On March 11, 2025 Mural Oncology plc (Nasdaq: MURA), a clinical-stage immuno-oncology company developing novel, investigational engineered therapies targeting cytokine pathways designed to address areas of unmet need for patients with a variety of cancers, reported its financial results for the fourth quarter and year ended December 31, 2024 and provided a business update (Press release, Mural Oncology, MAR 11, 2025, View Source [SID1234651070]).

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"By prioritizing operational efficiency and execution in 2024, we delivered on our milestones and positioned ourselves for a pivotal 2025, with several key inflection points anticipated for our nemvaleukin program. Late this quarter or early next quarter, we will report the interim overall survival analysis for ARTISTRY-7, a potentially registrational trial in platinum-resistant ovarian cancer. As is typical for interim analyses, the bar for success is high. We believe that either declaring the trial complete at the interim analysis or deciding to progress the trial to a final analysis based on the available overall survival data would be a meaningful step forward both for patients and for Mural. Additionally, topline data from ARTISTRY-6 in mucosal melanoma, expected in the second quarter of 2025, represents another potentially significant opportunity for impact and value creation," said Caroline Loew, Ph.D., CEO of Mural Oncology.

Recent Corporate Highlights

In January 2025, Mural announced that, consistent with the company’s prior timing projections, the phase 3 ARTISTRY-7 trial reached the 75% of overall survival (OS) events necessary for the planned interim analysis. This data remains blinded to the company until after the independent data monitoring committee (IDMC) has reviewed the interim analysis, which is expected to be in late Q1/early Q2 2025.

In January 2025, the company also announced that patient enrollment in cohort 3 of the phase 2 ARTISTRY-6 trial is now complete.

Mural expanded its pipeline in Q4 2024 by nominating two development candidates:

MURA-8518, the company’s interleukin-18 (IL-18) program, is designed to deliver a more sustained immune response by introducing half-life extension and resistance to IL-18 Binding Protein (IL-18BP), which otherwise neutralizes the native cytokine’s efficacy.
MURA-7012, Mural’s IL-12 program, is designed to leverage native IL-12’s anti-tumor potency while mitigating its hallmark toxicity. It splits the IL-12p70 heterodimer into two individual sub-units designed to preferentially self-assemble at the tumor site to limit systemic exposure.
Upcoming Milestones

Late Q1/early Q2 2025: Interim data readout of ARTISTRY-7

ARTISTRY-7 is a potentially registrational phase 3 trial evaluating nemvaleukin alfa in combination with pembrolizumab versus investigator’s choice single agent chemotherapy in patients with platinum-resistant ovarian cancer (PROC). Consistent with interim analyses, there is a higher statistical bar for success at the interim analysis compared to the final analysis. If the hazard ratio at the interim analysis meets this pre-specified higher bar for success at the interim analysis (0.727, or a 27.3% reduction in the risk of death assuming exactly 215 OS events), the company plans to submit a Biologics License Application (BLA) for nemvaleukin in combination with pembrolizumab for the treatment of PROC in 2025. If the hazard ratio does not meet the statistical threshold for success at the interim analysis and the company deems the study to have a high probability of success at the final analysis, Mural expects to continue the trial to the protocol-specified final OS. At the final OS analysis, the maximum hazard ratio for success is 0.788, or a 21.2% reduction in the risk of death, assuming exactly 286 events. In that scenario, the company expects to report final OS result in the second quarter of 2026, subject to event accrual.

Q2 2025: Top-line data readout of ARTISTRY-6, Cohort 2

ARTISTRY-6, cohort 2 is a potentially registrational phase 2 trial of nemvaleukin monotherapy in patients with unresectable or metastatic mucosal melanoma previously treated with immune checkpoint blockade. Nemvaleukin has been granted Orphan Drug Designation by the United States Food & Drug Administration (FDA) for the treatment of mucosal melanoma. The target response rate in the ARTISTRY-6 trial is 25%. Mural believes that in this rare and highly aggressive tumor, which has historically had poor outcomes even in the first line setting, demonstrating durable responses with a response rate of 20-25% would be meaningful for patients, and would support a discussion with the FDA regarding a BLA submission and potential accelerated approval.

