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    Mehdi Goudarzi • Truist Securities

    Mehdi Goudarzi's questions to Intellia Therapeutics Inc (NTLA) leadership

    Mehdi Goudarzi's questions to Intellia Therapeutics Inc (NTLA) leadership • Q2 2025

    Question

    On behalf of Joon Lee at Truist Securities, Mehdi Goudarzi inquired about how study blindness is maintained for LONVOZIE given its potentially binary response, and whether redosing is an option for patients with breakthrough attacks.

    Answer

    EVP & CMO David Lebwohl assured that the trial uses standard blinding procedures where neither the patient nor the physician knows the infusion content, and a placebo effect can also occur. CEO John Leonard added that the study's primary endpoint is discrete clinical events (attacks), which are objective and not subjective, reinforcing the study's integrity.

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    Mehdi Goudarzi's questions to Ultragenyx Pharmaceutical Inc (RARE) leadership

    Mehdi Goudarzi's questions to Ultragenyx Pharmaceutical Inc (RARE) leadership • Q2 2025

    Question

    Mehdi Goudarzi from Truist Securities asked for an explanation of the non-target site factors that contribute to the different dosing regimens between Ultragenyx's GTX-102 and a competitor's drug, both of which use LNA chemistry.

    Answer

    Emil Kakkis, Founder, President & CEO, explained that the LNA chemistry was selected for its superior potency, which allows for a lower and more advantageous dosing range of 5-14mg. He believes this lower chemical concentration reduces off-target effects, creating a better therapeutic window and contributing to the strong results that led to its Breakthrough Therapy Designation.

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    Mehdi Goudarzi's questions to Ultragenyx Pharmaceutical Inc (RARE) leadership • Q3 2024

    Question

    Mehdi Goudarzi, on for Joon Lee, asked for details on the Angelman syndrome Aurora study, including its diverse patient population and the optimal endpoint, questioning if it would be the Bayley-4 cognition score.

    Answer

    CEO Emil Kakkis clarified that the main Phase 3 study is Aspire, which focuses on a specific patient group. The Aurora study is a supportive, open-label trial designed to extend labeling to other populations, including different age groups and genetic types. Its goal is to demonstrate comparable safety and efficacy to the main trial, helping to cover various segments of the Angelman population.

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    Mehdi Goudarzi's questions to argenx SE (ARGX) leadership

    Mehdi Goudarzi's questions to argenx SE (ARGX) leadership • Q4 2024

    Question

    Mehdi Goudarzi, on for Joon Lee, requested more color on the plans for the early pipeline asset ARGX-109, an anti-IL-6 antibody, given its long history and recent return to the company.

    Answer

    CEO Tim Van Hauwermeiren characterized ARGX-109 as a best-in-class IL-6 blocker with high potency and a long half-life. He stated that argenx has a 'unique and novel angle' on IL-6 biology for new autoimmune indications, which are not yet disclosed. Phase I data is expected in the second half of the year.

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