Rona Therapeutics Co.,Ltd

11月17日，由研发客支持，上海市生物医药科技产业促进中心、易方达私募基金管理有限公司和上海国有资本投资有限公司共同承办的生物医药产业投融资论坛在上海张江科学会堂成功举办。本次论坛作为“2024中国医药工业发展大会 上海国际生物医药产业周”的系列活动之一，旨在推进创新端、产业端、资金端的深度融合，搭建本土创新药企业同跨国药企及投资机构交流与合作的桥梁。论坛邀请了国内外顶尖创新企业、专家学者、金融机构等，通过主旨演讲、高端对话和1对1商务洽谈等，深入剖析中国医药产业全球创新发展，切实促进产业链各方交流连接。论坛现场吸引了超过500名专业观众参加。 上海市科学技术委员会副主任朱启高 上海市科学技术委员会副主任朱启高在致辞中指出，生物医药是上海重点发展的三大先导产业之一，也是加快培育发展新质生产力的重要领域。上海始终牢记习近平总书记嘱托，瞄准打造世界级生物医药产业创新高地的重要使命，持续推进生物医药“研发+临床+制造+应用”全链条加速体系，打造“产学研医金”协同发展新生态。今年以来，上海已有6款1类创新药、10个Ⅲ类创新医疗器械获批上市；生物医药产业母基金和未来产业基金相继成立，产业生态持续优化；上海生物医药企业在科创板上市数、一级市场融资规模及平均融资额均处于全国首位。面对生物医药领域结构转型、破局新生之际，要持续推进创新、持续深化合作、持续推动改革，共同打造开放、包容、多元合作的产业投融资生态，助力生物医药创新企业能够行稳致远，加快构建世界级的生物医药产业集群。 易方达基金管理有限公司常务副总裁、易方达私募基金管理有限公司董事长马骏 易方达基金管理有限公司常务副总裁、易方达私募基金管理有限公司董事长马骏在致辞中表示，国内生物医药产业在全球创新中发挥着越来越重要的作用。根据易方达团队统计，中国创新药企业对外授权交易的金额从2016年至2023年底增长了131倍，达到421亿美元。国内完备的医药工业产业链及强大的工程化能力，高效且低成本地推动中国医药创新快速融入全球创新生态，与新能源、光伏等其他高端制造业一样，我国生物医药产业正在稳步进入到全球大市场。 上海未来产业基金总经理魏凡杰 上海未来产业基金总经理魏凡杰从颠覆性技术创新的内在属性、面临的挑战及加快发展的驱动因素三个方面分享了他的认识和思考。他表示，上海未来产业基金的愿景是希望成为Open Innovation平台，能够真正把人才、资本、技术和产业深度融合起来，基于未来产业基金这个大的母基金平台赋能本土的初创公司，未来给上海孵化出更多真正好的项目。 上海国投先导基金管理有限公司副总经理何苗 上海国投先导基金管理有限公司副总经理何苗围绕“发挥先导母基金担当，助力上海生物医药生态建设”这一主题进行了分享。她表示，发展新质生产力呼唤耐心资本，国有资本已成为国内耐心资本压舱石。打造母子基金矩阵，发挥母基金的杠杆作用，引导各类医疗子基金对潜力企业加强战略投资，可以更好地助力上海生物医药生态建设。 百利天恒创始人、董事长朱义 百利天恒创始人、董事长朱义在以“出海一一成为MNC”为主题的报告中分享了他对于本土公司如何成为具有全球能力和规模的跨国药企的关键成功要素，主要体现在具有潜在超级重磅药物&丰富的管线、全球合作和获得资本加持等方面。 高赢律师事务所合伙人、亚太区生命科学业务负责人潘文森，深信生物创始人、董事长兼CEO李林鲜，联影医疗董事、董事会秘书、首席投资官蔡涛，维亚生物创新中心负责人戴晗，辉瑞中国战略及业务发展副总裁任宇，默沙东亚太业务拓展与授权副总监祁昱，得时资本合伙人郭稼，摩根大通股票资本市场部主管陈晓颖共同围绕“创新+国际化，中国医药产业迎来新时代”展开圆桌讨论。 易方达私募医疗健康投资部研究员龚元超、维亚臻生物CEO邹晓明、深势科技创始人、CEO孙伟杰、诺泰生物首席投资官黎晌、石药集团投资总监梁益坚、祥峰投资合伙人刘天然、上海国投先导生物医药产业基金执行董事王珊、浦发银行科技金融部处长邹学诚共同围绕“生物科技创新加速，探索产业未来方向”展开圆桌讨论。 榜单发布/颁奖仪式 会议期间发布了“2024中国生物医药领跑者100榜单”，共包含生物医药、医疗器械、数字医疗三个重要领域。 本次会议还举行了第四季生物医药“明日之星”大赛颁奖仪式。 本次论坛还设置路演对接环节，标新生物、大睿生物、睿诺医疗、诗丹德、信念医药、英诺湖医药和长森药业展示了其在生物医药研发前沿领域的优势和潜力。论坛通过搭建优质项目与投资机构的交汇桥梁，促进资本与产业的交融，实现多方合作共赢。 现场观众参会拍摄瞬间 “创新持续升级，链接全球生态”是本次论坛的主题，也是生物医药企业在新时代赶考路上的全新挑战。本届2024中国医药工业发展大会生物医药产业投融资论坛的圆满举办，将为生物医药产业的未来发展注入新动力，助力上海加快打造具有全球影响力的生物医药产业创新高地。

