Geron Announces PDUFA Date for Imetelstat NDA in Lower Risk MDS

FOSTER CITY, Calif.--(BUSINESS WIRE)--Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, today announced that the United States Food and Drug Administration (FDA) has assigned a standard review and a Prescription Drug User Fee Act (PDUFA) action date of June 16, 2024 for Geron’s New Drug Application (NDA) for imetelstat for the treatment of transfusion-dependent anemia in patients with lower risk myelodysplastic syndromes (MDS). In addition, the FDA informed the Company that it is currently planning to hold an advisory committee meeting as part of the NDA review.

“We look forward to working with the FDA to complete the NDA review process on a timely basis,” said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. “With the PDUFA date now set, we continue to expect an imetelstat launch in the United States by the end of the first half of 2024, subject to FDA approval.”

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. 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 lower risk myelodysplastic syndromes (LR MDS), and in 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 FDA has assigned a PDUFA action date of June 16, 2024; (ii) that FDA is currently planning to hold an advisory committee meeting as part of the NDA review; (iii) that the Company looks forward to working with the FDA to complete the NDA review process on a timely basis; (iv) that the Company continues to expect an imetelstat launch in the United States by the end of the first half of 2024, subject to FDA approval; (v) that imetelstat may alter the underlying drivers of disease and has the potential to demonstrate disease-modifying activity in patients; and (vi) 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 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; (c) whether imetelstat actually demonstrates that it alters the underlying drivers of disease and has disease-modifying activity in patients; (d) whether the FDA will hold an advisory committee meeting and the discussions at and outcome of that meeting; and (e) whether the FDA will extend the PDUFA action date or approve imetelstat for the treatment of transfusion-dependent anemia in patients with lower risk MDS. 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 June 30, 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.