Antisense Therapeutics (ASX:ANP) has announced the phase 2 trial of its immunomodulatory therapy (ATL1102) for Duchenne Muscular Dystrophy (DMD) has met its primary endpoint and will now advance to a phase 2b study.
The company said the study has established the safety and tolerability of ATL1102. It added the therapy also met secondary endpoints that assessed its activity and efficacy. These include measuring the effects on immune cell numbers in the blood and measuring the participants’ functional capacity as evaluated via Performance of Upper Limb Test (PUL2.0), grip and pinch strength and distal mobility (using the MyoSet, MyoGrip, MyoPinch and MoviPlate tools, respectively).
The company reported that MRI assessment of the upper limb muscles of the patients with DMD has also shown apparent beneficial effects of ATL1102 in stabilising the fat fraction percentage within the muscles of the forearm (increase in fat levels is another key marker of disease progression in non-ambulant DMD boys).
"The data shows a stabilisation in the percentage of fat in the forearm muscles and an increase/maintenance of functional muscle mass, which is both outstanding and unexpected for a drug treating the inflammation (and not the muscle dystrophin loss)," it said.
ATL1102 is an inhibitor of CD49d expression on certain immune cells (e.g. T lymphocytes). Patients with DMD who have a greater number of CD4+ and CD8+T lymphocytes with high levels of CD49d have more severe and rapid disease progression.
The company said European Medicines Agency Scientific Advice due mid-year is the next milestone in preparation for submission of clinical trial application for a Phase 2b trial in Europe and UK. It is also preparing submissions for Orphan Drug Designation for ATL1102’s use in DMD in the US and the EU.
“Our focus is to continue planning and to methodically and efficiently move our DMD program through Phase IIb in Europe," said Mark Diamond, CEO of Antisense Therapeutics.
"There is great interest in the DMD space. As we gain further certainty from the regulatory process on the parameters of the next trial, which may lead to early market approval, we will assess funding and trial management options and also engage in discussions with interested potential partners ahead of commencement of Phase IIb.
This will allow the company to consider the most advantageous way to proceed with the pivotal trial on its own or with a partner. Potential partners’ input into the trial program may also be beneficial for future commercialisation plans. The company is now in the process of confirming the manufacture of additional clinical supplies of ATL1102 and will provide further updates in due course”.