Core Viewpoint - RenovoRx, Inc. is conducting its Q1 2025 earnings conference call, highlighting its financial and operational performance, with key leadership present to discuss the company's progress and future outlook [1][2]. Group 1: Company Leadership and Structure - The conference call features RenovoRx's leadership team, including Dr. Ramtin Agah (Founder and Chief Medical Officer), Shaun Bagai (CEO), and Ronald Kocak (VP Controller and Principal Accounting Officer) [2]. Group 2: Forward-Looking Statements - The company emphasizes that statements made during the call may be considered forward-looking, reflecting management's current views on market conditions and potential outcomes of clinical trials [3][4]. - Management's forward-looking statements are based on current plans and assumptions, which are subject to various risks and uncertainties [3].

Group 1 - The conference call is focused on BiomX's first quarter 2025 financial results and updates on business and programs [3] - The quarterly report on Form 10-Q will be filed with the Securities and Exchange Commission, and a press release was made available at 6:30 a.m. Eastern time [3] - A replay of the conference call will be accessible in the Investors section of the company's website [3] Group 2 - The call includes forward-looking statements regarding the company's cash sufficiency, pipeline, clinical trial designs, expected discussions with regulatory agencies, and potential benefits of product candidates [4] - The company emphasizes that past and current clinical trials do not guarantee future results [5]

Financial Data and Key Metrics Changes - For Q1 2025, the company reported R&D expenses of $9.4 million, an increase from $6.8 million in the same period last year, primarily due to share-based compensation and higher clinical trial expenses related to the PHOENIX trial [24] - General and administrative expenses rose to $6.1 million from $1.6 million year-over-year, also attributed to share-based compensation [24] - The net loss for the quarter was $14.3 million, compared to a net loss of $7.9 million in the prior year [25] - Operating cash outflow was approximately $8.3 million, with a cash increase of $12.3 million for the quarter, resulting in a total cash position of $157.4 million at the end of Q1 [26] Business Line Data and Key Metrics Changes - The company is advancing its lead product, Tenereband, which is in global Phase III trials for Stargardt disease and geographic atrophy, with promising interim results reported [4][5] - The DRAGON trial for Stargardt disease has maintained its sample size at 104 subjects after an interim analysis, with the Data Safety Monitoring Board recommending submission for regulatory review [6][14] - The PHOENIX trial for geographic atrophy has enrolled 464 subjects to date, with expectations to complete enrollment of 500 subjects by Q3 2025 [7][21] Market Data and Key Metrics Changes - The company is uniquely positioned in the market as there are currently no approved treatments for Stargardt disease and no approved oral treatments for geographic atrophy, highlighting a significant unmet need [5] - The average age of participants in the PHOENIX trial is around 80 years, indicating a focus on elderly patients [33] Company Strategy and Development Direction - The company aims to position Tenereband as the first oral treatment for degenerative retinal diseases, with a strong focus on advancing clinical trials and regulatory submissions [8] - The management is actively engaging with regulatory agencies to finalize development plans for Stargardt disease and monitor the impact of drug pricing policies on market prospects [36][47] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism regarding the interim results of the DRAGON trial, indicating a potential for significant efficacy based on the Data Safety Monitoring Board's recommendations [14][43] - The company expects to maintain a cash runway for four years, allowing it to complete all current clinical trials [26] Other Important Information - The company has received various designations for Tenereband, including rare pediatric disease and fast track designations in the US, and pioneer drug designations in Japan, underscoring the drug's potential impact [4][5] Q&A Session Summary Question: Can you provide data about the discontinuation rates and enrollment in the PHOENIX trial? - The dropout rate in the PHOENIX trial is approximately 20%, which is significantly lower than rates reported in other studies [32][33] Question: Any updates on