A Phase I, Dose Escalation and Dose Expansion, Safety and Tolerability Study of OnCARlytics (CF33-CD19), Administered Intravenously or Intratumorally in Combination with Blinatumomab in Adults with Advanced or Metastatic Solid Tumors (OASIS)

This is an open-label, dose escalation and dose expansion, multi-center phase I study evaluating the safety and tolerability of CF33-CD19 administered intravenously (IV) or intratumorally (IT) in combination with blinatumomab in adults with advanced or metastatic solid tumors.

开始日期2023-10-02

申办/合作机构

Imugene Ltd.

NCT05311176 / Completed临床2期

NextHERIZON: an Open-Label, Signal Generating, Phase 2 Study of HER-Vaxx in Combination with Chemotherapy or Pembrolizumab in Patients with Metastatic HER2/Neu Over-Expressing Gastric or Gastroesophageal Junction (GEJ) Adenocarcinomas Who Have Previously Received Trastuzumab and Progressed on This Treatment

This is a Phase 2, signal generating, open-label, 2-Arm, non-randomized study, in patients with metastatic HER2/neu over-expressing gastric cancer or gastroesophageal adenocarcinomas.

开始日期2022-08-17

申办/合作机构

Imugene Ltd.

NCT05346484 / Recruiting临床1期

A Phase I, Dose Escalation Safety and Tolerability Study of VAXINIA (CF33-hNIS), Administered Intratumorally or Intravenously as a Monotherapy or in Combination With Pembrolizumab in Adult Patients With Metastatic or Advanced Solid Tumors (MAST).

This is an open-label, dose-escalation, multi-center phase I study evaluating the safety of CF33-hNIS (hNIS - human sodium iodide symporter) administered via two routes of administration, intratumoral (IT) or intravenous (IV), either as a monotherapy or in combination with pembrolizumab in patients with metastatic or advanced solid tumors.

开始日期2022-05-17

申办/合作机构

Imugene Ltd.

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项与 Imugene Ltd. 相关的文献（医药）

2024-09-13·Clinical Cancer Research

Phase II Trial of HER-Vaxx, a B-cell Peptide-Based Vaccine, in HER2-Overexpressing Advanced Gastric Cancer Patients Under Platinum-Based Chemotherapy (HERIZON)

Article

作者: Wiedermann, Ursula ; Chawla, Tanuj ; Chong, Leslie Mi Ok ; Nikolić, Ivan ; Petrović, Zoran ; Ryspayeva, Dinara ; Yavrom, Sharon ; Nixon, Bonnie ; Ede, Nicholas J. ; Kundi, Michael ; Andrić, Zoran ; Maglakelidze, Marina ; Bulat, Iurie ; Garner-Spitzer, Erika ; Zielinski, Christoph C. ; Nagarkar, Rajnish ; Tobias, Joshua

AbstractPurpose:A multicenter, randomized, open-label, phase II study (HERIZON; NCT02795988) was conducted to evaluate the clinical and immunologic efficacy of HER-Vaxx (IMU-131), a B-cell, peptide-based vaccine targeting HER2 overexpressed in 6% to 30% of gastroesophageal adenocarcinomas (GEA).Patients and Methods:Patients (n = 36) with GEA were treated with standard-of-care chemotherapy (n = 17) or HER-Vaxx plus chemotherapy (n = 19), using the recommended phase 2 dose for the vaccine. Overall survival (OS; primary endpoint), safety, progression-free survival (PFS), clinical response (secondary endpoints), and vaccine-induced HER2-specific antibody levels in serum and correlation with tumor response rates (exploratory endpoints) were investigated.Results:A 40% OS benefit [HR, 0.60; median OS, 13.9 months; 80% confidence interval (CI), 7.52–14.32] for patients treated with HER-Vaxx plus chemotherapy compared with OS of 8.31 months (80% CI, 6.01–9.59) in patients that received chemotherapy alone. A 20% PFS difference was obtained for the vaccination arm (HR, 0.80; 80% CI, 0.47, 1.38). No additional toxicity due to HER-Vaxx was observed. The vaccine-induced high levels of HER2-specific total IgG and IgG1 antibodies (P < 0.001 vs. controls) that significantly correlated with tumor reduction (IgG, P = 0.001; IgG1, P = 0.016), had a significant capacity in inhibiting phosphorylation of the intracellular HER2-signaling pathways, mediated antibody-dependent cellular cytotoxicity, and decreased immunosuppressive FOXP3+ regulatory T cells.Conclusions:HER-Vaxx plus standard chemotherapy exhibits an excellent safety profile and improves OS. Furthermore, vaccine-induced immune response was significantly associated with reduced tumor size compared with standard-of-care chemotherapy. The presented vaccination approach may substitute for treatment with trastuzumab, upon unavailability or toxicity, based on further evidence of equivalent treatment efficacy.

