Antisense Therapeutics (ASX:ANP) has announced the presentation of additional data from seven patients who have completed their 24 weeks of dosing in the ATL1102 phase 2 DMD clinical trial.
The data was presented by principle investigator Dr Ian Woodcock at the 2019 Action Duchenne International Conference in the UK.
The company said the new clinical data included a more detailed safety overview and re-confirmation that no Serious Adverse Events (SAEs) have been reported to date and that the Data Safety Monitoring Board continues to have no safety concerns.
"In regard to the trial’s secondary endpoints that assess drug efficacy in terms of its effects on disease processes and progression (being the type of endpoints required for future product registration), Dr Woodcock presented new data on the functional capacity of the participants as evaluated via Performance of Upper Limb Test (PUL2.0)," said the company.
"PUL is a functional scale specifically designed for assessing upper limb function in DMD with the aim of reflecting the proximal to distal progression of muscle weakness typically observed in DMD. It includes three domains (shoulder, mid- and distal), each including items exploring activities easily related to activities of daily living that both patients and clinicians regard as relevant."
It continued, the PUL data presented by Dr Woodcock showed that the majority of participants have demonstrated either increases or no change in their PUL2.0 scores from baseline after 24 weeks of dosing with ATL1102 suggestive of an overall improvement in a key parameter of disease progression.
Muscle strength was also evaluated (as previously reported) via MyoGrip and MyoPinch assessments using the Myoset system with the data continuing to show an apparent improvement in muscle strength based on observed mean changes from baseline compared to the loss of muscle strength reported in the literature in similar patient populations, said the company.
The company said it is consulting with DMD experts in regard to the ongoing development of ATL1102 and the phase 2b clinical trial including Professor Thomas Voit MD, Director, NIHR GOSH Biomedical Research Centre, UK who is participating in all the planned SA meetings.
“I am most encouraged by the preliminary data that is emerging from the Phase II clinical trial of ATL1102 in non-ambulant DMD patients in particular its safety profile and apparent positive effects on disease progression endpoints that will be employed in the follow on clinical trial," said Dr Volt.
"There are a very few treatment alternatives being developed for the non-ambulant DMD population, so I would expect that these preliminary findings should help facilitate productive interactions with the regulatory authorities on a hopefully expedited development path to market for such an underserved patient group.”