Sign in

    Tommie Reerink • Goldman Sachs

    Tommie Reerink's questions to Ultragenyx Pharmaceutical Inc (RARE) leadership

    Tommie Reerink's questions to Ultragenyx Pharmaceutical Inc (RARE) leadership • Q2 2025

    Question

    Representing Salveen Richter of Goldman Sachs, Tommie Reerink asked about the regulatory and competitive bar for the upcoming Angelman syndrome data and whether there has been any updated feedback from the FDA on using the Bayley-4 scale as the primary endpoint.

    Answer

    Eric Crombez, Chief Medical Officer, stated that the bar for the Angelman program has not changed and that the company has full FDA buy-in on the study design. He expressed high confidence in the Bayley Cognition endpoint as foundational and noted that the rapid study enrollment reflects strong community enthusiasm for the therapy.

    Ask Fintool Equity Research AI

    Tommie Reerink's questions to BioMarin Pharmaceutical Inc (BMRN) leadership

    Tommie Reerink's questions to BioMarin Pharmaceutical Inc (BMRN) leadership • Q2 2025

    Question

    Tommie Reerink, on behalf of Salveen Richter at Goldman Sachs, asked for a breakdown of the factors contributing to the adjusted VOXZOGO guidance, including ex-U.S. order timing, demand, and U.S. expansion efforts.

    Answer

    Cristin Hubbard, EVP and Chief Commercial Officer, clarified that the guidance adjustment reflects better visibility with only five months left in the year. She attributed the change primarily to the shifting of some large OUS orders into later quarters, including into 2026. She emphasized that the updated guidance still represents significant 25% year-over-year growth at the midpoint.

    Ask Fintool Equity Research AI

    Tommie Reerink's questions to Sarepta Therapeutics Inc (SRPT) leadership

    Tommie Reerink's questions to Sarepta Therapeutics Inc (SRPT) leadership • Q4 2024

    Question

    Tommie Reerink, on for Salveen Richter at Goldman Sachs, asked about the key differentiators of Arrowhead's RNA platform and whether there is flexibility to expand infusion center capacity for ELEVIDYS.

    Answer

    Chief Scientific Officer Dr. Louise Rodino-Klapac highlighted Arrowhead's targeting ligands as a key differentiator, noting their effectiveness in reaching muscle tissue, along with a comprehensive and diligent approach to construct design. Chief Customer Officer Dallan Murray explained that expanding infusion capacity is not a simple lever to pull, as it's part of a complex cadence involving staffing, contracts, and finance, which dictates the overall pace of the launch.

    Ask Fintool Equity Research AI

    Tommie Reerink's questions to Sarepta Therapeutics Inc (SRPT) leadership • Q3 2024

    Question

    Tommie Reerink, on for Salveen Richter at Goldman Sachs, asked for quantification of the impact from patient infusions rescheduled from Q2 to Q3 and for the company's latest thinking on the long-term launch trajectory and peak year sales.

    Answer

    Chief Customer Officer Dallan Murray explained that the rescheduled infusions were already factored into the Q3 and Q4 guidance, so no specific quantification was necessary. President and CEO Doug Ingram reiterated the long-term plan to treat the prevalent Duchenne population by approximately 2030, with a period of significant growth throughout the decade.

    Ask Fintool Equity Research AI

    Tommie Reerink's questions to Alnylam Pharmaceuticals Inc (ALNY) leadership

    Tommie Reerink's questions to Alnylam Pharmaceuticals Inc (ALNY) leadership • Q3 2024

    Question

    On behalf of Salveen Richter, Tommie Reerink asked about the potential for a bolus of fast-progressing cardiomyopathy patients switching to AMVUTTRA, beyond the initial focus on first-line therapy.

    Answer

    Chief Commercial Officer Tolga Tanguler affirmed the strategy remains focused on establishing AMVUTTRA as a first-line therapy due to the disease's rapid progression. He acknowledged that physicians will identify patients progressing on stabilizers, but noted that a significant switching behavior has not yet been established in the market and will take time to develop.

    Ask Fintool Equity Research AI