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    Paul Matteis • Stifel, Nicolaus & Company, Incorporated

    Paul Matteis's questions to Rhythm Pharmaceuticals Inc (RYTM) leadership

    Paul Matteis's questions to Rhythm Pharmaceuticals Inc (RYTM) leadership • Q2 2025

    Question

    Paul Matteis of Stifel Financial Corp. asked about the RM-718 study, focusing on the expected patient mix and key factors for comparing its efficacy to other drugs. He also questioned whether the MC4R agonist mechanism's efficacy has been maximized.

    Answer

    David Meeker, Chairman, President & CEO, stated that the RM-718 trial will enroll patients aged 12 and older, and the key question is identifying the right dose for the weekly formulation, not its efficacy as an MC4 agonist. He affirmed his belief that the mechanism's efficacy has likely been maxed out, with dose adjustments potentially mattering only on the margins for patients with very different body weights.

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    Paul Matteis's questions to Rhythm Pharmaceuticals Inc (RYTM) leadership • Q1 2025

    Question

    Paul Matteis from Stifel asked about any safety signals, particularly idiosyncratic ones like liver issues, from the ongoing bivamelagon study and sought clarification on the 10% efficacy bar for the shorter Phase II trial.

    Answer

    David Meeker, CEO, confirmed that based on a blinded view, there are no significant safety signals of concern in the bivamelagon study, including no serious adverse events related to liver function tests. Regarding the efficacy bar, he explained that while 10% is a reasonable target based on setmelanotide's Phase II data, the full context of the data will be important, and he is more interested in individual patient responses than the mean value alone.

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    Paul Matteis's questions to Alnylam Pharmaceuticals Inc (ALNY) leadership

    Paul Matteis's questions to Alnylam Pharmaceuticals Inc (ALNY) leadership • Q2 2025

    Question

    Paul Matteis from Stifel Financial Corp. asked about the clinical criteria physicians are using to identify 'stabilizer progressors' and the potential percentage of stabilizer patients who could be candidates for Amvuttra.

    Answer

    CCO Tolga Tanguler cited research suggesting a third to half of patients on stabilizers eventually progress. CMO Pushkal Garg explained that physicians assess a constellation of factors, much like in general heart failure management—including biomarkers, echo data, and symptoms—rather than a single indicator, and expects formal guidelines to evolve over time.

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    Paul Matteis's questions to Alnylam Pharmaceuticals Inc (ALNY) leadership • Q1 2025

    Question

    Paul Matteis asked about the percentage of prescribers who can administer AMVUTTRA without a P&T committee review and the uptake from that group. He also questioned if Medicare fee-for-service patients could eventually receive their first dose on the same day of prescription.

    Answer

    Chief Commercial Officer Tolga Tanguler explained that same-day administration is not feasible due to the coordination required for a buy-and-bill, HCP-administered product. He did not provide a specific metric on P&T committees but emphasized that the process for getting patients on therapy is effective across all payer types, thanks to the drug's profile for a severe disease and the company's value-based agreements.

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    Paul Matteis's questions to Alnylam Pharmaceuticals Inc (ALNY) leadership • Q4 2024

    Question

    Paul Matteis asked if Alnylam expects insurers to cross-manage formularies between Part B and Part D drugs, and whether Part D stabilizers could be used as a step-edit for Part B AMVUTTRA.

    Answer

    Chief Commercial Officer Tolga Tanguler responded that despite noise, Alnylam expects payers to cover the product for this rare, devastating condition. He anticipates payer mix dynamics will be similar to what was seen with polyneuropathy and that AMVUTTRA's favorable patient affordability profile will be a key driver of uptake.

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    Paul Matteis's questions to Alnylam Pharmaceuticals Inc (ALNY) leadership • Q3 2024

    Question

    Paul Matteis asked for clarification on current prescribing dynamics, specifically the prior authorization hurdles a cardiologist might face when prescribing AMVUTTRA for a mixed-phenotype patient.

