Biotech Values

[pcrutch]

Re RNA/SRPT

Fast forward to 3 months later. Has SRPT disclosed what was in the FDA meeting minutes?

I suspect the FDA gave Prosensa/GSK its view in late December or early January given GSK walked soon after.

When was RNA supposed to have re-met with regulators? Sounded like they were planning a followup after the Jan16th press release. Any thoughts on Drisa re-dosing? Curious if they have considered doing IV vs the current subq.

-- Updates on the Drisapersen Development Program

On January 13, 2014, Prosensa and GlaxoSmithKline (GSK) announced that Prosensa regained all rights from GSK to drisapersen and retained rights to all other programs for the treatment of DMD. This transfer of rights represents the termination of the collaboration agreement between GSK and Prosensa executed in 2009 and gives Prosensa full, unencumbered rights to continue the development of drisapersen as well as each of its DMD programs.

On January 16, 2014, Prosensa announced initial findings from further analyses of the aggregate data obtained from the clinical development program of drisapersen for the treatment of DMD. The analyses, which are based on the results from three placebo controlled studies and two long term open label studies, suggest that treating earlier in DMD and treating longer shows a delay in the progression of the disease, as measured by the six-minute walk test (6MWT). Key safety findings are consistent with previous observations.

On February 18, 2014, the Investigational New Drug (IND) for drisapersen was transferred from GSK. As a first step in developing the path forward for drisapersen, Prosensa communicated to patient advocacy groups and investigators that it is exploring options for study participants to be re-dosed with drisapersen. Furthermore, it has initiated a survey to seek feedback from patients and investigators regarding the willingness and desire of patients to resume treatment. Based on the respondents to date, the majority of patients wish to be re-dosed with drisapersen.