Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
REGN17235 · 1 trial · 2 indications
| Arm | Type | Description |
|---|---|---|
| Part 1 Dose Escalation | EXPERIMENTAL | - |
| Part 2 Safety Expansion | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| REGN17235 | DRUG | Administered per the protocol |
Key Inclusion Criteria: 1. Presence of SF3B1 mutation in the bone marrow or peripheral blood AND diagnosis of low-risk MDS OR diagnosis of CCUS as defined by WHO 2022, 5th edition as described in the protocol 2. Adequate bone marrow function as described in the protocol; red blood cell transfusion ...
REGN17235 is an investigational small molecule being developed by Regeneron Pharmaceuticals for hematologic conditions marked by specific blood cell mutations. It is in Phase 1 clinical development for clonal cytopenia of undetermined significance, or CCUS, and is also being studied in low-risk myelodysplastic syndrome with an SF3B1 mutation.
REGN17235 is being studied as a treatment for clonal cytopenia of undetermined significance, a blood condition in which patients have low blood cell counts alongside an acquired mutation in blood-forming cells. It is investigational and has not been approved for this or any other use.
Regeneron Pharmaceuticals, Inc., which trades on Nasdaq under the ticker REGN, is the developer of REGN17235. The company is running the program in hematology, with the lead study enrolling adults who have clonal cytopenia of undetermined significance or low-risk myelodysplastic syndrome with an SF3B1 mutation.
REGN17235 is in Phase 1 clinical development. It is an investigational small molecule and has not been approved by the FDA. The program currently has one active Phase 1 trial that has not yet begun recruiting participants.
REGN17235 is being evaluated in the Phase 1 trial NCT07753148, titled Safety and Tolerability of REGN17235 in Adult Participants With Clonal Cytopenia of Undetermined Significance and Low-Risk Myelodysplastic Syndrome With SF3B1 Mutation. The study is not yet recruiting, is controlled, and plans to enroll 52 adults aged 18 years and older.
Yes. The REGN17235 Phase 1 trial NCT07753148 enrolls participants based on the presence of an SF3B1 mutation, so the study is biomarker selected. It includes adults with clonal cytopenia of undetermined significance or low-risk myelodysplastic syndrome who carry that mutation.