Submitted U.S. New Drug Application in lower
risk MDS in June 2023
Additional data and analyses from IMerge Phase
3 presented at medical meetings further strengthen value
proposition and differentiation of imetelstat
Potential U.S. commercial launch in lower risk
MDS expected in first half of 2024
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 highlights and financial results for the second
quarter of 2023.
“We progressed our business significantly this quarter, as we
evolve into a commercial company. Most notably, we submitted the
first New Drug Application to the FDA for a telomerase inhibitor, a
pioneering achievement that reflects our teams’ dedication to
ground-breaking and innovative drug development,” said John A.
Scarlett, M.D., Chairman and Chief Executive Officer. “Importantly,
new data and analyses from IMerge Phase 3 presented at ASCO and EHA
further strengthen the value proposition of imetelstat by
highlighting differentiated attributes of the drug, such as
unprecedented continuous durable transfusion independence,
responses across subgroups, patient-reported outcomes of improved
fatigue and strong evidence of disease-modifying activity.”
Dr. Scarlett also noted, “With approximately $400 million on the
balance sheet as of the end of the quarter, we have the financial
resources to not only fund a potential successful launch, but also
to support the first year of launch.”
Business Highlights
- Submitted New Drug Application (NDA) to the FDA based on
results from IMerge Phase 3 in lower risk myelodysplastic syndromes
(MDS).
- Presented new data and analyses from IMerge Phase 3 at ASCO and
EHA reporting robust durability of transfusion independence,
evidence of disease-modifying activity and favorable fatigue
patient-reported outcomes in imetelstat-treated lower risk MDS
patients versus placebo.
- Initiated Expanded Access Protocol (EAP) in June 2023, making
imetelstat available to clinicians and patients prior to potential
FDA approval.
- Achieved >40% enrollment in IMpactMF, Geron’s Phase 3 trial
of imetelstat in patients with myelofibrosis (MF)
relapsed/refractory to JAK-inhibitors. Based on projected planning
assumptions for enrollment and death rates in the trial, interim
analysis is expected in the first half of 2025 and final analysis
is expected in the first half of 2026.
- Dosed first patient in June 2023 in the investigator-led Phase
2 IMpress trial, evaluating imetelstat in patients with
relapsed/refractory acute myeloid leukemia or higher risk MDS.
- Obtained and reported significant market research insights
highlighting a potentially substantial commercial opportunity for
imetelstat in lower risk MDS.
- Appointed Scott Samuels as Executive Vice President, Chief
Legal Officer and Corporate Secretary, following Stephen
Rosenfield’s retirement at the end of July 2023. Mr. Samuels
recently served as the General Counsel of BeiGene, Ltd., where he
built a large, global legal and compliance team, oversaw launches
of three internally developed drug products in the U.S., Europe and
China and development of a global healthcare compliance program,
and led key strategic transactions with Amgen Inc., Novartis AG and
Celgene Corporation (now Bristol Myers Squibb Inc.).
Financial Resources to Support Potential Commercial Launch of
Imetelstat in Lower Risk Myelodysplastic Syndromes (MDS)
As of June 30, 2023, the Company had $400.2 million in cash,
cash equivalents, restricted cash and marketable securities. In the
second quarter of 2023, the Company received $17.8 million upon the
cash exercise of outstanding warrants. As of June 30, 2023,
warrants remaining outstanding are exercisable for potential future
proceeds of $31.5 million. Based on the Company’s current operating
plans and expectations regarding the timing of regulatory approval
and commercialization of imetelstat in the United States (U.S.) in
the first half of 2024, as well as revised guidance on interim and
final analyses from IMpactMF, Geron projects that its existing
financial resources plus potential future proceeds from remaining
warrants outstanding and estimated revenues from commercialization
will be sufficient to fund its projected operating requirements
through the end of 2025.
Second Quarter 2023 Financial Results
Revenues for the three and six months ended June 30, 2023, were
$29,000 and $50,000, respectively, compared to $73,000 and $196,000
for the comparable 2022 periods. Revenues in both years primarily
reflect estimated royalties from sales of cell-based research
products from the Company’s divested stem cell assets.
Total operating expenses for the three and six months ended June
30, 2023, were $52.0 million and $92.1 million, respectively,
compared to $28.0 million and $56.8 million for the comparable 2022
periods.
