Ann: Percheron launches post-trial access program, page-71

Thanks @MrLearner,
I wanted to lay out some facts and/or suppositions about post trial access because I believe it provides a novel insight into double blind placebo controlled trials. The effect of the Declaration of Helsinki and the principle of non-maleficence is to focus upon the provision of care for the patient with a serious or life threatening illness following their participation in a clinical trial.

How altogether reassuring that humanity triumphs over science even within the constraints of running clinical trials. It appears to me that trial investigators are not merely a cog within the clinical trial process but are a key flywheel to protect patients who they deem have benefited from an investigational drug or product and would be harmed if taken off treatment.

The trial investigators in the case of the clinical trial of avicursen in DMD have taken the decision to request post trial access for some of their patients even though;

• its a blinded treatment
• its a phase IIb and not a phase III
• the optimal biological dose is still unknown

Of course the trial investigators are not the arbitrators and care must be taken not to pre-empt the decision of the regulators. Its best stated in the announcement of 21 October 2024 when the Principal Investigator commented that should avicursen receive approval from regulatory agencies "we believe it may have a valuable role to play in the treatment of this very challenging disease". The formation of international Clinical Advisory Boards to discuss the development of avicursen in DMD, the requests for PTA by trial investigators and the remarkable data from the phase IIa, all combine to spell out an an amazing opportunity to participate as investors in this Aussie biotech story. Who knows, it could be bigger than Neuren.