Roger Song

Roger Song asked about the enrollment timing for TETON PPF, the read-through of TETON 2 data to IPF regarding mechanism of action, and the potential outcomes and expedited approval scenarios from the upcoming FDA meeting for IPF.

Answer

Martine Rothblatt, Founder, Chairman, and CEO, directed the questions to Dr. Leigh Peterson, EVP of Product Development and Xenotransplantation. Dr. Peterson stated TETON PPF enrollment is over halfway complete, with a 52-week follow-up period. She explained that similarities in underlying fibrosis between IPF and PPF, along with preclinical data showing treprostinil's anti-fibrotic and vasodilation effects, suggest similar mechanisms of action. Regarding regulatory path, she confirmed an agreement with the FDA to use both TETON 1 and TETON 2 data, with TETON 1 results expected in H1 2026. An FDA meeting is scheduled before year-end to discuss expediting the regulatory review process.

Roger Song asked about the data timing for TETON PPF enrollment, the read-through of TETON 2 data to IPF's mechanism of action, and the potential outcomes of an upcoming FDA meeting regarding expedited approval.

Answer

Martine Rothblatt, Founder, Chairman, and CEO, referred the question to Dr. Leigh Peterson, Executive Vice President of Product Development and Xenotransplantation. Dr. Peterson reported TETON PPF is over halfway enrolled with a 52-week follow-up. She explained that similarities in underlying fibrosis and preclinical data suggest an anti-fibrotic effect of treprostinil in both IPF and PPF. She also confirmed a meeting with the FDA before year-end to discuss expediting regulatory review using both TETON 1 and TETON 2 data.

Roger Song from Jefferies asked what would be considered a clinically meaningful FVC result from the TETON trial, what a 'homerun' scenario would entail, and what proportion of TETON patients have concomitant pulmonary hypertension.

Answer

Dr. C.Q. Deng, SVP of Biostatistics, responded that the TETON study was designed to test the anti-fibrotic effect of treprostinil, not its vasodilator effect. Therefore, the study enrolled a typical IPF population, and the company did not perform hemodynamic measures to determine if subjects had pulmonary hypertension.

Roger Song asked about Kiniksa's strategy for increasing penetration into the first recurrence patient population for ARCALYST, given the strong revenue growth and existing penetration.

Answer

Ross Moat (Chief Corporate and Commercial Officer, Kiniksa Pharmaceuticals) clarified that ARCALYST's label is agnostic to the number of flares, and while initial use was more in the two-plus recurrence group, physicians are increasingly prescribing it for first recurrence patients (now 20% of prescriptions). He attributed this shift to growing physician comfort, positive patient experiences, and a desire to prevent additional flares, highlighting the significant untapped opportunity in both first and multiple recurrence groups.

Roger Song from Jefferies asked about the target efficacy profile for KPL-387 that would be considered successful in the Phase 2 dose-finding portion, and also inquired about the potential indications for the company's quarterly IL-1 inhibitor.

Answer

EVP & CCO Ross Moat explained that for KPL-387, they are looking for a profile similar to Arcalis—the ability to treat acute flares and prevent subsequent ones—and will use the totality of data to select the dose. Regarding the quarterly IL-1 asset, he noted that while no specific indication has been announced, it has broad potential in chronic auto-inflammatory diseases, and the immediate focus is on IND-enabling studies.

Roger Song asked about the timing of updates on the four-week off-treatment follow-up and PK data from the Phase 2 oral study. He also inquired about the likelihood of testing weekly oral doses in a potential pivotal Phase 3 and how to think about low starting doses versus higher doses used in Phase 2.

Answer

Brian Lian (President and CEO, Viking Therapeutics) stated that the final PK report from the VENTURE-Oral Dosing Study is expected within the next couple of weeks. He explained that decisions on weekly oral doses for a potential pivotal Phase 3 would depend on the maintenance study results. If an extension were added to oral Phase 3 studies, a low-dose maintenance could be considered then.

Roger Song asked about updates on the four-week off-treatment follow-up and PK data from the phase 2 oral study, and how likely Viking would test weekly oral dosing in a potential pivotal phase three, considering the maintenance study. He also asked about the strategy for low starting doses versus higher doses in the next oral study.

Answer

Brian Lian (President and CEO, Viking Therapeutics) stated that the final PK report from the Venture Oral Dosing Study is expected within the next couple of weeks. He explained that decisions on weekly oral dosing and specific dose strategies for a potential pivotal phase three would be made after evaluating the maintenance study data, potentially adding low-dose maintenance to extension periods of oral studies.

Roger Song from Jefferies asked for expectations on the high-dose versus maintenance-dose cohorts in the upcoming Phase II oral data. He also inquired if a weekly oral dosing regimen would be considered for the maintenance study.

