Summary of Geron (NasdaqGS: GERN) FY Conference Call - December 02, 2025 Company Overview - **Company**: Geron Corporation - **Product**: Rytello (imetelstat), a first-in-class telomerase inhibitor targeting low-risk myelodysplastic syndromes (MDS) patients - **Sales Performance**: Reported $47.2 million in net sales in the U.S. for the last quarter, marking five quarters since launch [3][4][32] Key Points and Arguments Product Efficacy and Market Position - Rytello has demonstrated strong efficacy in clinical trials, particularly in durability and transfusion independence [4][10] - Despite strong clinical data, commercial uptake has been modest compared to expectations, especially when compared to competitors like Luspatercept, which generates over $2 billion annually [4][5] - The company aims to improve uptake by focusing on operational excellence and ensuring the right patients are prescribed Rytello [5][6] Market Strategy and Growth Potential - The company plans to target earlier lines of therapy to drive growth in 2026, moving from later-line patients to second-line patients as indicated by the IMerge study [7][9] - The NCCN guidelines have been updated to position Rytello favorably in relation to other therapies, enhancing its market potential [10] - The company has seen an increase in new accounts, with 1,150 accounts now ordering Rytello, up by 150 from the previous quarter [17] Challenges and Solutions - A significant challenge is the lack of awareness among U.S. physicians regarding Rytello, as most pivotal trial enrollments were conducted outside the U.S. [8][9] - The company is working to build relationships with key opinion leaders (KOLs) and ensure effective communication of trial data [11][12] - Engagement with academic medical centers (AMCs) is crucial, as these institutions are more likely to conduct their own experiments and publish results [21][22] Financial Management and Operational Efficiency - Geron has reduced its operational expenditure (OpEx) guidance for the year to between $250 million and $260 million, emphasizing the need for disciplined spending [32][33] - The company is focused on targeted investments rather than broad spending, aiming to maximize the impact of its resources [33][34] Future Outlook - The IMpactMF trial for myelofibrosis is fully enrolled, with interim analysis expected in H2 2026 and full analysis in H2 2028 [31] - The trial design aims to demonstrate that Rytello can transform myelofibrosis from a deadly disease to a manageable one, potentially doubling the addressable patient market [29][31] Additional Important Insights - The company is addressing concerns regarding cytopenia associated with Rytello, emphasizing the importance of managing this side effect to maintain patient adherence to therapy [25][26] - The focus on tailored messaging for different physician groups is critical for successful commercialization, particularly in the community setting [23][24] This summary encapsulates the key discussions and insights from the Geron FY Conference Call, highlighting the company's strategic direction, challenges, and future growth potential in the oncology market.

Core Insights - The biotech investment strategy focuses on a balanced portfolio with 85% in commercial stage and 15% in clinical stage companies, emphasizing disciplined position sizing and quality over quantity [5][6][8] - The community aspect of biotech investing is highlighted, where collaboration and feedback among members enhance investment decisions and strategies [9][10][15] - The current market environment presents opportunities in rare diseases and companies nearing growth stages, with a focus on those with approved drugs and clear paths to profitability [21][24][27] Group 1 - The ROTY Biotech Community has grown to nearly 500 members, including analysts and medical professionals, contributing to a collaborative investment environment [5][9] - The investment approach includes strict position sizing rules, with a maximum of 10% in commercial stage companies and 5% in clinical stage companies [6][20] - The strategy emphasizes long-term perspectives, focusing on sales projections for 2025 and beyond, and the importance of clinical momentum [8][21] Group 2 - Recent successes in the biotech sector, such as uniQure's advancements in gene therapy, have attracted more generalist investors to the space [16][18] - The investment community encourages a disciplined approach to trading, with a focus on maintaining a trade log to analyze performance and improve strategies [22][30] - The importance of understanding regulatory risks is emphasized, as even promising drugs can face unexpected challenges during the approval process [44][49] Group 3 - Syndax Pharmaceuticals is highlighted as a top holding, with a significant gain since entry, driven by positive developments in their lead drug [32][33] - Geron Corporation is presented as a potential investment opportunity, with a focus on its approved drug for blood cancer and a favorable enterprise value [49][52] - The current market dynamics suggest that while some biotech stocks are being overlooked, there are attractive opportunities in commercial stage companies [38][47]

