Madhu Kumar

Question 1

Madhu Kumar's questions to Passage BIO (PASG) leadership • Q1 2022

May 16, 2022

Question

Madhu Kumar from Goldman Sachs inquired about the hydrocephalus adverse event in the Krabbe disease trial, asking to distinguish between drug versus procedure-related causes and its potential impact on other programs using ICM injection. He also asked about key outcomes to expect from the upcoming ASGCT presentation on the GM1 program and how a competitor's recent discontinuation might affect GM1 trial recruitment.

Answer

Bruce Goldsmith, President and CEO, explained that while the root cause of the hydrocephalus event is difficult to determine, pre-dosing imaging showed some ventricle changes, and the event's temporal proximity led to classifying it as possibly related to the treatment or procedure. He emphasized that there is no evidence of inflammation and additional screening will be implemented, with no current indication of risk to other studies. For the ASGCT presentation, he highlighted that updated 12-month and 6-month data on biomarkers and clinical outcomes for GM1 would be shared. Regarding GM1 recruitment, Goldsmith stated they are open to collaborating with other centers but noted the primary rate-limiting step is the FDA-mandated 60-day interval between dosing patients.

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Question 2

Madhu Kumar's questions to Akari Therapeutics (AKTX) leadership • Q4 2017

Mar 21, 2018

Question

Madhu Kumar from B. Riley FBR sought clarification on the CAPSTONE trial's design, asking if the "standard of care" arm would include Eculizumab, and also inquired about the trial's powering assumptions and its primary endpoint.

Answer

CEO David Solomon clarified that the CAPSTONE trial is being conducted in geographies where Eculizumab (Soliris) is not reimbursed; therefore, the standard of care for the control arm is transfusion support. He explained that the primary endpoint will focus on transfusion reduction and hemoglobin levels, similar to Alexion's Triumph study, with LDH serving as a secondary endpoint. Solomon deferred providing specifics on the trial's powering assumptions at this time.