ONCOLOGY PIPELINE

Four Programs.
One Modular Platform.

KOP Therapeutics is advancing a pipeline of peptide-guided ASO programs across four solid tumor indications — all built on the same TGM delivery platform, enabling capital-efficient expansion from a single validated mechanism.

All programs are in preclinical development. IND-enabling studies are the next milestone for the lead program.

PROGRAM OVERVIEW

Active Pipeline Programs

PROGRAM
INDICATION
TARGET
STAGE
PROGRESS
KOP-101
LEAD
Prostate Cancer
CCND1 / BIRC5
In Vivo PoC
KOP-102
Breast Cancer
CCND1
In Vitro
KOP-103
Brain Cancer (GBM)
CCND1
In Vitro
KOP-104
Ovarian Cancer
CCND1
In Vitro
PROGRAM DETAILS

Program-by-Program Overview

KOP-101

Prostate Cancer

Lead Program

Target: CCND1 / BIRC5

Mechanism: CCND1 / BIRC5 ASO Knockdown

Lead program. In vivo proof-of-concept completed at Charles River Laboratories. Statistically significant TGI confirmed (p=0.049). 96-hr kill study (ED50/ED90) underway at Galenvs Sciences. Next: lead & construct optimization — finalize ASO leads and conjugate design, then advance 1–2 in vivo-ready conjugates into PK/PD studies by Q1 2027.

In Vitro Screen

In Vivo PoC (Charles River)

3

96-hr Kill Study (ED50/ED90) — Galenvs Sciences

4

Lead & Construct Optimization + Delivery Valency Selection

5

In Vivo PK/PD Studies — Q1 2027

6

IND-Enabling Studies

KOP-102

Breast Cancer

Active

Target: CCND1

Mechanism: CCND1 ASO Knockdown

In vitro knockdown confirmed across breast cancer cell lines. CCND1 overexpression is a validated driver in ER+ and HER2+ breast cancer subtypes.

In Vitro Screen

2

In Vivo PoC

3

96-hr Kill Study

4

IND-Enabling Studies

KOP-103

Brain Cancer (GBM)

Active

Target: CCND1

Mechanism: CCND1 ASO Knockdown

Glioblastoma multiforme (GBM) represents a high unmet need with limited treatment options. CCND1 overexpression is implicated in GBM proliferation and resistance.

In Vitro Screen

2

In Vivo PoC

3

96-hr Kill Study

4

IND-Enabling Studies

KOP-104

Ovarian Cancer

Active

Target: CCND1

Mechanism: CCND1 ASO Knockdown

Ovarian cancer has high CCND1 expression and limited targeted therapy options. KOP's peptide carrier targets LHRH receptors overexpressed on ovarian tumor cells.

In Vitro Screen

2

In Vivo PoC

3

96-hr Kill Study

4

IND-Enabling Studies

DEVELOPMENT ROADMAP

Path to IND-Enabling Studies

Completed

In Vitro Validation

Up to 95% knockdown · 95 constructs screened · 4 cancer types

Completed

In Vivo PoC (Charles River)

Statistically significant TGI · p=0.049 · PC3 xenograft · no observed toxicity

3

Active

96-hr Kill Study — Galenvs Sciences

ED50 / ED90 · Galenvs Sciences CRO · Montreal, QC · underway

4

Next

Lead & Construct Optimization

Finalize ASO leads · conjugate design · delivery valency selection · head-to-head uptake assay

5

Target: Q1 2027

In Vivo PK/PD Studies

1–2 in vivo-ready conjugates · PK/PD animal studies · candidate selection

6

Target

IND-Enabling Studies

GLP tox · CMC · FDA pre-IND meeting

Interested in the Pipeline?

Request our full partnering deck including pipeline details, preclinical data, and development milestones.

KOP Therapeutics

Advancing tumor-targeted RNA therapeutics toward IND-enabling development, partnership, and commercialization.

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