A Randomized, Double-blind, Placebo-Controlled, Phase III Study to Evaluate the Efficacy and Safety of Proxalutamide (GT0918) in Hospitalized COVID-19 Subjects

This study is an adaptive Phase III randomized double-blind placebo-controlled trial to evaluate the efficacy and safety of Proxalutamide (GT0918) in hospitalized adults diagnosed with COVID-19. The study is a multicenter trial that will be conducted globally. The study will compare GT0918 plus standard of care (SOC) with the placebo plus SOC. Approximately 762 subjects will be randomized in a 1:1 ratio to either GT0918 plus SOC or placebo plus SOC group.

开始日期2021-09-30

申办/合作机构

苏州开拓药业股份有限公司

NCT04869228 / Terminated临床3期

A Phase 3 Randomized, Double-Blind Placebo Controlled, Multi-regional Trial to Evaluate the Efficacy and Safety of GT0918 for the Treatment of Mild to Moderate COVID-19 Male Patients

The purpose of this study is to assess the efficacy and safety of Proxalutamide (GT0918) in male outpatients with mild to moderate COVID-19 disease

开始日期2021-08-04

申办/合作机构

苏州开拓药业股份有限公司

NCT05076851 / Unknown status临床2期IIT

A Randomized, Controlled, Double-blind, Single Center，Clinical Trial to Evaluate the Efficacy and Safety of Neoadjuvant Therapy With Proxalutamide Combined With Androgen Deprivation Therapy（ADT）for High Risk Prostate Cancer

This trial aims to evaluate the efficacy and safety of neoadjuvant therapy with Proxalutamide combined with androgen deprivation therapy（ADT）for High risk prostate cancer ，This trial is A randomized, controlled, double-blind, single center.Treatment cycle is 6 months,

开始日期2021-06-17

申办/合作机构-

100 项与普克鲁胺相关的临床结果

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100 项与普克鲁胺相关的转化医学

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100 项与普克鲁胺相关的专利（医药）

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项与普克鲁胺相关的文献（医药）

2024-03-01·International Journal of Antimicrobial Agents

Small-molecule antiviral treatments for COVID-19: A systematic review and network meta-analysis

Review

作者: Han, Bing ; Feng, Pinpin ; Zhang, Meiling ; Ying, Yin ; Yang, Bo ; Zhao, Qinqin ; Xin, Chuanwei ; Zhu, Jun ; Li, Gonghua ; Yang, Wenjuan ; Zheng, Bei

OBJECTIVEThis study aimed to explore the efficacy and safety of small-molecule antivirals for treating coronavirus disease 2019 (COVID-19).METHODSSeven databases were searched from their inception to 01 June 2023. The risk of bias in randomised controlled trials and retrospective studies was evaluated individually using the Cochrane risk-of-bias tool and Newcastle Ottawa Scale.RESULTSIn total, 160 studies involving 933 409 COVID-19 patients were evaluated. Compared with placebo or standard of care, proxalutamide demonstrated remarkable efficacy in reducing mortality rates, hospitalisation rates, serious adverse events, and the need for mechanical ventilation. Furthermore, it significantly enhanced both the rate of clinical improvement and expedited the duration of clinical recovery when compared with control groups. In patients with mild-to-moderate COVID-19, proxalutamide exhibited the above advantages, except for mortality reduction. Triazavirin was the most effective treatment for reducing the time required for viral clearance and improving the discharge rate. Leritrelvir and VV116 were ranked first in terms of enhancing the viral clearance rate on days 7 and 14, respectively. Molnupiravir was the most effective treatment for reducing the need for oxygen support. Overall, these findings remained consistent across the various subgroups.CONCLUSIONSA thorough evaluation of effectiveness, applicable to both mild-to-moderate and unstratified populations, highlights the specific advantages of proxalutamide, nirmatrelvir/ritonavir, triazavirin, azvudine, molnupiravir, and VV116 in combating COVID-19. Additional clinical data are required to confirm the efficacy and safety of simnotrelvir/ritonavir and leritrelvir. The safety profiles of these antivirals were deemed acceptable.

2023-08-15·International journal of cancer

Proxalutamide in metastatic castration-resistant prostate cancer: Primary analysis of a multicenter, randomized, open-label, phase 2 trial.

Article

作者: Hou, Jianguo ; Zhou, Tie ; Chen, Guanghua ; Sun, Zhongquan ; Sun, Weibing ; Xu, Weidong ; Li, Song ; Meng, Qinggui ; Wang, Jingen ; Liu, Ben ; Wang, Yinhuai ; He, Dalin ; Qin, Shengfei ; Yang, Yong ; Tang, Shouyan ; Zhang, Wei ; Ye, Dingwei ; Wang, Dongwen ; Hu, Zhiquan ; Fu, Cheng ; Chen, Lijun ; Shi, Guowei ; Song, Xishuang ; Gao, Xu ; Jin, Jie

We aim to assess the safety and efficacy of proxalutamide, a novel androgen receptor antagonist, for men with metastatic castration-resistant prostate cancer (mCRPC) in a multicenter, randomized, open-label, phase 2 trial. In our study, the enrolled mCRPC patients were randomized to 100, 200 and 300 mg dose groups at 1:1:1. The primary efficacy endpoint was prostate-specific antigen (PSA) response rate. The secondary endpoints included objective response rate (ORR), disease control rate (DCR) and time to PSA and radiographic progression. Safety and pharmacokinetics were also assessed. Finally, there were 108 patients from 17 centers being enrolled. By week 16, there were 13 (35.1%), 12 (36.4%) and 15 (42.9%) patients with confirmed 50% or greater PSA decline in 100 mg (n = 37), 200 mg (n = 33) and 300 mg (n = 35) groups, respectively. Among the 19 patients with target lesions at study entry, three (15.8%) had a partial response and 12 (63.2%) had stable disease. The ORRs of 20.0%, 22.2%, 0% and DCRs of 80.0%, 88.9%, 60.0% were, respectively, achieved in 100, 200 and 300 mg groups. By the maximum follow-up time of 24 weeks, there were 42.6% and 10.2% of cases experiencing PSA progression and radiographic progression, respectively. Overall, adverse events (AEs) were experienced by 94.4% of patients, most of which were mild or moderate. There were 28 patients experiencing ≥grade 3 AEs. The most common AEs were fatigue (17.6%), anemia (14.8%), elevated AST (14.8%) and ALT (13.0%), decreased appetite (13.0%). These findings preliminarily showed the promising antitumor activity of proxalutamide in patients with mCRPC with a manageable safety profile. The proxalutamide dose of 200 mg daily is recommended for future phase 3 trial (Clinical trial registration no. CTR20170177).

