FOSTER CITY, Calif.--(BUSINESS WIRE)--Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, today 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 (ESA)..

加利福尼亚州福斯特城（商业新闻短讯）--Geron Corporation（Nasdaq:GERN）是一家商业阶段的生物制药公司，旨在通过改变血癌的病程来改变生活，今天宣布，美国食品和药物管理局（FDA）已批准RYTELO™（imetelstat）用于治疗患有输血依赖性（TD）贫血的低至中1风险骨髓增生异常综合征（MDS）的成年患者，这些患者在八周内需要四个或更多的红细胞单位，但对红细胞生成刺激剂（ESA）无反应或失去反应或不合格。。

“With the approval and availability of RYTELO, we believe eligible patients with lower-risk MDS can potentially experience meaningful clinical benefit, particularly the potential for greater than 24 weeks of freedom from the burden of red blood cell transfusions and symptomatic anemia,” said John A.

“随着RYTELO的批准和可用性，我们相信符合条件的低风险MDS患者可能会获得有意义的临床益处，特别是可能超过24周免受红细胞输血和症状性贫血的负担，”John A。

Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. “The approval of RYTELO as the first telomerase inhibitor is a testament to the power of our science and the passion of our people to innovate in the field of blood cancer. As we celebrate today’s momentous milestone, I would like to thank the patients and families, advocates, clinicians, study coordinators and site personnel, scientists, and Geron employees and collaborators past and present whose participation was integral to this achievement and to supporting our transformation into a commercial company.”.

医学博士斯嘉丽是Geron的董事长兼首席执行官。“RYTELO被批准为第一种端粒酶抑制剂，这证明了我们科学的力量和我们人民在血癌领域创新的热情。在我们庆祝今天这一重要里程碑的时候，我要感谢患者和家属、倡导者、临床医生、研究协调员和现场人员、科学家以及Geron员工和合作者，他们的参与是这一成就的组成部分，也是支持我们向商业公司转型的关键。”。

Lower-risk myelodysplastic syndromes (LR-MDS) is a blood cancer that often progresses to require increasingly intensified management of key symptoms such as anemia and resulting fatigue1. These symptomatic LR-MDS patients frequently become red blood cell transfusion dependent, which has been shown to be associated with short- and long-term clinical consequences that reduce quality of life and shorten survival2,3.

低风险骨髓增生异常综合征（LR-MDS）是一种血癌，通常会发展为需要越来越多地加强对贫血和疲劳等关键症状的管理1。这些有症状的LR-MDS患者经常成为红细胞输注依赖性患者，这已被证明与降低生活质量和缩短生存期的短期和长期临床后果有关2,3。

There is a high unmet need for many LR-MDS patients, particularly those with characteristics having poorer prognosis. Current treatment options for those failing ESA are limited to select sub-populations and there is an unmet need for treatments that can provide extended and continuous red blood cell transfusion independence..

对于许多LR-MDS患者，特别是那些预后较差的患者，需求很高。目前针对ESA失败患者的治疗选择仅限于选择亚人群，并且对可以提供延长和持续的红细胞输血独立性的治疗需求尚未得到满足。。

Approval Based on Results from IMerge Phase 3 Clinical Trial

根据IMerge 3期临床试验的结果进行批准

“For patients with lower-risk MDS and anemia who are transfusion dependent, we have very few options today and often cycle through available therapies, making the approval of RYTELO potentially practice changing for us,” said Rami Komrokji, MD, Vice Chair, Malignant Hematology Department, Moffitt Cancer Center, who was an investigator of the pivotal IMerge clinical trial.

Moffitt癌症中心恶性血液科副主席Rami Komrokji医学博士说：“对于输血依赖的低风险MDS和贫血患者，我们现在几乎没有选择，经常循环使用可用的疗法，这使得RYTELO的批准可能会改变我们的做法。”。

“What is exciting about RYTELO is the totality of the clinical benefit across LR-MDS patients irrespective of ring sideroblast status or high transfusion burden, including sustained and durable transfusion independence and increases in hemoglobin levels, all within a well-characterized safety profile of generally manageable cytopenias.

