FDA Approves Geron's Rytelo for MDS

Geron Corporation has announced that the U.S. Food and Drug Administration (FDA) has approved Rytelo™ (imetelstat) for the treatment of adult patients with low* to intermediate-1 risk myelodysplastic syndromes (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).

The approval is based on the results from the imerge phase 3 clinical trial, where Rytelo demonstrated significantly higher rates of red blood cell transfusion independence (RBC-TI) versus placebo for at least eight consecutive weeks (Rytelo 39.8%; Placebo 15.0%) and for at least 24 weeks (Rytelo 28.0%; Placebo 3.3%). RBC-TI was durable and sustained in the Rytelo treated population, with a median RBC-TI duration for 8-week responders and 24-week responders of approximately 1 year and 1.5 years, respectively.

In an exploratory analysis of Rytelo-treated patients achieving ≥8-week RBC-TI, median increases in hemoglobin were 3.6 g/dl for Rytelo and 0.8 g/dl for placebo. The most common grade 3/4 adverse reactions were manageable and short-lived thrombocytopenia and neutropenia.

The company's Chairman and CEO, John A. Scarlett, M.D., stated that the approval and availability of Rytelo could potentially bring meaningful clinical benefit to eligible patients with lower-risk MDS, particularly the potential for greater than 24 weeks of freedom from the burden of red blood cell transfusions and symptomatic anemia.

Rytelo is an FDA-approved oligonucleotide telomerase inhibitor indicated to be administered as an intravenous infusion over two hours every four weeks. It is the first and only telomerase inhibitor approved by the FDA and aims to ensure broad access to eligible patients through the reach4rytelo™ patient support program.

The company is also conducting a pivotal phase 3 clinical trial of imetelstat in JAK-inhibitor relapsed/refractory myelofibrosis (R/R MF), as well as studies in other hematologic malignancies.

Geron Corporation is a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer. To learn more, visit www.geron.com or follow the company on LinkedIn. Today the company's shares have moved 18.0% to a price of $4.59. Check out the company's full 8-K submission here.

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