    Answer

    Chief Commercial Officer Tolga Tanguler explained that the primary difference lies in the diagnostic pathway, not payer access. Polyneuropathy requires genetic testing and a neurological workup, whereas cardiomyopathy often relies on scintigraphy. He emphasized that most patients ultimately secure reimbursement, making the diagnostic process the main distinguishing factor.

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    Paul Matteis's questions to Biogen Inc (BIIB) leadership

    Paul Matteis's questions to Biogen Inc (BIIB) leadership • Q2 2025

    Question

    Julian, on behalf of Paul Matteis at Stifel, asked for logistical details on how subcutaneous Leqembi initiation would work in practice, including the administration setting and its value proposition given ARIA concerns.

    Answer

    Priya Singhal, EVP & Head of Development, stated that trials were designed to allow for self-administration and that human factors studies were positive. Alisha Alaimo, President & Head of North America, explained the approach will be flexible and tailored to the patient and physician. Experienced prescribers may be comfortable with at-home initiation, while others may prefer in-office settings, with Biogen exploring home support options.

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    Paul Matteis's questions to Biogen Inc (BIIB) leadership • Q2 2025

    Question

    Julian, on behalf of Paul Matteis from Stifel, asked about the practical logistics of subcutaneous Leqembi initiation, specifically how it would be administered (in-center vs. at-home) given concerns over ARIA.

    Answer

    Priya Singhal, EVP & Head of Development, explained that trial data supports flexible administration options, including self-administration. Alisha Alaimo, President & Head of North America, elaborated that the choice will be tailored to the patient and physician's comfort level. Experienced prescribers may be comfortable with at-home initiation, while others may prefer an in-office start, ensuring flexibility based on individual patient needs.

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    Paul Matteis's questions to Biogen Inc (BIIB) leadership • Q4 2024

    Question

    Paul Matteis asked whether Biogen can achieve sustainable revenue growth with its current portfolio or if it requires a major pipeline success or external business development.

    Answer

    President and CEO Christopher Viehbacher stated that while the declining MS franchise is a headwind, the combination of growth from new launches, led by LEQEMBI, and the maturing pipeline can return the company to growth. He noted that for Biogen, a '$1 billion product really moves the needle,' suggesting that a combination of internal execution and potential BD will drive the future.

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    Paul Matteis's questions to Biogen Inc (BIIB) leadership • Q3 2024

    Question

    Paul Matteis asked whether Biogen is strategically shifting away from high-risk neuroscience towards immunology and if its business development focus reflects this change.

    Answer

    CEO Christopher Viehbacher clarified that Biogen remains heavily invested in neuroscience (Alzheimer's, Parkinson's) but is expanding into adjacent areas to diversify. He views immunology as a natural extension of their MS expertise and a key R&D growth area. The strategy is to leverage existing capabilities, like their rare disease commercial model, rather than making a wholesale shift away from neuroscience.

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    Paul Matteis's questions to Compass Pathways PLC (CMPS) leadership

    Paul Matteis's questions to Compass Pathways PLC (CMPS) leadership • Q2 2025

    Question

    Paul Matteis of Stifel Financial Corp. inquired about Compass's engagement with the FDA post-data, potential scenarios for an accelerated filing path for COMP360, and whether they would engage with senior FDA or HHS officials.

    Answer

    CEO Kabir Nath confirmed a Q3 meeting is requested with the FDA to discuss accelerated pathways, citing two positive late-stage studies. While not predicting the outcome, he noted supportive signals from the administration and confirmed engagement with senior officials. CCO Lori Englebert added that they are educating receptive members of Congress. Nath also revealed they have applied for the commissioner's national priority review voucher.

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    Paul Matteis's questions to Compass Pathways PLC (CMPS) leadership • Q1 2025

    Question

    Paul Matteis of Stifel inquired about the company's base case for the placebo effect in the Phase III trial and how the Data and Safety Monitoring Board (DSMB) would differentiate a true suicidality signal from statistical noise.

