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Chief Commercial Officer Joshua Schafer stated that the time from enrollment to paid drug is decreasing dramatically, sometimes to as little as 72 hours, though it varies by payer. President and CEO Neil McFarlane noted it's too early for formal persistency data on new patients but confirmed high retention among converted EAP patients. Regarding Olpruva, McFarlane emphasized the significant commercial synergies with Myclifa, stating the infrastructure is highly overlapped and the incremental investment is very limited.

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Chief Commercial Officer Joshua Schafer confirmed that new patients are coming from both the diagnosed and undiagnosed cohorts as a direct result of the company's awareness initiatives. Chief Financial Officer R. Clifton addressed inventory, stating that the company manages levels using a target days on hand and successfully maintained that target at the end of Q1, consistent with the end of Q4 2024.

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CFO R. Clifton explained that the $10.1 million in Q4 MIPLYFFA revenue included initial patient dispenses, refills for some early enrollees, and the establishment of target inventory levels at the specialty pharmacy. CCO Joshua Schafer noted the rapid EAP conversion and stated that future growth will focus on diagnosed but untreated patients and new diagnoses, implying a different cadence than the initial bolus.

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President and CEO Neil McFarlane confirmed that Zevra is on track with its 8-to-12-week post-approval timeline for getting MIPLYFFA into the channel, with no significant hurdles remaining besides logistics. Chief Commercial Officer Joshua Schafer added that the reimbursement process for patients new to the drug is identical to that for former EAP patients, with no additional steps or hurdles anticipated.

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CEO Daniel Barber confirmed there have been no major data requests from the FDA, only standard interactions. Chief Commercial Officer Sherry Korczynski detailed awareness efforts, including medical conferences and CME events, noting positive feedback from HCPs who believe Anafilm will improve patient carry rates and lower barriers to use.

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Executive Sherry Korczynski explained that awareness is growing significantly through medical team engagement at numerous conferences, with progress tracked against a baseline ATU trial from December 2024. For the launch curve, she noted the market's seasonality peaks during the back-to-school period. While acknowledging payer timelines can take months, she expressed confidence in achieving majority market access by the crucial August-September period, suggesting the adoption curve might be similar to neffy's.

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CEO Daniel Barber stated the immediate focus for Libervant is ensuring continued access for current patients (ages 2-5) while they contest the court ruling. For Anaphylm, Executive Sherry Korczynski outlined a comprehensive awareness strategy, including a significant presence at medical conferences, CME programs, publications, and building relationships with patient advocacy groups.

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Chief Medical Officer Carl Kraus stated that while the primary goal of the FDA meeting is to align on the program's completeness, the company sees opportunities to use data from studies like OASIS to differentiate the label. CEO Daniel Barber agreed, noting the potential for a unique label. CFO Ernie Toth commented on legacy revenues, explaining that while Suboxone remains an important cash generator, the company anticipates some market dislocation going forward.

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President and CEO Robert Blum and EVP & CCO Andrew Callos explained the EU launch is a country-by-country process gated by reimbursement negotiations, unlike the single U.S. launch. Germany is the initial focus. They also noted the EU manages risk via labeling rather than a formal REMS program, and spending is gated by reimbursement milestones.

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Fady Malik, EVP of R&D, emphasized that AMBER is a Phase II dose-finding study. Stuart Kupfer, SVP and CMO, clarified that results will be disclosed from the totality of the data, not by individual cohorts. He described the study as a careful, systematic dose-titration effort to identify the optimal benefit-risk profile, starting with lower doses.

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EVP of R&D Fady Malik explained that while the protocol amendment was implemented last year, not much data has yet accumulated with the 6-month monitoring schedule. He emphasized that the thousands of 3-month monitoring echoes showed very little impact on dosing or safety. He confirmed the overall safety profile of aficamten remains strong and consistent as more data is gathered, with no other protocol amendments currently planned for FOREST.

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Robert I. Blum, President and CEO, and Fady Malik, EVP of R&D, explained that CK-089 is a third-generation molecule from a different chemical scaffold. It is more potent, has better pharmaceutical properties, and is being developed for a specific muscular dystrophy, not ALS, applying key learnings from prior programs.

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CEO Catherine Owen Adams explained that the company is bifurcating its strategy between near-term commercial efforts and long-term brand maximization now possible through 2038. She confirmed the current DTC campaign is paying dividends and that the company was delighted to extend its agreement with Ryan Reynolds for the 'More to Parkinson's' campaign, which has been a key driver of awareness.

