Financial Data and Key Metrics Changes - As of March 31, 2025, the company had cash, cash equivalents, and short-term investments of approximately $27.1 million, down from $44.9 million as of December 31, 2024 [20] - Cash used in operations for the first quarter of 2025 was $18.1 million, compared to $13 million for the same period in 2024 [21] - The net loss for the first quarter of 2025 was $17.6 million, or $0.58 per basic and diluted share, compared to a net loss of $21.8 million, or $0.72 per basic and diluted share for the first quarter of 2024 [22] Business Line Data and Key Metrics Changes - Research and development expenses for the first quarter of 2025 totaled $12 million, down from $13.3 million in the first quarter of 2024, primarily due to lower study costs [21] - General and administrative expenses for the first quarter of 2025 were $6.3 million, compared to $9.7 million in the first quarter of 2024, driven by a decrease in stock-based compensation expense [22] Market Data and Key Metrics Changes - The market opportunity for NDV-one includes approximately 75,000 new cases of bladder cancer diagnosed each year in the US, with about 50% having high-grade disease at high risk of recurrence [8] - The potential market for sopranolone includes Prader Willi syndrome, which affects approximately 350,000 people worldwide, including about 20,000 in the US [17] Company Strategy and Development Direction - The company aims to advance its pipeline to important clinical milestones with a disciplined development plan and a clean balance sheet [6][20] - The strategic focus includes bringing NDV-one for bladder cancer and sopranolone for Prader Willi syndrome and Tourette syndrome to patients as soon as possible [5][24] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of NDV-one to improve care for bladder cancer patients, citing positive Phase two data presented at the AUA meeting [7][14] - The company plans to secure U.S. IND clearance for NDV-one in the second half of 2025 and is preparing for the next set of studies [15][24] Other Important Information - NDV-one is a novel sustained release formulation of gemcitabine and docetaxel, designed to maximize local dosage concentration while minimizing systemic toxicity [9] - The initial Phase two data for NDV-one showed an overall response rate of 85% at three months and 90% at any time point, with a 100% complete response rate in carcinoma in situ patients [12][13] Q&A Session Summary Question: What gives confidence that the current data from the Phase two study would be sufficient for the FDA to agree for NDV-one to move into registrational study? - Management highlighted the well-known efficacy and safety of the drug combination and the advantages of the new formulation that allows for easier administration [29][30] Question: Could you elaborate on scaling up supply? - The company is looking to secure manufacturing capacity for commercial needs and plans to have at least two manufacturers for risk management [47] Question: When should updates on the complete response rate for the entire population be expected? - The next data point will be the six-month results, expected around June or July, with further updates planned for nine and twelve months [52][53] Question: What would the Phase three trial design look like? - Management discussed potential routes for approval, including a single-arm trial or a randomized trial against placebo, depending on FDA feedback [62][64]