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)
Geron Corporation (Nasdaq: GERN), a commercial-stage
biopharmaceutical company aiming to change lives by changing the
course of blood cancer, today announced that the National
Comprehensive Cancer Network (NCCN) has updated its Clinical
Practice Guidelines in Oncology (NCCN Guidelines) for the treatment
of Myelodysplastic Syndromes (MDS) to recommend RYTELO™
(imetelstat) as a Category 1 and 2A treatment of symptomatic anemia
in patients with lower-risk MDS. Treatments are classified as
Category 1 and 2A when there is uniform NCCN consensus ≥85% that
the intervention is appropriate.
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 U.S.
Prescribing Information, and that these updates will help to
increase awareness and uptake of RYTELO as a compelling new
treatment option for these patients,” said Faye Feller, M.D.,
Geron’s Executive Vice President, Chief Medical Officer. “We are
encouraged by the increasing dialogue across hematologists
rethinking treatment approaches and sequencing given the
availability of RYTELO for eligible lower-risk MDS patients with
transfusion-dependent anemia.”
These updates to the MDS NCCN Guidelines follow the U.S. Food
and Drug Administration’s (FDA) approval in June 2024 of RYTELO
(imetelstat) for the treatment of adult patients with low- to
intermediate-1 risk MDS with transfusion-dependent (TD) 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 ESAs, as well as publication of the IMerge Phase 3 pivotal
trial data in The Lancet in December 2023.
National Comprehensive Cancer Network® (NCCN®) is a
well-recognized, not-for-profit alliance of leading cancer centers
in the United States. Its treatment practice guidelines, which are
reviewed and updated on a continual basis to reflect the most
current evidence, are widely respected and followed by the U.S.
physician community and serve to inform and facilitate coverage
decisions with payers for oncology therapies. 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.
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 U.S. Food and Drug Administration.
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 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%) and 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 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 U.S. Prescribing
Information, and that these updates will help to increase awareness
and uptake of RYTELO as a compelling new treatment option for these
patients; (ii) Geron being encouraged by the increasing dialogue
across hematologists rethinking treatment approaches and sequencing
given the availability of RYTELO for eligible lower-risk MDS
patients with transfusion-dependent anemia; (iii) an unmet need for
new treatments for patients with LR-MDS that can provide extended
and continuous red blood cell transfusion independence; (iv) that
inhibiting telomerase activity aims to potentially reduce
proliferation and induce death of malignant cells; (v) that Geron
aims to ensure broad access to RYTELO; (vi) that IMpactMF has
registrational intent; and (vii) other statements that are not
historical facts, constitute forward-looking statements. These
forward-looking statements involve risks and uncertainties that can
cause actual results to differ materially from those in such
forward-looking statements. These risks and uncertainties, include,
without limitation, risks and uncertainties related to: (a) whether
Geron is successful in commercializing RYTELO (imetelstat) for the
treatment of patients with LR-MDS with transfusion dependent
anemia; (b) whether Geron overcomes potential delays and other
adverse impacts caused by enrollment, clinical, safety, efficacy,
technical, scientific, intellectual property, manufacturing and
regulatory challenges in order to have the financial resources for
and meet expected timelines and planned milestones; (c) whether
regulatory authorities permit the further development of imetelstat
on a timely basis, or at all, without any clinical holds; (d)
whether any future safety or efficacy results of imetelstat
treatment cause the benefit-risk profile of imetelstat to become
unacceptable; (e) whether imetelstat actually demonstrates
disease-modifying activity in patients and the ability to target
the malignant stem and progenitor cells of the underlying disease;
(f) that Geron may seek to raise substantial additional capital in
order to continue the development and commercialization of
imetelstat; (g) whether Geron meets its post-marketing requirements
and commitments in the U.S. for RYTELO for the treatment of
patients with LR-MDS with transfusion dependent anemia; (h) whether
there are failures or delays in manufacturing or supplying
sufficient quantities of imetelstat or other clinical trial
materials that impact commercialization of RYTELO for the treatment
of patients with LR-MDS with transfusion dependent anemia or the
continuation of the IMpactMF trial; (i) that the projected timing
for the interim and final analyses of the IMpactMF trial may vary
depending on actual enrollment and death rates in the trial; and
(j) whether the EMA will approve RYTELO for the treatment of
patients with LR-MDS with transfusion dependent anemia and whether
the FDA and EMA will approve imetelstat for other indications on
the timelines expected, or at all. Additional information on the
above risks and uncertainties and additional risks, uncertainties
and factors that could cause actual results to differ materially
from those in the forward-looking statements are contained in
Geron’s filings and periodic reports filed with the Securities and
Exchange Commission under the heading “Risk Factors” and elsewhere
in such filings and reports, including Geron’s quarterly report on
Form 10-Q for the quarter ended March 31, 2024, and subsequent
filings and reports by Geron. Undue reliance should not be placed
on forward-looking statements, which speak only as of the date they
are made, and the facts and assumptions underlying the
forward-looking statements may change. Except as required by law,
Geron disclaims any obligation to update these forward-looking
statements to reflect future information, events, or
circumstances.
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
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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
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