There is no doubt that Amicus Therapeutics, Inc. (NASDAQ:FOLD) is on its best foot this year. Notably, the company offered its opinion on the coming quarters as well as a recap of the year just ended. Looking at the items the firm crossed of its list, it is evident that their growth strategy is robust.
Amicus accomplished all significant objectives for 2018
In particular, Amicus reported revenue for the full year 2018 as having surpassed guidance. Notably, the revenue improvement primarily comes from Galafold, a proprietary drug. Interestingly, revenue from sales of Galafold clocked $91 million for that period from a mere $36.9 million the previous year. This is also against the 2018 full year guidance of between $80 and $90 million. Although these are preliminary and therefore unaudited figures, they still represent a great effort on the part of Amicus. According to the firm, the revenue score meets one of its primary objectives for FY2019.
In the same year, Amicus achieved its objective of having Galafold approved in Japan and the U.S. By the end of the year; Amicus reported that 149 patients across the U.S. received Galafold prescription. Notably, the firm was also able to maintain a very healthy balance sheet with $505 million cash available for short-term commitments.
Substantial successes in CLN3 trials
Another crucial highlight from the year just ended is that Amicus expanded its gene therapy pipeline to 14. Notably, trials for a vital drug candidate for the treatment of CLN3 Batten disease are in Phase 1/2. According to Amicus’ Chief Medical Officer, Jay Barth, MD, “the first child has been dosed in the Phase 1/2 study for CLN3 Batten disease.”
The CLN3 Batten disease is a rare inherited disorder that affects the victim’s nervous system. Also known as neuronal ceroid lipofuscinoses (NCLs), the disease mostly begins in early childhood and is fatal. Interestingly, Amicus reported that initial trials on a patient showed positive results. Up to date, the firm does not see any adverse events that are worth reporting.
According to Dr. Barth, achievement of the positive results is no mean feat. Notably, Amicus is the pioneering entity to go deep into the study of the rare condition. This is to say that the resulting treatment will be the first of its kind in the pharmaceutical industry.