Salim Syed

Salim Syed asked about the flattish Q3 over Q2 performance of AUCATZYL sales, inquiring if seasonality or the CMS reimbursement policy change was a factor, and how this might impact Q4 sales and 2026 trajectory. He also asked CFO Rob Dolski about the handling of DNA costs in the cost of goods sold (COGS).

Answer

Dr. Christian Itin, CEO of Autolus Therapeutics, attributed the Q3 dynamic to the CMS reimbursement policy change impacting Q2 and early Q3 patient enrollment and manufacturing. He noted a healthy deferred revenue balance for Q4 but declined to give Q4 guidance due to holiday seasonality. Rob Dolski, CFO, clarified that depreciation from the Nucleus facility, non-cash stock-based compensation, and commercial milestone amortization are included in COGS, stating it's typical accounting treatment.

Salim Syed asked about the flat quarter-over-quarter performance of AUCATZYL sales despite an increase in authorized treatment centers, inquiring if seasonality or the CMS reimbursement policy change contributed to this trend and its implications for Q4 and deferred revenue. He also asked CFO Rob Dolski about the company's accounting treatment of DNA in the Cost of Goods Sold (COGS) and its impact on gross margins.

Answer

CEO Dr. Christian Itin explained that the CMS reimbursement policy change in Q2 led to a reduction in enrolled patients, impacting Q3 sales, but noted a healthy amount of deferred revenue for Q4. He stated it's too early to predict Q4 seasonality due to holidays and the ASH conference. CFO Rob Dolski clarified that the COGS includes typical depreciation, non-cash stock-based compensation, and commercial milestone amortization, but did not specifically break out DNA costs, indicating it's handled in a standard way.

Salim Syed asked about Atrobi's new patient share percentage, whether it has increased, and if it remains the primary focus for growth, or if there's an increase in the switch category.

Answer

Neil Kumar, CEO and Founder, BridgeBio Pharma, and Matt Outten, Chief Commercial Officer, BridgeBio Pharma, explained that the naive share is estimated to be well in the 20s, with strong double-digit script growth quarter-on-quarter, particularly in the MBRX setting. They noted a slight downturn in the switch category due to combination therapy use and reiterated the focus on maximizing new patient capture to achieve a 30+% market share goal within three to four years.

Salim Syed asked about the percentage of new patient share for Attruby, whether it has increased, and if it remains the primary focus for growth, or if there's an increase in the switch category.

Answer

Neil Kumar, CEO and Founder, BridgeBio Pharma, estimated Attruby's naive share is now well into the 20s, with significant overall script growth, particularly in the MBRX setting. He noted a slight downturn in the switch category due to combination use but emphasized maximizing new patient capture to achieve the goal of 30+% market share. Matt Outten, COO, BridgeBio Pharma, added that Pfizer's matching program has not impacted their strategy.

Salim Syed of Mizuho Securities inquired about the drivers behind the accelerated weekly patient additions for Atruvi and the expected trend for this metric, also asking for an estimate of the market share of naive patients captured in Q2.

Answer

Chief Commercial Officer Matthew Outten attributed the growth to strong momentum in treatment-naive patients, an expanding market from increased screening, and the drug's compelling clinical profile. CEO Neil Kumar estimated the naive patient share is approximately 18-20% and growing, highlighting the increasing scientific share of voice, particularly from recent publications on serum TTR levels, as a key factor.

Salim Syed of Mizuho Securities asked for more specific details on the key tailwinds driving the strong Q1 performance of Attruby, which significantly exceeded expectations.

Answer

CEO Neil Kumar attributed the success to three primary factors: Attruby's differentiated clinical efficacy, including its early 3-month separation and 42% relative risk reduction; continued growth in the overall ATTR-CM market driven by physician education; and effective patient access programs. He also noted a positive 'karma' from physician-friendly actions like providing free lifetime drugs to trial participants. CCO Matt Outten added that the quality of the commercial team was instrumental.

Representing Salim Syed from Mizuho, an analyst asked for details on the withdrawal of the European marketing application for Amtagvi, including EMA feedback and next steps, and inquired if the restructuring would reduce R&D expenses.

Answer

Interim CEO Frederick Vogt explained the EMA submission was withdrawn after learning that additional analysis, such as a virtual control arm, was required. The strategy is to resubmit without new clinical trials. He also confirmed that while the restructuring includes some R&D expense reductions, no significant changes to the clinical pipeline are expected.

Salim Syed of Mizuho Securities questioned the full-year 2025 revenue guidance, suggesting the underlying math implies flat quarter-over-quarter patient infusions after Q2.

