Swayampakula Ramakanth

Swayampakula Ramakanth asked about Humacyte's value proposition to Value Analysis Committees (VACs) and what resonates with them when dislodging autologous vein grafts. He also asked CFO Dale Sander about new cash use expectations, the runway provided by the current cash position, and if it includes the BLA submission for the dialysis indication.

Answer

President and CEO Laura Niklason explained that Humacyte's budget impact model, published in March 2025, demonstrates Symvess is cheaper even at its initial price point due to decreased amputation and infection costs, an argument strengthened by recent price reductions. CFO Dale Sander confirmed reduced operating expenses quarter-over-quarter, with R&D reductions expected to continue. He stated that the current cash position provides a runway exceeding 12 months, sufficient to cover interim V012 results, the dialysis BLA filing, and the commencement of human testing for cardiac bypass graft surgery.

Swayampakula Ramakanth inquired about Humacyte's value proposition to VAC committees, particularly against autologous vein grafts, and the company's new cash use expectations and runway with the current cash position, including key milestones like the dialysis BLA submission and CABG human testing.

Answer

CEO Laura Niklason referenced their March 2025 budget impact model, which demonstrated Symvess's cost-effectiveness even at its initial price due to reduced amputation and infection costs. She noted that with recent price reductions, this economic argument is even stronger and generally compelling for VACs, despite some hospitals' focus on initial acquisition cost. CFO Dale Sander confirmed significant operational expense reductions, including a $5 million decrease in R&D quarter-over-quarter. He stated that the $76 million cash on hand provides a runway exceeding 12 months, sufficient to cover interim VO12 results, the dialysis BLA filing, and the commencement of human testing for coronary artery bypass grafting (CABG).

Swayampakula Ramakanth asked about the tolerability profile of COM701 and its implications for combination therapies, particularly in less immune-inflamed tumors, and how Compugen plans to improve safety given the 16.7% grade 3 or higher treatment-related adverse events seen in the pooled analysis.

Answer

CMO Michelle Mahle stated that COM701 monotherapy is extremely well tolerated, with no discontinuations due to adverse events in the pooled analysis. She clarified that grade 3 adverse events in triplet combinations were consistent with the known safety profiles of Nivolumab and Pembrolizumab, positioning COM701 well for use as a monotherapy or in combination with standard of care or novel agents.

Swayampakula Ramakanth asked about COM701's tolerability profile and its influence on potential combination therapies, particularly in less immune-inflamed tumors, and how Compugen plans to improve safety regarding grade 3 or higher treatment-related adverse events seen in the pooled analysis.

Answer

Michelle Mahle, CMO of Compugen, stated that COM701 monotherapy is extremely well tolerated, with no discontinuations due to adverse events in the pooled analysis. She noted that grade 3 adverse events in triplet combinations were consistent with the respective labels for Nivolumab and Pembrolizumab, indicating COM701 is well-suited for combination with standard of care or novel agents.

Swayampakula Ramakanth inquired about Sanofi's progress with their BCMA-targeted ANKET asset, Innate Pharma's strategy for financing the lacutamab Phase III trial, and the potential indications and differentiation of IPH4502 based on preclinical data.

Answer

Jonathan Dickinson, CEO, confirmed Sanofi is progressing its BCMA-targeted ANKET in autoimmunity and updates are expected soon. He also noted active discussions with both investors and partners for lacutamab's Phase III, aiming for improved deal terms given the expanded market opportunity. Sonia Quaratino, CMO, highlighted IPH4502's potential in Nectin-4 expressing tumors, especially post-PADCEV urothelial cancer, for accelerated approval. Jonathan Dickinson added that IPH4502's payload and different resistance/toxicity profile should differentiate it from MMAE-based ADCs.

Swayampakula Ramakanth from H.C. Wainwright & Co. sought clarification on the potential indications for IPH4502 based on preclinical data and how its differentiation compares to other NECTIN-4 ADCs currently in clinical development.

Answer

CMO Sonia Quaratino highlighted urothelial cancer patients resistant to enfortumab vedotin as a key focus, noting the potential for accelerated approval. CEO Jonathan Dickinson added that IPH4502's differentiation stems from its payload and distinct resistance/toxicity profile compared to MMAE-based ADCs like PADCEV.

Swayampakula Ramakanth from H.C. Wainwright & Co. asked about Sanofi's progress with their BCMA-targeted ANKET® assets, the strategy for Lacutamab's Phase 3 initiation (partnering vs. investor funding), and the potential indications and differentiation of IPH4502 against other NECTIN-4 ADCs based on preclinical data.

Answer

Jonathan Dickinson, CEO & Director, confirmed Sanofi's continued development of the BCMA-targeted ANKET® for autoimmunity/immunology, with updates expected soon. For Lacutamab, he noted active discussions with both investors and partners to secure financing for Phase 3, aiming for improved deal terms due to increased market potential. Sonia Quaratino, EVP & Chief Medical Officer, identified urothelial cancer (especially post-EV refractory patients) and other NECTIN-4 expressing indications as key targets for IPH4502, highlighting its potential for accelerated approval. Jonathan Dickinson added that IPH4502's differentiation stems from its payload and distinct resistance/toxicity profile compared to MMAE-based ADCs.

Swayampakula Ramakanth asked about Innate's high-level strategy for allocating its cash resources across key assets like IPH6101, IPH6501, and IPH4502, especially in a challenging financing environment. He also questioned the importance of the upcoming long-term lacutamab data for partnership discussions.

Answer

Jonathan Dickinson, CEO, explained that the core strategy is to use the current cash, bolstered by Sanofi's investment, to reach key data inflection points for priority assets IPH4502 and IPH6501. He stressed that securing a partnership for lacutamab is essential to fund its future development. Dickinson confirmed the upcoming long-term data is critical for these partnership talks, as the improving data significantly strengthens the asset's value proposition.

Swayampakula Ramakanth requested commentary on the ongoing collaboration with Sanofi and asked about AstraZeneca's potential plans for Monalizumab following the upcoming data presentation at ASCO.

