2. Amicus Therapeutics, Inc. (NASDAQ:FOLD)
Number of Hedge Fund Holders In Q2 2024: 31
Average Analyst Share Price Target: $17.36
Average Analyst Share Price Target Upside: 55%
Amicus Therapeutics, Inc. (NASDAQ:FOLD) is a commercial stage biotechnology company. It develops treatments for Fabry disease and late onset Pompe disease. Despite the fact that it launched its Pompe treatment earlier this year, in a development that is typically beneficial for biotechnology stocks, Amicus Therapeutics, Inc. (NASDAQ:FOLD)’s shares are down by 52% year to date. Some of this is based on the fact that the Pompe treatment was soon met with a competitor called Nexviazyme. This drug is manufactured by Sanofi, which has considerably larger resources than Amicus Therapeutics, Inc. (NASDAQ:FOLD) and stands to better target the market through marketing and supply ramp. Subsequently, the key to the firm’s hypothesis is market surveys that indicate physician opinions on Pompe treatment. Additionally, Amicus Therapeutics, Inc. (NASDAQ:FOLD) also has to ensure strong performance of its Fabry disease treatment called Galafold especially since the treatment was launched eight years ago.
Amicus Therapeutics, Inc. (NASDAQ:FOLD)’s management shared details for its Pompe treatment during the Q2 2024 earnings call:
“Second, let me highlight the continued strong global commercial launch of Pombiliti and Opfolda, our novel therapy for late-onset Pompe disease.
Pombiliti and Opfolda has been and will continue to be a huge growth driver for us this year. We’ve already made great progress against our key performance indicators, which continue to demonstrate the strength of this launch. First and foremost, our number one focus for the year is to maximize the number of patients on therapy by year end. It’s great to report that the rate of new commercial patients coming on Pombiliti and Opfolda in 2024 continues to progress exceptionally well. In the second quarter, we saw the largest number of new commercial patients, meaning patients who were not in our clinical studies. And as of the end of July, we had 186 patients who had been treated or scheduled for treatment. We are incredibly pleased with the demand globally from patients and physicians from this new therapy, and consistently hear inspiring anecdotes from healthcare professionals around the world on how their patients are responding to Pombiliti and Opfolda, which will continue to fuel the momentum throughout the year and beyond.
Sebastien will provide more details in a moment, but the switch dynamics in the U.S., Europe and the U.K. continue to look strong, and we’re seeing great uptake in naïve patients and markets outside the U.S. as well. We’re also making significant progress on the reimbursement front globally and this includes moving patients more quickly through the insurance process in the U.S. as we’ve anticipated. Throughout the remainder of the year, we’ll focus on increasing patient access as we gain reimbursement and launch in additional countries throughout Europe. On the regulatory front, we’re pleased to announce that in July, the Swissmedic approved Pombiliti and Opfolda for adults living with late-onset Pompe disease in Switzerland. For the full year 2024, we’re well on track to deliver our guidance of $62 million to $57 million in global Pombiliti and Opfolda sales, which will be a significant contributor to our growth and set us on a great course to achieve our ambition for Pom-Opf to become the new standard of care treatment in this devastating disease.”
