Laura Chico

Laura Chico asked about the criteria for advancing the 4x10^13 vg/kg dose of UX701 in Wilson disease from the fourth cohort into future studies, specifically what the company would need to observe to gain confidence.

Answer

Emil Kakkis, CEO and President, and Eric Crombez, Chief Medical Officer, stated that they would need to see a substantial and compelling effect in the majority of patients, specifically patients coming off their current standard of care (chelators and zinc). They noted that increasing the dose and modifying the immunomodulation program are expected to maximize efficacy.

An analyst on behalf of Laura Chico asked for more detail on the key growth drivers for Crysvita during the quarter, with a specific focus on the Latin America and Turkey regions.

Answer

CCO Erik Harris and CEO Emil Kakkis attributed the strong growth to positive patient and physician experiences, which are driving broader adoption, including in adult patients. They highlighted that securing national reimbursement in Brazil and Mexico was a critical catalyst for uptake in Latin America. While Turkey remains a named-patient market, strong word-of-mouth and physician conviction are fueling demand. They noted this regional infrastructure bodes well for the future setrusumab launch.

Laura Chico asked about valbenazine in dyskinetic cerebral palsy, specifically what would constitute a meaningful change in CRIA score in this population, and the size of the opportunity and overlap with existing INGREZZA prescribers given the field force expansion.

Answer

Sanjay Keswani, Chief Medical Officer, explained that since there's no validated scale for dyskinetic cerebral palsy, they are borrowing from the UHDRS Huntington's scale, and a statistically significant effect would be meaningful. Kyle Gano, Chief Executive Officer, added that a robust data set would be taken to the agency for NDA submission, and the current sales force could cover an SNDA. Eric Benevich, Chief Commercial Officer, noted that this population is larger than Huntington's chorea but smaller than TD, and the expanded sales team could cover potential prescribers.

Laura Chico asked about valbenazine in dyskinetic cerebral palsy, specifically what would constitute a meaningful change in CRIA score, and the size of the opportunity and overlap with existing INGREZZA prescribers given the field force expansion.

Answer

Sanjay Keswani, Chief Medical Officer, explained that for dyskinetic cerebral palsy, there is no a priori validated scale, so they are borrowing from the UHDRS Huntington's scale, and a statistically significant effect would be meaningful. Kyle Gano, Chief Executive Officer, added that a robust data set would be taken to the agency for NDA submission, and the current sales force could cover an SNDA. Eric Benevich, Chief Commercial Officer, noted that the dyskinetic cerebral palsy population is larger than Huntington's chorea but smaller than TD, and the expanded sales team could cover all potential prescribers for this indication.

Laura Chico of Wedbush Securities asked if the majority of Crinesity clinical trial patients have transitioned to paid therapy and if there are any physician capacity constraints in managing these patients.

Answer

CFO Matt Abernethy first addressed capacity, stating there are no constraints as the treatment fits into existing care workflows. CEO Kyle Gano then reiterated that the number of transitioning trial patients is minimal—less than 40 in total, split across Q2 and Q3—and does not significantly impact the overall new patient numbers.

Laura Chico asked about INGREZZA's patient persistency and discontinuation rates, questioning if there were any differences across various channels like long-term care versus psychiatry.

Answer

Chief Commercial Officer Eric Benevich stated that INGREZZA's patient persistency has remained very steady since launch and does not differ meaningfully by channel. He noted that while Q1 reauthorizations were more challenging than in prior years, the impact was counterbalanced by the record number of new patient starts.

Laura Chico asked for clarification on where the impact of the INGREZZA sales force expansion is most likely to be felt, whether in driving new patient starts, channel expansion, or increasing adherence.

Answer

Chief Commercial Officer Eric Benevich stated that the primary benefit of the expansion is expected to be in driving new patient starts. He explained that the larger team will increase reach and frequency of contact with providers, particularly in the high-potential psychiatry and long-term care segments. He also noted that the full impact will take time to materialize, with the majority expected in the second half of the year.

Laura Chico of Wedbush Securities requested more color on the revised 800,000 tardive dyskinesia (TD) prevalence estimate, asking about the severity breakdown and its impact on the peak INGREZZA opportunity.