Answer

Chief Operating Officer Yannis Morel confirmed the Sanofi collaboration is very active, with two ANKET molecules prioritized in Sanofi's pipeline, and that he looked forward to potential updates this year. Regarding Monalizumab, he stated the ASCO presentation will be an update with longer-term data from the COAST trial but could not comment on AstraZeneca's future plans beyond the ongoing PACIFIC-9 trial.

Swayampakula Ramakanth of H.C. Wainwright & Co. inquired about the factors behind the CMS reimbursement rate increase, the commercial strategy for multi-unit sales, the clinical and regulatory path for the BPH indication, the status of the endometriosis trial, and the intended use of the proposed €36 million EIB financing.

Answer

CEO Ryan Rhodes stated that CMS considers clinical value, cost of service, and procedure volume for reimbursement, and that multi-unit sales to hospital networks validate the technology's value. He outlined plans for a US clinical study for BPH by early 2026 and noted that over 80% of sham-arm patients in the endometriosis study chose Focal One treatment post-unblinding. CFO Ken Mobeck clarified that no EIB funds have been received yet, while Rhodes added the capital is earmarked for accelerating HIFU commercial growth and clinical development.

Swayampakula Ramakanth of H.C. Wainwright & Co. asked about the commercialization strategy and timeline for endometriosis following the recent CE Mark approval. He also inquired about the expected timing for data from the PULS trial for pancreatic tumors and its geographic scope. Finally, he sought to clarify if the 16-25% HIFU revenue growth guidance for 2025 incorporates potential contributions from the increased CMS reimbursement or the new BPH CPT code.

Answer

Executive Ryan Rhodes explained that for endometriosis, the company is in the early stages of a controlled market entry in CE Mark countries, focusing on working with key opinion leaders to refine clinical development. For the pancreatic cancer PULS trial, he noted it's a small study conducted in France and is still in the early patient recruitment phase. Rhodes clarified that the 2025 HIFU revenue guidance is based purely on the core prostate cancer business, driven by the existing pipeline and market adoption, and does not yet factor in potential revenue from BPH or other new indications. Executive Ken Mobeck added that growth will also come from increasing service contract and disposable sales as the installed base matures.

Swayampakula Ramakanth of H.C. Wainwright inquired about EDAP's market penetration in top-tier U.S. cancer centers. He also asked about the timeline for international markets to become significant revenue contributors following new leadership appointments, and for details on the BPH trial's expansion and any required system modifications.

Answer

CEO Ryan Rhodes detailed their significant penetration in top cancer centers, noting they are in 7 of the top 10 U.S. News ranked hospitals and over 42% of NCI-designated centers, with more opportunity remaining. For international growth, he highlighted the new leadership and the importance of the forthcoming HIFI study publication for securing reimbursement in France, potentially by late 2024 or Q1 2025. For the BPH indication, Rhodes explained the current study is designed to support CE Mark/MDR approval and future FDA discussions. He confirmed the study protocol includes dose escalation to define optimal treatment parameters, which involves software and potentially hardware adjustments.

Swayampakula Ramakanth asked about the source of the Q2 revenue, the structure of the PANTHER registry study, potential tumor types and drugs to be explored in it, and the expected impact of hiring a new head of sales.

Answer

The company clarified that the $422k in Q2 revenue is entirely from new commercial sales, separate from the TIGER-PACT trial. The PANTHER study is an open registry without a fixed number of centers, designed to explore new tumor types like cholangiocarcinoma and other GI cancers, and new agents including immunotherapeutics. The new head of sales is tasked with ramping up commercial efforts immediately to drive revenue growth in 2025 and especially 2026, rather than just preparing for a post-approval launch.

Swayampakula Ramakanth of H.C. Wainwright & Co. asked if the reported Q2 revenue was from commercial sales or clinical trial purchases. He also sought details on the PANTHER registry study, including the number of centers, potential solid tumor applications beyond pancreatic cancer, the use of other drugs with Renovacath, and the expected impact of hiring a new Senior Director of Sales.

Answer

Shaun Bagai, CEO & Director, clarified that the $422,000 in Q2 revenue was entirely from new, non-trial commercial sales. He explained the PANTHER study is an open registry designed to collect real-world data on various solid tumors, such as cholangiocarcinoma and lung cancer, and with different agents, including immunotherapeutics. Bagai confirmed that hiring Phil Stockton as Senior Director of Sales is intended to immediately accelerate commercial efforts and build a team to drive significant revenue growth in 2026.

Asked about the payer distribution for the new ReWalk 7 placements, the practical benefits of the recent ALJ ruling, the reimbursement strategy for the advanced ReWalk 7, growth plans for the AlterG product line, and the rationale for lowering guidance.

Answer

The company responded that the 20 ReWalk 7 units were placed across all channels with a focus on Medicare. The ALJ ruling helps shape and formalize coverage policies and processes. The reimbursement for ReWalk 7 is currently the same as ReWalk 6 for Medicare, as the focus is on market penetration. AlterG growth will be driven by new channel partners and a renewed go-to-market strategy. The guidance was lowered to set realistic expectations regarding the timing of payer revenue cycles.

Swayampakula Ramakanth of H.C. Wainwright & Co. inquired about the payer mix for the 20 new ReWalk 7 placements, the benefit of the recent ALJ ruling, the reimbursement price for ReWalk 7, plans for AlterG growth, and the reasoning behind the updated guidance.

Answer

CEO Mark Grant stated the ReWalk 7 units were placed across all channels with a focus on Medicare, and that the ALJ ruling helps shape coverage policy. He noted the ReWalk 7 ASP is the same as ReWalk 6 for Medicare, with the current focus on penetration. Grant also outlined plans to expand AlterG via channel partners. On guidance, he cited the extended timing of payer cycles as the reason for the adjustment, not a change in the long-term outlook.

