Yale Jen

Yale Jen of Laidlaw & Co. followed up on the EBV vaccine, asking if Merck would provide the first data readout and if a definitive go/no-go decision had been made. He also inquired about the current status of the GLP-1 glucagon combo assets (oxyntomodulin) in collaboration with Entera Bio.

Answer

Dr. Phillip Frost, Chairman and CEO, stated that the decision and announcement regarding the EBV vaccine data readout and go/no-go decision rests with Merck, though initial data is encouraging. Dr. Elias Zerhouni, Vice Chairman and President, confirmed that the oxyntomodulin program is in the very late stages of IND submission, with an oral formulation being pursued with Entera and an injectable ready for Phase 1 after IND clearance.

Yale Jen followed up on the EBV vaccine, asking if Merck would provide the first data readout and if Merck had made a final go/no-go decision, or if the current studies were merely supplementary. He also inquired about the GLP-1 glucagon combo assets in collaboration with Entera Bio, requesting information on its current status.

Answer

Phillip Frost, Chairman and CEO, OPKO Health, stated that the decision and announcement regarding the EBV vaccine data readout and go/no-go decision rests with Merck, though OPKO is encouraged by the data and aims for a seamless transition to Phase 2 and beyond. Elias Zerhouni, Vice Chairman and President, OPKO Health, confirmed that the GLP-1 glucagon combo, referred to as oxyntomodulin, is in the very late stages of IND submission, with promising December results for the Entera formulation and preparations for an injectable Phase 1 trial.

Yale Jen of Laidlaw & Company asked for the estimated patient size for the Phase 1 study targeting both obesity and NASH. He also sought clarification on the differences between the GLP-1/glucagon agonist (OPK-88006) and the GLP-2 candidate for short bowel syndrome, as well as the clinical timeline for the latter.

Answer

President & Vice Chairman Dr. Elias Zerhouni estimated the Phase 1 study would enroll between 100 and 170 patients with biomarker evidence of F2-F4 stage NASH. He clarified that the GLP-2 agonist is a completely different molecule targeting intestinal absorption for the distinct unmet need of short bowel syndrome. He stated the short bowel syndrome program is expected to enter clinical studies next year, pending regulatory feedback.

Yale Jen of Laidlaw & Company inquired about the potential impact of government tariffs on operations and R&D, and also asked whether the MASH development program would target early-stage or later-stage fibrosis patients.

Answer

Executive Adam Logal assessed the tariff risk as manageable for the supply chain, while Executive Elias Zerhouni noted no significant impact is expected on R&D. Regarding the MASH program, Zerhouni specified that the focus is on later-stage patients with F3 and early F4 (pre-cirrhotic) NASH, where the unmet medical need is greatest.

Yale Jen of Laidlaw and Company asked a macro-level question regarding potential concerns about the impact of recent government changes on the future prospects for vaccine development and related government funding.

Answer

Executive Elias Zerhouni acknowledged the uncertainty but stated that OPKO has not received any indication of a slowdown from its partner Merck on the EBV vaccine or from BARDA on its antibody programs. He emphasized the continued importance of these programs for public health and pandemic preparedness, expressing no immediate worry about the current funding or program progression.

Yale Jen inquired if referral development would become a greater strategic emphasis going forward to enhance patient flow. He also asked whether existing treatment sites could further increase their patient volume or if they have reached a steady state, and what strategies are in place to deepen patient treatment at these sites. Finally, he asked for a prediction on the potential timing of NCCN guideline updates, specifically if they could occur later in 2026 or early 2027.

Answer

Gerard Michel, CEO, confirmed that referral development is a critical and increasing emphasis, especially as the company targets a broader patient population that may be less informed about treatment options, requiring early engagement with community oncologists. For existing sites, he explained that high-volume centers would need to expand capacity (e.g., book more room time, train new teams), while lower-share hospitals require education to broaden their patient criteria (e.g., treating extrahepatic disease) and adopt combination therapies, supported by CHOPIN data and potential guideline changes. Regarding NCCN guideline updates, Michel noted that while NCCN has off-cycle meetings, changes are primarily physician-initiated by KOLs, not company-driven, and expressed hope but no firm prediction on timing.

Yale Jen inquired if referral development would become a greater strategic emphasis going forward to increase patient flow, given its potential to tap into larger patient sources. He also asked whether existing treatment sites are expected to deepen their patient treatment volume or if they have largely reached a steady state. Finally, he questioned the likelihood and timing of potential NCCN guideline updates, specifically if they could occur later in 2026 or early 2027.

Answer

Gerard Michel, CEO, confirmed that referral development is a critical and increasing emphasis, particularly as Delcath delves deeper into the total addressable market (TAM). He explained the need to educate community oncologists who may not be familiar with liver-directed therapies to ensure early patient identification and referral. Regarding existing sites, Mr. Michel differentiated between high-volume centers that need to expand capacity (e.g., by booking more room time and training new teams) and lower-share hospitals where changing prescribing patterns (e.g., for patients with extrahepatic disease) through education and potential guideline modifications is key. For NCCN guidelines, Mr. Michel noted that while the typical schedule is November, off-cycle meetings are possible. He emphasized that guideline changes are physician-initiated, driven by Key Opinion Leaders (KOLs) based on available data, with Delcath's role being to foster conversation.