1H 2025: Preliminary data readout of ARTISTRY-6, Cohort 3

This trial is an evaluation of less-frequent intravenous (LFIV) dosing of nemvaleukin monotherapy in patients with cutaneous melanoma. The company is conducting the trial to evaluate the activity and further characterize the safety of nemvaleukin with LFIV dosing in patients with cutaneous melanoma.

2H 2025: Preliminary data readout of ARTISTRY-6, Cohort 4

This trial is an evaluation of LFIV dosing of nemvaleukin in combination with pembrolizumab in patients with cutaneous melanoma.

1H 2026: Submission of Investigational New Drug or Clinical Trial Application for a phase 1 trial of MURA-8518

MURA-8518 is Mural’s IL-18 development candidate. Native IL-18 is a potent immune-stimulating cytokine, but its activity is blunted by IL-18BP, a high affinity decoy protein that neutralizes IL-18, thereby rendering it ineffective. The native cytokine’s potency is also limited by its short half-life. MURA-8518 aims to address these shortcomings in two ways. First, through the introduction of mutations designed to minimally impact the native structure while eliminating binding to IL-18BP. Secondly, half-life extension via fusion to a protein scaffold increases the cytokine’s exposure, allowing for sustained immune stimulation. Together, these have demonstrated more durable immunological effects in preclinical studies.

Financial Results for the Quarter Ended December 31, 2024

Cash Position: As of December 31, 2024, cash, cash equivalents, and marketable securities were $144.4 million.

R&D Expenses: Research and development expenses were $28.7 million for the fourth quarter of 2024 compared to $42.2 million for the fourth quarter of 2023. This decrease was primarily due to a decrease in employee-related expenses, including a non-cash share-based employee compensation charge in the fourth quarter of 2023 as a result of the impact of the modification of our share based-awards in connection with the separation from Alkermes plc ("Alkermes"), our former parent.

In addition, the timing of patient enrollment in the ARTISTRY-7 trial, as well as the winding down of the ARTISTRY-1 and ARTISTRY-2 trials during 2024 also contributed to the decrease in R&D expenses in the fourth quarter of 2024, as compared to the fourth quarter of 2023.

G&A Expenses: General and administrative expenses were $7.2 million for the fourth quarter of 2024 compared to $16.3 million for the fourth quarter of 2023. This decrease in G&A expenses was primarily due to a decrease in employee-related expenses compared to those previously allocated to us by Alkermes prior to the separation and to one-time increases in employee related expenses in 2023, including a non-cash share-based employee compensation charge in the fourth quarter of 2023 as a result of the impact of the modification of our share based-awards in connection with the separation.

Net Loss: Net loss was $34.3 million for the fourth quarter of 2024 compared to $59.5 million for the fourth quarter of 2023. The net loss for the fourth quarter of 2023 included $11.7 million resulting from one-time charges related to the separation from Alkermes and conversion of Alkermes employee equity awards into Mural equity.

Financial Guidance: Mural’s cash, cash equivalents, and marketable securities as of December 31, 2024 are expected to fund its operations into the first quarter of 2026.

On March 11, 2025 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, reported that the European Medicines Agency (EMA) Pediatric Committee (PDCO) has approved the Pediatric Investigation Plan (PIP) for MaaT013 for the treatment of acute Graft-versus-Host Disease (aGvHD) (Press release, MaaT Pharma, MAR 11, 2025, View Source [SID1234651069]).

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"We are very pleased with the productive dialogue with the EMA Pediatric Committee and the positive PIP opinion. This approval marks a major regulatory milestone towards the submission of our Marketing Authorization dossier with the EMA," said Gianfranco Pittari, MD, PhD, Chief Medical Officer at MaaT Pharma. "Through our Early Access Program, we have already successfully and safely treated two pediatric patients with aGvHD. We are committed to bringing MaaT013 to pediatric patients suffering from aGvHD, who currently have limited options."

The EMA PDCO approved the clinical program to evaluate the safety and efficacy of MaaT013 in patients from 6 years old to less than 18 years old, with the initiation, in 2026, of a single-arm trial in third-line treatment for 18 patients with aGvHD and in line with the Company’s cash projections.

Based on this positive opinion, MaaT013 would be eligible for up to an additional two years of marketing exclusivity in Europe, on top of the ten-year European market exclusivity as an orphan drug if the Marketing Authorization is granted by the EMA. This also confirms the Company’s ability to reach the full patient population.

