Core Viewpoint - Kazia Therapeutics Limited has sold all intellectual property and trademark rights to its oncology drug candidate Cantrixil to Vivesto for USD $1 million, providing non-dilutive funding to advance its clinical-stage pipeline [1][3]. Group 1: Transaction Details - Kazia will receive USD $1 million from Vivesto for the rights to Cantrixil [1]. - Vivesto had previously licensed exclusive global development and commercialization rights for Cantrixil from Kazia in March 2021 [2]. - Vivesto has decided not to pursue Cantrixil for ovarian cancer and is now exploring it for hematological cancers [2]. Group 2: Company Overview - Kazia Therapeutics is an oncology-focused drug development company based in Sydney, Australia, with a lead program, paxalisib, targeting multiple forms of brain cancer [4]. - Paxalisib has undergone ten clinical trials and has received various designations from the FDA, including Orphan Drug Designation and Fast Track Designation for glioblastoma [4]. - The company is also developing EVT801, a small molecule inhibitor of VEGFR3, which has shown activity against a broad range of tumor types [4].

Financial Data and Key Metrics Changes - The company ended 2024 with $251 million in cash and cash equivalents, expected to fund operations into the second half of 2026 [24] - ZYNLONTA net product revenues for Q4 2024 were $16.4 million, slightly down from $16.6 million in Q4 2023, while full-year revenues were $69.3 million compared to $69.1 million in 2023 [25] - The net loss for Q4 2024 was $30.7 million, a significant improvement from a net loss of $85 million in Q4 2023 [26] - For the full year, the net loss was $157.8 million, down from $240.1 million in 2023 [26] - Non-GAAP operating expenses decreased by 15% year-over-year in Q4 2024, contributing to the reduced net loss [25][26] Business Line Data and Key Metrics Changes - ZYNLONTA maintained its position in the third-line-plus DLBCL market, achieving commercial brand profitability [9] - The company reported an overall response rate of 80% and a complete response rate of 50% in the LOTIS-5 trial, indicating strong efficacy [13] - Initial data from LOTIS-7 showed a best overall response rate of 94% and a complete response rate of 72% among relapsed or refractory DLBCL patients [14] Market Data and Key Metrics Changes - The company anticipates a peak revenue opportunity of $600 million to $1 billion in the US for ZYNLONTA, assuming regulatory approval and compendia listing [19][21] - The potential market opportunity for indolent lymphomas is estimated to be between $100 million and $200 million [22] Company Strategy and Development Direction - The company aims to expand the use of ZYNLONTA into earlier lines of DLBCL therapy through ongoing trials [13] - The strategy includes pursuing research collaborations to advance early-stage solid tumor pipelines [29] - The focus remains on hematology, with a disciplined capital allocation strategy to create value [28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's path forward, highlighting the importance of upcoming data readouts in 2025 [30] - The competitive landscape is expected to evolve with the introduction of new therapies, but management believes ZYNLONTA will maintain its market position [76] Other Important Information - The company achieved a double-digit reduction in operating expenses for the second consecutive year [12] - Management emphasized the importance of regulatory strategies and compendia listings for future growth [50] Q&A Session Summary Question: Update on LOTIS-7 - The company has not disclosed the specific forum or timing for sharing LOTIS-7 data but is on track to enroll 40 patients in Q2 [42] Question: Impact of ADCETRIS Approval - Management believes the approval of ADCETRIS will have limited impact on ZYNLONTA's market share, as it is likely to replace older regimens rather than significantly affect current usage [44] Question: Regulatory Meetings for LOTIS-7 - Discussions with regulatory authorities are planned for the second half of the year once sufficient data is available [50] Question: Market Opportunity in Indolent Lymphoma - The peak opportunity for indolent lymphomas is estimated to be between $100 million and $200 million [51] Question: LOTIS-5 and LOTIS-7 Strategy - Both LOTIS-5 and LOTIS-7 are seen as complementary approaches to address different patient needs in the DLBCL market [56] Question: Importance of Upcoming Presentations - The upcoming presentations at AACR are expected to provide differentiating data on the company's ADC platforms [60] Question: Competitive Landscape for DLBCL - The introduction of bispecifics has taken about a third of the third-line-plus market, but ZYNLONTA has maintained consistent sales [75] Question: Compendia Listing and Market Penetration - Peak penetration for ZYNLONTA is typically achieved within the first two years post-approval or listing [86]

