Antisense Therapeutics (ASX:ANP) has announced that seven patients are enrolled in the nine patient Phase 2 clinical trial of its immunomodulatory therapy, ATL1102, for Duchenne Muscular Dystrophy (DMD).
The company said one patient has completed dosing and the two month monitoring period. Five patients are in the treatment phase of the study with a seventh screened patient about to commence their dosing. Subject to meeting the eligibility criteria, the final two patients are anticipated to be enrolled into the trial this month with dosing completion to follow later in 2019, it said.
The open-label six-month dosing trial of ATL1102 in nine non-ambulant patients with DMD aged between 10 and 18 years is being conducted at the neuromuscular centre of the Royal Children’s Hospital in Melbourne (RCH), which operates the largest clinic in the southern hemisphere treating children with DMD.
The primary endpoints of the trial relate to the safety and tolerability of ATL1102. The efficacy of ATL1102 will also be assessed in terms of its effects on markers of inflammation, muscle damage and disease progression. The efficacy related endpoints will be assessed as changes from a patient’s baseline (start of the trial) measurements to the same measurements at the completion of their dosing.
ATL1102 is an inhibitor of CD49d expression on certain immune cells. 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 and do not appear to be as effective in patients with a greater number T cells that express high levels of CD49d.
The company said it expects to report trial results shortly after the completion of dosing. It has previously announced it has initiated a process focused on accelerating the development planning for ATL1102 that will include discussions with regulatory authorities, initially in Europe, on the design and conduct of the next clinical trial (Phase 2b) in DMD and on the development path for product registration.
According to CEO Mark Diamond, ”We are very happy with the progress being made in the Phase II clinical trial of ATL1102 in DMD patients at the RCH. Clinical trials for rare diseases such as DMD are notoriously challenging to recruit for, so we are pleased that we are close to having full enrolment in our trial with relatively minor delay. With no serious adverse events reported to date, and our clinical trial database continuing to grow with data on the longer term patient exposure to ATL1102, we are excited about the impending occasion of the reporting of our results and in turn the prospect that ATL1102 may play an important future role in the treatment of DMD”.