Exciting Milestone: FDA Greenlights Geron’s New Drug Application for Imetelstat, Marking a Leap Forward in Treating Lower Risk MDS

Turning the Tide: Geron’s Imetelstat Breakthrough Clears FDA Hurdle, Paving the Way for Innovative Treatment of Myelodysplastic Syndromes

FOSTER CITY, Calif.–(BUSINESS WIRE)– The stage is set for a medical revolution as Geron Corporation (Nasdaq: GERN), a trailblazing clinical biopharmaceutical company, proudly reveals the United States Food and Drug Administration’s (FDA) nod of acceptance for Geron’s New Drug Application (NDA) for the groundbreaking imetelstat. This first-of-its-kind telomerase inhibitor brings renewed hope for patients battling transfusion-dependent anemia within the realm of lower risk myelodysplastic syndromes (MDS).

With determination echoing through the corridors, Dr. John A. Scarlett, Geron’s Chairman and Chief Executive Officer, lauded the FDA’s embrace of their NDA as a pivotal marker on their resolute journey to deliver the transformative power of telomerase inhibition via imetelstat to the market. “We eagerly anticipate the ongoing partnership with the FDA, uniting our efforts to extend imetelstat’s reach to the countless patients who stand to gain a profound advantage from this treatment,” stated Dr. Scarlett.

Dr. Faye Feller, the Executive Vice President and Chief Medical Officer at Geron, emphasized the profound impact of this FDA acceptance on both Geron and the MDS community. As she noted, there is a glaring gap in treatment options, particularly for those with complex, hard-to-treat subtypes of this challenging condition. She envisions imetelstat as a beacon of hope, set to reshape the landscape of care standards for lower risk MDS, ushering in a new era.

The foundation of this momentous leap lies in the IMerge Phase 3 results. The study illuminated the unprecedented power of imetelstat, showcasing a statistically significant increase in 8-week transfusion independence (TI) compared to the placebo (p<0.001). With a median TI duration approaching one year for imetelstat’s 8-week TI responders, hope sparkles on the horizon. The hemoglobin levels of patients treated with imetelstat experienced a noteworthy surge (p<0.001) over time, distinguishing them from their placebo counterparts. Across critical MDS subgroups, irrespective of ring sideroblast (RS) status, baseline transfusion burden, or IPSS risk category, imetelstat delivered outcomes that shimmer with statistical significance and clinical significance alike. Patient-reported outcomes (PRO) unveiled a sustained, impactful alleviation of fatigue for those under imetelstat’s wing. The study’s safety results harmonize with the established imetelstat clinical track record.

The narrative takes an intriguing turn as the 21st Century Cures Act steps in, granting the FDA up to 74 days from the NDA submission date to unveil the Prescription Drug User Fee Act (PDUFA) action date. The moment this notification reaches Geron’s ears, the timeline for the NDA review will be revealed, igniting curiosity about the road ahead.

Eyes are also set on the European Union (EU) as Geron envisions submitting a Marketing Authorization Application (MAA) in the fourth quarter of 2023, furthering the global reach of imetelstat’s potential.

In this unfolding chapter of medical advancement, Geron’s imetelstat stands as a beacon, promising to rewrite the story of Myelodysplastic Syndromes, offering hope, innovation, and a brighter tomorrow.

About IMerge Phase 3

In the captivating Phase 3 segment of the IMerge Phase 2/3 study, an enigmatic journey unfolds. Picture a double-blind, 2:1 randomized, placebo-controlled clinical trial, like a tapestry woven with anticipation and discovery. Here, the spotlight falls on imetelstat—a beacon of hope—for patients with IPSS Low or Intermediate-1 risk (lower risk) transfusion-dependent MDS. These brave souls, relapsed after, refractory to, or ineligible for erythropoiesis-stimulating agent (ESA) treatment, embark on a quest for renewal. They stand untainted by prior encounters with HMAs or lenalidomide, and free from the grasp of del(5q).

Eligibility, a gatekeeper to this odyssey, demands a story of its own. Transfusion dependence, a call echoing for at least four units of packed red blood cells (RBCs) over eight weeks within the 16 weeks prior to entry. The canvas is set for heroes to rise.

Behold, the heart of the narrative—the primary efficacy endpoint. A beacon of light, it shines on the rate of red blood cell transfusion independence (RBC-TI), the pinnacle of triumph. It’s not just about liberation from transfusions but the endurance of eight weeks—a test of resilience, of newfound strength. Secondary characters emerge, each with its own allure—RBC-TI lasting 24 weeks, a test of time; the dance of duration; and the rhythmic rise of hematologic improvement erythroid (HI-E), a crescendo in hemoglobin, a tale of transformation.

