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- Antisense Therapeutics (ANP) receives a positive decision for the UK Paediatric Investigation Plan (PIP) submission for the development of its drug for Duchenne muscular dystrophy
- The decision came from the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK
- The plan aims to ensure necessary data is obtained through studies in children run in accordance with the European Medicines Agency and the MHRA’s expectations for future product approval
- A clinical trial will now kick off to determine the efficacy of the company’s lead drug candidate in patients with DMD
- Antisense Therapeutics last traded at 18.5 cents on December 13
Antisense Therapeutics (ANP) has received a positive decision for the UK Paediatric Investigation Plan (PIP) submission for the development of its drug for Duchenne muscular dystrophy.
The decision came from the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK, which the company considers a key location for the study.
The PIP is a development plan that addresses the entire paediatric development program for ATL1102 in Duchenne muscular dystrophy (DMD).
DMD is a disease that affects 1 in up to 6000 live male births, leading to ongoing deterioration in muscle strength. This can result in respiratory, muscular, cardiac and cognitive dysfunction. With no intervention, the average life expectancy of a patient with DMD is approximately 19 years.
The plan is aimed at ensuring necessary data is obtained through studies in children that are run in accordance with the European Medicines Agency and now the MHRA’s expectations for future product approval.
ATL1102 is Antisense’s lead drug candidate which returned highly promising Phase II trial results and has been shown to significantly reduce the number of brain lesions in patients with relapsing-remitting multiple sclerosis.
A Phase IIb/III clinical trial – a multicentre, randomised, double-blind, placebo-controlled study – will be used to determine the efficacy, safety and pharmacokinetic profile of ATL1102 in non-ambulatory participants with DMD, with a follow-on open label extension trial.
Clinical trial agreements will be executed with trial sites and separate trial applications will be made to national competent authorities of all participating countries.
Antisense Therapeutics last traded at 18.5 cents on December 13.
