Geron Announces FDA Acceptance of New Drug Application for Imetelstat for the Treatment of Lower Risk MDS

Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, today announced that the United States Food and Drug Administration (FDA) has accepted the filing of Geron’s New Drug Application (NDA) for imetelstat, its first-in-class telomerase inhibitor, for the treatment of transfusion-dependent anemia in patients with lower risk myelodysplastic syndromes (MDS).

“The FDA’s acceptance of our New Drug Application is an important landmark along our steadfast journey to bring telomerase inhibition with imetelstat to the market,” said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. “We look forward to continuing our collaboration with the FDA toward the goal of bringing imetelstat to the many patients for whom we believe this treatment could make a significant difference.”

“FDA acceptance of our NDA is a significant milestone for both Geron and the MDS community, as there remain few treatment options and significant unmet needs, particularly for patients with difficult-to-treat subtypes of this cancer,” said Faye Feller, M.D., Executive Vice President, Geron’s Chief Medical Officer. “We believe that the IMerge Phase 3 data reflect the truly unique attributes of imetelstat, and, if approved, we expect imetelstat will change the standard of care in lower risk MDS.”

The NDA submission is based on results from IMerge Phase 3, in which the primary endpoint of 8-week transfusion independence (TI) was significantly higher with imetelstat vs. placebo (p<0.001), with median TI duration approaching one year for imetelstat 8-week TI responders. Mean hemoglobin levels in imetelstat-treated patients increased significantly (p<0.001) over time compared to placebo patients. Statistically significant and clinically meaningful efficacy results were achieved across key MDS subgroups irrespective of ring sideroblast (RS) status, baseline transfusion burden and IPSS risk category. Patient-reported outcomes (PRO) data reported a sustained meaningful improvement in fatigue for imetelstat-treated patients vs. placebo. Safety results were consistent with prior imetelstat clinical experience.

As allowed under the 21^st Century Cures Act, the FDA has up to 74 days from the NDA submission date to notify Geron of the Prescription Drug User Fee Act (PDUFA) action date for the NDA. Upon receipt of this notification, Geron plans to disclose the timeline for the NDA review.

Additionally, Geron expects to submit a Marketing Authorization Application (MAA) in the European Union (EU) in the fourth quarter of 2023.

About IMerge Phase 3

The Phase 3 portion of the IMerge Phase 2/3 study is a double-blind, 2:1 randomized, placebo-controlled clinical trial to evaluate imetelstat in patients with IPSS Low or Intermediate-1 risk (lower risk) transfusion dependent MDS who were relapsed after, refractory to, or ineligible for, erythropoiesis stimulating agent (ESA) treatment, had not received prior treatment with either a HMA or lenalidomide and were non-del(5q). To be eligible for IMerge Phase 3, patients were required to be transfusion dependent, defined as requiring at least four units of packed red blood cells (RBCs), over an eight-week period during the 16 weeks prior to entry into the trial. The primary efficacy endpoint of IMerge Phase 3 is the rate of red blood cell transfusion independence (RBC-TI) lasting at least eight weeks, defined as the proportion of patients without any RBC transfusion for at least eight consecutive weeks since entry to the trial (8-week TI). Key secondary endpoints include the rate of RBC-TI lasting at least 24 weeks (24-week TI), the duration of TI and the rate of hematologic improvement erythroid (HI-E), which is defined under 2006 IWG criteria as a rise in hemoglobin of at least 1.5 g/dL above the pretreatment level for at least eight weeks or a reduction of at least four units of RBC transfusions over eight weeks compared with the prior RBC transfusion burden. A total of 178 patients were enrolled in IMerge Phase 3 across North America, Europe, Middle East and Asia.