Geron Announces U.S. Expanded Access Protocol for Imetelstat in Lower Risk MDS
29 Junho 2023 - 9:30AM
Business Wire
Geron Corporation (Nasdaq: GERN), a late-stage clinical
biopharmaceutical company, today announced that an Expanded Access
Protocol (EAP) is available for imetelstat, the Company’s
first-in-class telomerase inhibitor. As allowed under the U.S. Food
and Drug Administration (FDA) policies, companies may offer an EAP,
which permits an investigational medicinal product intended to
treat a serious or life-threatening condition, such as lower risk
myelodysplastic syndromes (MDS), to be used outside of a clinical
trial.1
“We have heard physicians in both academic and community
settings express the need for new treatment options for their lower
risk MDS patients, and they will now have an option to access
imetelstat through the EAP,” said Faye Feller, M.D., Executive Vice
President, Chief Medical Officer of Geron. “With positive IMerge
Phase 3 results submitted to the FDA, we now plan to be working
with clinicians, patients and the MDS patient advocacy community to
provide access to imetelstat for eligible patients through our EAP
that was accepted by the FDA.”
The imetelstat EAP is available for patients with transfusion
dependent non-del(5q) lower risk MDS who are refractory to,
relapsed after, or ineligible for erythropoiesis-stimulating agents
(ESAs), and naïve to lenalidomide and hypomethylating agents
(HMAs). Transfusion dependent is defined as requiring at least four
units of packed red blood cells (RBCs) over any eight-week period
during the 16 weeks prior to being considered for the EAP.
The Company recently announced the submission to the FDA of a
New Drug Application (NDA) for imetelstat, which is based on
results from the IMerge Phase 3 clinical trial. In that trial, 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.
About the Imetelstat EAP
The imetelstat EAP is a U.S.-only protocol for eligible adult
participants diagnosed with lower risk (very low, low, or
intermediate risk by IPSS-R) myelodysplastic syndromes (MDS) who
are red blood cell (RBC) transfusion dependent, have failed to
respond or have lost response or are ineligible for
erythropoiesis-stimulating agents (ESAs), had not received prior
treatment with either a hypomethylating agent or lenalidomide and
are non-del(5q). Additional information is planned to be available
on www.clinicaltrials.gov.
U.S.-based healthcare professionals seeking more information
about the imetelstat EAP can email GeronEAP@wepclinical.com.
Lower risk MDS patients who are interested in enrolling in the
imetelstat EAP should speak with their physician to understand if
imetelstat is an appropriate treatment option.
More information is available on Geron’s website under the
Patients tab.
Imetelstat is an investigational therapy and is not approved for
any indication in any markets.
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: (i) lower risk myelodysplastic
syndromes (LR MDS), and (ii) 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 plans
to be working with clinicians, patients and the MDS patient
advocacy community to provide access to imetelstat for eligible
patients through the FDA-reviewed EAP; (ii) 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; (iii) that Geron expects to submit a
Marketing Authorization Application in the EU in the second half of
2023; 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 continuation of the EAP or
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; and (c) whether imetelstat actually
demonstrates that it 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.
- FDA Expanded Access.
https://www.fda.gov/news-events/public-health-focus/expanded-access.
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version on businesswire.com: https://www.businesswire.com/news/home/20230629356623/en/
Aron Feingold Investor and Media Relations investor@geron.com
media@geron.com
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