Li Wang Watsek

Li Watsek from Cantor Fitzgerald asked whether the current Phase II ASPEN breast study could be registrational and how the trial design addresses the contribution of components for the drug combination.

Answer

CEO Jason Lettmann and CMO Alan Sandler explained that strong data, such as a response rate of 40% or higher, could open a path for accelerated approval discussions with the FDA. Dr. Sandler clarified that the contribution of components is addressed because the study adds evorpercept to a known standard of care (Herceptin plus chemotherapy), making its contribution directly evaluable.

Li Watsek asked for Kura's perspective on the competitor's Menin inhibitor launch in KMT2A-rearranged AML and sought details on the progress of Kura's Phase 3 frontline trials, including the company's confidence in achieving a first-in-class position.

Answer

Chief Commercial Officer Brian Powl stated that while the competitor's launch is positive for patients, Kura is confident in Zifdomenib's potentially best-in-class profile. Chief Medical Officer Dr. Mollie Leoni highlighted that the COMET-seventeen protocol's single design for two trials is attractive to sites, expressing high confidence in a second-half 2025 start and robust enrollment.

Li Wang Watsek of Morgan Stanley inquired about the assumptions behind the 2028 top-line guidance for ziftomenib in frontline intensive AML, including trial size and enrollment. She also asked about any remaining items needed for the NDA submission and feedback from the pre-NDA meeting.

Answer

CEO Troy Wilson explained that the 2028 timeline is a conservative estimate based on alignment with the FDA and EMA on accelerated approval pathways, with more trial details to be shared later. He confirmed that Kura has received constructive feedback and has alignment with the FDA for the NDA submission, with the Q2 timeline being driven by the final clinical data package.

Li Wang Watsek of Cantor Fitzgerald asked about Kura's strategy for using MRD negativity as a potential frontline endpoint for ziftomenib and inquired about the specifics of the upcoming 600mg cohort data presentation at ASH, including patient numbers and follow-up.

Answer

CEO Dr. Troy Wilson explained that while survival is the base case endpoint, they intend to discuss using MRD negativity as a surrogate with health authorities. EVP of Clinical Development, Dr. Mollie Leoni, added that they plan a central analysis of MRD samples for uniform reporting. Dr. Wilson confirmed the ASH update will include data from over 100 patients and is expected to show consistent activity and improved safety at higher doses.

Li Watsek of Cantor Fitzgerald inquired about the remaining steps required to file bemarituzumab in China and the potential timing for an NRDL listing. She also asked for Zai Lab's perspective on upcoming data from a competitor's DLL3 program and what additional information Zai Lab plans to share in its next data update for its own DLL3 ADC.

Answer

Josh Smiley, President & COO, stated that the immediate focus for bemarituzumab is securing regulatory approval, after which they will pursue NRDL listing as quickly as possible. Rafael Amado, President and Head of Global R&D, added they are working with partner Amgen to submit expeditiously. Regarding the DLL3 ADC, Dr. Amado expressed confidence in their asset's best-in-class profile and confirmed the next data update will include more follow-up on response durability, PFS, and a detailed analysis of intracranial activity.

Li Wang Watsek asked about the competitive landscape for VYVGART in China given potential new approvals, and questioned how geopolitical tensions are affecting big pharma's appetite for out-licensing assets to Chinese partners.

Answer

Joshua Smiley, President and COO, expressed confidence in VYVGART's position due to its strong data, safety, and NRDL status, suggesting new entrants will help grow the overall market. Jonathan Wang, CBO, reported that licensing is 'business as usual' with continued high interest from multinational companies in the China market.

Li Watsek questioned the expected durability of ZL-1310 compared to other modalities and the plan for its confirmatory study. She also asked for a Q4 breakdown of new versus maintenance patients for VYVGART and their expected future contribution.

Answer

Dr. Rafael Amado, President and Head of Global R&D, explained it was too early for median durability data on ZL-1310 but noted its efficacy appears independent of prior chemo sensitivity. Joshua Smiley, President and COO, addressed the VYVGART question, stating new patient initiations are around 1,000 per month, with an increasing number starting in the maintenance phase. He cited that 40% of patients who started in Q3 2023 had already returned for a second cycle in Q4 2023.

Li Watsek inquired if a priority review should be anticipated for the Vepdeq NDA submission and asked about the global filing strategy. She also asked about the key considerations Arvinas would prioritize when seeking a potential new partner for Vepdeq to maximize its value.

