Mayank Mamtani

Mayank Mamtani inquired about the specific endpoints, patient numbers, and criteria for clinical success in the GVHD and severe pulmonary inflammation trials, particularly relative to current standard of care. He also asked about manufacturing scale-up activities and potential non-dilutive financing for stocking requirements.

Answer

Dr. Jennifer Buell (President and CEO, MiNK Therapeutics) detailed that the GVHD trial, funded by a UW award and government grants, will evaluate dose and engraftment success, with GVHD presence/absence at day 100 as a key endpoint. She noted that a 20-50% reduction in GVHD incidence would be meaningful. For pulmonary disease, she mentioned interrogating survival, ventilator-free days, and prevention of secondary infections. Dr. Therese Hammond (Head of Development, MiNK Therapeutics) clarified that the primary endpoint for the pulmonary trial is 28-day mortality in patients with severe pneumonia and moderate to severe hypoxemic respiratory failure. Dr. Buell also addressed manufacturing, stating they can get billions of cells per donor, allowing for stockpiling, and are actively pursuing non-dilutive financing with public and private sector collaborators for future scale-up and stocking.

Mayank Mamtani from B. Riley asked for detailed information on the endpoints, patient characteristics, and clinical success metrics for the GVHD and severe pulmonary inflammation trials, in addition to an update on manufacturing scale-up, non-dilutive financing efforts, and potential stocking requirements for future commercialization.

Answer

President and CEO Jennifer Buell and Head of Development Therese Hammond outlined GVHD trial endpoints including dose and engraftment success, with day 100 GVHD presence/absence as a key readout, aiming for substantial reduction. For pulmonary disease, they noted 28-day mortality as the primary endpoint, with eligibility to be posted on clinicaltrials.gov. Ms. Buell also discussed manufacturing advancements yielding billions of cells per donor, enabling stockpiling, and confirmed active discussions for non-dilutive financing with public and private sector collaborators to support future scale and stocking.

Mayank Mantani of B. Riley Securities inquired about the trial design for the preventative Graft-versus-Host Disease (GVHD) study, including patient numbers and endpoints. He also asked for an update on the gastric cancer study and the company's plans for its targeted INKT program given its current cash position.

Answer

President and CEO Dr. Jennifer Buell explained that the Phase 1 GVHD trial will have a safety run-in followed by a 20-25 patient signal-seeking cohort at a target dose of one billion cells. For the gastric cancer program, she noted that mature follow-up data, including survival outcomes, is expected by year-end. Regarding the targeted INKT program, Dr. Buell stated that while the primary focus is on immune-mediated diseases, the engineered program has advanced significantly and can proceed to an IND with limited expense, potentially accelerated by a partnership.

Mayank Mamtani asked for investigator feedback on the recent ACR IO presentation, the potential for a registrational path for AGENT-797 based on current data, and how the PRAME TCR iNKT approach differentiates from competitors. He also asked for the IND timeline for next-gen programs.

Answer

President and CEO Dr. Jennifer Buell noted that the lead investigator is highly motivated and will present future data. Regarding a registrational path, she said the company will accumulate more data before advancing regulatory discussions. Dr. Buell explained that the PRAME TCR iNKT platform is differentiated by its ability to recruit T cells and NK cells and its favorable tolerability profile. She also confirmed the IND filing for program 215 is planned for 2025.

Mayank Mamtani asked about topics of interest for the end-of-phase two meeting for oral VK2735, the relevance of the subcutaneous safety package, and whether Viking is looking for anything from the oral semaglutide FDA review. He also asked about contemplated dose levels for the next oral study and the broader financial structure, including the CV outcome trial and other commercial studies.

Answer

Brian Lian (President and CEO, Viking Therapeutics) stated that the end-of-phase two meeting would discuss overall study design, duration, and safety package, leveraging the subcutaneous safety data for long-term toxicology. He did not disclose specific dose levels for the next oral study. Greg Zante (CFO, Viking Therapeutics) indicated that OpEx is expected to continue at higher levels due to phase three activities. Brian Lian added that blocking and tackling studies (renal/hepatic impairment, DDI) are expected, but no additional major phase three studies are contemplated beyond the current ones. He also noted increased interest in the MASH asset.

Mayank Mamtani asked about topics of interest for the end-of-Phase 2 meeting for oral VK2735, the relevance of the subcutaneous package, and contemplated dose levels for the next oral study. He also inquired if a CV outcome trial is still considered, and what other commercial studies (e.g., head-to-head) are being thought about for future spend.

Answer

Brian Lian (President and CEO, Viking Therapeutics) stated that the end-of-Phase 2 meeting would discuss overall study design, duration, and safety package, leveraging the subcutaneous safety package for long-term toxicology. He did not disclose specific dose levels for the next oral study. He mentioned that a CV outcome trial is not currently contemplated. Other expected studies for NDA filing include renal impairment, hepatic impairment, and drug-drug interaction studies, with no major additional Phase 3 studies planned.

Mayank Mamtani of B. Riley Securities questioned the rationale for selecting a 17.5mg top dose in the Phase III VANQUISH trial, up from 15mg in Phase II, and the reasoning behind the slower titration schedule. He also asked if a scenario exists where the oral formulation could be developed as a frontline therapy.

