Together, these results contributed to the validation of PCSK9-targeting siRNAs for development as a new therapeutic for use in LDL cholesterol–lowering therapy; specifically, small interfering RNA (siRNA) molecules of appropriate sequence and structure were sought and discovered as a means of decreasing PCSK9 levels, and the development of a clinical candidate ensued thereafter. Clinical study history
In 2019, The Medicines Company announced positive results from pivotal phase III study (all primary and secondary endpoints were met with efficacy consistent with Phase I and II studies). The company anticipated regulatory submissions in the U.S. in the fourth quarter of 2019, and in Europe in the first quarter of 2020. Inclisiran is being developed by The Medicines Company, a subsidiary of Novartis, which licensed the rights to inclisiran from Alnylam Pharmaceuticals.The effectiveness of inclisiran was studied in three randomized, double-blind, placebo-controlled trials that enrolled 3,457 adults with HeFH or clinical ASCVD. Enrolled participants were taking maximally tolerated statin therapy but required additional LDL-C lowering based on their risk for cardiovascular events. In all three studies, the main effectiveness outcome measure was the percent change in LDL-C from the beginning of the trial to day 510 (month 17). In each trial, participants received under-the-skin injections of either 284 mg inclisiran or a placebo on four separate days: day 1, day 90 (month 3), day 270 (month 9), and day 450 (month 15).Study 1 enrolled 1,561 adults with ASCVD.
When the patient began to relapse/remit, they were treated again, resulting in improvement. After this, the patient remained stable for roughly 7 months before suffering their third relapse/remit.This therapy may be a therapy option for patients with malignant MS.

See also
Tumefactive multiple sclerosis
Marburg acute multiple sclerosis
Multiple sclerosis
Autologous stem cell transplantation


== References ==