Yale Jen of Laidlaw & Company asked for specifics on the data to be presented from the CHOPEN trial at ESMO and sought more color on the reasons for lowering the site activation forecast.

Answer

CMO Vojislav Vukovic confirmed the CHOPEN presentation will include the primary endpoint of one-year progression-free survival, plus safety and secondary efficacy data. GM Kevin Muir and CEO Gerard Michel explained the activation slowdown is due to complexities at large institutions, citing perfusion services as a key hurdle that the team is now addressing earlier in the process.

Yale Jen asked about the number of sites for the CRC trial, the number of HEPZATO units sold, details on a recent price increase, the breakdown of patient referrals, and the meaning behind the consistent quarterly revenue growth.

Answer

CMO Vojo Vukovic stated over 20 sites are planned for the CRC trial. CFO Sandra Pennell detailed a price increase from $182.5k to $187.5k on February 1. General Manager Kevin Muir estimated 30-40% of patients are organic to treatment sites, with the rest referred in. CEO Gerard Michel explained the steady ~$4M quarterly revenue increase is a function of the consistent pace of new center activations, which is typical for this type of product ramp, but declined to extrapolate the trend.

Yale Jen of Laidlaw & Company inquired about the trial design for the upcoming metastatic colorectal cancer (CRC) study, including treatment cycles and sequencing. He also asked about revenue expectations and reimbursement strategy for European markets.

Answer

CMO Vojo Vukovic detailed the CRC trial design: two initial cycles of HEPZATO followed by standard of care, sequenced this way to address the liver-dominant disease first. CEO Gerard Michel projected "modest growth" for Europe in 2025, emphasizing its strategic importance for data generation over near-term revenue. He added that reimbursement efforts involve creating country-specific pharmacoeconomic models.

Yale Jen of Laidlaw & Company asked about the company's progress in engaging medical oncologists to build a referral network and inquired about the clinical data supporting the decision to pursue trials in colorectal and breast cancer.

Answer

CEO Gerard Michel confirmed that the commercial team is actively building a referral network, which is a key priority for success in 2025. He explained the colorectal cancer indication is supported by strong historical data from a similar surgical procedure that showed high response rates with melphalan. The breast cancer indication is based on promising results, including complete responses, observed in about a dozen patients treated with CHEMOSAT in Europe.

Yale Jen asked if acquiring the next wave of VYKAT XR patients would be more difficult or require a different approach, about potential next-generation products, and for further elaboration on DCCR's mechanism of action for Glycogen Storage Disease Type I (GSD I).

Answer

Meredith Manning, Chief Commercial Officer, noted that the company is seeing a broad spectrum of patients, including younger adults, and expects this to continue. Anish Bhatnagar, Chairman and CEO, mentioned internal lifecycle management programs for VYKAT XR to be discussed later in the year. He explained that DCCR suppresses insulin secretion to elevate glucose levels, reducing dependency on cornstarch for GSD I patients, leveraging its precise dosing and favorable side effect profile compared to the parent molecule.

Yale Jen asked if acquiring the 'next wave' of VYKAT XR patients would be more difficult or require a different approach after a strong initial launch, and also inquired about potential next-generation products and the mechanism of action for DCCR in GSD I.

Answer

Meredith Manning, Chief Commercial Officer, noted that the company is seeing a broad spectrum of patients, including younger adults, and expects this trend to continue. Anish Bhatnagar, Chairman and CEO, mentioned internal lifecycle management programs for VYKAT XR, to be discussed later in the year. He explained that DCCR's mechanism in GSD I involves suppressing insulin secretion to elevate glucose levels, reducing dependency on cornstarch and addressing life-threatening hypoglycemia.

Yale Jen from Laidlaw & Company asked for a breakdown of revenue between patients in the titration phase versus the maintenance phase. He also inquired about the payer mix between government and commercial plans.

Answer

CEO Anish Bhatnagar explained that since the vast majority of the 646 start forms were new to the drug, a significant number of patients were in the titration phase during the quarter. He stated it was too early to provide a steady-state payer mix but reiterated that the overall PWS population is split roughly into thirds across commercial, Medicare, and Medicaid.

Yale Jen from Laidlaw & Company asked for a breakdown of revenue between patients in the initial titration phase versus the maintenance phase, and also requested details on the current payer mix between government and commercial.

Answer

CEO Anish Bhatnagar clarified that with only about 60 former US trial patients, the vast majority of the 646 start forms were new patients, many of whom were still in the titration phase during Q2. He stated it was too early to provide a precise payer mix but reiterated the expected population split is roughly one-third each for commercial, Medicare, and Medicaid.

Yale Jen asked about the procedural steps for a new patient to obtain a Vykat XR prescription and sought an update on the company's European commercialization strategy, specifically regarding a potential partnership.

