MELBOURNE, Australia, Nov. 18, 2019 /PRNewswire/ -- Antisense Therapeutics (ASX: ANP/OTC: ATHJY) is pleased to advise that additional preliminary data analyses from the seven patients who have completed their 24 weeks of dosing in the ATL1102 Phase II DMD clinical trial was presented by Dr Ian Woodcock, the Principle Investigator of the ATL1102 Phase II trial at the 2019 Action Duchenne International Conference, Hinkley, UK on 15 November 2019.
The new clinical data (in addition to preliminary data reported by ANP on 18 September 2019) presented by Dr Woodcock 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). 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.
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.
These results continue to appear highly supportive of the Company's clinical development program, with plans for a Phase IIb clinical trial of ATL1102 in DMD presently being reviewed with European regulatory authorities at Scientific Advice (SA) meetings. The Company expects to report on these interactions after it receives written responses following the meetings.
The Company is consulting with internationally recognized DMD experts in regard to the ongoing development of ATL1102 and the Phase IIb clinical trial including Professor Thomas Voit MD, Director, NIHR GOSH Biomedical Research Centre, UK who is participating in all the planned SA meetings. Dr Voit had this to say about the preliminary trial results and their bearing on Phase IIb planning: "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. 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."
The ATL1102 Phase II DMD trial remains ongoing with dosing in all patients to be completed this month.
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About Antisense Therapeutics Limited (ANP.AX) is an Australian publicly listed biopharmaceutical company, developing and commercialising antisense pharmaceuticals for large unmet markets. The products are in-licensed from Ionis Pharmaceuticals Inc. (IONS), world leaders in antisense drug development and commercialisation. ATL1102 (injection) has successfully completed a Phase II efficacy and safety trial, significantly reducing the number of brain lesions in patients with relapsing-remitting multiple sclerosis (RRMS). ATL1103 drug designed to block GHr production successfully reduced blood IGF-I levels in Phase II clinical trials in patients with the growth disorder acromegaly. The Company is conducting a Phase II clinical trial of ATL1102 in DMD patients at the Royal Children's Hospital, Melbourne.