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    Ellen Horste • Cowen

    Ellen Horste's questions to Kura Oncology Inc (KURA) leadership

    Ellen Horste's questions to Kura Oncology Inc (KURA) leadership • Q2 2025

    Question

    Ellen Horste asked about the biggest potential risks to the pivotal program timelines that could delay data past 2028 and whether there is any risk associated with running combined studies for both KMT2A and NPM1 populations.

    Answer

    Chief Medical Officer Dr. Mollie Leoni responded that the 2028 timeline was set conservatively and they remain confident in meeting it. She added that based on robust data from the COMET-seven trial, the company has extensively modeled the combined populations and does not see it as a risk, viewing it as a strength of the trial design.

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    Ellen Horste's questions to BioMarin Pharmaceutical Inc (BMRN) leadership

    Ellen Horste's questions to BioMarin Pharmaceutical Inc (BMRN) leadership • Q2 2025

    Question

    Ellen Horste from TD Cowen asked what would constitute success for the BMN-401 (formerly INZ-701) ENERGY-3 trial, including functional endpoints, and inquired about the number of identified patients and the strategy for finding the estimated 2,000-2,500 total patients.

    Answer

    EVP & Chief R&D Officer Gregory Friberg explained that success involves co-primary endpoints of normalizing pyrophosphate levels and improving a radiologic index of bone quality. EVP & CCO Cristin Hubbard added that while Inozyme had identified over 600 patients, BioMarin will leverage its established infrastructure and diagnostic expertise within its enzyme therapy unit to find the remaining patient population, as the treating specialists overlap significantly.

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    Ellen Horste's questions to Syndax Pharmaceuticals Inc (SNDX) leadership

    Ellen Horste's questions to Syndax Pharmaceuticals Inc (SNDX) leadership • Q1 2025

    Question

    Ellen Horste from TD Cowen asked for color on the month-over-month trends for new Revuforj patient starts in Q1 and details on the free drug rate and its expected trajectory.

    Answer

    Chief Commercial Officer Steven Closter stated that while specific monthly numbers would not be provided, there was a strong ramp from Q4 2024 into Q1 2025. He mentioned it would take at least two full quarters to see a stable run rate. Regarding patient assistance, he reported that the free drug rate is very low, in the single digits, due to efficient execution, strong formulary coverage, and a well-defined patient assistance program.

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