Blog

Nov 06, 2024

Syndax strikes $350M deal with Royalty Pharma for Niktimvo

With its first FDA approval coming in August and a second expected next month for another of its treatments, these are exhilarating days for 19-year-old Syndax Pharmaceuticals as it transforms into a

With its first FDA approval coming in August and a second expected next month for another of its treatments, these are exhilarating days for 19-year-old Syndax Pharmaceuticals as it transforms into a commercial enterprise.

To help foot the bill to launch its approved graft-versus-host disease drug Niktimvo (axatilimab) and the expected launch of its leukemia treatment revumenib, Syndax has struck a $350 million deal with Royalty Pharma.

In exchange for the $350 million upfront, Syndax will pay a 13.8% royalty on U.S. sales of Niktimvo. The deal expires after Royalty gains 2.35 times its investment.

“We expect this transaction to fund us through profitability, while ensuring that we continue to participate in the profits from Niktimvo and retain the upside of its future growth. With this significant infusion of capital, we are well positioned to successfully launch two first-in-class medicines and expand their opportunity with additional indications,” Michael Metzger, Syndax’s CEO, said in a release.

With its strong profile as a third-line treatment for chronic graft-versus-host disease (cGVHD) and its potential to move into earlier lines, Niktimvo has been pegged with peak sales potential of $1 billion by Leerink Partners. Syndax plans to launch Niktimvo in early 2025.

Syndax and its partner on Niktimvo outside of the U.S., Incyte, have begun a phase 2 trial combining the monoclonal antibody with Incyte’s GVHD therapy Jakafi. The companies also are preparing to begin a phase 3 study pairing Niktimvo with steroids. Both trials are for cGVHD.

“(Niktimvo) has the potential to address the serious and devastating complications associated with chronic GVHD, where there is clear unmet need for additional treatment options,” Pablo Legorreta, Royalty Pharma’s founder and CEO, said in a statement.

In 2016, Syndax gained exclusive rights to develop and commercialize axatilimab from UCB. Five years later, Incyte bought in to co-develop and co-commercialize axatilimab in cGVHD and future indications.

Niktimvo, a colony-stimulating factor 1 receptor (CSF-1R) inhibitor, excelled in a phase 2 trial as a 0.3-mg/kg dose every two weeks, triggering an overall response rate of 75%. The median time to first response was 1.5 months, and the response lasted a median 1.9 months before disease progression, death or new systemic therapies. Among the responders, 60% of patients remained alive and didn’t need new systemic therapy for at least a year since the response.

Before Niktimvo, the FDA had approved Daiichi Sankyo’s Turalio as the first CSF-1R inhibitor but for a rare cancer called tenosynovial giant cell tumor.

Meanwhile, Syndax’s revumenib is on tap for an FDA decision by Dec. 26 to treat adults and pediatric patients with relapsed/refractory KMT2Ar acute leukemia. The small-molecule menin inhibitor would become the first therapy for the acute leukemias, which are associated with drug resistance and poor prognosis.

Syndax will reveal its quarterly financial report later today, with a conference call set for 4:30 p.m. ET.