Biren Amin • Piper Sandler Companies

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David Chang, Co-Founder, President, & CEO, affirmed that if MRD conversion is equated to complete remission, then a similar EFS benefit could be expected. Geoffrey Parker, EVP & CFO, stated that while the cash runway extends into 2027, its sufficiency for the EFS readout depends on trial enrollment pace, noting the 2027 timeframe is 'in the ballpark' for when the analyses could occur.

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Dr. Zachary Roberts, EVP of R&D and CMO, confirmed that sites are now generally well-staffed and operational, but deferred providing specific metrics on conversion rates and timing until more data is available. President and CEO Dr. David Chang added that the timeline from consent to randomization can vary as it depends on when a patient completes their 12-18 week frontline treatment before MRD testing can occur.

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President and CEO Dr. David Chang indicated that enrollment is "more or less tracking" with previous timelines, but the focus is on the unchanged data readout milestones. EVP of R&D and CMO Dr. Zachary Roberts added that while not exactly 50/50, they are seeing "robust activity" in patient enrollment from both community and academic centers without a significant skew.

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CEO Sean Nolan confirmed that the FDA has agreed the Part A and pivotal materials are analytically comparable. The pivotal trial product, manufactured using the planned commercial process at scale, has been cleared for clinical use. He also noted that the recent financing provides the flexibility to begin building commercial inventory to meet the large unmet need, should the program advance quickly.

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CEO Sean Nolan stated the transition will be seamless by leveraging existing, contracted sites, with the potential to add new ones to expedite enrollment. Regarding Europe, he noted it's a significant opportunity and that the regulatory team has been working in the background to enable the EMA pathway, which could be pursued more actively with additional capital.

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CEO Thomas Schuetz confirmed the Q1 projection is based on the current, slower mortality rate. He had no update on the IST. Regarding CTX-8371, he explained the responses were classified as partial (PR) instead of complete (CR) due to non-target lesions that did not disappear. He noted the TNBC patient had prior pembrolizumab and the NSCLC patient had low PD-L1 expression at original diagnosis, but patients were not re-biopsied for the study.

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CEO Neil Kumar stated that while the majority of Atruvi prescribing remains in Centers of Excellence (COEs) and affiliated practices, uptake in the community setting is growing. Justin To, CEO of QED Therapeutics, added that market research suggests an oral therapy like infigratinib could capture 60% of the achondroplasia market and expressed confidence in its potential for hypochondroplasia based on recent preclinical data.

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CCO Matt Outten stated the '3, 42, 50' data points (3-month separation, 42% risk reduction, 50% hospitalization reduction) are resonating strongly. CFO Tom Trimarchi explained that gross-to-net trended favorably this quarter due to lower-than-expected use of patient assistance programs but is expected to normalize. The floor for GTN is the 20% mandatory IRA rebate.

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Pravin Dugel, Executive Chairman, President & CEO, stated that enrollment for SOLAR is complete and the trial is proceeding well. He clarified the change in rescue criteria was a strategic decision made from a position of confidence based on masked data from the SOUL-one trial, not an FDA requirement. The goal is to enhance the trial's clinical relevance for physicians to support immediate adoption upon approval.

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President and CEO Dr. Pravin Dugel clarified that the week 76 redosing is to maximize drug exposure to satisfy FDA safety requirements. He confirmed all patients in SOL-1 will be redosed at weeks 52 and 76 with their originally randomized drug, regardless of prior rescue status. He affirmed the SOL-R size reduction was a direct result of fulfilling safety requirements across both studies. Regarding performance targets, Dr. Dugel stated that while FDA discussions are confidential, the company's confidence in the trials' success is 'higher than ever.'

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President and CEO Dr. Pravin Dugel explained that a differentiated label would address key clinical questions on durability, repeatability, and flexibility (6-to-9-month dosing). He added that if successful, the label could also include a unique superiority claim, providing both regulatory and commercial advantages.

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Dr. Pravin Dugel, President and CEO, stated that while the pace of SOL-1 enrollment is ahead of expectations, the official guidance for completion by the end of Q1 2025 has not been changed yet. He mentioned that conversations with the EMA are progressing well and updates will be provided when appropriate. Regarding SOL-R's retreatment criteria, Dr. Dugel explained they are still under discussion with the FDA but are expected to align with other non-inferiority studies.

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CFO Tim Sullivan explained that Q4 2024 is not a good comparable due to significant inventory stocking at physician offices, reiterating that injection growth is the key demand metric. For R&D, he advised that using the recent average is safer than the Q2 figure, as pivotal studies for DGF and FSGS have not yet fully ramped up.

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Executive Cedric Francois noted that physicians are using samples to ensure patient care continues despite co-pay challenges, indicating strong underlying demand. Executive David Acheson confirmed that samples are true demand and that Apellis is actively working to educate practices on benefit designs to help transition these patients to commercial doses. This effort has been underway since mid-Q1.

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COO Adam Townsend stated that while switching occurs, it is not frequent, and the company's focus is on bringing new prescribers into the market. For the nephrology launch, he noted they will leverage the existing PNH infrastructure and are currently determining the appropriate field force size. CEO Dr. Cedric Francois added that switches happen in both directions and the market is still settling.

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President & CEO Brian Lian confirmed the trial duration includes a titration window plus 52 weeks at the final dose, and that the trial listings would be posted 'very soon'. He also stated that manufacturing supply for a potential Phase III oral study would not be a gating factor for its initiation, as multiple batches are already in progress.

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Brian Lian, President and CEO, responded that the current oral formulation is 'pretty set,' though minor optimizations are always being explored. For the monthly regimen trial, he described it as a smaller, PK-focused study to evaluate exposures during the transition from weekly dosing. He estimated it would last slightly longer than three months to accommodate the necessary dose titration.

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CEO Brian Lian declined to disclose specific doses for the monthly study until initiation but mentioned a plan to use a weekly regimen for about four weeks before transitioning. For the amylin program, he confirmed that all IND-enabling work, particularly toxicology studies, must be completed to enable a filing later in the year.

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President and CEO Douglas Ingram clarified that aggregate capacity is good, but there's an imbalance, with top sites being fully booked for a year or more. The issue is making all sites more productive, which is a focus for the company. For risk mitigation, he highlighted the extensive monitoring in the label and the Sarepta Exchange program for real-time expert access, emphasizing that the greatest confidence will come from understanding the therapy's strong overall safety and efficacy data.

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Chief Scientific Officer Dr. Louise Rodino-Klapac confirmed they have seen similar expression levels in both ambulatory and non-ambulatory patients. She clarified that while the EMERGENE trial's primary endpoint is expression, the follow-on confirmatory study will analyze functional outcomes for the two populations separately and will use Sarepta's own JOURNEY natural history study as the comparator.

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President and CEO Doug Ingram explained that while they do not provide a granular breakdown, there is an expected bias towards ambulatory patients early in the launch. However, he stressed that a 'very significant percentage' of start forms are for late and non-ambulatory patients, and he is confident this segment will grow. Chief Customer Officer Dallan Murray added that this mix is not expected to fluctuate significantly quarter-to-quarter in the near term.

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CCO Dallan Murray corrected the premise, stating the number of sites has been very consistent at around 75. CEO Douglas Ingram added that this number already exceeds their original goals. Murray emphasized that the model is flexible, allowing them to rapidly bring on new sites, including adult neuromuscular centers, as needed to support patient demand, ensuring more than enough capacity.

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