Kristen Kluska • Cantor Fitzgerald

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CEO Vishwas Seshadri and CCO Madhav Vasanthavada explained that "identified patients" are those with severe RDEB and large, chronic wounds, as determined by physicians. They clarified that patients outside the initial QTCs are already being referred to the active centers. They also highlighted that testimonials from clinical trial participants in their "Strong Together" network have been highly effective in motivating new patients and families.

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CEO Vishwas Seshadri and CCO Madhav Vasanthavada responded that surgeons do not view the procedure as a barrier, as it is not technically difficult and is supported by experienced cell therapy coordinators at the target treatment centers. Regarding the FDA review, Vishwas Seshadri stated that while they don't have a definitive answer yet, the assumption is the review will primarily focus on the new information submitted to address the CRL items, as is standard for the process.

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CEO Stephen Dilly confirmed the strategy is to secure a large number of early-stage assets, which will generate significant value as they mature, with revenue shifting from milestones to direct material supply. He affirmed that partners strongly prefer fewer transitions, making an in-house path attractive. He also noted a recent targeted cash top-up was designed to fund this scaling. CFO Georgia Erbez added that more clarity on the financial impact will be provided with 2026 guidance.

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CEO Stephen Dilly explained that CDMOs are ideal early adopters due to their forward-planning nature and existing customer networks. He stated that to win over drug innovators directly, Codexis must execute on demonstrating key proof points: performance at scale, shorter production times, and the cost and speed advantages of building a GMP facility. Mr. Dilly emphasized that the company has moved from invention to execution, focusing on providing tangible evidence to potential customers.

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SVP of Research Stefan Lutz identified overall yield and material quality (impurity profile) as the two key success parameters for synthesis. COO Kevin Norrett explained that partnership disclosure details will vary by partner, but the focus is on advancing projects to revenue generation. President and CEO Dr. Stephen Dilly stated that the company is not assuming any future revenue from PAXLOVID.

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Chief Commercial Officer Joshua Schafer explained that diagnosed but untreated patients were often identified before therapies were available and are now being re-engaged as awareness grows. President and CEO Neil McFarlane pointed to the 26% quarter-over-quarter revenue growth to $21.5 million as the key metric for paid drug conversion. Schafer added that since Myclifa's label is for use with miglustat, they have seen very limited reimbursement pushback on the combination, successfully using medical exception pathways for patients not yet covered.

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President and CEO Neil McFarlane highlighted the European market potential with an estimated 1,100 patients and confirmed the plan to leverage MIPLYFFA's combination data with the established miglustat therapy. Chief Medical Officer Adrian Quartel added that the company is targeting an MAA filing in the second half of 2025, supported by new long-term data. Neil McFarlane also characterized the cash runway guidance, which excludes PRV proceeds, as an appropriately conservative estimate.

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CEO Daniel Barber stated the primary lesson was the importance of closely following FDA guidance. He noted the competitor's CRL appeared related to an incomplete study. He contrasted this by highlighting that Aquestive's package was designed to be comprehensive, citing the inclusion of a repeat dose arm in its oral allergy syndrome study as specifically requested by the FDA.

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CEO Daniel Barber clarified that their primary interactions have been with the deputy division head, who remains in place along with the rest of the review team, so they do not believe the division head's retirement will have an impact. He stated that from a review standpoint, everything appears to be in a 'normal state.' Barber expressed that the company is prepared for and hopes for an Ad Comm but has some doubts about the government's ability to organize one.

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CEO Daniel Barber confirmed they have hired external experts and have a dedicated internal team preparing for a potential Ad Comm. He also noted a significant opportunity exists among individuals never prescribed a rescue product. Executive Sherry Korczynski predicted the market will shift from a 'needle vs. non-needle' to a 'devices vs. oral film' dynamic, which she believes will expand the market due to Anaphylm's convenience.

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Dr. Elsa Rossignol, a Principal Investigator for the REVEAL trials, responded that improvements and milestone gains are observed continuously over time at most follow-up visits, not just at a single point. She stated that the benefits have not plateaued and that some critical functional gains, such as walking with a walker, are not even captured by the specific milestone scale, indicating broader, ongoing improvements.

