Michael Okunewitch

Michael Okunewitch asked if the current FDA administration has confirmed that the 12-month pain-only endpoint for the chronic lower back pain program is sufficient for approval. He also inquired about any outstanding items requested by the FDA that need to be finalized before the upcoming BLA submission for the Class 4 heart failure program next quarter.

Answer

CFO and Managing Director Silviu Itescu confirmed that the FDA has indeed confirmed the 12-month pain-only endpoint as an approvable endpoint, supported by recent guidance on single well-conducted randomized controlled trials. For the Class 4 heart failure BLA, he mentioned commercial manufacturing as a very important component, noting that the product was made at the same Lonza facility as Ryoncil, which was approved, suggesting a potential advantage.

Michael Okunewitch asked for confirmation from the FDA regarding the sufficiency of the 12-month pain-only endpoint for chronic lower back pain approval and inquired about any outstanding items requested by the FDA for the upcoming Class 4 heart failure program filing.

Answer

CEO Silviu Itescu confirmed that the FDA's current administration has affirmed the 12-month pain reduction endpoint as approvable for chronic lower back pain, and a single well-conducted randomized controlled trial could be sufficient. For the Class 4 heart failure filing, he highlighted commercial manufacturing as a key ongoing component, noting the product's manufacturing at the Lonza facility, which is already approved for Ryoncil, as a potential advantage, pending further agency confirmation.

Michael Okunewitch asked about the comprehensiveness of the Qkine collaboration, specifically if other commonly used cytokines might be subject to future agreements or M&A. He also asked if the exclusivity for certain cytokines was mutual (i.e., if Qkine was also exclusive to CellSeal for those products).

Answer

Chairman and CEO Roderick de Greef confirmed that other cytokines could be subject to future agreements, noting the current deal has narrow exclusivity for specific cytokines geared towards key customers and pipelines, with broader non-exclusive access. He clarified that the exclusivity is currently one-way (for BioLife relative to Qkine's cytokines) and that while there's a loose intent for CellSeal packaging, it's not yet papered. He doesn't anticipate agreements with other cytokine manufacturers for CellSeal vials at this time.

Michael Okunewitch inquired whether BioLife Solutions is currently observing any potential acquisition targets in the biopreservation area where valuations have become more attractive or normalized, prompting the company to consider pulling the trigger on such deals.

Answer

Chairman and CEO Roderick de Greef stated that while BioLife rigorously evaluates potential competitive technology in biopreservation, nothing has come to their attention that would offer a competitive advantage or value proposition beyond what they already provide, making the PanTHERA acquisition unique in that regard.

Michael Okunewitch asked for more detail on the allocation of increased R&D spending and inquired about BioLife's involvement with any mesenchymal stem cell (MSC) based products.

Answer

CEO Roderick de Greef clarified that the additional R&D spend is for product line expansion, not new products, focusing on enhancing the Sexton-acquired consumables like the CryoCase and the CT-5 automated fill device. He offered to follow up separately regarding the company's specific involvement with MSC-based therapies.

Michael Okunewitch asked about the most relevant endpoints for EscharEx's health economic benefit and any specific Phase 3 thresholds that could justify upside pricing. He also inquired about factors that might lead physicians to choose alternative debridement methods like sharp or autolytic over EscharEx, and if there are hard limits to EscharEx's market penetration beyond the 22.3% conversion estimate.

Answer

Barry Wolfenson, EVP of Strategy and Corporate Development, explained that health economic benefits are primarily linked to early wound closure, which reduces costs associated with nursing, physician time, product use, and risks like infection. He noted that payers indicated a potential pricing cap of approximately 50% premium over SANTYL, even if HEOR benefits are higher, and their model uses a conservative price within this range. He added that sharp debridement would persist, especially in DFU with peripheral neuropathy, and autolytic debridement would remain due to its lower cost, but EscharEx is expected to capture significant share from current enzymatic debridement.

