Incyte builds GvHD presence with Niktimvo approval
Incyte's chief executive Hervé Hoppenot
The FDA has approved Incyte's axatilimab as a treatment for chronic graft-versus-host disease (GvHD), making it a companion to the company's Jakafi for the disorder.
The US regulator has cleared the anti-CSF-1R antibody under the Niktimvo brand for the treatment of chronic GvHD after failure of at least two prior lines of systemic therapy in adult and paediatric patients weighing at least 40 kg.
Incyte licensed axatilimab from Syndax Pharma in a 2021 deal – worth up to $600 million – that included an upfront payment of $117 million and gave it worldwide rights to the drug in all indications. Syndax, which licensed it in turn from UCB, has co-promotion rights to the drug in the US.
GvHD is a life-threatening condition that can occur after a stem cell transplant from a donor in which the donated cells initiate an immune response and attack the transplant recipient’s organs. In 2021, Incyte's oral JAK inhibitor Jakafi (ruxolitinib) became the first treatment for steroid-refractory acute GvHD in adults and children 12 years and older.
Jakafi is Incyte's biggest seller – contributing $2.6 billion last year across its uses in myelofibrosis, polycythemia vera, and GvHD out of the company's total sales of $3.7 billion – and analysts think Niktimvo could add another $1 billion to that total at its peak.
That will likely depend on Incyte expanding its label to move it higher up the treatment pathway. The company has said it intends to start a phase 3 trial of Niktimvo in combination with corticosteroids and a phase 2 trial of Niktimvo plus Jakafi – both as front-line options for GvHD – before the end of this year. Syndax, meanwhile, is also planning a phase 2 trial of the drug in idiopathic pulmonary fibrosis (IPF).
"With the approval of Niktimvo, patients with chronic GvHD, whose disease has progressed after prior therapies, now have a new treatment option with a novel mechanism of action to help address the serious and devastating complications associated with this disease,” said Hervé Hoppenot, Incyte's chief executive.
The FDA's decision is based on the results of the phase 2 AGAVE-201 trial, which was still ongoing when Incyte licensed Niktimvo three years ago. The overall response rate to treatment was 75% with Niktimvo, with a median time to first response of around six weeks and a median duration of response of 1.9 months.
Among patients who achieved a response, there were no deaths or need for new systemic therapy in 60% of patients over 12 months, according to the FDA.
Incyte said that around 42% of donor stem cell transplants result in GvHD, affecting approximately 17,000 patients in the US. Some of those cases will resolve with acute treatment, but among those who develop chronic GvHD around 50% require at least three lines of treatment.
