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KalVista Pharmaceuticals, Inc.

KalVista 制药公司

(NASDAQ: KALV), today announced the presentation of novel data related to long-term prophylaxis and sebetralstat at the Western Society of Allergy, Asthma & Immunology (WSAAI) 2025 Annual Meeting taking place in Waimea, HI from February 9-13, 2025.

（纳斯达克股票代码：KALV）今天宣布，在2025年2月9日至13日于HI Waimea举行的西方过敏，哮喘和免疫学学会（WSAAI）2025年年会上，展示了与长期预防和sebetralstat相关的新数据。

Raffi Tachdjian, MD, MPH, Associate Clinical Professor of Medicine and Pediatrics in the Division of Allergy and Clinical Immunology at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA), presented data on the

加州大学洛杉矶分校（UCLA）大卫·格芬医学院（DavidGeffen School of Medicine）过敏与临床免疫学系医学与儿科副临床教授拉菲·塔奇安（Raffi Tachdjian）介绍了有关

Impact of Long-Term Prophylaxis (LTP) Adherence in Hereditary Angioedema Patients: Results of a Claims Database Analysis.

长期预防（LTP）依从性对遗传性血管性水肿患者的影响：索赔数据库分析的结果。

According to a US commercial claims analysis, nearly 40% of HAE patients who initiated LTP had substantial refill gaps in claims over 12 months, with more than half of those discontinuing LTP.

根据美国商业索赔分析，发起LTP的HAE患者中，近40%在12个月内索赔有较大的补充缺口，超过一半的患者停止了LTP。

For patients with substantial refill gaps, which increase the likelihood of non-adherence, on-demand claims remained unchanged before and after one year of starting LTP.

对于有大量补液间隙的患者，这增加了不依从的可能性，在开始LTP一年之前和之后，按需索赔保持不变。

“Despite guidelines emphasizing regular assessment of patients using LTP to confirm efficacy and safety of their therapy, these data suggest many patients experience substantial lapses in refilling their LTP, which may reflect non-adherence,” said Dr. Tachdjian. “We saw no decrease in on-demand claims for patients with LTP refill gaps.

Tachdjian博士说：“尽管指南强调定期评估使用LTP的患者以确认其治疗的有效性和安全性，但这些数据表明许多患者在重新填充LTP时出现了重大失误，这可能反映了不依从性。”。“我们发现LTP补充缺口患者的按需索赔没有减少。

These findings are important as most HAE patients in the US now receive LTP, and a greater focus on monitoring appears warranted, as LTP effectiveness is not a given. These data may also help to explain why on-demand treatment volumes in HAE have remained steady despite the advent of several effective subcutaneous and oral LTP options since 2017.”.

这些发现很重要，因为美国大多数HAE患者现在都接受了LTP，并且由于LTP的有效性尚未确定，因此有必要更加关注监测。这些数据也可能有助于解释为什么自2017年以来，尽管出现了几种有效的皮下和口服LTP选择，但HAE的按需治疗量保持稳定。”。

Marc A. Riedl, MD, Professor of Medicine and Clinical Director, US Hereditary Angioedema Association Center at the University of California, San Diego, presented data on the

加利福尼亚大学圣地亚哥分校美国遗传性血管性水肿协会中心医学教授兼临床主任MarcA.Riedl博士介绍了

Safety and Effectiveness of Sebetralstat in Patients with Hereditary Angioedema Receiving Long-Term Prophylaxis: Interim Analysis from the KONFIDENT-S Open-Label Study.

Sebetralstat在接受长期预防的遗传性血管性水肿患者中的安全性和有效性：来自KONFIDENT-S开放标签研究的中期分析。

Participants receiving LTP treated 382 attacks with sebetralstat (mean 1.7 attacks per month), of which more than half involved the abdomen and/or larynx.

接受LTP治疗的参与者用sebetralstat治疗了382次发作（平均每月1.7次发作），其中一半以上涉及腹部和/或喉部。

Sebetralstat enabled early treatment (median 6 minutes) and early symptom relief (median 1.3 hours), and was well-tolerated, regardless of LTP mechanism of action or route of administration.