Research and development expenses for the three and six months
ended June 30, 2023, were $35.5 million and $62.7 million,
respectively, compared to $20.6 million and $42.7 million for the
comparable 2022 periods. The increase in research and development
expenses for the three and six months ended June 30, 2023, compared
to the same periods in 2022, primarily reflects higher clinical
trial costs related to supporting IMerge Phase 3 and IMpactMF,
increased personnel-related expenses for additional headcount,
higher consulting costs to support regulatory submissions and
greater imetelstat manufacturing costs in preparation for potential
commercialization in lower risk MDS.
General and administrative expenses for the three and six months
ended June 30, 2023, were $16.5 million and $29.4 million,
respectively, compared to $7.4 million and $14.1 million for the
comparable 2022 periods. The increase in general and administrative
expenses for the three and six months ended June 30, 2023, compared
to the same periods in 2022, primarily reflects new costs for
commercial preparatory activities and higher personnel-related
expenses for additional headcount.
Interest income was $4.7 million and $8.6 million for the three
and six months ended June 30, 2023, respectively, compared to
$330,000 and $442,000 for the same periods in 2022. The increase in
interest income for the three and six months ended June 30, 2023,
compared to the same periods in 2022, primarily reflects higher
yields on the Company’s marketable securities as a result of rising
interest rates, as well as a larger investment portfolio with the
cash proceeds from the January 2023 public offering and warrant
exercises in the first half of 2023.
Interest expense was $2.0 million and $3.9 million for the three
and six months ended June 30, 2023, respectively, compared to $1.6
million and $3.1 million for the same periods in 2022. The increase
in interest expense for the three and six months ended June 30,
2023, compared to the same periods in 2022, primarily reflects
higher interest rates. Currently, the Company has $50.0 million in
principal debt outstanding.
Projected 2023 Financial Guidance Reaffirmed
For fiscal year 2023, under generally accepted accounting
principles (GAAP), the Company continues to expect total expenses
in the range of approximately $210 million to $220 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 expenses for
fiscal year 2023 to be in the range of approximately $200 million
to $210 million. This guidance excludes estimated non-cash items
such as: stock-based compensation expense, amortization of debt
discounts and issuance costs, as well as depreciation and
amortization.
The fiscal year 2023 financial guidance reflects costs to
support planned regulatory submissions in 2023; continued support
of ongoing clinical trials, IMerge Phase 3, IMpactMF, IMproveMF and
IMpress, as well as preclinical studies in lymphoid malignancies
and discovery research for a next generation telomerase inhibitor;
manufacturing commercial inventory of imetelstat; preparations for
potential U.S. commercial launch of imetelstat in lower risk MDS;
projected increases in headcount and interest payments on
outstanding debt.
As of June 30, 2023, the Company had 133 employees. The Company
plans to grow to a total of approximately 150 to 160 employees by
year-end 2023.
Conference Call
Geron will host a conference call at 4:30 p.m. ET on Thursday,
August 3, 2023 to discuss business updates, and second quarter
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.
Inducement Grants Under Nasdaq Listing Rule
5635(c)(4)
In connection with the commencement of Mr. Samuels’ employment
with the Company on August 1, 2023, the Company granted him
non-statutory stock options to purchase an aggregate of 1,600,000
shares of Geron common stock. Stock options representing an
aggregate of 1,350,000 shares have a 10-year term and vest over
four years, with 12.5% of the shares underlying the options vesting
on the six-month anniversary of commencement of employment and the
remaining shares vesting over the following 42 months in equal
installments of whole shares, subject to continued employment with
Geron through the applicable vesting dates. Stock options
representing an aggregate of 250,000 shares have a 10-year term and
vest in full upon achievement of a certain regulatory milestone,
subject to continued employment with Geron through the applicable
vesting date. All of the stock options were granted as a material
inducement to employment in accordance with Nasdaq Listing Rule
5635(c)(4) and are subject to the terms and conditions of the stock
option agreements covering the grants and Geron’s 2018 Inducement
Award Plan, which was adopted December 14, 2018 and provides for
the granting of stock options to new employees.
