- Subgroup analyses showed consistently higher transfusion
independence (TI) response rates than placebo across different risk
groups regardless of International Prognostic Scoring System
(IPSS), revised IPSS (IPSS-R) or IPSS molecular (IPSS-M)
- Robust TI rates with imetelstat treatment compared with placebo
in patients with poor prognosis mutational profiles
- For the nearly 20% of imetelstat-treated patients who achieved
greater than 1-year sustained TI, median duration of TI was more
than two years and median hemoglobin increase was more than 5
g/dL
- Consistent with prior imetelstat clinical experience, the most
common adverse events were thrombocytopenia and neutropenia that
were manageable and of short duration
- Imetelstat is currently under regulatory review by the FDA and
EMA for the treatment of transfusion-dependent anemia in adult
patients with lower risk MDS
Geron Corporation (Nasdaq: GERN), a late-stage clinical
biopharmaceutical company, today announced the publication of
abstracts from the IMerge Phase 3 clinical trial evaluating its
first-in-class investigational telomerase inhibitor imetelstat in
patients with lower risk myelodysplastic syndromes (MDS). Four
abstracts have been accepted for presentation at the 65th American
Society of Hematology (ASH) Annual Meeting taking place from
December 9-12 in San Diego, California and virtually.
“The 2023 ASH abstracts present data and analyses from the
IMerge Phase 3 clinical trial that reinforce the differentiated
clinical profile of imetelstat in lower risk MDS, and specifically
highlight that patients achieve TI irrespective of risk status
based on classification systems, or specific poor prognostic
mutation profiles. Of particular importance, the nearly 20% of
imetelstat-treated patients who achieved one year or greater
transfusion independence with accompanying hemoglobin rises of 5
g/dl and reduction of MDS-associated mutations speak to the
potential of this novel therapy to provide clinical benefits to
patients that have not been observed before in
transfusion-dependent lower risk MDS,” said Faye Feller, M.D.,
Geron’s Executive Vice President, Chief Medical Officer.
Imetelstat is currently under regulatory review by the U.S. Food
and Drug Administration (FDA) and by the European Medicines Agency
(EMA) for the treatment of transfusion dependent anemia in adult
patients with lower risk MDS who have failed to respond or have
lost response to or are ineligible for erythropoiesis-stimulating
agents (ESAs).
“The data from IMerge Phase 3 being presented at the ASH annual
meeting provides important insight into the breadth of effect of TI
achieved with imetelstat across different risk subgroups and across
the various underlying mutations associated with MDS, suggesting a
broad use for imetelstat in patients eligible for the study,
including in difficult-to-treat subgroups. Imetelstat also
demonstrated efficacy among patients who were reclassified as
higher risk using molecular international prognostic scoring system
(IPSS-M),” said Rami S. Komrokji, M.D., Vice Chair, Malignant
Hematology Department, Moffitt Cancer Center, one of the principal
investigators of IMerge Phase 3 and ASH presenter. “Additionally,
our abstract showcasing a real-world data population level analysis
of over 5,000 lower risk MDS patients adds to the significant
literature suggesting a correlation between TI and improvement in
survival. These data support the importance of TI to improve
outcomes for patients with lower risk MDS.”
Abstract #194: “Efficacy of Imetelstat in Achieving Red Blood
Cell Transfusion Independence (RBC-TI) Across Different Risk
Subgroups in Patients with Lower Risk Myelodysplastic Syndromes (LR
MDS) Relapsed/Refractory (R/R) to Erythropoiesis Stimulating Agents
(ESAs) in IMerge Phase 3 Study”
Oral Presentation on December 9, 2023, at
2:15 p.m. PT
This abstract evaluates TI rates in patients treated with
imetelstat vs. placebo across different risk subgroups as defined
by International Prognostic Scoring System (IPSS), revised IPSS
(IPSS-R) or IPSS molecular (IPSS-M) risk profiles and shows that
patients treated with imetelstat consistently had higher TI
response rates than placebo regardless of risk classification.
Further, in patients re-classified as high or very high risk using
IPSS-M, TI response rates with imetelstat (and not placebo) were
similar to TI response rates in lower risk subgroups. This analysis
suggests imetelstat has clinical activity in lower risk MDS
patients independent of risk categories.