Swayampakula Ramakanth from H.C. Wainwright asked for details on the new ReWalk 7, the expanded MyoCycle partnership, the CorLife relationship for workers' compensation, the new BARMER contract in Germany, and the performance of the AlterG business since its acquisition.

Answer

CEO Lawrence Jasinski provided a comprehensive overview, explaining that ReWalk 7 features significant software, mechanical, and power upgrades at the same price point. He noted the MyoCycle partnership offers immediate synergy with the existing ReWalk sales channel and access to the home use market. The CorLife partnership provides crucial access to the workers' compensation market, which represents about 6-7% of the total market. The new BARMER contract in Germany formalizes processes for training, refurbishment, and replenishment, setting a standard for other insurers. Lastly, he stated that after a slow start, the AlterG business is now accretive, with the new NEO product line driving 17-19% growth in the last two quarters.

Asked about the key learnings from the Medicare reimbursement process and how they apply to private payers, and questioned the strategy behind the increased focus on the workers' compensation market.

Answer

The Medicare reimbursement cycle has been longer than initially expected but is anticipated to shorten as CMS gains familiarity. The extensive data package developed for Medicare has proven beneficial for submissions to private payers, which tend to process more quickly. The increased focus on workers' compensation is driven by a strategic opportunity to leverage their product portfolio and organization for efficient growth and a faster path to profitability, rather than being a reaction to federal government policies.

Swayampakula Ramakanth of H.C. Wainwright & Co. asked about the company's key learnings from the Medicare reimbursement process and the strategy behind the increased focus on the workers' compensation market.

Answer

CEO Larry Jasinski stated that while the Medicare reimbursement cycle has been longer than expected, the comprehensive data package developed for CMS has been beneficial for submissions to commercial payers, who are processing claims more quickly. He clarified the focus on workers' compensation is driven by a significant market opportunity and is part of a broader strategy to leverage the company's portfolio for the most efficient path to profitability, not a hedge against federal reimbursement policies.

Swayampakula Ramakanth from H.C. Wainwright & Co. asked about the long-term development and commercialization plan for ABS-201 and whether Absci's future internal pipeline would focus on specific therapeutic areas.

Answer

CEO Sean McClain outlined a strategy for ABS-201 that includes a global Phase 2/3 trial after the initial Phase 1/2a, with a potential approval targeted for the 2030-2031 timeframe. He also clarified that while Absci partners across various indications, its internal pipeline is increasingly focused on I&I, metabolism, and cardiometabolic diseases, with more details to be shared at an upcoming R&D Day.

Swayampakula Ramakanth from H.C. Wainwright & Co. inquired about the long-term development plan and commercialization timeline for ABS-201, and the therapeutic focus for new internal programs.

Answer

CEO Sean McClain and SVP Christian Stegmann outlined a plan to move from the Phase 1/2a trial directly into a combined Phase 2/3 study, targeting potential approval in the 2030-2031 timeframe. Sean McClain also specified that while partnerships are broad, the internal pipeline will focus more on I&I, metabolism, and cardiometabolic diseases.

Swayampakula Ramakanth from H.C. Wainwright asked how data from the internal pipeline is used to strengthen the AI models and how conversations with potential partners have evolved since the company's early days.

Answer

CEO Sean McClain described the company's 'lab in the loop' process, a rapid 6-week cycle of wet lab data generation and model training that continually improves platform capabilities. CFO Zachariah Jonasson added that current pharma discussions are now highly centered on the platform's advanced de novo design capabilities for drugging difficult targets, a significant evolution from earlier partnership conversations.

Swayampakula Ramakanth questioned the process for expanding Candivy's label to patients under four years old, asking about the nature and timing of the required study. He also inquired about the potential increase in patient numbers once the broader label is approved.

Answer

CEO Sean Brynjelsen clarified that a new clinical study is not required for the Candivy label expansion. The process involves submitting a revised formulation with lower excipient concentrations. He stated the company plans to submit a prior approval supplement in Q1 2026, targeting an approval for the wider label by the end of 2026. Brynjelsen projected that with the expanded label, the combined adrenal insufficiency franchise could reach 1,500 to 2,000 patients.

Swayampakula Ramakanth of H.C. Wainwright & Co. requested feedback on the Galzin relaunch, including sales force commentary and adoption expectations for 2025. He also asked for current patient numbers for Galzin and an explanation for the increase in Medicaid rebates and receivables on the cash flow statement.

Answer

CEO Sean Brynjelsen explained that the Galzin relaunch is progressing better than planned, with a steady stream of patients converting due to improved access, a $0 co-pay, and strong relationships with patient advocacy groups. He deferred providing specific patient numbers until the transition is more complete. CFO James Gruber addressed the financials, stating the increase in receivables was driven by Increlex sales and the pending $4.3 million payment from the Esteve licensing deal, while the higher Medicaid liability was almost entirely due to the Increlex relaunch.

Swayampakula Ramakanth of H.C. Wainwright & Co. asked about commercial efforts for LIVMARLI in Europe and the potential patient population size that could be added from the EXPAND study.

Answer

Co-Founder & CEO Chris Peetz stated European performance has been driven by Alagille syndrome, with future growth expected from securing PFIC reimbursement. COO & President Peter Radovich added that the EXPAND study includes various ultra-rare populations, with biliary atresia being a key component, representing a significant opportunity.

Swayampakula Ramakanth of H.C. Wainwright & Co. asked if the site onboarding process has become more efficient, if the 340B program could increase treatment rates, and if European clinical trials could boost ChemoSat sales.

Answer

CEO Gerard Michel noted the team has learned lessons, such as addressing perfusion needs earlier, but hesitated to promise a faster process due to each institution's uniqueness. He does not expect a significant jump in volume from 340B but believes it removes a financial barrier. He confirmed that opening trial sites in Europe will help ChemoSat sales long-term by establishing trained teams in new countries.

Swayampakula Ramakanth asked about the frequency of repeat procedures, the drivers behind new center activations, the strategy for the European market, and the potential for new indications based on European data.