Answer

CCO Eric Benevich stated that while there's no standard definition, literature suggests half to two-thirds of patients are moderate-to-severe. He emphasized that even milder movements can be significantly impactful, and the focus remains on the large, untreated population. He noted the 800,000 figure is supportable and likely conservative, reinforcing the significant growth opportunity ahead as less than 20% of patients are treated.

Laura Chico from Wedbush Securities inquired about the potential for an accelerated regulatory pathway for zorrevonercin, given the strong three-year OLE data, high pre-screening numbers for the Phase 3 trial, and its existing breakthrough therapy designation.

Answer

Interim CEO Ian Smith confirmed that Stoke Therapeutics will present its comprehensive data, including the new 36-month OLE results, to the FDA in the second half of the year. He highlighted that the breakthrough designation was granted based on both seizure reduction and cognitive benefits. While acknowledging the responsibility to seek the fastest path to patients, he noted that the outcome of FDA discussions is uncertain and that the company will provide updates if timelines change.

Laura Chico of Wedbush Securities inquired about the expected timing for gaining insight into the FSGS advisory committee panel meeting date, noting that interactions seem to be on a regular cadence.

Answer

Dr. William Rote, Chief Research Officer, clarified that the specific date for the advisory committee is not yet known but will be shared once it is. He added that given the January 13 PDUFA date, it is reasonable to anticipate the meeting will occur sometime in Q4.

Laura Chico of Wedbush Securities inquired about the sustainability of the patient start form (PSF) metric, which saw an increase in the quarter, and its trajectory for the rest of 2025.

Answer

Chief Commercial Officer Peter Heerma and CEO Eric Dube both responded. Heerma highlighted that strong compliance and persistence build upon a robust base from new PSFs. Dube added that a key sustainable growth driver is upgrading the large number of patients currently on RAS inhibitors who are not yet at the new, more aggressive KDIGO guideline targets.

An analyst on behalf of Laura Chico inquired when Travere might provide revenue guidance for FILSPARI in the IgAN setting, given that the potential FSGS launch would alter the overall revenue trajectory.

Answer

CEO Eric Dube stated that while the company is pleased with the strong uptake in Q4 following full approval, it represents only a single data point. Management prefers to observe more trends before providing formal guidance but is consistently evaluating the possibility.

Laura Chico of Wedbush Securities asked for the company's thoughts on whether guideline bodies like KDIGO might update FSGS treatment guidelines in response to the new observational data presented by the PARASOL initiative.

Answer

Chief Medical Officer Dr. Jula Inrig stated that an update is a potential outcome. She noted that while FSGS guidelines were recently updated, guideline bodies typically revise them when a new treatment becomes available. She anticipates that a full FDA approval for sparsentan in FSGS would be the appropriate trigger for such an update.

Laura Chico from Wedbush Securities asked what underpins management's confidence in the new peak sales estimate for Xphozah and when guidance might be restored. She also inquired about the average duration of therapy for Ibsrela patients.

Answer

CEO Mike Raab expressed high confidence in the $750 million peak sales target for Xphozah, citing the large non-Medicare market and modest penetration required. He stated guidance would be provided after seeing more consistency. For Ibsrela, he noted its average duration of therapy is trending at or better than the market average of about five refills.

An analyst on behalf of Laura Chico from Wedbush Securities asked about the metrics used to monitor the expanded IBSRELA sales force and the source of confidence in achieving peak revenue expectations.

Answer

CEO Michael Raab and CCO Eric Foster identified key metrics as increased sales activity, growth in new and total prescribers, and a corresponding rise in new and refill prescriptions. They cited Q1 having the second-highest prescription demand quarter to date, despite market headwinds, as a primary reason for their confidence in reaching the greater than $1 billion peak sales target.

Laura Chico of Wedbush Securities requested quantitative metrics on XPHOZAH patient retention and free drug volume in 2025, and asked about the use of proceeds from the recent SLR Capital term loan.