2023-07-25·Proceedings of the National Academy of Sciences1区 · 综合性期刊

Proxalutamide reduces SARS-CoV-2 infection and associated inflammatory response

1区 · 综合性期刊

Article

作者: Pretto, Carla D. ; Parolia, Abhijit ; Ma, Liandong ; Su, Fengyun ; Qiao, Yuanyuan ; Sexton, Jonathan Z. ; Qin, Jun ; Cheng, Caleb ; Ellison, Stephanie J. ; Wang, Rui ; Eyunni, Sanjana ; Cao, Xuhong ; Zhou, Qianxiang ; He, Tongchen ; Wang, Yini ; Chinnaiyan, Arul M. ; Zhang, Yuping ; Zheng, Yang ; Yan, Honghua ; Wotring, Jesse W. ; Jiang, Xia ; Zhang, Charles J.

Early in the COVID-19 pandemic, data suggested that males had a higher risk of developing severe disease and that androgen deprivation therapy might be associated with protection. Combined with the fact that TMPRSS2 ( transmembrane serine protease 2 ), a host entry factor for the SARS-CoV-2 virus, was a well-known androgen-regulated gene, this led to an upsurge of research investigating androgen receptor (AR)-targeting drugs. Proxalutamide, an AR antagonist, was shown in initial clinical studies to benefit COVID-19 patients; however, further validation is needed as one study was retracted. Due to continued interest in proxalutamide, which is in phase 3 trials, we examined its ability to impact SARS-CoV-2 infection and downstream inflammatory responses. Proxalutamide exerted similar effects as enzalutamide, an AR antagonist prescribed for advanced prostate cancer, in decreasing AR signaling and expression of TMPRSS2 and angiotensin-converting enzyme 2 (ACE2) , the SARS-CoV-2 receptor. However, proxalutamide led to degradation of AR protein, which was not observed with enzalutamide. Proxalutamide inhibited SARS-CoV-2 infection with an IC 50 value of 97 nM, compared to 281 nM for enzalutamide. Importantly, proxalutamide inhibited infection by multiple SARS-CoV-2 variants and synergized with remdesivir. Proxalutamide protected against cell death in response to tumor necrosis factor alpha and interferon gamma, and overall survival of mice was increased with proxalutamide treatment prior to cytokine exposure. Mechanistically, we found that proxalutamide increased levels of NRF2, an essential transcription factor that mediates antioxidant responses, and decreased lung inflammation. These data provide compelling evidence that proxalutamide can prevent SARS-CoV-2 infection and cytokine-induced lung damage, suggesting that promising clinical data may emerge from ongoing phase 3 trials.

108

项与普克鲁胺相关的新闻（医药）

2024-10-15

·PRNewswire

Metastatic Castration-Sensitive Prostate Cancer Clinical Trial Pipeline Insights Featuring 35+ Companies | DelveInsight

