Daniel Bronder

Daniel Bronder from Cantor Fitzgerald asked for an update on the volrustomig plus casdatifan frontline study, a collaboration with AstraZeneca, specifically regarding its paused status and potential re-opening.

Answer

CEO Terry Rosen confirmed the study was paused but patients continued treatment with a reduced volrustomig dose, providing valuable insights. He noted no additional immunities since the dose reduction and no primary progression. Discussions with AstraZeneca are ongoing, and the ARC-20 IPI anti-PD-1 + CAS arm is open to inform Phase III decisions.

Daniel Bronder asked for more insight into the ARV-393 data planned for the second half of 2026, the basis for confidence in the molecule, and the types of endpoints to expect in the presentation.

Answer

Randy Teel (President and CEO) and Noah Berkowitz (CMO) reiterated the early responses observed in B- and T-cell lymphomas and robust BCL6 degradation. Noah Berkowitz expressed strong commitment to the program due to observed complete metabolic responses in patients with deadly disease, but did not guide to specific H2 2026 findings beyond that.

Daniel Bronder asked about the expected timeline for the next data cut for the spinal and bulbar muscular atrophy (SBMA) program (ARV-027) and what data from the healthy volunteer study, including muscle biopsy for AR concentrations, could help de-risk the program.

Answer

President and CEO Randy Teel stated it's too early to guide on the next data cut timeline for ARV-027. Chief Medical Officer Noah Berkowitz explained that the healthy volunteer study will establish dose range, PK, and a strong PD component through muscle biopsies to measure AR degradation, which will inform later trials.

Daniel Brander asked for insights into the expected launch trajectory of TransCon CNP, particularly where the majority of patients will be captured initially, given that few patients are currently on treatment for achondroplasia.

Answer

Jan Møller Mikkelsen, President and Chief Executive Officer, highlighted TransCon CNP's significant advantages, including a dramatically lower rate of injection site reactions (one every two years vs. 120 for competitors), no risk of hypotension, once-weekly dosing, and demonstrated benefits beyond linear growth such as improved leg bowing, muscle strength, and quality of life. He emphasized that every patient should have access to the best possible treatment option.

Daniel Brander, on behalf of Lee Watzek, asked for insights into the expected launch trajectory of TransCon CNP, particularly where the majority of patients will be captured initially, given that some patients are not currently on treatment.

Answer

Jan Møller Mikkelsen, President and Chief Executive Officer, highlighted TransCon CNP's differentiated profile, including its once-weekly regimen, significantly lower injection site reactions (one every two years versus 120), no risk of hypotension, and demonstrated benefits beyond linear growth such as improved leg bowing, muscle strength, and quality of life. He asserted that every patient should have access to the best possible treatment option.

Daniel Bronder of Cantor Fitzgerald, on for Lee Watsak, asked about patient compliance and pull-through for Yorvipath and sought clarification that the reported 1,500 patient figure represented net new patient starts.

Answer

President & CEO Jan Møller Mikkelsen cited long-term data from Europe showing an extremely low true discontinuation rate of only a few percent, which he attributed to the significant therapeutic benefit. He also confirmed that the enrollment figures refer to unique new patient prescriptions.

Speaking on behalf of Li Watsek, Daniel Bronder from Cantor Fitzgerald asked about the contribution of ibrutinib in the PCNSL trial and whether the FDA might question it. He also asked how Curis intends to prioritize its PCNSL program versus its AML and MDS programs.

Answer

President and CEO James Dentzer explained that the trial's design, which enrolls patients immediately after they progress on a BTK inhibitor, implicitly addresses the contribution of ibrutinib. He also cited historical data showing Curis's salvage line data is outperforming second-line BTK inhibitor data. Regarding prioritization, he called it a 'high-class headache' and confirmed that PCNSL is the top priority currently due to the ongoing FDA discussions and high unmet need.