Answer

CCO Eric Foster confirmed that both Medicare and non-Medicare patients from 2024 are successfully transitioning to 2025 access pathways, though specific retention metrics were not provided as it is too early. CFO Justin Renz explained the loan strengthens the balance sheet, providing strategic flexibility for growth and pipeline development.

Laura Chico requested clarification on the gross-to-net (GTN) improvement for XPHOZAH during the quarter and asked about the appropriate timing for the company to issue its 2025 guidance for IBSRELA.

Answer

Chief Financial and Operations Officer, Justin Renz, confirmed that XPHOZAH's GTN deduction improved from 21.4% to 19.3% in Q3, driven by patient payer mix and commercial co-pay utilization. President and CEO, Mike Raab, indicated that 2025 IBSRELA guidance would be provided in the early part of next year, after the impact of the expanded sales force becomes clearer.

Laura Chico of Wedbush Securities asked for commentary on Orlodeyo's real-world persistency rates in comparison to injectable prophylactic treatments for HAE.

Answer

President & CCO Charlie Gayer referenced a recent real-world data analysis showing Orlodeyo's one-year persistence rate is statistically identical to injectable competitors TAXHZYRO and HAEGARDA, at approximately 60%. He noted that Orlodeyo was numerically slightly better, which underscores that the drug's profile is highly competitive and meets patient needs effectively in a real-world setting.

Laura Chico inquired about the operational timelines for the pediatric ORLADEYO launch, including supply chain deployment, and how uptake dynamics might differ from the adult launch. She also asked for the starting dose of BCX17725 in the Netherton trial and the trigger for advancing to 12-week dosing.

Answer

Chief Commercial Officer Charlie Gayer stated the company would aim for a minimal lag between approval and commercial availability for pediatric ORLADEYO. He noted uptake dynamics will differ as an oral option could shift the treatment paradigm toward earlier prophylaxis in children. For the Netherton trial, Chief R&D Officer Dr. Helen Thackray confirmed the starting patient dose is 6 mg and clarified that the 12-week dosing portion is a parallel extension to gather longer-term data, not a phase triggered by a specific event.

Laura Chico asked about the characteristics of patients who might be better suited for seralutinib versus sotatercept, and inquired about the powering assumptions for the primary endpoint in the CIRANATA (PH-ILD) trial.

Answer

CEO Faheem Hasnain and CMO Dr. Richard Aranda projected that by seralutinib's launch, a large market of patients who failed, were ineligible for, or had waning effects from sotatercept would exist. They also see potential for combination use and positioning seralutinib as a first-choice therapy for new patients due to its safety profile. For the CIRANATA trial, Dr. Aranda explained they extrapolated from the INCREASE trial, assuming a sicker population and a 30-meter treatment difference, which provides over 94% power.

Laura Chico from Wedbush Securities asked if Sage has quantified its number of active prescribers and the change in average scripts per provider.

Answer

COO Chris Benecchi stated that Sage has not communicated the specific number of active prescribers. CEO Barry Greene added that the key dynamic driving growth is the continuous addition of new writers each quarter who then become repeat prescribers. While he could not quantify the scripts per provider, he reiterated that prescribing offices see significant growth in their PPD patient treatment and use ZURZUVAE as a frontline therapy over 70% of the time.

An analyst on behalf of Laura Chico asked about the potential for near-term revisions to PPD treatment guidelines from organizations like ACOG or AAP and how that might impact commercial uptake.

Answer

CEO Barry Greene stated that the current ACOG guidelines are already 'phenomenal,' as they were updated around the time of launch to specifically mention ZURZUVAE, which has been a significant advantage. COO Chris Benecchi added that these favorable guidelines were one of several key tailwinds supporting the launch, alongside strong media attention and broad payer access.

Laura Chico asked about the evolution of the turnaround time from a ZURZUVAE prescription being written to the medication being delivered to the patient.

Answer

CEO Barry Greene described this as an area of continuous improvement and a key part of ZURZUVAE's value proposition. He stated that while many patients receive the drug within 2-3 days, the company is not satisfied and is working to shorten the median delivery time for all patients. He characterized current performance as 'doing well' but with room to 'get better.'

