Kura Oncology(US:KURA)2021-05-09 13:47Financial Data and Key Metrics Changes - Research and development expenses for Q1 2021 were $20.3 million, up from $12.6 million in Q1 2020, primarily due to increased clinical trial costs and personnel expenses [15] - General and administrative expenses for Q1 2021 were $10.6 million, compared to $7.6 million in Q1 2020, mainly due to higher personnel costs and noncash share-based compensation [16] - Net loss for Q1 2021 was $30.7 million, or $0.46 per share, compared to a net loss of $19.2 million, or $0.42 per share, in Q1 2020 [16] - As of March 31, 2021, cash, cash equivalents, and short-term investments totaled $603.9 million, down from $633.3 million as of December 31, 2020, with sufficient funds projected to last into 2024 [16] Business Line Data and Key Metrics Changes - The KO-539 program is positioned as a potentially best-in-class menin inhibitor, with ongoing clinical trials aimed at optimizing Phase II dosing [6][7] - The company is also advancing the farnesyl transferase inhibitor program, with a focus on HRAS mutant head and neck squamous cell carcinoma, which received breakthrough designation from the FDA [10][11] Market Data and Key Metrics Changes - The menin inhibitor space is experiencing heightened activity, with new developments from competitors validating the therapeutic target in acute myeloid leukemia (AML) [5] - The company aims to expand its market presence by exploring additional genetic subtypes and indications, including acute lymphocytic leukemia and myelodysplastic syndrome [8] Company Strategy and Development Direction - The company plans to conduct a comprehensive clinical development plan for KO-539, including both monotherapy and combination studies [6] - The strategy includes a focus on identifying optimal dosing and expanding into additional genetic subtypes once the recommended Phase II dose is established [30] - The company is also preparing a Phase I/II proof-of-concept study for tipifarnib in combination with a PI3 kinase alpha inhibitor [11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the KO-539 program's potential, citing a favorable safety profile and wide therapeutic window [6][24] - The company is optimistic about the potential for tipifarnib to become the first approved small molecule targeted therapy in head and neck cancer [10] - Management highlighted the importance of defining the optimal Phase II dose and the potential for increased response rates in AML treatment [21][63] Other Important Information - The company is actively engaging with key opinion leaders and a global steering committee to refine its clinical development plans [7] - The anticipated milestones for 2021 include the initiation of genetically enriched Phase Ib expansion for KO-539 and the nomination of a development candidate for the next-generation farnesyl transferase inhibitor [17] Q&A Session Summary Question: Thoughts on Amgen's KRAS G12C inhibitor disclosures - Management discussed the FDA's initiative to define the optimum Phase II dose and the importance of identifying the lowest dose with maximum pharmacologic and clinical activity [19][21] Question: Confidence in KO-539 compared to competitors - Management highlighted the advantages of KO-539, including a wide therapeutic window and no dependence on CYP3A4, unlike competitors [22][24] Question: Data readouts and Phase Ib expansion - Management confirmed that updates on Phase Ib data will be provided later in the year, focusing on determining the recommended Phase II dose [27][28] Question: Expansion opportunities and genetic subtypes - Management indicated ongoing efforts to explore additional genetic subtypes and the potential for KO-539 to benefit MDS patients [30][31] Question: Financial guidance and R&D spending - Management acknowledged that Q1 expenses were higher due to one-off costs but anticipated continued increases in R&D spending throughout the year [45] Question: Properties of next-generation farnesyl transferase inhibitors - Management expressed the goal of improving pharmacokinetic properties and reducing dosing frequency compared to existing therapies [46] Question: Status of AIM study and site readiness - Management noted that patient reluctance due to COVID-19 has impacted enrollment but emphasized ongoing efforts to identify and screen eligible patients [49][50] Question: Pharmacokinetic and pharmacodynamic goals for Phase Ib - Management clarified that the focus will be on understanding interpatient variability and optimizing efficacy rather than targeting specific pharmacokinetic markers [52][53] Question: Comparison of combatant versus non-covalent inhibitors - Management discussed the decision to pursue a reversible inhibitor approach for KO-539, citing concerns over potential off-target toxicity with covalent inhibitors [56] Question: Targeting NPM1 versus MLLr populations - Management highlighted the negative prognosis associated with NPM1 mutations in relapsed/refractory settings and the potential for menin inhibitors to drive MRD-negative responses [60][63]