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REGN17235

Phase 1

Clonal Cytopenia of Undetermined Significance (CCUS) | Small molecule | Hematology |Regeneron Pharmaceuticals, Inc.|Last Updated: Aug 7, 2026

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment52

FDA Designations

No designations recorded

Clinical trial landscape

REGN17235 · 1 trial · 2 indications

Phase 1 1
NCT07753148Safety and Tolerability of REGN17235 in Adult Participants With Clonal Cytopenia of Undetermined Significance and Low-Risk Myelodysplastic Syndrome With SF3B1 MutationClonal Cytopenia of Undetermined Significance (CCUS)
NOT YET_RECRUITING52 Analytics
PHASE1NOT YET_RECRUITING
Safety and Tolerability of REGN17235 in Adult Participants With Clonal Cytopenia of Undetermined Significance and Low-Risk Myelodysplastic Syndrome With SF3B1 Mutation
Clonal Cytopenia of Undetermined Significance (CCUS)Unlock trial analytics

Study Endpoints

Primary Endpoints

Occurrence of Treatment Emergent Adverse Events (TEAEs)
Up to 5 years
Severity of TEAEs
Up to 5 years
Occurrence of Serious Adverse Events (SAEs)
Up to 5 years
Severity of SAEs
Up to 5 years

Secondary Endpoints

Hematologic improvement per International Working Group (IWG) 2018
Up to 5 years
Concentration of REGN17235 in serum
Up to 5 years
Occurrence of Anti-Drug Antibodies (ADA) to REGN17235 in serum
Up to 5 years
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part 1 Dose EscalationEXPERIMENTAL -
Part 2 Safety ExpansionEXPERIMENTAL -

Interventions

NameTypeDescription
REGN17235DRUGAdministered per the protocol
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo

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 ...

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Recent Changes (Last 90 Days)

LOWAug 7, 2026NCT07753148NEW_TRIAL: changed
LOWAug 7, 2026NCT07753148NEW_TRIAL: changed

Frequently asked questions about REGN17235

What is REGN17235 used for?

REGN17235 is an investigational small molecule being developed by Regeneron Pharmaceuticals for the treatment of Clonal Cytopenia of Undetermined Significance (CCUS) and low-risk myelodysplastic syndrome with SF3B1 mutation. It is currently in Phase 1 clinical development.

Who makes REGN17235?

REGN17235 is being developed by Regeneron Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker REGN. The drug is currently in Phase 1 clinical trials.

What phase is REGN17235 in?

REGN17235 is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. A Phase 1 trial is currently planned to evaluate its safety and tolerability in adult participants.

What clinical trials is REGN17235 in?

REGN17235 is being studied in a Phase 1 clinical trial with the identifier NCT07753148. The trial is designed to evaluate the safety and tolerability of REGN17235 in adult participants with Clonal Cytopenia of Undetermined Significance and low-risk myelodysplastic syndrome with SF3B1 mutation. The trial has not yet started recruiting.

Is REGN17235 the same as any other drug?

REGN17235 is the sole name provided for this investigational drug. It is not known to be marketed under any other name. The drug is being developed by Regeneron Pharmaceuticals for hematologic conditions.