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President and CEO Ian Mortimer stated that while they are confident in the Kv7 mechanism for MDD, the Biohaven data will be the first placebo-controlled results for that specific molecule. Chief Commercial Officer Darren Cline identified the main commercial headwind as overcoming the inertia of physicians accustomed to existing therapies, despite the significant opportunity for a novel mechanism after a long period without new branded launches.

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Fady Malik, EVP of R&D, detailed significant differences. In ACACIA, patients can down-titrate without interruption if LVEF is above 40%, and only interrupt for one week if it drops below 40%. This contrasts with the competitor's four-week interruption protocol. He also noted differences in dose ranges and patient selection.

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Robert I. Blum, President and CEO, and Fady Malik, EVP of R&D, clarified that the EMA uses a Risk Management Plan (RMP), not a REMS. An RMP was submitted as a standard part of the MAA filing, and they do not anticipate any issues or additional submissions related to it based on the day 120 questions received.

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EVP & CCO Andrew Callos estimated the current HCM diagnosis rate is about 30% and could reach 50% in the next 3-5 years, noting that nonobstructive HCM is growing at a double-digit rate. President & CEO Robert I. Blum added that the amyloidosis and pulmonary arterial hypertension markets serve as good proxies for how a next-in-class drug can increase diagnosis and category penetration.

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COO Ananth Sridhar explained that given the limited efficacy of the current standard of care, any successful study would be a 'home run.' He defined a clinically meaningful result as a majority of patients achieving concomitant normalization of both blood and urine calcium, noting that 69% of patients in the Phase 2 study achieved this endpoint versus zero on standard of care.

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CEO Neil Kumar agreed that the market is ramping faster than historically, driven by increased physician education and screening from multiple competitors entering the space. He stated the market could easily become a $20 billion opportunity and that he expects the high rate of new diagnoses to continue. VP of Strategic Finance Chinmay Shukla added that high-volume heart failure clinics are a key source of new diagnoses.

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CFO Ramón Zapata clarified that while the BMS collaboration is beneficial, overall R&D investments are set to increase in the second half of the year. He stated that spending will be focused on priority late-stage programs, including mRNA cancer immunotherapies and ADCs, while spending on non-priority areas will decrease.

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Chief Medical Officer Özlem Türeci called the competitor's data encouraging as it validates the VEGF/PD-L1 bispecific concept. However, she stated it has no direct impact on their Rosetta Lung-02 trial, which evaluates BNT327 in combination with chemotherapy in a different population, but viewed the news as a positive signal for the drug class overall.

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CMO Özlem Türeci confirmed the data would include Progression-Free Survival (PFS) and emerging Overall Survival (OS) data, though not median OS. Both she and CSO Ryan Richardson identified the benchmark comparator as the Tecentriq plus chemotherapy regimen from the IMpower133 trial.

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CEO Dr. Ugur Sahin explained that the current combination trial is designed to explore the safety profile, particularly whether there is additive toxicity, as the Trop-2 ADC is associated with stomatitis. He noted they do not expect other overlapping toxicities due to BNT327's favorable safety profile and will use a dose escalation approach to find a safe and effective dose.

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President & Chief Scientific Officer Dr. George Yancopoulos argued that the competitor data does not suggest an advantage and it will be a long time before any competitor can match Dupixent's extensive safety profile, which targets a largely non-essential immune pathway. CEO Dr. Leonard Schleifer added that Dupixent's ability to treat multiple comorbidities is a key commercial advantage.

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President & CSO Dr. George Yancopoulos argued that current OX40 data offers no clear advantages and emphasized Dupixent's superior and extensive safety profile, which targets a specific pathway unlike broader immunomodulators. President & CEO Dr. Leonard Schleifer added that Dupixent's ability to treat multiple common co-morbidities provides a significant commercial advantage.

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President and CSO, Dr. George Yancopoulos, stated that an overall survival benefit is not critical as a primary endpoint due to few events in this early-stage population, though the FDA will review the trend. EVP of Commercial, Marion McCourt, estimated the U.S. market opportunity at approximately 10,000 patients.

