CAMBRIDGE, Mass., June 12, 2024 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (the "Company") (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, today announced upcoming presentations to be made at the 31st Annual FSHD Society International Research Congress being held June 13-14, 2024 in Denver, Colorado, including an abstract outlining the baseline characteristics of patients enrolled in ...

Fulcrum Therapeutics, Inc. (FULC) closed the last trading session at $7.91, gaining 6.8% over the past four weeks, but there could be plenty of upside left in the stock if short-term price targets set by Wall Street analysts are any guide. The mean price target of $13.38 indicates a 69.2% upside potential.The average comprises eight short-term price targets ranging from a low of $5 to a high of $19, with a standard deviation of $5.13. While the lowest estimate indicates a decline of 36.8% from the current p ...

财务数据和关键指标变化 - 截至2024年3月31日,公司现金、现金等价物和可流通证券为2.133亿美元,较2023年12月31日的2.362亿美元减少2,290万美元,主要由于经营活动产生的净现金流出 [16] - 考虑到与Sanofi的合作协议中8,000万美元的里程碑付款,公司在2024年3月31日的现金、现金等价物和可流通证券达到2.933亿美元 [16][18] - 2024年第一季度,公司没有合作收入,而2023年同期为30万美元,减少是由于2023年第四季度完成了与Myocardia的研究服务 [16] - 2024年第一季度研发费用为1,980万美元,较2023年同期的1,670万美元增加310万美元,主要由于REACH临床试验推进成本增加 [17] - 2024年第一季度一般及行政费用为1,010万美元,较2023年同期的1,150万美元减少140万美元,主要由于员工薪酬成本降低 [17] - 2024年第一季度净亏损为2,690万美元,而2023年同期为2,480万美元 [17] 各条业务线数据和关键指标变化 - 公司目前最先进的项目是losmapimod,这是一种口服小分子选择性p38 alpha/beta MAP激酶抑制剂,可抑制DUX4表达,从而预防FSHD患者肌肉细胞死亡 [9] - 在2023年9月完成了REACH III期临床试验的入组,共260名患者,截至2024年4月底,已有146名患者完成48周治疗期,144名患者选择进入开放标签延长期 [10] - REACH III期临床试验的主要终点是相对表面积(RSA)的变化,这是一种定量评估可触及工作空间的指标,反映上肢活动能力,在II期试验中losmapimod相比安慰剂提高了10% [11] - 另一项目pociredir是一种口服HbF诱导剂,用于治疗镰状细胞病,2023年8月FDA解除了I期临床试验的临床hold,公司正在加快推进临床试验 [12][13][14] 各个市场数据和关键指标变化 - FSHD在美国的患病人群约为3万例,是一种罕见的肌肉营养不良症,导致上肢功能和活动能力严重受损,20%的患者最终需要使用轮椅 [9] - 镰状细胞病在美国约有10万例患者,全球约440万例,是最常见的非恶性血液疾病之一 [12] 公司战略和发展方向及行业竞争 - 公司与Sanofi达成合作,Sanofi获得losmapimod在美国以外的独家商业化权利,这将使公司能够专注于在美国市场的商业化准备 [5][6] - 与Sanofi合作可以更快将losmapimod带给全球患者,同时也降低了公司作为一家小型生物科技公司独自进行首个产品商业化的风险 [6] - Sanofi在神经肌肉疾病领域有丰富的经验和强大的商业能力,这将在监管、定价和报销等方面为公司提供宝贵的支持 [46][64] - 公司正在积极准备losmapimod在美国的商业化,包括引进有丰富罕见病上市经验的首席商务官,以及专注于确保基因检测不会成为准入障碍的市场准入团队 [45][46] - 在FSHD领域,Avidity正在开发一种直接抑制DUX4的候选药物,但其临床影响仍有待观察,公司认为losmapimod通过上游抑制DUX4并减少下游基因表达的方式也具有竞争优势 [36][37][38] 管理层对经营环境和未来前景的评论 - 公司选择Sanofi作为理想的合作伙伴,可以更快将losmapimod带给全球患者,同时也可以专注于在美国市场的商业化准备 [5][6] - 公司已完成REACH III期临床试验的入组,并有超过半数患者完成了48周治疗,这显示了患者对该药物的强烈需求 [10] - 公司已解决FDA对pociredir I期临床试验的临床hold,正在加快推进临床试验,认为这一口服HbF诱导剂有潜力为镰状细胞病患者提供一种差异化的治疗选择 [12][13][14] - 公司已延长现金储备至2027年,并引进了丰富罕见病上市经验的新任首席医疗官,为即将到来的关键时刻做好准备 [8][18] 问答环节重要的提问和回答 问题1 **Edward Tenthoff 提问** 关于losmapimod的制造和供应,公司与Sanofi的分工安排如何?在等待III期数据方面,还有哪些需要关注的重点工作? [25][28] **Paul Bruno 和 Iain Fraser 回答** 目前公司将继续负责全球的临床和商业供应,未来Sanofi也有机会接手制造和供应。在等待III期数据方面,临床试验进展顺利,最后几名患者的随访也在按计划进行,不会受到合作的影响。 [27][29][30] 问题2 **Gregory Renza 提问** 在FSHD竞争格局中,公司如何看待Avidity的候选药物对DUX4下游基因表达的影响,以及losmapimod在这方面的优势? [36] **Alex Sapir 和 Iain Fraser 回答** losmapimod通过上游抑制DUX4,也能够在体外实验中减少DUX4下游基因的表达。虽然Avidity的候选药物直接抑制DUX4,但其临床意义仍有待观察。公司在体外实验中观察到losmapimod能够浓度依赖性地降低DUX4及其下游基因的表达,为临床疗效奠定了基础。 [37][38][39] 问题3 **Corinne Johnson 提问** 公司计划如何利用Sanofi合作的资金支持在美国的商业化准备?Sanofi作为合作伙伴除了资金,还能为公司带来哪些其他优势? [45][

