Kura Oncology(KURA) - 2020 Q4 - Annual Report

Part I Business Kura Oncology is a clinical-stage biopharmaceutical company developing precision cancer medicines, with lead candidates tipifarnib and KO-539 in advanced clinical trials Overview and Pipeline Kura Oncology is a clinical-stage biopharmaceutical company with lead candidates tipifarnib for HRAS mutant HNSCC and KO-539 for AML, plus a next-generation FTI in discovery - Kura Oncology is a clinical-stage biopharmaceutical company developing small molecule product candidates targeting cancer signaling pathways, paired with molecular diagnostics[23] Clinical Development Pipeline as of December 31, 2020 | Program | Indication | Stage | | :--- | :--- | :--- | | Tipifarnib (FTI) | HRAS mutant HNSCC | Registration-Directed Trial (AIM-HN) Ongoing | | | PI3Kα mutant and HRAS overexpressed HNSCC | PI3Kα inhibitor combination study expected in 2H 2021 | | KO-539 (Menin Inhibitor) | Acute Myeloid Leukemia (AML) | Phase 1 expansion cohorts expected to begin mid-2021 | | Next-Generation FTI | Solid tumors | Development candidate nomination expected mid-2021 | Clinical Programs Clinical development focuses on tipifarnib for HRAS mutant HNSCC (Breakthrough Therapy Designation) and KO-539 for AML, with a next-generation FTI program initiated - The FDA granted Breakthrough Therapy Designation to tipifarnib for recurrent or metastatic HRAS mutant HNSCC with variant allele frequency ≥ 20% after progression on platinum-based chemotherapy, based on data from the Phase 2 RUN-HN trial[27][60] - The AIM-HN registration-directed trial for tipifarnib was amended in July 2020 to enroll patients with any HRAS mutation and to reflect evolving standards of care, which will require enrolling an increased number of patients; timelines are uncertain due to COVID-19[26][59] - Preliminary data from the KOMET-001 trial of KO-539 in AML, presented at ASH 2020, showed clinical or biological activity in 6 of 8 evaluable patients, including two complete remissions; the drug was well tolerated[31][75] - The company is advancing its KO-539 KOMET-001 trial, having completed the 600 mg dose cohort and currently evaluating an 800 mg dose, with plans to initiate Phase 1 expansion cohorts at lower doses in parallel with dose escalation[32][76] - A discovery-stage program for a next-generation Farnesyl Transferase Inhibitor (FTI) has commenced, with a development candidate expected to be nominated in mid-2021, aiming for improved pharmacokinetic and physicochemical properties[77][78] License and Asset Purchase Agreements Kura Oncology holds exclusive global rights for tipifarnib from Janssen and KO-539 from the University of Michigan, involving milestone and royalty payment obligations - Kura licensed exclusive global rights for tipifarnib from Janssen, with obligations to pay up to $25.0 million in regulatory milestones and up to $50.0 million in sales milestones, plus tiered royalties in the low teens percentages of net sales[79][80] - The company licensed exclusive worldwide rights for its menin-KMT2A program (including KO-539) from the University of Michigan, with obligations to pay up to $3.4 million in development and regulatory milestones, plus tiered royalties in the low single-digit percentages of net sales[82] Competition The company faces significant competition for tipifarnib from established HNSCC therapies and for KO-539 from other menin-KMT2A inhibitors - While no farnesyl transferase inhibitors are currently approved for cancer, tipifarnib will compete with established therapies for HNSCC, including cetuximab (Erbitux®), nivolumab (Opdivo®), and pembrolizumab (Keytruda®)[87][88] - In the menin-KMT2A inhibitor space, Kura faces competition from other companies developing similar targeted therapies, including Syndax and Biomea[89] Intellectual Property Kura's IP strategy combines licensed and owned patents, focusing on method-of-use patents for tipifarnib (composition-of-matter expired) and composition-of-matter patents for KO-539 - The composition-of-matter patents for tipifarnib expired in the U.S. and Europe in 2016, making the company's protection strategy reliant on method-of-use patents for specific patient populations, such as those with HRAS mutant HNSCC[99] - The company has issued U.S. patents covering the composition of matter for KO-539 and related compounds, as well as methods for their use in treating cancers[99] Government Regulation The company's products are subject to extensive FDA and global regulation, utilizing expedited programs like Fast Track and Breakthrough Therapy Designation to accelerate development - The FDA drug approval process involves preclinical testing, submitting an Investigational New Drug (IND) application, and conducting three sequential phases of clinical trials (Phase 1, 2, and 3) to establish safety and efficacy before submitting a New Drug Application (NDA)[108][111][113] - The company leverages FDA's expedited programs, with tipifarnib having received Fast Track Designation, which allows for more frequent FDA interaction and rolling review of the NDA[121][122] - Tipifarnib has also received Breakthrough