Geron Announces Submission of New Drug Application to FDA for First-in-Class Telomerase Inhibitor Imetelstat
June 20 2023 - 9:00AM
Business Wire
Geron Corporation (Nasdaq: GERN), a late-stage clinical
biopharmaceutical company, today announced the submission to the
United States Food and Drug Administration (FDA) of a New Drug
Application (NDA) for imetelstat for the treatment of
transfusion-dependent anemia in adult patients with low- to
intermediate-1 risk myelodysplastic syndromes (MDS) who have failed
to respond or have lost response to or are ineligible for
erythropoiesis-stimulating agents (ESAs).
“This pioneering achievement to submit the first New Drug
Application to the FDA for a telomerase inhibitor reflects the
dedication, commitment and teamwork of so many people who believed
targeting telomerase could make a significant difference for
patients,” said John A. Scarlett, M.D., Chairman and Chief
Executive Officer. “We are deeply committed to addressing the unmet
needs for lower risk MDS patients, who often suffer from
transfusion-dependent anemia.”
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. Further, statistically significant and clinically
meaningful efficacy results were achieved across key MDS subgroups:
ring sideroblast (RS) status, baseline transfusion burden and IPSS
risk category. Safety results were consistent with prior imetelstat
clinical experience.
As allowed under imetelstat’s Fast Track designation in lower
risk MDS, Geron has requested that the FDA grant Priority Review of
the NDA. Under standard practice, Geron expects FDA communication
in 60 days whether the NDA was accepted for review and the timeline
of such review (i.e., priority or standard). Additionally, based on
IMerge Phase 3, Geron expects to submit a Marketing Authorization
Application (MAA) in the EU in the second half 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. Geron submitted
a New Drug Application (NDA) in the U.S. in June 2023 and expects
to submit a Marketing Authorization Application (MAA) in the EU in
the second half of 2023 in the lower risk MDS indication.
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 Marketing Authorization Application in the EU
in the second half 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 Geron expects
FDA communication in 60 days whether the NDA was accepted for
review; 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 the FDA determines there is necessity to withhold their
acceptance of the NDA beyond the 60 days; and (d) whether
imetelstat actually demonstrates that it alters the underlying
drivers of disease and has disease-modifying activity in patients.
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, 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/20230620336071/en/
Aron Feingold Investor and Media Relations investor@geron.com
media@geron.com
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