JUPITER, Fla.--(BUSINESS WIRE)--Ligand Pharmaceuticals Incorporated (Nasdaq: LGND) today announced that its partner Travere Therapeutics, Inc. (Nasdaq: TVTX) has received full approval from the U.S. Food and Drug Administration (FDA) for FILSPARI® (sparsentan) to slow kidney function decline in adults with primary IgAN who are at risk of disease progression.

佛罗里达州朱庇特市（商业新闻短讯）--配体制药公司（纳斯达克股票代码：LGND）今天宣布，其合作伙伴Travere Therapeutics，Inc.（纳斯达克股票代码：TVTX）已获得美国食品和药物管理局（FDA）对FILSPARI®（sparsentan）的全面批准，以减缓有疾病进展风险的原发性IgAN成人的肾功能下降。

Ligand is entitled to milestone payments and a 9% royalty on worldwide net sales of FILSPARI..

Ligand有权获得里程碑付款和FILSPARI全球净销售额的9%版税。。

FILSPARI was granted accelerated approval in February 2023 based on the surrogate marker of proteinuria. Full approval is based on positive long-term confirmatory results from the PROTECT Study demonstrating that FILSPARI significantly slowed kidney function decline over two years compared to irbesartan..

2023年2月，基于蛋白尿的替代标志物，FILSPARI获得加速批准。完全批准是基于PROTECT研究的积极长期验证结果，该研究表明，与厄贝沙坦相比，FILSPARI在两年内显着减缓了肾功能下降。。

“This announcement is a momentous milestone for Travere and a positive development for people living with this rare kidney disease,” said Todd Davis, CEO of Ligand. “We are encouraged by Travere’s progress to drive adoption of FILSPARI in the U.S. and look forward to the European launch over the coming months.

Ligand首席执行官托德·戴维斯（ToddDavis）表示：“这一宣布对特拉维尔来说是一个重要的里程碑，对患有这种罕见肾脏疾病的人来说是一个积极的发展。”。“我们对Travere在推动美国采用FILSPARI方面取得的进展感到鼓舞，并期待着未来几个月在欧洲的推出。

FILSPARI is a core part of Ligand’s commercial-stage royalty portfolio, and we believe this treatment will be a significant driver of revenue for us over the next several years.”.

FILSPARI是Ligand商业舞台版税组合的核心部分，我们相信这种待遇将在未来几年为我们带来巨大的收入驱动力。”。

FILSPARI is the only oral, once-daily, non-immunosuppressive medication that directly targets glomerular injury in the kidney by blocking two critical pathways of IgAN disease progression (endothelin-1 and angiotensin II).

FILSPARI是唯一一种每日一次的口服非免疫抑制药物，通过阻断IgAN疾病进展的两个关键途径（内皮素-1和血管紧张素II）直接靶向肾脏肾小球损伤。

The two-year efficacy data contained in the FDA-approved label is a modified intention to treat (ITT) analysis, and as preferred by the FDA, evaluates data from all patients regardless of treatment discontinuation. In the final analysis of the 404 randomized patients, FILSPARI significantly reduced the rate of decline in kidney function from baseline to Week 110 compared to irbesartan.

FDA批准的标签中包含的两年疗效数据是一种改进的意向治疗（ITT）分析，并且FDA首选评估所有患者的数据，无论是否停止治疗。在404名随机患者的最终分析中，与厄贝沙坦相比，FILSPARI显着降低了从基线到第110周的肾功能下降率。

In the ITT analysis included in the label, the mean eGFR slope from baseline to Week 110 was -3.0 mL/min/1.73 m2/year for FILSPARI and -4.2 mL/min/1.73 m2/year for irbesartan, corresponding to a statistically significant treatment effect of 1.2 mL/ min/1.73 m2/year (p=0.0168). The positive treatment effects on proteinuria compared to the active control irbesartan that were observed at Week 36 were durable out to the two-year measurement period.

