Thomas Smith

Thomas Smith inquired about the expected increase in Cortrophin Gel utilization from acute gouty arthritis flares beyond the current 15% in 2025, and requested more details on the pilot programs executed in primary care and podiatry, including prescriber feedback and implementation learnings.

Answer

Nikhil Lalwani, President and CEO, stated that the 15% gout utilization is expected to increase significantly with the new sales force, but did not provide specific numbers. He highlighted that almost half of Cortrophin prescribers were new to ACTH, and the expansion will further reach new primary care and podiatry physicians. Learnings from pilot programs included identifying appropriate patients, streamlining office workflows for enrollment, and effectively targeting the 7,000 high-prescribing HCPs for severe acute gouty arthritis flares.

Thomas Smith from Leerink Partners asked for expectations regarding the percentage of Cortrophin Gel utilization derived from acute gouty arthritis in 2026 and 2027, following the sales force expansion. He also requested more details on the Cortrophin pilot programs in primary care and podiatry, including specific prescriber feedback and key learnings implemented for better targeting.

Answer

President and CEO Nikhil Lalwani stated that the 15% of current Cortrophin Gel volumes from gout is expected to increase significantly with the new investment, though specific numbers were not provided. He highlighted that almost half of Cortrophin prescribers were new to the ACTH category, and the pilot programs provided learnings on identifying appropriate patients, streamlining the enrollment-to-fulfillment process, and effectively targeting the 7,000 identified high-volume HCPs in primary care and podiatry.

Thomas Smith asked about the phase II-A results for empasiprubart in ALS, and any learnings applicable to the phase III CMS program or other indications.

Answer

Luc Truyen, CMO, acknowledged the ALS outcome was not as hoped, but valuable learnings were gained regarding novel endpoints, which will be shared. He clarified that there is no read-through to the CMS program due to fundamentally different treatment contexts and direct biological relevance for CMS.

Thomas Smith asked about the Phase 2A results for empasiprubart in ALS, inquiring about any learnings that could be applied to the Phase 3 CMS program or other indications.

Answer

Luc Truyen, Chief Medical Officer, stated that the ALS outcome was not as hoped, and the data does not support progression in this indication, though learnings on novel endpoints will be shared. He clarified that there is no read-through to the CMS program, which has a fundamentally different biological context, or to the SMA program, where empasiprubart is being evaluated for add-on efficacy to existing gene therapies.

Thomas Smith from Leerink Partners asked for comments on recent long-term follow-up data from a competitor's SRM in Graves' disease, inquiring about the importance of potential disease modification in argenx's decision to pursue thyroid eye disease (TED) with VYVGART. He also sought expansion on the market potential and initial assessment of the addressable market for Graves'.

Answer

Tim Van Hauwermeiren, Chief Executive Officer, acknowledged the competitor's data but cautioned that it was from a very small sample, warranting further investigation in larger trials. Karen Massey, Chief Operating Officer, explained that Graves' was selected based on strong biology, development feasibility, and a significant unmet patient need within a large prevalence, creating a franchise opportunity with TED.

Thomas Smith asked about recent long-term follow-up data from a competitor SRM in Graves disease suggesting potential for long-term disease remission. He inquired about the importance of disease modification in argenx's decision to pursue TED/Graves and sought an initial assessment of the addressable market opportunity.

Answer

Tim Van Hauwermeiren, Chief Executive Officer, acknowledged the competitor data as interesting but cautioned that it involved a very small sample, warranting further investigation in larger, controlled trials. Karen Massey, Chief Operating Officer, explained that Graves was selected based on biology, development feasibility, and a significant commercial opportunity driven by a large prevalence and a subset of patients with clear unmet needs, also noting the franchise opportunity with TED.

Thomas J. Smith from Leerink Partners asked for an update on the FDA FAERS safety signal regarding CIDP from June, including any visibility on resolution timelines and feedback from prescribers on the matter.

