Aspen Mori's questions to Boundless Bio (BOLD) leadership • Q1 2019
Question
Aspen Mori from Bank of America Merrill Lynch questioned the apparent disconnect between superior biopsy data in Cohort 2 and comparable functional scores for AT132 in XLMTM, and also asked about the competitive strategy for the DMD program.
Answer
Matt Patterson, Chairman and CEO, addressed the AT132 data by explaining that while biopsies show a dose-dependent effect, clinical measures have inherent variability, making it difficult to detect differences between doses when patients are achieving highly meaningful improvements and approaching normal function. For DMD, he stated the strategy is to be 'best-in-class' by producing a near-normal dystrophin protein, which they believe will prove superior to microdystrophin approaches over time. He emphasized that Audentes' manufacturing capabilities and a 'template approach' to development could accelerate timelines.