regulatory meetings for Stargardt disease? - The company is scheduling meetings with regulators to discuss the development plan for Stargardt disease [36] Question: Can you discuss recent interactions with the FDA and any perceived regulatory risk? - The company has not met with the FDA yet but plans to do so soon, expressing confidence that data will support their case despite changes at the agency [39][40] Question: What is the goalpost for the Phase III data? - The study is powered to detect a 35% treatment effect between placebo and active treatment, with expectations based on interim results [42][43] Question: Will operating expenses continue to rise in 2025? - Operating expenses are expected to be slightly higher than Q1 levels due to upcoming milestones in clinical studies [48] Question: Is the increase in stock compensation expected to continue? - The increase in stock compensation will depend on the allocation of expenses, but it is not expected to be as high moving forward [56] Question: How is the supply chain structured geographically? - Tenereband is manufactured in the US and other geographies, mitigating potential tariff impacts [58]

Financial Data and Key Metrics Changes - As of March 31, 2025, the company had cash and cash equivalents of $8 million, which is expected to fund operations into the third quarter of 2025 [15] - Research and development expenses for Q1 2025 were $6.1 million, up from $5.1 million in Q1 2024, driven by the ongoing SHIELD II Phase III trial [16] - The net loss for Q1 2025 was $8.3 million, compared to a net loss of $6.4 million in Q1 2024 [16] Business Line Data and Key Metrics Changes - The company is focused on the SHIELD II Phase III trial for DPLEX100, which has recently concluded enrollment with 800 patients, following a recommendation from the Data Safety Monitoring Board [5][6] - The anticipated top line data from the SHIELD II trial is expected by the end of the current quarter [15] Market Data and Key Metrics Changes - The total addressable market for DPLEX100 in the U.S. is estimated to be over 12 million surgeries annually, with approximately 4.4 million being abdominal soft tissue surgeries [11] - The company identified four groups of surgeons as potential users for DPLEX100, indicating a significant market opportunity [12] Company Strategy and Development Direction - The company aims to submit a New Drug Application (NDA) in early 2026, with preparations for regulatory submissions already underway [7][8] - The strategy includes finding a U.S. partner with an existing dedicated hospital product sales force to maximize sales potential [9][13] - An exclusive licensing agreement is already in place with Advanced Pharma for commercialization in Europe [14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term prospects of DPLEX100 and the potential to transform the surgical landscape [47] - The company is actively preparing for regulatory submissions and engaging in partnership discussions as they await SHIELD II trial results [8][13] Other Important Information - The company has received Fast Track and Breakthrough Therapy designations for DPLEX100, which may expedite the regulatory process [7] - The company is in advanced discussions with multiple potential partners in the U.S. as they approach pivotal data readout [13] Q&A Session Summary Question: Pricing strategy for DPLEX100 - Management has not finalized the pricing strategy but anticipates an average of 2.5 vials per procedure at a preliminary price of $600 per vial [20] Question: NDA filing requirements - The NDA will consist of three modules, with the company currently finalizing the CMC and preclinical modules [27] Question: Expectations for top line data - The company expects to report both primary and key secondary endpoints upon unblinding the data [32] Question: Inspection readiness for commercial manufacturing - The company is preparing for FDA inspections and plans to conduct several mock inspections prior to the review [40] Question: Comparison of SSI data pre-COVID and post-COVID - The CDC reported a 3% increase in SSIs in 2023 compared to 2022, marking the first uptick since the COVID pandemic [42]

