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ICON BIOSCIENCE
Creating Clinically Superior Ophthalmic Drugs
CONFIDENTIAL
Spring 2015
CONFIDENTIAL
Company Overview
2
Targets Large,
Unsatisfied Markets
• Ophthalmic drug markets generate $25+ billion in worldwide sales
annually
• Critical challenge: Drug delivery is key to compliance and duration
Verisome® Platform
• Improved products created through a combination of proprietary
Verisome® technology and proven compounds
• Enabling – easily administered, controlled, extended release drug
delivery mechanism
• Administered to >600 patients in multiple clinical trials in the U.S.
Advanced Lead Product
and Robust Pipeline
• IBI-10090 for post-cataract surgery inflammation
– Completed Phase 3; NDA filing late 2015 / early 2016
– Targets ~4 million patients annually in the U.S. alone
• Next drug Phase 2-ready and 5 additional programs in pipeline
Proven Management
• NEI/NIH
• Elan
• Oculex • Oceana Therapeutics
• Genentech / Roche• Onyx
• Valera
• Roberts Pharma
Ophthalmic Delivery
Focus
• Advanced clinical stage biopharma company focused on improving
eye health through more efficient delivery of ophthalmic drugs
• Expedited 505(b)(2) FDA approval pathway
CONFIDENTIAL
Verisome® Technology – Enabling Solution
Proprietary Delivery Technology
Vitreous
Injection
Anterior
Chamber
Injection
• True liquid injection
- Standard technique using small-gauge needle
- From 1 week to over 9 months duration with a single intraocular injection
• Biodegradable: Fully eliminated as drug is released
- No residual remains
• Physician control
- Visually monitor status and pace of delivery
- Treatment can be tailored to the individual patient
- May be removed, if needed
3
CONFIDENTIAL
Icon Bioscience Product Pipeline
4
IBI-10090
Post-cataract surgery
inflammation
Verisome® Dexamethasone
IBI-20089
DME, uveitis Verisome® Triamcinolone
IBI-60089
Glaucoma Verisome® Latanoprost
IBI-30089
PDR, wet AMD Verisome® Cyclosporine
NSAID
Post-cataract surgery
inflammation
Verisome® Undisclosed
IBI-70090
Uveitis, DME, dry AMD Verisome® Methotrexate
Phase
1
Phase
2
Phase
3
Pre-
Clinical
Product /
Indication
NDA
Filing
Delivery
Mechanism
Therapeutic
Active
IBI-80090
Retinoblastoma
(orphan); MSK partnership
Verisome® Melphalan
CONFIDENTIAL
Product Positioning:
• Convenience through one-time
administration by physician
• No patient compliance issues
• More rapid “quieting” of inflamed eye
Faster restoration of vision
• Side effects comparable to Steroid
drops
Potential Additional Indications:
• Anterior uveitis, post vitrectomy
inflammation
5
IBI-10090 Overview
Product Description:
• Dexamethasone + Verisome®
• One-time 5µL anterior segment
injection
Product Status:
• Phase 3 pivotal trial completed Oct
2014
• FDA “Type C” planning meeting
held July 2013
• Excellent efficacy and safety results
in clinical data
• NDA to submitted Q4/15-Q1/16
CONFIDENTIAL
• Multicenter, randomized, double-
masked, dose-ranging
• Initiated April 2012
• Completed March 2013
• 172 patients / 13 U.S. sites
• Dose groups / # patients
– 342µg dexamethasone / 58
– 517µg dexamethasone / 56
– 697µg dexamethasone / 58
• Primary efficacy endpoint:
– Proportion of patients with
anterior chamber cells (ACC)
clearing(1) at Day 8
• Secondary efficacy endpoints:
– ACC clearing over time
– Anterior chamber flare (ACF)
clearing
– ACF and ACC clearing
Trial Design Endpoints
6
IBI-10090 Phase 2 Trial Design
(1) ACC = 0.
