Ryan Deschner

Ryan Deschner asked about the expected breakout of pruritus and secondary data by indication for the EXPAND readout in Q4, and how the pruritus bar compares to PFIC and Alagille studies.

Answer

CEO Chris Peetz explained that the EXPAND study patient mix is approximately half biliary atresia with a long tail of other settings, and data will be broken out by relevant groups like biliary atresia. He noted that the commonality across settings is elevated bile acids and cholestatic pruritus, expecting similar treatment objectives and responses as seen in compassionate use.

Ryan Deschner asked about Mirum Pharmaceuticals' plans for breaking out pruritus and other secondary endpoint data by indication for the upcoming EXPAND study readout in Q4, and how the pruritus efficacy bar for EXPAND compares to what was observed in the PFIC and Alagille syndrome studies.

Answer

CEO Chris Peetz explained that the EXPAND study patient mix is roughly half biliary atresia, with other indications forming a longer tail. Data will be broken out in relevant ways, with biliary atresia being a primary focus. He highlighted the common underlying mechanism of elevated bile acids and cholestatic pruritus across these conditions, suggesting that treatment objectives and response potential should be similar to those observed in compassionate use and prior studies like PFIC and Alagille.

Ryan Deschner inquired about the rationale behind the BID (twice daily) dosing decision for the EXPAND study and how dosing instructions might appear on an expanded label for cholestatic pruritus patients. He also asked about any observed impact of the government shutdown on genetic screening programs for Alagille and PFIC.

Answer

CEO Chris Peetz explained that the BID dosing decision for the EXPAND study was empirical, based on observations from compassionate use settings at elevated dose levels that showed strong patient responses. He then confirmed that the company has seen no impact from the government shutdown on its interactions, customers, or business operations related to genetic screening programs.

Ryan Deschner of Raymond James Financial questioned the primary drivers behind the strong growth in LIVMARLI sales and the specific impact of the new tablet formulation on prescription volumes.

Answer

Co-Founder & CEO Chris Peetz and COO & President Peter Radovich attributed the growth to increased awareness and diagnosis of later-onset PFIC, which expanded the market beyond initial expectations. Radovich noted the tablet was launched in June, so its impact was not reflected in Q2 results, but early feedback is positive.

Ryan Deschner asked about the expected impact of the LIVMARLI tablet formulation on demand and competitive positioning, the proportion of Alagille patients likely to switch, and whether it could drive meaningful new patient demand.

Answer

CEO Christopher Peetz described the tablet as a highly attractive option for patients over 25 kilos due to reduced pill burden. He anticipates a substantial minority of patients will find it appealing and believes it could drive both switches from the liquid solution and new patient starts, especially among teenagers and young adults seeking convenience.

Ryan Deschner asked about anticipated commercial team expansion for a broader pruritus indication post-EXPAND study and the current sales impact from growing average patient weights.

Answer

CEO Chris Peetz stated that no field team expansion is expected for the potential new indication, as it leverages the existing prescriber base. Regarding patient weight, he described the impact as a modest and gradual increase in the average dispense value, which is tempered by new infant patient starts.

Ryan Deschner asked if there were any notable differences in the end-of-Phase II meeting minutes for oral VK2735 compared to the injectable version. He also inquired if any patients in the ongoing maintenance study would transition to using the autoinjector.

Answer

Viking Therapeutics' President and CEO, Brian Lian, stated that while details of FDA discussions couldn't be disclosed, the feedback for the oral end-of-Phase II meeting was consistent with what was heard for the subcutaneous formulation. He confirmed that patients in the maintenance study would not transition to the autoinjector, as the study is well underway using vial and syringe.

Ryan Deschner asked if there were any significant differences in the End-of-Phase 2 meeting minutes for oral VK2735 compared to the injectable version. He also inquired whether any participants in the ongoing maintenance study would transition to using the autoinjector device.

