Danielle Brill

Danielle Brill from Truist Securities asked about argenx's confidence in the commercial opportunity and regulatory approval for seronegative gMG, based on presented subgroup data. She also inquired about any Minimum Symptom Expression (MSE) data findings for this population, given its importance to prescribers.

Answer

Luc Truyen, Chief Medical Officer, expressed excitement about the ADAPT SERON study's outcome, which met its primary endpoint with clinically meaningful MG-ADL score reduction across all three seronegative subgroups. Karen Massey, Chief Operating Officer, highlighted the significant commercial opportunity in seronegative gMG, with 11,000 patients, and noted the rapid trial enrollment as an indicator of unmet need and prescriber enthusiasm. Tim Van Hauwermeiren, Chief Executive Officer, added that more MSE data will be disclosed in the future.

Danielle Brill asked about the commercial confidence and likelihood of approval for the seronegative gMG opportunity based on the subgroup data presented. She also inquired about any Minimum Symptom Expression (MSE) data findings, given its importance to prescribers.

Answer

Luc Truyen, Chief Medical Officer, expressed excitement for the Ceylon study, which met its primary endpoint with clinically meaningful MG-ADL reduction across all three seronegative subgroups (MuSK, triple seronegative, LRP4), reinforcing confidence for regulatory discussion. Tim Van Hauwermeiren, Chief Executive Officer, noted that more MSE data would be disclosed in the future. Karen Massey, Chief Operating Officer, highlighted the significant commercial opportunity with 11,000 patients and the rapid enrollment of the trial as indicators of unmet need.

Danielle Brill asked if the CIDP launch cadence is expected to be similar to gMG, citing physician checks suggesting a bolus of patients may be waiting for VYVGART.

Answer

COO Karen Massey acknowledged prescriber excitement but cautioned that a large bolus of waiting patients is not expected. She anticipates a potentially slower initial uptake than gMG due to the time required for payer policies to be established (approx. 2 quarters), patient reluctance to switch from stable IVIg therapy, and existing competition. She remains confident in the large, long-term opportunity.

Danielle Brill asked about the most important differentiating elements for Infigratinib in achondroplasia, considering efficacy, route of administration, and safety, and what level of hyperphosphatemia is acceptable.

Answer

Neil Kumar, CEO and Founder, BridgeBio Pharma, asserted that Infigratinib will be more efficacious, safer, and more convenient due to its oral route of administration. He highlighted its superior efficacy by targeting the condition at its source and providing proportionality impact, with no hypotensive results expected. Kumar indicated that Grade 1 hyperphosphatemia (15-20%) is not a significant concern for clinicians, who prioritize issues like hypergrowth or spinal complications.

Danielle Brill asked about the most important differentiating elements for Infogratinib in achondroplasia, specifically whether efficacy or route of administration is more critical, and discussed safety, including the acceptable level of hyperphosphatemia.

Answer

Neil Kumar, CEO and Founder, BridgeBio Pharma, stated that Infogratinib is expected to win on efficacy, safety, and convenience (oral administration). He emphasized superior efficacy demonstrated in preclinical, animal, and Phase 2 data, providing proportionality impact. He anticipates Infogratinib will be safer, without hypotensive results, and believes Grade 1 hyperphosphatemia (15-20%) is not a significant concern for clinicians compared to issues like hypergrowth or spinal complications.

Danielle Brill of Truist Securities asked about the enrollment pace for the Phase II TOYVA study in CVM, the rate-limiting factors for enrollment, and how to contextualize potential efficacy results compared to the MLM Phase II data, given the disease's heterogeneity.

Answer

President and CEO Wes Kaupinen confirmed the study is on track for a Q4 data readout and that the rate-limiting step is the careful selection of patients with significant cutaneous disease. He contextualized success by noting that in rare diseases with no approved therapies, an efficacy rate around 30%, combined with a clean safety profile, would be considered highly successful and supportive of advancing to a Phase III trial.

Danielle Brill from Truist Securities inquired about the potential market impact of new orexin-2 agonists on the Wakix franchise and sought clarification on Q2's sequential growth, questioning if it was solely due to an inventory drawdown given the high number of new patient adds.

