Vicuron Pharmaceuticals LLC

▎Armstrong2024年5月15日，黑石宣布创立Uniquity Bio，同时宣布核心管线TSLP抗体Solrikitug获得FDA批准开展二期临床试验，黑石将提供最高3亿美元的资金推进多个适应症的开发。Uniquity Bio的TSLP抗体从默沙东引进，几乎开发COPD、哮喘等适应症。默沙东TSLP抗体MK-8226此前曾开展特应性皮炎一期临床，后终止该项目，Solrikitug应即为MK-8226。黑石搭建了经验丰富的管理团队，首席执行官Brian Lortie曾在葛兰素史克工作15年，其后加入Endo Pharmaceuticals，加入Uniquity Bio之前为Onspira Therapeutics的CEO，该公司被Altavant（Roivant子公司）收购，后者又在2019年被住友收购（30亿美元收购了5家vant公司）。首席医学官Tim Henkel此前为VenatoRx Pharmaceuticals的CMO，还曾任职于Ceptaris Therapeutics (被Actelion收购), Ception Therapeutics (被Cephalon收购)，Vicuron Pharmaceuticals (被辉瑞收购)。首席科学官Hector Ortega此前为Gossamer Bio的临床开发副总裁，更早的时候就职于葛兰素史克，任职临床开发高级医学总监，在其领导下将IL-5抗体Nucala推向上市，以及之前在罗氏参与IL-13抗体、IgE抗体的临床开发。总结赛诺菲IL-4R抗体Dupixent在COPD的成功具有开创性意义，打开了一个巨大的市场，也吸引了更多追随者。Fairmount基金孵化的Apogee第2天管线即为长效IL-4R抗体，开发COPD适应症。黑石孵化的Uniquity Bio则选择引进默沙东高亲和力TSLP抗体，开发COPD适应症。Armstrong技术全梳理系列GPRC5D靶点全梳理；CD40靶点全梳理；CD47靶点全梳理；补体靶向药物技术全梳理；补体药物：眼科治疗的重要方向；Claudin 6靶点全梳理；Claudin 18.2靶点全梳理；靶点冷暖，行业自知；中国大分子新药研发格局；被炮轰的“me too”；佐剂百年史；胰岛素百年传奇；CUSBEA：风雨四十载；中国新药研发的焦虑；中国生物医药企业的研发竞争；中国双抗竞争格局；中国ADC竞争格局；中国双抗技术全梳理；中国ADC技术全梳理；Ambrx技术全梳理；Vir Biotech技术全梳理；Immune-Onc技术全梳理；亘喜生物技术全梳理；康哲药业技术全梳理；科济药业技术全梳理；恺佧生物技术全梳理；同宜医药技术全梳理；百奥赛图技术全梳理；腾盛博药技术全梳理；创胜集团技术全梳理；永泰生物技术全梳理；中国抗体技术全梳理；德琪医药技术全梳理；德琪医药技术全梳理2.0；和铂医药技术全梳理；荣昌生物技术全梳理；再鼎医药技术全梳理；药明生物技术全梳理；恒瑞医药技术全梳理；豪森药业技术全梳理；正大天晴技术全梳理；吉凯基因技术全梳理；基石药业技术全梳理；百济神州技术全梳理；百济神州技术全梳理第2版；信达生物技术全梳理；信达生物技术全梳理第2版；中山康方技术全梳理；复宏汉霖技术全梳理；先声药业技术全梳理；君实生物技术全梳理；嘉和生物技术全梳理；志道生物技术全梳理；道尔生物技术全梳理；尚健生物技术全梳理；康宁杰瑞技术全梳理；科望医药技术全梳理；科望医药技术全梳理2.0；岸迈生物技术全梳理；礼进生物技术全梳理；康桥资本技术全梳理；余国良的抗体药布局；荃信生物技术全梳理；安源医药技术全梳理；三生国健技术全梳理；仁会生物技术全梳理；乐普生物技术全梳理；同润生物技术全梳理；宜明昂科技术全梳理；派格生物技术全梳理；迈威生物技术全梳理；Momenta技术全梳理；NGM技术全梳理；普米斯生物技术全梳理；普米斯生物技术全梳理2.0；三叶草生物技术全梳理；贝达药业抗体药全梳理；泽璟制药抗体药全梳理；恒瑞医药抗体药全梳理；齐鲁制药抗体药全梳理；石药集团抗体药全梳理；豪森药业抗体药全梳理；华海药业抗体药全梳理；科伦药业抗体药全梳理；百奥泰技术全梳理；凡恩世技术全梳理。

— Santiago Arroyo, M.D., Ph.D. joined as CMO on November 7, 2022 — — Jeff W. Jacobs, Ph.D. to join as CSO on December 1, 2022 — CAMBRIDGE, Mass. –, Nov. 07, 2022 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced the appointment of Santiago Arroyo, M.D., Ph.D., as Chief Medical Officer, and Jeff W. Jacobs, Ph.D., as Chief Scientific Officer. “We are thrilled to welcome Drs. Arroyo and Jacobs to our executive team,” said Bryan Stuart, Chief Executive Officer at Fulcrum. “Dr. Arroyo has extensive experience leading clinical development at multiple biotech companies, most recently developing novel therapeutics to treat rare, immune-mediated diseases at Momenta Pharmaceuticals. Dr. Jacobs has a lengthy history in preclinical drug development, including spearheading the discovery of two drugs that have commercially launched. Their scientific and clinical expertise will be invaluable to Fulcrum as we advance our portfolio of transformative