Swayampakula Ramakanth • H.C. Wainwright & Co.

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CEO Sean Brynjelsen clarified that a new clinical study is not required for the Candivy label expansion. The process involves submitting a revised formulation with lower excipient concentrations. He stated the company plans to submit a prior approval supplement in Q1 2026, targeting an approval for the wider label by the end of 2026. Brynjelsen projected that with the expanded label, the combined adrenal insufficiency franchise could reach 1,500 to 2,000 patients.

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CEO Sean Brynjelsen explained that the Galzin relaunch is progressing better than planned, with a steady stream of patients converting due to improved access, a $0 co-pay, and strong relationships with patient advocacy groups. He deferred providing specific patient numbers until the transition is more complete. CFO James Gruber addressed the financials, stating the increase in receivables was driven by Increlex sales and the pending $4.3 million payment from the Esteve licensing deal, while the higher Medicaid liability was almost entirely due to the Increlex relaunch.

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Co-Founder & CEO Chris Peetz stated European performance has been driven by Alagille syndrome, with future growth expected from securing PFIC reimbursement. COO & President Peter Radovich added that the EXPAND study includes various ultra-rare populations, with biliary atresia being a key component, representing a significant opportunity.

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CEO Gerard Michel noted the team has learned lessons, such as addressing perfusion needs earlier, but hesitated to promise a faster process due to each institution's uniqueness. He does not expect a significant jump in volume from 340B but believes it removes a financial barrier. He confirmed that opening trial sites in Europe will help ChemoSat sales long-term by establishing trained teams in new countries.

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CEO Gerard Michel confirmed that repeat treatment frequency is tracking towards the clinical trial average of around four treatments per patient. He attributed new center growth to positive word-of-mouth among physicians rather than competitive pressure. Regarding Europe, he stated that while revenue is growing, the focus remains on generating clinical data due to significant pricing and reimbursement challenges. He also noted that while European sites use CHEMOSAT for other indications, the data is not yet substantial enough to drive new sponsored trials.

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CEO Gerard Michel confirmed a "fear of missing out" effect is helping drive interest from new centers. CFO Sandra Pennell projected gross margins could reach 90% by the end of 2025 due to operating leverage. Michel outlined an R&D strategy focused first on new cancer indications (CRC, breast), followed by combination therapies and new agents. He reiterated that the ex-U.S. strategy prioritizes strategic data generation over near-term revenue.

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CEO Gerard Michel confirmed that treatment intervals are generally 6-8 weeks, consistent with the trial, and shared anecdotal feedback from physicians who believe they are seeing even better response rates than in the FOCUS study. Regarding the European trials, Michel stated that a formal label expansion is not necessary, as physicians can already use the therapies in sequence. He noted the data would be important for informing clinical practice and potentially supporting future trials for patients failing immunotherapy.

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CEO Christopher Hall reiterated that the pharma business weakness is confined to the translational research segment due to macro headwinds, which he believes is temporary. He stressed that the strategic MRD pharma business is performing exceptionally well and is on track. CFO & COO Aaron Tachibana addressed the reimbursement question by stating that the key catalyst is receiving a favorable coverage decision from Medicare. He noted that the company's clinical revenue guidance of $3-6 million for the year already incorporates variables related to the timing and mechanics of revenue recognition post-coverage.

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Executive Christopher Hall expressed high confidence in the reimbursement timeline, clarifying that they submit to Palmetto, a private Medicare contractor, which insulates the process from direct federal government shifts. For the CRC indication, he outlined the required steps: maturing the current interim data, achieving publication in a peer-reviewed journal, and then submitting the complete data package to Medicare for reimbursement consideration, noting it is a long-term strategic priority.

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CEO Christopher Hall explained that the internal commercial team will remain small and the financial impact on SG&A will not be meaningful in the near term. Regarding reimbursement, he stated the typical timeline from submission to decision is about six months, plus or minus, and that while they expect at least two approvals in 2025, their internal goal is to submit and gain approval for all three key indications (breast, lung, IO therapy monitoring).

