Exciting updates have come out of Alnylam Pharmaceuticals. They just released data from one of their highly anticipated trials for its leading rare disease drug.
It looks like it did way better than its competitor’s drug which was made for the same disorder. The pharmaceuticals company added more than $1.5 billion to its market cap, as a result.
The trial was at its final Phase 3 for a drug called patisiran, which is being made to combat the rare disease called amyloidosis.
Amyloidosis occurs when abnormal proteins called amyloids gather and create massive deposits that clog vital organs like the kidney and the heart. As a result, the organs get incredibly stuffed and are unable to perform their basic motions, causing a number of problems. The disease currently affects 10,000 people. To learn more, click here.
In a September report, the company said that the study had exceeded their expectations and passed its goals. Now, it has shown overall positive results with patients, who have seen a degree of improvements on a neurological level when compared to a placebo.
Alnylam created this new drug in conjunction with Cambridge-based Sanofi Genzyme. It harnesses a unique cutting edge technology called RNA interference that dissolves genes that are in the business of creating diseases. There haven’t been any RNA treatments in the U.S. that have been approved which is why this has become a closely watched trial.
While all of this was occurring, another pharmaceutical company based out of California named Ionis Pharmaceuticals reported that its similar drug made for the same disease was performing exceptionally well during its late stage trial, but it failed to perform as well as that of Alnylm.
As of Thursday, Ainylam shares bumped up 11 percent which vastly increased the company’s market cap starting at $11.1 billion to $12.7 billion.
Head of research and development for Alnylam told the Boston Business Journal:
“The complete study results were a landmark achievement for the field of RNAi therapeutics, as we believe they demonstrate the transformational potential of this novel class of innovative medicines.”