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CEO Sean Nolan explained that Taysha will look for differentiation in the time to achieve functional gains and the overall magnitude of the response. President and Head of R&D Sukumar Nagendran added that translational data from animal models and the logical benefit of higher MECP2 protein levels also support the expectation of a superior clinical impact at the higher dose.

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CEO Sean Nolan explained that the strategy involves trial designs where the patient may serve as their own control, supported by a completed natural history data analysis. He highlighted a focus on objective, functional gains and milestones, such as hand and gross motor function. President and Head of R&D Sukumar Nagendran added that the totality of clinical evidence will be crucial in discussions with the FDA.

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CEO Sean Nolan stated that preclinical data showed increased survival and improved gait at the high dose, and they expect to see consistent effects across clinical domains. He acknowledged the low dose set a high bar. President and Head of R&D, Sukumar Nagendran, added that while preclinical work justifies testing a higher dose, the ultimate goal is to find the ideal dose that regulators will approve and that will make the most difference for patients, which will be driven by the clinical data.

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CEO Linda Marbán clarified that the successful resolution of the Form 483 observations was handled by the FDA's inspection team independently of the CRL. She confirmed the HOPE-three timeline is paused pending FDA guidance and stated the primary goal of the Type A meeting is to understand the FDA's resistance to the current data and define the clearest path to approval.

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CEO Linda Marbán highlighted the statistical significance of cardiac MRI data as a key objective measure that requires few patients to show a treatment effect. She confirmed that promising 4-year open-label extension data will be presented at the PPMD meeting in June. She also expressed confidence in the FDA process, noting that key reviewers remain on the file and are actively engaged.

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CEO Linda Marbán confirmed that most patients treated have been non-ambulatory, the population where cardiomyopathy is most manifest. She contrasted deramiocel's simple infusion and favorable safety profile with the complex care required for gene therapies, stating no extensive care team is anticipated. She also affirmed that the current facility, capable of servicing 250-500 patients annually, is sufficient to meet early launch demand until the expanded facility comes online.

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CEO Linda Marbán explained that while all patients showed benefit, the improvement was less pronounced in those with LVEF below 45% due to more existing scar tissue, highlighting the importance of early treatment. She described the patient journey, noting most children get a cardiac MRI before age 10, and that treatment could be triggered by either cardiac scar or a drop in ejection fraction. Marbán believes deramiocel's approval will accelerate and improve cardiac monitoring and treatment in the DMD community.

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CEO Dr. Matthew Klein stated the goal for the Huntington's program is to align with the FDA on the Phase III efficacy trial design and to clarify the pathway for accelerated approval, using either existing or future data from the open-label extension. Regarding Translarna, he explained that Article 117 allows individual European countries to make the drug commercially available despite the nonrenewal of the marketing authorization. He confirmed this has been adopted by about half the countries, securing roughly 25% of prior European revenue for the rest of 2025, but noted the duration is variable by country and does not require a centralized renewal.

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CEO Dr. Matthew Klein confirmed that labeling discussions with the FDA for Sephience are far along and feel ahead of schedule, with no impact from recent agency changes. CBO Eric Pauwels added that patient interest is strong across all types, including naive and previously treated, driven by social media and engagement with the company's disease awareness website.

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CEO Dr. Matthew Klein highlighted that upcoming data will show over 97% of PKU patients on sepiapterin can liberalize their diet, which is a key driver for therapy adoption. For vatiquinone, Dr. Klein noted its potential as the first therapy for pediatric FA patients, while CBO Eric Pauwels detailed the launch strategy focusing on the unmet need in pediatrics and adults who cannot tolerate current therapies.

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CEO Anish Bhatnagar confirmed that no new safety signals have emerged in the post-marketing setting compared to the clinical trials, and discontinuation rates are lower. CFO Jim Mackaness acknowledged the strong Q2 revenue but suggested that the initial momentum might normalize over the next couple of quarters, without providing specific forward-looking guidance.

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Executive Anish Bhatnagar stated that Soleno will have a significant presence at the conference, with commercial, medical affairs, and clinical teams attending. CCO Meredith Manning explained that Soleno's participation in the Medicaid Drug Rebate Program (MDRP) allows states to add Vykat XR as of May 1, but policy adoption timelines vary by state, with several already providing coverage.

