Geron Announces Updated NCCN Guidelines® Recommending RYTELO™ (imetelstat) for the Treatment of Symptomatic Anemia in Patients with Lower-Risk MDS
Geron (Nasdaq: GERN) announced that the National Comprehensive Cancer Network (NCCN) has updated its Clinical Practice Guidelines for Myelodysplastic Syndromes (MDS) to recommend RYTELO™ (imetelstat) as a Category 1 and 2A treatment for symptomatic anemia in lower-risk MDS patients. RYTELO is recommended as a Category 1 second-line treatment for both RS+ and RS- patients with serum EPO ≤500 mU/mL, and as a Category 2A first-line treatment for patients with serum EPO >500 mU/mL. This update follows the FDA's approval of RYTELO in June 2024 for treating adult patients with low- to intermediate-1 risk MDS with transfusion-dependent anemia. The company believes these updates will increase awareness and uptake of RYTELO as a new treatment option for eligible lower-risk MDS patients.
Geron (Nasdaq: GERN) ha annunciato che il National Comprehensive Cancer Network (NCCN) ha aggiornato le sue Linee Guida Cliniche per le Sindromi Mielodisplastiche (MDS) raccomandando RYTELO™ (imetelstat) come trattamento di Categoria 1 e 2A per l'anemia sintomatica nei pazienti MDS a basso rischio. RYTELO è raccomandato come un trattamento di seconda linea di Categoria 1 sia per i pazienti RS+ che per i RS- con EPO sierica ≤500 mU/mL, e come un trattamento di prima linea di Categoria 2A per i pazienti con EPO sierica >500 mU/mL. Questo aggiornamento segue l'approvazione della FDA per RYTELO nel giugno 2024 per il trattamento di pazienti adulti con MDS a rischio basso e intermedio-1 con anemia da trasfusione. L'azienda crede che questi aggiornamenti aumenteranno la consapevolezza e l'adozione di RYTELO come nuova opzione terapeutica per i pazienti MDS a basso rischio idonei.
Geron (Nasdaq: GERN) anunció que el National Comprehensive Cancer Network (NCCN) ha actualizado sus Guías de Práctica Clínica para los Síndromes Mielodisplásicos (MDS) para recomendar RYTELO™ (imetelstat) como tratamiento de Categoría 1 y 2A para la anemia sintomática en pacientes de bajo riesgo con MDS. RYTELO se recomienda como tratamiento de segunda línea de Categoría 1 tanto para pacientes RS+ como RS- con EPO sérica ≤500 mU/mL, y como tratamiento de primera línea de Categoría 2A para pacientes con EPO sérica >500 mU/mL. Esta actualización sigue a la aprobación de la FDA de RYTELO en junio de 2024 para tratar a pacientes adultos con MDS de bajo a intermedio-1 riesgo con anemia dependiente de transfuciones. La empresa cree que estas actualizaciones aumentarán la conciencia y la adopción de RYTELO como una nueva opción de tratamiento para los pacientes elegibles de bajo riesgo con MDS.
Geron (Nasdaq: GERN)은 국가 종합암 네트워크(NCCN)가 골수 이형성 증후군(MDS)에 대한 임상 실무 지침을 업데이트하여 RYTELO™ (imetelstat)를 저위험 MDS 환자의 증상성 빈혈에 대한 카테고리 1 및 2A 치료로 추천했다고 발표했습니다. RYTELO는 혈청 EPO가 ≤500 mU/mL인 RS+ 및 RS- 환자 모두에 대해 카테고리 1 2차 치료로 추천되며, 혈청 EPO가 >500 mU/mL인 환자에 대해서는 카테고리 2A 1차 치료로 추천됩니다. 이 업데이트는 2024년 6월 FDA가 수혈 의존성 빈혈을 가진 저위험 및 중간-1 위험 MDS 성인 환자 치료를 위해 RYTELO를 승인한 뒤 이루어진 것입니다. 회사는 이러한 업데이트가 저위험 MDS 환자에게 적합한 새로운 치료 옵션으로 RYTELO에 대한 인지도와 채택을 증가시킬 것이라고 믿고 있습니다.
Geron (Nasdaq: GERN) a annoncé que le National Comprehensive Cancer Network (NCCN) a mis à jour ses directives de pratique clinique pour les syndromes myélodysplasiques (MDS) afin de recommander RYTELO™ (imetelstat) comme traitement de catégorie 1 et 2A pour l'anémie symptomatique chez les patients MDS à faible risque. RYTELO est recommandé comme traitement de deuxième ligne de catégorie 1 pour les patients RS+ et RS- avec une EPO sérique ≤ 500 mU/mL, et comme traitement de première ligne de catégorie 2A pour les patients avec une EPO sérique > 500 mU/mL. Cette mise à jour fait suite à l'approbation par la FDA de RYTELO en juin 2024 pour le traitement des patients adultes atteints de MDS à risque faible à intermédiaire-1 avec une anémie dépendante de transfusion. L'entreprise croit que ces mises à jour augmenteront la sensibilisation et l'adoption de RYTELO en tant que nouvelle option de traitement pour les patients MDS à faible risque éligibles.
