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CEO Christopher Anzalone emphasized that the company is in the 'truth-seeking business' and does not pre-plan next steps before seeing the data. Interim Chief Medical Scientist Bruce Given added that because these are novel mechanisms, unlike another GLP-1 agonist, it is difficult to predict the development path without initial human data.

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An executive stated the FCS prevalence is estimated at 1 to 13 per million, translating to a few hundred to mid-single-digit thousands of patients in the U.S. He confirmed that internal analytics and marketing teams are using advanced technologies for patient-finding, which is critical for an ultra-rare condition.

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Chief Medical Officer Joanne Quan confirmed that the company is "good to go" with the trial, having received a "study may proceed" letter from the FDA after clearing the IND. She added that feedback from patient and physician communities has been incorporated, keeping the trial on track to enroll its first patient by year-end.

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President and COO Peter Radovich stated that literature estimates suggest 1,000 to 2,000 prevalent CTX patients in the U.S., with only about 10% currently diagnosed. He explained that an on-label approval would allow for active promotion aimed at increasing the diagnosis rate, which could have a meaningful impact on the product's current sales trajectory.

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David Meeker, Chairman, President & CEO, stated that patients will be evaluated at six months but will continue on treatment indefinitely if they are benefiting, as long-term data is valuable. He confirmed that while next-generation molecules are the future, a compelling result could lead to rapidly advancing setmelanotide for Prader-Willi, with next-gen compounds certainly following.

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David Meeker, CEO, responded that it is too early to provide a meaningful breakdown of the patient mix across different pituitary deficiency disorders. He mentioned that conditions like septo-optic dysplasia (SOD) and pituitary stalk interruption syndrome are among those being seen, but the study is in its very early stages.

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CCO Tolga Tanguler confirmed that they have seen a 'very small portion' of patients receiving combination therapy where access is permissible. He suggested this trend could become more prominent in the future as tafamidis becomes generic but noted it is currently difficult to quantify precisely.

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Chief Commercial Officer Tolga Tanguler stated that there is empirical evidence that more market participants accelerate category growth, pointing to the polyneuropathy market and the recent record growth of the stabilizer class. While he declined to predict a specific diagnosis rate for year-end, he expressed confidence that more voices in the space will help accelerate diagnosis.

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CEO Brett Monia explained that refining the timeline to September was simply due to operational progress in completing the studies and cleaning the data. He confirmed the top-line release will include a statement on AP findings, with the full dataset to be presented at a medical congress later in the year.

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CEO Brett Monia explained the more precise Q3 timing reflects increased clarity as the readout approaches. He confirmed the top-line data release for the CORE and CORE2 studies will focus on the primary endpoint of triglyceride reduction and safety. He stated that the company is still determining if the AP data analysis will be complete in time for the initial announcement but will share it if available.

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CEO Brett Monia explained that the ESSENCE study, while conducted in a lower-risk population, will provide important read-throughs on safety and target engagement (APOCIII reductions). He noted that while triglyceride reduction data will be an indicator, it is a less direct predictor for the sHTG population, with the primary focus being on validating the drug's safety profile.

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President & CEO Brian Lian described the maintenance study as complex and sizable, likely with more arms than the VENTURE oral study, as it will test monthly injections, daily orals, and a weekly oral. He indicated the post-transition evaluation period would be around three months but would provide more detail upon study initiation.

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Brian Lian, President and CEO, stated that it is difficult to project specific outcomes but suggested that achieving around 8% weight loss after 13 weeks would establish a competitive profile. He noted it was hard to predict the performance of lower doses but remained hopeful they would show good efficacy over the extended treatment period.

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CEO Brian Lian attributed the more precise timing to improved visibility on the logistical timeline for manufacturing the clinical drug product. He reassured that the program is proceeding according to plan without any issues.

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CEO Michael Rossi attributed the guidance to product seasonality and a particularly strong Q1 for ex-U.S. sales. CFO Peter Pfreundschuh elaborated that Q2 2024 included nearly $5 million in ex-U.S. stocking orders not expected in Q2 2025. He stressed the guidance is realistic and reiterated confidence in the full-year forecast of $75M-$90M, anticipating a U.S. rebound in the second half of the year.

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CEO Michael Rossi explained that while SADA R&D costs will increase with new targets, this is factored into the cash runway. He mentioned cost savings from the completion of the DANYELZA 201 study and a new, more efficient process for advancing SADA targets. CFO Peter Pfreundschuh clarified the OpEx guidance, suggesting analysts start with the 2024 SG&A figure of ~$55M, subtract ~$9M in one-time charges, and assume the rest of the guided OpEx is for R&D.

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Chief Medical Officer Dr. Vignesh Rajah confirmed that Cohort 5 is testing a dose of 1 milligram per kilogram with a 4-day interval. He explained that the decision to proceed to a Cohort 6 will depend entirely on the dosimetry results from the current cohort, and no immediate decision has been made.

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President and CEO Douglas Ingram responded that while it's too early to identify a precise trend, the company continues to see start forms coming in for both ambulatory and non-ambulatory patients. He reiterated that there is no data suggesting a differential safety risk based on ambulatory status, so there should be no reason for a significant difference in uptake between the groups once education is complete.

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CFO Ian Estepan stated the program has an 18-month timeframe. He explained that after funding the Arrowhead transaction, the company projects its cash reserves will return to year-end 2024 levels by the end of 2025 due to the strong ELEVIDYS launch. The company will look to be opportunistic in deploying capital for buybacks as cash reserves build, but will not telegraph the specific timing.

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CEO Douglas Ingram acknowledged that while the company has always modeled eventual robust cannibalization, what they have observed so far and are modeling for 2025 is very modest. He suggested that cannibalization might become more significant in the years after 2025, but also noted that their internal models may have been too aggressive on this front.

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Craig Tooman, President and CEO, stated that while he could not comment on specific ongoing discussions, the company remains actively engaged. He noted that the HORIZON data delay has spurred more funding interest in the Lp(a) space. Tooman emphasized that Silence is a portfolio company and the decision to wait for a partner before starting the Phase III trial allows for prudent resource management and focus on maximizing returns across all assets, including divesiran.

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Dr. Mark Eisner, Chief Medical Officer, described the meeting with the FDA as "very productive" and stated that Vir aligned closely with the agency on the clinical development plan. He confirmed that the final details will be revealed at the upcoming investor event.

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