Financial Data and Key Metrics Changes - The company ended 2024 with just under $103 million in cash and cash equivalents, which is expected to support operations into the first half of 2026 [24] - Net revenues from XOLREMDI were reported at $1.4 million for the fourth quarter and $2.6 million for the full year 2024 [25] - R&D expenditures totaled $21.7 million for the fourth quarter and $81.6 million for the full year, while SG&A expenses were $15.1 million and $61.5 million respectively [25] - The net loss for the fourth quarter was $39.8 million, and for the full year, it was $37.5 million, reflecting a one-time sale of a priority review voucher for $105 million in May of the previous year [25][26] Business Line Data and Key Metrics Changes - XOLREMDI sales topped $2.5 million for the 7.5 months since its launch in mid-May 2024 [10] - The company has successfully engaged with top-tier immunologists and hematologists, which is expected to continue to increase patient finding and shorten the time to prescription [11] Market Data and Key Metrics Changes - The company has made significant progress in expanding the global reach of mavorixafor, with the EMA accepting the submitted MAA for review, potentially leading to approval as early as Q1 2026 [12] - The company entered into a partnership with Norgine for commercialization in Europe, Australia, and New Zealand, receiving EUR 28.5 million upfront and potential milestone payments of up to EUR 226 million [13] Company Strategy and Development Direction - The company aims to maximize the opportunity for mavorixafor in chronic neutropenia, which is viewed as a larger market opportunity [22] - A strategic restructuring is expected to decrease annual spending by about $30 million to $35 million, implemented to sharpen focus on chronic neutropenia [22] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the engagement with regulatory agencies, indicating a supportive environment for the trial design [32][34] - The company is focused on building awareness and education around WHIM syndrome and chronic neutropenia, with plans to pivot efforts towards the patient community [86] Other Important Information - The company has initiated a peer-to-peer speaker program to enhance physician education regarding WHIM syndrome [7] - The 4WARD trial for chronic neutropenia has been activated at about 90% of targeted sites worldwide, with expectations for full enrollment by Q3 or Q4 of this year [22] Q&A Session Summary Question: Regulatory conversation regarding ANC threshold - Management indicated that the FDA is supportive of the trial design focusing on moderate to severe patients, and the change in ANC threshold is not expected to impact trial pace [32][33] Question: Inventory and discounting trends - The company noted that inventory reflects stocking up in Q4, and there are no current trends in discounting as they have a tight distribution channel [43][49] Question: Patient numbers and price increase for XOLREMDI - Management did not disclose patient numbers but confirmed a 7% price increase for the new year [54][56] Question: Commercialization experiences since launch - The company has successfully engaged physicians and identified additional patients, with ongoing efforts to build demand through conferences and education [70][71] Question: Expected patient numbers for WHIM and CN - Management reiterated the guidance of approximately 1,000 diagnosed WHIM patients in the U.S. and about 50,000 total chronic neutropenia patients, targeting the refractory severe end of that population [78]

Industry Overview - The psoriasis market is projected to reach $30 billion by 2030, with a significant shift towards oral drugs [1] Company Developments - Can-Fite BioPharma Ltd. has initiated a pivotal Phase 3 study for its oral drug Piclidenoson, targeting moderate to severe plaque psoriasis, with patient enrollment starting in Europe and expected to follow in the US and Canada [1][3] - The Phase 3 study is randomized, double-blind, and placebo-controlled, focusing on the safety and efficacy of Piclidenoson, with co-primary efficacy objectives including achieving a PASI score response of ≥75% and a sPGA score of 0 or 1 at Week 16 [2] - The FDA has requested two Phase 3 studies and encouraged the enrollment of adolescent patients due to the drug's strong safety profile [2] - Upon successful completion of the Phase 3 program, the company plans to submit a New Drug Application (NDA) to the FDA and a Marketing Authorization Plan (MAA) to the EMA [3] Product Information - Piclidenoson is a first-in-class A3 adenosine receptor agonist, demonstrating an excellent safety profile and efficacy in previous clinical studies, targeting inflammatory cytokines IL-17 and IL-23 [4] - Can-Fite's lead drug candidate, Piclidenoson, is part of a broader pipeline that includes other drugs like Namodenoson, which is being evaluated for various cancers and has received Orphan Drug Designation and Fast Track Designation from the FDA [5]

