Geron Corporation Reports Second Quarter 2024 Financial Results and Business Highlights
NCCN Guidelines® updated to include the use of RYTELO in both RS+ and RS- patients for second-line treatment of symptomatic anemia in patients with lower-risk MDS and for first-line treatment of patients who are
“We are thrilled to have begun the launch of RYTELO, our first commercial product, in June, and are encouraged by the early success we are seeing and the reception from the medical community over these first six weeks," said
Business Highlights
-
Received approval on
June 6, 2024 from theU.S. Food & Drug Administration (FDA) of RYTELO for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent (TD) anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs). -
Launched RYTELO commercially in the
U.S. , with both dosage strengths available for prescribers to order from specialty distributors as ofJune 27, 2024 . -
The National Comprehensive Cancer Network® (NCCN®) updated the MDS NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) to include RYTELO for the treatment of symptomatic anemia in patients with LR-MDS. For both RS+ and RS- patients, RYTELO has a Category 1 designation for second-line treatment regardless of first-line treatment and a Category 2A designation for first-line treatment of patients who are
ESA ineligible (serum EPO >500 mU/mL). -
Achieved approximately 70% enrollment in the Phase 3 IMpactMF trial of imetelstat as of
August 2024 , which has a primary endpoint of overall survival, in patients with myelofibrosis (MF) who are relapsed/refractory to JAK-inhibitors. As previously disclosed, based on the most recent planning assumptions for enrollment and death rates in the trial, an interim analysis is expected in early 2026 (when approximately 35% of planned enrolled patients have died) and final analysis is expected in early 2027 (when approximately 50% of planned enrolled patients have died). -
Expanded the dose level 4 cohort (imetelstat 9.4 mg/kg) in the Part 1 dose-finding stage of the Phase 1 IMproveMF study evaluating imetelstat as a combination therapy with ruxolitinib in patients with frontline MF. This followed a unanimous decision by the study’s Safety Evaluation Team (SET), who in
July 2024 reviewed data from the first three patients in the dose level 4 cohort and identified no dose-limiting toxicities.
Second Quarter 2024 Financial Results
As of
Net Loss
For the three and six months ended
Revenues
Total product revenue, net for the three and six months ended
Total net revenues for the three and six months ended
Operating Expenses
Total operating expenses for the three and six months ended
Cost of goods sold was approximately
Research and development expenses for the three months and six months ended
Selling, general and administrative expenses for the three and six months ended
Interest income was
Interest expense was
2024 Financial Guidance
For fiscal year 2024, we expect total operating expenses to be in the range of approximately
Based on our current operating plans and assumptions, we believe that our existing cash, cash equivalents, and marketable securities, together with projected revenues from
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/182024239
About RYTELO (imetelstat)
RYTELO (imetelstat) is an FDA-approved oligonucleotide telomerase inhibitor for the treatment of adult patients with low-to-intermediate-1 risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs). It is indicated to be administered as an intravenous infusion over two hours every four weeks.
RYTELO is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Telomeres are protective caps at the end of chromosomes that naturally shorten each time a cell divides. In LR-MDS, abnormal bone marrow cells often express the enzyme telomerase, which rebuilds those telomeres, allowing for uncontrolled cell division. Developed and exclusively owned by
About
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/study/NCT04576156.
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.
IMPORTANT SAFETY INFORMATION ABOUT RYTELO
WARNINGS AND PRECAUTIONS
Thrombocytopenia
RYTELO can cause thrombocytopenia based on laboratory values. In the clinical trial, new or worsening Grade 3 or 4 decreased platelets occurred in 65% of patients with MDS treated with RYTELO.
Monitor patients with thrombocytopenia for bleeding. Monitor complete blood cell counts prior to initiation of RYTELO, weekly for the first two cycles, prior to each cycle thereafter, and as clinically indicated. Administer platelet transfusions as appropriate. Delay the next cycle and resume at the same or reduced dose, or discontinue as recommended.
