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Quoin Pharmaceuticals宣布来自正在进行的儿童Netherton综合征研究的更多积极“全身性”临床数据,并获准开始对第二名儿童患者进行测试

Quoin Pharmaceuticals Announces Additional Positive ‘Whole Body’ Clinical Data from Ongoing Pediatric Netherton Syndrome Study and Approval to Initiate Testing of a Second Pediatric Patient

GlobeNewswire 等信源发布 2025-04-02 07:30

可切换为仅中文


Continued Clear Visual Evidence of Almost Completely Healed Skin After 6 weeks Treatment

治疗6周后皮肤几乎完全愈合的持续清晰视觉证据

High Durability of Treatment Effect of QRX003 with Continuous Daily Dosing

QRX003连续每日给药的治疗效果具有高持久性

Patient Continues Not to Require Previously Necessary Medications

患者继续不需要之前必要的药物治疗

With Patient’s Pruritus Almost Completely Eliminated, Patient Continues to Experience Zero Nightly Sleep Disturbance

患者的瘙痒几乎完全消除,夜间睡眠未再受到干扰。

No Adverse Events Reported from 6 Weeks of Whole Body Treatment with QRX003

QRX003全身治疗6周未报告不良事件

Approval Received to Initiate Whole Body Testing of a Second Pediatric Patient

已批准对第二名儿科患者进行全身测试

ASHBURN, Va., April 02, 2025 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a late clinical stage, specialty pharmaceutical company focused on the development and commercialization of therapeutic products that treat rare and orphan diseases, today announces additional highly positive clinical data from its ongoing Investigator Pediatric Netherton Syndrome (NS) study.

弗吉尼亚州阿什本,2025年4月2日(环球新闻社)——Quoin Pharmaceuticals Ltd.(纳斯达克股票代码:QNRX)(“公司”或“Quoin”),一家专注于开发和商业化治疗罕见和孤儿疾病的晚期临床阶段专业制药公司,今日宣布了其正在进行的儿科研究者Netherton综合征(NS)研究中额外的非常积极的临床数据。

After 6 weeks of continued whole body application of QRX003, the subject’s skin remains almost completely healed demonstrating the durability of ongoing daily treatment with the product. In addition, the patient has continued to have no requirement for previously necessary medications such as antibiotics, antivirals, antihistamines and glucocorticoids.

连续全身应用QRX003六周后,受试者的皮肤几乎完全愈合,显示出该产品持续每日治疗的持久效果。此外,患者继续无需使用以前必需的药物,如抗生素、抗病毒药、抗组胺药和糖皮质激素。

Importantly, with the patient’s pruritus or itch almost completely eliminated, she continues to experience zero nightly sleep disturbances without the need for any sedating medication, marking the first continuous period of uninterrupted sleep in the patient’s life. No adverse events have been reported to date after 6 weeks of whole body treatment with QRX003..

重要的是,患者的瘙痒几乎完全消除,她继续在没有任何镇静药物的情况下夜间睡眠未受干扰,这标志着患者生命中首次连续无间断的睡眠期。在使用QRX003进行全身治疗6周后,至今尚未报告任何不良事件。

Quoin CEO Dr. Michael Myers, said, “As we continue to monitor the progress of this first patient to receive whole body application with QRX003, we are very pleased to announce that after 6 weeks of dosing we are seeing continued positive improvement and the patient’s skin is almost completely healed now.

Quoin首席执行官迈克尔·迈尔斯博士表示:“在我们继续监测首位接受QRX003全身应用的患者进展时,我们非常高兴地宣布,在经过6周的给药后,我们看到了持续的积极改善,患者的皮肤现在几乎已完全愈合。"

The patient continues to remain off previously needed medications including antihistamines, antivirals, and glucocorticoids, and she has not needed any antibiotics in the 6 weeks since the initiation of dosing with QRX003. Very importantly also, with her pruritus almost completely eliminated, the patient is still experiencing zero nightly sleep disturbances without the need for any sedating medications.

