# Phase IIb trial of CD24Fc for GVHD prophylaxis, IND 127,363 (01/29/2016)

> **NIH FDA R01** · ONCOIMMUNE, INC. · 2020 · $485,432

## Abstract

Project Summary/Abstract
 Because of the graft vs leukemia effect (GVL), allogeneic hematopoietic stem cell transplantation
(HSCT) is the only established curative therapy for high risk leukemia and myelodysplasia in adults. However,
the promise of HSCT is severely limited by graft versus host disease (GVHD). Acute graft-versus-host disease
(aGVHD) is a principal contributor to transplant related mortality (TRM), and develops in approximately 60-80%
of recipients receiving unrelated donor hematopoietic stem cell transplantation (HSCT) despite standard
immunosuppressive prophylaxis. Thus, novel GVHD prophylaxis treatments which successfully attenuate
aGVHD and related complications such as infection are urgently needed.
 CD24Fc has a novel mechanism of action that specifically targets the processes initiating GvHD, which
is being developed for use in disease prophylaxis. CD24Fc has been demonstrated to be safe in healthy
human subjects in a Phase I clinical trial (ClinicalTrials.gov Identifier:
NCT02650895)
and the current study, a
multi-center Phase II clinical trial, aims to provide the first toxicity/safety, pharmacokinetics, pharmacodynamics
and biological activity data for CD24Fc in patients with hematological malignancies receiving allogeneic HSCT.
The clinical trial has three primary objectives addressed by two different phases: Phase IIa will determine the
safety and tolerability of CD24Fc in a single ascending dose-escalation study, and define the recommended
Phase II dose (or Maximum Tolerated Dose) for prophylaxis of GVHD in a large Phase IIb expansion trial;
Phase IIb will assess the in-human activity of CD24Fc in leukemia and myeloid dysplasia syndrome patients
receiving HSCT; and both Phases will assess the pharmacokinetics (PK), pharmacodynamics (PD), and
immunogenicity of CD24Fc in patients receiving HSCT.
 The ongoing Phase IIa portion of the trial (ClinicalTrials.gov Identifier: NCT02663622) is a randomized
double blind single ascending dose trial comprised of 3 dosing cohorts of 8 patients (3:1 treatment:placebo),
for a total planned enrollment of 24 subjects. CD24Fc is administered to patients on a background of standard
of care prophylaxis comprising a calcineurin inhibitor and methotrexate to maximize patient safety. To date we
have enrolled 6 patients in the Phase IIa trial with no drug related SAEs or DLTs. In addition, we propose an
adaptive trial design that allows transition to the Phase IIb trial should our Phase IIa data demonstrate safety
based on an interim analysis for all patients at 100 days, rather than waiting until the data on the secondary
endpoints at one year.
 The planned Phase IIb study will involve a randomized, double blind, component where subjects will
receive either standard GVHD prophylaxis with CD24Fc at the RP2D determined in Phase IIa (Arm A) versus
standard GVHD prophylaxis with placebo (Arm B), with a planned enrollment of 90 patients per arm. This
portion of the study will evaluate whether comb...

## Key facts

- **NIH application ID:** 9752976
- **Project number:** 5R01FD006089-02
- **Recipient organization:** ONCOIMMUNE, INC.
- **Principal Investigator:** Yang Liu
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** FDA
- **Fiscal year:** 2020
- **Award amount:** $485,432
- **Award type:** 5
- **Project period:** 2018-09-05 → 2021-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9752976

## Citation

> US National Institutes of Health, RePORTER application 9752976, Phase IIb trial of CD24Fc for GVHD prophylaxis, IND 127,363 (01/29/2016) (5R01FD006089-02). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9752976. Licensed CC0.

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