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
MYC
Non-small cell lung cancer
OTX-2101
Discovery
Preclinical
Phase 1/2
Phase 3
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
Learn more about our policy on expanded access to investigational drugs.