无论LTP的作用机制或给药途径如何，Sebetralstat均可实现早期治疗（中位数6分钟）和早期症状缓解（中位数1.3小时），并且耐受性良好。

“Treatment guidelines recommend that patients with HAE should consider treating all attacks regardless of LTP use and should treat attacks as early as possible,” noted Dr. Riedl. “These interim results suggest that, if approved, sebetralstat could be a safe and effective oral on-demand treatment for breakthrough attacks, regardless of severity, location, or type of LTP used.”.

Riedl博士指出：“治疗指南建议HAE患者应考虑治疗所有发作，无论是否使用LTP，并应尽早治疗发作。”。“这些中期结果表明，如果获得批准，无论使用的LTP的严重程度，位置或类型如何，西贝他司他都可能是一种安全有效的口服按需治疗突破性发作的药物。”。

Michael E. Manning, MD, allergist-immunologist at Allergy, Asthma and Immunology Associates, Ltd., Scottsdale, Arizona and Past President of WSAAI, presented

亚利桑那州斯科茨代尔过敏、哮喘和免疫协会有限公司过敏学家、免疫学家、医学博士迈克尔·曼宁（MichaelE.Manning）和WSAAI前任总裁介绍

Sebetralstat for the Treatment of HAE Attacks in Patients Receiving Berotralstat: Interim Analysis from the KONFIDENT-S Open-Label Study.

Sebetralstat治疗接受Berotralstat治疗的患者HAE发作：来自KONFIDENT-S开放标签研究的中期分析。

Participants receiving berotralstat treated 178 attacks with sebetralstat (mean 1.8 attacks per month).

接受berotralstat治疗的参与者用sebetralstat治疗了178次发作（平均每月1.8次发作）。

Sebetralstat enabled early treatment (median 20 minutes), early symptom relief (median 1.3 hours), and was well-tolerated with no increase in gastrointestinal side effects.

Sebetralstat能够早期治疗（中位数20分钟），早期症状缓解（中位数1.3小时），并且耐受性良好，胃肠道副作用没有增加。

“In general, patients prefer oral treatments over injectables, underscoring the strong interest in oral options for HAE,” said Dr. Manning. “These results demonstrate that sebetralstat, when used as an oral on-demand treatment for attacks occurring among patients receiving berotralstat for LTP, enabled rapid treatment and symptom improvement.

曼宁博士说：“一般来说，患者更喜欢口服治疗而不是注射剂，这突显了人们对HAE口服治疗的强烈兴趣。”。“这些结果表明，当使用西贝他司他作为口服按需治疗LTP患者中发生的发作时，能够快速治疗和症状改善。

If approved, sebetralstat could transform the treatment landscape for physicians and patients who prefer to manage HAE without needles.”.

如果获得批准，sebetralstat可以为更喜欢无针治疗HAE的医生和患者改变治疗格局。”。

“Injectable on-demand treatments have side effects and logistical obstacles that hinder adequate disease control and drive an over-reliance on LTP,” said Paul Audhya, MD, MBA, Chief Medical Officer of KalVista. “LTP does not, however, always yield the anticipated reductions in attacks, possibly due to adherence issues in a chronic lifelong setting.

KalVista首席医疗官、医学博士、MBA保罗·奥迪亚（PaulAudhya）表示：“按需注射治疗有副作用和后勤障碍，阻碍了疾病的充分控制，并导致过度依赖LTP。”。“然而，LTP并不总是产生预期的攻击减少，可能是由于长期终身环境中的依从性问题。

Sebetralstat has the potential to enable early treatment of attacks, thereby halting progression at an early stage and reducing morbidity, even among patients on LTP. By overcoming the barriers imposed by current injectable on-demand therapies, sebetralstat could shift the treatment paradigm and become the foundation of HAE management.”.

