Amicus (FOLD) announced the failure of its topical wound-healing product SD-101 for patients with epidermolysis bullosa (EB). The drug failure was revealed when top-line results from the randomized, double-blind, placebo-controlled Phase 3 clinical ESSENCE study SD-101 were announced.
The results showed that the drug did not demonstrate a statistically significant difference from placebo in the intent to treat (ITT) population. The first primary endpoint results were almost the same for the SD-101 product and the placebo, with the same to be said about the second primary endpoint and the secondary endpoints. endpoints. Encouraging trends in wound closure, however, were observed in certain sub-populations. We see no reason at this time to dwell on the adverse effects.
Epidermolysis bullosa (EB) is a rare genetic disorder that leads to severe skin blistering and open wounds that could show at birth. The Blistering can also occur in the mucous membrane of the mouth or of the intestines.
Amicus Therapeutics’ SD-101 drug’s failure was not followed by a selloff of the firm’s stock as usually happens when a late phase trial drug fails. The stock declined momentarily only in premarket hours, losing around 15% of its value. After the start of the trading hours, the stock recuperated its losses and closed at $13.52 UP $0.24 in midday.
Showing encouraging trends in a subpopulation of patients with EB did not change Amicus’ decision to stop the drug’s development.
The climbing in the stock price following the negative news did not surprise us, as it did not surprise many analysts who believed that the value of Amicus pipeline exceeds, by far the value of its market Cap.
What Kept Shareholders from Selling
Recent Great News
In mid-August, i.e., last month, the Australian Therapeutic Goods Administration (TGA) approved the oral precision medicine Galafold for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease and who have an amenable mutation. Amicus estimates that approximately 35%-50% of Fabry patients in Australia have an amenable mutation. The Australian approval of Galafold is not the first. The product was initially approved in Europe, including Switzerland and Israel, which also accepts the EU approval as the basis for submission and review.
More Recent Good News
In 2016, the FDA asked Amicus to conduct new clinical trials before submitting its NDA for the approval of its drug migalastat (the name of Galafold in the U.S.). In July 2017, the FDA reversed its decision, which opened the door wide for Amicus to seek regulatory approval for migalastat this year. The good news is that Amicus has prepared a New Drug Application for Fabry drug that could provide for accelerated approval. Amicus has based its NDA on existing data, including a reduction in the disease-causing substrate (GL-3), as well as the totality of data from completed clinical studies.
At the time, Jay Barth, Amicus’ chief medical officer, expressed its conviction that the firm has a robust data package for the NDA for the approval of migalastat. The approval will be a great event and a game changer in the life of this struggling firm.
An Interesting Story
On Rare Disease Day, President Trump referred to the hard work that John Crowley who is currently the CEO of Amicus has done developing of a treatment for Pompe disease when he established Novazyme Pharmaceuticals to save his two children who fell victims of this disease. President Trump also denounced the slow approval process at the FDA that prevents treatments from reaching the patients who could benefit from them. It is interesting to read the biography of Mr. John Francis Crowley.
Galafold will have to compete with Shire (SHPGY) drug replagel in the EU, However, the drug has a different mechanism for action, which means Galafold that it has the potential to be used alongside Shire’s drug.
Amicus Therapeutics has solid science and sound technologies. They have prominent researchers with publications about their science and achievements; a pipeline expected to have a near-term catalyst, i.e. the FDA acceptance of the NDA and then the approval in the U.S. of Galafold for Fabry disease. Short-term and long term catalysts will be related to trial results with positive data of the firm’s product ATB200/AT2221 from trials for Pompe disease, followed by mid-phase trial results and then by the approval of the drug.
The most important and near-term event now is the expected revenue to be generated from the countries that have already approved Galafold where the Amicus’ product for Fabry disease has been already approved.
The firm’s technology has been upgraded in all the investigational products.
Stock Symbol: FOLD
Market Cap.: $13.52 UP $024 on the day of the bad news
52-week Range: $4.40 — $14.37
|Percentage||Shares Held By|
|109.02%||Float held by institutions|
|215||Number of institutions holding shares|
Recognizing the increasing speed of the bio-revolution, Amicus can continue to bring more sophisticated products to the market of rare diseases as well as for common diseases.
For all of the above-mentioned reasons, we expect that investing in Amicus at this stage will be rewarding
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