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    Gena Wang • Barclays

    Gena Wang's questions to PTC Therapeutics Inc (PTCT) leadership

    Gena Wang's questions to PTC Therapeutics Inc (PTCT) leadership • Q2 2025

    Question

    Gena Wang from Barclays asked for confirmation on Sefiance's IP status, questioning if it relies solely on orphan designation. She also asked if revenue contribution would begin in Q3 2025 and requested more detail on the discussions during the vutiquinone late-cycle review.

    Answer

    CEO Dr. Matthew Klein corrected that Sefiance is protected by a composition of matter polymorph patent extending to 2038, with an expected extension to 2039, not just orphan exclusivity. He confirmed Q3 revenue is expected. Regarding vutiquinone, he said the late-cycle meeting focused on the evidence, with a key question being whether the data for upright stability, though not a pre-specified primary endpoint, is persuasive enough to support approval, especially given the FDA's evolving view on this endpoint.

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    Gena Wang's questions to PTC Therapeutics Inc (PTCT) leadership • Q1 2025

    Question

    An analyst on behalf of Gena Wang asked about the revenue sustainability for Translarna in ex-EU countries and the rationale for using different natural history controls for the 24-month Huntington's disease data.

    Answer

    CBO Eric Pauwels confirmed no revenue disruption for Translarna in ex-EU markets like Latin America. CEO Dr. Matthew Klein clarified for the Huntington's data that a direct comparison was only made for functional measures against the ENROLL-HD registry; the NFL data was contextualized with a reference publication, not a direct comparison.

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    Gena Wang's questions to PTC Therapeutics Inc (PTCT) leadership • Q3 2024

    Question

    Gena Wang of Barclays asked for confirmation of no AdCom for sepiapterin, the procedural steps following a potential negative EU decision on Translarna, an update on the renewal process in Brazil, and manufacturing readiness for AADC gene therapy.

    Answer

    CEO Matthew Klein confirmed no AdCom is expected for sepiapterin. For Translarna, he reiterated the EU timeline is approximate and next steps remain uncertain. He viewed continued bulk orders in Brazil as a positive sign for its independent renewal process. For AADC, he affirmed the company is fully prepared with more than adequate supply for a U.S. launch.

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    Gena Wang's questions to Cytokinetics Inc (CYTK) leadership

    Gena Wang's questions to Cytokinetics Inc (CYTK) leadership • Q2 2025

    Question

    Gena Wang of Barclays asked for more detail on why the MAPLE HCM trial data for aficamtan could be guideline-changing and what magnitude of benefit would be needed to drive first-line use over beta-blockers.

    Answer

    Fady Malik, EVP of R&D, explained that as a rare comparative efficacy trial, MAPLE HCM will illustrate the absolute benefit of aficamtan versus metoprolol, which could lead to a re-evaluation of its place in treatment algorithms beyond a last-line therapy. President and CEO Robert Blum noted full details would be presented at the upcoming ESC congress.

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    Gena Wang's questions to Ultragenyx Pharmaceutical Inc (RARE) leadership

    Gena Wang's questions to Ultragenyx Pharmaceutical Inc (RARE) leadership • Q2 2025

    Question

    Gena Wang of Barclays posed two statistical questions: one on the rationale for the COSMIC trial in osteogenesis imperfecta and the potential impact of its failure if the ORBIT trial succeeds, and another on the alpha split for the Phase III ASPIRE trial's primary endpoints.

    Answer

    Emil Kakkis, Founder, President & CEO, addressed both points. He explained that COSMIC evaluates severely affected young patients on bisphosphonates who couldn't enter a placebo trial. For ASPIRE, he clarified that splitting the primary alpha (90% for Bayley, 10% for MDRI) provides a more secure statistical design, ensuring the trial can succeed even if the Bayley endpoint alone does not hit, as it is not dependent on a sequential hierarchy.

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    Gena Wang's questions to Ultragenyx Pharmaceutical Inc (RARE) leadership • Q1 2025

    Question

    Gena Wang posed two questions: first, about the potential impact of a recent CBER nomination on the rare disease space, and second, whether skipping the bisphosphonate washout period for late-enrolling OI patients could negatively affect the placebo arm's fracture rate.

    Answer

    CEO Emil Kakkis addressed the CBER nomination by acknowledging industry concerns but expressed confidence in UX111's strong clinical data. Regarding the OI trial, both Kakkis and CMO Eric Crombez explained that the bisphosphonate washout effect would be present in both arms, but the transformative effect of setrusumab would far outweigh it. An executive added this factor was not included in the original power calculations and could represent a potential upside for the study's success.

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    Gena Wang's questions to Ultragenyx Pharmaceutical Inc (RARE) leadership • Q4 2024

    Question

    Huidong Wang, on behalf of Gena Wang, asked about the specific timing of the second interim OI update, the communication plan if it fails, and how quickly data would be shared if it succeeds.

    Answer

    CEO Emil Kakkis confirmed a mid-year timeframe for the interim analysis but did not commit to a specific month. He explained that if the analysis is successful, the company would be unblinded and could release top-line results relatively quickly, faster than after the first interim analysis.

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    Gena Wang's questions to BioCryst Pharmaceuticals Inc (BCRX) leadership

    Gena Wang's questions to BioCryst Pharmaceuticals Inc (BCRX) leadership • Q2 2025

    Question

    Gena Wang from Barclays requested an update on Q2 launch metrics like reimbursement and retention rates, the timing for data from two key pipeline assets, and the reasoning behind CEO Jon Stonehouse's planned transition.

