Expert Interview
Discussing the potential of Ulixacaltamide HCl for Essential Tremors following the Essential3 clinical trial topline data release from Praxis Precision Medicines
Ticker(s): PRAXInstitution: Beth Israel Deaconess Medical Center
- Physician-scientist with extensive experience in the pharmaceutical industry and the U.S. Food and Drug Administration (FDA), currently serving as a consultant and venture mentor at Beth Israel Deaconess Medical Center.
- Former FDA Medical Officer and Lead Medical Officer.
- Contributed to the review of numerous INDs and multiple NDA/BLA approvals across neurology, anesthesiology, and rare diseases.
What are your thoughts on FDA approvability, if IDMC used unblinded data to recommend statistical model changes?
Added By: max_adminGiven a majority of discontinued ulixacaltamide patients are TEAE-related, do you think this is an appropriate imputation method the FDA would approve?
Added By: max_adminGiven the overall efficacy /safety profile, do you think Ulixacaltamide trial and statistical analysis plan changes pose a risk onapprovability? Do you think the current FDA neurology division would approve Ulixacaltamide?
Added By: max_adminHow would FDA approach analyzing Praxis’s ET data set given trial design changes? Is there precedent neurology trials whereendpoints and SAP were modified that lead to an approved drug? (eg. aducanumab) and how does Praxis’ data compare in terms of approvability?
Added By: max_adminAre You Interested In These Questions?
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