Answer

President & CEO Brian Lian explained that the excellent tolerability at 15mg in Phase II suggested room to go higher. The titration was slowed to a four-week cadence to potentially improve tolerability further, aligning with current commercial standards. Regarding the oral formulation, he noted it's premature to map out next steps, but a frontline therapy scenario is possible pending the data.

Mayank Mamtani asked about the targeted cost of goods sold (COGS) under the CordenPharma manufacturing agreement and the objective of the 30mg maintenance dose arm in the VENTURE-Oral study.

Answer

Brian Lian, President and CEO, explained that while specific COGS figures are confidential, he expects margins to be consistent with other peptide products, benefiting from favorable tiered pricing at scale. He described the 30mg maintenance arm as a test to see if stepping down from a higher dose can prevent weight regain, exploring the viability of a low-dose oral maintenance therapy.

Mayank Mamtani asked about the 13-week weight loss expectations for the oral Phase II study, potential cost of goods, and plans to study lean muscle mass preservation. He also sought an update on partnering discussions for the NASH program, VK2809.

Answer

CEO Brian Lian stated it was too early to predict 13-week weight loss or discuss COGS. He confirmed that while DEXA scans for lean muscle mass will be part of Phase III, a specific muscle preservation study is not planned. For VK2809, he acknowledged ongoing partnering discussions but noted the Phase III biopsy requirement remains a complicating factor.

Mayank Mamtani of B. Riley Securities inquired about the most appealing deal structure for the norovirus program based on strategic feedback, sought input on the development plan and correlates of protection, asked about publication plans for avian flu data, and questioned the forward-looking R&D spend given the COVID trial's stop-work order.

Answer

CEO Steven Lo stated Vaxart is agnostic on deal structure for the norovirus program, prioritizing the advancement of the science. Chief Scientific Officer Dr. Sean Tucker explained the Phase 2b trial's key goal is gathering safety data for an end-of-Phase 2 FDA meeting and that the avian flu data will be published once the paper is written. CFO Jeroen Grasman clarified that BARDA will continue to fund the follow-up for the ~5,000 enrolled COVID trial participants and expects to collect over 50% of the original contract value.

Asked about preferred deal structures for the norovirus program, feedback on its development plan, publication and funding plans for the avian flu program, and the forward-looking R&D spending profile, especially concerning BARDA reimbursement for the halted COVID trial.

Answer

The company is agnostic on norovirus partnership structures and is focused on gathering safety data in the next phase. A publication for the avian flu data is planned. Regarding the COVID trial, BARDA will continue to fund the follow-up for the ~5,000 enrolled participants, and Vaxart anticipates collecting over 50% of the original contract value due to fixed costs.

Mayank Mamtani asked if any learnings from the 400-subject sentinel cohort of the COVID-19 trial were informing current discussions with BARDA and how the results from that cohort would be handled. He also inquired about the items reviewed by the DSMB for the Norovirus trial and how the complete data package, including prior generation data, is being assembled for a potential end-of-Phase II FDA meeting.

Answer

Chief Medical Officer Dr. James Cummings stated that for the main COVID-19 trial, the company is focused on reactivating sites and screening participants. Regarding the sentinel cohort, he noted the company is blinded and will engage BARDA on a potential interim analysis, with the 12-month safety follow-up concluding in December. For the Norovirus trial, Dr. Cummings confirmed the Safety Monitoring Committee reviewed safety data and found a clean profile. Founder and Chief Scientific Officer Dr. Sean Tucker added that the study will measure key immune parameters like functional antibodies and fecal IgA, with the hope that new constructs trend better than the old ones.

Inquired about the COVID-19 program, specifically whether learnings from the 400-subject sentinel cohort are informing discussions with BARDA and how those results will be handled. Also asked about the Norovirus program's recent DSMB review and the strategy for engaging regulators with the full data package for an end-of-Phase II meeting.

Answer

Regarding the COVID-19 sentinel cohort, the company is blinded and will discuss a potential interim analysis with BARDA; the study concludes in December. For the Norovirus program, the DSMB review confirmed a clean safety profile. The company plans to use the totality of data, including key immune parameters from the new study, to engage with regulators, hoping to show the new constructs are superior.

The analyst inquired about the specifics of the COVID-19 trial, including the DSMB safety review, management of the study blind, expectations for mucosal immunity endpoints, and clarification on the interim analysis. He also asked about the next-generation norovirus constructs, including their design, potential data publication, and IND filing timeline.

Answer

The company expects a favorable DSMB review with no safety concerns. They are collecting various samples to compare mucosal and systemic immune induction between their vaccine and the mRNA comparator. The interim analysis is planned after 255 cases, but the focus remains on the full 12-month data. For the next-gen norovirus constructs, preclinical data shows increased potency, but no guidance on an IND filing timeline was provided.

On behalf of Mayank Mamtani from B. Riley Securities, a representative asked if Vaxart's norovirus vaccine is expected to be more robust against emerging strains, and requested commentary on the recent trial failure by HilleVax and Vaxart's competitive positioning.

Answer

Dr. Sean Tucker, Founder and Chief Scientific Officer, responded that the HilleVax data underscores the potential importance of a mucosal immune response, which Vaxart's oral vaccine generates in addition to a serum response. He noted that Vaxart's focus on healthy adults differs from HilleVax's infant study. Dr. Tucker also stated that mucosal IgA responses have shown greater cross-reactivity, which could provide better protection against new norovirus strains.