Answer

CEO John Houston stated they are awaiting details from the FDA on the PDUFA date and will share them once available. CMO Noah Berkowitz added that they have not yet offered guidance on a global filing strategy. Regarding a new partner, Houston emphasized the goal is to place Vepdeq with a company, be it Pfizer or another, that is committed to launching, commercializing, and funding further development of the asset globally, with minimal future development involvement from Arvinas.

Li Watsek asked about Arvinas's business development strategy regarding in-licensing external assets, the company's conviction in its early-stage pipeline, and the expected timing and success bar for the KAT6 combination data.

Answer

CEO John Houston explained that while Arvinas is a platform-driven company, it remains open to in-licensing an asset that complements its lead programs. CMO Noah Berkowitz clarified that the KAT6 combination is an exploratory, all-comer Phase 1 trial run by Pfizer with no set timelines for data, as it is just beginning.

Li Wang Watsek questioned if the upcoming VERITAC-2 results would have any read-through for the two new Phase III trials planned for 2025. Additionally, she asked about the planned level of investment for pipeline programs, particularly for large indications like Parkinson's disease.

Answer

Chief Medical Officer Noah Berkowitz explained that any read-through from VERITAC-2 would primarily impact the second-line trial design rather than the first-line. CEO John Houston stated that Arvinas is well-capitalized to advance the LRRK2 program to a significant milestone internally, though a future strategic partnership remains a possibility.

Li Wang Watsek of Cantor Fitzgerald asked for more detail on the key regulatory inputs still needed to finalize the design of the Phase 3 combination trials and how dependent these designs are on upcoming data.

Answer

CMO Noah Berkowitz explained that following the VERITAC-2 results, Arvinas will engage in discussions with health authorities. Key topics will include the choice of comparator arm, the specific patient population, and, particularly for a first-line trial with two novel agents like atirmociclib and vepdegestrant, the regulatory requirement to demonstrate contribution of components.

Li Watsek of Cantor Fitzgerald inquired about the clinical bar for the BTK combination study in CLL, how emivosertib fits into the evolving CLL landscape, and what expectations to set for the upcoming AML triplet data.

Answer

CMO Ahmed Hamdy stated the goal in CLL is achieving MRD negative or complete remissions for time-limited treatment. CEO James Dentzer added that emivosertib can complement any BTK inhibitor or degrader. CDO Jonathan Zung confirmed plans to present efficacy and safety data from the AML triplet study's 7 and 14-day cohorts at the ASH conference.

Li Wang Watsek of Cantor Fitzgerald inquired about Curis's strategic prioritization between its lymphoma and AML programs and asked what actions are being taken to accelerate patient enrollment for the TakeAim lymphoma study.

Answer

President and CEO James Dentzer explained that while both programs are advancing simultaneously, the later-stage PCNSL lymphoma study is the primary focus and receives more resources. Chief Development Officer Jonathan Zung added that enrollment efforts include engagement with 37 major clinical sites in the U.S., Europe, and Israel. Chief Medical Officer Dr. Ahmed M. Hamdy noted that strategic prioritization is under continuous review.

Li Wang Watsek of Cantor Fitzgerald requested more detail on what the European Medicines Agency (EMA) would consider 'compelling and consistent data' regarding the response rate for emavusertib.

Answer

CEO James Dentzer stated that the goal is for the currently compelling data to remain consistent as enrollment completes. Chief Development Officer Jonathan Zung added that both the EMA and FDA acknowledge the lack of approved treatments in this space, and an Overall Response Rate (ORR) north of 25% should be viewed favorably by both agencies.

Li Wang Watsek requested details on the CD38-SADA Phase I trial, including the number of patients to be enrolled in Part A, the dosing regimen, and the expected timeline for data relative to the GD2-SADA trial.

Answer

CEO Michael Rossi stated that a comprehensive update will be provided at the R&D event on May 28. He noted that the trial is a platform safety study in a challenging relapsed/refractory non-Hodgkin's lymphoma population and is planned to enroll a smaller cohort of approximately 12 to 15 patients.

Li Wang Watsek from Cantor Fitzgerald questioned whether the nine GD2-positive patients in the SADA trial received more than one therapeutic lutetium dose and asked about the company's business development strategy for the new SADA targets it plans to unveil.

Answer

CEO Michael Rossi confirmed that all nine patients who showed significant uptake on the diagnostic scan received a follow-up therapeutic dose as per the protocol. Regarding business development, Rossi explained that the company has identified three core franchise areas for internal commercialization. Targets falling outside these areas would be ideal candidates for partnerships or out-licensing to maximize efficiency. He also mentioned openness to in-licensing targets from external partners.

