Geron Corporation Reports Business Highlights and Fourth Quarter and Full Year 2023 Financial Results
- Positive results from pivotal Phase 3 IMerge trial for imetelstat in TD LR-MDS patients.
- FDA acceptance of NDA for imetelstat with a PDUFA action date of June 16, 2024.
- European Medicines Agency validation of MAA for imetelstat in the same indication.
- Fifty percent enrollment achieved in Phase 3 IMpactMF trial for MF patients.
- Appointment of experienced professionals to the Board and executive positions.
- Strong financial position with $378.1 million in cash, cash equivalents, and marketable securities as of December 31, 2023.
- Projected financial resources to fund operating expenses into Q3 2025.
- Expected total operating expenses of $270 million to $280 million for fiscal year 2024.
- Plans to grow employee count to approximately 270 by year-end 2024.
- Conference call scheduled for February 28, 2024, to discuss business updates and financial results.
- Decline in revenues for the three and twelve months ended December 31, 2023, compared to the same periods in 2022.
- Increase in total operating expenses for the three and twelve months ended December 31, 2023, compared to the same periods in 2022.
- Higher interest expense for the three and twelve months ended December 31, 2023, compared to the same periods in 2022.
Insights
The financial results and business highlights reported by Geron Corporation reveal a strategic trajectory towards becoming a commercial entity, underscored by their progress with the imetelstat NDA. The company's financial health, as indicated by the $378.1 million in cash, cash equivalents and marketable securities, reflects a robust balance sheet that is expected to fund operations into the third quarter of 2025. This is a critical factor for investors, as it suggests that Geron has a sufficient runway to reach key milestones without the immediate need for additional capital-raising activities, which could dilute shareholder value.
The projected operating expenses for 2024, ranging from $270 million to $280 million, are substantial and should be considered in the context of the company's transition to a commercial-stage company. The increase in operating expenses year-over-year is aligned with the costs associated with preparing for commercial launch, which includes scaling up the workforce from 141 to an anticipated 270 employees. This planned growth in headcount is indicative of Geron's confidence in imetelstat's approval and commercial prospects.
However, the relatively low revenue figures reported for 2023, primarily from royalties of divested assets, highlight the company's current reliance on the successful commercialization of imetelstat. The financial guidance provided for 2024 will likely be adjusted post-approval, as the company transitions from a development-stage to a revenue-generating commercial entity.
The acceptance of the New Drug Application (NDA) by the FDA for imetelstat, a telomerase inhibitor, marks a significant milestone for Geron Corporation in the treatment of transfusion-dependent anemia in patients with lower-risk Myelodysplastic Syndromes (MDS). The Prescription Drug User Fee Act (PDUFA) action date set for June 16, 2024, represents a pivotal moment for the company, as approval would enable Geron to transition from a clinical-stage to a commercial-stage company.
The positive top-line results from the IMerge Phase 3 clinical trial and the subsequent detailed data presentations at major hematology meetings bolster the drug's profile. The clinical significance of imetelstat lies in its novel mechanism of action as a telomerase inhibitor, potentially offering a new therapeutic option for patients with lower-risk MDS, a group for whom treatment options are currently limited. The anticipation of commercial launch in the U.S., subject to FDA approval, could disrupt the MDS treatment landscape and offer substantial value creation for Geron.
Additionally, the progress in the IMpactMF clinical trial for patients with Myelofibrosis who are relapsed/refractory to JAK inhibitor treatment suggests that imetelstat could address unmet needs in a broader hematologic malignancy population. This expansion into additional indications could further enhance the drug's commercial potential.
Geron's pre-commercial activities, including securing a global trademark, establishing logistics and distribution networks and onboarding commercial and medical affairs teams, indicate a comprehensive strategy to ensure a successful market entry for imetelstat. The company's market research in transfusion-dependent lower-risk MDS (TD LR-MDS) is critical in understanding the target demographic and tailoring marketing strategies accordingly.
The expected commercial launch in the U.S., followed by a potential EU launch in 2025, subject to European Commission approval, represents a strategic move to capture key markets in the treatment of MDS. The engagement in marketing, commercial access, payer and reimbursement efforts is essential for market penetration and adoption of imetelstat, should it receive regulatory approval.