Michael Okunewitch asked about the most relevant endpoints for the health economic benefit in the new market research for EscharEx and if specific Phase III thresholds could justify upside pricing. He also questioned the factors that would lead physicians to choose alternative debridement methods like sharp or autolytic, seeking to understand potential hard limits for EscharEx beyond the 22.3% conversion estimate.

Answer

Barry Wolfenson, EVP of Strategy and Corporate Development, explained that the HEOR benefits primarily focus on early wound closure, leading to savings from reduced nursing/physician time, product use, and risks like infection. He noted that payers indicated a pricing cap at roughly a 50% premium over SANTYL, even if HEOR benefits are higher, and their model uses a conservative price within that range. He also stated that sharp debridement would persist, especially for DFU with peripheral neuropathy, and autolytic debridement would remain due to its lower cost, though EscharEx aims to capture significant share from both.

Michael Okunewitch questioned the potential pricing strategy for EscharEx against SANTYL, considering factors like faster debridement and reduced healthcare utilization. He also asked if new NexoBrid stockpiling programs would be supplied from the new Israeli facility or require separate dedicated manufacturing.

Answer

Barry Wolfenson (executive) addressed pricing, stating that while the initial model compares product costs, a full health economics (HEOR) study is underway to assess downstream savings, which could justify a higher price premium. Ofer Gonen (executive) added that the company's multiple manufacturing facilities provide flexibility to support both the EscharEx launch and NexoBrid demand, including commercial sales and stockpiling, from various sites including the planned U.S. facility.

Michael Okunewitch asked for the rationale behind partnering with Kerecis for the DFU study and sought clarification on how the company expects to achieve its 2025 incremental revenue growth given the new manufacturing facility won't be approved until 2026.

Answer

CEO Ofer Gonen stated the strategy is to partner with the best-in-class products for each indication, selecting Kerecis for DFU based on its strong clinical results, similar to choosing MIMEDX for VLU. He clarified that the 2025 revenue growth of approximately $4 million is not dependent on the new facility but will be driven by a few extra weeks of production from the current facility, price increases, and a strategic shift to more profitable territories.

Michael Okunewitch of Maxim Group asked for details on the clinical work required for the temperature-stable NexoBrid, the potential market size of the European stockpiling opportunity, and the role of partners like Solveton in the head-to-head VLU study.

Answer

Ofer Gonen (Executive) stated the expectation is for a single, small clinical trial for the temperature-stable formulation, contingent on completing nonclinical work. He sized the European stockpiling market in the 'low tens of millions of dollars' annually, with shipments likely starting in 2026. Ryan Zimmerman (Company Representative) clarified that collaborations with companies like 3M (for its Kovan products) are to standardize care and minimize variability by providing best-in-class compression therapy for patients in the trial.

Michael Okunewitch from Maxim Group asked about the distribution of RYONCIL sales throughout the launch quarter to gauge revenue trajectory and inquired about recent FDA feedback on a potential filing for Revascor in heart failure.

Answer

CEO Silviu Itescu and CCO Marcelo Santoro noted it was too early to provide sales guidance but highlighted the outstanding performance in the first quarter of launch. Regarding heart failure, Dr. Itescu described a 'terrific meeting' with the FDA, resulting in alignment on chemistry, manufacturing, and controls (CMC), potency assays, and the design of a confirmatory trial, paving the way for the BLA filing.

Michael Okunewitch followed up on pediatric dilated cardiomyopathy (DCM), asking what would constitute a clinically meaningful improvement in survival or time to transplant. He also requested an expansion on the newly licensed iPSC platform technology and its key differentiators.

Answer

Chief Medical Officer Nataliya Agafonova and Chief Science Officer Dr. Joshua Hare explained that in DCM, any measurable reduction in all-cause mortality or heart failure hospitalizations, or a prolongation of transplant-free survival, would be clinically meaningful. Dr. Hare then detailed the iPSC technology, stating it uses a patented method to select a subpopulation of cells that can form heart muscle without causing the dangerous arrhythmias observed with other iPS cell transplant approaches, which has been a major barrier to clinical translation.

