Antisense Therapeutics updates on preliminary results from ATL1102 study

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Antisense Therapeutics (ASX:ANP) has announced that a review of preliminary data from the six patients who have completed their 24 weeks of dosing in the phase 2 clinical trial of its immunomodulatory therapy, ATL1102 for Duchenne Muscular Dystrophy (DMD) therapy, is indicative of a positive drug effect.

ATL1102 is an inhibitor of CD49d expression on certain immune cell. It has been reported that patients with DMD who have a greater number of T cells with high levels of CD49d on their surface have more severe and rapid disease progression.

ATL1102 is being developed as a novel treatment for the inflammation that exacerbates muscle fibre damage in DMD patients, currently treated with corticosteroids. Corticosteroids have a range of serious side effects when used for a prolonged period as required in DMD.

The primary endpoints of the trial relate to the safety and tolerability of ATL1102 with efficacy in DMD assessed in terms of its effects on disease processes and progression.

The company said that, with respect to the safety related trial data, no serious adverse events have been reported to date.

The company also said it is highly encouraged the data to date is indicative of a drug effect, particularly considering the small number of patients evaluated.

"Early indications of an immunomodulatory effect are underpinned via observations that certain immune cell numbers (in particular those expressing the CD49d, the biological target of ATL1102) are trending downward during the treatment phase while returning to around starting levels post dosing," said the company.

It said the results appear supportive of its plans for a phase 2b clinical trial of ATL1102 in DMD.

“Given that there is currently no effective treatment for non-ambulant DMD patients, we are particularly encouraged by the preliminary data from the first six patients in this trial, which suggests a positive drug effect and may also demonstrate a meaningful slowing of disease progression compared to what might otherwise have been expected," said Mark Diamond, CEO of Antisense Therapeutics. 

"We expect this preliminary data to assist us in our planned regulatory interactions on the design and conduct of the Phase IIb clinical trial that should allow examination of dosages of 25mg and higher to determine the optimal dosage”.