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BEAM-103

Phase 1

Undisclosed | Small molecule | Other |Beam Therapeutics Inc.|Trials Updated: Dec 26, 2025

BEAM-103 development status

Highest phase Phase 1
Registered trials 1 across 1 sponsor since Dec 2025

BEAM-103 target and mechanism

Molecular targetCD117
Target classProtein
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedPLACEBO_CONTROLLED
Total Trials1
Total Enrollment30

FDA Designations

No designations recorded

BEAM-103 clinical trials

BEAM-103 · 1 trial · 2 indications

Phase 1 1
NCT07304791This is a Phase 1, Randomized, Single-blind, Placebo-controlled Study to Assess the Safety, Pharmacokinetics (PK), and Pharmacodynamics (PD) of BEAM-103 in Healthy SubjectsHealthy Subjects
RECRUITING30 Analytics
PHASE1RECRUITING
This is a Phase 1, Randomized, Single-blind, Placebo-controlled Study to Assess the Safety, Pharmacokinetics (PK), and Pharmacodynamics (PD) of BEAM-103 in Healthy Subjects
Healthy SubjectsUnlock trial analytics

Study Endpoints

Primary Endpoints

To determine the safety and tolerability of single ascending doses of BEAM-103
from enrollment to the end of study at 5 months

Incidence of treatment-emergent AEs, serious AEs, severe AEs, AEs related to BEAM-103

Secondary Endpoints

Change in Free Stem Cell Factor (SCF) in Peripheral Blood Over Time
from the start of treatment to the end of study at 5 months
Change in Tryptase Level in Peripheral Blood Over Time
from the start of treatment to the end of study at 5 months
Change in Reticulocyte Count in Peripheral Blood Over Time
From enrollment to end of study at 5 months
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Study Design & Arms

AllocationRANDOMIZED
MaskingSINGLE
ModelSEQUENTIAL
PurposeBASIC_SCIENCE

Treatment Arms

ArmTypeDescription
BEAM-103 Drug ProductEXPERIMENTALBEAM-103 is a monoclonal antibody that will be administered in single dose ascending dose cohorts as an intravenous infusion through the arm.
PlaceboPLACEBO_COMPARATORNormal Saline Solution will be administered as a matching place in single dose ascending dose cohorts as an intravenous infusion through the arm.

Interventions

NameTypeDescription
BEAM-103DRUG• BEAM-103 is a monoclonal antibody administered as a single intravenous dose
Placebo ComparatorDRUG• The matching placebo comparator is administered as a single intravenous dose
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Eligibility Criteria

Age Range18 Years to 55 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * Ages 18 to ≤55 years * Body mass index (BMI) of 18.5 to 25 kg/m2 * Morning neutrophil count ≥2.6 × 109 cells/L for Black/African American subjects or ≥3.5 ×109 cells/L for Caucasian/Other subjects * Baseline hemoglobin value of ≥14 g/dL for males and ≥12 g/dL for females * Bas...

Countries:United Kingdom
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Frequently asked questions about BEAM-103

What is BEAM-103?

BEAM-103 is an investigational small molecule developed by Beam Therapeutics Inc. It is being studied in a Phase 1 clinical trial in healthy adult subjects in the United Kingdom. The study is designed to assess safety, pharmacokinetics, and pharmacodynamics of the drug candidate.

What does BEAM-103 target?

BEAM-103 targets CD117, a protein also known as KIT. CD117 is a receptor tyrosine kinase. By binding this protein target, BEAM-103 is designed to modulate CD117-related signaling, which is the basis for its evaluation in early-stage clinical testing.

Who makes BEAM-103?

BEAM-103 is developed by Beam Therapeutics Inc., a biotechnology company that trades on the Nasdaq under the ticker BEAM. The company is the sponsor of the Phase 1 study evaluating BEAM-103 in healthy volunteers.

What phase is BEAM-103 in?

BEAM-103 is in Phase 1 clinical development. It is investigational and has not been approved by the FDA. The ongoing Phase 1 trial is a randomized, single-blind, placebo-controlled study in healthy subjects.

What clinical trials is BEAM-103 in?

BEAM-103 is being evaluated in one clinical trial, NCT07304791. This Phase 1 study is randomized, single-blind, and placebo-controlled, with an planned enrollment of 30 healthy subjects. It is currently recruiting participants in the United Kingdom and is designed to assess safety, pharmacokinetics, and pharmacodynamics.