CONFIDENTIAL
0
10
20
30
40
50
60
70
80
90
100
342 µg 517 µg 697 µg
60.3%
IBI-10090 Dexamethasone Dose Group
50.0% 51.8%
(n=58) (n=56) (n=58)
Note: The last-observation-carried-forward (LOCF) method was used to impute missing data.
(1) The p-value for the overall comparison among all dose groups is based on a two-sided Fisher's exact test and was not statistically significant (p >0.50) at Day 8.
Proportion of Patients with ACC=0 at Day 8
%
IBI-10090 Phase 2 Trial Primary Efficacy Endpoint
CONFIDENTIAL
7
Primary efficacy endpoint of ACC clearance at Day 8: The ACC=0 rates for the 342µg, 517µg and 697µg dose
groups were 50.0%, 51.8% and 60.3% of patients at Day 8, respectively
• There was no statistically significant difference(1) among the three dose groups
ACC clearing rate was significantly above the ~20-30% range observed for current eye drop treatments
CONFIDENTIAL
0
10
20
30
40
50
60
70
80
90
100
Day 1 Day 3 Day 8 Day 15 Day 30
342 µg dexamethasone
517 µg dexamethasone
697 µg dexamethasone
Study Visit Day
Note: Vertical bars are +/-1 standard error of the unadjusted mean.
The last-observation-carried-forward (LOCF) method was used to impute missing data.
(1) The p-value for the overall comparison among all dose groups is based on a two-sided Fisher's exact test and was not statistically significant (p >0.30 in all cases)
at any time point.
%
IBI-10090 Phase 2 Trial Secondary Efficacy Endpoint
CONFIDENTIAL
8
Proportion of Patients with ACC=0
Secondary efficacy endpoint of ACC clearing over time: The ACC=0 rates for the 342µg, 517µg and 697µg
dose groups increased to 65.5%, 78.6% and 77.6% of patients at Day 30, respectively
• There was no statistically significant difference(1) among the three dose groups at any time point
CONFIDENTIAL
IBI-10090 Phase 3 Trial Design
• Multicenter, randomized, double-
masked, dose-ranging
• Initiated December 2013
• Completed October 2014
• Total 394 patients / 28 U.S. sites
• Dose groups/# patients
– 342µg dexamethasone / 158
– 517µg dexamethasone / 156
– Placebo (0 dexamethasone) / 80
• Primary efficacy endpoint:
– Proportion of patients with
anterior chamber cell (ACC)
clearing(1) at Day 8
• Secondary efficacy endpoints:
– ACC clearing over time
– Anterior chamber flare (ACF)
clearing
– ACF and ACC clearing
Trial Design Endpoints
9
(1) ACC = 0.
CONFIDENTIAL
IBI-10090 Phase 3 Trial Efficacy and Safety
Efficacy:
• The study met all primary and secondary endpoints with statistically significant
differences between the placebo group and the two active dose groups
• The percentage of patients with ACC=0 at Day 8 was 25.0% in the placebo group,
and 63.1% and 66.0% in the 342µg and 517µg dexamethasone dose groups,
respectively
Safety:
• No ocular serious adverse events were reported
• Ocular adverse events for the IBI-10090 active dose groups were similar to the
placebo group and to the ocular adverse events stated in the label for Durezol
– These ocular adverse events for IBI-10090 occurred in 5-15% of patients
• Most of the adverse events observed for IBI-10090 may have been the
consequence of the surgical procedure
10
CONFIDENTIAL
IBI-10090 Commercial Opportunity
U.S. Ex-U.S.
~4 Million ~21 Million
3% 3%
~$400 TBD
2017 >2017
40% 20%
>$500 Million $TBD
Cataract Surgeries(1)
Growth Rate
Price / Injection
Anticipated Launch
Maximum Penetration
Peak Sales
11
Source: Marketscope.
(1) Estimated number of cataract surgeries in 2017.
