Ann: Trial Met Primary & Exceeded Secondary Endpoint Expectations, page-10

WOW what a great result

additional data fantastic news

The stabilisation of the % FF combined with the observed increase/maintenance of the lean muscle mass suggests ATL1102 preserves functional muscle mass.

ATL1102 PRESERVES FUNCIONAL MUSCLE MASS

I am tipping companies will be knocking on our door after this

INCREASE / MAINTENANCE of lean muscle MASS

TAKE THE ABOVE PARAGRAPH and DIGEST

DO NOT LET ANYONE TALK THIS DOWN

THIS IS MAJOR BREAKTHROUGH in DMD at these levels

I dont suppose i was far away when i suggested repairing dystrophin or distrophin related as in

DMD occurs because the mutated DMD gene fails to produce virtually any functional dystrophin. Individuals with BMD genetic mutations make dystrophin that is partially functional, which protects their muscles from degenerating as badly or as quickly as in DMD.

It would appear that this trial may not repair or replace Dystrophin but from the above paragraph it would appear that 1102 is actually preserving existing muscle mass, and in my reading a future trial this could lead to preservation of the patients health as when he were to start his medication

I am speaking as a layman from what i have garnered from this report

dont listen to my ramblings or any other expert that frequents this forum

Read what the experts in this field have had to say in this report

Dr Ian Woodcock, Paediatric Neurologist and Honorary Fellow at The Murdoch Children’s Research Institute, Melbourne and the Principal Investigator of the ATL1102 Phase II DMD trial said, “the study met its primary endpoint showing ATL1102 to be safe and well tolerated with no serious adverse events being reported and no participant withdrawing from the study. With very few treatment options for boys with Duchennes who are no longer ambulant, it has been great to enable the boys to participate in this clinical trial and I am most encouraged by the outcomes of the study”

Dr Jean-Yves Hogrel, PhD, Director of the Neuromuscular Physiology and Evaluation Laboratory, Institute of Myology, France, a developer of the MyoSet assessment tools stated that “First, I would like to highlight the quality of the data that proves the expertise and dedication of evaluators in following standardised operating procedures. The intra-individual variability is very moderate and reflects this measurement quality. My observations based on the MyoSet data from the study suggest that the patients were generally stable.”

Dr Valeria Ricotti MD, Researcher and Honorary Clinical Lecturer, Great Ormond Street Institute of Child Health University College London, UK, stated that “Based on the MRI data from the study, the observed stabilisation in the percentage fat fraction with ATL1102 treatment would not be expected in the natural course of disease in DMD even under corticosteroid treatment. Furthermore, the stabilisation of fat fraction percentage combined with the observed maintenance/increase of remaining muscle area is suggestive that ATL1102’s effect could preserve the contractile muscle mass.”

Further data analyses performed by Dr Jean-Yves Hogrel looking at the correlation between MRI results for remaining muscle area (removing the fat fraction) with the results of the MyoGrip assessments for the individual participants has shown a highly significant positive correlation between these measures. Dr Hogrel stated that “this positive correlation of remaining muscle area (the lean muscle), with grip strength suggests a consistency of the results across the different parameters of muscle structure and muscle force.”


Professor Thomas Voit MD, Director, NIHR GOSH Biomedical Research Centre, UK had this to say about the trial results and the efficacy being observed in this Phase II trial of ATL1102: “The data certainly suggests an overall ‘stabilisation’ in disease progression at the very least which of itself is a very positive clinical outcome. MRI data confirms the positive changes at a muscular/cellular level and supports the observed physical stabilisation/improvements in muscle strength and function. The consistency of positive clinically relevant effects of ATL1102 treatment across muscle measures of structure, strength and function are very pleasing and provide great encouragement for the treatment of non-ambulant patients with DMD”

Dr Gil Price M.D., ANP’s Consultant Medical Director, said: “In a small study with nine boys of only six months duration at a single low dose we not only achieved our primary endpoint of safety we also i) achieved significance in demonstrating an effect on our secondary endpoint relating to modulation of CD49d; ii) achieved positive outcomes in the important upper body function parameter of the PUL 2.0 test, and finally iii) achieved fat fraction percentage reduction in important upper body muscles. That each of these three effects were observed in an initial study in DMD boys at the lowest dose where we might have anticipated a biological effect is truly surprising and highly encouraging”.

who knows what a larger does is going to achieve it does actually state INCREASE lean muscle mass at these levels WOW

This has to be exciting news not only for us as shareholders but the DMD community as a whole

See what hapens Tommorrow great report

GL to everyone especially the LTs amongst us