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Note: This story was updated Sept. 25, 2023, to correct that TW001 uses the same active ingredient as Radicava and Radicava ORS but is a different therapy.
<a href="https://treeway.nl">Treeway</a> has chosen <a href="https://stabiopharma.com">Specialised Therapeutics Asia</a>, known as ST, as its partner to market TW001, an oral formulation of edaravone, for the treatment of <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) in Australia and New Zealand.
Under the licensing deal, ST will be responsible for marketing and distributing TW001 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 <a href="https://www.prnewswire.com/news-releases/specialised-therapeutics-acquires-commercialisation-rights-to-new-oral-mnd-therapy-301909816.html">ST press release</a>.
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.
Edaravone is available in the U.S. as <a class="c-link" href="https://alsnewstoday.com/radicava-edaravone-for-als/" target="_blank" rel="noopener noreferrer" data-stringify-link="https://alsnewstoday.com/radicava-edaravone-for-als/" data-sk="tooltip_parent">Radicava</a>, which is infused into the bloodstream, and as Radicava ORS, an oral suspension taken by mouth or via a feeding tube, but is not approved in Europe for treating ALS. Its intravenous, or into-the-vein, formulation was <a class="c-link" href="https://www.tga.gov.au/resources/auspmd/radicava" target="_blank" rel="noopener noreferrer" data-stringify-link="https://www.tga.gov.au/resources/auspmd/radicava" data-sk="tooltip_parent">approved in Australia</a> earlier this year.
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<h3>Phase 3 trial underway to test TW001 edaravone therapy in early ALS</h3>
Evidence that Radicava slows the progression of ALS is conflicting and it is possible that Radicava <a href="https://alsnewstoday.com/news/radicava-suggests-more-real-world-data-needed-to-determine-efficacy-of-edaravone-in-als/">may not work well</a> for everyone. Moreover, it&#8217;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&#8217;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 <a href="https://www.ferrer.com/en">Ferrer</a>, which <a href="https://treeway.nl/ferrer-and-treeway-enter-into-licence-agreement-for-the-development-and-commercialisation-of-tw001-fnp122-for-amyotrophic-lateral-sclerosis/">acquired the rights</a> to develop and market the medication in Europe and certain Asian territories.
Ferrer now is conducting a Phase 3 study, dubbed <a href="https://www.clinicaltrials.gov/study/NCT05178810">ADORE (NCT05178810)</a>, that is testing TW001 against a placebo in approximately 300 people with early ALS. For this study, patients&#8217; 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.
[quote=&#8221;center&#8221;]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.[/quote] 
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 <a href="https://clinicaltrials.gov/study/NCT05866926">ADOREXT (NCT05866926)</a>, 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.
<a href="https://www.mndaustralia.org.au/">MND Australia</a>, 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&#8217;s executive director of research.

New deal aims to bring edaravone for ALS to Australia, New Zealand

A Phase 3 clinical trial that&#8217;s testing FNP122, <a href="https://www.ferrer.com/en" rel="nofollow">Ferrer’</a>s oral formulation of edaravone, in people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) is now rolling participants into its open-label extension study.
The <a href="https://www.clinicaltrials.gov/study/NCT05178810">ADORE trial (NCT05178810)</a> 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&#8217;s 48 weeks now have the opportunity to join an extension study called <a href="https://classic.clinicaltrials.gov/ct2/show/NCT05866926">ADOREXT (NCT05866926)</a>, where all will receive FNP122 for up to three years. The goal is to assess the treatment&#8217;s long-term safety.
“The ADOREXT study allows the company&#8217;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&#8217;s chief research and development officer, said in a <a href="https://www.businesswire.com/news/home/20230621955112/en/Ferrer-Initiates-its-ADOREXT-Extension-Study-in-Amyotrophic-Lateral-Sclerosis-ALS">company press release</a>.
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<h3>Edaravone, as the oral treatment Radicava ORS, is not available in Europe</h3>
Edaravone is an <a href="https://alsnewstoday.com/approved-treatments-for-als/">ALS treatment</a> 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: <a href="https://alsnewstoday.com/radicava-edaravone-for-als/">Radicava</a>, 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 <a href="https://alsnewstoday.com/news-posts/2020/03/30/radicava-effective-slowing-als-progression-across-1-year-use-patients-trial-data/">slow functional decline</a> 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 <a href="https://alsnewstoday.com/news/first-patient-enrolled-phase-3-adore-trial-fnp122-oral-form-edaravone-europe/">assess FNP122&#8217;s safety and efficacy</a> 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&#8217;s ability to perform daily activities, as measured with the ALS Functional Rating Scale &#8211; Revised (ALSFRS-R). The medication&#8217;s effects on survival also are being determined.
<h3>TRICALS also supporting the long-term ADOREXT study</h3>
All patients completing ADORE&#8217;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 <a href="https://www.tricals.org/en/">Treatment Research Initiative to Cure ALS</a> (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.

