更新于：2024-11-01
Mosliciguat
更新于：2024-11-01
概要
基本信息
结构
分子式C41H36ClF3N2O5 |
InChIKeyHFCWZVGKWGOBKV-QNGWXLTQSA-N |
CAS号2231749-54-3 |
关联
2
项与 Mosliciguat 相关的临床试验A Phase 2, Randomized, Placebo-Controlled Trial to Assess the Efficacy and Safety of Mosliciguat in Participants With Pulmonary Hypertension Associated With Interstitial Lung Disease
This is a Phase 2, randomized, double-blind, placebo-controlled, multi-center clinical study to evaluate the safety and efficacy of inhaled mosliciguat in participants with pulmonary hypertension associated with interstitial lung disease (PH-ILD).
开始日期2024-11-01 |
申办/合作机构 |
A Non-randomized Two Part Multi-center, Open-label, Single Dose Trial With an Escalation Part in Untreated Pulmonary Arterial Hypertension (PAH) and Chronic Thromboembolic Pulmonary Hypertension (CTEPH) Patients (Part A), Followed by a Parallel-group Part in Untreated and Pre-treated Patients With PAH and CTEPH (Part B) to Investigate the Safety, Tolerability and Pharmacodynamics of Inhaled BAY1237592
In this trial the effects of the inhaled drug BAY1237592 will be studied in patients with high blood pressure in the pulmonary blood vessels due to Pulmonary Arterial Hypertension (PAH) and due to Chronic Thromboembolic Pulmonary Hypertension (CTEPH). Pulmonary hypertension is characterized by the elevation of pressure in the pulmonary arteries (PAP) and of the pulmonary vascular resistance (PVR) leading to increased workload of the right chamber of the heart to eject blood against this elevated resistance. The goal of this study is to measure the safety and tolerability of the drug as well as the reduction of the PVR at different doses
In Part A patients without specific treatment for PH (untreated patients) will be tested. In Part B also patients stably pre-treated with specific PH drugs will be studied in combination with the new inhaled drug
In Part A patients without specific treatment for PH (untreated patients) will be tested. In Part B also patients stably pre-treated with specific PH drugs will be studied in combination with the new inhaled drug
开始日期2019-01-21 |
申办/合作机构 |
100 项与 Mosliciguat 相关的临床结果
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100 项与 Mosliciguat 相关的转化医学
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100 项与 Mosliciguat 相关的专利（医药）
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1
项与 Mosliciguat 相关的文献（医药）Respiratory Research
Inhaled mosliciguat (BAY 1237592): targeting pulmonary vasculature via activating apo-sGC
Article
作者: Hüser, Jörg ; Truebel, Hubert K F ; Marquardt, Tobias ; Kopf, Johannes ; Weimann, Gerrit ; Dietz, Lisa ; Delbeck, Martina ; Nagelschmitz, Johannes ; Mondritzki, Thomas ; Becker-Pelster, Eva M ; Kraemer, Thomas ; Nikkho, Sylvia M ; Wunder, Frank ; Tinel, Hanna ; Pires, Philippe V ; Hahn, Michael G ; Schuhmacher, Joachim ; Sandner, Peter ; Stasch, Johannes-Peter
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项与 Mosliciguat 相关的新闻（医药）2024-10-28
The acquisition expands Organon’s dermatology capabilities with a nonbiologic, non-steroidal topical treatment in the U.S.BASEL, Switzerland and LONDON and NEW YORK, Oct. 28, 2024 (GLOBE NEWSWIRE) -- Organon (NYSE: OGN), a global healthcare company with a mission to improve the health of women throughout their lives, today announced the successful completion of its acquisition of Dermavant Sciences Ltd. from Roivant (NASDAQ: ROIV). Dermavant is a company dedicated to developing and commercializing innovative therapeutic solutions in immuno-dermatology. Please see our prior announcement for a summary of the transaction terms. VTAMA® (tapinarof) cream, 1%, is a novel nonbiologic, non-steroidal topical therapy approved by the U.S. Food and Drug Administration (FDA) for treatment of mild, moderate, and severe plaque psoriasis in adults with no safety label warnings or precautions and without restrictions on location and duration of use or body surface area. The FDA is reviewing a supplemental New Drug Application (sNDA) for VTAMA cream as a potential treatment for atopic dermatitis (AD) in adults and children two years of age and older, with Prescription Drug User Fee Act (PDUFA) action expected in the fourth quarter of