Specialised Therapeutics Nabs Australian Approval For Chronic Graft-Versus-Host Disease Therapy In Patients Receiving Blood Stem Cells

Key Points

• Specialised Therapeutics has received TGA approval for NIKTIMVO (axatilimab) in chronic graft-versus-host disease after at least two prior lines of systemic therapy.
• The approval covers adults and children ages 6 and older who weigh at least 40 kg, and makes Australia the first country to authorize the therapy after its FDA approval.
• The decision was based on phase 2 AGAVE-201 data, where the approved dose showed a 74% overall response rate and 60% of responders maintained that response at 12 months.

The FDA has approved hematopoietic (blood) stem cell transplants for a range of blood disorders, including leukemias, lymphomas, and severe aplastic anemia, and it appears to work quite well. However, sometimes patients end up with chronic graft-versus-host disease (cGVHD) following transplant.

cGVHD causes donor immune cells to attack healthy tissues in the recipient and can affect multiple organs, leading to inflammation, fibrosis, pain, stiffness, weakness, reduced function, and, in severe cases, multi-organ failure. Australia performs about 600 allogeneic stem cell transplants each year, and cGVHD affects about 40% to 50% of recipients.

In 2024, the FDA approved a couple of therapies for cGVHD, including Ryoncil, a mesenchymal stem cell therapy, and NIKTIMVO, an anti-CSF-1R antibody. The Australian TGA (their equivalent to the FDA) just announced NIKTIMVO is now approved there as well.

NIKTIMVO is a colony-stimulating factor-1 receptor (CSF-1R) blocking antibody. The company says it is the first approved anti-CSF-1R antibody for cGVHD. According to the company, scientists at QIMR Berghofer identified a cellular process involved in cGVHD and developed an antibody approach to block it, whereas Ryoncil likely acts as an immunomodulator through MSC paracrine signaling.

Clinical data and access

The TGA decision was based on the Phase 2 AGAVE-201 trial, which enrolled 241 adult and some pediatric patients with recurrent or refractory active cGVHD after at least two prior systemic therapies. The study included 121 sites across 16 countries, including Australia, Singapore, South Korea, and Taiwan.

• At the approved dose of 0.3 mg/kg every 2 weeks, the overall response rate was 74% (95% CI 63, 83).
• Among responders, 60% maintained a response at 12 months.
• The most common adverse events were dose-dependent transient laboratory abnormalities related to CSF-1R blockade.
• Infusion-related reactions occurred in 18% of patients in AGAVE-201, with Grade 3 or 4 reactions in 1.3%.
• In the 0.3 mg dose group, adverse events leading to discontinuation occurred in 6% of patients.

The company reported that the treatment was generally well tolerated, with a safety profile consistent with CSF-1R inhibition. Specialised Therapeutics is now exploring reimbursement through the Pharmaceutical Benefits Scheme, which subsidizes drugs for Australian patients.

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