- Approval across ESA ineligible and ESA relapsed/refractory
patients with LR-MDS with transfusion-dependent anemia, regardless
of ring sideroblast (RS) status
- Durable and sustained red blood cell transfusion independence,
increases in hemoglobin levels and reduction in transfusion burden
observed across key LR-MDS subgroups in the IMerge Phase 3 clinical
trial; the most common Grade 3/4 adverse reactions were
thrombocytopenia and neutropenia, which were generally manageable
and short-lived
- Lower-risk MDS is a progressive blood cancer with high unmet
need, where many patients with anemia become dependent on red blood
cell transfusions, which can be associated with clinical
consequences and decreased quality of life
- Conference call with Geron management scheduled at 8am ET on
Friday, June 7, 2024
Geron Corporation (Nasdaq: GERN), a commercial-stage
biopharmaceutical company aiming to change lives by changing the
course of blood cancer, today announced that the U.S. Food and Drug
Administration (FDA) has approved RYTELO™ (imetelstat) for the
treatment of adult patients with low- to intermediate-1 risk
myelodysplastic syndromes (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 erythropoiesis-stimulating agents (ESA).
“With the approval and availability of RYTELO, we believe
eligible patients with lower-risk MDS can potentially experience
meaningful clinical benefit, particularly the potential for greater
than 24 weeks of freedom from the burden of red blood cell
transfusions and symptomatic anemia,” said John A. Scarlett, M.D.,
Geron’s Chairman and Chief Executive Officer. “The approval of
RYTELO as the first telomerase inhibitor is a testament to the
power of our science and the passion of our people to innovate in
the field of blood cancer. As we celebrate today’s momentous
milestone, I would like to thank the patients and families,
advocates, clinicians, study coordinators and site personnel,
scientists, and Geron employees and collaborators past and present
whose participation was integral to this achievement and to
supporting our transformation into a commercial company.”
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.
Approval Based on Results from IMerge Phase 3 Clinical
Trial
“For patients with lower-risk MDS and anemia who are transfusion
dependent, we have very few options today and often cycle through
available therapies, making the approval of RYTELO potentially
practice changing for us,” said Rami Komrokji, MD, Vice Chair,
Malignant Hematology Department, Moffitt Cancer Center, who was an
investigator of the pivotal IMerge clinical trial. “What is
exciting about RYTELO is the totality of the clinical benefit
across LR-MDS patients irrespective of ring sideroblast status or
high transfusion burden, including sustained and durable
transfusion independence and increases in hemoglobin levels, all
within a well-characterized safety profile of generally manageable
cytopenias. The treatment goal for patients with LR-MDS and anemia
is transfusion-independence and before today, this wasn’t possible
for many patients.”
The FDA approval of RYTELO is based on results from the IMerge
Phase 3 clinical trial, published in The Lancet4. The IMerge trial
met its primary and key secondary endpoints, with RYTELO
demonstrating significantly higher rates of red blood cell
transfusion independence (RBC-TI) versus placebo for at least eight
consecutive weeks (RYTELO 39.8% [95% CI 30.9–49.3]; placebo 15.0%
[7.1–26.6]; p<0.001) and for at least 24 weeks (RYTELO 28.0%
[95% CI 20.1-37.0]; placebo 3.3% [95% CI 0.4-11.5]; p<0.001).
RBC-TI was durable and sustained in the RYTELO treated population,
with a median RBC-TI duration for 8-week responders and 24-week
responders of approximately 1 year and 1.5 years, respectively.
In an exploratory analysis of RYTELO-treated patients achieving
≥8-week RBC-TI, median increases in hemoglobin were 3.6 g/dL for
RYTELO and 0.8 g/dL for placebo. Clinically meaningful efficacy
results were observed across key MDS subgroups irrespective of ring
sideroblast (RS) status, baseline transfusion burden and IPSS risk
category.
In the IMerge trial, the safety profile of RYTELO was
well-characterized with generally manageable and short-lived
thrombocytopenia and neutropenia, which are familiar side effects
for hematologists who are experienced with managing cytopenias. The
most common Grade 3/4 adverse reactions were neutropenia (72%) and
thrombocytopenia (65%), which lasted a median duration of less than
two weeks, and in more than 80% of patients were resolved to Grade
< 2 in under four weeks. Cytopenias
were generally manageable with dose modifications. The intravenous
administration of RYTELO every four weeks aligns to routine blood
count monitoring for these patients.
The most common adverse reactions (incidence ≥10% with a
difference between arms of >5% compared to placebo), including
laboratory abnormalities, were decreased platelets
(thrombocytopenia), decreased white blood cells, decreased
neutrophils (neutropenia), increased aspartate aminotransferase
(AST), increased alkaline phosphatase (ALP), increased alanine
aminotransferase (ALT), fatigue, prolonged partial thromboplastin
time, arthralgia/myalgia, COVID-19 infections, and headache.
Clinically relevant adverse reactions in < 5% of patients who
received RYTELO included febrile neutropenia, sepsis,
gastrointestinal hemorrhage, and hypertension.
Conference Call Details
A conference call with Geron management is scheduled at 8am
Eastern Time on Friday, June 7, 2024, to discuss the FDA approval
and launch of RYTELO. To access the webcast and slides, please
visit the Investors & Media page. Participants may access the
webcast by registering online using the following link,
https://events.q4inc.com/attendee/923992744.
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 eligible patients with lower-risk MDS can potentially
experience meaningful clinical benefit with RYTELO, particularly
the potential for greater than 24 weeks of freedom from the burden
of red blood cell transfusions and symptomatic anemia; (ii) an
unmet need for new treatments for patients with LR-MDS that can
provide extended and continuous red blood cell transfusion
independence; (iii) that RYTELO could be practice-changing for
hematologists who treat patients with lower-risk MDS and anemia who
are transfusion dependent; (iv) the potential for RYTELO to offer a
totality of clinical benefit across LR-MDS patients irrespective of
ring sideroblast status or high transfusion burden, including
sustained and durable transfusion independence and increases in
hemoglobin levels, all within a well-characterized safety profile
of generally manageable cytopenias; (v) that inhibiting telomerase
activity aims to potentially reduce proliferation and induce death
of malignant cells; (vi) that Geron aims to ensure broad access to
RYTELO; (vii) that imetelstat has the potential to demonstrate
disease-modifying activity in patients; (viii) that IMpactMF has
registrational intent; and (ix) 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 4 Platzbecker, U. 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. The Lancet. Volume 403, Issue
10423, P249-260. Jan 20, 2024.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240606850162/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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