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    Ananda Ghosh • H.C. Wainwright & Co.

    Ananda Ghosh's questions to Amylyx Pharmaceuticals Inc (AMLX) leadership

    Ananda Ghosh's questions to Amylyx Pharmaceuticals Inc (AMLX) leadership • Q2 2025

    Question

    Ananda Ghosh questioned Amylyx's strategy for educating primary care physicians (PCPs) on PBH diagnosis and asked how Key Opinion Leaders (KOLs) are defining clinical significance for a potential therapy.

    Answer

    Co-CEO & Co-Founder Josh Cohen clarified that the launch will be endocrinologist-focused, as they are the primary treaters. He stated that KOLs consider the prevention of even a single severe hypoglycemic event to be clinically meaningful, given the potentially life-altering consequences of such events.

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    Ananda Ghosh's questions to Amylyx Pharmaceuticals Inc (AMLX) leadership • Q1 2025

    Question

    Ananda Ghosh from H.C. Wainwright & Co. asked about the Phase I LUMINA trial for AMX0114 in ALS. Given the preclinical data and the importance of neurofilament light chain (NFL) as a biomarker, she questioned if the trial's inclusion criteria required patients to have elevated NFL levels at baseline.

    Answer

    Co-CEO Justin Klee confirmed this was a key design consideration but explained that the trial does not have an inclusion criterion for high NFL levels. He reasoned that NFL levels in ALS patients are typically already very high, and for a first-in-human study, the company wanted to avoid over-subsetting the population to ensure they could observe signals across a broader patient group. They will, however, analyze outcomes based on baseline NFL levels.

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    Ananda Ghosh's questions to WAVE Life Sciences Ltd (WVE) leadership

    Ananda Ghosh's questions to WAVE Life Sciences Ltd (WVE) leadership • Q1 2025

    Question

    Ananda Ghosh of H.C. Wainwright & Co. asked about the potential for pleiotropic effects with the INHBE target and how Wave's AATD program differentiates from siRNA approaches like fasisiran.

    Answer

    Chief Scientific Officer Dr. Erik Ingelsson explained that the INHBE ligand-receptor pair is highly specific, so they expect only positive downstream metabolic effects, not primary pleiotropy. President and CEO Dr. Paul Bolno added that for AATD, an siRNA approach is suboptimal as it simply silences the protein, creating a need for IV replacement therapy to protect the lungs. He contrasted this with Wave's RNA editing approach, which aims to restore functional protein, thereby addressing both the liver (aggregate) and lung (protective) aspects of the disease with a single therapeutic.

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