Geron Announces FDA Acceptance of New Drug Application for Imetelstat for the Treatment of Lower Risk MDS
21 Agosto 2023 - 9:00AM
Business Wire
Geron Corporation (Nasdaq: GERN), a late-stage clinical
biopharmaceutical company, today announced that the United States
Food and Drug Administration (FDA) has accepted the filing of
Geron’s New Drug Application (NDA) for imetelstat, its
first-in-class telomerase inhibitor, for the treatment of
transfusion-dependent anemia in patients with lower risk
myelodysplastic syndromes (MDS).
“The FDA’s acceptance of our New Drug Application is an
important landmark along our steadfast journey to bring telomerase
inhibition with imetelstat to the market,” said John A. Scarlett,
M.D., Geron’s Chairman and Chief Executive Officer. “We look
forward to continuing our collaboration with the FDA toward the
goal of bringing imetelstat to the many patients for whom we
believe this treatment could make a significant difference.”
“FDA acceptance of our NDA is a significant milestone for both
Geron and the MDS community, as there remain few treatment options
and significant unmet needs, particularly for patients with
difficult-to-treat subtypes of this cancer,” said Faye Feller,
M.D., Executive Vice President, Geron’s Chief Medical Officer. “We
believe that the IMerge Phase 3 data reflect the truly unique
attributes of imetelstat, and, if approved, we expect imetelstat
will change the standard of care in lower risk MDS.”
The NDA submission 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. Safety results were
consistent with prior imetelstat clinical experience.
As allowed under the 21st Century Cures Act, the FDA has up to
74 days from the NDA submission date to notify Geron of the
Prescription Drug User Fee Act (PDUFA) action date for the NDA.
Upon receipt of this notification, Geron plans to disclose the
timeline for the NDA review.
Additionally, Geron expects to submit a Marketing Authorization
Application (MAA) in the European Union (EU) in the fourth quarter
of 2023.
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 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 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 biopharmaceutical company pursuing
therapies with the potential to extend and enrich the lives of
patients living with hematologic malignancies. Our first-in-class
telomerase inhibitor, imetelstat, harnesses Nobel Prize-winning
science in a treatment that may alter the underlying drivers of
disease. Geron currently has two Phase 3 pivotal clinical trials
underway evaluating imetelstat in lower risk myelodysplastic
syndromes (LR MDS), and 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 to submit a MAA in the EU in the fourth quarter of 2023;
(ii) that imetelstat may alter the underlying drivers of disease
and has the potential to demonstrate disease-modifying activity in
patients; (iii) that imetelstat could make a significant difference
to patients and has the potential to change the standard of care in
lower risk MDS; (iv) that Geron plans to disclose the timeline for
the NDA review upon notification from the FDA of the PDUFA date;
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
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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Aron Feingold Investor and Media Relations investor@geron.com
media@geron.com
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