PART I. FINANCIAL INFORMATION [ITEM 1. Condensed Financial Statements (unaudited)](index=3&type=section&id=ITEM%201.%20Condensed%20Financial%20Statements%20(unaudited)) This section presents the unaudited condensed financial statements, including balance sheets, statements of operations and comprehensive loss, stockholders' equity, and cash flows, along with detailed notes explaining the company's organization, accounting policies, investments, fair value measurements, balance sheet specifics, leases, stockholders' equity changes, share-based compensation, and significant subsequent events [Condensed Balance Sheets](index=3&type=section&id=Condensed%20Balance%20Sheets) | (In thousands) | September 30, 2022 (Unaudited) | December 31, 2021 | Change (2022 vs 2021) | | :-------------------------------- | :------------------------------- | :---------------- | :-------------------- | | **Assets** | | | | | Cash and cash equivalents | $90,937 | $90,672 | +$265 | | Short-term investments | $336,838 | $427,288 | -$90,450 | | Total current assets | $436,391 | $522,289 | -$85,898 | | Total assets | $447,988 | $534,051 | -$86,063 | | **Liabilities and Stockholders' Equity** | | | | | Total current liabilities | $24,745 | $22,455 | +$2,290 | | Total liabilities | $28,367 | $27,442 | +$925 | | Total stockholders' equity | $419,621 | $506,609 | -$86,988 | [Condensed Statements of Operations and Comprehensive Loss](index=4&type=section&id=Condensed%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) | (In thousands, except per share data) | Three Months Ended Sep 30, 2022 | Three Months Ended Sep 30, 2021 | Nine Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2021 | | :------------------------------------ | :------------------------------ | :------------------------------ | :----------------------------- | :----------------------------- | | Research and development | $24,973 | $22,367 | $70,144 | $63,765 | | General and administrative | $11,621 | $11,310 | $34,565 | $34,455 | | Total operating expenses | $36,594 | $33,677 | $104,709 | $98,220 | | Interest and other income, net | $1,090 | $311 | $1,983 | $911 | | Net Loss | $(35,504) | $(33,366) | $(102,726) | $(97,723) | | Net loss per share, basic and diluted | $(0.53) | $(0.50) | $(1.54) | $(1.47) | | Comprehensive Loss | $(37,105) | $(33,167) | $(110,353) | $(97,963) | [Condensed Statements of Stockholders' Equity](index=5&type=section&id=Condensed%20Statements%20of%20Stockholders'%20Equity) | (In thousands) | Balance at Dec 31, 2021 | Share-based compensation expense | Issuance of common stock under equity plans | Other comprehensive loss | Net loss | Balance at Sep 30, 2022 | | :----------------------------- | :---------------------- | :------------------------------- | :------------------------------------------ | :----------------------- | :------- | :---------------------- | | Common Stock (Shares) | 66,572 | — | 322 | — | — | 66,894 | | Common Stock (Par Value) | $7 | — | — | — | — | $7 | | Additional Paid-In Capital | $941,359 | $19,513 | $3,852 | — | — | $964,724 | | Accumulated Other Comprehensive Loss | $(1,789) | — | — | $(7,627) | — | $(9,416) | | Accumulated Deficit | $(432,968) | — | — | — | $(102,726) | $(535,694) | | Total Stockholders' Equity | $506,609 | $19,513 | $3,852 | $(7,627) | $(102,726) | $419,621 | [Condensed Statements of Cash Flows](index=7&type=section&id=Condensed%20Statements%20of%20Cash%20Flows) | (In thousands) | Nine Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2021 | | :------------------------------------------ | :----------------------------- | :----------------------------- | | Net cash used in operating activities | $(83,532) | $(81,185) | | Net cash provided by (used in) investing activities | $79,945 | $(200,896) | | Net cash provided by (used in) financing activities | $3,852 | $(4,229) | | Net increase (decrease) in cash, cash equivalents and restricted cash | $265 | $(286,310) | | Cash, cash equivalents and restricted cash at end of period | $91,147 | $39,393 | [Notes to Condensed Financial Statements (unaudited)](index=8&type=section&id=Notes%20to%20Condensed%20Financial%20Statements%20(unaudited)) [1. Organization and Basis of Presentation](index=8&type=section&id=1.%20Organization%20and%20Basis%20of%20Presentation) **Kura Oncology, Inc.** is a clinical-stage biopharmaceutical company focused on precision medicines for cancer, developing small molecule product candidates targeting specific cancer signaling pathways. The unaudited condensed financial statements are prepared in accordance with **GAAP** for interim information, reflecting management's estimates and assumptions, and acknowledge the ongoing uncertainty regarding the impact of the **COVID-19** pandemic - **Kura Oncology, Inc.** is a clinical-stage biopharmaceutical company dedicated to precision medicines for cancer, developing small molecule product candidates[24](index=24&type=chunk) - The financial statements are prepared using **GAAP** for interim information, relying on management's estimates and assumptions, which are subject to inherent uncertainty[26](index=26&type=chunk)[27](index=27&type=chunk) - The company acknowledges the highly uncertain and unpredictable impact of the **COVID-19** pandemic on its business and operations[28](index=28&type=chunk) [2. Summary of Significant Accounting Policies](index=9&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies) This section outlines key accounting policies, including reclassifications, the treatment of restricted cash pledged for an office lease, the methodology for assessing allowance for credit losses on available-for-sale securities, and the calculation of net loss per share, noting that common stock equivalents are anti-dilutive due to net losses - Restricted cash of **$0.2 million** as of **September 30, 2022**, is pledged as collateral for an office lease[30](index=30&type=chunk)[31](index=31&type=chunk) - For available-for-sale securities in an unrealized loss position, the company assesses intent/requirement to sell before recovery; credit-related losses are recorded through an allowance, while other unrealized losses are in other comprehensive income (loss)[32](index=32&type=chunk) - Basic and diluted net loss per share are the same for the periods presented due to net losses, with approximately **9.1 million** common stock equivalents excluded from diluted EPS calculation as they would be anti-dilutive[36](index=36&type=chunk) [3. Investments](index=10&type=section&id=3.%20Investments) The company invests in available-for-sale securities, primarily money market funds, U.S. Treasury, corporate debt, commercial paper, non-U.S. government, supranational debt, and U.S. Agency bonds. As of September 30, 2022, the fair value of short-term investments was $336.8 million, with $9.4 million in unrealized losses, primarily due to interest rate changes, not credit risk | (In thousands) | September 30, 2022 (Fair Value) | December 31, 2021 (Fair Value) | | :------------------------------------ | :------------------------------ | :----------------------------- | | Money market funds | $80,905 | $79,895 | | U.S. Treasury securities | $142,940 | $134,833 | | Corporate debt securities | $122,207 | $207,172 | | Commercial paper | $37,718 | $53,439 | | Non-U.S. government and supranational debt securities | $25,213 | $22,908 | | U.S. Agency bonds | $8,760 | $8,936 | | Total short-term investments | $336,838 | $427,288 | | Total investments (including cash equivalents) | $417,743 | $507,183 | - As of **September 30, 2022**, the company held **35 available-for-sale securities** with a fair value of **$299.1 million** in gross unrealized loss positions, with **$110.6 million** in a continuous unrealized loss position for over **12 months**[42](index=42&type=chunk) - Unrealized losses at **September 30, 2022**, were primarily attributed to changes in interest rates, not increased credit risks, and the company does not intend to sell these securities before recovery of their amortized cost basis[42](index=42&type=chunk) [4. Fair Value Measurements](index=11&type=section&id=4.