Asked about the required commercial infrastructure for HLHS, the impact of Alzheimer's partnering on manufacturing strategy, and the specific design of the planned adaptive Phase II/III trial for Alzheimer's disease.

Answer

A small, focused commercial team of 12-15 people will be needed for HLHS, as the 12 trial centers cover ~80% of the surgical volume. The manufacturing decision for HLHS is distinct from Alzheimer's, as the latter will require a larger commercial-scale facility (likely a CDMO or partner's). The Alzheimer's trial is a seamless adaptive Phase II/III design where positive interim data from the first 600 patients could support an accelerated BLA submission.

The analyst asked about any new FDA interactions that might support an expedited pathway, the specifics of the Phase 2 trial's comparison against placebo, and the potential timing for the release of unblinded data.

Answer

The company reported no new direct interactions with the FDA confirming a further expedited pathway beyond their current Fast Track designation. They clarified that the trial compares responder rates (based on >30% improvement from baseline) against a sham control group, with safety being the primary endpoint. The decision on unblinding data is pending the outcome of the upcoming Type B meeting with the FDA, as they might be able to proceed to the next stage without an interim analysis.

Michael Okunewitch of Maxim Group asked for an update on any new FDA interactions that might support an expedited regulatory pathway for BRTX-100, clarification on the trial's efficacy endpoints versus placebo, and the potential timing for unblinding trial data.

Answer

Chairman and CEO Lance Alstodt acknowledged anecdotal support for an accelerated pathway but confirmed no new formal designations from the FDA beyond Fast Track. Chief Scientist Francisco Silva clarified that the trial compares responder rates between the treatment and sham control groups. Alstodt added that a decision on unblinding data will follow the upcoming Type B meeting with the FDA, as they may be able to proceed to Phase 3 based on blinded data, which would preserve the trial's statistical power.

Inquired about the magnitude of response in the 52-week data, the potential to file for accelerated approval given the current FDA climate, and the secondary endpoints that will be analyzed in the final unblinded data.

Answer

The executives confirmed a continuing trend of significant response in 3 of the 4 patients at 52 weeks (over 60% improvement). They are optimistic about the potential for the Phase II data to be used for a pivotal study and accelerated approval. They also plan to analyze secondary endpoints, including radiographic improvements via MRI, to provide objective evidence of efficacy beyond the primary pain and function measures.

Michael Okunewitch asked for details on the magnitude of response in the 52-week data, particularly the separation between the top three responders and the fourth. He also explored the possibility of filing for accelerated approval based on strong pain and function data, and inquired if secondary endpoints would include percent change from baseline between groups.

Answer

VP of R&D Francisco Silva confirmed a continuing trend of significant separation, with three patients showing over 60% improvement compared to one with a lesser effect. Both Silva and CEO Lance Alstodt expressed optimism that the data could potentially be used for a pivotal trial, noting the FDA's increasingly supportive view of cell therapies. They also confirmed that radiographic endpoints and other measures are being tracked to provide objective evidence of efficacy, with Alstodt highlighting that MRI images have not yet been released.

Michael Okunewitch of Maxim Group sought confirmation from the current FDA administration regarding a single pivotal trial design for full approval. He also asked about the potential scale of the commercial force needed for a market launch.

Answer

Chairman & CEO Tom Lin affirmed that the path for a single-study approval is 'very clear and very straightforward' provided they meet a robust statistical significance of p < 0.01. CFO & Director Hao-Yuan Chuang noted that the current four-year cash runway forecast does not yet include the full costs of commercialization, which are being budgeted.

Michael Okunewitch of Maxim Group requested more details on the timing and conditions for the interim analysis of the Geographic Atrophy (PHOENIX) trial and asked if the 500-patient enrollment target was final.

Answer

Chairman and CEO Dr. Tom Lin stated that the company would not disclose details on the GA interim analysis until receiving an official response from regulators. Both Dr. Lin and Chief Scientific Officer Dr. Nathan Mata confirmed there is no current plan for a sample size reestimation in the PHOENIX trial, as they are capitalizing on smooth enrollment to add more subjects upfront to strengthen the study.

