Encinitas, California--(Newsfile Corp. - October 3, 2023) - Kiora Pharmaceuticals (NASDAQ: KPRX) has received Investigational New Drug Application approval in Australia to enroll up to six additional patients in the ABACUS study of KIO-301. ABACUS was initially designed to evaluate patients with Retinitis Pigmentosa (RP).

加利福尼亚州恩西尼塔斯-（Newsfile Corp.-2023年10月3日）-Kiora Pharmaceuticals（纳斯达克股票代码：KPRX）已在澳大利亚获得研究性新药申请批准，可在KIO-301的ABACUS研究中招募多达6名患者。ABACUS最初设计用于评估色素性视网膜炎（RP）患者。

Based on encouraging findings previously shared, Kiora sought this approval to evaluate additional patients with late-stage RP as well as those with late-stage Choroideremia (CHM), a rare form of hereditary retinal degeneration. Plans remain for Kiora to report topline results on the first six RP patients in the ABACUS study on November 4th at the American Academy of Ophthalmology retina subspecialty day..

基于先前共享的令人鼓舞的发现，Kiora寻求获得此批准，以评估其他晚期RP患者以及晚期脉络膜血症（CHM）（一种罕见的遗传性视网膜变性）患者。Kiora仍计划于11月4日在美国眼科学会视网膜亚专科日在ABACUS研究中报告前6名RP患者的一线结果。。

Kiora previously reported preliminary data in late-stage RP patients finding KIO-301 safe and tolerable with promising signs of efficacy. KIO-301 is a small molecule photoswitch, a first-in-class therapeutic with the potential to restore vision across several inherited retinal diseases (IRDs), regardless of underlying gene mutations..

Kiora先前报道了晚期RP患者的初步数据，发现KIO-301安全且可耐受，具有良好的疗效迹象。KIO-301是一种小分子光开关，是一流的治疗药物，无论潜在的基因突变如何，都有可能在几种遗传性视网膜疾病（IRD）中恢复视力。。

'Data collected from additional patients will provide further insight to guide continued stages of clinical development,' explained Eric Daniels, M.D., Chief Development Officer at Kiora. 'We are now in a position to expand this 'gene mutation-agnostic approach' to generate further proof-of-concept in patients with late-stage CHM.

“从其他患者收集的数据将提供进一步的见解，以指导临床开发的持续阶段，”Kiora首席开发官Eric Daniels博士解释说我们现在能够扩展这种“基因突变不可知论方法”，为晚期CHM患者提供进一步的概念验证。

The common denominator in both RP and CHM is rod and cone degeneration, and that is precisely why KIO-301 has a good opportunity to make an impact.'.

RP和CHM的共同点是杆和锥的退化，这正是为什么KIO-301有很好的机会产生影响。

Choroideremia is a rare X-linked, recessive, inherited retinal disorder affecting approximately 1 in 50,000 individuals. It is caused by a loss-of-function mutation in the CHM gene. Initial symptoms include loss of night vision and often leads to severe vision impairment and blindness. Kiora is working with the Choroideremia Research Foundation (CRF), the world's largest not-for-profit organization dedicated finding a cure for CHM, to assist in a deeper understanding of the disease as well as access to CRFs patient database and thought leaders to assist in trial enrollment and future study designs..

脉络膜血症是一种罕见的X连锁，隐性遗传性视网膜疾病，大约每50000个人中就有1个人受到影响。它是由CHM基因的功能丧失突变引起的。最初的症状包括夜视丧失，并经常导致严重的视力障碍和失明。Kiora正在与世界上最大的非营利组织脉络膜血症研究基金会（CRF）合作，致力于寻找治疗CHM的方法，以帮助更深入地了解该疾病以及访问CRF患者数据库和思想领袖协助在试验注册和未来的研究设计。。

'Adding CHM into ABACUS is consistent with our sharpened focus in addressing orphan retinal diseases,' added Dr. Daniels. 'Running programs in multiple IRDs will allow for operational efficiencies while developing therapeutics for a major unmet need, like CHM. Our work with CRF brings the top thought leaders in the space and access to patient databases to ensure we are giving the best chance of success to the development of KIO-301 in CHM.'.

Daniels博士补充说：“将CHM添加到ABACUS中与我们在解决孤儿视网膜疾病方面的重点相一致。”在多个IRD中运行程序将允许操作效率，同时为CHM等主要未满足需求开发治疗方法。我们与CRF的合作为空间中的顶级思想领袖带来了访问患者数据库的机会，以确保我们能够为CHM中KIO-301的开发提供最佳成功机会。

About Kiora Pharmaceuticals

关于Kiora制药公司

Kiora Pharmaceuticals is a clinical-stage biotechnology company developing and commercializing products for the treatment of orphan retinal diseases. KIO-301 is being developed for the treatment of Retinitis Pigmentosa, and Kiora also plans to develop KIO-301 for Choroideremia and Stargardt's Disease.