Metastatic castration-sensitive prostate cancer (mCSPC), also known as metastatic hormone-sensitive prostate cancer (mHSPC), refers to prostate cancer that has spread beyond the prostate but still responds to therapies that lower testosterone (castration) with an aging population, the incidence of prostate cancer is rising, leading to an increased demand for advanced therapies to treat metastatic forms of the disease. LAS VEGAS, Oct. 15, 2024 /PRNewswire/ -- DelveInsight's ' Metastatic Castration-Sensitive Prostate Cancer Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline mCSPC therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the mCSPC pipeline domain. Key Takeaways from the Metastatic Castration-Sensitive Prostate Cancer Pipeline Report DelveInsight's mCSPC pipeline report depicts a robust space with 35+ active players working to develop 40+ pipeline therapies for mCSPC treatment. Key mCSPC companies such as Tavanta Therapeutics, AstraZeneca, Regeneron Pharmaceuticals, Astellas Pharma, Propella Therapeutics, POINT Biopharma, Amgen, Janssen Research & Development, LLC, Bayer, Beigene, Amgen, Merck Sharp & Dohme LLC, Jiangsu HengRui Medicine Co., Ltd., Suzhou Kintor Pharmaceutical Inc, Astellas Pharma, and others are evaluating new mCSPC drugs to improve the treatment landscape. Promising mCSPC pipeline therapies such as TAVT-45 (abiraterone acetate), Saruparib (AZD5305), Capivasertib, REGN2810, Abiraterone decanote + dexamethasone or prednisone (PRL-02/ASP5541), [Ac-225]-PSMA-62, Xaluritamig, Apalutamide, Darolutamide, Luteinizing Hormone Releasing Hormone, Pembrolizumab, Dalpiciclib isethionate, GT0918, PRL-02, and others are under different phases of mCSPC clinical trials. In October 2024, Apalutamide (Erleada) was found to provide a significant improvement in overall survival (OS) at 24 months compared with enzalutamide (Xtandi) in the treatment of androgen receptor pathway inhibitor (ARPI)-naïve patients with metastatic castration-sensitive prostate cancer (mCSPC). In April 2024, Saruparib (AZD5305) demonstrated encouraging efficacy, favorable safety, a wide therapeutic index, and improved pharmacokinetic properties compared with currently approved PARP inhibitors in patients with advanced solid tumors harboring BRCA1/2, PALB2, or RAD51C/D mutations, according to findings from the Phase I/IIa PETRA study. In February 2024, Capivasertib combined with docetaxel to enhance anti-tumour activity through inhibition of AKT-mediated survival mechanisms in prostate cancer. Capivasertib can enhance anti-tumour effects of docetaxel by targeting residual docetaxel-persister cells, independent of PTEN status, to induce apoptosis and DNA damage in part through GSK3β. In January 2023, Tavanta Therapeutics announced positive top-line results from its pivotal, global Phase III clinical trial, evaluating the safety and efficacy of TAVT-45 (abiraterone acetate). The trial met its primary objective of establishing therapeutic equivalence of TAVT-45 to Zytiga® in patients with metastatic castrate-resistant prostate cancer (mCRPC) and metastatic high-risk castrate-sensitive prostate cancer (mCSPC), in addition to demonstrating a comparable safety profile. Request a sample and discover the recent advances in mCSPC treatment drugs @ Metastatic Castration-Sensitive Prostate Cancer Pipeline Report The mCSPC pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage mCSPC drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the mCSPC clinical trial landscape. Metastatic Castration-Sensitive Prostate Cancer Overview Metastatic castration-sensitive prostate cancer (mCSPC) is an advanced form of prostate cancer in which the cancer cells have spread beyond the prostate to other parts of the body, such as bones or lymph nodes, but remain responsive to hormone therapy. This means that reducing testosterone levels can still effectively slow the growth of cancer cells. mCSPC often arises when prostate cancer is initially diagnosed or when early-stage prostate cancer recurs after treatment. It is distinct from castration-resistant prostate cancer (CRPC), where the cancer continues to progress despite low testosterone levels. The causes of mCSPC primarily involve the spread of prostate cancer cells beyond the local prostate region, a process called metastasis. Although the exact mechanisms are complex, certain risk factors increase the likelihood of developing prostate cancer and its progression to metastatic disease. These risk factors include advanced age, a family history of prostate cancer, genetic mutations such as BRCA1/BRCA2, and race. Symptoms of mCSPC can include bone pain, urinary issues, fatigue, weight loss, and general malaise, especially if the cancer has spread to bones or lymph nodes. However, in many cases, early stages of metastasis may be asymptomatic, making regular screening essential for those at risk. Diagnosis of mCSPC typically involves a combination of blood tests, such as measuring prostate-specific antigen (PSA) levels, imaging studies, and biopsy to confirm the presence of cancer cells. Treatment for mCSPC revolves around androgen deprivation therapy to lower testosterone levels, often combined with other agents like chemotherapy (e.g., docetaxel) or second-generation androgen receptor inhibitors (e.g., abiraterone or enzalutamide). In recent years, clinical trials have shown the benefit of combining ADT with novel therapies to improve survival rates and manage symptoms, with personalized treatment approaches based on genetic testing becoming more common. Find out more about mCSPC treatment drugs @ Drugs for Metastatic Castration-Sensitive Prostate Cancer Treatment A snapshot of the mCSPC Pipeline Drugs mentioned in the report: Learn more about the emerging mCSPC pipeline therapies @ Metastatic Castration-Sensitive Prostate Cancer Clinical Trials Metastatic Castration-Sensitive Prostate Cancer Therapeutics Assessment The mCSPC pipeline report proffers an integral view of the mCSPC emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Metastatic Castration-Sensitive Prostate Cancer Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous Therapeutics Assessment By Molecule Type: Small molecule, Cell therapy, Peptides, Polymer, Small molecule, Gene therapy Therapeutics Assessment By Mechanism of Action: Steroidal inhibitor of CYP17A1, PARP1 inhibitor, Proto-oncogene protein c-akt inhibitors, CYP17 lyase inhibitor, Antibody-dependent cell cytotoxicity, Immunostimulants, T lymphocyte stimulants, Ionizing radiation emitters. Key Metastatic Castration-Sensitive Prostate Cancer Companies: Tavanta Therapeutics, AstraZeneca, Regeneron Pharmaceuticals, Astellas Pharma, Propella Therapeutics, POINT Biopharma, Amgen, Janssen Research & Development, LLC, Bayer, Beigene, Amgen, Merck Sharp & Dohme LLC, Jiangsu HengRui Medicine Co., Ltd., Suzhou Kintor Pharmaceutical Inc, Astellas Pharma, and others. Key Metastatic Castration-Sensitive Prostate Cancer Pipeline Therapies: TAVT-45 (abiraterone acetate), Saruparib (AZD5305), Capivasertib, REGN2810, Abiraterone decanote + dexamethasone or prednisone (PRL-02/ASP5541), [Ac-225]-PSMA-62, Xaluritamig, Apalutamide, Darolutamide, Luteinizing Hormone Releasing Hormone, Pembrolizumab, Dalpiciclib isethionate, GT0918, PRL-02, and others. Dive deep into rich insights for new drugs for mCSPC treatment, visit @ Metastatic Castration-Sensitive Prostate Cancer Drugs Table of Contents For further information on the mCSPC Cancer pipeline therapeutics, reach out @ Metastatic Castration-Sensitive Prostate Cancer Treatment Drugs Related Reports Metastatic Castration-Sensitive Prostate Cancer Market Metastatic Castration-Sensitive Prostate Cancer Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key mCSPC companies including Myovant Sciences, Pfizer, Bayer, Orion, Novartis, AstraZeneca, Pfizer, Janssen, Merck, Eli Lilly, among others. Metastatic Prostate Cancer Market Metastatic Prostate Cancer Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key metastatic prostate cancer companies including AstraZeneca, Arvinas, Madison Vaccines, Phosplatin Therapeutics, Hinova Pharmaceuticals, Bristol Myers Squibb, Merck, MacroGenics, Daiichi Sankyo, Seagen, Taiho Pharmaceutical, Modra Pharmaceuticals, Xencor, Point Biopharma, Lantheus Holdings, Zenith Epigenetics, Essa Pharma, Telix Pharmaceuticals, Kintor Pharmaceutical, AB Science, Eli Lilly and Company, Exelixis among others. Metastatic Castration-Resistant Prostate Cancer Market Metastatic Castration-Resistant Prostate Cancer Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key mCRPC companies including AstraZeneca, Merck Sharp & Dohme, Hinova Pharmaceuticals, Pfizer, Astellas Pharma, Modra Pharmaceuticals, AB Science, Eli Lilly and Company, Zr Pharma & GmbH, Bristol-Myers Squibb, Ipsen, Exelixis, Takeda, Janssen Research & Development, Tesaro, Lantheus Holdings, Kintor Pharmaceutical, MacroGenics, Daiichi Sankyo, Madison Vaccines, Novartis, Point Biopharma, Xencor, Essa Pharma, Telix International, Bayer, Arvinas, among others. Metastatic Castration-Resistant Prostate Cancer Pipeline Metastatic Castration-Resistant Prostate Cancer Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key mCRPC companies, including AstraZeneca, Merck Sharp & Dohme, Hinova Pharmaceuticals, Pfizer, Astellas Pharma, Modra Pharmaceuticals, AB Science, Eli Lilly and Company, Zr Pharma & GmbH, Bristol-Myers Squibb, Ipsen, Exelixis, Takeda, Janssen Research & Development, Tesaro, Lantheus Holdings, Kintor Pharmaceutical, MacroGenics, Daiichi Sankyo, Madison Vaccines, Novartis, Point Biopharma, Xencor, Essa Pharma , Telix International, Bayer, Arvinas, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床3期临床结果