“RYTELO令人兴奋的是，LR-MDS患者的总体临床获益，无论环铁粒幼细胞状态或高输血负担如何，包括持续和持久的输血独立性以及血红蛋白水平的升高，所有这些都在一般可控制的血细胞减少症的特征明确的安全性范围内。

The treatment goal for patients with LR-MDS and anemia is transfusion-independence and before today, this wasn’t possible for many patients.”.

LR-MDS和贫血患者的治疗目标是输血独立，在今天之前，这对许多患者来说是不可能的。”。

The FDA approval of RYTELO is based on results from the IMerge Phase 3 clinical trial, published in The Lancet4. The IMerge trial met its primary and key secondary endpoints, with RYTELO demonstrating significantly higher rates of red blood cell transfusion independence (RBC-TI) versus placebo for at least eight consecutive weeks (RYTELO 39.8% [95% CI 30.9–49.3]; placebo 15.0% [7.1–26.6]; p<0.001) and for at least 24 weeks (RYTELO 28.0% [95% CI 20.1-37.0]; placebo 3.3% [95% CI 0.4-11.5]; p<0.001).

FDA对RYTELO的批准是基于发表在Lancet4上的IMerge 3期临床试验的结果。IMerge试验达到了主要和关键的次要终点，RYTELO连续至少8周显示红细胞输血独立性（RBC-TI）明显高于安慰剂（RYTELO 39.8%[95%CI 30.9-49.3]；安慰剂15.0%[7.1-26.6]；p＜0.001）和至少24周（RYTELO 28.0%[95%CI 20.1-37.0]；安慰剂3.3%[95%CI 0.4-11.5]；p＜0.001）。

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..

RBC-TI在接受RYTELO治疗的人群中持久且持续，8周应答者和24周应答者的中位RBC-TI持续时间分别约为1年和1.5年。。

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. Clinically meaningful efficacy results were observed across key MDS subgroups irrespective of ring sideroblast (RS) status, baseline transfusion burden and IPSS risk category..

在对RYTELO治疗的患者达到≥8周RBC-TI的探索性分析中，RYTELO的血红蛋白中位数增加为3.6 g/dL，安慰剂为0.8 g/dL。无论环铁粒幼细胞（RS）状态，基线输血负担和IPSS风险类别如何，在关键MDS亚组中观察到临床上有意义的疗效结果。。

In the IMerge trial, the safety profile of RYTELO was well-characterized with generally manageable and short-lived thrombocytopenia and neutropenia, which are familiar side effects for hematologists who are experienced with managing cytopenias. The most common Grade 3/4 adverse reactions were neutropenia (72%) and thrombocytopenia (65%), which lasted a median duration of less than two weeks, and in more than 80% of patients were resolved to Grade < 2 in under four weeks.

在IMerge试验中，RYTELO的安全性得到了很好的表征，其血小板减少症和中性粒细胞减少症通常可以控制且寿命短，这是具有血细胞减少症管理经验的血液学家熟悉的副作用。最常见的3/4级不良反应是中性粒细胞减少症（72%）和血小板减少症（65%），中位持续时间不到两周，超过80%的患者在四周内达到2级以下。

Cytopenias were generally manageable with dose modifications. The intravenous administration of RYTELO every four weeks aligns to routine blood count monitoring for these patients..

血细胞减少症通常可以通过剂量调整来控制。每四周静脉注射RYTELO与这些患者的常规血细胞计数监测相一致。。

The most common adverse reactions (incidence ≥10% with a difference between arms of >5% compared to placebo), including laboratory abnormalities, were decreased platelets (thrombocytopenia), decreased white blood cells, decreased neutrophils (neutropenia), increased aspartate aminotransferase (AST), increased alkaline phosphatase (ALP), increased alanine aminotransferase (ALT), fatigue, prolonged partial thromboplastin time, arthralgia/myalgia, COVID-19 infections, and headache.

包括实验室异常在内，最常见的不良反应（发生率≥10%，与安慰剂组相比差异>5%）是血小板减少（血小板减少），白细胞减少，中性粒细胞减少（中性粒细胞减少），天冬氨酸转氨酶增加（AST），碱性磷酸酶（ALP）增加，丙氨酸转氨酶（ALT）增加，疲劳，部分凝血活酶时间延长，关节痛/肌痛，COVID-19感染和头痛。

Clinically relevant adverse reactions in < 5% of patients who received RYTELO included febrile neutropenia, sepsis, gastrointestinal hemorrhage, and hypertension..

