Precision BioSciences (DTIL) Conference May 21, 2025 03:15 PM ET Speaker0 Welcome everyone to this session of our May twenty twenty five MicroCap conference. I'm Alex Hanman, and I serve as an equity research analyst here at Sidoti and Company. Today, we're pleased to be in conversation with CFO Alex Kelly and VP of Investor Relations, Naresh Thanna, of Precision Biosciences, ticker d t I l. During the presentation, please feel welcome to submit questions using the Zoom Q and A interface at the bottom of yo ...

Summary of Clene (CLNN) Conference Call - May 21, 2025 Company Overview - Clene Inc. (CLNN) is a late clinical stage biopharmaceutical company focused on improving mitochondrial health and protecting neuronal function to treat neurodegenerative diseases [1] Core Drug and Therapeutic Areas - The primary drug discussed is CMA-eight, targeting neurodegenerative diseases, specifically amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS) [3][4] - CMA-eight has shown potential in remyelination and neuron protection in MS, with data presented at the American Academy of Neurology [4] Regulatory Pathway and FDA Meetings - Clene has a meeting scheduled with the FDA in June to discuss the statistical analysis plan for neurofilament data, a biomarker indicative of neuron damage [6][10] - The company is preparing to file a New Drug Application (NDA) under the accelerated approval pathway, potentially by Q4 2025 [12][13] - The FDA's evaluation will include data from a $45 million NIH-funded compassionate use program involving 170 participants with ALS [8][9] Clinical Trial Insights - The ALS study duration was shorter (6-9 months) due to the urgent needs of patients, while the MS study is planned for 12 months [19][20] - The ALS study focuses on global functional scales and survival, whereas the MS study looks at visual and cognitive changes [20] Current Market Landscape - The main approved drug for ALS is riluzole, which has been available since 1995, providing limited clinical benefit [21][22] - Clene's drug aims to improve neuronal energy and function, potentially offering significant advantages over existing treatments [23] Market Potential and Comparables - Clene believes it could achieve sales of up to $400 million in the first year post-approval, which would significantly increase its market capitalization [24][25] - A comparable ALS company previously achieved a market cap of over $2 billion after FDA approval, highlighting the potential for Clene [24] Additional Insights - The company is also exploring strategic partnerships related to its MS program, although commercialization for MS is still years away [16] - The focus remains primarily on ALS due to a quicker regulatory path [16] Conclusion - Clene is positioned to make significant advancements in the treatment of neurodegenerative diseases, with ongoing discussions with the FDA and promising clinical data supporting its drug candidates [26]

CorMedix (CRMD) 2025 Conference Summary Company Overview - CorMedix is a publicly traded, commercial stage biotech company with a market cap of approximately $900 million [4] - The lead product, DefendCath, is designed to reduce the risk of catheter-related bloodstream infections in patients undergoing chronic hemodialysis [4] Product Details - DefendCath is a catheter lock solution combining heparin and a proprietary chemical entity, tyrolidine, which has broad-spectrum antimicrobial activity [5] - A phase three clinical study showed a 71% reduction in the risk of catheter-related bloodstream infections [5] - The product was commercialized in mid-2024, with net sales guidance of approximately $70 million for the first half of 2025 [6] Market Position and Competitive Landscape - DefendCath is the only FDA-approved catheter lock solution demonstrating antimicrobial activity [8] - The outpatient hemodialysis market is concentrated among five major dialysis operators, with CorMedix having contracts with four of them [12] - Initial adoption by US Renal Care has been successful, with onboarding of additional operators [13] Financial Guidance and Sales Trajectory - The company provided guidance for the first half of 2025, indicating strong sales performance and growth in utilization among existing customers [16][17] - The company is cautious about providing full-year guidance due to variability in the onboarding of large dialysis operators [15] Inpatient and Outpatient Dynamics - In April, 6% of unit shipments were directed to the inpatient segment, with expectations for growth as new accounts are added [20][21] - The partnership with Syneos for inpatient sales is progressing well, with a focus on quality account management [19] Reimbursement Landscape - The current TDAPA reimbursement process is a five-year adjustment to the dialysis bundle, incentivizing innovative products [22] - CorMedix is conducting a real-world evidence study to demonstrate the cost-effectiveness of DefendCath, aiming for sustainable reimbursement [24] - The company plans to contract directly with Medicare Advantage plans for long-term reimbursement strategies [25] Total Addressable Market (TAM) and Future Opportunities - The TAM for total parenteral nutrition (TPN) is estimated between $500 million and $750 million, with potential peak sales for DefendCath projected at $520 million [29] - A new study for TPN has commenced, with the first patient dosed, aiming for commercialization by late 2027 or early 2028 [26] Operational Efficiency and Cost Structure - CorMedix operates with a lean SG&A structure, with no expected significant increases in spending for commercialization [47] - R&D spending for the TPN study is projected at $1.2 million [47] Key Metrics and Future Outlook - The company aims to demonstrate a material impact on infection rates and potentially mortality rates through ongoing studies [41] - Future guidance on revenue expectations is anticipated in the upcoming quarterly report [42] Conclusion - CorMedix is positioned for growth with DefendCath, leveraging its unique market position and ongoing studies to enhance its product offerings and reimbursement strategies [56]