2024-03-29

·美通社

开拓药业2023年度业绩：探索皮科多渠道商业化路径，深挖多管线研发及商业价值

苏州 2024年3月29日 /美通社/ -- 北京时间2024年3月29日，开拓药业（股票代码：9939.HK），一家专注于潜在同类首创和同类最佳创新药物研发及产业化的生物制药公司，公布了近期业务亮点及截至2023年12月31日全年业绩。 2023年度业绩总览业务概览皮科双核心产品全球临床研发持续推进在研多管线显露研发及商业合作价值多项研究成果入选重要学术会议及于核心期刊发表财务概览资金使用效率有效提升，在手现金支持公司战略布局奖项荣誉斩获认可，屡次入选生科公司榜单一、业务概览作为专注于潜在同类首创和同类最佳创新药物研发及产业化的生物制药公司，开拓药业前瞻性布局了包含小分子创新药、生物创新药及联合疗法的多元化产品管线，目前拥有6款在中国和全球开展临床I-III期研究的新药项目。 1. 皮科双核心产品全球临床研发持续推进我们在皮科领域拥有丰富的研发经验，目前两款核心药物KX-826及GT20029已分别推进至临床III期及临床II期，并正积极探索核心产品于皮科领域的商业化路径。此外，我们亦在开发更多化合物，拓展皮科领域的适应症布局。 KX-826 ，公司自主开发的外用雄激素受体（AR）拮抗剂，超千人使用显示良好的安全性和有效性，连续使用6个月后，患者目标区域内的平均非毳毛数（TAHC）较基线增加最高可达22.7根/cm 2 。 2023年，KX-826完成了中国男性脱发III期临床试验、美国男性脱发II期临床试验及中国痤疮II期临床试验，同时，我们启动了中国脱发长期安全性III期临床试验及KX-826与米诺地尔联合使用的中国脱发Ib/III期临床试验。针对脱发适应症，我们已验证KX-826在不同的人群中的良好安全性及有效性。长期安全性III期临床试验预计将为公司提供长期使用KX-826的安全性及有效性的数据支持。与米诺地尔联合疗法的探索将进一步挖掘KX-826于脱发领域的治疗效果。针对痤疮适应症，II期临床试验的结果为公司后续研究开展奠定了基础。 2024年2月1日，公司自主研发的KX-826和米诺地尔联合治疗中国男性患者雄激素性脱发Ib/III期临床试验获得中国国家药品监督管理局（NMPA）批准开展。 2023年11月27日，公司公布KX-826治疗男性脱发的中国III期临床试验顶线数据。分析结果显示，该试验整体安全性优良，KX-826展示了极佳的安全性能。治疗24周后，KX-826组TAHC结果显示，KX-826与基线相比促进了毛发生长，具有统计学意义（ p <0.0001）。与安慰剂相比，KX-826 0.5%BID组TAHC在各个访视点均有提高，显示疗效趋势，但差异在统计学上未达到显著性。 2023年11月15日，KX-826治疗脱发的长期安全性III期临床试验完成全部患者入组。该试验于2023年4月18日获得NMPA批准开展。 2023年8月28日，公司宣布KX-826治疗痤疮的中国II期临床试验已成功完成。结果显示KX-826具有良好的安全性和有效性。 2023年5月11日，公司宣布KX-826治疗男性脱发的美国II期临床试验已成功完成。与基线相比，治疗24周后的结果具有统计学和临床意义，且安全性良好。 GT20029 ，外用AR-PROTAC化合物，基于公司自有PROTAC平台开发，是全球首个进入II期临床试验的外用PROTAC化合物。 2023年，GT20029完成了美国脱发及痤疮的I期临床试验。其于中国开展的脱发II期临床试验预计将在近期读出顶线结果。基于GT20029治疗痤疮于中国及美国I期临床试验的结果，我们正在计划开展GT20029用于痤疮治疗的II期临床试验。 2023年8月22日，GT20029治疗男性脱发的中国II期临床试验完成全部180名患者入组，公司正在清理各个入组中心的数据，预计将在近期读出顶线结果，并据此确定后续的临床策略，如开展中国脱发男性III期临床试验等。 2023年2月10日，公司公布GT20029治疗脱发及痤疮的美国I期临床试验的顶线结果。结果显示，GT20029在健康受试者、脱发受试者和痤疮受试者中均展示良好的安全性、耐受性和药代动力学特征。基于GT20029治疗痤疮于中国及美国I期临床试验的结果，我们正在准备开展GT20029用于痤疮治疗的II期临床试验，进一步拓展我们在痤疮治疗领域的研究范围。 2. 在研多管线显露研发及商业合作价值通过多年积累，公司拥有另外4款处于临床阶段的产品，前期的临床试验已初步验证该等产品具有良好的安全性及疗效。公司亦开展多项临床前研究，潜在化合物展现出优异的开发及临床合作价值。 GT1708F ，公司自主开发的针对多种实体瘤的创新药物，并首次将适应症扩大至罕见病领域 2023年10月，GT1708F治疗IPF的中国II期临床试验获得NMPA有条件批准开展。 2023年5月8日，公司宣布GT1708F治疗恶性血液疾病的中国I期临床试验成功完成。结果显示，GT1708F展现出良好的安全性和耐受性，所有患者均未发生剂量限制性毒性（DLT）和与研究药物有关的严重不良事件（SAE）。在剂量递增阶段，自180mg剂量组起，在多线治疗失败的急性骨髓性白血病（AML）患者中观察到初步疗效，AML患者髓系原始细胞较基线最高下降了62%。此外，我们还拥有公司license in的首款大分子药物，具有极佳的联合用药潜力的 ALK-1 ，公司自主开发的有潜力成为同类最佳药物的二代AR抑制剂的普克鲁胺（ GT0918），公司自主开发的分子胶化合物，在多种癌细胞中表现出纳摩尔级别的抗肿瘤活性的 c-Myc分子胶。 3. 科研学术影响力不断增强 2023年以来，开拓药业多个产品管线研究进展入选国际学术大会和在期刊上发表，公司创新能力持续提升，进一步验证公司产品管线First-in-Class的潜力。 KX-826和GT20029治疗雄激素性脱发和痤疮的临床进展在2023年美国皮肤病学会年会（AAD 2023）备受关注 Hedgehog/SMO抑制剂GT1708F治疗急性髓系白血病（AML）的最新临床前研究结果入选第65届美国血液学年会（ASH 2023）及2023年美国癌症研究协会年会（AACR 2023）普克鲁胺治疗转移性乳腺癌Ic期临床试验结果于第46届圣安东尼奥乳腺癌研讨会（SABCS 2023）及2023年欧洲肿瘤内科学会年会（ESMO 2023）亮相 ALK-1联合Nivolumab治疗晚期肝细胞癌的Ib/II期临床研究获《BMC医学》（影响因子11.806)发表 c-Myc分子胶研究获《Nature》子刊《Nature Communications》（影响因子16.6）发表 c-Myc分子胶研究结果于ASH 2023亮相二、财务概览 1. 研发费用 *：下降66.6% 截至2023年12月31日，撇除减值及跌价影响，公司的研发成本共计人民币245.8百万元，同比截至2022年12月31日人民币735.0百万元下降66.6%。研发成本减少主要是由于报告期内公司聚焦核心皮科管线，该等投入较其余管线有极大节约。 * 研发费用已撇除减值及跌价影响 2. 行政开支 *：下降34.6% 截至2023年12月31日，撇除减值影响，公司的行政开支共计人民币86.4百万元，同比截至2022年12月31日人民币132.2百万元下降34.6%。行政开支减少主要是由于报告期内员工薪酬及股权激励费用减少。公司已于2023年底进行雇员规模及薪酬调整，并将视乎业务推进情况采取进一步行动。 *行政开支已撇除减值影响 3. 在手现金截至2023年12月31日，公司持有现金和现金等价物以及定期存款为人民币456.3百万元，另外，公司持有动用的银行授信为人民币110.5百万元。在手现金能够支持公司的临床和研发推进，以及为实现产品商业化的必要性支出。三、奖项荣誉斩获认可，屡次入选生科公司榜单开拓药业凭借其在药物创新研发领域长期的钻研不懈，获得业界的广泛认可，荣膺多项行业和资本市场奖项，包括第十届港股100强"生物科技股15强"、入选"中国小分子药物企业创新力TOP30"排行榜（米内网2022年度中国生物医药企业创新力百强系列榜单）、智通财经评选"最佳中小市值公司"等。四、董事长寄语创始人、董事长兼首席执行官童友之博士表示：新药研发之路注定布满荆棘。2023年对于开拓而言，是在荆棘之路不断磨砺、在重重迷雾中坚定脚步的历程。虽然面临重重挑战，开拓药业始终没有放弃对新药研发的追求。公司上下怀揣着推出首款创新药物的共同目标，付诸自己的努力与汗水。我们在皮科领域的探索始终处于前沿地位，两款皮科核心药物KX-826及GT20029具备优秀临床和商业化潜质。我们已在超1,000位受试者中验证了KX-826的安全性和有效性，得益于我们的药物，这些受试者的平均TAHC较基线增加最高可达22.7根/cm 2 。GT20029计划在近期披露中国脱发II期临床试验的顶线结果并准备开展用于痤疮的II期临床试验。此外，我们亦在开发更多化合物，拓展皮科领域的适应症布局。在非皮科领域，我们正通过联合开发或对外授权方式挖掘更高的药物价值，为患者提供更多的用药选择。展望2024年，我们将继续发挥公司在皮科领域的独特优势，积极探索多方位的商业化路径。同时，我们会提高资金使用效率，期待通过长期可持续发展，为患者、股东创造更多价值。 * 详情请参阅公司于2024年3月28日发布的2023年度业绩公告

