Osaka, Japan, March 12, 2025 - Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; “ONO”) announced that it has entered into a license agreement with Ionis Pharmaceuticals, Inc. (Carlsbad, CA,U.S.; CEO, Brett P. Monia, Ph.D.; “Ionis”) for sapablursen, an investigational RNA-targeted medicine for the treatment of polycythemia vera (PV).

日本大阪，2025年3月12日 — 小野药品工业株式会社（总部：日本大阪；总裁兼首席运营官：武野藤一；“ONO”）宣布已与Ionis Pharmaceuticals, Inc.（美国加利福尼亚州卡尔斯巴德；首席执行官：布雷特·P·莫尼亞博士；“Ionis”）就sapablursen达成许可协议。Sapablursen是一种用于治疗真性红细胞增多症（PV）的在研RNA靶向药物。

Sapablursen is currently being evaluated in adult patients with PV in the fully enrolled Phase 2 IMPRSSION study. Sapablursen was granted Fast Track designation in January 2024 and orphan drug designation in August 2024 by the U.S. Food and Drug Administration (FDA)..

Sapablursen目前正在完全招募的2期IMPRSSION研究中对真性红细胞增多症（PV）成年患者进行评估。Sapablursen于2024年1月获得美国食品药品监督管理局（FDA）的快速通道资格，并于2024年8月获得孤儿药资格。

Under the agreement, ONO will obtain an exclusive license to develop and commercialize sapablursen worldwide. Ionis will remain responsible for the completion of the ongoing Phase 2 IMPRSSION study, while ONO will be solely responsible for subsequent development, regulatory filings and commercialization..

根据协议，ONO将获得在全球范围内开发和商业化sapablursen的独家许可。Ionis将继续负责完成正在进行的2期IMPRSSION研究，而ONO将全权负责后续的开发、监管申报和商业化工作。

ONO will make an upfront payment of 280 million US dollars, with up to a maximum of 660 million US dollars in additional payments based on the achievement of development, regulatory and sales milestones. ONO will also pay to Ionis royalties in the mid-teens on annual net sales of sapablursen. The transaction is subject to the requirements of the Hart-Scott-Rodino Antitrust Improvements Act of 1976 (HSR Act)..

ONO将支付2.8亿美元的预付款，并根据开发、监管和销售里程碑的达成情况，额外支付最高可达6.6亿美元的款项。此外，ONO还将按sapablursen年度净销售额的十几%向Ionis支付特许权使用费。本次交易需符合《1976年哈特-斯科特-罗迪诺反垄断改进法》（HSR法案）的要求。

“We are delighted to enter into this collaboration with Ionis Pharmaceuticals, a pioneer in RNA-targeted medicines, for sapablursen. This partnership aligns with our strategy to strengthen our pipeline in hematology. We expect sapablursen to become a new treatment option for PV patients worldwide,” said Toichi Takino, President and COO of ONO..

“我们很高兴与RNA靶向药物的先驱Ionis Pharmaceuticals就Sapablursen展开合作。这一合作伙伴关系符合我们加强血液学领域产品线的战略。我们预计Sapablursen将成为全球PV患者的一种新治疗选择，”ONO总裁兼首席运营官Toichi Takino表示。

“Ionis and ONO share a commitment to advancing innovative medicines to help meet the needs of people living with serious diseases. We are pleased to entrust sapablursen to ONO, whose unique capabilities will help maximize its value by ensuring broad access for people living with PV,” said Brett P. Monia, Ph.D., CEO of Ionis..

“Ionis和ONO都致力于推进创新药物，以帮助满足患有严重疾病的人们的需求。我们很高兴将sapablursen托付给ONO，其独特的能力将通过确保PV患者广泛获得该药物，从而最大化其价值。”Ionis首席执行官Brett P. Monia博士表示。

The impact of this matter on the Company’s consolidated financial results for the current fiscal year ending March 31, 2025, is expected to be minor. Should there be any events to be disclosed in the future, we will announce them in a timely manner.

该事项对本公司截至2025年3月31日的当前财政年度合并财务结果的影响预计较小。若未来有任何需披露的事件，我们将及时公布。

About Sapablursen

关于Sapablursen

Sapablursen is designed to reduce the production of TMPRSS6 resulting in increased expression of hepcidin, which is the key regulator of iron homeostasis. By increasing production of hepcidin, sapablursen has the potential to positively impact blood diseases such as PV.

Sapablursen旨在减少TMPRSS6的产生，从而增加铁调素（hepcidin）的表达，而铁调素是铁稳态的关键调节因子。通过增加铁调素的产生，Sapablursen有可能对诸如PV等血液疾病产生积极影响。

About Polycythemia Vera

关于真性红细胞增多症

Polycythemia Vera (PV) is a rare and potentially life-threatening hematologic disease characterized by the overproduction of red blood cells, which significantly increases the risk of serious blood clots, especially in critical organs like the lungs, heart and brain. Patients with PV also experience severe iron deficiency and commonly have symptoms of fatigue, which can lead to reduce quality of life (QOL)..

真性红细胞增多症 (PV) 是一种罕见且可能危及生命的血液疾病，其特征是红细胞过度生成，这会显著增加严重血栓的风险，尤其是在肺、心脏和大脑等关键器官中。PV 患者还经历严重的铁缺乏，并常出现疲劳症状，这可能导致生活质量 (QOL) 下降。

About Ionis Pharmaceuticals Inc.

关于Ionis制药公司

For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has six marketed medicines and a leading pipeline in neurology, cardiology, and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing.

三十年来，Ionis 发明了为严重疾病患者带来更好未来的药物。Ionis 目前拥有六种已上市的药物，并在神经病学、心脏病学以及一些高需求患者领域拥有领先的管线。作为 RNA 靶向药物的先驱，Ionis 除了推进基因编辑的新方法外，还继续推动 RNA 疗法的创新。

A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. For further information, visit .

对疾病生物学的深刻理解和行业领先的技术推动了我们的工作，同时我们充满热情并迫切希望能够为患者带来改变生命的进步。欲了解更多信息，请访问。