Core Insights - The biotech investment strategy focuses on a mix of commercial stage (85%) and clinical stage (15%) companies, emphasizing disciplined position sizing and a long-term perspective on sales growth and clinical momentum [5][8][21]. Investment Strategy - The investment approach is characterized by strict position sizing rules, with a maximum of 10% in any commercial stage company and 5% in clinical stage companies [6]. - The strategy involves trading infrequently, typically two to four times a month, to avoid overtrading and to focus on quality investments [7][8]. - The community aspect of the ROTY Biotech Community allows for collaborative idea sharing and constructive criticism, enhancing investment decisions [9][10][15]. Market Trends - Recent successes in gene therapy and rare diseases are attracting more generalist investors to the biotech sector, creating a "halo effect" that could lead to increased investment [17][21]. - The FDA's favorable regulatory environment for rare diseases is seen as a significant tailwind for biotech investments [21][41]. Company Examples - Syndax Pharmaceuticals (SNDX) has shown a 60% gain since entry, driven by the approval of their lead drug and positive market reception [32][33]. - Geron Corporation (GERN) is highlighted as a potential investment opportunity due to its novel drug for blood cancer, which is currently undervalued relative to its peak sales potential [49][51]. Risk Management - Investors are advised to maintain a trade log to analyze performance and replicate successful strategies while avoiding past mistakes [22]. - The importance of understanding personal risk tolerance is emphasized, particularly in a volatile sector like biotech [30][31].

Financial Data and Key Metrics Changes - Q2 Rytello net revenues were $49 million, representing an increase of approximately 24% over the first quarter [5][29] - As of June 30, 2025, cash and marketable securities were approximately $433 million, up from $503 million as of December 31, 2024 [29] - Research and development expenses for Q2 were $22 million, down from $31 million for the same period in 2024, primarily due to lower clinical trial costs [30] Business Line Data and Key Metrics Changes - Demand growth in Q2 was 17% higher compared to Q1, with approximately 1,000 sites of care utilizing Rytello, an increase of about 400 new sites since the beginning of the year [13][14] - Approximately two-thirds of accounts that previously ordered Rytello have reordered in Q2 [14] - Payer access improved, with approximately 90% of US covered lives now under favorable Rytello medical coverage policies, up from 85% in Q1 [16] Market Data and Key Metrics Changes - The company is focused on increasing brand awareness among US hematologists treating lower-risk MDS patients, with positive feedback on educational efforts [7][23] - The company plans to expand its commercial sales force and customer-facing roles by over 20% to enhance KOL support and advocacy [8][20] Company Strategy and Development Direction - The primary strategy remains the successful commercialization of Rytello in the US, with a focus on increasing brand awareness and physician education [5][10] - The company is preparing for the launch of Rytello in select EU markets next year, maintaining financial discipline in investments for this launch [10][22] - The appointment of Harout Semerjian as the new President and CEO is expected to drive shareholder value and enhance the company's strategic direction [4][12] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the execution of commercial strategies and the potential for continued growth, particularly in the US market [11][54] - The company is optimistic about the enrollment momentum in the Phase III IMPACT MF trial, expecting completion by year-end [11][25] Other Important Information - The company has doubled the size of its medical affairs team to enhance community awareness and KOL advocacy [23] - The gross-to-net percentage remained in the mid-teens from Q1 to Q2, consistent with previous guidance [29] Q&A Session Summary Question: How many active patients are now receiving Rytello? - The company estimates that there are at least 600 active patients based on the number of accounts that have prescribed Rytello [36] Question: How