Daunorubicin Hydrochloride

Study of uproleselan combined with chemotherapy did not meet its primary endpoint of overall survival in the intent to treat population Adverse events were consistent with known side effect profiles of chemotherapy used in the study Comprehensive data analysis with medical, statistical, and regulatory experts underway and will be shared as appropriate; company will submit results for presentation at an upcoming medical meeting National Cancer Institute (NCI) Phase 2/3 study in newly diagnosed AML patients remains ongoing Conference call and webcast to be hosted today, May 6, 2024, at 8:30 a.m. ET. ROCKVILLE, Md.--(BUSINESS WIRE)-- GlycoMimetics, Inc. (Nasdaq: GLYC), a late clinical-stage biotechnology company discovering and developing glycobiology-based therapies for cancers and inflammatory diseases, today announced topline results from its Phase 3 global pivotal study of uproleselan in 388 patients with R/R AML. In the study, uproleselan combined with chemotherapy did not achieve a statistically significant improvement in overall survival in the intent to treat population versus chemotherapy alone. Patients treated with uproleselan had a median overall survival of 13 months, compared to 12.3 months in the placebo arm. Adverse events were consistent with known side effect profiles of chemotherapy used in the study. “While the outcome of our Phase 3 study in R/R AML is not what we hoped, we wish to thank the investigators, the participating patients and their families for their dedication to this large, well-controlled randomized study,” said Harout Semerjian, Chief Executive Officer of GlycoMimetics. “We are thoroughly analyzing the data in collaboration with medical, statistical and regulatory experts and are committed to submitting a comprehensive data analysis for presentation at an upcoming medical meeting.” The randomized, double-blind, placebo-controlled Phase 3 clinical study evaluated uproleselan in combination with MEC (mitoxantrone, etoposide and cytarabine) or FAI (fludarabine, cytarabine and idarubicin) in patients with R/R AML. Patients received either uproleselan or placebo for 8 days over 1 cycle of an induction and, if applicable, up to 3 cycles of consolidation. The primary endpoint of the study was overall survival without censoring for transplant. Secondary endpoints included incidence of severe oral mucositis, complete remission rate and remission rate. A total of 388 patients across 70 sites in nine countries were randomized 1:1 between treatment and placebo arms. The NCI and the Alliance for Clinical Trials in Oncology are conducting an adaptive Phase 2/3 study of uproleselan in adults with newly diagnosed AML who are 60 years or older and fit for intensive chemotherapy. The randomized, controlled study is evaluating the addition of uproleselan to a standard cytarabine/daunorubicin regimen (7+3) versus chemotherapy alone. The Phase 2 portion of the study completed enrollment of 267 patients in December 2021. Results of the pre-planned Phase 2 event free survival interim analysis will be reported when available. First Quarter 2024 Preliminary Financial Results Today, the company also disclosed its preliminary financial results for the first quarter of 2024. Cash position: As of March 31, 2024, GlycoMimetics had cash and cash equivalents of $31.3 million, compared to $41.8 million as of December 31, 2023. R&D Expenses: The company’s research and development expenses increased to $6.0 million for the quarter ended March 31, 2024, as compared to $5.4 million for the same period in 2023. These increases were due to raw material acquisition costs for future manufacturing batches. G&A Expenses: The company’s general and administrative expenses decreased to $5.1 million for the quarter ended March 31, 2024, compared to $5.5 million for the same period in 2023. The decrease was due to lower personnel-related and external consulting expenses. Shares Outstanding: Shares of common stock outstanding as of March 31, 2024, were 64,450,835. Conference Call Information The company will host a conference call and webcast today at 8:30 a.m. ET. To access the call by phone, please go to this registration link and you will be provided with dial in details. Participants are encouraged to connect 15 minutes in advance of the scheduled start time. A live webcast of the call will be available on the “Investors” tab