2024-04-05·Cancer Research

Abstract CT182: Oncolytic virus CF33-hNIS for the treatment of advanced cancer

作者: Wilkinson, Grey A. ; Lim, Emerson ; Shields, Anthony F. ; Spira, Alex ; Leddon, Jennifer ; Li, Daneng ; Fein, Seymour ; Merchan, Jaime ; Flores, Oscar ; Hamilton, Stanley ; Wright, Gavin ; Austria, Janet ; Mamdani, Hirva ; Daniels, Gregory ; Akerley, Wallace ; Byon, John ; Yavrom, Sharon ; Woodard, Paul ; Egelston, Colt ; Kumar, Abhijeet ; Travis, Patrick ; Seiz, Amanda

AbstractBackground: CF33-hNIS is a novel vaccinia virus engineered with the human sodium-iodide symporter (hNIS) gene. CF33-hNIS selectively replicates in tumor cells and promotes anti-tumor immunity. Here, we report results on a first-in-human phase 1 dose escalation study of CF33-hNIS, administered intratumorally (IT) or intravenously (IV) in adult patients with metastatic or advanced solid tumors (MAST).Methods: The MAST study is evaluating the safety of CF33-hNIS administered IT or IV, alone or in combination with pembrolizumab in patients with advanced or metastatic solid tumors with ≥ 2 prior lines of therapy (NCT05346484). CF33-hNIS is administered in 21-day cycles on C1D1 and C1D8, then D1 of each cycle thereafter. Pembrolizumab begins C2D1 for the combination groups and is administered Q3W. The study consists of two parts. Part 1 follows a 3+3 dose escalation scheme independent of each route of CF33-hNIS administration (IT and IV) and for each therapy regimen (monotherapy and combination therapy) with up to 7 dose levels of CF33-hNIS ranging from 8.6x105 to 3.0x109 PFU. Part 2 is a cohort expansion in select indications at the optimal dose. The co-primary endpoints are safety and identification of the recommended phase 2 dose. Secondary endpoints include objective response rate according to RECIST v1.1 and iRECIST, and assessment of viral replication in tumor lesions via Single-Photon Emission Computerized Tomography (SPECT).Results: As of Dec 2023, 36 patients have been treated in Part 1 of the study with a median age of 59 (range 23-81). Twenty-two (61%) patients were heavily pretreated with ≥3 prior lines of systemic therapy, and 14 (39%) received prior treatment with checkpoint blockade. Treatment-related adverse events consisted predominantly of ‘flu-like’ symptoms, such as grade 1/2: fatigue (31%), pyrexia (22%), and chills (14%). No dose-limiting toxicities have occurred. The highest dose treated to date is 1.1x108 PFU by IT and IV delivery; dose escalation continues. In the IT cohorts (14 patients), 7 of 15 (47%) injected lesions had a reduction in tumor burden, three lesions were completely eradicated. Three patients (21%) had an objective response: one complete response by iRECIST in a patient with cholangiocarcinoma; and two partial responses in patients with melanoma by RECIST. In IV cohorts (17 patients), 53% of patients achieved stable disease as their best response. Patients who received prior checkpoint blockade therapy derived clinical benefit with and without pembrolizumab. Viral replication, assessed by SPECT was higher in patients that saw a reduction in tumor burden. Immunological changes in peripheral blood and tumor biopsies will be presented.Conclusions: CF33-hNIS alone or in combination with pembrolizumab is a safe treatment option for advanced cancer patients. Encouraging efficacy has warranted advancement to part 2 of this study with a cohort expansion of patients with cholangiocarcinoma and other tumor types.Citation Format: Daneng Li, Anthony F. Shields, Hirva Mamdani, Patrick Travis, Gavin Wright, Wallace Akerley, Alex Spira, Gregory Daniels, Jaime Merchan, Abhijeet Kumar, Emerson Lim, Colt Egelston, Oscar Flores, Stanley Hamilton, Janet Austria, Amanda Seiz, Sharon Yavrom, Seymour Fein, John Byon, Paul Woodard, Grey A. Wilkinson, Jennifer Leddon. Oncolytic virus CF33-hNIS for the treatment of advanced cancer [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 2 (Late-Breaking, Clinical Trial, and Invited Abstracts); 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84(7_Suppl):Abstract nr CT182.

