New First-in-Class Therapy for Chronic Graft-Versus-Host Disease Approved in Australia[1]

• NIKTIMVO^® (axatilimab) approved by the Therapeutic Goods Administration (TGA) under Priority Review for the treatment of eligible Australian patients with chronic graft-versus-host disease^[2]
• Australia becomes the first country to obtain Marketing Authorisation for NIKTIMVO since approval by the US FDA^[3]
• Australian-led research played key role in the development of NIKTIMVO, a first-in-class novel colony stimulating factor-1 receptor (CSF-1R) blocking antibody.^[1],[4]

SINGAPORE, May 3, 2026 /PRNewswire/ -- Independent biopharmaceutical company Specialised Therapeutics is pleased to announce that NIKTIMVO^® (axatilimab) has been approved for use in Australia by the Therapeutic Goods Administration (TGA) for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and paediatric patients 6 years and older weighing at least 40 kg.

NIKTIMVO was assessed through the TGA's Priority Review pathway and is the first approved anti-colony stimulating factor-1 receptor (anti-CSF-1R) antibody for cGVHD.^[1],[2],[5] Through its novel mechanism of action, NIKTIMVO specifically targets the key drivers of inflammation and fibrosis in tissues throughout the body, helping to address the serious and potentially life-threatening complications associated with cGVHD.^[6],[7]

"Chronic GVHD is a significant long-term concern in patients with blood cancer requiring stem cell or bone marrow transplantation, and is characterised by multi-organ inflammation and fibrosis, leading to increased morbidity and non-relapse mortality," said Professor Jeff Szer, Clinical Haematologist at Peter MacCallum Cancer Centre and The Royal Melbourne Hospital.

"The Australian approval of NIKTIMVO is welcome news and will provide an important new treatment option for patients with chronic GVHD who continue to progress on other therapies, experience a higher symptom burden, and a decline in function and quality of life," said Professor Szer. "I urge the Government and Specialised Therapeutics to determine an appropriate pathway to reimbursement to ensure eligible patients can have equitable access to this therapy as soon as possible."

Graft-versus-host disease (GVHD) occurs when donated stem cells (the graft) mature into immune cells and attack the healthy cells in the patient's body (the host).^[8] Approximately  600 allogeneic stem cell transplants — where a patient receives stem cells from a donor to replace their diseased blood cells and repair their bone marrow — are carried out in Australia each year.^[8],[9] Chronic GVHD can occur at any time but typically develops 4-6 months after the transplant and affects approximately 40-50% of transplant recipients.^[7],[8],[10] In the majority of cases, three or more organs are involved, ranging from mild skin rashes, to muscle weakness, pain and joint stiffness, which can impair overall functional capacity, to potentially life-threatening muti-organ failure.^[8],[11]

In June 2025, Specialised Therapeutics (ST) entered into an exclusive partnering agreement with Incyte (NASDAQ:INCY) to commercialise NIKTIMVO in Australia, New Zealand and Singapore.

"Chronic GVHD remains an area of significant unmet need for Australians with blood cancer who have received donor stem cell or bone marrow transplants," said Carlo Montagner, ST Chief Executive Officer. "For patients who develop chronic GVHD, almost 50% will require at least three different therapies^{[7],[10],[11]}, reinforcing the need for additional effective treatment options."

The development of NIKTIMVO was made possible due to important pre-clinical Australian research, which commenced in 2014.^[4] Scientists from QIMR Berghofer made the world-first discovery, identifying both the cellular process that causes cGVHD, and the antibody that could block this process and prevent the disease's development.^[4]

"We are extremely proud to have secured local approval for NIKTIMVO, which would not have been possible without the pioneering work led by these Australian scientists over a decade ago," said Mr Montagner. "Chronic GVHD may lead to debilitating symptoms that can be extremely difficult to treat.^[9] As the first country to obtain Marketing Authorisation for NIKTIMVO since it was approved by the US FDA in August 2024^[3], we are pleased that it will soon be available for Australian prescribers and patients in need of new therapeutic options."

ST is currently exploring the reimbursement of NIKTIMVO through the Pharmaceutical Benefits Scheme (PBS) for eligible adults and patients 6 years and older with cGVHD.

The TGA approval of NIKTIMVO was based on data from the pivotal phase 2 global AGAVE-201 clinical trial, which evaluated its safety and efficacy in 241 adult and some paediatric patients with refractory cGVHD who received at least two prior lines of systemic therapy.^[12] AGAVE-201 involved 121 clinical trial sites across 16 countries, including patients from Australia, Singapore, South Korea and Taiwan.^[12]

The overall response rate (ORR) among patients in the study who received NIKTIMVO was 74% (95% CI 63, 83) at the approved dose (0.3 mg/kg every 2 weeks).^[13],[14] Additionally, 60% of patients maintained a response at 12 months.^[13],[14] In clinical studies, treatment with NIKTIMVO was generally well-tolerated, with a safety profile that was manageable and consistent with the mechanism of action of CSF-1R inhibition.^{[6],[13],[14]} The most common adverse events were dose-dependent transient laboratory abnormalities related to CSF-1R blockade.^{[6],[13],[14]}

For further details on NIKTIMVO, contact your healthcare professional and please refer to the approved Australian Consumer Medicine Information or Product Information available from ST Medical Information on 1300 798 820.