Blade Air Mobility (BLDE) Conference Summary Company Overview - Blade Air Mobility has been operational for eleven years, initially focusing on helicopter passenger transport in New York and expanding to European markets such as Monaco, Nice, Cannes, and Geneva [1][2] - The company aims to transition from helicopters to electric vertical takeoff and landing (eVTOL) aircraft, leveraging an asset-light model that allows for future aircraft swaps [2][4] Industry Dynamics - The transition to eVTOL is expected to occur soon, with potential commercialization timelines for manufacturers like Adobe and Archer projected for late 2025 to early 2026 in the Middle East and late 2027 to early 2028 in the U.S. [10][11] - Blade is well-positioned to capitalize on this transition due to its established infrastructure and technology stack, which includes heliports in key markets [12][14] Key Business Segments Passenger Transport - Blade has successfully penetrated the passenger market, offering competitive pricing that has attracted a significant customer base [11] - The company has broken through pricing barriers, offering a $95 annual airport pass for frequent flyers [11] Medical Transport - Blade is the largest air transporter of human organs in the U.S., with a business generating approximately $150 million, surpassing its passenger transport revenue [5][6][37] - The medical segment is expected to grow in double digits, with a target margin expansion from 15% to high teens due to increased control over owned jets [34][35] - Blade holds about 30-35% of the organ transport market, indicating significant growth potential [37] Technological Advancements - The company is exploring the integration of AI in operations, aiming for enhanced safety and efficiency in future eVTOL aircraft [21][22] - Blade does not manufacture its own aircraft but collaborates with established manufacturers like Bell and Airbus [23] Financial Performance - Blade is ahead of its profitability projections, with adjusted EBITDA expected to exceed $10 million in 2025, up from just over $1 million last year [43][45] - The company maintains a strong financial position with $120 million in cash and zero debt, focusing on strategic acquisitions in the medical sector [40][42] Strategic Initiatives - Blade is actively seeking acquisitions that can enhance its medical transport capabilities and leverage existing hospital relationships [40][42] - The company is involved in various high-profile events to increase brand recognition and attract new customers, such as the Ryder Cup and major music festivals [28][29] Conclusion - Blade Air Mobility is strategically positioned to lead the transition to eVTOL aircraft while maintaining a strong foothold in the medical transport sector, showcasing impressive financial growth and operational efficiency [43][45]

Summary of NCS Multistage (NCSM) Conference Call - May 21, 2025 Company Overview - NCS Multistage is a technology-focused oil field services and equipment company, primarily selling to exploration and production (E&P) companies such as Chevron, Conoco, Oxy, BP, and others [3][4] - The company competes with larger, established firms like Schlumberger, Halbern, and Core Lab [4] Core Business Strategies - NCS Multistage has three core business strategies: 1. **Build on Leading Market Positions**: Focus on fracturing systems, Canadian completions, and tracer diagnostics [8] 2. **Capitalize on Offshore and International Opportunities**: International markets are growing faster than North America, allowing for stronger customer relationships based on technical characteristics [9] 3. **Commercialize Innovative Solutions**: Understanding customer needs and delivering solutions that provide tangible value [9] Financial Performance - Revenue grew by approximately 14% or $20 million in 2024, with expectations for continued growth in 2025 despite a challenging market [10] - Gross margin is around 40%, with an improvement of approximately 250 basis points in 2024 compared to the previous year [11] - The company operates with a capital-light model, expecting to convert about 50% to 60% of adjusted EBITDA to free cash flow [11] Market Environment and Challenges - The market is expected to be flat or slightly declining in the U.S., with slight growth in Canada and opportunities outside North America [15] - Spot oil prices are about $10 per barrel lower, leading to reduced drilling and completion spending by customers [16] - Customer consolidation in Canada may lead to fewer wells being drilled, reducing market opportunities [16] Cash Management and Future Plans - As of March 31, the company had approximately $23 million in cash and $27 million available through a revolving credit facility [13] - Priorities for cash usage include investing in internal R&D for organic growth and exploring strategic acquisitions [20][21] - If no suitable M&A opportunities arise, the company may consider returning capital to shareholders [21] Conclusion - NCS Multistage presents a compelling investment opportunity with a strong organic growth track record, innovative technology, and a robust balance sheet [12] - The company is focused on navigating market uncertainties while pursuing attractive commercial opportunities [19]