Answer

Interim CEO Frederick Vogt refuted the idea of flat growth, stating the guidance implies over 500 infusions for the fiscal year, representing significant year-over-year growth. Chief Commercial Officer Daniel Kirby reinforced this, expecting continued growth driven by increased ATC adoption and new center additions.

An analyst on behalf of Salim Syed asked about the efficacy benchmark for the registrational Phase II trial in post-PD-1 non-small cell lung cancer (NSCLC).

Answer

Interim CEO Frederick Vogt and CMO Dr. Friedrich Graf Finckenstein explained that the FDA has permitted a single-arm trial. The benchmark is the current standard of care (docetaxel +/- ramucirumab), which has response rates in the low 20s with poor durability. They believe a response rate in the mid-20s with the superior durability characteristic of TIL therapy should be sufficient for approval.

Salim Syed from Mizuho Securities questioned what the company expects to learn from the September late-cycle meeting and how much of the REMS negotiation typically occurs after this meeting.

Answer

President and CEO Robert Blum expressed hope that the meeting will confirm alignment with the FDA and a clear path to approval, with no new issues arising. He noted that prior REMS discussions were fruitful and that he hopes the meeting validates their progress and moves them toward final label negotiations.

Salim Syed asked for clarification on the FDA's decision to request a REMS for aficamten after initially accepting the NDA filing without one.

Answer

Robert I. Blum, President and CEO, explained that unlike for mavacamten, the FDA did not provide prior guidance to submit a REMS. Based on pre-NDA discussions, Cytokinetics believed it was reasonable to propose risk mitigation through labeling alone. The FDA later requested a formal REMS during the review process, a contingency for which the company was well prepared.

Salim Syed from Mizuho asked for Cytokinetics' perspective on the upcoming 28-day data from competitor Edgewise and whether there's a possibility their drug could proceed without a REMS program.

Answer

President & CEO Robert I. Blum declined to comment on a competitor's program, emphasizing it's early days and data should speak for itself. EVP of R&D Fady Malik added that understanding a drug's safety profile requires data from hundreds of patients, making it premature to comment on a potential REMS. Blum reiterated confidence in aficamten's efficacy, safety, and ease-of-use profile.

Salim Syed of Mizuho Financial Group asked about the potential timeline for publishing positive MAPLE-HCM results and their subsequent inclusion in treatment guidelines, particularly if it could precede a supplementary approval.

Answer

Robert I. Blum, President and CEO, affirmed that the company will maintain its aggressive publication strategy to ensure data is peer-reviewed and available for consideration. He noted that guidelines are updated continuously and that while publishing quickly is a goal, he could not commit to a timeline relative to a potential approval.

Salim Syed presented a calculation suggesting that the 2026 consensus sales forecast for Nyktymbo appears very low based on current run rates. He also asked about the rationale for updating the NPM1 patient population estimate.

Answer

CFO Keith Goldan agreed that Nyktymbo could be a 'several hundred million dollar product' in the next few years, referencing partner Incyte's positive outlook. CEO Michael Metzger addressed the NPM1 estimate, stating that confidence in capturing the higher end of the range comes from seeing the drug used in earlier lines of therapy. He also highlighted the potential for Nyktymbo's treatment duration to extend for years, boosting its value.

Salim Syed from Mizuho asked for a breakdown of Revuforj sales by SKU to better understand the blended WAC. He also sought clarification on the Niktimvo launch metric, asking if the 1,250 infusions were as of March 31 and if it represented infusions or unique patients.

Answer

Chief Commercial Officer Steven Closter stated that a specific SKU breakdown for Revuforj would not be provided yet, but confirmed the majority of use is the 110mg and 160mg doses. For Niktimvo, he clarified the 1,250+ figure was through the end of March and represents the number of infusions, not unique patients, as it is an estimate based on product shipped to distributors.

An associate for Salim Syed of Mizuho Securities asked about the timing, addressable market size, and strategic approach for launching Revuforj outside the United States.

Answer

CEO Michael Metzger stated that ex-U.S. plans are not finalized but will be tied to the frontline development strategy. He estimated the ex-U.S. market to be about half the size of the U.S. market and confirmed the strategy is to find a partner for Revuforj's ex-U.S. development and commercialization, similar to their existing ex-U.S. partnership with Incyte for Niktimvo.

Salim Syed from Mizuho Securities questioned the decision to expand cohort two of the WVE-007 study, asking if it was driven by protocol requirements, data variability, or a strategic move to accelerate development. He also asked if any GSK milestones are tied to the upcoming WVE-006 data.

Answer

President & CEO Paul Bolno clarified the WVE-007 cohort expansion was a strategic decision based on robust and statistically significant Activin E reduction in cohort one, which translated well from preclinical models. This gave them confidence to enrich the dataset at a dose modeled for efficacy. Regarding GSK, he confirmed that milestone payments are reasonably expected in 2025-2026 but could not specify if they are tied directly to this upcoming data release.

