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    Sami Corwin • William Blair & Company

    Sami Corwin's questions to Allogene Therapeutics Inc (ALLO) leadership

    Sami Corwin's questions to Allogene Therapeutics Inc (ALLO) leadership • Q2 2025

    Question

    Sami Corwin asked about the change in stance on sharing MRD conversion rates, given previous concerns about influencing physician behavior. She also inquired if the ALLO-329 autoimmune disease update would include data from both the cyclophosphamide-only and no-lymphodepletion cohorts.

    Answer

    David Chang, CEO, explained that sharing the secondary biomarker data on a limited number of patients is not expected to compromise the trial's integrity. He confirmed the ALLO-329 update in H1 2026 will include biomarker and early clinical data from both lymphodepletion cohorts, with more details to be provided as the study progresses.

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    Sami Corwin's questions to Allogene Therapeutics Inc (ALLO) leadership • Q1 2025

    Question

    Representing Sami Corwin of William Blair, an analyst asked if site-related challenges from ALPHA3 are expected at ex-U.S. sites and if the ALLO-329 autoimmune study will also expand internationally.

    Answer

    Dr. Zachary Roberts, EVP of R&D and CMO, stated they expect a smoother rollout for ALPHA3 ex-U.S. due to lessons learned and less fragmented lymphoma care abroad. For ALLO-329, he said the near-term focus is on U.S. sites, but the company is open to ex-U.S. expansion in the future given the competitive landscape.

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    Sami Corwin's questions to Zevra Therapeutics Inc (ZVRA) leadership

    Sami Corwin's questions to Zevra Therapeutics Inc (ZVRA) leadership • Q2 2025

    Question

    Sami Corwin of William Blair inquired about the number of unique Myclifa prescribers, how that figure has trended, and the potential for further expanding the percentage of covered lives.

    Answer

    Chief Commercial Officer Joshua Schafer explained that the number of unique prescribers has grown significantly as the launch moves beyond EAP investigators to community clinicians treating fewer patients, a trend he expects to continue. He noted that covered lives stand at 52%, which is typical for this stage of a launch, and anticipates this will increase as more health plans complete their reviews, using Olpruva's 79% coverage as a potential long-term analog.

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    Sami Corwin's questions to PTC Therapeutics Inc (PTCT) leadership

    Sami Corwin's questions to PTC Therapeutics Inc (PTCT) leadership • Q2 2025

    Question

    Sami Corwin from William Blair requested an update on the FDA's review of Translarna in the U.S., including any recent interactions. She also asked about the company's perspective on additional business development opportunities given its strong balance sheet.

    Answer

    CEO Dr. Matthew Klein reported that the Translarna NDA remains under active review, with clinical site inspections completed in the spring and easily addressed information requests received in the early summer. Regarding business development, CFO Pierre Gravier and Dr. Klein stated that with a strong financial position, PTC is actively looking for disciplined, value-creating opportunities. They are seeking assets that could fit into their existing global commercial infrastructure or complement their R&D portfolio, focusing on their sweet spot of rare diseases.

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    Sami Corwin's questions to Ultragenyx Pharmaceutical Inc (RARE) leadership

    Sami Corwin's questions to Ultragenyx Pharmaceutical Inc (RARE) leadership • Q2 2025

    Question

    Sami Corwin of William Blair asked if the leadership changes at CBER would impact the UX-111 CRL resolution and whether Ultragenyx is considering modifying its AAV immunosuppression regimens in light of recent industry safety events.

    Answer

    Emil Kakkis, Founder, President & CEO, stated he does not expect the CBER leadership change to affect the CRL resolution, as it's a practical process handled by the existing team. Regarding AAV safety, he noted their programs use lower doses and have not seen major issues. While they are exploring enhanced immunomodulation to optimize efficacy, it is not a response to major safety concerns.

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    Sami Corwin's questions to Krystal Biotech Inc (KRYS) leadership

    Sami Corwin's questions to Krystal Biotech Inc (KRYS) leadership • Q2 2025

    Question

    Sami Corwin from William Blair inquired about the European launch, specifically if patients require an initial physician office visit and if there is a patient onboarding goal similar to the U.S. target.

    Answer

    SVP & GM for Europe Laurent Goux confirmed that an initial appointment at a specialty center is customary in Europe. He stated there is no specific patient ramp number communicated, but the strategic goal is to reach 60% of the eligible patient pool within two to three years.

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    Sami Corwin's questions to Uniqure NV (QURE) leadership

    Sami Corwin's questions to Uniqure NV (QURE) leadership • Q2 2025

    Question

    Sami Corwin asked what the FDA is looking for in the NfL biomarker data for it to be considered supportive and whether hospital capacity could be a limiting factor for the commercial launch of AMT-130.

    Answer

    CMO Walid Abi-Saab stated there have been no specific FDA discussions on NfL data requirements but expressed confidence it will support the primary endpoint. CEO Matt Kapusta said that hospital bed capacity is not expected to be a significant launch constraint, as the procedure is short and patients are typically discharged within 24 hours.

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    Sami Corwin's questions to Sarepta Therapeutics Inc (SRPT) leadership

    Sami Corwin's questions to Sarepta Therapeutics Inc (SRPT) leadership • Q1 2025

    Question

    An analyst on behalf of Sami Corwin asked what specific data sets families want to see to gain confidence in ELEVIDYS after the safety event, particularly regarding older, non-ambulatory patients.

    Answer

    President and CEO Douglas Ingram speculated that families need context: understanding the event was one in over 800 patients dosed and that safety signals do not differ by ambulatory status. He stressed the importance of communicating the full efficacy picture, especially the muscle MRI data, which shows the urgency of treatment to prevent irreversible muscle damage. Chief Customer Officer Dallan Murray pointed to new educational tools available on elevidys.com.

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    Sami Corwin's questions to Beam Therapeutics Inc (BEAM) leadership

    Sami Corwin's questions to Beam Therapeutics Inc (BEAM) leadership • Q3 2024

    Question

    Sami Corwin from William Blair & Company inquired about the rationale for expanding the ESCAPE platform to beta-thalassemia and asked about the planned location for its clinical trials.

    Answer

    President Dr. Giuseppe Ciaramella explained that ESCAPE's improved safety profile could make it a viable option for a broader population of beta-thalassemia patients, beyond the most severe cases. He noted that the location for the initial healthy volunteer study (U.S. vs. ex-U.S.) has not been decided and will be based on what is most efficient.

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