    Answer

    President & CCO Charlie Gayer confirmed the one-year patient retention rate is stable at 60%. Chief R&D Officer Dr. Helen Thackray stated that data for both the Netherton syndrome and DME programs remain on track for year-end. CEO Jon Stonehouse explained his retirement timing is personal, as he is turning 65 after nearly 19 years with BioCryst, and that the board's multi-year succession plan led to the unanimous selection of Charlie Gayer as the next CEO.

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    Gena Wang's questions to Alnylam Pharmaceuticals Inc (ALNY) leadership

    Gena Wang's questions to Alnylam Pharmaceuticals Inc (ALNY) leadership • Q2 2025

    Question

    Gena Wang from Barclays sought to clarify if over half of the 1,400 patients treated so far were first-line and asked how many of these patients received free drugs through assistance programs.

    Answer

    CCO Tolga Tanguler described the patient mix as 'broad and balanced' but did not provide a specific percentage split, reiterating the goal is to establish Amvuttra as the first-line choice. CFO Jeff Poulton confirmed that the use of the Quick Start (free drug) program was 'de minimis' and very low.

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    Gena Wang's questions to Alnylam Pharmaceuticals Inc (ALNY) leadership • Q3 2024

    Question

    Gena Wang asked about the ALN-HTT02 program for Huntington's disease, questioning if a 50% knockdown is the benchmark and what other endpoints will be assessed in the Phase I study.

    Answer

    Chief Medical Officer Pushkal Garg detailed that the Phase I study for ALN-HTT02 will assess safety, tolerability, PK/PD, and changes in mutant Huntington levels, along with biomarkers like neurofilament light chain (NFL). He highlighted the program's unique targeting of the exon 1 fragment, which could offer distinct efficacy, and noted they are proceeding with the study despite a dose cap in the U.S.

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    Gena Wang's questions to BioMarin Pharmaceutical Inc (BMRN) leadership

    Gena Wang's questions to BioMarin Pharmaceutical Inc (BMRN) leadership • Q2 2025

    Question

    Gena Wang of Barclays asked about the BMN-333 program, specifically the target Area Under Curve (AUC) in patients, any upper safety limits, and the historical AUC comparison between VOXZOGO's low and high doses.

    Answer

    Gregory Friberg, EVP and Chief R&D Officer, explained that they anticipate selecting doses for the patient study that are at or above the 3x AUC level. He noted that calculating a precise AUC for the original VOXZOGO studies is difficult due to its very short half-life (minutes vs. days for BMN-333), making a direct, meaningful comparison challenging. The final dose selection for BMN-333 will occur after the current study is complete.

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    Gena Wang's questions to Legend Biotech Corp (LEGN) leadership

    Gena Wang's questions to Legend Biotech Corp (LEGN) leadership • Q1 2025

    Question

    Gena Wang of Barclays PLC inquired about the CARVYKTI price difference between the U.S. and ex-U.S. markets, the FDA approval process for the new Raritan facility expansion, and the expected timeline for data from pipeline assets.

    Answer

    Alan Bash, President of CARVYKTI, stated the U.S. vs. ex-U.S. price differential is approximately 30%. Guowei Fang, President of R&D, and Mythili Koneru, Chief Medical Officer, detailed upcoming clinical readouts for the in vivo CAR-T and allogeneic platforms, as well as for Claudin 18.2 and DLL-3 programs later in the year. CEO Ying Huang added that the Raritan expansion will use the CB30 pathway, expecting on-time approval without delays.

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    Gena Wang's questions to Legend Biotech Corp (LEGN) leadership • Q4 2024

    Question

    On behalf of Gena Wang, an analyst asked for a breakdown of CARVYKTI revenue between second/third-line patients versus later-line patients and for more color on market demand, including physician capacity.

    Answer

    Executive Alan Bash stated that the company has seen strong uptake for the second- to fourth-line indication, with nearly 60% of usage already converted to this earlier-line population. He highlighted positive receptivity to the clinical profile. Executive Ying Huang added that the combined field forces are targeting 8,000 hematologists in 3,000 clinics to drive deeper penetration into the community setting.

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    Gena Wang's questions to Sarepta Therapeutics Inc (SRPT) leadership

    Gena Wang's questions to Sarepta Therapeutics Inc (SRPT) leadership • Q1 2025

    Question

    Tony, on behalf of Gena Wang, asked for color on payer coverage amid suggestions some are not covering ELEVIDYS, and requested an update on the clinical hold in the EU.

    Answer

    President and CEO Douglas Ingram stated that while some payers always require more work, ELEVIDYS policies are generally very good, and their ultimate success rate in gaining access is currently 100%. Head of R&D Dr. Louise Rodino-Klapac said the EU restart requires a substantial amendment with a typical 95-day review, so approval to restart is expected around the end of summer, noting the trial continues to enroll outside the EU.

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    Gena Wang's questions to Intellia Therapeutics Inc (NTLA) leadership

    Gena Wang's questions to Intellia Therapeutics Inc (NTLA) leadership • Q3 2024

    Question

    A representative for Gena Wang asked about Intellia's assumptions for the TTR silencer drop-in rate in its ATTR study and the strategies to manage this if the rate is higher than anticipated.

    Answer

    CMO David Lebwohl stated that this possibility has been factored into the trial design. He believes a high drop-in rate is unlikely based on recent competitor data, which suggests payers would not cover both a silencer and gene editing therapy. He does not expect potential switches to affect the primary endpoint.

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