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Incyte and Syndax Pharmaceuticals has received FDA approval for a new treatment for chronic graft-versus-host disease. Representatives forecast the drug will earn $650 million in sales. | DBT FILE PHOTO[/caption]
WILMINGTON — Earlier this month, Incyte and Syndax Pharmaceuticals have secured approval from the U.S. Food & Drug Administration as a treatment of chronic graft-versus-host disease (cGVHD), a potentially fatal complication that could occur after a bone marrow transplant or a stem cell transplant.
The drug Niktimvo expands on the Wilmington-based biotechnology company’s offerings to treat the cGVHD beyond its blockbuster drug Jakafi, which first received regulator approval back in 2011. Down the line, it may even replace Jakafi, said Incyte CEO Hervé Hoppenot.
“What we’re doing here is delivering a new mechanism, a very different one, to help patients that may not see that disease well controlled by Jakafi,” Hoppenot told the Delaware Business Times. “It's a franchise strategy and maybe over time Niktimvo could replace Jakafi because our team thinks it has characteristics that could make it a better product. Or it could be combined in treatments.”
Niktimvo is expected to hit the market in early 2025, just in time for the clock to start ticking on Incyte’s exclusive hold on Jakafi. The company holds exclusive rights to the drug until 2028.
Incyte had reported revenues topping $1 billion for the first time in the second quarter of 2024, with the small-molecule drug Jakafi royalties and sales bringing in $706 million. Jakafi, known generically as ruxolitinib, is used in the treatment of rare bone marrow cancer, blood diseases and in certain bone marrow donations.
Chronic graft-versus-host disease occurs in transplants when donor cells recognize the recipient's tissue cells as different, and attack them. That triggers a range of symptoms including skin rashes, liver dysfunction, gastrointestinal issues and other symptoms. More than 17,000 people in the United States are affected by it, according to Incyte representatives.
Of the patients who develop the cGVHD, nearly 50% require at least three lines of treatment, emphasizing the need for additional effective treatment options.
“It can lead to major quality of life decline, and even though it’s a rare disease, we think it’s important to make a massive impact on the lives of the people who do have it,” Hoppenot said.
Incyte formed a partnership with Syndax Pharmaceuticals to develop the drug back in 2021. Later, the two companies launched a three-year study on Niktimvo, and the results were strong. Among 75% of patients responded to the treatment in the first six months.
Among the responders, 60% of patients remained alive and did not need a new therapy for at least a year.
Niktimvo’s response rate did drop over time at higher doses. The drug is approved for use with adults and children who weigh at least 88.2 pounds, after the failure of at least two previous systemic therapies.
Hoppenot noted that the drug is unique, as it binds to the CSF-1R protein, which is used in the creation and survival of a type of white blood cells. These white blood cells can be drivers to the process involved in inflammation and fibrosis in cGVHD.
First treatments for cGVHD include steroids, but about half typically don’t respond so patients turn to more targeted treatments.
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Incyte CEO Herve Hoppenot addresses the crowd at the ribbon cutting for the company's new Wilmington lab in 2022. | DBT FILE PHOTO PHOTO BY JACOB OWENS[/caption]
“Only medicine can deal with the inflammation coming from that situation, and when we develop new data on how to use Niktimvo earlier in the disease, and we already have two studies we’re starting,” he said. “The life of this franchise is to ideally move from the late state to the early stage.”
Incyte is leading the commercialization of Niktimvo, leading 70% of the sales effort to tap into its existing customer relationships. Syndax has predicted that the drug will see a net profit of $650 million, with the profits split between the two companies.
What’s Next for Incyte
Looking ahead, Incyte is continuing to explore other ways to diversify its pipeline, and additional news about positive topline data on a third phase trail on its antibody Monjuvi is one strong line of business.
Monjuvi, a treatment for large B-cell lymphoma, showed positive landmarks in a inMIND trial for relapsed or follicular lymphoma. The study, which evaluated Monjuvi in combination with a combination of two other drugs, met primary endpoints for patients with the blood cancer without it worsening.
The goal is to target more patients at the start of care for lymphoma, and with Incyte’s results to prove Monjuvi can control the disease's progression. Hoppenot said that Incyte will be seeking FDA approval to expand the label on the drug.
“In a way, that’s typical of Incyte. It takes time, and while what we do may not always be successful, it's all part of the model we are following: innovate and go to places where no one has been before,” he said. “We have $1.7 billion per year in research and development, and we’re investing in early research for medicines that may hit the market in 2032.”
As the biopharmaceutical sector is known to need lot of lead time and significant funding up-front, Hoppenot said he sees Incyte’s pipeline of development stronger than it has been before, with drugs in long-term development as well as continuing studies on existing drugs to find surprising treatment pathways.
“It’s all based on the quality of science. I want to say you don’t have to be lucky, but I do think you have to be looking at places where the chance of being successful is the best,” Hoppenot said.
He pointed to Incyte’s franchises with autoimmune diseases and vitiligo treatments, like Opzelura, noting that researchers focused on those areas because of their understanding of connections between immunity and certain types of diseases.
“Once you realize that some diseases are in fact autoimmune, you can test hypotheses that are blocking the immune system in a certain way. We were one of the first companies to be in the field to be connecting those dots,” he said. “I think that’s what the quality of the science is at the end of the day, and what will make it successful. You have to be in the right place in the right field.”