FNP122, edaravone in oral form, enters Phase 3 extension trial

An oral suspension form of <a href="https://alsnewstoday.com/radicava-edaravone-for-als/">Radicava</a> (edaravone), an approved treatment of <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS), was favored for patient reimbursement in Canada by a branch of the Canadian Agency for Drugs and Technologies in Health (CADTH).
The opinion issued by the CADTH Canadian Drug Expert Committee recommended that Radicava Oral Suspension be reimbursed through the public drug plans of the country&#8217;s provinces and territories.
Certain conditions must be met, including a prescription for this formulation and the cost of oral Radicava not exceeding that of Radicava in its initial intravenous formulation.
&#8220;The positive recommendation from CADTH is an important first step in ensuring access to Radicava Oral Suspension for all eligible Canadians living with ALS,&#8221; Atsushi Fujimoto, president of <a href="https://www.mt-pharma-ca.com/" rel="nofollow">Mitsubishi Tanabe Pharma Canada</a>, said in a <a href="https://www.newswire.ca/news-releases/radicava-r-edaravone-oral-suspension-receives-positive-recommendation-from-cadth-for-the-treatment-of-patients-with-als-882129765.html">company press release</a>.
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<h3>Oral Radicava can be taken at the home by mouth or feeding tube</h3>
CADTH&#8217;s recommendation is nonbinding, with the public drug plans of each province and territory deciding whether to list the oral medication based on the agency&#8217;s recommendation and factors such as its own priorities and resources.
Mitsubishi Tanabe Pharma Canada, a subsidiary of <a href="https://www.mt-pharma-america.com/" rel="nofollow">Mitsubishi Tanabe Pharma America</a> that markets Radicava in Canada,  stated it will continue to work with insurance companies and public agencies to make Radicava Oral Suspension available to patients via public formularies and private insurance. Public formularies are lists of prescription treatments covered as benefits for those eligible.
&#8220;While we are delighted by this milestone, we know there is more work to do, and we look forward to continued discussions with stakeholders to secure coverage for Radicava Oral Suspension on public formularies,&#8221; Fujimoto said.
Oxidative stress is seen as a central mechanism in the neuronal damage taking place in ALS. Radicava is a free-radical scavenger, meaning it helps to remove the reactive molecules that can contribute to oxidative stress to lessen nerve damage and slow ALS progression.
The intravenous (IV) formulation was <a href="https://alsnewstoday.com/news/radicava-now-approved-to-treat-als-patients-in-canada/">approved in Canada</a> in 2018, and patients using it are <a href="https://als.ca/blogs/access-to-therapies-radicava-edaravone-update/">reimbursed through public formularies</a> across Canada, as well as via the Veterans Affairs Canada and Indigenous Services Canada.
Radicava Oral Suspension, <a href="https://alsnewstoday.com/news/radicava-oral-suspension-approved-canada-treat-als/#:~:text=Radicava%20Oral%20Suspension%2C%20an%20oral%20formulation%20of%20edaravone%2C,in%20Canada%20for%20treating%20amyotrophic%20lateral%20sclerosis%20%28ALS%29.">approved by Health Canada</a> in November, was developed to be as effective as Radicava IV but more convenient.  It n can be self-administered at home, either by mouth or via a feeding tube, while Radicava IV is infusion treatment administered at a center, with each infusion lasting about one hour.
Available as <a href="https://www.radicava.com/patient/taking-radicava-ors/">Radicava ORS</a> in the U.S., the medication comes in a portable bottle and includes a 5 mL oral syringe for accurate dosing. It should be taken at a dose of 105 mg (the equivalent to 5 mL of the solution) in the morning after overnight fasting. Patients are recommended to take only water for one hour after administration.
The oral formulation is given in the same dosing regimen as Radicava IV: in a first cycle, patients receive daily dosing for 14 days, followed by a two-week treatment-free period. In subsequent cycles, the medication is given for 10 of the first 14 days, followed by two weeks with no treatment.
U.S. and Canadian approvals of Radicava Oral Suspension were supported by clinical trials showing that the oral formulation delivers similar amounts of edaravone, its active ingredient, to the body.
They also were based on interim results of the open-label <a href="https://clinicaltrials.gov/ct2/show/NCT04165824">Phase 3 trial (NCT04165824)</a> in 185 ALS patients, which showed that six months of treatment with Radicava Oral Suspension was <a href="https://alsnewstoday.com/news/oral-edaravone-appears-safe-patients-interim-trial-data/">generally well tolerated and slowed functional decline</a>.

Tag: oral edaravone

New deal aims to bring edaravone for ALS to Australia, New Zealand

FNP122, edaravone in oral form, enters Phase 3 extension trial

Radicava Oral Suspension Favored for Reimbursement in Canada