Core Insights - Syndax Pharmaceuticals is experiencing significant growth driven by its products Revuforj and Niktimvo, with a strong commercial performance expected to continue into 2026 [4][7]. Product Performance - Revuforj has shown a 38% growth in the fourth quarter, attributed to its use in KMT2A-rearranged acute myeloid leukemia (AML) and new momentum in NPM1-mutant AML following recent guideline inclusion and approval [2][3]. - Niktimvo has generated $152 million in net sales within its first 11 months on the market for chronic graft-versus-host disease (GVHD), indicating strong uptake [3][7]. - The company anticipates Revuforj's average treatment duration to increase from 4-6 months to 6-12 months as maintenance adoption rises from 35-40% to an expected 70-80% [6][12]. Market Opportunities - The U.S. market for Niktimvo in the third-line-plus setting is estimated at approximately 6,500 patients, with a frontline opportunity of around 17,000 patients, suggesting a potential approach to $1 billion in sales [5][15]. - Syndax expects to dominate the market share in NPM1-mutant AML, although it is currently too early to quantify [8]. Financial Outlook - The company has guided flat operating expenses at $400 million for 2026 and is fully funded, indicating no immediate need for additional cash [23]. - Syndax's collaboration with Incyte involves a 50/50 profit split, with expectations that 25-30% of Incyte's reported net sales will contribute to Syndax's revenue [16][17]. Clinical Development - Syndax is advancing a Phase II trial in idiopathic pulmonary fibrosis (IPF) with results expected in the second half of 2026, and a Phase II trial combining Niktimvo with Jakafi is anticipated to read out in early 2027 [18][20]. - The company is also exploring myelofibrosis treatment options, with preclinical work showing promising results [13][14].

Core Insights - Oryzon Genomics reported promising clinical data for iadademstat, a selective LSD1 inhibitor, in treating acute myeloid leukemia (AML) at the 67th ASH Annual Meeting, indicating potential for significant clinical benefits in combination with standard therapies [1][4] Group 1: Clinical Study Results - The ALICE-2 study showed a 100% overall response rate (ORR) and a 90% composite complete remission (CCR) rate in newly diagnosed AML patients treated with iadademstat, azacitidine, and venetoclax [2][5] - In the FRIDA study, 67% CCR and 47% combined CR and CRh were observed in patients with FLT3-mutant relapsed/refractory AML, demonstrating the efficacy of iadademstat combined with gilteritinib [3][5] Group 2: Safety and Tolerability - Both clinical studies reported that iadademstat was safe and well tolerated, with adverse event profiles comparable to other combination treatments in AML [2][3] - The ongoing dose-finding studies are focused on determining the maximum tolerated dose (MTD) for iadademstat in these patient populations [2][3] Group 3: Future Development and Strategic Partnerships - The CEO of Oryzon emphasized the potential of iadademstat as a best-in-class combination agent, indicating plans for future clinical development and exploration of strategic partnerships to maximize its value [4][6] - Oryzon is also expanding iadademstat's clinical applications beyond hematological cancers, with ongoing trials in sickle cell disease and essential thrombocythemia [7]

Core Insights - Telomir Pharmaceuticals, Inc. has announced new in vitro results indicating that Telomir-1 adds KDM5 family inhibition to its previously reported KDM2/KDM6 and DNA methylation activity, suggesting a potential advancement in epigenetic therapy [1] Company Summary - Telomir Pharmaceuticals, Inc. is a preclinical-stage biotechnology company focused on developing therapies that target the root causes of cancer, aging, and age-related diseases [1] - The company is listed on NASDAQ under the ticker TELO [1] Industry Summary - The results from Telomir-1 may represent a novel frontier in epigenetic therapy, an area where no existing candidates have demonstrated comparable breadth [1] - The inhibition of three members of the KDM5 histone demethylase family by Telomir-1 highlights the potential for innovative therapeutic approaches in the field of epigenetics [1]

Summary of Prelude Therapeutics (PRLD) FY Conference Call - June 11, 2025 Company Overview - **Company**: Prelude Therapeutics (PRLD) - **Focus**: Development of targeted therapies, specifically SMARCA2 and CAT6 degraders for cancer treatment Key Points on SMARCA2 Degraders - **Agents in Development**: Two SMARCA2 degraders are in the clinic, one oral and one intravenous (IV) [2][3] - **IV Agent**: The IV agent has shown proof of concept for the novel pathway, with ongoing dose escalation and a maximum dose selected at 500 mg/m² [6][12] - **Efficacy Data**: An objective response rate of approximately 23% was reported for the IV agent, significantly higher than the expected 5-10% for the patient population [10][11] - **Patient Population**: Focus on upper gastrointestinal (GI) and non-small cell lung cancer (NSCLC) patients, particularly those with SMARCA4 mutations [8][9] - **Next Steps**: Full data set expected by the end of the year, with plans for a combination study with docetaxel [12][13] Key Points on Oral Degrader - **Development Status**: The oral agent is in dose escalation at 60 mg, with expectations to select a dose by the end of the year [23] - **Advantages**: Oral administration is preferred by patients for convenience and continuous dosing, potentially increasing therapeutic activity [16][17] - **Safety Profile**: Both agents have shown a clean safety profile with no dose-limiting toxicities (DLTs) reported [25][26] CAT6 Degrader Insights - **Target**: CAT6 is an epigenetic target with a focus on ER-positive breast cancer due to observed amplifications [34][37] - **Mechanism of Action**: The company is developing selective degraders for CAT6, aiming to reduce toxicities associated with existing therapies [38][39] - **Development Plans**: Plans to advance CAT6 into IND (Investigational New Drug) application next year, depending on capital allocation [45] Combination Strategies - **Potential Combinations**: Preclinical studies indicate potential synergies with immunotherapies like pembrolizumab and chemotherapy [28][29] - **Target Patient Populations**: Focus on frontline NSCLC patients, as they have limited treatment options and poor progression-free survival [30][31] Financial Overview - **Cash Position**: As of the latest report, the company has $103 million in cash, expected to last into Q2 2026, covering ongoing projects including SMARCA4, oral agents, and CAT6 [51] Additional Considerations - **ADC Pipeline**: The company is exploring antibody-drug conjugates (ADCs) to deliver degraders directly to tumors, enhancing treatment specificity [46][48] - **Strategic Collaborations**: Collaboration with Absella for antibody development is underway, aiming to expand treatment options for aggressive cancers [50] This summary encapsulates the critical insights and developments discussed during the conference call, highlighting Prelude Therapeutics' strategic focus on innovative cancer therapies and their ongoing clinical programs.