Financial Data and Key Metrics Changes - As of March 31, 2025, the company's cash position was $47.9 million, expected to support operations into Q1 2027 [23] - Total revenues for Q1 2025 were $1.5 million, a net increase of $0.1 million compared to $1.4 million for the same period in 2024, primarily driven by collaboration revenue from Roche [24] - Operating expenses for Q1 2025 were $8 million, a decrease of $0.1 million compared to $8.1 million in Q1 2024 [24] - The net loss for Q1 2025 was $4.1 million or $0.02 per share, compared to a net loss of $6.5 million or $0.04 per share for the same period in 2024 [25] Business Line Data and Key Metrics Changes - OpRegen is currently in a Phase 2a study called the GALET study, which is exploring various parameters of surgical delivery [7] - The OPC1 program has been tested in 30 individuals with severe spinal cord injuries, with promising long-term safety and efficacy data [18] Market Data and Key Metrics Changes - The company is encouraged by recent positive data from competing RPE companies, which supports the potential of RPE transplants to drive clinical outcomes beyond currently approved therapies [13] Company Strategy and Development Direction - The company aims to capitalize on investments in cell manufacturing and expand capabilities through partnerships beyond OpRegen and OPC1 [27] - The manufacturing platform is seen as a competitive advantage, allowing for low-cost production of allogeneic therapies [15] Management's Comments on Operating Environment and Future Outlook - Management remains confident in OpRegen's potential to drive positive clinical outcomes in dry AMD and is encouraged by partners' commitment to the program [27] - The company is preparing for success by seeking additional partnerships and funding opportunities [27] Other Important Information - The company has completed GMP production runs capable of producing millions of doses from a single cell line, marking a significant achievement in manufacturing capabilities [16] Q&A Session Summary Question: Comparison of manufacturing capabilities with peers - Management expressed frustration over the lack of detailed comparisons from peers but emphasized their differentiated capabilities in manufacturing [31][33] Question: Potential tariff impacts due to manufacturing site in Israel - Management does not expect tariff impacts and has measures in place to mitigate production issues [36][37] Question: Thoughts on potential partnerships around new manufacturing capabilities - Management indicated that partnerships would vary by opportunity, focusing on success payments and ownership in assets rather than becoming a contract manufacturer [44] Question: Expected data from the OPC1 program - Initial data from the dose study is expected to be available within 30 days, with functional assessments potentially lagging by several months [54] Question: Design of the spinal cord injury trial - The trial will enroll between six and ten patients, with a staggered approach for thoracic and cervical patients [90]

Company Strategy & Portfolio - Whitehawk Therapeutics is transitioning into an ADC-focused company, leveraging advanced ADC technology and targeting significant patient populations[6, 7] - The company's portfolio includes three assets (HWK-007, HWK-016, and HWK-206) rapidly progressing to clinical trials, with all three INDs anticipated in the next 15 months[7, 9] - Whitehawk's ADC platform is engineered for minimal off-target toxicity, greater stability, and a higher therapeutic index[9, 12] ADC Technology & Efficacy - Whitehawk utilizes a proprietary TOPO1 inhibitor payload and a highly stable, cleavable linker to improve the therapeutic index of its ADCs[13] - Switching to an advanced ADC platform technology delivers efficacy gains compared to 1st generation ADCs, as demonstrated by increased Overall Response Rates (ORR) in various cancer indications[17] - For example, in SEZ6 targeting for Small Cell Lung Cancer (SCLC), next-wave ADCs like ABBV-706 achieved a 61% ORR, compared to 19% with 1st generation ADCs[17] Target & Clinical Development - HWK-007 targets PTK7, which is overexpressed in a broad spectrum of cancers, including Non-Small Cell Lung Cancer (NSCLC) and Ovarian cancer, with potential to outperform current ADC benchmarks[20, 29, 31] - HWK-016 targets MUC16, a glycoprotein overexpressed in cancers affecting women, with a Phase 1 trial planned in ovarian cancer[40, 50] - HWK-206 targets SEZ6, a CNS protein upregulated in tumors of neuroendocrine origin, with a Phase 1 trial planned in SCLC and Neuroendocrine Neoplasms (NENs)[55, 65] Financial Outlook - The company's cash runway is expected to fund operations into 2028, including anticipated key clinical data[3, 67]