"With this approval of our Pediatric Investigation Plan, we are now on track to submit our Marketing Authorization dossier in June this year. If approved, the Company could be positioned to generate revenues as soon as late 2026 with MaaT013 in third-line treatment in aGvHD," stated Hervé Affagard co-founder and CEO of MaaT Pharma. "Additionally, the Company will continue to provide the product through its Early Access Program for all patients in need."

About the Pediatric Committee (PDCO)
The Pediatric Committee (PDCO) is the European Medicines Agency’s (EMA) scientific committee responsible for activities on medicines for children and to support the development of such medicines in the European Union by providing scientific expertise and defining pediatric needs. The PDCO issues an opinion on PIP as part of the regulatory process and the EMA adopts a final decision based on the PDCO’s opinion.

About the Pediatric Investigation Plan (PIP)
A pediatric investigation plan (PIP) is a development plan aimed at ensuring that the necessary data are obtained through studies in children, to support the authorization of a medicine for children. As part of the regulatory process for the registration of new medicines in Europe, the EMA requires pharmaceutical companies to provide a PIP detailing their strategy for investigation of the new medicinal product in the pediatric population. An approved PIP is a prerequisite for filing a Marketing Authorization Application (MAA).

About acute Graft-versus-Host Disease

Acute Graft-versus-Host Disease occurs in patients within 100 days of undergoing a stem cell or bone marrow transplant, where the transplanted cells initiate an immune response and attack the transplant recipient’s organs, causing inflammation of the skin, liver and/or gastro-intestinal tract and leading to significant morbidity and mortality. GI involvement is associated with severe complications such as profound diarrhea, abdominal pain, intestinal bleeding, and death. These complications are often life-threatening, with increased mortality risk, due to the challenges of managing severe GI inflammation and the associated risks of infection, malnutrition, and organ failure. The standard first line therapy for treating aGvHD is the use of systemic steroids. If patients do not respond to steroids, they are considered Steroid Resistant (SR) and other agents can be administered. Currently, the second-line treatment for steroid-refractory acute graft-versus-host disease (SR aGvHD) is ruxolitinib. Recently, remestemcel—L-rknd was approved in December 2024 in the US specifically for use in the paediatric population as a second-line treatment.

About MaaT013

MaaT Pharma’s Microbiome Ecosystem TherapiesTM (MET) are designed to leverage a full microbiome ecosystem to restore balance and maximize clinical benefits for patients with severe, treatment-induced dysbiosis in acute diseases. MaaT013 is a full-ecosystem, off-the-shelf, standardized, pooled-donor, enema Microbiome Ecosystem TherapyTM for acute, hospital use. It is characterized by a consistently high diversity and richness of microbial species and the presence of ButycoreTM (a group of bacterial species known to produce anti-inflammatory metabolites). MaaT013 aims to restore the symbiotic relationship between the patient’s functional gut microbiome and their immune system to correct the responsiveness and tolerance of immune functions and thus reduce steroid-resistant, gastrointestinal (GI)-aGvHD. MaaT013 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

On March 11, 2025 Lyell Immunopharma, Inc. (Nasdaq: LYEL), a clinical-stage company advancing a pipeline of next-generation CAR T-cell therapies for patients with cancer, reported financial results and business highlights for the fourth quarter and year ended December 31, 2024 (Press release, Lyell Immunopharma, MAR 11, 2025, View Source [SID1234651068]).

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"Last year was transformative for Lyell and now, based on promising emerging clinical data, we are poised to initiate pivotal development of IMPT-314, our next-generation dual-targeting CD19‌/‌CD20 CAR T-cell product candidate for patients with aggressive large B‑cell lymphoma," said Lynn Seely, M.D., Lyell’s President and CEO. "We believe IMPT-314 has the potential to deliver improved outcomes for patients by increasing complete response rates and prolonging the duration of response over approved CD19 CAR T-cell therapies, and this year we expect to share more mature data from the ongoing Phase 1/2 trial of IMPT-314. We also plan to initiate two pivotal programs for IMPT-314: one for patients in the 3rd line and later setting by the middle of this year and a second program for patients in the 2nd line setting by early 2026. In addition, we expect to submit a new IND in 2026 for a next-generation solid tumor CAR T-cell product candidate with a new target that is fully-armed with a suite of technologies, including our proprietary clinically-validated anti-exhaustion technology. Our strong cash position enables us to advance our pipeline through important clinical milestones and fund operations into 2027."

