- Antisense Therapeutics (ANP) has submitted its ATL1102 inhibitor toxicology protocol to the US FDA to support extended clinical dosing
- The nine-month chronic monkey toxicology study will support the dosing of patients with the inhibitor beyond six months in the US for Duchenne Muscular Dystrophy (DMD)
- FDA interactions on the development pathway for DMD will continue in parallel with the conduct of phase 2b/three trial in Europe
- The company says it may receive a priority review voucher (PRV), given EU trial timing and US Congress extension of PRV sunset provisions
- Antisense Therapeutics shares have been trading up 8.33 per cent at 19.5 cents
Antisense Therapeutics (ANP) submitted its ATL1102 inhibitor toxicology protocol to the US Food and Drug Administration (FDA) to support extended clinical dosing in US studies.
The nine-month chronic monkey toxicology study will support the dosing of patients with ATL1102 beyond six months in the US for Duchenne Muscular Dystrophy (DMD).
The company said it expects feedback from the FDA on the protocol in the first quarter of 2022.
Guidance meetings held with the FDA earlier this year, provided the company with clarity on study requirements and design of a phase 2b/three for the US.
The company said it may have the potential to take data from the phase 2b/three Europe trial to the FDA for a future marketing application or a possible approval of ATL1102 for DMD without further trials.
Given the alignment of phase 2b/three between the European Medicines Agency (EMA) and the FDA, the feedback allows the company to engage with regulatory processes and push overall global clinical development plans.
Should the phase 2b/three trial in Europe be successful, the company believes it could be in a position to receive a rare pediatric disease priority review voucher (PRV), if it obtains FDA approval before September 30 2026, given EU trial timing and US congress extension of PRV sunset provisions.
The company said it may then decide to sell its PRV as a source non-dilutive capital.
Antisense Therapeutics shares were up 8.33 per cent at 19.5 cents at 12:01 pm AEDT.