Acute myeloid leukaemia with 11q23 abnormality

– BLA filing for axatilimab in chronic GVHD granted Priority Review; PDUFA action date set for August 28, 2024 – – NDA for revumenib in R/R KMT2Ar acute leukemia submitted under FDA's RTOR program; PDUFA date assignment expected in 1Q24 – – Enrollment in AUGMENT-101 mNPM1 patient cohort expected to complete in late 1Q/early 2Q; topline data in 4Q24 – – Initiated revumenib Phase 1 combination trial with 7+3 chemotherapy in newly diagnosed acute leukemias – – Axatilimab Phase 2 IPF trial underway – – $600 million in cash and cash equivalents expected to provide runway through 2026 – – Company to host conference call today at 4:30 p.m. ET – WALTHAM, Mass., Feb. 27, 2024 /PRNewswire/ -- Syndax Pharmaceuticals (Nasdaq: SNDX), a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today reported its financial results for the fourth quarter and full year ended December 31, 2023, and provided a clinical and business update. "2023 was a landmark year for Syndax marked by execution across all of our major clinical, regulatory, and operational milestones," said Michael A. Metzger, Chief Executive Officer. "With the axatilimab BLA filing under FDA Priority Review and receipt of a PDUFA action date for revumenib anticipated later this quarter, preparations are well underway for the potential launch of both first- and best-in-class drugs later this year. We are committed to maximizing the full potential of these therapies beyond their initial approvals through expansion into earlier lines of therapy in combination with standard of care agents and into new indications. With a strong cash position that is expected to fund operations through 2026, Syndax is well positioned for continued success and long-term growth." Recent Pipeline Progress and Anticipated Milestones Revumenib On December 29, 2023, the Company submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for revumenib, a potent, selective small molecule menin inhibitor, for the treatment of adult and pediatric relapsed or refractory (R/R) KMT2A-rearranged (KMT2Ar) acute leukemia. The NDA submission is being reviewed under the FDA's Real-Time Oncology Review (RTOR) program, and the Company expects to receive a Prescription Drug User Fee Act (PDUFA) action date from the FDA this quarter. The Company expects to complete enrollment in the AUGMENT-101 pivotal trial cohort of patients with R/R mutant nucleophosmin (mNPM1) acute myeloid leukemia (AML) later this quarter or early in the second quarter of 2024. Topline data is expected in the fourth quarter of 2024 and could support a supplemental NDA (sNDA) filing for revumenib in R/R mNPM1 AML in the first half of 2025. Positive results from the Phase 1 and Phase 2 portions of the Company's AUGMENT-101 trial were featured throughout multiple sessions at the 65th American Society of Hematology (ASH) Annual Meeting in December 2023, including results from the pivotal portion of the trial, which were highlighted as a late-breaking oral presentation. At the ASH Annual Meeting and during the Company's accompanying investor event, investigators presented compelling data from multiple Phase 1 combination trials of revumenib in mNPM1 and KMT2Ar acute leukemia across the treatment landscape. The trials are expanding to validate recommended Phase 2 doses, with additional data expected in the second half of 2024. These trials include: BEAT AML: Evaluating the combination of revumenib with venetoclax and azacitidine in front-line AML patients. This trial is being conducted as part of the Leukemia & Lymphoma Society's Beat AML® Master Clinical Trial. SAVE: Evaluating the all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in R/R AML or mixed phenotype acute leukemias. The trial is being conducted by investigators from the MD Anderson Cancer Center. AUGMENT-102: Evaluating the combination of revumenib with fludarabine and cytarabine in patients with R/R acute leukemias. In February 2024, the Company initiated a Phase 1 trial of revumenib in combination with 7+3 chemotherapy followed by maintenance treatment in newly diagnosed patients with mNPM1 or KMT2Ar acute leukemias. The Company plans to initiate a pivotal trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar acute leukemia patients unable to receive intensive chemotherapy by year-end 2024. Enrollment is ongoing in a Phase 1 proof-of-concept clinical trial of revumenib in patients with unresectable metastatic microsatellite stable colorectal cancer. The Company expects to provide an update on the trial in the second quarter of 2024. Axatilimab The FDA has accepted the Biologics License