Incyte and Syndax Announce U.S. Food and Drug Administration (FDA) Approval of Niktimvo™ (axatilimab-csfr) 9 mg and 22 mg Vial Sizes

– U.S. launch expected in early February –

– Niktimvo is the first and only approved treatment for chronic GVHD that targets CSF-1R to reduce the drivers of inflammation and fibrosis –

– Pivotal data from the AGAVE-201 trial supporting FDA approval show treatment with Niktimvo resulted in durable responses across all organs studied and patient subgroups –

WILMINGTON, Del. and WALTHAM, Mass., Jan. 15, 2025 /PRNewswire/ -- Incyte (Nasdaq:INCY) and Syndax Pharmaceuticals (Nasdaq:SNDX) today announced that the U.S. Food and Drug Administration (FDA) has approved Niktimvo™ (axatilimab-csfr) in 9 mg and 22 mg vial sizes. The Companies expect product to be available for order in the U.S. in early February. Niktimvo is approved for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs). Niktimvo is the first and only FDA-approved prescription treatment for chronic GVHD that targets CSF-1R to reduce the drivers of inflammation and fibrosis.

"We are thrilled to build on our strong commitment to the GVHD community with the U.S. launch of Niktimvo, a first-in-class therapeutic agent that has demonstrated remarkable responses in patients with chronic GVHD whose response was suboptimal after at least two prior lines of systemic therapy," said Hervé Hoppenot, Chief Executive Officer, Incyte. "Our deep understanding of chronic GVHD and our connections in the clinical community will support a successful launch, in partnership with Syndax, of this important medicine for patients."

Niktimvo was approved by the FDA on August 14, 2024. The approval was based on positive data from the global AGAVE-201 trial, which were published in the New England Journal of Medicine in September 2024.1 The trial met the primary endpoint across all cohorts receiving Niktimvo with 75% of patients who received 0.3 mg/kg every two weeks achieving a response at six months of treatment (N=79).

"As the first and only FDA-approved anti-CSF-1R antibody targeting the drivers of inflammation and fibrosis in chronic GVHD, Niktimvo represents a major breakthrough for patient care," said Michael Metzger, Chief Executive Officer, Syndax. "Together with Incyte, we look forward to executing a robust commercial launch and advancing the treatment paradigm for patients with chronic GVHD who have progressed after at least two lines of systemic therapy."

Serious adverse reactions occurred in 44% of patients who received Niktimvo (N=79). Serious adverse reactions in > 2 patients included infection (pathogen unspecified) (14%), viral infection (14%), and respiratory failure (5.1%). Permanent discontinuation of Niktimvo due to an adverse reaction occurred in 10% of patients and dose reduction due to adverse reaction occurred in 8% of patients. Dose interruptions due to an adverse reaction occurred in 44% of patients. The adverse reactions leading to dose interruption in >2 patients were viral infection, infection (pathogen unspecified), bacterial infection, musculoskeletal pain and pyrexia.

The most common (≥15%) adverse reactions, including laboratory abnormalities, were increased aspartate aminotransferase (AST), infection (pathogen unspecified), increased alanine aminotransferase (ALT), decreased phosphate, decreased hemoglobin, viral infection, increased gamma glutamyl transferase (GGT), musculoskeletal pain, increased lipase, fatigue, increased amylase, increased calcium, increased creatine phosphokinase (CPK), increased alkaline phosphatase (ALP), nausea, headache, diarrhea, cough, bacterial infection, pyrexia and dyspnea.

The approved dose of Niktimvo for adults and pediatric patients weighing at least 40 kg is 0.3 mg/kg, up to a maximum dose of 35 mg, as an intravenous infusion over 30 minutes every two weeks. Niktimvo will be available for healthcare providers to order through a network of specialty distributors in both 9 mg vial and 22 mg vial sizes to facilitate patient dosing.

