saving sight through transformative science
TRANSCRIPT
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Securities Litigation Reform Act of 1995, including, but not limited to statements regarding our clinical pipeline, our ability to advance GB-102, GB-103, GB-
401, or any future product candidate through clinical development, our ability to achieve our anticipated milestones within the timing outlined above or at all,
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2
Extensive experience in ophthalmology and healthcare
Novartis
Worldwide Head
Ophthalmology
Alcon
Global Franchise Head
Pharmaceuticals
Led extension of Novartis
ophthalmology pipeline:
Encore Vision, Lubricin®,
Luxturna®, Xiidra®
Novartis
VP and Global Head, Clinical
Development and
Therapeutic Area Head,
Ophthalmology
Ophthalmologist from
Moorfield’s Eye Hospital, UK
Ocular immunologist from
Schepens Eye Research
Institute, a Harvard affiliated
institute
Corium
CFO, IPO through PE sale
Codexis
CFO, Private to IPO
Aerogen
CFO, Public through Public
Sale
Ophthotech Corporation
Technical Operations
Aptalis Pharmaceuticals,
Global Supply Chain and
Manufacturing
Launched over 30 global
products including Zenpep®,
Lamictal®, Amrix®, Canasa®
and Metadate CD®
Fred Guerard
PharmD, CEORobert Breuil
CFO
Parisa Zamiri
MD, PhD, CMODan Salain
Chief Tech Ops OfficerBettina Maunz
Chief People Officer
Novartis
Global Head Enterprise
Communications
Alcon
VP and Global Head
Communications, President
Alcon Foundation
Led teams and culture
change as part of significant
business transformations
across Pharma, Biotech and
Medical Device industry
3
Dr. Rick Ferris
Ophthalmic Research
Consultants
National Eye Institute
(retired)
4 |
Dr. Jeff Heier
Ophthalmic Consultants
of Boston
Dr. Arshad Khanani
Sierra Eye Associates
University of Nevada,
Reno School of Medicine
Dr. Carl Regillo
Retina Service Wills
Eye Hospital
Thomas Jefferson
University School of
Medicine
Dr. David Boyer
Retina-Vitreous
Associates Medical
Group, California
USC/Keck School of
Medicine
Dr. Rishi Singh
Cole Eye Institute,
Cleveland Clinic
Lerner College of
Medicine
4
Our Scientific Advisory Board
Graybug investment highlights
5
Potentially transformative, long-acting treatments for vision-threatening diseases
Differentiated clinical-stage candidates targeting $15B+ markets
• GB-102 for wet age-related macular degeneration (wet AMD)
• GB-102 for diabetic macular edema (DME)
• GB-103 for diabetic retinopathy (DR)
• GB-401 for primary open-angle glaucoma (POAG)
Patent protection: GB-102/GB-103 through 2039, GB-401 through 2041
Numerous near-term milestones
5
2Q 2021: GB-102 Phase 2b results 2H 2021: Initiate 4 new trials
Proprietary ocular technologies promote controlled, sustained drug delivery
Extended durability & sustained drug deliveryDifferentiated mechanisms of action
Designed with safety in mindVersatile proprietary technologies
6
Particle aggregation and bioabsorption with GB-102 (1 mg) Observed over 6 months
Baseline 2 Weeks 1 Month 2 Months
4 Months3 Months 5 Months 6 Months
7
GB-102 depot images using wide field color fundus photography throughout the 6-month observation period in the ME study
Our pipeline of long-acting ocular drug candidates
8
GB-102(sunitinib)
6-month dosing
GB-103(sunitinib)
12-month dosing
GB-401(pro-drug)
6-month dosing
Wet Age-Related Macular
Degeneration (wAMD)
Diabetic Retinopathy (DR)
Primary Open-Angle Glaucoma
(POAG)
Ph2b ALTISSIMO Topline (wAMD)
2021 2022
1H 2H 1H
Initiate Ph3 Global Trials (wAMD)
Initiate Ph2b (DME)
Initiate Ph1/2a (DR)
IND Submission (POAG)
Program Indication Anticipated Milestone
Diabetic Macular Edema (DME)
Diabetic Retinopathy (DR) and Diabetic Macular Edema (DME)
Leading causes of acquired vision loss in young & middle-age adults
Age-related Macular Degeneration (AMD)
Leading cause of vision loss in the elderly worldwide
Retina: A growing $11B+ market with significant unmet need
|
10
Existing standard of care presents high burden of up to 12 intravitreal injections per year
US and Global AMD reference: Pennington and DeAngelis Eye and Vision (2016)
10-15% patients
with wAMD
~11M AMD patients in U.S.