Application (BLA) filing for axatilimab, an anti-CSF-1R antibody, in patients with chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy. The application has been granted Priority Review and assigned a PDUFA action date of August 28, 2024. Results from the pivotal AGAVE-201 trial were featured in the Plenary Scientific Session at the 65th ASH Annual Meeting in December 2023. The Company exercised its option under its collaboration agreement with Incyte to co-commercialize axatilimab in the U.S. and will provide 30% of the commercial effort. Enrollment is ongoing in a randomized, double-blinded, placebo-controlled Phase 2 trial of axatilimab in patients with idiopathic pulmonary fibrosis (IPF). Incyte plans to initiate two combination trials with axatilimab in chronic GVHD in mid-2024, including a Phase 2 combination trial with ruxolitinib and a Phase 3 combination trial with steroids. Corporate Update In December of 2023, Syndax closed an underwritten public offering of 12,432,431 shares of its common stock at $18.50 per share. This total included the exercise in full by the underwriters of their option to purchase up to 1,621,621 additional shares of common stock. Additionally, the Company issued and sold 2,719,744 shares pursuant to its ATM facility. Aggregate net proceeds from these offerings were approximately $258.1 million after deducting underwriting discounts and sales agent commissions and estimated offering expenses payable by Syndax. Fourth Quarter and Full Year 2023 Financial Results As of December 31, 2023, Syndax had cash, cash equivalents, and short and long-term investments of $600.5 million and 85.1 million common shares and prefunded warrants outstanding. Fourth quarter 2023 research and development expenses increased to $55.1 million from $31.8 million, and for the full year increased to $163.0 million compared to $118.5 million for 2022. The year-over-year increase in research and development expenses was primarily due to increased employee-related expenses and professional fees as well as increased clinical and manufacturing expenses. Fourth quarter 2023 selling, general and administrative expenses increased to $22.8 million from $10.2 million, and for the full year increased to $66.9 million compared to $33.3 million for 2022. The year-over-year increase in selling, general and administrative expenses was primarily due to increased employee-related expenses and professional fees as well as increased commercialization activities for revumenib and axatilimab. For the three months ended December 31, 2023, Syndax reported a net loss attributable to common stockholders of $72.5 million, or $1.00 per share, compared to a net loss attributable to common stockholders of $39.2 million, or $0.62 per share, for the comparable prior year period. For the year ended December 31, 2023, Syndax reported a net loss attributable to common stockholders of $209.4 million or $2.98 per share, compared to a net loss attributable to common stockholders of $149.3 million or $2.46 per share for the comparable prior year period. Financial Guidance For the first quarter of 2024, the Company expects research and development expenses to be $56 to $62 million and total operating expenses to be $82 to $88 million. For the full year of 2024, the Company expects research and development expenses to be $240 to $260 million and total operating expenses to be $355 to $375 million, which includes an estimated $43 million in non-cash stock compensation expense. The Company believes that it has sufficient cash runway to fund its research, clinical development and commercial operations through 2026. Conference Call and Webcast In connection with the earnings release, Syndax's management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Tuesday, February 27, 2024. The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company's website. Alternatively, the conference call may be accessed through the following: Conference ID: Syndax4Q23 Domestic Dial-in Number: 800-590-8290 International Dial-in Number: 240-690-8800 Live webcast: For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company's website at approximately 24 hours after the conference call and will be available for 90 days following the call. About Revumenib Revumenib is a potent, selective, small molecule inhibitor of the menin-KMT2A binding interaction that is being developed for the treatment of KMT2A-rearranged, also known as mixed lineage leukemia rearranged or MLLr, acute leukemias including ALL and AML, and NPM1-mutant AML. Positive topline results from the Phase 2 AUGMENT-101 trial in R/R KMT2Ar acute leukemia