on the GlycoMimetics website. A webcast replay will be available for 30 days following the call. Please note this call will replace the previously announced First Quarter 2024 Financial Results call scheduled for May 9, 2024 at 8:30 a.m. ET. About AML AML is the most common acute leukemia in adults. A cancer of the bone marrow, nearly 21,000 people in the United States are diagnosed with AML each year. Despite the availability of multiple treatments, disease prognosis is poor, and new treatment options are needed to improve outcomes. Newly diagnosed AML has the lowest 5-year survival rate of all leukemias at 31.7%. The five-year survival rate for people with relapsed/refractory disease is only 10%. About Uproleselan Discovered and developed by GlycoMimetics, uproleselan (yoo’ pro le’se lan) is an investigational, first-in-class E-selectin antagonist. GlycoMimetics has received Breakthrough Therapy and Fast Track designations from the U.S. Food and Drug Administration (FDA) and Breakthrough Therapy designation from the Chinese National Medical Products Administration for uproleselan as a potential treatment for adult AML patients with relapsed or refractory disease. E-selectin is a leukocyte adhesion molecule constitutively expressed on endothelial cells of the vasculature and bone marrow. In AML, there is evidence that E-selectin–ligand interaction between endothelial cells in the protective niche of the Bone Marrow microEnvironment (BME) and leukemic stem cells and blasts promotes leukemic cell survival and hides them from AML therapies. Uproleselan is designed to disrupt E-selectin binding and prevent leukemic myeloid cells using the protective niche of the BME. About GlycoMimetics, Inc. GlycoMimetics is a late clinical-stage biotechnology company discovering and developing glycobiology-based therapies for cancers, including AML, and for inflammatory diseases. The company’s scientific approach is based on an understanding of the role that carbohydrates play in cell recognition. Its specialized chemistry platform is being deployed to discover small molecule drugs, known as glycomimetics, that alter carbohydrate-mediated recognition in diverse disease states, including cancers and inflammation. GlycoMimetics is leveraging its differentiated expertise with this scientific approach in order to advance its pipeline of wholly owned drug candidates. The company’s goal is to develop transformative therapies for diseases with high unmet medical need. GlycoMimetics is headquartered in Rockville, MD in the BioHealth Capital Region. Learn more at . Forward-Looking Statements This press release contains forward-looking statements. These forward-looking statements may include, but are not limited to, statements regarding the conduct of, and timing for analysis and presentation of data from, clinical trials; potential development and regulatory activities; and the potential benefits and impact of uproleselan. Actual results may differ materially from those described in these forward-looking statements. For a further description of the risks associated with these statements, as well as other risks facing GlycoMimetics, please see the risk factors described in the company’s Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) on March 27, 2024, and other filings GlycoMimetics makes with the SEC from time to time. Forward-looking statements speak only as of the date of this release, and GlycoMimetics undertakes no obligation to update or revise these statements, except as may be required by law. View source version on businesswire.com: Contacts Investor Contact: Argot Partners Leo Vartorella 212-600-1902 Glycomimetics@argotpartners.com Source: GlycoMimetics, Inc. View this news release online at:

– Breakthrough Therapy Designation for ziftomenib in NPM1-mutant AML – – Registration-directed trial of ziftomenib in NPM1-mutant AML on track to complete enrollment by mid-2024 – – Positive preliminary combination data for ziftomenib in NPM1-mutant and KMT2A-rearranged AML – – First patient dosed with KO-2806 and cabozantinib in renal cell carcinoma – – $527 million in cash, cash equivalents and investments provide runway into 2027 – – Management to host webcast and conference call today at 4:30 p.m. ET – SAN DIEGO, May 02, 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 first quarter 2024 financial results and provided a corporate update. “Ziftomenib continues