PASADENA, Calif.--(

加利福尼亚州帕萨迪纳--(

BUSINESS WIRE

商业热线

)--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the global licensing and collaboration agreement with Sarepta Therapeutics (NASDAQ: SRPT) announced on November 26, 2024, has now closed. Closing of the transaction was subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions..

)--箭头制药公司（NASDAQ:ARWR）今天宣布，2024年11月26日宣布的与Sarepta Therapeutics（NASDAQ:SRPT）的全球许可和合作协议现已结束。根据1976年《哈特·斯科特·罗迪诺反托拉斯改进法案》和其他习惯条件，交易的结束取决于等待期的到期或终止。。

Summary Financial Terms

财务条款摘要

Upon closing, Arrowhead receives a $500 million upfront payment and $325 million through the purchase by Sarepta of Arrowhead common stock priced at $27.25 per share. Arrowhead will also receive $250 million to be paid in annual installments of $50 million over 5 years. Arrowhead also has the potential to receive $300 million in near-term payments associated with the continued enrollment of certain cohorts of a Phase 1/2 study of ARO-DM1, which Arrowhead is on track to achieve during the next 12 months..

交割后，Arrowhead收到了5亿美元的预付款，并通过Sarepta购买了3.25亿美元的Arrowhead普通股，每股价格为27.25美元。。Arrowhead还有可能获得3亿美元的短期付款，这些款项与ARO-DM1 1/2期研究的某些队列的继续登记有关，Arrowhead有望在未来12个月内实现这一目标。。

Arrowhead is eligible to receive development milestone payments of between $110 million and $410 million per program and sales milestone payments of between $500 million and $700 million per program. Arrowhead is also eligible to receive tiered royalties on commercial sales up to the low double digits..

Arrowhead有资格获得每个项目1.1亿至4.1亿美元的开发里程碑付款，以及每个项目5亿至7亿美元的销售里程碑付款。Arrowhead也有资格获得商业销售的分层版税，最高可达两位数。。

Summary of Programs under License and Collaboration Agreement

许可和合作协议下的计划摘要

Clinical Stage

临床分期

ARO-DUX4, which is designed to target the gene that encodes the DUX4 protein as a potential treatment for patients with facioscapulohumeral muscular dystrophy type 1, currently dosing patients in a Phase 1/2 clinical study.

ARO-DUX4旨在靶向编码DUX4蛋白的基因，作为1型面肩肱型肌营养不良症患者的潜在治疗方法，目前正在进行1/2期临床研究。

ARO-DM1, which is designed to reduce expression of the dystrophia myotonica protein kinase (DMPK) gene in skeletal muscle as a potential treatment for patients with type 1 myotonic dystrophy, currently dosing patients in a Phase 1/2 clinical study.

ARO-DM1旨在降低骨骼肌中肌营养不良肌强直蛋白激酶（DMPK）基因的表达，作为1型肌强直性营养不良患者的潜在治疗方法，目前正在进行1/2期临床研究。

ARO-MMP7, which is designed to reduce expression of matrix metalloproteinase 7 (MMP7) in the lung as a potential treatment for idiopathic pulmonary fibrosis, currently dosing patients in a Phase 1/2 clinical study.

ARO-MMP7旨在降低肺中基质金属蛋白酶7（MMP7）的表达，作为特发性肺纤维化的潜在治疗方法，目前正在1/2期临床研究中给患者服用。

ARO-ATXN2, which is designed to silence expression of the toxic ATXN2 protein in the CNS as a potential treatment for spinocerebellar ataxia 2 (SCA2), currently in a Phase 1/2 study that is open for enrollment.

ARO-ATXN2，旨在沉默中枢神经系统中有毒ATXN2蛋白的表达，作为脊髓小脑共济失调2（SCA2）的潜在治疗方法，目前正在进行1/2期研究，该研究已开放供登记。

Preclinical Stage

临床前阶段

ARO-HTT for patients Huntington’s disease expected to be CTA-ready in 2025

亨廷顿舞蹈症患者的ARO-HTT有望在2025年准备好CTA

ARO-ATXN1 for patients with spinocerebellar ataxia 1 (SCA1) expected to be CTA-ready in 2026

脊髓小脑共济失调1（SCA1）患者的ARO-ATXN1有望在2026年准备好CTA

ARO-ATXN3 for patients with spinocerebellar ataxia 3 (SCA3) expected to be CTA-ready in 2026

ARO-ATXN3用于脊髓小脑共济失调3（SCA3）患者，预计在2026年准备好CTA

Discovery

发现

During the five-year term, Sarepta can propose up to six new CNS or muscle targets for which Arrowhead will perform discovery and preclinical development. Sarepta would then receive an exclusive license to those programs and be responsible for clinical development and commercialization.