Answer

CEO Gerard Michel confirmed that repeat treatment frequency is tracking towards the clinical trial average of around four treatments per patient. He attributed new center growth to positive word-of-mouth among physicians rather than competitive pressure. Regarding Europe, he stated that while revenue is growing, the focus remains on generating clinical data due to significant pricing and reimbursement challenges. He also noted that while European sites use CHEMOSAT for other indications, the data is not yet substantial enough to drive new sponsored trials.

Swayampakula Ramakanth of H.C. Wainwright & Co. asked about the influence of existing sites on new center adoption, the outlook for gross margins, the mandate for the expanded R&D team, and the ex-U.S. geographic expansion strategy.

Answer

CEO Gerard Michel confirmed a "fear of missing out" effect is helping drive interest from new centers. CFO Sandra Pennell projected gross margins could reach 90% by the end of 2025 due to operating leverage. Michel outlined an R&D strategy focused first on new cancer indications (CRC, breast), followed by combination therapies and new agents. He reiterated that the ex-U.S. strategy prioritizes strategic data generation over near-term revenue.

Swayampakula Ramakanth of H.C. Wainwright asked about the real-world interval between treatments compared to the FOCUS trial, sought anecdotal feedback on physician experience, and questioned if data from the SCANDIUM and CHOPIN trials would be sufficient for an FDA indication expansion.

Answer

CEO Gerard Michel confirmed that treatment intervals are generally 6-8 weeks, consistent with the trial, and shared anecdotal feedback from physicians who believe they are seeing even better response rates than in the FOCUS study. Regarding the European trials, Michel stated that a formal label expansion is not necessary, as physicians can already use the therapies in sequence. He noted the data would be important for informing clinical practice and potentially supporting future trials for patients failing immunotherapy.

Swayampakula Ramakanth of H.C. Wainwright & Co. asked for more color on the weakness in the pharma business and inquired about the logistics of revenue recognition if reimbursement is granted in Q4.

Answer

CEO Christopher Hall reiterated that the pharma business weakness is confined to the translational research segment due to macro headwinds, which he believes is temporary. He stressed that the strategic MRD pharma business is performing exceptionally well and is on track. CFO & COO Aaron Tachibana addressed the reimbursement question by stating that the key catalyst is receiving a favorable coverage decision from Medicare. He noted that the company's clinical revenue guidance of $3-6 million for the year already incorporates variables related to the timing and mechanics of revenue recognition post-coverage.

Swayampakula Ramakanth questioned the company's confidence in its reimbursement timelines amid potential federal regulatory changes and asked about the necessary next steps to advance the colorectal cancer (CRC) indication.

Answer

Executive Christopher Hall expressed high confidence in the reimbursement timeline, clarifying that they submit to Palmetto, a private Medicare contractor, which insulates the process from direct federal government shifts. For the CRC indication, he outlined the required steps: maturing the current interim data, achieving publication in a peer-reviewed journal, and then submitting the complete data package to Medicare for reimbursement consideration, noting it is a long-term strategic priority.

Swayampakula Ramakanth of H.C. Wainwright & Co. asked about the potential SG&A impact of building out an internal commercial team and sought clarity on the expected timeline for a Medicare reimbursement decision for breast cancer and subsequent submissions.

Answer

CEO Christopher Hall explained that the internal commercial team will remain small and the financial impact on SG&A will not be meaningful in the near term. Regarding reimbursement, he stated the typical timeline from submission to decision is about six months, plus or minus, and that while they expect at least two approvals in 2025, their internal goal is to submit and gain approval for all three key indications (breast, lung, IO therapy monitoring).

Swayampakula Ramakanth asked if the new acquisition models are increasing sales leads and whether the company is gathering enough feedback to improve products and pivot its customer focus away from capital-constrained segments.

Answer

President & CEO Jeff Hawkins clarified that the new models are primarily improving the conversion rate of existing leads who are sold on the technology but lack capital, rather than generating new leads. He stated the goal is not to abandon the academic market but to enhance product capabilities, like the V3 library prep kit and PTM analysis, to serve all segments based on direct customer feedback and current market opportunities.

Swayampakula Ramakanth of H.C. Wainwright & Co. questioned the improvements in upcoming sequencing kits, the strategy for maintaining a competitive lead versus conserving cash, and the effectiveness of peer-to-peer marketing efforts like academic webinars.

Answer

President and CEO Jeffrey Hawkins explained that new sequencing kits are released when combined improvements offer a meaningful performance lift, such as increased amino acid detection. He affirmed the company's strategy is to maintain its technology leadership through focused R&D investment while being prudent with capital, noting they are capitalized into H2 2027. Hawkins also highlighted the value of peer-to-peer marketing, including customer webinars and user group meetings, as a powerful complement to direct sales.

Swayampakula Ramakanth of H.C. Wainwright & Co., LLC asked for an update on the current enrollment percentage and the expected timeline for top-line data from the OCU400 Limelight trial. He also inquired about future DSMB reviews for the study and questioned how Ocugen plans to secure non-dilutive funding from the OrthoCelix spin-off and other sources to advance its three main programs.

Answer

Chief Medical Officer Dr. Huma Qamar stated that enrollment for the Limelight trial is on track for a 2026 BLA filing, but as a blinded study, efficacy data will only be released after the last patient's final visit. She noted another safety update is planned for Q4. CEO Shankar Musunuri addressed funding, explaining that Ocugen is actively pursuing non-dilutive funding through strategic partnerships to minimize shareholder dilution, while remaining opportunistic about equity financing as needed. He clarified that OrthoCelix will seek its own funding independently post-merger.

Swayampakula Ramakanth asked if the OCU400 liMeliGhT study has an interim analysis, sought clarification on its 2:1 randomization design, and inquired about expected clinical data updates for the modifier gene therapy programs in 2025.

Answer

Chief Medical Officer Dr. Huma Qamar confirmed the liMeliGhT study is blinded with no interim analysis, with full data available upon completion. CEO Dr. Shankar Musunuri clarified the 2:1 randomization is for OCU400, while a 1:1:1 ratio applies to the OCU410 dry AMD trial. Dr. Musunuri outlined 2025 data updates, including interim results for OCU410 for dry AMD in the fall, 12-month Phase I data for OCU410ST, and Phase I data for OCU200 late in the year.