    Answer

    Dr. Guy Goodwin, Chief Medical Officer, responded that the placebo effect estimate is based on previous studies and their own Phase IIb data, noting that a MADRS score difference over 3 would be clinically significant. Regarding suicidality, he clarified that the DSMB's assessment is a nuanced medical judgment based on the severity, timing, and evolution of each event, not just on raw numbers.

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    Paul Matteis's questions to BioMarin Pharmaceutical Inc (BMRN) leadership

    Paul Matteis's questions to BioMarin Pharmaceutical Inc (BMRN) leadership • Q2 2025

    Question

    Paul Matteis of Stifel asked for confirmation that BMN-333's competing program is TransCon CNP and inquired about the drug's safety profile, particularly the risk of hypotension with increased exposure.

    Answer

    Gregory Friberg, EVP and Chief R&D Officer, confirmed the competing agent was the one referenced in the cited pharmacology journal. He stated that BMN-333's safety profile in the healthy volunteer study showed nothing unexpected and highlighted that genetic data from individuals with naturally high CNP levels is reassuring, as they primarily exhibit increased height without other organ system issues.

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    Paul Matteis's questions to BioMarin Pharmaceutical Inc (BMRN) leadership • Q1 2025

    Question

    Paul Matteis from Stifel asked for clarification on two points: whether VOXZOGO's global net price has changed recently and if the planned pivotal trial for BMN 333 is designed to show superiority or non-inferiority to VOXZOGO.

    Answer

    Brian Mueller, CFO, confirmed that recent VOXZOGO revenue trends are due to order timing, not pricing changes. Greg Friberg, Chief R&D Officer, provided a direct answer on the pipeline question, stating, 'Yes, we are looking for superiority' for BMN 333 in its comparative trial against VOXZOGO.

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    Paul Matteis's questions to BioMarin Pharmaceutical Inc (BMRN) leadership • Q3 2024

    Question

    Paul Matteis asked if the reaffirmation of long-term guidance, despite increased competition in achondroplasia, implies the original guidance was conservative. He also asked what aspects of the long-term vision the Street might be missing.

    Answer

    EVP, Chief Financial Officer Brian Mueller stated the guidance was not conservative but that the company's model could absorb the new competitive data by adjusting assumptions. President and CEO Alexander Hardy added that the long-term outlook, including the mid-teen CAGR and $5 billion skeletal conditions opportunity, is based on modest penetration in new indications and excludes the potential impact of IP defense.

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    Paul Matteis's questions to Neurocrine Biosciences Inc (NBIX) leadership

    Paul Matteis's questions to Neurocrine Biosciences Inc (NBIX) leadership • Q2 2025

    Question

    Paul Matteis from Stifel Financial Corp. asked about the concentration of Crinesity prescribing, questioning whether it's mainly from academic centers of excellence or more broadly distributed in the community.

    Answer

    CEO Kyle Gano and CCO Eric Benevich explained that the CAH patient population is not highly concentrated. While Centers of Excellence are important, many patients are treated by pediatric and community endocrinologists who may only have one or two CAH patients. They noted most prescribers to date have initiated one or two patients, and the new patient additions have been very steady week-over-week, indicating broad, consistent adoption rather than a concentrated bolus.

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    Paul Matteis's questions to Neurocrine Biosciences Inc (NBIX) leadership • Q1 2025

    Question

    Paul Matteis inquired about INGREZZA's growth outlook for the second quarter and beyond, and asked whether the strong initial number of CRENESSITY prescriptions represented a one-time bolus or a sustainable trend.

    Answer

    CEO Kyle Gano confirmed that while Q1 had challenges, a record number of new patient starts for INGREZZA provides strong momentum for growth acceleration in Q2 and the rest of 2025. Chief Commercial Officer Eric Benevich added that for CRENESSITY, it is too early to define a trend after one quarter, but the initial adoption exceeded the company's expectations.

