创新疗法开发商Paradigm Therapeutics获得1250万美元融资，以支持完成SD-101（Zorblisa）的开发-动脉网

Paradigm Therapeutics, Inc. Announces Investment by Eshelman Ventures, LLC to Support Completion of Development of SD-101 (Zorblisa™), A Topical Therapy for Treatment Across the Entire Skin Surface of All Subtypes of Epidermolysis Bullosa (EB)

CISION 等信源发布 2025-05-06 22:12

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First Topical Therapy Developed to Treat the Wounds and Lesions on the Entire Skin Surface Across All EB Subtypes

首个用于治疗所有EB亚型患者全身皮肤表面伤口和病变的局部疗法问世

Funding to be Used for Completion of All Activities to Support Planned NDA Submission in the Second Half of 2025, Along with Plans for Commercialization in the U.S.

资助将用于完成所有活动以支持计划于2025年下半年提交的新药申请（NDA），以及美国市场商业化的相关计划。

MT PLEASANT, S.C.

南卡罗来纳州，芒特普莱森特

，

May 6, 2025

2025年5月6日

/PRNewswire/ -- Paradigm Therapeutics, Inc., a biopharmaceutical company, announces a

/PRNewswire/ -- Paradigm Therapeutics, Inc.，一家生物制药公司，宣布

$12.5 Million

1250万美元

investment by Eshelman Ventures, LLC and appointment of Dr. Eshelman as a strategic advisor for Paradigm. SD-101 (Zorblisa™) is being developed as a topical whole-body treatment for all subtypes of Epidermolysis Bullosa (EB) patients. Funds will be used to complete all activities to support an NDA submission in the later part of 2025. .

Eshelman Ventures, LLC 进行投资，并任命 Eshelman 博士为 Paradigm 的战略顾问。SD-101（Zorblisa™）正在开发作为针对所有亚型的表皮松解症（EB）患者的局部全身治疗药物。资金将用于完成支持 2025 年下半年提交新药申请（NDA）的所有活动。

'We are excited about the opportunity to complete the development activities for SD-101 (Zorblisa™) and to move a path forward towards registration of this therapy for patients with all EB subtypes,' said

“我们对完成SD-101（Zorblisa™）的开发活动并为所有EB亚型患者推进该疗法的注册路径感到兴奋，”

Robert Ryan

罗伯特·瑞安

., Ph.D., Chief Executive Officer of Paradigm Therapeutics. 'There is a tremendous need for novel whole skin surface treatment options for treating the debilitating skin effects across all patients with Epidermolysis Bullosa. The Paradigm Therapeutics team will be working passionately to provide patients with a new therapeutic alternative in this disease with limited whole-body treatment options.

博士，Paradigm Therapeutics首席执行官。他表示：“对于所有患有表皮松解症的患者而言，目前急需全新的全身皮肤表面治疗方案来应对这些严重的皮肤问题。Paradigm Therapeutics团队将全力以赴，为这类疾病提供一种新的治疗选择，因为当前全身治疗方案非常有限。”

Paradigm's goal is to provide this therapy to patients on a global basis and is working in parallel with potential partners to work towards this goal as quickly as possible.'.

Paradigm的目标是为全球患者提供这种疗法，并正在与潜在合作伙伴并行工作，以尽快实现这一目标。

Dr. Ryan added, 'Epidermolysis Bullosa is a devastating disease. We believe the clinical data show beneficial effects of the use of SD-101 across the range of EB patients, and strongly support progression towards registration. Results from the completed Phase II and Phase III trials indicate that SD-101 demonstrated a broad benefit with whole body treatment of the lesions and wounds on the entire skin surface in pediatric and adult patients across all EB patients, who have limited whole body treatment alternatives.

瑞安博士补充道：“表皮松解症是一种毁灭性的疾病。我们相信临床数据展示了SD-101在各类EB患者中的有益效果，并强烈支持向注册推进。已完成的II期和III期试验结果表明，SD-101在全身治疗中表现出广泛的益处，可治疗儿童和成人患者全身皮肤表面的病灶和伤口，而这些EB患者目前的全身治疗选择非常有限。”

Results also demonstrated that SD-101 has a favorable safety profile when used long term.'.

结果还表明，SD-101 长期使用时具有良好的安全性。

'Eshelman Ventures is honored to invest in Paradigm Therapeutics and play a pivotal role in accelerating the development of SD-101 as a transformative treatment for Epidermolysis Bullosa,' said Dr. Eshelman, founder of Eshelman Ventures. 'As an investor and strategic advisor, I am deeply committed to working closely with this exceptional team as we work tirelessly to bring this innovative therapy to patients in need.

“Eshelman Ventures很荣幸能够投资Paradigm Therapeutics，并在加速开发SD-101作为治疗表皮松解症的变革性疗法中发挥关键作用，”Eshelman Ventures创始人埃舍尔曼博士表示。“作为一名投资者和战略顾问，我深感有责任与这支卓越的团队紧密合作，全力以赴将这一创新疗法带给有需要的患者。”

The urgency of addressing the challenges faced by EB patients is what drives us; our goal is to provide an improved quality of life in this area of unmet need. Together, we will strive to revolutionize treatment options and make a meaningful impact on the lives of those affected by this debilitating disease.'.

解决EB患者面临的挑战的紧迫性驱使着我们；我们的目标是在这一未满足需求的领域提供更好的生活质量。我们将共同努力，彻底改变治疗选择，并对受这种衰弱性疾病影响的人们的生活产生有意义的影响。

About Epidermolysis Bullosa (EB)

关于表皮松解症（EB）

Epidermolysis Bullosa is a rare, devastating genetic disorder causing very fragile skin that leads to severe skin blistering and open wounds often beginning at birth. There are no approved therapies that were developed to treat the entire skin surface of patients across all subtypes of EB patients, including wounds and lesional skin, which affects children and adults throughout their lifetimes. EB is a chronic disease, potentially disfiguring, and in some cases fatal.

