Product Description
Tamibarotene is an orally active, synthetic retinoid, developed to overcome all-trans retinoic acid (ATRA) resistance, with potential antineoplastic activity. As a specific retinoic acid receptor (RAR) alpha/beta agonist, tamibarotene is approximately ten times more potent than ATRA in inducing cell differentiation and apoptosis in HL-60 (human promyelocytic leukemia) cell lines in vitro. Due to a lower affinity for cellular retinoic acid binding protein (CRABP), tamibarotene may show sustained plasma levels compared to ATRA. In addition, this agent may exhibit a lower toxicity profile than ATRA, in part, due to the lack of affinity for the RAR-gamma receptor, the major retinoic acid receptor in the dermal epithelium. (Sourced from: https://pubchem.ncbi.nlm.nih.gov/compound/Tamibarotene)
Mechanisms of Action: RAR Agonist
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: Fast Track - Acute Myeloid LeukemiaFast Track - Myelodysplastic SyndromeOrphan Drug - Myelodysplastic Syndrome *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Syros
Company Location: CAMBRIDGE MA 02140
Company CEO: Nancy Simonian
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Austria, Belgium, Canada, Czech Republic, France, Germany, Hungary, Israel, Italy, Japan, Poland, Spain, United Kingdom, United States
Active Clinical Trial Count: 6
Highest Development Phases
Phase 3: Myelodysplastic Syndrome|Preleukemia
Phase 2: Acute Myeloid Leukemia|Arthrogryposis|Kidney Diseases, Cystic|Polycystic Kidney Diseases|Polycystic Kidney, Autosomal Dominant
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| SELECT-MDS-1 | P3 |
Active, not recruiting |
Myelodysplastic Syndrome |
2028-05-07 |
|
| SY-1425-202 | P2 |
Active, not recruiting |
Acute Myeloid Leukemia |
2028-04-01 |
|
| RP014-01 | P2 |
Recruiting |
Arthrogryposis|Polycystic Kidney Diseases|Kidney Diseases, Cystic|Polycystic Kidney, Autosomal Dominant |
2025-12-01 |
|
| SELECT MDS-1 | P3 |
Recruiting |
Preleukemia|Myelodysplastic Syndrome |
2024-11-15 |