Answer

Executive James MacKaness outlined the process: a physician fills out a start form, which is sent to a specialty pharmacy to initiate patient access. Executive Anish Bhatnagar stated that Soleno is still evaluating both a partnership and a self-commercialization strategy for Europe, with a decision to be made following the planned MAA submission to the EMA this quarter.

Yale Jen asked about the procedural steps for a new patient to obtain a Vykat XR prescription and sought an update on the company's decision regarding a European partnership versus a direct launch.

Answer

CFO James MacKaness explained the process: a physician fills out a start form, which is sent to a specialty pharmacy to initiate patient access. CEO Anish Bhatnagar confirmed that both European commercialization options are still under evaluation, with the immediate priority being the MAA submission to the EMA, which is planned for the current quarter.

Yale Jen followed up on safety, specifically regarding potential discontinuation rates for Palatin's MC4R agonists in Prader-Willi syndrome patients compared to currently approved drugs. He also asked how hyperphagia and weight reduction metrics would be assessed in Phase 1/Phase 2 studies, the timeline and resource allocation for aggressive Prader-Willi syndrome development, and the anticipated role of GLP-1 combination therapy in future product development.

Answer

Carl Spana, President and CEO, stated that Phase 1 studies will provide a good look at tolerability and safety, expecting controllable GI side effects and lower discontinuation rates due to the mechanism and MCR1 avoidance. He explained that Phase 1 MAD in healthy obese patients would show target engagement and expected reductions in food intake and body mass, which should translate to a strong signal in PWS patients, though direct PWS studies are needed for confirmation. Steve Wills, CFO and COO, clarified that initial Phase 1 SAD/MAD studies for both the oral small molecule and long-acting peptide are funded, with data readouts by year-end (oral) and H1 next year (peptide), but Phase 2/3 studies in HO and PWS patients will not start before mid-2027. Spana added that GLP-1 combination therapy is a long-term strategy, especially for severe hyperphagia, to optimize patient therapy, acknowledging that while initial development focuses on monotherapy, combination is likely in the marketplace.

Yale Jen inquired about Palatin's assessment of the safety profile for their MC4R agonists in Prader-Willi syndrome, particularly regarding discontinuation rates compared to currently approved drugs. He also asked how hyperphagia and weight reduction metrics would be assessed in Phase 1/Phase 2 studies, the anticipated timeline for aggressive PWS development, and the role of GLP-1 combination therapy in future product development.

Answer

Carl Spana (President and CEO, Palatin) stated that Phase 1 studies would provide a good look at tolerability and safety, expecting lower GI side effects and discontinuation rates due to MC4R selectivity. He explained that while Phase 1 MAD in healthy obese patients would show target engagement and initial efficacy, definitive hyperphagia control in PWS requires studies in the actual patient population. Steve Wills (CFO and COO, Palatin) clarified that Phase 1 SAD/MAD studies are funded, with data by year-end for oral and H1 next year for peptide, and Phase 2/3 studies in HO and PWS are not expected before mid-2027. Carl Spana added that GLP-1 combination therapy is a long-term strategy for optimized, aggressive therapy in chronic conditions.

Yale Jen asked for more details on the Gen 2 system's extended service intervals, whether this extension is a prerequisite for FDA review, and if Beyond Air's increased cash position would lead to the Phase 1B oncology study starting later in the current calendar year.

Answer

CEO Steve Lisi explained that Gen 1 systems require service every 1,000 hours, while Gen 2 is expected to extend this to at least 3,000 hours, improving hospital convenience and gross margins. He clarified that the extended service interval is not an FDA gating factor, as reliability hurdles have already been met. Regarding the oncology study, Lisi stated no commitment to a timeline, emphasizing the current focus on commercial operations and that Beyond Air would likely not fully fund the study at this stage.

Yale Jen asked for an update on sales performance in the current quarter, the total number of hospitals with LungFit PH installed, and the company's current expectations and priorities for the cardiac surgery and Gen 2 device PMA filings.

Answer

CEO Steve Lisi declined to provide intra-quarter sales figures but reaffirmed full-year guidance to show confidence. He stated that "dozens and dozens" of hospitals are now using LungFit PH. Regarding regulatory filings, Lisi clarified that the company's primary focus is on the second-generation machine, as it is strategically more important at this time than the cardiac indication.

Sought confirmation on the number of new hospital contracts, asked about the turnaround time from contract signing to revenue generation, inquired about the company's long-term debt status, and questioned how new indications would apply to the next-generation device.

Answer

The company confirmed 6 new hospitals and 2 renewals. Most hospitals start generating revenue within 30-60 days of signing a contract, making the turnaround relatively short. The company clarified it has $11 million in debt, but payments (structured as a royalty on sales) do not begin until October 2026. The next-generation device, being a PMA supplement, would automatically inherit any indications approved for the current system.

Asked for clarification on the 60% customer growth metric, the annualized revenue run rate, the international expansion strategy, and the required regulatory approvals for ex-U.S. sales.

Answer

The 60% growth was quarter-over-quarter. The revenue run rate is expected to increase quarterly. International expansion is managed by an intermediary firm, BAC, who works with local distributors. A mix of FDA, CE Mark, MDSAP, and country-specific approvals are needed for global sales.