2024-04-05·Cancer Research

Abstract CT215: Frontline vaccination with the B-cell peptide compound HER-Vaxx (IMU-131), combined with standard-of-care chemotherapy induces high levels of HER2-specific antibodies mediating ADCC and intracellular phosphorylation inhibition resulting in overall survival benefit in patients with HER2+ metastatic or advanced gastric/GEJ adenocarcinoma - Final results from Phase II/HERIZON study

作者: Nixon, Bonnie ; Andric, Zoran ; Zielinski, Christoph ; Garner-Spitzer, Erika ; Nagarkar, Rajnish ; Wiedermann, Ursula ; Ede, Nicholas J. ; Petrovic, Zoran ; Maglakelidze, Marina ; Kundi, Michael ; Chong, Leslie Mi Ok ; Chawla, Tanuj ; Yavrom, Sharon ; Tobias, Joshua

AbstractBackground: HER2, a member of the epidermal growth factor receptor (EGFR) family, is overexpressed in approximately 30% of gastric cancers. HER-Vaxx is a B cell peptide-based anti-HER2 vaccine (IMU-131) comprising trastuzumab’s binding site. Here, we present the results of the randomized open-label, multicenter phase 2 HERIZON trial (NCT02795988), which compared HER-Vaxx plus chemotherapy to chemotherapy alone. The study aimed to evaluate clinical (primary endpoint) and antibody (secondary endpoint) responses in patients with HER2-overexpressing gastric/gastro-esophageal junction (GC) cancer naïve to anti HER2 therapy.Methods: Patients were randomized to chemotherapy alone (n=17) or HER-Vaxx (50µg, recommended phase 2 dose) plus chemotherapy (n=19). In the HER-Vaxx group, patients received the vaccine at days 0, 14, 35, and 77, then every 63 days until disease progression. Chemotherapy consisted of oxaliplatin plus capecitabine and was started at day 0, repeated every 21 days for a maximum of 6 cycles. The clinical responses were assessed by RECIST 1.1 criteria. FACS analysis of PBMCs for immunophenotyping was carried out, as well as in vitro phosphorylation inhibition assays of HER2 and the signaling pathway kinases Akt and MAPK, and mediation of ADCC to characterize the vaccine-induced IgG antibodies.Results: A 42% survival benefit for patients treated with HER-Vaxx plus chemotherapy [median overall survival: 13.9 months (7.5, 14.3)] compared to chemotherapy alone [mOS: 8.3 months (6.0, 9.6)] was shown, which translated into an OS HR of 0.580 (80% 2-sided CI: 0.362, 0.927). A highly significant HER2-specific IgG and IgG1 antibody response at all time points, particularly after 3 or more doses of HER-Vaxx were induced (P<0.001). The HER-Vaxx antibody response correlated well with mediating ADCC (IgG, P=0.003; IgG1, P=0.05) and the anti-tumor effect (IgG, P=0.001; IgG1, P=0.016). Moreover, the HER-Vaxx-induced IgG antibodies exhibited binding to the gastric cancer cell line N87, with capacity to inhibit HER2 signaling pathway kinases Akt and MAPK phosphorylation. It also demonstrated decreased levels of Foxp3+ Tregs (P=0.0013).Conclusions: The previously shown safety profile of HER-Vaxx, along with the improved clinical and antibody responses in HER2-overexpressing GC patients shown here, validate the proof of concept for a first-in-class B-cell immunotherapy based on HER2 B-cell peptides. Clinical trials evaluating further treatment with HER-Vaxx in HER2+ GC are ongoing.Citation Format: Joshua Tobias, Michael Kundi, Erika Garner-Spitzer, Christoph Zielinski, Marina Maglakelidze, Zoran Andric, Zoran Petrovic, Rajnish Nagarkar, Tanuj Chawla, Leslie Mi Ok Chong, Bonnie Nixon, Sharon Yavrom, Nicholas J. Ede, Ursula Wiedermann. Frontline vaccination with the B-cell peptide compound HER-Vaxx (IMU-131), combined with standard-of-care chemotherapy induces high levels of HER2-specific antibodies mediating ADCC and intracellular phosphorylation inhibition resulting in overall survival benefit in patients with HER2+ metastatic or advanced gastric/GEJ adenocarcinoma - Final results from Phase II/HERIZON study [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 2 (Late-Breaking, Clinical Trial, and Invited Abstracts); 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84(7_Suppl):Abstract nr CT215.