%20Fair%20Value%20Measurements) The company measures cash equivalents and short-term investments at fair value on a recurring basis, categorizing them into **Level 1** (U.S. Treasury securities, money market funds) and **Level 2** (corporate debt, commercial paper, non-U.S. government/supranational debt, U.S. Agency bonds) inputs based on the fair value hierarchy | (In thousands) | September 30, 2022 (Total Fair Value) | Level 1 | Level 2 | | :------------------------------------ | :------------------------------------ | :-------- | :-------- | | Money market funds | $80,905 | $80,905 | $— | | U.S. Treasury securities | $142,940 | $142,940 | $— | | Corporate debt securities | $122,207 | $— | $122,207 | | Commercial paper | $37,718 | $— | $37,718 | | Non-U.S. government and supranational debt securities | $25,213 | $— | $25,213 | | U.S. Agency bonds | $8,760 | $— | $8,760 | | Total | $417,743 | $223,845 | $193,898 | - **Level 2** securities' fair values are determined using third-party valuations based on market pricing or prices for similar instruments, validated against a third-party pricing source[45](index=45&type=chunk) [5. Balance Sheet Detail](index=12&type=section&id=5.%20Balance%20Sheet%20Detail) This section provides a detailed breakdown of property and equipment, which increased to $2.7 million net, and accounts payable and accrued expenses, which rose to $22.4 million, primarily driven by accrued clinical trial R&D expenses | (In thousands) | September 30, 2022 | December 31, 2021 | | :------------------------------------ | :----------------- | :---------------- | | Property and equipment, net | $2,734 | $2,673 | | Accounts payable and accrued expenses | $22,441 | $20,192 | | - Accrued clinical trial R&D expenses | $5,471 | $2,619 | | - Accrued compensation and benefits | $7,966 | $7,923 | [6. Leases](index=13&type=section&id=6.%20Leases) The company holds **three operating leases** for administrative and R&D space expiring between **July 2024** and **November 2025**. Total operating lease liabilities were $5.4 million as of **September 30, 2022**, with a weighted-average remaining lease term of **2.6 years** and a discount rate of **5.5%** - The company has **three operating leases** for office and lab space in San Diego and Boston, expiring between **July 2024** and **November 2025**[49](index=49&type=chunk) | (In thousands) | Amount | | :----------------------- | :----- | | Total operating lease liabilities (Sep 30, 2022) | $5,384 | | Weighted-average discount rate (Sep 30, 2022) | 5.5% | | Weighted-average remaining lease term (Sep 30, 2022) | 2.6 years | | Total cash paid for operating lease liabilities (Nine Months Ended Sep 30, 2022) | $1,700 | | Total operating lease expense (Nine Months Ended Sep 30, 2022) | $1,500 | [7. Stockholders' Equity](index=13&type=section&id=7.%20Stockholders'%20Equity) In **February 2022**, the company established a new **ATM Facility** allowing the sale of up to **$150.0 million** in common stock, though no shares have been sold under this facility to date - A new **ATM Facility** was established in **February 2022**, enabling the company to offer and sell up to **$150.0 million** of common stock[51](index=51&type=chunk) - No shares have been sold under the **ATM Facility** as of the reporting date[51](index=51&type=chunk) [8. Share-Based Compensation](index=13&type=section&id=8.%20Share-Based%20Compensation) Share-based compensation expense for the nine months ended **September 30, 2022**, increased to **$19.5 million**, with R&D accounting for **$7.5 million** and G&A for **$12.0 million**. Unrecognized compensation expense for stock options and restricted stock units totaled **$48.4 million** and **$10.8 million**, respectively, to be recognized over approximately **2.6 years** | (In thousands) | Three Months Ended Sep 30, 2022 | Three Months Ended Sep 30, 2021 | Nine Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2021 | | :------------------------------------ | :------------------------------ | :------------------------------ | :----------------------------- | :----------------------------- | | Research and development | $2,492 | $2,059 | $7,545 | $5,277 | | General and administrative | $3,863 | $4,055 | $11,968 | $11,905 | | Total share-based compensation expense | $6,355 | $6,114 | $19,513 | $17,182 | - As of **September 30, 2022**, unrecognized compensation expense was approximately **$48.4 million** for stock options and **$10.8 million** for restricted stock units, expected to be recognized over a weighted average period of **2.6 years**[52](index=52&type=chunk) [9. Subsequent Events](index=14&type=section&id=9.%20Subsequent%20Events) Subsequent to the quarter end, the company entered into a Loan and Security Agreement with **Hercules Capital** for up to **$125.0 million** in term loans, drawing an initial **$10.0 million**. Additionally, it entered a Securities Purchase Agreement with **Bristol-Myers Squibb Company** (**BMS**) for a **$25.0 million** registered direct offering of common stock - On **November 2, 2022**, the company entered a Loan Agreement with **Hercules Capital, Inc.** for up to **$125.0 million** in term loans, with an initial **$10.0 million** borrowed[54](index=54&type=chunk) - The Loan Agreement includes additional tranches of up to **$115.0 million**, subject to certain conditions and approvals, with a maturity date of **November 2, 2027**[54](index=54&type=chunk) - On **November 2, 2022**, the company also entered a Securities Purchase Agreement with **BMS** to sell **1,370,171 shares** of common stock for approximately **$25.0 million** in gross proceeds[57](index=57&type=chunk) [ITEM 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=15&type=section&id=ITEM%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section provides an overview of the company's clinical-stage biopharmaceutical operations, focusing on its product candidates **ziftomenib**, **tipifarnib**, and **KO-2806**, and their development progress. It details the financial results, including research and development and general and administrative expenses, and discusses the company's liquidity, capital resources, contractual obligations, and critical accounting policies [Overview](index=15&type=section&id=Overview) - **Kura Oncology** is a clinical-stage biopharmaceutical company focused on precision medicines for cancer, with lead product candidates **ziftomenib** and **tipifarnib**, and preclinical candidate **KO-2806**[62](index=62&type=chunk) - **ziftomenib** (KOMET-001 trial) received orphan drug designation for **AML**, completed Phase 1b enrollment, and will present updated data at **ASH 2022**, with Phase 2 initiation anticipated in **H1 2023**[65](index=65&type=chunk)[71](index=71&type=chunk)[73](index=73&type=chunk) - **tipifarnib** received **Breakthrough Therapy Designation** for **HRAS mutant HNSCC**, but the AIM-HN trial was closed due to feasibility challenges; new trials include KURRENT-HN (with alpelisib) and KURRENT-LUNG (with osimertinib)[79](index=79&type=chunk)[80](index=80&type=chunk)[81](index=81&type=chunk)[84](index=84&type=chunk) - The company had **$427.8 million** in cash, cash equivalents, and short-term investments as of **September 30, 2022**, and recently secured a **$125.0 million** loan facility and a **$25.0 million** stock purchase agreement with **BMS**[86](index=86&type=chunk)[87](index=87&type=chunk)[88](index=88&type=chunk) [Financial Operations Overview](index=19&type=section&id=Financial%20Operations%20Overview) - Research and development expenses include personnel costs, clinical trial costs, manufacturing, and fees to external service providers, all expensed as incurred[90](index=90&type=chunk) - The timing, duration, and completion costs of preclinical studies and clinical trials are inherently unpredictable, depending on factors like trial complexity, patient enrollment, and regulatory requirements[91](index=91&type=chunk)[93](index=93&type=chunk) - General and