在五年任期内，Sarepta可以提出多达六个新的中枢神经系统或肌肉靶标，Arrowhead将对其进行发现和临床前开发。。

Drug Manufacturing

药品生产

Under the agreement, Arrowhead will manufacture clinical drug supply for all programs arising out of the license and collaboration, and commercial drug product for the four programs currently in clinical trials.

根据该协议，箭头将为许可和合作产生的所有项目制造临床药物供应，并为目前正在进行临床试验的四个项目制造商业药物产品。

About Arrowhead Pharmaceuticals

关于箭头制药

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes.

箭头制药公司（Arrowhead Pharmaceuticals）通过沉默导致顽固性疾病的基因来开发治疗顽固性疾病的药物。使用广泛的RNA化学组合和有效的递送方式，箭头疗法触发RNA干扰机制，以诱导靶基因的快速，深入和持久的敲低。

RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing..

RNA干扰或RNAi是活细胞中存在的一种机制，可抑制特定基因的表达，从而影响特定蛋白质的产生。Arrowhead基于RNAi的疗法利用了这种基因沉默的自然途径。。

For more information, please visit

有关更多信息，请访问

www.arrowheadpharma.com

www.arrowheadpharma.com

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@箭头制药公司

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Safe Harbor Statement under the Private Securities Litigation Reform Act:

私人证券诉讼改革法案下的安全港声明：

This news release contains forward-looking statements within the meaning of the 'safe harbor' provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this release except for historical information may be deemed to be forward-looking statements. Without limiting the generality of the foregoing, words such as “may,” “will,” “expect,” “believe,” “anticipate,” “hope,” “intend,” “plan,” “project,” “could,” “estimate,” “continue,” “target,” “forecast” or “continue” or the negative of these words or other variations thereof or comparable terminology are intended to identify such forward-looking statements.

本新闻稿包含1995年《私人证券诉讼改革法案》中“安全港”条款所指的前瞻性声明。除历史信息外，本版本中包含的任何声明都可能被视为前瞻性声明。在不限制上述一般性的情况下，诸如“可能”、“将”、“预期”、“相信”、“预期”、“希望”、“打算”、“计划”、“项目”、“可能”、“估计”、“继续”、“目标”、“预测”或“继续”等词语或其其他变体或类似术语的否定词旨在识别此类前瞻性陈述。

In addition, any statements that refer to projections of our future financial performance, trends in our business, expectations for our product pipeline or product candidates, including anticipated regulatory submissions and clinical program results, prospects or benefits of our collaborations with other companies, or other characterizations of future events or circumstances are forward-looking statements.

此外，任何涉及我们未来财务业绩预测、业务趋势、对我们产品线或候选产品的期望（包括预期的监管提交和临床计划结果）、我们与其他公司合作的前景或益处，或未来事件或情况的其他特征的声明都是前瞻性声明。

These forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; our expectations regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions we have entered into or may enter into in the future; our beliefs and expectations regarding milestone, royalty or other payments that could be due to or from third parties under existing agreements; and our estimates regarding future revenues, research and development expenses, capital requirements and payments to third parties.

这些前瞻性陈述包括但不限于关于我们的临床前研究和临床试验以及我们的研究和开发计划的开始，时间，进展和结果的陈述；我们对合作伙伴关系、许可和/或合作安排以及我们已经达成或将来可能达成的其他战略安排和交易的潜在利益的期望；；以及我们对未来收入、研发费用、资本要求和向第三方付款的估计。

These statements are based u.

这些声明基于美国。

Source:

资料来源：

Arrowhead Pharmaceuticals, Inc.

箭头制药公司。