calendar year 2024. Plaque psoriasis and atopic dermatitis-commonly known as eczema-are common chronic inflammatory skin diseases affecting millions of people in the U.S. and around the globe.1,2 Psoriasis presents a significant impact to quality of life and atopic dermatitis is associated with a higher disease burden for women compared to men.3,4“The future of dermatology depends on innovative treatments like VTAMA, and Organon’s acquisition of Dermavant allows us to further expand our existing portfolio of established brands and biosimilar dermatology treatments,” said Kevin Ali, Chief Executive Officer of Organon. “Integrating the expertise of Dermavant into Organon’s U.S. organization marks the beginning of a new chapter in dermatology. We are excited to bring this nonbiologic non-steroidal topical option to the millions of patients suffering from a chronic skin condition like plaque psoriasis and, potentially in the future, atopic dermatitis.” “I would like to thank Kevin and the entire Organon team for their partnership in the acquisition of Dermavant,” said Mayukh Sukhatme, MD, President and Chief Investment Officer of Roivant. “This deal represents a true win-win outcome for Organon and Roivant in our mutual goal to address patient needs and is emblematic of Roivant’s ability to form non-traditional, value-enhancing collaborations on important medicines. We believe that Organon’s strong global commercial footprint will maximize the impact of VTAMA for patients globally, and we are excited to continue to share meaningfully in the success of VTAMA along the way.” About VTAMA® (tapinarof) cream, 1%VTAMA cream is a non-steroidal once-daily topical treatment. The safety and effectiveness of VTAMA cream was evaluated in randomized, double-blind, vehicle-controlled trials, PSOARING-1 and 2 for plaque psoriasis. The safety and efficacy of VTAMA for the treatment of atopic dermatitis was also evaluated in ADORING-1 and ADORING-2 Phase III clinical studies and is currently under review with the FDA. Important Safety Information Indication: VTAMA® (tapinarof) cream, 1% is an aryl hydrocarbon receptor agonist indicated for the topical treatment of plaque psoriasis in adults. VTAMA cream is for use on the skin (topical) only. Do not use VTAMA cream in your eyes, mouth, or vagina. Adverse Events: The most common adverse reactions (incidence ≥ 1%) in subjects treated with VTAMA cream were folliculitis (red raised bumps around the hair pores), nasopharyngitis (pain or swelling in the nose and throat), contact dermatitis (skin rash or irritation, including itching and redness, peeling, burning, or stinging), headache, pruritus (itching), and influenza (flu). You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. See full Prescribing Information and Patient Information. About Organon Organon is an independent global healthcare company with a mission to help improve the health of women throughout their lives. Organon’s diverse portfolio offers more than 60 medicines and products in women’s health, biosimilars, and a large franchise of established medicines across a range of therapeutic areas. In addition to Organon’s current products, the company invests in innovative solutions and research to drive future growth opportunities in women’s health and biosimilars. In addition, Organon is pursuing opportunities to collaborate with biopharmaceutical partners and innovators looking to commercialize their products by leveraging its scale and agile presence in fast growing international markets. Organon has geographic scope with significant reach, world-class commercial capabilities, and approximately 10,000 employees with headquarters located in Jersey City, New Jersey. For more information, visit http://www.organon.com and connect with us on LinkedIn, Instagram, X (formerly known as Twitter) and Facebook. Cautionary Note Regarding Forward-Looking StatementsExcept for historical information, this press release includes “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995, including, but not limited to, statements about management’s expectations about Organon’s acquisition of Dermavant (including statements regarding the timing, benefits, and financial impact of such acquisition), potential regulatory approvals and other actions relating to VTAMA (including the expected timeframe thereof), and Organon’s expected financial results and condition. Forward-looking statements may be identified by words such as “pursuing,” “foresees,” “future,” “potential,” “potentially,” “expected,” “expects,” “will” or words of similar meaning. These statements are based upon the current beliefs and expectations of Organon’s management and are subject to significant risks and uncertainties. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward- looking statements. Risks and uncertainties include, but are not limited to, an inability to execute on our business development strategy or realize the benefits of our acquisition of Dermavant or any other planned acquisitions; weakening of economic conditions that could adversely affect the level of demand for Dermavant’s products; the risk that the business will not be integrated successfully; risks related to the ability to realize the anticipated benefits of the acquisition, including the possibility that the expected benefits from the acquisition will not be realized or will not be realized within the expected time period; the ability to retain key personnel; unknown liabilities; the risk of litigation and/or regulatory actions related to the completed acquisition; pricing pressures globally, including rules and practices of managed care groups, judicial decisions and governmental laws and regulations related to Medicare, Medicaid and health care reform, pharmaceutical reimbursement and pricing in general; an inability to fully execute on Organon’s product development and commercialization plans in the United States, Europe, and elsewhere internationally; an inability to adapt to the industry-wide trend toward highly discounted channels; changes in tax laws or other tax guidance which could adversely affect Organon’s cash tax liability, effective tax rates, and results of operations and lead to greater audit scrutiny; expanded brand and class competition in the markets in which Organon operates; and governmental initiatives that adversely impact Organon’s marketing activities. Organon undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in Organon’s filings with the Securities and Exchange Commission (“SEC”), including Organon’s most recent Annual Report on Form 10-K and subsequent SEC filings, available at the SEC’s Internet site www.sec.gov. References and links to websites have been provided for convenience, and the information contained on any such website is not a part of, or incorporated by reference into, this press release. Organon is not responsible for the contents of third-party websites About Roivant Roivant is a biopharmaceutical company that aims to improve the lives of patients by accelerating the development and commercialization of medicines that matter. Roivant’s pipeline includes IMVT-1402 and batoclimab, fully human monoclonal antibodies targeting FcRn in development across several IgG-mediated autoimmune indications; brepocitinib, a potent small molecule inhibitor of TYK2 and JAK1 in development for the treatment of dermatomyositis and non-infectious uveitis; and mosliciguat, an inhaled soluble sGC activator in development for pulmonary hypertension associated with interstitial lung disease, in addition to other clinical stage molecules. We advance our pipeline by creating nimble subsidiaries or “Vants” to develop and commercialize our medicines and technologies. Beyond therapeutics, Roivant also incubates discovery-stage companies and health technology startups complementary to its biopharmaceutical business. For more information, www.roivant.com. Roivant Forward-Looking Statements This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intends,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “would” and variations of such words or similar expressions. The words may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. Our forward-looking statements include, but are not limited to, statements regarding our or our management team’s expectations, hopes, beliefs, intentions or strategies regarding