Alex Thompson

Alex Thompson asked if the 200 enrollment forms recorded in Q4 2025 for PALSONIFY represent a reasonable run rate moving forward, and also inquired about the typical time from enrollment form submission to commercial therapy.

Answer

CFO Toby Schilke stated it's premature to extrapolate the 200 enrollment forms due to early launch dynamics. He noted that approximately 50% of patients are reimbursed commercially or through government programs, while the other 50% utilize the QuickStart program, with initial bottles lasting 30 days and check-ins every 15 days.

Alex Thompson inquired whether the 200 enrollment forms recorded in Q4 2025 for PALSONIFY represent a reasonable run rate moving forward, and if not, asked about the typical time from enrollment form submission to commercial therapy.

Answer

Toby Schilke, Chief Financial Officer, stated it is premature to extrapolate the 200 enrollment forms as a run rate due to various launch dynamics. He noted that approximately 50% of patients are reimbursed commercially or through Medicare/Medicaid, while the other 50% utilize the Quick Start program, but did not provide specific guidance on the duration from Quick Start to reimbursed care.

An analyst on behalf of Alex Thompson from Stifel Financial Corp. asked how many patients are currently in the open-label extension (OLE) for paltusotine and how quickly they are expected to transition to commercial drug post-launch.

Answer

CEO R. Scott Struthers noted that OLE patients are located globally. Chief Medical & Development Officer Dana Pizzuti clarified that a decision on the transition will be made after the PDUFA date and that only a small number of the total OLE patients are in the U.S. and would be eligible to switch to the commercial supply.

Alex Thompson inquired about the regulatory discussion status for starting the pivotal Graves study and whether a single pivotal trial would suffice for an sBLA.

Answer

Karen Massey, COO, confirmed the Graves program is well underway. Luc Truyen, CMO, clarified that while a single study can be sufficient, the specific division has requested two trials, which argenx is executing on.

Alex Thompson asked for an update on regulatory discussions for the Graves' disease program, specifically whether a single pivotal study would be sufficient for an sBLA or if two studies are required.

Answer

Karen Massey, Chief Operating Officer, confirmed the Graves' program is well underway. Luc Truyen, Chief Medical Officer, clarified that the specific regulatory division has requested two trials, which argenx is currently executing, despite the possibility of a single study being sufficient in other contexts.

Alex Thompson from Stifel asked about the discontinuation of empasiprubart in dermatomyositis (DM), specifically inquiring if enrollment issues were related to the IV presentation and how this compared to efgartigimod's myositis trial. He also sought an update on empasiprubart's CIDP enrollment and any potential impacts.

Answer

Luc Truyen, Chief Medical Officer, explained that the empasiprubart DM trial faced enrollment challenges in a competitive environment, leading to reprioritization despite DM remaining an important indication for argenx, as evidenced by strong enrollment in the ALKIVIA study with efgartigimod. Karen Massey, Chief Operating Officer, noted that it's too early to comment on empasiprubart's CIDP enrollment as those trials have just begun.

Alex Thompson asked about the reasons behind the discontinuation of empasiprubart in dermatomyositis (DM), specifically if enrollment issues were related to the IV presentation and how this compared to the efgartigimod myositis trial. He also questioned the enrollment status of empasiprubart in CIDP.

Answer

Luc Truyen, Chief Medical Officer, explained that the DM trial was a POC study facing a highly competitive enrollment environment, which was not helped by negative C5 readouts. He noted that robust inclusion criteria made enrollment challenging, leading to reprioritization. Karen Massey, Chief Operating Officer, added that it is too early to comment on empasiprubart's CIDP enrollment as those trials have just begun.

Alex Thompson from Stifel Institutional inquired about the evolution of Vyvgart cycles per year in Myasthenia Gravis (MG) and whether a higher gross price per patient was a key driver of the increased gross-to-net ratio.

Answer

CFO Karl Gubitz clarified that net revenue per patient for MG and CIDP remains consistent, with variables like product mix and utilization offsetting the gross-to-net impact, and confirmed no US price increases in 2025. COO Karen Massey added that while the company guides to five cycles per year for MG, the convenience of the prefilled syringe (PFS) could drive higher utilization as patients aim to maintain Minimum Symptom Expression (MSE).

Alex Thompson inquired about the consistency of interactions among members of the FDA's Cardio-Renal division regarding the FSGS filing over the last few months.

Answer

Bill Rote, Chief Research Officer, noted recent changes within the review team but also highlighted continuity, especially among those involved in the PARASOL project. He emphasized that division-level leadership has remained consistent and engaged throughout the process.

Alex Thompson asked about the consistency of interactions among members of the Cardio-Renal division at the FDA related to the FSGS filing over the last few months.