therapies.” Santiago Arroyo, M.D., Ph.D. – Chief Medical OfficerDr. Arroyo brings to Fulcrum nearly 20 years of biopharmaceutical industry experience, leading the clinical development of therapeutics across multiple disease areas. In his role as Chief Medical Officer at Momenta Pharmaceuticals (acquired by Johnson & Johnson), Dr. Arroyo led the clinical development of the company’s broad portfolio of hematology, neurology, immunology, and materno-fetal therapeutics. Previously, he was the Chief Medical Officer of Boston Pharmaceuticals. Before that, he held multiple clinical roles at Bristol-Myers Squibb and Pfizer, where he was the Head of Clinical Research and Chief Medical Officer for Pharmacotherapeutics. Earlier, Dr. Arroyo held clinical development roles at Eisai Global Clinical Development and Schwarz Biosciences. Dr. Arroyo began his career as an academic neurologist at the Medical College of Wisconsin, Johns Hopkins Hospital and Medical School, and the Hospital Clinic de Barcelona in Spain. He earned a Ph.D. from the Universidad de Barcelona and an M.D. from the Universidad Autonóma de Madrid. “I am pleased to have joined Fulcrum at this key juncture,” commented Dr. Arroyo. “With the FSHD Phase 3 program that could deliver a first to market therapy for this rare and devastating disease, and a Phase 1b sickle cell disease program that has shown compelling proof-of-concept as a potential first oral fetal hemoglobin inducer, I am eager to leverage my experience to bring these two exciting medicines to patients.” Jeff Jacobs, Ph.D. – Chief Scientific Officer Dr. Jacobs has more than 25 years of experience in drug discovery and target identification, and has led, co-led and mentored multiple discovery programs from concept stage through development candidate nomination, and from investigational new drug enabling studies through to pivotal clinical trials. Dr. Jacobs was most recently Chief Scientific Officer at Goldfinch Bio, where he led the research and discovery program to help build its portfolio of therapeutics for rare kidney diseases. Before Goldfinch, Dr. Jacobs held roles of increasing responsibilities at Ardelyx, Inc., and was promoted to Chief Scientific Officer. During his time at Ardelyx, he led the discovery effort for IBSRELA®, an oral small molecule for the treatment of irritable bowel syndrome with constipation, that became its first U.S. Food and Drug Administration (FDA) approved product. Earlier, Dr. Jacobs held senior roles in development chemistry at Sunesis Pharmaceuticals, along with similar roles leading chemistry teams at Versicor, Inc. (acquired by Pfizer), and Affymax (acquired by Glaxo Wellcome). Dr. Jacobs earned a Ph.D. in bioorganic