CONFIDENTIAL
IBI-10090 Reimbursement
12
Site of Use:
• IBI-10090 will primarily be used in the ambulatory surgery center setting –
Medicare Part B
• Cataract surgery billed – CPT for physician service and APC fee for facility
• Icon will initially seek pass through designation (C code) for IBI-10090 to provide
separate payment
Pass Through Designation:
• Pass through status is temporary – 2-3 years
• Intended to provide separate reimbursement for novel technologies until:
– Cost data can either be incorporated into a primary procedure, raising the
reimbursement rate for the standard of care (in this case cataract surgery)
– It is determined that IBI-10090 should be paid separately as a separate service
in the future
Permanent J Code
• Once pass through in place, work with reimbursement consultants, KOLs, ASCRS to
get permanent APC payment via J code
CONFIDENTIAL
Product Status:
• Pre-clinical studies complete
• Active IND with two small Phase 2 studies completed
– 10-patient study in cystoid macular edema following retinal vein occlusion
demonstrated that a single intravitreal injection of IBI-20089 resulted in the
controlled and extended delivery of triamcinolone over 6-12 months
– 10-patient study in wet AMD demonstrated that a single intravitreal injection
of IBI-20089 in combination with a single intravitreal injection of Lucentis
resulted in the controlled and extended delivery of triamcinolone over 6-12
months
13
IBI-20089 Overview
Product Positioning:
• Convenience with 6-12 month
duration
• Improved compliance
Product Description:
• Triamcinolone + Verisome®
• 25-50µL posterior segment
injection
CONFIDENTIAL
Product Description:
• Latanoprost formulated + Verisome® technology
• 20.0 - 50.0µL intravitreal injection
• Administration: standard injection procedure – 30G needle
Product Status:
• Preclinical (kinetics / toxicology / stability) results are positive
• Second preclinical trial initiation pending
• 3 month dog study to be conducted at Calvert, PA
• IND to filed Q1 2016
Product Positioning:
• Patient convenience & compliance
• Potential for superior efficacy compared to eye drops regardless of active drug
14
IBI-60089 Overview
CONFIDENTIAL
Near-term Revenue Generation
Robust Pipeline
Widely Applicable Verisome® Platform
Proven Management Team
Large Market Opportunity
Takeaway
15

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Creating Clinically Superior Ophthalmic Drugs Through Extended-Release Delivery

  • 1. ICON BIOSCIENCE Creating Clinically Superior Ophthalmic Drugs CONFIDENTIAL Spring 2015
  • 2. CONFIDENTIAL Company Overview 2 Targets Large, Unsatisfied Markets • Ophthalmic drug markets generate $25+ billion in worldwide sales annually • Critical challenge: Drug delivery is key to compliance and duration Verisome® Platform • Improved products created through a combination of proprietary Verisome® technology and proven compounds • Enabling – easily administered, controlled, extended release drug delivery mechanism • Administered to >600 patients in multiple clinical trials in the U.S. Advanced Lead Product and Robust Pipeline • IBI-10090 for post-cataract surgery inflammation – Completed Phase 3; NDA filing late 2015 / early 2016 – Targets ~4 million patients annually in the U.S. alone • Next drug Phase 2-ready and 5 additional programs in pipeline Proven Management • NEI/NIH • Elan • Oculex • Oceana Therapeutics • Genentech / Roche• Onyx • Valera • Roberts Pharma Ophthalmic Delivery Focus • Advanced clinical stage biopharma company focused on improving eye health through more efficient delivery of ophthalmic drugs • Expedited 505(b)(2) FDA approval pathway
  • 3. CONFIDENTIAL Verisome® Technology – Enabling Solution Proprietary Delivery Technology Vitreous Injection Anterior Chamber Injection • True liquid injection - Standard technique using small-gauge needle - From 1 week to over 9 months duration with a single intraocular injection • Biodegradable: Fully eliminated as drug is released - No residual remains • Physician control - Visually monitor status and pace of delivery - Treatment can be tailored to the individual patient - May be removed, if needed 3