Answer

Bill Rote (Chief Research Officer) stated that while there have been some recent changes within the review team, there is also continuity, especially with those involved in the PARASOL project. He noted that recent interactions have included consistent and engaged division-level leadership.

Alex Thompson from Stifel inquired about the launch progress by partner CSL Vifor in Europe and when Travere might expect to see meaningful royalty revenue from ex-U.S. sales.

Answer

President & CEO Dr. Eric Dube deferred to CSL Vifor for specific performance commentary. CFO Chris Cline added that as CSL Vifor progresses through the country-by-country reimbursement process, revenues and subsequent royalties are expected to pick up, and the company will share more information as it becomes available.

Alex Thompson asked about potential challenges in the reimbursement process for keeping Rezdiffra patients on drug for extended periods, particularly if fibrosis scores improve and patients technically fall outside initial label requirements.

Answer

Bill Sibold, Chief Executive Officer, explained that reauthorizations are routine, often involving physician attestation or evidence of stabilization/improvement via non-invasive tests. He emphasized that recent AASLD data showing disease return upon discontinuation reinforces the belief that Rezdiffra is a chronic therapy, and the community generally uses it as such, mitigating concerns about patients falling outside label requirements due to improvement.

Alex Thompson asked about potential challenges in the reimbursement process for long-term Rezdiffra use, particularly if patients' fibrosis scores improve, potentially moving them outside initial label requirements.

Answer

CEO Bill Sibold stated that reauthorizations are routine, often based on physician attestation or stabilization/improvement in non-invasive tests. He highlighted AASLD data showing disease recurrence upon discontinuation, reinforcing the belief that Rezdiffra is a chronic therapy and that patients' best interest lies in continued treatment, even after initial response.

Alex Thompson asked about the expected OpEx trajectory for 2026, considering the TransCon CNP launch and the SKYTROFA label expansion, including both monotherapy and combination pivotal studies.

Answer

Scott Smith, Chief Financial Officer, suggested using Q4 OpEx as a run rate for the full year, with updates to be provided if circumstances change. He noted that TransCon CNP-related details would be discussed further following approval. Jan Møller Mikkelsen, President and Chief Executive Officer, added that R&D expenses have remained constant for the past three to four years, and the global commercialization infrastructure is mature, meaning additional personnel for new products will not significantly impact overall OpEx.

Alex Thompson asked Scott Smith about the OpEx trajectory for 2026, considering the TransCon CNP launch and the SKYTROFA label expansion, including both monotherapy and combination pivotal studies.

Answer

Scott Smith, Chief Financial Officer, suggested using Q4 2025 OpEx as a run rate for the full year 2026, with updates to be provided if circumstances change. He reiterated that specific details related to TransCon CNP would be discussed post-approval. Jan Møller Mikkelsen, President and Chief Executive Officer, added that R&D expenses have remained constant over the past three to four years, and the global commercialization infrastructure is already mature, so additional personnel for new launches would not significantly impact OpEx.

Alex Thompson from Stifel asked about the proportion of Yorvipath prescribers who have multiple patients on therapy and how that trend might be evolving.

Answer

President & CEO Jan Møller Mikkelsen stated that the company does not yet have sufficient data to comment on the trend of prescribers treating multiple patients.

Alex Thompson, Managing Director at Stifel, asked about the competitive landscape in Graves' disease, specifically how Roivant plans to maintain its lead against Argenx, which is entering the area, and whether Argenx's potential for shorter or fewer studies could impact Roivant's timeline.

Answer

Matt Gline, CEO of Roivant Sciences, acknowledged that the extent of Roivant's lead would depend on Argenx's study design, but he expects a significant lead regardless. He highlighted Roivant's strong relationships with KOLs, the 26-week duration of one of their own studies, and the belief that IMVT-1402's deeper IgG suppression will matter for remission in Graves' disease.

Alex Thompson asked about the competitive landscape in Graves' disease with Argenx entering the area, and Roivant's confidence in maintaining its lead if Argenx were to run shorter studies.

Answer

CEO Matt Gline stated that the extent of Roivant's lead in Graves' will depend on Argenx's study design, but they expect a significant lead regardless. He highlighted Roivant's strong KOL relationships, existing 26-week study (2503), and the expectation that deeper IgG suppression from their drug will matter for remission in this large and exciting population.

Alex Thompson of Stifel asked about the chemical scaffold of the next-generation STAT6 degrader compared to KT-621 and inquired about the timing for a potential decision to split indications between the two assets.

Answer

CEO Nello Mainolfi described their chemistry platform as having a "plethora" of different scaffolds and binding moieties, providing significant IP protection. He explained that it is too early to define a strategy for splitting indications, and the company is focused on maintaining maximum optionality, especially now that its recent financing enables the initiation of multiple Phase 3 studies.