(~170M WW)
8% annual growth
$7.9B WW
~30M diabetic patients in U.S.
(~460M WW)
700M diabetic
patients WW in 2035
$3.7B WW
~1.5M DME
patients in U.S.
(~20M WW)
~10M DR patients in U.S.
(~90M WW)
Hariprasad SM et al. Journal of Ophthalmology, Volume 2012, Article ID 690641
SAILOR PRN
PIER Q12 w
ANCHOR Q4 w
MARINA Q4 w
Stability and maintenance of vision requires frequent injectionsLonger-acting therapies are #1 unmet need for retina specialists
Low visual outcomes due to high burden of up to 12 intravitreal injections per year
Begin dosing quarterly (PIER) and as-needed (SAILOR)
ANCHOR (dosed every 4 weeks, n=279)
Me
an
VA
* c
ha
ng
e
fro
m b
as
eli
ne (
lett
ers
)MARINA (dosed every 4 weeks, n=478)
SAILOR (dosed as needed, n=946)
0 3 6 9 12 15 18 21 24
15
10
5
0
-5
Months*Visual Acuity
All studies were conducted with ranibizumab (Lucentis®)
Each square, rhombus, or triangle represents an anti-VEGF injection; each circle represents a measurement visit
Vision declines without regular injections
PIER (dosed every 12 weeks; n=121)
11
Higher fluctuations in retinal thickness correlate with poor visual outcomes
Decreasing Vision
12
Increasing Fibrosis
12
High
Low
High
Low
Fluctuation:
Fluctuation:
Association between variation in retinal thickness and visual function, Evans R, Chakravathy U, Reeves, BC, ARVO 2019 poster #3456
High Retinal Thickness
Normal Retinal Thickness
Pan-VEGF Inhibitor Neuroprotectant
Sunitinib is a potent pan-VEGF inhibitor and neuroprotectant
13
DLK: Dual Leucine Zipper Kinase
Oral sunitinib inhibits
CNV in murine model
(Takahashi 2006)
Oral sunitinib inhibits
CNV in cancer patients
(Hulpus et al 2014)
No ocular toxicity in long-term
safety surveillance studies
(Motzer et al 2013 Porta et al 2016)
Potent anti-angiogenic
in laser-CNV model
(Campochiaro et al 2019)
CNV: Choroidal Neovascularisation
10
10.5
11
11.5
12
12.5
13
13.5
14
Control 40 80 150CN
V V
olu
me (
log c
onvert
ed)
Dose (mg/kg)
Sunitinib has well documented safety and efficacy in choroidal
neovascularization
14
Baselin
e
Month 8
0.00
0
0.01
0.02
0.015
0.03
0.025
Mouse Laser CNV Model Aflibercept vs. GB-102
*
Day 0:• Aflibercept x 1 dose
Intravitreal• GB-102 x 1 dose Intravitreal
**
Mean
CN
V A
rea
(mm
2)
0.5M 2M 3.5M 5M 6M
Months
* p<0.05 (GB-102 or aflibercept vs. fellow eye control)
*GB-102
No Effect Range
(Placebo)
aflibercept
*
Baseline
8 months
Competitive advantages of GB-102Microparticle depot formulation of sunitinib
Extended durability demonstrated
in Phase 1/2a
Twice a year versus up to 12 times
Reduction of fluctuation in retinal thickness
Differentiated mechanisms of action
Blocks all VEGF receptor types allowing for
broader MOA
Dual Leucine Zipper Kinase (DLK) inhibitor
may have neuro-protective effect
15 |
Versatile proprietary technologies
Tailored durability
Tunable drug elution profile
Designed with safety in mind
Biodegradable/bioabsorbable
Approx. 100 patients exposed
15
≤ 3 months duration
≥ 8 months duration 4-5 months duration
≥ 6 months durationAnti-VEGF injection prior to study entry GB-102 injection Patient leaving study unrelated to treatment
16
Time to first use of
supportive therapy
Percentage of
patients (n)
≤ 3M 12% (1/8)
≥ 4M 88% (7/8)
≥ 6M 88% (7/8)
≥ 8M 50% (4/8)
GB-102 1 mg
Open label, dose ranging, safety, durability and pharmacodynamics study
Phase 1/2a ADAGIO: single injection of GB-102 in patients with wet AMD previously treated with short-acting anti-VEGFs
GB-102 1 mg single injection maintained visual acuity for 6+ months
17
Anti-VEGF injections GB-102 injection
GB-102 1 mg (n=8)• Mean Pre anti-VEGF/year = 8.8 ± 1.9• Mean Pre BCVA = 61 ± 12 (6/8)• Mean Final BCVA = 65 ± 11
30
40
50
60
70
80
90
30
40
50
60
70
80
90
Pre Baseline 1M 2M 3M 4M 5M 6M 7M 8M
30
40
50
60
70
80
90
30
40
50
60
70
80
90
Mean
BC
VA
(E
TD
RS±
SD
)O
bserv
ed d
ata