showing the trial met its primary endpoint were recently presented at the 65th American Society of Hematology Annual Meeting and data from the Phase 1 portion of AUGMENT-101 in acute leukemia was published in Nature. Revumenib was granted Orphan Drug Designation by the FDA and European Commission for the treatment of patients with AML, and Fast Track designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation. Revumenib was granted BTD by the FDA for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement. About Axatilimab Axatilimab is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages along with their monocyte precursors, which has been shown to play a key role in the fibrotic disease process underlying diseases such as chronic graft-versus-host disease (GVHD) and idiopathic pulmonary fibrosis (IPF). Positive topline results from the Phase 2 AGAVE-201 trial showing the trial met its primary endpoint were recently presented at the 65th American Society of Hematology Annual Meeting and Phase 1/2 data of axatilimab in chronic GVHD were published in the Journal of Clinical Oncology. Axatilimab was granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with chronic GVHD and IPF. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab. Syndax has exercised its option under the collaboration agreement to co-commercialize axatilimab in the U.S. and will provide 30% of the commercial effort. Axatilimab is being developed under an exclusive worldwide license from UCB entered into between Syndax and UCB in 2016. About RTOR RTOR provides a more efficient review process for oncology drugs to ensure that safe and effective treatments are available to patients as early as possible, while improving review quality and engaging in early iterative communication with the applicant. Specifically, it allows for close engagement between the sponsor and the FDA throughout the submission process and it enables the FDA to review individual sections of modules of a drug application rather than requiring the submission of complete modules or a complete application prior to initiating review. Additional information about RTOR can be found at: About Syndax Syndax Pharmaceuticals is a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies. Highlights of the Company's pipeline include revumenib, a highly selective inhibitor of the menin–KMT2A binding interaction, and axatilimab, a monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor. For more information, please visit or follow the Company on Twitter and LinkedIn. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipate," "believe," "could," "estimate," "expects," "intend," "may," "plan," "potential," "predict," "project," "should," "will," "would" or the negative or plural of those terms, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Syndax's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, clinical development and scope of clinical trials, the reporting of clinical data for Syndax's product candidates, the sufficiency of the Company's cash runway to fund its research, clinical development and commercial operations through 2026, the potential use of its product candidates to treat various cancer indications and fibrotic diseases, and Syndax's expected first quarter and full year research and development expenses, and expected first quarter and full year total operating expenses. Many factors may cause differences between current expectations and actual results, including: unexpected safety or efficacy data observed during preclinical or clinical trials; clinical trial site activation or enrollment rates that are lower than expected; changes in expected or existing competition; changes in the regulatory environment; failure of Syndax's collaborators to support or advance collaborations or product candidates; and unexpected litigation or other disputes. Other factors that may cause Syndax's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Syndax's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Syndax assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available. Syndax Contact Sharon Klahre Syndax Pharmaceuticals, Inc. [email protected] Tel 781.684.9827 SNDX-G SOURCE Syndax Pharmaceuticals, Inc.