Answer

CMO Luc Truyen contextualized the FAERS signal, noting its limitations (e.g., lack of denominators) and classifying it as a 'potential' signal requiring a 12-month monitoring period. He stated that argenx's internal data shows a low event rate (<2%) and the benefit-risk profile remains unchanged. COO Karen Massey added that prescriber feedback is very positive, with a high grant rate for patient requests for Vyvgart, indicating continued physician confidence.

Thomas Smith asked about the lupus nephritis (LN) proof-of-concept study, inquiring about the baseline characteristics of the patients enrolled in China by Zai Lab and the company's bar for success.

Answer

CEO Tim Van Hauwermeiren praised partner Zai Lab and explained the trial is a true proof-of-concept designed to answer the primary question of whether the IgG-lowering mechanism works in LN patients. He stated that if the data is positive, argenx will advance to a global Phase III trial designed to account for different global treatment regimens.

Thomas Smith asked for elaboration on the anticipated launch cadence for the Acquired Hypothalamic Obesity (HO) expansion, specifically how it might compare to the launch of IMCIVREE for Bardet-Biedl Syndrome (BBS), considering regulatory interactions, reimbursement, and patient activation.

Answer

Jennifer Lee, Executive Vice President and Head of North America, outlined both similarities and differences between the HO and BBS launches. She noted ongoing opportunities for diagnosis and a similar 3-9 month timeline for payer policy updates. Key differences for HO include higher precision in targeting physicians and the ability to identify and engage incident patients earlier in their journey through specialized medical centers.

Thomas Smith asked for elaboration on the anticipated launch cadence for the Acquired Hypothalamic Obesity (HO) expansion, particularly how it compares to the Bardet-Biedl Syndrome (BBS) launch, considering ongoing regulatory interactions, reimbursement, payer coverage, and site activation efforts.

Answer

Jennifer Lee, Executive Vice President and Head of North America, highlighted similarities with the BBS launch in the need for patient diagnosis and the time required for payer policy updates, despite a strong foundation from BBS discussions. She noted key differences for HO, including greater precision in identifying target physicians and medical centers, allowing for earlier identification of incident patients, which provides more confidence in the launch strategy compared to BBS.

Thomas Smith inquired about the contribution of the Germany launch to 2025 worldwide revenues and 2026 expectations, and the strategy for mid-stage Phase 2 combination studies for pipeline assets like Ervogastat, specifically regarding the need for liver histology via biopsy versus non-invasive tests (NITs).

Answer

Bill Sibold, Chief Executive Officer, stated that Germany's contribution was negligible in 2025 and not expected to be significant in 2026, with the U.S. remaining the primary growth driver. David Soergel, Chief Medical Officer, indicated that while definitive plans for Phase 2 combination studies require FDA discussion, NITs are expected to play a major role in assessment, reflecting evolving clinical care beyond biopsy. Tina Ventura, Chief Investor Relations Officer, added that robust growth is expected for 2026, aligning with current consensus.

Thomas Smith inquired about the revenue and patient contribution from the Germany launch in 2025 and 2026, and the strategy for pipeline combination programs, specifically regarding the need for liver histology via biopsy versus non-invasive tests (NITs) in future Phase 2 and pivotal studies for Ervogastat.

Answer

CEO Bill Sibold clarified that Germany's contribution was negligible in 2025 and not expected to be significant in 2026, with the US remaining the primary growth driver. Chief Medical Officer David Soergel indicated that while definitive plans require FDA discussion, NITs are expected to play a major role in Phase 2 assessments, reflecting a shift away from biopsy in clinical care.

Thomas Smith asked for comments on the recent Aetna formulary coverage decision and its potential impact, and for an explicit goal or expectation for the percentage of commercial lives that will have broad first-line access to Rezdiffra for 2026.

Answer

CEO Bill Sibold stated Madrigal expects broad commercial live coverage for 2026. He clarified that Rezdiffra was not on Aetna's formulary in 2025 and remains so for 2026, thus expecting no meaningful impact as it will still be available via prior authorization or medical exception, with Madrigal's patient support team assisting.

