Syndax(SNDX)
搜索文档
Syndax Announces Positive Pivotal Topline Results from Relapsed or Refractory mNPM1 AML Cohort in AUGMENT-101 Trial of Revumenib
Prnewswire· 2024-11-12 20:00
– Primary endpoint met with CR/CRh rate of 23% in pivotal Ph 2 cohort of R/R mNPM1 AML patients (p-value = 0.0014) –– 47% (30/64) ORR in a heavily pre-treated population, including 75% with prior venetoclax exposure in the efficacy population –– Favorable safety and tolerability profile; only 5% of patients discontinued due to treatment-related adverse events –– Results highlight consistency of revumenib's compelling clinical profile –– sNDA filing in R/R mNPM1 AML expected in 1H25, ...
Syndax(SNDX) - 2024 Q3 - Earnings Call Transcript
2024-11-06 09:39
财务数据和关键指标变化 - 截至2024年9月30日,公司现金、现金等价物及短期和长期投资总额为3.996亿美元,结合与Royalty Pharma达成的3.5亿美元的特许权使用费协议,预计将足够支持公司实现盈利 [52][53] - 第三季度运营费用为1.021亿美元,其中研发费用为7100万美元,销售、一般和行政费用为3110万美元 [55] 各条业务线数据和关键指标变化 - Niktimvo于2024年8月获得FDA批准,成为治疗慢性移植物抗宿主病的首个CSF-1R抗体,预计将在2025年第一季度向患者提供 [41] - Revumenib预计将在2024年12月26日获得FDA批准,针对复发或难治性KMT2A重排急性白血病,且在AUGMENT-101试验中显示出64%的总体反应率 [13][22] 公司战略和发展方向和行业竞争 - 公司计划通过与Incyte的合作,利用其在慢性GVHD市场的深厚理解和长期关系,推动Niktimvo的商业化 [11][41] - Revumenib的市场机会被认为是巨大的,针对KMT2A和NPM1突变的急性白血病患者,预计市场机会接近20亿美元 [50] 管理层对经营环境和未来前景的评论 - 管理层对未来前景持乐观态度,认为Niktimvo和Revumenib的推出将显著改善患者的治疗选择,并推动公司长期增长 [49][51] - 管理层强调,Niktimvo的市场需求强劲,预计将占据慢性GVHD治疗市场的显著份额 [43] 其他重要信息 - 公司在第三季度取得了多个重要里程碑,标志着从研发组织向综合商业阶段公司的转变 [7][8] - 公司与Royalty Pharma的协议进一步增强了财务状况,为未来的药物开发和市场推广提供了资金支持 [8][53] 问答环节所有提问和回答 问题: 关于Revumenib的KMT2A和NPM1数据的指南进入过程 - 管理层表示,药物获得批准后,需快速发布数据并提交指南,预计能在两到三周内进入指南 [60] 问题: AUGMENT-101试验中维护治疗的患者比例 - 管理层认为,随着药物的批准,维护治疗的患者比例可能会增加,具体情况将根据实际治疗经验而定 [64] 问题: NPM1数据的临床标准 - 管理层表示,NPM1的CR/CRh率在20%到30%之间被视为可批准的标准,且对数据的预期持乐观态度 [81][107] 问题: NPM1的突破性疗法认定 - 管理层确认未申请NPM1的突破性疗法认定,因缺乏足够的数据支持,但未来可能会考虑申请 [105] 问题: KMT2A数据的更新 - 管理层表示,预计不会再更新KMT2A的数据集,专注于即将到来的NPM1数据 [111]
Syndax(SNDX) - 2024 Q3 - Earnings Call Presentation
2024-11-06 07:14
现金流与费用 - Syndax在2024年9月30日的现金及现金等价物为3.996亿美元[34] - 2024财年的研究与开发费用指导为2.45亿至2.5亿美元[35] - 总运营费用预计为3.65亿至3.7亿美元[35] 产品与临床数据 - Revumenib在R/R KMT2Ar急性白血病的PDUFA行动日期为2024年12月26日,随后将立即推出[36] - Revumenib在R/R KMT2Ar急性白血病的临床数据将在2024年ASH会议上展示[36] - Revumenib在R/R KMT2Ar急性白血病的整体反应率为64%[7] - Revumenib在R/R KMT2Ar急性白血病患者中的完全缓解率为42%[9] - Revumenib与venetoclax/HMA联合使用的患者中,整体反应率为88%[26] - Niktimvo(axatilimab-csfr)已获得FDA批准,用于治疗慢性移植物抗宿主病,预计在2025年第一季度早期推出[32] 市场与销售策略 - Niktimvo的销售努力在美国为30%,在美国以外为50%[31]
Syndax Pharmaceuticals (SNDX) Reports Q3 Loss, Tops Revenue Estimates
ZACKS· 2024-11-06 07:10
Syndax Pharmaceuticals (SNDX) came out with a quarterly loss of $0.98 per share versus the Zacks Consensus Estimate of a loss of $1.13. This compares to loss of $0.73 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 13.27%. A quarter ago, it was expected that this biopharmaceutical company would post a loss of $0.91 per share when it actually produced a loss of $0.80, delivering a surprise of 12.09%.Over the last four quarters, ...
