbmp-2/acs Induced bone Longitudinal section core biopsy Samples taken at time of dental implant placement (6-12 months) 10x Normal Trabecular Bone Native Bone Residual Bone Graft fragments were included in the quantitative histomorphometric measurements 37

Bone Graft Sinus Augmentation Studies Bone Quality Supports Dental Implant Similar Trabecular Volume and Thickness Both with 90-95% lamellar bone with small amounts of immature bone Residual lamellar bone fragments in Bone Graft group may have accounted for statistical differences Differences did not affect clinical outcomes INFUSE Bone Graft 38

Sinus Augmentation De Novo Bone Formation Bone Graft rhbmp-2/acs Labeling Yellow - 10 days post-op (tetracycline hydrochloride) Orange - 3 months post-op (democlocycline) rhbmp-2/acs demonstrated substantial, rapid de novo bone formation Bone Graft group demonstrated variable de novo bone 39

Histology Summary autogenous bone and rhbmp-2/acs grafted sites resulted in significant formation of new trabecular bone comparable in density and structure to the host site. The bone that formed was biologically and structurally normal Dr. Stephen Cook, Independent Histology Reviewer, Tulane University 40

Sinus Augmentation Clinical Effectiveness Summary INFUSE Bone Graft: Combined Study Result: 79.6% Success Rate (exceeded target success rate) Induced de novo bone formation Exceeded primary outcome objective Clinically effective for: Generating bone in the maxillary sinus allowing for Dental restoration 41

David L. Cochran, DDS, PhD, MMSc Chairman Department of Periodontics UT Health Sciences Center at San Antonio 42

Extraction Socket Augmentation Clinical Need Goal: Restore ridge width and preserve height to replace missing teeth 43

Extraction Socket Augmentation Methods and Definition Prospective, randomized, multi-center (8), doubleblinded controlled 80-patient human clinical trial providing high level of evidence Buccal wall defect (~ 50% of the extraction socket depth) Effectiveness endpoint: adequate alveolar bone formation for implant placement Safety Endpoints 44

Extraction Socket Augmentation Studies Baseline rhbmp-2/acs Graft Bone Formation Biopsy Dental Implant and Osseointegration Functional Loading T=0 4 mos ~ 8 mos ~ 16 mos 18-36 mos CT Scan CT Scan Biopsy Periapical X-ray Periapical X-ray CT Scan Safety Evaluation Throughout the Study 45

Preceded by Pilot Study at 0.43 mg/ml Study Design Schematic Extraction Socket Study N=80 Acute Safety Established Cohort 2 --1.5 1.5 mg/ml rhbmp-2 (20) ACS Alone (10) Cohort 1 --0.75 0.75 mg/ml Unfilled (10) rhbmp-2 (20) ACS Alone (10) Unfilled (10) 46

Prospective, Controlled Extraction Socket IDE Studies Extraction Socket (N=92) N Dosing Study (80) rhbmp-2/acs (1.5 mg/ml) 21 Unfilled Control Treatment 20 ACS Alone (0.0 mg/ml) 17 rhbmp-2/acs (0.75 mg/ml) 22 rhbmp-2/acs Effectiveness N= 21 Pilot Study (12) rhbmp-2/acs (0.43 mg/ml) 12 47

Extraction Socket Augmentation Baseline No Buccal Wall Palatal Wall 16-weeks Post rhbmp-2/acs Placement (1.5 mg/ml) De Novo Bone 48

INFUSE Bone Graft Patient Baseline Buccal Wall Palatal Wall 16-weeks Post rhbmp-2/acs Placement (1.5 mg/ml) De Novo Bone 49

Patient with Unfilled Defect Buccal Wall Baseline Extraction Site Palatal Wall Marker 16-weeks Post Extraction 50

Clinically Significant Results vs. Unfilled Unfilled Defect/ Standard of Care INFUSE Bone Graft Baseline Marker Unfilled defect at time of extraction INFUSE defect at time of placement 16-weeks Post De Novo Bone forms in INFUSE group Unfilled defect 16 weeks post extraction INFUSE defect 16 weeks post placement 51

Patient Therapeutic Benefit Demonstrated Dental implant placed in de novo bone. 52

Extraction Socket Augmentation CT Scan Measurement Methodology Pre-op 16-week post rhbmp-2/acs Alveolar ridge height = D1 + D2 Width: perpendicular to line D2 at ¼, ½, and ¾ 53