– Positive preliminary combination data from KOMET-007 trial of ziftomenib in NPM1-m and KMT2A-r AML, including effective mitigation of differentiation syndrome – – First patient dosed in KOMET-008 trial of ziftomenib in combination with additional standards of care, including gilteritinib, FLAG-IDA and LDAC, in AML – – Completion of enrollment in registration-directed trial of ziftomenib in NPM1-m AML anticipated by mid-2024 – – $570 million in pro forma cash provides runway into 2027 – – Management to host webcast and conference call today at 4:30 p.m. ET – SAN DIEGO, Feb. 27, 2024 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, today reported fourth quarter and full year 2023 financial results and provided a corporate update. “Given its best-in-class safety and efficacy pro well as optimal pharmaceutical properties, we believe ziftomenib is well positioned to become a cornerstone of therapy for patients with acute leukemias,” said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. “This belief is supported by strong enthusiasm among both physicians and patients, as evidenced by rapid enrollment across our ongoing ziftomenib studies. We continue to be encouraged by the rate of enrollment in KOMET-001, our Phase 2 registration-directed trial of ziftomenib in patients with relapsed/refractory NPM1-mutant AML, and we remain on pace to complete enrollment of all 85 patients in the trial by the middle of this year. We also are seeing robust enrollment in our ongoing KOMET-007 study, which is investigating ziftomenib in combination with current standards of care in the frontline and relapsed/refractory settings. And with our recent financing, we remain in a strong financial position, which enables us to invest aggressively in research, development and pre-commercial activities to maximize the value of ziftomenib and support our other pipeline assets.” Recent Highlights Encouraging safety and tolerability pro ziftomenib in combination with 7+3 and ven/aza – In January 2024, Kura reported preliminary clinical data from the first 20 patients in KOMET-007, a Phase 1 dose-escalation trial of ziftomenib in combination with standards of care, including cytarabine/daunorubicin (7+3) and venetoclax/azacitidine (ven/aza), in patients with NPM1-mutant (NPM1-m) and KMT2A-rearranged (KMT2A-r) AML. The first 20 patients were enrolled between July 2023 and November 2023 and included five newly diagnosed patients with adverse risk NPM1-m or KMT2A-r AML and 15 patients with refractory/relapsed (R/R) NPM1-m or KMT2A-r AML. Continuous daily dosing of ziftomenib at 200 mg was well tolerated, and the safety pro consistent with features of underlying disease and backbone therapies. No differentiation syndrome events of any grade were reported, and no dose-limiting toxicities, evidence of QTc prolongation, drug-drug interactions or additive myelosuppression were observed. Encouraging evidence of clinical activity for ziftomenib combinations in NPM1-m and KMT2A-r AML – As of the data cutoff on January 11, 2024, all five newly diagnosed patients treated with ziftomenib and 7+3 achieved a complete remission with full count recovery, for a complete remission (CR) rate of 100%. The overall response rate (ORR) among the 15 R/R patients treated with ziftomenib and ven/aza was 53%, including a 40% ORR in the 10 patients who had received prior venetoclax. The CR/CRh (CR with partial hematologic recovery) rate among the nine R/R patients who were menin inhibitor naïve was 56%. As of the data cutoff, 16 of the first 20 patients remained on trial, including all 11 NPM1-mutant patients. To date, enrollment at the 400 mg dose of ziftomenib is ongoing in the R/R ven/aza cohorts and in the frontline NPM1-mutant 7+3 cohort. First patient dosed in KOMET-008 trial of ziftomenib in combination with additional standards of care in AML – Yesterday, Kura announced dosing of the first patient in its KOMET-008 trial of ziftomenib in combination with the FLT3 inhibitor gilteritinib, FLAG-IDA or LDAC for the treatment of relapsed/refractory NPM1-m or KMT2A-r AML. Roughly half of patients with relapsed or refractory hNPM1-mutant AML have co-occurring FLT3 mutations, and the prognosis for these patients is poor. Preclinical data for ziftomenib in combination with FLT3 inhibitors demonstrate strong synergistic effects compared to either single agent alone. Completion of enrollment in registration-directed trial of ziftomenib in NPM1-m AML anticipated by mid-2024 – The KOMET-001 registration-directed trial of ziftomenib in NPM1-m R/R AML is expected to enroll a total of 85 patients in the U.S. and Europe. In the Phase 1 trial, ziftomenib demonstrated a 35% CR rate and 45% overall response rate in 20 patients with NPM1-mutant AML treated at the recommended Phase 2 dose (RP2D). NPM1-mutant AML accounts for approximately 30% of new AML cases annually and represents a disease of significant unmet need for which no approved targeted therapy exists. Addressing the continuum of care for patients with acute leukemias– Kura remains