在标签中包含的ITT分析中，FILSPARI从基线到第110周的平均eGFR斜率为-3.0 mL/min/1.73 m2/年，厄贝沙坦为-4.2 mL/min/1.73 m2/年，相当于1.2 mL/min/1.73 m2/年的统计学显着治疗效果（p=0.0168）。与在第36周观察到的活性对照厄贝沙坦相比，对蛋白尿的积极治疗效果持续到两年的测量期。

Additional results from the PROTECT Study demonstrated the benefit of FILSPARI on absolute eGFR accrued over time and by Week 110 resulted in a 3.8 mL/min/1.73 m2 difference in the mean change from baseline between FILSPARI and irbesartan..

PROTECT研究的其他结果表明，FILSPARI对绝对eGFR的益处随着时间的推移而累积，到第110周，FILSPARI和厄贝沙坦之间的平均基线变化差异为3.8 mL/min/1.73 m2。。

Results from the PROTECT Study showed that FILSPARI was well tolerated with a clearly defined safety profile that has been consistent across all clinical trials conducted to date. Following engagement with the FDA, the Company expects to submit an sNDA for a potential modification to the liver-monitoring REMS..

PROTECT研究的结果表明，FILSPARI耐受性良好，安全性明确，迄今为止所有临床试验均一致。在与FDA接洽后，该公司预计将提交一份sNDA，以对肝脏监测REMS进行潜在的修改。。

About IgA Nephropathy

关于IgA肾病

IgA nephropathy (IgAN), also called Berger's disease, is a rare progressive kidney disease characterized by the buildup of immunoglobulin A (IgA), a protein that helps the body fight infections, in the kidneys. The deposits of IgA cause a breakdown of the normal filtering mechanisms in the kidney, leading to blood in the urine (hematuria), protein in the urine (proteinuria) and a progressive loss of kidney function.

IgA肾病（IgAN），也称为伯杰病，是一种罕见的进行性肾脏疾病，其特征是免疫球蛋白a（IgA）在肾脏中积聚，免疫球蛋白a是一种帮助身体抵抗感染的蛋白质。IgA的沉积导致肾脏正常过滤机制的破坏，导致尿液中的血液（血尿），尿液中的蛋白质（蛋白尿）和肾功能的进行性丧失。

Other symptoms of IgAN may include swelling (edema) and high blood pressure..

IgAN的其他症状可能包括肿胀（水肿）和高血压。。

IgAN is the most common type of primary glomerulonephritis worldwide and a leading cause of kidney failure due to glomerular disease. IgAN is estimated to affect up to 150,000 people in the U.S. and is one of the most common glomerular diseases in Europe and Japan.

IgAN是全球最常见的原发性肾小球肾炎类型，也是肾小球疾病导致肾衰竭的主要原因。据估计，IgAN在美国影响多达15万人，是欧洲和日本最常见的肾小球疾病之一。

About the PROTECT Study

关于PROTECT研究

The PROTECT Study is one of the largest interventional studies to date in IgA nephropathy (IgAN) and the only Phase 3 head-to-head trial in this rare kidney disease. It is a global, randomized, multicenter, double-blind, parallel-arm, active-controlled clinical trial evaluating the safety and efficacy of 400 mg of FILSPARI (sparsentan), compared to 300 mg of irbesartan, in 404 patients ages 18 years and up with IgAN and persistent proteinuria despite receiving at least 50% of max label dose and maximally tolerated ACE or ARB therapy..

PROTECT研究是迄今为止IgA肾病（IgAN）中最大的介入研究之一，也是这种罕见肾脏疾病中唯一的3期头对头试验。这是一项全球性，随机，多中心，双盲，平行组，主动对照临床试验，评估了404名18岁及以上的IgAN和持续性蛋白尿患者中400 mg FILSPARI（sparsentan）与300 mg厄贝沙坦相比的安全性和有效性，尽管接受了至少50%的最大标记剂量和最大耐受性ACE或ARB治疗。。

The primary efficacy endpoint for the interim analysis was the change from baseline in urine protein/creatinine ratio at Week 36. The key secondary efficacy endpoint for the final analysis was the rate of change in eGFR over a 110-week period following initiation of randomized therapy.