Financial Data and Key Metrics Changes - For Q4 2024, the company reported a net loss of $11.4 million, compared to a net loss of $7.8 million in Q4 2023 [25] - R&D expenses increased to $9.3 million in Q4 2024 from $6.5 million in Q4 2023, primarily due to higher clinical trial costs [25][26] - G&A expenses rose to $2.9 million in Q4 2024 from $2.4 million in the same period in 2023, mainly due to increased stock-based compensation [26] - As of December 31, 2024, cash, cash equivalents, and marketable securities totaled $107.6 million, bolstered by a $50 million offering completed in December [26][27] Business Line Data and Key Metrics Changes - The company achieved three positive data readouts in 2024, including studies on Human Abuse Potential, the CORAL study in chronic cough patients with IPF, and the RIVER study in patients with refractory chronic cough [6][20] - The RIVER trial met its primary endpoint with a statistically significant reduction in 24-hour objective cough frequency, achieving a p-value of less than 0.0001% [17] Market Data and Key Metrics Changes - The RIVER study targets a patient population of approximately 2 million to 3 million in the US suffering from refractory chronic cough, which currently has no approved therapies [16] - The company plans to focus on patients who have failed prior therapies, addressing a significant unmet need in the market [18] Company Strategy and Development Direction - The company aims to develop Haduvio as a first-in-class therapy for chronic cough conditions, with a focus on both IPF and RCC [23][24] - Plans include preparing for an End-of-Phase 2 meeting with the FDA by the end of 2025 to discuss pivotal program designs and safety databases [21] - The company intends to release more data from the RIVER trial at upcoming medical conferences, indicating a proactive approach to stakeholder engagement [22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the execution of clinical trials and the potential of Haduvio to address significant unmet needs in chronic cough conditions [6][23] - The company remains optimistic about the upcoming data readouts and the potential for Haduvio to be positioned as a best-in-class therapy [23] Other Important Information - The company expects cash burn of approximately $12 million to $14 million per quarter in Q1 and Q2 of 2025, with guidance to be updated as trial designs are finalized [28] - The current fully diluted shares outstanding are approximately 137 million, including about 10 million stock options [29] Q&A Session Summary Question: Could you speak about the patients enrolled in the CORAL study? - Management confirmed that no changes were made to the study protocol or sites after the sample size estimation, maintaining consistency in enrollment statistics [33][34] Question: What are the placebo response expectations for the IPF chronic cough Phase 2 study? - Management clarified that the study includes a two-week titration period, and previous studies indicated a placebo effect ranging from 15% to 23% [42][43] Question: How will the RCC patient population be split between P2X3 and Haduvio? - Management indicated that Haduvio would be positioned as a second or third-line therapy for patients who have failed P2X3 treatments, addressing a significant unmet need [46][48] Question: What exploratory metrics are expected at the time of the IPF chronic cough readout? - Management confirmed that key secondary endpoints will include the EXACT-2 and CS-VAS metrics, but specifics on additional secondary endpoints were not provided [82] Question: Will the next RCC trial have any changes in inclusion criteria? - Management stated that the inclusion criteria will remain similar to the previous study, focusing on patients with a minimum level of cough [60]