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President & CEO Jeff Hawkins clarified that the new models are primarily improving the conversion rate of existing leads who are sold on the technology but lack capital, rather than generating new leads. He stated the goal is not to abandon the academic market but to enhance product capabilities, like the V3 library prep kit and PTM analysis, to serve all segments based on direct customer feedback and current market opportunities.

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President and CEO Jeffrey Hawkins explained that new sequencing kits are released when combined improvements offer a meaningful performance lift, such as increased amino acid detection. He affirmed the company's strategy is to maintain its technology leadership through focused R&D investment while being prudent with capital, noting they are capitalized into H2 2027. Hawkins also highlighted the value of peer-to-peer marketing, including customer webinars and user group meetings, as a powerful complement to direct sales.

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Chief Medical Officer Dr. Huma Qamar stated that enrollment for the Limelight trial is on track for a 2026 BLA filing, but as a blinded study, efficacy data will only be released after the last patient's final visit. She noted another safety update is planned for Q4. CEO Shankar Musunuri addressed funding, explaining that Ocugen is actively pursuing non-dilutive funding through strategic partnerships to minimize shareholder dilution, while remaining opportunistic about equity financing as needed. He clarified that OrthoCelix will seek its own funding independently post-merger.

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Chief Medical Officer Dr. Huma Qamar confirmed the liMeliGhT study is blinded with no interim analysis, with full data available upon completion. CEO Dr. Shankar Musunuri clarified the 2:1 randomization is for OCU400, while a 1:1:1 ratio applies to the OCU410 dry AMD trial. Dr. Musunuri outlined 2025 data updates, including interim results for OCU410 for dry AMD in the fall, 12-month Phase I data for OCU410ST, and Phase I data for OCU200 late in the year.

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CEO Dr. Shankar Musunuri cited the company's track record and recent FDA alignment on OCU410ST as reasons for confidence in the BLA timelines. CMO Dr. Huma Qamar highlighted OCU410's superior safety profile and strong patient interest as key differentiators from approved GA therapies. Both executives confirmed that ongoing data, including 3-year durability data for OCU400, will be available before its BLA filing.

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CEO Joseph Belanoff acknowledged the rapid demand from CATALYST outpaced their pharmacy's capacity. Sean Maduck, President of Corcept Endocrinology, explained the guidance cut reflects the cumulative impact of patient start delays. Chief Development Officer William Guyer noted the BELLA trial will have results in late 2026 and that oncology plans include other gynecological cancers. Chief Business Officer Charles Robb stated the FDA meeting for dazicorilant in ALS is scheduled for August 2025.

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Chief Development Officer William Guyer responded that the company expects to meet the PFS endpoint, but if it were missed, the OS data would provide a second chance for a positive study approximately one year later. Sean Maduck, President of Endocrinology, explained that while a small impact from CATALYST is being seen, a more significant effect on medical practice is anticipated in the second half of the year and beyond as the data is more broadly published and communicated.

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CEO Joseph K. Belanoff and Chief Development Officer William Guyer explained that while they are on track to have enough progression events by year-end, the exact timing for data analysis and release is uncertain, which is typical for event-driven oncology trials. Guyer noted that a longer timeline could indicate better patient outcomes. They committed to releasing the results publicly as soon as the analysis is complete.

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CFO Joe Mara cited stronger-than-average conversion of Epicel biopsies and continued NexoBrid momentum as drivers for outperformance. CEO Dominick Colangelo added that any potential Q4 BARDA revenue is pure upside to the current guide but declined to quantify it. He also clarified that surgeons often take biopsies before training, so there is no adoption lag time post-training.

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CEO Dominick Colangelo stated that P&T committee approval is not a factor for MACI Arthro, though some hospitals review the new instruments. He also highlighted the fully staffed burn care sales force as a key growth driver. CFO Joe Mara added that while the new facility's costs will be absorbed, they do not expect a material negative impact on the gross margin trajectory toward the high 70s target.

