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Eflapegrastim

Phase 2

Solid Tumors | Small molecule | Oncology |Assertio Holdings, Inc.|Last Updated: May 22, 2026

Target and mechanism

Molecular targetCSF3R
Target classAgonist
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment40

FDA Designations

No designations recorded

Clinical trial landscape

Eflapegrastim · 2 trials · 4 indications

Phase 2 1Phase 1 1
NCT04570423A Study to Evaluate the Safety and Pharmacokinetics of Eflapegrastim in Pediatric Participants With Solid Tumors or Lymphomas and Treated With Myelosuppressive ChemotherapySolid Tumors
RECRUITING40 Analytics
PHASE2RECRUITING
A Study to Evaluate the Safety and Pharmacokinetics of Eflapegrastim in Pediatric Participants With Solid Tumors or Lymphomas and Treated With Myelosuppressive Chemotherapy
Solid TumorsUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Treatment Emergent Adverse Events (TEAEs)
From first dose of study drug to 35 days after the last dose of the study drug (Up to approximately 16 months)

An AE is defined as any untoward medical occurrence in a participant or clinical investigation participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. A TEAE is any AE that occurs from the first dose of the study drug until 35 days after the last dose of study drug, or on the day a new/additional chemotherapy regimen, or on the day another granulocyte-colony stimulating factor (G-CSF) is administered.

Time to Recovery of Absolute Neutrophil Count (ANC) From Nadir to ≥1.5×10^9/L in Cycle 1
Cycle 1 is 21 days

Time to ANC Recovery is defined as the time from chemotherapy administration until the patient's ANC increases to ≥1.5×10\^9/liter (L) after the expected nadir.

Secondary Endpoints

Percentage of Participants With Severe Neutropenia in Cycle 1
Cycle 1 (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy selected)
Time to Absolute Neutrophil Count (ANC) Recovery of Severe Neutropenia in Cycle 1
Cycle 1 (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy selected)
Number of Participants With Febrile Neutropenia in Cycle 1
Cycle 1 (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy selected)
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort 1: ≥12 to <17 yearsEXPERIMENTALParticipants will receive a SC injection of eflapegrastim after completion of each cycle of chemotherapy up to four cycles of treatment (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy regimen selected).
Cohort 2: ≥6 to <12 yearsEXPERIMENTALParticipants will receive a SC injection eflapegrastim after completion of each cycle of chemotherapy up to four cycles of treatment (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy regimen selected).
Cohort 3: ≥2 to <6 yearsEXPERIMENTALParticipants will receive a SC injection eflapegrastim after completion of each cycle of chemotherapy up to four cycles of treatment (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy regimen selected).
Cohort 4: ≥1 month to <2 yearsEXPERIMENTALParticipants will receive a SC injection eflapegrastim after completion of each cycle of chemotherapy up to four cycles of treatment (cycle length may vary and can be up to 28 days or more based on the type of chemotherapy regimen selected).
Early Phase: Eflapegrastim @ 30mins post TCEXPERIMENTALEflapegrastim (13.2 mg/0.6 mL fixed dose, equivalent to 3.6 mg granulocyte colony-stimulating factor \[G-CSF\]). Supplied in prefilled single-use syringes for subcutaneous injection. Cycle 1: Administered on the same day as TC chemotherapy, 30 minutes from the end of TC administration. Cycles 2-4: Administered 24 hours after TC chemotherapy administration. Each cycle is 21 days.
Early Phase: Eflapegrastim @ 3 hours post TCEXPERIMENTALEflapegrastim (13.2 mg/0.6 mL fixed dose, equivalent to 3.6 mg G-CSF). Supplied in prefilled single-use syringes for subcutaneous injection. Cycle 1: Administered on the same day as TC chemotherapy, 3 hours from the end of TC administration. Cycles 2-4: Administered 24 hours after TC chemotherapy administration. Each cycle is 21 days.
Early Phase: Eflapegrastim @ 5 hours post TCEXPERIMENTALEflapegrastim (13.2 mg/0.6 mL fixed dose, equivalent to 3.6 mg G-CSF). Supplied in prefilled single-use syringes for subcutaneous injection. Cycle 1: Administered on the same day as TC chemotherapy, 5 hours from the end of TC administration. Cycles 2-4: Administered 24 hours after TC chemotherapy administration. Each cycle is 21 days.
Expansion Phase: Eflapegrastim @ 30 mins post TCEXPERIMENTALEflapegrastim (13.2 mg/0.6 mL fixed dose, equivalent to 3.6 mg G-CSF). Supplied in prefilled single-use syringes for subcutaneous injection. Cycles 1-4: Administered on the same day as TC chemotherapy, 30 minutes following the end of TC administration. Each cycle is 21 days.

Interventions

NameTypeDescription
EflapegrastimDRUGEflapegrastim supplied in prefilled, single-use syringes for SC injection.
ChemotherapyDRUGChemotherapy agents may include doxorubicin, ifosfamide, docetaxel, CHOP regimen, etoposide, cyclophosphamide and vincristine which will be administered as per standard of care per the Primary Care physician's treatment plan.
DocetaxelDRUG75 mg/m\^2 IV infusion. Administered on Day 1 of each cycle.
CyclophosphamideDRUG600 mg/m\^2 IV infusion. Administered on Day 1 of each cycle.
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Eligibility Criteria

Age Range1 Month to 17 Years
SexALL
Healthy VolunteersNo
Study Sites5

Inclusion Criteria: 1. Participant must have a pathologic/histologic confirmed newly diagnosed/relapsed/recurrent solid tumor or lymphoma without bone marrow involvement. 2. Participant must be a candidate to receive myelosuppressive chemotherapy, with a febrile neutropenia rate of at least 20% as ...

Countries:United States
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Competitive Landscape -Other Solid Tumors 9 trials (matched to "Solid Tumors")

Frequently asked questions about Eflapegrastim

What is Eflapegrastim used for?

Eflapegrastim is an investigational monoclonal antibody being studied for the treatment of neutropenia and solid tumors. It is being evaluated in patients with breast cancer who develop neutropenia after chemotherapy, as well as in pediatric patients with solid tumors or lymphomas receiving myelosuppressive chemotherapy.

Who is developing Eflapegrastim?

Eflapegrastim is being developed by Assertio Holdings, Inc., a company traded on the stock exchange under the ticker ASRT. The company is conducting clinical trials to evaluate the drug's safety and efficacy in oncology settings.

What phase is Eflapegrastim in?

Eflapegrastim is in clinical development. A Phase 1 study in patients with breast cancer and neutropenia has been completed, and a Phase 2 study in pediatric patients with solid tumors or lymphomas is currently recruiting participants. The drug is investigational and not yet approved.

What clinical trials is Eflapegrastim in?

Eflapegrastim has been studied in two clinical trials. NCT04187898 is a completed Phase 1 study evaluating the duration of severe neutropenia after same-day dosing in breast cancer patients. NCT04570423 is a recruiting Phase 2 study assessing safety and pharmacokinetics in pediatric patients with solid tumors or lymphomas.

How does Eflapegrastim work?

Eflapegrastim is a monoclonal antibody designed to target and modulate biological pathways involved in neutrophil production. By binding to specific molecular targets, it aims to reduce the severity and duration of neutropenia caused by myelosuppressive chemotherapy in cancer patients.

Is Eflapegrastim the same as other drugs?

Eflapegrastim is a distinct investigational drug developed by Assertio Holdings. It is not known to be identical to any other approved medication. Its unique mechanism and formulation are being studied to determine its potential role in managing chemotherapy-induced neutropenia.