Core Insights - Soligenix, Inc. is focused on developing and commercializing products for rare diseases with unmet medical needs, reporting recent accomplishments and financial results for Q1 2025 [1][9] Recent Accomplishments - The company anticipates significant milestones, including top-line results in 2026 from a Phase 3 study of HyBryte™ for early-stage cutaneous T-cell lymphoma and results from Phase 2 studies of SGX945 in Behçet's disease and SGX302 in psoriasis in the second half of 2025 [2] Financial Results - For the quarter ended March 31, 2025, Soligenix reported no revenue, a decrease from $0.1 million in the same quarter of 2024, primarily due to a reduction in revenue from a zero-margin grant for HyBryte™ [5] - The net loss for Q1 2025 was $3.2 million, or ($1.06) per share, compared to a net loss of $1.9 million, or ($2.91) per share, in Q1 2024, attributed to increased operating expenses and a decrease in other income [6] - Research and development expenses rose to $2.2 million in Q1 2025 from $1.1 million in Q1 2024, driven by costs related to ongoing clinical trials [7] - General and administrative expenses increased to $1.1 million in Q1 2025 from $1.0 million in Q1 2024, mainly due to higher professional expenses and taxes [8] Cash Position - As of March 31, 2025, the company's cash position was approximately $7.3 million, which is expected to provide sufficient operating runway through December 2025 [3][8]

Financial Data and Key Metrics Changes - The company ended the first quarter with a cash position of $204.1 million, which includes approximately $65.5 million in net proceeds from a public offering closed in January 2025 [23] - Total operating expenses for the quarter were $37.8 million, down 82% from the same period in 2024 [25] - Research and development expenses were $22.1 million compared to $36.6 million in Q1 2024, primarily due to decreased spending on AMX-thirty five and reduced payroll costs [25] - Selling, general and administrative expenses were $15.7 million compared to $57.8 million in Q1 2024, mainly due to decreased payroll and consulting costs [25] Business Line Data and Key Metrics Changes - The company is advancing three potential therapies across four clinical trials, including Avexatide for post bariatric hypoglycemia (PBH), AMX-thirty five for Wolfram syndrome and progressive supranuclear palsy (PSP), and AMX-one hundred fourteen for ALS [7][8] - Avexatide has shown promise in treating PBH, with a pivotal Phase III trial (Lucidity) initiated, and the first participant dosed [10][18] - AMX-thirty five is being evaluated in two indications, with positive Phase II data reported for Wolfram syndrome and ongoing trials for PSP [12][14] Market Data and Key Metrics Changes - There are approximately 160,000 people in the U.S. living with PBH, a condition with no approved treatment options [9][19] - The American Society for Metabolic and Bariatric Surgery reported around 270,000 new bariatric surgeries in 2023, indicating a stable market for potential PBH patients [20] - The company estimates that 8% of patients who undergo bariatric surgery will develop PBH within one to three years [48][50] Company Strategy and Development Direction - The company aims to develop novel therapies for diseases with high unmet needs, focusing on execution across its clinical trials [28] - The company is preparing for a potential commercial launch of Avexatide in 2027 if approved [10] - The company is actively engaging with clinical trial sites and expects to complete recruitment for the Lucidity trial by the end of 2025 [22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving clinical milestones through the end of 2026, supported by a strong cash position [23][24] - The company is focused on educating the medical community and patients about PBH, given the lack of awareness and treatment options [34][46] - Management highlighted the importance of access to treatments for patients suffering from debilitating conditions like PBH [65] Other Important Information - The company plans to present week 48 data from the HELIOS trial in Wolfram syndrome at an upcoming conference [12][28] - The company is preparing for potential FDA discussions regarding trial designs for its various programs [56] Q&A Session Summary Question: Will a large patient education campaign be required for Avexatide? - Management acknowledged the need for education due to the rarity of PBH and the lack of prior treatments, emphasizing the importance of informing both medical professionals and patients [32][34] Question: What is the expected efficacy for the PSP trial? - Management stated the study has 80% power to detect a 30% effect on the PSP rating scale, with multiple endpoints being considered for decision-making [37][39] Question: How many of the 160,000 PBH patients are seeking treatment? - Management indicated that a larger group, potentially up to 1 million, may experience