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 and in the USA it was approved under the name Radicava® in May 2017. Radicut® and Radicava® are 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.
In 2015, Treeway demonstrated in clinical trials that adequate levels of edaravone can be obtained in the blood by oral administration of edaravone. At Treeway, orally administered edaravone is referred to as TW001. Subsequently, a development plan was made to bring TW001 to the market. Currently, this plan is being executed. The approval of Radicava® does not interfere with Treeway’s plans for TW001.
Treeway is developing Edaravone as a formulation so patients can take it via the mouth (oral) each day as a sustained delivery medicine. 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.
We are determined to make TW001 available to the patients as quickly as possible so ALS patients all over the world can take this drug and thereby delay the disease process.
Patients interested in participating in a clinical study with TW001 can find more information at www.tricals.org
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.
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. These opportunities are evaluated on a case by case basis.