  • 4. CONFIDENTIAL Icon Bioscience Product Pipeline 4 IBI-10090 Post-cataract surgery inflammation Verisome® Dexamethasone IBI-20089 DME, uveitis Verisome® Triamcinolone IBI-60089 Glaucoma Verisome® Latanoprost IBI-30089 PDR, wet AMD Verisome® Cyclosporine NSAID Post-cataract surgery inflammation Verisome® Undisclosed IBI-70090 Uveitis, DME, dry AMD Verisome® Methotrexate Phase 1 Phase 2 Phase 3 Pre- Clinical Product / Indication NDA Filing Delivery Mechanism Therapeutic Active IBI-80090 Retinoblastoma (orphan); MSK partnership Verisome® Melphalan
  • 5. CONFIDENTIAL Product Positioning: • Convenience through one-time administration by physician • No patient compliance issues • More rapid “quieting” of inflamed eye Faster restoration of vision • Side effects comparable to Steroid drops Potential Additional Indications: • Anterior uveitis, post vitrectomy inflammation 5 IBI-10090 Overview Product Description: • Dexamethasone + Verisome® • One-time 5µL anterior segment injection Product Status: • Phase 3 pivotal trial completed Oct 2014 • FDA “Type C” planning meeting held July 2013 • Excellent efficacy and safety results in clinical data • NDA to submitted Q4/15-Q1/16
  • 6. CONFIDENTIAL • Multicenter, randomized, double- masked, dose-ranging • Initiated April 2012 • Completed March 2013 • 172 patients / 13 U.S. sites • Dose groups / # patients – 342µg dexamethasone / 58 – 517µg dexamethasone / 56 – 697µg dexamethasone / 58 • Primary efficacy endpoint: – Proportion of patients with anterior chamber cells (ACC) clearing(1) at Day 8 • Secondary efficacy endpoints: – ACC clearing over time – Anterior chamber flare (ACF) clearing – ACF and ACC clearing Trial Design Endpoints 6 IBI-10090 Phase 2 Trial Design (1) ACC = 0.
  • 7. CONFIDENTIAL 0 10 20 30 40 50 60 70 80 90 100 342 µg 517 µg 697 µg 60.3% IBI-10090 Dexamethasone Dose Group 50.0% 51.8% (n=58) (n=56) (n=58) Note: The last-observation-carried-forward (LOCF) method was used to impute missing data. (1) The p-value for the overall comparison among all dose groups is based on a two-sided Fisher's exact test and was not statistically significant (p >0.50) at Day 8. Proportion of Patients with ACC=0 at Day 8 % IBI-10090 Phase 2 Trial Primary Efficacy Endpoint CONFIDENTIAL 7 Primary efficacy endpoint of ACC clearance at Day 8: The ACC=0 rates for the 342µg, 517µg and 697µg dose groups were 50.0%, 51.8% and 60.3% of patients at Day 8, respectively • There was no statistically significant difference(1) among the three dose groups ACC clearing rate was significantly above the ~20-30% range observed for current eye drop treatments
  • 8. CONFIDENTIAL 0 10 20 30 40 50 60 70 80 90 100 Day 1 Day 3 Day 8 Day 15 Day 30 342 µg dexamethasone 517 µg dexamethasone 697 µg dexamethasone Study Visit Day Note: Vertical bars are +/-1 standard error of the unadjusted mean. The last-observation-carried-forward (LOCF) method was used to impute missing data. (1) The p-value for the overall comparison among all dose groups is based on a two-sided Fisher's exact test and was not statistically significant (p >0.30 in all cases) at any time point. % IBI-10090 Phase 2 Trial Secondary Efficacy Endpoint CONFIDENTIAL 8 Proportion of Patients with ACC=0 Secondary efficacy endpoint of ACC clearing over time: The ACC=0 rates for the 342µg, 517µg and 697µg dose groups increased to 65.5%, 78.6% and 77.6% of patients at Day 30, respectively • There was no statistically significant difference(1) among the three dose groups at any time point
  • 9. CONFIDENTIAL IBI-10090 Phase 3 Trial Design • Multicenter, randomized, double- masked, dose-ranging • Initiated December 2013 • Completed October 2014 • Total 394 patients / 28 U.S. sites • Dose groups/# patients – 342µg dexamethasone / 158 – 517µg dexamethasone / 156 – Placebo (0 dexamethasone) / 80 • Primary efficacy endpoint: – Proportion of patients with anterior chamber cell (ACC) clearing(1) at Day 8 • Secondary efficacy endpoints: – ACC clearing over time – Anterior chamber flare (ACF) clearing – ACF and ACC clearing Trial Design Endpoints 9 (1) ACC = 0.