Li Wang Watsek asked about the key factors influencing Q4 revenue, the company's confidence in hitting the lower end of its guidance, and the radiosensitivity of the various tumor types being studied in the GD2-SADA trial.

Answer

Chief Financial Officer Pete Pfreundschuh expressed confidence in achieving the lower half of the $87-$95 million revenue guidance, noting that Q3 results were impacted by the timing of a SciClone sale and a Medicaid adjustment, which will positively affect Q4. Chief Medical Officer Dr. Vignesh Rajah commented that it is too early to determine radiation sensitivity across the different tumor types in the GD2-SADA trial, which include osteosarcoma, melanoma, and small cell lung cancer, but the company will provide updates as more data becomes available.

Li Wang Watsek asked about the placebo group's response, questioning if the observed effect was typical for atopic dermatitis trials. She also inquired if there was a differential response observed in patients based on their history of prior systemic treatments.

Answer

Richard Miller, an executive, explained that the placebo response was typical, noting the wide standard error bars indicated variability. He pointed out that unlike longer studies for DUPIXENT or JAK inhibitors, no placebo patients in this 28-day trial reached the key EASI 75 or IGA 0/1 endpoints. Regarding prior treatments, Miller stated it was too early for a definitive conclusion but highlighted two DUPIXENT-refractory patients in Cohort 3 who responded significantly to soquelitinib, suggesting a non-overlapping mechanism of action.

Representing Li Watsek, an analyst inquired about Corvus's strategy for prioritizing soquelitinib's development between oncology and inflammatory diseases as it moves to later-stage trials. A follow-up question asked if the delayed presentation of Cohort 4 data was due to enrollment issues or a decision to await Cohort 3 results.

Answer

Executive Richard Miller clarified that the strategy is to 'push on all fronts aggressively' to secure approvals in both lymphoma and immune diseases, citing Rituxan as a precedent for a drug used in both areas. He noted that at the right time, the company would consider a partnership for the vast autoimmune disease opportunity. Regarding Cohort 4, Miller confirmed there is 'no delay in enrollment' and the company is simply waiting to see the full data from Cohort 3 before making a final decision.

Li Wang Watsek of Cantor Fitzgerald inquired about the clinical response kinetics of soquelitinib in atopic dermatitis, its potential for continued improvement post-treatment, and the benchmark for success as an oral therapy.

Answer

Richard Miller, an executive at Corvus, explained that clinical improvement is rapid, seen within the first week. He noted expectations for a durable effect based on the drug's mechanism and stated that the company will compare its 28-day data against approved agents like Dupixent to define success.

Li Wang Watsek asked for expectations on the casdatifan combination with AstraZeneca's VORO in the IO-naive setting and what the comparator would be in a potential pivotal trial.

Answer

President Juan Jaen explained that while full details are not yet disclosed for competitive reasons, a key goal is to lower the high rate of primary progressive disease seen with current IO/IO therapies like ipi/nivo. The initial focus will be on safety and early efficacy signals, particularly the rate of progressive disease. He noted significant investigator interest in a TKI-free frontline option.

Li Wang Watsek of Cantor Fitzgerald asked about the timeline for a pivotal trial in IO-naive RCC with AstraZeneca, sought clarification on optimizing the statistical plan for the domvanalimab lung cancer study, and questioned the clinical relevance of stable disease in the casdatifan study.

Answer

CEO Terry Rosen deferred comment on the AstraZeneca collaboration's timeline until the trial is public and clarified that while statistical plans are always under review, no changes are currently being made for domvanalimab. CMO Dimitry Nuyten emphasized that stable disease is highly relevant for patients and equally important as a response for the registrational endpoint of progression-free survival (PFS).

An analyst from Cantor Fitzgerald & Co., on for Li Watsek, asked about pipeline prioritization. She inquired how Arcus is thinking about its next-generation programs, including its inflammation portfolio, while simultaneously executing on multiple late-stage oncology trials.

Answer

CEO Terry Rosen explained that the collaboration with Gilead was designed to function as an R&D engine, enabling continued investment in discovery alongside late-stage development. He highlighted the AXL inhibitor as a notable early-stage program that recently entered the clinic. Rosen also mentioned that while the company has a strong immunology biology group and will likely increase its focus on immunology and inflammation (I&I), the primary driver for new projects remains identifying the best targets, regardless of therapeutic area.

Li Wang Watsek requested guidance on the gross-to-net trend for YORVIPATH for the remainder of the year and asked about the dosing and titration plan for SKYTROFA within the Phase II COACH combination trial.

Answer

CFO Scott Smith stated that the largest gross-to-net driver will be mandatory government rebates in the low 20% range. EVP Jay Wu added that commercial contracting is expected to be minimal and not materially impact the overall figure. Regarding the COACH trial, Chief Medical Officer Aimee Shu explained there is a safe, universal starting dose for TransCon Growth Hormone, with physicians having the flexibility to titrate based on clinical response and IGF-1 levels.

Li Watsek asked about Yorvipath gross-to-net trends and the SKYYTOVA dosing scheme in the COACH trial.

Answer

CFO Scott Smith discussed government rebates. EVP Jay Wu noted minimal contracting changes. CMO Aimee Shu discussed the starting dose.

Li Wang Watsek asked about the potential revenue contribution for SKYTROFA from the adult and Turner syndrome markets, and the opportunity presented by the planned basket trial.

Answer

Jan Mikkelsen, President and CEO, described the adult growth hormone deficiency market as a highly underpenetrated segment that could rival the pediatric market in size. He explained the basket trial (including ISS, SGA, and SHOX/Turner) is a key part of the strategy to cover the entire growth hormone market and build SKYTROFA into a blockbuster.

Li Wang Watsek asked for the assumptions behind the lowered SKYTROFA guidance and inquired about potential cost reductions and the possibility of achieving cash flow breakeven in 2024.

Answer

President and CEO Jan Mikkelsen and CFO Scott Smith explained the guidance was based on a conservative projection of Q1-to-Q2 volume growth, without assuming a seasonal second-half uplift. On breakeven, Mikkelsen pointed to strong expense control and potential revenue upside from YORVIPATH. Smith added that the new $150M financing provides a buffer while they maintain cost scrutiny, with the company still guiding to quarterly operating cash flow breakeven in 2024 or 2025.

Asked about the upcoming AFM28 data at ASH, specifically regarding durability, patient characteristics (mutational status, prior treatments), and the potential for a registrational monotherapy path with the FDA, including the target patient population.

Answer

The company stated that the initial six patients will have meaningful durability data for ASH, while the additional six are too recent. They detailed the heavily pre-treated nature of the patients (prior HMA/venetoclax, anthracyclines, stem cell transplants). A registrational path would be discussed with the FDA after collecting more data from higher dose cohorts, targeting a later-line AML population with unmet medical need.

Asked about the AFM13 (acimtamig) study, inquiring about patient status, durability, response kinetics for partial responders, and the reasons for improved enrollment progress in recent cohorts.

Answer

The company responded that follow-up is too short for definitive durability data, but most responding patients remain on study. The 6 CRs occurred after the first cycle. Enrollment has improved due to more sites being activated and trained, which is encouraging for future study phases. They believe the current enrollment pace is good.

Asked about the number of treatment cycles the 7 reported patients have received and for a comparison of the patient baseline characteristics, particularly prior lines of therapy, between the current LuminICE study and the previous MD Anderson study.

Answer

Patients are at various stages, with some in their second or third cycle and one proceeding to a stem cell transplant after a complete response; treatment has been well tolerated. The current study has patients with fewer prior lines of therapy (median of 4) compared to the MD Anderson study, which is seen as a positive development as physicians refer patients earlier. All patients still meet the FDA's criteria for unmet medical need.

Inquired about the new AlloNK cohort, asking about the criteria for patient crossover, whether crossover patients would be included in the primary efficacy analysis, and the number of clinical sites needed for enrollment.

Answer

Patients in the AlloNK/IL-2 cohort can cross over to the combination treatment if they do not achieve a partial or complete response. However, these crossover patients will not be included in the primary efficacy analysis cohort to avoid bias. The company anticipates needing 5 to 6 well-recruiting sites for the initial phase of the study.

Li Watsek of Cantor Fitzgerald asked about other potential autoimmune indications for FT522, the strategic positioning between FT522 and FT819, and whether amending the FT819 protocol for alternative conditioning regimens would boost patient enrollment.

Answer

CEO Scott Wolchko declined to disclose specific expansion strategies but confirmed guidance for a clinical update on the first 3-5 FT819 patients by year-end. He emphasized that moving away from cy/flu conditioning is critical for the autoimmune setting and believes offering alternatives like Cytoxan-only will be key to driving adoption and realizing the therapy's potential.