INDICATION
RYTELO™ (imetelstat) is indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA). See more
GIVE YOUR ESA-RELAPSED/REFRACTORY RS-, RS+, OR ESA-INELIGIBLE
LR-MDS PATIENTS WITH TRANSFUSION-DEPENDENT ANEMIA
THE POSSIBILITY OF
ZERO TRANSFUSIONS1
In the IMerge phase 3, double-blind, placebo-controlled clinical trial, 39.8% (n=47/118) (95% CI, 30.9, 49.3) of patients who received RYTELO achieved red blood cell transfusion independence for ≥8 consecutive weeks vs 15% (n=9/60) with placebo (95% CI, 7.1, 26.6) (P<0.001)1
- Primary Endpoint: 39.8% achieved RBC-TI ≥8 consecutive weeks with RYTELO (95% CI, 30.9, 49.3) vs 15% with placebo (95% CI, 7.1, 26.6) (P<0.001)
- Secondary Endpoint: 28% (n=33/118) of patients treated with RYTELO achieved ≥24-week RBC-TI (95% CI, 20.1-37.0) vs 3.3% (n=2/60) of patients with placebo (95% CI, 0.4-11.5) (P<0.001)
- Secondary Endpoint: RYTELO ≥8-week RBC-TI responders experienced mDoR of 51.6-week RBC-TI (95% CI, 26.9, 83.9) vs 13.3-week RBC-TI with placebo (95% CI, 8.0, 24.9) (HR, 0.2; 95% CI, 0.1, 0.6)‡
Limitations: This analysis was prespecified, descriptive in nature, did not control for type 1 error, and should be interpreted with caution.
- Treatment was discontinued due to adverse reactions in 15% (n=17) of patients receiving RYTELO
- 7.6% (n=9) of RYTELO patients discontinued due to cytopenias vs 0% on placebo
- Most patients who experienced treatment-emergent adverse reactions were managed with dose delay and/or reductions
- 68.6% (n=81) of patients receiving RYTELO had a dose delay due to Grade ≥3 adverse reactions vs 23.7% (n=14) on placebo
- 49.2% (n=58) of patients receiving RYTELO had a dose reduction due to Grade ≥3 adverse reactions vs 6.8% (n=4) on placebo
- Serious adverse reactions occurred in 32% of patients who received RYTELO. Serious adverse reactions in >2% of patients included sepsis (4.2%), fracture (3.4%), cardiac failure (2.5%), and hemorrhage (2.5%). Fatal adverse reactions occurred in 0.8% of patients who received RYTELO, including sepsis (0.8%)
‡Not all patients treated with RYTELO experienced 51.6 weeks of continuous RBC-TI.3
ESA, erythropoiesis-stimulating agent; HR, hazard ratio; LR-MDS, lower-risk myelodysplastic syndromes; mDoR, median duration of response; NCCN, National Comprehensive Cancer Network; RBC-TI, red blood cell transfusion independence; RS, ring sideroblasts.
References: 1. RYTELO. Prescribing information. Geron Corp.; 2024. 2. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Myelodysplastic Syndromes V.1.2025. © National Comprehensive Cancer Network, Inc. 2024. All rights reserved. Accessed November 19, 2024. To view the most recent and complete version of the guideline, go online to NCCN.org. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way. 3. Platzbecker U and Santini V, et al. Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge): a multinational, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2024;403(10423):249-260. 4. Data on file. Geron Corporation. Foster City, CA.
INDICATION
RYTELO™ (imetelstat) is indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA).
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
Thrombocytopenia
RYTELO can cause thrombocytopenia based on laboratory values. In the clinical trial, new or worsening Grade 3 or 4 decreased platelets occurred in 65% of patients with MDS treated with RYTELO.
Monitor patients with thrombocytopenia for bleeding. Monitor complete blood cell counts prior to initiation of RYTELO, weekly for the first two cycles, prior to each cycle thereafter, and as clinically indicated. Administer platelet transfusions as appropriate. Delay the next cycle and resume at the same or reduced dose, or discontinue as recommended.
Neutropenia
RYTELO can cause neutropenia based on laboratory values. In the clinical trial, new or worsening Grade 3 or 4 decreased neutrophils occurred in 72% of patients with MDS treated with RYTELO.
Monitor patients with Grade 3 or 4 neutropenia for infections, including sepsis. Monitor complete blood cell counts prior to initiation of RYTELO, weekly for the first two cycles, prior to each cycle thereafter, and as clinically indicated. Administer growth factors and anti-infective therapies for treatment or prophylaxis as appropriate. Delay the next cycle and resume at the same or reduced dose, or discontinue as recommended.
Infusion-Related Reactions
RYTELO can cause infusion-related reactions. In the clinical trial, infusion-related reactions occurred in 8% of patients with MDS treated with RYTELO; Grade 3 or 4 infusion-related reactions occurred in 1.7%, including hypertensive crisis (0.8%). The most common infusion-related reaction was headache (4.2%). Infusion-related reactions usually occur during or shortly after the end of the infusion.
Premedicate patients at least 30 minutes prior to infusion with diphenhydramine and hydrocortisone as recommended and monitor patients for one hour following the infusion as recommended. Manage symptoms of infusion-related reactions with supportive care and infusion interruptions, decrease infusion rate, or permanently discontinue as recommended.
Embryo-Fetal Toxicity
RYTELO can cause embryo-fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with RYTELO and for 1 week after the last dose.
ADVERSE REACTIONS
Serious adverse reactions occurred in 32% of patients who received RYTELO. Serious adverse reactions in >2% of patients included sepsis (4.2%), fracture (3.4%), cardiac failure (2.5%), and hemorrhage (2.5%). Fatal adverse reactions occurred in 0.8% of patients who received RYTELO, including sepsis (0.8%).
Most common adverse reactions (≥10% with a difference between arms of >5% compared to placebo), including laboratory abnormalities, were decreased platelets, decreased white blood cells, decreased neutrophils, increased AST, increased alkaline phosphatase, increased ALT, fatigue, prolonged partial thromboplastin time, arthralgia/myalgia, COVID-19 infections, and headache.
Please see full Prescribing Information, including Medication Guide.
You are encouraged to report adverse events related to Geron products by calling 1-855-437-6664 (1-855-GERON-MI) (US only). If you prefer, you may contact the US Food and Drug Administration (FDA) directly. Visit www.fda.gov/MedWatch or call 1-800-FDA-1088.