HT-100 – A powerful Anti-Inflammatory and Anti-Fibrotic – £20k grant 

Alongside other Duchenne charities, Alex’s Wish are proud supporters and investors of this exciting clinical trial which is being run by Alkashi Therapeutics.

This is a promising new treatment and should it prove successful, will be eligible for all boys with Duchenne Muscular Dystrophy regardless of their mutation.

In non clinical studies (animal studies), HT-100 an oral drug, has been shown to decrease fibrosis, decrease inflammation and muscle degeneration, increase muscle regeneration and improve motor, pulmonary and cardiac function. Akashi Therapeutics is now running clinical trials in boys with Duchenne Muscular Dystrophy.

To date, early preliminary data on HT-100 appears to be well tolerated, with no early safety signals. Akashi Therapeutics expects to release more clinical data in Q1 2015 following an ongoing Phase 1a/2a clinical study (30 boys with Duchenne aged 6-20 both ambulatory and non-ambulatory, enabling investigators to evaluate a broad range of patient population) to evaluate safety and tolerability of increasing doses of HT-100, and explore trends in a range of efficacy endpoints including motor function and muscle strength tests as well as measures of pulmonary and cardiac function.

We will keep you posted on progress.11