Salim Syed of Mizuho Securities sought clarity on the INHBE data release, asking if the timing was undecided or just undisclosed, and questioned whether the plan to use dystrophin for accelerated approval in DMD was definitively locked in with the FDA.

Answer

President and CEO Dr. Paul Bolno confirmed that for the INHBE program, the data cutoff time point is predefined but simply has not been publicly disclosed, and the company is not viewing blinded data. Regarding the DMD regulatory path, he stated that the responsible FDA division has affirmed dystrophin as a clinical surrogate endpoint. While acknowledging that policies can change, he views it as highly unlikely that the agency would overturn this precedent, emphasizing that the focus is on executing a robust confirmatory study.

Salim Syed from Mizuho Group asked about the Duchenne muscular dystrophy (DMD) program, specifically whether regulator discussions are based on 24- or 48-week data, the potential outcomes of the upcoming 48-week readout, the role of PMO switchers, and the company's preliminary pricing strategy.

Answer

President and CEO Paul Bolno clarified that regulatory engagement was based on the 24-week data, with feedback expected alongside the 48-week results. He outlined that the upcoming data will assess dystrophin kinetics, muscle architecture, and functional trends. Bolno noted a substantial opportunity exists with both PMO switchers and untreated patients but declined to comment on specific pricing plans at this stage.

Salim Syed of Mizuho Securities questioned whether Wave has 'full agreement' from the FDA on using caudate atrophy as an endpoint for WVE-003, how the pre-symptomatic Huntington's disease population is being factored into the trial design, and if the trial might require an extended duration without full certainty on the endpoint.

Answer

Chief Development Officer Anne-Marie Li-Kwai-Cheung clarified that the FDA does not give 'full agreement' without data in hand, but their supportive feedback on being open to the endpoint is the best possible outcome at this stage. The planned study would be for accelerated approval, with a subsequent confirmatory study for full approval. She and CEO Paul Bolno confirmed the trial design intends to include earlier-stage, pre-symptomatic patients, which is enabled by the allele-selective approach's favorable benefit/risk profile.

Salim Syed from Mizuho Securities questioned the drivers behind Nyctivo's strong quarterly performance, asking about intra-quarter dynamics following its J-code implementation and the impact of channel inventory.

Answer

CEO Bill Meury highlighted strong underlying momentum, with 10% penetration in the third-line plus market and high patient retention. EVP & Head of U.S. Oncology Mohamed Issa added that inventory accounted for less than 5% of Q2 sales, confirming that the strong performance was driven primarily by patient demand.

Salim Syed inquired about the drivers of Niktimvo's strong $14 million Q1 sales, asking about any one-time dynamics and the impact of its J-code activation in April.

Answer

Executive Mohamad attributed the strong launch to high unmet need in fourth-line plus GVHD patients. He identified two temporary factors: about one-third of Q1 sales was from inventory build, and only half of the EAP patients converted to paid drug in Q1, with the remainder expected in Q2. He noted the inventory level is expected to stabilize.

Representing Salim Syed from Mizuho, an analyst asked for guidance on modeling Jakafi sales through 2025, specifically regarding the growth rate versus 2024 and the expected cadence of sales throughout the year.

Answer

Christiana Stamoulis, CFO, reiterated that the guidance implies 5-7% year-over-year growth for Jakafi. She advised that Q1 is consistently the lowest quarter of the year due to the reset of patient deductibles and that Q1 2025 net sales should be modeled to be lower than Q4 2024.

Salim Syed from Mizuho asked about Kura's updated market share expectations for ziftomenib in the relapsed/refractory NPM1 AML setting, considering the perception that competing menin inhibitors have similar efficacy rates.

Answer

CEO Troy Wilson emphasized that ziftomenib's complete profile, including safety and tolerability, will be key differentiators, especially in combination settings. Executive Brian Powl added that internal demand studies show a competitive space, but Kura is confident in its profile and will leverage its partnership with Kyowa Kirin to compete for every patient.

Salim Syed from Mizuho sought more detailed insights into the macro environment, particularly the nature of discussions with officials in Washington D.C. regarding vaccine policy, PCVs specifically, and the recent ACIP meeting delay.

Answer

CEO Grant Pickering suggested the ACIP meeting delay could be a simple procedural issue and not a cause for concern. Executive Andrew Guggenhime emphasized that Vaxcyte has had constructive, bipartisan conversations that affirm the critical public health and economic role of vaccines. He noted that PCVs are considered a cornerstone of immunization and advised stakeholders to focus on official actions rather than speculation, highlighting broad support for the existing science-driven regulatory framework.