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Niktimvo™ (axatilimab-csfr) was studied in a randomized, open-label, multicenter phase 2 trial1,2

Niktimvo was approved by the FDA for cGVHD after 2 lines of systemic therapy in adults and pediatric patients weighing at least 40 kg, based on positive results from the AGAVE-201 trial1,3

 

Patients with recurrent or refractory cGVHD

Randomization*

Recommended therapeutic dose:

0.3 mg/kg IV Q2W (N=79)

*Patients were randomized to 1 of 3 treatment groups that investigated a distinct dose of Niktimvo administered at 0.3 mg/kg Q2W, 1.0 mg/kg Q2W, and 3.0 mg/kg Q4W until disease progression, lack of response by 9 months, or unacceptable toxicity.3

Niktimvo 0.3 mg/kg IV Q2W

was selected as the recommended therapeutic dose1

Primary endpoint1,3:

  • ORR by cycle 7, day 1 of treatment. Responses were defined by 2014 NIH consensus criteria

Secondary and exploratory endpoints2-4:

  • mLSS, DOR, and FFS

Niktimvo was studied in a diverse range of patients, the majority of whom had previous exposure to ruxolitinib (72%) or another T- and B-cell–modulating therapy1,3

Demographics and baseline characteristics of patients with cGVHD10.3 mg/kg Q2W 
(N=79)
Median age, years (range)50 (7-76)
Age ≥65 years, n (%)21 (27)
Male, n (%)46 (58)
Race, n (%) 
White67 (85)
Asian4 (5)
Black2 (3)
Other1 (1)
Not reported5 (6)
Median number of months (range) from cGVHD diagnosis47 (4-211)
≥4 organs involved, n (%)45 (57)
Median number of prior lines of therapy (range)4 (2-12)
Number of prior lines of therapy, n (%) 
211 (14)
314 (18)
417 (22)
≥537 (47)
Prior cGVHD treatment with ibrutinib, n (%)a27 (34)
Prior cGVHD treatment with ruxolitinib, n (%)a57 (72)
Prior cGVHD treatment with belumosudil, n (%)a16 (20)
Refractory to last therapy, n (%)37 (47)
Severe cGVHD, n (%)63 (80)
Median Lee Symptom Scale score at baseline (range)24 (4-55)

aNo systemic cGVHD treatments other than those listed were allowed in the study.1,3

A high percentage of patients enrolled in the study had a wide range of organ involvement and organs prone to inflammation and fibrosis at baseline, including skin (80%), lungs (40%), and joints/fascia (69%)3

cGVHD=chronic graft-versus-host disease; DOR=duration of response; FFS=failure-free survival; IV=intravenous; mLSS=modified Lee Symptom Scale; NIH=National Institutes of Health; ORR=overall response rate; Q2W=every 2 weeks; Q4W=every 4 weeks.

AXA_Macrophage_BrandArt_silo

References: 1. Niktimvo Prescribing Information. Wilmington, DE: Incyte Corporation. 2. A study of axatilimab at 3 different doses in participants with chronic graft versus host disease (cGVHD) (AGAVE-201). ClinicalTrials.gov. Updated June 14, 2024. Accessed August 17, 2024. https://clinicaltrials.gov/study/NCT04710576. 3. Wolff D, Cutler C, Lee SJ, et al; for the AGAVE-201 Investigators. Axatilimab in recurrent or refractory chronic graft-versus-host disease. N Engl J Med. 2024;391(11):1002-1014. Supplementary appendix available at: https://www.nejm.org/doi/full/10.1056/NEJMoa2401537. 4. Data on file. Incyte Corporation. Wilmington, DE.

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Indications and Usage

Niktimvo™ (axatilimab-csfr) is a colony stimulating factor-1 receptor (CSF-1R)-blocking antibody indicated for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg.

Important Safety Information

WARNINGS AND PRECAUTIONS

Infusion-Related Reactions

Niktimvo™ (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 Toxicity

Based 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 REACTIONS

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.

Clinically relevant adverse reactions in < 10% of patients who received Niktimvo included:

  • Eye disorders: periorbital edema
  • Skin and subcutaneous skin disorders: pruritus
  • Vascular disorders: hypertension

Immunogenicity: Anti-Drug Antibody–Associated Adverse Reactions

Across treatment arms in patients with cGVHD who received Niktimvo in clinical trials, among the patients who developed anti-drug antibodies (ADAs), hypersensitivity reactions occurred in 26% (13/50) of patients with neutralizing antibodies (NAb) and in 4% (2/45) of those without NAb.

USE IN SPECIFIC POPULATIONS

Lactation

Because of the potential for serious adverse reactions in a breastfed child, advise women not to breastfeed during treatment and for 30 days after the last dose of Niktimvo.

Females and Males of Reproductive Potential

Pregnancy Testing

Verify pregnancy status in females of reproductive potential prior to initiating Niktimvo.

Contraception

Females
Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose of Niktimvo.

DOSAGE AND ADMINISTRATION

Dosage Modifications for Adverse Reactions

Monitor aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP), creatine phosphokinase (CPK), amylase, and lipase prior to the start of Niktimvo therapy, every 2 weeks for the first month, and every 1 to 2 months thereafter until abnormalities are resolved. See Table 1 in the Prescribing Information for more recommendations.

Please see Full Prescribing Information for Niktimvo.

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Indications and Usage

Niktimvo™ (axatilimab-csfr) is a colony stimulating factor-1 receptor (CSF-1R)-blocking antibody indicated for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg.

Important Safety Information

WARNINGS AND PRECAUTIONS

Infusion-Related Reactions

Niktimvo™ (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 Toxicity

Based 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 REACTIONS

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.

Clinically relevant adverse reactions in < 10% of patients who received Niktimvo included:

  • Eye disorders: periorbital edema
  • Skin and subcutaneous skin disorders: pruritus
  • Vascular disorders: hypertension

Immunogenicity: Anti-Drug Antibody–Associated Adverse Reactions

Across treatment arms in patients with cGVHD who received Niktimvo in clinical trials, among the patients who developed anti-drug antibodies (ADAs), hypersensitivity reactions occurred in 26% (13/50) of patients with neutralizing antibodies (NAb) and in 4% (2/45) of those without NAb.

USE IN SPECIFIC POPULATIONS

Lactation

Because of the potential for serious adverse reactions in a breastfed child, advise women not to breastfeed during treatment and for 30 days after the last dose of Niktimvo.

Females and Males of Reproductive Potential

Pregnancy Testing

Verify pregnancy status in females of reproductive potential prior to initiating Niktimvo.

Contraception

Females
Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose of Niktimvo.

DOSAGE AND ADMINISTRATION

Dosage Modifications for Adverse Reactions

Monitor aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP), creatine phosphokinase (CPK), amylase, and lipase prior to the start of Niktimvo therapy, every 2 weeks for the first month, and every 1 to 2 months thereafter until abnormalities are resolved. See Table 1 in the Prescribing Information for more recommendations.

Please see Full Prescribing Information for Niktimvo.

Niktimvo and the Niktimvo logo are trademarks of lncyte.

lncyte and the lncyte logo are registered trademarks of lncyte.

Niktimvo (axatilimab) is licensed from Syndax.

© 2024, Incyte. MAT-AXA-00044  09/24