Geron Announces U.S. Expanded Access Protocol for Imetelstat in Lower Risk MDS

Rhea-AI Summary

Geron Corporation announces availability of Expanded Access Protocol for imetelstat, a telomerase inhibitor for lower risk myelodysplastic syndromes (MDS). Positive IMerge Phase 3 results submitted to FDA. NDA also submitted.

Positive

• Positive IMerge Phase 3 results submitted to FDA
• Mean hemoglobin levels in imetelstat-treated patients increased significantly over time compared to placebo patients
• Statistically significant and clinically meaningful efficacy results achieved across key MDS subgroups

Negative

• None.

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, today announced that an Expanded Access Protocol (EAP) is available for imetelstat, the Company’s first-in-class telomerase inhibitor. As allowed under the U.S. Food and Drug Administration (FDA) policies, companies may offer an EAP, which permits an investigational medicinal product intended to treat a serious or life-threatening condition, such as lower risk myelodysplastic syndromes (MDS), to be used outside of a clinical trial.¹

“We have heard physicians in both academic and community settings express the need for new treatment options for their lower risk MDS patients, and they will now have an option to access imetelstat through the EAP,” said Faye Feller, M.D., Executive Vice President, Chief Medical Officer of Geron. “With positive IMerge Phase 3 results submitted to the FDA, we now plan to be working with clinicians, patients and the MDS patient advocacy community to provide access to imetelstat for eligible patients through our EAP that was accepted by the FDA.”

The imetelstat EAP is available for patients with transfusion dependent non-del(5q) lower risk MDS who are refractory to, relapsed after, or ineligible for erythropoiesis-stimulating agents (ESAs), and naïve to lenalidomide and hypomethylating agents (HMAs). Transfusion dependent is defined as requiring at least four units of packed red blood cells (RBCs) over any eight-week period during the 16 weeks prior to being considered for the EAP.

The Company recently announced the submission to the FDA of a New Drug Application (NDA) for imetelstat, which is based on results from the IMerge Phase 3 clinical trial. In that trial, 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. Further, statistically significant and clinically meaningful efficacy results were achieved across key MDS subgroups: ring sideroblast (RS) status, baseline transfusion burden and IPSS risk category. Safety results were consistent with prior imetelstat clinical experience.

About the Imetelstat EAP

The imetelstat EAP is a U.S.-only protocol for eligible adult participants diagnosed with lower risk (very low, low, or intermediate risk by IPSS-R) myelodysplastic syndromes (MDS) who are red blood cell (RBC) transfusion dependent, have failed to respond or have lost response or are ineligible for erythropoiesis-stimulating agents (ESAs), had not received prior treatment with either a hypomethylating agent or lenalidomide and are non-del(5q). Additional information is planned to be available on www.clinicaltrials.gov.

U.S.-based healthcare professionals seeking more information about the imetelstat EAP can email GeronEAP@wepclinical.com.

Lower risk MDS patients who are interested in enrolling in the imetelstat EAP should speak with their physician to understand if imetelstat is an appropriate treatment option.

More information is available on Geron’s website under the Patients tab.

Imetelstat is an investigational therapy and is not approved for any indication in any markets.

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.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic malignancies. Data from non-clinical studies and clinical trials of imetelstat provide strong evidence that imetelstat targets telomerase to inhibit the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies resulting in malignant cell apoptosis and potential disease-modifying activity. Imetelstat has been granted Fast Track designation by the U.S. Food and Drug Administration for both the treatment of adult patients with transfusion dependent anemia due to Low or Intermediate-1 risk MDS that is not associated with del(5q) who are refractory or resistant to an erythropoiesis stimulating agent, and for adult patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus associated kinase (JAK) inhibitor treatment. Geron submitted a New Drug Application (NDA) in the U.S. in June 2023 and expects to submit a Marketing Authorization Application (MAA) in the EU in the second half of 2023 in the lower risk MDS indication. Imetelstat is currently not approved by any regulatory authority.

About Geron

Geron is a late-stage biopharmaceutical company pursuing therapies with the potential to extend and enrich the lives of patients living with hematologic malignancies. Our first-in-class telomerase inhibitor, imetelstat, harnesses Nobel Prize-winning science in a treatment that may alter the underlying drivers of disease. Geron currently has two Phase 3 pivotal clinical trials underway evaluating imetelstat in: (i) lower risk myelodysplastic syndromes (LR MDS), and (ii) relapsed/refractory myelofibrosis (MF). To learn more, visit www.geron.com or follow us on LinkedIn.

Use of Forward-Looking Statements

Except for the historical information contained herein, this press release contains forward-looking statements made pursuant to the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such statements, include, without limitation, those regarding: (i) that Geron plans to be working with clinicians, patients and the MDS patient advocacy community to provide access to imetelstat for eligible patients through the FDA-reviewed EAP; (ii) that imetelstat targets telomerase to inhibit the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies resulting in malignant cell apoptosis and potential disease-modifying activity; (iii) that Geron expects to submit a Marketing Authorization Application in the EU in the second half of 2023; and (iv) other statements that are not historical facts, constitute forward-looking statements. These forward-looking statements involve risks and uncertainties that can cause actual results to differ materially from those in such forward-looking statements. These risks and uncertainties, include, without limitation, risks and uncertainties related to: (a) whether regulatory authorities permit the continuation of the EAP or further development of imetelstat on a timely basis, or at all, without any clinical holds; (b) whether any future safety or efficacy results cause the benefit-risk profile of imetelstat to become unacceptable; and (c) whether imetelstat actually demonstrates that it has disease-modifying activity in patients. Additional information on the above risks and uncertainties and additional risks, uncertainties and factors that could cause actual results to differ materially from those in the forward-looking statements are contained in Geron’s filings and periodic reports filed with the Securities and Exchange Commission under the heading “Risk Factors” and elsewhere in such filings and reports, including Geron’s quarterly report on Form 10-Q for the quarter ended March 31, 2023 and future filings and reports by Geron. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made, and the facts and assumptions underlying the forward-looking statements may change. Except as required by law, Geron disclaims any obligation to update these forward-looking statements to reflect future information, events or circumstances.

1. FDA Expanded Access. https://www.fda.gov/news-events/public-health-focus/expanded-access.

 

View source version on businesswire.com: https://www.businesswire.com/news/home/20230629356623/en/

Aron Feingold

Investor and Media Relations

investor@geron.com

media@geron.com

Source: Geron Corporation

FAQ

What is the Expanded Access Protocol for imetelstat?

The Expanded Access Protocol allows for the use of imetelstat, a telomerase inhibitor, outside of a clinical trial for lower risk MDS patients.

What were the results of the IMerge Phase 3 clinical trial?

The primary endpoint of 8-week transfusion independence was significantly higher with imetelstat compared to placebo. Mean hemoglobin levels in imetelstat-treated patients increased significantly over time. Statistically significant and clinically meaningful efficacy results were achieved across key MDS subgroups.

What is the status of the New Drug Application (NDA) for imetelstat?

A NDA for imetelstat has been submitted to the FDA.