Yale Jen of Laidlaw & Company sought clarification on the 60% increase in customers, asking for the baseline period. He also asked about the annualized revenue run rate mentioned in the press release and inquired about the strategy for international expansion, including the role of partners and the regulatory requirements in ex-U.S. countries.

Answer

Executive Steven Lisi clarified the 60% increase was a quarter-over-quarter comparison of total customers from June 30 to September 30, 2024. He confirmed the revenue run rate is expected to rise from the current quarter's level. For international strategy, he explained that the firm BAC acts as an intermediary to manage local distributors. He also noted that while FDA and CE Mark approval cover many regions, most countries still require their own licensing process, which can take several months to a year.

Yale Jen asked if a larger-scale maintenance study would be necessary following the current one, and if so, whether it would run concurrently with the oral VK2735 Phase 3 study. He also inquired about potential supply issues once the subcutaneous program fully transitions to autoinjectors.

Answer

Brian Lian, President and CEO, indicated that a larger subsequent maintenance study would likely be required to fully understand long-term maintenance and optimal dosing, potentially as an extension or standalone trial. He confirmed no anticipated supply issues with the autoinjectors, citing the supplier's high production capacity.

Yale Jen asked if a larger-scale maintenance study would be needed following the current one to finalize the path forward, especially while the oral Phase III study is ongoing. He also inquired about potential supply issues with autoinjectors once the transition to them is complete for all subcutaneous dosing.

Answer

Viking Therapeutics' President and CEO, Brian Lian, indicated that a larger subsequent study, either as part of an extension or standalone, would likely be required to fully understand longer-term maintenance and ideal dosing. He does not anticipate any supply issues with the autoinjectors, as the supplier has very high production capacity.

Yale Jen asked if a potential Phase 3 for oral VK2735 would involve an oral-to-oral (high dose to low dose) or subcutaneous-to-oral transition, similar to the maintenance study. He also inquired whether data from VANQUISH-1 and VANQUISH-2 would be reported simultaneously or sequentially, given the enrollment time gap.

Answer

Brian Lian (President and CEO, Viking Therapeutics) clarified that a traditional oral Phase 3 would likely involve titrating up to a level and maintaining it for 52 weeks, not an oral-to-oral or subcutaneous-to-oral transition. Regarding VANQUISH data, he stated that the reporting would depend on the actual time difference in data availability, and historically, companies report data when available, potentially separately if there's a few months' difference, to allow each study its own focus.

Yale Jen asked if a potential phase three for oral VK2735 would consider an oral-to-oral or subcutaneous-to-oral transition, similar to the maintenance study. He also inquired whether data from Vanquish One and Vanquish Two studies would be reported simultaneously or sequentially given the enrollment time gap.

Answer

Brian Lian (President and CEO, Viking Therapeutics) stated that a traditional oral phase three would likely involve titrating up to a level and maintaining it for 52 weeks, not an oral-to-oral or subcutaneous-to-oral transition. Regarding Vanquish data, he couldn't confirm simultaneous or sequential reporting, noting that companies typically report data when available, and separate press releases might be preferred if there's a significant time difference.

Yale Jen from Laidlaw & Company asked about the potential impact of upcoming Phase III data from Lilly's oral GLP-1 on Viking's oral program. He also inquired about the status of the calcitonin receptor aspect of Viking's amylin program.

Answer

President & CEO Brian Lian expressed that the obesity market is large enough to accommodate multiple agents, so they are not overly concerned about a single competitor's data. Regarding the amylin program, he clarified that their compounds are balanced agonists of both the amylin and calcitonin receptors, as their preclinical work showed this balanced approach yielded the best results for weight and food consumption.

Yale Jen asked about the number of clinical sites prepared for the upcoming Phase III study and requested a status update on potential partnerships for the VK0214 and VK2809 programs.

Answer

Brian Lian, President and CEO, did not disclose the specific number of sites for the large Phase III program. Regarding the VK0214 (X-ALD) and VK2809 (NASH) programs, he confirmed both are available for licensing and are attracting interest, but he could not provide details on any ongoing discussions.

Yale Jen asked if the Phase III study in type 2 diabetes patients could support a standalone diabetes indication. He also inquired about preparations for an auto-injector and whether a bridging study would be necessary.

Answer

CEO Brian Lian clarified that the study is intended to support an obesity label for patients with type 2 diabetes, not a separate diabetes treatment indication. He confirmed that Viking plans to introduce an auto-injector during the Phase III program and would likely conduct a bioequivalence bridging study to support it.

Yale Jen asked if there were any surprises in the recent FDA feedback for VK2809 and its potential impact on partnering, and also inquired about how recent competitor data on amylin agonists might influence Viking's own program strategy.

Answer

Brian Lian, an executive, reported that while the company was still reviewing the FDA feedback for VK2809, there were no surprises so far. Regarding the competitive landscape, he stated that exciting data from other amylin programs is viewed positively, as it validates the mechanism and bodes well for Viking's own combination strategy with VK2735.