临床3期临床2期临床成功

2024-01-03

·赛柏蓝

2024｜医药领域的机会、风险

2023年是中国成为医药创新发源地的第一年，医药魔方数据显示，中国2023年创新药license out（国内转国外）交易总金额超350亿美元，比2022年增长27%以上。底层原因既有自研进步、创新前置的理想叙事，也有投融资艰难、以BD首付款获得输血的现实主义悲歌。部分Biotech把核心管线卖得干干净净，虽然活下来，却要从头再来。2024年，医药行业澎湃的内生增长动力，将遇到两个冰冷的现实。海外资本寒冬正在结束，CXO、XBI、IBB自2023年11月1日以来狂飙突进，而国内未盈利创新药企IPO及再融资阻滞局面，还没有结束的时间表。在大国科技角力的背景下，这将长期损害中国生物科技的活力。很多人还没注意到，医保局官网的基本医疗保险基金收支情况，从2023年10月起不再公布同比增速数据。随着低基数效应消失，预计2024年医保支出将回归高个位数增长（2018~2022医保支出CAGR9.6%）。当然，国家维持卫生费用增速接近GDP增速2倍，也是尽力了。在投融资及支付环境紧平衡的条件下，存量及结构性增量争夺战将更加惨烈，2024年呈现极致分化的局面，让我们由寒及热，快速俯瞰新一年的可能性。0110亿市值以下10亿港元市值以下的港股18A公司增至9家，占带有B标识的生物科技公司比例17%。参考2004-2018年间通过纳斯达克IPO上市的424家创新药企，至今不再存续的比例达47%，且存续企业有37%市值小于1亿美元，预计港股带有B标识的生物科技公司，市值小于10亿港元的比例长期向30%靠拢。在接近半数Biotech消失后，美国创新药产业没有完蛋，而是更加强大，所以不必自作多情。而国内生物科技要形成正常的新陈代谢（退出）生态，需要10亿港元以下市值结构扩容，以降低并购门槛，让管线实现货币化流动。对10亿港元以下Biotech不必悲观，只要现金储备充裕，管线有临床价值，还有重塑自身的机会。而且10亿港元，对国内大型综合药企已不构成资金门槛，参考美股亘喜生物，一个并购可以引发脉冲式爆发。02平静等待结局开拓药业正在平静等待自己的结局。曾经5个月10倍，成为全球医药股涨幅王，即使在随后漫长的下跌途中也充满诱惑力，出现约10次单日上涨超10%的机会。相比最高点89元，已经跌去98%。Biotech可以犯几次错？开拓药业经历过3次III期临床失败，普克鲁胺蹭疫情热度失败了，现在连治疗前列腺癌、脱发的基本盘都丢失了。2023H1，亏损2亿元，现金储备7亿元，一年内或按要求偿还借款1亿元。还有逆天改命的奇迹吗？开拓药业股价冲天时，没有从新冠疫情取得收益，资本市场的迷人之处在于，画饼更能充饥，算出来的钱比拿到手的钱更值钱。生物科技领域，随着少数老板和背后运作团队做局能力的升维，画饼现在很难被识破。在一门遵循证据的科学中，任何高端局终将败露，早期临床数据不规范或有水分，但III期注册临床揭盲将飞出黑天鹅。0310亿大单品有多难？2024年，才是Biotech真正的商业化验证期，我们可能会发现一个单品要逾越10亿门槛有多难？随着支付政策对创新药倾斜，长周期大品种的销售峰值将边际改善，但在质变发生之前，动辄10亿到50亿的预测模型，是否过于理想化？Biotech商业化进程已出现危险信号，部分上市3年左右的明星药物有停止爬坡的迹象。再鼎医药核心产品PARP抑制剂则乐2022年刚刚跨过10亿大关，2023Q3销售额环比下降3.3%；荣昌生物ADC药物维迪西妥单抗2021年6月获批上市，至今距离10亿大关遥不可及，维迪西妥单抗+泰它西普2023前三季度合计销售额7.7亿元，付出的代价是5.4亿元销售费用。荣昌生物是一家令人困惑的公司，IPO两次，目前现金却只能撑半年，2023前三季度亏损10.3亿元，货币资金+交易性金融资产7.9亿元，自免重磅药物泰它西普或眼科重磅药物RC28可能不得不寻求对外BD，以补充现金流。中腰部Biotech摊子铺得太大，一旦造血能力没有跟上消耗，不排除阶层跌落的可能。04防御型资产2024年，传统药企将大举反攻。商业变现能力强、现金流充裕是基本盘，部分传统药企有了本质进化，对Biotech的玩法、套路都学得差不多了，在管线立项、BD能力上都日益成熟，进可攻，退可守。传统药企的创新模式主要有三种。一是以百洋医药、康哲药业为代表，基于对终端医疗场景的深刻洞察，以商业化基因指导前端管线立项。百洋医药集团承担产业投资人和孵化器的角色，在创新药、高端医疗器械、基础研究平台上广泛布局，创新产品培育成熟一个，优先考虑向上市公司输送一个，上市公司基本与创新风险隔离，并从源源不断注入的创新产品获得长期成长动力。