PBS Information: NIKTIMVO is not listed on the Pharmaceutical Benefits Scheme (PBS).

Important safety Information on NIKTIMVO^[14]

WARNINGS AND PRECAUTIONS: Infusion-Related Reactions – NIKTIMVO can cause infusion-related reactions. Infusion-related reactions, including hypersensitivity reactions, occurred in 18% of patients who received NIKTIMVO in the clinical trial (AGAVE-201), with Grade 3 or 4 reactions in 1.3%.

ADVERSE REACTIONS: The safety of axatilimab was evaluated in 79 patients with cGVHD enrolled in the AGAVE-201 study. No fatal or serious adverse reactions occurred in patients receiving axatilimab. No patients permanently discontinued axatilimab due to an adverse reaction. Dose interruptions due to an adverse reaction occurred in 3.8% of patients. The adverse reactions leading to dose interruption were elevated liver enzymes, amylase increased, fatigue, and lipase increased (1.3% each). The most common (≥ 15%) adverse reactions were fatigue, elevated liver enzymes, and infusion related reactions. Immunogenicity: Antidrug antibodies (ADAs) were assessed in 276 patients with cGVHD who received axatilimab. The incidence of axatilimab treatment-emergent ADAs was 33.7% (93/276) using a bridging enzyme-linked immunosorbent assay following a median exposure time of 7.8 months. Neutralising antibodies were detected in 47 of 93 cGVHD patients with treatment-emergent ADAs. There was no clinically significant effect of anti-axatilimab antibodies or neutralising antibodies on the pharmacokinetics, pharmacodynamics, safety, or effectiveness of axatilimab with the 0.3 mg/kg every 2 weeks dosage regimen.

USE IN SPECIFIC POPULATIONS: Lactation – Because of the potential for serious adverse reactions in a breastfed child, women should not breastfeed during treatment and for 30 days after the last dose of NIKTIMVO. Females and Males of Reproductive Potential – Pregnancy Testing, verify pregnancy status in females of reproductive potential prior to initiating NIKTIMVO; Contraception, females of reproductive potential should use effective contraception during treatment with NIKTIMVO and for 30 days after the last dose of NIKTIMVO. Embryo-Foetal Toxicity – based on its mechanism of action, NIKTIMVO may cause foetal harm when administered to a pregnant woman.

About NIKTIMVO^® (axatilimab)^[14]

NIKTIMVO is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody approved for use in the US in August 2024 for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and paediatric patients weighing at least 40 kg (88.2 lbs).^[1],[3]

In Australia, NIKTIMVO is indicated for the treatment of chronic graft-versus host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and paediatric patients 6 years and older weighing at least 40 kg.^[14]

▼ This medicine is included in the TGA Black Triangle Scheme. Please report suspected adverse events to the TGA.

In 2016, Syndax licensed exclusive worldwide rights to develop and commercialise NIKTIMVO from UCB. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialisation license agreement for NIKTIMVO in chronic GVHD and any future indications.

NIKTIMVO is being studied in frontline combination trials in chronic GVHD – a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774) are underway. NIKTIMVO is also being studied in an ongoing Phase 2 trial in patients with idiopathic pulmonary fibrosis (NCT06132256).

NIKTIMVO is a trademark of Incyte.

All other trademarks are the property of their respective owners.

About the AGAVE-201 Study^[13],[14]

AGAVE-201 was a global Phase 2, open-label, randomised, multicentre dose-ranging trial that evaluated the efficacy, safety and tolerability of axatilimab in 241 adult and paediatric patients with recurrent or refractory active chronic GVHD whose disease had progressed after two prior therapies. Patients were randomised to one of three treatment groups that investigated a distinct dose of axatilimab administered at 0.3 mg/kg every two weeks, 1.0 mg/kg every two weeks or 3.0 mg/kg every four weeks. The trial's primary endpoint was the proportion of patients in each dose group who achieved an objective response as defined by 2014 NIH Consensus Criteria for chronic GVHD by cycle 7 day 1. Secondary endpoints include duration of response, percent reduction in daily steroids dose, organ specific response rates and validated quality-of-life assessments using the modified Lee Symptom Scale. Adverse events leading to discontinuation of axatilimab occurred in 6% of the patients in the 0.3-mg dose group, 22% in the 1-mg dose group, and 18% in the 3-mg dose group. Visit https://www.clinicaltrials.gov/study/NCT04710576 for further information.

About Specialised Therapeutics

Founded in 2007, Specialised Therapeutics is an independent specialty pharmaceutical company, providing new therapies and technologies to patients in Australia, New Zealand and across Southeast Asia. Headquartered in Singapore, ST partners with global pharmaceutical, biotech and diagnostic companies to bring novel healthcare opportunities to patients who are impacted by a range of diseases. ST has built a strong track record of success, navigating complex regulatory, reimbursement and commercialisation environments in its diverse regions. The ST mission is to provide specialty therapies where there is an unmet need. The company's broad therapeutic portfolio currently includes novel agents in oncology, haematology, CNS, neurology, endocrinology, ophthalmology and supportive care, although it is not confined to these areas.     

Additional information can be found at www.stbiopharma.com.  

SOURCE Specialised Therapeutics