Height Change (Loss) at 4 months D1 + D2-1.17-1.00-0.02 0.0070 0.0270 54

Width Change at ¼ at 4 Months Mean Width Change at ¼ 3.27 0.57 0.82 1/4 0.0002 0.0010 55

Width Change at 1/2 at 4 Months Mean Width Change at 1/2 3.97 1.62 1.79 1/2 0.0009 0.0026 56

INFUSE Extraction Socket Augmentation Bone Growth and Implant Placement Mean Change (mm) ± SD No Implant Implant P Value Height -1.21 ± 1.34-0.36 ± 1.28 0.0085 Width at ¼ 0.74 ± 1.76 2.17 ± 2.45 0.0049 Width at ½ 1.79 ± 1.80 2.89 ± 2.37 0.0280 Width at ¾ 2.12 ± 1.61 2.06 ± 1.56 0.8862 Greater bone growth is associated with greater implant placement. 57

Functional Loading Endpoint, By Patient Time Post Functional Loading Unfilled ACS Only (0.0 mg/ml) INFUSE Bone Graft P Value Unfilled / ACS Only vs. INFUSE Bone Graft Dental Implant Placement 47% (9/19) 59% (10/17) 86% (18/21) 0.0171 / 0.0780 Received prosthesis 41% (7/17) 56% (9/16) 76% (16/21) 0.0458 / 0.2913 6 month 38% (6/16) 50% (7/14) 74% (14/19) 0.0442 / 0.2728 12 month 38% (6/16) 50% (7/14) 72% (13/18) 0.0824 / 0.2769 18 month 38% (6/16) 50% (7/14) 69% (11/16) 0.1556 / 0.4572 24 month 38% (6/16) 50% (7/14) 67% (10/15) 0.1556 / 0.4621 INFUSE Bone Graft is more successful. 58

Dental Implant Placement w/o Augmentation: INFUSE Bone Graft vs. ACS only vs. Unfilled by Patient 90% 86% 80% 70% 60% 59% 50% 47% 40% 30% Unfilled (N=19) ACS Only (0.0 mg/ml) (N=17) INFUSE Bone Graft (N=21) 59

6 month Functional Loading: INFUSE Bone Graft Compared with ACS only and Unfilled by Patient 80% 74% 70% 60% 50% 50% 40% 38% 30% 20% Unfilled (N=19) ACS Only (0.0 mg/ml) (N=17) INFUSE Bone Graft (N=21) 60

Same Histology for Both Indications Extraction Socket INFUSE BONE GRAFT Samples taken approx. 7 months post-op Similar Qualitative Parameters including Trabecular Volume, Thickness, and Number Sinus Augmentation 61

Extraction Socket Representative Histology rhbmp-2/acs induced bone Vascular marrow space Lamellar and immature bone Similar to the bone formed in the sinus studies H & E stain, 10 X magnification 62

Density Comparison Extraction Socket Sinus Studies Density Density (mg/cc) 0 100 200 300 400 500 Unfilled 0.00 mg/ml 1.50 mg/ml Bone Graft 1.50mg/ml Bone Graft 1.50mg/ml rhbmp-2/acs rhbmp-2/acs rhbmp-2/acs rhbmp-2/acs 4 months Post Grafting 4 months Post Grafting 6 months Post Prosthesis Extraction Socket Sinus 4 months post grafting 4 months post grafting 6 months post prosthesis 63

INFUSE Bone Graft: Summary of Clinical Effectiveness: Extraction Socket Augmentation Induced de novo bone formation Is clinically effective following tooth extraction for: Preservation of the alveolar ridge and Dental restoration 64

SAFETY OF INFUSE Bone Graft 65

Safety Profile Two PMA approvals More than 300,000 kits distributed worldwide 437 patients received rhbmp-2/acs in IDE clinical trials which supported two PMAs More Level I clinical evidence than any other bone grafting agent 1,070 patients studied in FDA clinical trials 66

Prospective, Controlled IDE Studies (N=312) Safety Data Sinus Augmentation (N=220) N rhbmp-2/acs (1.5 mg/ml) 99 Bone Graft 91 rhbmp-2/acs (0.43; 0.75 mg/ml) 30 Extraction Socket (N=92) rhbmp-2/acs (1.5 mg/ml) 21 Unfilled Control Treatment 20 rhbmp-2/acs (0.43; 0.75 mg/ml) 34 Safety: All rhbmp-2/acs N= 184 1.5 mg/ml rhbmp-2/acs N=120 ACS Alone (0.0mg/ml) 17 67