Fourth Quarter Updates and Recent Business Highlights

Lyell is advancing a pipeline of next-generation CAR T-cell product candidates. Its lead program, IMPT-314, is in Phase 1/2 clinical development for relapsed or refractory aggressive large B-cell lymphoma (LBCL) and its preclinical programs target solid tumor indications. Lyell’s programs target cancers with large unmet need with substantial patient populations.

IMPT-314: A next-generation dual-targeting CD19/CD20 CAR T-cell product candidate designed to increase complete response rates and prolong the duration of response as compared to the approved CD19‑targeted CAR T-cell therapies for the treatment of LBCL

IMPT-314 is an autologous CAR T-cell product candidate with a true ‘OR’ logic gate to target B cells that express either CD19 or CD20 with full potency and is manufactured with a process that enriches for CD62L+ cells to generate more naïve and central memory CAR T cells with enhanced stemlike features and antitumor activity. The ongoing Phase 1/2 clinical trial is a multi-center, open-label study designed to evaluate the tolerability and clinical benefit of IMPT-314 in patients with relapsed/refractory LBCL and determine a recommended Phase 2 dose. IMPT-314 has received Fast Track Designation from the U.S. Food and Drug Administration for the treatment of relapsed/refractory aggressive B-cell lymphoma in the 3rd line and later (3rd line+) setting.

A Phase 1/2 clinical trial is ongoing and currently enrolling patients in the 3rd line+ and 2nd line settings who have not previously received CAR T-cell therapy.
Initial data from the Phase 1/2 trial was presented at the American Society for Hematology 2024 Annual Meeting on December 9, 2024. Data from 23 patients with relapsed or refractory, CAR T-naive LBCL who received IMPT-314 were reported. The efficacy evaluable population consisted of 17 patients. The overall response rate was 94% (16/17 patients), with 71% (12/17 patients) achieving a complete response by three months. The median follow up was 6.3 months (range 1.2 – 12.5 months) and 71% of patients were experiencing a response at last follow-up. In the safety evaluable population of 23 patients, no Grade 3+ CRS was reported. Grade 3 ICANS was reported in 13% (3/23) of patients with a median time to ICANS resolution of 5 days, and rapid improvement to Grade 2 or lower with standard therapy.
More mature data from the ongoing Phase 1/2 trial in the 3rd line+ setting and initial data from patients in the 2nd line setting are expected to be presented in mid-2025.
Pivotal trial in the 3rd line+ setting is expected to be initiated in mid-2025 in patients with relapsed/refractory aggressive LBCL who have not previously received CAR T-cell therapy.
Pivotal trial in the 2nd line setting expected to be initiated by early 2026 in patients with relapsed/refractory aggressive LBCL who have not previously received CAR T-cell therapy.
Preclinical Pipeline, Technologies and Manufacturing Protocols

The first IND for a fully-armed CAR T-cell product candidate with an undisclosed target for solid tumors is expected in 2026. Lyell is advancing next-generation fully-armed CAR T-cell product candidates, meaning they are armed with multiple technologies, each designed to address different barriers to effective cell therapies, including T-cell exhaustion, lack of durable stemness, as well as immune suppression within the hostile tumor microenvironment.
Presented nonclinical and clinical data from cell therapy product candidates incorporating anti‑exhaustion and manufacturing technologies that demonstrated the potential of Lyell’s technologies to improve T‑cell function in solid tumors. These presentations, from multiple scientific conferences throughout the year, can be found on the Lyell website here.
Corporate Updates

Streamlined expenses and expect net cash use in 2025 to be between $175 million – $185 million. The disciplined expense management will be accomplished by focusing clinical development efforts on the pivotal trials of IMPT-314 and research efforts on developing next-generation fully-armed CAR T-cell programs for solid tumors.
Fourth Quarter and Full Year 2024 Financial Results

Lyell reported a net loss of $191.9 million and $343.0 million for the fourth quarter and year ended December 31, 2024, respectively, compared to a net loss of $52.9 million and $234.6 million for the same periods in 2023. Net loss for the fourth quarter and year ended December 31, 2024 included $87.2 million in acquired in-process research and development (IPR&D) expense as part of our acquisition of ImmPACT Bio USA Inc (ImmPACT Bio) and $51.3 million of long‑lived asset impairment expense. Non‑GAAP net loss, which excludes stock-based compensation, non-cash expenses related to the change in the estimated fair value of success payment liabilities, acquired IPR&D expense, long‑lived asset impairment expense and certain non-cash investment gains and charges, was $45.9 million and $159.5 million for the fourth quarter and year ended December 31, 2024, respectively, compared to $43.9 million and $177.4 million for the same periods in 2023.

