Antisense Therapeutics Limited (ASX:ANP) has announced that it intends to conduct a double-blind, placebo-controlled six-month dosing trial (PUL2.0) of ATL1102 followed by a six-month open-label phase 2b trial in non-ambulant boys with Duchenne’s Muscular Dystrophy (DMD).
The company said the primary endpoint of PUL2.0 will be assessed after six months of treatment (versus 12 months in the Phase IIb/III study). It said the Phase 2b study aims to enrol and randomize 45 non-ambulant boys with DMD.
“Following the initial six-month regimen of either placebo, 25 mg or 50 mg once weekly, participants will be invited into a further six-month open-label follow-up treatment period in which all boys will be on active treatment (25 or 50mg),” said the company. “This additional time period will be used to demonstrate longevity of response as well as collect additional safety data and facilitate streamlining and de-risking of a Phase III study, the most expensive phase of the drug’s development (i.e., may reduce the number of dosing arms).”
The company said the revised trial design has allowed for the opportunity to incorporate Australian sites alongside key trial centres in Europe.
Antisense Therapeutics chair Dr Charmaine Gittleson said, “The Board, management and the Company’s clinical advisors remain focused on bringing ATL1102 to the non-ambulant DMD population. We believe that the Phase IIb study provides a valuable and significant de-risking step toward achieving this outcome. It allows us to build upon past work and re-enter the clinic, within the current market’s fiscal constraints, to obtain robust clinical outcome data that will either stand-alone or form part of a broader regulatory submission. We believe this represents significant value to the DMD community and our shareholders.”
