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Chief Medical Officer Mythili Koneru defined 'high-risk' by cytogenetics and 'functional high-risk' by rapid disease progression after frontline therapy. She noted that upcoming ASCO data will provide subgroup analysis but that both populations do well with CARVYKTI. Alan Bash, President of CARVYKTI, added that physicians often start with these high-risk categories in earlier lines before expanding to a broader patient population.

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Executive Alan Bash stated that while they don't break down centers specifically, the majority of patients (a little over half) are already receiving CARVYKTI in the outpatient setting. Executive Ying Huang addressed the in vivo question, noting it's an early-stage technology with potential advantages and liabilities. He mentioned Legend is also actively pursuing its own differentiated in vivo platform.

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CEO John Leonard explained that the Phase III trial is providing crucial operational learnings. He emphasized that Intellia's commercial team has deep experience with one-time therapies and that NTLA-2002's simple outpatient infusion process is a significant advantage over more complex gene therapies, suggesting a more efficient and straightforward market entry.

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CEO John Leonard acknowledged the gene is driven by the albumin promoter and would not have a typical response to infection. He stated the priority is to normalize the protein level, which is the primary defect in the disease and the main determinant of the product's success.

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President and CEO Douglas Ingram declined to comment on the specifics of ex-U.S. sales, deferring to their partner, Roche. He stated he would be surprised if the decline related to the same cycle time or safety event issues seen in the United States but recommended asking Roche directly for details.

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CEO Douglas Ingram noted that while it's still early days with less than 5% of the addressable population treated with ELEVIDYS, they have seen instances of payers reimbursing for both therapies. He emphasized that there has not been significant net cannibalization of the PMO franchise, which continues to see good growth ex-U.S. Chief Customer Officer Dallan Murray confirmed these points.

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President and CEO Doug Ingram stated that the company is in a very good position with its manufacturing capacity to serve both its own and its partner Roche's needs. Regarding the suspension process, he confirmed it is proceeding well with a bridging study planned for 2025, but a decision on a commercial supplier has not yet been made.

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Greg Friberg, Chief R&D Officer, differentiated the two drugs by explaining the difficulty in capturing VOXZOGO's pulsatile PK profile versus the expected continuous exposure from BMN 333. He argued that rather than a direct comparison, BMN 333's potential is modeled on preclinical data from other long-acting CNPs, with the goal of testing if sustained, higher exposure leads to better outcomes.

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Chief R&D Officer Gregory Friberg reiterated that their current candidate, BMN 349, is focused on the liver manifestation of the disease. He noted that due to its selectivity, the therapy could potentially be used in conjunction with enzyme replacement therapies that address the lung issues, but the company's current R&D strategy is centered on the liver-focused approach.

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EVP, Worldwide R&D Greg Freiberg stated that the company is focused on collecting final adult height data for Voxzogo's full approval. Regarding Palynziq, he positioned it for a different patient segment with higher phenylalanine levels, noting that new options are good for the heterogeneous patient population. He also pointed to a competitor's recent standard FDA review as an objective indicator of its perceived improvement over available therapies.

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President and CEO Craig Tooman explained that Phase III readiness activities are set to complete by mid-year, providing a window for a partner to offer input and make 'fine-tuning' adjustments. Steven Romano, Chief R&D Officer, added that while the current focus for divesiran is polycythemia vera (PV), its mechanism is applicable to other conditions like HH, and the company is actively exploring opportunities to expand the program.

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President and CEO Craig Tooman confirmed that Phase III readiness activities are on track for completion by mid-year, which allows for some flexibility for a partner to provide input on the design. Steven Romano, Chief R&D Officer, added that while the current focus for divesiran is polycythemia vera (PV), they recognize its mechanistic potential in other conditions like HH and are exploring opportunities to expand the program.

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Craig Tooman, President and CEO, explained that Phase III readiness activities are on track to conclude by mid-year, which allows a window for a potential partner to provide input on the trial design. Regarding divesiran, Steven Romano, Chief R&D Officer, confirmed that while the current focus is on polycythemia vera (PV), the company recognizes the drug's mechanism could apply to other conditions like HH and is exploring opportunities to expand the program.

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The company is on track to complete Phase III readiness activities by mid-year, which allows some flexibility for a partner's input on the design. Regarding divesiran, while the current focus is on polycythemia vera (PV), they acknowledge its potential mechanism applies to other conditions like hereditary hemochromatosis (HH) and are exploring expansion opportunities.

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Chief Commercial Officer Tolga Tanguler stated that based on pre-pricing discussions, payers recognize the value of AMVUTTRA and the predictability of its Part B positioning. He expects uptake to be consistent with the past experience in polyneuropathy and does not anticipate significant access hurdles like step-edits.

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CEO Yvonne Greenstreet stated that while the company is privileged to have a rich internal pipeline, their business development focus is on opportunities that enable their RNAi platform and facilitate access to new tissues. The primary focus remains on executing the internal pipeline and ensuring a successful AMVUTTRA launch in cardiomyopathy.

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President Dr. Giuseppe Ciaramella confirmed that no off-target biology of concern has been identified in extensive preclinical work. CEO John Evans described a life-cycle strategy where BEAM-101 is a 'Wave 1' therapy, which he expects would be overtaken by the 'Wave 2' ESCAPE program if it successfully eliminates chemotherapy. He noted ESCAPE could expand the market three- to four-fold and that development synergies between the two programs will accelerate ESCAPE's path to market.

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President Dr. Giuseppe Ciaramella confirmed that extensive preclinical analysis showed no off-target biology of concern. CEO John Evans described the programs as a life cycle strategy, expecting ESCAPE to eventually replace BEAM-101 if its profile is successful. He reiterated that the initial market for BEAM-101 is the ~10% of severe patients already considering transplant.

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President Dr. Giuseppe Ciaramella confirmed no off-target biology of concern was found in extensive preclinical analyses. CEO John Evans described a life-cycle strategy where ESCAPE, if successful, would likely replace BEAM-101 by expanding the market up to fourfold. He emphasized the significant development synergies between the two programs, which accelerate and de-risk ESCAPE.

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