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Corporate News Blog - Amicus Therapeutics Reports Phase-3 Results for Sd-101 in Epidermolysis Bullosa did not Meet Primary Endpoints

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LONDON, UK / ACCESSWIRE / September 15, 2017 / Pro-Trader Daily takes a look at the latest corporate events and news making the headlines for Amicus Therapeutics, Inc. (NASDAQ: FOLD), following which we have published a free report that can be viewed by signing up at http://protraderdaily.com/optin/?symbol=FOLD. The Company announced on September 13, 2017, the top-line data from the randomized placebo-controlled Phase-3 clinical study, i.e. ESSENCE, SD-005. The study was conducted to evaluate the efficacy and safety of the new topical wound-healing agent SD-101. The ESSENCE study concluded that SD-101 did not meet the primary endpoints or secondary endpoints in participants with EpidermolysisBullosa (EB). For immediate access to our complimentary reports, including today's coverage, register for free now at:

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No Proper Treatment of EB As Yet

Epidermolysis Bullosa is a rare, devastating genetic disorder, which affects thousands of children and adults throughout their lifetimes. It is chronic, potentially disfiguring, and in some cases even fatal. Although there are several genetic and symptomatic variations of EB, but all forms share the common symptom of fragile skin that blisters and tears, sometimes from the slightest friction or trauma. These often begin at birth itself.

Currently, there are no approved therapies for the treatment of Epidermolysis Bullosa. The current standard of care comprises only pain management and the bandaging and cleaning of open wounds to prevent infection.

ESSENCE Study Design

ESSENCE (SD-005) is a randomized, multicenter, double blind, placebo-controlled Phase-3 study in patients with a documented diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB. However, for the study entry, target wounds were required to be chronic, i.e. at least 21 days old and between 10 and 50 square cm in size. For the study, a total of 169 patients were randomized on a 1:1 basis to SD-101 treatment (n=82) or placebo (n=87) for a three-month primary treatment period. This was followed by a continuing open-label extension study (SD-006) in which all patients receive SD-101 treatment.

Study did not Meet Primary Endpoints

  • SD-101 did not show a statistically significant difference from placebo in the intent to treat (ITT) population (n=169).
  • The first primary endpoint, which was the time to target wound closure within 3 months, was not different between groups (Hazard ratio=1.004, p=0.985).
  • The second primary endpoint that is the percentage of patients achieving target wound closure by month 3. It was also not distinct between groups (49% SD-101; 54% placebo; p=0.390). Likewise, the secondary endpoints did not reach statistical significance versus placebo.
  • Nevertheless, some encouraging trends were reported in wound closure for certain sub-populations.
  • Moreover, treatment-emergent adverse events (TEAEs) were analogous across both the SD-101 and placebo groups. Common TEAEs include nasopharyngitis (common cold), pruritis (itchy skin), and pyrexia (fever).

Comments from Amicus Chairman

John F. Crowley, Chairman and Chief Executive Officer at Amicus Therapeutics, shared his disappointment on Phase-3 study of SD-101 not meeting the primary endpoints in EB, which is a distressing rare genetic disorder with no approved treatment options.

He highlighted Amicus' strong commitment to the EB community and mentioned that the Company would continue to work closely with investigators and other leading experts to understand and to share these data. He extended a sincere acknowledgment for the patients, families, clinical investigators, regulators, and the Amicus team involved in this EB program. In trying to develop new, high-quality therapies for people living with devastating diseases, Amicus might have failed. But it believes it is better to be the first to fail than be the last to try.

He reiterated that the vision of Amicus remains steadfast and it is dedicated to building a leading global biotechnology company that delivers noteworthy benefits for people living with rare diseases.

Further Analysis and Discussion of Phase 3 ESSENCE Results

Amicus intends to further analyze and share the Phase-3 ESSENCE results with key stakeholders in the EB community such as physicians, patient organizations, and regulators. In the meanwhile, in consultation with their physicians, the ongoing extension studies (SD-004 and -006) will continue being treated with SD-101. However, the Company currently has no plans for investing in any additional clinical studies or commercial preparation activities for SD-101

About Amicus Therapeutics

Amicus Therapeutics is a biotechnology Company which is always at the forefront of therapies for rare and orphan diseases. The Company has a strong pipeline of advanced therapies for a broad range of human genetic diseases. Its lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases.

Last Close Stock Review

On Thursday, September 14, 2017, the stock closed the trading session at $13.42, slightly down 0.74% from its previous closing price of $13.52. A total volume of 3.41 million shares have exchanged hands. Amicus Therapeutics' stock price soared 52.67% in the last three months, 88.48% in the past six months, and 93.37% in the previous twelve months. Furthermore, since the start of the year, shares of the Company have skyrocketed 170.02%. At Thursday's closing price, the stock's net capitalization stands at $2.22 billion.

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