Sebetralstat有可能早期治疗发作，从而在早期停止进展并降低发病率，即使在LTP患者中也是如此。通过克服当前可注射按需治疗所带来的障碍，塞贝特拉司他可以改变治疗范式，成为HAE管理的基础。”。

Links to all presentations can be found on the KalVista website under

所有演示文稿的链接都可以在KalVista网站上找到

Publications

出版物

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About the KONFIDENT Phase 3 Trial

关于KONFIDENT 3期试验

The KONFIDENT phase 3 clinical trial was a randomized, double-blind, 3-way crossover trial evaluating the safety and efficacy of sebetralstat 300 mg and 600 mg versus placebo for the on-demand treatment of HAE in adult and pediatric patients aged 12 years and older. The trial randomized 136 HAE patients from 66 clinical sites across 20 countries, making it the largest clinical trial ever conducted in HAE.

KONFIDENT 3期临床试验是一项随机、双盲、三向交叉试验，评估了塞贝他300 mg和600 mg与安慰剂对12岁及以上成人和儿童HAE按需治疗的安全性和有效性。该试验将来自20个国家66个临床地点的136名HAE患者随机分组，使其成为HAE有史以来规模最大的临床试验。

In the trial, participants treated each eligible attack with up to two doses of study drug and treated up to three attacks over the course of the study. The trial included Type I and Type II HAE patients who had at least two documented HAE attacks 90 days prior to randomization and also included patients receiving long-term prophylaxis..

在试验中，参与者用最多两剂研究药物治疗每一次符合条件的发作，并在研究过程中治疗最多三次发作。该试验包括I型和II型HAE患者，他们在随机分组前90天至少有两次记录的HAE发作，还包括接受长期预防的患者。。

About the KONFIDENT-S Trial

关于KONFIDENT-S审判

KONFIDENT-S is an open-label extension trial with numerous real-world elements evaluating the long-term safety and efficacy of sebetralstat for the on-demand treatment of HAE attacks in adults and pediatric patients aged 12 years and older with HAE Type I or Type II. KalVista is transitioning ongoing trial participants to a novel oral disintegrating tablet (ODT) formulation to support a planned 2026 sNDA filing.

KONFIDENT-S是一项开放标签的扩展试验，有许多实际因素评估了sebetralstat在成人和12岁及以上HAE I型或II型儿科患者中按需治疗HAE发作的长期安全性和有效性。KalVista正在将正在进行的试验参与者转换为一种新型口腔崩解片（ODT）配方，以支持计划的2026年sNDA申请。

If approved, the ODT formulation would provide people living with HAE with an alternative, novel option for oral, on-demand treatment..

如果获得批准，ODT配方将为HAE患者提供另一种新颖的口服按需治疗选择。。

About Sebetralstat

关于Sebetralstat

Sebetralstat is an investigational, novel oral plasma kallikrein inhibitor for the treatment of hereditary angioedema (HAE). We have filed multiple regulatory applications seeking approval of sebetralstat as the first oral, on-demand treatment for HAE in individuals aged 12 and older, with ongoing studies exploring its use in children aged 2 to 11.

Sebetralstat是一种用于治疗遗传性血管性水肿（HAE）的研究性新型口服血浆激肽释放酶抑制剂。我们已经提交了多项监管申请，寻求批准sebetralstat作为12岁及以上人群HAE的第一种口服按需治疗，正在进行的研究正在探索其在2至11岁儿童中的应用。

If approved, sebetralstat has the potential to become the foundational therapy for HAE management worldwide..

如果获得批准，sebetralstat有可能成为全球HAE管理的基础疗法。。

About Hereditary Angioedema

关于遗传性血管性水肿

Hereditary angioedema (HAE) is a rare genetic disease resulting in deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system. People living with HAE experience painful and debilitating attacks of tissue swelling in various locations of the body that can be life-threatening depending on the area affected.

遗传性血管性水肿（HAE）是一种罕见的遗传性疾病，导致C1酯酶抑制剂（C1INH）蛋白缺乏或功能障碍，并随后导致激肽释放酶-激肽系统的不受控制的激活。患有HAE的人会在身体的不同部位经历痛苦和衰弱的组织肿胀发作，根据受影响的部位，这些组织肿胀可能危及生命。

All currently approved on-demand treatment options require either intravenous or subcutaneous administration..