committed to developing new treatment options across the continuum of care for patients with acute leukemias, where poor outcomes and significant unmet medical need remain. In December 2023, the Company announced that ziftomenib was selected by the Leukemia & Lymphoma Society for the Pediatric Acute Leukemia (PedAL) Master Clinical Trial. As part of the study, ziftomenib will be evaluated in combination with chemotherapy in pediatric patients with R/R KMT2A-r, NPM1-m or NUP98-rearranged acute leukemia. In addition, Kura recently began dosing with ziftomenib in patients with R/R KMT2A-r acute lymphoblastic leukemia, a relatively small population with a large unmet medical need. Positive results from registration-directed trial of tipifarnib in HRAS-mutant HNSCC – In October 2023, Kura presented positive results from its AIM-HN registration-directed trial of tipifarnib as a monotherapy in patients with HRAS-mutant head and neck squamous cell carcinoma (HNSCC). The Company continues to evaluate tipifarnib in combination with alpelisib in patients with PIK3CA-dependent HNSCC as part of its ongoing KURRENT-HN dose-escalation trial. Dose escalation continues in first-in-human trial of KO-2806 – In October 2023, Kura announced that the first patient was dosed in its FIT-001 Phase 1 dose-escalation trial of its next-generation FTI, KO-2806. Concurrent with dose escalation as a monotherapy in the FIT-001 trial, the Company also plans to evaluate KO-2806 in dose-escalation combination cohorts with cabozantinib in clear cell renal cell carcinoma (ccRCC) and with adagrasib in KRASG12C-mutated non-small cell lung cancer (NSCLC). Preclinical data support clinical combinations of KO-2806 with targeted therapies – In October 2023, Kura presented preclinical data supporting its rationale to combine KO-2806 with cabozantinib in ccRCC and with adagrasib in KRASG12C-mutated NSCLC. The new findings illustrate the potential for FTIs to drive enhanced antitumor activity and address mechanisms of innate and adaptive resistance to targeted therapies such as tyrosine kinase inhibitors and KRAS inhibitors. Clinical collaboration with Mirati to evaluate KO-2806 and adagrasib in KRASG12C-mutated NSCLC – In November 2023, Kura announced a clinical collaboration and supply agreement with Mirati Therapeutics to evaluate the combination of KO-2806 and adagrasib in patients with KRASG12C-mutated NSCLC. Kura anticipates dosing the first patients with KO-2806 and adagrasib in KRASG12C-mutated NSCLC by mid-2024. Financial Results Research and development (R&D) expenses for the fourth quarter of 2023 were $32.5 million, compared to $22.7 million for the fourth quarter of 2022. R&D expenses for the full year 2023 were $115.2 million, compared to $92.8 million for the prior year. General and administrative (G&A) expenses for the fourth quarter of 2023 were $14.2 million, compared to $12.5 million for the fourth quarter of 2022. G&A expenses for the full year 2023 were $50.6 million, compared to $47.1 million for the prior year. Net loss for the fourth quarter of 2023 was $42.8 million, compared to a net loss of $33.1 million for the fourth quarter of 2022. Net loss for the full year 2023 was $152.6 million, compared to a net loss of $135.8 million for the prior year. Net loss for the fourth quarter and full year 2023 included non-cash, share-based compensation expense of $7.2 million and $28.1 million, respectively. This compares to $6.8 million and $26.3 million for the same periods in 2022. As of December 31, 2023, Kura had cash, cash equivalents and short-term investments of $424.0 million, compared to $438.0 million as of December 31, 2022. Pro forma for $146 million in approximate net proceeds from the company’s private placement completed in January 2024, Kura had $570 million in cash, cash equivalents and short-term investments at December 31, 2023. Based on its operating plan, management expects that cash, cash equivalents and short-term investments will fund current operations into 2027. Forecasted Milestones Initiate the post-transplant maintenance program for ziftomenib in the first quarter of 2024. Complete enrollment of 85 patients in the KOMET-001 registration-directed trial of ziftomenib in NPM1-m AML by mid-2024. Initiate an expansion cohort evaluating ziftomenib as a monotherapy in patients who have neither NPM1-mutant nor KMT2A-rearranged AML by mid-2024. Determine the RP2D for ziftomenib in combination with ven/aza and initiate dose validation/expansion in frontline AML by mid-2024. Determine the RP2D for ziftomenib in combination with 7+3 by mid-2024. Dose the first patients in the FIT-001 