Prostate specific membrane antigen tumour activated T-cell engager therapy (Janux Therapeutics/Merck & Co)

声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。 标准普尔500指数是衡量美国股市整体表现的一个重要指标，包含了美国股市中市值最大、流动性最好的500家上市公司，标准普尔500指数被视为全球经济的晴雨表，其涨跌不仅影响美国股市，还可能对全球股市产生联动效应。 标普500指数自2024年7月继续上涨，因为第二季度美国GDP增长超出预期。大多数投资者还预计美联储将在9月份降息。截至7月31日，标普500指数上涨15.8%，投资者希望未来几个月通胀前景将进一步改善。以下是2024年在美国主要交易所交易且市值至少为10亿美元的公司中表现最佳的6只医药股： TOP6 Arcutis Biotherapeutics Arcutis Biotherapeutics专注于治疗免疫介导的皮肤病和病症。Arcutis利用其独特的皮肤疾病开发平台，结合其皮肤病领域的专业知识，针对生物学验证的靶点构建了差异化疗法，使得其产品管线不断丰富。Arcutis正在开展的多个炎症性皮肤疾病的临床试验，包括头皮和身体银屑病、特应性皮炎、脂溢性皮炎和斑秃。 该公司的局部罗氟司特乳膏（ARQ-151）和局部罗氟司特泡沫剂（ARQ-154）已分别获得美国FDA的批准，用于治疗斑块状银屑病和脂溢性皮炎。今年7月，美国FDA再次批准0.15%的罗氟司特乳膏用于治疗6岁及以上用户的特应性皮炎；此外罗氟司特泡沫（0.3%，每日1次）治疗头皮银屑病和全身银屑病的新药补充申请（sNDA）获FDA受理。积极的头条新闻和围绕罗氟司特潜在市场扩张的乐观情绪，使Arcutis的股价今年飙升了211.8%。 TOP5 Dyne Therapeutics Dyne Therapeutics是一家针对肌肉疾病的早期临床阶段药物开发商，包括1型强直性肌营养不良（DM1）、杜氏肌营养不良（DMD）和面肩肱型肌营养不良（FSHD）。该公司的主要资产是针对DM1的DYNE-101和DYNE-251。 1月初，Dyne的股价飙升，当时公司报告了Dyne-251在适合外显子51跳跃的Duchenne患者中的DELIVER试验的积极初步临床数据。据报道，Dyne-251治疗后显示出比竞争对手Exondys 51更低的剂量和更高的疗效，后者由Sarepta Therapeutics生产。 该公司在5月份发布了DYNE-101和DYNE-251的积极1/2期临床试验积极试验数据，延续了其势头。在1/2期临床试验DELIVER中，DYNE-251（10 mg/kg）在6个月时将抗肌萎缩蛋白（dystrophin）的表达水平提高到接受标准治疗患者的10倍。多种功能性检测也显示出接受DYNE-251治疗患者的功能改善。2024年，该股上涨了222.6%。 TOP4 Janux Therapeutics Janux Therapeutics成立于2017年，是一家临床阶段的创新生物制药公司，开发针对癌症的肿瘤激活免疫疗法，通过肿瘤特异性免疫反应，在不破坏患者健康组织的情况下攻击和杀死肿瘤。Janux的目标是为癌症患者提供安全有效的药物，指导和引导患者的免疫系统根除肿瘤，同时最大限度地减少安全问题。 2月27日，Janux报告了其正在进行的治疗转移性去势耐受性前列腺癌症的JANX007和治疗晚期实体瘤的JANX008试验的令人鼓舞的安全性和有效性数据后，Janux股价暴涨229.47%，随后公司宣布公开发行普通股，以推进其内部产品管线的临床开发，并用于一般企业用途。该公司声称JANX007具有潜在的同类最佳疗效和安全性。Janux也一直是收购传闻的对象，收购可能会使股价在任何时候飙升。Janux股价今年已上涨278.3%。 TOP3 Summit Therapeutics Summit Therapeutics是一家生物制药公司，其2024年的巨大收益与SMT112（ivonescimab，依沃西单抗）有关。 2024年5月，康方生物和Summit共同宣布全球首创双特异性抗体新药依沃西单抗注射液单药对比帕博利珠单抗（Keytruda，K药）一线治疗PD-L1表达阳性（PD-L1 TPS≥1%）的局部晚期或转移性非小细胞肺癌（NSCLC）的注册性III期临床研究（HARMONi-2或AK112-303）达到无进展生存期（PFS）的主要研究终点。依沃西成为全球首个且唯一在III期单药头对头临床研究中证明疗效显著优于帕博利珠单抗的药物。 受此消息影响Summit股价大幅上涨，盘中涨幅高达272.06%。截至7月31日，这一消息使Summit股价飙升313.7%。 TOP2 Avidity Biosciences Avidity Biosciences利用其专有的抗体寡核苷酸偶联物平台(AOCs™)设计RNA疗法，将单克隆抗体的特异性与寡核苷酸疗法的精度结合起来，能够递送给以前无法达到的组织和细胞类型，以靶向以前RNA疗法无法治疗的疾病，并更有效地针对疾病的潜在遗传驱动因素。 该公司有三个临床开发项目：AOC-1001用于治疗1型强直性肌营养不良症，AOC-1044用于治疗杜氏肌营养不良和AOC-1020用于治疗面肩肱型肌营养不良病。 6月，Avidity报道AOC-1020在中期临床试验中表现出前所未有的疗效，显著下调DUX4相关基因，Avidity计划2025年上半年启动关键三期临床试验。受此消息影响Avidity Biosciences当天股价大涨33%，Avidity股价今年已经上涨了403.6%。 TOP1 Longboard Pharmaceuticals Longboard Pharmaceuticals是一家临床阶段的生物制药公司，专注于开发神经系统疾病的疗法。该公司的Bexicaserin（LP352）候选药物正在接受测试，用于治疗与发育性和癫痫性脑病相关癫痫发作，如Dravet综合征、Lennox-Gastaut综合征（LGS）、结节性硬化症（TSC）、CDKL5缺乏症（CDD）和其他癫痫疾病。 今年7月，Longboard 宣布其研发药物Bexicaserin（LP352）获得了美国FDA的突破性疗法认定，用于治疗发育性和癫痫性脑病（DEEs），适用于两岁以上患者。今年1月，Longboard公布了1b/2a期PACIFIC研究的积极顶线数据，初步结果显示达到了研究的主要疗效终点，并且安全数据表明与现有治疗方法相比具有潜在优势。基于这些结果，该公司计划开展全球3期临床试验。 受此消息影响公司股价飙升，该股从2024年开始上涨了近300%，到7月31日为止已上涨了451.2%。 参考资料：https://money-usnews-com.libproxy1.nus.edu.sg