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President and CEO Dr. Leonard Schleifer reiterated that the company does not comment on the specific mechanics or timing of share repurchases. Regarding a dividend, he repeated prior commentary that the most opportune time to consider it would be after the Sanofi development balance is paid off, which is anticipated around the end of 2026.

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Gregory Friberg, EVP and Chief R&D Officer, explained that while short-term growth increases with such combinations are not surprising, the long-term persistence of this effect is unproven in achondroplasia, which is not a growth hormone-deficient condition. He noted that long-term follow-up on growth hormone alone has shown minimal final height benefit and lacks the broader health improvements, like facial morphology changes, seen with CNP therapy.

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Cristin Hubbard, Chief Commercial Officer, explained that the revenue split fluctuates quarterly but is currently around 75% ex-U.S. and 25% U.S. She stated that in the long term, the split is expected to normalize and mirror BioMarin's overall portfolio, which is approximately two-thirds of revenue from outside the U.S. and one-third from the U.S.

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Chief R&D Officer Gregory Friberg explained that the 25-week data will provide a 'clear line of sight' on whether the 10% target is achievable at steady state, based on their predictive models. He emphasized this time point will be a very solid read on their trajectory and noted that they are also advancing a higher 9 mg/kg dose cohort, providing another path to achieving their goal.

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EVP, Worldwide R&D Greg Freiberg explained that while the company is tracking final adult height data as part of its post-marketing commitment, it will not release specific numbers yet. Regarding the DMD program, he stated that the company will have dystrophin data from the first six patients in-house mid-year and will decide on public release based on the quality and meaningfulness of the data.

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CEO Kyle Gano clarified that the number of patients transitioning from the adult OLE is very small, totaling less than 40 patients. He explained that these transitions are split between Q2 and Q3, making their impact 'de minimis' relative to the more than 600 new patient starts in the quarter.

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Chief Financial Officer Matthew Abernethy confirmed there was very little inventory stocking for CRENESSITY in the quarter. Chief Medical Officer Dr. Eiry Roberts stated that one-year data from the open-label extension, covering androgen control, glucocorticoid levels, and other clinical endpoints, will be presented at upcoming medical meetings.

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Chief Commercial Officer Eric Benevich explained that CRENESSITY is initially non-formulary, so a 'Fast Start' program provides free product while reimbursement is secured, a process expected to take one to two months for most patients early in the launch. Chief Financial Officer Matthew Abernethy added that early signs from payers are positive, citing a recent favorable coverage policy from a major plan.

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CCO Eric Benevich estimated that the dozen or so CAH Centers of Excellence manage about 10-15% of the total patient population, with the majority being cared for in community and pediatric endocrinology practices. CMO Eiry Roberts added there is significant interest in the pediatric setting, where managing androgen levels is critical for normal growth and development.

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Executive Dave Gancarz deferred commentary on HARMONi-2 OS subgroup data until their partner Akeso releases it. Chief Medical Officer Dr. Allen Yang addressed safety, stating they are pleased with the profile from randomized trials and do not expect significant differences between China and the U.S., aside from minor cultural variations in reporting lab abnormalities. Dr. Jack West, VP and Thoracic Oncology TA Head, added that the most concerning adverse events are not subject to nuanced interpretation and the data seen to date is very reassuring to clinicians.

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Executive Dave Gancarz deferred comment on HARMONi-2 OS subgroups to their partner, Akeso. Chief Medical Officer Dr. Allen Yang stated he does not expect significant safety differences between China and global populations, noting only minor cultural differences in reporting non-impactful lab abnormalities. Dr. H. Jack West, VP, added that serious adverse events are not subject to nuanced interpretation and the data seen so far is reassuring.

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President Stephen Hoge stated that the exact procedural path is not yet determined and will depend on consultations with the FDA after the flu data is finalized. He noted that while an amendment to the existing BLA is a pragmatic option, a resubmission is also possible. The review of other components of the file is already progressing.

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CFO Jamey Mock clarified there was no change to expense guidance, explaining the potential confusion between GAAP costs and the company's defined 'cash costs.' President Stephen Hoge confirmed that a reduction in MDEs is the pivotal endpoint for the PA program, as agreed with regulators, and the trial will compare MDE rates before and after treatment.

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