Dublin, Ireland-based Avadel Pharmaceuticals Plc (AVDL:NASDAQ), which focuses on developing once-nightly sodium oxybate, FT218, for narcolepsy, today announced that the U.S. Food and Drug Administration (FDA) has agreed to the firm's proposed amendments to the statistical analysis plan and protocol under its special protocol assessment agreement (SPA) for its REST-ON study. The changes will result in a lower sample size needed to demonstrate significance for both excessive daytime sleepiness and cataplexy in narcolepsy patients. In the report the company advised that there were no modifications made to the fundamental design of the study, including the primary or secondary endpoints, dosing scheme or duration of the study, and the SPA remains intact.
The company states that the REST-ON study will now target enrolling 205 patients. Based on this updated target sample size and enrollment currently at 193 patients (94%), the company now expects to complete enrollment by the end of 2019 and have topline data in the second quarter of 2020. This is up to a year ahead of expectations to complete enrollment for the previous target of 264 patients for the study. The firm notes that even with this change, the REST-ON clinical trial remains one of the largest studies conducted to date for this indication.
Avadel's CEO Greg Divis commented, "This REST-ON clinical trial update is a direct result of an overall strategic review of the entire FT218 program. The addition of our recently appointed medical and clinical team members was instrumental in this important development and their contributions have put us on track to save significant time, resources and capital in the completion of the REST-ON clinical trial."
The firm explains that the REST-ON study is a double-blind, randomized, placebo-controlled Phase 3 trial to assess the efficacy and safety of once-nightly FT218, a formulation of sodium oxybate using Avadel's proprietary Micropump technology for extended-release oral suspension in the treatment of excessive daytime sleepiness and cataplexy in patients suffering from narcolepsy.
According to the company, dosed once nightly, FT218 has been granted Orphan Drug Designation from the FDA for the treatment of narcolepsy. The designation was granted on the plausible hypothesis that once-nightly FT218 may be clinically superior to a formulation of sodium oxybate that is already approved by the FDA for the same indication. In particular, once-nightly FT218 may be safer due to ramifications associated with the dosing regimen of the previously approved product. The twice-nightly sodium oxybate market is currently valued at an estimated annualized rate of $1.6 billion. Avadel's market research leads it to believe that FT218, if approved by the FDA, has the potential to take a significant share of this market.
Avadel Pharmaceuticals indicates that it is a branded specialty pharmaceutical company committed to providing solutions for overlooked and unmet medical needs through patient-focused, innovative products. The company's primary focus is on the development and potential FDA approval for FT218. Additionally, Avadel develops and markets a portfolio of sterile injectable drugs used in the hospital setting.
Avadel started the day with a market cap of approximately $126 million and a short interest of about 18.5%. AVD shares opened today at $4.35 (+$0.98, +29.08%) over Friday's closing price of $3.37. The stock has traded today on much higher than average volume between $4.13 and $5.37/share and currently is trading at $4.47 (+$1.10, +32.64%).[NLINSERT]
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