Geron (Nasdaq: GERN) gab bekannt, dass das National Comprehensive Cancer Network (NCCN) seine klinischen Praxisrichtlinien für myelodysplastische Syndrome (MDS) aktualisiert hat, um RYTELO™ (imetelstat) als Behandlung der Kategorie 1 und 2A für symptomatische Anämie bei MDS-Patienten mit niedrigem Risiko zu empfehlen. RYTELO wird als zweitlinige Behandlung der Kategorie 1 für sowohl RS+ als auch RS- Patienten mit Serum-EPO ≤500 mU/mL empfohlen, sowie als erstlinige Behandlung der Kategorie 2A für Patienten mit Serum-EPO >500 mU/mL. Dieses Update folgt auf die Genehmigung von RYTELO durch die FDA im Juni 2024 zur Behandlung von erwachsenen Patienten mit niedrigem bis intermediärem Risiko MDS mit transfusionsabhängiger Anämie. Das Unternehmen ist der Meinung, dass diese Updates das Bewusstsein und die Verwendung von RYTELO als neue Behandlungsoption für geeignete MDS-Patienten mit niedrigem Risiko erhöhen werden.
- RYTELO received Category 1 and 2A recommendations in NCCN Guidelines for lower-risk MDS patients
- RYTELO is recommended as a first-line treatment for patients with serum EPO >500 mU/mL
- The update may increase awareness and uptake of RYTELO among hematologists
- FDA approval of RYTELO in June 2024 for treating adult patients with low- to intermediate-1 risk MDS
- None.
The inclusion of RYTELO™ (imetelstat) in the updated NCCN Guidelines for Myelodysplastic Syndromes (MDS) is a significant development for Geron As a Medical Research Analyst, I find this update particularly impactful for several reasons:
- RYTELO has received a Category 1 recommendation for second-line treatment, indicating a high level of evidence and uniform consensus among NCCN experts. This strong endorsement could significantly influence treatment decisions and potentially increase RYTELO's adoption rate.
- The Category 2A recommendation for first-line treatment in ESA-ineligible patients opens up a new market segment for RYTELO, potentially expanding its patient pool.
- The guidelines now recommend RYTELO for both RS+ and RS- patients, broadening its applicability across different MDS subtypes.
- Importantly, the guidelines do not specify red blood cell transfusion burden, which could allow for more flexible use of RYTELO in clinical practice.
These updates reflect the strength of Geron's Phase 3 data and could accelerate RYTELO's integration into standard treatment protocols. However, it's important to monitor real-world effectiveness and safety data as RYTELO's usage expands.
From a financial perspective, Geron 's announcement about RYTELO's inclusion in the NCCN Guidelines is highly positive. Here's why this news is significant for investors:
- The NCCN Guidelines are widely respected and often influence insurance coverage decisions. This inclusion could potentially lead to broader reimbursement for RYTELO, directly impacting Geron's revenue potential.
- The Category 1 and 2A recommendations position RYTELO favorably against competitors, potentially increasing its market share in the MDS treatment landscape.
- The expanded indications (RS+ and RS- patients, first and second-line treatments) could significantly enlarge RYTELO's addressable market, potentially boosting future sales.
- This update comes shortly after FDA approval and publication in The Lancet, creating a strong foundation for RYTELO's commercial success.
While it's too early to quantify the exact financial impact, this development could accelerate RYTELO's revenue ramp-up. Investors should closely monitor upcoming quarterly reports for early indicators of RYTELO's market penetration and sales growth. However, it's important to consider that market adoption of new therapies can take time and competition in the MDS space remains fierce.
As an oncologist specializing in hematological malignancies, I find the updated NCCN Guidelines for RYTELO™ (imetelstat) in MDS treatment to be a game-changer. Here's my clinical perspective:
- The Category 1 recommendation for second-line treatment is based on high-level evidence, which is rare for MDS therapies. This gives clinicians more confidence in prescribing RYTELO after first-line treatments fail.
- RYTELO's recommendation for both RS+ and RS- patients is crucial, as it addresses a broader patient population than some existing therapies.
- The guideline's flexibility regarding serum EPO levels (
≤500 mU/mL and>500 mU/mL ) allows for more personalized treatment decisions. - Importantly, RYTELO offers a new option for patients with high serum EPO levels who are often challenging to treat effectively.
However, it's essential to note that while these guidelines are promising, long-term follow-up data and real-world evidence will be important to fully understand RYTELO's place in MDS treatment. Clinicians should carefully consider individual patient factors, including comorbidities and treatment history, when deciding on RYTELO use. As we integrate RYTELO into clinical practice, monitoring for any unexpected side effects or long-term outcomes will be vital.