A diverse cast of 178 patients grace this stage, drawn from the corners of North America, Europe, Middle East, and Asia. Their stories intermingle, united by a common thread of courage and hope. As the curtain rises on this global symphony of healing, the IMerge Phase 3 saga unfolds—a saga painted in the hues of science, resilience, and the human spirit.

About Imetelstat

Enter the world of Imetelstat, a trailblazing protagonist in the realm of medical marvels. Imagine a treasure held close by the hands of Geron, a whispered secret shared only among those who seek the frontiers of hematologic advancements. This is the story of a pioneer, a first-of-its-kind telomerase inhibitor that dances on the edge of possibility.

The tale is woven from threads of non-clinical studies and clinical trials, each revealing a tapestry of promise. Imetelstat strides forward, a guardian of balance, striking at the heart of unbridled growth in myeloid hematologic malignancies. A symphony of science, it orchestrates the fall of malignant stem and progenitor cells, the swan song of unchecked proliferation. The result? A ballet of apoptosis, a dance of demise, and whispers of transformative potential.

Amidst whispers, Imetelstat takes its place under the spotlight, a Fast Track designation granted by the U.S. Food and Drug Administration. A standing ovation for its dual role—savior to those with transfusion-dependent anemia due to Low or Intermediate-1 risk MDS without del(5q), and a beacon of hope for those navigating the treacherous waters of Intermediate-2 or High-risk MF. A second chance, a lifeline, for those whose journey had been thwarted by resistance.

But hold your breath, for this tale is still being written. Imetelstat, a celestial voyager, is yet to receive the stamp of approval from the guardians of regulation. The story, at its precipice, invites you to witness the birth of a revolution, where science and determination intertwine, forging a path toward a future where disease bends to the will of human ingenuity.

About Geron

Geron stands as a beacon of hope in the world of biopharmaceuticals, dedicated to uncovering therapies that hold the promise of extending and enriching the lives of those battling hematologic malignancies. At the heart of our mission lies imetelstat, a groundbreaking telomerase inhibitor that draws upon the brilliance of Nobel Prize-winning science. With the potential to rewrite the story of these diseases, imetelstat seeks to unravel the very threads of their origin.

Currently, the stage is set for a captivating journey as Geron spearheads two Phase 3 pivotal clinical trials. One explores the transformative power of imetelstat in lower risk myelodysplastic syndromes (LR MDS), while the other delves into its potential for relapsed/refractory myelofibrosis (MF), all in the pursuit of rewriting the future of healthcare.

Use of Forward-Looking Statements

Step into the world of possibilities as we unveil our vision for the future! This press release is a journey through innovation and hope, guided by the forward-looking statements under the protective umbrella of the Private Securities Litigation Reform Act of 1995.

Prepare to be captivated by what lies ahead:

(i) Geron’s exciting plan to submit a MAA in the EU by the fourth quarter of 2023;

(ii) the groundbreaking potential of imetelstat to redefine the course of diseases by addressing their root causes;

(iii) the revolutionary impact that imetelstat could bring to patients, potentially elevating the standard of care for lower risk MDS;

(iv) the anticipation building as Geron prepares to unveil the timeline for NDA review, a secret guarded until the magical moment of the FDA’s PDUFA date notification; and

(v) a tapestry of forward-looking statements that transcend history, igniting a beacon of change in the medical landscape.

Yet, amidst this thrilling voyage lie challenges—risks and uncertainties that dare us to push the boundaries:

(a) the delicate balance of regulatory authorities nurturing imetelstat’s evolution without delay or clinical holds;

(b) the meticulous dance between efficacy and safety results, shaping imetelstat’s destiny in the eyes of benefit-risk analysis;

(c) the unfolding mystery of whether imetelstat truly holds the power to reshape diseases at their core; and

(d) the ultimate question of whether the FDA’s seal of approval will grace imetelstat’s journey towards treating transfusion-dependent anemia in lower risk MDS patients.

As you dive into this immersive experience, remember that these forward-looking statements are the stars guiding us, accompanied by the realization that even stars change their courses. While we can’t predict the future, we can dream it. Step alongside us as we venture into the uncharted territories of tomorrow, ready to adapt, evolve, and shine.

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