Financial Data and Key Metrics Changes - Total revenue for Q4 2024 increased significantly to $10.8 million from $4.8 million in Q4 2023, primarily due to higher license and milestone revenue [25][26] - Full year net loss for 2024 was $62 million or $0.61 per share, compared to a net loss of $50.6 million or $0.51 per share in 2023 [30][31] - Operating expenses for the full year 2024 were lower compared to 2023, with R&D expenses flat and G&A expenses decreasing [29][30] Business Line Data and Key Metrics Changes - The number of active partners grew by 18% year-over-year, reaching 91 active partners as of December 31, 2024 [11][12] - Active programs increased by 12% year-over-year, totaling 362 active programs as of December 31, 2024 [13][14] - Over 98% of active programs have contracted future economics, positioning the company well for future value creation [14] Market Data and Key Metrics Changes - The company noted a decline in royalty revenue due to competitive dynamics in the PD-1, PD-L1 market in China, impacting product sales [28][29] - The company has seen strong interest from partners in various therapeutic areas, including oncology and CNS, with new partnerships being formed [12][74] Company Strategy and Development Direction - The company is focused on expanding its pipeline and advancing clinical programs, with expectations for 5 to 7 new clinical entrants in 2025 [15][66] - The introduction of new technologies, such as OmniDAV and OmniHub, is aimed at enhancing partnerships and driving growth [42][44] - The strategic shift from small molecules to antibodies is expected to align staffing needs and reduce operating expenses [34][35] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's resilience and ability to perform well in various market cycles [46] - The company anticipates continued growth driven by pipeline expansion and advancements in clinical programs [24][33] - Management highlighted the importance of innovation and collaboration with partners as key competitive advantages [42][44] Other Important Information - The company exited 2024 with $59.4 million in cash, at the top end of the guidance range provided [31][32] - The 2025 revenue guidance is projected to be between $20 million and $25 million, with a significant portion of 2024 revenue being non-cash [33][34] Q&A Session Summary Question: What are the reasons behind the attrition rates? - Management clarified that attrition is primarily due to Big Pharma pipeline realignment and normal drug development processes, not technical issues [39][40] Question: Can you provide more details on new technology rollouts? - Management indicated that new technologies, including OmniDAV and OmniHub, are expected to enhance discovery and attract new partners [42][44] Question: Is there potential for additional cash deployment from partners in the current market? - Management believes the business is well-positioned to thrive in various market conditions, indicating resilience and adaptability [46] Question: Can you clarify the 2025 revenue guidance regarding cash and non-cash components? - The guidance is a GAAP number, with a significant portion of 2024 revenue being non-cash due to service revenue amortization [52][53] Question: How is the academic partner ecosystem being affected by NIH funding concerns? - Management has not seen a significant impact on academic collaborations despite concerns over NIH funding, with ongoing discussions with prospective partners [58][59] Question: Has the productive fourth quarter continued into the first quarter? - Management indicated that while trends are positive, revenue can be lumpy due to the timing of partner reports [61][62]

Financial Data and Key Metrics Changes - Total revenue for Q4 2024 increased significantly to $10.8 million from $4.8 million in Q4 2023, primarily due to higher license and milestone revenue driven by new deals and clinical advancements [25][26] - Full year net loss for 2024 was $62 million or $0.61 per share, compared to a net loss of $50.6 million or $0.51 per share in 2023 [30][34] - Operating expenses for 2024 were lower compared to 2023, with R&D expenses flat and G&A expenses decreasing mainly due to non-recurring costs [29][34] Business Line Data and Key Metrics Changes - The number of active partners grew by 18% year-over-year, reaching 91 active partners as of December 31, 2024 [11] - Active programs increased by 12% year-over-year, totaling 362 programs net of attrition as of December 31, 2024 [13] - Over 98% of active programs have contracted future economics, positioning the company well for future value creation [14] Market Data and Key Metrics Changes - The company noted a decline in royalty revenue compared to the previous year, primarily due to competitive dynamics in the PD-1/PD-L1 market in China [28] - The company is observing strong interest from partners across various therapeutic areas, including oncology and immunology, with a notable increase in CNS targets [76] Company Strategy and Development Direction - The company is focused on pipeline expansion and advancement, with significant progress expected