Swayampakula Ramakanth from H.C. Wainwright inquired about the company's confidence in its aggressive goal of three BLA filings by 2028, the competitive positioning of OCU410 for Geographic Atrophy (GA), and expected data from the OCU400 Phase I/II study.

Answer

CEO Dr. Shankar Musunuri cited the company's track record and recent FDA alignment on OCU410ST as reasons for confidence in the BLA timelines. CMO Dr. Huma Qamar highlighted OCU410's superior safety profile and strong patient interest as key differentiators from approved GA therapies. Both executives confirmed that ongoing data, including 3-year durability data for OCU400, will be available before its BLA filing.

Swayampakula Ramakanth of H.C. Wainwright & Co. questioned the timing of adding a second pharmacy, the rationale for the revised revenue guidance, the timeline for the BELLA trial, the scope of relacorilant's oncology development beyond prostate cancer, and the status of FDA discussions for dazicorilant in ALS.

Answer

CEO Joseph Belanoff acknowledged the rapid demand from CATALYST outpaced their pharmacy's capacity. Sean Maduck, President of Corcept Endocrinology, explained the guidance cut reflects the cumulative impact of patient start delays. Chief Development Officer William Guyer noted the BELLA trial will have results in late 2026 and that oncology plans include other gynecological cancers. Chief Business Officer Charles Robb stated the FDA meeting for dazicorilant in ALS is scheduled for August 2025.

Swayampakula Ramakanth of H.C. Wainwright sought clarification on the ROSELLA trial's dual endpoint structure, asking what would happen if the Progression-Free Survival (PFS) endpoint is missed and how long the wait would be for Overall Survival (OS) data. He also questioned if the CATALYST study data is already impacting patient screening and Korlym use.

Answer

Chief Development Officer William Guyer responded that the company expects to meet the PFS endpoint, but if it were missed, the OS data would provide a second chance for a positive study approximately one year later. Sean Maduck, President of Endocrinology, explained that while a small impact from CATALYST is being seen, a more significant effect on medical practice is anticipated in the second half of the year and beyond as the data is more broadly published and communicated.

Swayampakula Ramakanth from H.C. Wainwright & Co. sought clarification on the ROSELLA study timeline, asking if the data would be analyzed and released by year-end or if the company would only reach the required number of progression events by then.

Answer

CEO Joseph K. Belanoff and Chief Development Officer William Guyer explained that while they are on track to have enough progression events by year-end, the exact timing for data analysis and release is uncertain, which is typical for event-driven oncology trials. Guyer noted that a longer timeline could indicate better patient outcomes. They committed to releasing the results publicly as soon as the analysis is complete.

Swayampakula Ramakanth asked about the factors that could lead the burn franchise to outperform its new guidance, the potential revenue from the BARDA contract, and the time it takes for a surgeon to adopt MACI Arthro post-training.

Answer

CFO Joe Mara cited stronger-than-average conversion of Epicel biopsies and continued NexoBrid momentum as drivers for outperformance. CEO Dominick Colangelo added that any potential Q4 BARDA revenue is pure upside to the current guide but declined to quantify it. He also clarified that surgeons often take biopsies before training, so there is no adoption lag time post-training.

Swayampakula Ramakanth asked about operational details, including whether MACI Arthro requires separate hospital committee approvals, if the new manufacturing facility would initially dip gross margins, and what the sustainable growth drivers are for the Burn Care franchise.

Answer

CEO Dominick Colangelo stated that P&T committee approval is not a factor for MACI Arthro, though some hospitals review the new instruments. He also highlighted the fully staffed burn care sales force as a key growth driver. CFO Joe Mara added that while the new facility's costs will be absorbed, they do not expect a material negative impact on the gross margin trajectory toward the high 70s target.

Swayampakula Ramakanth asked a high-level question about operating margin synergies from MACI Arthro adoption and whether these financial gains would be realized in the near or long term.

Answer

CFO Joe Mara explained that MACI Arthro has significant built-in synergies by leveraging the existing commercial infrastructure without materially changing the P&L profile per case. He noted that while there is minor incremental revenue from instrument sales, the primary benefit is driving top-line growth, which in turn provides operating leverage for the business rather than changing the margin on a per-procedure basis.

Swayampakula Ramakanth of H.C. Wainwright & Co. asked about the effectiveness of new commercial partnerships for personal devices, the immediate strategy to return the Enterprise Health segment to growth, and the proportion of enterprise clients that rely on federal grants for purchases.

Answer

CEO Scott Davis explained that partnerships with Pria, Bionic P&O, and NSM are progressing well, with a quarter-over-quarter increase in patient claims being submitted. To get the Enterprise segment back on track, he stated the focus is on closing the deferred deals from Q2, including a large IDN deal over $1 million expected in Q3. Davis also mentioned leveraging a new third-party financing partner to help clients convert capital expenses to operational ones. He conservatively estimated that historically, around 10% of North American enterprise customers use grants.

Swayampakula Ramakanth asked for details on the new distribution partnerships with National Seating & Mobility and Bionic P&O, specifically questioning if geographical overlap was a concern and how the partnerships impact G&A expenses versus distributor margin sharing.

Answer

CEO Scott Davis clarified that the partnerships with NSM and Bionic P&O are complementary and enhance value by leveraging their expertise in claims processing and patient coordination, which is more efficient than building the capability in-house. He stated that while there is a trade-off in margin, ongoing operational efficiencies and cost controls are expected to allow Ekso to support its distribution network while maintaining strong corporate gross margins in the high-50s as volume scales.

Swayampakula Ramakanth of H.C. Wainwright & Co. inquired about the geographic breakdown of the 33 device placements in Q3, the status of CMS claims filed for the Ekso Indego Personal, and the nature of the customer purchasing delays and the resulting order backlog.