Michael Okunewitch from Maxim Group asked about the statistical design of the DRAGON study, specifically inquiring about the difference in lesion growth between the placebo and treatment arms that the trial is powered to detect.

Answer

Chief Scientific Officer Dr. Nathan Mata answered directly, stating that the DRAGON study is powered for a 40% treatment effect with 80% power to detect that effect at the two-year endpoint.

Michael Okunewitch asked about the necessity of the DRAGON 2 trial for a U.S. filing, the potential for a broad label in GA, and the key differentiators between Tinlarebant and Alkeus's therapy.

Answer

Executive Tom Lin explained that DRAGON 2 is primarily for Japanese approval but could serve as a second pivotal study if needed by the FDA, and that they expect a broad GA label due to the convenience of an oral therapy. Chief Scientific Officer Nathan L. Mata detailed that Tinlarebant reduces all toxic vitamin A byproducts, including all-trans retinal, whereas Alkeus's drug increases vitamin A levels, which he believes is a critical mechanistic advantage for Tinlarebant.

Michael Okunewitch of Maxim Group inquired about Ocugen's strategy for future deals, such as regional licenses or spin-outs, following the OrthoCelix and Korea agreements. He also asked for details on the Stargardt Phase 2/3 trial, including the number of sites and potential enrollment challenges, and sought context on what a 27% reduction in geographic atrophy (GA) lesion growth means for patients' vision.

Answer

CEO Shankar Musunuri confirmed that Ocugen is continuously exploring regional partnership opportunities for all its gene therapy programs. Chief Medical Officer Dr. Huma Qamar stated that the Stargardt trial has 15 centers, is on track for a 2027 BLA filing, and faces no anticipated enrollment challenges due to the large patient population and lack of approved treatments. Chief Scientific Officer Dr. Arun Upadhyay explained that the 27% lesion growth reduction in GA is significant and expected to preserve and enhance photoreceptor function, ultimately leading to gains in functional vision for patients over time.

Michael Okunewitch inquired about the enrollment status and timeline for the OCU400 liMeliGhT study, the manufacturing capabilities required for filing and commercialization, and the expected timing for the next update on the OCU200 program.

Answer

Chief Medical Officer Dr. Huma Qamar confirmed the OCU400 trial is on track for enrollment in the first half of 2025, aligning with BLA submission plans. CEO Dr. Shankar Musunuri and CSO Dr. Arun Upadhyay detailed the manufacturing strategy, noting process validations are on target for this year, with initial supply from partner CanSino Bio before transitioning to their in-house Malvern facility. Dr. Musunuri also stated that the OCU200 Phase I trial should complete in late 2025, with a data update expected before year-end.

Michael Okunewitch of Maxim Group asked about Ocugen's financial runway, the timeline for data from the OCU200 DME program, and the funding status for the OCU500 COVID-19 vaccine trial.

Answer

CEO Dr. Shankar Musunuri confirmed the current cash runway guidance already accounts for the newly planned OCU410ST Phase II/III study. Chief Medical Officer Dr. Huma Qamar stated that safety and efficacy data for the OCU200 Phase I trial are expected by the end of 2025. Dr. Musunuri also affirmed that the NIAID remains on track to initiate the Phase I trial for OCU500.

Michael Okunewitch of Maxim Group asked about MindMed's intellectual property strategy in the context of potential partnerships and inquired whether the company is exploring an accelerated approval pathway for its lead asset.

Answer

CEO & Board Director Robert Barrow expressed strong confidence in the company's IP strategy and patent portfolio. Regarding accelerated approval, he noted that the existing Breakthrough Therapy Designation already offers avenues for acceleration and that the company is exploring all options, while also emphasizing the importance of providing a robust and comprehensive data package from its two pivotal trials.