to demonstrate a best-in-class safety and efficacy profile as well as optimal pharmaceutical properties, which we believe will enable it to become a cornerstone of therapy in acute leukemias and beyond,” said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. “This belief is backed by increasing investigator enthusiasm, as evidenced by rapid enrollment across our ongoing ziftomenib studies, and further supported by the FDA’s decision to grant Breakthrough Therapy Designation (BTD) to ziftomenib, making it the first investigational treatment to be granted BTD for the treatment of NPM1-mutant acute myeloid leukemia (AML). In the near term, we look forward to completing enrollment in our registration-directed trial of ziftomenib in NPM1-mutant AML and working closely with FDA to bring this potentially innovative medicine to patients in urgent need of effective treatments.” Recent Highlights Breakthrough Therapy Designation for ziftomenib in NPM1-mutant AML – Last month, the U.S. Food and Drug Administration (FDA) granted BTD to ziftomenib for the treatment of relapsed/refractory (R/R) NPM1-mutant AML. FDA granted BTD based on data from Kura’s ongoing KOMET-001 trial of ziftomenib in patients with R/R NPM1-mutant AML. BTD is awarded for a drug that treats a serious or life-threatening condition and may demonstrate substantial improvement on one or more clinically significant endpoints over available therapies.Registration-directed trial of ziftomenib in NPM1-mutant AML nearing completion – Kura remains on track to complete enrollment of 85 patients in its KOMET-001 registration-directed trial of ziftomenib in NPM1-mutant R/R AML by mid-2024. In the Phase 1 trial, ziftomenib demonstrated a 35% CR rate and 45% overall response rate in 20 heavily pretreated patients with NPM1-mutant AML treated at the recommended Phase 2 dose. 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.Positive preliminary combination data for ziftomenib in NPM1-mutant and KMT2A-rearranged AML – In January 2024, Kura reported preliminary data from the KOMET-007 dose-escalation trial of ziftomenib in combination with venetoclax/azacitidine or cytarabine/daunorubicin (7+3) in patients with NPM1-mutant or KMT2A-rearranged 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 (CR) with full count recovery, for a CR rate of 100%. The overall response rate among the 15 R/R patients treated with ziftomenib and venetoclax/azacitidine was 53%. Continuous daily dosing of ziftomenib at 200 mg was well tolerated. 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. As of the data cutoff, 16 of the first 20 patients remained on trial, including all 11 NPM1-mutant patients.Dose escalation continues in KOMET-007 combination trial of ziftomenib – To date, the 400 mg dose of ziftomenib has been cleared in three of the four cohorts in the KOMET-007 trial: 1) in combination with venetoclax/azacitidine in R/R NPM1-mutant AML, 2) in combination with venetoclax/azacitidine in R/R KMT2A-rearranged AML and 3) in combination with 7+3 in newly diagnosed adverse risk NPM1-mutant AML. Enrollment at the 600 mg dose is ongoing in all three cohorts. Enrollment continues at the 400 mg dose in combination with 7+3 in newly diagnosed adverse risk KMT2A-rearranged AML.First patients dosed in KOMET-008 combination trial of ziftomenib – In February, Kura began dosing patients in its KOMET-008 trial of ziftomenib in combination with additional standards of care, including the FLT3 inhibitor gilteritinib, FLAG-IDA or LDAC, for the treatment of R/R NPM1-mutant or KMT2A-rearranged AML. Preclinical data for ziftomenib in combination with FLT3 inhibitors demonstrate strong synergistic effects compared to either single agent alone. Roughly half of patients with R/R NPM1-mutant AML have co-occurring FLT3 mutations, and the prognosis for these patients is poor.First patients dosed with KO-2806 and cabozantinib in renal cell carcinoma – In March, Kura announced dosing of the first patient with KO-2806, the Company’s next-generation farnesyl transferase inhibitor (FTI), in combination with cabozantinib in clear cell renal cell carcinoma, just four months after dosing the first patients with KO-2806 as a monotherapy in the FIT-001 dose-escalation trial. The Company remains on track to dose the first patient in combination with adagrasib in KRASG12C-mutated non-small cell lung cancer by the middle of this year, as