Laura Chico sought clarification on whether the patient enrollment percentage for the ulixacaltamide interim analysis trigger has changed, the rationale for separating the result releases for Study 1 and 2, and the mechanistic read-through for restarting the Parkinson's program.

Answer

CEO Marcio Souza clarified that the information fraction for the interim analysis (50-75% of patients) has not changed. He explained the separation of study results is a cautious measure to protect the integrity of Study 2 from potential patient bias. The Parkinson's program restart is driven by a strong scientific rationale and a desire to be prepared to launch immediately following positive essential tremor results, leveraging go-to-market synergies.

Laura Chico from Wedbush Securities sought two clarifications: whether the cash runway guidance incorporates a C3G launch and its revenues, and how the average number of injections per patient has trended during the launch.

Answer

CFO Timothy Sullivan confirmed the cash runway guidance 'absolutely does' include the C3G launch, noting the incremental costs are not massive. COO Adam Townsend addressed the second point, stating that the use of every-other-month dosing has been very stable, accounting for 80% to 90% of all SYFOVRE prescriptions, and is expected to remain at that level.

Laura Chico from Wedbush Securities asked about the target patient populations and therapeutic windows for the partnered GBA1 and Friedreich's ataxia programs, and also requested an update on any remaining 2024 milestones.

Answer

CMO Dr. Toby Ferguson deferred specific questions on the partnered programs to Neurocrine, which controls their development, but noted that other approved drugs have paved a path by targeting broad patient populations. He declined to comment on future milestones. CEO Dr. Al Sandrock added that Voyager's frataxin-replacement therapy for Friedreich's ataxia has a distinct mechanism from the approved drug, suggesting potential for additive benefit.

Laura Chico of Wedbush asked a housekeeping question about any additional milestone payments to watch for in 2024 and a strategic question about how positive data from the VY-TAU01 antibody program would impact Voyager's focus relative to its Tracer programs.

Answer

Executive Robin Swartz stated that while a milestone was achieved, the company does not provide guidance on future potential milestone payments, noting they are not included in the cash runway guidance into 2027. Executive Alfred Sandrock addressed the strategic question by explaining that a positive result for VY-TAU01 would likely lead to seeking a partner for late-stage development, given the scale of Alzheimer's trials. He also noted the option to vectorize the antibody for a 'once and done' treatment remains a high-interest possibility.

Laura Chico of Wedbush Securities asked about the definition of success for the upcoming THRIVE trial data, the potential impact of reduced drug exposure on hearing impairment rates, and the rationale for including an active control arm in the STRIVE safety study.

Answer

President and CEO Stephen Mahoney stated that a successful outcome for the THRIVE trial would be an efficacy and safety profile similar to TEPEZZA. He noted that lower drug exposure and a lower Cmax could potentially improve the hearing safety profile, but this remains to be seen. Mahoney explained that the STRIVE study is a standard requirement to build the safety database for a BLA submission, and the active control arm is necessary for it to be a well-controlled study.

Posed two questions: one on the expected kinetics and pace of exon skipping in CONNECT1, and another on what level of splicing correction is considered meaningful for the DM1 program and which functional assessments will be reported from the FREEDOM1 study.

Answer

On exon skipping, the executive noted that NHP data shows the largest increase between the first and second doses, with levels plateauing by the fourth dose, which is expected to be reflective of long-term results. For DM1, greater than 25% splicing correction is considered important, as preclinical models show this leads to robust myotonia correction. The FREEDOM1 update will include functional assessments like vHoT (myotonia), strength tests, and walk tests, though meaningful changes in some functional measures may require multiple doses.

Laura Chico of Wedbush inquired how the changes to the GM1 trial's inclusion criteria might affect potential labeling and asked about the potential impact of non-human primate (NHP) supply chain constraints on preclinical programs.

Answer

CEO Will Chou explained that protocol amendments are subject to regulatory review, but the goal is to treat patients at an earlier stage to maximize the risk-benefit profile. Chief Medical Officer Mark Forman stated that NHP availability has not impacted their preclinical pipeline to date, though it is being carefully monitored.