Mayank Mamtani of B. Riley Securities inquired about the timeline for final VERSAL-2 data, its impact on VERSAL-3 enrollment, the competitive landscape for HPV16-positive head and neck cancer trials, and progress on the Mayo Clinic neoadjuvant study.

Answer

CEO Frank Bedu-Addo stated that final VERSAL-2 data is expected for publication by late 2025 or early 2026. CMO Dr. Kirk Shepard confirmed that VERSAL-3 enrollment is on schedule, driven by strong Phase 2 results and tolerability. Dr. Shepard also noted a favorable competitive landscape with few trials targeting the specific HPV16-positive population and highlighted that the KEYNOTE-689 trial is not comparable to their patient group.

Asked about the timeline for final data from the VERSAL-two trial, its impact on enrollment for the VERSAL-three trial, the competitive landscape, and an update on the Mayo Clinic neoadjuvant study.

Answer

The company expects to publish final VERSAL-two data by late 2025 or early 2026, which is helping drive strong enrollment for the VERSAL-three trial. They see limited direct competition from other trials. The Mayo Clinic study showed promising early activity, and they clarified that the KEYNOTE-689 trial is not applicable to their target HPV16-positive patient population.

Mayank Mamtani inquired about the potential impact of the recent KEYNOTE-689 trial data on patient enrollment for PDSB's VERSATILE-003 trial, particularly for the HPV16-positive subset. He also asked what new information on treatment durability could be expected from the upcoming ASCO poster presentation and how it might compare to emerging EGFR targeted therapies.

Answer

Chief Medical Officer Kirk Shepard and Executive Frank Bedu-Addo clarified that the KEYNOTE-689 trial should have a negligible impact on VERSATILE-003 enrollment, as it primarily involved HPV-negative patients eligible for surgery, a small fraction of the HPV-positive population. Regarding the ASCO data, Frank Bedu-Addo stated that PDS Biotech will present an updated data cut on the significant durability and survival benefits observed in the VERSATILE-002 trial, which has shown a median overall survival of 30 months versus the 12-month standard of care.

The analyst, speaking on behalf of Mayank Mamtani, asked about future plans for a Phase III trial for IMMUNOCERV in cervical cancer and how PDS Biotech positions its head and neck cancer therapy against Merus's EGFR bi-specific drug.

Answer

Regarding Merus, PDS Biotech differentiates itself by focusing specifically on the HPV16-positive population and emphasizing that their own data shows deep, durable responses and a strong survival signal, which is the FDA's key endpoint, unlike objective response rate. For IMMUNOCERV, the company is highly encouraged by the Phase II data, which compares favorably to the standard of care. They are currently consulting with an advisory board of experts to determine the next steps for its development in cervical cancer and will provide an update later.

Speaking on behalf of Mayank Mamtani from B. Riley Securities, an associate asked about potential Phase III plans for IMMUNOCERV following promising data presented at ASTRO. They also inquired how PDS Biotech positions its therapy against Merus' EGFR bi-specific, which has shown a high overall response rate in first-line head and neck cancer.

Answer

Executive Frank Bedu-Addo differentiated PDS's HPV16-specific immunotherapy from Merus's approach, emphasizing that the FDA prioritizes median overall survival (mOS) over objective response rates (ORR) in this indication. He highlighted PDS's superior data on deep tumor regression and durability from a larger patient cohort with longer follow-up. Executive Kirk Shepard added that Merus's data is primarily in HPV-negative patients and lacks survival data. Regarding IMMUNOCERV, Frank Bedu-Addo confirmed the encouraging survival data and stated the company is actively consulting with key opinion leaders to define the next steps for its development path.

Inquired about the strategic positioning of the doublet vs. triple combination therapies, details of the VERSATILE-003 trial protocol and statistical plan, the implications of recent biomarker data on trial design, and the status of strategic collaboration discussions.

Answer

The company stated that the doublet in ICI-naive patients offers the most straightforward regulatory path, while the triple combination in ICI-resistant patients provides the quickest path to approval in that setting. The VERSATILE-003 trial is statistically overpowered as a risk mitigation strategy, but specific design details are not public. Recent data has clarified the distinct roles of PDS0101 (survival) and PDS-01ADC (ORR), which has been beneficial for partnership discussions. The company is evaluating suggestions from potential partners.

Asked for clarification on reconciling Amrozi's prescription volume with reported sales, considering channel stocking and payer rebates. Also inquired about year-end market share goals, early refill and adherence trends, and a comparison to the historical launch of Oracea.

Answer

The company explained that the relationship between prescription volume and revenue is complex due to channel stocking in Q1 and the evolving mix of co-pay assistance versus managed care reimbursement, which takes time to stabilize. They are not providing specific year-end market share goals or refill rate metrics yet. A comparison to the Oracea launch from 20 years ago is not considered a strong analog due to the vastly different payer environment today.

Mayank Mamtani of B. Riley Securities sought to reconcile Amrozi's strong prescription volume with reported sales, asking about channel stocking and payer rebating dynamics. He also inquired about year-end market share goals and any early data on patient refill and adherence trends.