  • 10. CONFIDENTIAL IBI-10090 Phase 3 Trial Efficacy and Safety Efficacy: • The study met all primary and secondary endpoints with statistically significant differences between the placebo group and the two active dose groups • The percentage of patients with ACC=0 at Day 8 was 25.0% in the placebo group, and 63.1% and 66.0% in the 342µg and 517µg dexamethasone dose groups, respectively Safety: • No ocular serious adverse events were reported • Ocular adverse events for the IBI-10090 active dose groups were similar to the placebo group and to the ocular adverse events stated in the label for Durezol – These ocular adverse events for IBI-10090 occurred in 5-15% of patients • Most of the adverse events observed for IBI-10090 may have been the consequence of the surgical procedure 10
  • 11. CONFIDENTIAL IBI-10090 Commercial Opportunity U.S. Ex-U.S. ~4 Million ~21 Million 3% 3% ~$400 TBD 2017 >2017 40% 20% >$500 Million $TBD Cataract Surgeries(1) Growth Rate Price / Injection Anticipated Launch Maximum Penetration Peak Sales 11 Source: Marketscope. (1) Estimated number of cataract surgeries in 2017.
  • 12. CONFIDENTIAL IBI-10090 Reimbursement 12 Site of Use: • IBI-10090 will primarily be used in the ambulatory surgery center setting – Medicare Part B • Cataract surgery billed – CPT for physician service and APC fee for facility • Icon will initially seek pass through designation (C code) for IBI-10090 to provide separate payment Pass Through Designation: • Pass through status is temporary – 2-3 years • Intended to provide separate reimbursement for novel technologies until: – Cost data can either be incorporated into a primary procedure, raising the reimbursement rate for the standard of care (in this case cataract surgery) – It is determined that IBI-10090 should be paid separately as a separate service in the future Permanent J Code • Once pass through in place, work with reimbursement consultants, KOLs, ASCRS to get permanent APC payment via J code
  • 13. CONFIDENTIAL Product Status: • Pre-clinical studies complete • Active IND with two small Phase 2 studies completed – 10-patient study in cystoid macular edema following retinal vein occlusion demonstrated that a single intravitreal injection of IBI-20089 resulted in the controlled and extended delivery of triamcinolone over 6-12 months – 10-patient study in wet AMD demonstrated that a single intravitreal injection of IBI-20089 in combination with a single intravitreal injection of Lucentis resulted in the controlled and extended delivery of triamcinolone over 6-12 months 13 IBI-20089 Overview Product Positioning: • Convenience with 6-12 month duration • Improved compliance Product Description: • Triamcinolone + Verisome® • 25-50µL posterior segment injection
  • 14. CONFIDENTIAL Product Description: • Latanoprost formulated + Verisome® technology • 20.0 - 50.0µL intravitreal injection • Administration: standard injection procedure – 30G needle Product Status: • Preclinical (kinetics / toxicology / stability) results are positive • Second preclinical trial initiation pending • 3 month dog study to be conducted at Calvert, PA • IND to filed Q1 2016 Product Positioning: • Patient convenience & compliance • Potential for superior efficacy compared to eye drops regardless of active drug 14 IBI-60089 Overview
  • 15. CONFIDENTIAL Near-term Revenue Generation Robust Pipeline Widely Applicable Verisome® Platform Proven Management Team Large Market Opportunity Takeaway 15