RN-0191

Single dose of RN0191 achieved up to 95% individual maximum and 87% mean maximum PCSK9 reduction, and up to 74% individual maximum and 56% mean maximal LDL-C lowering Robust and sustainable treatment effects support at least bi-annual dosing regimen Best-in-class potential of novel PCSK9 siRNA RN0191 supports further development as single agent or combination to further reduce atherosclerosis cardiovascular risk SHANGHAI, Nov. 18, 2024 /PRNewswire/ -- 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/m2. 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). Mean maximum reduction of PCSK9 >85% and LDL-C >55% have been achieved. Significant and durable LDL-C reduction is achieved up to 42% through Day180, supporting a bi-annual dosing regimen for future development Significant lowering of ApoB, Lp(a), non-HDL-C and total cholesterol are also noted Remarkable treatment effects with reduction of PCSK9, LDL-C and the other lipid parameters maintained to 6 months. 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: or contact: [email protected] WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Financing led by LongRiver Investments with participation by new and existing global investment funds Rona will use proceeds to progress leading metabolic siRNA pipeline programs into global development and expand extra-hepatic delivery platform in CNS and beyond SHANGHAI, July 19, 2024 /PRNewswire/ -- Rona Therapeutics, a clinical stage pioneering platform company in nucleic acid drug research and development, today announced the completion of $35 Million Series A+ financing. This round was led by LongRiver Investments, with participation from investors including Zhaode Investment, BioTrack Capital, Zhongqi Capital and Lilly Asia Ventures. LongRiver Investments will join the board of Rona in connection with the Series A+ financing, commented, "Rona Therapeutics has successfully brought two best-in-class siRNA metabolic programs into global development and demonstrated differentiated clinical data and platform value. We are impressed by the progress Rona has made in developing innovative siRNA pipeline and expanding into extrahepatic delivery in CNS and beyond. We look forward to partnering with Rona team to unlock potential of RNA medicine as transformative therapies to patients globally." Ms. Stella SHI, CEO and Founder of Rona Therapeutics, commented, "We are thrilled to welcome LongRiver Investments and our other new investors as we progress more best-in-class and first-in-class siRNA programs into global development and catalyzing potential business development opportunities. Rona Therapeutics remains committed to pushing boundaries of RNA medicine in metabolic, obesity, renal and neuro degenerative diseases with Rona's proprietary RNA platform." Rona is advancing a pipeline of best and first-in-class siRNA programs generated from next generation siRNA platform. The company's first program RN0191, a best-in-class PCSK9 siRNA program, has completed Australia and China Ph1 clinical studies and ready for Ph2 clinical development for hypercholesterolemia. RN0361 is the company's second program to enter clinic in 2024Q2 for hypertriglyceridemia. The company is also advancing innovative pipeline at IND enabling stage for kidney disease, obesity and cardiometabolic diseases. About Rona Therapeutics Rona Therapeutics is a global leader in nucleic acid innovative drug platform company, specializing in the treatment of metabolic diseases and CNS degenerative diseases. Rona Therapeutics is always committed to developing the best and first-in-class siRNA drugs, and has unique differentiation and innovation in its liver-targeted metabolism-related pipeline products in cardiovascular diseases, MASH, obesity, and kidney diseases. In addition, Rona Therapeutics is focusing on the potential of extra-hepatic nucleic acid delivery in CNS degenerative diseases and has successfully established a proprietary extra-hepatic delivery platform to treat a range of complex diseases, such as ALS, Alzheimer's disease, etc. which are difficult to treat with traditional drug therapies. About LongRiver Investments LongRiver Investments is a forward-thinking venture capital firm dedicated to empowering entrepreneurs and driving technological and industrial advancements. With a robust investment strategy and a commitment to excellence, LongRiver Investments aims to deliver superior returns to its investors while making a positive impact on the global market. For Further information, Please visit: or contact: [email protected]