Financial Data and Key Metrics Changes - The company reported a significant reduction in non-GAAP operating expenses by 50% year-on-year in 2024, focusing on key priorities to enhance efficiency [17] - An equity offering was announced to extend the company's cash runway, with proceeds expected to last until late Q3 2025 [16][12] Business Line Data and Key Metrics Changes - The capsid engineering platform saw a new licensing agreement with Eli Lilly, which includes an $18 million upfront fee and potential total payments of up to $1.4 billion across five targets [6][7] - The neurology pipeline is advancing, with preparations for a Phase 1/2 study of ST503 for chronic neuropathic pain expected to begin in mid-2025 [8][10] Market Data and Key Metrics Changes - The company is actively engaging in discussions with potential partners for its Fabry disease program, with a focus on securing a commercial partnership that aligns with its neurology mission [11][19] Company Strategy and Development Direction - The long-term vision is to position Sangamo as a neurology-focused genomic medicine company, aiming to deliver transformational therapies and significant shareholder value [15] - The company is committed to operating efficiently while advancing its neurology programs, with a disciplined approach to capital allocation [17] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism regarding the FDA's engagement and the clarity provided during the Type B meeting, which is crucial for the BLA submission process [32] - The company remains focused on securing funding and partnerships to support its neurology pipeline and Fabry disease program [19] Other Important Information - The company plans to present data at the ASGCT Annual Meeting, showcasing advancements in its neurology pipeline and the effectiveness of its technologies [19][9] Q&A Session Summary Question: Can you provide more color on the top line eGFR data? - Management confirmed that the updated mean eGFR slope will be shared, along with additional information at a later date [24] Question: How many potential partners are you currently in conversations with for STABBI? - Management indicated that multiple potential partners are being engaged, and the Type B meeting was beneficial for discussions [27] Question: Has the pace of conversations with potential partners changed due to the macro landscape? - Management noted that interactions with the FDA have been positive and have not impacted the program [33] Question: Will you file based on 52-week eGFR data? - Management confirmed that they are pursuing the agreement with the FDA to use 52-week eGFR data for the entire patient population [38] Question: Are all patients who were on ERT still off ERT? - Management confirmed that all 18 patients who started on ERT are still off ERT [47]

Core Insights - Roche has launched the Elecsys® PRO-C3 test, a diagnostic solution for assessing liver fibrosis severity in patients with metabolic dysfunction-associated steatotic liver disease (MASLD) [1][3] Group 1: Product Launch and Features - The Elecsys PRO-C3 test is developed in partnership with Nordic Bioscience and provides a simple method for identifying liver fibrosis severity, enabling timely intervention [1][3] - The test delivers results in just 18 minutes using Roche's cobas® analysers, streamlining the diagnostic process and reducing costs compared to existing tests [4][9] - The test, when used with the ADAPT formula, distinguishes between different severities of fibrosis, which is crucial for determining appropriate treatment pathways [4][9] Group 2: Market Context and Importance - MASLD affects approximately 30% of the population and is a leading cause of chronic liver disease, primarily due to diabetes and obesity [2] - Liver fibrosis associated with MASLD is often asymptomatic until advanced stages, leading to severe consequences if unmanaged [2] - The introduction of the Elecsys PRO-C3 test addresses an urgent need for accurate and early detection of liver fibrosis, especially as new drug treatments for MASLD are emerging [3][9] Group 3: Future Developments - Initially, the ADAPT score will be calculated manually, but Roche plans to launch software later this year to automate this process, further streamlining liver fibrosis diagnosis [5]

Core Insights - ArriVent BioPharma, Inc. has appointed Dr. Merdad Parsey to its Board of Directors, bringing extensive experience in global clinical development from his previous role at Gilead Sciences, Inc. [1][2] - Dr. Parsey expressed enthusiasm about ArriVent's potential to transform cancer treatment with its lead candidate firmonertinib and its pipeline of next-generation antibody drug conjugates (ADCs) [2][3] - ArriVent is focused on developing differentiated medicines to meet unmet medical needs in cancer treatment, leveraging its team's drug development expertise [3] Company Overview - ArriVent is a clinical-stage biopharmaceutical company dedicated to the identification, development, and commercialization of innovative therapeutics for cancer patients [3] - The company aims to maximize the potential of its lead candidate firmonertinib and advance its ADC pipeline through approval and commercialization [3] Leadership Experience - Dr. Parsey has held significant roles in the biopharmaceutical industry, including Senior Vice President at Genentech and CEO of 3-V Biosciences, Inc. [2] - His academic background includes an M.D. and Ph.D. in immunology, with training at prestigious institutions such as Stanford University and the University of Colorado [2]

Core Insights - Jazz Pharmaceuticals is presenting significant clinical trial data at the ASCO Annual Meeting, highlighting the potential of Zepzelca® (lurbinectedin) in combination with atezolizumab (Tecentriq®) for first-line maintenance therapy in extensive-stage small cell lung cancer (ES-SCLC) [1][2] - The company is also showcasing long-term outcomes for Ziihera® (zanidatamab-hrii) in HER2-positive gastroesophageal cancer, indicating its potential to change treatment paradigms [1][2] - Efficacy and safety data for dordaviprone (ONC201) in recurrent H3 K27M-mutant diffuse glioma patients are also being presented, addressing a significant unmet medical need [1][2] Group 1: Zepzelca® (lurbinectedin) - The Phase 3 IMforte trial data shows statistically significant and clinically meaningful progression-free survival (PFS) and overall survival (OS) for Zepzelca in combination with atezolizumab for ES-SCLC patients [2][4] - The FDA has received a supplemental New Drug Application based on this trial data, marking a milestone for Zepzelca's approval process [2][4] - An investor webcast is scheduled for June 10 to review Zepzelca data, featuring commentary from experts [3] Group 2: Ziihera® (zanidatamab-hrii) - Updated long-term outcomes and median overall survival findings from the Phase 2 trial of Ziihera in combination with chemotherapy for HER2-positive metastatic gastroesophageal adenocarcinoma will be presented [2][4] - Ziihera is positioned as a differentiated HER2-targeted therapy, with further Phase 3 findings expected later this year [2][4] Group 3: Dordaviprone (ONC201) - New efficacy and safety findings for dordaviprone in adult and pediatric patients with recurrent H3 K27M-mutant diffuse glioma are being highlighted [2][4] - The drug addresses a significant unmet need, as there are currently no FDA-approved therapies for this patient population [2][4] Group 4: Company Overview - Jazz Pharmaceuticals is committed to advancing targeted treatment options that address pressing patient needs, with a focus on oncology and neuroscience [55] - The company has a diverse portfolio of marketed medicines and is dedicated to developing potentially life-changing therapies for serious diseases [55]

