Alnylam Pharmaceuticals Rallied 32.7% in September.
RNAi treatement is a kind of gene suppression.
Alnlyam Pharmaceuticals (NASDAQ:ALNY) focuses on RNAi treatments. RNAi interference is used in research into immune respone to viruses and other foreign genetic material.
Alnlyam announced encouraging phase 3 clinical research results for an important pipeline drug. As a result, shares of Alnlyam gained 33% in September.
September began in a very negative way for Alnylam. The company halted all clinical development on Fitusiran. This is a treatment candidate for the blood-clotting disorder hemophilia. A patient died during one of its trials.
However, a few days later Alnylam and its partner Sanofi (NYSE:SNY) released phase 3 clinical data on a drug called patisiran. This is a treatment for hereditary ATTR amyloidosis (hATTR) with polyneuropathy. This is a rare disease which affects about 50,000 people worldwide. These patients currently have limited treatment options.
While patisiran looks promising, it is important for investors to realize that the drug won’t likely enjoy monopoly status. Ionis Pharmaceuticals (NASDAQ:IONS) has a drug called inotersen that they will submit for FDA approval later this year. This drug also represents a hopeful treatment for the same disease state. Patisiran’s safety data is expected to give it an edge over inotersen. Nevertheless, it would be a mistake to ignore the potential competition completely.
Most noteworthy, if patisiran wins approval, it will still likely bring in hundreds of millions of dollars in revenue per year.