Investors should note the potential for significant commercial opportunities, considering the rarity of new entrants in the MDS treatment space and the therapeutic promise of imetelstat. The company's focus on TD LR-MDS and relapsed/refractory Myelofibrosis positions it within niche markets that often face limited competition and high unmet medical need, which could translate into favorable pricing and reimbursement scenarios.
June 16, 2024 PDUFA date for imetelstat NDA for the treatment of transfusion-dependent anemia in adult patients with lower-risk MDS
“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 John A. Scarlett, M.D., Chairman and Chief Executive Officer. “We believe that we are in a strong position for value creation, based on our differentiated product candidate, the potential for significant commercial opportunities in transfusion-dependent, lower-risk MDS and relapsed/refractory MF, the excellence and experience of our employees, and the strength of our balance sheet to support a potential
2023 Business Highlights
Transfusion-Dependent Lower-Risk Myelodysplastic Syndromes (TD LR-MDS)
- 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 the European Hematology Association Annual Meeting in June 2023 and at the American Society of Hematology Annual Meeting and published in The Lancet in December 2023.
-
In August 2023, the
U.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 of June 16, 2024 to the NDA. - On January 30, 2024, the FDA provided notice in the Federal Register that it has scheduled a public Oncologic Drugs Advisory Committee to be held virtually on March 14, 2024, as part of the imetelstat NDA review.
- In September 2023, the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for imetelstat in the same proposed indication as the NDA.
Myelofibrosis
- 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
- In November 2023, Geron appointed Gaurav 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 appointed Scott Samuels as Executive Vice President, Chief Legal Officer and Corporate Secretary, and Michelle Robertson as Executive Vice President, Chief Financial Officer and Treasurer. Both Mr. Samuels and Ms. Robertson join Geron with a track record of excellence in commercial-stage biopharmaceutical companies.
Anticipated Upcoming Milestones
-
U.S. commercial launch of imetelstat upon potential FDA approval (PDUFA date June 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, Geron completed several long-lead time pre-commercial activities, including securing a global trademark for the imetelstat brand name; finalizing third party logistics, our distribution network, patient support providers; and onboarding highly experienced commercial and medical affairs teams. Other pre-commercial preparations for the
Fourth Quarter and Full Year 2023 Financial Results
As of December 31, 2023, the Company had
Revenues for the three and twelve months ended December 31, 2023, were
Total operating expenses for the three and twelve months ended December 31, 2023, were
Research and development expenses for the three and twelve months ended December 31, 2023, were
General and administrative expenses for the three and twelve months ended December 31, 2023, were
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 December 31, 2023, the Company had 141 full-time employees. Subject to approval of imetelstat in the
Conference Call
Geron will host a conference call at 8:00 am ET on Wednesday, February 28, 2024, to discuss business updates and fourth quarter and full year 2023 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://events.q4inc.com/attendee/964382933.
About Imetelstat
Imetelstat is a novel, first-in-class investigational 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 suggesting potential disease-modifying activity. Imetelstat has been granted Fast Track designation by the
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 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. Our first-in-class investigational telomerase inhibitor, imetelstat, harnesses Nobel Prize-winning science in a treatment that may alter the underlying drivers of disease. The New Drug Application (NDA) for imetelstat for the treatment of transfusion dependent anemia in patients with lower-risk myelodysplastic syndromes (TD LR-MDS) who have failed to respond or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs), based on the results from the Phase 3 IMerge clinical trial, is currently under review by the United States Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) target action date of June 16, 2024. In addition, an MAA is under review in the European Union for the same proposed indication. Furthermore, Geron currently has an ongoing pivotal Phase 3 clinical trial evaluating imetelstat in relapsed/refractory myelofibrosis (R/R 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) 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.
GERON CORPORATION |
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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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December 31, |
|
December 31, |
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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 |
) |
||||
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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|
December 31, |
December 31, |
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(In thousands) |
2023 |
2022 |
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|
|
|
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Current assets: |
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|
||||
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/
Aron Feingold
Vice President, Investor Relations and Corporate Communications
Kristen Kelleher
Senior Manager, Investor Relations
investor@geron.com
media@geron.com
Source: Geron Corporation
FAQ
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