Conference
Call Scheduled for 4:30 p.m. ET today
Geron Corporation (Nasdaq: GERN), a late-stage clinical
biopharmaceutical company developing a first-in-class telomerase
inhibitor, imetelstat, to treat hematologic myeloid malignancies,
today reported financial results for the fourth quarter and year
ended December 31, 2020. The Company ended fiscal year 2020 with
$260 million in cash and marketable securities, which is expected
to fund operations until the end of 2022.
“Geron’s vision is to be recognized as a leader in the treatment
of hematologic malignancies by changing the course of these
diseases and thereby improving and extending the lives of
patients,” said John A. Scarlett, M.D., Chairman and Chief
Executive Officer. “In 2020, we made important progress toward
realizing that vision through two ongoing Phase 3 clinical trials,
presentation of new data and analyses providing strong evidence of
imetelstat’s disease-modifying potential that differentiates
imetelstat from other treatments today, and raising the capital
necessary to support future activities. With this momentum from
2020, in 2021, we plan to complete enrollment in IMerge Phase 3,
advance site initiation and enrollment in IMpactMF, and begin
activities to prepare for Geron’s first NDA filing and potential
commercial launch. With a strong team in place and the financial
resources to support these plans, we believe our vision can be
attained.”
Dr. Scarlett added, “We are hopeful that clinical site
operations will normalize in the next several months since vaccine
distribution has commenced in many countries and the number of
COVID-19 cases is declining. However, the fluid and dynamic nature
of the pandemic continues to create uncertainty and
unpredictability on our clinical trial activities. Taking into
account these challenges and under current planning assumptions, we
expect IMerge Phase 3 to be fully enrolled in the second half of
2021. Depending on the timing of full enrollment, we expect
top-line results from IMerge Phase 3 to be available from the end
of 2022 to the first half of 2023. For IMpactMF, under current
planning assumptions, we expect the pace of enrollment to be slower
and now project the event-driven analyses for the trial to occur in
2024 for the interim analysis and 2025 for the final analysis.”
Company Highlights and Upcoming Milestones
Ongoing Phase 3 Clinical Trials
IMerge in Lower Risk Myelodysplastic
Syndromes (MDS)
In December 2020, 50% of the planned enrollment in IMerge was
achieved and today, 65% have been enrolled. In the fourth quarter
of 2020, the first meeting of the Independent Data Monitoring
Committee (IDMC) was held, and the IDMC recommended that the trial
continue without modification.
In the past few winter months, the resurgence of COVID-19 cases
caused delays in clinical site openings, as well as patient
screening and enrollment, and such delays may be ameliorated with
broader vaccine distribution and other public health safety
measures. With the current decline in the number of COVID-19 cases,
the Company expects its trial operations to begin to normalize in
the next several months. However, the pace at which any
normalization may occur remains uncertain and unpredictable. Taking
into account these dynamic and evolving circumstances, under
current planning assumptions, the Company expects IMerge Phase 3 to
be fully enrolled in the second half of 2021. Depending on the
timing of full enrollment, the Company expects top-line results
from IMerge Phase 3 to be available during the time period from the
end of 2022 to first half of 2023. To address the delays in
enrollment, the Company continues to pursue enrollment boosting
activities, including opening new sites, engaging clinical science
liaisons and employing a social media campaign. Currently, 105
sites are open for enrollment, and the Company expects to open the
remaining 15 additional planned sites over the next few months.
IMpactMF in Refractory Myelofibrosis
(MF)
IMpactMF, the only Phase 3 clinical trial in MF with overall
survival (OS) as a primary endpoint, opened for screening and
enrollment in December 2020 with three clinical sites in the United
States. However, as with IMerge Phase 3, COVID-19 has negatively
impacted clinical trial activities. In addition, in 2020 a number
of competing trials were initiated in MF and other oncology
indications in the countries where IMpactMF is planned to be
conducted. As a result of these factors, site personnel resources
are constrained at many clinical sites, causing delays in site
initiation activities. Although the Company has expanded the number
of countries and sites planned to conduct the trial, the Company
now expects IMpactMF to be fully enrolled in 2024. The Company
currently plans to engage over 180 sites to participate in IMpactMF
across North America, South America, Europe, Australia and
Asia.