the future, and statements that are not historical facts, including statements about the clinical and therapeutic potential of our product candidates, the availability and success of topline results from our ongoing clinical trials and any commercial potential of our product candidates following applicable regulatory approvals. In addition, any statements that refer to projections, forecasts or other characterizations of future events, results or circumstances, including any underlying assumptions, are forward-looking statements. Actual results may differ materially from those contemplated in these statements due to a variety of risks, uncertainties and other factors, including (i) the possibility that the transaction may involve unexpected costs or liabilities, (ii) the risk that disruptions from the transaction will harm relationships with employees, customers and suppliers and other business partners or (iii) the risk that the achievement of the specified milestones or royalties described in the definitive agreement for the transaction may take longer to achieve than expected or may never be achieved and the resulting contingent milestone payments or royalties may never be realized. Although we believe that our plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, those risks set forth in the Risk Factors section of our filings with the U.S. Securities and Exchange Commission. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of our management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. Contacts: InvestorsKeyur Parekh keyur.parekh@roivant.com MediaStephanie Leestephanie.lee@roivant.com 1 Armstrong, A., Mehta, M., Schupp, C., Gondo, C., Bell, S., Griffiths, C. Psoriasis Prevalence in Adults in the United States. JAMA Dermatol. 2021 Aug; 157(8):1-7. doi:10.1001/jamadermatol.2021.2007 2 Eczema Prevalence. National Eczema Foundation. Eczema Prevalence, Quality of Life and Economic Impact.3 Carole, G., Corsin, S., Meienberger, N., Valeska Maul, L., Maul, J-T. The Impact of Gender and Sex in Psoriasis: What to be Aware of When Treating Women with Psoriasis. Int J Women’s Dermatol. 2022 Jun; 8(2): e010. doi: 10.1097/JW9.0000000000000010. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9112394/ 4 Urban, K., Chu, S., Giesey, RL., Mehrmal, S., Uppal, P., Nedley, N., Delost, GR. The Global, Regional, and National Burden of Atopic Dermatitis in 195 Countries and Territories: An ecological study from the Global Burden of Disease Study 2017. 2021 Mar; 2: 12-18. JAAD International. doi: https://doi.org/10.1016%2Fj.jdin.2020.10.002. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8362298/
临床3期并购上市批准临床结果
2024-09-15
·药明康德
▎药明康德内容团队编辑
本期看点
1. 口服人类表皮生长因子受体2（HER2）酪氨酸激酶抑制剂（TKI）zongertinib治疗非小细胞肺癌（NSCLC）患者在一项早期临床试验中获积极结果，94%患者的肿瘤有缩小。
2. HER2靶向疗法BAY 2927088在一项1/2期临床试验中治疗携带HER2 YVMA插入突变的晚期NSCLC患者，客观缓解率（ORR）达90%。
3. 口服靶向抗癌化合物ibrilatazar联用化疗（紫杉醇/卡铂）在一项1/2期临床试验中使子宫内膜癌患者的无进展生存期（PFS）相比历史对照组延长了40%。
药明康德内容团队整理
Zongertinib：公布1b期临床试验的初步数据
勃林格殷格翰（Boehringer Ingelheim）公布了旗下口服HER2靶向疗法zongertinib（BI 1810631）在NSCLC患者中的最新积极临床结果。Zongertinib不与野生型表皮生长因子受体（EGFR）结合，进而相关毒性较低。Zongertinib于2023年获得美国FDA授予快速通道资格，并随后于2024年获得美国FDA和中国国家药监局药品审评中心（CDE）授予的突破性疗法认定，用于治疗曾接受过全身性疗法、携带HER2激活突变的晚期NSCLC成人患者。
此次公布的结果显示，截至2024年5月，共132例患者每天接受一次120 mg/240 mg zongertinib治疗（n=75/n=57）。经盲法独立中央审评（BICR）评估，试验达到主要终点，患者的确认ORR为66.7%（97.5% CI：53.8–77.5，p<0.0001）。在所有剂量的患者中，94%的患者观察到任何程度的肿瘤缩小。
此外，数据还显示，zongertinib具有初步的脑活性。根据BICR评估的RANO-BM（神经肿瘤脑转移瘤的疗效评估），33%（120 mg，n=27）和40%（240 mg，n=25）的无症状脑转移患者达成确认的客观缓解，疾病控制率（DCR）分别为74%和92%。Zongertinib在120 mg和240 mg剂量下的耐受性通常良好，没有因治疗导致的死亡，且导致剂量减少（11%）和停药（3%）的不良事件发生率低。
BAY 2927088：公布1/2期临床试验数据
拜耳（Bayer）公布了1/2期SOHO-01研究扩展部分的积极结果。该研究评估了其口服、可逆的HER2靶向疗法BAY 2927088在携带HER2变异的晚期NSCLC患者中的安全性和初步疗效。入组患者在接受≥1种晚期疾病全身治疗后病情出现进展，但未曾接受过HER2靶向治疗。BAY 2927088能够强效抑制突变的HER2，包括HER2外显子20插入突变和HER2点突变，同时也能抑制EGFR，对突变型EGFR具有高选择性，且不和野生型EGFR相结合。美国FDA和中国药品审评中心于2024年授予BAY 2927088突破性疗法认定，用于治疗携带HER2激活突变且曾接受过全身治疗的不可切除或转移性NSCLC成年患者。
此次公布的结果显示，43名可评估疗效患者的确认ORR为72.1%，1名（2.3%）患者实现完全缓解（CR）。中位缓解持续时间（DOR）和PFS分别为8.7个月和7.5个月。在携带HER2 YVMA插入突变（最常见的变异）的患者中，ORR为90.0%，DOR为9.7个月，PFS为9.9个月。此外，BAY 2927088的安全性是可控的，并且与之前的报告一致。
Ibrilatazar（ABTL0812）：公布1/2期临床试验数据