二是以恒瑞医药为代表，对创新药所有高景气赛道全布局，ADC、GLP-1、自免、阿尔茨海默症、痛风无一遗漏，并且进度领先。三是以华东医药为代表，跨越多线，形成创新药（GLP-1、ADC）、医美（全产品线）、工业微生物的稳定架构。血制品依靠资源禀赋、刚需属性，龙头不仅是龙头，还会成为寡头，将成为跨越周期的防御型资产。主打平价替代、下沉市场的首仿和生物类似药将有强劲的市场需求。以仿制药为代表的板块业绩增速企稳，2023年利润规模有望达到集采以来的最高点。仿制药CRO龙头百诚医药、阳光诺和业绩增速亮眼，在手订单充沛，也验证了仿制药企盈利能力稳定、不受投融资环境影响的逻辑。部分仿制药企正向仿创升级，手握创新药大单品，进一步增加抗风险能力。05ADC继续最热中国企业部分ADC产品已具备BIC优势。目前，国内进入全球前三的ADC资产已经快被MNC抢光。据InScienceWeTrust BioAdvisory 统计，在过去两年，除罗氏以外，所有肿瘤领域的MNC都已完成ADC引进交易。中国Biotech成为全球早期ADC资产的主要来源，2023年，中国Biotech ADC对外授权交易的首付款总额是2022年的5倍 (57亿美元vs12亿美元)。InScienceWeTrust BioAdvisory创始人唐钧博士预计ADC将在2024年甚至更远的时间里继续成为肿瘤学最热门的领域。ADC赛道持续升温，乐普生物性价比凸显，研发进展不断，EGFR ADC新药MRG003获得FDA授予快速通道资格，用于治疗复发性或转移性鼻咽癌，TF靶向ADC新药MRG004A用于治疗胰腺癌获FDA授予孤儿药资格认定。06头部格局初定2015年，中国进入创新药元年，经历10年迷雾和混战，各个细分领域的头部正在明朗。新生代创新药企已呈现两巨头格局。百济神州2024年创新药收入或将超过恒瑞，如果能平衡好研发与财务，市值有希望超过恒瑞医药。信达生物体现出超强的临床开发效率，在国内KRAS G12C新药竞争胶着状态中，上市许可申请率先获得受理。信达生物的受试者入组速度可能是国内最快之一，临床研究III期的患者招募期间，CRA（临床监查员）一周七天可能都在出差，每天在医院工作到十二点。猎头专门来挖信达这批CRA，已经出去的CRA每介绍一个项目组内的CRA成功入职就能拿到3000美金的推荐费。12月26日，信达生物启动GLP-1双靶点减肥药玛仕度肽头对头挑战司美格鲁肽的III期临床试验，如果有战胜司美格鲁肽的成绩背书，玛仕度肽成为中国药王将无悬念。创新药有多个高景气赛道，以下只分析少数，并且广泛布局的综合型药企不再重复列举。血液瘤领域头部为诺诚健华、亚盛医药，这一赛道特点是存量患者多，生存期长，药物有较长销售爬坡期，容易诞生爆款。肾科领域头部为云顶新耀、信瑞诺医药，这一赛道特点是患者基数大且多为35岁以下，为避免肾功能衰竭，有着急迫的治疗需求。自免领域头部为康诺亚、康方生物、三生国健、诺诚健华、再鼎医药、泽璟制药、荃信生物，这个赛道的特点是自免疾病之间存在共患率，一款创新药甚至可以捆绑约10个适应症，一旦首个适应症获批，就有成为集束炸弹的可能，2024年，首个国产白介素家族、首个国产JAK抑制剂新药将获批上市。阿尔茨海默症领域存在预期差，国产药物布局较弱，临床诊断的商业价值可能更高。Science最近一篇论文提到，Aβ单抗治疗阿尔茨海默症，为确保安全性，全程检查费是药费的3倍。阿尔茨海默症检测金标准是 PET/CT 脑显像或脑脊液检测，但脑脊液检测需要穿刺，侵入性低的PET/CT 脑显像将成为主流，东诚药业靶向 Aβ的18F-洛贝平注射液已向CDE 申报上市，Tau 蛋白PET示踪剂18F-Florzolotau处于全球 III 期临床阶段，有望复制福瑞股份在NASH检测领域的逻辑。血液生物标志物检测是最新趋势，侵入性低，成本低，可及性更高，结合淀粉样斑块、Tau 病理、神经损伤共三种血液标志物来识别评估与阿尔茨海默症相关的生物学变化。亚辉龙、诺唯赞、热景生物、金域医学和迪安诊断正在布局 AD 血液检测试剂和诊断服务市场。2024H1，礼来靶向Aβ单抗Donanemab获批上市将成为赛道催化剂。医药生物即将超越银行，成为A股行业市值第一，其内部有着复杂多变的现实，极寒极热并存，风险机会共生，2024年将是一个重要节点。END作者 | 阿基米德君来源 | 阿基米德Biotech医药代表交流群扫描下方二维码加入医药代理商转型交流群扫描下方二维码加入