administrative expenses primarily cover executive, finance, business development, and support personnel costs, patent portfolio maintenance, and professional services[92](index=92&type=chunk) [Results of Operations](index=20&type=section&id=Results%20of%20Operations) [Comparison of the Three Months Ended September 30, 2022 and 2021](index=20&type=section&id=Comparison%20of%20the%20Three%20Months%20Ended%20September%2030%2C%202022%20and%202021) For the three months ended **September 30, 2022**, total operating expenses increased by **$2.9 million** to **$36.6 million**, primarily due to higher personnel costs and increased research activities for discovery-stage programs, partially offset by decreased **tipifarnib**-related clinical costs. Net loss increased to **$35.5 million** from **$33.4 million** in the prior year | (In thousands) | Three Months Ended Sep 30, 2022 | Three Months Ended Sep 30, 2021 | Change | | :------------------------------------ | :------------------------------ | :------------------------------ | :----- | | Research and development expenses | $24,973 | $22,367 | +$2,606 | | General and administrative expenses | $11,621 | $11,310 | +$311 | | Other income (expense), net | $1,090 | $311 | +$779 | | Net Loss | $(35,504) | $(33,366) | $(2,138) | | Net loss per share, basic and diluted | $(0.53) | $(0.50) | $(0.03) | - The increase in R&D expenses was driven by higher personnel costs and increased research activities for **KO-2806**, partially offset by decreased clinical costs for **tipifarnib**[96](index=96&type=chunk) - Other income (expense), net increased primarily due to higher interest income[98](index=98&type=chunk) [Comparison of the Nine Months Ended September 30, 2022 and 2021](index=21&type=section&id=Comparison%20of%20the%20Nine%20Months%20Ended%20September%2030%2C%202022%20and%202021) For the nine months ended **September 30, 2022**, total operating expenses increased by **$6.5 million** to **$104.7 million**, primarily due to increased **ziftomenib**-related clinical trial costs, higher personnel expenses, and expanded discovery-stage programs, partially offset by reduced **tipifarnib**-related costs. Net loss increased to **$102.7 million** from **$97.7 million** in the prior year | (In thousands) | Nine Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2021 | Change | | :------------------------------------ | :----------------------------- | :----------------------------- | :----- | | Research and development expenses | $70,144 | $63,765 | +$6,379 | | General and administrative expenses | $34,565 | $34,455 | +$110 | | Other income (expense), net | $1,983 | $497 | +$1,486 | | Net Loss | $(102,726) | $(97,723) | $(5,003) | | Net loss per share, basic and diluted | $(1.54) | $(1.47) | $(0.07) | - **ziftomenib**-related R&D expenses increased by **$3.0 million** due to Phase 1/2 clinical trial costs, while **tipifarnib**-related R&D decreased by **$9.7 million** due to reduced clinical and manufacturing activities[100](index=100&type=chunk) - Other income (expense), net increased by **$1.5 million**, driven by higher interest income and a decrease in interest expense following a term loan payoff in **May 2021**[101](index=101&type=chunk) [Liquidity and Capital Resources](index=21&type=section&id=Liquidity%20and%20Capital%20Resources) - As of **September 30, 2022**, the company had **$427.8 million** in cash, cash equivalents, and short-term investments, and an accumulated deficit of **$535.7 million**[86](index=86&type=chunk)[108](index=108&type=chunk) - The company expects to fund operations into **2026**, assuming the full draw of its **$125.0 million** term loan facility with **Hercules Capital**, entered into on **November 2, 2022**[103](index=103&type=chunk)[109](index=109&type=chunk) | (In thousands) | Nine Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2021 | Change | | :------------------------------------ | :----------------------------- | :----------------------------- | :----- | | Net cash used in operating activities | $(83,532) | $(81,185) | $(2,347) | | Net cash provided by (used in) investing activities | $79,945 | $(200,896) | +$280,841 | | Net cash provided by (used in) financing activities | $3,852 | $(4,229) | +$8,081 | [Contractual Obligations and Commitments](index=23&type=section&id=Contractual%20Obligations%20and%20Commitments) - The company has non-cancelable operating leases for office and laboratory space, with total lease payments of **$5.8 million** and total operating lease liabilities of **$5.4 million** as of **September 30, 2022**[50](index=50&type=chunk)[115](index=115&type=chunk) - The company may be required to pay up to approximately **$80.0 million** in milestone payments, plus sales royalties, upon achieving regulatory and commercial milestones under its in-license agreements[118](index=118&type=chunk) [Critical Accounting Policies and Management Estimates](index=24&type=section&id=Critical%20Accounting%20Policies%20and%20Management%20Estimates) - There have been no material changes to the company's critical accounting policies and estimates since the Annual Report on **Form 10-K** for the fiscal year ended **December 31, 2021**[120](index=120&type=chunk) [ITEM 3. Quantitative and Qualitative Disclosures about Market Risk](index=24&type=section&id=ITEM%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) The company is exposed to interest rate risk primarily through its investment portfolio of cash, cash equivalents, and short-term investments, and its variable-rate term loans. A **10.0%** change in interest rates is not expected to materially affect the fair value of the investment portfolio. Inflation has not had a material effect on the business - The company's investment portfolio, consisting of money market funds, U.S. Treasury securities, corporate debt, commercial paper, and other debt securities, is subject to interest rate risk[121](index=121&type=chunk) - A **10.0%** change in interest rates on **September 30, 2022**, would not have had a material effect on the fair value of the investment portfolio[121](index=121&type=chunk) - The Term Loans bear interest at a variable rate (greater of prime rate minus **6.25%** plus **8.65%** or **8.65%**), exposing the company to interest expense fluctuations[122](index=122&type=chunk) - Inflation has not had a material effect on the company's business, financial condition, or results of operations during any presented periods[123](index=123&type=chunk) [ITEM 4. Controls and Procedures](index=25&type=section&id=ITEM%204.%20Controls%20and%20Procedures) The company's disclosure controls and procedures were evaluated and deemed effective at a reasonable assurance level as of **September 30, 2022**. During the quarter, a new **ERP system** was implemented, leading to updates in internal control over financial reporting processes, but no other material changes were identified - Disclosure controls and procedures were evaluated and concluded to be effective at the reasonable assurance level as of **September 30, 2022**[126](index=126&type=chunk) - A new **ERP system** was implemented during the quarter ended **September 30, 2022**, resulting in updated internal control over financial reporting processes[127](index=127&type=chunk) - No other changes in internal control over financial reporting materially affected, or are reasonably likely to materially affect, internal control over financial reporting during the quarter[128](index=128&type=chunk) PART II. OTHER INFORMATION [ITEM 1. Legal Proceedings](index=26&type=section&id=ITEM%201.%20Legal%20Proceedings) The company is not currently a party to any legal proceedings that, in management's opinion, would have a material adverse effect on its results of operations or financial position - The company is not involved in any legal proceedings that would individually or in aggregate have a material adverse effect on its financial condition or results of operations[131](index=131&type=chunk) [ITEM 1A. Risk Factors](index=26&type=page&id=ITEM%201A.