所有目前批准的按需治疗方案都需要静脉或皮下给药。。

About KalVista Pharmaceuticals, Inc.

关于KalVista Pharmaceuticals，Inc。

KalVista Pharmaceuticals, Inc., is a global biopharmaceutical company dedicated to developing and delivering life-changing oral therapies for individuals affected by rare diseases with significant unmet needs. Our lead investigational product is sebetralstat, a novel, oral, on-demand treatment for hereditary angioedema (HAE).

KalVista Pharmaceuticals，Inc.是一家全球生物制药公司，致力于为受罕见疾病影响且需求未得到满足的个体开发和提供改变生命的口服疗法。我们的主要研究产品是sebetralstat，一种针对遗传性血管性水肿（HAE）的新型口服按需治疗药物。

Sebetralstat is under regulatory review by the U.S. FDA, with a PDUFA goal date of June 17, 2025. In addition, we have completed Marketing Authorization Applications for sebetralstat to the European Medicines Agency and multiple other global regulatory authorities..

Sebetralstat正在接受美国FDA的监管审查，PDUFA的目标日期为2025年6月17日。此外，我们已经向欧洲药品管理局和其他多个全球监管机构完成了sebetralstat的上市授权申请。。

For more information about KalVista, please visit

有关KalVista的更多信息，请访问

www.kalvista.com

www.kalvista.com

or follow us on social media at

或者在社交媒体上关注我们

@KalVista

@KalVista

and

和

LinkedIn

LinkedIn

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.

Forward-Looking Statements

前瞻性声明

This press release contains 'forward-looking' statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: 'anticipate,' 'intend,' 'plan,' 'goal,' 'seek,' 'believe,' 'project,' 'estimate,' 'expect,' 'strategy,' 'future,' 'likely,' 'may,' 'should,' 'will' and similar references to future periods.

本新闻稿包含1995年《美国私人证券诉讼改革法案》安全港条款所指的“前瞻性”声明。前瞻性陈述可以用“预期”、“打算”、“计划”、“目标”、“寻求”、“相信”、“项目”、“估计”、“预期”、“战略”、“未来”、“可能”、“可能”、“应该”、“将会”等词来识别，以及对未来时期的类似引用。

These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, timing or outcomes of communications with the FDA, our expectations about safety and efficacy of our product candidates and timing of clinical trials and its results, our ability to commence clinical studies or complete ongoing clinical studies, including our KONFIDENT-S and KONFIDENT-KID trials, and to obtain regulatory approvals for sebetralstat and other candidates in development, the success of any efforts to commercialize sebetralstat, the ability of sebetralstat and other candidates in development to treat HAE or other diseases, and the future progress and potential success of our oral Factor XIIa program.

这些声明受到许多风险和不确定性的影响，可能导致实际结果与我们的预期存在重大差异。前瞻性陈述的例子包括，与FDA沟通的时间或结果，我们对候选产品的安全性和有效性的期望以及临床试验的时间安排及其结果，我们开始临床研究或完成正在进行的临床研究（包括我们的KONFIDENT-S和KONFIDENT-KID试验）的能力，以及为开发中的西贝塔司他和其他候选药物获得监管部门批准的能力，西贝塔司他商业化的任何努力的成功，西贝塔司他和其他候选药物治疗HAE或其他疾病的能力，以及我们口服因子XIIa计划的未来进展和潜在成功。

Further information on potential risk factors that could affect our business and financial results are detailed in our filings with the Securities and Exchange Commission, including in our annual report on Form 10-K for the year ended April 30, 2024, our quarterly reports on Form 10-Q, and our other reports that we may make from time to time with the Securities and Exchange Commission.

有关可能影响我们业务和财务业绩的潜在风险因素的更多信息，请参阅我们向证券交易委员会提交的文件，包括截至2024年4月30日的10-K表年度报告、10-Q表季度报告以及我们可能不时向证券交易委员会提交的其他报告。

We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new info.

我们没有义务公开更新任何前瞻性声明，无论是书面的还是口头的，无论是由于新信息而不时做出的。