Therapy Designation, designed to expedite the development and review of drugs for serious conditions where preliminary clinical evidence shows substantial improvement over available therapies[125][126] - KO-539 has received Orphan Drug Designation for AML, which provides incentives such as tax credits and eligibility for seven years of market exclusivity in the U.S. upon approval for that indication[127][128] Human Capital As of December 31, 2020, Kura Oncology employed 89 people, with a focus on R&D, and established a Culture and Inclusion Leadership Committee in 2020 - As of December 31, 2020, the company had 89 employees, with 59 in R&D and supply chain and 30 in commercial and G&A roles[166] - In 2020, the company established a Culture and Inclusion Leadership Committee to obtain employee feedback and focus on corporate culture, diversity, and inclusion[169] Risk Factors Kura Oncology faces significant risks including clinical trial success dependency, COVID-19 impacts, financial losses, reliance on third parties, and limited intellectual property protection for tipifarnib - The COVID-19 pandemic could continue to adversely impact clinical trial conduct, including delaying site startups, slowing patient enrollment, and disrupting the supply of product candidates[175] - The company is highly dependent on the success of its two lead product candidates, tipifarnib and KO-539, which are still in clinical development and may never receive regulatory approval[176] - The company has a history of losses, expects to incur losses for the foreseeable future, and will need to obtain substantial additional capital, which may cause dilution to stockholders or restrict operations[216][225] - The company relies on third-party contractors for manufacturing and to conduct clinical trials, which reduces control over these activities and introduces risks related to performance, quality, and timelines[229][235] - The composition of matter patents for tipifarnib expired in 2016, making the company reliant on method-of-use patents, which may provide weaker protection and expose it to competition from generic versions for other indications[245][280] Properties Kura Oncology leases corporate office spaces in San Diego (13,420 sq. ft.) and Boston (16,541 sq. ft.), deemed sufficient for current operations - The company leases 13,420 sq. ft. for its headquarters in San Diego, CA (lease expires Nov 2025) and 16,541 sq. ft. in Boston, MA (lease expires July 2024)[366] Legal Proceedings Kura Oncology is not currently involved in any material legal proceedings - The company is not currently a party to any material legal proceedings[367] Part II Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities Kura Oncology's common stock trades on Nasdaq under "KURA"; the company has never paid and does not intend to pay cash dividends - The company's common stock has been listed on the Nasdaq Global Select Market under the symbol "KURA" since November 5, 2015[371] - The company has never paid cash dividends and does not intend to in the foreseeable future, with future earnings to be retained for business growth[373] Management's Discussion and Analysis of Financial Condition and Results of Operations Kura Oncology's net loss increased to $89.6 million in 2020 due to higher R&D and G&A expenses, but its financial position was strengthened by $459.0 million in public offerings, providing liquidity into 2024 Results of Operations In 2020, R&D expenses increased to $60.4 million due to KO-539 and discovery programs, while G&A expenses rose to $31.5 million from higher personnel and professional costs Results of Operations (in thousands) | | 2020 | 2019 | Change | | :--- | :--- | :--- | :--- | | Research and development expenses | $60,397 | $47,826 | $12,571 | | General and administrative expenses | $31,502 | $19,653 | $11,849 | | Other income, net | $2,274 | $4,339 | $(2,065) | Research and Development Expenses Breakdown (in thousands) | Category | 2020 | 2019 | Change | | :--- | :--- | :--- | :--- | | Tipifarnib-related costs | $26,025 | $26,517 | $(492) | | KO-539-related costs | $6,629 | $2,496 | $4,133 | | KO-947-related costs | $2,301 | $3,416 | $(1,115) | | Discovery stage programs | $2,255 | $318 | $1,937 | | Personnel costs and other expenses | $19,227 | $11,652 | $7,575 | | Share-based compensation expense | $3,960 | $3,427 | $533 | | Total R&D Expenses | $60,397 | $47,826 | $12,571 | - The increase in R&D expenses was primarily due to higher costs for the Phase 1/2 clinical trial of KO-539, which began in September 2019, and increased activities for new discovery stage programs[402] - The increase in G&A expenses was driven by rises of $2.9 million each in share-based compensation, pre-commercial planning, and personnel costs, plus a $2.3 million increase in professional and legal services[403] Liquidity and Capital Resources As of December 31, 2020, Kura Oncology held $633.3 million in cash and investments, secured through $459.0 million in public offerings, expected to fund operations into 2024 - The company ended 2020 with $633.3 million in cash, cash equivalents, and short-term investments[394][412] - In 