Answer

Brian Lian, President and CEO, stated that while he couldn't detail FDA discussions, the feedback from the oral and subcutaneous End-of-Phase 2 meetings was consistent. He confirmed that patients in the maintenance study would not transition to the autoinjector, as the study is already in progress using vial and syringe.

Ryan Deschner asked about the next clinical steps for validating a maintenance regimen and potentially getting it on a future label, including the feasibility of adding an expansion arm to the VANQUISH study. He also inquired about any impact of the government shutdown on VANQUISH enrollment or the amyloid program timing.

Answer

Brian Lian (President and CEO, Viking Therapeutics) stated that adding an expansion arm to VANQUISH is challenging. The next step for maintenance would likely be a longer Phase 2b or Phase 3 study, depending on the data. He reported no impact from the government shutdown on VANQUISH enrollment or the amyloid program, but noted a potential impact on the timing of the end-of-Phase 2 meeting for oral VK2735.

Ryan Deschner asked about the next clinical steps for validating a maintenance regimen if the current study shows compelling evidence, and whether an expansion arm could be added to the Vanquish study. He also inquired about any impact of the government shutdown on Vanquish enrollment or the amyloid program timing.

Answer

Brian Lian (President and CEO, Viking Therapeutics) stated that adding an expansion arm to Vanquish is challenging. The next step for maintenance would likely be a longer study (phase 2B or phase 3) depending on the data. He reported no impact from the government shutdown on enrollment or the amyloid program, but noted potential delays for the end-of-phase two meeting for oral VK2735.

Ryan Deschner of Raymond James inquired about the potential for increased patient satiety in the longer Phase II oral study of VK2735 and asked if the upcoming data readout would include results from all cohorts, including the maintenance dosing arm.

Answer

President & CEO Brian Lian responded that while satiety signals can be inconsistent across studies, they were observed in the Phase I trial. He confirmed that the Phase II data release will include all parallel cohorts simultaneously, noting the maintenance arm that steps down in dosage will be particularly interesting.

Ryan Deschner questioned the potential clinical positioning of Viking's amylin receptor agonist candidate and asked if the company would evaluate it as a co-formulation with VK2735 early in its development.

Answer

Brian Lian, President and CEO, explained that the initial clinical study for the amylin candidate will be a standard single-ascending and multiple-ascending dose (SAD/MAD) trial. While the company is interested in evaluating a combination regimen with VK2735, this would likely occur next year, after establishing the single-agent profile.

Ryan Deschner inquired about the specifics from the end-of-Phase II meeting for VK2735, seeking details on the number of patients, study size, and potential comparator arms for the upcoming Phase III trials.

Answer

CEO Brian Lian explained that full study design details will be provided upon trial initiation. He confirmed the program will adhere to regulatory guidance, requiring at least 4,500 participants across two studies: one for obesity and another for obesity with type 2 diabetes. These trials will utilize multiple doses and have a 52-week treatment duration.

Ryan Deschner of Raymond James Financial inquired about the target age range for the MLM label, the age distribution of patients enrolled in the CELVA trial, and whether the trial's over-enrollment was more a reflection of high demand or easier-than-expected patient identification.

Answer

President and CEO Wes Kaupinen explained that the CELVA trial enrolls patients aged three and up, but the primary analysis for the label will focus on the six-and-older cohort, with potential label expansion to younger patients pending data. He attributed the over-enrollment to a combination of high demand, effective patient identification, and the strong execution of Palvella's internal clinical operations team.

Ryan Deschner of Raymond James Financial questioned the timeline for feeling the full impact of key growth drivers like the DTC campaign and ALK co-promotion, their potential to sustain growth through Q4 seasonality, and what indicators suggest Nefi is capturing the untapped market of non-epinephrine users.

Answer

Co-Founder, President, and CEO Richard Lowenthal expressed confidence that the drivers would have a notable impact during the back-to-school season and would sustain quarter-over-quarter growth through Q4. He noted that penetration into the untapped market is currently supported by anecdotal evidence of new patients, with plans for a more thorough market analysis later in the year to quantify this trend.

