Darolutamide

The PIK3CA wild-type cohort of the Phase 3 VIKTORIA-1 trial is 100% enrolled; expect to report topline data for this cohort in late Q1 2025 or Q2 2025Approximately $264 million in cash, cash equivalents and investments at end of Q3 2024 expected to fund current clinical development program activities through 2026 Management to host webcast and conference call today, November 14, 2024, at 4:30 p.m. ET MINNEAPOLIS, Nov. 14, 2024 (GLOBE NEWSWIRE) -- Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, today announced financial results for the third quarter ended September 30, 2024 and other recent business developments. “Enrollment in our VIKTORIA-1 study remains robust and on-track. The PIK3CA wild-type cohort is 100% enrolled, and enrollment in the PIK3CA mutant cohort is on plan,” said Brian Sullivan, CEO and co-founder of Celcuity. “Based on our current forecast of reaching the event thresholds that will trigger primary analysis in both the PIK3CA wild-type and mutant cohorts, we expect to report topline data for the PIK3CA wild-type cohort sometime in late Q1 2025 or during Q2 2025 and to report topline data for the PIK3CA mutant cohort in the second half of 2025.” Third Quarter 2024 Business Highlights and Other Recent Developments The VIKTORIA-1 Phase 3 clinical trial expects to provide topline data for the PIK3CA wild-type cohort in late Q1 2025 or during Q2 2025 and for the PIK3CA mutant cohort in the second half of 2025. VIKTORIA-1 is evaluating gedatolisib in combination with fulvestrant with and without palbociclib in adults with HR+, HER2- advanced breast cancer who have received prior treatment with a CDK4/6 inhibitor.The PIK3CA wild-type cohort is 100% enrolled and enrollment of the PIK3CA mutant cohort is on-track relative to plan. The VIKTORIA-2 Phase 3 clinical trial remains on track to enroll its first patient in Q2 2025. The VIKTORIA-2 study is a global Phase 3 open-label randomized clinical trial evaluating the efficacy and safety of gedatolisib in combination with fulvestrant plus a CDK4/6 inhibitor, either ribociclib or palbociclib, in comparison to fulvestrant plus a CDK4/6 inhibitor as a first-line treatment for patients with HR+/HER2- advanced breast cancer who are endocrine therapy resistant.Prior to the initiation of the Phase 3 portion of the trial, a safety run-in study will be conducted in 12-36 participants to assess the safety profile of gedatolisib in combination with ribociclib and fulvestrant.Site qualification activities to support activation of up to 200 sites across North America, Europe, Latin America, and Asia are on track. The Phase 1b/2 clinical trial, evaluating gedatolisib in combination with darolutamide for the treatment of patients with metastatic castration resistant prostate cancer (mCRPC), is ongoing and expected to report preliminary data in Q2 2025. Overall survival data from the B2151009 Phase 1b clinical trial will be presented at the San Antonio Breast Cancer Symposium (SABCS), taking place December 10-13, 2024. Details of the poster presentation are as follows: Abstract Title: Overall survival in patients with HR+/HER2- advanced breast cancer treated in a phase 1b trial evaluating gedatolisib in combination with palbociclib and endocrine therapy (SESS-1510)Presentation Number: P4-08-25Date/Time: Thursday, December 12, 5:30 PM CST Additional nonclinical data further characterizing the mechanism of action of gedatolisib and its effect on breast cancer cell metabolic functions will also be presented at the SABCS. Abstract Title: Mechanism of action of gedatolisib in combination with fulvestrant and/or palbociclib in estrogen receptor positive breast cancer models (SESS-989)Abstract Title: Different effects of gedatolisib versus single-node PI3K/AKT/mTOR pathway inhibitors on breast cancer cell metabolic functions (SESS-997) In October, Cancers published results of nonclinical studies in gynecological cancer cell line models highlighting the differences between single-node inhibitors of the PI3K/AKT/mTOR pathway and gedatolisib. The published manuscript is available online and on the publications section of Celcuity’s website. Third Quarter 2024 Financial Results Unless otherwise stated, all comparisons are for the third quarter ended September 30, 2024, compared to the third quarter ended September 30, 2023. Total operating expenses were $30.1 million for the third quarter of 2024, compared to $18.9 million for the third quarter of 2023. Research and development (R&D) expenses were $27.6 million for the third quarter of 2024, compared to $17.5 million for the prior-year period. Of the approximately $10.1 million increase in R&D expenses, $6.3 million primarily related to activities supporting the VIKTORIA-1 Phase 3 trial, the Phase 1b/2 trial and the initiation of the VIKTORIA-2 Phase 3 trial, and $3.8 million was related to increased employee and consulting expenses. General and administrative (G&A) expenses were $2.5 million for the third quarter of 2024, compared to $1.4 million for the prior-year