患者继续停止使用之前所需的药物,包括抗组胺药、抗病毒药和糖皮质激素,并且在开始使用QRX003剂量后的6周内,她没有需要任何抗生素。非常重要的是,由于她的瘙痒几乎完全消除,患者仍然没有夜间睡眠障碍,且无需任何镇静药物。

For a majority of Netherton Syndrome patients, the continuous presence of chronically debilitating pruritus is the worst symptom of this disease and it severely impacts their ability to sleep or to even sit still for any period of time. Often heavy sedation is required to allow patients to sleep and sit still, which in turn has a major negative impact on their quality of life.

对于大多数尼思顿综合征患者来说,持续存在的慢性衰弱性瘙痒是该疾病最严重的症状,严重地影响了他们的睡眠能力,甚至无法静坐一段时间。通常需要重度镇静才能让患者入睡或静坐,这反过来又对他们的生活质量产生了重大的负面影响。

The almost complete healing of the skin and the elimination of this chronically debilitating pruritus provides strong clinical evidence that QRX003 is successfully tackling the root causes of this disease and not merely providing symptomatic relief. Furthermore, I am very pleased to report that no adverse events have been reported after six full weeks of whole-body application of QRX003.

皮肤几乎完全愈合,这种慢性致残性瘙痒的消除提供了强有力的临床证据,表明QRX003正在成功解决该疾病的根源,而不仅仅是提供症状缓解。此外,我非常高兴地报告,在全身应用QRX003六周后,没有报告任何不良事件。

Based on these positive results, approval has been received to initiate whole body application of QRX003 to a second pediatric Netherton patient. Testing is expected to commence in just a few weeks and we look forward to providing updates on both patients progress as we continue with our plans to further expand thi.

基于这些积极的结果,已获准开始对第二名患有Netherton综合征的儿童患者进行QRX003的全身应用。预计测试将在几周内开始,我们期待在继续推进计划的同时,提供两名患者进展的更新。

About Netherton Syndrome

关于尼瑟顿综合症

Netherton Syndrome, a form of Ichthyosis, is a rare hereditary skin disorder caused by a mutation in the SPINK5 gene (serine protease inhibitor, Kazal Type 5) that leads to severe skin barrier defects and recurring infections, as well as a pronounced predisposition to allergies, asthma, and eczema. Patients often suffer from severe dehydration, chronic skin inflammation and stunted growth.

Netherton综合征是一种鱼鳞病,是由SPINK5基因(丝氨酸蛋白酶抑制剂,Kazal 5型)突变引起的罕见遗传性皮肤病,会导致严重的皮肤屏障缺陷和反复感染,同时伴有显著的过敏、哮喘和湿疹倾向。患者常遭受严重脱水、慢性皮肤炎症和生长迟缓的困扰。

Currently, there is no cure for Netherton Syndrome, nor are there any approved therapeutic treatments..

目前,尼瑟顿综合症尚无治愈方法,也无任何获批的治疗方法。

About QRX003

关于 QRX003

QRX003 is a topical lotion formulated with a proprietary delivery technology that contains a broad-spectrum serine protease inhibitor, whose mechanism of action is intended to perform the function of a specific protein called LEKTI. The absence of LEKTI in Netherton patients leads to excessive skin shedding, resulting in a highly porous and compromised skin barrier.

QRX003是一种外用乳液,采用专有递送技术配方,含有广谱丝氨酸蛋白酶抑制剂,其作用机制旨在实现一种名为LEKTI的特定蛋白质的功能。Netherton综合征患者缺乏LEKTI,导致皮肤过度脱落,从而形成高度多孔且受损的皮肤屏障。

QRX003 is designed to promote a more normalized skin-shedding process and the formation of a stronger and more effective skin barrier. For more information about Quoin’s current clinical trials please visit: .

QRX003旨在促进更正常的皮肤脱落过程,并形成更强效的皮肤屏障。有关Quoin当前临床试验的更多信息,请访问:。

https://www.nethertonsyndromeclinicaltrials.com/

https://www.nethertonsyndromeclinicaltrials.com/

About Quoin Pharmaceuticals Ltd.

关于Quoin Pharmaceuticals Ltd.