About Imetelstat
Imetelstat is a novel, first-in-class telomerase inhibitor
exclusively owned by Geron and being developed in hematologic
malignancies. Data from non-clinical studies and clinical trials of
imetelstat 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 U.S. Food and Drug Administration (FDA) for both
the treatment of adult patients with transfusion dependent anemia
due to Low or Intermediate-1 risk MDS that is not associated with
del(5q) who are refractory or resistant to an erythropoiesis
stimulating agent, and for adult patients with Intermediate-2 or
High-risk MF whose disease has relapsed after or is refractory to
janus associated kinase (JAK) inhibitor treatment. Geron submitted
a New Drug Application (NDA) in the U.S. in June 2023 and expects
to submit a Marketing Authorization Application (MAA) in the EU in
the fourth quarter of 2023 in the lower risk MDS indication.
Imetelstat is currently not approved by any regulatory
authority.
About IMerge Phase 3
The Phase 3 portion of the IMerge Phase 2/3 study is a
double-blind, 2:1 randomized, placebo-controlled clinical trial to
evaluate imetelstat in patients with IPSS Low or Intermediate-1
risk (lower risk) transfusion dependent MDS who were relapsed
after, refractory to, or ineligible for, erythropoiesis stimulating
agent (ESA) treatment, had not received prior treatment with either
a HMA or lenalidomide and were non-del(5q). To be eligible for
IMerge Phase 3, patients were required to be transfusion dependent,
defined as requiring at least four units of packed red blood cells
(RBCs), over an eight-week period during the 16 weeks prior to
entry into the trial. The primary efficacy endpoint of IMerge Phase
3 is the rate of red blood cell transfusion independence (RBC-TI)
lasting at least eight weeks, defined as the proportion of patients
without any RBC transfusion for at least eight consecutive weeks
since entry to the trial (8-week TI). Key secondary endpoints
include the rate of RBC-TI lasting at least 24 weeks (24-week TI),
the duration of TI and the rate of hematologic improvement
erythroid (HI-E), which is defined under 2006 IWG criteria as a
rise in hemoglobin of at least 1.5 g/dL above the pretreatment
level for at least eight weeks or a reduction of at least four
units of RBC transfusions over eight weeks compared with the prior
RBC transfusion burden. A total of 178 patients were enrolled in
IMerge Phase 3 across North America, Europe, Middle East and
Asia.
About IMpactMF Phase 3
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 relapsed after or refractory to prior
treatment with a JAK inhibitor, also referred to as
relapsed/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 IMpress Phase 2
IMpress Phase 2 (NCT05583552) is an open-label, single-arm,
multicenter study aiming to enroll approximately 45 AML and higher
risk MDS patients who are relapsed, refractory, or intolerant to
HMAs. The objective of this trial is to evaluate the efficacy, in
terms of hematologic improvement, of imetelstat in this patient
population. The primary endpoint of this trial is overall response
rate. The combined response assessment criteria for MDS and AML
based on IWG 2018 criteria (MDS) and the criteria of the European
LeukemiaNet (AML) will be used to define responders. Study sites
will be located in Australia, France and Germany.
IMpress Phase 2 is an investigator-led study being led by The
European Myelodysplastic Neoplasms Cooperative Group (EMSCO) and
Australasian Leukaemia & Lymphoma Group (ALLG).
About Geron
Geron is a late-stage clinical biopharmaceutical company
pursuing therapies with the potential to extend and enrich the
lives of patients living with hematologic malignancies. The
Company’s investigational first-in-class telomerase inhibitor,
imetelstat, harnesses Nobel Prize-winning science in a treatment
that may alter the underlying drivers of disease. Geron currently
has a Phase 3 clinical trial underway evaluating imetelstat in each
of: (i) lower risk myelodysplastic syndromes (LR MDS), and (ii)
relapsed/refractory myelofibrosis (MF). To learn more, visit
www.geron.com or follow us on LinkedIn.