Answer

CEO Claude Maraoui explained that Q1 included channel stocking and that the dynamic between co-pay assistance and managed care reimbursement is still evolving. COO Ramsey Alloush added that strong demand is aiding payer negotiations, but it takes time for coverage to convert to revenue. Regarding market share, Claude Maraoui noted the 10% NRx share among target dermatologists is a fantastic start and that refill uptake is strong and growing, but declined to provide specific future goals or ratios, stating the launch is still very new.

Mayank Mamtani of B. Riley FBR asked a series of questions regarding QBREXZA's commercial performance, the clinical assumptions for the DFD-29 trials, and the company's timeline for providing financial guidance.

Answer

Claude Maraoui, Co-Founder President and CEO, detailed that a digital marketing campaign for QBREXZA drove a 25% sequential increase in prescriptions, with new prescriptions rising to 5,400 in June. Dr. Srinivas Sidgiddi, VP of Clinical Development, explained that DFD-29's Phase 3 trial assumptions are based on Phase 2 data showing 66% IGA treatment success versus 33% for Oracea. Finally, both Maraoui and CFO Ernest De Paolantonio indicated that financial guidance could be expected in the next two quarters as the new product portfolio normalizes.

Mayank Mamtani sought confirmation that VEVYE's Q2 Average Selling Price (ASP) has reset to a new sustainable level and asked about the end-of-quarter stocking dynamics for IHEZO compared to Q4 2024.

Answer

CEO Mark Baum confirmed they are comfortable with VEVYE's current ASP levels and see a bias for improvement, noting the current average is well ahead of pre-launch plans. He stated there were no significant stocking dynamics for IHEZO or other products in Q2, and highlighted that IHEZO adoption is accelerating in retina practices, with Q3 new account starts already surpassing the entire second quarter.

Mayank Mamtani questioned the IHEEZO revenue decline despite stable unit demand, asking about account dynamics. For VEVYE, he asked about the progress of transitioning Klarity-C patients and its market share. He also requested an update on debt refinancing.

Answer

CEO Mark L. Baum attributed IHEEZO's revenue variance to ASP management for buy-and-bill products but noted positive demand trends from retina accounts. For VEVYE, he expressed high confidence in winning the cyclosporine market due to its superior clinical profile and competitive pricing, without giving specific transition numbers. CFO Andrew Boll confirmed that Harrow is in active, positive discussions with lenders and expects to complete a debt refinancing by late summer or early fall 2025.

Mayank Mamtani asked about Q1 trends versus seasonality, the net price and profitability impact of the 'VEVYE Access For All' program, and for an update on the company's 5-year plan in light of its debt maturity and acquisition strategy.

Answer

CEO Mark L. Baum suggested viewing the business in two halves, with Q1 typically being lighter due to deductible resets. He boldly predicted the VEVYE access program would increase both unit volume and average selling price (ASP) within six months. CFO Andrew Boll stated the company has great options to refinance its debt. Baum confirmed the 5-year plan is intact without needing acquisitions, though the company remains open to intelligent, value-creating deals like Melt.

Mayank Mamtani questioned the strategy for improving VEVYE's gross-to-net margin and asked about potential DTC advertising. He also inquired about the procedural overlap between IHEEZO and TRIESENCE, the expected net price for TRIESENCE, and the potential value creation from the upcoming MELT-300 data.

Answer

CEO Mark Baum stated Harrow will not pursue DTC advertising for VEVYE but will improve gross-to-net through payer contracting, driven by strong clinical data, with the goal of meaningfully improving ASP. CFO Andrew Boll added the focus is shifting from volume to margin improvement. Baum noted IHEEZO and TRIESENCE are bundled for GPOs but declined to comment on TRIESENCE's net price. Regarding MELT-300, he said positive data, expected next week, could create a nearly $100 million annual revenue opportunity for Harrow.

Mayank Mamtani of B. Riley Securities asked about the baseline EASI scores of enrolled patients, screen failure rates, the method for measuring skin degradation, and any learnings from the 4-month toxicology studies.

Answer

CEO Nello Mainolfi and CMO Jared Gollob stated the entry criterion is an EASI score of 16 or higher but did not comment on the baseline of enrolled patients or screen failure rates. They confirmed skin degradation is measured by mass spectrometry on biopsies. From the toxicology studies, Nello Mainolfi confirmed that STAT6 degradation was complete and sustained throughout the study period.

Mayank Mamtani of B. Riley Securities posed a multi-part question regarding the Phase III plan, asking about the expected duration of the induction efficacy endpoint, the strategy to demonstrate a remittive effect, and for an update on the Lilly litigation.

Answer

Chief Research & Development Officer Jonathan Zalevsky explained that the induction duration for Phase III will be finalized after the FDA meeting, noting that responses may deepen beyond 16 weeks. For remission, he highlighted the 52-week off-drug follow-up period in the current Phase IIb study, which is designed to assess durability. President & CEO Howard Robin addressed the litigation, stating Nektar is highly committed to pursuing the lawsuit against Lilly and believes the strong Respag data reinforces their position and the damages incurred.

Mayank Mamtani of B. Riley Securities asked about the target baseline EASI score for the REZOLVE-AD study, the importance of the 16-week induction period, the alopecia study's patient severity and response kinetics, and for an update on the Lilly litigation.

Answer

Executive Jonathan Zalevsky said they aim for a baseline EASI of 25-30 and noted the 16-week induction provides more time for efficacy to emerge. For alopecia, he explained response kinetics are slower due to hair growth cycles. Executive Howard W. Robin commented on the litigation, stating they are aggressively pursuing the case and that REZPEG's outcome does not impact the damages claim.