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Executive Brendan Teehan responded that it is still early days for the new campaigns. So far, the prescriber mix reflects previous quarters, and there has not been a substantial change in the age of patients. He noted the caregiver campaign helps families identify subtle changes in loved ones more readily.

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CFO Nicole LaBrosse clarified that while current year guidance is updated quarterly, long-term guidance is updated annually at the start of the year. President and CEO Dr. Helen Torley reported that for Ocrevus, Roche has indicated about 50% of new subcutaneous patients are new to the brand, with the other 50% switching from IV. Dr. Torley also confirmed strong partner interest in the high-volume auto-injector, with multiple discussions underway.

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President and CEO Dr. Helen Torley confirmed the auto-injector component work is designed to be suitable for multiple partners. CFO Nicole LaBrosse explained that while the ASR is ongoing, approximately 80% of the shares were delivered and retired in December 2024, with final settlement occurring by the end of Q1 2025.

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President and CEO Dr. Helen Torley confirmed the PDUFA action date is expected in April 2025. She clarified that while Halozyme advises on the ENHANZE formulation, Argenx is leading the development and regulatory work for the device itself, and she could not share further details on the process.

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Executive Joseph Todisco confirmed CorMedix is providing significant support to the contracted LDO and remains hopeful for a midyear implementation start. He noted the potential usage volume could change and that the company is working to accelerate the rollout. Regarding new customers, Todisco mentioned progress with another LDO has been slow, but they are now working through JV partners and are actively building inroads with smaller customers.

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CEO Joseph Todisco explained that DefenCath adoption is almost entirely driven by top-down institutional protocols, which takes longer to set up but leads to a faster patient conversion ramp. He noted that claims data lags, but the initial trend shows facilities starting with fee-for-service patients before expanding to other payers. Regarding expenses, CFO Matt David and CEO Joseph Todisco confirmed they expect Q4 operating expenses to be in the 15% to 17% range, with significant R&D upticks not expected until 2025.

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CEO Joseph Todisco stated that consistent, repeat orders from several hundred clinics suggest strong patient pull-through with minimal trade stocking in the $5.2 million figure. He affirmed that reaching breakeven EBITDA by the end of 2024 is achievable, assuming the current sales trajectory from small and mid-sized operators continues and new, larger accounts are onboarded, which would cover the guided quarterly OpEx of $15 million to $18 million.

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Chief Medical Officer Michael Shetzline confirmed that patients with both stoma and colon-in-continuity (CIC) are achieving enteral autonomy. He stated that the data shows a numerical benefit across all subpopulations. Shetzline also noted that patients who achieved enteral autonomy during the double-blind period have maintained it in the open-label extension study, with the total number of patients reaching autonomy now at 27.

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CEO Thomas McCourt identified LINZESS demand drivers as overall market growth, increased patient care-seeking, broader physician adoption, the pediatric indication, and the future potential for an OTC switch. For apraglutide, he detailed ongoing market preparation, including disease awareness programs and the creation of a critical patient services hub to support the launch.

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Chief Medical Officer Michael Shetzline stated that remaining work includes finalizing the commercial supply chain and supporting CMC documentation for the enhanced vial kit. He added that feedback from KOLs is excellent, praising the once-weekly dosing and tolerability. CEO Thomas McCourt and CBO Andrew Davis added that market preparation is leveraging new ICD-10 codes to identify patients and guide the go-to-market strategy.

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CEO Thomas McCourt detailed the pre-launch strategy for apraglutide, which includes a disease education program targeting the GI community and patients to raise awareness and help identify eligible patients. CFO Sravan Emany addressed the pediatric launch, stating that while still early, they are encouraged by strong new-to-brand prescription growth, noting that a large portion of the 18% NBRx growth is from the 72-microgram dose approved for pediatric use.

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President and CEO Gene G. Kinney emphasized two key factors: the risk-benefit profile (ARIA vs. amyloid reduction) and reducing treatment burden via a once-monthly, at-home subcutaneous injection. Executive Mark Johnson added that initial data from ~225 non-homozygous APOE4 patients will be shared mid-year, with data from homozygous carriers and long-term extensions to follow. The primary goal is to define the optimal dose for a registration-enabling trial.

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Phil Tennant, Chief Commercial Officer, stated he was attracted by the high unmet need in RRP, which aligns with his experience in rare oncology. His priorities are to right-size the commercial footprint and build an integrated commercial and medical affairs team. Dr. Helen Sabzevari, President and CEO, added that while the UltraCAR-T platform remains a priority, pausing two trials was a strategic decision to focus resources on PRGN-2012. She announced that Phase Ib enrollment for PRGN-3006 in AML is complete, and the company is preparing for FDA discussions.

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