Thomas Smith sought comments on the recent Aetna formulary coverage decision and its potential impact, as well as Madrigal's explicit goal or expectation for the percentage of commercial lives that will have broad first-line access to Rezdiffra in 2026.

Answer

Bill Sibold, Chief Executive Officer, stated that Madrigal expects broad commercial live coverage for 2026. He clarified that Rezdiffra was not on Aetna's formulary in 2025 or 2026, so there is no change or meaningful impact, as access remains available through prior authorization or medical exception, supported by Madrigal's patient support team.

Thomas J. Smith of Leerink Partners inquired about the gross-to-net and inventory dynamics during the quarter, asking for clarification on the apparent improvement in net pricing versus Q1 and the outlook for the remainder of the year.

Answer

CFO Mardi Dier explained that the quarter's results were driven by strong demand and reiterated that gross-to-net will be choppy early in the launch. She noted that while contracting began in April, the more significant impact is expected in the second half of the year, keeping the metric within the typical specialty pharma range. CEO Bill Sibold added that the company takes a long-term, disciplined view on gross-to-net to preserve value.

Thomas Smith of Leerink Partners inquired about the Rezdiffra launch momentum in early 2025, asking for commentary on new patient additions, any changes in cadence, and the impact of Q1 insurance dynamics. He also sought color on Q1 and full-year 2025 performance relative to successful specialty drug launch analogs.

Answer

CEO William Sibold confirmed that the strong momentum from 2024 has continued into 2025 with steady patient and prescriber growth, while acknowledging the typical 'Q1 dynamic' that the company is managing well. CFO Mardi Dier added that while not providing specific guidance, they expect the Q1 2025 consensus to narrow and increase, and that the launch continues to track well against their basket of top specialty launch analogs.

Thomas Smith inquired about early visibility into patient persistence and refill rates for Rezdiffra, and also asked about the company's business development strategy for building a pipeline beyond Rezdiffra.

Answer

CEO William Sibold responded that it is too early to quantify persistence but that early indicators are optimistic. On business development, he affirmed Madrigal's goal to be the leading NASH company by building a pipeline. He noted the company is evaluating opportunities across all stages and is receiving inbound interest due to its leadership position, with a focus on finding the right assets to add to the portfolio.

Thomas Smith inquired about any notable differences in baseline characteristics between the maintenance study and the prior VENTURE subQ or oral studies. He also asked about the amylin program's design and execution, specifically if it would mirror the VK2735 Phase I experience in Australia, and expectations for weight loss from the MAD portion of the amylin study.

Answer

Viking Therapeutics' President and CEO, Brian Lian, stated that the amylin study would target U.S.-based clinical sites, not Australia. He noted that while it's early to interpret efficacy from a single dose, preclinical data in primates suggest the amylin agonist is potent, potentially more so than VK2735. He expects the amylin program to follow a SAD then MAD study design, similar to VK2735. Regarding maintenance study demographics, he expects them to be similar to VENTURE Phase II, primarily normoglycemic individuals with BMI > 30.

Thomas Smith asked about any significant differences in baseline characteristics for the VK2735 maintenance study compared to the VENTURE subcutaneous or oral studies. He also inquired if the amylin program's Phase 1 design and execution would mirror VK2735's Phase 1 in Australia, and what weight loss expectations exist for the multiple ascending dose (MAD) portion of the amylin study.

Answer

Brian Lian, President and CEO, clarified that the amylin study would target U.S.-based clinical sites, not Australia, and would follow a single ascending dose (SAD) then multiple ascending dose (MAD) design. He noted preclinical primate data suggested high potency, potentially exceeding VK2735, but human data is pending. For the maintenance study, he anticipated demographics similar to VENTURE Phase 2, primarily normoglycemic individuals, but did not have precise figures.

Thomas Smith asked about the baseline patient characteristics in the Phase II VENTURE oral study and how they compare to the prior injectable study. He also inquired about the need for additional manufacturing capacity or redundancy beyond the CordenPharma agreement.

