KalVista Pharmaceuticals, Inc.,

KalVista Pharmaceuticals，股份有限公司。，

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

宣布于2025年2月9日至13日在夏威夷州瓦伊梅亚举行的西方过敏、哮喘和免疫学学会（WSAAI）2025年年会上介绍了与长期预防和西贝他司他相关的新数据

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

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）介绍了长期预防（LTP）对遗传性血管性水肿患者依从性影响的数据：索赔数据库分析的结果。

i. 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.  ii. 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..

i、 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。二。对于有大量补液间隙的患者，这会增加不依从的可能性，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

塔奇坚博士

. “We saw no decrease in on-demand claims for patients with LTP refill gaps. 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..

“我们发现LTP补液缺口患者的按需索赔没有减少。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的按需治疗量仍保持稳定。。

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

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Marc A.Riedl医学博士介绍了西贝他司他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数据：来自KONFIDENT-S开放标签研究的中期分析。。

i. 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.

i、 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。

ii. 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.

二。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..

。“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 Sebetralstat for the Treatment of HAE Attacks in Patients Receiving Berotralstat: Interim Analysis from the KONFIDENT-S Open-Label Study. i. Participants receiving berotralstat treated 178 attacks with sebetralstat (mean 1.8 attacks per month).

“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前任总裁介绍了西贝特拉司他治疗接受贝罗他治疗的患者HAE发作：来自KONFIDENT-S开放标签研究的中期分析。i、 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）。

ii. 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..

二。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

“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，”他说

Dr. Manning

曼宁博士

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

“这些结果表明，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。如果获得批准，西贝他司他可以改变医生和喜欢无针治疗HAE的患者的治疗格局。”。

“Injectable on-demand treatments have side effects and logistical obstacles that hinder adequate disease control and drive an over-reliance on LTP,” said

他说：“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。”

Paul Audhya

保罗 奥迪亚

, MD, MBA, Chief Medical Officer of

KalVista

KalVista

. “LTP does not, however, always yield the anticipated reductions in attacks, possibly due to adherence issues in a chronic lifelong setting. 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.

“然而，LTP并不总是产生预期的发作减少，这可能是由于慢性终身环境中的依从性问题。西贝特拉司他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。

By overcoming the barriers imposed by current injectable on-demand therapies, sebetralstat could shift the treatment paradigm and become the foundation of HAE management.”.

。

About the KONFIDENT Phase III Trial; The KONFIDENT phase III 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.

关于KONFIDENT III期试验；KONFIDENT III期临床试验是一项随机、双盲、三因素交叉试验，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。

The trial randomized 136 HAE patients from 66 clinical sites across 20 countries, making it the largest clinical trial ever conducted in HAE. 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..

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。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; 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.

关于KONFIDENT-S审判；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。。

Condition:

条件：

Hereditary Angio-Oedema

遗传性血管水肿

Type:

drug

药物