Warsaw, 21st May 2024 – Molecure S.A. (‘Molecure’, WSE ticker: MOC), a biotechnology company that discovers and develops drugs to the clinical stage, leveraging its globally unique expertise in medicinal chemistry and biology to explore and develop first-in-class small molecule drugs that directly modulate protein activity and mRNA function for the treatment of multiple incurable diseases, announces today that it has received national regulatory approvals from Denmark, France, Greece, Germany and Norway to conduct a Phase II clinical trial for OATD-01, a first-in-class chitotriosidase 1 (CHIT1) inhibitor with disease-modifying potential in pulmonary sarcoidosis..

2024年5月21日，华沙——Molecure S.A.（“Molecure”，WSE股票代码：MOC）是一家发现和开发临床药物的生物技术公司，利用其在药物化学和生物学方面的全球独特专业知识，探索和开发直接调节蛋白质活性和mRNA功能以治疗多种不治之症的一流小分子药物，今天宣布，该公司已获得丹麦、法国、希腊、德国和挪威的国家监管批准，将对OATD-01进行II期临床试验，OATD-01是一种一流的壳三糖苷酶1（CHIT1）抑制剂，具有改善肺结节病疾病的潜力。。

Marcin Szumowski, Molecure’s CEO said: “An important milestone has been reached in the Phase II clinical trial of OATD-01 with the first patient administration in March this year at the Royal Infirmary in Edinburgh. We are pleased to receive further regulatory approvals from selected EU countries and Norway to conduct this breakthrough clinical study.

Molecure首席执行官马辛·苏莫夫斯基（MarcinSzumowski）表示：“OATD-01的II期临床试验已经达到了一个重要的里程碑，今年3月在爱丁堡的皇家医院首次进行了患者给药。我们很高兴获得选定欧盟国家和挪威的进一步监管批准，以进行这项突破性的临床研究。

The first dose is expected to be administered to patients in the selected countries in the third quarter of this year. The Phase II clinical trials to be conducted in both the USA and Europe, the two most commercially important markets, will enable us to collect the necessary data that, if positive, will confirm clinical proof of concept in a pulmonary sarcoidosis patient population that currently has few treatment options with limited efficacy in disease modification.

预计第一剂将于今年第三季度在选定国家的患者中服用。将在美国和欧洲这两个商业上最重要的市场进行的II期临床试验将使我们能够收集必要的数据，如果这些数据呈阳性，将证实肺结节病患者群体的临床概念证明，目前很少有治疗选择，在疾病改变方面疗效有限。

OATD-01 has demonstrated remarkable potential in preclinical studies, suggesting it could redefine the standard of care for pulmonary sarcoidosis. The results of these studies will be crucial for further development and commercialization of OATD-01 in this and other potential indications such as NASH/MASH, IPF or inflammatory bowel disease (IBD).

OATD-01在临床前研究中显示出显着的潜力，表明它可以重新定义肺结节病的护理标准。这些研究的结果对于OATD-01在NASH/MASH，IPF或炎症性肠病（IBD）等其他潜在适应症中的进一步开发和商业化至关重要。

We look forward to presenting the unblinded results of this study in late 2025.”.

我们期待着在2025年末公布这项研究的非盲结果。”。

The Phase II clinical trial for OATD-01 is a randomized, double-blinded, placebo-controlled, multicenter study to evaluate the safety and efficacy of OATD-01 in approximately 100 patients with active pulmonary sarcoidosis, including patients previously receiving other therapies with no clinical improvement and patients previously untreated..

OATD-01的II期临床试验是一项随机，双盲，安慰剂对照的多中心研究，旨在评估OATD-01在大约100例活动性肺结节病患者中的安全性和有效性，包括先前接受其他治疗但没有临床改善的患者和先前未经治疗的患者。。

Due to the requirement for double blinding in the study, the final unblinded results publication will occur after its completion and is scheduled for the end of 2025. To measure efficacy of OATD-01 in the study, an innovative primary endpoint has been agreed upon with the regulatory authorities, which is the response to 12-week administration of OATD-01, measured by the degree of granulomatous inflammation reduction in the lung parenchyma, assessed by PET/CT imaging.

由于该研究需要双盲，最终的非盲结果发布将在完成后进行，计划于2025年底发布。为了测量OATD-01在研究中的疗效，与监管机构达成了一项创新的主要终点，这是对OATD-01 12周给药的反应，通过PET/CT成像评估肺实质肉芽肿炎症减轻程度。

Following the completion of full dosing along with a monitoring period involving approximately 50 patients, an interim analysis (intermediate checkpoint) is planned to evaluate the statistical results by an independent committee and make decisions regarding the study's continuation in terms of patient numbers in early Q1 of 2025..

在完成全面给药以及涉及大约50名患者的监测期后，计划进行中期分析（中间检查点），以评估独立委员会的统计结果，并在2025年第一季度初就患者人数继续进行研究做出决定。。

The study will involve approximately 20-30 centers in the USA, European Union, Norway, and the United Kingdom. The renowned Contract Research Organization (CRO) responsible for organizing and conducting the comprehensive study is Simbec Orion.