Core Viewpoint - Roche announced a commitment to invest USD 50 billion in the United States over the next five years, aimed at enhancing its manufacturing and R&D capabilities, which is expected to create over 12,000 new jobs [1][2][6]. Company Investment and Expansion - The investment will expand Roche's existing footprint of more than 25,000 employees across 24 sites in eight US states [2][6]. - New facilities will include state-of-the-art R&D sites and expanded manufacturing capabilities in Indiana, Pennsylvania, Massachusetts, and California, with an additional site to be announced [6][7]. Job Creation - The investment is projected to create more than 12,000 new jobs, including nearly 6,500 construction jobs and 1,000 jobs at new and expanded facilities [1][6]. Manufacturing and R&D Focus - Roche will enhance its manufacturing and distribution capabilities for innovative medicines and diagnostics in states such as Kentucky, Indiana, New Jersey, Oregon, and California [7]. - A new gene therapy manufacturing facility will be established in Pennsylvania, along with a 900,000 square foot manufacturing center for next-generation weight loss medicines [7]. Commitment to Innovation - The investment is positioned as a foundation for Roche's next era of innovation and growth, benefiting patients in the US and globally [2][6]. - Roche's CEO emphasized the company's long-standing commitment to research, development, and manufacturing in the US, highlighting its 110-year legacy in the country [2].

Core Insights - The SB221 study demonstrates positive safety results for SON-1010 in combination with atezolizumab for patients with platinum-resistant ovarian cancer (PROC) [1][5] - The maximum tolerated dose (MTD) of SON-1010 was established at 1200 ng/kg without dose-limiting toxicity or cytokine release syndrome [1][3] - The study showed a partial response (PR) in one patient and stable disease (SD) in 33% of evaluable patients at four months [1][5] Study Design and Results - The SB221 study aimed to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of SON-1010 with atezolizumab, focusing on establishing the MTD [2][8] - A total of 19 subjects were treated, with one patient achieving a PR at the highest dose [2] - Common adverse effects included fatigue, fevers, and gastrointestinal symptoms, with no significant toxicity observed [3][5] Clinical Implications - The results indicate that SON-1010 may enhance the efficacy of immune checkpoint inhibitors (ICIs) in treating 'cold' tumors like ovarian cancer [4][5] - The study's findings are encouraging given the historical safety concerns associated with rhIL-12, suggesting a potential for improved tumor control [5][6] - The ongoing trial will assess longer-term safety and tumor responses, with a focus on expanding the dataset for efficacy evaluation [6][8] Company Overview - Sonnet BioTherapeutics is focused on developing targeted biologic drugs using its Fully Human Albumin-Binding (FHAB) platform [10][11] - SON-1010 is designed to deliver IL-12 to tumor tissues, potentially improving the safety and efficacy profile of immunomodulators [7][10] - The company is also exploring partnerships to support the later stages of SON-1010's development [6][11]

Core Viewpoint - Inhibrx Biosciences, Inc. announces significant leadership changes with the departure of co-founder and Chief Scientific Officer Dr. Brendan Eckelman, who will establish a new private biotechnology company, and the appointments of Dr. Carlos Bais as CSO and David Matly as President [1][2][3][4]. Leadership Changes - Dr. Brendan Eckelman is leaving Inhibrx to become the founder and CEO of a new biotechnology company, with an exclusive license agreement in place for certain technologies previously pursued by Inhibrx [2]. - Dr. Carlos Bais, previously Executive Vice President of Translational Sciences, will take over as CSO, bringing a strong scientific background and expertise in late-stage development [5][6]. - David Matly has been promoted to President while retaining his roles as Chief Commercial and Business Development Officer, overseeing various functions including clinical development and operations [4][5]. Financial and Strategic Implications - The agreement with Dr. Eckelman’s new company includes an upfront payment upon the completion of initial funding and future development milestones, indicating potential financial implications for Inhibrx [2]. - David Matly played a key role in the asset sale of INBRX-101 to Sanofi for up to $2.2 billion in 2024, highlighting his experience in commercial strategy and business development [4]. Company Overview - Inhibrx Biosciences is a clinical-stage biopharmaceutical company focused on developing novel biologic therapeutic candidates, utilizing diverse protein engineering methods [9]. - The current clinical pipeline includes ozekibart (INBRX-109) and INBRX-106, with key data readouts expected in 2025 [9].

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].