Inquired about the specifics of Medicare revenue for the quarter, the size of the lead pipeline, the impact of tariffs on the business, and the achievability of the revised 2025 revenue guidance.

Answer

The company clarified that Q2 2024 included a one-time revenue item, and excluding that, Medicare sales grew year-over-year. The lead pipeline in the U.S. exceeds 130 and is growing. The impact from tariffs is immaterial as the ReWalk exoskeleton has an exemption. Management affirmed their expectation for continued ReWalk revenue growth in the second half of the year, supporting the guidance.

Yale Jen of Laidlaw & Company asked for specifics on Q2 Medicare revenue, clarification on the lead pipeline size in the U.S. and Germany, the impact of tariffs, and the achievability of the 2025 guidance for the ReWalk product line.

Answer

CFO Almog Adar clarified that Q2 2024 included a one-time $700,000 Medicare revenue item, and excluding that, Medicare sales grew year-over-year. CEO Mark Grant confirmed the U.S. pipeline has over 130 qualified leads. Adar added that tariffs have an immaterial impact and that the company expects continued ReWalk revenue growth in the second half of 2025.

Yale Jen of Laidlaw & Company inquired about the potential impact of recent tariffs on Lifeward's international business, the company's confidence in its full-year revenue guidance despite a seasonally slow Q1, and the key drivers behind the forecast for a significantly reduced adjusted operating loss of approximately $1 million by Q4 2025.

Answer

CEO Lawrence Jasinski stated that the company's global supply and revenue diversification, with ReWalk produced in Israel and strong markets in the US and Europe, helps mitigate tariff risks. He affirmed confidence in the annual guidance, citing strong growth momentum in the AlterG line (up 17-19% in the last two quarters) and a record ReWalk pipeline. An executive, likely CFO Michael Lawless, detailed the path to a lower Q4 loss, explaining it will be driven by revenue growth towards a breakeven run rate of about $10 million per quarter, combined with the full impact of cost-saving initiatives phasing in throughout the year.

Asked about the composition and conservatism of the 2025 guidance, the impact and terms of the new CorLife and MYOLYN partnerships, and the launch timeline for the next-generation ReWalk system.

Answer

The 2025 guidance reflects balanced growth across all product lines, with a conservative approach that balances top-line growth and spending discipline. The CorLife partnership provides efficient, profitable access to the workers' compensation market without increasing marketing expenses. The expanded MYOLYN agreement opens up the larger home-use market, which is expected to increase revenue without raising marketing costs. The next-generation ReWalk 7.0 is on track for clearance and launch by mid-2025.

Yale Jen of Laidlaw & Company asked about the component growth within the 2025 guidance, the potential impact of the CorLife partnership, the launch timeline for the next-generation ReWalk, and the financial terms of the CorLife and MYOLYN agreements.

Answer

CFO Mike Lawless stated that 2025 guidance reflects balanced growth across all product lines, with a focus on profitability. CEO Larry Jasinski explained the CorLife partnership provides efficient, low-cost access to the workers' compensation market and confirmed the next-gen ReWalk 7.0 is expected to launch by mid-2025. Both executives clarified that the new partnerships are designed to increase revenue and profitability without increasing marketing expenses.

Yale Jen from Laidlaw and Company questioned the rationale for selecting the 15mg dose in the CF trial and asked if additional data for both the CF and OTC programs would be released before regulatory meetings.

Answer

President and CEO Joseph Payne explained that the 5, 10, and 15mg dosing strategy was based on extensive experience from the Phase 1 and 1b studies and was agreed upon with the FDA. He confirmed that it is logical to expect further data releases for both programs as the Phase 2 trials are completed, which for CF is expected by year-end, and for OTC is contingent on the decision regarding the higher dose cohort.

Yale Jen inquired about the competitive positioning of Arcturus's CF program against a rival with a similar data timeline, asked about the target strains for the new 2-dose COVID vaccine, and sought clarification on the KOSTAIVE revenue-sharing structure.

Answer

President and CEO Joseph Payne stated that while near-term competition will focus on FEV data, the long-term focus for a daily therapy will shift to safety and tolerability, an area of strength for their LUNAR platform. He clarified the new vaccine vial application precedes the selection of the updated viral strain. CFO Andrew Sassine confirmed the $28 million in shared profit represents a 3-way split between Arcturus, CSL, and Meiji.

Yale Jen asked about the timing of KOSTAIVE revenue recognition from Japan, whether the recent lower R&D expense trend will continue, and how to think about the future of the ARCT-2303 combination vaccine with QIV.

Answer

CFO Andrew Sassine advised to anticipate KOSTAIVE revenue in the first half of 2025 due to reporting lags and cost offsets. He reiterated cash runway guidance into Q1 2027, implying an annual burn around $100M, rather than providing quarterly R&D guidance. CEO Joseph Payne confirmed that partner CSL will determine all future commercial strategies for combination vaccines.

Yale Jen of Laidlaw and Company asked if a 21-day dosing regimen is still planned for the AML triplet study, how Curis is prioritizing its CLL and AML programs, and for an update on investigator-sponsored solid tumor studies.