表皮松解症是一种罕见的、严重的遗传性疾病，会导致皮肤非常脆弱，从而引发严重的水疱和开放性伤口，通常从出生时便开始。目前尚无获批的疗法能够针对所有亚型的表皮松解症患者治疗其整个皮肤表面，包括伤口和病灶皮肤，这种疾病会影响儿童和成人的一生。表皮松解症是一种慢性疾病，可能导致毁容，在某些情况下甚至可能致命。

There are many genetic and symptomatic variations of EB, but all forms share the common symptom of fragile skin that blisters and tears, sometimes from the slightest friction or trauma..

EB有许多遗传和症状变异，但所有形式都有一个共同的症状，即皮肤脆弱，容易起水泡和撕裂，有时甚至是最轻微的摩擦或创伤也会导致这种情况。

About SD-101 (Zorblisa™)

关于SD-101（Zorblisa™）

SD-101 (Zorblisa™) is a topical cream that has demonstrated potential to provide improvement in treating the severe skin disruptions seen in patients across all EB subtypes. Phase II and Phase III studies were conducted in children and adults with either Simplex, Recessive Dystrophic (RDEB), or Junctional EB.

SD-101（Zorblisa™）是一种局部用药的乳膏，已在治疗所有EB亚型患者中出现的严重皮肤损伤方面显示出改善的潜力。II期和III期研究针对患有单纯型、隐性营养不良型（RDEB）或交界型EB的儿童和成人展开。

A total of 217 EB patients were included in these studies. SD-101 was applied topically over the entire body daily in adults and children as young as 21 days old for a period of three months. The primary outcome measurement was assessment of target wound closure, in addition to secondary measurements including reduction in body surface area (BSA) coverage of blisters and wounds..

这些研究共纳入了217名EB患者。SD-101每天全身局部应用于成人及年仅21天的儿童，持续三个月。主要结局指标是评估目标伤口闭合情况，次要指标包括水疱和伤口覆盖体表面积（BSA）的减少。

SD-101 (Zorblisa™) was the first EB treatment to receive the FDA's Breakthrough Therapy designation. SD-101 (Zorblisa™) received Rare Pediatric Disease designation from the U.S. Food and Drug Administration (FDA) covering the broad treatment of EB. In addition to the Rare Pediatric Disease designation, SD-101 was granted Orphan Drug designation from the FDA and the European Commission (EC).

SD-101（Zorblisa™）是首个获得FDA突破性疗法认定的EB治疗药物。SD-101（Zorblisa™）获得了美国食品药品监督管理局（FDA）授予的罕见儿科疾病认定，涵盖EB的广泛治疗。除了罕见儿科疾病认定外，SD-101还获得了FDA和欧洲委员会（EC）授予的孤儿药认定。

Upon approval, Paradigm will be eligible to receive a Priority Review Voucher (PRV). .

一经批准，Paradigm将有资格获得优先审查券（PRV）。

About Paradigm Therapeutics, Inc.

关于Paradigm Therapeutics公司

Paradigm Therapeutics, Inc. is a privately held, clinical-stage pharmaceutical company focused on developing innovative therapies to address critical medical needs in the treatment of rare diseases. Paradigm's main focus is the treatment of Epidermolysis Bullosa (EB). SD-101 (Zorblisa™), its leading therapy for the treatment of skin effects in patients with Epidermolysis Bullosa, has completed Phase II and Phase III clinical trials.

Paradigm Therapeutics, Inc. 是一家私人持有的临床阶段制药公司，专注于开发创新疗法以满足罕见病治疗中的关键医疗需求。Paradigm的主要关注点是大疱性表皮松解症（EB）的治疗。其领先的疗法SD-101（Zorblisa™），用于治疗大疱性表皮松解症患者的皮肤症状，已经完成了II期和III期临床试验。

SD-101 (Zorblisa™) is a topical cream that requires no special handling and can be stored at room temperature and has demonstrated potential to provide improvement in treating the severe skin effects seen in patients across all EB subtypes. .

SD-101（Zorblisa™）是一种局部用药膏，无需特殊处理，可在室温下储存，并已显示出在治疗所有EB亚型患者中出现的严重皮肤症状方面的改善潜力。

Forward Looking Statement

前瞻性声明

Except for the historical information contained herein, the matters discussed in this press release are forward-looking statements that involve risks and uncertainties, including: our dependence on third parties for the development, regulatory approval and successful commercialization of our products, the inherent risk of failure in developing product candidates based on new technologies, risks associated with the costs of clinical development efforts, as well as other risks.

本新闻稿中除包含的历史信息外，所讨论的事项均为涉及风险和不确定性的前瞻性陈述，包括：我们依赖第三方进行产品的开发、监管批准和成功商业化，基于新技术开发产品候选物的固有失败风险，与临床开发工作成本相关的风险，以及其他风险。

Actual results may differ materially from those projected. These forward-looking statements represent our judgment as of the date of the release. Paradigm disclaims any intent or obligation to update these forward-looking statements..

实际结果可能与预测的结果有重大差异。这些前瞻性陈述代表了我们截至发布日期的判断。Paradigm 不承担更新这些前瞻性陈述的意图或义务。

Company Contact

公司联系人

Dr. Robert Ryan

罗伯特·瑞安博士

Chief Executive Officer

首席执行官

rryan@paradigmtherapeutics.com

(919)-274-0703

SOURCE Paradigm Therapeutics, Inc.

来源：Paradigm Therapeutics, Inc.

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