Suzanne van Voorthuizen

Suzanne van Voorthuizen asked about empasiprubart (EMPA) in MMN, specifically how argenx navigated risks introduced by the dosing regimen change between Phase 2 and 3, the head-to-head study design, and the setting of the non-inferiority margin.

Answer

Luc Truyen, Chief Medical Officer, explained that the Phase 3 dosing regimen was chosen based on modeling exposure-response relationships from ADHERE data. The head-to-head design was selected over a placebo-controlled study due to the progressive nature of MMN and the high likelihood of rescue therapy. The non-inferiority margin was determined using available IVIG grip strength data, with confidence to meet or exceed IVIG's efficacy given EMPA's continuous grip strength improvement.

Suzanne van Voorthuizen asked about empasiprubart (empa) in MMN, specifically how argenx navigates risks from dosing regimen changes between phase II and III, the head-to-head design, comfort with non-inferiority, and the non-inferiority margin setting.

Answer

Luc Truyen, CMO, explained the dosing regimen was chosen based on modeling exposure-response from ADHERE data. The head-to-head design against IVIG was chosen to avoid many rescue events in a placebo-controlled study. The non-inferiority margin was determined using available IVIG grip strength data. He expressed confidence in meeting, and potentially beating, IVIG due to continuous grip strength improvement seen with empasiprubart.

Suzanne van Voorthuizen asked about the relative positioning of empasiprubart (empa) versus VYVGART in CIDP, given their different pivotal trial designs, and the respective commercial opportunities for each molecule.

Answer

Chief Executive Officer Tim Van Hauwermeiren described CIDP as a heterogeneous disease where pathogenic IgGs, targeted by VYVGART, drive disease in about 70-75% of patients. He noted there is a suspicion that complement, targeted by empa, is at play in other patients. The strategy is not to niche empa in a refractory setting but to give it the fullest chance of success with a head-to-head trial against IVIg, letting the data determine its position in a market large enough for multiple innovative molecules.

Suzanne van Voorthuizen inquired about the long-term strategy for the FcRn franchise, the potential shift in focus to the next-generation molecule ARGX-213, and whether development timelines for it could be shortened by leveraging VYVGART's experience.

Answer

CEO Tim Van Hauwermeiren described FcRn as a franchise opportunity too large for a single molecule. He explained that ARGX-213 provides optionality to either 'roll up' existing indications with faster development by leveraging existing knowledge, or to open new indications with different pricing. The immediate focus is on generating Phase I data.

Cara Montiania, on behalf of Suzanne van Voorthuizen, asked for a comparison between the discontinued ANCA vasculitis (AAV) program and the newly prioritized systemic scleroderma (SSc) program.

Answer

CEO Tim Van Hauwermeiren explained that while the opportunities are comparable in size, SSc offers a more straightforward clinical development path. He noted that AAV, despite strong biology, was discontinued due to the unmanageable risk of confounding effects from mandated high-dose steroid background medication, a challenge not present to the same degree in SSc.

Suzanne van Voorthuizen asked if a Phase 1/2 data update for petosemtamab in head and neck cancer should be expected at a medical conference this year, particularly regarding mature frontline data since ASCO last year, and what expectations should be for duration, metrics, and survival.

Answer

Chief Development Officer Judith Klimovsky reinforced that the last readout for the petosemtamab-pembrolizumab combination, presented at ASCO 2025 with approximately 15 months of follow-up, showed 79% of patients with 12-month overall survival. She indicated that this presentation from Merus is already very informative regarding the probability of success for the Phase 3 trials, suggesting sufficient data is available.

Suzanne van Voorthuizen asked whether a Phase I/II data update for petosemtamab in head and neck cancer should be expected this year, especially regarding mature frontline data since ASCO last year, and what expectations should be on duration metrics and survival.

Answer

CEO Jan van de Winkel and Chief Development Officer Judith Klimovsky responded. Klimovsky reinforced that the last readout for the petosemtamab/pembro combination (ASCO 2025 presentation from Merus) was very informative, with around 15 months follow-up and 79% overall survival at 12 months. She indicated that this existing data is sufficient for market understanding ahead of Phase III readouts and did not commit to a new Phase I/II update.

Suzanne van Voorthuizen of VLK asked for more details on the tisotumab vedotin (Tivdak) data set in head and neck cancer to be presented at ASCO. She inquired about the drug's potential in this indication and what to expect from the update regarding sample size, efficacy, and follow-up.

Answer

Chief Development Officer Judith Klimovsky clarified that the upcoming ASCO presentation is based on a Phase II study and will be a more substantial data set with longer follow-up compared to the initial data presented at ASTRO. She reiterated that the study population consists of patients with head and neck cancer who have failed standard-of-care therapies, including PD-1 inhibitors, chemotherapy, and cetuximab.

Suzanne van Voorthuizen asked for details on preliminary commercial plans for AMT-130 in the U.S. and inquired about the company's current thinking on a potential European filing or ex-U.S. partnership.

