Plus Therapeutics(US:PSTV)2022-02-25 04:09Financial Data and Key Metrics Changes - As of December 31, 2021, cash and cash equivalents were $18.4 million, compared to $8.3 million as of December 31, 2020, indicating a significant increase in liquidity [38] - Cash used in operations for the year ended December 31, 2021, was $10.3 million, up from $8.4 million in 2020, reflecting increased operational activities [38] - Net loss for 2021 was $13.4 million, compared to a net loss of $8.2 million in 2020, primarily due to increased R&D and G&A expenses [42] Business Line Data and Key Metrics Changes - Research and development expenses rose to $5.3 million in 2021 from $2.7 million in 2020, driven by continued development of RNL to GMP standards [39] - General and administrative expenses increased to $6.9 million in 2021 from $6.4 million in 2020, attributed to new patent filings and professional fees related to the BAM transaction [40] Market Data and Key Metrics Changes - The initial indication for RNL targets recurrent glioblastoma, with an estimated 30,000 patients annually in the U.S. and a similar number in Europe, highlighting a significant market opportunity [8] - The ReSPECT-GBM trial has shown promising interim data, with a mean overall survival of 38 weeks and a median of 50 weeks for treated patients [15] Company Strategy and Development Direction - The company aims to become a global leader in precision targeted radio therapeutics for cancer, focusing on the development of RNL and expanding its pipeline with new technologies like BAM [7][21] - An agreement with the University of Texas for a worldwide exclusive license to develop biodegradable alginate microspheres (BAM) was announced, which will enhance the company's therapeutic offerings [22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing clinical trials and the potential for RNL to address significant unmet needs in cancer treatment, particularly in recurrent glioblastoma and leptomeningeal metastases [27][34] - The company plans to meet with the FDA midyear to propose a Phase 2 clinical plan and explore the use of real-world data to facilitate trial enrollment [28][44] Other Important Information - The company raised approximately $28.5 million of capital in early 2022, strengthening its balance sheet and providing over two years of operating cash [38] - The company is on track to deliver GMP RNL by mid-2022, with ongoing CMC activities [20][43] Q&A Session Summary Question: How much of a barrier to potential adoption would physician training be? - Management acknowledged a barrier but stated it is solvable, emphasizing that existing neurosurgical techniques can be leveraged for catheter placement [50][51] Question: Can you provide additional color on a regulatory precedent for using a synthetic control arm? - Management noted that real-world data and synthetic control arms have established precedents with the FDA, citing successful studies in ovarian cancer and glioblastoma [55][56] Question: Given the greater overall survival rate in higher dose cohorts, what is the dosing strategy for the next trial? - The dosing plan includes a recommended Phase 2 dose of 8.8 CC and approximately 21 millicuries, targeting 50% to 75% of patients with recurrent glioblastoma [62][63] Question: Can you do a registration study with a single arm of 100 patients? - Management indicated that approximately 100 patients is the expected size for the trial, with fast track status from the FDA potentially facilitating the process [68][70] Question: What is the rationale behind the BAM product? - Management highlighted BAM's mature delivery technology and its alignment with existing RNL technology, providing a logical expansion of the company's capabilities [84][86]