Answer

Brian Lian, President and CEO, stated he had not yet reviewed the demographic data for the oral study but expected it to be similar to past trials. He confirmed that Viking does plan to establish redundancy across its entire supply chain to ensure capacity and mitigate risks.

Thomas Smith asked for an update on the review of the 100mg oral VK2735 cohort data, whether higher doses might be explored, what additional analyses for VK2809 in NASH could be expected at the upcoming AASLD meeting, and the current strategy for partnering the NASH program.

Answer

Executive Brian Lian deferred detailed comments on the 100mg cohort until the ObesityWeek data release but noted nothing precluded dosing higher. For VK2809, he said to expect more granular data on histologic changes at AASLD. He reiterated that the company's strategy for the NASH program remains to advance it in collaboration with a larger pharmaceutical partner.

Thomas Smith asked about the data release timing for the 1402 D2T-RA study (whether open-label and randomized data would be reported together), and the expectation for a Graves' launch by the end of 2028 but not MG, inquiring if this was due to data timing or other strategic considerations.

Answer

CEO Matt Gline indicated that a final decision on RA data release timing hasn't been made, but it's reasonably likely they will wait for the randomized withdrawal period. He clarified that the exclusion of MG from the 2028 launch timeline should not be read into, as there's a possibility it could also launch then, with more guidance to come once study timelines are firm.

Thomas Smith, Senior Research Analyst at Leerink Partners, sought clarification on the data release timing for IMVT-1402's DTT-RA study (open-label and randomized data together or separately) and asked about the expectation for a Graves' launch by the end of 2028 but not MG, despite both having phase 3 data in 2027.

Answer

Matt Gline, CEO of Roivant Sciences, indicated that while a final decision on RA data release timing hasn't been made, it's reasonably likely they will wait for the randomized withdrawal period before discussing results. He clarified that the exclusion of MG from the 2028 launch timeline should not be read into, as there's a possibility it could also launch then, with more specific guidance to follow after study timelines are firm.

Thomas Smith asked for clarification on whether both open-label and randomized data from the IMVT-1402 D2T-RA study would be reported together, and about the expectation for a Graves' launch by the end of 2028 but not MG, questioning if this is due to data timing or other strategic considerations.

Answer

Matt Gline, CEO of Roivant, stated that a final decision on the IMVT-1402 RA data release timing is pending, but it's reasonably likely they will wait for the randomized withdrawal period. He noted that there isn't much to read into the exclusion of MG from the 2028 launch timeline, and MG could potentially launch in 2028 as well, with more guidance to follow once data and timelines are firm.

Thomas Smith asked if both the open-label and randomized data from the IMVT-1402 D2T-RA study would be reported together, and about the expectation for a Graves' launch by the end of 2028 but not MG, despite both having Phase 3 data in 2027.

Answer

Matt Gline, CEO of Roivant, said a final decision on RA data release timing hasn't been made, but it's likely they'll wait for the randomized withdrawal period. He clarified that the exclusion of MG from the 2028 launch timeline on slide 31 shouldn't be read into, as MG could also launch in 2028, with more guidance to follow after data and study timelines are clear.

Thomas Smith asked about the TED program, specifically the IL-6 class data and its approvability, expectations for metoclopramide, and an update on overseas 1402 studies and timing for additional indications.

Answer

CEO Matt Gline noted a notably high placebo response in the IL-6 study and acknowledged the competitive intensity in TED, focusing on Graves' opportunity to impact the disease earlier. For overseas 1402 studies, he mentioned they are ongoing global registrational programs, with small POCs informing bigger studies, and updates will be shared when available.

Thomas Smith asked for comments on the IL-6 class data in TED, its approvability, expectations for metoclopramide, and updates on 1402's overseas studies.

Answer

CEO Matthew Gline declined to comment on the approvability of other mechanisms but noted a high placebo response in the IL-6 study. He reiterated the competitive intensity in TED and Roivant's focus on Graves' as an earlier intervention. For overseas studies, he stated they inform indication selection or design decisions for bigger studies, with updates shared if and when available.