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 plans to submit a Marketing Authorization
Application in the EU in the fourth quarter of 2023 and is
preparing for a potential launch in lower risk MDS in the U.S. in
the first half of 2024; (ii) that imetelstat has the potential to
demonstrate disease-modifying activity in patients; (iii) that for
fiscal year 2023, under GAAP the Company expects total expenses to
be in the range of $210 to $220 million, and non-GAAP total
expenses to be in the range of $200 to $210 million; (iv) that the
Company believes the approximately $400 million on the balance
sheet at the end of the second quarter provides the financial
resources to fund a potential successful launch of imetelstat in
lower risk MDS and also to support the first year of launch; (v)
that the IMpactMF interim analysis is expected in the first half of
2025 and the final analysis is expected in the first half of 2026;
(vi) that there is a potentially substantial commercial opportunity
for imetelstat in lower risk MDS; (vii) that IMpactMF has
registrational intent; (viii) that Geron projects its existing
financial resources plus potential future proceeds from remaining
warrants outstanding and estimated revenues from commercialization
will be sufficient to fund its projected operating requirements
through the end of 2025; and (ix) 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
geopolitical events 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 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,
planned milestones and expenses in (i) and (v) 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 has demonstrated sufficient safety, efficacy and
clinical benefit in IMerge Phase 3 to enable regulatory approval;
(e) whether any future safety or efficacy results of imetelstat
treatment 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 additional capital in
order to complete the development and commercialization of
imetelstat to meet the expected timelines, planned milestones and
expenses in (i) and (v) above; (h) whether regulatory authorities
require an additional imetelstat lower risk MDS clinical trial for
approval, or post-approval; (i) whether there are failures or
delays in manufacturing or supplying sufficient quantities of
imetelstat or other clinical trial materials that impact a
commercial launch in lower risk MDS or the continuation of the
IMpactMF trial; (j) whether the follow-up period of 12 months for
the IMerge Phase 3 primary analysis was sufficient to demonstrate
safety and efficacy, including transfusion independence and
clinical benefit, and obtain regulatory approval; (k) for IMerge
Phase 3, the FDA may require Geron to submit additional information
or require advisory committee procedures that could cause a
regulatory approval, if any, to be delayed; and (l) that the timing
in (v) above for IMpactMF may vary depending on actual enrollment
and death rates in the trial. 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, 2023 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 presenting non-GAAP total
expenses, which excludes stock-based compensation expense,
amortization of debt discounts and issuance costs and depreciation
and amortization, from GAAP total 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 non-GAAP financial measure is in
addition to, not a substitute for, or superior to, measures of
financial performance prepared 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 CONSOLIDATED
STATEMENTS OF OPERATIONS
Three Months Ended
Six Months Ended
June
30,
June
30,
(In thousands, except share and per share
data)
2023
2022
2023
2022
Revenues:
Royalties
$
29
$
73
$
50
$
196
Operating expenses:
Research and development
35,490
20,606
62,709
42,705
General and administrative
16,490
7,443
29,384
14,142
Total operating expenses
51,980
28,049
92,093
56,847
Loss from operations
(51,951
)
(27,976
)
(92,043
)
(56,651
)
Interest income
4,738
330
8,591
442
Interest expense
(2,003
)
(1,581
)
(3,925
)
(3,060
)
Other income and expense, net
(11
)
1,110
28
1,054
Net loss
$
(49,227
)
$
(28,117
)
$
(87,349
)
$
(58,215
)
Basic and diluted net loss per
share:
Net loss per share
$
(0.09
)
$
(0.07
)
$
(0.16
)
$
(0.16
)
Shares used in computing net loss per
share
547,280,946
403,868,713
553,772,809
368,166,148
CONDENSED CONSOLIDATED BALANCE
SHEETS
June 30,
December 31,
(In thousands)
2023
2022
(Unaudited)
(Note 1)
Current assets:
Cash, cash equivalents and restricted
cash
$
57,438
$
57,209
Current marketable securities
314,475
115,901
Other current assets
5,366
7,136
Total current assets
377,279
180,246
Noncurrent marketable securities
28,281
—
Property and equipment, net
1,147
793
Deposits and other assets
8,589
9,536
$
415,296
$
190,575
Current liabilities
$
72,569
$
76,694
Noncurrent liabilities
44,300
33,883
Stockholders’ equity
298,427
79,998
$
415,296
$
190,575
Note 1: Derived from audited financial statements included in
the Company’s annual report on Form 10-K for the year ended
December 31, 2022.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230803480128/en/
Aron Feingold Investor and Media Relations investor@geron.com
media@geron.com
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