Under current planning assumptions around enrollment and median
overall survival for each treatment arm, the Company expects to
conduct the interim analysis in 2024 and the final analysis in
2025. The final analysis for OS is planned to be conducted after
more than 50% of the patients enrolled in the trial have died. An
interim analysis of OS is planned to be conducted after
approximately 70% of the total projected number of events for the
final analysis have occurred. If the pre-specified, statistically
significant difference in OS between the two treatment arms is met
at the interim analysis, it is possible that data from the interim
analysis could support a registration filing. Both the planned
interim and final analyses are event-driven and could occur on
different timelines than currently expected.
New Drug Application (NDA) and Commercial Readiness
Activities in 2021
The Company has begun preparations for the future submission of
an NDA for imetelstat in lower risk MDS. Assuming the results of
IMerge Phase 3 are supportive, the Company plans to submit the
completed NDA in 2023.
The Company is beginning pre-commercial activities, including
building the internal infrastructure to support a commercial
launch. Assuming approval of the NDA, the Company forecasts the
commercial launch of imetelstat in lower risk MDS in the United
States to be in 2024.
Presentations and Publications of Phase 2 Data and
Analyses
More Mature Clinical Data from IMerge
Phase 2 Continue to Differentiate Imetelstat in Lower Risk
MDS
In October 2020, more mature data of 38 patients in IMerge Phase
2 were published in the Journal of Clinical Oncology (JCO)
highlighting the durability of transfusion independence achievable
with imetelstat treatment in the trial. Durability of transfusion
independence continues to differentiate imetelstat with 29% (11/38)
of patients being transfusion-free for more than one year, and a
median duration of transfusion independence of 20 months. Such
durability provides significant and meaningful clinical benefit to
lower risk MDS patients given their chronic anemia. In addition,
the JCO publication reported observations from the IMerge Phase 2
of depletion of cytogenetic abnormalities and reductions in key
driver mutations for lower risk MDS which provide strong evidence
of disease-modifying activity of imetelstat. These observations
were further strengthened through the correlation of molecular data
with clinical benefits of transfusion independence.
The data from the JCO publication were also reported in an oral
presentation at the American Society of Hematology (ASH) Annual
Meeting in December 2020. Further analyses of IMerge Phase 2 data
are expected to be presented at medical conferences in 2021.
New Analyses of IMbark Phase 2 Data
Support OS Outcome and Disease-Modifying Potential of
Imetelstat
In December 2020, new analyses of IMbark Phase 2 data were
reported in oral and poster presentations at the ASH Annual
Meeting. These analyses described dose-related improvements in OS
that were correlated to other clinical benefits, such as symptom
response and spleen volume reduction, which support previously
reported improvement in OS for MF patients in the trial.
Furthermore, (i) reductions in the variant allele frequency of key
driver mutations in MF, indicating imetelstat targets the
underlying malignant clone, and (ii) improvements in bone marrow
fibrosis were correlated to improvement in OS, which provide
further evidence of imetelstat’s disease-modifying potential.
Fourth Quarter and Full Year 2020 Results
For the fourth quarter of 2020, the Company reported a net loss
of $23.8 million, or $0.07 per share, compared to $29.1 million, or
$0.15 per share, for the fourth quarter of 2019. Net loss for the
full year of 2020 was $75.6 million, or $0.28 per share, compared
to $68.5 million, or $0.36 per share, for the full year of
2019.
Revenues for the three and twelve months ended December 31, 2020
were $50,000 and $253,000, respectively, compared to $171,000 and
$460,000 for the same periods in 2019. Revenues in 2020 and 2019
primarily reflect estimated royalties from sales of cell-based
research products from the Company’s divested stem cell assets. In
connection with the divestiture of Geron’s human embryonic stem
cell assets, including intellectual property and proprietary
technology, to Lineage Cell Therapeutics, Inc. (formerly BioTime,
Inc., which acquired Asterias Biotherapeutics, Inc.) in 2013, Geron
is entitled to receive royalties on sales from certain research or
commercial products utilizing Geron’s former intellectual
property.