Abstract #4603: “Impact of Mutational Status on Clinical
Response to Imetelstat in Patients with Lower Risk Myelodysplastic
Syndromes in the IMerge Phase 3 Study”
Poster Presentation on December 11, 2023,
from 6-8 p.m. PT
This abstract evaluates the impact of MDS-associated mutations
on clinical efficacy of imetelstat for the 165 of 178 patients for
whom mutation data were available and shows that RBC-TI response
rates in patients receiving imetelstat occurred regardless of the
presence of baseline MDS associated mutations that have a poor
prognosis (either specific mutations or multiple concurrent
mutations). In patients with mutations associated with poor
prognosis (TP53, ETV6, RUNX1, ASXL1, or EZH2), ≥8-week and
≥24-week, TI was observed in 31.8% and 9.1% of imetelstat-treated
patients vs 0 on placebo. The 8-week TI rate for patients with 3 or
more mutations was 55.6% with imetelstat compared to 14.3% with
placebo. This analysis suggests clinical benefit of imetelstat
across different molecularly defined subgroups and independent of
the underlying molecular pattern.
Abstract #4605: “Durable Continuous Transfusion Independence
(TI) With Imetelstat in IMerge Phase 3 for Patients with Heavily
Transfused Non-Del(5q) Lower Risk Myelodysplastic Syndromes (LR
MDS) Relapsed/Refractory (R/R) to or Ineligible for
Erythropoiesis-Stimulating Agents (ESAs)”
Poster Presentation on December 11, 2023,
from 6-8 p.m. PT
This abstract evaluates the 1-year TI responders in IMerge Phase
3, including 17.8% of imetelstat-treated patients (21/118; 95% CI,
11.4-25.9) and 1.7% of patients on placebo (1/60; 95% CI, 0-8.9).
The median duration of TI for imetelstat ≥1-year TI responders was
123 weeks (95% CI, 80.4 to not evaluable); the median increase in
hemoglobin during the longest TI interval was 5.18 g/dL (range,
2.67-13.76 g/dL) for the imetelstat group vs 1.67 g/dL for the
placebo patient. Of the 18/21 imetelstat-treated 1-year TI
responders for whom mutation data were available, 72.2% achieved
≥50% SF3B1 variant allele frequency (VAF) reduction, including 7
patients in whom there was complete elimination of MDS associated
mutations. The abstract concludes that the long-term durable TI,
robust increases in hemoglobin and meaningful reductions in
mutational burden suggest imetelstat may have disease-modifying
activity. Grade 3-4 thrombocytopenia and neutropenia occurred in 14
(67%) and 20 (95%) patients with ≥1-year TI, respectively, and the
mean duration of grade 3/4 thrombocytopenia and neutropenia events
was 2.25 and 1.78 weeks, respectively. 89% of grade 3/4
thrombocytopenia and 81% of grade 3/4 neutropenia was reduced to
grade 1/2 within 4 weeks.
Abstract #2440: “Durable Transfusion Independence in Lower Risk
Myelodysplastic Syndrome (LR MDS) Is Associated with Better
Survival: A Population Level Analysis Based on a Large US Health
Insurance Claims Database”
Poster Presentation on December 11, 2023,
from 6-8 p.m. PT
This abstract describes a population level analysis of 5,662
lower risk MDS patients identified through Optum Clinformatics®
between October 2015 and June 2022. Among these patients, 35% and
49% were transfusion-dependent before first and second lines of
therapy, respectively. The median overall survival from start of
second line therapy was 23.4 months overall, and 37.9 months vs 9.3
months among responders becoming transfusion independent vs
non-responders, respectively (P < .0001). Despite the currently
available therapies, transfusion dependence after any line of
therapy is associated with poorer outcomes. Achievement of durable
TI was associated with improved survival, supporting the clinical
benefit of achieving transfusion independence in lower risk
MDS.
These abstracts are available on the ASH 2023 Meeting website at
https://www.hematology.org/meetings/annual-meeting/abstracts.
