Geron Corporation Reports Business Highlights and Fourth Quarter and Full Year 2023 Financial Results
“Geron’s progress and execution throughout 2023 has paved the way for a potentially transformational 2024, as we plan for the transition to becoming a commercial company,” said
2023 Business Highlights
Transfusion-Dependent Lower-Risk Myelodysplastic Syndromes (TD LR-MDS)
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In
January 2023 ,Geron reported positive top-line results from the pivotal IMerge Phase 3 clinical trial evaluating imetelstat in patients with TD LR-MDS. Additional data including subgroup analyses and patient-reported outcomes were subsequently reported at theEuropean Hematology Association Annual Meeting inJune 2023 and at theAmerican Society of Hematology Annual Meeting and published inThe Lancet inDecember 2023 . -
In
August 2023 , theU.S. Food & Drug Administration (FDA) accepted the New Drug Application (NDA) for imetelstat for the treatment of TD anemia in adult patients with low- to intermediate-1 risk myelodysplastic syndromes, who have failed to respond, or have lost response to, or are ineligible for erythropoiesis-stimulating agents (ESAs). The FDA assigned a Prescription Drug User Fee Act (PDUFA) action date ofJune 16, 2024 to the NDA. -
On
January 30, 2024 , the FDA provided notice in theFederal Register that it has scheduled a publicOncologic Drugs Advisory Committee to be held virtually onMarch 14, 2024 , as part of the imetelstat NDA review. -
In
September 2023 , theEuropean Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for imetelstat in the same proposed indication as the NDA.
Myelofibrosis
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In
December 2023 ,Geron achieved fifty percent enrollment in the Phase 3 IMpactMF clinical trial investigating imetelstat versus best available therapy (BAT) in patients with intermediate-2 or high-risk Myelofibrosis (MF) who are relapsed/refractory myelofibrosis (R/R MF) to Janus kinase (JAK) inhibitor treatment. -
In
January 2024 , dosing in the Phase 1 IMproveMF study evaluating imetelstat as a combination therapy with ruxolitinib in patients with intermediate-2 or high-risk MF (frontline MF) was escalated to the third of four doses following a decision by the study’s independent Safety Evaluation Team (SET).
Corporate
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In
November 2023 ,Geron appointedGaurav Aggarwal , M.D., a prominent investor in the life sciences sector for more than two decades who has a history of expertise in financial and corporate strategy, as well as business development, to Geron’s Board of Directors. -
In August and
September 2023 , respectively,Geron appointedScott Samuels as Executive Vice President, Chief Legal Officer and Corporate Secretary, andMichelle Robertson as Executive Vice President, Chief Financial Officer and Treasurer. BothMr. Samuels andMs. Robertson joinGeron with a track record of excellence in commercial-stage biopharmaceutical companies.
Anticipated Upcoming Milestones
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U.S. commercial launch of imetelstat upon potential FDA approval (PDUFA dateJune 16, 2024 ) for the treatment of transfusion-dependent anemia in adult patients with LR-MDS who have failed to respond, or have lost response to, or are ineligible for ESAs. -
Review of the imetelstat MAA for the same indication as the NDA expected to be completed in early 2025. Subject to approval by the
European Commission , EU commercial launch of imetelstat could occur in 2025. - Interim analysis from the Phase 3 IMpactMF trial in R/R MF expected in the first half of 2025, with a final analysis from the study expected in the first half of 2026.
Throughout 2023,
Fourth Quarter and Full Year 2023 Financial Results
As of
Revenues for the three and twelve months ended
Total operating expenses for the three and twelve months ended
Research and development expenses for the three and twelve months ended
General and administrative expenses for the three and twelve months ended
Interest income was
Interest expense was
Projected 2024 Financial Guidance
For fiscal year 2024, the Company expects total operating expenses to be in the range of approximately
The fiscal year 2024 financial guidance reflects costs to support regulatory processes with the FDA and EMA in 2024; continued support of ongoing clinical trials; manufacturing of commercial inventory of imetelstat; continued build out of our commercial organization to support the potential
As of
Conference Call
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://events.q4inc.com/attendee/964382933.
About Imetelstat
Imetelstat is a novel, first-in-class investigational telomerase inhibitor exclusively owned by
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
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 MF who are relapsed after or refractory to prior treatment with a JAK inhibitor, also referred to as R/R MF. Patients will be randomized to receive either imetelstat or BAT. 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.
