Geron Receives Positive Opinion from the EMA Committee for Orphan Medicinal Products for Orphan Drug Designation in the Europ...
July 01 2020 - 8:30AM
Geron Corporation (Nasdaq: GERN), a late-stage clinical
biopharmaceutical company, today announced that the European
Medicines Agency (EMA) Committee for Orphan Medicinal Products
(COMP) issued a positive opinion on the Company’s application for
orphan drug designation of its first-in-class telomerase inhibitor,
imetelstat, as a potential treatment for myelodysplastic syndromes
(MDS). The Company expects that the European Commission, based on
this positive opinion of the COMP, will formally grant the orphan
drug designation for the European Union (EU) by the end of July.
Imetelstat has already been granted orphan drug designation by the
United States Food and Drug Administration as a potential treatment
for MDS.
“The positive opinion from the COMP acknowledges the compelling
clinical data from our Phase 2 clinical trial that imetelstat has
the potential to provide a clinically relevant advantage to lower
risk MDS patients who are transfusion dependent and have failed to
respond to treatment with erythropoiesis stimulating agents,” said
John A. Scarlett, Chairman and Chief Executive Officer. “Our
ongoing IMerge Phase 3 clinical trial in lower risk MDS is being
conducted at multiple sites around the world, and data from this
trial are intended to support global regulatory filings.”
To qualify for orphan drug designation in the EU, an
investigational medicine must be intended to treat a seriously
debilitating or life-threatening condition that affects fewer than
five in 10,000 people in the EU, and there must be sufficient
non-clinical or clinical data to suggest the investigational
medicine may produce clinically relevant outcomes. EMA orphan drug
designation provides companies with certain benefits and
incentives, including clinical protocol assistance, differentiated
evaluation procedures for Health Technology Assessments in certain
countries, access to a centralized marketing authorization
procedure valid in all EU member states, reduced regulatory fees
and ten years of market exclusivity.
Ongoing IMerge Phase 3 Clinical Trial
The IMerge Phase 3 clinical trial is a double-blind, randomized,
placebo-controlled clinical trial with registration intent. The
trial is designed to enroll approximately 170 patients with lower
risk transfusion dependent MDS who are relapsed or refractory to an
ESA, have not received prior treatment with either a
hypomethylating agent (HMA) or lenalidomide and who are
non-del(5q). Geron expects to complete patient enrollment by the
end of the first quarter of 2021 and top-line results to be
available in the second half of 2022.
About Myelodysplastic Syndromes
MDS is a group of blood disorders in which the proliferation of
malignant progenitor cells produces multiple malignant cell clones
in the bone marrow resulting in disordered and ineffective
production of the myeloid lineage, which includes red blood cells,
white blood cells and platelets. Chronic anemia is the predominant
clinical problem in patients who have lower risk MDS. Many of these
patients become dependent on red blood cell transfusions due to low
hemoglobin. Serial red blood cell transfusions can lead to elevated
levels of iron in the blood and other tissues, which the body has
no normal way to eliminate. Iron overload is a potentially
dangerous condition. Studies in patients with MDS have shown that
iron overload resulting from regular red blood cell transfusions is
associated with lower quality of life, shorter overall survival and
a higher risk of developing acute myeloid leukemia.
About Imetelstat
Imetelstat is a novel, first-in-class telomerase inhibitor
exclusively owned by Geron and being developed in hematologic
myeloid malignancies. Early clinical data suggest imetelstat may
have disease-modifying activity through the apoptosis of malignant
stem and progenitor cells, which allows potential recovery of
normal hematopoiesis. Geron’s imetelstat development program
includes two ongoing or planned registration-enabling studies,
IMerge, an ongoing Phase 2/3 clinical trial in lower risk
myelodysplastic syndromes (MDS), and a planned Phase 3 clinical
trial in refractory myelofibrosis (MF) expected to be open for
patient screening and enrollment in the first quarter of 2021.
Imetelstat has been granted Fast Track designation by the U.S. Food
and Drug Administration for both the treatment of patients with
non-del(5q) lower risk MDS who are refractory or resistant to an
erythropoiesis-stimulating agent and for patients with
Intermediate-2 or High-risk MF whose disease has relapsed after or
is refractory to janus kinase (JAK) inhibitor treatment.
About Geron
Geron is a late-stage clinical biopharmaceutical company focused
on the development and potential commercialization of a
first-in-class telomerase inhibitor, imetelstat, in hematologic
myeloid malignancies. For more information about Geron, visit
www.geron.com.
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) IMerge, Geron
expects to complete enrollment in the first quarter of 2021 and
have top-line results in the second half of 2022; (ii) that data
from the IMerge Phase 3 clinical trial will support global
regulatory filings; (iii) the period of potential market
exclusivity in the EU with orphan drug designation; (iv) potential
disease-modifying activity of imetelstat; (v) that the European
Commission will grant the orphan drug designation; and (vi) 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 the evolving effects of
the COVID-19 pandemic and resulting global economic and financial
disruptions will materially and adversely impact Geron’s business
and business prospects, its financial condition and the future of
imetelstat; (b) whether Geron overcomes all of the potential delays
and other adverse impacts caused by the evolving effects of the
COVID-19 pandemic, and overcomes the clinical, safety, efficacy,
technical, scientific, intellectual property, manufacturing and
regulatory challenges in order to meet the expected timelines and
planned milestones in (i) above; (c) whether regulatory authorities
permit the further development of imetelstat on a timely basis, or
at all, without any clinical holds; (d) whether imetelstat is
demonstrated to be safe and efficacious in clinical trials; (e)
whether any future efficacy or safety results may cause the
benefit-risk profile of imetelstat to become unacceptable; (f)
whether imetelstat actually demonstrates disease-modifying activity
in patients; (g) Geron’s need to raise substantial capital in order
to complete the development and commercialization of imetelstat,
including to meet all of the expected timelines and planned
milestones in (i) above; (h) whether Geron can accurately project
or attain complete enrollment in the IMerge Phase 3 clinical trial,
whether due to the evolving effects of the COVID-19 pandemic or
otherwise; (i) whether EU regulatory authorities in the future
decide that there are other drugs so similar to imetelstat that its
orphan drug designation is not warranted and removed; and (j)
whether there are failures or delays in manufacturing sufficient
quantities of imetelstat or other clinical trial materials in a
timely manner, whether due to the evolving effects of the COVID-19
pandemic or otherwise. 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, 2020. 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.
CONTACT:
Suzanne MessereInvestor and Media
Relationsinvestor@geron.commedia@geron.com
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