。最终分析的关键次要疗效终点是随机治疗开始后110周内eGFR的变化率。

The trial met the pre-specified primary endpoint which showed that after 36 weeks patients receiving FILSPARI achieved a mean reduction in proteinuria from baseline of 49.8%, compared to a mean reduction in proteinuria from baseline of 15.1% for irbesartan-treated patients (p<0.0001).

该试验符合预先规定的主要终点，该终点显示36周后，接受FILSPARI治疗的患者的蛋白尿平均从基线水平降低了49.8%，而厄贝沙坦治疗的患者的蛋白尿平均从基线水平降低了15.1%（p<0.0001）。

The two-year efficacy data contained in the FDA-approved label is a modified intention to treat (ITT) analysis, and as preferred by the FDA, evaluates data from all patients regardless of treatment discontinuation. In the final analysis of 404 randomized patients, FILSPARI reduced the rate of decline in kidney function from baseline to Week 110 compared to irbesartan.

FDA批准的标签中包含的两年疗效数据是一种改进的意向治疗（ITT）分析，并且FDA首选评估所有患者的数据，无论是否停止治疗。在404名随机患者的最终分析中，与厄贝沙坦相比，FILSPARI降低了从基线到第110周的肾功能下降率。

The mean eGFR slope from baseline to Week 110 was -3.0 mL/min/1.73 m2/year for FILSPARI and -4.2 mL/min/1.73 m2/year for irbesartan, corresponding to a statistically significant treatment effect of 1.2 mL/ min/1.73 m2/year (p=0.0168)..

从基线到第110周的平均eGFR斜率为FILSPARI为-3.0 mL/min/1.73 m2/年，厄贝沙坦为-4.2 mL/min/1.73 m2/年，相当于1.2 mL/min/1.73 m2/年的统计学显着治疗效果（p=0.0168）。。

Additional results from the PROTECT Study demonstrated the benefit of FILSPARI on absolute eGFR accrued over time and by Week 110 resulted in a 3.8 mL/min/1.73 m2 difference in the mean change from baseline between FILSPARI and irbesartan.

PROTECT研究的其他结果表明，FILSPARI对绝对eGFR的益处随着时间的推移而累积，到第110周，FILSPARI和厄贝沙坦之间的平均基线变化差异为3.8 mL/min/1.73 m2。

Patients who completed the PROTECT double-blind portion of the study on treatment were eligible to participate in the open-label extension of the trial.

完成治疗研究的PROTECT双盲部分的患者有资格参加试验的开放标签延期。

About Ligand Pharmaceuticals

关于配体药物

Ligand is a biopharmaceutical company enabling scientific advancement through supporting the clinical development of high-value medicines. Ligand does this by providing financing, licensing our technologies or both. Its business model seeks to generate value for stockholders by creating a diversified portfolio of biopharmaceutical product revenue streams that are supported by an efficient and low corporate cost structure.

Ligand是一家生物制药公司，通过支持高价值药物的临床开发实现科学进步。配体通过提供资金、许可我们的技术或两者来实现这一目标。其商业模式旨在通过创建由高效和低公司成本结构支持的生物制药产品收入流的多元化组合，为股东创造价值。

Ligand’s goal is to offer investors an opportunity to participate in the promise of the biotech industry in a profitable and diversified manner. Its business model focuses on funding programs in mid- to late-stage drug development in return for economic rights, purchasing royalty rights in development stage or commercial biopharmaceutical products and licensing its technology to help partners discover and develop medicines.

