FNP122, edaravone in oral form, enters Phase 3 extension trial
ADOREXT study in Europe to determine therapy's long-term safety
A Phase 3 clinical trial that’s testing FNP122, Ferrer’s oral formulation of edaravone, in people with amyotrophic lateral sclerosis (ALS) is now rolling participants into its open-label extension study.
The ADORE trial (NCT05178810) is testing FNP122 against a placebo in about 300 patients across Europe whose symptoms started less than two years earlier. Those who completed the trial’s 48 weeks now have the opportunity to join an extension study called ADOREXT (NCT05866926), where all will receive FNP122 for up to three years. The goal is to assess the treatment’s long-term safety.
“The ADOREXT study allows the company’s research team to continue in its aim to bring significant and differential value to people suffering from serious diseases such as ALS,” Tatjana Naranda, Ferrer’s chief research and development officer, said in a company press release.
Edaravone, as the oral treatment Radicava ORS, is not available in Europe
Edaravone is an ALS treatment that works to slow disease progression by reducing oxidative damage to cells. Oxidative stress, an imbalance between the production of potentially harmful free radicals and a cell’s ability to detoxify them, is believed to be a key driver of nerve cell death in the disease.
The medication is currently approved in the U.S. in two distinct formulations: Radicava, which is administered through intravenous (into-the-vein) infusions, and Radicava ORS, which is an oral suspension taken by mouth or via a feeding tube.
Both are given in 28-day cycles. After an initial cycle of treatment for 14 consecutive days, followed by a two-week period without treatment, the therapy is taken daily over 10 of the next 14 days, followed by two weeks of no treatment.
While the two formulation can safely and effectively slow functional decline in ALS patients, the administration schedule may pose some challenges. These Radicava formulations also are not approved in the European Union, where Ferrer is based.
In a previous Phase 1 trial, FNP122 was reported to be well tolerated and to have a higher bioavailability — a measure of how much of the medication is distributed in the body to reach its target areas — than Radicava.
The ADORE trial is ongoing at more than three dozens of clinical sites across Europe to assess FNP122’s safety and efficacy in adults, ages 18 to 80, with ALS. Participants were randomly assigned in a 2-to-1 ratio to the edaravone formulation, a daily oral dose of 100 mg, or to a placebo, for 48 weeks (nearly one year).
Its main goal is changes with treatment in a patient’s ability to perform daily activities, as measured with the ALS Functional Rating Scale – Revised (ALSFRS-R). The medication’s effects on survival also are being determined.
TRICALS also supporting the long-term ADOREXT study
All patients completing ADORE’s 48 weeks, including those who stopped treatment for other than safety reasons but remained on the trial, are invited to join the extension study.
According to Ferrer, enrollment for ADOREXT began in March, and several patients across nine countries — Spain, France, Italy, Belgium, Germany, the Netherlands, Poland, Ireland, and Sweden — are now in the extension. Participants from other European countries are expected to join next.
Both trials are being supported by the Treatment Research Initiative to Cure ALS (TRICALS), the largest European research initiative working to find a cure for ALS.
The protocol for the extension study was developed taking into consideration patients’ needs and perspectives, which were collected in an ALS Patient Representatives Advisory Board (PAB), Ferrer reported. Eight patient organizations from Europe and North America participated in the advisory board.