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Emil Kakkis, Founder, President & CEO, affirmed that the FDA considers the totality of data for rare disease programs. He stated that strong supportive data, such as on pain, would be helpful in any scenario. However, he reiterated his confidence that the fracture data will be strong, based on Phase II results showing very few fractures in patients established on the treatment.

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CEO Emil Kakkis and CMO Eric Crombez responded that the majority of patients in the trial are pediatric, with a smaller number of adults included to support a broad label. Regarding the data release, Kakkis stated that the reviews for the Orbit and Cosmic studies are not scheduled for the same day and the company has not yet decided if the results will be announced together.

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CEO Emil Kakkis and CMO Eric Crombez explained that patients are not expected to titrate down standard care during the blinded period due to safety concerns. A meaningful primary endpoint is the reduction of chronically elevated, cognitively impairing ammonia levels to the normal range. They drew a parallel to their GSD1A program, where patients reduced standard care only after entering an unblinded follow-up period.

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CEO Emil Kakkis explained the 20% fracture reduction estimate for bisphosphonates comes from the two successful of five randomized studies. He noted the 67% reduction seen in their Phase 2 was on top of bisphosphonates. He agreed that increased travel for clinic visits could actually increase fracture incidence in the placebo arm, potentially widening the detectable difference between the groups.

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CEO Jack Khattar attributed KELBRI's success to a combination of factors, including a favorable label update clarifying its unique mechanism of action and a strong push into the adult segment. Regarding M&A, he confirmed that Women's Health is now a key area of interest alongside CNS. CFO Timothy Dec stated the company's pro forma cash position is between $240 million and $260 million.

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CEO Jack Khattar noted that comorbidities like mood disorders affect 40-60% of ADHD patients. He explained that while the company cannot promote off-label, the label update provides important scientific information for physicians about Qelbree's unique mechanism. For business development, he said the company is interested in specialty areas like dermatology or urology and rare diseases, provided the opportunities offer scale through multi-asset situations.

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President and CEO Bradley Campbell described the switching decision as a continuum, noting that earlier switches are often patients experiencing a clear decline on their current therapy. He expressed a long-term goal for Pombility/OpFolda to be established as the best therapy, leading to proactive switches. Chief Development Officer Dr. Jeff Castelli added that the pool of potential switchers will naturally grow over time as more patients on existing therapies experience disease progression.

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Chief Development Officer Dr. Jeff Castelli explained that the DMX-200 trial targets the majority of FSGS patients who still have significant proteinuria despite standard care. He stated that higher proteinuria levels are likely indicative of an uncontrolled inflammatory component, making those patients prime candidates. While precise percentages are not yet known, they believe it is a significant portion of the over 40,000 FSGS patients in the U.S.

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Chief Financial Officer Simon Harford attributed the reduced guidance to disciplined expense management, especially limiting new headcount by leveraging the existing Fabry commercial infrastructure for the Pombiliti launch. He confirmed that all key activities, such as registry studies and clinical trials, remain fully funded, and this financial discipline is a key factor in achieving non-GAAP profitability.

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President and CEO Bradley Campbell reported a 50% increase in global prescribers in Q2, indicating strong growth in both breadth (new prescribers) and depth (repeat prescribers). He explained the mix varies by market: concentrated markets like the U.K. have all key centers prescribing, while in distributed markets like the U.S. and Germany, they are seeing strong uptake from top centers and are now penetrating more community-based physician practices.

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Chief Medical Officer Walid Abi-Saab explained that after assessing various natural history databases, uniQure proposed using the ENROL-HD dataset, and the FDA agreed it was the most appropriate comparator. He confirmed the September data update will feature a comparison to a propensity score-matched control from ENROL-HD.

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CEO Dr. Laura Niklason and CCO BJ Scheessele confirmed the dialogue with VACs is consistent, focusing on clinical data and price. Mr. Scheessele noted the company's published Budget Impact Model effectively addresses cost concerns and that the ongoing NTAP application is also key. He stated that the minority (less than 50%) of the 45 hospitals were prior clinical trial sites, indicating strong interest from new centers.

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Executive Laura Niklason affirmed Humacyte's commitment to the PAD program, highlighting the significant clinical need for patients who lack vein and require below-the-knee revascularization. However, she stated that the company is currently evaluating its cash position and long-term spending priorities following a recent capital raise before committing to a specific timeline for initiating the Phase III trial.