项与 Imugene Ltd. 相关的新闻（医药）

2024-10-24

·EndPoints

Exclusive: Moldova clears first gene editing study for HBV in humans

Precision Biosciences has received clearance from regulators in the Eastern European country of Moldova to begin a human trial of a gene editor for chronic hepatitis B, marking the first such test in the world. Precision’s ultimate goal is to use gene editing to functionally cure chronic hepatitis B, which to date is treatable but not curable. It’s a herculean task — a cure thus far has been elusive because the hepatitis B virus hides in a stable form in liver cells where it continues to make more copies of itself. “This is a disease indication where new therapies have really had a challenge, and patients have really been looking for better treatment options for a really long time,” Precision CEO Michael Amoroso said. People with chronic HBV can receive daily antiviral pills and interferon injections to treat the disease. Functional cure rates are around 2% on the antiviral pills alone and around 10% when combined with the injections but with significant side effects. The company’s Phase 1 trial will recruit from Moldova and up to four other countries. Amoroso declined to name those countries in an interview ahead of the announcement, though he confirmed Precision is pursuing an IND in the US. Precision uses its own gene editing platform it calls ARCUS, which was developed by North Carolina scientists who founded the company in 2006. Instead of CRISPR/Cas9, ARCUS uses an editing enzyme derived from algae. For chronic hepatitis B, Precision calls its experimental therapy PBGENE-HBV. The gene editor is delivered via a fatty acid envelope called a lipid nanoparticle, which accumulates in the liver. The gene editor is targeted to a sequence in HBV DNA that codes for the virus’ polymerase, which is an enzyme that helps make more copies of viral DNA. In doing so, Precision hopes gene editing can eliminate two forms of the hepatitis B viral DNA in liver cells that other approaches can’t get to: covalently closed circular DNA, or cccDNA, as well as HBV DNA that has integrated into the host DNA. cccDNA is a feature of certain viruses including HBV where its genetic code makes a stable ring inside the cell, and it also makes the virus very hard to cure. “The factory that makes replicating virus is that cccDNA,” Amoroso said. “So we’re not looking to cure symptoms, we’re looking to go at the root cause.” In May, Excision BioTherapeutics reported that its attempt to gene edit HIV failed. Like Precision, Excision’s goal is to use gene editing to excise hidden viruses from cells. However, there are some key differences — including that HIV is found in cells beyond the liver and Excision used viral envelopes called AAVs to try to reach those cells. Excision is likewise developing a gene editing therapy for HBV, for which it hopes to start clinical studies in 2026. Beam Therapeutics was also working on a gene editing approach to HBV but the company stopped work on the program in October last year as part of larger cost cuts and layoffs. It’s been a long road to this moment for Precision, which once developed CAR-T therapies before offloading that work to Imugene and in April saw Eli Lilly end a partnership on three programs. Gilead had signed on to Precision’s hepatitis B program in 2018, but axed the partnership in 2020, returning the rights to the gene editing company. In a May note to investors, BMO Capital Markets analyst Kostas Biliouris wrote that Precision “remains a ‘show me’ story wherein positive clinical data from the PBGENE-HBV program are needed to improve investor sentiment.”