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    Paul Matteis's questions to Neurocrine Biosciences Inc (NBIX) leadership • Q4 2024

    Question

    Paul Matteis questioned the INGREZZA sales guidance, highlighting the significant deceleration from 26% growth in 2024 to a guided sub-10% sequential growth, asking if this signals a fundamental change in the market.

    Answer

    Chief Commercial Officer Eric Benevich reiterated the record growth of INGREZZA in 2024 and stated the 2025 guidance reflects continued VMAT2 category growth while accounting for an increasingly complex payer environment and competitive dynamics. CEO Kyle Gano added that the Q4 sales force expansion caused some disruption but is expected to drive growth in the second half of 2025 and beyond, emphasizing the product's differentiated profile and the large untreated patient population.

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    Paul Matteis's questions to Neurocrine Biosciences Inc (NBIX) leadership • Q3 2024

    Question

    Paul Matteis from Stifel asked about Neurocrine's business development philosophy, specifically its capacity and strategic appetite for larger, transformative deals versus its historical strategy of smaller acquisitions.

    Answer

    CEO Kyle Gano responded that the company feels no immediate pressure to pursue deals of any size due to its deep and diversified internal pipeline, with a current focus on executing late-stage programs. CFO Matt Abernethy added that Neurocrine maintains strong financial flexibility, with significant cash and a clean balance sheet, providing capacity for future opportunities.

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    Paul Matteis's questions to Uniqure NV (QURE) leadership

    Paul Matteis's questions to Uniqure NV (QURE) leadership • Q2 2025

    Question

    Paul Matteis asked about the rationale for using a propensity score-matched analysis instead of a weighted one, whether this was an FDA request, and how it might impact data comparability. He also asked about the company's view on the efficacy bar for disease slowing.

    Answer

    Chief Medical Officer Walid Abi-Saab explained the shift to propensity score matching aligns with the FDA's stated preference. He reassured that internal modeling shows both methods yield very similar results, so the data should not be materially different. He also noted that key opinion leaders view a 25-30% reduction in disease progression as highly meaningful for patients.

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    Paul Matteis's questions to Uniqure NV (QURE) leadership • Q1 2025

    Question

    Paul Matteis questioned the company's approach in its recent statistical analysis plan (SAP) meeting with the FDA, the rationale for using 3-year versus 2-year natural history data, and whether waiting for meeting minutes is standard procedure or indicates a need for clarification.

    Answer

    CEO Matthew Kapusta clarified that waiting for formal meeting minutes is simply 'good housekeeping' and standard best practice. CMO Dr. Walid Abi-Saab explained that the 3-year data is the most relevant dataset as the majority of patients will have reached that timepoint, making it crucial to demonstrate that the promising effects seen at 2 years are sustained.

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    Paul Matteis's questions to Alkermes Plc (ALKS) leadership

    Paul Matteis's questions to Alkermes Plc (ALKS) leadership • Q2 2025

    Question

    Paul Matteis of Stifel Financial Corp. asked about the focus on visual adverse events (AEs) with the Orexin program, questioning if the attention is warranted and how to differentiate between a benign versus a clinically significant signal for regulatory and commercial purposes.

    Answer

    Richard Pops, Chairman & CEO, stated the focus on visual AEs is primarily from Wall Street, not clinicians, and highlighted that the VIBRANCE-one study's rigorous ophthalmic exams showed no changes. Marcus Yountz, VP of Clinical Development, added that mild AEs not interfering with daily activity are not overly concerning. Craig Hopkinson, EVP and Chief Medical Officer, noted the Data Safety Monitoring Board's approval to proceed. Pops also clarified the full safety database is not yet closed and will be presented at World Sleep.

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    Paul Matteis's questions to Alkermes Plc (ALKS) leadership • Q1 2025

    Question

    James Condulis, on behalf of Paul Matteis, asked for an update on the safety profile of the orexin program on a blinded basis, with a specific focus on the instances of visual disturbances observed in prior data.