Answer

Scott Davis (executive) explained that approximately half of the device placements were in Europe, with several in APAC, making the majority international. He noted that personal products represented about 20% of total revenue. Regarding CMS, Davis stated that multiple claims were submitted, with one reimbursed and two requiring resubmission, acknowledging the process is taking time. He attributed purchasing delays to North American IDNs being in an 'off-cycle' year after strong sales in 2022-2023, with a return to normal cycles anticipated for Q4 and 2025.

Swayampakula Ramakanth asked about the company's strategy for NexoBrid stockpiling, the geographic distribution of sites in the EscharEx Phase III trial, the potential for early study completion, and the relative result timelines for the Phase II head-to-head and Phase III studies.

Answer

Ofer Gonen (executive) explained that treating patients is the current priority over stockpiling, with any related revenue already factored into existing guidance. He detailed that approximately 17-20 of the 40 EscharEx trial sites are in the U.S. and that the enrollment pace is deliberately managed to ensure data quality, making an early finish unlikely. Gonen also clarified the Phase II head-to-head study is simpler and expected to produce results before the Phase III trial concludes.

Swayampakula Ramakanth questioned how EscharEx's performance on granulation tissue formation compares to SANTYL under the new LCDs, whether the VLU study could enroll faster than expected, and the current status of the planned U.S. facility in collaboration with BARDA.

Answer

Executive Barry Wolfenson referenced Phase II data showing EscharEx's superiority over SANTYL in achieving complete debridement and wound closure more quickly. CEO Ofer Gonen stated that they are deliberately not accelerating VLU trial enrollment to ensure the recruitment of high-quality patients with hard-to-heal wounds. He also clarified the BARDA collaboration is a long-term project for a backup U.S. facility, with a timeline extending to 2027-2028, and does not impact near-term guidance.

Swayampakula 'RK' Ramakanth of H.C. Wainwright & Co. questioned the near-term benefit of the new Category III CPT code for NexoBrid, the necessary steps to initiate the DFU study, and the key takeaways from the FDA Type C meeting regarding the temperature-stable NexoBrid formulation.

Answer

Ofer Gonen (Executive) responded that the CPT code is not expected to have a significant short-term impact on market penetration. For the DFU study, he outlined that the company must first develop a protocol, gain alignment from KOLs, and secure FDA/EMA clearance, a process expected to take about a year. For the temperature-stable NexoBrid, Gonen detailed that the path to a 2026 trial start involves building a dedicated manufacturing facility and completing nonclinical and CMC data requests from the FDA.

Swayampakula Ramakanth from H.C. Wainwright & Co. asked for clarification on whether the chronic neutropenia (CN) patient population figures were U.S.-only, what the target label population would be, and if the Norgine partnership covers both WHIM and CN indications.

Answer

CEO Paula Ragan confirmed the 50,000 diagnosed and 15,000 high-unmet-need patient figures are for the U.S. only. Executive Christophe Arbet-Engels explained that despite the trial's focus on severe patients, the company will seek a broad label for the entire CN population. CFO Adam Mostafa clarified that the Norgine agreement is a license covering both the WHIM and CN indications.

Swayampakula Ramakanth of H.C. Wainwright & Co. asked for an update on XOLREMDI's market uptake, including the number of patients on the drug, the commercialization setup, and the expected timeline for broader adoption.

Answer

Chief Commercial Officer Mark Baldry stated that the company is currently 'laser-focused' on raising disease awareness and driving screening for WHIM syndrome, as no approved treatment existed before XOLREMDI. He explained this foundational work is crucial for building momentum into 2025, at which point more details on the commercial ramp-up would be shared.

Swayampakula Ramakanth inquired about the feedback from physicians and patients at recent medical conferences regarding XOLREMDI adoption and what the company has learned to improve uptake. He also asked if any other data from the 4WARD study would be presented before the full Phase II data release in November.

Answer

President and CEO Dr. Paula Ragan and Chief Commercial Officer Mark Baldry highlighted the excitement among physicians for an oral, targeted therapy backed by strong Phase III data showing infection reduction. Dr. Ragan confirmed that the next data release for the chronic neutropenia program will be the full Phase II dataset in November, with no other interim updates planned before then.

Swayampakula Ramakanth of H.C. Wainwright inquired about the accounting treatment for bona fide service fees paid to Venture Medical, the expected timeline for resolution with the SEC, the relevance of the recent FDA warning letter, and the sustainability of Q4 revenue performance into 2025.

Answer

CFO Michael Fortunato explained the accounting issue is a matter of classification on the income statement—whether payments to Venture are a sales expense or a reduction of revenue—and would not materially affect net income or EBITDA. He could not provide a specific timeline for the SEC resolution. CEO Jason Matuszewski clarified that the FDA warning letter pertained to four products that were already discontinued and does not impact the company's current product portfolio. Both executives declined to provide specific 2025 guidance but expressed confidence in continued progress.

Swayampakula Ramakanth from H.C. Wainwright & Co. asked about potential non-dilutive financing options to extend the cash runway into 2027. He also questioned the potential reasons NBTXR3 might not reach the market despite its promising profile and inquired about the gating event for initiating a clinical program with the Curadigm asset.

Answer

CFO and CBO Bart Van Rhijn discussed financing, indicating a preference for non-dilutive options like royalty financing, noting only 'low teens' of millions would be needed to extend the runway into 2027. Co-Founder and CEO Laurent Levy addressed program risk, stating that major hurdles like manufacturing and partnering are now behind them, leaving the program in a 'very derisked situation.' Regarding Curadigm, Levy explained that the main gating factor is timing, as they have sufficient results and are actively pursuing partnerships.

Asked about the extent to which the validation from NBTXR3 can be applied to the new Curadigm and Oocuity platforms, whether new data is needed for partnerships, and if preclinical data can be expected in the next year.

Answer

While NBTXR3 provides broad validation for nano physics-based therapies, the new platforms are different and will require their own specific data to secure partnerships. These platforms offer multiple opportunities, including technology licensing, not just product co-development. The company confirmed it will be able to share new data in the second half of the year.