chemistry from the University of California, Berkeley and a B.S. in chemistry from Gonzaga University. Dr. Jacobs added, “Fulcrum has an impressive discovery engine that has identified novel targets across multiple therapeutic areas, and notably, led to the development of two clinical programs. I look forward to building upon Fulcrum’s expertise in target discovery to develop a robust and diverse pipeline within the company’s three core areas of focus: rare muscle, hematologic and neurologic diseases, and to delivering the company’s next IND in 2023.” About Fulcrum TherapeuticsFulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum’s two lead programs in clinical development are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy (FSHD), and FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and other hemoglobinopathies, including beta-thalassemia. Fulcrum’s proprietary product engine, FulcrumSeek™, identifies drug targets that can modulate gene expression to treat the known root cause of gene mis-expression. For more information, visit www.fulcrumtx.com and follow us on Twitter @FulcrumTx and LinkedIn. Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release are forward-looking statements, including statements regarding Dr. Jacobs role at Fulcrum, including start date; the benefits of their expertise; potential for losmapimod and ability to bring FTX-6058 to market; building on Fulcrum’s discovery efforts; and submission of next IND; among others. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with integration of new employees; Fulcrum’s ability to continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod, FTX-6058 and its other product candidates; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; realize the anticipated benefits of the strategic realignment; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Fulcrum’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties, and other important factors, in Fulcrum’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Fulcrum’s views as of the date hereof and should not be relied upon as representing Fulcrum’s views as of any date subsequent to the date hereof. Fulcrum anticipates that subsequent events and developments will cause Fulcrum’s views to change. However, while Fulcrum may elect to update these forward-looking statements at some point in the future, Fulcrum specifically disclaims any obligation to do so. ISBREYLA® is a registered trademark of Ardelyx, Inc. Contact: Investors:Stephanie AscherStern Investor Relations, Inc.stephanie.ascher@sternir.com 212-362-1200 Media:Dee SmithExecutive Director, Corporate Communications Fulcrum Therapeutics, Inc.dsmith@fulcrumtx.com202-746-1324