Laura Chico of Wedbush Securities questioned how the changes to the GM1 trial's inclusion criteria might affect the potential product labeling. She also inquired about the impact of reported non-human primate (NHP) supply chain constraints on the company's preclinical programs.

Answer

CEO Will Chou explained that any protocol amendments are subject to regulatory review and that the goal is to treat patients at an earlier stage to maximize the risk-benefit profile. CMO Mark Forman addressed the second question, stating that NHP availability has not impacted the advancement of their preclinical pipeline to date, though it is being carefully monitored.

Laura Chico of Wedbush asked about the assumed screening failure rates for FTD patients, whether the 60-day waiting period between subjects applies to subsequent cohorts, and the specific points of alignment sought from regulators for the GM1 program.

Answer

Chief Medical Officer Mark Forman explained that he expects a low screening failure rate for FTD due to broad inclusion criteria. He confirmed the 60-day waiting period between patients is in effect for the entire trial to ensure safety. For GM1, he stated the company seeks regulatory alignment on the use of external cohorts and the overall registrational study design.

Laura Chico from Wedbush sought clarification on the main bottleneck for FTD recruitment, asking if it was primarily a diagnosis issue or related to the competitive landscape. Additionally, she requested an update on the ongoing search for a permanent CEO.

Answer

Interim CEO Edgar Cale addressed the CEO search, confirming the Board has engaged Russell Reynolds, the search is active, and they are making good progress in evaluating candidates. Chief Medical Officer Mark Forman explained that FTD recruitment challenges are multifactorial, involving patient identification, competition from other trials, and patients meeting specific eligibility criteria. However, he reiterated that their outreach and genetic testing initiatives are proving successful.

Laura Chico of Wedbush asked for more detail on the pipeline, questioning how the clinical differentiation of BHV-7000, specifically its lack of GABA activation, would be demonstrated in trials. She also inquired about the communication strategy for the upcoming verdiperstat topline results, given its inclusion in a multi-therapy platform study.

Answer

President of Biohaven Labs Michael Bozik explained that by avoiding GABA activation, BHV-7000 is expected to have a superior side effect profile (less somnolence, vertigo) and better compliance, which will be a key point of differentiation measured in clinical trials. CEO Vlad Coric clarified that for the HEALEY ALS platform trial, Biohaven will receive and announce topline data for its asset, verdiperstat, as soon as it reads out around mid-year, without waiting for other assets in the study.

Laura Chico from Wedbush Securities asked if a U.S. co-promotion with Pfizer was considered, given their primary care strength. She also inquired about expected differences in pricing and market access dynamics for the European launch compared to the U.S.

Answer

CEO Dr. Vlad Coric clarified that the company's strategic priority was to secure an ex-U.S. partner, as the U.S. launch is already outperforming expectations, and Biohaven retains full U.S. optionality. For Europe, he explained that while pricing processes are ongoing, the core value proposition of reducing migraine-related disability will be a central argument for payers, similar to the successful strategy employed in the U.S.

Laura Chico of Wedbush Securities asked about patient continuation rates for NURTEC ODT, referencing third-party data that suggested higher continuation for a competitor, and inquired how this aligns with Biohaven's expectations.

Answer

CCO BJ Jones did not provide specific data but suggested competitor data could be skewed by multi-dose regimens. He stressed that Biohaven sees a high degree of patient satisfaction with NURTEC ODT. CEO Vlad Coric added that NURTEC's single-dose efficacy is a more efficient treatment approach and cautioned against misinterpreting script data that reflects the need for second doses with other therapies.

Laura Chico from Wedbush Securities noted a disconnect in market research showing higher familiarity with a competitor but more favorable views on NURTEC's profile, asking how Biohaven plans to modify outreach. She also inquired about capital allocation between EU expansion and pipeline investment.

Answer

CEO Vladimir Coric attributed NURTEC's favorable views to its differentiated label, which he believes will drive adoption in primary care. CCO BJ Jones added that as markets reopen, they will focus on activating patients and broadening physician communication to increase productivity. The capital allocation question was not directly addressed.