AbilityPharma公司公布了ibrilatazar联用化疗（紫杉醇/卡铂）治疗晚期或复发性子宫内膜癌患者的1/2期临床试验ENDOLUNG的最终数据。Ibrilatazar是一种潜在“first-in-class”、差异化的口服靶向抗癌化合物，通过诱导内质网应激和抑制PI3K/Akt/mTOR通路来引发自噬。在临床试验中，ibrilatazar对子宫内膜癌和肺癌患者显示出临床益处。此外，它在包括肺癌、子宫内膜癌、胰腺癌、神经母细胞瘤和胶质母细胞瘤等癌症类型的动物模型中展示了强有力的临床前概念验证。
此次公布的结果显示，ibrilatazar联用化疗的总缓解率为65.8%，其中13.2%的患者达到CR，52.6%的患者达到部分缓解（PR），中位DOR为7.4个月。中位PFS为9.8个月，相比历史对照组延长了40%。中位总生存期（OS）为23.6个月。这些结果表明，接受ibrilatazar联用化疗治疗的患者的ORR、PFS和OS相较于历史对照组（分别为51%、7.1个月和20.4个月）均有所提高。
安全性方面，ibrilatazar联用化疗治疗显示出良好的安全性，未引入与化疗相关的重大不良事件，其安全性与历史对照组一致。药代动力学参数与临床前试验中观察到的靶点作用相符，血液药效学生物标志物表明治疗开始后至少28天内靶点调节持续存在。
RLY-2608：公布首个人体试验数据
Relay Therapeutics公司公布了其在研疗法RLY-2608的首个人体试验的积极中期数据。RLY-2608是Relay Therapeutics开发针对PI3Kα突变体的选择性抑制剂。该激酶是所有癌症中最常出现突变的激酶之一，约在14%的实体瘤患者检测出PI3Kα的致癌突变。传统上，PI3Kα抑制剂的开发聚焦于蛋白的活性位点。然而，这些抑制剂缺乏对突变型PI3Kα的选择性，导致治疗效果欠佳。为了增强PI3Kα抑制剂的选择性，Relay Therapeutics解析了PI3Kα的全长蛋白冷冻电镜结构，以阐明野生型和突变型PI3Kα的构象差异，并利用这些见解支持RLY-2608的设计。
此次公布的数据显示，此前接受过大量治疗，携带PI3Kα突变的HR阳性、HER2阴性局部晚期或转移性乳腺癌患者在接受RLY-2608（600 mg）联合fulvestrant治疗后，表现出具有临床意义的PFS。中位随访时间为7.5个月时，所有携带PI3Kα突变患者的中位PFS为9.2个月。在30名具有可测量疾病的患者中，1/3的患者达到PR，ORR为33%。新闻稿指出，RLY-2608是首个针对PI3Kα，具有突变体和异构体（isoform）选择性的别构抑制剂。
▲RLY-2608让73%患者的肿瘤缩小（图片来源：Relay Therapeutics公司官网）
Mosliciguat：公布1b期临床试验数据
Roivant公司公布了其肺动脉高压（PH）疗法mosliciguat的1b期概念验证试验的结果。Mosliciguat是一种每日给药一次、潜在"first-in-class"和“best-in-class”的吸入性可溶性鸟苷酸环化酶（sGC）激动剂，可靶向递送至肺部。sGC是一种一氧化氮（NO）/环磷酸鸟苷（cGMP）信号通路中的关键酶，可催化cGMP生成，从而增加血管扩张、减少炎症和细胞凋亡、逆转血管重塑和抗纤维化作用。与需要减少血红素和NO才能对sGC产生作用的sGC激动剂不同，mosliciguat是一种独立于血红素和NO的sGC激活剂。这使mosliciguat可能在典型PH患者体内高氧化环境中保持有效性。该疗法目前正在开发用于治疗与间质性肺病（PH-ILD）相关的肺动脉高压。
此次公布的结果显示，1.0、2.0和4.0毫克mosliciguat分别使患者的肺血管阻力（PVR）相对于基线的平均最大峰值降低达25.9%、38.1%和36.3%，超过预定主要结果≥20%降幅的阈值。此外，该疗法的耐受性良好，治疗引起的不良事件发生率低。根据新闻稿，这是迄今为止PH相关试验中观察到PVR的最大降低幅度之一。Mosliciguat的全球2期PHocus研究即将启动。
CT-996：公布1期临床试验数据
罗氏（Roche）公布了其每日一次的口服胰高血糖素样肽-1（GLP-1）受体小分子激动剂CT-996的1期临床试验结果。罗氏在去年12月与Carmot Therapeutics达成合并协议，斥资约31亿美元收购了Carmot公司，囊获包括CT-996在内的多款处于临床开发阶段的皮下和口服肠促胰岛素疗法。
此次公布的结果显示，CT-996在未患有2型糖尿病的肥胖人群中，在4周内将受试者体重减轻7.3%（约7公斤），安慰剂同期将体重降低1.2%。与安慰剂相比，CT-996提供了具有临床意义的体重减轻。安全性方面，所有的治疗伴发不良事件（TEAE）均为轻度或中度，没有发现3级以上TEAE。没有出现与在研药物相关的停止用药。最常见的TEAE为胃肠道相关不良事件，与其它肠促胰岛素疗法类似。接受更高剂量治疗的参与者TEAE更多，不过更缓慢的剂量滴定可能改善耐受性。基于这些积极结果，该公司计划在2025年启动2期临床试验。
▲CT-996的减重效果（图片来源：参考资料[5]）
Radiprodil：公布1b期临床试验数据
GRIN Therapeutics公司公布了其正在进行的全球1b期开放标签临床试验的初步结果。该试验的两个部分正在评估radiprodil的安全性、耐受性、药代动力学以及疗效。Radiprodil是一种在研选择性强效N-甲基-D-天冬氨酸受体亚基2B（GluN2B）的负变构调节剂，用于治疗GRIN相关神经发育障碍。GRIN相关神经发育障碍是一组由GRIN基因突变引起的罕见儿科神经发育障碍，目前尚无批准的疗法。
此次公布的结果显示，radiprodil在具有GRIN基因功能增加突变的患者中，无论基线时是否存在可数的运动性癫痫发作（CMS），均表现出良好的耐受性。具有CMS患者的中位癫痫发作频率与基线相比降低了86%，超过了背景抗癫痫药物，其中71%的患者癫痫发作频率降低了50%以上。这些结果预计将支持GRIN Therapeutics与监管机构的讨论，以推动radiprodil进入3期关键性试验。
Elraglusib：公布1/2期临床试验的新数据
Actuate Therapeutics公司更新了elraglusib用于治疗复发/难治性尤文氏肉瘤（r/r EWS）的1/2期临床试验进展。该研究旨在评估elraglusib在复发/难治性恶性肿瘤儿科患者中的安全性和疗效，涵盖EWS及EWS相关的小圆细胞肉瘤。所有患者均接受了elraglusib与环磷酰胺/拓扑替康的联合治疗。
此次公布的结果显示，在首批8名复发/难治性EWS及相关肉瘤患者中，两名患者正在经历持续的CR，两名患者实现疾病稳定（SD），1名患者取得了PR，肿瘤缩小了52%。Elraglusib展现了抗肿瘤活性，DCR为62.5%。该研究招募正在进行，预计2025年上半年有望公布顶线数据。
▲Elraglusib联用环磷酰胺/拓扑替康的1/2期临床试验结果（图片来源：参考资料[6]）
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参考资料（可上下滑动查看）
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[9] Seres Therapeutics Reports SER-155 Phase 1b Placebo-Controlled Cohort 2 Study Safety and Clinical Results in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplant (allo-HSCT). Retrieved September 13, 2024, from https://www.globenewswire.com/news-release/2024/09/12/2945049/0/en/Seres-Therapeutics-Reports-SER-155-Phase-1b-Placebo-Controlled-Cohort-2-Study-Safety-and-Clinical-Results-in-Patients-Undergoing-Allogeneic-Hematopoietic-Stem-Cell-Transplant-allo-.html
[10] Vironexis Biotherapeutics Launches with FDA Clearance of IND Application for First-Ever Clinical Trial of an AAV-delivered Cancer Immunotherapy. Retrieved September 13, 2024, from https://www.globenewswire.com/news-release/2024/09/12/2945063/0/en/Vironexis-Biotherapeutics-Launches-with-FDA-Clearance-of-IND-Application-for-First-Ever-Clinical-Trial-of-an-AAV-delivered-Cancer-Immunotherapy.html