Syndax(SNDX) - 2024 Q3 - Quarterly Report
2024-11-06 05:03
产品上市与研发管线 - 公司获得FDA批准上市首款商业化产品Niktimvo,用于治疗慢性移植物抗宿主病[49] - 公司正在开发一款针对KMT2A重排和NPM1突变急性白血病的创新型候选药物revumenib,正在进行多项临床试验[50,51] - 公司预计Niktimvo将于2025年初在美国上市销售,同时还在开发适应症扩展[49,52,53] - 公司正在评估revumenib在转移性微卫星稳定性结直肠癌中的应用[51] - 公司计划在2024年底前启动一项revumenib联合venetoclax和azacitidine用于新诊断的NPM1突变或KMT2A重排急性白血病患者的关键性试验[51] 商业化与合作 - 公司与Incyte公司合作在美国共同推广Niktimvo,并已提交FDA申请获批两种更小规格的Niktimvo制剂[49] - 公司与Royalty Pharma签署350万美元的特许权使用费融资协议,基于Niktimvo在美国的销售额[54] - 公司有望通过合作协议获得版税收入、研发资金以及里程碑和其他或有付款[75] 财务情况 - 公司研发费用主要用于临床试验、生产工艺开发和临床供应等,未来会继续大幅增加研发投资[57,58,59] - 公司预计未来销售、管理和行政费用会随着Niktimvo商业化和revumenib研发推进而增加[60] - 2024年第三季度和前九个月的总收入分别为1,250万美元和1,600万美元[65] - 2024年前九个月研发费用增加6,821万美元,主要由于revumenib和axatilimab项目的持续推进[67] - 2024年前九个月销售、一般及管理费用增加3,904.6万美元,主要由于商业化准备活动的增加[68] - 2024年前九个月利息收入净额增加355.6万美元,主要由于现金等价物和投资的平均余额增加以及利率上升[69] - 2024年前九个月经营活动产生的现金流出增加9,826.3万美元,主要由于经营亏损增加、预付费用和应收合作款项增加以及应付款项减少[72] - 2024年前九个月投资活动产生的现金流入减少8,470.1万美元,主要由于可供出售证券的购买增加[73] - 2024年前九个月筹资活动产生的现金流入增加293万美元,主要由于行权和ESPP的收入增加[74] - 公司预计未来12个月内现金、现金等价物和短期及长期投资将为其预计的运营费用和资本支出需求提供资金[75] - 公司有望获得最高8.225亿美元的未来付款[75] - 公司未来资本需求取决于多个因素,包括Niktimvo的销售情况、临床试验进展、监管审批、知识产权维护成本等[76] - 公司预计未来至少两年内将持续亏损,需要通过股权融资、债务融资或其他方式筹集资金[79] - 公司设立了2023年ATM计划,可在未来发行最高2亿美元的股票[80] - 公司的现金和投资组合利率变动不会对其运营业绩和现金流产生重大影响[81] - 通胀和价格变动对公司业绩影响不大[82]
Syndax(SNDX) - 2024 Q3 - Quarterly Results
2024-11-06 05:02
Exhibit 99.1 Syndax Reports Third Quarter 2024 Financial Results and Provides Business Update – New revumenib and Niktimvo™clinical data will be highlighted at 66 th ASH Annual Meeting – – mNPM1 AML topline data from AUGMENT-101 expected in 4Q24; potential sNDA filing in 1H25 – – Revumenib NDA in R/R KMT2Ar acute leukemia is being reviewed under RTOR; PDUFA action date of December 26, 2024 – – Niktimvo approved by U.S. FDA for treatment of chronic GVHD after failure of at least two prior lines of systemic t ...
Syndax Reports Third Quarter 2024 Financial Results and Provides Business Update
Prnewswire· 2024-11-06 05:01
– New revumenib and Niktimvo™ clinical data will be highlighted at 66th ASH Annual Meeting –– mNPM1 AML topline data from AUGMENT-101 expected in 4Q24; potential sNDA filing in 1H25 –– Revumenib NDA in R/R KMT2Ar acute leukemia is being reviewed under RTOR; PDUFA action date of December 26, 2024 –– Niktimvo approved by U.S. FDA for treatment of chronic GVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg –– $350 million royalty funding ...
Syndax Pharmaceuticals and Royalty Pharma Enter into $350 Million Royalty Funding Agreement for Niktimvo™
Prnewswire· 2024-11-05 05:01
Proceeds expected to support the upcoming planned launches and fund the continued development of Niktimvo and revumenibExpected to fund Company through profitability; proforma cash approaching $800 million as of June 30WALTHAM, Mass. and NEW YORK, Nov. 4, 2024 /PRNewswire/ -- Syndax Pharmaceuticals (Nasdaq: SNDX) and Royalty Pharma plc (Nasdaq: RPRX) today announced that Syndax has entered into a $350 million synthetic royalty funding agreement with Royalty Pharma based on U.S. net sales of Niktimvo™ (axati ...
Syndax Announces Participation in November Investor Conferences
Prnewswire· 2024-11-01 19:00
WALTHAM, Mass., Nov. 1, 2024 /PRNewswire/ -- Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced that Michael A. Metzger, Chief Executive Officer of Syndax, as well as members of the Syndax management team, will participate in the following upcoming investor conferences: Guggenheim's Inaugural Healthcare Innovation Conference in Boston, MA with a fireside chat on Wednesday, November 13, 2024, at 1:30 p.m. ...
Syndax to Announce Third Quarter 2024 Financial Results and Host Conference Call and Webcast on November 5, 2024
Prnewswire· 2024-10-29 19:00
WALTHAM, Mass., Oct. 29, 2024 /PRNewswire/ -- Syndax Pharmaceuticals (Nasdaq: SNDX), a commercialstage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced that it will report its third quarter 2024 financial results and provide a business update on Tuesday, November 5, 2024. In connection with the earnings release, Syndax's management will host a conference call and live audio webcast at 4:30 p.m. ET on Tuesday, November 5, 2024 to discuss the Company's financial ...