Adverse Events > 10% Patients COSTART TERM EDEMA FACE EDEMA INFECTION PAIN ORAL EDEMA ORAL ERYTHEMA MOUTH PAIN ECCHYMOSIS Bone Graft (n=91) 37.4% 57.1% 42.9% 50.5% 64.8% 61.5% 83.5% 23.1% INFUSE Bone Graft (n=120) 1.7% 67.5% 25.0% 21.7% 67.5% 47.5% 85.0% 15.8% 68

Adverse Events > 10% Patients (cont.) COSTART TERM HYPERGLYCEMIA ARTHRALGIA BONE DISORDER ABNORMAL GAIT HYPESTHESIA SINUSITIS ERYTHEMA Bone Graft (n=91) 16.5% 26.4% 12.1% 40.7% 16.5% 16.5% 37.4% INFUSE Bone Graft (n=120) 6.7% 11.7% 11.7% 0 4.2% 9.2% 7.5% 69

Adverse Events > 10% Patients (P<0.05) 50 40 30 20 10 0 70 Arthalgia Abnormal Gait Hypesthesia Erythema Edema Infection Pain Hyperglycemia Bone Graft INFUSE Bone Graft Adverse Event (%)

Adverse Event Summary INFUSE Bone Graft and Bone Graft Sinus Augmentation Extraction Socket Augmentation Bone Graft N=91 rhbmp-2/acs 1.5 mg/ml N=99 rhbmp-2/acs 1.5 mg/ml N=21 At Least One Event 99% 100% 100% rhbmp-2/acs Procedure Related Not Applicable 17% 24% Grade 3 or 4 18% 10% 10% Grade 3 or 4 rhbmp- 2/ACS Procedure Related Not Applicable 0 0 One death unrelated to INFUSE Bone Graft at 3 years post-implantation 71

rhbmp-2 and Collagen Antibodies Immune response evaluated in 184 rhbmp- 2/ACS patients and 91 bone graft patients Anti-rhBMP-2 Antibody Bone Graft 0.0% rhbmp- 2/ACS 2.2%* Anti-bovine Type I collagen 31% 20% Anti-human Type I collagen None None *Low titers, transient effect, clinically insignificant 72

Bone Graft Patients: Harvest Site Pain and Morbidity N=91 Harvest Site Iliac Crest (n=18) Conditions Pain Sensory Loss Gait Disturbance 2 days 89.9% 0% 55.6% 10 days 44.4% 0% 44.4% 6 months 0% 11.1% 5.6% Tibial Plateau (n=33) Pain Sensory Loss Gait Disturbance 66.7% 0% 72.7% 51.5% 3% 45.5% 3.1% 0% 3.1% Intra-Oral Bone (n=30) Pain Sensory Loss Gait Disturbance 58.6% 27.6% 0% 27.6% 34.5% 0% 0% 17.2% 0% 73

Timeline of Harvest Site Adverse Events 100 Adverse Events % 80 60 40 20 Bone Graft No harvest site for INFUSE Bone Graft patients! 0 0 20 40 60 80 100 120 140 160 180 200 Days After Harvest 74

Safety Summary Established safety profile Thoroughly evaluated in these RCT Significantly fewer adverse events than bone graft Eliminates bone harvesting morbidity 75

Conclusions Ed Chin, DPh Group Director, Regulatory Affairs Medtronic Spinal and Biologics 76

Proposed Indication For Use Statement "INFUSE Bone Graft is indicated as an alternative to autogenous bone graft for sinus augmentations and localized alveolar ridge augmentations for defects associated with extraction sockets." 77

Sinus Augmentation Extraction Socket Augmentation Implant and Tooth Restoration 78

Sinus Augmentation Following INFUSE Bone Graft, multiple implants, and prosthetic teeth replacement Large bone loss on maxillary ridge 79

Extraction Socket Augmentation Following INFUSE Bone Graft, dental implants, and prosthetic teeth replacement Non restorable teeth 80

SUMMARY INFUSE induced new bone allowing for dental restoration in 2 separate IDE evaluations INFUSE is Safe and Effective for Sinus Augmentation and Extraction Socket INFUSE eliminates the need to harvest autogenous bone graft in oral maxillofacial procedures 81

INFUSE Bone Graft SAFE and EFFECTIVE 82

Thank You 83