[11] Cytonics Announces Completion Of Enrollment For Phase 1 Clinical Study Evaluating CYT-108, A Novel Recombinant Protease Inhibitor, In Patients Suffering From Osteoarthritis Of The Knee. Retrieved September 13, 2024, from https://www.prnewswire.com/news-releases/cytonics-announces-completion-of-enrollment-for-phase-1-clinical-study-evaluating-cyt-108-a-novel-recombinant-protease-inhibitor-in-patients-suffering-from-osteoarthritis-of-the-knee-302239925.html
[12] Phanes Therapeutics Announces First Patient Dosed in Clinical Study of PT886 in Combination with Chemotherapy. Retrieved September 13, 2024, from https://www.prnewswire.com/news-releases/phanes-therapeutics-announces-first-patient-dosed-in-clinical-study-of-pt886-in-combination-with-chemotherapy-302242421.html
[13] Upstream Bio Announces Oral Presentation of New Data from a Phase 1b Multiple Ascending Dose Trial of Verekitug (UPB-101) in Adults with Asthma at the European Respiratory Society Congress. Retrieved September 13, 2024, from https://www.globenewswire.com/news-release/2024/09/09/2942765/0/en/Upstream-Bio-Announces-Oral-Presentation-of-New-Data-from-a-Phase-1b-Multiple-Ascending-Dose-Trial-of-Verekitug-UPB-101-in-Adults-with-Asthma-at-the-European-Respiratory-Society-Co.html
[14] CytomX Therapeutics Announces First Patient Dosed with CX-801, a Dually-Masked Interferon-Alpha 2b PROBODY®, in a Phase 1 Study in Patients with Solid Tumors. Retrieved September 13, 2024, from https://www.globenewswire.com/news-release/2024/09/09/2942754/37704/en/CytomX-Therapeutics-Announces-First-Patient-Dosed-with-CX-801-a-Dually-Masked-Interferon-Alpha-2b-PROBODY-in-a-Phase-1-Study-in-Patients-with-Solid-Tumors.html#:~:text=SOUTH%20SAN%20FRANCISCO%2C%20Calif.%2C%20Sept.%2009%2C%202024%20%28GLOBE,1%20study%20%28NCT06462794%29%20in%20patients%20with%20solid%20tumors.
[15] ORYZON announces first patient dosed in an Investigator-initiated Phase Ib study of iadademstat in first-line acute myeloid leukemia. Retrieved September 13, 2024, from https://www.oryzon.com/en/news-events/news/oryzon-announces-first-patient-dosed-investigator-initiated-phase-ib-study
[16] PolTREG Treg cell therapy for patients with type-1 diabetes shows long-term clinical remission and insulin independence. Retrieved September 13, 2024, from https://poltreg.com/poltreg-treg-cell-therapy-for-patients-with-type-1-diabetes-shows-long-term-clinical-remission-and-insulin-independence/
[17] Centessa Announces Positive Interim Phase 1 Clinical Data with its Novel Orexin Receptor 2 (OX2R) Agonist, ORX750, in Acutely Sleep-Deprived Healthy Volunteers. Retrieved September 13, 2024, from https://investors.centessa.com/news-releases/news-release-details/centessa-announces-positive-interim-phase-1-clinical-data-its
[18] Cytokinetics Announces Data From Phase 1 Study of CK-4021586. Retrieved September 13, 2024, from https://www.globenewswire.com/news-release/2024/09/09/2942724/35409/en/Cytokinetics-Announces-Data-From-Phase-1-Study-of-CK-4021586.html
[19] Antennova to Present Latest Data of ATN-037 in a Mini Oral Presentation at ESMO Congress. Retrieved September 13, 2024, from https://www.prnewswire.com/news-releases/antennova-to-present-latest-data-of-atn-037-in-a-mini-oral-presentation-at-esmo-congress-2024-302240448.html
[20] TILT Biotherapeutics Presents Clinical Data on TILT-123 After Fully Intravenous Delivery Regimen at ESMO 2024. Retrieved September 13, 2024, from https://www.globenewswire.com/news-release/2024/09/09/2942525/0/en/TILT-Biotherapeutics-Presents-Clinical-Data-on-TILT-123-After-Fully-Intravenous-Delivery-Regimen-at-ESMO-2024.html
[21] Terns Pharmaceuticals Announces Positive Phase 1 Clinical Trial Results with TERN-601 Once-Daily Oral GLP-1R Agonist for the Treatment of Obesity. Retrieved September 9, 2024 from https://ir.ternspharma.com/news-releases/news-release-details/terns-pharmaceuticals-announces-positive-phase-1-clinical-trial
[22] Zealand Pharma announces positive topline results from 13-week Phase 1b multiple ascending dose clinical trial with GLP-1/GLP-2 receptor dual agonist dapiglutide. Retrieved September 9, 2024 from https://www.globenewswire.com/news-release/2024/09/09/2943004/0/en/Zealand-Pharma-announces-positive-topline-results-from-13-week-Phase-1b-multiple-ascending-dose-clinical-trial-with-GLP-1-GLP-2-receptor-dual-agonist-dapiglutide.html