sustainable is the 17% quarter-over-quarter demand growth? - The company focuses on business drivers such as new patient starts and duration of treatment, with positive trends observed in physician prescribing behavior [42][44] Question: What was the biggest issue that held back the launch in Q4 and Q1? - Management identified the need to increase awareness, ensure prescribing comfort, and engage KOLs as key strategies that have been pivoted since March [52][53] Question: Any qualitative insights on early Q3 demand trends? - The company sees conscious optimism in demand trends based on internal sales data and claims data [62] Question: Any anecdotes about the type of physicians currently prescribing Rytello in earlier lines? - The company noted that earlier line use is seen among physicians who have had patient success in later lines of therapy [71] Question: What is the EU commercialization strategy? - The company is focused on securing the highest possible reimbursable rate and is engaged with potential partners for commercialization in the EU [81][84]

Financial Data and Key Metrics Changes - As of March 31, 2025, the company had approximately $457.5 million in cash and equivalents, down from $502.9 million as of December 31, 2024 [42] - Total net product revenue for Q1 2025 was $39.4 million, a decrease of $8 million from Q4 2024, attributed to inventory drawdown among distributors [7][42] - Research and development expenses for Q1 2025 were $15.1 million, significantly lower than $29.4 million for the same period in 2024, due to reduced clinical trial costs [43] - Selling, general, and administrative expenses increased to $40 million in Q1 2025 from $27.1 million in the same period last year, primarily due to higher personnel expenses [44] Business Line Data and Key Metrics Changes - Q1 sales of Rytello were $39.4 million, with demand relatively flat at a 1% increase compared to the previous thirteen weeks [7] - Approximately 900 sites of care have utilized Rytello, an increase of nearly 300 new sites since the end of Q4 2024 [24] - Demand grew approximately 10% in the four-week period ending April 25, 2025, compared to the prior four-week period, marking the highest month-over-month growth since October 2024 [25] Market Data and Key Metrics Changes - The estimated U.S. total addressable market for Rytello is approximately 15,400 lower-risk MDS patients, with significant opportunities to expand use in earlier lines of treatment [15] - In Europe, the total addressable market for Rytello is estimated to be about 80% of the U.S. market [15] - Approximately 85% of U.S. covered lives are now under favorable Rytello medical coverage policies consistent with FDA labels and NCCN guidelines [26] Company Strategy and Development Direction - The primary corporate priority is the successful U.S. commercialization of Rytello, with a focus on increasing brand awareness and refining marketing efforts [6][10] - The company plans to work with experienced third parties for commercialization outside the U.S. to minimize investment while focusing on U.S. efforts [11] - The late-stage clinical development program aims to expand Imetel for broader use within hematology, potentially doubling the commercial opportunity of Rytello if successful [12][48] Management's Comments on Operating Environment and Future Outlook - Management acknowledged that Q1 sales were modest and do not reflect Rytello's true commercial potential, emphasizing the commitment to making it part of the standard of care for eligible patients [46] - The company is encouraged by early performance indicators and is prepared to make adjustments to improve commercial execution [47] - The pivotal Phase III trial evaluating Imetelstat in relapsed-refractory myelofibrosis is expected to provide interim analysis data in the second half of 2026, which could significantly impact commercial potential [48] Other Important Information - The process to identify a permanent CEO is ongoing, with a decision expected within the next six months [12] - The company is expanding its medical affairs team to enhance community awareness and HCP confidence in prescribing Rytello [29] Q&A Session Summary Question: Expectations for Q2 and year cadence - Management noted that inventory issues are behind them, and they anticipate continued momentum building into Q2 and throughout 2025 [50][53] Question: Commercial efforts to promote switching to Rytello - Management highlighted that awareness and education are key to driving physician perceptions and utilization of Rytello, with positive shifts noted in recent market research [56][60] Question: Inventory dynamics and corrections - Management explained that higher inventory levels were due to several factors, including seasonality and anticipation of pricing issues, but they believe the situation has stabilized [64][66] Question: Reasons for physicians not reordering Rytello - Management indicated that discontinuations are largely reflective of clinical trial data, with no new surprises in market research [87] Question: Demand increase sustainability - Management expressed optimism about sustaining the recent 10% demand increase, attributing it to effective messaging and physician engagement [97]