Answer

President & CEO Brian Lian confirmed that a weekly oral cohort will be included in the upcoming maintenance study. Regarding the Phase II oral study, he highlighted the cohort that titrates up to 90mg and then steps down to 30mg as particularly informative for a maintenance strategy. He reiterated that achieving mid-to-high single-digit weight loss, similar to the 8% seen in Phase I, would be a strong result.

Roger Song of Jefferies questioned the consistency of data across different CD47 expression cutoffs, the applicability of the 10% cutoff to other programs, and the biological rationale for this expression level.

Answer

CEO Jason Lettmann stated that the positive effect was robust and consistent across various cutoffs, with full data on ORR, DOR, PFS, and OS to be presented in Q4. He noted the cutoff for future studies is to be determined. Both Lettmann and CMO Alan Sandler referenced literature supporting CD47 as a well-studied negative prognostic marker, providing a strong biological basis for the strategy.

Roger Song from Jefferies asked about the remaining work required before requesting the end-of-Phase 2 meeting and how much 48-week data is necessary for the Phase 3 proposal. He also inquired how the new Chairman appointment might alter the company's strategy on partnerships and commercialization.

Answer

Chief Medical Officer Dr. M. Scott Harris expressed confidence in holding the FDA meeting by year-end, noting they have sufficient data. CEO Dr. Vipin Garg described the Chairman change as a 'natural evolution' to leverage the new appointee's experience in liver disease and M&A as the company advances toward Phase 3.

Roger Song of Jefferies questioned which of the dual primary endpoints in the IMPACT study is historically harder to achieve and asked about the correlation between the recently presented MASH resolution index and fibrosis improvement.

Answer

CBO Ray Jordt acknowledged that fibrosis improvement has historically been the more challenging endpoint to hit. He explained that the MASH resolution index is highly predictive of MASH resolution and emphasized that liver fat reduction, where pemvidutide is a leader, is the primary driver for both MASH resolution and fibrosis improvement, giving them confidence in the trial's success.

Roger Song inquired about the criteria for the recently decided RCC expansion cohort for KO-2806, the next steps for the PIK3CA and KRAS programs, and whether there have been any recent FDA interactions regarding the Phase 3 pivotal design for Zifdomenib.

Answer

Chief Medical Officer Dr. Mollie Leoni explained that the RCC expansion cohort decision was based on meeting pre-set criteria and is designed to fulfill Project Optimus requirements. Regarding the FDA, she confirmed interactions are routine for an agreed-upon design and that everything is proceeding as planned for the COMET-seventeen trials.

Roger Song from Jefferies inquired about key launch metrics for Vaseo, including patient segmentation between home use and high ESA users, the expected impact of adding larger dialysis organizations like DaVita, and the payer mix between Medicare Advantage and fee-for-service. He also asked for the current average dose strength.

Answer

Nicholas Grund, SVP & Chief Commercial Officer, explained that protocols are broad, with home use patients representing about 12% of scripts, and Medicare Advantage accounting for about 20% of prescriptions, a figure expected to grow. He noted that refill doses are up 28% from the starting dose. CEO John Butler added that as new patient cohorts begin treatment, the average dose may temporarily decrease before titrating up.

Roger Song asked about the expected evolution of Vafseo's net price as large dialysis organizations (LDOs) increase adoption, the future mix of new versus refill prescriptions, and management's confidence in initiating the NDD trial in the second half of 2025 amid changes at the FDA.

Answer

CEO John Butler stated that while the net price per patient for Vafseo will likely decrease over time with higher volumes, there will be 'lumpiness' as LDOs come on board. Chief Commercial Officer Nicholas Grund added that the new-to-refill script ratio will also be lumpy with new launches, but highlighted that dose titration on refills is increasing revenue per patient. Butler affirmed confidence in the H2 2025 NDD trial start, noting their FDA review team is stable and they are proceeding with preparatory work.

Roger Song of Jefferies inquired about Novavax's regulatory filing and supply readiness for the 2025-2026 COVID-19 season, and also asked for an update on the progress and timeline for securing a partnership for its COVID-influenza combination (CIC) vaccine.

Answer

EVP, CFO & Treasurer Jim Kelly confirmed that the JN.1 vaccine's BLA is approved and current regulatory efforts are focused on extending its shelf life. President, CEO & Director John Jacobs stated that while active discussions with multiple potential partners for the CIC vaccine are underway, details remain confidential. He highlighted positive commentary from Sanofi on the asset's potential. EVP, Head of Research & Development Ruxandra Draghia-Akli added that the recent trial was a Phase 1 cohort to strengthen the data package for potential partners, who would conduct any future registrational trials.

Roger Song from Jefferies asked for guidance on expectations for the upcoming WVE-006 (AATD) data, specifically the differences between the multi-dose and single-dose cohorts and whether to focus on absolute M-protein levels or percentage of total protein. He also inquired about the follow-up period for the WVE-007 (Inhibin E) data readout in Q4.

