Innovation Passport Designation Awarded to
Imetelstat for the Treatment of Lower Risk Myelodysplastic
Syndromes
Pathway Intended to Expedite Regulatory Review
Process
Geron Corporation (Nasdaq: GERN), a late-stage biopharmaceutical
company focused on the development and commercialization of
treatments for hematologic malignancies, today announced that
imetelstat, the Company’s first in class telomerase inhibitor, has
been granted an Innovation Passport, which is the first prescribed
entry point to the Innovative Licensing and Access Pathway (ILAP)
launched in the United Kingdom (UK) by the Medicines and Healthcare
products Regulatory Agency (MHRA) in January 2021, post-Brexit. The
objective of this new licensing and access pathway is to reduce the
time to market for innovative medicines. Key benefits of being
within ILAP include a 150-day accelerated assessment and rolling
review of a Marketing Authorization Application (MAA), as well as
opportunities for frequent interactions with the review staff at
the MHRA and its partner agencies, including the National Institute
for Health and Care Excellence (NICE), to discuss imetelstat’s
development, regulatory plans and reimbursement plans.
“We are pleased to participate in this new expedited review
pathway established by MHRA and look forward to working with them
and their partner agencies as we collaborate to bring imetelstat to
UK patients,” said Sharon McBain, Geron’s Vice President, Global
Regulatory Affairs. “We are pleased imetelstat met the three
qualifying criteria for the Innovation Passport and believe that
because of the durable transfusion independence across different
patient subgroups and strong evidence of disease-modifying activity
observed in our IMerge Phase 2 study, imetelstat’s novel telomerase
inhibitor approach has the potential to be an important drug in
this patient population.”
Patients from the IMerge Phase 2 clinical trial achieved durable
transfusion independence with imetelstat treatment, including
transfusion-free periods greater than one year, irrespective of the
disease subgroup, such as ringed sideroblast positive or ringed
sideroblast negative. Such durability provides significant and
meaningful clinical benefit to lower risk MDS patients given their
chronic anemia and the debilitating impact of serial blood
transfusions. In addition, depletion of cytogenetic abnormalities
and reductions in key driver mutations associated with lower risk
MDS were observed, and these results were also correlated with
transfusion independence. Taken together, the durability, molecular
and cytogenetic data from IMerge Phase 2 provide strong evidence
for disease-modifying activity of imetelstat which has the
potential to differentiate it from other currently approved and
investigational treatments in lower risk MDS today.
Imetelstat is currently being studied in two Phase 3 clinical
trials, IMerge Phase 3 in lower risk MDS and IMpactMF in refractory
myelofibrosis. Based upon current planning assumptions, Geron
expects top-line results for the IMerge Phase 3 clinical trial to
be available at the beginning of January 2023.
About ILAP and Innovation Passport
The Innovation Passport is awarded by the UK’s Innovation
Licensing and Access Pathway Steering Group, which consists of
representatives from MHRA, NICE, the Scottish Medicines Consortium
(SMC) and the National Health Service (NHS) England. To enter ILAP
and receive an Innovation Passport, an experimental drug needs to
meet the following public health and/or patient-centric criteria:
(1) the condition is life-threatening or seriously debilitating;
(2) the program fulfills at least one of the following: innovative
medicine, clinically significant new indication or, it is intended
for a special population; and (3) the medicine has the potential to
offer benefits to patients. The ILAP aims to accelerate the time to
market and facilitate patient access to medicines through the
development of a target development profile (TDP) that outlines a
unique product-specific roadmap for regulatory and development
milestones. Other benefits provided by ILAP include the potential
for a 150-day accelerated assessment of a Marketing Authorization
Application (MAA) and rolling review. The ILAP also provides
opportunities for frequent interactions with the review staff at
the MHRA and its partner agencies to discuss the drug’s development
and regulatory plans, as well as reimbursement.
About IMerge Phase 3
IMerge Phase 3 is a double-blind, randomized, placebo-controlled
Phase 3 clinical trial with registrational intent. The trial is
designed to enroll approximately 170 transfusion dependent patients
with Low or Intermediate-1 risk myelodysplastic syndromes (MDS),
also referred to as lower risk MDS, who have relapsed after or are
refractory to prior treatment with an erythropoiesis stimulating
agent (ESA). The primary endpoint is the rate of red blood cell
(RBC) transfusion independence (TI) for any consecutive period of
eight weeks or longer, or 8-week RBC-TI rate. Key secondary
endpoints include the rate of RBC-TI lasting at least 24 weeks, or
24-week RBC-TI rate, and the rate of hematologic
improvement-erythroid (HI-E), defined as a reduction of at least
four units of RBC transfusions over eight weeks compared with the
prior RBC transfusion burden.
IMerge Phase 3 is fully enrolled and patient enrollment has been
closed. For additional information about IMerge Phase 3, visit
ClinicalTrials.gov/NCT02598661.
About Myelodysplastic Syndromes
Myelodysplastic syndromes are a group of diverse blood disorders
that develop because bone marrow cells do not mature into healthy
blood cells. Many patients develop chronic anemia, the predominant
clinical problem in lower risk MDS, and become dependent on red
blood cell transfusions which leads to iron overload, heart and
kidney complications, decreases in quality of life and shorter
overall survival. Approximately 70% of MDS patients are categorized
in the lower risk groups at diagnosis, according to the
International Prognostic Scoring System that assigns relative risk
of progression to acute myelogenous leukemia and overall survival
by taking into account the presence of a number of disease factors,
such as cytopenias and cytogenetics.
About Imetelstat
Imetelstat is a novel, first-in-class telomerase inhibitor
exclusively owned by Geron and being developed in myeloid
hematologic malignancies. Data from Phase 2 clinical trials 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 United States 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 associated 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 myeloid
hematologic malignancies. The Company currently is conducting two
Phase 3 clinical trials: IMerge in lower risk myelodysplastic
syndromes and IMpactMF in refractory myelofibrosis. 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) that top-line
results for IMerge Phase 3 be available at the beginning of January
2023; (ii) that imetelstat has potential disease-modifying activity
in patients and target the malignant stem and progenitor cells of
the underlying disease; and (iii) 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 current or 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 current or evolving effects of the COVID-19 pandemic,
and overcomes all the enrollment, clinical, safety, efficacy,
technical, scientific, intellectual property, manufacturing and
regulatory challenges in order to have the financial resources for,
and to meet the expected timeline 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
IMerge Phase 3; (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 and the ability to target
the malignant stem and progenitor cells of the underlying disease;
(g) whether regulatory authorities require an additional clinical
trial for approval even if IMerge Phase 3 meets its primary
endpoints; and (h) whether there are failures or delays in
manufacturing or supplying sufficient quantities of imetelstat or
other clinical trial materials in a timely manner. 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, 2021
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/20211025005733/en/
Olivia Bloom Chief Financial Officer investor@geron.com
media@geron.com
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