GB-102 0.25 mg (n=8)• Mean Pre anti-VEGF/year = 8.8 ± 1.9 • Mean Pre BCVA = 57 ± 16 (7/8)• Mean Final BCVA = 47 ± 17
GB-102 0.5 mg (n=8)• Mean Pre anti-VEGF/year = 7.2 ± 2.8 • Mean Pre BCVA = 61 ± 12 (4/8)• Mean Final BCVA = 70 ± 13
GB-102 2 mg (n=8)• Mean Pre anti-VEGF/year = 9.9 ± 2.0 • Mean Pre BCVA = 59 ± 8 (5/8)• Mean Final BCVA = 58 ± 13
BCVA = Best corrected visual acuity
≥
M = month
≥
≥
≥
GB-102 1 mg single injection maintained retinal thickness for 6+ months
18 |18
GB-102 1 mg (n=8)• Mean Pre anti-VEGF/year = 8.8 ± 1.9• Mean Pre CST = 376 ± 133• Mean Final CST = 287 ± 85
100
200
300
400
500
Pre Baseline 1M 2M 3M 4M 5M 6M 7M 8M
100
200
300
400
500
100
200
300
400
500
100
200
300
400
500
C O N F I D E N T I A L A N D P R O P R I E T A R Y
GB-102 0.25 mg (n=8)• Mean Pre anti-VEGF/year = 8.8 ± 1.9 • Mean Pre CST = 394 ± 141• Mean Final CST = 312 ± 208
GB-102 0.5 mg (n=8)• Mean Pre anti-VEGF/year = 7.2 ± 2.8 • Mean Pre CST = 401 ± 89• Mean Final CST = 266 ± 76
GB-102 2 mg (n=8)• Mean Pre anti-VEGF/year = 9.9 ± 2.0 • Mean Pre CST = 410 ± 144 • Mean Final CST = 293 ± 78
Mean
CS
T (μ
m±
SD
)O
bserv
ed
data
CST = Central sub-field thickness
M = month
≥
≥
≥
≥
Anti-VEGF injections GB-102 injection
20 |19
GB-102 1 mg controlled ~80% of wAMD patients for 6 months or moreWhile being well tolerated
• GB-102 1 mg single injection in patients previously on ~8 injections of anti-VEGF annually (ADAGIO)
Reduced mean frequency of anti-VEGF injections by 80%
Controlled 7/8 patients for 6 months or more and 4/8 patients for more than 8 months
Maintained retinal thickness and visual acuity for 6 months or more
• GB-102 1 mg met primary endpoint of safety and tolerability (ME trial)
No ocular SAE or dose-limiting toxicity
Majority of patients had no drug-related AEs, including no inflammation
All AEs were mild to moderate without long-term consequences
• Phase 2b 12-month treatment phase completed in December 2020 (ALTISSIMO)
Top-line read-out expected in Q2 2021
58% of patients have entered 6-month extension period to observe further durability of GB-102 1 mg
GB-102 Phase 2b trial in wet AMD (ALTISSIMO) will read-out in Q2 2021
20
Extension study to provide information on GB-102 1mg beyond month 12
Primary endpoint: time to first use of additional supportive therapy
Monitoring Visit
50 out of 56 patients completed 12-
month treatment phase*
(LPV Dec 2020)
58% of patients who completed
Month 12 visit were eligible and
agreed to continue clinical monitoring
in six-month trial extension
*6 patients withdrew for reasons unrelated to their treatment
Lucentis($3,386M WW2)
BevacizumabOff-label
Commercialized
Products
FaricimabPhase 3 Completed
GB-102Phase 2b Treatment Completed +
Extension Study Ongoing
Ranibizumab(Surgical Implant of Port Delivery System)
Phase 3 Completed
In Development
GB-102 most clinically-advanced IVT drug targeting ≥6-month duration1
Longest lasting therapy currently approved is Beovu with 55% patients reaching 3 months
1 Products and product candidates shown are in different stages of development and have been studied in different numbers and types of patients, so direct comparisons of efficacy, duration, or commercial success may not be possible2 2020 global sales from GlobalData as of February 2021
KSI-301Phase 3 Initiated
Eylea($8,285M WW2)
Beovu($190M WW2)
21
Duration(Primary endpoint)
Safety
Control
Patients treatment-free for 6 months
>50% of patients treatment-free for 4 months
No irreversible vision-threatening inflammation
<15% of 1mg patients have medication present in the anterior chamber
BCVA and CST trending in parallel to control arm
ALTISSIMO outcomes required to be competitive with market leaders
22
Glaucoma: Significant unmet need due to lack of patient compliance
wAMD
~ 76M glaucoma patients worldwide
~$3.8B in 2026
~2.7M patients in U.S.