Incyte and Syndax are committed to supporting patients and removing barriers to ensure access to Niktimvo. Eligible patients in the U.S. who are prescribed Niktimvo have access to IncyteCARES (Connecting to Access, Reimbursement, Education and Support), a comprehensive program offering personalized patient support, including financial assistance for eligible patients and ongoing education and additional resources. More information about IncyteCARES is available by visiting http://www.incytecares.com or calling 1-855-452-5234.

On August 30, 2024, axatilimab-csfr (Niktimvo) was added to the latest NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) as a category 2A recommendation for the treatment of chronic GVHD after the failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg.2 Treatments are classified as category 2A when there is uniform NCCN consensus that the intervention is appropriate, based on lower-level evidence. The updated NCCN guidelines are available at www.nccn.org.

In the U.S., Incyte and Syndax are co-commercializing Niktimvo. Incyte has exclusive commercialization rights for Niktimvo outside of the U.S.

About Chronic Graft-Versus-Host Disease (GVHD)

Chronic GVHD is a serious condition that can occur after an allogeneic stem cell transplant (the transfer of stem cells from a donor) in which the donated cells initiate an immune response and attack the transplant recipient's organs. Chronic GVHD is a leading cause of significant morbidity and mortality after an allogeneic stem cell transplant and is estimated to develop in approximately 42% of transplant recipients, affecting approximately 17,000 patients in the U.S.3 Of those patients who develop chronic GVHD, nearly 50% require at least three lines of treatment, emphasizing the need for additional effective treatment options.4

About AGAVE-201

The global AGAVE-201 dose-ranging trial evaluated the efficacy, safety, and tolerability of axatilimab in 241 adult and pediatric patients with recurrent or refractory active chronic GVHD (GVHD) whose disease had progressed after two or more prior therapies. Patients were randomized 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 included duration of response, percent reduction in daily steroid dose, organ specific response rates and validated quality-of-life assessments using the Modified Lee Symptom Scale.

For more information about AGAVE-201, visit https://www.clinicaltrials.gov/study/NCT04710576.

About Niktimvo™ (axatilimab-csfr)

Niktimvo (axatilimab-csfr) is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody approved for use in the U.S. for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs).

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

Axatilimab 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. Axatilimab 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.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONSInfusion-Related ReactionsNiktimvo™ (axatilimab-csfr) 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%.

Premedicate with an antihistamine and an antipyretic for patients who have previously experienced an infusion-related reaction to Niktimvo. Monitor patients for signs and symptoms of infusion-related reactions, including fever, chills, rash, flushing, dyspnea, and hypertension. Interrupt or slow the rate of infusion or permanently discontinue Niktimvo based on severity of the reaction.

Embryo-Fetal ToxicityBased on its mechanism of action, Niktimvo may cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose.

ADVERSE REACTIONSSerious adverse reactions occurred in 44% of patients who received Niktimvo (N=79). Serious adverse reactions in >2 patients included infection (pathogen unspecified) (14%), viral infection (14%) and respiratory failure (5.1%). Permanent discontinuation of Niktimvo due to an adverse reaction occurred in 10% of patients and dose reduction due to adverse reaction occurred in 8% of patients. Dose interruptions due to an adverse reaction occurred in 44% of patients. The adverse reactions leading to dose interruption in >2 patients were viral infection, infection (pathogen unspecified), bacterial infection, musculoskeletal pain, and pyrexia.

The most common (≥15%) adverse reactions, including laboratory abnormalities, were increased aspartate aminotransferase (AST), infection (pathogen unspecified), increased alanine aminotransferase (ALT), decreased phosphate, decreased hemoglobin, viral infection, increased gamma glutamyl transferase (GGT), musculoskeletal pain, increased lipase, fatigue, increased amylase, increased calcium, increased creatine phosphokinase (CPK), increased alkaline phosphatase (ALP), nausea, headache, diarrhea, cough, bacterial infection, pyrexia, and dyspnea.

Clinically relevant adverse reactions in