Neutropenia
RYTELO can cause neutropenia based on laboratory values. In the clinical trial, new or worsening Grade 3 or 4 decreased neutrophils occurred in 72% of patients with MDS treated with RYTELO.
Monitor patients with Grade 3 or 4 neutropenia for infections, including sepsis. Monitor complete blood cell counts prior to initiation of RYTELO, weekly for the first two cycles, prior to each cycle thereafter, and as clinically indicated. Administer growth factors and anti-infective therapies for treatment or prophylaxis as appropriate. Delay the next cycle and resume at the same or reduced dose, or discontinue as recommended.
Infusion-Related Reactions
RYTELO can cause infusion-related reactions. In the clinical trial, infusion-related reactions occurred in 8% of patients with MDS treated with RYTELO; Grade 3 or 4 infusion-related reactions occurred in 1.7%, including hypertensive crisis (0.8%). The most common infusion-related reaction was headache (4.2%). Infusion-related reactions usually occur during or shortly after the end of the infusion.
Premedicate patients at least 30 minutes prior to infusion with diphenhydramine and hydrocortisone as recommended and monitor patients for one hour following the infusion as recommended. Manage symptoms of infusion-related reactions with supportive care and infusion interruptions, decrease infusion rate, or permanently discontinue as recommended.
Embryo-Fetal Toxicity
RYTELO can cause embryo-fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with RYTELO and for 1 week after the last dose.
ADVERSE REACTIONS
Serious adverse reactions occurred in 32% of patients who received RYTELO. Serious adverse reactions in >2% of patients included sepsis (4.2%) and fracture (3.4%), cardiac failure (2.5%), and hemorrhage (2.5%). Fatal adverse reactions occurred in 0.8% of patients who received RYTELO, including sepsis (0.8%).
Most common adverse reactions (≥10% with a difference between arms of >5% compared to placebo), including laboratory abnormalities, were decreased platelets, decreased white blood cells, decreased neutrophils, increased AST, increased alkaline phosphatase, increased ALT, fatigue, prolonged partial thromboplastin time, arthralgia/myalgia, COVID-19 infections, and headache.
Please see RYTELO (imetelstat) full Prescribing Information, including Medication Guide, available at https://pi.geron.com/products/US/pi/rytelo_pi.pdf.
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 Company’s views, estimates and expectations concerning the commercial launch of RYTELO, including estimates of accounts reached and patients receiving RYTELO; (ii) the potential impact on clinical decision-making, prescriber behavior and reimbursement decisions of the inclusion of RYTELO in the NCCN Guidelines as a Category 1 and 2A treatment of symptomatic anemia in patients with lower-risk MDS; (iii) the Company seeing increasing dialogue among hematologists rethinking treatment approaches for eligible patients with lower-risk MDS with transfusion-dependent anemia, regardless of ring sideroblast status, and the Company’s belief that RYTELO can become part of the standard-of-care for these patients; (iv) that the interim analysis of IMpactMF is expected in early 2026 and the final analysis is expected in early 2027; (v) the Company’s projections and expectations regarding the sufficiency of its cash resources and expected available resources to fund its projected operating requirements into Q2 2026, and the assumptions underlying such projections and expectations; (vi) the Company’s projections for total operating expenses for fiscal 2024 and employee headcount as of the end of 2024; (vii) that inhibiting telomerase activity aims to potentially reduce proliferation and induce death of malignant cells; (viii) that