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CFO Joe Mara explained that MACI Arthro has significant built-in synergies by leveraging the existing commercial infrastructure without materially changing the P&L profile per case. He noted that while there is minor incremental revenue from instrument sales, the primary benefit is driving top-line growth, which in turn provides operating leverage for the business rather than changing the margin on a per-procedure basis.

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Ofer Gonen (executive) explained that treating patients is the current priority over stockpiling, with any related revenue already factored into existing guidance. He detailed that approximately 17-20 of the 40 EscharEx trial sites are in the U.S. and that the enrollment pace is deliberately managed to ensure data quality, making an early finish unlikely. Gonen also clarified the Phase II head-to-head study is simpler and expected to produce results before the Phase III trial concludes.

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Executive Barry Wolfenson referenced Phase II data showing EscharEx's superiority over SANTYL in achieving complete debridement and wound closure more quickly. CEO Ofer Gonen stated that they are deliberately not accelerating VLU trial enrollment to ensure the recruitment of high-quality patients with hard-to-heal wounds. He also clarified the BARDA collaboration is a long-term project for a backup U.S. facility, with a timeline extending to 2027-2028, and does not impact near-term guidance.

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Ofer Gonen (Executive) responded that the CPT code is not expected to have a significant short-term impact on market penetration. For the DFU study, he outlined that the company must first develop a protocol, gain alignment from KOLs, and secure FDA/EMA clearance, a process expected to take about a year. For the temperature-stable NexoBrid, Gonen detailed that the path to a 2026 trial start involves building a dedicated manufacturing facility and completing nonclinical and CMC data requests from the FDA.

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CEO Paula Ragan confirmed the 50,000 diagnosed and 15,000 high-unmet-need patient figures are for the U.S. only. Executive Christophe Arbet-Engels explained that despite the trial's focus on severe patients, the company will seek a broad label for the entire CN population. CFO Adam Mostafa clarified that the Norgine agreement is a license covering both the WHIM and CN indications.

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Chief Commercial Officer Mark Baldry stated that the company is currently 'laser-focused' on raising disease awareness and driving screening for WHIM syndrome, as no approved treatment existed before XOLREMDI. He explained this foundational work is crucial for building momentum into 2025, at which point more details on the commercial ramp-up would be shared.

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President and CEO Dr. Paula Ragan and Chief Commercial Officer Mark Baldry highlighted the excitement among physicians for an oral, targeted therapy backed by strong Phase III data showing infection reduction. Dr. Ragan confirmed that the next data release for the chronic neutropenia program will be the full Phase II dataset in November, with no other interim updates planned before then.

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CFO Michael Fortunato explained the accounting issue is a matter of classification on the income statement—whether payments to Venture are a sales expense or a reduction of revenue—and would not materially affect net income or EBITDA. He could not provide a specific timeline for the SEC resolution. CEO Jason Matuszewski clarified that the FDA warning letter pertained to four products that were already discontinued and does not impact the company's current product portfolio. Both executives declined to provide specific 2025 guidance but expressed confidence in continued progress.

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Chief Science Officer Birgitte Rono detailed the planned EVX-01 biomarker analysis, which includes T cell profiling and soluble analytes. Both Rono and CEO Christian Kanstrup indicated it was too early to announce other 2025 data milestones. Kanstrup acknowledged the potential for another BD deal in 2024 but cautioned that timing is uncertain.

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Dr. Helen Sabzevari, President and CEO, stated that no further data from the pivotal trials is planned for release as the complete dataset was presented at ASCO and met FDA requirements, though durability follow-up is ongoing. She confirmed active engagement with ex-U.S. authorities and readiness for a global launch. Phil Tennant, Chief Commercial Officer, described the planned commercial team as 'small but perfectly formed,' likely in the 'tens' of representatives, integrated with medical affairs for the rare disease launch.

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