▎Armstrong2022年7月12日，美国疾控中心（CDC）发布了一份特别报告，新冠疫情之下超级细菌显著增加。2020年，耐药细菌感染和死亡增长了15%。从2020年3月到10月，80%的新冠住院患者都接受了抗生素治疗，2020年，以下18种耐药细菌或真菌导致的感染，大部分都有显著的增加。从古至今，感染始终是威胁人类健康和生命的主要因素。1928年青霉素的发现改变了人类对抗微生物感染的历史，青霉素的大规模生产在二战期间及以后挽救了无数人的生命。然而，几十年来抗生素的广泛使用乃至滥用，导致耐药细菌也就是超级细菌泛滥，普通抗生素已经无法应付。在这种背景下，研发针对耐药细菌的超级抗生素，成为挽救许多感染患者健康与生命的最后一道防线。在国内，普通抗生素成为“限抗令”整顿的对象，但真正存在巨大未满足需求的超级抗生素，仍存在明显的空白，许多耐药感染患者亟需特效的超级抗生素。2022年7月13日，证监会同意上海盟科药业科创板IPO，意味着超级抗生素第一股盟科药业很快将登陆科创板正式上市。盟科药业成立于2007年，创始人袁征宇博士有二十余年的抗菌药物研发经验，参与创办的抗菌新药研发公司Vicuron于2000 年在纳斯达克上市，并于2005 年6 月以19 亿美元的价格被辉瑞收购，袁征宇博士参与研发的两款抗菌新药—抗真菌药阿尼芬净和抗革兰阳性球菌感染的达巴万星现已在美国上市。盟科药业经过十余年的发展已经建立起丰富的研发管线，超级抗生素是其核心标签。康替唑胺是新一代噁唑烷酮类抗菌药，通过抑制细菌蛋白质的合成达到抗菌作用，于2021年6月获批上市，用于治疗复杂性皮肤和软组织感染。康替唑胺对中国和美国临床分离得到的革兰阳性菌，均表现出良好的抗菌活性，其中对金黄色葡萄球菌的抗菌活性优于利奈唑胺。在已完成的首个针对复杂性皮肤和软组织感染的III 期临床试验中，康替唑胺达到了和利奈唑胺相当的临床疗效。安全性方面，已完成的临床前和临床研究显示，康替唑胺未见肾毒性、肌肉毒性、骨髓抑制毒性、周围神经和视神经毒性等潜在风险，安全性优于已上市抗耐药革兰阳性菌药物。在给药途径方面，康替唑胺采取口服剂型，使用更加方便。康替唑胺不容易诱发耐药，天然耐药频率低。相比之下，利奈唑胺在连续传代试验中从第13代及开始产生耐药。MRX-4 为基于康替唑胺结构设计和开发的水溶性前药，已开发注射和口服两种剂型。MRX-4 于2019 年完成了美国II期临床试验，于2021 年7月完成了中国I期临床试验。此外公司已开展注射用MRX-4 和康替唑胺片序贯使用的全球多中心III 期临床试验，并计划其完成III 期临床试验后于美国、欧盟及中国等地申请上市。MRX-8为多黏菌素类的新型抗菌药，其阳离子及脂肪酸侧链与革兰阴性杆菌细胞外膜带阴离子的脂多糖和磷脂之间相结合，竞争性取代膜脂磷酸基团上的2价阳离子（钙离子和镁离子），从而导致细胞外膜溶解用于治疗多重耐药革兰阴性菌感染。MRX-8对美国临床分离得到的多种革兰阴性菌均表现出很好的抗菌活性。MRX-8对碳青霉烯耐药阴性菌仍然有效，这也是临床上最为严重的耐药细菌感染。安全性方面，肾毒性是限制多黏菌素类药物使用最主要的不良反应。MRX-8即是针对克服该类药物安全性问题进行的优化，经过优化得到的MRX-8对HK-2肾细胞无显著毒性，安全性显著由于多粘菌素B。盟科药业最新股权架构如下图。盟科药业此次科创板IPO拟募资12.5亿元，其中9.1亿元用于创新药研发，1.4亿元用于营销渠道升级和学术推广，2亿元用于补充流动资金。总结盟科药业所处的超级抗生素赛道属于典型的高壁垒领域，全球范围内的玩家也不多，中国更是屈指可数。盟科药业经过十余年的积累，聚焦超级抗生素的研发，研发管线覆盖革兰氏阳性菌、革兰氏阴性菌等多种主要细菌感染原。临床上，耐药细菌日益成为严重威胁感染患者健康与生命的因素，在新冠疫情中更是有快速抬头趋势。作为耐药细菌感染的最后一道防线，临床上亟需更多的超级抗生素维护患者的健康与生命。盟科药业即将登陆科创板成为中国超级抗生素第一股，其产品具有疗效与安全性的双重优势，在满足临床需求的同时也将不断开拓具有广阔前景的市场空间。同时，盟科药业在临床开发上注重中美同步推进，技术和产品管线具有全球竞争力，国际化前景值得期待。----------------------感兴趣的读者可以加入“医药笔记”知识星球。Armstrong技术全梳理系列GPRC5D靶点全梳理；CD40靶点全梳理；CD47靶点全梳理；补体靶向药物技术全梳理；补体药物：眼科治疗的重要方向；Claudin 6靶点全梳理；Claudin 18.2靶点全梳理；靶点冷暖，行业自知；中国大分子新药研发格局；被炮轰的“me too”；佐剂百年史；胰岛素百年传奇；CUSBEA：风雨四十载；中国新药研发的焦虑；中国生物医药企业的研发竞争；中国双抗竞争格局；中国ADC竞争格局；中国双抗技术全梳理；中国ADC技术全梳理；Ambrx技术全梳理；Vir Biotech技术全梳理；Immune-Onc技术全梳理；科济药业技术全梳理；恺佧生物技术全梳理；同宜医药技术全梳理；百奥赛图技术全梳理；腾盛博药技术全梳理；创胜集团技术全梳理；永泰生物技术全梳理；中国抗体技术全梳理；德琪医药技术全梳理；德琪医药技术全梳理2.0；和铂医药技术全梳理；荣昌生物技术全梳理；再鼎医药技术全梳理；药明生物技术全梳理；恒瑞医药技术全梳理；豪森药业技术全梳理；正大天晴技术全梳理；吉凯基因技术全梳理；基石药业技术全梳理；百济神州技术全梳理；百济神州技术全梳理第2版；信达生物技术全梳理；信达生物技术全梳理第2版；中山康方技术全梳理；复宏汉霖技术全梳理；先声药业技术全梳理；君实生物技术全梳理；嘉和生物技术全梳理；志道生物技术全梳理；道尔生物技术全梳理；尚健生物技术全梳理；康宁杰瑞技术全梳理；科望医药技术全梳理；科望医药技术全梳理2.0；岸迈生物技术全梳理；礼进生物技术全梳理；康桥资本技术全梳理；余国良的抗体药布局；荃信生物技术全梳理；安源医药技术全梳理；三生国健技术全梳理；仁会生物技术全梳理；乐普生物技术全梳理；同润生物技术全梳理；宜明昂科技术全梳理；派格生物技术全梳理；迈威生物技术全梳理；Momenta技术全梳理；NGM技术全梳理；普米斯生物技术全梳理；普米斯生物技术全梳理2.0；三叶草生物技术全梳理；贝达药业抗体药全梳理；泽璟制药抗体药全梳理；恒瑞医药抗体药全梳理；齐鲁制药抗体药全梳理；石药集团抗体药全梳理；豪森药业抗体药全梳理；华海药业抗体药全梳理；科伦药业抗体药全梳理；百奥泰技术全梳理；凡恩世技术全梳理。