%20Risk%20Factors) This section details the significant risks and uncertainties facing the company, encompassing challenges in product development, financial stability, reliance on third parties, regulatory compliance, intellectual property protection, commercialization, human resources, and macroeconomic conditions, all of which could materially impact its business, financial condition, and stock price [Risk Factor Summary](index=26&type=section&id=Risk%20Factor%20Summary) - The company's ability to conduct clinical trials has been and could continue to be adversely impacted by **COVID-19**[132](index=132&type=chunk) - High dependence on the success of lead product candidates, **ziftomenib** and **tipifarnib**, which are still in clinical development and require regulatory approval for commercialization[132](index=132&type=chunk) - Expectation of incurring losses for several years and the need for substantial additional capital, which may cause dilution to stockholders or restrict operations[132](index=132&type=chunk) - Reliance on third-party contractors for clinical trials and manufacturing increases risks of insufficient quantities, unacceptable cost, or quality issues[132](index=132&type=chunk) - Challenges in obtaining and maintaining intellectual property protection, as well as potential market acceptance issues and substantial competition for commercialized products[132](index=132&type=chunk) [Risks Related to the Discovery and Development of Our Product Candidates](index=28&type=section&id=Risks%20Related%20to%20the%20Discovery%20and%20Development%20of%20Our%20Product%20Candidates) - **COVID-19** has adversely impacted and could continue to impact the company's ability to conduct clinical trials, potentially causing delays in site startup, patient enrollment, and protocol compliance[135](index=135&type=chunk) - The company is highly dependent on the successful development and regulatory approval of **ziftomenib** and **tipifarnib**, with no guarantee of approval or timely commercialization[136](index=136&type=chunk)[139](index=139&type=chunk) - Clinical drug development is lengthy, expensive, and uncertain; preclinical and early clinical trial results may not predict later-stage outcomes, and preliminary data do not guarantee final results[155](index=155&type=chunk) - Product candidates may cause serious adverse events or unacceptable side effects, potentially delaying, limiting, or preventing their development or leading to trial suspension/termination[169](index=169&type=chunk)[170](index=170&type=chunk) - Failure to develop, validate, and obtain regulatory approval for companion diagnostic testing platforms could harm the drug development strategy and operational results[175](index=175&type=chunk)[177](index=177&type=chunk) [Risks Related to Our Financial Position and Need for Additional Capital](index=37&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) - The company expects to incur significant expenses and increasing operating losses for the foreseeable future and may never achieve or maintain profitability[181](index=181&type=chunk)[182](index=182&type=chunk) - Substantial additional capital will be needed for continuing operations, which may be raised through equity offerings, debt financings, or collaborations, potentially leading to stockholder dilution or restrictive covenants[191](index=191&type=chunk) - The Loan Agreement with **Hercules Capital** includes affirmative and restrictive covenants, and a default could accelerate repayment obligations or allow lenders to take control of pledged assets[193](index=193&type=chunk) - The company's limited operating history makes it difficult to evaluate future success or viability, as it has not yet demonstrated the ability to successfully complete clinical trials, obtain marketing approvals, or commercialize products[188](index=188&type=chunk) [Risks Related to Our Dependence on Third Parties](index=39&type=section&id=Risks%20Related%20to%20Our%20Dependence%20on%20Third%20Parties) - The company relies heavily on third-party contractors (CROs, data managers, medical institutions) to conduct preclinical and clinical trials, reducing control and posing risks of delays or non-compliance[195](index=195&type=chunk)[198](index=198&type=chunk)[199](index=199&type=chunk) - Dependence on third parties for manufacturing product candidates for preclinical, clinical, and commercial use increases the risk of insufficient quantities, unacceptable cost, or quality issues[204](index=204&type=chunk) - Manufacturing pharmaceutical products is complex, with risks including production difficulties, quality control issues, and compliance with cGMP regulations, which could lead to delays or regulatory sanctions[205](index=205&type=chunk)[208](index=208&type=chunk) - Geopolitical events and the **COVID-19** pandemic could negatively impact third-party manufacturers and supply chains, potentially causing interruptions in drug supply for clinical trials[210](index=210&type=chunk) [Risks Related to Regulatory Approval of Our Product Candidates and Other Legal Compliance Matters](index=43&type=section&id=Risks%20Related%20to%20Regulatory%20Approval%20of%20Our%20Product%20Candidates%20and%20Other%20Legal%20Compliance%20Matters) - Obtaining marketing approval from regulatory authorities (**FDA**, **EMA**) is expensive, lengthy, and uncertain, with no guarantee of success, potentially impairing revenue generation[212](index=212&type=chunk)[213](index=213&type=chunk) - The company may not benefit from available regulatory exclusivity periods for **tipifarnib** if another company obtains approval first, potentially reducing or eliminating its exclusivity and commercial prospects[214](index=214&type=chunk)[215](index=215&type=chunk) - **Orphan drug exclusivity** for **ziftomenib** (**AML**) may be lost if approved for a broader indication or if a competitor's different drug for the same condition is approved[218](index=218&type=chunk) - **Fast Track** and **Breakthrough Therapy Designations** do not guarantee faster development, review, or approval, nor do they increase the likelihood of marketing approval[220](index=220&type=chunk)[223](index=223&type=chunk) - Relationships with healthcare professionals and business operations are subject to fraud and abuse laws (**Anti-Kickback**, **False Claims**, **HIPAA**, **Sunshine Act**), privacy laws (**CCPA**, **GDPR**), and other healthcare regulations, with potential for significant penalties for non-compliance[230](index=230&type=chunk)[232](index=232&type=chunk)[234](index=234&type=chunk)[235](index=235&type=chunk)[236](index=236&type=chunk)[240](index=240&type=chunk) - Recently enacted and future healthcare legislation (**ACA**, **IRA**) and governmental scrutiny over drug pricing could increase costs, restrict sales, and adversely affect revenues and profitability[241](index=241&type=chunk)[243](index=243&type=chunk)[244](index=244&type=chunk)[245](index=245&type=chunk)[247](index=247&type=chunk)[248](index=248&type=chunk) [Risks Related to Our Intellectual Property](index=52&type=section&id=Risks%20Related%20to%20Our%20Intellectual%20Property) - Inability to obtain and maintain broad intellectual property protection for product candidates could allow competitors to commercialize similar products, impairing the company's ability to commercialize its own[255](index=255&type=chunk) - Patent rights for **tipifarnib** are limited, as composition of matter patents expired in **2016**, and method of treatment patents may not prevent competitors from marketing **tipifarnib** for other indications[259](index=259&type=chunk)[260](index=260&type=chunk) - The company depends on licensors (**University of Michigan**, **Janssen**) to prosecute and maintain material patents, and any failure by them to effectively protect these rights could adversely impact the business[264](index=264&type=chunk) - Breaching license agreements could lead to the loss of critical commercialization rights for product candidates, materially harming the business[266](index=266&type=chunk) - Changes