dose-escalation trial of KO-2806 in combination with cabozantinib in ccRCC by mid-2024. Dose the first patients in the FIT-001 dose-escalation trial of KO-2806 in combination with adagrasib in KRASG12C-mutated NSCLC by mid-2024. Complete enrollment of two expansion cohorts to support determination of the optimal biologically active dose for tipifarnib in combination with alpelisib by the end of 2024. Conference Call and Webcast Kura’s management will host a webcast and conference call at 4:30 p.m. ET / 1:30 p.m. PT today, February 27, 2024, to discuss the financial results for the fourth quarter and full year 2023 and to provide a corporate update. The live call may be accessed by dialing (888) 886-7786 for domestic callers and (416) 764-8658 for international callers and entering the conference ID: 02911668. A live webcast and archive of the call will be available online from the investor relations section of the company website at . About Kura Oncology Kura Oncology is a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer. The Company’s pipeline consists of small molecule drug candidates that target cancer signaling pathways. Ziftomenib is a once-daily, oral drug candidate targeting the menin-KMT2A protein-protein interaction for the treatment of genetically defined AML patients with high unmet need. Kura is currently enrolling patients in a Phase 2 registration-directed trial of ziftomenib in NPM1-m R/R AML (KOMET-001). The Company is also conducting a series of studies to evaluate ziftomenib in combination with current standards of care, beginning with ven/aza and 7+3 in NPM1-m and KMT2A-r newly diagnosed and R/R AML (KOMET-007). Tipifarnib, a potent and selective FTI, is currently in a Phase 1/2 trial in combination with alpelisib for patients with PIK3CA-dependent HNSCC (KURRENT-HN). Kura is also evaluating KO-2806, a next-generation FTI, in a Phase 1 dose-escalation trial as a monotherapy and in combination with cabozantinib in ccRCC and with adagrasib in KRASG12C-mutated NSCLC (FIT-001). For additional information, please visit Kura’s website at and follow us on X and LinkedIn. Forward-Looking Statements This news release contains certain forward-looking statements that involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. Such forward-looking statements include statements regarding, among other things, the efficacy, safety and therapeutic potential of Kura’s product candidates, ziftomenib, tipifarnib and KO-2806, progress and expected timing of Kura’s drug development programs and clinical trials and submission of regulatory filings, the presentation of data from clinical trials, plans regarding regulatory filings and future clinical trials, the regulatory approval path for tipifarnib, the strength of Kura’s balance sheet and the sufficiency of cash, cash equivalents and short-term investments to fund its current operating plan to 2027. Factors that may cause actual results to differ materially include the risk that compounds that appeared promising in early research or clinical trials do not demonstrate safety and/or efficacy in later preclinical studies or clinical trials, the risk that Kura may not obtain approval to market its product candidates, uncertainties associated with performing clinical trials, regulatory filings, applications and other interactions with regulatory bodies, risks associated with reliance on third parties to successfully conduct clinical trials, the risks associated with reliance on outside financing to meet capital requirements, and other risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. You are urged to consider statements that include the words “may,” “will,” “would,” “could,” “should,” “believes,” “estimates,” “projects,” “promise,” “potential,” “expects,” “plans,” “anticipates,” “intends,” “continues,” “designed,” “goal,” or the negative of those words or other comparable words to be uncertain and forward-looking. For a further list and description of the risks and uncertainties the Company faces, please refer to the Company's periodic and other filings with the Securities and Exchange Commission, which are available at Such forward-looking statements are current only as of the date they are made, and Kura assumes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. KURA ONCOLOGY, INC. Statements of Operations Data (unaudited) (in thousands, except per share data) Three Months Ended Year Ended December 31, December 31, 2023 2022 2023 2022 Operating Expenses: Research and development $ 32,533 $ 22,668 $ 115,235 $ 92,812 General and administrative 14,229 12,488 50,569 47,053 Total operating expenses 46,762 35,156 