Kiora Pharmaceuticals是一家临床阶段生物技术公司，致力于开发和商业化治疗孤儿视网膜疾病的产品。KIO-301正在开发用于治疗色素性视网膜炎，Kiora还计划开发用于脉络膜血症和Stargardt病的KIO-301。

It is a molecular photoswitch that has the potential to restore vision in patients with inherited and/or age-related retinal degeneration. Kiora plans to develop KIO-104 for the treatment of posterior non-infectious uveitis. It is a next-generation, non-steroidal, immuno-modulatory and small molecule inhibitor of Dihydroorotate Dehydrogenase (DHODH) with what Kiora believes is best-in-class picomolar potency and a validated immune modulating mechanism (blocks T cell proliferation and proinflammatory cytokine release)..

它是一种分子光开关，具有恢复遗传性和/或年龄相关性视网膜变性患者视力的潜力。Kiora计划开发KIO-104用于治疗后部非感染性葡萄膜炎。它是二氢乳清酸脱氢酶（DHODH）的下一代非甾体免疫调节和小分子抑制剂，Kiora认为它具有一流的皮摩尔效力和经过验证的免疫调节机制（阻断T细胞增殖和促炎细胞因子释放））。。

In addition to news releases and SEC filings, we expect to post information on our website, www.kiorapharma.com, and social media accounts that could be relevant to investors. We encourage investors to follow us on Twitter and LinkedIn as well as to visit our website and/or subscribe to email alerts..

除了新闻稿和SEC文件外，我们还希望在我们的网站www.kiorapharma.com和可能与投资者相关的社交媒体帐户上发布信息。我们鼓励投资者在Twitter和LinkedIn上关注我们，并访问我们的网站和/或订阅电子邮件警报。。

Forward-Looking Statements

前瞻性声明

Some of the statements in this press release are 'forward-looking' and are made pursuant to the safe harbor provision of the Private Securities Litigation Reform Act of 1995. These 'forward-looking' statements include statements relating to, among other things, the development and commercialization efforts and other regulatory or marketing approval efforts pertaining to Kiora's development-stage products, including KIO-301 and KIO-104, as well as the success thereof, with such approvals or success may not be obtained or achieved on a timely basis or at all, and Kiora's ability to reach a quorum at the adjourned Special Meeting.

本新闻稿中的一些陈述是“前瞻性的”，是根据1995年“私人证券诉讼改革法”的安全港规定制定的。这些“前瞻性”陈述包括与Kiora开发阶段产品（包括KIO-301和KIO-104）有关的开发和商业化努力以及其他监管或市场批准努力等方面的陈述，以及其中的成功，如果这些批准或成功可能无法及时或根本获得或实现，和Kiora在休会特别会议上达到法定人数的能力。

These statements involve risks and uncertainties that may cause results to differ materially from the statements set forth in this press release, including, among other things, the ability to conduct clinical trials on a timely basis, the ability to obtain any required regulatory approvals, market and other conditions and certain risk factors described under the heading 'Risk Factors' contained in Kiora's Annual Report on Form 10-K filed with the SEC on March 23, 2023, or described in Kiora's other public filings.

这些陈述涉及可能导致结果与本新闻稿中所述陈述大不相同的风险和不确定性，其中包括及时进行临床试验的能力，获得任何必要的监管批准的能力，市场和其他条件以及某些风险因素在2023年3月23日提交给SEC的Kiora年度报告10-K标题“风险因素”中描述，或在Kiora的其他公共文件中描述。

Kiora's results may also be affected by factors of which Kiora is not currently aware. The forward-looking statements in this press release speak only as of the date of this press release. Kiora expressly disclaims any obligation or undertaking to release publicly any updates or revisions to such statements to reflect any change in its expectations with regard thereto or any changes in the events, conditions, or circumstances on which any such statement is based, except as required by law..

Kiora的结果也可能受到Kiora目前不知道的因素的影响。本新闻稿中的前瞻性声明仅在本新闻稿发布之日起发表。Kiora明确表示不承担任何义务或承诺公开发布对此类声明的任何更新或修订，以反映其对此类声明的期望的任何变化，或任何此类声明所基于的事件，条件或情况的任何变化，除非另有要求法律。。

Contact

联系

investors@kiorapharma.com

investors@kiorapharma.com

To view the source version of this press release, please visit https://www.newsfilecorp.com/release/182690

要查看此新闻稿的源版本，请访问https://www.newsfilecorp.com/release/182690