in U.S. or foreign patent laws (e.g., **Leahy-Smith Act**, **Unitary Patent Court**) or their interpretation could diminish patent value, increase prosecution costs, and reduce protection[269](index=269&type=chunk)[270](index=270&type=chunk)[275](index=275&type=chunk)[276](index=276&type=chunk) - Failure to protect trade secrets through non-disclosure and confidentiality agreements could result in misappropriation or independent development by competitors, harming the company's competitive position[285](index=285&type=chunk) [Risks Related to the Commercialization of Our Product Candidates](index=59&type=section&id=Risks%20Related%20to%20the%20Commercialization%20of%20Our%20Product%20Candidates) - Even if approved, product candidates may fail to achieve sufficient market acceptance by physicians, patients, and third-party payors, impacting revenue generation and profitability[289](index=289&type=chunk) - The company currently lacks internal sales and market access personnel and may struggle to establish effective capabilities or secure third-party agreements, delaying or limiting commercialization[292](index=292&type=chunk) - The drug development market is highly competitive, with major pharmaceutical and biotechnology companies developing competing products that may be safer, more effective, or less expensive[293](index=293&type=chunk)[295](index=295&type=chunk)[296](index=296&type=chunk) - Uncertainty regarding insurance coverage and adequate reimbursement by governmental and private payors could limit market access and decrease revenue for new products[297](index=297&type=chunk)[298](index=298&type=chunk) - Product liability lawsuits related to clinical trials or commercial sales could result in substantial liabilities, reputational harm, and limitations on commercialization[304](index=304&type=chunk) [Risks Related to Employee Matters, Managing Growth and Macroeconomic Conditions](index=62&type=section&id=Risks%20Related%20to%20Employee%20Matters%2C%20Managing%20Growth%20and%20Macroeconomic%20Conditions) - **COVID-19** precautionary measures, such as remote work, could disrupt operations, limit interaction with third parties, and negatively affect employee recruitment and engagement[306](index=306&type=chunk) - The company is highly dependent on its Chief Executive Officer and other key management/scientific personnel, and the inability to retain or attract qualified individuals could harm business strategy[307](index=307&type=chunk)[308](index=308&type=chunk) - Anticipated significant growth in employees and operations (development, regulatory, commercial) may be difficult to manage effectively due to limited financial resources and management experience[309](index=309&type=chunk) - Failure to meet evolving environmental, social, and governance (**ESG**) expectations could negatively impact reputation, employee attraction/retention, and investment desirability[311](index=311&type=chunk)[313](index=313&type=chunk) - Unfavorable global economic conditions, including volatility from **COVID-19**, interest rates, and inflation, could adversely affect the ability to raise capital and strain suppliers[314](index=314&type=chunk) - Compromised information technology systems or data, including those of third parties, could lead to security incidents, regulatory actions, litigation, and business disruptions[315](index=315&type=chunk)[316](index=316&type=chunk)[318](index=318&type=chunk)[319](index=319&type=chunk) [Risks Related to Ownership of our Common Stock](index=66&type=section&id=Risks%20Related%20to%20Ownership%20of%20our%20Common%20Stock) - The company's stock price may fluctuate significantly due to various factors, including clinical trial results, regulatory decisions, competition, and general market conditions, making it difficult for stockholders to sell shares[324](index=324&type=chunk)[325](index=325&type=chunk)[326](index=326&type=chunk) - Future sales and issuances of common stock or rights to purchase common stock, including under equity incentive plans or the **ATM Facility**, could result in dilution to existing stockholders and cause the stock price to fall[332](index=332&type=chunk)[333](index=333&type=chunk)[337](index=337&type=chunk) - Anti-takeover provisions in charter documents and Delaware law could delay or prevent a change of control, limiting the market price of common stock and potentially frustrating stockholder attempts to replace management[338](index=338&type=chunk)[340](index=340&type=chunk) - Changes in tax laws or regulations, such as the Tax Cut and Jobs Act or the **IRA**, could adversely affect the company's business, cash flow, and financial condition, and limit the use of net operating loss carryforwards[344](index=344&type=chunk)[346](index=346&type=chunk) - The company does not intend to pay cash dividends on its capital stock in the foreseeable future, with any future payments dependent on financial condition and board discretion[347](index=347&type=chunk) [General Risk Factors](index=71&type=section&id=General%20Risk%20Factors) - If securities or industry analysts publish inaccurate or unfavorable research, the company's stock price and trading volume could decline[348](index=348&type=chunk) - Actions by activist stockholders could divert management's attention, incur significant costs, and negatively impact the company's reputation and stock value[349](index=349&type=chunk) - Securities class action litigation, common in the biotechnology industry, could divert management's attention, harm the business, and lead to significant liabilities[350](index=350&type=chunk) - Misconduct by employees, contractors, or other third parties, including noncompliance with regulatory standards or fraud and abuse laws, could result in significant penalties and reputational harm[351](index=351&type=chunk) - Non-compliance with U.S. and foreign export/import controls, sanctions, anti-corruption, and anti-money laundering laws could lead to criminal liability, fines, and harm to the business[352](index=352&type=chunk) [ITEM 6. Exhibits](index=73&type=section&id=ITEM%206.%20Exhibits) This section lists all exhibits filed as part of the **Form 10-Q**, including organizational documents, warrant agreements, securities purchase agreements, loan and security agreements, certifications, and XBRL-related documents - Key exhibits include the Amended and Restated Certificate of Incorporation and Bylaws, Warrant Agreements (including one issued to **Hercules Capital, Inc.** on **November 2, 2022**), Securities Purchase Agreement with **Bristol-Myers Squibb Company**, and the Loan and Security Agreement with **Hercules Capital, Inc.**[355](index=355&type=chunk) - Certifications of Principal Executive and Financial Officer pursuant to **Sections 302** and **906** of the **Sarbanes-Oxley Act** of **2002** are included[355](index=355&type=chunk) - Inline XBRL documents (Instance, Schema, Calculation, Definition, Label, Presentation Linkbase Documents) are provided for interactive data filing[355](index=355&type=chunk) [Signatures](index=74&type=section&id=Signatures) The report is officially signed on behalf of **Kura Oncology, Inc.** by its President and Chief Executive Officer, **Troy E. Wilson, Ph.D., J.D.**, who also serves as the **Principal Executive and Financial Officer** - The report was signed on **November 3, 2022**, by **Troy E. Wilson, Ph.D., J.D.**, President and Chief Executive Officer, acting as the **Principal Executive and Financial Officer**[360](index=360&type=chunk)

Kura Oncology, Inc. (NASDAQ:KURA) Q2 2022 Earnings Conference Call August 3, 2022 4:30 PM ET Company Participants Pete De Spain - Senior Vice President of Investor Relations Troy Wilson - President and Chief Executive Officer Tom Doyle - Senior Vice President of Finance and Accounting Conference Call Participants Jonathan Chang - SVB Securities Tiago Fauth - Credit Suisse Peter Lawson - Barclays Roger Song - Jefferies Li Watsek - Cantor Fitzgerald Reni Benjamin - JMP Securities Eva Privitera - Cowen, Inc. O ...