Mayank Mamtani of B. Riley Securities asked about the screen failure rate from the trial's stringent criteria, the structure of the protocol's escape arm, and the potential translational read-through from the atopic dermatitis data to the alopecia areata study.

Answer

Chief Research and Development Officer Dr. Jonathan Zalevsky deferred commenting on screen failure rates until data presentation. He explained that patients not achieving an EASI-50 response at 16 weeks can enter an escape arm to receive the highest dose of REZPEG. He noted that alopecia areata was selected as a key dermatological indication with a strong biological rationale for Treg modulation, and that correlative biomarkers will be analyzed across both studies to understand the drug's activity.

On behalf of Mayank Mamtani from B. Riley Securities, an analyst inquired about the potential for ZW191 data at upcoming fall conferences like ESMO and asked for an estimate of how many dose escalation cohorts would be completed by that time.

Answer

CEO Kenneth Galbraith reiterated that while the company is eager to share initial ZW191 data, it will only do so at a peer-reviewed medical meeting once an abstract is accepted. He declined to provide a specific timeline or commit to a particular conference, stating a data release is possible in 2025 or early 2026.

Mayank Mamtani from B. Riley Securities asked about the dose levels in the two ongoing Phase 1 studies, how dose-finding work informs the company's IP strategy, and whether FVC data from the CORAL trial would be presented at CHEST.

Answer

CEO Jennifer Good confirmed that FVC data will not be presented, as the study was too short for it to be meaningful, and that all study data is reviewed for new IP opportunities. Chief Development Officer James Cassella specified that the TITLE study is a dose-escalation trial, while the DDI study uses a single strength of Haduvio.

Mayank Mamtani asked about plans for further dose exploration for RCC, the potential trial design for a broader interstitial lung disease (ILD) study, and details regarding the recent FDA Type C meeting.

Answer

Executive Jennifer Good stated that decisions on RCC dose exploration will follow the IPF data readout. Chief Development Officer Dr. James Cassella and Chief Commercial Officer Farrell Simon indicated a future ILD trial would likely use a crossover design and target a broad population with both ILD and chronic cough. Regarding the FDA, management confirmed the Type C meeting was a technical query on validating the cough count endpoint, and the agency provided clear, timely guidance.

Mayank Mamtani from B. Riley Securities inquired about the placebo response expectations for the IPF chronic cough study, the nature of the two-week run-in period, and how baseline cough counts might differ from the prior CANAL study. He also asked about the potential market positioning of Haduvio versus P2X3 antagonists for RCC.

Answer

Executive Jennifer Good clarified the two-week period is for dose titration, not a placebo run-in, and noted the study's powering assumption of a 30% placebo effect is conservative compared to the 15-23% seen in prior IPF cough studies. For market positioning, she explained Haduvio would target RCC patients who have failed other therapies, including potential P2X3 antagonists, thereby addressing a significant unmet need.

Mayank Mamtani from B. Riley Securities asked if comparable CD4 T-cell superiority data could be expected from Sanofi's study and questioned the importance of B-strain immunogenicity. He also inquired about the process of converting Material Transfer Agreements (MTAs) into financial transactions and Novavax's understanding of the evolving BARDA framework.

Answer

EVP, Head of Research & Development Ruxandra Draghia-Akli stated she could not comment on Sanofi's data but noted their own T-cell data is encouraging, while strain-specific performance is variable year-to-year. President, CEO & Director John Jacobs explained that MTAs allow potential partners to validate Novavax's technology, which can lead to licensing deals. EVP, Chief Corporate Affairs & Advocacy Officer Silvia Taylor added that Novavax continues to engage with BARDA on potential funding for its pandemic influenza asset, emphasizing the value of its technology platform.

Mayank Mamtani from B. Riley Securities asked about the regulatory weight of real-world quality-of-life data, the potential design of a pivotal trial for the CIC/Flu vaccine, the timeline for IND filings for early-stage programs, and whether potential new partnerships are included in financial guidance.

Answer

Head of R&D Dr. Ruxandra Draghia-Akli noted that real-world data like the SHIELD study primarily informs consumers, as regulators focus on controlled trial data. CEO John Jacobs added that any pivotal trial for CIC/Flu would be designed with a future partner. CFO James Kelly confirmed the current revenue framework excludes any yet-to-be-signed partnership agreements.

Mayank Mamtani asked for an update on BLA-related interactions with the FDA, plans for the CDC ACIP meeting, visibility on H5N1 pandemic preparedness funding, and a preview of the second-half 2025 R&D Day.

Answer

President and COO John Trizzino described FDA interactions as 'positive and productive' ahead of the April PDUFA date. Head of R&D Ruxandra Draghia-Akli highlighted positive non-human primate data for its H5N1 vaccine. President and CEO John Jacobs added that the company is seeking government funding for H5N1 and that it's too early to preview the R&D day, but the goal is a comprehensive update on all new programs.

Mayank Mamtani from B. Riley Securities asked about the expected trends for net pricing and distribution channels once Sanofi takes over commercialization in 2025. He also inquired about the company's steady-state operating expense structure for 2026-2027 and sought clarity on the next IND filing and any insight into Sanofi's pipeline prioritization.