Financial table follows.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
|
|||||||||||||||
|
Three Months Ended |
Six Months Ended |
|||||||||||||
|
|
|
|||||||||||||
(In thousands, except share and per share data) |
2024 |
|
2023 |
|
2024 |
|
2023 |
|
|||||||
|
(Unaudited) |
(Unaudited) |
(Unaudited) |
(Unaudited) |
|||||||||||
Revenues: |
|
|
|
|
|||||||||||
Product revenue, net |
$ |
780 |
|
$ |
- |
|
$ |
780 |
|
$ |
- |
|
|||
Royalties |
|
102 |
|
|
29 |
|
|
406 |
|
|
50 |
|
|||
|
|
882 |
|
|
29 |
|
|
1,186 |
|
|
50 |
|
|||
Operating expenses: |
|
|
|
|
|
|
|
|
|||||||
Cost of goods sold |
|
17 |
|
|
- |
|
|
17 |
|
|
- |
|
|||
Research and development |
|
30,779 |
|
|
35,490 |
|
|
60,152 |
|
|
62,709 |
|
|||
Selling, general and administrative |
|
39,419 |
|
|
16,490 |
|
|
66,484 |
|
|
29,384 |
|
|||
Total operating expenses |
|
70,215 |
|
|
51,980 |
|
|
126,653 |
|
|
92,093 |
|
|||
|
|
|
|
|
|
|
|
|
|||||||
Loss from Operations |
|
(69,333 |
) |
|
(51,951 |
) |
|
(125,467 |
) |
|
(92,043 |
) |
|||
|
|
|
|
|
|
|
|
|
|||||||
Interest income |
|
5,332 |
|
|
4,738 |
|
|
9,571 |
|
|
8,591 |
|
|||
Interest expense |
|
(3,319 |
) |
|
(2,003 |
) |
|
(6,752 |
) |
|
(3,925 |
) |
|||
Other income and (expense), net |
|
(63 |
) |
|
(11 |
) |
|
(125 |
) |
|
28 |
|
|||
Net loss |
$ |
(67,383 |
) |
$ |
(49,227 |
) |
$ |
(122,773 |
) |
$ |
(87,349 |
) |
|||
|
|
|
|
|
|||||||||||
Basic and diluted net loss per share: |
|
|
|
|
|
|
|
|
|||||||
Net loss per share |
$ |
(0.10 |
) |
$ |
(0.09 |
) |
$ |
(0.19 |
) |
$ |
(0.16 |
) |
|||
Shares used in computing net loss per share |
|
653,904,978 |
|
|
547,280,946 |
|
|
628,699,214 |
|
|
553,772,809 |
|
CONDENSED CONSOLIDATED BALANCE SHEETS |
||||||||
|
|
|
|
|
|
|
||
(In thousands) |
|
2024 |
|
|
2023 |
|
||
|
|
(Unaudited) |
|
|
(Note 1) |
|
||
Current assets: |
|
|
|
|
|
|
||
Cash, cash equivalents and restricted cash |
|
$ |
118,068 |
|
|
$ |
71,138 |
|
Current marketable securities |
|
|
245,789 |
|
|
|
263,676 |
|
Other current assets |
|
|
9,451 |
|
|
|
6,534 |
|
Total current assets |
|
|
373,308 |
|
|
|
341,348 |
|
|
|
|
|
|
|
|
||
Noncurrent marketable securities |
|
|
66,505 |
|
|
|
43,298 |
|
Property and equipment, net |
|
|
1,626 |
|
|
|
1,177 |
|
Deposits and other assets |
|
|
7,960 |
|
|
|
8,253 |
|
|
|
$ |
449,399 |
|
|
$ |
394,076 |
|
|
|
|
|
|
|
|
||
Current liabilities |
|
$ |
103,540 |
|
|
$ |
108,070 |
|
Noncurrent liabilities |
|
|
39,164 |
|
|
|
38,057 |
|
Stockholders’ equity |
|
|
306,695 |
|
|
|
247,949 |
|
|
|
$ |
449,399 |
|
|
$ |
394,076 |
|
Note 1: Derived from audited financial statements included in the Company’s annual report on Form 10-K for the year ended |
View source version on businesswire.com: https://www.businesswire.com/news/home/20240808881582/en/
Vice President, Investor Relations and Corporate Communications
Associate Director, Investor Relations and Corporate Communications
investor@geron.com
media@geron.com
Source: