Eric Schmidt

Eric Schmidt of Cantor Fitzgerald asked for details on the CDMO inspection observations, whether a reinspection would be required prior to approval, and if the facilities are currently operational.

Answer

CEO David Hallal explained that receiving observations is common and that a "very constructive" late-cycle FDA meeting was held after the observations were noted. He confirmed both facilities continue to operate and manufacture products, and that the agency is still working toward the September 22 PDUFA date. He also noted Scholar Rock's launch supply is manufactured and ready.

Eric Schmidt asked about Legend Biotech's strategies to mobilize CARVYKTI demand now that supply constraints are easing, and the timeline for achieving near full utilization of the 10,000-dose capacity.

Answer

Alan Bash, President of CARVYKTI, outlined plans to educate physicians on the benefits of earlier treatment, expand the community strategy, and leverage community and regional hospitals. He expects full utilization of the 10,000-dose capacity in 2026. Ying Huang, CEO, added that all four nodes are currently running at nearly 100% capacity utilization.

Eric Schmidt asked about the most important strategies Legend Biotech is implementing to mobilize demand for CARVYKTI now that supply constraints have eased, and the expected timeline to achieve near full utilization of the 10,000 doses capacity.

Answer

Alan Bash, President of CARVYKTI, outlined plans to educate physicians on the benefits of earlier treatment, drive referrals from community physicians, and engage community networks for CAR-T administration. Ying Huang, CEO, added that all four manufacturing nodes are currently running at nearly 100% capacity utilization and are expected to maintain high utilization in 2026, aligning with consensus revenue projections.

Eric Schmidt from Cantor Fitzgerald asked for scientific insights gained from the Oxbryta withdrawal, particularly concerning its mechanism of action compared to mitapivat, and how this might affect the probability of success for the RISE UP study.

Answer

CMO Dr. Sarah Gheuens emphasized that mitapivat has a very different mechanism of action, working to reduce 2,3-DPG and increase ATP to address both hemolysis and sickling. She contrasted this with Oxbryta's mechanism and pointed to mitapivat's robust, placebo-controlled Phase II data as strong support for its distinct and promising profile.

Eric Schmidt of Cantor Fitzgerald inquired about Agios's strategy for communicating any future safety updates for mitapivat, particularly regarding liver injury, and asked for details on how the company constructs its multibillion-dollar peak sales estimate for the drug in thalassemia and sickle cell disease.

Answer

Chief Medical Officer Dr. Sarah Gheuens confirmed that Agios would update the investor community on any changes to the drug's safety profile, consistent with past practices. CEO Brian Goff reiterated strong conviction in the multibillion-dollar potential, citing the significant unmet need in thalassemia, where two-thirds of U.S. patients lack approved therapies, and the growing need in sickle cell disease. Goff noted that more specific guidance would follow regulatory approvals and key data readouts.

Eric Schmidt of Cantor Fitzgerald inquired about the company's planned discussions with the FDA regarding the LOTUS-7 trial, asking what outcomes are hoped for and how results might be communicated. He also asked whether the next data update would be at a medical conference or a company-sponsored event.

Answer

CEO Ameet Mallik stated that with accelerated enrollment in the 100-patient cohort, the company is pursuing a compendia listing strategy while also planning to engage the FDA on potential regulatory paths in second-line or frontline settings. Chief Medical Officer Mohamed Zaki added that discussions would focus on expediting patient access. Regarding the next data update, Mallik noted the company is weighing options, considering that medical congresses have early data cutoff dates which could limit the maturity of the data presented.

Eric Schmidt of Cantor Fitzgerald inquired about the number of additional patients expected in the upcoming EHA data presentation for LOTIS-7 and asked for the company's perspective on the combination's overall profile, particularly its efficacy and safety compared to other therapies.

Answer

CEO Ameet Mallik confirmed more patients would be included in the EHA update but could not disclose the exact number due to embargo rules, though he affirmed it was in the ballpark of 10-15 more than the December update. He emphasized the combination's highly encouraging efficacy, manageable safety profile with only low-grade CRS, and its novel mechanism of action as key differentiators.

Eric Schmidt inquired about the specific timing and format for the anticipated Q2 data update for the LOTIS-7 trial.

Answer

CEO Ameet Mallik began to respond, but the connection was lost due to an audio gap in the transcript, preventing the full answer from being recorded.

Eric Schmidt asked about the key learnings from the decision to discontinue the ADCT-601 AXL program and sought more specific timelines for the next solid tumor IND filing or a potential partnership. He also requested quantification of the net pricing gains for ZYNLONTA.

Answer

CEO Ameet Mallik explained that the ADCT-601 program was discontinued due to an unfavorable benefit-risk profile observed during dose optimization, despite early positive signals, reflecting a disciplined go/no-go decision. He indicated a partnership was more likely a 2025 event. CFO Jose Carmona added that the timeline from drug candidate selection to IND is roughly 18 months and clarified that ZYNLONTA pricing involves low single-digit increases twice a year, compounding to a mid-single-digit annual increase.

Eric Schmidt inquired about the plan to update investors following the upcoming FDA meeting, the specific discussion points for the agency, and whether the company intends to apply for a priority review voucher for CD388.

Answer

President and CEO Dr. Jeffrey Stein confirmed the FDA meeting is scheduled for August and that the company will communicate the outcome in detail after receiving the official meeting minutes. He noted the primary goal is to corroborate the previous Type C meeting agreement in light of the new Phase 2b data. Dr. Stein also confirmed that a statement of interest for a voucher has been submitted, agreeing that CD388 appears to be a good fit.

Eric Schmidt asked for clarification on whether discussions with the FDA regarding the statistical analysis plan for the Phase IIb study were ongoing or complete, and inquired about the specific definition of the 'high-risk' patient population for the planned Phase III trial.

Answer

Executive Jeffrey Stein confirmed that discussions with the FDA have already taken place and that the updated statistical analysis plan, along with a more detailed definition of the high-risk patient population, will be shared at the company's R&D Day on May 22.

Eric Schmidt of Cantor Fitzgerald asked about the TEBI-AM trial, questioning if hitting on the combination arm but missing on monotherapy would provide enough evidence of Chemtrec's contribution for the FDA. He also requested quantification of the impact from previously deferred revenue in Europe.

Answer

David Berman, EVP of R&D, expressed confidence, noting the trial's eligibility criteria and the expectation that the control arm will reflect real-world retreatment with anti-PD-1, providing the necessary contribution of components. Travis Coy, CFO, quantified the total revenue reserves from 2024 at approximately $18 million, which was spread roughly evenly throughout that year.

Eric Schmidt from Cantor Fitzgerald & Co. asked about the brenetafusp PRISM-MEL study, specifically the follow-up duration for the first 90 patients in the dose selection analysis and whether a decision could be based on early disease control. He also inquired about a potential update on Phase I data for brenetafusp in second-line melanoma.

Answer

Ralph Torbay, Head of Commercial, responded that the 90-patient dose selection analysis involves an 8 to 12-week follow-up period. The review will assess both safety and initial response rates, looking for significant differences between the two active doses.

Eric Schmidt of Cantor Fitzgerald asked about the potential peak sales ceiling for KIMMTRAK in its current uveal melanoma indication, given its strong growth and longer-than-expected duration of therapy.

Answer

Ralph Torbay, Head of Commercial, stated that future growth in the current indication will be driven by the U.S., but the larger opportunity lies in label expansions. CFO Brian Di Donato added that the extending duration of therapy, now over 11 months, represents a significant upside with an unknown ceiling.

Eric Schmidt of Cantor Fitzgerald asked about the potential peak sales ceiling for KIMMTRAK in its current uveal melanoma indication, considering its strong growth, $300M annualized run rate, and longer-than-expected duration of therapy.

Answer

Ralph Torbay, Head of Commercial, stated that future growth in the current indication will be driven by the U.S. due to European reimbursement challenges, but highlighted the much larger opportunity from label expansions (TEBE-AM and ATOM trials). CFO Brian Di Donato added that the extending duration of therapy, now over 11 months, represents a key upside with an unclear ceiling.

Asked about the peak sales potential for KIMMTRAK in its current uveal melanoma indication.

Answer

While U.S. growth and extending duration of therapy provide upside in the current indication, the major future growth is expected from label expansions into cutaneous melanoma and adjuvant uveal melanoma, potentially reaching up to 6,000 patients across all indications.

Eric Schmidt from Cantor Fitzgerald questioned the competitive dynamics for Leqembi in the U.S., asking about potential market share shifts and the impact of a competitor's recent label update for safety.

Answer

Alisha Alaimo, President & Head of North America, stated that while competition is expanding the overall market (which grew ~15% in Q2), Leqembi maintains a majority market share of nearly 70%. She noted the competitor's growth is primarily from existing Leqembi prescribers. Priya Singhal, EVP & Head of Development, added that an independent analysis suggested the competitor's label update may have limited impact.

Eric Schmidt from Cantor Fitzgerald questioned the U.S. market dynamics for Leqembi, specifically its market share against donanemab and the potential impact of a competitor's recent safety label update.

Answer

Alisha Alaimo, President & Head of North America, stated that while a competitor helped grow the market by 15% in Q2, Leqembi maintains nearly 70% market share and is driving new prescriber growth. She noted it's too early to assess the label update's impact. Priya Singhal, EVP & Head of Development, added that an independent analysis suggested the competitor's modified dosing benefits were confined to a small patient subset.

Eric Schmidt asked if the Overall Survival (OS) data from the HARMONi trial will be mature at its mid-2025 readout and what the FDA requires for a U.S. approval. He also questioned whether the preliminary 0.777 OS hazard ratio from HARMONi-2 is likely to mature in a favorable or unfavorable direction over time.

Answer

Executive Dave Gancarz stated that while data on both primary endpoints (PFS and OS) will be available for HARMONi, the company has not disclosed specifics on OS maturity. Regarding the HARMONi-2 hazard ratio, Gancarz emphasized the encouraging nature of the early positive trend. Dr. Jack West added that clinicians are not deterred by the preliminary nature and view a hazard ratio below 0.8 against a strong comparator like pembrolizumab as highly positive and clinically meaningful, suggesting strong potential for ivonescimab.

Eric Schmidt asked if the HARMONi overall survival data will be mature at readout, what is required for U.S. approval, and whether the HARMONi-2 OS hazard ratio of 0.777 is likely to change over time.

Answer

Executive Dave Gancarz stated that while data on both primary endpoints will be available, the maturity level hasn't been disclosed, and approval will depend on a 'total package' review. Regarding the hazard ratio, he noted the early data is encouraging but has variability. Dr. H. Jack West, VP, added that clinicians view a hazard ratio below 0.8 against a strong comparator like pembrolizumab as highly positive and potentially practice-changing, even if preliminary.

Representing Eric Schmidt from Cantor Fitzgerald, an analyst asked what would be considered meaningful and competitive data for HiSCR75 in the povorcitinib HS trial. They also asked what would constitute meaningful data for tafasitamab in first-line DLBCL over the current standard of care.

Answer

Pablo Cagnoni, President, Head of R&D, responded that for povorcitinib, results in the vicinity of the strong Phase II data would be very important. For tafasitamab in first-line DLBCL, he explained that because it's a curative setting, even a modest improvement in progression-free survival could be highly meaningful and lead to wide adoption, referencing the market uptake of Polivy as a benchmark.

Eric Schmidt from Cantor Fitzgerald inquired about the progress on formulary access and reimbursement efforts for Opzelura ahead of its potential launch in pediatric atopic dermatitis (AD).

Answer

Matteo Trotta, an executive at Incyte, expressed excitement about the opportunity in pediatric AD, noting the high unmet need among 2 million patients. He conveyed confidence that Opzelura will maintain competitive overall formulary coverage for 2025, which would include the pediatric indication upon its potential approval.

Eric Schmidt asked for an estimated BLA timeline for reni-cel now that adolescent cohort enrollment is complete, and what new information to expect from the late 2024 data update.

Answer

Chief Medical Officer Baisong Mei stated that while no specific BLA timeline has been shared pending final FDA alignment, the company is optimistic and using the CASGEVY approval as a benchmark. He added that the year-end update will feature data from more patients and longer follow-up, with over 10 patients having one-year exposure, creating a data package similar to the CASGEVY BLA.

Asked for the specific definition of a 'new start', inquired about Q1 financial trends like COGS and OpEx amidst the restatement, and asked for a timeline on when the restated financials will be released.

Answer

A 'new patient start' is defined as the first unique, completed cell collection. The company declined to comment on specific financial trends or provide a timeline for the restatement, but reiterated that cash and key operational metrics are not impacted.

Eric Schmidt of Leerink Partners LLC inquired about the enrollment of adolescent patients in the STARS study, the level of detail expected in the Q3 top-line data release, and the company's business development goals for 2018.

Answer

Executive Amit Rakhit explained that while specific numbers weren't being disclosed, strong demand from the adolescent community led to over-enrollment in the STARS study, and baseline demographics would be shared at the upcoming AAN meeting. He also confirmed the Q3 top-line release would provide a clear sense of the safety and efficacy data. Executive Yaron Werber, prompted by CEO Jeremy Levin, described business development as an active and ongoing process with compelling opportunities, advising to 'stay tuned' for updates.

Eric Schmidt from Cowen & Company asked about the expected patient enrollment rate for the SNS-062 trial and sought confirmation on the timeline between dose cohorts, wanting to understand the feasibility of presenting data from several cohorts by Q2 2018.

Answer

President and CEO Dan Swisher confirmed that all five trial sites are now open and enrolling. He stated there is a one-month safety evaluation period between cohorts and estimated an overall 8-10 week timeline per cohort. This pace supports the goal of having data from several initial cohorts, likely through the active dose levels, available for presentation in the spring of 2018.

Eric Schmidt from Cowen and Company asked about the activation status of clinical trial sites for SNS-062, the initial starting dose and its expected target coverage, and the projected timeline for the first data release from the study.

Answer

Linda Neuman, VP of Clinical Development, confirmed that sites at U.C. Irvine and The Ohio State University have been initiated and are identifying patients. She also verified the 25mg BID starting dose, which achieved complete BTK inhibition in the Phase 1A study. Dan Swisher, President and CEO, added that they plan to present initial data toward the end of the year, with a more robust dataset expected by mid-2018.

Eric Schmidt from Cowen and Company inquired about the upcoming AACR Meeting presentation for SNS-062, asking if new preclinical data would be shared, and questioned the rate-limiting steps and timeline for initiating the Phase II study.

Answer

President and CEO Daniel Swisher confirmed the AACR presentation would detail SNS-062's efficacy against the C481S mutation. VP of Clinical Development Linda Neuman added that trial initiation in Q2 is pending vendor and lab setup and coordination with five top CLL centers.

Eric Schmidt from Cowen & Company asked about the company's communication plan for the Day 180 questions from the CHMP, the status of the SNS-062 study regarding multiple ascending dose cohorts, the selected dose for the Phase 1B trial, and whether an investor event was planned around the upcoming ESH meeting.

Answer

President and CEO Dan Swisher explained they will announce when the regulatory clock restarts. Debbie Thomas, SVP of Regulatory Affairs, added that they expect the Day 180 list of issues in mid-December and that it should be shorter than the Day 120 list. Ms. Thomas and Mr. Swisher clarified that the healthy volunteer study was single-dose only and the company is now moving into a Phase 1B/2 study in patients. Mr. Swisher declined to disclose the starting dose pending IND filing but confirmed a dinner event for investors at the ESH meeting.