IPO临床3期引进/卖出

100 项与普克鲁胺相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	普克鲁胺	-	DB16065

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
新型冠状病毒感染	巴拉圭	苏州开拓药业股份有限公司	2021-07-16

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
新型冠状病毒感染	临床2期	巴西	Sutro Biopharma, Inc.	2020-11-01
新型冠状病毒感染	临床2期	巴西	Applied Biology Co. SAS	2020-11-01
严重急性呼吸综合征	临床2期	巴西	Applied Biology Co. SAS	2020-11-01
转移性去势抵抗性前列腺癌	临床2期	中国	中国药科大学	2019-04-30
转移性去势抵抗性前列腺癌	临床2期	中国	苏州开拓药业股份有限公司	2019-04-30
新型冠状病毒感染	药物发现	巴西	Applied Biology Co. SAS	2020-11-01
新型冠状病毒感染	药物发现	巴西	Sutro Biopharma, Inc.	2020-11-01
严重急性呼吸综合征	药物发现	巴西	Applied Biology Co. SAS	2020-11-01
转移性去势抵抗性前列腺癌	药物发现	中国	中国药科大学	2019-04-30
转移性去势抵抗性前列腺癌	药物发现	中国	苏州开拓药业股份有限公司	2019-04-30

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临床结果

适应症

分期

评价

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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


