
GENERATION 2.0
Inspired to build the world’s leading high-science retina pipeline
OUR PIPELINE
Our pipeline is designed to address key limitations of today’s therapies and bring new science to the treatment of retinal diseases.

OUR CANDIDATES

Tarcocimab tedromer
1 Molecule, 1 Target

KSI-501
1 Molecule, 2 Targets

Triplet Medicines
1 Molecule, Many targets
An investigational anti-VEGF biologic designed for long-interval dosing
Tarcocimab tedromer is designed to maintain potent and effective drug levels in ocular tissues for longer than existing anti-VEGF therapies.
Built on our proprietary Antibody Biopolymer Conjugate (ABC) Platform, tarcocimab tedromer is purposefully designed with a science for durability and is being studied as a 6-month durability therapy with a strong focus in diabetic eye disease.
Unique potential for long-interval dosing while
preserving the flexibility to dose monthly

- Across five tarcocimab tedromer pivotal studies for diabetic macular edema, diabetic retinopathy retinal vein occlusion and wet age-related macular degeneration.
- According to the product label for aflibercept, ranibizumab, faricimab and expectations for high-dose aflibercept (reported clinical data).
Five Phase 3 studies across high-prevalence retinal diseases with a focus in diabetic eye disease





Results from our Phase 1b and Phase 3 BEACON studies
Tarcocimab tedromer has consistently shown strong durability data in early and late-stage clinical studies.
BEACON Phase 3 study in RVO:
Tarcocimab Q8W was non-inferior to aflibercept Q4W1

- Tarcocimab Q8W is the first anti-VEGF therapy to demonstrate non-inferiority to monthly aflibercept while doubling the treatment interval from monthly to every-other-month dosing
- Tarcocimab Q8W improved BCVA and OCT CST comparably to aflibercept Q4W from baseline to Week 24 in all RVO patients
- Primary results available here
- The Phase 3 BEACON study is a global, multi-center, randomized study designed to evaluate the durability, efficacy and safety of tarcocimab tedromer Q8W vs. aflibercept Q4W in patients with macular edema due to RVO. NCT04592419 (https://clinicaltrials.gov/show/NCT04592419)
Phase 1b study in DME, RVO and wet AMD:
Over 2/3 of patients were on a 6-month or longer treatment-free interval at Year 12

- 69% of patients with DME were on a 6-month or longer treatment-free interval at Year 1 after 3 loading doses. Mean BCVA gain of +7.6 ETDRS letters
- 66% of patients with RVO and wet AMD were on a 6-month or longer treatment-free interval at Year 1 after 3 loading doses
- One-year results available here
- Phase 1b is a randomized, open label study to evaluate multidose safety, efficacy and durability in wet AMD, DME and RVO. 2.5 & 5 mg doses pooled. Includes only patients that received all (3) loading doses and either a) received a dose before Week 52 or b) did not receive a dose and were followed for at least six months after the last loading dose (Week 32 visit). Interval at Year 1 reflects the treatment interval ongoing at the Week 52 visit (where available) or the last interval before Week 52.
Tarcocimab tedromer and the ABC Platform are purposefully designed for increased durability

Tarcocimab tedromer aims to maintain therapeutic activity for longer.

An investigational first-in-class dual inhibitor biologic designed to address both vascular permeability and inflammation
Inflammation has been shown to play a significant role in retinal vascular disease. However, no treatments exist that concurrently address vascular permeability and inflammation.
KSI-501, our second investigational medicine built on our Antibody Biopolymer Conjugate (ABC) platform, is designed to inhibit VEGF and IL-6, a pro-inflammatory cytokine and immune growth factor, combining two powerful mechanisms of action to address retinal vascular disease and the underlying inflammatory cascade.
KSI-501: two powerful mechanisms of action that may enhance efficacy beyond anti-VEGFs

IL-6 levels are significantly elevated in eyes with retinal vascular disease and are implicated in anti-VEGF treatment resistance
Vitreous IL-6 levels in patients with retinal vascular disease vs. control1

DME: diabetic macular edema; PDR: prolifera tive diabetic retinopathy; BRVO: branch retinal vein occlusion; CRVO: central retinal vein occlusion; RD: retinal detachment.
1.Yoshimura et al. (2009). PLoS ONE 4(12): e8158.
Aqueous humor IL-6 levels significantly correlate with anti-VEGF treatment response in wet AMD2


A significant portion of DME patients (30%-66%) have evidence of persistent disease activity despite frequent anti-VEGF treatment
In pre-clinical studies, dual inhibition of VEGF and IL-6 by KSI-501 demonstrated normalization of inner and outer blood retinal barriers compared to anti-VEGF or anti-IL-6 monotherapy

Inner blood-retinal barrier: leakage from vascular endothelium disruption leads to macular edema and hemorrhage
Outer blood-retinal barrier: RPE integrity prevents choroidal vascularization from invading the retina
Orphan development path with concurrent exploration in high-prevalence retinal diseases
The ongoing Phase 1 study is in DME initially with the possibility to expand into uveitic macular edema (UME).

The initial pivotal program is likely to be in UME because there is a high unmet need for patients with UME:



Additional indications to be explored include DME and wet AMD. In DME, there is strong preclinical and clinical evidence on the role of IL-6 in driving inflammation and anti-VEGF treatment response. In wet AMD, there is preclinical evidence IL-6 is implicated in the development of exudative choroidal neovascularization and clinical evidence suggesting elevated aqueous IL-6 levels in patients with low or no anti-VEGF treatment response.
Our goal with KSI-501 is to develop a first-in-class biologic by combining two powerful mechanisms of action – anti-immune (new) and anti-permeability (core of therapy today) – for high-prevalence retinal disease and orphan disease UME
Multi-mechanism, multi-modality targeted biologic for complex retinal and systemic diseases
Triplet medicines combine the benefits of our Antibody Biopolymer Conjugate (ABC) Platform for long-interval dosing of biologics with a new feature that adds sustained release of 100s of small molecules to target three or more mechanisms of action, enabling treatment of complex, multifactorial diseases.
Designing a new generation of targeted therapy for high-prevalence multifactorial diseases

Our triplet ABC medicines aim to broaden what’s possible with antibody conjugate therapies
Antibody Drug Conjugate (ADC) therapies are revolutionizing the way cancer is treated today by delivering highly potent cancer-killing agents directly to cancer cells via a targeted antibody. With our ABC triplet medicines, we aim to build on this foundation in notable ways:
ADC
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ABC Triplet Medicine
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Our goal with our triplet medicines is to deliver greater therapeutic benefit for multifactorial diseases in the eye and systemically by modulating multiple distinct pathological processes in parallel