First-in-class opportunity
Represents a first-in-class therapeutic opportunity to directly target miR-221 in oncology.
Pipeline
Using Precision LNA Modified ASO Chemistry to treat diseases.
Our pipeline spans cancer 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 | Cancer (HCC and CRC) | |||||
| PRX-334 | Cancer | |||||
| PRX-376 | Cancer & Fibrosis |
Lead Candidate
PRX-474 Inhibits miR-221
microRNA-221 (miR-221) simultaneously silences multiple tumor suppressor targets — a single hub controlling proliferation, survival, and apoptosis, and is widely expressed across multiple solid tumor types.
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
55+months durable response