Ann: Webinar Presentation - Phase IIb study of avicursen, page-3

First 15mins and we are rehashing a disease we have no interest in anymore. Anyway; my notes fwiw

- Subjective on behalf of the patient. Nevertheless best thing we have
- placebo shows same result as drug
- consistency of results show nothing
- no discontinuations due to adverse events
- adverse events inline with if the drug worked
- pk and pd effects; cd49d 50mg showed a reduction of 17%, 25mg 0%, placebo 18%. Decrease in Serum Creatine Kinase 50mg -18%.
- moving off placebo did nothing

Patients knew they were on active drug for ph2a = Hopium.
Dropping of cd49d is not enough to have a substantial outcome

MRI - didn't talk about the Ph2a result, just how it was difficult to manage in ph2b multisite. My question not answered, why MRI showed proof beyond placebo in ph2a.
Post trial access; some patients are doing well and the hope that the drug is the cause... there's so little else to hope on.
Longer study wouldn't have showed a difference. Higher drug dose may show signals, but safety issues come into play
Shelf life; doesn't require controlled storage conditions, not fragile (seemingly!?), checked some vials of drugs for damage, concentration looked exactly as it should.
Lots of audits along the way eg. no sabotage possibilities
First injection at clinical site, taught caregivers how to give injection at home.
Dr Voit's response; shared disappointment
Abbreviated end of study report to close study
ATL1102 is clinically dead.