~$2.9B in 2026
50% of patients
stop treatment
within
6 months
>50% non-compliance
https://www.nei.nih.gov/learn-about-eye-health/resources-for-health-educators/eye-health-data-and-statistics/glaucoma-data-and-statistics
24
GB-401 could provide at least 6 months of sustained intraocular pressure
(IOP) control
32 | C O N F I D E N T I A L A N D P R O P R I E T A R Y
• Preclinical pharmacological effect similar to timolol eye drops
• At least six-month therapeutic drug level demonstrated in animal models
Phase 1/2a clinical trial in POAG planned to be initiated in 2H 2021
*
*Ki = Inhibition Constant
25
Large number of patent families protected through 2041
• Sole owner of ten patent families with 83 granted or pending patent applications
• Licensed five patent families from Johns Hopkins with 50 granted or pending patent applications
GB-102/GB-103
6 issued orange bookable patents
GB-401
3 issued orange bookable patents
Expiration window of issued patents and patent applications
2039
2041
27
Manufacturing process amenable to scale-up
28
• Currently relying on third-party manufacturing at Lubrizol Life Science Health for clinical trial supply
• Intention to build own manufacturing capabilities:
Gain more control and protect know-how
Reduce cycle times
Increase robustness and consistency of process
Reduce cost of goods for commercial production
Optimize commercial scale process
Recent IPO funds current operations
• Priced IPO at $16 on September 25, 2020
• Net proceeds totaled $92.1M, including full over-allotment option exercised in October 2020
• Cash and cash investments of $95.0M at September 30, 2020
• Sufficient to begin patient enrollment in both Phase 3 trials of GB-102 in wAMD in Q4 2021
• 21.0M shares outstanding as of November 6, 2020
• All common, no preferred, no debt
• >10% shareholders: Deerfield, Orbimed, Fidelity1
29
1 As reported on Form 13-G/A filed Feb 8, 2021
Our pipeline of long-acting ocular drug candidates
31
GB-102(sunitinib)
6-month dosing
GB-103(sunitinib)
12-month dosing
GB-401(pro-drug)
6-month dosing
Wet Age-Related Macular
Degeneration (wAMD)
Diabetic Retinopathy (DR)
Primary Open-Angle Glaucoma
(POAG)
Ph2b ALTISSIMO Topline (wAMD)
2021 2022
1H 2H 1H
Initiate Ph3 Global Trials (wAMD)
Initiate Ph2b (DME)
Initiate Ph1/2a (DR)
IND Submission (POAG)
Program Indication Anticipated Milestone
Diabetic Macular Edema (DME)
Graybug investment highlights
3
2
Potentially transformative, long-acting treatments for vision-threatening diseases
Differentiated clinical-stage candidates targeting $15B+ markets
• GB-102 for wet age-related macular degeneration (wet AMD)
• GB-102 for diabetic macular edema (DME)
• GB-103 for diabetic retinopathy (DR)
• GB-401 for primary open-angle glaucoma (POAG)
Patent protection: GB-102/GB-103 through 2039, GB-401 through 2041
Numerous near-term milestones
2Q 2021: GB-102 Phase 2b results 2H 2021: Initiate 4 new trials
32