RYTELO, for both RS+ and RS- patients, has a Category 1 recommendation for second-line treatment and a Category 2A recommendation for first-line treatment of patients who are ESA ineligible (serum EPO >500 mU/mL)
The MDS NCCN Guidelines categorize lower-risk MDS patients without the del(5q) abnormality and with symptomatic anemia on the basis of ring sideroblasts (RS) percentage and serum EPO levels, without specifying red blood cell transfusion burden. For RS- lower-risk MDS patients with symptomatic anemia, RYTELO is recommended as a Category 1 second-line treatment after either erythropoiesis-stimulating agents (ESAs) or luspatercept in patients with serum EPO ≤500 mU/mL, and as a Category 2A first-line treatment in patients with serum EPO >500 mU/mL and unlikely to respond to immunosuppressive therapy. For RS+ lower-risk MDS patients with symptomatic anemia, RYTELO is recommended as a Category 1 second-line treatment after luspatercept in patients with serum EPO ≤500 mU/mL, and as a Category 2A first-line treatment in patients with serum EPO >500 mU/mL.
“We believe that the placement of RYTELO in the updated MDS NCCN Guidelines reflects the strength of our Phase 3 data and the
These updates to the MDS NCCN Guidelines follow the
National Comprehensive Cancer Network® (NCCN®) is a well-recognized, not-for-profit alliance of leading cancer centers in
About Lower-Risk Myelodysplastic Syndromes (LR-MDS)
Lower-risk myelodysplastic syndromes (LR-MDS) is a blood cancer that often progresses to require increasingly intensified management of key symptoms such as anemia and resulting fatigue1. These symptomatic LR-MDS patients frequently become red blood cell transfusion dependent, which has been shown to be associated with short- and long-term clinical consequences that reduce quality of life and shorten survival2,3. There is a high unmet need for many LR-MDS patients, particularly those with characteristics having poorer prognosis. Current treatment options for those failing ESA are limited to select sub-populations and there is an unmet need for treatments that can provide extended and continuous red blood cell transfusion independence.
About RYTELO™ (imetelstat)
RYTELO™ (imetelstat) is an FDA-approved oligonucleotide telomerase inhibitor for the treatment of adult patients with low-to-intermediate-1 risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs). It is indicated to be administered as an intravenous infusion over two hours every four weeks.
RYTELO is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Telomeres are protective caps at the end of chromosomes that naturally shorten each time a cell divides. In LR-MDS, abnormal bone marrow cells often express the enzyme telomerase, which rebuilds those telomeres, allowing for uncontrolled cell division. Developed and exclusively owned by Geron, RYTELO is the first and only telomerase inhibitor approved by the
Geron aims to ensure broad access to RYTELO for eligible patients. Accordingly, our REACH4RYTELO™ Patient Support Program provides a range of resources that support access and affordability to eligible patients prescribed RYTELO.
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
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
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
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
Most common adverse reactions (≥
Please see RYTELO (imetelstat) full Prescribing Information, including Medication Guide, available at https://pi.geron.com/products/US/pi/rytelo_pi.pdf.
About Geron
Geron is a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer. Our first-in-class telomerase inhibitor RYTELO™ (imetelstat) is FDA-approved for the treatment of adult patients with lower-risk MDS with transfusion dependent anemia. We are also conducting a pivotal Phase 3 clinical trial of imetelstat in JAK-inhibitor relapsed/refractory myelofibrosis (R/R MF), as well as studies in other hematologic malignancies. Inhibiting telomerase activity, which is increased in malignant stem and progenitor cells in the bone marrow, aims to potentially reduce proliferation and induce death of malignant cells. To learn more, visit www.geron.com or follow us on LinkedIn.
Use of Forward-Looking Statements
Except for the historical information contained herein, this press release contains forward-looking statements made pursuant to the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such statements, include, without limitation, those regarding: (i) Geron’s belief that placement of RYTELO in the updated MDS NCCN Guidelines reflects the strength of its Phase 3 data and the
1 Lewis R, Bewersdorf JP, Zeidan AM. Clinical Management of Anemia in Patients with Myelodysplastic Syndromes: An Update on Emerging Therapeutic Options. Cancer Manag Res. 2021 Jan 25;13:645-657. doi: 10.2147/CMAR.S240600. PMID: 33531837; PMCID: PMC7846829.
2 Cogle CR, Reddy SR, Chang E, et al. Early treatment initiation in lower-risk myelodysplastic syndromes produces an earlier and higher rate of transfusion independence. Leuk Res. 2017;60:123-128.
3 Balducci, L. (2006), Transfusion independence in patients with myelodysplastic syndromes. Cancer, 106: 2087-2094. https://doi.org/10.1002/cncr.21860
View source version on businesswire.com: https://www.businesswire.com/news/home/20240726376477/en/
Aron Feingold
Vice President, Investor Relations and Corporate Communications
Kristen Kelleher
Associate Director, Investor Relations and Corporate Communications
investor@geron.com
media@geron.com
Source: Geron
FAQ
What is the new NCCN recommendation for RYTELO (imetelstat) in treating lower-risk MDS patients?
When was RYTELO (imetelstat) approved by the FDA for MDS treatment?
How might the NCCN Guidelines update affect Geron (GERN) and RYTELO adoption?