Clinical programs

TW001: Oral formulation of edaravone. Edaravone is a free radical scavenger that targets oxidative stress, a process known to play an important role in the pathogenesis of ALS. In 2015, the intravenous formulation of edaravone, known as Radicut®, was approved in Japan for the indication ALS. Radicut® is administered intravenously and the dosing regimen consists of 10 days of treatment, over a period of two weeks, followed by a 16-day drug holiday, a period in which the patient is not given edaravone. Treeway believes that sustained exposure levels are required to more efficiently target a chronic process such as oxidative stress. The intravenous formulation and the dosing regimen are believed to have limitations, including the invasive route of administration and the suboptimal exposure levels associated with the drug holidays. Treeway is developing a patient friendly oral formulation of edaravone to overcome the problems associated with intravenous administration. TW001 is currently in clinical stage. TW001 was awarded Orphan Drug Designation status by the European Medicines Agency in 2014 and by the Food and Drug Administration in 2015.

Patients interested in participating in a clinical study with TW001 can find more information at

Preclinical programs

TW002: AAV5-GDNF gene therapy. GDNF is a neurotrophic factor with neuroprotective properties. Proof of Concept studies with TW002 are currently ongoing. TW002 is being developed in collaboration with uniQure.

Discovery programs 

The Discovery Research of Treeway is focused on 3 main programs or “Research Lines” that approach the disease from different angles: 1) Project MinE (focused on the discovery of new more upstream disease pathways and new disease targets by integrating big data obtained through different “omics” approaches; Project MinE is the project that collects ALS-associated genetic information, including gene mutations, risk genes and sequences, that will provide important information on possible causative disease pathways.),  2) motor neuron signalling (focused on the earliest deficiencies in the motor neuron physiology that might also be caused by non-cell-autonomous factors) and 3) the immune system (focused on shifting the balance of the immune response from the pro-inflammatory response back towards its protective function). In addition to these 3 main research lines, Treeway is involved in an European research program, called NaNOals, that aims to replace lost motor neurons in ALS patients as a potential cure of the disease. These different approaches not only enhance the chance of success, but also enable the development of combination therapies that in the end might be required to fully control the disease.

For its Discovery Research activities Treeway has established or will establish close and long-term relationships with selected academic partners with strong and long-standing expertise in the field of ALS or drug development. Collaborations with the University Medical Centre (UMC) in Utrecht and the Leiden Academic Center for Drug Research (LACDR) in Leiden have been established. In addition, Treeway embraces new research opportunities via collaborations with other pharma or biotech companies or academic institutions.