[23] BridgeBio Receives FDA’s Regenerative Medicine Advanced Therapy (RMAT) Designation for BBP-812 Canavan Disease Gene Therapy Program. Retrieved September 11, 2024 from https://www.globenewswire.com/news-release/2024/09/10/2944157/0/en/BridgeBio-Receives-FDA-s-Regenerative-Medicine-Advanced-Therapy-RMAT-Designation-for-BBP-812-Canavan-Disease-Gene-Therapy-Program.html
[24] NKGen Biotech’s Positive Phase 1 Clinical Data in Moderate Alzheimer’s Disease Advances Troculeucel into Phase 2 with First Patient Dosed in Phase 1/2a Trial. Retrieved September 12, 2024, from https://www.globenewswire.com/news-release/2024/09/12/2945163/0/en/NKGen-Biotech-s-Positive-Phase-1-Clinical-Data-in-Moderate-Alzheimer-s-Disease-Advances-Troculeucel-into-Phase-2-with-First-Patient-Dosed-in-Phase-1-2a-Trial.html
[25] HotSpot Therapeutics Presents First-in-Human Phase 1 Clinical Data on its Novel CBL-B Inhibitor, HST-1011, at ESMO Congress 2024. Retrieved September 13, 2024, from https://www.prnewswire.com/news-releases/hotspot-therapeutics-presents-first-in-human-phase-1-clinical-data-on-its-novel-cbl-b-inhibitor-hst-1011-at-esmo-congress-2024-302246957.html
[26] Sernova Announces New Positive Data from Phase I/II Trial Regarding Islet Survival and Function. Retrieved September 13, 2024, from https://www.globenewswire.com/news-release/2024/09/12/2945121/0/en/Sernova-Announces-New-Positive-Data-from-Phase-I-II-Trial-Regarding-Islet-Survival-and-Function.html
[27] VYNE Therapeutics Announces Positive Phase 1a SAD Data for VYN202, a Novel BD2-Selective BET Inhibitor. Retrieved September 13, 2024, from https://www.globenewswire.com/news-release/2024/09/12/2945109/0/en/VYNE-Therapeutics-Announces-Positive-Phase-1a-SAD-Data-for-VYN202-a-Novel-BD2-Selective-BET-Inhibitor.html
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突破性疗法临床结果快速通道临床2期
2024-09-14
本周，生物医药创投领域活跃。Insight 数据库显示，本周全球范围内共发生了 20 多项医药交易和 20 多起新药投融资事件。
在授权合作方面，赛诺菲、拜耳、吉利德科学、诺和诺德、辉瑞等跨国药企动作频繁，合作大多数围绕临床阶段的在研项目进行，并且以开发和商业化合作类型居多。其中，以赛诺菲围绕放射性配体疗法的合作首付款金额最高，达到 1 亿欧元。
在新锐融资方面，细胞和基因治疗（CGT）、T 细胞连接器（TCE）、双抗等领域尤其被资本看好，这些获得融资的 Biotech 主要来自美国和中国。其中，又以自免新锐 Candid Therapeutics 最引人注目，不仅是因为其融资金额高达 3.7 亿美元以上，而且其通过收购获得的两款 TCE 产品均来自中国 Biotech。
下面，Insight 数据库将分别节选部分备受关注的融资交易案例做介绍，仅供读者参阅。
授权合作
赛诺菲就放射性配体疗法达成合作
赛诺菲宣布，已与 RadioMedix 和 Orano Med 达成独家许可协议，将共同开发基于同位素铅的放射性配体疗法（RLT）用于癌症治疗。根据协议，RadioMedix 和 Orano Med 将获得 1 亿欧元的预付款和高达 2.2 亿欧元的销售里程碑等款项。
此次合作主要聚焦于后期临床项目 AlphaMedix，这是一种基于α粒子的靶向疗法（TAT），已在早期临床中被证明耐受性良好，可显著减轻患者的肿瘤负担，用药后患者缓解率达 62.5%。美国 FDA 已在近日授予该产品突破性疗法认定，用以治疗未接受过肽受体放射性疗法的胃肠胰神经内分泌肿瘤患者，目前该药 2 期临床即将完成。
Roivant Sciences 与拜耳达成合作
Roivant Sciences 宣布获得拜耳可溶性鸟苷酸环化酶（sGC）激活剂 Mosliciguat 的全球授权。为此，拜耳将收到 1400 万美元预付款，以及未来高达 2.8 亿美元的开发、监管和商业化里程碑付款。同日，Roivant Sciences 宣布新设立子公司 Pulmovant，后者将专注于开发 Mosliciguat。
目前，Mosliciguat 正在开发用于治疗与间质性肺病（PH-ILD）相关的肺动脉高压，1b 期概念验证 ATMOS 研究结果显示，在 1 mg、2 mg 和 4 mg 剂量下，Mosliciguat 的肺血管阻力平均峰值分别较基线降低 25.9%、38.1% 和 36.3%。
吉利德科学与Genesis合作开发新型小分子疗法
吉利德科学与 Genesis Therapeutics 宣布达成战略合作，以发现和开发跨多个靶点的新型小分子疗法。此次合作将利用 Genesis 公司的人工智能平台 GEMS，以协助为吉利德选择的靶标生成和优化分子。两家公司将在临床前研究活动上密切合作，吉利德将拥有合作化合物潜在临床开发和商业化的独家权利。
根据协议，Genesis 公司将获得 3500 万美元预付款，同时有资格获得额外的临床前、开发、监管和商业里程碑付款。如果吉利德成功将合作的产品商业化，Genesis 公司也有资格获得净销售额的分层特许权使用费。
欧加隆中国与宝济药业及其子公司达成合作
欧加隆中国宣布与宝济药业及其全资子公司晟济药业就辅助生殖领域新药 SJ02 达成供应和转让协议。根据协议，欧加隆中国将获得 SJ02 在中国大陆的独家商业化权益，宝济药业及其子公司将获 1200 万美元的首付款，并将根据商业化进程等获得额外款项。
SJ02 是由晟济药业研发的一款长效重组人促卵泡激素-CTP 融合蛋白注射液（FSH-CTP），适用于接受辅助生殖技术治疗的女性中，与促性腺激素释放激素拮抗剂联合使用进行控制性卵巢刺激，诱导多个滤泡发育。该产品可在卵巢内启动并维持卵泡生长 7 天，目前已在中国申报上市。
ENCell 与 Lucy Biotech 达成合作
ENCell 公司宣布与 Lucy Biotech 签署下一代间充质干细胞疗法 EN001 的许可和战略联盟协议，授予后者在六个亚洲国家和地区（中国香港、台湾、澳门，以及越南、泰国和新加坡）开发和商业化 EN001 的权利。根据协议，ENCell 公司将获得 150 万美元的预付款，并将获得高达 1800 万美元的里程碑付款及基于销售的特许权使用费。
EN001 是一款临床阶段的间充质干细胞治疗候选药物，使用早期培养阶段获得的间充质造血干细胞生产。临床前研究显示，该产品在再生受损神经和肌肉方面具有一定潜力，目前已完成针对夏科-马里-图斯病 1a 型患者的 1a 期临床试验，并正在开展治疗腓骨肌萎缩症 1A 型患者的 1b 期试验。
ITM 公司与 Debiopharm 公司达成合作
ITM 公司宣布与 Debiopharm 公司达成合作，获得后者放射性药物项目的全球独家开发和商业化权，该药物结合了治疗化合物 Debio 0228 和诊断成像剂 Debio 0328，靶向碳酸酐酶 IX（CA IX）。根据协议，Debiopharm 公司将获得约 3 亿欧元的前期、开发和监管里程碑付款，以及商业里程碑和未来潜在销售的低两位数特许权使用费。
Debio 0228/0328 是一对研究性治疗药物，其中，Debio 0328 是一种可单独使用的 PET 成像剂，旨在识别癌症过度表达 CA IX 的患者。一旦识别，这些患者可以用镥标记的放射性配体 Debio 0228 治疗，该配体旨在向肿瘤输送靶向辐射，目的是从内部将其摧毁。目前，该药物正在 1/2 期临床中被评估用于透明细胞肾细胞癌、胰腺导管腺癌和结直肠癌癌症的治疗。
诺和诺德再次与 Kallyope 公司达成合作