hypoglycemia post-bariatric surgery, but only about 160,000 have persistent symptoms warranting treatment [46][47] Question: What are the remaining questions with the FDA regarding trial design? - Management noted that the week 48 data will inform the Phase III program, but specific details of FDA discussions were not disclosed [56] Question: Will there be subgroup analysis in the Avexatide study? - Management confirmed that the study will focus on a population experiencing frequent hypoglycemic events, with no anticipated step therapy restrictions [61][62] Question: What gives confidence that Avexatide won't cause safety issues? - Management highlighted the good safety profile observed in both non-clinical and clinical data, noting that Avexatide is a competitive antagonist rather than a GLP-1 agonist [70] Question: How does Avexatide compare to other drugs in the space? - Management emphasized Avexatide's strong profile and breakthrough therapy designation, noting that other programs are earlier in development and face hurdles [82][84]

Financial Data and Key Metrics Changes - The company achieved $317 million in revenue for Q1 2025, reflecting a 9% increase on a constant currency basis and a 10% increase on an organic basis compared to the prior year [29] - Adjusted operating income rose to $65 million, up from $53 million in Q1 2024, resulting in an adjusted operating income margin of 20% compared to 18% in the prior year [31] - Adjusted diluted earnings per share increased to $0.88 from $0.73 in Q1 2024, driven by operating income growth [32] - Cash balance at March 31 was $738 million, up from $429 million at year-end 2024, primarily due to the reclassification of $295 million of restricted cash [32][33] Business Line Data and Key Metrics Changes - Cardiopulmonary segment revenue was $176 million, a 15% increase year-over-year, with heart-lung machine revenue growing approximately 30% [11] - Epilepsy segment revenue increased by 4%, with U.S. revenue growth at 2% and Europe and rest of world regions combined growth at 13% [14][16] - The company expects cardiopulmonary revenue to grow 9% to 10% for the full year 2025, up from a previous forecast of 7% to 8% [12] Market Data and Key Metrics Changes - The company reported strong demand for oxygenated products, with growth in the high single digits driven by customer demand and pricing [11] - The U.S. epilepsy business faced procedure deferrals due to a voluntary field safety notification, impacting revenue by less than $2 million [15] - The company anticipates low single-digit growth in the U.S. epilepsy market, with Europe and rest of world regions expected to grow in low double digits [16] Company Strategy and Development Direction - The company is focused on expanding its portfolio into areas of high unmet clinical need, particularly in neuromodulation and OSA [52][43] - The company is investing in additional manufacturing capacity to support growth in cardiopulmonary products and anticipates sustaining double-digit growth in heart-lung machines [13][58] - The company is pursuing Medicare coverage for VNS therapy for treatment-resistant depression, which represents a significant growth opportunity [27][43] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's ability to manage tariff impacts, estimating a negligible effect on adjusted operating income for the year [39][40] - The company believes the Italian Supreme Court's decision regarding the SNEA matter effectively removes a long-standing overhang, allowing for greater capital allocation flexibility [37][43] - Management raised the overall organic growth outlook by 100 basis points to between 7% and 8% for 2025, reflecting strong first-quarter performance [19] Other Important Information - The company recorded a liability of $360 million related to the SNEA litigation, which is expected to impact adjusted diluted earnings per share by approximately $0.20 for the remainder of 2025 [36][37] - The company is implementing a tariff mitigation plan that includes a holistic assessment of its supply chain and potential pricing actions [38][39] Q&A Session Summary Question: Can you help us frame where the OSSPREY data puts you on a market competitive basis? - Management highlighted the strength of the clinical data, noting that the trial enrolled more severe patients and achieved a rapid therapeutic response, with 65% of patients responding at twelve months [47][48] Question: What is the outlook for the HLM business and Essence placement rollout? - Management indicated that Essence represented 