Swayampakula Ramakanth from H.C. Wainwright questioned how much validation from the NBTXR3 program could be applied to the newer Curadigm and Oocuity platforms and whether new data would be required to attract partners for these platforms. He also asked about the timeline for preclinical data.

Answer

Co-Founder and CEO Laurent Levy emphasized that while NBTXR3 provides broad validation for nano physics-based medicine, the new platforms are distinct and will require their own data to secure partnerships. He clarified that Curadigm and Oocuity are technology platforms with diverse opportunities, including product co-development and technology licensing. Levy confirmed that the company plans to share preclinical data for these platforms, with initial updates expected in the second half of 2024.

Swayampakula Ramakanth from H.C. Wainwright questioned if the multiple data looks for the NANORAY-312 trial would impact the statistical alpha for a potential accelerated approval filing, and also asked about Janssen's interest in pursuing other indications from the MD Anderson collaboration, such as pancreatic cancer.

Answer

CEO Laurent Levy confirmed that the multiple data analyses (futility, interim, final) do influence the statistical alpha, and this was factored into the trial's design and power calculations. Regarding pipeline strategy, CFO Bart Van Rhijn explained that the MD Anderson collaboration is separate but compatible with the J&J license, serving to find efficacy signals that inform discussions with J&J about future development pathways beyond head & neck and lung cancer.

Swayampakula Ramakanth inquired about the new $8-$10 million partnership, asking about the timing of the revenue drawdown, the potential for a second purchase order, and whether this deal structure will be a model for future collaborations. He also asked for clarification on how the Vultr partnership impacts margins and about the company's expected cost structure following the potential divestiture of its European operations.

Answer

CEO Jennifer Bath explained that the initial $8 million purchase order will be recognized over an 18-month period via weekly drawdowns based on work completion, with revenue recognition already starting in the current quarter. She confirmed this cash-based, execution-focused deal is a repeatable model for future large-scale partnerships. Regarding the Vultr collaboration, she stated it primarily benefits the AI programs by reducing costs and increasing speed, which indirectly impacts wet lab projects as AI becomes more integrated. Finally, she confirmed that post-divestiture, the company's overall operating costs are expected to be significantly lower, with any revenue impact anticipated to be temporary.

The analyst asked for the strategic rationale behind the headquarters move to Austin and cost containment measures, the expected financial contributions from different business entities following the restructuring, and the development timeline for key assets like the LILRB2 and Trk programs.

Answer

The CEO explained that the Austin move is strategic for accessing AI/biotech talent and non-dilutive funding. The divestiture of European labs aims to reduce redundancies with the more profitable and efficient BioStrand platform and generate capital. Future growth will be driven by the Canadian lab, a new low-capital investment in the Boston area, and especially the high-margin BioStrand platform. Updates on existing assets are forthcoming, and new assets developed entirely by BioStrand will be unveiled to showcase its revolutionary capabilities.

Swayampakula Ramakanth asked for an update on the base business, specifically the performance of the Vancouver and Utrecht facilities, and inquired about the inventory build-up at the Utrecht site, its potential revenue impact, and timing. He also sought clarification on how LENSai technology is enhancing the Talem portfolio and whether this was driven by partner requests. Finally, he questioned if the LENSai platform had started generating fee-for-service revenue.

Answer

CEO Jennifer Bath confirmed double-digit growth at the Victoria and Oss discovery labs, while the Utrecht site focused on rebuilding inventory, with resulting sales already occurring in Q2. CFO Kristin Taylor added that the Utrecht site experiences more timing variability due to quicker project turns. Regarding the Talem portfolio, Jennifer Bath explained that LENSai enhancements increase asset value and showcase the platform's capabilities, driven by both internal strategy and partner feedback, with a full update expected in 6-8 weeks. She also confirmed that LENSai has generated fee-for-service revenue and that its current sales pipeline is the strongest to date.

Asked about strategies to accelerate VA revenue growth, potential for price increases in FSS contracts, details on prescriber adoption in the managed care system, the nature and future cadence of military orders, growth levers for Truvaga Plus, and when the company might provide top-line guidance.

Answer

VA growth is driven by direct sales efforts and a potential future PTSD label. The company plans to hold pricing constant in new FSS contracts. The 25 prescribers in the managed care system are revenue-generating, primarily in California where the product is on formulary. The recent military order was for the new TAC-STIM model, and revenue from this channel is expected to remain variable until 2026-2027. Truvaga Plus growth will be pursued through e-commerce, affiliate platforms like Perks at Work, and an Amazon launch. The company will reevaluate providing top-line guidance in the new year but has no immediate plans to do so.

Swayampakula Ramakanth of H.C. Wainwright asked about strategies to accelerate growth in the VA channel, the potential for price increases in new VA contracts, details on prescribers in the managed care system, specifics of the post-quarter TAC-STIM order, growth levers for Truvaga Plus, and when the company might provide top-line guidance.

Answer

CEO Daniel Goldberger explained that VA channel growth is driven by scaling their commission-based sales force and that a future PTSD label could substantially increase the addressable market. He stated they are holding pricing constant in VA contract renewals. Regarding the managed care partner, he noted the 25 prescribers are primarily in California where gammaCore is on formulary. He confirmed the large post-quarter order was for TAC-STIM and that revenue from this channel will remain variable. For Truvaga, new channels like affiliates and Amazon are key growth levers. CFO Joshua Lev added that providing top-line guidance is not planned but will be re-evaluated for the new year.

Swayampakula Ramakanth asked if the current number of devices in the U.K. is sufficient to generate the data required for NICE designation and whether this data would be presented publicly. He also questioned the status of the company's Diabetic Foot Ulcer (DFU) indication program.

