Icon Bioscience

ICON BIOSCIENCECreating Clinically Superior Ophthalmic Drugs

CONFIDENTIAL

Spring 2015

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Company Overview

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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

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Verisome® Technology – Enabling Solution

Proprietary Delivery Technology

VitreousInjection

Anterior ChamberInjection

• 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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CONFIDENTIAL

Icon Bioscience Product Pipeline

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IBI-10090Post-cataract surgery inflammation

Verisome® Dexamethasone

IBI-20089DME, uveitis Verisome® Triamcinolone

IBI-60089Glaucoma Verisome® Latanoprost

IBI-30089PDR, wet AMD Verisome® Cyclosporine

NSAIDPost-cataract surgery inflammation

Verisome® Undisclosed

IBI-70090Uveitis, DME, dry AMD Verisome® Methotrexate

Phase 1

Phase 2

Phase 3

Pre-Clinical

Product / Indication

NDA Filing

Delivery Mechanism

Therapeutic Active

IBI-80090Retinoblastoma (orphan); MSK partnership

Verisome® Melphalan

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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

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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

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• Multicenter, randomized, double-masked, dose-ranging

• Initiated April 2012

• Completed March 2013

• 172 patients / 13 U.S. sites

• Dose groups / # patients

– 342µg 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

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IBI-10090 Phase 2 Trial Design

(1) ACC = 0.

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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 EndpointCONFIDENTIAL

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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

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0

10

20

30

40

50

60

70

80

90

100

Day 1 Day 3 Day 8 Day 15 Day 30

342 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

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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

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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



– 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

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(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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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

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Source: Marketscope.(1) Estimated number of cataract surgeries in 2017.

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IBI-10090 Reimbursement

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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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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-20089 Overview


• Convenience with 6-12 month duration

• Improved compliance


• Triamcinolone + Verisome®

• 25-50µL posterior segment injection

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• 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


• Patient convenience & compliance

• Potential for superior efficacy compared to eye drops regardless of active drug

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IBI-60089 Overview

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Near-term Revenue Generation

Robust Pipeline

Widely Applicable Verisome® Platform

Proven Management Team

Large Market Opportunity

Takeaway

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Icon Bioscience

Healthcare