    Answer

    CEO Richard Pops stated it was inappropriate to comment on blinded data but confirmed that an independent Data Safety Monitoring Board (DSMB) is actively overseeing safety. He noted the DSMB has met regularly and has consistently recommended the study continue without any modifications.

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    Paul Matteis's questions to Alkermes Plc (ALKS) leadership • Q4 2024

    Question

    Paul Matteis inquired about the orexin program, ALKS 2680, asking how Alkermes is monitoring adverse events and retention in the blinded NT2 study and about the company's confidence in the drug's therapeutic index.

    Answer

    CEO Richard Pops explained that while the studies are blinded, safety is continuously monitored by an internal team and an independent Data Safety Monitoring Board (DSMB). He stated that the company expects high retention and has seen no data to suggest otherwise, expressing confidence in the therapeutic index based on a clear dose response observed in the Phase Ib program.

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    Paul Matteis's questions to Alkermes Plc (ALKS) leadership • Q3 2024

    Question

    Paul Matteis followed up on the 2025 EBITDA guidance, asking for the biggest mismatch versus consensus beyond royalty revenues, and inquired about the biggest perceived risk in translating early ALKS 2680 data to Phase 2 success in NT2 and IH.

    Answer

    COO Blair Jackson reiterated that the primary mismatch in Street models is the misunderstanding of the manufacturing and royalty revenue transition, along with increased R&D spend for ALKS 2680. CEO Richard Pops identified the biggest risk for ALKS 2680's Phase 2 studies as the inherent variability of the NT2 and IH patient populations, though he noted the drug has already shown a meaningful, dose-dependent effect on the MWT endpoint.

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    Paul Matteis's questions to Xenon Pharmaceuticals Inc (XENE) leadership

    Paul Matteis's questions to Xenon Pharmaceuticals Inc (XENE) leadership • Q1 2025

    Question

    Paul Matteis asked about the potential timeline from receiving top-line epilepsy data for azetukalner to filing an NDA, and requested color on the key metrics being tracked in the X-TOLE2 study.

    Answer

    President and CEO Ian Mortimer stated that the timeline from top-line data to an NDA filing is approximately six months, with the clinical data being the critical path item. He explained that key metrics tracked for study quality include patient baseline characteristics, diary compliance, and rollover rates into the open-label extension, all of which are showing consistency with the successful Phase IIb X-TOLE study.

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    Paul Matteis's questions to Xenon Pharmaceuticals Inc (XENE) leadership • Q4 2024

    Question

    Paul Matteis asked about the enrollment status and timing for the first Phase III epilepsy data readout and whether potential negative results from a competitor's Kv7 trial in bipolar mania would affect Xenon's plans for bipolar depression.

    Answer

    President and CEO Ian Mortimer confirmed the guidance for Phase III epilepsy data remains in the second half of 2025 and will be narrowed closer to the date. He stated that competitor results in bipolar mania will not impact their plans, as Xenon believes the scientific rationale for Azetukalner is stronger in bipolar depression. Chief Medical Officer Dr. Chris Kenney added that genetic links between Kv7 and bipolar disorder appear even stronger than for MDD, and prior data for other Kv7 drugs in mania was unconvincing, making depression a more compelling target.

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    Paul Matteis's questions to ACADIA Pharmaceuticals Inc (ACAD) leadership

    Paul Matteis's questions to ACADIA Pharmaceuticals Inc (ACAD) leadership • Q1 2025

    Question

    Julian, on behalf of Paul Matteis from Stifel, asked about the primary risks associated with the European approval and reimbursement for trofinetide, and inquired about the role of business development in the company's current priorities.

    Answer

    CEO Catherine Owen Adams expressed high confidence in the EMA approval process, stating it is proceeding as expected. Regarding business development, she confirmed that Acadia is 'very interested in transacting' for assets in high-unmet-need areas, including rare diseases beyond neurology. CFO Mark Schneyer added that the company is well-positioned to act on strategic deals that offer good financial value.

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