Database for
Therapeutic Antibodies
Therapeutic Antibodies
| Entry ID | 1741 |
| INN | Axatilimab |
| Status | Approved |
| Drug code(s) | SNDX-6352, UCB6352, INCA034176 |
| Brand name | Niktimvo |
| mAb sequence source | mAb humanized |
| General Molecular Category | Naked monospecific |
| Format, general category | Full length Ab |
| Format details | None |
| Isotype (Fc) | IgG4 |
| Light chain isotype | kappa |
| Linker | None |
| Ave. DAR | None |
| Conjugated/fused moiety | None |
| Discovery method/technology | None |
| Target(s) | CSF-1R |
| Indications of clinical studies | Idiopathic Pulmonary Fibrosis, Chronic Graft-versus-host-disease, COVID-19, solid tumors, Hodgkin's lymphoma, |
| Primary therapeutic area | Immune-mediated / inflammatory disorders |
| Most advanced stage of development (global) | Approved US |
| Status | Active |
| Start of clinical phase (IND filing or first Phase 1) | October 15, 2016 |
| Start of Phase 2 | May 30, 2020 |
| Start of Phase 3 | |
| Date BLA/NDA submitted to FDA | December 28, 2023 |
| Year of first approval (global) | 2024 |
| Date of first US approval | August 14, 2024 |
| INN, US product name | Axatilimab, axatilimab-csfr |
| US or EU approved indications | Treatment of chronic graft-vs-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. |
| Company | UCB |
| Licensee/Partner | Syndax Pharmaceuticals Inc, Incyte |
| Comments about company or candidate | Sep 2024: Pivotal study results published https://www.nejm.org/doi/full/10.1056/NEJMoa2401537 On August 14 2024, the U.S. Food and Drug Administration (FDA) approved axatilimab-csfr (Niktimvo), a colony-stimulating factor–1 receptor–blocking antibody, for the treatment of chronic graft-vs-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. U.S. Food and Drug Administration has accepted for Priority Review the Biologics License Application (BLA) for axatilimab, an anti-CSF-1R antibody, for the treatment of chronic graft-versus-host disease after failure of at least two prior lines of systemic therapy. The Prescription Drug User Fee Act (PDUFA) date for the FDA decision is August 28, 2024. Jan 2 2024 press release: The Biologics License Application for axatilimab, an anti-CSF-1R antibody, in adult and pediatric patients six years or older with chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy was submitted to the FDA on December 28, 2023. Syndax also announced that it has exercised its option under the Company's 2021 collaboration agreement with Incyte to co-commercialize axatilimab in the U.S. July 2023: Syndax Pharmaceuticals and Incyte announced positive topline data from the pivotal AGAVE-201 trial of axatilimab, an anti-CSF-1R antibody, in adult and pediatric patients with chronic graft-versus-host disease (GVHD) following two or more prior lines of therapy. Based on these results, and pending agreement from the U.S. Food and Drug Administration (FDA), Syndax and Incyte intend to submit a Biologics License Application (BLA) to the FDA by year-end 2023. Sep 2022: The Company and its partner, Incyte, announced completion of enrollment in the pivotal AGAVE-201 trial evaluating axatilimab in patients with cGVHD following two or more prior lines of therapy. The trial is evaluating the safety and efficacy of three dosing regimens of axatilimab. The primary endpoint will assess objective response rate based on the 2014 NIH consensus criteria for cGVHD, with key secondary endpoints including duration of response and improvement in modified Lee Symptom Scale score. The Company expects to report topline data in mid-2023, with the expectation for a BLA filing later in 2023. Q2 2022: Top line results from Pivotal Phase 2 (3L+ cGVHD) expected in 2023. Syndax Pharmaceuticals and Incyte have signed an exclusive global partnership and licence agreement to develop and market the former’s anti-CSF-1R monoclonal antibody, axatilimab. Incyte will oversee the international (outside US) commercialisation activities of axatilimab for all indications. NCT04415073 in COVID-19 Suspended in Aug 2020. (Given enrollment challenges, partly attributable to the constantly changing COVID-19 treatment landscape, the trial has been suspended.); it was A Phase 2 Study to Evaluate Axatilimab for Hospitalized Patients With Respiratory Involvement Secondary to COVID-19. NCT03604692 Phase 1 in graft vs. host disease started in Nov 2018. Jan 2018: Syndax and AstraZeneca will collaborate on a non-exclusive basis to evaluate the combination of SNDX-6352 and durvalumab in multiple solid tumor types. Syndax expects to initiate a Phase Ib study in the first half of 2018 to establish the safety and recommended dose regimen of SNDX-6352 in combination with durvalumab. Data from this study will enable both companies to sponsor, design and initiate subsequent Phase II studies aimed at exploring the safety and efficacy of the combination across a number of defined tumor types. According to Nov 10, 2016 press release, Syndax commenced enrollment in the Phase 1 single ascending dose clinical trial of SNDX-6352 in healthy volunteers to determine the safety and pharmacokinetics of the anti-CSF-1R monoclonal antibody. SNDX-6352 is expected to be developed to treat a variety of cancers Axatilimab was granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with chronic GVHD and IPF. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab. Axatilimab is being developed under an exclusive worldwide license from UCB entered into between Syndax and UCB in 2016. |
| Full address of company | Allée de la Recherche, 60 1070 Brussels Belgium Europe Belgium https://www.ucb.com/contact |
Hinge stabilized (S228P) Syndax licensed UCB's preclinical CSF1R mAb in July 2016. UCB6352 is a high affinity antibody targeting the colony stimulating factor 1 receptor (CSF-1R) with preclinical evidence of anti-tumor and anti-metastatic efficacy. CSF-1R is expressed on monocytes and macrophages and activated through its ligands, IL-34 and CSF-1. UCB6352 inhibition of CSF-1R signaling results in an enhanced preclinical anti-tumor immune response through the reduction in the number and activation status of immunosuppressive tumor promoting macrophages.
| Anticipated events | None |
| Factor(s) contributing to discontinuation | None |