Treeway’s Oral Formulation of Edavarone for ALS Shows Promise in Phase 1 Trial

Treeway’s Oral Formulation of Edavarone for ALS Shows Promise in Phase 1 Trial

An experimental oral formulation of edavorone for amyotrophic lateral sclerosis (ALS), called TW001, demonstrated promising pharmacological and safety data in a Phase 1 clinical trial, according to the biotech Treeway.

The randomized trial compared TW001 to Radicava (Mitsubishi Tanabe Pharma America), an intravenous medication approved in the U.S. in 2017 for the treatment of ALS.

Both TW001 and Radicava have edavarone as the active ingredient.

Edavarone is a neuroprotective compound that decreases oxidative stress, a crucial element in the death of neurons in ALS.

Specifically, scientists compared a single oral dose of 140 mg TW001 to a one-hour infusion of 60 mg Radicava in 18 healthy participants. They then analyzed the medications’ bioavailability in each group, which refers to the proportion that reaches circulation after administration.

Results showed that TW001, Treeway’s lead compound, had higher bioavailability than Radicava. The treatment candidate was well-tolerated and did not raise safety concerns.

Ongoing studies also suggest that the oral formulation is stable. These findings indicate that TW001 has the potential to improve treatment of ALS patients, researchers believe.

“It is Treeway’s mission to identify novel treatment routes for ALS patients and we believe that this study indicates that an oral formulation of edavarone may offer patients a better quality of life through a simpler and more effective route of administration,” Ronald van der Geest, chief development officer of the Netherlands-based Treeway, said in a press release.

“Based on the positive results we have seen in this comparative study, we can initiate a pivotal Phase 3 study and move rapidly toward bringing TW001 to patients,” van der Geest added.

Merit Cudkowicz, MD, director of the ALS Multidisciplinary Clinic at Massachusetts General Hospital and a key adviser to Treeway, said the Phase 1 trial results “are a promising sign for ALS patients, who are in high need of better and more patient-friendly treatments for their disease.

“I regard this study to be a key milestone for the development of TW001 in ALS and I look forward to supporting the company’s scientific and clinical progress,” she added.

Treeway is developing TW001 as a disease-modifying therapy for convenient daily treatment of patients with ALS. In clinical trials, edavarone slowed the progression of ALS by one-third.


  1. stuart says:

    Shows promise in phase 1, says it all really, phase 1 haha.
    Please spare us the false optimism. It shouldn’t even come into discussion until its passed a 2nd phase trial.

  2. WayneG says:

    I’d rather hear about everything that’s happening in possible new ALS treatments than be left in the dark until it gets to Phase 2 or 3.

  3. Steve G UK Cambridge says:

    How can I get in on this action? I have the slow form of ALS, been living with it since 2005 and it is now starting to have an effect. My ex wife uses it to withhold access to my four year old daughter citing I too disabled to look after her at weekends despite support in place to help me. I have just given up driving, now walk with a limp, have very thin arms and started to record my voice. I am sliding further and further into debt with solicitors fighting to keep my connection and anything to slow down progression would help. Not only am I dealing with this horrific illness I also have to fend off attacked after an attack from a venomous ex partner trying to erase me from my daughter’s history.

    • Adele says:

      It is a hard time for you but believe the miracles do happen. I hope that your ex will realise that being kind is much better and more rewarding than being mean. One day she will have to answer to your daughter ” What did you do for my sick father” Concentrate on your well being, take supplements. The best wishes.

  4. Jim Peckenpaugh says:

    Even the Japan Radicava study? Where is a recent progress report of those patients?? Why is ALSA not reporting or questioning this??

  5. Seamus O Hagan says:

    What ever has happened to Treeway have they disappeared ? I am suffering with als for the past 3/4 years. I am 49 married with 4 kids.As with all of us patients I was delighted with their phase 1 results but what has happened since ?
    I am lucky that I attend a top neurologist here in Ireland. We will never access EDARAVONE in its current form as it wont suit our healthcare system in Europe so we NEED oral version if possible. I have emailed Treeway politely just for any info but have heard nothing back or can find nothing online since this trial. What I would like to know when will it be available in oral form if ever? and does it work surely people have been on it since 2017 so any results?
    Like us all I feel so frustrated at my life wasting away and spend a lot of my time wishing for anything to help.
    Best wishes

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