Ryan Deschner inquired about the impact of inventory on Q1 2025 sales figures and the change in the gross-to-net discount from Q4 2024 to Q1 2025.

Answer

President and CEO Richard Lowenthal stated that inventory had very little influence on Q1 sales. CFO Kathleen Scott explained that the gross-to-net discount was over 60% in Q1, down from Q4, and is expected to trend towards 50% as insurance coverage improves.

Ryan Deschner of JMP Securities inquired about the anticipated sales ramp for the 1-milligram neffy dose and sought an update on ARS Pharma's progress toward its 80% unrestricted market access goal.

Answer

Richard Lowenthal, Co-Founder, President and CEO, explained that the 1mg dose, representing 23% of the market, is additive and expected to significantly boost the sales ramp, as early adoption is heavily weighted toward children. He stated that ARS Pharma will achieve 51% unrestricted commercial access by April 1 and is on track to reach nearly 80% by July 1, following a key agreement with Zinc which covers CVS Caremark.

Ryan Deschner from Raymond James inquired about the early launch split between cash-pay and co-pay prescriptions for neffy and asked how multiple-unit prescriptions are logged in third-party data.

Answer

Executive Eric Karas explained that cash payments are slightly higher than expected, driven by strong patient interest. He detailed that about a third of scripts are covered directly, with prior authorizations succeeding 50-55% of the time. Executives Richard Lowenthal and Eric Karas clarified that while data providers like IQVIA log each device as a unit, their co-pay program can consolidate charges for patients, and the number of units per prescription aligns with their expectations for patients needing multiple devices.

Ryan Deschner of Raymond James Financial asked about the timeline for feeling the full impact of the DTC campaign, the AOK promotion, and the 1mg dose, questioning if they would align with the August sales peak. He also asked if these factors could sustain Q4 growth and what indicators would signal penetration into the untapped market of non-epinephrine users.

Answer

CEO Richard Lowenthal stated he expects the DTC campaign to have a notable impact on the August-September peak due to pre-scheduled appointments and expressed confidence in sustained quarter-over-quarter growth through Q4. He noted that while current data on market expansion is anecdotal, it suggests a healthy mix of new patients and switchers, with a more formal analysis planned for later in the year once a larger market share is achieved.

Ryan Deschner of Raymond James inquired about the proportion of Yutrepia patient starts associated with paid drug versus free drug programs and the breakdown of starts between new patients and switches from other treprostinil products.

Answer

CFO & COO Michael Kaseta reported that less than 50% of starts utilized the free 28-day voucher program, which is in line with launch expectations. CCO Scott Moomaw explained that while the focus is on new-to-prostacyclin patients, they have been surprised by the high volume of switches from Tyvaso DPI and other products. CEO Roger Jeffs added this switch volume from intolerant patients was a key driver of the launch's upside.

Ryan Deschner from Raymond James inquired about the anticipated payer mix between commercial and public for PAH and PH-ILD, and asked for details on the recently filed '494 patent infringement suit.

Answer

Chief Commercial Officer Scott Moomaw provided the expected payer mix: approximately 50% Medicare, 35% commercial, 10% Medicaid, and 5% other. On the legal question, General Counsel Russell Schundler declined to comment on the specifics of the active '494 patent litigation, stating that the company's position is outlined in the public complaint.

Ryan Deschner from Raymond James asked for more details on the next-generation nebulizer included in the Pharmosa agreement, specifically its development stage and whether it is currently used with other commercial products.

Answer

CEO Dr. Roger Jeffs explained that specific details about the nebulizer are being withheld at this time due to its proprietary nature and ongoing intellectual property efforts, with more information to be disclosed in coming quarters. He reiterated its key features as an iPhone-sized, portable, breath-actuated device that allows for precise drug delivery and dosing.

