Geron Announces EMA Validation of Marketing Authorization Application for Imetelstat for the Treatment of Lower Risk MDS
29 Septiembre 2023 - 7:00AM
Business Wire
Geron Corporation (Nasdaq: GERN), a late-stage clinical
biopharmaceutical company, today announced that the European
Medicines Agency (EMA) has validated the Marketing Authorization
Application (MAA) for imetelstat, a first-in-class investigational
telomerase inhibitor, for the treatment of transfusion-dependent
anemia in patients with lower risk myelodysplastic syndromes (MDS).
The MAA was submitted earlier this month and is now under
regulatory review by the European Committee for Medicinal Products
for Human Use (CHMP) under the centralized procedure, which applies
to all 27 EU member states, Iceland, Norway and Liechtenstein.
Review of the MAA is expected to be approximately 14 months.
“The EMA validation of the marketing authorization application
for imetelstat brings us one step closer to potentially offering
this first-in-class therapeutic to lower risk MDS patients in the
EU suffering from anemia,” said John A. Scarlett, M.D., Geron’s
Chairman and Chief Executive Officer. “Based on the clinical
profile of imetelstat to date, we are optimistic about its
potential to become a standard of care and address longstanding
unmet needs of lower risk MDS patients.”
The MAA is based on results from IMerge Phase 3, in which the
primary endpoint of 8-week transfusion independence (TI) was
significantly higher with imetelstat vs. placebo (p<0.001), with
median TI duration approaching one year for imetelstat 8-week TI
responders. Mean hemoglobin levels in imetelstat-treated patients
increased significantly (p<0.001) over time compared to placebo
patients. Statistically significant and clinically meaningful
efficacy results were achieved across key MDS subgroups
irrespective of ring sideroblast (RS) status, baseline transfusion
burden and IPSS risk category. Patient-reported outcomes (PRO) data
reported a sustained meaningful improvement in fatigue for
imetelstat-treated patients vs. placebo. Consistent with prior
imetelstat clinical experience, the most common serious adverse
events were primarily short-lived, manageable cytopenias.
The MAA validation follows the acceptance by the U.S. Food and
Drug Administration (FDA) of a New Drug Application (NDA), which
was assigned a Prescription Drug User Fee Act (PDUFA) action date
of June 16, 2024.
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 in lower risk myelodysplastic syndromes (LR MDS),
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, those regarding: (i) that Geron
expects review of the MAA in the EU to be approximately 14 months;
(ii) that imetelstat may alter the underlying drivers of disease in
myeloid hematologic malignancies and has the potential to
demonstrate disease-modifying activity in patients; (iii) that
imetelstat has the potential to become the standard of care and
address longstanding unmet needs of lower risk MDS patients; and
(iv) 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 June
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.
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version on businesswire.com: https://www.businesswire.com/news/home/20230929279253/en/
Aron Feingold Vice President, Investor Relations and Corporate
Communications
Kristen Kelleher Senior Manager, Investor Relations
investor@geron.com media@geron.com
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