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David Chang, CEO, stated that the MRD testing window is well-defined and not expected to significantly impact outcomes. Zachary Roberts, CMO, added there is no data suggesting timing would influence EFS. For the efficacy bar, Dr. Roberts referenced the approximately 30% efficacy delta seen in second-line CAR T trials as the benchmark, noting ALPHA-three is pioneering this prospective analysis.

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CEO Nello Mainolfi and CMO Jared Gollob explained that if conjunctivitis is a true on-mechanism effect of IL-4/13 pathway blockade, they would expect to see it. They noted it hasn't appeared in preclinical or healthy volunteer studies and have no reason to expect a different rate than dupilumab. Nello Mainolfi added that he doesn't see why the pathway blockade would be different from an antibody and highlighted KT-621's exceptional tolerability in long-term tox studies.

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CEO Nello Mainolfi confirmed that several time points will be measured but cautioned against over-relying on healthy volunteer biomarker data for dose selection, citing dupilumab's data as an example. He reiterated that the key criteria for dose selection are robust STAT6 degradation (90%+) and a safe, well-tolerated profile.

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Interim CFO Jessie Yeung confirmed that earlier-line treatment improves the safety profile, with Parkinsonism rates dropping from 6% in CARTITUDE-1 to 1% in CARTITUDE-4, largely due to better bridging therapy options. President of CARVYKTI Alan Bash added that earlier treatment also leads to fitter T-cells, lower out-of-spec rates, and better efficacy. CEO Ying Huang noted the percentage incidence rate of neurotoxicities is decreasing.

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Alan Bash, President of CARVYKTI, confirmed the Tech Lane facility is on track for commercial production by year-end, supporting European launches, and mentioned a joint $150 million investment for further capacity. Guowei Fang, President of R&D, detailed the in vivo CAR-T platform's advantages, including better T-cell phenotype, an off-the-shelf approach without lymphodepletion, and scalable manufacturing.

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Executive Jessie Yeung noted that bispecifics are approved in later lines and their trials exclude prior BCMA agents, reinforcing the IMWG recommendation for CAR-T first in eligible patients. Executive Ying Huang emphasized CARVYKTI's unmatched benefits, including the depth and durability of response and the convenience of a one-time treatment that provides a long treatment-free period and improved quality of life.

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CBO Eric Pauwels explained that access speed for Sefiance depends on the patient's profile, with previously documented failures potentially moving through payer approvals more quickly. CFO Pierre Gravier confirmed the $650M-$800M guidance includes new product launches, with the upper end allowing for potential upside. CEO Dr. Matthew Klein reported a constructive late-cycle meeting for vutiquinone, where the FDA confirmed the application is under active review.

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CEO Dr. Matthew Klein noted Sephience shows superior phenylalanine lowering even in patients on existing therapy. CBO Eric Pauwels added that payer research indicates they view Sephience as highly differentiated and superior, with diet liberalization being a key factor. He anticipates minimal reimbursement hurdles due to the large untreated or failed-treatment patient population.

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CEO Matthew Klein highlighted that the strong Q3 results prompted an increase in the full-year 2024 revenue guidance to $750-$800 million. He anticipates continued strong contributions in Q4, with Emflaza performing well against generic competition and Translarna revenue expected to be stable, though subject to typical quarterly lumpiness.

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President & CEO Jan Møller Mikkelsen noted that while they lack precise real-world data on titration duration, the process appears successful given low patient dropout rates. He clarified that the current U.S. approval is for doses up to 30mcg. A trial to support a 60mcg dose has been initiated, but it is too early to comment on the pricing or reimbursement for a potential higher dose.

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President and CEO Jan Mikkelsen clarified that patient severity (e.g., controlled vs. uncontrolled) is not a factor in reimbursement discussions. He emphasized that the primary driver for uptake is the profound personal benefit patients experience, rather than long-term health economic outcomes. Chief Medical Officer Aimee Shu added that existing literature on the benefits of reducing conventional therapy might preclude the need for a new trial.