Thomas J. Smith of Leerink Partners asked for the rationale behind the IMVT-1402 trial design in CIDP, which omits a washout period and doesn't measure response rates. He also inquired about patient selection optimization and how the resulting data will position the drug commercially.

Answer

CEO & Director Matt Gline explained the design was driven by three factors: FDA preference for direct placebo-controlled trials, investigator and patient aversion to washouts, and improved ability to select the right patients. He noted that data from their previous batoclimab study showed they can effectively identify patients who flare upon withdrawal, making a patient-friendly "no washout" design feasible and competitive.

Inquired about the status of partnership discussions for vidofludimus and the progress on Phase II plans for IMU-856, including regulatory feedback and gating factors for starting the next study.

Answer

The company is actively exploring various partnership structures (full licensing, regional deals, profit-sharing) for vidofludimus while also building in-house commercial capabilities. For IMU-856, Phase II development across several potential GI indications is contingent on securing additional financing, a license, or a partnership, as the company's primary focus remains on its MS programs.

Thomas Smith of Leerink Partners questioned the specifics of the neurofilament light chain (NfL) assay used and its variability. He also asked about life cycle management, specifically regarding additional intellectual property for vidofludimus calcium or other preclinical Nurr1 activator compounds.

Answer

CEO Dr. Daniel Vitt explained that the company uses the state-of-the-art Simoa technology by Quanterix with a dual-antibody test system to ensure high precision and low variability in NfL measurements. On life cycle management, he confirmed that the discovery of Nurr1 activation has created an attractive platform. Immunic is actively working on the biology and chemistry, developing derivatives, filing new IP, and exploring applications in other neurodegenerative diseases like Parkinson's.

Inquired about any preclinical data comparing ATI-2138 to upadacitinib (a JAK1 inhibitor), the rationale for choosing an open-label trial design for the Phase IIa study, and the expected timeline for top-line data from that study.

Answer

The company stated the key differentiator is ITK inhibition, which they believe will provide an additive effect to outperform competitors, though they have no direct preclinical comparisons to upadacitinib. The open-label design was chosen for speed and cost-efficiency in enrollment, as they believe the required efficacy bar is well understood. They are not giving specific guidance on data timing but expect it within a year.

Thomas Smith asked about any preclinical data comparing ATI-2138 to upadacitinib (a JAK1 inhibitor), the rationale for an open-label versus placebo-controlled trial design, and the expected timeline for top-line data.

Answer

Interim CEO Neal Walker stated he was unaware of direct comparisons to a pure JAK1 inhibitor but emphasized the hypothesis that the dual ITK/JAK3 mechanism should yield an additive effect. He defended the open-label design as more cost-efficient and faster for enrollment, which is ideal for signal-finding in a well-understood indication like atopic dermatitis. Regarding timing, Walker declined to give specific guidance but indicated data would be available within a year.

Thomas Smith asked if Aclaris had any preclinical data comparing ATI-2138 to the selective JAK1 inhibitor upadacitinib and how it might position against it in atopic dermatitis. He also questioned the choice of an open-label trial design over a placebo-controlled one and asked for a timeline for top-line data.

Answer

Interim CEO Neal Walker responded that the key differentiator is ITK inhibition, which directly targets Th2 cytokines and is expected to provide an additive effect to outperform drugs like upadacitinib, though he noted no direct head-to-head preclinical data exists. Regarding the trial design, Walker explained that an open-label study is faster and more cost-efficient for signal-finding, given the competitive enrollment environment and well-understood efficacy benchmarks. He did not provide a specific timeline for data but stated it would be available 'within a year of this call.'

Thomas Smith from SVB Securities asked for details on the planned Phase 1b data update, including potential venues and expected patient follow-up. He also inquired about the key differences between Tegoprubart and Sanofi's competing molecule, and the company's current view on broader strategic interest and business development.