GAAP and Non-GAAP Operating Expenses

Research and development (R&D) expenses were $48.7 million and $171.6 million for the fourth quarter and year ended December 31, 2024, respectively, compared to $47.0 million and $182.9 million for the same periods in 2023. The increase in fourth quarter 2024 R&D expenses of $1.7 million was primarily due to increased facilities costs. The decrease in annual 2024 R&D expenses of $11.3 million was primarily driven by a $14.0 million decrease in personnel-related expenses mainly due to lower headcount following the Company’s November 2023 reduction in workforce, partially offset by a $3.2 million increase in research activities primarily driven by clinical trial activity. Non‑GAAP R&D expenses, which exclude non-cash stock-based compensation and non-cash expenses related to the change in the estimated fair value of success payment liabilities, for the fourth quarter and year ended December 31, 2024 were $45.4 million and $157.3 million, respectively, compared to $42.9 million and $165.7 million for the same periods in 2023. The $2.5 million increase in fourth quarter 2024 non-GAAP R&D expenses was primarily driven by increased facilities costs. The $8.3 million decrease in annual 2024 non-GAAP R&D expenses was primarily driven by the decrease in personnel-related expenses mainly due to lower headcount following the Company’s November 2023 reduction in workforce.
General and administrative (G&A) expenses were $14.5 million and $52.0 million for the fourth quarter and year ended December 31, 2024, respectively, compared to $13.2 million and $67.0 million for the same periods in 2023. The decrease in annual 2024 G&A expenses of $14.9 million was primarily driven by a decrease in non‑cash stock-based compensation. Non‑GAAP G&A expenses, which exclude non-cash stock‑based compensation, for the fourth quarter and year ended December 31, 2024 were $9.7 million and $33.5 million, respectively, compared to $8.5 million and $38.1 million for the same periods in 2023. The $1.3 million increase in fourth quarter 2024 non-GAAP G&A expenses was primarily driven by acquisition-related personnel expenses. The $4.6 million decrease in annual 2024 non-GAAP G&A expenses was primarily driven by the decrease in personnel-related expenses mainly due to lower headcount following the Company’s November 2023 reduction in workforce.
Operating expenses for the fourth quarter and year ended December 31, 2024, include $87.2 million of acquired IPR&D expenses recognized as a part of the acquisition of ImmPACT Bio. Additionally, operating expenses for the fourth quarter and year ended December 31, 2024, include an impairment charge of $51.3 million for long-lived assets, resulting from the continued decline in our stock price and related market capitalization.
A discussion of non-GAAP financial measures, including reconciliations of the most comparable GAAP measures to non‑GAAP financial measures, is presented below under "Non-GAAP Financial Measures."

Cash, cash equivalents and marketable securities

Cash, cash equivalents and marketable securities as of December 31, 2024 were $383.5 million compared to $562.7 million as of December 31, 2023. Lyell believes that its cash, cash equivalents and marketable securities balances will be sufficient to meet working capital and capital expenditure needs into 2027.

On March 11, 2025 Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, reported that the European Commission (EC) has granted marketing authorization for RYTELO (imetelstat) as a monotherapy for the treatment of adult patients with transfusion-dependent (TD) anemia due to very low, low or intermediate risk myelodysplastic syndromes (lower-risk MDS or LR-MDS) without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy (ESAs) (Press release, Geron, MAR 11, 2025, View Source [SID1234651067]). Lower-risk MDS is a progressive blood cancer with high unmet need, where many patients with anemia become dependent on red blood cell transfusions, which can be associated with clinical consequences and decreased quality of life.