Clinical Development - Kura Oncology has two clinical-stage product candidates: ziftomenib and tipifarnib, with a preclinical-stage candidate KO-2806 currently in IND-enabling studies[57]. - Ziftomenib targets approximately 35% of acute myeloid leukemia (AML), including NPM1-mutant and KMT2A-rearranged AML, with KMT2A rearrangements found in 70-80% of infant leukemias[58]. - The KOMET-001 trial for ziftomenib reported clinical activity in 6 out of 8 efficacy-evaluable patients, including 2 complete remissions[61]. - Tipifarnib has been studied in over 5,000 cancer patients, showing durable anti-cancer activity with a manageable side effect profile[67]. - The AIM-HN trial for tipifarnib in HRAS mutant head and neck squamous cell carcinoma (HNSCC) has been amended to include patients with any HRAS mutation, potentially increasing the number of evaluable patients[70]. - Tipifarnib received Breakthrough Therapy Designation from the FDA for recurrent or metastatic HRAS mutant HNSCC with variant allele frequency ≥ 20%[71]. - Kura Oncology is collaborating with Novartis to evaluate the combination of tipifarnib and alpelisib in patients with HRAS overexpression and/or PIK3CA mutations[72]. - Preclinical data suggest that tipifarnib may prevent resistance to EGFR-targeted therapies in non-small cell lung cancer (NSCLC)[74]. - Kura Oncology is developing a next-generation farnesyl transferase inhibitor, KO-2806, which has improved potency and pharmacokinetic properties compared to tipifarnib[75]. - The company plans to initiate the KURRENT-LUNG trial of tipifarnib in combination with osimertinib in treatment-naïve locally advanced/metastatic EGFR mutated NSCLC in Q3 2022[74]. Financial Performance - As of June 30, 2022, the company had cash, cash equivalents, and short-term investments totaling $450.3 million[77]. - The company reported research and development expenses of $24.3 million for the three months ended June 30, 2022, an increase of 15.1% compared to $21.1 million for the same period in 2021[86]. - For the six months ended June 30, 2022, total research and development expenses were $45.2 million, up from $41.4 million in the same period in 2021, reflecting a 9.1% increase[90]. - The company has an accumulated deficit of $500.2 million as of June 30, 2022, indicating ongoing operating losses since inception[95]. - The company has not generated any revenues from product sales and does not have any approved products as of June 30, 2022[96]. - The company anticipates requiring significant additional financing in the future to continue operations and fund research and development activities[95]. - The company expects general and administrative expenses to increase in future periods to support planned research and development activities[88]. - The company has entered into an ATM Facility allowing for the sale of up to $150.0 million in common stock, but has not yet sold any shares under this agreement[94]. - The increase in ziftomenib-related research and development expenses for the three months ended June 30, 2022, was primarily due to costs associated with the Phase 1/2 clinical trial[86]. - The company expects its existing cash and investments to be sufficient to fund operating expenses through 2024, but future capital requirements will depend on various factors[96]. Cash Flow and Investments - Net cash used in operating activities for the six months ended June 30, 2022 was $62,522,000, an increase of $5,931,000 compared to $56,591,000 in 2021[98]. - Net cash provided by investing activities for the six months ended June 30, 2022 was $8,135,000, a significant change of $231,576,000 from a net cash used of $223,441,000 in 2021[98]. - Net cash provided by financing activities for the six months ended June 30, 2022 was $2,972,000, compared to a net cash used of $6,487,000 in 2021, reflecting a change of $9,459,000[100]. - The company may be required to pay up to approximately $80,000,000 in milestone payments plus sales royalties if regulatory and commercial milestones under in-license agreements are achieved[103]. - The increase in net cash used in operating activities was primarily due to changes of $3,000,000 in prepaid expenses and other current assets[98]. - The company does not believe that a 10.0% change in interest rates would have a material effect on the fair value of its investment portfolio as of June 30, 2022[107]. - Inflation has not had a material effect on the company's business, financial condition, or results of operations during the periods presented[108]. - The company has established guidelines regarding approved investments and maturities of investments to maintain safety and liquidity[107]. - The company has short-term and cancellable agreements with clinical sites and CROs for clinical research studies, generally outstanding for periods less than one year[102]. - There have been no material changes to the company's critical accounting policies and estimates from the previous fiscal year[105].

......... DEVELOPING PRECISION MEDICINES FOR THE TREATMENT OF CANCER Corporate Presentation – May 2022 Forward-Looking Statements This presentation contains forward-looking statements. Such statements include, but are not limited to, statements regarding our research, preclinical and clinical development activities, plans and projected timelines for ziftomenib, tipifarnib and KO-2806, plans regarding regulatory filings, our expectations regarding the relative benefits of our product candidates versus compet ...

Financial Data and Key Metrics Changes - Research and development expenses for Q1 2022 were $20.9 million, an increase from $20.3 million in Q1 2021, primarily due to higher clinical trial and personnel costs [14] - General and administrative expenses rose to $11.9 million in Q1 2022 from $10.6 million in Q1 2021, mainly due to increased professional fees and non-cash share-based compensation [14] - The net loss for Q1 2022 was $32.5 million, compared to a net loss of $30.7 million in Q1 2021, which included non-cash share-based compensation of $6.7 million versus $5.1 million in the prior year [15] - As of March 31, 2022, cash, cash equivalents, and short-term investments totaled $480.1 million, down from $518 million as of December 31, 2021, with a cash runway expected to fund operations through 2024 [15] Business Line Data and Key Metrics Changes - The Menin Inhibitor program, Ziftomenib, is progressing with the completion of enrollment in the Phase 1b study, with top-line data expected in Q3 2022 and a full data presentation planned for Q4 2022 [4][6] - The Farnesyl Transferase Inhibitor (FTI) programs are being developed, with ongoing trials for Tipifarnib in combination with Alpelisib targeting head and neck squamous cell carcinoma (HNSCC) [8][10] Market Data and Key Metrics Changes - The company is expanding its clinical development strategy in the U.S. and Europe, anticipating a larger patient population for Ziftomenib through combination therapies [8] - The combination of Tipifarnib and Alpelisib is expected to increase the total addressable patient population for Tipifarnib to as much as 50% of HNSCC patients [10] Company Strategy and Development Direction - The company aims to register Ziftomenib as a monotherapy while exploring combination opportunities to enhance treatment efficacy [8] - A comprehensive development strategy is being pursued for the FTI programs, focusing on both monotherapy and combination therapies to address larger patient populations [11][47] Management's Comments on Operating Environment and Future Outlook - Management remains optimistic about the company's position despite a challenging market environment, highlighting a strong team and a well-designed clinical strategy [4] - The anticipated milestones for 2022 include identifying the recommended Phase 2 dose for Ziftomenib and initiating several key trials [16] Other Important Information - The company has implemented an enhanced mitigation strategy for managing differentiation syndrome associated with Ziftomenib, which is seen as a marker of clinical activity [23][60] - The company is preparing to submit an IND application for its next-generation FTI candidate, KO-2806, in Q4 2022 [13] Q&A Session Summary Question: What can investors expect in the top-line third quarter data versus the medical meeting presentation in the fourth quarter? - The top-line data will focus on safety, tolerability, and clinical activity at the recommended Phase 2 dose, while the medical meeting will provide a more comprehensive dataset including genetic subtype breakdowns [18] Question: Can you provide details on the enrollment completion and patient demographics in the Phase 1b expansion cohorts? - There is a good balance between the two populations (NPM1 mutation and KMT2A rearrangement), with no disproportionate enrollment observed [19] Question: How much safety data will be available in the Q3 update, and what is the management strategy for differentiation syndrome? - The safety data will be high-level, with an encouraging safety profile reported, and an enhanced mitigation strategy in place for differentiation syndrome [23] Question: What are the top combination studies being considered for Ziftomenib? - Venetoclax and FLT3 inhibitors are identified as attractive combination partners, with ongoing activities to support these studies [46] Question: What are the primary endpoints for the current lung trial? - The primary endpoint is progression-free survival (PFS), with safety and tolerability also being key metrics [55]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2022 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ________ to ________ Commission File Number: 001-37620 KURA ONCOLOGY, INC. (Exact name of registrant as specified in its charter) Delaware 61-1547851 12730 Hi ...

