)--Opna Bio, a clinical-stage biopharmaceutical company focused on the discovery and development of novel oncology therapeutics, announced today that the FDA has granted orphan drug designation (ODD) to one of its lead programs, OPN-6602, for the treatment of multiple myeloma (MM). OPN-6602 is an oral, small molecule inhibitor of the E1A binding protein (EP300) and CREB-binding protein (CBP) currently being tested in a .

)--Opna Bio是一家专注于发现和开发新型肿瘤治疗药物的临床阶段生物制药公司，今天宣布，FDA已将孤儿药指定（ODD）授予其主要项目之一OPN-6602，用于治疗多发性骨髓瘤（MM）。OPN-6602是目前正在a中测试的E1A结合蛋白（EP300）和CREB结合蛋白（CBP）的口服小分子抑制剂。

Phase 1 trial

第一阶段试验

in patients with relapsed or refractory MM.

复发或难治性MM患者。

Multiple myeloma is a rare and aggressive cancer of the plasma cells in the bone marrow that often leads to serious complications such as bone damage, kidney failure and immune suppression. The disease is typically diagnosed in older adults, with limited treatment options available for patients with relapsed or refractory disease..

多发性骨髓瘤是一种罕见的侵袭性骨髓浆细胞癌，通常会导致严重的并发症，如骨损伤、肾衰竭和免疫抑制。该疾病通常在老年人中被诊断出来，复发或难治性疾病患者的治疗选择有限。。

“We are pleased to have received ODD for OPN-6602 for the treatment of multiple myeloma, a further validation of the drug’s therapeutic potential in patients with this disease who have limited treatment options once they have relapsed,” said Gideon Bollag, PhD, chief scientific officer.

首席科学官吉迪恩·博拉格（GideonBollag）博士说：“我们很高兴收到用于治疗多发性骨髓瘤的OPN-6602的ODD，这进一步验证了该药物在复发后治疗选择有限的这种疾病患者中的治疗潜力。”。

Orphan Drug Designation is granted by the FDA to encourage the development of therapies for rare diseases, which are defined as those affecting fewer than 200,000 people in the U.S. The designation provides several benefits, including tax credits for clinical trial costs, a waiver of certain FDA fees, and eligibility for seven years of market exclusivity upon approval..

美国食品和药物管理局（FDA）授予孤儿药名称，以鼓励开发罕见疾病的治疗方法，这些疾病被定义为影响美国不到20万人的疾病。该名称提供了多种好处，包括临床试验费用的税收抵免，免除某些FDA费用，以及获得批准后七年市场排他性的资格。。

Opna recently presented

Opna最近推出

data

数据

at the American Society of Hematology (ASH) meeting in December 2024 showing that in human-derived multiple myeloma models, OPN-6602 suppresses tumor growth, while downregulating key MM driving genes. Synergistic effects were observed with OPN-6602 in combination with dexamethasone, pomalidomide and mezigdomide.

在 2024 年 12 月举行的美国血液学会（ASH）会议上，OPN-6602 证明在人源多发性骨髓瘤模型中，OPN-6602 能抑制肿瘤生长，同时下调关键的多发性骨髓瘤驱动基因。OPN-6602 与地塞米松、泊马度胺和美沙多米联合用药可产生协同效应。

OPN-6602’s distinct pharmacokinetic profile allows for continuous daily dosing that potentially results in a lower incidence of toxicities and improved efficacy..

OPN-6602独特的药代动力学特征允许每天连续给药，这可能会降低毒性发生率并提高疗效。

The Phase 1 study in patients with relapsed or refractory multiple myeloma (NCT06433947) is taking place at multiple sites in the U.S. Opna Bio expects to complete the single agent, dose-escalation phase of the trial in 2026. Further development of OPN-6602 in combination with other standard-of-care agents in multiple myeloma is planned..

复发或难治性多发性骨髓瘤患者的1期研究（NCT06433947）正在美国的多个地点进行。Opna Bio预计将于2026年完成该试验的单药剂量递增阶段。计划进一步开发OPN-6602与其他多发性骨髓瘤标准治疗药物的组合。。

About Opna Bio

Opna生物

Opna Bio is a clinical-stage biopharmaceutical company focused on the discovery and development of novel oncology therapeutics. The company’s broad portfolio targets multiple drivers of cancer, including a novel oncology discovery program focused on the fragile-X multifunctional RNA-binding protein (FMRP) and a diversified pipeline of promising oncology assets.

Opna Bio是一家临床阶段的生物制药公司，专注于发现和开发新型肿瘤治疗药物。该公司的广泛投资组合针对多种癌症驱动因素，包括一项专注于脆性X多功能RNA结合蛋白（FMRP）的新型肿瘤学发现计划和一系列有前途的肿瘤学资产。

The Opna team has a proven track record of scientific expertise and commercial value creation, having advanced multiple FDA-approved drugs to market. Opna’s lead clinical compounds include OPN-2853, a potentially best-in-class BET bromodomain inhibitor, being evaluated in patients with myelofibrosis in combination with ruxolitinib, and OPN-6602, a dual EP300/CBP inhibitor, currently being studied in a first-in-human Phase 1 clinical trial in patients with multiple myeloma.

Opna团队在科学专业知识和商业价值创造方面有着良好的记录，已经将多种FDA批准的药物推向市场。Opna的主要临床化合物包括OPN-2853，一种潜在的最佳BET溴结构域抑制剂，正在与ruxolitinib联合用于骨髓纤维化患者的评估，以及OPN-6602，一种双重EP300/CBP抑制剂，目前正在多发性骨髓瘤患者的首次人体1期临床试验中进行研究。