Pipeline Pipeline Overview

We leverage our OMEGA platform to advance a robust pipeline of development candidates that target a broad range of diseases.

Target Gene(s)

Indication

OEC

Discovery

Preclinical

Phase 1/2

Phase 3

Oncology

MYC

Hepatocellular carcinoma

OTX-2002*

Discovery

Preclinical

Phase 1/2

Phase 3

Phase 1/2 MYCHELANGELO™ Study

MYC

Non-small cell lung cancer

OTX-2101

Discovery

Preclinical

Phase 1/2

Phase 3

IND-Enabling Studies Ongoing

Undisclosed

Small cell lung cancer

Discovery

Preclinical

Phase 1/2

Phase 3

Multigenic Diseases

CXCL 1-8

Potential franchise of programs**

Discovery

Preclinical

Phase 1/2

Phase 3

Undisclosed

Idiopathic pulmonary fibrosis

Discovery

Preclinical

Phase 1/2

Phase 3

Regenerative Medicine

HNF4A

Liver regeneration

Discovery

Preclinical

Phase 1/2

Phase 3

Undisclosed

Corneal regeneration

Discovery

Preclinical

Phase 1/2

Phase 3

Monogenic Diseases

SFRP1

Alopecia

Discovery

Preclinical

Phase 1/2

Phase 3

Route of Administration (top to bottom): IV (HCC), IV (NSCLC), IV (SCLC), IV/Pulmonary, IV/Pulmonary (IPF), IV (Liver regeneration), Topical (Corneal regeneration), Topical (Alopecia)
*In November 2022, OTX-2002 received Orphan Drug Designation from the FDA for the treatment of hepatocellular carcinoma
**Potential indications for CXCL-targeting OEC include neutrophilic asthma, acute respiratory distress syndrome (including COVID-related), oncology, and dermatological and rheumatological indications

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