Answer

CEO Matthew Kapusta expressed excitement about the commercial potential for AMT-130 as a first-in-class, disease-modifying therapy. He confirmed the current focus is on U.S. regulatory interactions but they will engage with European regulators. He acknowledged significant strategic interest from large pharma and biotech, stating uniQure will always act in the best interest of shareholders.

On behalf of Suzanne van Voorthuizen, a question was asked about what to expect from the petrelintide Phase IIb top-line data release, what would be considered a good result, and if body composition would be measured.

Answer

CEO Adam Steensberg defined a 'good result' as data supporting a 15-20% weight loss profile for Phase III, highlighting that the tolerability profile and trajectory are as crucial as the absolute weight loss figure. Chief Medical Officer David Kendall confirmed that body composition will be measured by MRI to assess fat mass and that clear dose separation would be a key success factor for designing the Phase III program.

Suzanne van Voorthuizen asked for a mechanistic explanation for the heart rate reduction seen with petrelintide and for a comparison with AstraZeneca's amylin data, focusing on the reasons for differing efficacy and adverse event profiles.

Answer

CEO Adam Steensberg and CMO David Kendall responded. Dr. Kendall speculated the heart rate reduction could stem from increased vagal tone, a positive differentiator from GLP-1s. Regarding the AstraZeneca asset, Mr. Steensberg noted its shorter half-life and different receptor profile. Both executives reiterated their confidence in petrelintide's best-in-class potential, citing its balanced signaling and longer half-life as key advantages for tolerability and efficacy.

Suzanne van Voorthuizen from Van Lanschot Kempen asked for the mechanistic explanation behind the heart rate reduction observed with petrelintide. She also questioned what factors, such as receptor potency or half-life, might explain the different risk-benefit profile seen in AstraZeneca's amylin data compared to petrelintide.

Answer

President and CEO Adam Steensberg highlighted that avoiding the heart rate increase seen with GLP-1s is a key benefit. Chief Medical Officer David Kendall speculated that the heart rate reduction could be linked to increased vagal tone but stated the exact mechanism is unknown. Regarding the competitor asset, he pointed to petrelintide's longer half-life and balanced amylin-calcitonin signaling as key differentiators that improve tolerability and metabolic effects, giving them confidence in their best-in-class approach.

Samuel, on behalf of Suzanne van Voorthuizen from Van Lanschot Kempen, asked for color on the Lyme disease infection rate in the Phase III study and whether it's in line with expectations. He also inquired if the first study cohort is receiving a second booster and if data on this will be reported at year-end. Lastly, he asked about the potential business impact of recent news from vaccine policymakers.

Answer

CEO Thomas Lingelbach stated that while the company monitors Lyme cases on an ongoing basis and has seen a consistent profile, he would not comment on specific numbers but noted no concerns. He confirmed the first cohort is not receiving a second booster this season. Regarding vaccine policy news, Lingelbach commented that it is too early to speculate on the potential impact of political dynamics in the U.S. on the vaccine market and that the company will wait for more defined policies.

Representing Suzanne van Voorthuizen of Van Lanschot Kempen, Samuel asked for an update on the infection rate in the Lyme Phase III study, whether the first cohort is receiving a second booster, and for commentary on how recent news from vaccine policymakers might affect Valneva's business.

Answer

CEO Thomas Lingelbach stated that while Lyme cases are monitored, the company will not comment on specific numbers but noted the case profile has been consistent and raises no concerns. He confirmed the first cohort is not receiving a second booster at this time. Regarding policy, he commented that it is too early to speculate on the potential impact of political dynamics in the United States on the vaccine market.

Suzanne van Voorthuizen requested clarification on the provision for contractual disputes, asking for the exact amount, whether more charges are expected, and if it relates to ongoing patent disputes with other mRNA companies.

Answer

CFO Jens Holstein confirmed the 'other operating result' reflects provisions for contractual disputes with licensors and collaborators. He stated that due to the ongoing legal situation, he could not provide precise details but confirmed the company has accrued approximately €600 million year-to-date, which they believe is an accurate amount at this time.

Suzanne van Voorthuizen sought clarification on the duration data to be included in the Q2 HB-200 update, the target conference for a later presentation, and the company's broader business development strategy for future partnerships.

Answer

Chief Medical Officer Katia Schlienger clarified that while a median duration of response is unlikely by the Q2 update, they will report on objective response rates, disease control, safety, and immunogenicity, with a future conference yet to be decided. CEO Joern Aldag discussed partnership strategy, stating that while they aim to retain program value, they remain open to new collaborations that could provide capital, given current market conditions.

Suzanne van Voorthuizen of Kempen & Co asked about the specific benchmarks for success for the newly added third arm of the HB-101 trial, which enrolls lower-risk, CMV-positive patients.

Answer

CMO Igor Matushansky responded that for this cohort, the company is looking for an intra-patient increase in CMV-specific antibody and CD8 T cell responses above baseline. He specified that a two- to three-fold improvement over baseline would be considered a good barometer for success.