Summary of GRI Bio Conference Call Company Overview - **Company**: GRI Bio - **Focus**: Clinical stage biotech company targeting inflammatory fibrotic and autoimmune diseases through regulation of invariant natural killer T (NKT) cells [2][5] Core Program - **Lead Program**: GRI o six two one (GRI621) - **Type**: Small molecule inhibitor of type one invariant NKT cells - **Indication**: Treatment of idiopathic pulmonary fibrosis (IPF) - **Current Stage**: Phase 2a study with top line data expected in Q3 2025 [6][17] Mechanism of Action - **NKT Cells**: Critical lymphocytes involved in chronic inflammation and fibrosis - Inhibition of NKT cells shown to be therapeutic across various preclinical models of fibrosis [8][9] - Increased NKT cell activity correlates with worsening fibrotic disease, suggesting potential as a biomarker [9][12] Clinical Trial Details - **Trial Design**: - 36 patients, randomized 2:1 (GRI621 vs. placebo) - Duration: 12 weeks, with patients allowed to continue background therapy [18][19] - Primary endpoints: Safety, tolerability, pharmacokinetics, biomarkers of fibrosis, and pulmonary function tests [19][25] - **Enrollment Status**: Approximately 80% enrolled [20] Interim Results - **Recent Findings**: Positive recommendation from the Independent Data Monitoring Committee (IDMC) for continued trial [24] - **Early Data**: Indication of antifibrotic signal observed in initial 12 patients after two weeks [24] Challenges in IPF Treatment - **Current Landscape**: - Existing treatments (nantenadib and profinidone) slow lung function decline but do not improve overall survival [32] - Significant side effects limit patient compliance [32] - **Focus on Upstream Targets**: GRI Bio aims to address earlier stages in the inflammatory cascade rather than late-stage targets like TGF beta [33][34] Market Potential - **Market Size**: IPF is an orphan indication; nantenadib generated approximately $4 billion in sales last year [38] - **Competitive Landscape**: Other drugs in development face challenges, including drug-drug interactions and tolerability issues [39][40] Future Considerations - **Regulatory Pathways**: Potential for expedited approval due to existing safety data from other indications [47] - **Pipeline Opportunities**: GRI Bio is developing additional programs, including GRI o eight zero three (GRI803) targeting systemic lupus erythematosus [49][50] Conclusion - GRI Bio is positioned to address significant unmet needs in the treatment of IPF through innovative targeting of NKT cells, with ongoing clinical trials and a robust pipeline of future therapies [52]

Summary of GRI Bio Conference Call Company Overview - **Company**: GRI Bio - **Focus**: Clinical stage biotech company targeting inflammatory fibrotic and autoimmune diseases through regulation of NKT cells [2][5] Lead Program - **Product**: GRI o six two one - **Type**: Type one invariant natural killer T cell antagonist - **Indication**: Treatment of idiopathic pulmonary fibrosis (IPF) - **Current Stage**: Phase 2a study with top line data expected in Q3 2025 [6][18] Mechanism of Action - **NKT Cells**: Critical lymphocytes involved in chronic inflammation and fibrosis - **Therapeutic Approach**: Inhibiting NKT cell activity to interrupt disease progression and restore immune homeostasis [5][8] - **Biomarker Potential**: Increased NKT cells correlate with worsening fibrotic disease, suggesting their role as a useful biomarker [9][39] Clinical Trial Details - **Trial Design**: - 36 patients, 2:1 randomization (24 on GRI o six two one, 12 on placebo) - Duration: 12 weeks, with primary endpoints focusing on safety and tolerability [16][18] - **Enrollment Status**: Approximately 80% enrolled, with interim data expected in Q2 2025 [18][22] Data and Results - **Interim Analysis**: Positive recommendation from the Independent Data Monitoring Committee (IDMC) after 12 patients completed two weeks [21] - **Early Signals**: Antifibrotic signal observed in pro-collagen type III (pro C3) after two weeks [21][26] Market Context - **Market Size**: IPF is an orphan indication; Nintanadib, one of the approved drugs, generated approximately $4 billion in sales last year [35] - **Challenges**: Current treatments slow disease progression but do not improve overall survival; significant side effects limit patient compliance [29][30] Competitive Landscape - **Current Treatments**: Nintanadib and Perfinetone are the two approved drugs, both with limitations in efficacy and tolerability [35][36] - **Future Positioning**: GRI o six two one aims to address the disease earlier in the inflammatory cascade compared to existing therapies [30][31] Regulatory Considerations - **Expedited Approval**: Potential for a single registration trial due to existing safety data from other indications [43][44] Pipeline Opportunities - **Future Programs**: GRI o eight zero three targeting type two NKT cells for systemic lupus erythematosus; additional 500 compounds in the library for future development [46][47] Conclusion - GRI Bio is positioned to potentially disrupt the IPF treatment landscape with GRI o six two one, focusing on early intervention in the inflammatory process and leveraging NKT cells as biomarkers for disease progression [5][39]