DEVELOPING PRECISION MEDICINES FOR THE TREATMENT OF CANCER ......... Corporate Presentation – February 2022 Forward-Looking Statements This presentation contains forward-looking statements. Such statements include, but are not limited to, statements regarding our research, preclinical and clinical development activities, plans and projected timelines for ziftomenib, tipifarnib and KO-2806, plans regarding regulatory filings, our expectations regarding the relative benefits of our product candidates versus c ...

Kura Oncology, Inc. (NASDAQ:KURA) Q4 2021 Earnings Conference Call February 24, 2022 4:30 PM ET Company Participants Troy Wilson – Chairman, Chief Executive Officer & President Tom Doyle – Senior Vice President of Finance and Accounting Pete De Spain – Vice President, Investor Relations & Corporate Communications Conference Call Participants Jonathan Chang – SVB Leerink Peter Lawson – Barclays Bank Tiago Fauth – Crédit Suisse Ren Benjamin – JMP Securities Roger Song – Jefferies Phil Nadeau – Cowen and Compa ...

PART I [Business Overview](index=3&type=section&id=Item%201.%20Business) Kura Oncology is a clinical-stage biopharmaceutical company focused on developing precision medicines for cancer - Kura Oncology is a clinical-stage biopharmaceutical company developing precision medicines for cancer[16](index=16&type=chunk) - The pipeline includes clinical-stage ziftomenib and tipifarnib, preclinical KO-2806, and discovery-stage programs[16](index=16&type=chunk) - The company owns global commercial rights to all programs and plans to advance them through internal development and strategic partnerships[16](index=16&type=chunk) [Forward-Looking Statements](index=3&type=section&id=Forward-Looking%20Statements) - The report contains forward-looking statements regarding future events, financial performance, and known/unknown risks, uncertainties, and other factors[11](index=11&type=chunk) - Key forward-looking statements include the impact of COVID-19, clinical trial progress, regulatory approvals, commercialization, intellectual property, financing, and personnel[11](index=11&type=chunk) - Readers are cautioned not to unduly rely on these statements, as actual results may differ materially, particularly due to factors discussed in the 'Risk Factors' section[12](index=12&type=chunk)[13](index=13&type=chunk) [Risk Factor Summary](index=4&type=section&id=Risk%20Factor%20Summary) - Investment in common stock is speculative due to material factors summarized, including COVID-19 impact on clinical trials[14](index=14&type=chunk)[15](index=15&type=chunk) - High dependence on lead product candidates (ziftomenib and tipifarnib) which are still in clinical development and may not receive regulatory approval[15](index=15&type=chunk) - Risks include lengthy and expensive clinical development, potential for serious adverse events, need for substantial additional capital, and reliance on third-party contractors[15](index=15&type=chunk)[17](index=17&type=chunk) [Company Overview](index=5&type=section&id=Overview) - Kura Oncology is a clinical-stage biopharmaceutical company focused on precision medicines for cancer, with small molecule product candidates targeting cancer signaling pathways[16](index=16&type=chunk) - The company has two clinical-stage candidates (ziftomenib, tipifarnib), one preclinical (KO-2806), and additional discovery-stage programs, holding global commercial rights[16](index=16&type=chunk) [The COVID-19 Pandemic](index=8&type=section&id=The%20COVID-19%20Pandemic) - COVID-19 has disrupted business operations, including slower patient screening and enrollment in clinical trials, and challenges at clinical sites[35](index=35&type=chunk) - The company implemented precautionary measures like remote working and a contingency plan to minimize impact, but expects continued disruption[35](index=35&type=chunk)[37](index=37&type=chunk) [Precision Medicines in Cancer Treatment](index=9&type=section&id=Precision%20Medicines%20in%20Cancer%20Treatment) - Precision medicine aims to improve patient outcomes and reduce healthcare costs by matching targeted therapeutics to patients with specific cancers based on molecular diagnostics[38](index=38&type=chunk) - This approach offers potential for higher translatability from preclinical to clinical studies, increased response rates, and expedited clinical development[40](index=40&type=chunk) [Our Approach to Development of Precision Medicines in Oncology](index=9&type=section&id=Our%20Approach%20to%20Development%20of%20Precision%20Medicines%20in%20Oncology) - Translational research, synthesizing basic, preclinical, and clinical data, guides the company's precision medicine approach, using in vitro and in vivo experiments, including patient-derived xenograft (PDX) models[41](index=41&type=chunk) - The strategy involves using existing diagnostic tools (NGS, RNA expression profiling) to identify patient subsets and developing companion diagnostics for potential registration and marketing[42](index=42&type=chunk) - A disciplined clinical development approach evaluates product candidates in well-defined patient populations to identify early response signals and reduce development risks[43](index=43&type=chunk)[44](index=44&type=chunk)[45](index=45&type=chunk) [Clinical Programs and Pipeline](index=10&type=section&id=Clinical%20Programs%20and%20Pipeline) Kura Oncology Pipeline Status | Program | Preclinical | Phase 1 | Phase 2 | Registration Directed | | :--- | :--- | :--- | :--- | :--- | | Ziftomenib (KO-539) Menin Inhibitor | Acute Myeloid Leukemia (AML) KOMET-001 Trial | Enrollment in Phase 1b expansion cohorts ongoing | | | | Tipifarnib Farnesyl Transferase Inhibitor (FTI) | | HRAS mutant Head and Neck Squamous Cell Carcinoma (HNSCC) AIM-HN Trial (Enrollment ongoing) | PIK3CA/HRAS Dependent HNSCC KURRENT Trial (Enrollment ongoing) | | | KO-2806 Next-Generation FTI | Solid Tumors (IND enabling studies ongoing) | | | | [Ziftomenib (KO-539) – A Selective Inhibitor of the Menin-KMT2A Interaction](index=10&type=section&id=Ziftomenib%20%28KO-539%29%20%E2%80%93%20A%20Selective%20Inhibitor%20of%20the%20Menin-KMT2A%20Interaction) - Ziftomenib is an orally bioavailable small molecule inhibitor of the menin-KMT2A interaction, developed for genetically defined subsets of acute leukemias (AML, ALL)[46](index=46&type=chunk) - Preclinical data supports anti-tumor activity in KMT2A-rearranged and NPM1-mutant acute leukemias, potentially addressing **~35% of AML cases**[18](index=18&type=chunk)[49](index=49&type=chunk) - The KOMET-001 Phase 1/2 trial for relapsed/refractory AML reported preliminary clinical activity in **6 of 8 evaluable patients** (including **2 complete remissions**) and a manageable safety profile as of Nov 2020[20](index=20&type=chunk)[51](index=51&type=chunk) - The KOMET-001 trial was placed on a partial clinical hold in Nov 2021 due to a Grade 5 adverse event, but the hold was lifted in Jan 2022 after agreement on a mitigation strategy for differentiation syndrome[25](index=25&type=chunk)[56](index=56&type=chunk) - Preclinical data from Dec 2021 showed synergistic activity of ziftomenib in combination with the BCL2 inhibitor venetoclax in AML cells[26](index=26&type=chunk)[57](index=57&type=chunk) [Tipifarnib – An Oral Farnesyl Transferase Inhibitor](index=11&type=section&id=Tipifarnib%20%E2%80%93%20An%20Oral%20Farnesyl%20Transferase%20Inhibitor) - Tipifarnib is a potent, selective, orally bioavailable farnesyl transferase inhibitor, previously studied in over **5,000 