NCT03899467 (CTgov)	临床2期	转移性去势抵抗性前列腺癌	61	GT0918 (Arm 1: 400 mg /Day of GT0918)	(糧簾齋鹹構遞網繭窪築) = 壓簾選膚鹽蓋遞積壓醖製廠願構鬱顧淵衊壓艱 (窪窪獵範顧壓選鹽淵鹹, 壓衊鹹觸積顧觸淵鹽齋 ~ 衊衊構壓繭獵夢顧壓繭) 更多	-	2024-01-02
				GT0918 (Arm 2: 500 mg/Day of GT0918)	(糧簾齋鹹構遞網繭窪築) = 膚膚憲構鑰糧願願廠願製廠願構鬱顧淵衊壓艱 (窪窪獵範顧壓選鹽淵鹹, 鏇遞齋壓製構鑰鹹簾壓 ~ 鹽製糧製衊鑰觸鹽艱積) 更多
NCT04870606 (MRCT, CTgov)	临床3期	新型冠状病毒感染	733	Proxalutamide (GT0918) (GT0918+ Standard of Care)	鑰夢夢範顧鑰鏇繭獵鏇(繭齋範鹹遞積襯願鹽憲) = 廠憲鬱醖顧衊遞選鏇範淵鬱獵壓觸築廠蓋淵鬱 (製淵蓋積鏇淵壓鏇膚繭, 築衊齋夢顧鹹顧繭醖遞 ~ 蓋衊願蓋夢襯廠齋淵構) 更多	-	2024-01-02
				Placebo (Placebo+ Standard of Care)	鑰夢夢範顧鑰鏇繭獵鏇(繭齋範鹹遞積襯願鹽憲) = 築衊顧夢鑰廠網獵積築淵鬱獵壓觸築廠蓋淵鬱 (製淵蓋積鏇淵壓鏇膚繭, 衊夢膚鹹積築淵積壓窪 ~ 築鏇憲衊夢餘遞觸壓選) 更多
CTR20191063 (ESMO2023) 人工标引	临床1期	AR阳性/ER阳性/HER2阴性乳腺癌 HR positive \| HER2 negative \| AR positive	37	proxalutamide plus fulvestrant	(築憲夢蓋壓鬱廠糧顧鹽) = 構顧淵鬱網願製餘繭蓋選壓選夢鹽網廠襯糧繭 (鏇壓鏇鹽蓋構憲繭鏇觸 ) 更多	积极	2023-10-21
				proxalutamide plus fulvestrant (part 1)	(襯鏇築選鏇淵願構願築) = 壓鹹糧憲觸顧糧餘鏇製襯繭範夢選構鹽鏇齋衊 (膚壓選糧網襯壓觸淵壓 ) 更多
NCT04103853 \| CTR20170757 (Pubmed)	临床1期	转移性乳腺癌	63	Proxalutamide 200 mg	(壓製淵選衊網蓋鹹構廠) = 鏇繭顧窪窪繭簾築壓鹽餘餘窪鹽構糧鑰憲鹽鏇 (糧餘醖鹹網網壓夢願鑰, 11.9 ~ 39.4) 更多	-	2022-09-28
				Proxalutamide 300 mg	(壓製淵選衊網蓋鹹構廠) = 繭範襯壓觸憲醖齋簾選餘餘窪鹽構糧鑰憲鹽鏇 (糧餘醖鹹網網壓夢願鑰, 11.9 ~ 39.4) 更多
NCT04870606 (MRCT, NEWS) 人工标引	临床3期	新型冠状病毒感染	733	Standard of Care+Proxalutamide	艱醖簾窪衊鏇築鑰鹽蓋(鹽壓製壓鬱選築艱淵夢) = reduced the risk of hospitalization/death by100% 鏇襯餘鹽製鏇選範鹽蓋 (醖鏇製餘鹽襯夢遞糧膚 ) 更多	积极	2022-04-06
				Standard of Care+Placebo
NCT04728802 \| NCT05126628 (Pubmed) 人工标引	临床3期	新型冠状病毒感染	778	Proxalutamide	壓遞鹽鹹繭範範築膚餘(蓋顧鑰齋簾網淵繭簾餘) = 齋壓憲膚鏇觸顧衊鏇窪艱鹽壓鹹鏇製願蓋構艱 (壓憲選鑰鏇膚顧顧壓蓋 ) 更多	积极	2021-12-25
				Placebo	壓遞鹽鹹繭範範築膚餘(蓋顧鑰齋簾網淵繭簾餘) = 簾顧鹽襯鬱築鹹觸齋獵艱鹽壓鹹鏇製願蓋構艱 (壓憲選鑰鏇膚顧顧壓蓋 ) 更多
CSCO2021	临床2期	转移性去势抵抗性前列腺癌	61	GT0918	範糧膚選鏇衊鬱壓願願(廠築製構醖餘獵襯鏇窪) = 壓蓋鹽齋襯鑰顧網蓋繭觸選廠鹹顧淵醖積遞顧 (糧艱簾網鑰鏇觸壓網願, 99.5 ~ 150)	-	2021-09-25
CSCO2021	临床2期	转移性去势抵抗性前列腺癌	108	GT0918 therapy, 100 mg/day	(廠蓋遞鹹鏇繭襯選網夢) = 憲壓網夢鏇膚鬱襯襯膚簾繭糧構繭製鏇鏇鑰壓 (網範餘選簾蓋製網網醖, 240 ~ 477) 更多	-	2021-09-25
NCT04446429 (Pubmed \| Front Med (Lausanne)) 人工标引	临床2期	新型冠状病毒感染	268	proxalutamide	(構願構蓋艱簾糧顧繭築) = 鏇鏇憲襯壓膚範淵鬱艱廠夢製觸蓋廠憲簾範鏇 (夢顧鬱餘遞鏇窪願鏇繭 )	积极	2021-07-19
				Placebo	(構願構蓋艱簾糧顧繭築) = 衊壓繭獵積糧選醖糧繭廠夢製觸蓋廠憲簾範鏇 (夢顧鬱餘遞鏇窪願鏇繭 ) 更多
NCT04728802 (CTgov)	临床3期	新型冠状病毒感染 \| 严重急性呼吸综合征	645	Proxalutamide (Proxalutamide + Usual Care)	願糧積衊繭艱廠鑰網鬱(繭築簾選憲積窪顧齋醖) = 夢鑰艱製鹹蓋構淵築選憲簾築襯鹽積鹽廠簾觸 (製顧夢壓廠艱窪醖願夢, 顧範積糧鏇淵蓋遞膚醖 ~ 壓膚淵鬱憲憲淵膚餘廠) 更多	-	2021-06-23
				Placebo (Placebo + Usual Care)	願糧積衊繭艱廠鑰網鬱(繭築簾選憲積窪顧齋醖) = 鏇憲憲築艱憲齋簾窪鑰憲簾築襯鹽積鹽廠簾觸 (製顧夢壓廠艱窪醖願夢, 衊築簾製鑰選窪廠憲願 ~ 廠鏇醖鹹糧憲窪襯鏇構) 更多