Geron Shares Rise on Favorable Rytelo Drug Data at ASH Conference
New analysis suggests a key side effect of the MDS treatment is linked to its clinical effectiveness, bolstering the drug's profile among clinicians.
Shares of Geron Corporation (NASDAQ: GERN) climbed in Monday trading after the biotechnology firm presented new data that positively reframes the side effect profile of its flagship blood cancer drug, Rytelo (imetelstat). The findings, unveiled at the American Society of Hematology (ASH) 2025 Annual Meeting, suggest that a common side effect is directly associated with the drug's clinical benefits.
The stock rose as much as 3.2% to $1.31 a share, reflecting investor optimism about the drug's commercial prospects. The new analysis from the Phase 3 IMerge trial indicates that treatment-emergent cytopenias—a decrease in the number of mature blood cells—are an 'on-target' effect of Rytelo's mechanism of action in patients with lower-risk myelodysplastic syndromes (MDS).
This is a significant development for Geron, as it helps clinicians understand that these manageable side effects are a sign the drug is working as intended. "This new analysis provides physicians with important information to help them interpret treatment patterns and manage their patients," the company stated in a press release detailing the ASH presentation.
Rytelo, a first-in-class telomerase inhibitor, was approved by the U.S. Food and Drug Administration (FDA) in June 2024 for the treatment of adult patients with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks. The drug targets a specific population with lower-risk MDS who have not responded to or are ineligible for standard erythropoiesis-stimulating agents.
The data from the pivotal IMerge trial was a key factor in its approval, showing that nearly 40% of patients treated with Rytelo achieved at least eight weeks of transfusion independence, compared to just 15% on placebo, according to findings published by the National Institutes of Health.
The presentations at the ASH conference, one of the premier events for hematology research, serve to strengthen Rytelo's position in a competitive market. Geron is strategically aiming to establish the drug as a preferred second-line therapy. The company has been executing a "surround sound approach" to boost physician awareness, and the new data provides a compelling clinical argument for its adoption.
Analysts have set a consensus price target of $3.60 for GERN, suggesting significant potential upside from its current trading level. The company, with a market capitalization of approximately $836 million, is focused on the successful commercial launch and uptake of Rytelo. The findings presented at ASH are expected to support this effort by building confidence among hematologists in managing patients treated with the novel therapy.
In addition to the analysis on cytopenias, Geron announced it would present four other posters at the conference, covering long-term survival outcomes and translational biomarker analyses. These presentations aim to build a more comprehensive understanding of Rytelo's biological impact and long-term benefits for patients with myeloid hematologic malignancies.
The company's focused strategy on Rytelo underscores its transition from a clinical-stage to a commercial-stage biopharmaceutical entity. As Geron executives outlined in recent investor conferences, the successful rollout and physician adoption of Rytelo are the company's primary objectives. The positive reception of its latest clinical data provides a tailwind for these efforts.