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    Lydia Erdman • Goldman Sachs

    Lydia Erdman's questions to Agios Pharmaceuticals Inc (AGIO) leadership

    Lydia Erdman's questions to Agios Pharmaceuticals Inc (AGIO) leadership •

    Question

    Lydia Erdman of Goldman Sachs asked if Agios could provide details on baseline VOCs for the sickle cell trial now that enrollment is complete, and also inquired about the timing and content of the upcoming data release from the Phase IIb tebapivat study.

    Answer

    CMO Dr. Sarah Gheuens responded that baseline characteristics for the sickle cell trial will not be disclosed until the top-line data readout. Regarding tebapivat, she stated that more details on the timing of the Phase IIb data update will be provided next year.

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    Lydia Erdman's questions to Agios Pharmaceuticals Inc (AGIO) leadership • Q4 2024

    Question

    Lydia Erdman, on behalf of Salveen Richter at Goldman Sachs, asked how the expansion into the pediatric population could affect the total market opportunity for PYRUKYND in PK deficiency (PKD).

    Answer

    Chief Commercial Officer Tsveta Milanova stated that the pediatric segment represents approximately 20% of the ultra-rare PKD market. CEO Brian Goff added that while it's an important future addition for patients, the company's focus on the thalassemia launch means they expect overall PKD revenues to be relatively flat in 2025 compared to 2024.

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    Lydia Erdman's questions to Taysha Gene Therapies Inc (TSHA) leadership

    Lydia Erdman's questions to Taysha Gene Therapies Inc (TSHA) leadership • Q2 2025

    Question

    Lydia Erdman of Goldman Sachs asked if the 100% response rate for the pivotal trial's primary endpoint observed in the Part A study is the expected bar for the Part B pivotal trial.

    Answer

    CEO Sean Nolan explained that while 100% is a difficult standard, the statistical plan for Part B is robust. He noted the trial uses a null hypothesis of 6.7%, making the statistical bar for success relatively low at approximately 33%. This gives the company high confidence based on the compelling and consistent results from Part A.

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