Answer

EVP & CCO Adam Zaeske expressed confidence in Wakix's durability due to its differentiated non-scheduled profile and potential for synergistic use. CFO & CAO Sandip Kapadia confirmed that underlying demand was strong and that a typical Q2 trade inventory drawdown of a few days impacted reported net revenue. He reiterated confidence in the full-year guidance and expects strong quarter-over-quarter growth ahead.

Danielle Brill questioned the implied slowdown in Q4 patient adds based on year-end guidance and asked for the rationale behind pursuing separate DEE indications for EPX-100 instead of a basket study.

Answer

CFO Sandip Kapadia and CCO Jeffrey Dierks clarified that the 'approximately 7,000' patient guidance is an approximation and that they expect Q4 growth momentum to be similar to prior quarters. Chief Medical and Scientific Officer Dr. Kumar Budur explained the DEE strategy is a measured approach, focusing on distinct Dravet and LGS populations first before exploring broader basket studies, with learnings to be applied to future programs.

Danielle Brill from Truist Securities asked for feedback from the ENDO conference on Crinesity's one-year data, specifically regarding glucocorticoid dose changes and insulin resistance, and inquired about future data releases.

Answer

Strategic Advisor Eiry Roberts reported a very positive response from clinicians at ENDO. She explained that the one-year data showed a consistent, modest, but clinically beneficial effect across metabolic parameters like weight and HOMA-IR, which results from controlling androgens and enabling steroid reduction. She confirmed that Neurocrine intends to continue publishing long-term clinical data, including outcomes like bone age, from its ongoing open-label studies.

Danielle Brill asked about the potential impact on azetukalner's value proposition if the efficacy effect size in the X-TOLE2 study is less robust than what was observed in the original X-TOLE trial.

Answer

CEO Ian Mortimer emphasized that the X-TOLE data has already set a very high efficacy bar and those results are fixed. CMO Dr. Christopher Kenney noted the primary goal is a positive confirmatory trial. CCO Christopher Von Seggern added that the drug's value proposition is based on the totality of its data, including durable open-label results, seizure freedom, rapid onset, and ease-of-use attributes, not just a single efficacy number.

An analyst on behalf of Danielle Brill of Raymond James inquired about the level of effort to get patients who previously discontinued Daybue back on treatment, given the ongoing learnings about mitigating adverse events.

Answer

Executive Brendan Teehan explained that the company stays in contact with every family. For those who discontinued early due to tolerability, the team works to educate them and their HCPs on GI management and alternative dosing strategies. However, he noted that to date, patient restarts represent significantly less than 10% of the overall patient base, though it remains an area of focus.

Danielle Brill of Raymond James questioned the rate of patient uptake for ELEVIDYS, noting her math suggested a smaller-than-expected increase in treated patients from Q2 to Q3, and asked about the primary bottlenecks in the launch.

Answer

President and CEO Doug Ingram responded that the company uses revenue as its key performance metric and that the launch is proceeding exactly as planned. He identified three main factors governing the growth trajectory: ensuring high-quality site capacity, managing manufacturing and batch releases, and productive payer interactions, all of which are progressing well.

Danielle Brill from Raymond James posed broader questions about the rationale for prioritizing the GM1 and FTD programs and inquired about the biggest bottlenecks in recruitment for these trials, such as site activation or patient identification.

Answer

CEO William Chou clarified that the program prioritization was a resource-driven decision to focus on the most advanced clinical programs (GM1, FTD) and key preclinical assets. Chief Medical Officer Mark Forman added that the main bottlenecks are patient identification for GRN mutations and opening trial sites, both of which are being addressed through multi-pronged initiatives.

Danielle Brill of Raymond James asked about the FTD program, specifically how many patients have been screened with the company-provided genetic tests and the resulting GRN mutation positivity rate. She questioned if the prevalence of FTD-GRN might be lower than estimated and asked about any gating factors for activating additional trial sites.

Answer

Bruce Goldsmith, President and CEO, stated that the company has not disclosed specific numbers on genetic screening results. He elaborated on a multi-step strategy to boost patient identification, which includes outreach to non-trial centers, providing free test kits, and building referral networks. He mentioned that activating more sites is a matter of logistics and operations, not any specific issue. Goldsmith also conceded that the current prevalence estimates for FTD-GRN are based on published literature and that the actual number will become clearer as more testing is conducted.