Rajan Sharma

Rajan Sharma of Goldman Sachs asked for the target profile of asfotase alfa (Amphicosalpha) and the residual unmet need it aims to address in the context of the existing therapy, Strensiq.

Answer

CEO of Alexion, Marc Dunoyer, explained that asfotase alfa is being developed for both adult and pediatric populations with a goal of wider geographic coverage than Strensiq. He highlighted a key patient benefit: a less frequent dosing schedule of one injection every two weeks, compared to six or twelve for Strensiq in the same period. He noted Phase 3 results are expected by year-end.

Rajan Sharma asked about the potential filing strategy for the DESTINY-Breast09 (DB09) combination therapy and sought clarification on whether overall survival data from the TROPION-Lung01 QCS biomarker analysis would be presented at ASCO.

Answer

EVP, Oncology R&D, Susan Galbraith stated that discussions with regulators regarding the DB09 filing are ongoing, noting the data's "highly clinically meaningful results" will be influential. She clarified that the upcoming ASCO presentation will feature a QCS biomarker analysis from the TROPION-Lung02 dataset, not TROPION-Lung01, which will provide additional supportive evidence for the biomarker's utility.

Rajan Sharma from Goldman Sachs asked if a cardiovascular outcomes trial is necessary for meaningful uptake of the oral PCSK9 inhibitor and whether a positive PFS result from the AVANZAR trial would be sufficient for a conditional approval in lung cancer.

Answer

Sharon Barr, EVP of BioPharmaceuticals R&D, deferred comment on specific outcomes trial plans for the oral PCSK9, noting it's still early in development. Susan Galbraith, EVP of Oncology R&D, explained that for a first-line lung cancer filing based on PFS, regulatory agencies generally also want to see a positive trend in overall survival and reasonable data maturity.

Rajan Sharma inquired about the implications of recent competitor data for Wainua's CARDIO-TTRansform trial and asked about the market dynamics and patient sources for Ultomiris in myasthenia gravis.

Answer

EVP, BioPharmaceuticals R&D, Sharon Barr, said that positive competitor data validates the drug's mechanism, increasing confidence in the ongoing CARDIO-TTRansform study. EVP, Rare Disease, Marc Dunoyer, explained that the myasthenia gravis market is highly dynamic and that Ultomiris's strong 36% growth was driven predominantly by its neurology indications, a trend he expects to continue.

Rajan Sharma from Goldman Sachs inquired about the growth drivers for VYVGART, specifically asking if IV and other formulations were still growing alongside the pre-filled syringe (PFS) in gMG and CIDP. He also sought argenx's perspective on Sjögren's disease, recent Novartis ianalumab Phase III data, and VYVGART's potential differentiation and clinically meaningful signals in this indication.

Answer

Karen Massey, Chief Operating Officer, confirmed that while the PFS is the major growth driver, the IV formulation continues to be an important contributor to VYVGART's performance in gMG. Tim Van Hauwermeiren, Chief Executive Officer, acknowledged the positive Novartis data for Sjögren's, noting a 2-point improvement as clinically meaningful, and expressed conviction in efgartigimod's precision tool approach for the disease.

Rajan Sharma inquired about the growth drivers for VYVGART, specifically if the IV and other formulations are still growing alongside the pre-filled syringe (PFS) in gMG and CIDP. He also sought insights on Sjogren's disease, the recent Novartis Ianalumab Phase 3 data, potential differentiation for VYVGART, and what constitutes a clinically meaningful signal in this indication.

Answer

Karen Massey, Chief Operating Officer, confirmed that while the pre-filled syringe is the major growth driver, the IV formulation continues to be an important contributor in gMG. Tim Van Hauwermeiren, Chief Executive Officer, welcomed the Novartis data for Sjogren's, noting a 2-point improvement as clinically meaningful and expressed strong conviction in efgartigimod's precision approach based on Phase 2 data.

Speaking for Rajan Sharma from Goldman Sachs, an analyst asked if the near doubling of global CIDP patients on Vyvgart (to 2,500) between January and June 2025 serves as a reliable proxy for the growth rate for the remainder of the year.

Answer

COO Karen Massey confirmed the 2,500 global patient number reflects strong momentum that is expected to continue. She highlighted that this figure includes fast starts in newly launched markets like Japan and Germany, demonstrating a significant unmet need for innovation in CIDP globally, which supports continued growth.

