Treeway

[GregK]

Interesting.
This outfit has 3 ALS treatments in the pipeline. Treeway Pipeline - Treeway
One, TW001, is a mod of the recent drug approved in Japan, edaravone. They state it should hit the market in 2017.

Treeway’s pipeline is anchored on the role of immunology and inflammation in the disease progression of ALS. Near-term focus for 2015 is on a series of complementary assets that together provide a robust portfolio with a balanced risk-reward profile:

TW001; Oral formulation of edaravone. Edaravone is a neuroprotective agent and acts as a potent radical scavenger, thereby protecting against oxidative stress and neuronal apoptosis. In Japan, it was approved under the brand name Radicut® for the indication stroke in 2001 and for the indication ALS in June 2015. However, Radicut® is an intravenous drug and administered for 14 days followed by 14 days drug holiday. To optimize the efficacy, and to be able to administer in a more convenient way and on a daily basis, Treeway has developed an oral formulation of edaravone (TW001). TW001 is currently in clinical stage and Treeway aims to develop TW001 globally. Treeway was awarded Orphan Drug Designation for edaravone by the EMA in November 2014 and FDA in January 2015.Reformulated therapeutic drug that has shown clinical efficacy in previous clinical trials. Start of clinical program in first half of 2015

TW002i; AAV5-GDNF Gene Therapy; novel administration modality available for clinical use; preparation of Proof-of-Concept clinical trial; co-development partnership with uniQure

TW003; Vaccine therapy development to permanently restore immune balance; co-development partnership with Amarna Therapeutics

TW004: Rational therapy development research program towards combination therapy to restore immune balance and neuropathology; collaborations with international academia

The balanced-risk reward profile, taking into account development time (speed), reward, risk and costs is shown beside.

Treeway focuses on ALS therapies, with TW001 as the first drug available on the market by 2017. TW001 will be protective, and thus significantly delay disease progression and increase the patient’s lifespan. Development time is short, and costs are low.

TW002 and TW004 will not only be protective but also capable of controlling the disease. We foresee a development time of 4 years for TW002, and 6 years for TW004 We aim for TW003 to be a cure for ALS or at least significantly delay disease progression by a minimum of 5 years. This will revolutionize the treatment and diagnostics for ALS. We anticipate, in case of successful proof of concept (PoC), to enter efficacy clinical trials within 3 years.

In all cases Treeway aims to have an early access program available when possible.

 

[GregK]

Their TW-001 is moving along:

Treeway Announces Positive Data from Two Separate Phase I TW001 Clinical Trials | Small Molecules | News Channels

ROTTERDAM, The Netherlands I December 10, 2015 I Treeway, a biotech company developing therapies against amyotrophic lateral sclerosis (ALS), today announces the completion of two successful phase I trials with their lead compound TW001, a new more patient friendly oral formulation of the Japanese stroke and ALS therapy edaravone. Treeway, founded by two Dutch ALS patients, aims at speeding up ALS research in collaboration with academic and research centers around the globe.

TW001 was tested in both single and multiple dose phase l studies in healthy volunteers and in ALS patients. In the two Phase 1 clinical studies TW001 was shown to be safe and well tolerated and adequate exposure levels with the oral formulation were detected. Based on these trial data, the company is now aiming to start a pivotal phase 2/3 study in 2016. Earlier, Treeway obtained orphan drug designation for edaravone of ALS from both the FDA and EMA.

Inez de Greef, CEO of Treeway comments on the trial outcome: “The positive results of our Phase I program are an important milestone for the development of TW001 for ALS. This outcome is not only represents a significant achievement for our company, but first offers important new patient friendly aspects to the treatment of ALS”. We look forward to sustaining this momentum in the coming months, as preparations for the pivotal Phase II/III clinical study are now ongoing.’

Lead compound

TW001, Treeway’s lead compound, is an oral formulation of edaravone. Edaravone is a well-established free radical scavenger, which targets oxidative stress, a process that plays an important role in ALS. Intravenously administered edaravone has been shown to slow down disease progression in ALS patients in previous Japanese clinical trials.

Treeway has developed an oral formulation (TW001) to overcome the problems associated with intravenous administration. The reformulated drug allows for chronic daily use and its route of administration (oral) is patient friendly.

 

[affected]

thanks Greg!

 

[Nikki J]

The PALS from the executive board was the final speaker at the MNDA symposium yesterday