Top-Line
Results from IMerge Phase 3 Trial in Lower Risk MDS Expected in
Early January 2023
Geron Corporation (Nasdaq: GERN), a late-stage clinical
biopharmaceutical company developing a first-in-class telomerase
inhibitor, imetelstat, to treat hematologic malignancies, today
reported business updates, upcoming milestones and financial
results for the fourth quarter and year ended December 31, 2021.
The Company ended 2021 with $212.7 million in cash and marketable
securities, which the Company believes is sufficient to fund
current operations through the end of the first quarter of
2023.
“We made significant accomplishments in 2021 to advance the
development of imetelstat toward two important Phase 3 data
readouts, which if positive, have the potential to transform
patient care in lower risk MDS and refractory MF,” said John A.
Scarlett, M.D., Chairman and Chief Executive Officer. “With
completion of enrollment in IMerge Phase 3 last October, we look
forward to top-line results from that trial in early January 2023.
If those results confirm similar safety, as well as the depth,
breadth and durability of transfusion independence that was
observed in our Phase 2 trial, then upon approval of an NDA, we
expect our U.S. commercial launch of imetelstat in lower risk MDS
to occur as early as the first half of 2024.”
Dr. Scarlett added, “In 2021, we also made progress with the
IMpactMF Phase 3 trial in refractory MF, by opening 50% of the
planned clinical sites to patient enrollment. In 2022, we plan to
open the rest of the selected clinical sites to enable the planned
interim analysis in 2024.”
Dr. Scarlett also noted, “Building on the strong evidence of
imetelstat’s disease modification potential from our Phase 2
trials, we started several new programs in 2021. We believe that
the data to be generated from these programs over the next few
years will provide strategic value to the imetelstat franchise
through the potential expansion into new myeloid and lymphoid
hematologic indications, as well as into combination regimens.”
Key Upcoming Milestones Expected
- Top-line results from IMerge Phase 3 in early January 2023
- Open the rest of the selected clinical sites in IMpactMF for
enrollment to enable the planned interim analysis in 2024
- Expand into additional indications and treatment combinations:
- Start of IMproveMF Phase 1 study of imetelstat + ruxolitinib in
frontline MF in 1H 2022
- Start of IMpress Phase 2 study of single-agent imetelstat in
R/R AML and HR MDS in 2H 2022
- Start of TELOMERE Phase 1/2 study of imetelstat + HMAs or
venetoclax in R/R AML in 2H 2022
Top-Line Results Expected in Early January 2023 from IMerge
Phase 3 in Lower Risk Myelodysplastic Syndromes (MDS)
Assuming the results of IMerge Phase 3 support regulatory
submissions, Geron plans to submit a New Drug Application (NDA) in
the United States in the first half of 2023 and a Marketing
Authorization Application (MAA) in Europe in the second half of
2023 for imetelstat in lower risk MDS. Upon potential approval by
the United States Food and Drug Administration (FDA) of the NDA,
the Company expects that its commercial launch of imetelstat in
lower risk MDS in the United States could occur as early as the
first half of 2024.
Planned Interim Analysis in 2024 from IMpactMF in Refractory
Myelofibrosis (MF)
IMpactMF, the only Phase 3 clinical trial in MF with overall
survival (OS) as a primary endpoint, is planned to engage
approximately 180 clinical sites across North America, South
America, Europe, Australia and Asia to facilitate patient
recruitment and enrollment into the trial. The Company expects to
open for enrollment the remaining approximately 50% of selected
clinical sites in 2022.
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.
Pipeline Expansion to Enhance Imetelstat Potential
In November 2021, the Company unveiled three new clinical
programs and one preclinical program for imetelstat. These programs
explore the use of imetelstat as a single agent and in combination
with current standard of care therapies to expand the potential
application of imetelstat.
In the first half of 2022, Geron plans to initiate IMproveMF, a
company-sponsored Phase 1 study of imetelstat in combination with
ruxolitinib in frontline MF patients. In addition, in collaboration
with key opinion leaders with expertise in acute myeloid leukemia
(AML), the Company is supporting investigator-led studies in
relapsed/refractory AML and higher risk MDS (HR MDS) to evaluate
imetelstat as a single agent and in combination with venetoclax or
hypomethylating agents (HMAs). These studies are expected to start
in the second half of 2022. The preclinical program in lymphoid
malignancies is being conducted at MD Anderson Cancer Center, and
the Company expects preliminary results from ongoing in vitro and
in vivo experiments to be available by the end of 2022.
Fourth Quarter and Full Year 2021 Results
For the fourth quarter of 2021, the Company reported a net loss
of $32.0 million, or $0.10 per share, compared to $23.8 million, or
$0.07 per share, for the fourth quarter of 2020. Net loss for the
full year of 2021 was $116.1 million, or $0.35 per share, compared
to $75.6 million, or $0.28 per share, for the full year of
2020.