in clinical and future royalty programs [24] - New technologies and enhancements are being introduced to attract new partners and drive efficiencies, with a commitment to innovation seen as a competitive advantage [42][44] - The company aims to leverage its ecosystem of partners to analyze economic returns for technology expansion and launch options [24] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's plans for 2025, highlighting the resilience of the business model amid industry volatility [46] - The company anticipates several catalysts for growth in 2025, including new deals and the launch of novel technologies [24][36] - Management noted that while revenue may decrease due to non-cash components, cash received from partners is expected to increase in 2025 compared to 2024 [34] Other Important Information - The company introduced its first revenue guidance for 2025, expecting revenue in the range of $20 to $25 million [33] - The company exited 2024 with $59.4 million in cash, at the top end of the guidance range provided in the previous earnings call [25] Q&A Session Summary Question: What are the reasons behind the attrition rates observed? - Management clarified that attrition is primarily due to Big Pharma pipeline realignment and normal drug development processes, not technical issues [39][40] Question: Can you provide more details on the new technology rollouts planned for this year? - Management highlighted ongoing investments in innovation, including the launch of OmniDAV and OmniHub, which are expected to enhance partner collaboration and drive growth [42][44] Question: How does the company expect to see cash deployment from partners in the current market? - Management indicated that the business has shown resilience across various cycles, positioning it well for continued partner engagement and cash deployment [46] Question: Can you clarify the revenue guidance for 2025 regarding cash and non-cash components? - Management confirmed that the guidance is a GAAP number, with a significant portion of 2024 revenue being non-cash, particularly from service revenue amortization [52][54] Question: Is there any impact from NIH funding changes on academic partnerships? - Management noted that, despite concerns about NIH funding, they have not seen a negative impact on collaborations with academic partners [58] Question: Has the productive addition of partners continued into the first quarter of 2025? - Management indicated that while the trend is positive, the timing of reports from partners can vary, making it difficult to assess specific quarterly performance [62]

Core Insights - Lexicon Pharmaceuticals reported positive topline results from the Phase 2b PROGRESS study of pilavapadin, a novel non-opioid therapy for diabetic peripheral neuropathic pain (DPNP), indicating a meaningful pain reduction at the 10 mg dose, which is set to advance into Phase 3 development in 2025 [3][7] - The company is focusing on its R&D pipeline, with significant progress on three key programs: pilavapadin for DPNP, LX9851 for obesity, and sotagliflozin for hypertrophic cardiomyopathy (HCM) [3][4] - Financial results for Q4 2024 showed a substantial increase in revenues, driven by sales of INPEFA and a $25 million upfront payment from a licensing agreement [11][12] Group 1: Clinical Development - Pilavapadin (LX9211) demonstrated meaningful pain reduction and was well-tolerated in the PROGRESS Phase 2b study, supporting its advancement to Phase 3 trials [3][7] - LX9851 is progressing towards an IND filing in 2025, targeting obesity and related metabolic disorders [5] - Enrollment is ongoing for the SONATA Phase 3 study of sotagliflozin in HCM, with a target of 500 patients [8] Group 2: Financial Performance - Q4 2024 revenues increased to $26.6 million from $0.7 million in Q4 2023, and full-year revenues rose to $31.1 million from $1.2 million in 2023, reflecting increased sales of INPEFA and the licensing agreement [11] - R&D expenses for Q4 2024 rose to $26.7 million from $14.8 million in Q4 2023, primarily due to investments in clinical trials [12] - The net loss for Q4 2024 was $33.8 million, an improvement from a net loss of $49.8 million in Q4 2023 [14] Group 3: Strategic Focus - The company is strategically repositioning to prioritize R&D efforts, aiming to address significant unmet medical needs in its pipeline [3][4] - Lexicon's focus on generating clinical data for sotagliflozin aims to differentiate it in the market, particularly in reducing cardiovascular risks [18] - The company has a strong cash position of $238 million as of December 31, 2024, up from $170 million a year earlier, providing a solid foundation for ongoing development [15]