Michael Okunewitch from Maxim Group asked about MindMed's intellectual property strategy in the context of potential partnerships and whether the company is exploring an accelerated regulatory pathway.

Answer

CEO Robert Barrow expressed high confidence in the company's IP strategy and patent portfolio. Regarding regulatory pathways, he noted that their Breakthrough Therapy Designation already offers opportunities for acceleration, which they are exploring, but stressed the primary goal is to deliver a robust and comprehensive data package from their two pivotal GAD trials.

Michael Okunewitch from Maxim Group inquired about the company's cash runway, asking if a potential confirmatory MDD study would run concurrently with other programs and if its costs are factored into current financial projections.

Answer

CEO Robert Barrow confirmed that all planned development activities, including the full MDD program, are factored into their financial projections and cash runway guidance, which extends into 2027. He noted that while they are mindful of operational efficiencies, they are also being financially prudent, and the precise timing of the second MDD study will be informed by ongoing progress and regulatory interactions.

Michael Okunewitch, on behalf of Jason McCarthy, asked if a competitor's data delay poses a risk to the expansion of delivery infrastructure for psychedelics and whether MM-120's lack of a psychotherapy component could broaden its potential delivery settings.

Answer

Executive Robert Barrow expressed confidence in MindMed's ability to shape the market and set the standard for real-world delivery, regardless of other organizations' progress. He affirmed that the decision to eliminate the psychotherapy component was intentional and by design, specifically to maximize patient access and enable a broader impact.

Michael Okunewitch of Maxim Group questioned what defines a successful outcome in the OPC1 DOSED study, the nature of the 'immediate use' formulation, and if Roche's testing of new surgical devices for OpRegen was a new development.

Answer

CEO Brian Culley clarified that success in the DOSED study is primarily the safe and effective use of the new, more convenient external delivery device. He confirmed 'immediate use' is synonymous with their 'thaw-and-inject' formulation, which simplifies preparation. He also confirmed that Roche's evaluation of two new proprietary surgical devices is a recent, positive development for the OpRegen program.

Michael Okunewitch of Maxim Group asked if the FDA would require a safety run-in for the OPC1 DOSED study before full enrollment could begin. He also questioned the company's capital plans and whether there was consideration to accelerate the ReSonance auditory neuron program given the recently strengthened balance sheet.

Answer

CEO Brian Culley confirmed the DOSED study has a staged enrollment process, starting with thoracic injury patients (AIS-A) to establish safety before opening to broader enrollment. Regarding capital allocation, he stated that while the ReSonance program is important, the company remains prudent with spending in the current market, preferring to wait for a potentially more attractive cost of capital after further advancement of the lead OpRegen program.

Michael Okunewitch from Maxim Group asked if Lineage would initiate the OPC1 DOSED study before securing the CIRM grant and whether the development process for in-house programs like ANP1 is expected to be faster due to prior learnings.

Answer

CEO Brian Culley confirmed they would balance moving the OPC1 study forward with prudent cash management, performing necessary start-up activities while awaiting the CIRM grant portal to reopen to maximize potential reimbursement. He responded with an emphatic "Absofreakinglutely" that in-house programs like ANP1 benefit from a massively accelerated development timeline, citing the ability to go from program initiation to animal testing in 12 months by applying learnings from OpRegen.

Michael Okunewitch of Maxim Group asked about the company's strategy for advancing preclinical programs like ANP1 into IND-enabling studies, considering the focus on careful spending in the current market. He also requested more color on the type of manufacturing data for ANP1 that will be presented at an upcoming conference.

Answer

CEO Brian Culley described the company's strategy as finding an 'efficient frontier' of spending. He stated they are making modest but impactful investments in the pipeline to build value while awaiting key OpRegen data, which they hope will lead to more affordable capital for larger trials later. Regarding the ANP1 data, Culley clarified that the upcoming presentation will focus on process development metrics like scale and purity, not functional data from the ongoing animal model study.

Asked where the company is making its initial inroads in the AYA setting (NCI centers vs. community) and whether the operating hours of community infusion centers remain a challenge for adoption.