165,804 139,865 Other income, net 3,976 2,042 13,173 4,025 Net loss $ (42,786 ) $ (33,114 ) $ (152,631 ) $ (135,840 ) Net loss per share, basic and diluted $ (0.55 ) $ (0.49 ) $ (2.08 ) $ (2.03 ) Weighted average number of shares used in computing net loss per share, basic and diluted 77,337 67,781 73,229 66,990 KURA ONCOLOGY, INC. Balance Sheet Data (unaudited) (in thousands) December 31, December 31, 2023 2022 Cash, cash equivalents and short-term investments $ 423,957 $ 437,985 Working capital 397,218 422,369 Total assets 448,935 456,306 Long-term liabilities 16,399 11,971 Accumulated deficit (721,439 ) (568,808 ) Stockholders’ equity 397,273 420,278 Contacts Investors: Pete De Spain Executive Vice President, Investor Relations & Corporate Communications (858) 500-8833 pete@kuraoncology.com Media: Alexandra Weingarten Associate Director, Investor Relations & Corporate Communications (858) 500-8822 alexandra@kuraoncology.com

– KOMET-008 is evaluating ziftomenib in combination with gilteritinib, FLAG-IDA or LDAC in patients with relapsed/refractory NPM1-mutant or KMT2A-rearranged AML – SAN DIEGO, Feb. 26, 2024 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, today announced that the first patient has been dosed in KOMET-008, the Company’s Phase 1 trial of its menin inhibitor ziftomenib, in combination with gilteritinib, FLAG-IDA or LDAC for the treatment of NPM1-mutant or KMT2A-rearranged acute myeloid leukemia (AML). “Roughly half of patients with relapsed or refractory NPM1-mutant AML have co-occurring FLT3 mutations, and the prognosis for these patients is particularly poor,” said Stephen Dale, M.D., Chief Medical Officer of Kura Oncology. “Given the potential best-in-class safety and tolerability profile as well as the robust monotherapy activity observed in our Phase 1 study of ziftomenib, we believe an all-oral combination of ziftomenib and gilteritinib may provide an attractive treatment option for these patients.” KOMET-008 is a Phase 1 study designed to assess safety and tolerability, pharmacokinetics and evidence of clinical activity of ziftomenib in combination with gilteritinib, FLAG-IDA or LDAC for two genetically defined cohorts, NPM1-mutant AML and KMT2A-rearranged AML, in the relapsed/refractory setting. Trial participants will be enrolled in one of five dose escalation cohorts, including a cohort of NPM1-mutant AML patients with a documented FLT3 co-mutation, who will be treated in combination with the FLT3 inhibitor gilteritinib. For more information regarding KOMET-008, please visit www.clinicaltrials.gov (identifier: NCT06001788). Kura is conducting a series of studies to evaluate ziftomenib in combination with current standards of care in earlier lines of therapy and across multiple patient populations. In July, the Company began dosing patients in the first of these studies, KOMET-007, in combination with venetoclax and azacitidine in patients with relapsed/refractory NPM1-mutant and KMT2A-rearranged AML or in combination with standard induction cytarabine/daunorubicin chemotherapy (7+3) in patients with previously untreated NPM1-mutant and KMT2A-rearranged AML. Kura reported positive preliminary data from 20 patients in KOMET-007 on January 30, 2024. Preclinical data for menin inhibitors in combination with multiple FLT3 inhibitors demonstrate strong synergistic effects compared to either single agent alone. Currently there are no other actively recruiting clinical trials evaluating the combination of a menin inhibitor with a FLT3 inhibitor for the treatment of AML. About Acute Myeloid Leukemia AML is the most common acute leukemia in adults and begins when the bone marrow makes abnormal myeloblasts (white blood cells), red blood cells or platelets. Despite the many available treatments for AML, prognosis for patients remains poor and a high unmet need remains. The menin pathway is considered a driver for multiple genetic alterations of the disease, of which NPM1 mutations are among the most common, representing approximately 30% of AML cases and KMT2A rearrangements represent approximately 5-10% of AML cases. While patients with NPM1-m AML have high response rates to frontline therapy, relapse rates are high and survival outcomes are poor, with only 30% overall survival at 12 months in the R/R setting. Additionally, NPM1 mutations frequently occur with co-mutations in other disease-associated genes, including FLT3, DNMT3A and IDH1/2, with prognosis heavily influenced by the presence of co-occurring mutations. Adult patients with NPM1-m AML and select co-mutations and/or R/R disease have a poor prognosis, with median overall survival of