Answer

CEO James Dentzer deferred comment on specific AML dosing regimens until data is public but expressed optimism. He emphasized that the company is making disciplined, capital-efficient decisions on pipeline prioritization. He also noted there are no updates on the five ISTs as Curis does not control their timelines.

Yale Jen asked about the start date for the BX004 Phase II trial in cystic fibrosis, the potential for the BX211 program to proceed directly to a pivotal study, the required size of the safety database for phage therapy, and sought clarification on the pro forma cash balance.

Answer

CEO Jonathan Solomon confirmed the BX004 trial is on track for a Q1 2026 data readout with no impediments and strong physician interest. Regarding BX211, he stated there is a chance for a direct-to-pivotal study pending FDA discussions. He also noted they hope for a smaller-than-typical safety database due to the therapy's strong safety profile. Executive Marina Wolfson clarified the $21.2M cash balance already includes the recent $12M financing, with another $12M potentially available from unexercised warrants.

Asked a series of detailed questions covering financial reporting (EPS calculation, R&D spending trends, reverse split share count) and clinical pipeline specifics for both BX004 (FDA meeting status, trial timeline) and BX211 (next steps after 13-week data, definition of a 'good outcome', potential for expanded efficacy at 52 weeks, and ultimate approval endpoints).

Answer

The company confirmed EPS is based on net income, R&D burn will decrease to meet cash runway guidance through Q4 2025, and the reverse split share count was for outstanding shares. For BX004, the FDA meeting was successful with plans unchanged. For BX211, a ~70% ulcer size reduction in the treatment arm versus ~40% in placebo would be considered a good signal at 13 weeks. The 52-week data will focus on amputation trends, which is the conservative pivotal endpoint, though other endpoints are being explored.

Yale Jen of Laidlaw & Company asked if the proposed Phase II study for BX004 might include an extension for longer treatment, and also posed a fundamental question on how BiomX ensures its phage products remain in the functional lytic state rather than the lysogenic state.

Answer

CEO Jonathan Solomon responded that while the Phase IIb trial already has a long two-month duration, a longer follow-up and a potential treatment extension are being discussed, particularly for patients who experience conversion. On the scientific question, he assured that all phages are fully sequenced to confirm they do not contain any lysogenic genes, which is a strict FDA requirement.

Yale Jen followed up on a previous question, asking for confirmation that the data releases for the LUGANO and LUCIA trials would be approximately two months apart.

Answer

President and CEO Dr. Jay Duker confirmed that a data release separation of approximately two months is likely. He stated the plan is to release the top-line data for each trial as it becomes available, reflecting the staggered start times and similar enrollment rates.

Yale Jen of Laidlaw & Company followed up on the data release plan for 2026, asking if the top-line results for the two Phase III trials would be released approximately two months apart.

Answer

President and CEO Dr. Jay Duker confirmed that a roughly two-month separation between the data releases for the LUGANO and LUCIA trials is a likely scenario. He stated that the company plans to release the data for each trial as it becomes available, and the approximate two-month gap is based on the staggered trial start times and similar enrollment patterns.

Yale Jen asked for a comment on EyePoint's first-to-market potential given a competitor's timeline and whether the company had analyzed patient characteristics from earlier studies to predict which patients might not need supplemental treatment.

Answer

President and CEO Dr. Jay Duker reiterated confidence in being first-to-market, noting the critical factor is the last patient in the second trial, LUCIA, which is recruiting rapidly. Chief Medical Officer Ramiro Ribeiro explained that due to the small size of earlier studies, it is difficult to predict supplement-free patients, but it's something they may analyze with the larger Phase III database.

Yale Jen of Laidlaw & Company (UK) Ltd. inquired about the competitive landscape following a competitor's CRL and the discontinuation of a compassionate use program for domperidone. He also asked for an update on the expansion of the Medicaid pharmacy network.

Answer

Executive Matthew D'Onofrio acknowledged it's difficult to quantify the market share gain from the domperidone discontinuation but expects many patients will turn to GIMOTI. Executive Christopher Quesenberry added that this solidifies Evoke's market leadership. Quesenberry also confirmed the pharmacy expansion strategy is working, noting that Medicaid's business contribution grew from approximately 2% to a range of 5-7% during 2024, which helped boost script conversion rates.

Inquired about the potential size of a pivotal study for AFM28 if it pursues an accelerated approval path, and asked for the reason behind AFM28's apparently stronger monotherapy activity compared to AFM13.

Answer

The company estimated that a pivotal trial for accelerated approval would likely require 80 to 100 patients. They attributed the stronger monotherapy signal of AFM28 not to the molecule itself, but to the underlying biology of AML, which is inherently more susceptible to NK cell-mediated killing compared to the Hodgkin lymphoma targeted by AFM13.

Asked about the rollout timeline for the new version 4.0, its availability for new customers, the impact of the business model shift on the product revenue line in financial reporting, and the reason for the continued high number of perpetual deals.

