Geron(GERN)2022-11-03 17:07Financial Data and Key Metrics - As of September 30, 2022, the company had approximately $195 million in cash and marketable securities, including $4 million from warrant exercises [53] - Operating expenses increased for the three and nine-month periods ended September 30, 2022, due to higher activity levels and a $7 million accrual for a class action lawsuit settlement [54][56] - Non-GAAP total operating expenses for 2022 are expected to be up to $150 million, with $140 million in cash and marketable securities projected at the time of the low-risk MDS top-line results in early 2023 [57] - The company expects up to $171 million in additional funding in 2023, including $121 million from warrant exercises and $50 million from potential debt drawdowns [58] Business Line Data and Key Metrics - The company is focused on developing and commercializing imetelstat, a telomerase inhibitor, for hematologic malignancies, particularly lower-risk MDS and relapsed/refractory myelofibrosis (MF) [7][8] - In the Phase 2 lower-risk MDS study, approximately one-third of patients achieved one year or more of transfusion independence, with a median duration of 1.8 years [13][27] - In the Phase 2 relapsed/refractory MF study, median overall survival improved almost twice as long as reported in medical literature [14] - The company is preparing for the top-line results of the IMerge Phase 3 trial in lower-risk MDS, expected in early January 2023, with regulatory submissions planned for 2023 [15][16] Market Data and Key Metrics - The company is targeting a market for imetelstat that is approximately three times larger than the patient group indicated for luspatercept, which is limited to RS-positive patients [69] - The IMpactMF Phase 3 trial, targeting relapsed/refractory MF, is the first and only Phase 3 MF trial with overall survival as the primary endpoint, with an interim analysis expected in 2024 [35][36] - The company is exploring imetelstat in combination with ruxolitinib for frontline MF, with preliminary data expected by the end of 2023 [38][40] Company Strategy and Industry Competition - The company aims to become a leader in the treatment of hematologic malignancies, with imetelstat as a key driver of this strategy [7][61] - Imetelstat's unique mechanism of action, targeting malignant stem and progenitor cells, differentiates it from other therapies and addresses unmet needs in lower-risk MDS and relapsed/refractory MF [9][11] - The company is preparing for potential commercialization of imetelstat, including hiring key personnel and building a foundation for a successful launch in lower-risk MDS [17] Management Commentary on Operating Environment and Future Outlook - Management is optimistic about the upcoming milestones, including the IMerge Phase 3 top-line results and regulatory submissions, which could unlock significant value for patients and shareholders [15][61] - The company is actively preparing for commercialization and regulatory submissions, with a focus on building a strong foundation for imetelstat's potential launch [16][17] - Management highlighted the durability of clinical effects observed in Phase 2 studies, which they believe will translate into a highly differentiated clinical profile in Phase 3 trials [14][32] Other Important Information - The company is exploring imetelstat in other indications, including acute myeloid leukemia (AML) and lymphoid malignancies, with non-clinical data presented at ASH 2022 [41][46] - A next-generation telomerase inhibitor program is underway, with lead compounds expected to be identified in 2023 [50] - The company is supporting investigator-led studies in relapsed/refractory AML and higher-risk MDS, with the first site expected to open by the end of 2022 [43][44] Q&A Session Summary Question: Implications of Bristol Myers Squibb's COMMAND study on imetelstat's path [64] - The company believes imetelstat will be part of the standard of care for lower-risk MDS, addressing a broader patient population than luspatercept [65][69] Question: Level of detail expected in the IMerge Phase 3 top-line results [70] - The top-line results will include primary and secondary endpoints, such as 24-week transfusion independence and safety data, but one-year TI rates may not be included [70][71] Question: Subgroup analysis in the IMerge Phase 3 top-line results [74] - Subgroup analysis will be provided at the time of the top-line results, with additional data presented at future medical meetings [75] Question: Enrollment milestones for the IMpactMF trial [79] - The company plans to communicate enrollment milestones, such as 50% enrollment, but the interim analysis is event-driven and expected in 2024 [80][81] Question: Potential for an overall survival study in MDS [85] - Overall survival and progression-free survival are secondary endpoints in the IMerge Phase 3 trial, and the company will closely monitor these metrics [87] Question: DSMB review of the IMerge Phase 3 trial [94] - An independent data monitoring committee has reviewed safety data and recommended the study proceed as planned [94] Question: Enrollment progress in the IMproveMF Phase 1 trial [104] - Two of three trial sites are open, and enrollment is progressing as expected, with preliminary safety data expected in 2023 [106][108] Question: Combination therapy in AML [89] - The company is exploring imetelstat in combination with other therapies, with a focus on safety and potential efficacy signals [89][90]