Geron’s RYTELO gains EC approval for certain blood cancers

Published 11/03/2025, 21:38
Geron’s RYTELO gains EC approval for certain blood cancers

FOSTER CITY, Calif. - Geron Corporation (NASDAQ:GERN), a biopharmaceutical company focused on blood cancer treatments with a market capitalization of $1 billion, announced today that the European Commission (EC) has authorized its drug RYTELO® (imetelstat) for specific cases of myelodysplastic syndromes (MDS). According to InvestingPro analysis, the company currently appears undervalued, with analysts setting price targets ranging from $2 to $7. The approval covers adult patients with transfusion-dependent anemia who do not have the deletion 5q cytogenetic abnormality and are unresponsive or ineligible for erythropoiesis-stimulating agents (ESAs).

This EC endorsement follows RYTELO’s U.S. approval and makes it the sole telomerase inhibitor sanctioned for use in both markets. Telomerase inhibitors target the enzyme telomerase, which cancer cells often use to maintain their uncontrolled growth. By inhibiting this enzyme, RYTELO aims to reduce the proliferation and survival of malignant cells.

The approval is supported by the IMerge Phase 3 clinical trial results, which showed RYTELO’s efficacy in reducing the necessity for red blood cell transfusions compared to a placebo during the first 24 weeks of treatment. This development comes as InvestingPro data shows the company maintains a strong liquidity position with a current ratio of 5.56, though it faces near-term profitability challenges. The safety profile was considered manageable, with the most common adverse reactions being neutropenia and thrombocytopenia.

Geron plans to commercialize RYTELO in select EU countries starting in 2026, subject to individual reimbursement decisions. The company is also exploring Expanded Access Programs to make the drug available to patients on a case-by-case basis before commercial release.

The EC’s marketing authorization extends to all 27 EU member states, as well as Iceland, Norway, and Liechtenstein. The drug’s orphan designation in the EU may provide ten years of market exclusivity post-approval, with patent exclusivity expected until 2038 upon approval of a patent term extension.

LR-MDS often progresses to a point where patients require frequent red blood cell transfusions, negatively impacting their quality of life. RYTELO’s approval offers a new treatment option for this patient group, particularly for those with a poor prognosis and limited treatment alternatives.

This article is based on a press release statement from Geron Corporation. Investors can access comprehensive analysis and 12 additional ProTips for Geron through InvestingPro, including detailed insights on the company’s financial health, growth prospects, and market positioning. The next earnings report is expected on May 12, 2025, which could be a significant catalyst for the stock.

In other recent news, Geron Corporation reported $47.5 million in net product revenue for the fourth quarter of 2024, slightly exceeding internal estimates. Despite this, analysts have expressed concerns over the flattening sales of its drug Rytelo, which is primarily used for third-line therapy in lower-risk myelodysplastic syndromes. Stifel analysts lowered their price target for Geron to $4, citing a tempered outlook, but maintained a Buy rating. Similarly, Scotiabank also reduced its price target to $4 while keeping an Outperform rating, indicating a positive outlook despite the slowdown in Rytelo’s market momentum.

On the other hand, H.C. Wainwright downgraded Geron from a Buy to a Neutral rating due to stagnant revenue trends and revised its revenue projections, lowering the 2025 estimate to $212 million. TD Cowen also adjusted its price target from $10 to $5 but retained a Buy rating, highlighting the need for increased adoption of Rytelo in earlier treatment stages. The company is focusing on strategic engagement with healthcare professionals to counteract the flattening sales trend and expand Rytelo’s market position. Meanwhile, Geron announced a leadership transition with Dawn Carter Bir stepping in as Interim President and CEO, following Dr. John Scarlett’s departure. The company continues to explore the potential of Rytelo for additional indications, including its ongoing Phase 3 trial for myelofibrosis.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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