Total operating expenses for the three and twelve months ended
December 31, 2020 were $23.3 million and $77.2 million,
respectively, compared to $30.2 million and $73.0 million for the
same periods in 2019. Research and development expenses for the
three and twelve months ended December 31, 2020 were $16.2 million
and $51.5 million, respectively, compared to $24.9 million and
$52.1 million for the same periods in 2019. The overall decrease in
research and development expenses, compared to the same periods in
2019, primarily reflects the net result of closing of the IMbark
Phase 2 clinical trial, completion of the transition of the
imetelstat program and reduced purchases of raw materials, drug
substance and drug product, partially offset by increased costs for
IMerge Phase 3 and start-up costs for IMpactMF. General and
administrative expenses for the three and twelve months ended
December 31, 2020 were $7.1 million and $25.7 million,
respectively, compared to $5.3 million and $20.9 million, for the
same periods in 2019. The overall increase in general and
administrative expenses, compared to the same periods in 2019,
primarily reflects increased personnel-related expenses for
additional general and administrative headcount to support the
development organization and higher legal costs.
Interest income for the three and twelve months ended December
31, 2020 was $243,000 and $1.8 million, respectively, compared to
$925,000 and $4.2 million for the same periods in 2019. The overall
decrease in interest income, compared to the same periods in 2019,
primarily reflects lower yields on the Company’s marketable
securities portfolio due to declining interest rates.
Interest expense for the three and twelve months ended December
31, 2020 was $754,000 and $760,000, respectively. In September
2020, the Company secured a new debt facility for up to $75
million.
The Company ended the 2020 fiscal year with $260 million in cash
and marketable securities, which the Company believes is sufficient
for its operations until the end of 2022.
Projected 2021 Financial Guidance
For fiscal year 2021, the Company expects its operating expense
burn to range from $108 to $112 million, which includes costs for
the two ongoing Phase 3 clinical trials; producing validation
batches of imetelstat at contract manufacturers to enable future
production of imetelstat for clinical and commercial purposes; and
preparatory activities for NDA and commercial readiness.
As of December 31, 2020, the Company had 55 employees. The
Company plans to grow to a total of approximately 80 to 85
employees by year-end 2021, of which the majority will be research
and development personnel.
Conference Call
Geron will host a conference call at 4:30 p.m. ET on Thursday,
March 11, 2021 to discuss fourth quarter and full year 2020
financial results and recent events.
A live, listen-only webcast will be available on the Company’s
website at www.geron.com/investors/events. An archive of the
webcast will be available on the Company’s website for 30 days.
Participants may access the conference call live via telephone
by pre-registering online using the following link,
http://www.directeventreg.com/registration/event/3329959. Upon
registration, a phone number, Direct Event Passcode and unique
Registrant ID will be sent via email. This information will be
needed in order to enter the conference call. Participants are
advised to pre-register at least 10 minutes prior to joining the
call.
About Imetelstat
Imetelstat is a novel, first-in-class telomerase inhibitor
exclusively owned by Geron and being developed in hematologic
myeloid 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 hematologic myeloid 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 kinase (JAK) inhibitor treatment.
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 currently enrolling patients. For further
information about IMerge Phase 3, including enrollment criteria,
locations and current status, visit
ClinicalTrials.gov/NCT02598661.
About IMpactMF
IMpactMF is an open label, randomized, controlled Phase 3
clinical trial with registrational intent. The trial is designed to
enroll approximately 320 patients with Intermediate-2 or High-risk
myelofibrosis who are refractory to prior treatment with a JAK
inhibitor, also referred to as refractory MF. Patients will be
randomized to receive either imetelstat or best available therapy.
The primary endpoint is overall survival (OS). Key secondary
endpoints include symptom response, spleen response, progression
free survival, complete response, partial response, clinical
improvement, duration of response, safety, pharmacokinetics, and
patient reported outcomes.
IMpactMF is currently enrolling patients. For further
information about IMpactMF, including enrollment criteria,
locations and current status, visit
ClinicalTrials.gov/NCT04576156.