Rajan Sharma asked for clarification on the relative magnitude of the headwinds to Q1 revenue growth, specifically between typical seasonality (reverification) and the impact from the Medicare redesign.

Answer

Chief Financial Officer Karl Gubitz explained that while the Medicare redesign accelerated the shift to Part D and increased the gross-to-net, the majority of the Q1 impact was due to typical industry seasonality related to patient benefit reverification. Chief Operating Officer Karen Massey reinforced this, noting the dynamic was similar to the previous year and that the company expects continued growth throughout the year, driven by the PFS launch.

Rajan Sharma asked for an update on the myasthenia gravis (MG) market expansion, specifically how much of the potential 25,000-patient market growth has been realized since the 2021 launch.

Answer

Chief Operating Officer Karen Massey responded that while market expansion is occurring, they are still in the early stages of the growth curve. She cited the fact that 60% of new VYVGART patients come directly from oral therapies as evidence of penetrating this expanded market, but believes there is still a long way to go.

Rajan Sharma inquired about the regulatory steps, timeline, and potential for a faster review process for the VYVGART prefilled syringe (PFS) following recent data collection.

Answer

CEO Tim Van Hauwermeiren confirmed that Argenx has successfully collected all necessary bioequivalence and human factors study data for the PFS. He stated the company is on track to submit the dossier to the FDA by the end of June, expressing confidence in the data's quality while noting the final review timeline is at the FDA's discretion.

Rajan Sharma of Goldman Sachs questioned the confidence in a 2025 Lyme disease data readout given a 2026 completion date on clinicaltrials.gov. He also asked about the efficacy bar for success and approvability compared to previous Lyme vaccines.

Answer

CEO Thomas Lingelbach clarified that the clinicaltrials.gov date includes follow-up periods and that Pfizer, who controls the process, reaffirmed the 2026 submission timeline. He stated there is no pre-aligned efficacy bar with regulators but noted that the trial was designed for success with a 3+1 dose schedule, and direct comparisons to older vaccines are challenging due to different study designs.

Rajan Sharma from Goldman Sachs asked for clarification on the $143 million milestone from Pfizer for the Lyme vaccine, questioning if it's tied to Phase III results or commercial approvals. He also sought Valneva's perspective on the potential impact of tariffs on its business and manufacturing footprint.

Answer

CFO Peter Buhler clarified that the $143 million milestone is tied to commercialization, not Phase III results. It is split into three parts triggered by first commercial sales in the U.S., first commercial sales in Europe, and an ACIP opinion, all expected in 2027. He also mentioned an additional $100 million in commercial milestones and royalties of 14-22%. Regarding tariffs, Buhler noted the company is monitoring the situation but it's too early to assess the impact. He confirmed Valneva's vaccines are manufactured in Europe (Scotland, Germany, Sweden), with IP also in Europe.

Rajan Sharma from Goldman Sachs inquired about the timing and triggers for the $143 million milestone payment from Pfizer for the Lyme vaccine and asked for Valneva's perspective on the potential impact of tariffs on its manufacturing operations.

Answer

CFO Peter Buhler clarified that the $143 million milestone is tied to the first commercialization of the Lyme vaccine, expected in 2027, and is triggered by first sales in the U.S. and Europe, plus an ACIP opinion. He also noted further commercial milestones and royalties. Regarding tariffs, Mr. Buhler stated it is too early to assess the impact but confirmed Valneva's vaccines are manufactured in Europe (Scotland, Germany, and Sweden), which they are monitoring closely.

Rajan Sharma of Goldman Sachs asked about the launch strategy for Epkinley in second-line follicular lymphoma, whether its label would require hospitalization, and the rationale for discontinuing the Hexabody OX40 program.

Answer

Chief Commercial Officer Brad Bailey outlined the launch strategy, focusing on expanding into the community setting. Chief Medical Officer Tahamtan Ahmadi confirmed the label would not require hospitalization and explained the OX40 discontinuation was a portfolio prioritization decision, not a failure of the Hexabody platform. CEO Jan van de Winkel reinforced confidence in the platform.

Rajan Sharma of Goldman Sachs asked about the launch strategy for Epkinley in second-line follicular lymphoma, its potential revenue contribution, and hospitalization requirements. He also questioned the rationale for discontinuing the Hexabody OX40 program and its implications for the platform.

