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ICON BIOSCIENCECreating 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
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
3
CONFIDENTIAL
Icon Bioscience Product Pipeline
4
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
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 EndpointCONFIDENTIAL
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