Alnylam Pharmaceuticals draws attention after rival trial failure shifts ATTR CM expectations

Arcturus Therapeutics Holdings Inc. announced it will host a virtual presentation on Wednesday, September 23, 2026, at 4:30 p.m. ET covering the ARCT-810 Phase 2 clinical program for Ornithine transcarbamylase deficiency and the company's mRNA liver therapeutics platform. The company will issue a press release summarizing the presentation prior to the call. Marshall Summar, M.D., a founding member and Executive Committee member of the NIH UCD Consortium and a recognized expert in rare diseases and OTC deficiency, will participate in the presentation. ARCT-810 is an intravenously administered investigational mRNA therapeutic designed to express normal functional OTC enzyme in the liver of individuals with OTC deficiency, and it holds Orphan Medicinal Product Designation and an approved pediatric investigation plan from the European Medicines Agency, plus Orphan Drug Designation, Fast Track Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration. In Europe and the U.S., approximately 10,000 people have OTC deficiency.

On September 18, the Ministry of Industry and Information Technology, the National Development and Reform Commission, and eight other ministries jointly released the 15th Five-Year Plan for the Development of the Pharmaceutical Industry, proposing that by 2030, biopharmaceutical R&D and application will rank among the world's leaders, and the biopharmaceutical industry will accelerate its rise as a national emerging pillar industry. The plan sets 10 expected indicators covering industrial scale and efficiency, innovative development, enterprise cultivation, and cluster development, including operating revenue of pharmaceutical industrial enterprises above designated size exceeding 3.5 trillion yuan, an average annual growth rate of the innovative drug industry scale exceeding 20 percent, more than 5 products with global annual sales exceeding 1 billion US dollars, first-in-class innovative drugs accounting for more than 25 percent of the global total, more than 200 innovative medical devices launched, 50 pharmaceutical industrial enterprises with annual operating revenue exceeding 10 billion yuan, and 20 pharmaceutical industrial parks at the 100-billion-yuan level. Boosted by this news, the three major stock indices all rose more than 1 percent in early trading that day. Huatian Technology hit the daily limit up, with turnover of 6.065 billion yuan, nearly 1.47 million lots of sealed buy orders on the limit-up board, and nearly 3.3 billion yuan of main capital rushing in, ranking first in the A-share market. Zhou Sicong, fund manager of Ping An Pharmaceutical Select, believes innovative drugs are likely to become an important growth theme, and CITIC Securities said China's innovative drugs have entered a stage of global value realization. According to statistics from Securities Times Data Treasure, since September, Chengdu Leading Pharmaceutical, Asymchem Laboratories, Porton Pharma Solutions, and Hunan Warrant Pharmaceutical have led gains, all rising more than 10 percent, with Chengdu Leading Pharmaceutical up a cumulative 16.87 percent.