40% of all HLM placements in 2024 and is expected to reach 60% in 2025, with strong demand and regulatory approvals in new markets like China [58][59] Question: Can you provide more detail on the U.S. Neuromodulation business and updated guidance? - Management noted improved commercial execution in Europe and rest of the world, with a focus on maximizing VNS therapy access in the underpenetrated drug-resistant epilepsy population [64][66] Question: What is the impact of tariffs on guidance? - Management confirmed that the estimated tariff impact on adjusted operating income is less than $5 million, with mitigation strategies potentially offsetting some costs [70][74] Question: How does the company view its leverage ratio and capital allocation flexibility? - Management stated that the leverage ratio is healthy, allowing for continued investment in core businesses and potential inorganic growth opportunities [88][90]

Financial Data and Key Metrics Changes - Total revenue for Q1 2025 was $149.8 million, compared to $143.6 million in Q1 2024, reflecting an increase driven by core products [14] - Total revenues excluding Trokendi XR and Oxtellar XR increased by 26% year-over-year [6][15] - GAAP net loss for Q1 2025 was $11.8 million, or loss per diluted share of $0.21, compared to a GAAP net earnings of $124,000 in the prior year [16] - Adjusted operating earnings for Q1 2025 were $25.9 million, up from $22.3 million in Q1 2024 [17] - Cash and cash equivalents as of March 31, 2025, were approximately $463.6 million, an increase from $453.6 million at the end of 2024 [17] Business Line Data and Key Metrics Changes - KELBRE saw a 22% increase in prescriptions and a 44% increase in net sales, with March 2025 prescriptions reaching an all-time high of 75,277 [7] - GOCOVRI prescriptions increased by 12% and net sales increased by 16% compared to the same quarter last year [8] - Combined net sales of Trokendi XR and Oxtellar XR were down 46% in Q1 2025 [10] Market Data and Key Metrics Changes - The Medicare Inflation Reduction Act led to a 42% decline in GOCOVRI's Medicare co-pay, resulting in increased prescriptions among Medicare patients [8] - 84% of GOCOVRI's Medicare prescriptions cost patients less than $25 by March 2025 [8] Company Strategy and Development Direction - The company aims to drive growth from core products while investing in its pipeline and launching new products like NAPCO [9][57] - Corporate development remains a top priority to strengthen future growth through strategic opportunities [11][57] - The company plans to initiate a follow-on phase 2b trial for SPN-eight twenty in adults with major depressive disorder [10] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the growth trajectory of KELBRE, emphasizing that growth will be driven more by volume than price [21] - The company is confident in its ability to navigate the current environment, including potential tariff impacts, as most products are shielded from tariffs [12] - Management reiterated guidance for total revenues in 2025 to range from $600 million to $630 million [18] Other Important Information - The company is focused on enhancing its infrastructure for new product launches, ensuring a smooth transition from patient enrollment to product delivery [30] - The company has established a strong support system for patients and physicians, which is seen as a competitive advantage [45] Q&A Session Summary Question: Key growth drivers for KELBRE in 2025 - Growth will be driven by both volume and a small price increase, with a strong prescription growth observed in Q1 [21] Question: Timing for data readout from the study on SPN-eight twenty - The study is expected to start before year-end 2025, with data potentially available in about 18 months [25] Question: Impact of Q1 dynamics on KELBRE net pricing - Q1 typically sees pressure on gross to net pricing, but improvements are expected in subsequent quarters [29] Question: Reimbursement discussions for ONAPCO - The company expects a high percentage of enrollment forms to be fulfilled, with a strong support system in place for reimbursement [34] Question: Competitive dynamics regarding ONAPCO - Initial feedback from physicians has been positive, and the product is seen as a strong alternative to existing therapies [45] Question: Potential indications for SBN-eight twenty - The company is considering multiple indications, including ADHD and sleep-wake disorders, but has not finalized the lead indication yet [48]