Summary of Aardvark Therapeutics (AARD) Conference Call Company Overview - **Company**: Aardvark Therapeutics (AARD) - **Focus**: Development of ARTOR 101, a therapeutic targeting appetite regulation through gut hormones Key Points Therapeutic Mechanism and Approach - ARTOR 101 was developed to inhibit bitter taste receptors to suppress appetite, initially theorized to reduce calorie intake by using a bitter compound that is non-toxic [3][4] - The drug stimulates the release of gut peptide hormones such as CCK, GLP-1, GLP-2, and possibly PYY, which act locally and are rapidly degraded [5][6] - The distinction between hunger (pain avoidance pathway) and appetite (pleasure-seeking pathway) is emphasized, with ARTOR 101 allowing users to skip meals without feeling hungry [8][9][12] Indication and Market Opportunity - The primary indication is for Prader-Willi syndrome, characterized by hyperphagia, which severely impacts patients and families [18][19] - Recent approval of Celino Therapeutics' drug VICAT (DCCR) for hyperphagia presents competition, but Aardvark believes their approach directly addresses the cause of hyperphagia, offering a significant market opportunity [20][21] Clinical Trials and Data - A phase two trial showed a drop of nine points on the HQCT scale, indicating a robust signal of efficacy [23][24] - The phase three trial design includes dose escalation and stricter inclusion criteria to ensure a more reliable data package [36][39] - The trial aims for a clinically meaningful change of five points on the HQCT scale, with 90% power requiring 32 patients per arm [46][47] Regulatory Pathway - The FDA indicated that one statistically significant study could suffice for submission as a pivotal trial [54][55] - Ongoing safety data collection from open-label extensions and additional trials in hypothalamic obesity and general obesity [55] Competitive Landscape - Aardvark anticipates that ARTOR 101 will replace rather than combine with existing treatments due to pricing and efficacy concerns [57][58] - The company is aware of the challenges faced by existing treatments, with a significant dropout rate observed in Celino's studies, indicating potential limitations in efficacy [58][59] Future Directions - Plans to explore hypothalamic obesity and general obesity, leveraging the drug's mechanism to enhance CCK signaling to the hypothalamus [61][63] - A trial comparing ARTOR 101 with GLP-1 drugs is planned to assess weight loss potential and rebound effects post-treatment [65][66] Additional Insights - The importance of consistent patient rating for HQCT scale assessments was highlighted, as variability can affect data reliability [38] - The potential for ARTOR 101 to suppress ghrelin levels, which rise with GLP-1 treatments, could provide a unique advantage in managing weight rebound [67] This summary encapsulates the key discussions and insights from the Aardvark Therapeutics conference call, focusing on the company's innovative approach to appetite regulation and the clinical and market strategies moving forward.