SHANGHAI, China I November 17, 2024 I Rona Therapeutics, a leader in innovative RNA-based therapies, today presented positive results from the Phase 1 clinical trial of RN0191 at the American Heart Association’s (AHA) Annual Scientific Sessions in Chicago, IL, November 16th, 2024. RN0191 is a proprietary GalNAc conjugated PCSK9 siRNA designed to significantly lower low-density lipoprotein cholesterol (LDL-C) and other lipid parameters. The Ph1 study was a randomized, single-dose ascending, placebo-controlled study to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics in healthy subjects with elevated LDL-C. The demographics included adults aged 18 to 60 years, spanning a BMI range of 19-30 kg/m 2 . All results are based on data in the database as of Oct. 14, 2024. A total of 32 subjects were randomized and treated with RN0191 from 60mg to 600mg, respectively. The baseline LDL-C mean level was ranging from 110-130 mg/dL. After a single-dose subcutaneous injection, RN0191 showed a favorable safety profile with no serious adverse events and only mild, transient adverse events reported across all dose- levels. Dose-dependent, significant and durable changes in PCSK9, LDL-C and other lipid parameters were observed (see table below). Stella Shi, CEO of Rona Therapeutics, shared, “We are thrilled to report that RN0191 has demonstrated best-in-class PCSK9 siRNA potential. These results highlight RN0191’s potential as a transformative siRNA therapy for global patients with elevated LDL-C, a major risk factor for atherosclerotic cardiovascular disease either as single agent therapy or as combinatory backbone to improve cardiovascular outcome. “ About Rona Therapeutics Rona Therapeutics is a global leader in nucleic acid innovative drug platform company, specializing in the treatment of metabolic diseases and neurological diseases. Rona Therapeutics is always committed to developing the best and first-in-class siRNA drugs with differentiation and innovation to address unmet needs and improve outcome in cardiovascular diseases, obesity, and MASH. In addition, Rona Therapeutics is unlocking potential of extra-hepatic delivery for neurological disorders and adipose/muscle delivery or various metabolic syndromes. For Further information, Please visit: www.ronatherapeutics.com or contact: BD@ronatherapeutics.com SOURCE: Rona Therapeutics

Rona Therapeutics unveiled promising early-stage findings for its PCSK9-targeted siRNA therapy, dubbed RN0191, at the American Heart Association's (AHA) annual scientific sessions on Sunday, demonstrating notable reductions in PCSK9 and low-density lipoprotein cholesterol (LDL-C) after a single dose. The Shanghai-based biotech gained the asset securing exclusive global rights to Sanofi’s siRNA platform in 2022. Touting 'best-in-class' prospects, Rona CEO Stella Shi, said the “results highlight RN0191's potential as a transformative siRNA therapy for global patients with elevated LDL-C…either as single agent therapy or as combinatory backbone to improve cardiovascular outcome."The Phase I study randomised 32 healthy participants aged 18 to 60, to receive a single dose of RN0191 in the range of 60 mg to 600 mg or placebo. Results showed that the RN0191 group achieved up to 95% maximum reduction in PCSK9 and a 74% drop in LDL-C, with average reductions of 87% and 56%, respectively, compared with placebo. Moreover, RN0191 maintained up to 42% LDL-C reduction at six months, indicating suitability for bi-annual dosing. Additionally, RN0191 demonstrated significant reductions in ApoB, Lipoprotein(a), non-high-density lipoprotein cholesterol and total cholesterol. In terms of safety, the therapy had no serious adverse events, with only mild and short-term side effects reported at all doses.Following a $33-million Series A round in 2022, Rona secured another $35 million this year to advance RN0191 and another lipid-lowering siRNA against APOC3, named RN0361, into mid-stage testing. Besides dyslipidaemia, the company’s pipeline features a range of liver-directed siRNA therapies being developed for obesity, metabolic dysfunction-associated steatohepatitis, atherosclerotic cardiovascular disease, IgA nephropathy and hypertension.