Answer

Dr. Michael DiMaio, Chairman of the Board, stated he could not confirm the exact number of images needed for NICE approval but stressed that initial feedback from U.K. doctors has been 'overwhelmingly positive.' He affirmed a publication plan is in place to present data at conferences and in medical journals. Regarding the DFU program, Dr. DiMaio explained it is in a 'holding pattern' as the company's immediate priority is focusing on and delivering the BARDA-funded burn indication product.

Swayampakula Ramakanth asked for details on the upcoming EVX-01 biomarker data, inquired about other potential data releases in the next six months, and questioned the likelihood of closing another business development deal before year-end 2024.

Answer

Chief Science Officer Birgitte Rono detailed the planned EVX-01 biomarker analysis, which includes T cell profiling and soluble analytes. Both Rono and CEO Christian Kanstrup indicated it was too early to announce other 2025 data milestones. Kanstrup acknowledged the potential for another BD deal in 2024 but cautioned that timing is uncertain.

Swayampakula Ramakanth asked if additional PRGN-2012 data would be released before the confirmatory study, questioned plans for European regulatory submissions, and inquired about the planned size of the commercial team.

Answer

President and CEO Dr. Helen Sabzevari stated that no further data is planned for release, as the Phase I and II data presented at ASCO is complete for the BLA submission, though durability follow-up continues. She confirmed active engagement with ex-U.S. authorities and readiness for global supply in 2025. Chief Commercial Officer Phil Tennant described the commercial team as being precise and small, likely in the 'tens' of representatives, integrated with medical affairs for a 'small but perfectly formed' approach.

Swayampakula Ramakanth asked if additional data for PRGN-2012 would be released before the confirmatory study, about plans for regulatory submissions in Europe, and about the planned size of the commercial team for the launch.

Answer

President and CEO Dr. Helen Sabzevari stated that no further data would be released as the Phase I and II data sets are complete for the BLA submission, though durability follow-up is ongoing. She confirmed active engagement for ex-U.S. markets, including Europe. Chief Commercial Officer Phil Tennant added that the commercial team will be precise and small, numbering in the 'tens' rather than hundreds, and will be tightly integrated with medical affairs.

Swayampakula Ramakanth asked if additional data for PRGN-2012 would be released before the confirmatory study, about plans for regulatory submissions in Europe, and about the planned size of the commercial team.

Answer

President and CEO Dr. Helen Sabzevari stated that no further data is needed for the BLA submission, as the Phase I and II data sets are complete, though durability follow-up is ongoing. She confirmed active engagement with ex-U.S. authorities and manufacturing readiness for a global launch. Chief Commercial Officer Phil Tennant added that the commercial team will be a precise, small footprint of "tens" of sales representatives, integrated with medical affairs.

Asked about the commercialization strategy for PRGN-2012, including the sales force approach, and for details on the upcoming preliminary data and development timeline for the UltraCAR-T PRGN-3007 program.

Answer

The commercial strategy for PRGN-2012 involves a small, specialty sales team targeting a concentrated group of laryngologists. For the UltraCAR-T PRGN-3007, an interim data update is expected by the end of 2024, after which the trial will move to a Phase Ib expansion phase upon dose selection and regulatory discussion.

The analyst asked about the regulatory pathway and timeline for the breast implant program, and the monetization strategy for the gut-on-a-chip program.

Answer

The company stated the breast implant regulatory pathway is likely a PMA, with CBER leading. The upcoming large animal study is crucial for verifying tissue quality and surgical protocols before a pivotal study. It's too early to provide a timeline for the PMA study as they need to consult with the FDA first. For the gut-on-a-chip program, the plan is to produce the scaffolds and provide them to a CRO partner who will handle patient biopsies and testing.

Swayampakula Ramakanth asked for more specifics on the IND amendments that accelerated trial enrollment and the potential number of patients in the Q3 data update. He also inquired about the strategy for generating non-dilutive funding from the Hunter platform and sought clarification on whether the released restricted cash was included in the Q1 cash balance.

Answer

CEO Kevin Boyle detailed the IND amendments, including combining screening and treatment protocols, removing the tumor retesting requirement, and adding cryopreservation, which all increased efficiency. VP of Research & Development Drew Deniger explained that the Hunter platform discovers proprietary TCRs that could be out-licensed for various modalities, creating partnership opportunities. VP of Finance Mike Wong confirmed the $13.9 million in restricted cash was included in the total cash balance of $37.4 million as of March 31, 2023.

Swayampakula Ramakanth from H.C. Wainwright asked about the current bed-to-bed manufacturing time for the TCR-T doses and the potential for its reduction. He also inquired about the Solasia Pharma relationship, specifically their commercialization timeline and the royalty terms.

Answer

CEO Kevin Boyle stated that the current manufacturing time is approximately 30 days, and the company sees a clear path to reducing it by about half through ongoing process development. Regarding the Solasia partnership, Boyle deferred to Solasia for their commercialization plans but clarified that Alaunos expects to receive low single-digit royalties. He characterized this as helpful non-dilutive capital but not a transformational amount for the company's balance sheet.

Swayampakula Ramakanth of H.C. Wainwright requested the rationale for the EOS-448 and iberdomide combination in multiple myeloma and asked for details on the biomarkers being used in the inupadenant trials.

Answer

CMO Joe Lager explained the multiple myeloma combination is supported by preclinical collaboration data showing synergy between an FC-engaging TIGIT antibody and an IMiD like iberdomide. Regarding biomarkers for inupadenant, he referenced the ASCO data showing A2A receptor expression levels (IHC) correlated with clinical benefit, and noted they are also evaluating other IHC and mRNA markers.

Swayampakula Ramakanth from H.C. Wainwright & Co. requested details on the target indications and biomarkers for the inupadenant program and asked about the types of assets iTeos might acquire to expand its pipeline.

Answer

Chief Medical Officer Joe Lager and President and CEO Michel Detheux explained that the A2AR expression biomarker is guiding indication selection, with plans confirmed for anti-PD-1 resistant melanoma and an update on other solid tumors coming later in the year. Detheux added that pipeline expansion will focus on internal discovery targeting new immune pathways and opportunistic external innovation for mechanisms targeting different immune cells.