接受RYTELO治疗的患者中，少于5%的临床相关不良反应包括发热性中性粒细胞减少症，败血症，胃肠道出血和高血压。。

Conference Call Details

电话会议详细信息

A conference call with Geron management is scheduled at 8am Eastern Time on Friday, June 7, 2024, to discuss the FDA approval and launch of RYTELO. To access the webcast and slides, please visit the Investors & Media page. Participants may access the webcast by registering online using the following link, https://events.q4inc.com/attendee/923992744..

定于2024年6月7日（星期五）东部时间上午8点与Geron管理层举行电话会议，讨论FDA批准和推出RYTELO的事宜。要访问网络广播和幻灯片，请访问投资者和媒体页面。参与者可以通过以下链接在线注册来访问网络广播，https://events.q4inc.com/attendee/923992744..

About RYTELO™ (imetelstat)

关于RYTELO™（imetelstat）

RYTELO™ (imetelstat) is an FDA-approved oligonucleotide telomerase inhibitor for the treatment of adult patients with low-to-intermediate-1 risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent 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).

RYTELO™（imetelstat）是一种FDA批准的寡核苷酸端粒酶抑制剂，用于治疗患有输血依赖性贫血的低至中1风险骨髓增生异常综合征（LR-MDS）的成年患者，需要四个或更多红细胞单位超过八周，对红细胞生成刺激剂（ESA）没有反应或失去反应或不合格。

It is indicated to be administered as an intravenous infusion over two hours every four weeks..

建议每四周静脉输注两小时以上。。

RYTELO is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Telomeres are protective caps at the end of chromosomes that naturally shorten each time a cell divides. In LR-MDS, abnormal bone marrow cells often express the enzyme telomerase, which rebuilds those telomeres, allowing for uncontrolled cell division.

RYTELO是一种通过抑制端粒酶酶活性起作用的一流治疗方法。端粒是染色体末端的保护帽，每次细胞分裂时都会自然缩短。在LR-MDS中，异常的骨髓细胞通常表达端粒酶，端粒酶可以重建这些端粒，从而使细胞分裂不受控制。

Developed and exclusively owned by Geron, RYTELO is the first and only telomerase inhibitor approved by the U.S. Food and Drug Administration..

RYTELO由Geron开发并独家拥有，是美国食品和药物管理局批准的第一种也是唯一一种端粒酶抑制剂。。

Geron aims to ensure broad access to RYTELO for eligible patients. Accordingly, our REACH4RYTELO™ Patient Support Program provides a range of resources that support access and affordability to eligible patients prescribed RYTELO.

Geron旨在确保符合条件的患者广泛使用RYTELO。因此，我们的REACH4RYTELO™患者支持计划提供了一系列资源，支持符合条件的患者使用RYTELO并负担得起。

About Geron

关于Geron

Geron is a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer. Our first-in-class telomerase inhibitor RYTELO™ (imetelstat) is FDA-approved for the treatment of adult patients with lower-risk MDS with transfusion dependent anemia. We are 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是一家商业化阶段的生物制药公司，旨在通过改变血癌的病程来改变生活。我们的一流端粒酶抑制剂RYTELO™（imetelstat）被FDA批准用于治疗输血依赖性贫血的低风险MDS成年患者。我们还正在进行imetelstat在JAK抑制剂复发/难治性骨髓纤维化（R/R MF）中的关键3期临床试验，以及其他血液系统恶性肿瘤的研究。

Inhibiting telomerase activity, which is increased in malignant stem and progenitor cells in the bone marrow, aims to potentially reduce proliferation and induce death of malignant cells. To learn more, visit www.geron.com or follow us on LinkedIn..

抑制端粒酶活性，其在骨髓中的恶性干细胞和祖细胞中增加，旨在潜在地减少增殖并诱导恶性细胞死亡。要了解更多信息，请访问www.geron.com或在LinkedIn上关注我们。。