SAN DIEGO--( BUSINESS WIRE )-- Janux Therapeutics, Inc. (Nasdaq: JANX) (Janux), a clinical-stage biopharmaceutical company developing a broad pipeline of novel immunotherapies by applying its proprietary technology to its Tumor Activated T Cell Engager (TRACTr) and Tumor Activated Immunomodulator (TRACIr) platforms, today reported financial results for the second quarter ended June 30, 2024, and provided a business update. “ We are pleased with the progress we have made this quarter, particularly with the ongoing enrollment of our PSMA-TRACTr (JANX007) and EGFR-TRACTr (JANX008) clinical trials. These advancements, along with the $7.5 million milestone payment from Merck, underscore the potential of our TRACTr and TRACIr platforms to develop transformative cancer therapies,” said David Campbell, Ph.D., President and CEO of Janux Therapeutics. “ We are also excited to welcome Eric Dobmeier and Natasha Hernday to our Board of Directors. Their extensive experience and proven leadership in the biopharmaceutical industry will be invaluable as we continue to advance our pipeline and strategic goals.” RECENT BUSINESS HIGHLIGHTS AND FUTURE MILESTONES: The company continues to enroll patients in the first-in-human Phase 1 clinical trial of JANX007 in mCRPC (NCT05519449) and JANX008 in advanced or metastatic solid tumors (NCT05783622). Janux anticipates providing an update on JANX007 data and doses selected for expansion cohorts in the second half of 2024. An update on JANX008 data is expected in 2025. Board strengthened with key appointments. Eric Dobmeier, J.D., has more than 20 years of experience in the biotechnology industry as both an executive and board member. Most recently, he was the President and CEO of Chinook Therapeutics, where he led the company through multiple strategic growth initiatives ultimately leading to its acquisition by Novartis in 2023 for $3.5 billion. Prior to Chinook, Mr. Dobmeier spent 16 years in a series of positions of increasing responsibility at Seattle Genetics, including Chief Operating Officer, during the company’s growth from 60 to 1,200 employees, from a market cap of $150 million to over $8 billion and through its transition to a commercial company with FDA approval and launch of Adcetris, a novel lymphoma drug. During his career, Mr. Dobmeier has been directly involved in raising more than $2 billion in equity capital and led negotiation of many corporate alliances with leading biotechnology and pharmaceutical companies. He is currently a venture partner at Samsara Biocapital and serves on the boards of directors of Structure Therapeutics and Abdera Therapeutics. Natasha Hernday previously served as Chief Business Officer and a member of the Executive Committee at Seagen Inc. (formerly Seattle Genetics) and was pivotal in driving business development, including alliance management, strategic partnerships, mergers, and acquisitions. Ms. Hernday led the acquisition of Cascadian Therapeutics in 2018 for approximately $614 million and a global strategic oncology collaboration with Merck in 2020 for over $1.5 billion in upfront cash and equity. Ms. Hernday’s strategic insight and successful track record in transactions culminated in Seagen's acquisition by Pfizer in 2023 for $43 billion. Prior to her role at Seagen, Ms. Hernday spent 16 years at Amgen, where