Answer

Version 4.0 will start shipping in mid-December and will be the default for new customers. In financial reporting, the product revenue line includes perpetual, subscription, and cloud revenue; the main driver of change is the volume of perpetual deals. The GE partnership is 100% perpetual for new deals, explaining the persistence of that deal type.

Asked for clarification on the ARR comparison starting from Q1 2022, the differences between cloud and subscription offerings, the expected growth trajectory of cloud, and the future visibility of revenue trends.

Answer

Executives explained that Q1 2022 was the starting point for their subscription model, making it the appropriate baseline for showing the shift to recurring revenue. They clarified that subscription is on-premise software with recurring payments, while cloud is a true SaaS model hosted by iCAD. They anticipate cloud will grow faster than subscription due to its convenience, which will negatively impact short-term GAAP revenue but build a more predictable and stable recurring revenue base (ARR) over time, improving future visibility.

Inquired about the trend of perpetual versus subscription deals, the commercial readiness and adoption hurdles for the new ProFound Cloud platform, and whether historical revenue seasonality still applies to the business.

Answer

The company is pushing for more recurring revenue through both subscription and the new cloud model, and the sales team is incentivized accordingly. The ProFound Cloud platform is commercially available, and the first deal was signed in Q2. Historical seasonality, driven by customer budget cycles, still applies, with business often skewed towards the end of the calendar year.

Yale Jen of Laidlaw & Company inquired about MEI's financial runway, future revenue expectations, and the specific data anticipated from the upcoming voruciclib and ME-344 clinical trial readouts, including biomarker details and the rationale for the ME-344 progression-free survival (PFS) threshold.

Answer

President and CEO David Urso characterized the cash runway guidance of at least 12 months as appropriate, covering current Phase 1 work, and confirmed that revenue from the Kyowa Kirin collaboration is not expected to continue. Chief Medical Officer Dr. Richard Ghalie detailed that the voruciclib readout will include safety, PK, a recommended Phase 2 dose, and biomarker data such as MCL1 and MYC expression. David Urso added that the ME-344 trial's 20% PFS threshold was established with clinicians, but its significance will depend on the specific patient population enrolled.

Yale Jen of Laidlaw & Company asked about the company's cash runway guidance, the potential for future top-line revenue, and the specific data expected from the upcoming voruciclib Phase 1 readout, including key biomarkers. He also questioned the rationale behind the 20% PFS threshold for the ME-344 trial.

Answer

President and CEO David Urso described the cash runway guidance of at least 12 months as appropriate, covering current Phase 1 plans, and confirmed that revenue from the Kyowa Kirin collaboration is not expected to continue. Chief Medical Officer Dr. Richard Ghalie detailed that the voruciclib data will include safety, PK, and biomarker analysis, specifically MCL1 expression and MYC. David Urso added that the 20% PFS threshold for ME-344 was established with clinicians and its significance will depend on the patient population enrolled.

Yale Jen of Laidlaw & Co. asked for guidance on fiscal 2023 revenue, the expected timing for the full TIDAL study data release, and an update on patient enrollment for the COASTAL study.

Answer

President and CEO Dan Gold clarified that quarterly revenue fluctuates based on R&D expense reimbursements from partner Kyowa Kirin as clinical activities ramp up or down. He suggested it is 'reasonable to expect' the final TIDAL data at the upcoming ASH meeting. Regarding COASTAL, Gold declined to give specific numbers but confirmed over 135 sites are open globally, keeping the study on track for completion in 2024.

Yale Jen from Laidlaw & Company asked about the potential regulatory filing timeline following a positive TIDAL readout, the expected start date for the upcoming CLL trial, and when to anticipate an update on the full 120-patient TIDAL cohort.

Answer

Dan Gold, President and CEO, stated that the filing timeline for TIDAL is contingent on pre-NDA discussions with the FDA, which will focus on overall response, safety, and durability. He projected the CLL trial would begin in the first half of the next calendar year, hopefully in Q1, and that a more robust update on the full TIDAL dataset could be presented around mid-year, possibly at ASCO.

Yale Jen of Laidlaw & Company followed up on the marginal zone lymphoma cohort, asking if the trial would evaluate monotherapy or a combination and if the regimen would change. He also asked if the projected data timeline would allow for a presentation at the year-end ASH meeting.

Answer

President and CEO Dan Gold clarified that the planned amendment for the marginal zone arm will evaluate zandelisib as a monotherapy using the same dosing schedule as the main TIDAL study. He added that the subsequent confirmatory study with rituximab is planned to include both follicular and marginal zone lymphoma patients. Regarding a potential presentation at ASH, Gold simply stated, 'Well, we’re certainly trying.'

Yale Jen from Laidlaw & Company inquired about the status of the INVIGORATE-2 study, including its timeline and enrollment, and asked what to anticipate from the upcoming data readout for ADX-2191 in retinitis pigmentosa.

Answer

President and CEO Dr. Todd Brady stated that the INVIGORATE-2 allergen chamber trial has been enrolling during winter seasons to avoid confounding pollen and that enrollment is expected to be complete soon, with data in the first half of the year. For retinitis pigmentosa, he highlighted it as a key milestone for the ADX-2191 franchise, noting it is the largest of the three indications for the drug and that results from the open-label trial are also expected in the first half of the year.