only approximately 7.8 months in 2nd line, 5.3 months in 3rd line and 3.5 months following the 4th line1. Adult patients with KMT2A-r AML have a poor prognosis with high rates of resistance and relapse following standard of care, with median overall survival for this patient population of only 6 months following 2nd line and 2.4 months following 3rd line2. No FDA-approved therapies targeting NPM1-m and KMT2A-r AML currently exist. About Ziftomenib Ziftomenib is a novel, once-daily, oral investigational drug candidate targeting the menin-KMT2A/MLL protein-protein interaction for treatment of genetically defined AML patients with high unmet need. Ziftomenib inhibits the KMT2A/MLL protein complex and exhibits downstream effects on HOXA9/MEIS1 expression and potent anti-leukemic activity in genetically defined preclinical models of AML. Ziftomenib has received Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of AML. Additional information about clinical trials for ziftomenib can be found at kuraoncology.com/clinical-trials/#ziftomenib. About Kura Oncology Kura Oncology is a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer. The Company’s pipeline consists of small molecule drug candidates that target cancer signaling pathways. Ziftomenib is a once-daily, oral drug candidate targeting the menin-KMT2A protein-protein interaction for the treatment of genetically defined AML patients with high unmet need. Kura is currently enrolling patients in a Phase 2 registration-directed trial of ziftomenib in NPM1-mutant relapsed or refractory AML (KOMET-001). The Company is also conducting a series of studies to evaluate ziftomenib in combination with current standards of care, beginning with venetoclax and azacitidine and 7+3 in NPM1-mutant and KMT2A-rearranged newly diagnosed and relapsed/refractory AML (KOMET-007). Tipifarnib, a potent and selective farnesyl transferase inhibitor (FTI), is currently in a Phase 1/2 trial in combination with alpelisib for patients with PIK3CA-dependent head and neck squamous cell carcinoma (KURRENT-HN). Kura is also evaluating KO-2806, a next-generation FTI, in a Phase 1 dose-escalation trial as a monotherapy and in combination with adagrasib in KRASG12C-mutated non-small cell lung cancer and cabozantinib in clear cell renal cell carcinoma (FIT-001). For additional information, please visit Kura’s website at www.kuraoncology.com and follow us on X and LinkedIn. Forward-Looking Statements This news release contains certain forward-looking statements that involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. Such forward-looking statements include statements regarding, among other things, the efficacy, safety and therapeutic potential of ziftomenib, potential benefits of combining ziftomenib with appropriate standards of care, and progress and expected timing of the ziftomenib program and clinical trials. Factors that may cause actual results to differ materially include the risk that compounds that appeared promising in early research or clinical trials do not demonstrate safety and/or efficacy in later preclinical studies or clinical trials, the risk that Kura may not obtain approval to market its product candidates, uncertainties associated with performing clinical trials, regulatory filings, applications and other interactions with regulatory bodies, risks associated with reliance on third parties to successfully conduct clinical trials, risks associated with reliance on outside financing to meet capital requirements, and other risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. You are urged to consider statements that include the words “may,” “will,” “would,” “could,” “should,” “believes,” “estimates,” “projects,” “promise,” “potential,” “expects,” “plans,” “anticipates,” “intends,” “continues,” “designed,” “goal,” or the negative of those words or other comparable words to be uncertain and forward-looking. For a further list and description of the risks and uncertainties the Company faces, please refer to the Company's periodic and other filings with the Securities and Exchange Commission, which are available at www.sec.gov. Such forward-looking statements are current only as of the date they are made, and Kura assumes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. Contacts Investors:Pete De SpainExecutive Vice President, Investor Relations &Corporate Communications(858) 500-8833pete@kuraoncology.com Media:Alexandra WeingartenAssociate Director, Corporate Communications & Investor Relations(858) 500-8822alexandra@kuraoncology.com 1 Issa GC, et al. Blood Adv. 2023;7(6):933-42.2 Issa GC, et al. Blood Cancer J. 2021;11(9):162.