Ryan Deschner of Raymond James Financial asked about any other notable trends from post-hoc analyses of the CORAL or RIVER trials that have influenced dosing strategy and requested specifics on data to be presented at the upcoming ERS conference.

Answer

Chief Development Officer James Cassella confirmed that deep-dive analyses reinforce that the 54mg dose is the key anchor dose. CEO Jennifer Good added that there were no surprises in the safety profile. She clarified that RCC data will be presented at ERS, while the more extensive IPF data from the CORAL trial is slated for presentation at the CHEST conference in October.

Ryan Deschner from Raymond James asked which exploratory metrics, such as EXACT or sleep cough frequency, would be included in the upcoming Q2 IPF chronic cough data readout. He also inquired about potential patient stratifications for a future late-stage RCC study.

Answer

Executive Jennifer Good responded that the top-line Q2 IPF data will include the primary endpoint and key secondary endpoints like EXACT2 and CSS, but she was uncertain about other exploratory metrics. For future RCC studies, she stated there are no plans to stratify by cough frequency (e.g., moderate vs. severe), as the drug showed a strong effect across the entire range of patients.

Ryan Deschner from Raymond James inquired about the timeline for a DEA decision on scheduling after the HAP results are available and asked about the specific dose of the comparator drug, butorphanol, used in the study.

Answer

CEO Jennifer Good explained that the DEA review process occurs late in the drug approval cycle, as part of the NDA review, and sometimes a referral to the DEA is not even made by the FDA. She stated the butorphanol dose is a 6-milligram infusion over one hour, a dosage level that was agreed upon with the FDA to mimic the pharmacokinetics of an existing inhaled version while allowing for proper study blinding.

Ryan Deschner of Raymond James Financial asked how much of Ibsrela's Q2 sales growth was attributable to the expanded sales team and whether to expect continued script pull-through acceleration. He also inquired about future Q1 seasonality.

Answer

CCO Eric Foster confirmed the expanded sales team's increased activity was a significant driver of growth in prescribers and prescriptions, and he expects this to continue. Regarding seasonality, he acknowledged that Q1 is typically the most impacted quarter and that the company remains mindful of potential summer vacation impacts in Q3.

Ryan Deschner from Raymond James asked for more details on the Q1 IBS-C market contraction, the long-term gross-to-net target for XPHOZAH, and the status of the product's launch in China.

Answer

CEO Michael Raab and CCO Eric Foster attributed the market contraction to a consistent seasonal trend that is more apparent as IBSRELA's volume grows. Regarding gross-to-net for XPHOZAH, CFO Justin Renz stated it was 32% in Q1 (excluding the reserve release), and Raab advised modeling 30% +/- 5% going forward. Raab noted there were no new updates to share on the China launch.

Ryan Deschner of Raymond James inquired about XPHOZAH's promotional sensitivity compared to IBSRELA's early launch and asked for expectations on XPHOZAH's long-term gross-to-net percentage.

Answer

CCO Eric Foster indicated that XPHOZAH's promotional sensitivity remains strong and consistent with 2024 levels. CFO Justin Renz stated it is too early to provide long-term gross-to-net guidance but expects it to be less favorable in 2025 than in 2024 due to the changing payer mix.

Ryan Deschner inquired how much of the recent IBSRELA script uptick is attributable to the expanded sales team, whether the team is fully deployed, and asked for updates on ex-U.S. partnerships for XPHOZAH, including the timeline for Chinese approval.

Answer

President and CEO, Mike Raab, and Chief Commercial Officer, Eric Foster, confirmed the sales team is fully trained and deployed. They noted that early positive impacts are visible from the first hires, which gives them confidence for future growth. Raab also stated that Chinese approval for XPHOZAH is still anticipated by year-end, with no new updates on other partnerships.

Ryan Deschner from Raymond James Financial asked if Empaveli launch metrics will be broken out by indication and inquired about the expected burden of prior authorization for C3G and ICMPGN patients.