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Executive Laura Niklason confirmed the trial had a co-primary endpoint at 6 and 12 months and a Special Protocol Assessment (SPA) with the FDA, increasing confidence in its design. She noted they will present the totality of data from both the V007 and older V006 trials to the FDA to support a filing. Regarding surgeon feedback, she observed that enrollment in the subsequent V012 women-only trial appears to be accelerating, suggesting increased investigator enthusiasm since the V007 results were publicized.

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President and CEO Douglas Ingram explained it was a specific administrative hiccup between sites in Los Angeles County and Medi-Cal regarding single-case agreements, which sites require for reimbursement assurance on high-cost therapies. This was not an access or prior authorization issue, but a delay in paperwork that pushed scheduled infusions from Q1 into Q2. Chief Customer Officer Dallan Murray added that such delays have a tenfold financial impact for a one-time gene therapy versus a chronic one.

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President and CEO Doug Ingram highlighted the significance of the pipeline, stating that Sarepta's current LGMD portfolio represents an opportunity equivalent to about 70% of the Duchenne market. He specified that the near-term sarcoglycan programs alone are comparable to over 25% of the Duchenne opportunity, agreeing it is a major area of focus and value.

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CEO Douglas Ingram clarified that the provided 2025 revenue guidance is for U.S. sales only and does not assume any impact from a European approval. He explained that the revenue curve to peak sales is a function of the entire process, including site capacity and payer interactions, and reiterated that while 2025 will be strong, peak sales will occur some years after that as they treat the large prevalent population over the decade.

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Executive Alexander Sapir attributed the strong enrollment to a combination of having the 'right' clinical sites, the unavailability of voxelotor (Oxbryta) prompting patients to seek alternatives, and overall excitement for HbF induction as a therapeutic strategy. Iain Fraser, Head of Development, added that overcoming the initial lag in site activation was key. Regarding translatability, Mr. Fraser noted that encouraging HbF responses were seen in less severe patients in the initial study, suggesting the benefit would extend to a broader population.

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Executive Iain Fraser stated it is encouraging that other companies are using the RSA endpoint, reinforcing that the FDA has not suggested an alternative. CEO Alexander Sapir added that the other data validates the DUX4 pathway and the RSA endpoint. Sapir highlighted the large safety database of over 3,600 patients, and Fraser noted that extensive open-label data from both the Phase II (over 3 years) and Phase III REACH studies will be included in the filing.

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President and CEO Adam Grossman stated that the onboarding of third-party supply centers is progressing rapidly, exceeding 50% completion and beating internal estimates, which could lead to upside in 2026 guidance. He confirmed the queue of prospective ASCENIV patients is growing, and the company is accelerating new patient starts as plasma supply increases. Grossman also noted that physicians who try ASCENIV with one or two patients often queue up several more, indicating significant growth potential within existing clinics.

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President and CEO Adam Grossman explained that strong demand, coupled with a VIP donor program and the ADMAlytics platform, is accelerating the identification and retention of high-titer plasma donors, which is the primary constraint on ASCENIV's growth. He noted the company has strategically shifted production from BIVIGAM to ASCENIV and is working with third-party collectors to expand supply. While declining to share specific metrics on the VIP program, he confirmed it is active in all 10 centers and is improving donor frequency and retention. CFO Brad Tade added that the company will pursue the $1 billion target as rapidly as possible, with further growth headroom expected thereafter.

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CEO Paula Ragan and Chief Medical Officer Christophe Arbet-Engels explained that physicians' confidence to lower G-CSF doses stemmed from the high and robust Absolute Neutrophil Count (ANC) responses observed after adding mavorixafor. They noted that this decision was at the discretion of physicians and patients, with 75% choosing to reduce the dose. They stated it was too early to provide specific anecdotes on patient-reported outcomes like pain reduction.

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Chief Commercial Officer Mark Baldry explained that physicians are primarily focused on XOLREMDI's impact on reducing infections, which is the key value driver. Chief Financial Officer Adam Mostafa confirmed that partnership discussions are ongoing and that interest is growing following the U.S. approval and positive Phase II data in chronic neutropenia.

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