Roche & Ionis early Huntington's disease test results positive
March 02, 2018 Source: Sina Pharmaceutical
Window._bd_share_config={ "common":{ "bdSnsKey":{ },"bdText":"","bdMini":"2","bdMiniList":false,"bdPic":"","bdStyle":" 0","bdSize":"16"},"share":{ }};with(document)0[(getElementsByTagName('head')[0]||body).appendChild(createElement('script')) .src='http://bdimg.share.baidu.com/static/api/js/share.js?v=89860593.js?cdnversion='+~(-new Date()/36e5)];Recently, Ionis and partner Roche announced biomarker data for an I/II study that drove pharmaceutical giant Roche to continue paying $45 million in option fees to IONIS-HTT Rx (RG6042).
The researchers said on Thursday night that they found that patients who used the therapy had an average 40% reduction in mutated Huntingtin after receiving the highest dose, and some patients had a 60% reduction. And they added that achieving this effect is the first time for any Huntington drug. It is important to understand that this toxic protein causes damage to neurons in the brains of Huntington's patients, causing severe neurodegenerative diseases. The main symptoms are decreased muscle coordination and cognitive ability, which usually causes patients to be at 20 Slowly died during the year.
If the efficacy of the drug is finally confirmed, the researchers may have discovered the world's first potential drug that could alter the disease process, although some of the safety and efficacy hurdles remain to be clarified.
The team added that dose-dependent responses showed that levels of mutant proteins continued to decline after the last test, which also increased their expectations for better outcomes. The open label extension for this study is ongoing. Roche now chooses to participate in the research and development of drugs and is responsible for conducting key tests.
IONIS-HTTRx, jointly developed by Ionis and Roche, is an innovative antisense antisense therapy targeting HTT proteins. By binding to mRNA encoding the HTT protein, IONIS-HTTRx is able to reduce the occurrence of all HTT protein translations, so the drug can reduce the production of toxic mHTT regardless of where the gene mutation encoding HTT occurs. It has been granted orphan drug status by the US FDA and the European Medicines Agency.
Ionis' recent performance has been rising due to the launch of the spinal muscular atrophy (SMA) drug SPINRAZA. The biotech company is also under review for two drugs, including Inostersen, which is believed to be threatened by the Alnylam competition drug Patisiran.
Sarah Tabrizi, professor of clinical neurology at the University of London's Huntington's Disease Center and director of global research, said, "In the past 20 years, I have seen many families suffer losses from this progressive neurodegenerative disease. With IONIS-HTTRx, Huntington The patient has new hopes for treating the disease, can reduce the cause of HD, and therefore may slow down the progress and possibly prevent the disease of the offspring. This is truly groundbreaking progress. I am looking forward to a long-term, larger scale. Studies to determine the benefits of reducing the toxic mutant Huntington's protein in Huntington's patients." (Sina Medical Compilation / Fan Dongdong)
Article reference source: Roche, Ionis herald a big win in early-stage Huntington's disease trial, demonstrate disease modifying potential
Tetanus Vaccine,Hepatitis B Vaccine For Adults,Tetanus Booster,Td Vaccine
FOSHAN PHARMA CO., LTD. , https://www.foshanmedicine.com