Revenues for the three and twelve months ended December 31, 2021
were $1.0 million and $1.4 million, respectively, compared to
$50,000 and $253,000 for the same periods in 2020. Revenues in 2021
and 2020 primarily reflect estimated royalties from sales of
cell-based research products from the Company’s divested stem cell
assets. In connection with the 2013 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.), Geron is entitled to receive royalties on sales from certain
research or commercial products utilizing Geron’s former
intellectual property. The increase in revenues in 2021 compared to
2020 primary reflects retroactive royalties of approximately
$911,000 on product sales of cell-based research products.
Total operating expenses for the three and twelve months ended
December 31, 2021 were $32.0 million and $115.4 million,
respectively, compared to $23.3 million and $77.2 million for the
same periods in 2020.
Research and development expenses for the three and twelve
months ended December 31, 2021 were $24.2 million and $85.7
million, respectively, compared to $16.2 million and $51.5 million
for the same periods in 2020. The increase in research and
development expenses for the three and twelve months ended December
31, 2021, compared to the same periods in 2020, primarily reflects
increased clinical development costs associated with conducting two
Phase 3 clinical trials, higher imetelstat manufacturing costs for
producing validation batches at contract manufacturers to enable
future production of imetelstat for clinical and commercial
purposes and higher personnel-related costs for additional
headcount.
General and administrative expenses for the three and twelve
months ended December 31, 2021 were $7.9 million and $29.7 million,
respectively, compared to $7.1 million and $25.7 million, for the
same periods in 2020. The increase in general and administrative
expenses for the three and twelve months ended December 31, 2021,
compared to the same periods in 2020, primarily reflects new costs
in connection with pre-commercial activities, including modernizing
the internal infrastructure to support a commercial launch, and
higher legal costs.
Interest income for the three and twelve months ended December
31, 2021 was $106,000 and $527,000, respectively, compared to
$243,000 and $1.8 million for the same periods in 2020. The
decrease in interest income for the three and twelve months ended
December 31, 2021, compared to the same periods in 2020, primarily
reflects lower yields on the Company’s marketable securities
portfolio.
Interest expense for the three and twelve months ended December
31, 2021 was $1.1 million and $3.7 million, respectively, compared
to $754,000 and $760,000 for the same periods in 2020. In September
2020, the Company secured a new debt facility for up to $75
million. As of December 31, 2021, a total of $50 million has been
drawn down under the facility.
Financial Resources and Projected 2022 Financial
Guidance
The Company ended 2021 with $212.7 million in cash and
marketable securities, which the Company believes is sufficient to
fund current operations through the end of the first quarter of
2023.
For fiscal year 2022, under generally accepted accounting
principles (GAAP), the Company expects total operating expenses in
the range of approximately $155 million to $165 million, which
includes non-cash items such as: stock-based compensation expense,
amortization of debt discounts and issuance costs and depreciation
and amortization. The Company expects non-GAAP total operating
expenses for fiscal year 2022, which excludes estimated non-cash
items such as: stock-based compensation expense, amortization of
debt discounts and issuance costs and depreciation and
amortization, in the range of approximately $140 million to $150
million.
The fiscal year 2022 financial guidance reflects costs to
support preparatory activities for top-line results in lower risk
MDS; NDA and commercial readiness; two ongoing Phase 3 clinical
trials and new clinical studies expanding the pipeline; finalizing
validation batches of imetelstat at contract manufacturers to
enable future production of imetelstat for clinical and commercial
purposes; projected increases in headcount and interest payments on
outstanding debt.
As of December 31, 2021, the Company had 72 employees. The
Company plans to grow to a total of approximately 90 to 100
employees by year-end 2022.
Conference Call
Geron will host a conference call at 4:30 p.m. ET on Thursday,
March 10, 2022 to discuss business updates, expected upcoming
milestones and fourth quarter and full year 2021 financial
results.
A live webcast of the conference call and related presentation
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 webcast by registering online using
the following link, https://conferencingportals.com/event/SmvlMvWL.
Participants that are unable to register online can access the
conference call via telephone by dialing domestically +1 (888)
330-2434 or internationally +1 (240) 789-2725. The conference ID is
67335.
