Rare diseases and common diseases, all-round RNAi therapy start, Novo Nordisk, Roche, Novartis, Regenerative...

Read: RNAi has the potential to change the medical landscapeAlnylam published positive clinical results of its RNAi therapy Phase IIIprimary glyphual acidicin i (PH1) is a rare genetic liver disease that, in severe cases, often requires liver and kidney transplantsRecently, Alnylam Pharmaceuticals released the latest clinical data from a Phase III clinical study of its third RNA interference (RNAi) drug lumasiran for the treatment of primary hypergrassyorice (PH1)The results showed that the level of oxalic acid decreased by 65.4% in PH1 patients under treatment, and 53.5% lower than placebo, the study reached the primary endpoint84% of PH1 patients had levels of oxalic acid at or near normal levels, more than half of the patients reached normal levels, and Lumasiran had good safety and toleranceit is understood that Alnylam has submitted a new drug application (NDA) to the U.SFood and Drug Administration (FDA), which has approved the NDA's priority reviewIn addition, lumasiran's Marketing Authorization Request (MAA) has been submitted to the European Medicines Agency (EMA)two RNAi treatments have been approved for the treatment of rare diseases
only two of the world's approved RNAi therapies, all from Alnylam, have been on the market in the past two years2018, Alnylam's Onpattro (patisiran) was approved in the United States for the treatment of peripheral neuropathy (also known as multiple neuropathy) caused by hereditary transthyroidin intheminin (hATTR) in adultsThe world's first approved RNAi drug is currently approved in the United States, the European Union, Japan, Switzerland and Brazil(hATTR amyloid degeneration is a serious and fatal rare disease with a life expectancy of only 2 to 15 years from the onset of symptoms.)November 2019, the second RNAi drug givlaari (givosiran) was approved by the FDA for the treatment of acute hepatic disease (AHP) in adults and obtained a marketing license from the European Commission (EC) in March 2020(AHP is a rare genetic disease, and the majority of patients are female.) Prior to Givlaari's approval, there had been no approved treatment for the diseaseit is understood that the first RNAi product Onpattro sales reached $112.5 million in the first year of the market, and 2019 net income of $166 million Givlaari, the second RNAi product, is expected to generate sales of more than $500 million in 2025! the positive results of this Phase III clinical study, if successfully approved, it means that the world's first three RNAi drugs will be covered by Alnylam, indications are rare diseases RNAi therapy to treat for public diseases Alnylam believes RNAi therapy represents a highly modular and repeatable approach to drug discovery and development Currently, the company is developing innovative RNAi drugs in four areas: genetic diseases, heart metabolic diseases, liver infectious diseases, and central nervous system (CNS) and eye diseases , the existing partnership with VirBiotechnology has expanded to allow the development of up to six siRNAs to treat infectious diseases, including the development and commercialization of siRNAs that target highly conservative areas of coronavirus RNA photo source: Alnylam Pharmaceuticals website inclisiran is specifically targeted to the intracellular PCSK9 synthesis through RNA interference technology to help remove bad cholesterol in the blood 2019, MDCO published comprehensive clinical data on inclisiran, which showed effective and long-lasting LDL-C (LOW density lipoprotein cholesterol) reduction and good safety and tolerance in more than 3,600 high-risk patients with ASCVD (atheroscleostic cardiovascular disease) and FH (family hypercholesterolemia) In addition, inclisiran can be administered during routine treatment, twice a year, helping to improve patient compliance and consistently reduce LDL-C levels is currently under review by regulators, if successfully launched, it will mark the huge potential of RNAi technology drugs to treat mass diseases with more patients! Fierce Pharma forecast selling in Clisiran to $1.529 billion in 2024 RNAi Global Layout currently leads the industry with three ashes players in the RNAi field: Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals and Dicerna Pharmaceuticals However, with the launch of two RNAi drugs, more and more pharmaceutical companies are seeing their potential value and beginning to lay out, including pharmaceutical giants such as Novartis and Roche Arrowhead Pharmaceuticals
Arrowhead has the broadest product line, including liver disease, tumors, pulmonary cystic fibrosis, hepatitis B and cardiovascular disease, and is currently not available, with the fastest-moving project being ARO-AAT for the treatment of trypsin deficiency (AATD) Dicerna Pharmaceuticals
Dicerna also has no products on the market, and the fastest-moving project is Nedosiran, which is used to treat three types of primary hyperuric acid (PH1, PH2, PH3), the only drug in the research that can treat three PhMs simultaneously Compared to Alnylam's LUMASIRAN, which can only treat type 1 PH, there are significant clinical advantages Blackstone Blackstone Group and Alnylam Pharmaceuticals have agreed a $2 billion strategic financing partnership to accelerate the development of RNA interference (RNAi) therapeutics The financing is one of the largest private financings ever made by biotech companies Novo Nordisk November 2019 November November November November November November November November November November November November November November November November November November, Novo Nordisk and Dicerna Pharmaceuticals announced a research agreement to jointly develop RNAi therapies for the treatment of liver-related heart metabolism diseases using Dicerna's proprietary GalXCRNAi technology platform The partnership explores more than 30 liver cell targets that have the potential to provide a variety of clinical candidates for chronic liver disease, non-alcoholic fatty hepatitis (NASH), type 2 diabetes, obesity and rare diseases Novartis Novartis acquired Medicines Company for $9.7 billion in November 2019 for the innovative RNAi therapy, which was previously developed by TheMedicines in conjunction with Alnylam and is a long-acting RNAi drug in the form of siRNA-GalNAc Roche In October 2019, Roche and Dicerna Pharmaceuticals signed a research partnership and license agreement for the GalXCRNAi Technology Platform to develop and commercialize DCR-HBVS, a candidate based on that platform Regenerative dollars April 2019, Regenerative Yuan signed an $800 million cooperation agreement with Alnylam to discover RNAi drugs specifically for eye and central nervous system diseases (CNS) , Lilly has entered into a global licensing and research and development partnership with Dicerna Pharmaceuticals, And Johnson and Arrowhead have signed an ARO-HBV development and commercialization cooperation agreement, with a growing number of pharmaceutical companies looking to the RNAi field..