she began her career in discovery research, then held various leadership positions in corporate development and corporate strategy, including as Director, Mergers & Acquisitions and as Director, Out-Partnering, playing a key role in numerous high-value transactions. Additionally, Ms. Hernday served on the Board of Alpine Immune Sciences, which was acquired by Vertex Pharmaceuticals in 2024 for $4.9 billion. Janux received a $7.5 million payment from Merck triggered by the achievement of an undisclosed development milestone for the first collaboration target under the companies’ 2020 Research Collaboration and Exclusive License Agreement. SECOND QUARTER 2024 FINANCIAL RESULTS: Cash and cash equivalents and short-term investments: As of June 30, 2024, Janux reported cash and cash equivalents and short-term investments of $646.3 million compared to $344.0 million at December 31, 2023. Research and development expenses: Research and development expenses for the quarter ended June 30, 2024, were $14.9 million compared to $14.9 million for the comparable period in 2023. General and administrative expenses: General and administrative expenses for the quarter ended June 30, 2024, were $7.8 million compared to $6.9 million for the comparable period in 2023. Net loss: For the quarter ended June 30, 2024, Janux reported a net loss of $6.0 million compared to a net loss of $17.5 million for the comparable period in 2023. Janux’s TRACTr and TRACIr Pipeline Janux’s first clinical candidate, JANX007, is a TRACTr that targets PSMA and is being investigated in a Phase 1 clinical trial in adult subjects with metastatic castration-resistant prostate cancer (mCRPC). Janux’s second clinical candidate, JANX008, is a TRACTr that targets EGFR and is being studied in a Phase 1 clinical trial for the treatment of multiple solid cancers including colorectal cancer, squamous cell carcinoma of the head and neck, non-small cell lung cancer, and renal cell carcinoma. We are also generating a number of additional TRACTr and TRACIr programs for potential future development, some of which are at development candidate stage or later. We are currently assessing priorities in our preclinical pipeline. About Janux Therapeutics Janux is a clinical-stage biopharmaceutical company developing tumor-activated immunotherapies for cancer. Janux’s proprietary technology enabled the development of two distinct bispecific platforms: Tumor Activated T Cell Engagers (TRACTr) and Tumor Activated Immunomodulators (TRACIr). The goal of both platforms is to provide cancer patients with safe and effective therapeutics that direct and guide their immune system to eradicate tumors while minimizing safety concerns. Janux is currently developing a broad pipeline of TRACTr and TRACIr therapeutics directed at several targets to treat solid tumors. Janux has two TRACTr therapeutic candidates in clinical trials, the first targeting PSMA is in development for prostate cancer, and the second targeting EGFR is being developed for colorectal, lung, head and neck, and renal cancers. For more information, please visit www.januxrx.com and follow