Answer

President and COO John Trizzino expressed confidence that Sanofi's global marketing strength would have a 'dramatic impact' on demand and confirmed Novavax's strategy is to partner late-stage assets rather than build a commercial team. CEO John Jacobs highlighted that approximately $30 million in quarterly commercial spend will be eliminated, supporting cost reduction goals. Chief Medical Officer Dr. Robert Walker identified the RSV combination and pandemic influenza programs as the next candidates moving toward IND-enabling activities.

Mayank Mamtani of B. Riley Securities requested more detail on the claims of the newly issued patent extending to 2045, specifically its basis in weight-based dosing, and asked about plans to further build IP with emerging F4C outcomes data.

Answer

CEO Bill Sibold clarified that the strong new patent is based on the clear finding from the MAESTRO-NASH trial that weight-based dosing optimizes ResDiffera's efficacy and safety, a regimen adopted in the FDA label. He confirmed that 2045 is now the company's base case for all strategic planning. While they will look for future IP opportunities with the F4C indication, it is premature to detail those plans.

Mayank Mamtani asked for commentary on patient segmentation in light of new semaglutide data and the implications of upcoming F4 data for European pricing and launch strategy.

Answer

CEO William Sibold stated the new competitor data doesn't change their view, citing real-world adherence challenges for GLP-1s. He noted Rezdiffra is used across F2 and F3 stages and aims to be the foundational therapy from F2 to F4c. For Europe, he believes Rezdiffra's innovation will be recognized in pricing and that the two drug classes can coexist, as many patients still have MASH despite prior GLP-1 exposure.

Mayank Mamtani asked about plans to generate real-world evidence for Revuforge as a post-transplant maintenance therapy and the expected duration in that setting. He also inquired about development milestones for moving Nyktymbo into earlier lines of GVHD.

Answer

CMO Dr. Nick Botwood confirmed that preliminary real-world data, including post-transplant use, will be presented later this year. CEO Michael Metzger reiterated that physicians anticipate a maintenance duration of one to two years. For Nyktymbo, Dr. Botwood highlighted ongoing trials in earlier-line GVHD, including a Phase 3 combo with steroids and a Phase 2 with ruxolitinib, as key to future expansion.

Mayank Mamtani asked about the company's assumptions for the overall IV anti-CD20 market trends and new patient starts for competitors. He also inquired about how upcoming competitor data in CD19 CAR-T for MS might influence TG's development strategy for its own azacel program.

Answer

Chief Commercial Officer Adam Waldman stated that BRIUMVI continues to see strong growth in new patient enrollments with limited impact from competitors, and that the IV market has stabilized at around 60% of the total CD20 market. CEO Michael Weiss added that upcoming competitor CAR-T data is expected to be very early and is unlikely to alter their well-defined development plan for azacel.

Mayank Mamtani asked a series of questions regarding physician feedback on the 30-minute infusion, the scope of upcoming Phase III trials, drivers of R&D and SG&A spend, future profitability, and the status of the North Carolina manufacturing plant.

Answer

CEO Michael Weiss and CCO Adam Waldman responded. Waldman noted positive feedback on the 30-minute infusion's convenience. Weiss clarified plans for two pivotal trials (simplified dosing and 30-min infusion), explained that higher R&D spend was due to subcu manufacturing materials, and stated the focus is not on profitability targets this year. He also confirmed the North Carolina plant is a long-term, multi-year project for manufacturing backup.

Mayank Mamtani asked for the revenue split between new and maintenance BRIUMVI patients, details on dynamic market share, and an update on the size and objectives of the allogeneic CD19 (azer-cel) study.

Answer

Chief Commercialization Officer Adam Waldman did not provide a specific revenue split but expects maintenance prescriptions to become the majority in early 2025, and noted BRIUMVI is capturing about 1 in 4 new IV anti-CD20 patients. CEO Michael Weiss stated the azer-cel study is ready to enroll and is focused on dose-finding and establishing the right conditioning regimen, with the ultimate goal of slowing or stopping MS progression.

Mayank Mamtani from B. Riley Securities asked about the revenue split between new and maintenance patients, market share in newly diagnosed patients, the size of the intra-CD20 switch market, and the long-term supply goals for the dual-facility manufacturing setup.

Answer

Chief Commercialization Officer Adam Waldman noted that new patients still drive most revenue but expects repeat prescriptions to become the majority in the next few quarters. He confirmed a sizable portion of business comes from OCREVUS and KESIMPTA switches. CEO Michael Weiss stated that long-term supply goals between the two manufacturing sites are not yet defined but are being secured for long-term needs.

Mayank Mamtani of B. Riley Securities asked about the expected placebo response range, baseline demographics, which secondary endpoints will be in the top-line release, and pemvidutide's potential impact on bone and muscle health.

Answer

An Altimmune executive projected a placebo response for fibrosis between 7% and 13% due to their biopsy rereading method but deferred on specific demographics until final data. The top-line release will include weight loss, liver fat reduction, NITs like FibroScan and ELF, and safety data. The executive also highlighted pemvidutide's class-leading lean mass preservation (21.9% of weight loss) as a key differentiator against other metabolic drugs.

William, on behalf of Mayank Mamtani from B. Riley Securities, asked what specific details Altimmune is looking for in the full ESSENCE trial data presentation and how discussions with potential strategic partners have evolved recently.

