Geron Announces Fast Track Designation Granted to Imetelstat for Relapsed/Refractory Myelofibrosis
September 30 2019 - 4:19PM
Geron Corporation (Nasdaq: GERN) today announced that the United
States Food and Drug Administration (FDA) has granted Fast Track
designation to imetelstat for the treatment of adult patients with
Intermediate-2 or High-risk myelofibrosis (MF) whose disease has
relapsed after or is refractory to janus kinase (JAK) inhibitor
treatment, or relapsed/refractory MF. The Fast Track designation
includes patients with primary MF and MF developed after essential
thrombocythemia or polycythemia vera. This is the same patient
population that was studied in Geron’s IMbark Phase 2 clinical
trial. There are currently no marketed drugs specifically approved
for relapsed/refractory MF, representing a significant unmet
medical need. Geron plans to conduct an End of Phase 2 meeting with
the FDA by the end of the first quarter of 2020 to determine if
there is a regulatory path forward for imetelstat in
relapsed/refractory MF.
The FDA's Fast Track Program is designed to facilitate the
development and expedite the review of new drugs that are intended
to treat serious conditions and supported by data that demonstrate
the potential to address an unmet medical need. Fast Track
designation provides opportunities for frequent interactions with
FDA review staff, including meetings to discuss the drug's
development plan and to ensure the collection of appropriate data
needed to support approval. Through the Fast Track Program, a
product candidate may be eligible for priority review, if supported
by the clinical data, and for the ability to submit completed
sections of a New Drug Application (NDA) on a rolling basis as data
become available prior to completion of the full application.
About Myelofibrosis
Myelofibrosis (MF), a type of myeloproliferative neoplasm, is a
chronic blood cancer in which abnormal or malignant precursor cells
in the bone marrow proliferate rapidly, causing scar tissue to form
(fibrosis). As a result, normal blood production in the bone marrow
is impaired and may shift to other organs, such as the spleen and
liver, which can cause them to enlarge substantially. People with
MF may have abnormally low or high numbers of red blood cells,
white blood cells or platelets in the blood or bone marrow. MF
patients can also suffer from debilitating constitutional symptoms,
such as fever, weight loss, night sweats, and itching (pruritus).
MF patients have shortened survival, and their disease may
transform to acute myeloid leukemia.
The number of people living with MF in the United States is
estimated at 13,000 patients, with approximately 3,000 new cases
diagnosed each year. There are currently only two drugs approved by
the FDA for treating MF patients. The most widely used drug therapy
for MF has a discontinuation rate of 75% after five years of
treatment. Once patients discontinue treatment with this drug due
to failure or lack of response, there are no specifically approved
therapies, and the median overall survival for these MF patients is
approximately 14 to 16 months, representing a significant unmet
medical need.
About IMbark
IMbark is a Phase 2 clinical trial to evaluate two starting dose
levels of imetelstat (either 4.7 mg/kg or 9.4 mg/kg administered by
intravenous infusion every three weeks) in patients with
Intermediate-2 or High-risk MF who have relapsed after or are
refractory to prior treatment with a JAK inhibitor. The co-primary
efficacy endpoints for the trial are spleen response rate and
symptom response rate. Key secondary endpoints are safety and
overall survival. IMbark is closed to new patient enrollment.
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 suppression of
malignant progenitor cell clone proliferation, which allows
potential recovery of normal hematopoiesis. Ongoing clinical
studies of imetelstat consist of IMerge, a Phase 2/3 trial in lower
risk myelodysplastic syndromes (MDS), and IMbark, a Phase 2 trial
in Intermediate-2 or High-risk myelofibrosis (MF). 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 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 statements in this
press release regarding: (i) Geron’s plan to conduct an End of
Phase 2 meeting with the FDA by the end of the first quarter of
2020; (ii) that imetelstat may have disease-modifying activity; and
(iii) other statements that are not historical facts, constitute
forward-looking statements. These 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: (i) Geron may not be able to prepare timely for an End
of Phase 2 meeting by the end of the first quarter of 2020; and
(ii) imetelstat may not actually demonstrate disease-modifying
activity in clinical trials. Additional information on the
above-stated 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 periodic reports filed with the Securities and
Exchange Commission under the heading “Risk Factors,” including
Geron’s quarterly report on Form 10-Q for the quarter ended June
30, 2019. 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
CG Capital877-889-1972
Geron (NASDAQ:GERN)
Historical Stock Chart
From Mar 2024 to Apr 2024
Geron (NASDAQ:GERN)
Historical Stock Chart
From Apr 2023 to Apr 2024