Geron(US:GERN)2023-08-04 01:23Financial Data and Key Metrics Changes - As of June 30, 2023, the company had approximately $400.2 million in cash and marketable securities, which includes about $17.8 million from warrant exercises in Q2 2023 [10][59] - Operating expenses increased to approximately $52 million in Q2 2023 from about $40 million in Q1 2023, primarily due to higher clinical trial costs and increased personnel-related expenses [82][124] - The company maintains its non-GAAP total operating expenses guidance of up to $210 million for the full year of 2023 [83][124] Business Line Data and Key Metrics Changes - The IMerge Phase 3 trial data showed statistically significant improvements in transfusion independence (TI) rates for patients treated with imetelstat compared to placebo [17][19] - Nearly 20% of imetelstat-treated patients experienced a one-year TI, representing approximately 60% of 24-week responders [44] - The IMpactMF trial is the first Phase 3 study in myelofibrosis with overall survival as a primary endpoint, with strong evidence of disease-modifying potential [9][24][52] Market Data and Key Metrics Changes - The total addressable market for imetelstat in lower risk MDS is estimated to be approximately $3.5 billion by 2033 [34] - Market research indicates that imetelstat is likely to become a new standard of care in post-ESA experienced and luspatercept-experienced frontline patients [81] - The company expects to be ready for a US commercial launch upon potential approval in early 2024 [34][54] Company Strategy and Development Direction - The company aims to transition from a late-stage clinical development company to one with substantial commercial capabilities [5] - There is a focus on preparing for the potential launch of imetelstat, with significant regulatory progress and market research supporting its value proposition [27][34] - The company is also expanding its commercial team, expecting to grow to 160 employees by the end of the year, excluding the sales force [116] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential of imetelstat to transform the treatment landscape for lower risk MDS patients [61][87] - The company is actively addressing physician concerns regarding cytopenias associated with imetelstat treatment, emphasizing the manageable nature of these side effects [94][112] - The anticipated timeline for NDA approval is under priority review, potentially in Q1 2024, with a standard review expected in Q2 2024 [40][41] Other Important Information - The company initiated an expanded access program in June 2023 to provide imetelstat to patients prior to FDA approval [41][115] - The first patient was dosed in the Phase 2 IMpress trial for relapsed refractory acute myeloid leukemia or high-risk MDS [26] Q&A Session Summary Question: What early launch metrics will be important for low risk MDS? - The company emphasized the importance of full coverage across reimbursement policies and monitoring uptake across academic and community channels [90] Question: Is enrollment in the IMpactMF study being impacted by competition from other clinical studies? - Management noted that while there are ongoing trials, the main challenge is limited research staff resources rather than competition for patients [91] Question: How will the company address doctor concerns about temporary cytopenias associated with imetelstat? - The company has shared the full safety profile with physicians and received positive feedback regarding the manageable nature of cytopenias [94][112] Question: What is the expected growth in terms of employees for the launch? - The company plans to expand its sales force in alignment with the PDUFA date, aiming for a total of 100 to 120 full-time employees [99][116] Question: Can you discuss the opportunity to capture market share in RS negative patients? - Management indicated strong physician preference for imetelstat in RS negative patients, especially those previously treated with ESA [123]