该研究将涉及美国，欧盟，挪威和英国的大约20-30个中心。Simbec Orion是著名的合同研究组织（CRO），负责组织和进行综合研究。

About OATD-01

关于OATD-01

OATD-01 is an oral, once-daily, first-in-class, and highly selective CHIT1 inhibitor for potential use in the treatment of sarcoidosis. The CHIT1 enzyme is a promising molecular target due to its role in transforming local anti-inflammatory macrophages into pro-inflammatory and pro-fibrotic types. Blocking CHIT1 activity by OATD-01 has resulted in documented anti-inflammatory and anti-fibrotic effects..

OATD-01是一种口服，每日一次，一流的高选择性CHIT1抑制剂，可用于治疗结节病。CHIT1酶是一种有前途的分子靶标，因为它在将局部抗炎巨噬细胞转化为促炎和促纤维化类型中起作用。OATD-01阻断CHIT1活性已导致记录的抗炎和抗纤维化作用。。

The OATD-01 molecule has shown strong anti-inflammatory and anti-fibrotic effects in various disease models and has high therapeutic potential in diverse inflammatory and fibrotic diseases with unmet medical needs, such as sarcoidosis, as well as idiopathic pulmonary fibrosis (IPF) and non-alcoholic steatohepatitis (NASH), recently relabeled as Metabolic Dysfunction-Associated Steatohepatitis (MASH)..

OATD-01分子在各种疾病模型中显示出强大的抗炎和抗纤维化作用，并且在各种炎症和纤维化疾病中具有很高的治疗潜力，这些疾病的医疗需求尚未得到满足，例如结节病，特发性肺纤维化（IPF）和非酒精性脂肪性肝炎（NASH），最近被重新标记为代谢功能障碍相关脂肪性肝炎（MASH）。。

Molecure has obtained orphan drug designation (ODD) from the FDA for OATD-01 in the indications of sarcoidosis and idiopathic pulmonary fibrosis and has received approval to initiate a Phase II clinical trial for the treatment of pulmonary sarcoidosis in the US, UK, selected countries of the European Union, and Norway..

Molecure已从FDA获得OATD-01结节病和特发性肺纤维化适应症的孤儿药名称（ODD），并已获得批准，在美国、英国、欧盟选定国家和挪威启动治疗肺结节病的II期临床试验。。

About sarcoidosis

关于结节病

Sarcoidosis is a multi-organ disease of unknown etiology characterized by the formation of granulomatous structures in various organs, primarily in the lungs and lymphatic system.

结节病是一种病因不明的多器官疾病，其特征是在各种器官中形成肉芽肿结构，主要在肺和淋巴系统中。

It is a globally occurring disease affecting both men and women with an estimated incidence of 5-50 cases per 100,000 population, with 70% of patients being between 25-45 years old.

这是一种全球性疾病，影响男性和女性，估计每10万人口中有5-50例发病，其中70%的患者年龄在25-45岁之间。

The most serious and common complication of sarcoidosis is pulmonary fibrosis, usually associated with significant impairment of lung function. Pulmonary fibrosis is the cause of most sarcoidosis-related deaths in Western countries.

结节病最严重和最常见的并发症是肺纤维化，通常伴有严重的肺功能损害。肺纤维化是西方国家大多数结节病相关死亡的原因。

About Molecure S.A.

关于Molecure S.A。

Molecure S.A. is a biotechnology company discovering and developing drugs to the clinical stage, leveraging its unique expertise in medicinal chemistry and biology to search for and develop first-in-class small molecule drugs that, through direct modulation of previously unexplored protein and RNA targets, can provide therapy for many incurable diseases..

Molecure S.A.是一家发现和开发临床药物的生物技术公司，利用其在药物化学和生物学方面的独特专业知识，寻找和开发一流的小分子药物，通过直接调节先前未开发的蛋白质和RNA靶标，可以为许多不治之症提供治疗。。

Molecure has generated a diversified portfolio of seven distinct programs with the support of leading academic institutions worldwide, including Yale University, Rutgers University, the Flemish Institute for Biotechnology (VIB) in Ghent, the University of Michigan, and the International Institute of Molecular and Cell Biology in Warsaw (MIBMiK)..

Molecure在耶鲁大学、罗格斯大学、根特佛兰德生物技术研究所（VIB）、密歇根大学和华沙国际分子与细胞生物学研究所（MIBMiK）等全球领先学术机构的支持下，推出了七个不同项目的多元化组合。。

The most advanced drug candidate developed by Molecure is OATD-01, a first-in-class CHIT1 inhibitor for the treatment of interstitial lung diseases such as sarcoidosis and idiopathic pulmonary fibrosis.

Molecure开发的最先进的候选药物是OATD-01，一种一流的CHIT1抑制剂，用于治疗结节病和特发性肺纤维化等间质性肺病。

The second drug candidate is OATD-02, an oral, selective, first-in-class, dual arginase inhibitor (ARG1 and ARG2) for the treatment of cancers, with Phase I clinical trials commencing with the first patient administration in the first quarter of 2023.

第二种候选药物是OATD-02，一种用于治疗癌症的口服，选择性，一流的双精氨酸酶抑制剂（ARG1和ARG2），I期临床试验从2023年第一季度的第一次患者给药开始。

Molecure's headquarters and laboratories are located in Warsaw and Łódź. The company is listed on the Warsaw Stock Exchange (ticker: MOC).

Molecure的总部和实验室位于华沙和Łódź。该公司在华沙证券交易所上市（股票代码：MOC）。