Quoin Pharmaceuticals Ltd. is a late clinical-stage specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases. We are committed to addressing unmet medical needs for patients, their families, communities and care teams. Quoin’s innovative pipeline comprises four products in development that collectively have the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, SAM Syndrome,  Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa, Microcystic Lymphatic Malformations, Venous Malformations, Angiofibroma and others.

Quoin Pharmaceuticals Ltd. 是一家处于后期临床阶段的专科制药公司,专注于开发和商业化治疗罕见病和孤儿病的治疗产品。我们致力于满足患者、他们的家庭、社区和护理团队未被满足的医疗需求。Quoin 的创新研发管线包括四种在研产品,这些产品共同有可能针对多种罕见和孤儿病适应症,包括尼泽顿综合征、脱皮皮肤综合征、SAM 综合征、掌跖角化病、硬皮病、表皮松解症、微囊性淋巴管畸形、静脉畸形、血管纤维瘤等。

For more information, visit: .

欲了解更多信息,请访问:。

www.quoinpharma.com

www.quoinpharma.com

or

LinkedIn

领英

for updates.

更新。

Cautionary Note Regarding Forward Looking Statements

关于前瞻性陈述的注意事项

The Company cautions that statements in this press release that are not descriptions of historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words referencing future events or circumstances, such as “expect,” “intend,” “hope,” “plan,” “potential,” “anticipate,” “look forward,” “believe,” “may,” and “will,” among others.

公司提醒,本新闻稿中的声明如果不是对历史事实的描述,则属于1995年《私人证券诉讼改革法案》所指的前瞻性陈述。前瞻性陈述可以通过使用引用未来事件或情况的词语来识别,例如“预期”、“打算”、“希望”、“计划”、“潜力”、“预计”、“期待”、“相信”、“可能”和“将”等。

All statements that reflect the Company’s expectations, assumptions, projections, beliefs, or opinions about the future, other than statements of historical fact, are forward-looking statements, including, without limitation, statements relating to: the potential efficacy of QRX003 as a treatment for Netherton Syndrome; testing expected to commence in a few weeks on a second pediatric patient; continue with our plans to further expand this study to include additional pediatric subjects; and Quoin’s products in development collectively having the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, SAM Syndrome,  Palmoplantar Keratoderma, Scleroderma, Epidermolysis Bullosa, Microcystic Lymphatic Malformations, Venous Malformations, Angiofibroma and others.

所有反映公司对未来预期、假设、预测、信念或意见的声明,而非历史事实的陈述,均为前瞻性声明,包括但不限于以下相关声明:QRX003作为Netherton综合征治疗的潜在疗效;预计几周内将对第二名儿科患者开始测试;继续推进我们的计划以进一步扩展这项研究,纳入更多的儿科受试者;以及Quoin正在开发的产品总体上具有针对多种罕见和孤儿适应症的潜力,包括Netherton综合征、剥脱性皮炎综合征、SAM综合征、掌跖角化病、硬皮病、表皮松解症、微囊性淋巴管畸形、静脉畸形、血管纤维瘤等。

Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon the Company’s current expectations and involve assumptions that may never materialize or may prove to be incorrect.

由于这些陈述受到风险和不确定性的约束,实际结果可能与这些前瞻性陈述所表达或暗示的结果有重大差异。这些前瞻性陈述基于公司的当前预期,涉及的假设可能永远不会实现或可能被证明是不正确的。

Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties including, bu.

实际结果和事件发生的时间可能与这些前瞻性陈述中预期的有重大差异,原因是存在各种风险和不确定性,包括但不限于以下因素。

For further information, contact:

如需更多信息,请联系:

Quoin Pharmaceuticals Ltd.

奎因制药有限公司

Michael Myers, Ph.D., CEO

迈克尔·迈尔斯,博士,首席执行官

mmyers@quoinpharma.com

mmyers@quoinpharma.com

Investor Relations

投资者关系

PCG Advisory

PCG咨询公司

Jeff Ramson

杰夫·拉姆森

jramson@pcgadvisory.com

jramson@pcgadvisory.com

(646) 863-6341

(646) 863-6341