Ligand的目标是为投资者提供一个机会，以盈利和多样化的方式参与生物技术行业的承诺。其商业模式侧重于为中晚期药物开发项目提供资金，以换取经济权利，购买开发阶段或商业生物制药产品的特许使用权，并授权其技术以帮助合作伙伴发现和开发药物。

Ligand partners with other pharmaceutical companies to leverage what they do best (late-stage development, regulatory management and commercialization) in order to generate its revenue. Ligand’s Captisol® platform technology is a chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs.

配体与其他制药公司合作，利用他们最擅长的（后期开发，监管管理和商业化）来产生收入。配体的Captisol®平台技术是一种化学修饰的环糊精，其结构旨在优化药物的溶解度和稳定性。

Ligand has established multiple alliances, licenses and other business relationships with the world’s leading biopharmaceutical companies including Amgen, Merck, Pfizer, Jazz, Takeda, Gilead Sciences, and Baxter International. For more information, please visit at www.ligand.com. Follow Ligand on X @Ligand_LGND..

。有关更多信息，请访问www.ligand.com。关注X@ligand\u LGND上的配体。。

We use our investor relations website and X as a means of disclosing material non-public information and for complying with our disclosure obligations under Regulation FD. Investors should monitor our website and our X account, in addition to following our press releases, SEC filings, public conference calls and webcasts..

。投资者除了关注我们的新闻稿、SEC文件、公开电话会议和网络广播外，还应监控我们的网站和X账户。。

Forward-Looking Statements

前瞻性声明

This news release contains forward-looking statements by Ligand that involve risks and uncertainties and reflect Ligand's judgment as of the date of this release. Words such as “plans,” “believes,” “expects,” “anticipates,” “potential,” “will” and similar expressions are intended to identify forward-looking statements.

本新闻稿包含配体的前瞻性声明，涉及风险和不确定性，并反映了配体截至本新闻稿发布之日的判断。诸如“计划”、“相信”、“期望”、“预期”、“潜在”、“将”等词语以及类似的表达方式旨在识别前瞻性陈述。

These forward-looking statements include, without limitation, statements regarding: the timing of the commercial launch of FILSPARI by Travere and the potential contribution it is expected to bring to Ligand; the timing and amount of milestone payments in connection with FILSPARI that Ligand expects; and the potential royalties to be paid on sales of FILSPARI by Travere.

这些前瞻性声明包括但不限于以下方面的声明：Travere商业推出FILSPARI的时间安排及其预计对配体的潜在贡献；配体预期的与FILSPARI相关的里程碑付款的时间和金额；以及Travere出售FILSPARI可能支付的特许权使用费。

Actual events or results may differ from Ligand’s or its partner’s expectations due to risks and uncertainties inherent in Ligand’s and its partner’s business, including, without limitation: Travere may not be able to successfully commercialize FILSPARI which will depend on a number of factors including coverage and reimbursement levels from governmental authorities and health insurers as well as market acceptance by healthcare providers; the market size for FILSPARI may be smaller than estimated; Ligand is dependent on Travere for the commercialization of FILSPARI; and other risks described in Ligand’s prior press releases and filings with the Securities and Exchange Commission available at www.sec.gov.

由于配体及其合作伙伴业务固有的风险和不确定性，实际事件或结果可能不同于配体或其合作伙伴的预期，包括但不限于：Travere可能无法成功商业化FILSPARI，这将取决于许多因素，包括政府当局和健康保险公司的覆盖率和报销水平以及医疗保健提供者的市场接受程度；FILSPARI的市场规模可能比估计的要小；配体依赖于Travere进行FILSPARI的商业化；以及Ligand之前的新闻稿和提交给证券交易委员会的文件中描述的其他风险，请访问www.sec.gov。

The failure to meet expectations with respect to any of the foregoing matters may reduce Ligand’s stock price. Ligand disclaims any intent or obligation to update these forward-looking statements beyond the date of this release. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995..

未能满足上述任何事项的预期可能会降低Ligand的股价。配体否认在本发布日期之后更新这些前瞻性声明的任何意图或义务。这一警告是根据1995年《私人证券诉讼改革法案》的安全港条款作出的。。