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We are a clinical-stage biopharmaceutical company focused on developing precision medicines for cancer treatment Our pipeline features small molecule candidates targeting key cancer signaling pathways with strong scientific and clinical justifications We aim to personalize therapies by pairing these treatments with molecular or cellular diagnostics to identify patients most likely to benefit Our strategy involves advancing product candidates through internal development and strategic partnerships, while retaining significant rights for development and commercialization.
Tipifarnib, our lead product candidate, is a potent, selective, and orally bioavailable farnesyl transferase inhibitor with a proven track record in cancer treatment Previously studied in over 5,000 cancer patients, tipifarnib demonstrated durable and compelling anti-cancer activity, particularly in specific patient populations, with a manageable side effect profile We are actively exploring the potential of tipifarnib across multiple solid tumor and hematologic cancer indications to expand its therapeutic applications.
Our most advanced solid tumor indication targets patients with head and neck squamous cell carcinoma (HNSCC) harboring HRAS mutations In September 2017, we announced that our Phase 2 clinical trial of tipifarnib in HRAS mutant HNSCC met its primary efficacy endpoint Based on feedback from the FDA and other regulators, we launched a registration-directed trial in November 2018, a global, multicenter, open-label study enrolling at least 59 patients who previously received platinum-based therapy, expected to complete enrollment in two years After successful results in the initial trial, we expanded to include patients with other HRAS mutant squamous cell carcinomas (SCCs) We anticipate sharing additional data from this Phase 2 trial in the second half of 2019.
We are advancing our tipifarnib development program targeting HRAS mutant solid tumors and exploring its potential use in various hematologic and solid tumor indications through CXCL12 pathway biomarkers In December 2018, we reported preliminary results from our Phase 2 trial of tipifarnib in patients with relapsed or refractory peripheral T-cell lymphomas (PTCL), highlighting a significant link between CXCL12 expression and clinical benefit Notably, the data provided clinical proof-of-concept in patients with angioimmunoblastic T-cell lymphoma (AITL), an aggressive PTCL subtype often exhibiting high CXCL12 levels We expect to release additional results from this study by mid-2019.
We are investigating the potential of CXCL12 pathway biomarkers to enhance patient selection in relapsed or refractory acute myeloid leukemia (AML) and chronic myelomonocytic leukemia (CMML) through an ongoing Phase 2 clinical trial We expect to receive additional data from the CMML study in 2019, which may further inform the development of targeted treatment strategies for these hematologic malignancies.
In 2019, we identified a potential link between CXCL12 expression and clinical benefits from tipifarnib in pancreatic cancer patients These findings suggest that tipifarnib could be effective across a broader range of hematologic and solid tumor indications, particularly in cancers where the CXCL12 pathway influences tumor initiation and progression We are actively exploring further clinical development opportunities to expand the therapeutic applications of tipifarnib in these settings.
KO-947 is a potent and selective small molecule inhibitor of ERK, being developed as a targeted treatment for tumors with dysregulated MAPK pathway activity caused by mutations or other mechanisms Preclinical data demonstrates KO-947's anti-tumor activity in KRAS- and BRAF-mutant adenocarcinomas, as well as certain squamous cell carcinoma subsets An ongoing Phase 1 clinical trial in patients with solid tumors aims to evaluate its safety and efficacy, with data from the dose-escalation phase expected in 2019.
KO-539 is a potent and selective small molecule inhibitor targeting the menin-MLL protein-protein interaction, demonstrating promising anti-tumor activity in preclinical studies It has shown effectiveness against genetically defined subsets of acute leukemia, including MLL gene rearrangements and NPM1 mutations The FDA has cleared our IND application for KO-539, and we are preparing to initiate Phase 1 clinical trials.
1 clinical trial in relapsed or refractory AML in the second quarter of 2019.
Kura Oncology, Inc is a Delaware corporation incorporated in August 2014, referenced as "we," "us," and "our" in this Annual Report for periods prior to its reverse merger on March 6, 2015 Following the Merger, the company is also known as Kura Oncology, Inc., a Delaware corporation originally incorporated in November 2007 and formerly named Zeta Acquisition Corp III.
Our strategy focuses on discovering, acquiring, developing, and commercializing innovative anti-cancer agents targeted at oncology indications with substantial unmet medical needs and strong commercial potential The company's approach emphasizes identifying promising therapies, acquiring promising assets, developing them effectively, and bringing innovative solutions to market to address crucial gaps in cancer treatment This comprehensive strategy aims to deliver impactful cancer treatments while maximizing commercial opportunities in high-need areas.
Focus on developing novel, small molecule product candidates for the treatment for cancer;
Identify molecular, genetic or other tumor-related characteristics to identify patients more likely to benefit from our product candidates;
Leverage clinical and pathology trends towards comprehensive tumor profiling and the use of companion diagnostics;
Prioritize development of tipifarnib in clinical indications of high unmet need where improved outcomes are associated with specific biomarkers;
Advance our programs through a combination of internal development and strategic partnerships;
Maintain significant development and commercial rights to our product candidates; and
Precision Medicines in Cancer Treatment
Advancements in cancer genetics and innovative molecular diagnostic tools are essential for understanding why certain patients respond well to specific therapies, while others experience minimal benefit This personalized approach in cancer treatment enhances the potential for developing safer, more targeted, and more effective therapies The new era of cancer drug discovery is focused on leveraging genetic insights to revolutionize treatment options for patients with various cancer types.
Our goal is to improve patient outcomes and reduce healthcare costs by matching targeted therapeutics to the patients who will benefit most through precision medicine We are developing a pipeline of small molecule candidates that inhibit mutated or abnormal cellular pathways responsible for cancer growth, paired with molecular diagnostics to identify patients with tumors most likely to respond This personalized treatment approach aims to enhance effectiveness and optimize cancer care.
Precision medicine has revolutionized cancer treatment with the development of targeted therapies such as small molecule inhibitors against the epidermal growth factor receptor (EGFR) for advanced lung cancer Patients with EGFR mutations treated with these inhibitors experience response rates around 65%, significantly higher than the approximately 10% seen in unselected lung cancer patients Erlotinib (Tarceva®) was approved in the U.S as a first-line treatment for non-small cell lung cancer (NSCLC) with EGFR mutations Other targeted therapies approved through precision medicine include inhibitors targeting ALK, BCR-ABL, BRAF, and TRK, demonstrating the impact of personalized approaches in improving cancer outcomes.
Precision medicine offers significant advantages over traditional drug development by enabling the evidence-based selection of patients most likely to respond to targeted therapies, leading to higher success rates in clinical trials and requiring fewer participants This approach promises faster clinical development, especially in areas with high unmet medical needs, while reducing development costs and timelines Success in precision medicine relies on a deep understanding of tumor biology and the mechanisms of action of drug candidates, which we have achieved through extensive translational research on each of our programs.
Our Approach to Development of Precision Medicines in Oncology
Translational research combines basic, preclinical, and clinical data to develop a comprehensive understanding of potential therapeutics, guiding precision medicine approaches We assess product candidates through in vitro and in vivo experiments, utilizing tools like patient-derived xenograft (PDX) models, which retain the key histologic and genetic features of donor tumors PDX models are valuable for predicting clinical outcomes and are increasingly used in preclinical drug evaluation, biomarker discovery, and personalized medicine Our preclinical PDX studies aim to validate clinical data and identify the most promising indications for our product candidates.