Answer

Brad Bailey, EVP & CCO, outlined the launch strategy, focusing on expanding into the community setting. Tahamtan Ahmadi, EVP & CMO, clarified that no hospitalization is required for the FL label and explained the OX40 discontinuation was a portfolio prioritization decision, not a failure of the platform. CEO Jan van de Winkel reinforced confidence in the Hexabody platform, noting a new asset is advancing to the clinic.

Rajan Sharma of Goldman Sachs asked to reconcile a discrepancy between the company's guided 2026 readout for the EPCORE DLBCL-2 trial and the mid-2027 date listed on clinicaltrials.gov.

Answer

CMO Tahamtan Ahmadi advised that clinicaltrials.gov is not always the most updated source for timelines. He explained that trial readouts are event-driven and accrual for this specific study was significantly faster than projected, potentially leading to an earlier result than the official registry date suggests.

Rajan Sharma of Goldman Sachs asked about the company's confidence in having sufficient U.S. trial sites and representative patient demographics for the TEBY-AM and ATOM trials to satisfy the FDA. He also inquired about Chemtrac's penetration in Europe versus the U.S.

Answer

David Berman, EVP of R&D, expressed confidence, stating they have enough U.S. sites and that most international sites are in regions with the same standard of care. Ralph Torbay, EVP of Commercial, noted that penetration in mature European markets like Germany and France is over 80%, which serves as a guide for the potential in the U.S. as they continue to penetrate the community setting.

Rajan Sharma of Goldman Sachs asked about long-term benchmarks for the atopic dermatitis program and whether the upcoming HIV data could provide a positive cross-read to the HBV program and the platform's broader potential.

Answer

David Berman, Head of R&D, said that for atopic dermatitis, the initial focus is on refractory patients to establish a signal before defining a commercial TPP. He views the HIV and HBV data as a package that will be important for validating the ImmTAV platform in chronic viral diseases. CEO Bahija Jallal added that the CD1a target in atopic dermatitis has potential across multiple indications.

Rajan Sharma from Goldman Sachs asked about the long-term pricing evolution for KIMMTRAK with potential label expansions, whether folate receptor alpha positive patients are included in the ovarian trial, and the potential maximum dose in the HIV trial.

Answer

Ralph Torbay, Head of Commercial, said pricing depends on the benefit shown and expects no significant U.S. price erosion, though Europe is challenging. Mohammed Dar, an executive, confirmed they don't exclude prior folate receptor alpha patients. Regarding the HIV trial, he stated that while the maximum dose is unknown, their platform generally does not require high doses to be effective.

Asked about long-term pricing for KIMMTRAK with new indications, inclusion of folate receptor alpha positive patients in the ovarian trial, and the potential maximum dose for the HIV program.

Answer

U.S. pricing is expected to hold firm with good data, while Europe is uncertain. The ovarian trial does not exclude folate receptor alpha positive patients. The HIV dose will be escalated as needed, but the platform is known to be potent at low doses.

Rajan Sharma from Goldman Sachs asked about the long-term pricing evolution for KIMMTRAK with new indications, whether folate receptor alpha positive patients are included in the ovarian trial, and the potential maximum dose for the HIV program.

Answer

Ralph Torbay, Head of Commercial, expects minimal U.S. price erosion but noted European viability depends on future data. Mohammed Dar, an executive, confirmed folate receptor alpha patients are included and that the HIV program likely won't require high milligram doses, as the platform is potent at microgram levels.

Rajan Sharma requested an update on the launch timeline for Bligepa (Bluejepa) and its expected trajectory, and also asked for the key drivers behind the company's increased R&D expense guidance.

Answer

CFO Julie Brown attributed the higher R&D spend to accelerating the ADC portfolio, particularly B7-H3, and funding four new pivotal trials in H2. CCO Luke Miels stated the Bligepa launch is set for later in the year with a full push in Q1 2026 to align with payer formulary timing, targeting a population of 3 million patients with resistant UTIs.

Rajan Sharma inquired about potential REMS requirements for Blenrep and their influence on uptake, and asked if lower market valuations might spur more business development activity.

Answer

CEO Emma Walmsley confirmed that BD remains a high priority. CSO Tony Wood noted that REMS are common in oncology and the U.K. approval provides a potential model. CCO Luke Miels added that the launch strategy is built around supporting physicians in managing Blenrep's known side effects, leveraging collaborations with eye care professionals to ensure a smooth process.

