First-in-class opportunity
Represents a first-in-class therapeutic opportunity to directly target miR-221 in oncology.
Pipeline
Advancing LockMiR to treat multiple diseases
Our pipeline spans oncology, CNS diseases, and fibrosis, with lead candidate PRX-474 in clinical development for advanced solid tumors and emerging evidence of benefit in chemotherapy-induced peripheral neuropathy.
| Program | Indication | Preclinical | IND Enabling | Phase 1 | Phase 2 | Phase 3 |
|---|---|---|---|---|---|---|
| PRX-474 | Advanced Solid Tumors | |||||
| PRX-334 | CNS Diseases | |||||
| PRX-376 | Cancer & Fibrosis |
Lead Candidate
PRX-474 Inhibits miR-221
Broad therapeutic potential across multiple tumor types, covering 75-80% of all cancer indications.
Widely overexpressed
Overexpressed across multiple cancer types including hematological malignancies and solid tumors.
Suppresses tumor suppressors
Directly suppresses CDKN1B/p27 for cell cycle control and PTEN for tumor suppression.
Strong preclinical evidence
Inhibiting miR-221 using PRX-474 leads to impressive anti-tumor activity and upregulation of tumor suppressor genes.
Phase 1a Results: PRX-474
0grade 3-4 toxicities
56%disease control rate
44+months durable response