PLX-2853

SAN DIEGO--( BUSINESS WIRE )--Opna Bio, a clinical-stage biopharmaceutical company focused on the discovery and development of novel oncology therapeutics, announced interim data from its lead clinical program, OPN-2853, in patients with advanced myelofibrosis showing encouraging levels of spleen reduction, with minimal toxicities and adverse events. Preclinical data from a second program, OPN-6602, showed significant tumor regression in multiple myeloma models as a single agent and in combination with other therapeutics. The presentations took place this weekend at the American Society of Hematology (ASH) annual meeting, December 7-10, 2024, in San Diego. OPN-2853 Shows Spleen Reduction in Patients with Advanced Myelofibrosis OPN-2853, a small molecule bromodomain and extra-terminal motif (BET) inhibitor, is being evaluated in an ongoing Phase 1 investigator-led study in patients with advanced myelofibrosis who are no longer responding to ruxolitinib alone. Myelofibrosis is a type of blood cancer that causes fibrosis in the bone marrow, anemia and enlarged spleen, amongst other symptoms. The study is testing three dose levels of OPN-2853 (20 mg, 40 mg and 80 mg), given orally once daily, in combination with ruxolitinib. As of February 2024, the cut-off date, 16 patients had been enrolled at different dose levels, across multiple sites in the United Kingdom, coordinated by the Cancer Research UK (CRUK) Clinical Trials Unit at the University of Birmingham. In 12 evaluable patients, the median spleen size was reduced from baseline with spleens no longer palpable in 50% of evaluable patients. The combination dose has been well tolerated, and the majority of patients have completed eight cycles of combination treatment. “We are very encouraged by these data to date, which demonstrate that daily dosing of OPN-2853 in combination with ruxolitinib was well tolerated, and showed spleen reduction in patients with myelofibrosis who have very limited options once they have progressed,” said principal investigator Adam Mead, PhD, MRCP, FRCP, professor of hematology at the University of Oxford and CRUK senior cancer research fellow. “We are enthusiastic about the OPN-2853 and ruxolitinib combination and expect to have a recommended Phase 2 dose in early 2025.” Opna Bio is planning further clinical development with OPN-2853. OPN-6602 Combinations Demonstrate Complete Tumor Regression in Preclinical Multiple Myeloma Models OPN-6602, an oral, small molecule inhibitor of the E1A binding protein (EP300) and CREB-binding protein (CBP), is currently being tested in a Phase 1 trial in patients with multiple myeloma (MM) . MM is a type of blood cancer derived from malignant plasma cells in the bone marrow. In MM mouse xenograft models, OPN-6602 showed 71% tumor suppression as a single agent, and 100% tumor regression when combined with dexamethasone, pomalidomide or mezigdomide with a sustained duration of response. RNA sequencing of treated tumors showed that OPN-6602 downregulated key MM driver and signature genes, and has the potential to overcome resistance mechanisms to standard-of-care regimens. “We are very encouraged by the strength of the OPN-2853 clinical and OPN-6602 preclinical data, which highlights the potential of their unique pharmacokinetic profiles and validates our ‘safety by design’ approach. Both drug candidates have a high Cmax and short half-life, which allows for continuous daily dosing and effective target engagement with rapid clearance to mitigate toxicities,” said Gideon Bollag, PhD, co-founder and chief scientific officer of Opna Bio. “This confers a significant advantage to these drugs when used as single agents and a potentially synergistic effect when used in combination with other therapies.” About Opna Bio 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. 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 multiple myeloma patients. For more information, please visit opnabio.com .

▲5月30-31日 第八届广州生物医药创新者峰会 扫码立即报名 注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需得到授权。2022年1月12日，日本制药巨头第一三共（Daiichi Sankyo）宣布决定关闭其位于美国三藩市南部的研发子公司Plexxikon，以最大限度地将投资用于研发旗下的多种抗体偶联药物开发（当时的3ADC—ENHERTU®、Dato DXd和HER3 DXd），部分研发管线转送至总部进行开发。Plexxikon于2022年3月31日正式永久暂停运行。Plexxikon始创于2000年，是第一三共在2011年以9.35亿美元的价格收购的子公司，当时拥有两款已上市抗肿瘤药物（Zelboraf/Vemurafenib与罗氏共同开发推广；和Turalio/Pexidartinib治疗腱鞘巨细胞瘤/TGCT）和6款已经启动临床试验的药物。药融云数据www.pharnexcloud.com显示：原Plexxikon团队从公司收购了部分资产(5款)，于2022年3月份成立了新的小分子新药公司Opna Bio（Opna-IO LLC ），产品线处于临床阶段的有：OPN-2853 (此前的PLX2853), 一款BET候选新药；OPN-7486 (此前的PLX7486), 一款CSF-1R/TRK候选新药。公司临床前阶段的资产有：EP300抑制剂；CD73抑制剂；TEAD抑制剂；FMRP（Fragile X mental retardation protein，脆性 X 染色体智力迟滞蛋白）等等。2022年11月，公司完成3800万美元A轮融资，现有投资人有：Longitude Capital、Northpond Ventures、Menlo Ventures等等。笔者认为，从此类关停或者破产清算BIOTECH公司购买合适资产去开发的模式，或者适合当下的中国创业者或者药企。原来存在一些庞大公司里的资产过于复杂，开发也许并未深究。参考：NMPA/CDE；药融云数据www.pharnexcloud.com；FDA/EMA/PMDA；相关公司公开披露；opnabio.com；https://www.plexxikon.com/；https://www.biocentury.com/article/646652/opna-bio-targeting-fmrp-to-treat-cancer；https://precoro.com/blog/opna-bio-sa-case-study/；https://www.businesswire.com/news/home/20221121005179/en/;https://www.daiichisankyo.co.jp/files/news/pressrelease/pdf/202201/20220112_J.pdf；https://www.daiichisankyo.co.jp/rd/pipeline/；  等等。本文仅用于向医疗卫生专业人士提供科学信息，不代表平台立场，不作任何用药推荐 活动推荐  5月 • 第八届大湾区生物医药创新者峰会  关键词：  小分子，XDC，多肽及GLP-1热点话题，80+行业专家（点击下方图片查看详情）▼【关于药融圈】药融圈PRHub旨在帮助生物医药科技型企业进行品牌推广及商务拓展服务，针对客户的真实需求制定系统化解决方案，通过“翻译-降维-场景化”将客户的品牌信息以直白易懂的方式被公众知悉，同时在流量渠道覆盖100万+垂直用户基础上实现合作目的，帮助合作伙伴完成从品牌开始到商务为终的闭环营销服务。我们已经完成了数十场线下1000人规模的生物医药研发类会议，涵盖小分子新药，大分子新药，改良型新药，BD跨境交易等多个领域，服务了百余家上市/独角兽/生物技术/制药企业。