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CEO William Lewis affirmed that the initial market size estimate of 250,000 patients is based on data showing documented exacerbations. He highlighted the larger, untapped opportunity in patients with comorbid COPD or asthma who have not yet been diagnosed with bronchiectasis via a CT scan. He believes the availability of an approved treatment will incentivize physicians to conduct more CT scans, thereby expanding the diagnosed population.

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CEO Mark Goldsmith declined to provide specific updates on regulatory interactions, stating it's not typically helpful. However, he emphasized that the company is making very good progress and reiterated the timeline for initiating the trial, implying that necessary steps are being successfully completed.

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Dr. Mark Goldsmith, Chairman and CEO, responded that Tango's compound is interesting and that positive preclinical data from their joint work supported the collaboration. However, he emphasized that this partnership does not preclude future collaborations with other PRMT5 inhibitors, as the company is actively seeking the best combination partners and it is too early to make a final determination.

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Pravin Dugel, Executive Chairman, President & CEO, explained the extension study is a strategic effort to generate long-term data on non-pulsatile dosing and support a superiority claim. He argued that the key to market dominance is not just hitting a specific visual acuity number but achieving a superiority label, which would place axpaxly in a 'different orbit' from competitors and protect it from pricing pressures.

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CCO Steve Closter stated that the average treatment duration is tracking within the projected four-to-six-month range for the first year and is expected to expand to six-to-twelve months in the second year as the drug is used earlier. CFO Keith Goldan clarified expense guidance, indicating that Q3 operating expenses (less non-cash stock comp) are projected to be $95-$100 million, with Q4 expected to be at a similar level.

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President and Head of R&D Dr. Neil Gallagher explained that for unfit patients, FLT3 status is not a selection criterion, but for fit patients, an approved FLT3 inhibitor is typically used first. CCO Steven Closter reported that physician feedback on the overall profile is "great," with safety aspects being well-understood and manageable, supported by the company's clinical nurse educators.

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Chief Medical Officer Dr. Allen Yang confirmed that HARMONi-3 has enrollment objectives for both histologies to ensure sufficient data, with an expected near-equal split between squamous and non-squamous patients. Regarding HARMONi-7, executive Dave Gancarz noted a hazard ratio around or under 0.80 is a key focus. Dr. Jack West added that if the trial is statistically positive as designed, it will be considered clinically meaningful and practice-changing, as clinicians generally look for a 2-3 month OS improvement as a benchmark.

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Chief Medical Officer Dr. Allen Yang confirmed HARMONi-3 has enrollment objectives for both histologies to ensure sufficient data, aiming for a roughly equal split. Executive Dave Gancarz noted the company targets an OS hazard ratio around or under 0.80. Dr. H. Jack West, VP, and Dr. Yang added that if the trial is positive as designed, it will be clinically meaningful and practice-changing, with a 2-3 month OS improvement being a general benchmark for clinicians.

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Dr. Allen Yang, Chief Medical Officer, confirmed the primary analysis for the HARMONi trial is planned for the total study population, although regional differences will be examined. An executive clarified that 'mid-year' refers to a broader Q2/Q3 2025 timeframe. Regarding new trial arms, an executive stated they are exploring all opportunities to accelerate development and believe EV is important in bladder cancer, promising more details in the coming months.

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Wang Lai, Global Head of R&D, explained that both are single-arm Phase II studies where the primary endpoints are Overall Response Rate (ORR) and Duration of Response (DOR), which could support accelerated approval. For the sonrotoclax filing in China, he noted it has received priority review and anticipates a potential approval in the first half of the next year.

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Lai Wang, Global Head of R&D, clarified that for both single-arm Phase II studies, the primary endpoints are Overall Response Rate (ORR) and Duration of Response (DOR), which can support accelerated approval. He added that the sonrotoclax filing in China has received priority review, with a potential approval anticipated in the first half of 2026.

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Steven Stein, an executive, explained that the mild-to-moderate HS patient population can have a notable placebo effect. Using the higher bar of HiSCR 75 as the endpoint helps control for this placebo response, addresses the significant unmet need, and increases the probability of a successful trial outcome. He confirmed this endpoint was agreed upon with regulatory agencies.

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