Kura Oncology(US:KURA)2021-02-24 18:43Financial Data and Key Metrics Changes - Research and Development (R&D) expenses for Q4 2020 were $17.5 million, up from $13.5 million in Q4 2019. Full year R&D expenses for 2020 were $16.4 million compared to $47.8 million in 2019 [25]. - General and Administrative (G&A) expenses for Q4 2020 were $8.8 million, compared to $5.5 million in Q4 2019. Full year G&A expenses for 2020 were $31.5 million, up from $19.7 million in 2019 [26]. - Net loss for Q4 2020 was $26.2 million, compared to a net loss of $17.9 million in Q4 2019. The full year net loss for 2020 was $89.6 million, compared to $63.1 million in 2019 [27][28]. - Cash, cash equivalents, and short-term investments were $633.3 million as of December 31, 2020, compared to $236.9 million as of December 31, 2019 [29]. Business Line Data and Key Metrics Changes - The menin inhibitor KO-539 showed promising preliminary clinical data in patients with relapsed or refractory acute myeloid leukemia, demonstrating single-agent activity and a favorable safety profile [7][8]. - The farnesyl transferase inhibitor tipifarnib received breakthrough therapy designation from the FDA for treating recurrent or metastatic HRAS mutant head and neck squamous cell carcinoma, with an objective response rate of approximately 50% [14][15][17]. Market Data and Key Metrics Changes - The breakthrough therapy designation for tipifarnib acknowledges the significant unmet need for patients with HRAS mutant head and neck squamous cell carcinoma [16]. - The total addressable population for tipifarnib may be as high as 50% of head and neck squamous cell carcinoma patients [19]. Company Strategy and Development Direction - The company is focused on advancing its clinical-stage oncology drug candidates through an accelerated development and fast-to-market strategy [5]. - Plans include initiating genetically enriched Phase 1 expansion cohorts for KO-539 and a Phase 1/2 proof-of-concept study for tipifarnib in combination with a PI3 kinase alpha inhibitor [31]. Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's financial position, with over $600 million in cash to support ongoing programs into 2024 [6][29]. - The company aims to refine its clinical development plans based on FDA feedback, focusing on identifying a minimum safe and efficacious dose for KO-539 [50][52]. Other Important Information - The company is exploring the development of a next-generation farnesyl transferase inhibitor with improved properties compared to tipifarnib [22]. - The anticipated milestones for 2021 include the initiation of Phase 1 expansion cohorts for KO-539 and the combination study for tipifarnib [31]. Q&A Session Summary Question: What drove the decision to dose escalate to 800 milligrams? - Management indicated that the decision was based on the lack of observed toxicity and the potential for good safety and tolerability, allowing for continued dose escalation [34][35]. Question: Will the 600 milligram dose be used in the expansion cohorts? - Management stated that they are evaluating both lower and higher doses in the expansion cohorts to determine the optimal dose for efficacy [37][39]. Question: What is the rationale for the change in protocol? - The change was driven by discussions with the FDA, focusing on identifying a minimum safe and efficacious dose to maximize therapeutic benefit [46][50]. Question: When will the recommended Phase 2 dose be determined? - Management indicated that the timing for determining the recommended Phase 2 dose is still pending further data from the ongoing studies [61][63]. Question: What is the expected timeline for filing for tipifarnib? - Management expressed optimism about the ongoing registrational study and the potential for future combination studies, but did not provide specific timelines for filing [64].