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. 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 imetelstat
has potential disease-modifying activity; (ii) that Geron’s current
financial resources of $260 million in cash and marketable
securities are expected to fund its operations until the end of
2022; (iii) that Geron plans to engage over 180 sites for IMpactMF;
(iv) that Geron expects to submit the completed NDA for lower risk
MDS in 2023, and assuming FDA approval of the NDA, forecasts a
commercial launch of imetelstat in the United States in 2024; (v)
that Geron expects IMerge Phase 3 to be fully enrolled in the
second half of 2021; (vi) that Geron expects top-line results for
IMerge Phase 3 will be available from the end of 2022 to first half
of 2023; (vii) that the ten remaining additional IMerge Phase 3
sites will be open for screening and enrollment over the next few
months; (viii) the possibility that data from the IMpactMF interim
analysis could support a registration filing; (ix) that under
current planning assumptions for IMpactMF, Geron projects and
expects enrollment to be complete in 2024, to conduct an interim
analysis in 2024 and a final analysis in 2025; (x) that Geron’s
2021 operating expense burn will range from $108 to $112 million;
(xi) that Geron expects to grow to 80-85 employees in 2021; (xii)
that Geron expects to present further analyses of IMerge Phase 2 at
medical conferences in 2021; and (xiii) 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 meet the expected timelines and
planned milestones in (iii), (iv), (v), (vi), (vii) and (ix) 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 and IMpactMF trials to enable
regulatory approval; (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) that Geron will 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 (iii), (iv), (vi) and
(ix) above; (h) whether regulatory authorities require an
additional clinical trial for approval even if IMerge Phase 3 or
IMpactMF meet their respective primary endpoint(s); (i) whether
there are failures or delays in manufacturing or supplying
sufficient quantities of imetelstat or other clinical trial
materials in a timely manner, whether due to the current or
evolving effects of the COVID-19 pandemic or otherwise; (j) whether
imetelstat is able to maintain patent protection and have freedom
to operate; (k) whether there are cost overruns in 2021 due to the
current or evolving effects of the COVID-19 pandemic or otherwise;
(l) whether Geron can accurately project the timing of, or attain
complete enrollment in IMerge Phase 3 or IMpactMF, whether due to
the current or evolving effects of the COVID-19 pandemic or
otherwise; and (m) whether Geron is able to enroll IMpactMF at a
pace that would enable an interim analysis in 2024 and a final
analysis in 2025. 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 annual report on Form 10-K for the year
ended December 31, 2020 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.
Financial table follows.
GERON CORPORATION
CONDENSED STATEMENTS OF
OPERATIONS
(UNAUDITED)
Three Months Ended
Year Ended
December
31,
December
31,
(In thousands, except share and per share
data)
2020
2019
2020
2019
Revenues:
License fees and royalties
$
50
$
171
$
253
$
460
Operating expenses:
Research and development
16,228
24,923
51,488
52,072
General and administrative
7,088
5,256
25,678
20,893
Total operating expenses
23,316
30,179
77,166
72,965
Loss from operations
(23,266)
(30,008)
(76,913)
(72,505)
Interest income
243
925
1,828
4,221
Interest expense
(754)
—
(760)
—
Change in fair value of equity
investment.
(49)
—
60
(195)
Other income and (expense), net
39
13
168
(69)
Net loss
$
(23,787)
$
(29,070)
$
(75,617)
$
(68,548)
Basic and diluted net loss per
share:
Net loss per share
$
(0.07)
$
(0.15)
$
(0.28)
$
(0.36)
Shares used in computing net loss per
share
318,813,084
198,447,315
271,460,265
190,160,311
CONDENSED BALANCE
SHEETS
December 31,
December 31,
(In thousands)
2020
2019
Current assets:
Cash, cash equivalents and restricted
cash
$
10,288
$
13,914
Current marketable securities
186,350
125,681
Other current assets
3,219
2,013
Total current assets
199,857
141,608
Noncurrent marketable securities
63,387
19,651
Property and equipment, net
658
408
Other assets
6,826
3,850
$
270,728
$
165,517
Current liabilities
$
30,940
$
28,162
Noncurrent liabilities
28,841
2,200
Stockholders’ equity
210,947
135,155
$
270,728
$
165,517
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210311005912/en/
Olivia Bloom Chief Financial Officer investor@geron.com
media@geron.com
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