Answer

President & CEO Paul Bolno stated that the 200 mg multi-dose cohort is expected to have greater liver exposure than the 400 mg single-dose, making it a key focus. He anticipates seeing higher protein levels than the initial single-dose data, which already achieved MZ phenotype levels. Bolno emphasized that M-protein is a critical, standardized measure to follow. For WVE-007, he confirmed at least three months of follow-up data would be available for cohorts one and two at the time of the Q4 readout.

Roger Song from Jefferies inquired about the pricing strategy for INO-3107 given its long-term durability data, and asked about the costs and funding plans for the confirmatory study and commercialization.

Answer

CCO Steven Egge indicated that based on payer research, a price analogous to SpringWorks Therapeutics' OGSIVEO ($360,000/year) is being considered for the initial treatment and is seen as appropriate by payers. CEO Jacqueline Shea stated that Inovio plans to self-commercialize in the U.S. with a small field force and is open to ex-U.S. partnerships for funding and market access.

Roger Song from Jefferies asked about the dropout rate in the Phase Ib atopic dermatitis trial versus expectations for Phase II, the strategy for studying post-biologic populations in Phase III, and the company's plans for a potential partnership for REZPEG.

Answer

Executive Jonathan Zalevsky noted the Phase Ib dropout rate was 20-30% and Phase II rates will be reported in June. He stated the Phase III program will include biologic-experienced patients. Executive Howard W. Robin added that Nektar will seek a collaboration for Phase III to minimize dilution while retaining significant ownership, ruling out a simple out-licensing deal.

Jiale Song asked about the specific criteria for the go/no-go decision on the glioblastoma (GBM) program, whether preclinical models show different activity based on the number of antigens expressed in the squamous lung cancer program, and what is included in the cash runway guidance to 2028.

Answer

CSO Myriam Mendila specified that the go-forward criteria for the GBM program is a median overall survival of 15 months or longer, or an overall response rate around 20%. She clarified that animal models cannot determine efficacy based on the number of human antigens. CFO Axel-Sven Malkomes stated the cash runway into 2028 includes the planned clinical phases for the current core pipeline, though some trial phases may extend beyond that timeframe.

Asked about the potential value and next steps for out-licensing luvelta, and the timeline for preclinical and initial clinical data for STRO-004.

Answer

The company is actively seeking a partner to take over luvelta development to realize its full potential, noting that the decision to deprioritize was due to resource constraints, not a lack of belief in the drug. For STRO-004, an IND will be filed in the second half of the current year, with initial clinical data expected in 2026-2027, supported by encouraging preclinical data showing a superior therapeutic index.

On behalf of Roger Song, Cha Cha Yang asked for details on the planned Phase II trial for soquelitinib in atopic dermatitis, specifically regarding the target patient subpopulations, including prior medication use, baseline EASI scores, and any geographic or demographic considerations.

Answer

Executive Richard Miller provided a preliminary outline, stating that while Phase I is not yet complete, the Phase II trial would likely target patients with moderate-to-severe AD who have failed topical or systemic therapies. He suggested a standard design, possibly a three-arm study with two dose levels plus placebo, enrolling around 200 patients total. The primary endpoint would likely be EASI 75 or IGA 0/1, which are standard for registration.

Inquired about the extent to which the VAX-24 regulatory package, particularly for CMC, can be leveraged for VAX-31, and asked for details on the high-dose cohort being tested for VAX-31.

Answer

The company expects to leverage a significant portion of the VAX-24 study designs and CMC processes for VAX-31, as the programs are very similar. They believe FDA feedback on VAX-24's CMC will be applicable to VAX-31. Regarding the VAX-31 high dose, they declined to provide specific details for competitive reasons but confirmed they are testing a range of doses to ensure a clear path forward to Phase III.

At which of the two interim analyses will efficacy be compared against the FDA's statistical hurdle? Also, when should the full data from the trial be expected, given completion is slated for the end of 2024?

Answer

The second interim analysis, which uses the primary endpoint of independently assessed ORR, is when the data will be tested against the study's statistical plan. The company will not disclose the specific hurdle but will comment on efficacy and trial continuation. Full data is expected around the end of 2024 or, more likely, in early 2025.

Roger Song from Jefferies asked if IGM would continue dose escalation for IGM-8444 beyond 10 mg/kg and questioned the strategy of targeting "severe" forms of lupus and RA for imvotamab's initial studies.

Answer

Chief Medical Officer Dr. Chris Takimoto indicated that 10 mg/kg is considered a good dose for IGM-8444 with no strong need to go higher. CEO Fred Schwarzer added that a timeline for releasing more single-arm data has not been set. President of Autoimmunity and Inflammation Dr. Mary Beth Harler clarified that they are starting with severe patient populations to establish safety and activity, with the full intention of later moving into milder, broader populations.