dose escalation of KO-2806 as a monotherapy continues in parallel. Financial Results Research and development expenses for the first quarter of 2024 were $36.3 million, compared to $25.2 million for the first quarter of 2023.General and administrative expenses for the first quarter of 2024 were $18.2 million, compared to $11.4 million for the first quarter of 2023.Net loss for the first quarter of 2024 was $49.5 million, compared to a net loss of $34.1 million for the first quarter of 2023. This included non-cash share-based compensation expense of $8.5 million, compared to $6.8 million for the same period in 2023.As of March 31, 2024, Kura had cash, cash equivalents and short-term investments of $527 million, compared to $424 million as of December 31, 2023. This includes net proceeds of approximately $145.8 million from the Company’s private placement in January 2024.Based on its operating plan, management expects that cash, cash equivalents and short-term investments will fund current operations into 2027. Forecasted Milestones Complete enrollment of 85 patients in the KOMET-001 registration-directed trial of ziftomenib in NPM1-mutant R/R AML by mid-2024.Identify the recommended Phase 2 dose of ziftomenib in combination with venetoclax and azacitidine by mid-2024.Identify the recommended Phase 2 dose of ziftomenib in combination with 7+3 by mid-2024.Initiate Phase 1b expansion study of ziftomenib in combination with standards of care, including venetoclax/azacitidine in newly diagnosed NPM1-mutant or KMT2A-rearranged AML, in the second half of 2024.Submit an investigational new drug application for ziftomenib in a solid tumor indication and present preclinical data at a medical meeting in the second half of 2024.Dose the first patients with KO-2806 and adagrasib in KRASG12C-mutated non-small cell lung cancer by mid-2024.Complete enrollment of two expansion cohorts in KURRENT-HN and identify the optimal biologically active dose of tipifarnib and alpelisib by the end of 2024.Present data from the KURRENT-HN trial of tipifarnib in combination with alpelisib in PIK3CA-dependent head and neck squamous cell carcinoma (HNSCC) in the first half of 2025. 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, May 2, 2024, to discuss the financial results for the first quarter 2024 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: 20226736. A live webcast and archive of the call will be available online from the investor relations section of the company website at www.kuraoncology.com. 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, a once-daily, oral drug candidate targeting the menin-KMT2A protein-protein interaction, has received Breakthrough Therapy Designation for the treatment of R/R NPM1-mutant AML. Kura is currently enrolling patients in a Phase 2 registration-directed trial of ziftomenib in NPM1-mutant R/R AML (KOMET-001). The Company is also conducting a series of clinical trials to evaluate ziftomenib in combination with current standards of care in NPM1-mutant and KMT2A-rearranged newly diagnosed and R/R AML. 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 targeted therapies (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 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 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. KURA ONCOLOGY, INC. Statements of Operations Data (unaudited) (in thousands, except per share data) Three Months Ended March 31, 2024 2023 Operating Expenses: Research and development $36,268 $25,192 General and administrative 18,184 11,374 Total operating expenses 54,452 36,566 Other income, net 4,927 2,497 Net loss $(49,525) $(34,069)Net loss per share, basic and diluted $(0.59) $(0.50)Weighted average number of shares used in computing net loss per share, basic and diluted 83,905 68,403 KURA ONCOLOGY, INC. Balance Sheet Data (unaudited) (in thousands) March 31, December 31, 2024 2023 Cash, cash equivalents and short-term investments $527,122 $423,957 Working capital 505,569 397,218 Total assets 553,908 448,935 Long-term liabilities 16,558 16,399 Accumulated deficit (770,964) (721,439)Stockholders’ equity 505,084 397,273 Contacts Investors: Pete De Spain Executive Vice President, Investor Relations & Corporate Communications(858) 500-8833 pete@kuraoncology.com Media:Alexandra WeingartenAssociate Director, Investor Relations & Corporate Communications(858) 500-8822alexandra@kuraoncology.com