2020, the company completed two public offerings, raising net proceeds of approximately $324.1 million in December and $134.9 million in May[406][407] - Management believes that existing cash, cash equivalents, and short-term investments are sufficient to fund operating expenses and capital expenditure requirements into 2024[412] Summary of Net Cash Flow (in thousands) | | 2020 | 2019 | Change | | :--- | :--- | :--- | :--- | | Net cash used in operating activities | $(69,830) | $(54,760) | $(15,070) | | Net cash used in investing activities | $(99,936) | $(46,325) | $(53,611) | | Net cash provided by financing activities | $469,334 | $111,101 | $358,233 | Quantitative and Qualitative Disclosures About Market Risk Kura Oncology's primary market risk is interest rate risk on its investments and term loan, though a 10% rate change is not expected to be material - The company's main market risk is interest rate risk on its cash, cash equivalents, short-term investments, and its term loan facility with SVB[427][428] - Management does not believe a hypothetical 10% change in interest rates as of December 31, 2020, would have a material effect on the fair value of its investment portfolio or its interest expense[427][428] Financial Statements and Supplementary Data This section presents Kura Oncology's audited financial statements for 2020, including the independent auditor's report, detailing financial position, operations, equity, and cash flows Report of Independent Registered Public Accounting Firm Ernst & Young LLP issued unqualified opinions on Kura Oncology's 2020 financial statements and internal controls, identifying accrued clinical trial R&D expenses as a Critical Audit Matter - The independent auditor, Ernst & Young LLP, issued an unqualified opinion on the company's financial statements and the effectiveness of its internal control over financial reporting as of December 31, 2020[441][475] - The audit identified "Clinical Trial Research and Development Expenses and Accruals" as a Critical Audit Matter due to the challenging and subjective judgments required to estimate the progress and stage of completion of R&D activities[478][480] Financial Statements Kura Oncology's 2020 financial statements show total assets grew to $647.2 million, with a net loss of $89.6 million (or $1.69 per share), driven by increased expenses Balance Sheet Highlights (in thousands) | | Dec 31, 2020 | Dec 31, 2019 | | :--- | :--- | :--- | | Cash and cash equivalents | $325,493 | $26,135 | | Short-term investments | $307,827 | $210,756 | | Total Assets | $647,212 | $241,972 | | Total Liabilities | $36,307 | $23,191 | | Total Stockholders' Equity | $610,905 | $218,781 | Statement of Operations Highlights (in thousands, except per share data) | | 2020 | 2019 | | :--- | :--- | :--- | | Total operating expenses | $91,899 | $67,479 | | Net Loss | $(89,625) | $(63,140) | | Net loss per share, basic and diluted | $(1.69) | $(1.51) | Statement of Cash Flows Highlights (in thousands) | | 2020 | 2019 | | :--- | :--- | :--- | | Net cash used in operating activities | $(69,830) | $(54,760) | | Net cash used in investing activities | $(99,936) | $(46,325) | | Net cash provided by financing activities | $469,334 | $111,101 | Controls and Procedures As of December 31, 2020, management and auditors concluded Kura Oncology's disclosure controls and internal control over financial reporting were effective, with no material changes - Management, including the CEO and CFO, concluded that the company's disclosure controls and procedures were effective as of December 31, 2020[433] - Management concluded that internal control over financial reporting was effective as of December 31, 2020, based on the COSO framework; this assessment was audited by Ernst & Young LLP, which issued an unqualified opinion[434][436] - There were no changes in internal control over financial reporting during the fourth quarter of 2020 that materially affected, or are reasonably likely to materially affect, such controls[437] Part III Directors, Executive Officers, Corporate Governance, Compensation, and Principal Accounting Fees Information for Items 10-14, covering governance, compensation, and related matters, is incorporated by reference from the company's forthcoming 2021 Proxy Statement - Information regarding Directors, Executive Officers, Corporate Governance (Item 10), Executive Compensation (Item 11), Security Ownership (Item 12), Certain Relationships and Related Transactions (Item 13), and Principal Accounting Fees and Services (Item 14) is incorporated by reference from the company's forthcoming 2021 Proxy Statement[451][453][454][455][456] Part IV Exhibits, Financial Statement Schedules This section lists filed financial statements and provides a comprehensive index of all exhibits, including corporate documents and material contracts - This item contains the list of financial statements filed with the annual report and an index of all exhibits, including material contracts and corporate governance documents[458][460] Form 10-K Summary No summary is provided for the Form 10-K - No summary is provided for the Form 10-K[464]