Answer

The company is seeing growth in both NCI centers and large community practices. The challenge of limited infusion center hours is being addressed by the enhanced Fennec HEARS program, which facilitates seamless home health administration of PEDMARK, an area they see as a big opportunity.

Michael Okunewitch asked about the company's partnering strategy for Japan, specifically whether data from the STS-J01 trial is required to secure a partner. He also inquired about how Fennec prioritizes its commercial efforts for the AYA segment between community centers and the major academic institutions.

Answer

CEO Jeffery Hackman stated that while having the final data from the Japanese trial is preferable for partnering, discussions with potential partners have already begun. He believes positive data signals will enhance the value of a potential deal. Regarding AYA commercialization, Hackman explained that patients are distributed across both academic and community settings, making both critical targets. He emphasized the significant opportunity in raising awareness among community physicians who are often unaware of PEDMARK as a treatment option.

Speaking for Jason McCarthy, Michael Okunewitch of Maxim Group asked about Bionano's plans for new customer acquisition, the specificity of the new Category 1 CPT code for OGM, and the mechanics of how VIA software adoption increases utilization and menu expansion.

Answer

CEO Erik Holmlin responded that new customer acquisition will be selective, focusing on sites that fit the 'routine use' profile where acquisition costs are acceptable, particularly catalyzed by the new CPT code. He clarified the CPT code is specific to hematologic malignancies. Dr. Holmlin detailed that VIA software drives utilization by automating the time-consuming manual processes of variant curation and reporting, thereby increasing lab capacity to run more samples and adopt new applications.

Speaking on behalf of Michael Okunewitch, Chad from Maxim Group asked for a comparison of the weight loss and speed of titration for ProGen's PG-102 against Zealand's competing GLP-1/GLP-2 agonist.

Answer

Executive Talat Imran cautioned against direct cross-trial comparisons but highlighted that ProGen's preclinical data showed more sustained weight loss for PG-102. He emphasized that a key advantage of PG-102, an FC-fusion protein, is its rapid titration schedule, which allowed it to reach the maximum dose within a month in clinical studies, a potentially significant patient benefit.

Michael Okunewitch, on behalf of Jason McCarthy, asked about the company's pipeline plans beyond TRD, such as for PTSD and anorexia, and whether the market adoption of SPRAVATO could help prepare the market for COMP360.

Answer

CEO Kabir Nath stated that the company's primary development focus is on TRD and PTSD, with data from a completed anorexia study expected in 2025. An executive confirmed that they believe SPRAVATO's success is beneficial, as it has educated healthcare providers on TRD and has only captured a small fraction of the large addressable patient population, indicating a significant opportunity for COMP360.

Michael Okunewitch from Maxim Group sought clarification on the nature of the 'additional registrational opportunity' from Cohort 5 of the GOBLET study and asked about the expected size of the pivotal study in breast cancer.

Answer

Chief Medical Officer Dr. Tom Heineman explained that the GOBLET Cohort 5 data is considered signal-finding and would not support an accelerated approval on its own. However, positive results could lead to the combination being evaluated in a larger platform study, similar to the GCAR collaboration. For the breast cancer study, he estimated a size of sub-200 patients, following the model of initial approvals for IBRANCE and ENHERTU.

Michael Okunewitch of Maxim Group asked about the strategic goal of the EMBARK program and the rationale for shifting focus from the initial sublingual psilocybin program to earlier-stage analogs. He also sought clarification on whether the Phase I/IIa trial for CYB003 would require a healthy volunteer cohort before enrolling MDD patients.

Answer

CEO Douglas Drysdale explained that EMBARK is being developed as an open-source tool to support its proprietary drug candidates, not to get a separate psilocybin program approved. He stated the strategic shift to CYB003 was driven by its superior product profile, which offers a better patient experience and stronger IP, justifying the development timeline. He also confirmed the plan is to move directly into MDD patients for the Phase I/IIa trial, which would provide preliminary PK and safety data by year-end.