Answer

CEO David-Alexandre Gros stated that the company is targeting a medical meeting like Kidney Week towards the end of the year for the data update, by which time the six currently enrolled patients would have follow-up data ranging from over 90 days to a year. He clarified that while both Eledon's and Sanofi's molecules are anti-CD40 ligands, the strategic difference is Eledon's focus on transplantation versus Sanofi's focus on larger indications like MS. Gros concluded that the company is not currently seeking to in-license assets and is focused on executing its own trials with its new capital.

Thomas Smith from SVB Leerink Securities asked for more context on the reported eGFR rates compared to standard of care, details on any drug-related adverse events, and expectations for the pace of enrollment for the ongoing Phase Ib study.

Answer

CEO David-Alexandre Gros stated that the observed eGFRs are notably higher than the typical standard of care, which is in the 50s. He clarified that one drug-related adverse event was BK viremia, a common occurrence in immunosuppressed transplant patients. Regarding enrollment, he expects the Phase Ib study to complete enrollment by year-end, noting that the pace can be variable due to the unpredictable nature of cadaveric organ availability, but that the company has sufficient sites activated.

Thomas Smith of SVB Securities inquired about Eledon's perspective on recent developments in the CD40 landscape, including data from Horizon and Novartis. He also asked for an update on the plans and gating factors for initiating the Phase 2 kidney transplant study in the U.S.

Answer

President and CSO Steve Perrin noted that recent data from competitors validates the CD40 pathway. CEO David-Alexandre Gros reiterated Eledon's focus on its current indications, citing financial constraints for expansion. Perrin and CMO Jeff Bornstein detailed the scientific rationale for targeting CD40 ligand over the receptor in transplants, highlighting differences in expression, pathway blocking, and T-cell repolarization, and noted they can learn from Novartis's study design. Gros confirmed there are no major gating items for starting the U.S. Phase 2 kidney study and that the team is working with CROs on execution.

Thomas Smith of SVB Securities inquired about the updated clinical trial data timelines, asking for the expected number of patients in the Q1 2023 datasets for transplant and IgAN. He also sought details on the islet cell transplant program's University of Chicago team and the rationale for running the Phase 2 renal transplant study in parallel with the ex-US Phase 1b.

Answer

CEO David-Alexandre Gros confirmed the patient numbers remain consistent with previous guidance, with the timeline shifting to Q1 2023 to ensure sufficient data. He and CMO Jeff Bornstein highlighted the University of Chicago's expertise in islet cell transplants. Dr. Bornstein explained the Phase 2 kidney trial's superiority design was guided by the FDA for efficiency, while Dr. Gros noted running it in parallel with the Phase 1b allows for continuous data reporting.

Thomas Smith of SVB Securities asked for more details on regulatory interactions with China's CDE for CBP-201, the company's partnership and commercialization strategy, and the next steps for the CBP-174 program.

Answer

Co-Founder and CEO Dr. Zheng Wei described the CDE's feedback as a positive development, potentially accelerating the BLA submission timeline in China pending positive data. He stated the company is open to both regional and global partnerships for CBP-201. Regarding CBP-174, Dr. Wei and Chief Medical Officer Dr. Chin Lee noted the positive Phase 1 safety data and explained that next steps involve careful planning, including potential formulation optimization and a study design beyond a typical multiple ascending dose trial.

Thomas Smith of SVB Leerink inquired about the enrollment status of the 4.5 mg/kg dose cohort in the Phase 1b CSU study and the expected timing for data from the 1.5 mg/kg cold urticaria cohort. He also asked for an overview of the remaining and planned reproductive toxicology work for CDX-0159.

Answer

SVP and CMO Diane Young stated that the 4.5 mg/kg cohort is still enrolling to gather more high-exposure data, and the cold urticaria data will be presented at a future meeting once sufficient data is collected. Co-Founder and CSO Tibor Keler detailed the planned reproductive toxicology work, which includes standard ePPND studies to evaluate effects during pregnancy and birth, as well as juvenile toxicology studies required before initiating pediatric trials.