us on 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, Janux’s ability to bring new treatments to cancer patients in need, expectations regarding the timing, scope and results of Janux’s development activities, including its ongoing and planned preclinical studies and clinical trials, the timing of and plans for regulatory filings, the potential benefits of Janux’s product candidates and platform technologies, expectations regarding the use of Janux’s platform technologies to generate novel product candidates and the strength of Janux’s balance sheet and the adequacy of cash on hand. Factors that may cause actual results to differ materially include the risk that compounds that appear promising in early research do not demonstrate safety and/or efficacy in later preclinical studies or clinical trials, the risk that Janux may not obtain approval to market its product candidates, uncertainties associated with performing clinical trials, regulatory filings and applications, 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 Janux faces, please refer to Janux’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 Janux assumes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. Janux Therapeutics, Inc. Condensed Balance Sheets (in thousands) June 30, 2024 December 31, 2023 Assets (unaudited) Current assets: Cash and cash equivalents $ 14,662 $ 19,205 Accounts receivable 7,500 — Short-term investments 631,631 324,823 Prepaid expenses and other current assets 8,146 5,213 Total current assets 661,939 349,241 Restricted cash 816 816 Property and equipment, net 5,940 7,003 Operating lease right-of-use assets 20,077 20,838 Other long-term assets 2,640 2,509 Total assets $ 691,412 $ 380,407 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $ 1,253 $ 2,424 Accrued expenses 8,568 7,387 Current portion of deferred revenue 94 1,705 Current portion of operating lease liabilities 1,630 1,517 Total current liabilities 11,545 13,033 Operating lease liabilities, net of current portion 22,190 23,025 Total liabilities 33,735 36,058 Total stockholders’ equity 657,677 344,349 Total liabilities and stockholders’ equity $ 691,412 $ 380,407 Janux Therapeutics, Inc. Unaudited Condensed Statements of Operations and Comprehensive Loss (in thousands, except share and per share data) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Collaboration revenue $ 8,897 $ 1,057 $ 10,149 $ 3,105 Operating expenses: Research and development 14,898 14,924 28,968 30,789 General and administrative 7,821 6,881 15,164 13,345 Total operating expenses 22,719 21,805 44,132 44,134 Loss from operations (13,822 ) (20,748 ) (33,983 ) (41,029 ) Total other income 7,863 3,240 13,264 6,062 Net loss $ (5,959 ) $ (17,508 ) $ (20,719 ) $ (34,967 ) Other comprehensive gain (loss): Unrealized gain (loss) on available-for-sale securities, net (1,092 ) (321 ) (2,281 ) 475 Comprehensive loss $ (7,051 ) $ (17,829 ) $ (23,000 ) $ (34,492 ) Net loss per common share, basic and diluted $ (0.11 ) $ (0.42 ) $ (0.40 ) $ (0.84 ) Weighted-average shares of common stock outstanding, basic and diluted 54,451,666 41,836,238 51,750,690 41,800,304