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Tuesday, 01/02/2024 4:01:48 PM

Tuesday, January 02, 2024 4:01:48 PM

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In addition to the Rett efficacy + safety results and biometrics, the FDA will most certainly value the RWE data...

Over 91% of patients completing the trial continued into a 48-week open-label extension study (OLE), which is ongoing. Upon patient’s caregivers and investigators request, Anavex has established Compassionate Use Programs in Canada, Australia, and the UK for pediatric patients upon completion of the OLE study, similarly to its existing program for adult patients with Rett syndrome. To date, of the pediatric patients who completed the OLE, 93% have joined the Compassionate Use Program. This rate is comparable to the Compassionate Use level seen for adult patients which is over 96%. As of today, some patients with Rett syndrome have been on ANAVEX®2-73-treatment for over 4 years, combined OLE and Compassionate Use Program.

The high enrollment rates in the OLE and the high level of requests for the Compassionate Use Program provide solid numerical evidence for the reported positive Real World Evidence (RWE) from patients with Rett syndrome under Compassionate Use Authorization. Families whose children were previously on drug or placebo in the placebo-controlled trial commented favorably on the improvement of their child’s daily life due to ANAVEX®2-73 treatment in the Compassionate Use Program. E.g.:

Brigitte: “We did get a surprise once with her mobility. We heard a noise from our family room, and next we looked, and Madeline had climbed twelve steps upstairs to her bedroom by herself.”

Jayne: “Within a week of starting the Anavex open label extension, she only had one seizure and then she went three months without a seizure.”

See related link for more video comments from parents at RSAA/parent stories.

“We believe that ANAVEX®2-73, as a new, potential convenient treatment option in the future, can contribute to patients and healthcare professionals by addressing unmet needs in the treatment of Rett syndrome,” stated Christopher U Missling, PhD, President and Chief Executive Officer. “Based on these study results, we will continue to be committed to the Rett syndrome and rare disease community, given also the prior successful two placebo-controlled studies in adult patients with Rett syndrome. We express our deep gratitude for the commitment of the study participants and their caregivers, whose dedication and generous participation in clinical trials made this research possible.”

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