About Imetelstat
Imetelstat is a novel, first-in-class telomerase inhibitor
exclusively owned by Geron and being developed in 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 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, duration of TI 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 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 (MF) 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 remission, partial remission, 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
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 for IMerge
Phase 3, Geron expects top-line results to be available in early
January 2023, regulatory filings in the U.S. in the first half of
2023 and the EU in the second half of 2023, and a potential launch
in lower risk MDS in the U.S. in the first half of 2024 and in the
EU in the second half of 2024; (ii) for IMpactMF, that Geron
expects to conduct an interim analysis in 2024 and a final analysis
in 2025; (iii) that Geron plans to open the remaining approximately
50% of the selected IMpactMF sites in 2022; (iv) that Geron
believes its financial resources are sufficient to fund current
operations through the end of the first quarter of 2023; (v) that
Geron expects total operating expenses in 2022 to be approximately
$155 to $165 million; (vi) that IMerge Phase 3 and IMpactMF have
registrational intent; (vii) that imetelstat has the potential to
demonstrate disease-modifying activity; (viii) that the Company
believes the data from the additional imetelstat indications,
treatment combinations and programs will provide strategic value to
the imetelstat franchise; (ix) that the Company expects IMproveMF
to start in the first half of 2022; (x) that the Company expects
IMpress to start in the second half of 2022; (xi) that the Company
expects TELOMERE to start in the second half of 2022; (xii) that
the Company expects preliminary results from the preclinical
program in lymphoid malignancies to be available at the end of
2022; 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/or the
Russia/Ukraine conflict cause global economic and financial
disruptions that 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/or the Russia/Ukraine conflict, 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 timelines and planned milestones in (i) to
(iii) and (viii) to (xii) 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 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 and the ability
to target the malignant stem and progenitor cells of the underlying
disease; (g) that Geron may seek to raise substantial capital in
order to complete the development and commercialization of
imetelstat to meet all of the expected timelines and planned
milestones in (i) to (iii) and (viii) to (xii) above; (h) whether
regulatory authorities require an additional clinical trial for
approval even if IMerge Phase 3 or IMpactMF meet their respective
primary endpoints; (i) whether there are failures or delays in
manufacturing or supplying sufficient quantities of imetelstat or
other clinical trial materials in a timely manner, or at all; (j)
whether the patient follow-up period of 12 months in IMerge Phase 3
results in not obtaining adequate data to demonstrate safety and
efficacy, including transfusion independence, for achieving success
in the primary analysis; (k) whether Geron can accurately project
the timing of enrollment in its clinical trials, whether due to the
current or evolving effects of the COVID-19 pandemic, the
Russia/Ukraine conflict, or otherwise; (l) whether Geron is able to
enroll its clinical trials at a pace that would enable the
financial resources for, and to meet the expected timelines and
planned milestones in (i) to (iii) and (viii) to (xii) above; and
(m) if the FDA does not grant priority review for the IMerge data,
then the launch date in lower risk MDS may be later than the first
half of 2024. 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, 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.
Non-GAAP Financial Measure
To supplement our financial results and guidance presented in
accordance with GAAP, the Company is providing projected non-GAAP
total operating expenses, which excludes stock-based compensation
expense, amortization of debt discounts and issuance costs and
depreciation and amortization, from projected GAAP total operating
expenses. The Company believes this non-GAAP financial measure,
when considered together with other financial information prepared
in accordance with GAAP, can enhance investors’ and analysts’
ability to meaningfully compare Geron’s results from period to
period and to projected forward-looking guidance, and to identify
operating trends in Geron’s business. The exclusion of non-cash
items, such as stock-based compensation expense, amortization of
debt discounts and issuance costs and depreciation and
amortization, does not directly or immediately relate to the
operational performance for the periods presented. This projected
non-GAAP financial measure is in addition to, not a substitute for,
or superior to, measures of financial performance projected in
accordance with GAAP. Geron encourages investors to carefully
consider the Company’s results under GAAP, as well as the
supplemental non-GAAP financial information, to more fully
understand Geron’s business.
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)
2021
2020
2021
2020
Revenues:
License fees and royalties
$
1,040
$
50
$
1,393
$
253
Operating expenses:
Research and development
24,150
16,228
85,727
51,488
General and administrative
7,872
7,088
29,665
25,678
Total operating expenses
32,022
23,316
115,392
77,166
Loss from operations
(30,982
)
(23,266
)
(113,999
)
(76,913
)
Interest income
106
243
527
1,828
Interest expense
(1,135
)
(754
)
(3,740
)
(760
)
Change in fair value of equity
investment
—
(49
)
—
60
Other income and (expense), net
(6
)
39
1,100
168
Net loss
$
(32,017
)
$
(23,787
)
$
(116,112
)
$
(75,617
)
Basic and diluted net loss per
share:
Net loss per share
$
(0.10
)
$
(0.07
)
$
(0.35
)
$
(0.28
)
Shares used in computing net
loss per share
330,833,782
318,813,084
327,631,814
271,460,265
CONDENSED BALANCE
SHEETS
December 31,
December 31,
(In thousands)
2021
2020
Current assets:
Cash, cash equivalents and
restricted cash
$
35,235
$
10,288
Current marketable
securities
148,851
186,350
Other current assets
3,120
3,219
Total current assets
187,206
199,857
Noncurrent marketable securities
28,651
63,387
Property and equipment, net
650
658
Other assets
9,527
6,826
$
226,034
$
270,728
Current liabilities
$
45,521
$
30,940
Noncurrent liabilities
54,097
28,841
Stockholders’ equity
126,416
210,947
$
226,034
$
270,728
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220310005190/en/
Olivia Bloom Chief Financial Officer investor@geron.com
media@geron.com
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