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"As the first and only treatment of its kind, RYTELO represents an important new option – significantly reducing the need for red blood cell transfusions for people living with LR-MDS who are battling debilitating symptoms like anemia and fatigue," said Joseph Eid, M.D., Geron’s Executive Vice President, Research and Development. "This approval from the European Commission, just nine months following approval in the U.S., underscores the positive benefit for these patients demonstrated in our clinical trials and we look forward to making this innovative therapy accessible to eligible patients in Europe."

"I am thrilled that the European Commission has approved RYTELO in LR-MDS. The long-term and durable responses observed in the Phase 3 IMerge study reinforce the practice-changing potential of telomerase inhibition as a clinically meaningful and differentiated option for the treatment of lower-risk MDS," said Uwe Platzbecker, M.D., Chief Medical Officer at the University Hospital Carl Gustav Carus Dresden in Germany, who was an IMerge investigator and a co-lead author of the Phase 3 results published in The Lancet . "Physicians and patients in Europe are now one step closer to accessing a novel treatment that, in addition to having a generally manageable safety profile, has the potential to provide extended and continuous red blood cell transfusion independence."

The marketing authorization of RYTELO approved by the EC is supported by data from the IMerge Phase 3 clinical trial, which demonstrated the significant clinical benefit of RYTELO in patients with transfusion-dependent anemia due to LR-MDS, reducing the need for red blood cell transfusions in the first 24 weeks of treatment compared to placebo, as observed in the double-blind controlled study. The safety profile of RYTELO was well-characterized with generally manageable and short-lived thrombocytopenia and neutropenia, which are familiar side effects for hematologists who are experienced with managing cytopenias. The most commonly reported adverse reactions ≥ Grade 3 were neutropenia (69%), thrombocytopenia (63%), which lasted a median duration of less than two weeks, and in more than 80% of patients were resolved to Grade < 2 in under four weeks.

RYTELO is the first and only telomerase inhibitor approved by the EC, and the marketing authorization applies to all 27 European Union member states, and Iceland, Norway and Liechtenstein. Geron is preparing to commercialize RYTELO in select EU countries beginning in 2026, pending country-by-country reimbursement. Additionally, Geron is exploring opportunities to make RYTELO available to eligible patients through Expanded Access Programs (EAP), including Named Patient Programs (NPP), which are designed to support access for individual patients on a case-by-case basis.

In connection with the approval, the EMA’s Committee of Orphan Medicinal Products (COMP) reviewed and issued a positive opinion to maintain RYTELO’s orphan drug designation in the EU for MDS, which is expected to provide market exclusivity for ten years after approval, subject to maintaining orphan designation. Patent exclusivity in the EU for LR-MDS is anticipated into 2038, subject to approval of patent term extension by the European Patent Office.

About Lower-Risk Myelodysplastic Syndromes (LR-MDS)

Lower-risk myelodysplastic syndromes (LR-MDS) is a blood cancer that often progresses to require increasingly intensified management of key symptoms such as anemia and resulting fatigue1 . These symptomatic LR-MDS patients frequently become red blood cell transfusion dependent, which has been shown to be associated with short- and long-term clinical consequences that reduce quality of life and shorten survival2,3 . There is a high unmet need for many LR-MDS patients, particularly those with characteristics having poorer prognosis. Current treatment options for those failing ESA are limited to select sub-populations and there is an unmet need for treatments that can provide extended and continuous red blood cell transfusion independence.

About RYTELO (imetelstat)

RYTELO is an oligonucleotide telomerase inhibitor approved in the U.S. 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.

In addition, RYTELO is approved in the European Union as a monotherapy for the treatment of adult patients with transfusion-dependent anemia due to very low, low or intermediate risk myelodysplastic syndromes without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.

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 Geron, RYTELO is the first and only telomerase inhibitor approved by the U.S. Food and Drug Administration and the European Commission.

U.S. IMPORTANT SAFETY INFORMATION

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, available at View Source

The Summary of Product Characteristics (SmPC) for RYTELO in the EU is available at View Source

On March 11, 2025 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that members of its management team will present an overview of the company at the upcoming 37th Annual Roth Conference (Press release, Cellectar Biosciences, MAR 11, 2025, View Source [SID1234651066]). Details are as follows:

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Date: Monday, March 17
Time: 8:30 AM Eastern Time
Webcast: Click HERE

A replay of the corporate presentation will be available on the Events section of the company’s Investor Relations website.