About IMerge Phase 3
The Phase 3 portion of the IMerge Phase 2/3 study is a
double-blind, 2:1 randomized, placebo-controlled clinical trial to
evaluate imetelstat in patients with IPSS Low or Intermediate-1
risk (lower risk) transfusion dependent MDS who were relapsed
after, refractory to, or ineligible for, erythropoiesis stimulating
agent (ESA) treatment, had not received prior treatment with either
a HMA or lenalidomide and were non-del(5q). To be eligible for
IMerge Phase 3, patients were required to be transfusion dependent,
defined as requiring at least four units of packed red blood cells
(RBCs), over an eight-week period during the 16 weeks prior to
entry into the trial. The primary efficacy endpoint of IMerge Phase
3 is the rate of red blood cell transfusion independence (RBC-TI)
lasting at least eight weeks, defined as the proportion of patients
without any RBC transfusion for at least eight consecutive weeks
since entry to the trial (8-week TI). Key secondary endpoints
include the rate of RBC-TI lasting at least 24 weeks (24-week TI),
the duration of TI and the rate of hematologic improvement
erythroid (HI-E), which is defined under 2006 IWG criteria as a
rise in hemoglobin of at least 1.5 g/dL above the pretreatment
level for at least eight weeks or a reduction of at least four
units of RBC transfusions over eight weeks compared with the prior
RBC transfusion burden. A total of 178 patients were enrolled in
IMerge Phase 3 across North America, Europe, Middle East and
Asia.
About Imetelstat
Imetelstat is a novel, first-in-class investigational telomerase
inhibitor exclusively owned by Geron and being developed in
hematologic malignancies. Data from non-clinical studies and
clinical trials of imetelstat provide strong evidence that
imetelstat targets telomerase to inhibit the uncontrolled
proliferation of malignant stem and progenitor cells in myeloid
hematologic malignancies resulting in malignant cell apoptosis and
potential disease-modifying activity. Imetelstat has been granted
Fast Track designation by the U.S. Food and Drug Administration for
both the treatment of adult patients with transfusion dependent
anemia due to Low or Intermediate-1 risk MDS that is not associated
with del(5q) who are refractory or resistant to an erythropoiesis
stimulating agent, and for adult patients with Intermediate-2 or
High-risk myelofibrosis (MF) whose disease has relapsed after or is
refractory to Janus associated kinase (JAK) inhibitor treatment.
Imetelstat is currently not approved by any regulatory
authority.
About Geron
Geron is a late-stage clinical biopharmaceutical company
pursuing therapies with the potential to extend and enrich the
lives of patients living with hematologic malignancies. Our
first-in-class investigational telomerase inhibitor, imetelstat,
harnesses Nobel Prize-winning science in a treatment that may alter
the underlying drivers of disease. The New Drug Application (NDA)
for imetelstat for the treatment of transfusion dependent anemia in
adult patients with lower risk myelodysplastic syndromes (LR MDS)
who have failed to respond or have lost response to or are
ineligible for erythropoiesis-stimulating agents (ESAs), based on
the results from the Phase 3 IMerge clinical trial, is currently
under review by the United States Food and Drug Administration
(FDA) with a Prescription Drug User Fee Act (PDUFA) target action
date of June 16, 2024. In addition, an MAA is under review in the
European Union for the same proposed indication. Furthermore, Geron
currently has an ongoing pivotal Phase 3 clinical trial evaluating
imetelstat in relapsed/refractory myelofibrosis (MF). 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: (i) that data from the IMerge Phase 3
clinical trial in the ASH abstracts reinforce the differentiated
clinical profile of imetelstat in lower risk MDS; (ii)that the
nearly 20% of imetelstat-treated patients who achieved one year or
greater transfusion independence with accompanying hemoglobin rises
of 5 g/dl and reduction of MDS-associated mutations speak to the
potential of imetelstat to provide clinical benefits to patients
that have not been observed before in transfusion-dependent lower
risk MDS; (iii) that data from IMerge Phase 3 associated with the
ASH annual meeting suggests a broad use for imetelstat in patients
eligible for the study, including in difficult-to-treat subgroups;
(iv) that a real-world data population level analysis of over 5,000
lower risk MDS patients adds to the significant literature
suggesting a correlation between transfusion independence and
improvement in survival and support the importance of transfusion
independence to improve outcomes for patients with lower risk MDS;
and (v) 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 regulatory authorities
permit the further development of imetelstat on a timely basis, or
at all, without any clinical holds; (b) whether any future safety
or efficacy results cause the benefit-risk profile of imetelstat to
become unacceptable; (c) whether imetelstat actually demonstrates
that it alters the underlying drivers of disease and has
disease-modifying activity in patients; and (d) whether the FDA and
EMA will approve imetelstat for the treatment of
transfusion-dependent anemia in patients with lower risk MDS.
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
September 30, 2023 and future 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231102642785/en/
Aron Feingold Vice President, Investor Relations and Corporate
Communications
Kristen Kelleher Senior Manager, Investor Relations
investor@geron.com media@geron.com
Geron (NASDAQ:GERN)
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