Answer

EVP David Acheson stated that initially, total start forms will be reported, with more granular data provided over time. CMO Dr. Caroline Baumal explained that the REMS certification is a standard, one-time process for physicians and is not expected to be a significant burden.

Ryan Deschner from Raymond James Financial asked if future Empaveli launch metrics, like start forms and REMS enrollments, will be broken out by indication (PNH, C3G, ICMPGN). He also inquired about the anticipated burden of prior authorization for the new indications.

Answer

EVP David Acheson stated that initially, total start forms will be reported, with a potential breakdown by indication over time as more data becomes available. CMO Dr. Caroline Baumal added that the one-time REMS certification is standard for complement inhibitors and is not expected to be a significant burden for physicians.

Ryan Deschner from Raymond James Financial asked if future Empaveli launch metrics will be broken out by indication (PNH, C3G, IC-MPGN) and about the expected burden of prior authorization for the new indications.

Answer

EVP David Acheson stated that initially, they will report total start forms and will break down the data by indication over time as more information becomes available. CMO Dr. Caroline Baumal commented that the REMS certification is a one-time process for physicians and is not expected to be a significant burden.

Ryan Deschner from Raymond James asked if future Empaveli launch metrics will be broken out by indication and inquired about the expected burden of prior authorization for C3G and IC-MPGN patients.

Answer

EVP David Acheson said that initially, total start forms will be reported, with breakdowns by indication to follow over time. CMO Dr. Caroline Baumal noted that the one-time REMS certification is standard for complement inhibitors and is not expected to be a significant burden for physicians.

Ryan Deschner asked about the likelihood of inventory drawdowns and co-pay funding shortages having a significant impact on future first-quarter sales and inquired about the impact of the new DTC campaign.

Answer

Executive Timothy Sullivan stated that the Q4 inventory build was a specific event tied to the competitor's CRL and is now settled. He views the co-pay issue as a one-time event with a continuing effect, not something that would cause a similar inventory dynamic in the future. An unnamed executive added that the DTC campaign has been very positive, driving a 30% higher average quarterly traffic to the website in Q1 2025 compared to the 2024 average.

Ryan Deschner from Raymond James asked if gross-to-net expectations for 2025 have evolved and about the expected impact of the new DTC campaign.

Answer

CFO Tim Sullivan confirmed that the gross-to-net guidance for 2025 remains unchanged in the low-to-mid 20% range. EVP of Commercial David Acheson noted it was early to measure the full DTC impact but shared a positive early indicator: a single TV spot during a high-profile event drove approximately 3,000 visits to the company's website.

Ryan Deschner from Raymond James asked about the late-stage clinical strategy for the WVE-007 obesity program, inquiring about the potential design of a maintenance therapy trial and how the drug might be positioned on its initial label (e.g., monotherapy, add-on, or maintenance).

Answer

President and CEO Paul Bolno stated that the company is actively planning for late-stage studies, including an 'off-ramp' trial design where patients stable on a GLP-1 would be switched to WVE-007. He noted these studies are being designed to generate meaningful data for clinical use and to support a broad label covering monotherapy and maintenance use cases for reimbursement.

Ryan Deschner of Raymond James inquired about the use of concomitant therapies like vitamin E, patient lifestyle management, details on NASH resolution, weight loss effects, the interpretation of PRO-C3 biomarker data, and potential correlations with ALT reduction.

Answer

Professor Vlad Ratziu clarified that patients on other therapies were effectively non-responders and received standard lifestyle counseling, which is typical for NASH trials. President and CEO Allen Baharaff added that data on other histological endpoints like NASH resolution is still being analyzed and is forthcoming. He also noted that the drug appears to be weight-neutral, and while PRO-C3 data is encouraging, it is viewed as supportive evidence for now, cautioning against over-interpretation. He confirmed that detailed correlation analyses for ALT reduction have not yet been performed.