period. Employee and consulting related expenses accounted for $0.9 million of the increase. Professional fees and other administrative expenses accounted for the remaining increase of approximately $0.2 million. Net loss for the third quarter of 2024 was $29.8 million, or $0.70 loss per share, compared to a net loss of $18.4 million, or $0.83 loss per share, for the third quarter of 2023. Non-GAAP adjusted net loss for the third quarter of 2024 was $27.6 million, or $0.65 loss per share, compared to non-GAAP adjusted net loss of $17.3 million, or $0.78 loss per share, for the third quarter of 2023. Non-GAAP adjusted net loss excludes stock-based compensation expense, non-cash interest expense, and non-cash interest income. Because these items have no impact on Celcuity’s cash position, management believes non-GAAP adjusted net loss better enables Celcuity to focus on cash used in operations. For a reconciliation of financial measures calculated in accordance with generally accepted accounting principles in the United States (GAAP) to non-GAAP financial measures, please see the financial tables at the end of this press release. Net cash used in operating activities for the third quarter of 2024 was $20.6 million, compared to $12.7 million for the third quarter of 2023. At September 30, 2024, Celcuity reported cash, cash equivalents and short-term investments of $264.1 million. Webcast and Conference Call Information The Celcuity management team will host a webcast/conference call at 4:30 p.m. ET today to discuss the third quarter 2024 financial results and provide a corporate update. To participate in the teleconference, domestic callers should dial 1-800-717-1738 or 1-646-307-1865. A live webcast presentation can also be accessed using this weblink: https://viavid.webcasts.com/starthere.jsp?ei=1688854&tp_key=0b14db1255. A replay of the webcast will be available on the Celcuity website following the live event. About Celcuity Celcuity is a clinical-stage biotechnology company focused on development of targeted therapies for treatment of multiple solid tumor indications. The company's lead therapeutic candidate is gedatolisib, a potent, pan-PI3K and mTOR inhibitor. Its mechanism of action and pharmacokinetic properties are highly differentiated from other currently approved and investigational therapies that target PI3K or mTOR alone or together. A Phase 3 clinical trial, VIKTORIA-1, evaluating gedatolisib in combination with fulvestrant with or without palbociclib in patients with HR+/HER2- advanced breast cancer is currently enrolling patients. More detailed information about the VIKTORIA-1 study can be found at ClinicalTrials.gov. A Phase 1b/2 clinical trial, CELC-G-201, evaluating gedatolisib in combination with darolutamide in patients with metastatic castration resistant prostate cancer, is enrolling patients. A Phase 3 clinical trial, VIKTORIA-2, evaluating gedatolisib plus a CDK4/6 inhibitor and fulvestrant as first-line treatment for patients with HR+/HER2- advanced breast cancer is expected to begin enrolling patients in the second quarter of 2025. Celcuity is headquartered in Minneapolis. Further information about Celcuity can be found at www.celcuity.com. Follow us on LinkedIn and Twitter. Forward-Looking Statements This press release contains statements that constitute "forward-looking statements" including, but not limited to, the design of our clinical trials; the timing of initiating and enrolling patients in, and receiving results and data from, our clinical trials; the costs and expected results from any ongoing or planned clinical trials; the market opportunity for gedatolisib; revenue expectations; our strategy, marketing and commercialization plans, including the benefits of strategic decisions regarding studies and trials; other expectations with respect to Celcuity's lead product candidate, gedatolisib, and its CELsignia platform; our anticipated use of cash; and the strength of our balance sheet. In some cases, you can identify forward-looking statements by terminology such as "may," "should," "expects," "plans," "anticipates," "believes," "estimates," "predicts," "potential," "intends" or "continue," and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. Forward-looking statements are subject to numerous risks, uncertainties, and conditions, many of which are beyond the control of Celcuity. These include, but are not limited to, unforeseen delays in our clinical trials, our ability to obtain and maintain regulatory approvals to commercialize our products, and the market acceptance of such products, the development of therapies and tools competitive with our products, our ability to access capital upon favorable terms or at all, and those risks set forth in the Risk Factors section in Celcuity's Annual Report on Form 10-K for the year ended December 31, 2023 filed with the Securities and Exchange Commission on March 27, 2024. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Celcuity undertakes no obligation to update these statements for revisions or changes after the date of this press release, except as required by law. View source version of release on GlobeNewswire.com Contacts: Celcuity Inc. Brian Sullivan, bsullivan@celcuity.com Vicky Hahne, vhahne@celcuity.com (763) 392-0123 ICR HealthcarePatti Bank, patti.bank@icrhealthcare.com(415) 513-1284 Celcuity Inc.Condensed Balance Sheets September 30, 2024 December 31, 2023 (unaudited) Assets Current Assets: Cash and cash equivalents$12,603,289 $30,662,774 Investments 251,455,468 149,919,974 Other current assets 8,379,682 10,007,849 Total current assets 272,438,439 190,590,597 Property and equipment, net 338,787 228,782 Operating lease right-of-use assets 259,744 400,019 Total Assets$273,036,970 $191,219,398 Liabilities and Stockholders' Equity: Current Liabilities: Accounts payable$9,158,778 $5,076,699 Operating lease liabilities 175,226 184,950 Accrued expenses 16,974,725 8,927,094 Total current liabilities 26,308,729 14,188,743 Operating lease liabilities 95,699 225,922 Note payable, non-current 96,923,914 37,035,411 Total Liabilities 123,328,342 51,450,076 Total Stockholders' Equity 149,708,628 139,769,322 Total Liabilities and Stockholders' Equity$273,036,970 $191,219,398 Celcuity Inc.Condensed Statements of Operations(unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Operating expenses: Research and development$27,587,483 $17,488,236 $70,732,017 $42,512,811 General and administrative 2,472,416 1,409,801 6,104,803 3,988,248 Total operating expenses 30,059,899 18,898,037 76,836,820 46,501,059 Loss from operations (30,059,899) (18,898,037) (76,836,820) (46,501,059) Other (expense) income Interest expense (3,343,989) (1,372,132) (7,005,284) (3,929,140)Interest income 3,612,099 1,865,629 8,716,040 5,499,555 Other income, net 268,110 493,497 1,710,756 1,570,415 Net loss before income taxes (29,791,789) (18,404,540) (75,126,064) (44,930,644)Income tax benefits - - - - Net loss$(29,791,789) $(18,404,540) $(75,126,064) $(44,930,644) Net loss per share, basic and diluted$(0.70) $(0.83) $(1.96) $(2.05) Weighted average common shares outstanding, basic and diluted 42,793,047 22,117,626 38,299,548 21,920,147 Cautionary Statement Regarding Non-GAAP Financial Measures This press release contains references to non-GAAP adjusted net loss and non-GAAP adjusted net loss per share. Management believes these non-GAAP financial measures are useful supplemental measures for planning, monitoring, and evaluating operational performance, as they exclude stock-based compensation expense, non-cash interest expense, and non-cash interest income from net loss and net loss per share. Management excludes these items because they do not impact Celcuity’s cash position, which management believes better enables Celcuity to focus on cash used in operations. However, non-GAAP adjusted net loss and non-GAAP adjusted net loss per share are not recognized measures under GAAP and do not have a standardized meaning prescribed by GAAP. As a result, management’s method of calculating non-GAAP adjusted net loss and non-GAAP adjusted net loss per share may differ materially from the method used by other companies. Therefore, non-GAAP adjusted net loss and non-GAAP adjusted net loss per share may not be comparable to similarly titled measures presented by other companies. Investors are cautioned that non-GAAP adjusted net loss and non-GAAP adjusted net loss per share should not be construed as alternatives to net loss, net loss per share or other statements of operations data (which are determined in accordance with GAAP) as an indicator of Celcuity’s performance or as a measure of liquidity and cash flows. Celcuity Inc.Reconciliation of GAAP Net Loss to Non-GAAP Adjusted Net Loss andGAAP Net Loss Per Share to Non-GAAP Adjusted Net Loss Per Share Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 GAAP net loss$(29,791,789) $(18,404,540) $(75,126,064) $(44,930,644)Adjustments: Stock-based compensation Research and development(1) 1,190,424 660,706 3,000,641 1,954,689 General and administrative(2) 740,777 447,931 1,672,418 1,704,213 Non-cash interest expense(3) 730,741 520,794 1,891,139 1,523,699 Non-cash interest income(4) (473,584) (480,520) (1,112,420) (439,331)Non-GAAP adjusted net loss$(27,603,431) $(17,255,629) $(69,674,286) $(40,187,374) GAAP net loss per share - basic and diluted$(0.70) $(0.83) $(1.96) $(2.05)Adjustment to net loss (as detailed above) 0.05 0.05 0.14 0.22 Non-GAAP adjusted net loss per share$(0.65) $(0.78) $(1.82) $(1.83) Weighted average common shares outstanding, basic and diluted 42,793,047 22,117,626 38,299,548 21,920,147 (1) To reflect a non-cash charge to operating expense for Research and Development stock-based compensation.(2) To reflect a non-cash charge to operating expense for General and Administrative stock-based compensation.(3) To reflect a non-cash charge to other expense for amortization of debt issuance and discount costs and PIK interest related to the issuance of a note payable.(4) To reflect a non-cash adjustment to other income for accretion on investments.