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, 2024 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: 001-38978 FULCRUM THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 47-4839948 (State or other jur ...

Exhibit 99.1 Fulcrum Therapeutics Announces Recent Business Highlights and Financial Results for First Quarter 2024 ― Entered into a collaboration and ex-U.S. license agreement with Sanofi for the development and commercialization of losmapimod ― ― On track to report topline data for Phase 3 REACH trial of losmapimod in facioscapulohumeral muscular dystrophy (FSHD) in the fourth quarter of 2024 ― ― Industry veteran, Patrick Horn M.D., Ph.D., appointed as chief medical officer ― ― Conference call and webcast ...

― Sanofi receives exclusive rights to commercialize losmapimod in all territories outside the U.S.; Fulcrum retains full U.S. commercialization rights ― ― Fulcrum will receive an upfront payment of $80.0 million, and is eligible to receive $975.0 million in potential milestones, plus royalties on ex-U.S. product sales; parties will share future global development costs 50:50 ― ― Conference call and webcast scheduled for 8:00 a.m. ET today to discuss the collaboration and other recent corporate developments, ...

― Phase 2b ReDUX4 clinical trial indicates improvements in functional, structural, and patient-reported outcomes; findings informed the design and choice of efficacy endpoints in the ongoing Phase 3 clinical trial― ― Topline data for the Phase 3 REACH clinical trial of losmapimod in FSHD expected during 4Q'24― CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically def ...

CAMBRIDGE, Mass., May 06, 2024 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (the "Company") (NASDAQ:FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, today announced that its first quarter 2024 financial results will be released on Monday, May 13, 2024 before the U.S. financial markets open. Management will host a conference call and webcast at 8:00 a.m. ET to discuss the results and provide an ...

―Industry veteran with late-stage clinical development, medical affairs, and regulatory experience; well-positioned to advance losmapimod towards a potential regulatory submission and approval ― ― Interim Chief Medical Officer, Iain Fraser, MBChB, DPhil, will continue to serve on Fulcrum's executive leadership team as senior vice president (SVP) of early development ― CAMBRIDGE, Mass., March 18, 2024 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Fulcrum) (NASDAQ:FULC), a clinical-stage biopharmaceutical ...

Fulcrum Therapeutics, Inc.公司概况 - Fulcrum Therapeutics, Inc.是一家临床阶段的生物制药公司，专注于开发小分子药物，改善患有基因定义罕见疾病的患者的生活[1] Fulcrum员工股票期权 - Fulcrum向两名新员工授予了非法定股票期权，共计85,600股，行权价格为每股11.33美元，期限为十年，分四年解锁，符合Nasdaq上市规则[2] Fulcrum临床开发项目 - Fulcrum的两个主要临床开发项目是losmapimod和pociredir，分别用于治疗肌肉萎缩症和镰状细胞病[4]