Rajan Sharma of Goldman Sachs asked about the drivers of the positive channel mix in HIV and its sustainability, and sought to reconcile the £2B uplift in 2031 guidance with Blenrep's £3B peak sales potential, also asking about the key differences versus consensus estimates.

Answer

CEO Emma Walmsley reiterated the 'more than £40 billion' 2031 outlook and confirmed no change to Blenrep's peak sales ambition. Chief Financial and Administrative Officer David Redfern noted the main channel mix headwind in HIV is the rise of 340B, but other trends are stable. CFO Julie Brown explained the guidance delta versus consensus is primarily in Oncology (Blenrep, Jemperli) and RI&I (camlipixant, depemokimab), where the market is likely awaiting further data and launch traction.

Rajan Sharma asked for the rationale behind developing a B7-H3 targeted ANKET (IPH62) with Sanofi, given the increasingly crowded landscape for this target, and questioned why the ANKET platform might be a superior modality compared to an ADC.

Answer

Yannis Morel, Chief Operating Officer, clarified that the program originated from strong preclinical efficacy data that prompted Sanofi to license it. He noted that while the B7-H3 space is competitive, particularly with ADCs, utilizing the ANKET platform provides an alternative and orthogonal immune-based mechanism via NK cells, which could be a valuable way to address the target.

Rajan Sharma asked about the initial development plan for the ADC IPH-45, its potential to differentiate on efficacy and safety, and for more detail on the fast-to-market strategy for Lacutamab in Sezary syndrome.

Answer

Chief Medical Officer Sonia Quaratino explained that the IPH-45 plan will be refined after the initial dose-escalation study but could target tumors with low Nectin-4 expression, not just PADCEV-refractory patients. For Lacutamab, she confirmed that an accelerated approval pathway in Sezary syndrome remains an option, which would require 12-month durability data and an ongoing registrational trial that would also support approval in MF.

Max, on behalf of Rajan Sharma, asked about the expected progression of R&D costs in 2025, the design of the lung cancer trial regarding response assessment, and the significance of the slight decline in mRNA integrity shown in the stability data.

Answer

CSO Myriam Mendila clarified that in the NSCLC trial, efficacy endpoints like tumor shrinkage and PFS will be measured after adding the vaccine to stable patients. She also noted the mRNA stability at room temperature remains within regulatory specifications. CFO Axel-Sven Malkomes added that the company continues to focus on cost efficiency while funding its key pipeline programs.

Rajan Sharma of Goldman Sachs asked about the expected ramp-up of authorized treatment centers to the year-end target of 60, questioning if it would be linear or occur in blocks. He also inquired about the potential impact of tariffs on the product, given its manufacturing in the U.K.

Answer

Executive Christian Itin explained that the center activation has been a 'pretty continuous kind of flow' and he expects a 'reasonably steady process' through the year, driven by individual hospital timelines. On tariffs, Itin stated it is 'way too early' to speculate, but noted that historically, pharmaceutical and blood products are often excluded from such tariffs to avoid impacting medication supply.

Rajan Sharma inquired about the go-to-market strategy for glepaglutide if launched without a partner, the necessity of a head-to-head trial for petrelintide, and whether a gender-based difference in weight loss was observed in the petrelintide trial.

Answer

CEO Adam Steensberg, CCO Eric Cox, and CMO David Kendall addressed the questions. Mr. Cox outlined a focused, rare-disease commercial model for a potential solo glepaglutide launch while partnership talks continue. Mr. Cox and Dr. Kendall agreed a head-to-head trial for petrelintide would be important for differentiation, with timing to be determined post-Phase II. Mr. Steensberg confirmed that while detailed data is pending, they anticipate females will show greater weight loss, consistent with other obesity drugs.

Rajan Sharma of Goldman Sachs inquired about the potential go-to-market strategy for glepaglutide if launched without a partner, including expected SG&A costs. He also asked if a head-to-head trial against GLP-1s is necessary for petrelintide to become a foundational therapy, and if there was a gender-based difference in weight loss in the Phase Ib data.

Answer

President and CEO Adam Steensberg noted that detailed gender-split data for petrelintide was not yet public but expects females to show greater weight loss. Chief Commercial Officer Eric Cox explained that an initial glepaglutide launch would have a focused footprint with a modest SG&A increase, while they actively seek a partner. He and Chief Medical Officer David Kendall confirmed a head-to-head trial for petrelintide is under consideration for Phase III or IIIb, pending dose finalization from the Phase IIb study.