点击上方蓝字 关注我们 编者按：前列腺癌是男性泌尿生殖系统最常见的恶性肿瘤之一， 晚期前列腺癌患者的预后较差，是临床治疗的一大难点。中国前列腺癌研究协作组制定了《前列腺癌药物去势治疗随访管理中国专家共识（2024版）》指出，雄性激素剥夺治疗（ADT）是晚期转移性前列腺癌患者的主要全身性基础治疗手段。即便前列腺癌发展到去势抵抗阶段，也应在维持去势治疗的基础上开启后续治疗。为进一步落实共识指导思想，结合临床实践，本期“百家泌闻”栏目中，北京大学肿瘤医院杜鹏教授分享了一例老年晚期前列腺癌伴多发骨转移病例，在参与内分泌治疗临床研究进展为去势抵抗后，转换治疗方案为戈舍瑞林微球+达罗他胺+多西他赛方案，逐步控制PSA。目前该病例仍在随访中，希望能为临床治疗带来参考。 ▌基本信息 患者，男，55岁（1968年生）。 主诉：2023年1月31日，因外院穿刺诊断前列腺癌20余天前来就诊。 现病史：因反复尿潴留行穿刺活检，病理报告示前列腺癌，Gleason评分 4+5=9（预后分级分组为5/5），外院骨扫描考虑多发骨转移。 既往史：高血压7年余（2级），口服硝苯地平血压控制良好。余正常。 治疗史：未行其他抗肿瘤治疗，醋酸戈舍瑞林缓释植入剂 3.6mg 1/4w 2023.1.20皮下注射。 其他：否认家族肿瘤史、遗传病史、传染病史等。既往吸烟史、饮酒史。 ▌体格检查 体重：72kg 身高：170c ECOG评分：0分 血压：117/78 mmHg 查体：发育良，营养好，神志清，步入病房，查体合作。未触及肿大淋巴结。头、颈、胸、腹、躯干及四肢、神经系统正常。 专科情况：双肾区无异常隆起，双肾下极未触及，双肾区无压痛和叩击痛。双输尿管走形区无压痛。耻骨上区无压痛，膀胱未充盈。外生殖器发育正常。 ▌相关检查 病理检查：前列腺12点穿刺活检显示 -前列腺腺泡腺癌，Gleason评分：5+4=9分，见神经侵犯，WHO预后分组为V/V组； -原单位免疫组化：PSA+，CK+，CD56-，CgA-，Syn-，HER2 2+，Ki67 10%，NKX3.1+ 总前列腺特异性抗原（TPSA） 273 ng/ml 游离前列腺特异性抗原（FPSA）24.35 ng/ml ▌疾病诊断 1. 前列腺恶性肿瘤（腺泡腺癌）（T3N0M1）； 2. 高血压。 ▌治疗过程 2023年1月31日 治疗 加入临床研究 参与“一项在高风险转移性激素敏感性前列腺癌男性患者中评价阿贝西利联合阿比特龙及泼尼松的3期、随机、双盲、安慰剂对照研究”，签署知情同意书并入组。 2023年2月15日 治疗 （临床研究方案C1D1） 戈舍瑞林缓释植入剂+阿贝西利/安慰剂+阿比特龙+泼尼松 2024年1月23日 随访 PSA 4ng/ml；未见新发骨转移，暂不建议更改治疗方案；血尿（1级）。 2024年5月14日 随访（临床研究C17 D2） 全身骨扫描显示：颅骨、脊柱、双侧肋骨、胸骨、双侧锁骨、双侧肩胛骨、骨盆、四肢骨可见多发性放射性分布浓聚灶，呈“超级骨显像”。对比2023年12月20日骨扫描结果为全身多发骨转移瘤，呈“超级骨显像”，骨盐代谢大致同前。 诊断为：前列腺癌（转移性，多发骨转移，去势抵抗） 2024年5月27日 治疗 PSA 16ng/ml；出组临床研究。 更改治疗方案为：戈舍瑞林微球+达罗他胺（末次给药6月22日） 2024年7月11日（多西他赛C1）、8月1日（多西他赛C2）治疗 PSA进展（7月2日为45.3ng/ml） 更改治疗方案为：戈舍瑞林微球+达罗他胺+多西他赛（6个周期） 2024年8月1日 治疗（多西他赛C2） 戈舍瑞林微球+达罗他胺+多西他赛 2024年8月13日 随访 化疗第4天恶心呕吐、食欲下降、高热、疼痛、恶心、纳差、体温最高39.6度，WBC升高。 