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    Steven Seedhouse • Raymond James

    Steven Seedhouse's questions to Apellis Pharmaceuticals Inc (APLS) leadership

    Steven Seedhouse's questions to Apellis Pharmaceuticals Inc (APLS) leadership • Q1 2025

    Question

    Steven Seedhouse asked if Apellis is in dialogue with other industry members to resolve the co-pay assistance funding shortage and whether the company is willing to contribute to a new or existing charity fund.

    Answer

    Executive Cedric Francois stated that Apellis is not in such dialogues. He affirmed that the company respects the full independence of co-pay assistance organizations and keeps its contributions entirely separate from its commercial operations, though it does contribute to them periodically.

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    Steven Seedhouse's questions to BioCryst Pharmaceuticals Inc (BCRX) leadership

    Steven Seedhouse's questions to BioCryst Pharmaceuticals Inc (BCRX) leadership • Q1 2025

    Question

    Steven Seedhouse inquired about the potential market for the pediatric version of ORLADEYO, asking about the number of patients currently on competitor TAKHZYRO and the expected mix of patient switches versus new starts. He also asked what would constitute a successful initial data readout for the avoralstat program in Diabetic Macular Edema (DME).

    Answer

    Chief Commercial Officer Charlie Gayer estimated about 500 pediatric HAE patients in the U.S., with at least 200 suitable for prophylaxis, a number he believes will grow with an oral option. CEO Jon Stonehouse highlighted the significant advantage of a granule formulation over injections for children. For the DME program, Chief R&D Officer Dr. Helen Thackray stated that success would be defined by observing a clear effect on reducing retinal thickness, with CEO Jon Stonehouse adding that the drug's long-lasting concentration could yield significant data even from a single dose.

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    Steven Seedhouse's questions to WAVE Life Sciences Ltd (WVE) leadership

    Steven Seedhouse's questions to WAVE Life Sciences Ltd (WVE) leadership • Q3 2024

    Question

    Steven Seedhouse of Raymond James asked about key efficacy assessments for the WVE-006 AATD program's multi-dose cohorts, the potential to assess protein levels during infections, the estimated cost of the WVE-003 Huntington's disease Phase II/III program, and potential partnership structures.

    Answer

    Chief Development Officer Anne-Marie Li-Kwai-Cheung clarified that the RestorAATion-2 study for WVE-006 is focused on safety, PK, and PD, not efficacy, with exploratory endpoints like FibroScan being captured. CEO Paul Bolno added that while not prospectively planned, they could correlate protein levels with any patient infections that occur. Regarding the WVE-003 Huntington's program, Dr. Bolno stated that owning the asset and having a clearer regulatory path based on caudate atrophy opens up more flexible partnership structures, including geographic or financial collaborations, compared to previous deals designed for larger, longer trials.

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    Steven Seedhouse's questions to Viking Therapeutics Inc (VKTX) leadership

    Steven Seedhouse's questions to Viking Therapeutics Inc (VKTX) leadership • Q3 2024

    Question

    Steven Seedhouse questioned the potential inclusion of an active control arm in the VK2735 Phase III study, the strategy for testing the new amylin agonist in combination with other therapies, its potential for oral co-formulation, and the specific data supporting continued weight loss with longer oral dosing.

    Answer

    Executive Brian Lian clarified that initial Phase III studies will be placebo-controlled, with active comparator trials as a future possibility. He emphasized the amylin agonist's value is in combination therapies, which would be explored rapidly. He also confirmed the observation of a non-plateauing negative weight loss slope in the 28-day study supports expectations for further benefit with longer dosing.

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