Financial Data and Key Metrics Changes - Total product revenues for Q1 2025 were $121.5 million, representing a year-over-year growth of 62% compared to $75 million in Q1 2024 [18][21] - Net loss for Q1 2025 was $59.4 million or $1.22 per share, an improvement from a net loss of $68.4 million or $1.44 per share in Q1 2024 [21] - Cash and cash equivalents at the end of Q1 2025 were $300.9 million, down from $315.4 million at year-end [21] Business Line Data and Key Metrics Changes - Avelity net product sales were $96.2 million for Q1 2025, representing 80% year-over-year growth from $53.4 million in Q1 2024 [18] - Sunosi net product revenues were $25.2 million for Q1 2025, a 17% year-over-year growth from $21.6 million in Q1 2024 [18] Market Data and Key Metrics Changes - Avelity led the market in TRx growth in Q1 with approximately 167,000 prescriptions, reflecting 76% year-over-year growth [23] - Sunosi total prescriptions were over 46,000, representing a 12% growth versus Q1 2024 [25] Company Strategy and Development Direction - The company aims to advance three novel NDA stage product candidates: AXS-fourteen for fibromyalgia, AXS-five for Alzheimer's disease agitation, and AXS-twelve for narcolepsy [8][12] - The company is focused on executing multiple Phase III clinical programs to broaden the potential of current products and candidates [8][14] - The company anticipates a strong position with potentially five marketed products across six indications by 2026 [17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's ability to navigate trade policy developments, stating that any potential impact would be immaterial [15] - The company expects 2025 to be a catalyst-rich year with significant updates on product launches and clinical trials [17] Other Important Information - The company is preparing for the launch of Cymbravo, with positive feedback from healthcare providers regarding its clinical profile [26] - The company is actively negotiating to expand payer coverage for Avelity and Sunosi [68] Q&A Session Summary Question: FDA interactions regarding developmental agents - Management indicated that dialogue with the FDA remains status quo and no changes have been noted regarding review teams or agency responsiveness [32] Question: DTC advertising for Avelity - A national campaign is expected to launch later this year [35] Question: Coverage evolution for Avelity - Management is focused on increasing covered lives and reducing utilization management [68] Question: Expected cadence of coverage for Cymbravo - Management stated it is premature to specify the cadence but is focused on securing access quickly [48] Question: Sales opportunity for AXS-fourteen in fibromyalgia - Management is enthusiastic about the potential impact, given the large market size and lack of recent therapeutic innovation [62] Question: Profitability expectations for 2025 - Management remains confident in achieving cash flow positivity but has not provided specific guidance on profitability [94]

Core Viewpoint - Abivax has completed enrollment for the Phase 3 ABTECT trials in patients with moderately to severely active ulcerative colitis, marking a significant milestone for the company and the patient community [2][3]. Group 1: Trial Enrollment and Characteristics - A total of 1,275 participants were successfully enrolled in the Phase 3 ABTECT trials, exceeding the target enrollment of 1,224 by 4% [6]. - The blinded baseline characteristics of participants in the Phase 3 ABTECT trials closely align with those observed in the Phase 2b trial, reinforcing confidence in the drug's potential efficacy and safety [3][9]. - Key baseline characteristics include: - 65% of participants had a baseline MMS score of 7-9 in Phase 3 compared to 71% in Phase 2b - 48% had prior advanced therapy in both trials - 42% of Phase 3 participants had previously failed corticosteroid treatment, compared to 52% in Phase 2b [3][6]. Group 2: Upcoming Milestones - Top-line results from the 8-week induction trials are anticipated in Q3 2025, with 44-week maintenance data expected in Q2 2026 [6][9]. - If the results confirm the safety and efficacy of obefazimod, a New Drug Application (NDA) submission to the FDA is planned for the second half of 2026 [3][9]. Group 3: Company Overview - Abivax is a clinical-stage biotechnology company focused on developing therapeutics that stabilize the immune response in patients with chronic inflammatory diseases [7]. - The lead drug candidate, obefazimod (ABX464), is currently in Phase 3 clinical trials for the treatment of moderately to severely active ulcerative colitis [7].