cancer patients** with a manageable side effect profile[27](index=27&type=chunk)[58](index=58&type=chunk) - The company is evaluating tipifarnib in HRAS mutant HNSCC (AIM-HN registration-directed trial) and in combination with alpelisib (PI3 kinase alpha inhibitor) in the KURRENT trial (collaboration with Novartis)[28](index=28&type=chunk)[32](index=32&type=chunk)[74](index=74&type=chunk)[78](index=78&type=chunk) - Tipifarnib received Breakthrough Therapy Designation from the FDA in Feb 2021 for recurrent or metastatic HRAS mutant HNSCC (variant allele frequency **≥ 20%**) after platinum-based chemotherapy[30](index=30&type=chunk)[73](index=73&type=chunk) - Clinical data from a Phase 2 study in relapsed/refractory PTCL showed an overall response rate of **56%** and median overall survival of **32.8 months** in advanced AITL patients[33](index=33&type=chunk)[84](index=84&type=chunk) - The company is collaborating with Illumina, Inc. to develop and commercialize a companion diagnostic test for HRAS mutations in HNSCC patients for use with tipifarnib[81](index=81&type=chunk) [Next-Generation Farnesyl Transferase Inhibitor](index=15&type=section&id=Next-Generation%20Farnesyl%20Transferase%20Inhibitor) - Kura Oncology is developing KO-2806, a next-generation farnesyl transferase inhibitor, which has advanced into IND-enabling studies[34](index=34&type=chunk)[86](index=86&type=chunk) - KO-2806 is intended for innovative biology and larger disease indications in combination with other targeted therapies, with an IND submission expected by the end of 2022[34](index=34&type=chunk)[86](index=86&type=chunk) [License Agreements](index=15&type=section&id=License%20Agreements) - The company holds exclusive worldwide rights for its menin-KMT2A program (including ziftomenib) from the University of Michigan, involving upfront fees, annual maintenance, and milestone/royalty payments[87](index=87&type=chunk) - Exclusive global rights for tipifarnib (excluding virology) were licensed from Janssen Pharmaceutica NV, with obligations for development, commercialization, regulatory/sales milestone payments (up to **$75 million aggregate**), and tiered royalties[89](index=89&type=chunk)[90](index=90&type=chunk) [Competition](index=16&type=section&id=Competition) - The cancer treatment market is intensely competitive, with Kura Oncology facing major pharmaceutical, specialty pharmaceutical, and biotechnology companies globally[92](index=92&type=chunk) - Competitors for menin-KMT2A inhibitors include Syndax, Biomea, Janssen, Sumitomo Dainippon, and Daiichi Sankyo, with other products in clinical development for KMT2A-r or NPM1 leukemias[97](index=97&type=chunk) - For farnesyl transferase inhibitors (

Financial Data and Key Metrics Changes - Research and development expenses for Q3 2021 were $22.4 million, up from $16.6 million in Q3 2020, primarily due to increased clinical trial costs and personnel expenses [30] - General and administrative expenses for Q3 2021 were $11.3 million, compared to $7.6 million in Q3 2020, driven by higher personnel costs and professional fees [31] - Net loss for Q3 2021 was $33.4 million or $0.50 per share, compared to a net loss of $23.8 million or $0.42 per share in Q3 2020 [31] - As of September 30, 2021, cash, cash equivalents, and short-term investments totaled $543.4 million, down from $633.3 million as of December 31, 2020 [32] Business Line Data and Key Metrics Changes - KO-539 showed encouraging single-agent activity in relapsed and/or refractory AML, with a favorable safety profile and no evidence of QTC prolongation [9][10] - The Phase 1b study of KO-539 is currently enrolling two expansion cohorts, with preliminary results indicating activity at both doses [12][13] - Tipifarnib has been awarded breakthrough therapy designation from the FDA for HRAS mutant HNSCC, based on data from the Phase 2 RUN-HN trial [20][21] Market Data and Key Metrics Changes - The overall response rate for tipifarnib in patients with angioimmunoblastic T-cell lymphoma was reported at 56.3%, with a median overall survival of 32.8 months [20] - The company is preparing for a Phase 1/2 study of tipifarnib in combination with alpelisib in HNSCC, with the first clinical site activated [24][25] Company Strategy and Development Direction - The company remains focused on three main programs: KO-539 in acute leukemia, tipifarnib in head and neck squamous cell carcinoma, and KO-2806 in solid tumors [8] - The development strategy for KO-539 aims to register it as a monotherapy while exploring combination therapies to expand treatment options [16][75] - The company is actively preparing for combination trials and is exploring partnerships to enhance development opportunities [63][75] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the progress of clinical trials and the potential of their drug candidates to create significant value for patients and shareholders [8][16] - The company anticipates sufficient cash reserves to fund operations into 2024, despite the increase in operating expenses [32][33] - Management remains optimistic about the clinical activity of KO-539 and the potential for registration in both NPM1 and KMT2A populations [46] Other Important Information - The company plans to provide updates on the Phase 1 study results at future medical meetings, pending the determination of the recommended Phase 2 dose [15] - The company has identified opportunities for farnesyl transferase inhibitors in combination with other targeted therapies in large solid tumor indications [27] Q&A Session Summary Question: Can you provide more details on the evidence of activity and safety profile? - Management indicated that they are encouraged by the clinical activity observed in both dose cohorts but could not provide more granular details at this stage due to the nature of the Phase 1b study [38][39] Question: What is the latest thinking on KO-539's activity in different genetic subtypes? - Management stated that they have not observed any significant differences in activity between NPM1 and KMT2A populations and expect the recommended Phase 2 dose to support efficacy in both [41][43] Question: How do you interpret competitor data in the context of the relapsed/refractory patient population? - Management noted that while competitor data showed lower response rates, it is early in the study, and they remain optimistic about their own data and the potential for longer duration of response [52][54] Question: What are the go/no-go criteria for moving forward with KO-539? - Management confirmed that they have established go/no-go criteria based on achieving a sufficient level of CR/CRH to support confidence in a properly powered trial [70] Question: How will the company approach combination trials once the recommended Phase 2 dose is established? - Management indicated a desire to maintain control over trial design and execution while exploring opportunities for investigator-sponsored trials (ISTs) [64][75] Question: Have global supply chain issues impacted the company's activities? - Management reported no significant impact from supply chain issues, attributing their resilience to the nature of their drug candidates being small molecules that can be administered on an outpatient basis [85] Question: How is duration of response measured in trials, particularly regarding transplant? - Management clarified that duration of response is tracked until the point of transplant, at which data are censored to avoid confounding results [91]