New deal aims to bring edaravone for ALS to Australia, New Zealand
Treeway partners with ST Asia on therapy, sold in US as Radicava ORS
Treeway has chosen Specialised Therapeutics Asia, known as ST, as its partner to market TW001, an oral formulation of edaravone, for the treatment of amyotrophic lateral sclerosis (ALS) in Australia and New Zealand.
Under the licensing deal, ST will be responsible for marketing and distributing TW001 — sold in the U.S. as Radicava ORS — in this region of the Southern hemisphere. The regulatory submission of the edaravone therapy for ALS, a form of motor neurone disease (MND), also will be handled by ST.
“ST is [focusing] on bringing new therapies to the market for diseases with a high medical need and therefore ST fits well as a licensing partner for Treeway,” Inez de Greef, CEO of Treeway, said in a ST press release.
TW001, also known as FNP122, is an oral formulation of edaravone designed to protect nerve cells by targeting unstable molecules called reactive oxygen species and making them harmless. An abnormal buildup of these molecules can damage nerve cells and contribute to the neurodegeneration seen in ALS.
Available in the U.S. as Radicava, which is infused into the bloodstream, and as Radicava ORS, an oral suspension taken by mouth or via a feeding tube, edaravone is not approved in Europe for treating ALS. Its intravenous or into-the-vein formulation was approved in Australia earlier this year.
Phase 3 trial underway to test TW001 edaravone therapy in early ALS
Evidence that Radicava slows the progression of ALS is conflicting and it is possible that Radicava may not work well for everyone. Moreover, it’s given on an on-and-off administration schedule, which may present some challenges to patients.
“Better disease modifying therapies are urgently needed to slow and potentially halt this disease,” said Susan Mathers, MD, a neurologist at Monash Health and assistant professor at Monash University’s School of Clinical Sciences, in Australia.
TW001 is another formulation of edaravone that’s well tolerated and has a higher availability than Radicava for ALS — suggesting similar effects may be achieved with lower doses of the medication.
It originally was developed by Treeway and is being advanced through clinical testing by Ferrer, which acquired the rights to develop and market the medication in Europe and certain Asian territories.
Ferrer now is conducting a Phase 3 study, dubbed ADORE (NCT05178810), that is testing TW001 against a placebo in approximately 300 people with early ALS. For this study, patients’ symptoms must have started within the previous two years.
The participants, all adults, were enrolled at nearly 40 sites across Europe. Each was randomly assigned, in a 2-to-1 ratio, to receive a once-daily oral dose of 100 mg TW001 or the placebo, for 48 weeks, or nearly one year.
The main goal is to evaluate 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 will be investigated.
We look forward to further results from the ADORE study and then working with ST to make our therapy available for all eligible patients in Australia and New Zealand who may benefit.
Participants completing ADORE — including those who discontinued the medication for other than safety issues but remained on the trial — have the option to enter an open-label extension study in which all will receive TW001. That extension, called ADOREXT (NCT05866926), will provide patient dosing up to three years.
“We look forward to further results from the ADORE study and then working with ST to make our therapy available for all eligible patients in Australia and New Zealand who may benefit,” de Greef said.
As an oral formulation, TW001 could be taken at home, making it easier for patients to manage their symptoms.
“Oral therapies like edaravone present the opportunity for a simple to manage therapy which can be taken at home and monitored through each person’s local health care provider,” Mathers said.
MND Australia, a patient advocacy organization, noted that TW001 could provide another treatment option for ALS patients in the country.
“Oral edaravone would complement the recent approval of intravenous edaravone in Australia and broaden the patient base able to access treatment,” said Gethin Thomas, PhD, MND Australia’s executive director of research.