About IMproveMF
IMproveMF is a single arm, open label, two-part Phase 1 study to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of imetelstat in combination with ruxolitinib as a frontline treatment in patients with Intermediate-2 or High-risk MF (frontline MF). In both parts, patients will receive ruxolitinib followed by imetelstat, a dosing schedule that showed synergistic and additive effects of the two agents in preclinical experiments. Part 1 will enroll up to 20 frontline MF patients who, at the time of enrollment, have received an optimized dose of ruxolitinib, to which imetelstat treatment will be added at increasing dose levels based on safety and tolerability. The primary purpose of Part 1 is to identify a safe dose for treating frontline MF patients with a combination of imetelstat and ruxolitinib. If a safe dose is identified in Part 1, participants in Part 2 will be JAK inhibitor naïve and will receive treatment with ruxolitinib after screening and enrollment at a starting dose based on standard of care or local prescribing information. Treatment with single-agent ruxolitinib will continue for at least 12 weeks, including four consecutive weeks at a stable dose prior to the addition of imetelstat. Part 2 is designed to confirm the safety profile of imetelstat in combination with ruxolitinib and to evaluate for preliminary clinical activity of the combination.
About
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) the potential for a transformational year for the Company in 2024, the Company’s plans to transition to becoming a commercial company, and the potential for value creation and significant commercial opportunities; (ii) plans for a potential launch in TD LR-MDS in the
Financial tables follow.
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CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS |
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(Unaudited) |
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Three Months Ended |
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Year Ended |
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|
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(In thousands, except share and per share data) |
|
2023 |
|
2022 |
|
2023 |
|
2022 |
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|
|
|
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Revenues: |
|
|
|
|
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Royalties |
$ |
23 |
|
$ |
103 |
|
$ |
237 |
|
$ |
596 |
|
||||
|
|
|
|
|
|
|
|
|
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Operating expenses: |
|
|
|
|
|
|
|
|
||||||||
Research and development |
|
32,911 |
|
|
28,210 |
|
|
125,046 |
|
|
95,518 |
|
||||
General and administrative |
|
21,401 |
|
|
13,844 |
|
|
69,135 |
|
|
43,628 |
|
||||
Total operating expenses |
54,312 |
|
42,054 |
|
194,181 |
|
139,146 |
|
||||||||
Loss from operations |
|
(54,289 |
) |
|
(41,951 |
) |
|
(193,944 |
) |
|
(138,550 |
) |
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|
|
|
|
|
|
|
|
|
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Interest income |
|
4,595 |
|
|
1,235 |
|
|
18,152 |
|
|
2,529 |
|
||||
Interest expense |
|
(2,321 |
) |
|
(2,005 |
) |
|
(8,312 |
) |
|
(6,822 |
) |
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Other income and expense, net |
|
41 |
|
|
86 |
|
|
(23 |
) |
|
1,002 |
|
||||
Net loss |
$ |
(51,974 |
) |
$ |
(42,635 |
) |
$ |
(184,127 |
) |
$ |
(141,901 |
) |
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|
|
|
|
|
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Basic and diluted net loss per share: |
|
|
|
|
|
|
|
|
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Net loss per share |
$ |
(0.09 |
) |
$ |
(0.10 |
) |
$ |
(0.32 |
) |
$ |
(0.37 |
) |
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Shares used in computing net loss per share |
|
594,977,503 |
|
|
408,143,890 |
|
|
570,645,405 |
|
|
380,784,846 |
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CONDENSED CONSOLIDATED BALANCE SHEETS |
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(In thousands) |
2023 |
2022 |
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|
|
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Current assets: |
|
|
||||
Cash, cash equivalents and restricted cash |
$ |
71,138 |
$ |
57,209 |
||
Current marketable securities |
|
263,676 |
|
115,901 |
||
Other current assets |
|
6,534 |
|
7,136 |
||
Total current assets |
|
341,348 |
|
180,246 |
||
|
|
|
||||
Noncurrent marketable securities |
|
43,298 |
|
— |
||
Property and equipment, net |
|
1,177 |
|
793 |
||
Deposits and other assets |
|
8,253 |
|
9,536 |
||
|
$ |
394,076 |
$ |
190,575 |
||
|
|
|
||||
Current liabilities |
$ |
108,070 |
$ |
76,694 |
||
Noncurrent liabilities |
|
38,057 |
|
33,883 |
||
Stockholders’ equity |
|
247,949 |
|
79,998 |
||
|
$ |
394,076 |
$ |
190,575 |
View source version on businesswire.com: https://www.businesswire.com/news/home/20240228164846/en/
Vice President, Investor Relations and Corporate Communications
Senior Manager, Investor Relations
investor@geron.com
media@geron.com
Source: