1. health and fitness
2. drugs

ICON BIOSCIENCE
Creating Clinically Superior Ophthalmic Drugs
CONFIDENTIAL
Spring 2015
Company Overview
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
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
Proven Management
• 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
• NEI/NIH
• Oculex
• Roberts Pharma • Elan
• Valera
• Oceana Therapeutics
• Onyx
• Genentech / Roche
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Verisome® Technology – Enabling Solution
Vitreous
Injection
Anterior
Chamber
Injection
Proprietary Delivery Technology
•
•
•
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
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Icon Bioscience Product Pipeline
Product /
Indication
Delivery
Mechanism
Therapeutic
Active
Verisome®
Dexamethasone
Verisome®
Triamcinolone
Verisome®
Latanoprost
Verisome®
Melphalan
Verisome®
Cyclosporine
Verisome®
Undisclosed
Verisome®
Methotrexate
PreClinical
Phase
1
Phase
2
Phase
3
NDA
Filing
IBI-10090
Post-cataract surgery
inflammation
IBI-20089
DME, uveitis
IBI-60089
Glaucoma
IBI-80090
Retinoblastoma
(orphan); MSK partnership
IBI-30089
PDR, wet AMD
NSAID
Post-cataract surgery
inflammation
IBI-70090
Uveitis, DME, dry AMD
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IBI-10090 Overview
Product Description:
Product Positioning:
•
Dexamethasone + Verisome®
•
•
One-time 5µL anterior segment
injection
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
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
drops
Potential Additional Indications:
•
Anterior uveitis, post vitrectomy
inflammation
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IBI-10090 Phase 2 Trial Design
Trial Design
•
Multicenter, randomized, doublemasked, dose-ranging
•
Initiated April 2012
•
Completed March 2013
•
172 patients / 13 U.S. sites
•
Dose groups / # patients
– 342µg dexamethasone / 58
– 517µg dexamethasone / 56
Endpoints
•
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
– 697µg dexamethasone / 58
(1) ACC = 0.
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CONFIDENTIAL
IBI-10090 Phase 2 Trial Primary Efficacy Endpoint
100
Proportion of Patients with ACC=0 at Day 8
90
80
70
%
60
50
60.3%
50.0%
51.8%
342 µg
(n=58)
517 µg
(n=56)
40
30
20
10
0
697 µg
(n=58)
IBI-10090 Dexamethasone Dose Group
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
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.
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CONFIDENTIAL
IBI-10090 Phase 2 Trial Secondary Efficacy Endpoint
Proportion of Patients with ACC=0
100
90
80
70
%
342 µg dexamethasone
60
50
517 µg dexamethasone
40
30
697 µg dexamethasone
20
10
0
Day 1
Day 3
Day 8
Day 15
Day 30
Study Visit Day
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
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.
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IBI-10090 Phase 3 Trial Design
Trial Design
•
Multicenter, randomized, doublemasked, dose-ranging
•
Initiated December 2013
•
Completed October 2014
•
Total 394 patients / 28 U.S. sites
•
Dose groups/# patients
Endpoints
•
Primary efficacy endpoint:
– Proportion of patients with
anterior chamber cell (ACC)
clearing(1) at Day 8
•
Secondary efficacy endpoints:
– ACC clearing over time
– 517µg dexamethasone / 156
– Anterior chamber flare (ACF)
clearing
– Placebo (0 dexamethasone) / 80
– ACF and ACC clearing
– 342µg dexamethasone / 158
(1) ACC = 0.
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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
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IBI-10090 Commercial Opportunity
U.S.
Ex-U.S.
~4 Million
Cataract Surgeries(1)
~21 Million
3%
Growth Rate
3%
~$400
Price / Injection
TBD
2017
Anticipated Launch
>2017
40%
Maximum Penetration
20%
>$500 Million
Peak Sales
$TBD
Source: Marketscope.
(1) Estimated number of cataract surgeries in 2017.
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IBI-10090 Reimbursement
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
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IBI-20089 Overview
Product Description:
Product Positioning:
•
Triamcinolone + Verisome®
•
•
25-50µL posterior segment
injection
Convenience with 6-12 month
duration
•
Improved compliance
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
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IBI-60089 Overview
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
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Takeaway
Large Market Opportunity
Near-term Revenue Generation
Robust Pipeline
Widely Applicable Verisome® Platform
Proven Management Team
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