Answer

CMO Dr. Scott Harris responded that while they will review the full ESSENCE data, the modest top-line results were predictable and reinforce their confidence in pemvidutide's potential for a superior profile. CBO Ray Jordt noted that partners are still defining their obesity strategies, and Altimmune's continued data generation on differentiating factors like lean mass preservation and MASH efficacy strengthens their position. CEO Dr. Vipin Garg added that the recent FDA alignment provides crucial clarity for these ongoing discussions.

Mayank Mamtani from B. Riley Securities asked for learnings from the obesity study's SAD data, whether MAD and combo cohorts are parallel, and which milestones are in the 2028 cash runway guidance.

Answer

An executive confirmed the multi-dose cohorts are enrolling in parallel and deferred sharing SAD data until later in the year. Outgoing CFO Ken Myszkowski stated that the cash runway guidance into 2028 includes near-term milestones expected from Sarepta and other milestones deemed probable, without providing specifics.

Mayank Mamtani sought clarification on the timing of data disclosures for the Sarepta-partnered programs, ARO-DM1 and ARO-DUX4. He also asked for a comparison of the target patient population for zodasiran in HoFH versus evinacumab.

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An executive stated that they could not provide guidance on data readouts for the Sarepta-partnered programs, as the timing of disclosure is Sarepta's decision. Regarding zodasiran, another executive explained the target HoFH population is very similar to evinacumab's but highlighted potential advantages for zodasiran, including quarterly subcutaneous dosing versus monthly IV infusion and a different safety profile.

An analyst on behalf of Mayank Mamtani asked about key factors being monitored in the neuromuscular landscape that could influence ARO-DM1 and DUX4 development, the clinical timeline for dimer candidates, and the timeline for the adipose tissue targeting program.

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Dr. James Hamilton, Chief of Discovery and Translational Medicine, stated they follow all competitor programs closely. President and CEO Dr. Christopher Anzalone added that they aim to be best-in-class by learning from others' regulatory paths. He expects the first dimer (PCSK9/APOC3) in the clinic next year and guided for a CTA filing for the first adipose-targeted candidate this year, with more details at the August 14 webinar.

Mayank Mamtani asked about potential development plan changes following recent AACR data, the focus of the upcoming Type B FDA meeting, its timing, its interdependence with strategic discussions, and for an update on European regulatory engagement.

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Dr. Richard Goldberg, Chief Development Officer, confirmed the immediate focus remains on colorectal cancer (CRC) as the shortest path to approval. Dr. Jennifer Buell, Chairman of the Executive Board, clarified that the strategic discussions are not dependent on the FDA meeting outcome. Dr. Buell also disclosed that previous interactions with Europe's CHMP were 'incredibly productive,' with agreement on key trial design elements, and that a conditional approval pathway in Europe is a possibility being pursued.

Mayank Mamtani asked for an update on the monetization process for noncore assets and their potential economics. He also sought clarity on the company's latest thinking for a BOT/BAL registrational program across late-line, frontline, and neoadjuvant settings.

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Chairman and CEO Garo Armen confirmed active discussions for monetizing the West Coast manufacturing facility, building on a prior mortgage. For the BOT/BAL program, Garo Armen, CMO Steven O’Day, and CCO Robin Taylor collectively detailed the strategy. They highlighted strong, durable responses in late-stage MSS CRC, 'unambiguous' complete pathological responses in the neoadjuvant setting, and the potential for organ-sparing outcomes in rectal cancer, which is a primary regulatory target.

Mayank Mamtani of B. Riley Securities asked about the confidence in the submitted REMS being accepted, what to expect after the June late-cycle meeting, and the potential value of an Adcom.

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Robert I. Blum, President and CEO, noted that REMS negotiations are iterative, which is likely why the FDA designated the submission a major amendment, allowing sufficient time for discussion. He confirmed the late-cycle meeting is expected in June and dismissed the idea of an Adcom, as the FDA has not planned one and such meetings rarely focus on REMS implementation.

Mayank Mamtani from B. Riley Securities asked about the CK-586 study in HFpEF, inquiring if the high-dose cohort is contingent on results from the first two, and what the company aims to learn about tolerability and biomarkers.

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SVP & CMO Stuart Kupfer confirmed the study has a flexible design, and the decision to proceed with the third cohort will be based on data from the first two, which are expected to complete enrollment by year-end. He stated the trial's primary focus is on safety and tolerability, but it will also collect data on cardiac biomarkers like NT-proBNP, pharmacokinetics, and ejection fraction to inform future development in this dose-finding study.

Mayank Mamtani of B. Riley Securities inquired about the CK-586 Phase II trial, asking about expectations for functional improvement and how the drug compares to GLP-1s in the HFpEF space.

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Fady Malik, EVP of R&D, and Stuart Kupfer, SVP & CMO, responded. They noted the 12-week trial is sufficient for dose-finding and observing signals, similar to the nHCM cohort in REDWOOD. They positioned CK-586 as complementary to GLP-1s, which target obesity-related HFpEF, whereas CK-586 is aimed at a hypercontractile phenotype that may not be obesity-driven.

Mayank Mamtani asked about the expected discontinuation rate in the LORIKEET study, what learnings informed the program, the progress of the MGC028 Phase 1 study, and if any tumor-type enrichment is being implemented.