Kallyope 公司宣布与诺和诺德签订许可协议，后者行使选择权，允许由 Kallyope 公司鉴定的配体用于疗法开发，包括潜在的肥胖治疗。诺和诺德将负责临床前和临床开发、制造和商业化，而 Kallyope 公司将收到预付费，并可能根据里程碑和销售版税获得额外付款。
此前，双方曾达成合作，以利用 Kallyope-Klarity 平台来鉴定可能改善肥胖治疗前景的基于肽的潜在疗法。研究合作期间产生的临床前数据表明，由 Kallyope 公司鉴定的配体可能对肥胖患者产生有益影响。
辉瑞与 HK inno.N 公司达成合作
HK inno.N 宣布与辉瑞签署协议，在韩国供应一种新的新冠肺炎疫苗 Comirnaty JN1 注射液。根据协议，HK inno.N 将负责在国内向公众分发该疫苗。
Comirnaty JN1 是一种 mRNA 疫苗，设计用于针对新冠奥密克戎变异株 JN.1。它以 0.3 毫升的单次肌内注射形式给药，无需稀释，以预防 12 岁及以上人群中由 SARS-CoV-2 病毒引起的 COVID-19。8 月 30 日，Comirnaty JN1 获得了韩国食品药品安全部的批准。
诺纳生物与 Umoja Biopharma 达成合作
诺纳生物宣布，与 Umoja Biopharma 达成多靶点抗体开发合作。该合作将利用诺纳生物专有的全人源重链抗体（HCAb）平台技术生产新型体内 CAR-T 细胞疗法，并进一步扩大该创新递送技术的潜在应用范围。全人源 HCAb 具有小巧的体积、简单的结构及精准的结合能力，能显著降低抗体免疫原性，同时提高 CAR 功能设计的灵活性和多样性。
新锐融资
Candid 公司完成超过 3.7 亿美元融资
Candid Therapeutics 宣布走出隐匿模式，并完成超过 3.7 亿美元融资。该公司正在开发能够清除特定 B 淋巴细胞群的 T 细胞连接器（TCE）抗体，拟用于治疗自身免疫疾病。通过三方合并和多步融资，Candid 公司收购了 Vignette Bio 和 TRC 2004 公司，从而获得两款 TCE 产品——CND106 和 CND261。
其中，CND106 是一款 BCMA/CD3 双抗，Vignette Bio 通过与岸迈生物合作获得其在大中华区以外开发和商业化的独家权利。CND261 是一款 CD20/CD3 双抗，TRC 2004 公司通过与嘉和生物合作获得了其在大中华区以外的全球独家许可权益。目前，这两款产品均已完成了 1 期剂量递增研究。
君合盟完成超 2 亿元 B+融资
君合盟宣布，继今年 5 月底获得通化东宝 1 亿元 B 轮融资后，再次完成由国投创合领投的超 2 亿元 B+融资。本轮募集资金将主要用于进一步推进该公司在研管线，并加快产业化落地及商业化推广。
君合盟成立于 2020 年 11 月，专注于开发重组蛋白创新药物及合成生物学领域创新产品，围绕严肃医疗、消费医疗、代谢等领域。目前，该公司主要在研产品有重组 I 型人胶原蛋白、重组 III 型人胶原蛋白、重组 A 型肉毒毒素、重组人生长激素注射液和重组长效生长激素。其中，重组 A 型肉毒毒素用于成人中重度眉间纹的 2 期临床已接近尾声，即将进入 3 期临床。
贝斯生物完成数千万元 A2 轮融资
贝斯生物宣布完成由 AEF 大湾区创业基金（由戈壁大湾区（GBA）管理）领投的数千万元 A2 轮融资，融资资金将用于加强研发团队、优化产品管线、加速临床试验等。
贝斯生物成立于 2021 年，专注于基因编辑领域基础创新，其开发的 AccuBase 碱基编辑系统可实现体外和体内高效的基因编辑且零脱靶。另外，该公司还开发了先导编辑技术 ePE、CasH 基因编辑技术、BEAT-CART 技术、体内靶向递送技术等。
基于这些技术，贝斯生物正在开发 BASE-NK 和体内碱基编辑产品管线，其中进展最快的 NK510 细胞注射液已在中国递交 IND，这是一款基于碱基编辑技术的自然杀伤细胞（NK）产品，拟开发用于治疗晚期实体肿瘤。
晶蛋生物完成数千万元 A 轮融资
晶蛋生物宣布完成数千万元 A 轮融资。本轮融资由勤智资本领投，老股东隆门资本、粤科金融继续追加投资，融资所得将主要用于推动核心管线的研发进展，并加速与跨国药企的合作。
晶蛋生物成立于 2019 年，专注于研发神经精神疾病及慢性肾病的离子通道创新药。该公司核心团队来自哈佛大学和上海药物所，同时邀请了诺奖得主离子通道领域奠基人 Erwin Neher 博士担任首席科学顾问。目前，该公司已建立离子通道靶点小分子和蛋白药物技术平台，并配套四大工具平台，解决了药物发现成功率低和副作用大等行业瓶颈。
Superluminal 完成 1.2 亿美元 A 轮融资
Superluminal Medicines 宣布完成 1.2 亿美元的 A 轮融资。本轮融资由 RA Capital Management 领投，Insight Partners、NVentures、礼来、Gaingels 等参与投资，融资所得将支持该公司先导项目进入临床开发阶段，并增加专注于高价值 G 蛋白偶联受体（GPCR）小分子药物发现项目的数量。
Superluminal 公司成立于 2023 年，研究重点是介导细胞信号传导和人类生理学基础反应过程的膜受体，并构成主要类别的药物靶点，包括 GPCR、离子通道和转运蛋白。该公司的技术平台通过利用生物学和化学专业知识、机器学习和专有大数据基础设施的全面结合，快速开发候选化合物。目前，该公司正在推进 6 个小分子项目。
Epsilogen 完成 1250 万英镑的 B 轮拓展融资
Epsilogen 宣布完成 1250 万英镑的 B 轮拓展融资，这使其 B 轮融资总额达到 4325 万英镑。本轮融资由 British Patient Capital、诺华风险投资基金、Epidarex Capital、3B Future Health Fund、ALSA Ventures 等参与投资。
本轮融资所得将主要用于推进免疫球蛋白 E（IgE）抗体 MOv18 IgE 治疗铂耐药复发性卵巢癌（PROC）的 1b 期试验以获得概念验证，以及进一步开发该公司基于 IgE 的管线。MOv18 IgE 是首款进入临床的治疗性 IgE 抗体，靶向叶酸受体α（FRα）。在 1 期试验中，该产品已展现出安全性和耐受性良好，同时具有抗肿瘤活性。
Bambusa 完成约 1500 万美元的种子轮融资
Bambusa Therapeutics 宣布完成约 1500 万美元的种子轮融资，本轮融资由 BVF Partners 和 Dawn Biopharma 共同领投，Salvia GmbH 和 INCE Capital 参与投资，募集资金将用于加速 Bambusa 公司双特异性抗体管线的开发，以进行 1 期临床研究。
Bambusa 公司由一群行业专家和科学创新者创立，他们的愿景是改变免疫和炎症 疾病的治疗格局。该公司利用经过验证的靶点，通过创新的抗体工程创建双特异性抗体疗法。
Vironexis 完成 2600 万美元的种子轮融资
Vironexis Biotherapeutics 宣布走出隐匿模式，并完成 2600 万美元的种子轮融资。本轮融资由 Drive Capital 和 Future Ventures 领投，Moonshots Capital、Capital Factory 参与投资。Vironexis 公司有 TransJoin 腺相关病毒（AAV）基因疗法平台，以及治疗血液癌症、预防实体瘤转移及癌症疫苗等十余款候选产品。
其中，首个候选基因疗法 VNX-101 已获得 FDA 授予的快速通道资格和罕见儿科疾病资格，其治疗 CD19 阳性急性淋巴细胞白血病的 IND 申请也已获 FDA 许可。第二个项目 VNX-202 用于预防 HER2 阳性癌症（包括乳腺癌及其他类型肿瘤）的转移，预计将在 2025 年启动 1/2 期临床试验。
封面来源：站酷海洛 Plus
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放射疗法突破性疗法并购基因疗法引进/卖出
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 纤维化 | 临床2期 | 美国 | 2024-11-01 | |
| 肺性高血压 | 临床2期 | 美国 | 2024-11-01 | |
| 间质性肺疾病 | 临床2期 | 美国 | 2024-11-01 | |
| 血管疾病 | 临床2期 | 美国 | 2024-11-01 | |
| 间质性肺病导致的肺动脉高压 | 临床2期 | - | - | |
| 慢性血栓栓塞性肺动脉高压 | 临床1期 | 奥地利 | 2019-01-21 | |
| 肺动脉高压 | 临床1期 | 波兰 | 2019-01-21 | |
| 肺动脉高压 | 临床1期 | 德国 | 2019-01-21 | |
| 肺动脉高压 | 临床1期 | 奥地利 | 2019-01-21 | |
| 肺动脉高压 | 临床1期 | 捷克 | 2019-01-21 |
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临床结果
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| 研究 | 分期 | 人群特征 | 评价人数 | 分组 | 结果 | 评价 | 发布日期 |
|---|
临床1期 | 38 | (構觸襯壓簾齋觸鹽繭衊) = 願觸廠繭齋觸簾憲遞觸 鏇醖構鹽廠選選衊築窪 (鹹鏇憲餘鬱艱壓鏇膚艱 ) | 积极 | 2024-09-10 | |||
(構觸襯壓簾齋觸鹽繭衊) = 願膚顧壓鏇膚範顧簾繭 鏇醖構鹽廠選選衊築窪 (鹹鏇憲餘鬱艱壓鏇膚艱 ) |
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