Executives from Geron (NASDAQ:GERN) outlined their commercial priorities for RYTELO (imetelstat) and provided updates on the company’s pipeline and financial outlook during a discussion hosted by Barclays.
Leadership says commercial plan is being refined as RYTELO enters second year
Harout Semerjian, who joined as president and CEO in August, said he was drawn to Geron by what he characterized as “a drug that works,” referring to imetelstat as a new option for patients with lower-risk myelodysplastic syndromes (MDS). Semerjian said the company has been focused on building a “more robust and refined commercialization plan” aimed at reaching more patients.
On expenses, Semerjian pointed to operating expense guidance of $230 million to $240 million for 2026, describing it as a reduction versus prior levels as major trials, including in myelofibrosis (MF), are now fully enrolled.
R&D chief describes MDS patient journey and RYTELO’s positioning
Joseph Eid, executive vice president of R&D and chief medical officer, described MDS as a spectrum of disease in which patients often present with fatigue and declining quality of life, are diagnosed through blood counts showing low hemoglobin, and undergo bone marrow biopsy to confirm disease and mutations.
He said patients typically start with erythropoietin-stimulating agents (ESAs) such as Procrit or Aranesp, and noted that luspatercept has more recently moved into the first-line setting. For patients with specific mutations such as deletion 5q, he said lenalidomide can be an option. Over time, Eid said many patients become increasingly transfusion-dependent and may then move to other therapies, including hypomethylating agents (HMAs), which he described as a mainstay after initial treatments stop working.
Eid said RYTELO was approved “in the first line for ESA-ineligible or unlikely to respond” patients—such as those with high erythropoietin levels (500 and beyond) or high transfusion burden—as well as in second line and beyond.
He differentiated imetelstat from therapies focused on raising hemoglobin, arguing that RYTELO “works on the disease, not on the symptom,” by targeting the clone driving MDS. Eid said this mechanism can allow normal blood formation to recover. He also referenced a “landmark analysis” beyond 40 months in which overall survival, progression-free survival, and conversion to leukemia were “all trending in favor of” the RYTELO arm.
Eid emphasized that despite the “low-risk” label, MDS should be viewed as cancer, citing quality-of-life impairment, complications from transfusion dependence (including iron overload and transfusion allergies), and shortened survival.
Early commercial metrics: demand growth, new accounts, and line-of-therapy mix
In discussing uptake, management said it has been sharing three key performance indicators with the market:
- Demand growth: The company reported 9% growth in the most recent quarter discussed (Q4 2025).
- New purchasing accounts: Management said the number of accounts that have bought RYTELO since launch is about 1,300, including 150 new accounts added recently.
- Line-of-therapy mix: Management said about 30% of business has been in first line and second line combined, based on a 12-month look-back as of the last quarter reported.
Semerjian said Geron is focusing on a clearer second-line opportunity as luspatercept moves earlier and HMAs shift later. He added that the company is simplifying messaging to support community adoption, noting that about 80% of patients are seen in the community setting rather than at academic centers.
Physician education focus includes managing cytopenias
Eid said that as a first-in-class telomerase inhibitor, RYTELO initially had limited hands-on experience among U.S. physicians, given that clinical development largely enrolled patients outside the U.S. He said one area the company has worked to address is physician understanding of treatment-related cytopenias.
According to Eid, the company has educated physicians that cytopenias are “on target,” predictable, resolvable, and manageable, and he said data presented at ASH linked cytopenia with response—describing it as akin to a “clinical biomarker.” Eid added that long-term follow-up has helped provide confidence in safety and benefit over time.
Ex-U.S. optionality and MF catalyst later this year
Semerjian said Geron holds worldwide rights to RYTELO and reported the company has approximately $400 million in cash, which he said provides flexibility as it evaluates international commercial plans. He noted the company already has European Medicines Agency (EMA) approval and is currently engaging with health technology assessment (HTA) processes across Europe to support reimbursement discussions, whether Geron ultimately commercializes directly or pursues partnering options.
On the pipeline, Eid reviewed the company’s Phase 3 IMPACT MF trial of imetelstat in relapsed/refractory myelofibrosis. He said the study uses a 2:1 randomization of imetelstat versus best available therapy in patients previously treated with JAK inhibitors who failed or were intolerant. Eid said the primary endpoint is overall survival, with symptom relief as a secondary endpoint, and that an interim analysis is expected in the second half of the year, triggered by death events.
Management said the decision to use overall survival as the primary endpoint was supported by Phase 2 results that showed improved survival at a higher dose given every three weeks, as well as confirmation using a historical matched cohort. Eid also described MF biology and said prior studies showed fibrosis resolution, which he said is not seen with JAK inhibitors, along with improvements that can translate into symptom relief over time.
In closing comments, Semerjian pointed to the 2025 sales baseline, the 2026 growth guidance, the company’s cash position, and the fully enrolled MF trial as key elements of the company’s opportunity, while emphasizing that execution on commercial performance remains the immediate priority.
About Geron (NASDAQ:GERN)
Geron Corporation (NASDAQ: GERN) is a clinical-stage biotechnology company dedicated to developing and commercializing novel treatments that target telomerase, an enzyme critical to cancer cell immortality. The company’s research is focused on hematologic malignancies and solid tumors, with a pipeline designed to address diseases that have historically had limited therapeutic options.
The lead product candidate, imetelstat, is a first-in-class telomerase inhibitor currently in Phase II and Phase III clinical trials for myelofibrosis and myelodysplastic syndromes.