2024年8月22日（多西他赛C3）、9月12日（多西他赛 C4） 、10月8日（多西他赛 C5）治疗 戈舍瑞林微球+达罗他胺+多西他赛 2024年9月23日 随访 PSA 32.5 ng/ml 睾酮 ＜0.025 ng/ml 患者疾病进展后使用戈舍瑞林微球+达罗他胺+多西他赛期间PSA变化 当前状况： 患者病情稳定，仍在治疗中，建议继续实施戈舍瑞林微球+达罗他胺+多西他赛方案，多西他赛化疗实施至6个周期完成。 病例小结 该患者为前列腺腺泡腺癌，Gleason评分 5+4=9分，确诊分期为T3N0M1，伴有多发骨转移。转移性前列腺癌一般预后较差，5年生存率仅有34.1%。我国卫健委颁布的《前列腺癌诊疗指南（2022）》指出，ADT是晚期转移性前列腺癌患者的主要全身性基础治疗手段，也是各种新型联合治疗方案的基础。该患者在就诊前即已于外院接受了戈舍瑞林缓释植入剂初步治疗，且有机会参与到戈舍瑞林+阿贝西利/安慰剂+阿比特龙+泼尼松的临床研究中。基于前期的LATITUDE研究和STAMPEDE研究，阿比特龙+泼尼松已然是转移性前列腺癌的标准治疗方案之一，虽然在患者所参与的临床研究在揭盲前暂不明确是否使用了阿贝西利，但无论如何，患者均接受了有效的内分泌治疗。从2023年2月参与临床研究到2024年5月，患者使用戈舍瑞林缓释植入剂+阿贝西利/安慰剂+阿比特龙+泼尼松方案共17个周期，前期状况维持相对较好，PSA得到了有效控制，但后期无奈发生了全身多发骨转移。 在2024年5月患者出组后换用戈舍瑞林微球+达罗他胺无法控制疾病，且出现了PSA再次上升，达到45.3ng/ml，可见患者出现了明显的去势抵抗。去势抵抗型晚期前列腺癌侵袭性强，整体预后差，该阶段是前列腺癌临床治疗策略的重要切换点。《前列腺癌药物去势治疗随访管理中国专家共识（2024版）》指出，对于去势抵抗型晚期前列腺癌，应在维持去势治疗的基础上，开启后续治疗，其主要治疗目的为延长生存，改善生活质量。《前列腺癌诊疗指南（2022）》推荐以多西他赛为基础的化疗方案是去势抵抗型前列腺癌的标准治疗方案之一，可以有效控制疾病进展，减轻疼痛，提高生活质量。于是将该患者的治疗方案转换为戈舍瑞林微球+达罗他胺+多西他赛方案，目前已经持续治疗5个周期，患者PSA持续稳步下降，且睾酮水平持续维持在理想状态，反映了稳定的控制效果。后续该患者需要继续完成多西他赛6个周期的治疗，并需要定期、规律随访，以防止疾病的进一步进展以及预防骨相关不良事件的发生。总之，密切关注患者疾病发展状况，及时调整治疗方案，将最大化患者的治疗获益。 该患者的整个治疗过程中戈舍瑞林持续发挥着药物去势作用，并维持着稳定的治疗效果。值得一提的是，在“以患者为中心”的理念推动下，戈舍瑞林等GnRHa的给药剂型也在不断改良中。新近作为我国2.2类改良型新药、并首次使用“二代微球技术”而上市的戈舍瑞林微球具有稳定、等效、安全和便捷等优势，可为前列腺癌患者提供更加持续耐受、稳定去势的治疗效果。在戈舍瑞林微球新剂型可及性不断增强的背景下，期待其能够在临床实践中发挥更大的作用，助力前列腺癌患者获得更好的生存、更优的生活质量。 杜鹏 教授 医学博士、主任医师、教授、博士生导师 北京大学肿瘤医院泌尿外科副主任 北京抗癌协会常务理事 北京抗癌协会早癌筛查专业委员会主任委员 《泌尿外科杂志》（电子版）执行主编 《坎贝尔泌尿外科》医生手册主译 中国医师协会泌尿外科分会机器人学组委员 北京医学会泌尿外科分会机器人学组委员 国家医学考试中心命题专家 （来源：《肿瘤瞭望》编辑部） 声 明 凡署名原创的文章版权属《肿瘤瞭望》所有，欢迎分享、转载。本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。