Summary of Cora Bio Conference Call Company Overview - **Company**: Cora Bio - **Industry**: Biotechnology, specifically focusing on genetic medicines for chronic diseases [1][2] Core Points and Arguments - **Mission and Strategy**: Cora Bio aims to develop genetic medicines targeting complex chronic diseases by leveraging pharmacology and genetic insights. The focus is on creating a toolbox for biological pathway activation that is not available with small molecules [3][4] - **RNA Editing Technology**: The company utilizes RNA editing through oligonucleotides to modify specific genes, aiming for high levels of editing (above 50%) to restore normal protein function in patients [4][5] - **Pipeline Development**: Cora Bio has a three:two:one strategy, planning to advance three candidates into clinical trials across two tissue types. The third indication is expected to target the central nervous system (CNS) for ALS [6][7] - **Regulatory Environment**: The company has engaged with the FDA, receiving orphan drug designation and conducting pre-IND meetings. There is concern about the increasing regulatory bar but confidence in the data driving approval processes [10][12][13] - **Preclinical Data**: Cora Bio presented promising preclinical data showing significant editing efficiency (60-65%) in animal models, which translates well to protein levels in circulation [18][20] - **Lipid Nanoparticle Delivery**: The company uses lipid nanoparticles for drug delivery, which have shown safety in patients with liver conditions. The focus is on optimizing dosing to achieve therapeutic protein levels [21][23][25] Important but Overlooked Content - **Patient Population Insights**: The company discussed the baseline liver function levels in patients with ZZ mutations, indicating that many have normal liver function, which may mitigate concerns about toxicity from lipid nanoparticles [22][23] - **Dosing Strategy**: Cora Bio aims for a dosing frequency of every three to six weeks, with the goal of achieving therapeutic protein levels that are comparable to normal individuals [26] - **Regulatory Pathway Confidence**: The company believes that demonstrating protein levels close to normal and achieving significant editing levels could support discussions for accelerated approval with regulators [39][40] - **Financial Position**: Cora Bio has a cash runway into 2027, with $139 million on the balance sheet and potential milestones from collaborations, indicating a solid financial foundation for ongoing development [47]

Matrix Service Company (MTRX) Conference Call Summary Company Overview - **Company Name**: Matrix Service Company (MTRX) - **Industry**: Engineering and construction services for the oil and gas, utility, and industrial markets - **Key Executives Present**: CEO John Hewitt, CFO Kevin Cavanaugh, Director of Investor Relations Kelly Smythe - **Core Values**: Emphasis on safety and operational excellence [1][4] Financial Highlights - **Recent Performance**: - Q3 fiscal 2025 revenue increased by 21% to $200 million compared to $166 million in Q3 fiscal 2024 [17] - Gross margin improved to 6.4% from 3.4% year-over-year [17] - Net loss reduced to $3.4 million ($0.12 per share) from $14.6 million ($0.53 per share) in the previous year [18] - Adjusted EBITDA reached breakeven compared to a loss of $10 million in the prior year [18] - **Revenue Guidance**: Projected revenue for fiscal 2025 is between $770 million and $800 million, up from $728 million in fiscal 2024 [8] Backlog and Growth Opportunities - **Backlog**: - Current backlog stands at $1.4 billion, with project awards of $301 million in Q3, resulting in a book-to-bill ratio of 1.5 [13] - Backlog has doubled since fiscal 2022, with a significant portion attributed to storage and terminal solutions [13][14] - **Opportunity Pipeline**: - A robust $7 billion pipeline of project opportunities, with many expected to be bid and awarded in the next 12 to 18 months [15] - Approximately 90% of revenue comes from recurring customers, providing long-term visibility for growth [15] Market Position and Strategy - **Market Focus**: - Specializes in complex infrastructure projects, particularly in LNG, ammonia, and renewable fuels [9][24] - Positioned to capitalize on multiyear spending cycles in energy and industrial markets [5][12] - **Competitive Environment**: - Favorable conditions for larger projects with fewer competitors capable of handling specialty vessel work [28] - Shift from crude-related storage to specialty vessel-related storage has been noted [24] Financial Management and Capital Allocation - **Balance Sheet Strength**: - Zero debt with available liquidity of $247 million [19] - Focus on managing working capital effectively, with net cash provided by operating activities of $31.2 million in Q3 [19] - **Capital Allocation Strategy**: - Emphasis on returning to profitability and considering strategic acquisitions or stock buybacks in the future [34] Long-term Goals and Margin Expectations - **Margin Targets**: - Long-term gross margin targets remain in the range of 10% to 12% [21] - Weighted average margin of current backlog is above 10%, supporting long-term margin expectations [26][27] - **Revenue Growth**: - Anticipated revenue growth to continue, with a target of reaching $250 million per quarter to leverage construction overhead effectively [29][30] Conclusion - **Outlook**: - Matrix Service Company is well-positioned for growth with a strong backlog, disciplined capital allocation, and a focus on high-margin projects [20][21] - **Market Confidence**: - Executives express confidence in the company's ability to achieve long-term targets and navigate the evolving market landscape [41]