Yale Jen asked for details on the endpoints for the ADX-629 trial in acute alcoholic hepatitis and questioned how prior work on a topical drug for Sjögren-Larsson Syndrome influences the current development of oral ADX-629 for the same indication.

Answer

President and CEO Dr. Todd Brady detailed that endpoints for alcoholic hepatitis include both symptoms and objective signs like intoxication tests and metabolic profiling. For Sjögren-Larsson Syndrome, he explained the strategic shift from a topical to an oral systemic treatment (ADX-629) to address both the skin (ichthyosis) and neurological aspects of the disease, building on positive proof-of-concept data from the earlier topical program.

Yale Jen inquired if the Sjögren-Larsson syndrome study would remain single-center, whether the FDA discussion for PVL would focus on PK/PD, and if there would be sales force synergies between reproxalap and ADX-2191.

Answer

President & CEO Dr. Todd Brady confirmed the SLS study is currently restricted to Dr. Rizzo's center, which has one of the world's largest patient cohorts. He clarified the FDA discussion for PVL is not about PK/PD but about the safety of the novel vitreous-compatible formulation. Regarding commercialization, he stated there are back-office synergies but the sales forces would be separate, as retinal surgeons (for ADX-2191) and anterior segment ophthalmologists (for reproxalap) are distinct call points. He added that the ADX-2191 sales effort would be small and focused on distribution to a limited number of specialists.

Yale Jen of Laidlaw & Company asked about the study size and data timing for the two reproxalap backup trials, and requested details on the study design and endpoints for the ADX-629 trials in ethanol toxicity and chronic cough.

Answer

CEO Dr. Todd Brady stated the crossover backup trial will enroll about 60 subjects, while the Schirmer test trial will enroll hundreds. Data is expected shortly after TRANQUILITY-2. He confirmed the ADX-629 trials for ethanol toxicity and chronic cough are both placebo-controlled crossover designs with 30-50 subjects. Endpoints will include both objective signs (e.g., acetaldehyde levels, cough counts) and subjective symptoms (e.g., nausea, quality of life).

Yale Jen from Laidlaw & Company asked for more details on the functional characteristics of the observed TCR-T cells, inquired about potential business development opportunities, and questioned the company's strategy for managing its debt.

Answer

CEO Kevin Boyle confirmed that translational assessments show persisting T-cells with limited exhaustion and that cells grown from post-treatment biopsies are functional. He also noted active discussions for business development, highlighting the Hunter platform's potential. VP of Finance Mike Wong addressed the debt, stating the $16.7 million balance is expected to be fully repaid by August 2023.

Yale Jen asked for clarification on the dose for the next patient, the specific configurations of the two new TCRs being added to the library, and whether the revenue from Solasia Pharma should be considered a recurring event.

Answer

CEO Kevin Boyle Senior stated the next patient would be treated at dose level 2, which ranges from 10 to 70 billion cells. VP of Research and Development, Drew Deniger, noted that the identity of the new TCRs is not yet disclosed pending FDA clearance but they will significantly expand the addressable patient population. VP of Finance, Mike Wong, clarified that the Q3 payment from Solasia is expected to be the most meaningful payment for the next few years.

Yale Jen of Laidlaw & Company questioned if manufacturing capacity was a rate-limiting factor for patient enrollment, inquired about the rationale for the rapid move to the second dose level, and asked for the anticipated total number of patients for the Phase 1/2 study.

Answer

CEO Kevin Boyle clarified that manufacturing is not the current rate-limiting step, and the company is proactively expanding capacity and developing cryopreservation techniques. He attributed the swift progression to the second dose level to the trial's design and the therapy's strong safety profile, as it targets hotspot mutations exclusive to tumor cells. Boyle stated that the total number of patients is not predetermined but will be guided by the science and the trial's adaptive Bayesian design.

Yale Jen from Laidlaw and Company requested details on prescription script numbers for the second quarter, asked for color on Q3 revenue trends, and questioned how the recent ACA mandate guidance might impact GTN in the second half of the year.

Answer

CEO Saundra Pelletier highlighted 11-12% quarter-over-quarter script growth and noted a 16% spike in demand from a recent media campaign. CFO Jay File reiterated the full-year revenue guidance of $30-$35 million, expecting strong sequential growth in H2 2022, supported by a major PBM contract win and the ACA mandate. He confirmed the ACA guidance should drive continued improvement in GTN as government enforcement increases.

Yale Jen of Laidlaw and Co. asked if the company could name the large PBM that lifted restrictions, requested the number of units shipped in the quarter, and inquired whether the Q1 sales figures met internal expectations.

Answer

CEO Saundra Pelletier stated that while she could not name the PBM due to confidentiality, it is one of the top three in the nation and the change will provide unrestricted access for 28 million lives. CFO Jay File reported that 27,053 units were dispensed in Q1. Pelletier confirmed that the Q1 sales figures were exactly in line with the company's expectations and that prescription trends rebounded strongly in March and April.