Financial Data and Key Metrics Changes - As of March 31, 2025, the company had approximately $457.5 million in cash, cash equivalents, restricted cash, and marketable securities, down from $502.9 million as of December 31, 2024 [43] - Total net product revenue for Q1 2025 was $39.4 million, a decrease of $8 million from Q4 2024, attributed to an inventory drawdown among distributors [43][44] - Research and development expenses for Q1 2025 were $15.1 million, compared to $29.4 million for the same period in 2024, primarily due to lower clinical trial costs [44][45] - Selling, general, and administrative expenses increased to $40 million in Q1 2025 from $27.1 million in the same period last year, mainly due to higher personnel expenses [45] Business Line Data and Key Metrics Changes - Q1 Rytello net revenues were $39.4 million, down from Q4, with demand relatively flat at a 1% increase compared to the previous thirteen weeks [6][44] - Approximately 900 sites of care utilized Rytello as of April 2025, an increase of almost 300 new sites since the end of Q4 [24] - Demand grew approximately 10% in the four-week period ending April 25 compared to the prior four-week period, marking the highest month-over-month growth since October 2024 [25] Market Data and Key Metrics Changes - The estimated U.S. total addressable market for Rytello is approximately 15,400 lower-risk MDS patients, with significant opportunities to expand use in earlier lines of treatment [15] - In Europe, the estimated total addressable market for Rytello is approximately 80% of the U.S. market [15] - Approximately 85% of U.S. covered lives are now under favorable Rytello medical coverage policies consistent with FDA label and NCCN guidelines [26] Company Strategy and Development Direction - The company's top priority is the successful commercialization of Rytello in the U.S., with plans to increase brand awareness and refine marketing efforts [5][17] - The company is pursuing a disciplined path for EU commercialization, planning to engage third-party partners to minimize investment while focusing on U.S. efforts [11][28] - The ongoing Phase III trial for Imetelstat in relapsed-refractory myelofibrosis could potentially double the commercial opportunity for Rytello if successful [12][48] Management's Comments on Operating Environment and Future Outlook - Management acknowledged that Q1 sales were modest and do not reflect the product's true commercial potential, emphasizing the commitment to making Rytello part of the standard of care [47] - The company is encouraged by early performance indicators and is ready to make adjustments to improve commercial execution [47] - The interim analysis for the Phase III trial is expected in the second half of 2026, with management optimistic about the potential outcomes [49] Other Important Information - The company is in the process of identifying a permanent CEO, with a decision expected within the next six months [12] - The company plans to maintain financial discipline while investing in the commercialization strategy for Rytello [46] Q&A Session Summary Question: What are the expectations for Q2 given the inventory impacts and demand improvements? - Management noted that inventory issues have been addressed and expects continued momentum into Q2, with new commercial representatives expected to impact later in the year [50][53] Question: Can you comment on commercial efforts to promote switching to Rytello? - Management highlighted that awareness and education are key, with positive shifts in perceptions regarding Rytello's efficacy and cytopenia management among physicians [56][58] Question: What is the current status of inventory levels? - The inventory at the end of Q1 was on the lower end of two to two and a half weeks, indicating a drawdown from the higher levels seen previously [93] Question: Are new patient starts exceeding discontinuations? - Management indicated that while there is no perfect data, new patient starts are promising, with approximately 25% of new starts occurring in the first and second lines of therapy [96] Question: What drove the incremental cash burn in Q1? - The cash burn was higher due to payouts of bonuses and early investments in inventory, typical for Q1 [97]