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Dr. Stephen Eck, SVP, Clinical Development and Chief Medical Officer, said they anticipate a low discontinuation rate for lorigerlimab due to its good tolerability. He noted the MGC028 study just began and that patients are not being preselected for ADAM9 expression. Dr. Scott Koenig, President and CEO, added that the decision to study ovarian cancer was based on the totality of prior data, including a response in a similar tumor type in a Phase 1 study. He also clarified that MGC028 enrollment is initially limited to tumors with known ADAM9 upregulation, such as pancreatic, lung, and cholangiocarcinoma.

Mayank Mamtani from B. Riley Securities asked several questions, including whether MacroGenics might shift its strategic focus away from prostate cancer, when to expect specific data for MGC026, learnings from TAMARACK regarding B7-H3 expression, and baseline characteristics for the LORIKEET trial.

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Dr. Scott Koenig, President and CEO, affirmed the company's broad focus on solid tumors, noting the prostate cancer programs were driven by unmet need. For MGC026, he said the Phase I study includes mini-expansions to explore various tumor types, with data expected in 2025. He also confirmed that LORIKEET's patient population is characteristic of second-line mCRPC patients and that OS data would likely be too immature to report in 2025.

Asked about expectations for the upcoming rPFS data, how the drug might be positioned relative to PSMA and STEAP1-targeted therapies in a future Phase III trial, and for clarification on the number of Pluvicto-exposed patients in the current study.

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The executive expressed optimism for achieving a strong rPFS result (baseline expectation of 6+ months), with data expected at a conference in the second half of 2024. He clarified that there were very few Pluvicto-exposed patients in the trial due to the geographic enrollment distribution (mostly Western Europe).

Mayank Mamtani from B. Riley Securities, Inc. asked about expectations for the upcoming rPFS data, the potential positioning of vobra duo relative to PSMA and STEAP1 therapies, and the number of Pluvicto-exposed patients in the study.

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Dr. Scott Koenig, President and CEO, expressed encouragement that the rPFS data, expected in H2 2024, could meet or exceed the baseline expectation of 6+ months. He confirmed that very few patients in the study had prior exposure to Pluvicto, primarily due to the trial's geographic enrollment being concentrated in Western Europe.

Mayank Mamtani inquired about the tau cutoff threshold for the next Alzheimer's trial, its implications for the ongoing early AD study, and any enrollment updates. He also asked for physician feedback on the SHIMMER data for DLB, the publication strategy, and the potential structure of a partnership deal.

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Executive Anthony Caggiano confirmed the company plans to enrich for lower tau in the next study, similar to the SHINE trial, but an exact level is not yet determined. He clarified the early AD study does not have a specific tau cut. CEO Lisa Ricciardi stated that while there is significant interest from potential partners for one or both indications, no deal is finalized. She added that feedback from KOLs and payers has been consistently strong. Anthony Caggiano noted the SHIMMER data publication is underway.

Asked about the SHINE study's expected ARIA rate and patient severity split, key biomarkers to watch, investigator feedback and enrollment for the MAGNIFY study, and how grant funding impacts the cash runway guidance.

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The company does not expect ARIA in the SHINE study and will analyze results by patient severity. Key biomarkers will be similar to the preliminary read. The MAGNIFY study has strong investigator interest but no enrollment updates were given. Grant funding is drawn down as studies progress, impacting the runway accordingly.

Mayank Mamtani of B. Riley Securities inquired about the manufacturing processes of competing RPE programs, asking for comparisons on scale, cell source, and dosage. He also asked for potential insights into the design of a future controlled study for OpRegen, including patient exposure and efficacy endpoints.

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CEO Brian Culley noted that while he lacks specific details on competitors' manufacturing, Lineage's own process is highly advanced. He hypothesized that competitors may be achieving strong results by applying learnings from Lineage's public data. Regarding a future trial, Culley stated that Genentech could decide to launch a controlled study at any time, as the ongoing GAlette study is focused on surgical optimization and not necessarily a prerequisite for advancing the program.

Asked about the functional endpoints in the Phase II CALLIPER study, the relationship between brain volume changes and NfL, and the potential scenarios for the Phase III ENSURE sample size reassessment, including whether the program could be expedited.

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The company detailed the functional endpoints for CALLIPER, including EDSS, the 25-foot walk test, and cognition tests. They noted that brain volume change is a robust measure expected to correlate with disability and NfL. For the ENSURE futility analysis, it was clarified that the FDA does not permit acceleration for positive signals; the possible outcomes are to proceed as planned, adjust the sample size if viable, or stop the study.

Mayank Mamtani from B. Riley Securities asked about the functional endpoints in the Phase II CALLIPER study and their potential link to vidofludimus's dual mechanism. He also inquired about the relationship between brain volume changes and NfL, and the possible scenarios for the Phase III ENSURE sample size reassessment.

Answer

CEO Dr. Daniel Vitt detailed that beyond the key secondary endpoint of confirmed disability worsening (EDSS), the CALLIPER study measures functional outcomes like the 25-foot walk test, cognition, and the nine-hole peg test. He noted brain volume change is a robust long-term measure of neurodegeneration expected to correlate with disability and NfL. For the ENSURE futility analysis, he reiterated that the FDA prohibits stopping for early efficacy, limiting outcomes to proceeding as planned, a viable sample size increase, or stopping for futility.