基因疗法

2024-09-17

·FiercePharma

CDMO Kincell inks deal with new client to expand work at Florida cell therapy facility

Cell therapy CDMO Kincell Bio has brought on an unnamed client to a multi-year deal to manufacture personalized, clinical-grade regenerative treatments. Kincell Bio, a CDMO focused on producing cell therapies, signed an unnamed client to a multi-year deal to manufacture personalized, clinical-grade regenerative treatments.The partnership is slated to add work at Kincell's site in Gainesville, Florida, the company said in a Sept. 16 press release. The client was described by Kincell as a clinical-stage cell therapy company investigating regenerative medicines for a variety of “critical” indications. Financial details of the deal weren’t disclosed.“We’re delighted to welcome another client to our Gainesville facility,” Bruce Thompson, Kincell’s chief executive, said in the release. “Our facility is GMP-ready, and our team thrives on providing critical CMC development and manufacturing expertise to support our clients’ clinical development plans to help patients.” Back in April, Kinsell said it would shell out $6 million over the next three years and kick in milestone-driven payments to take over immuno-oncology biotech Imogene’s North Carolina cell manufacturing facility and hire the site’s associated staffers.Under that deal, Kincell picked up a new 32,800-square-foot site in Research Triangle Park. Imugene, for its part, aimed to save $32 million through staff cost cuts, manufacturing efficiencies and overhead savings.

细胞疗法

2024-09-08

·药明康德

改善视力指标100倍的基因疗法；达完全缓解的CAR-T疗法…… | 一周盘点

▎药明康德内容团队编辑本期看点 1. 现货型同种异体嵌合抗原受体（CAR）-T细胞疗法azer-cel在一项1b期临床试验中使3名复发/难治性弥漫性大B细胞淋巴瘤（DLBCL）患者获得完全缓解（CR），CR率为33%。 2. 治疗因GUCY2D双等位基因突变引起的Leber先天性黑蒙（LCA1）患者的在研基因疗法ATSN-101的早期临床试验结果积极，高剂量组患者的一项视力指标改善了100倍。 3. 治疗杜氏肌营养不良（DMD）的在研抗体偶联寡核苷酸疗法DYNE-251在一项早期临床试验中使多个队列患者的抗肌萎缩蛋白表达达到前所未有的水平，且患者的临床功能获得改善。药明康德内容团队整理 Azer-cel：公布1b期临床试验数据 Imugene公司宣布，其现货型同种异体CD19靶向CAR-T细胞疗法azercabtagene zapreleucel（azer-cel）治疗复发/难治性弥漫性大B细胞淋巴瘤（一种非霍奇金淋巴瘤）的1b期临床试验取得了令人鼓舞的初步结果。该研究针对的是接受自体CAR-T细胞疗法治疗后复发的DLBCL患者，所有患者在试验前均接受过4-5线治疗。截至目前，已有10名患者接受了azer-cel治疗，A队列的6名患者接受了azer-cel+清淋化疗治疗，B队列的4名患者接受了azer-cel+清淋化疗+白介素-2（IL-2）治疗。研究结果显示，所有接受azer-cel和清淋化疗的患者都显示出可接受的安全性。此外，azer-cel+清淋化疗+IL-2的治疗在DLBCL患者中显示出了临床上有意义的疗效和持续性。在所有接受治疗的患者中，总缓解率为44%，其中3名患者达到了CR，1名患者达到了部分缓解（PR）。队列B中获得CR的两名患者已分别持续缓解超过90天和120天。目前，B队列的4名患者均仍在试验中，1名患者在等待评估。 ▲Azer-cel的1b期研究结果（图片来源：参考资料[1]）除了研究azer-cel在血液癌症中的疗效外，Imugene公司还计划将azer-cel与其新颖的onCARlytics结合，用于治疗实体肿瘤患者。 ATSN-101：公布1/2期临床试验数据 Atsena Therapeutics公司公布了其在研基因疗法ATSN-101治疗因GUCY2D双等位基因突变引起的Leber先天性黑蒙患者的1/2期临床试验数据。ATSN-101是一种以腺相关病毒5（AAV5）为载体的基因治疗药物，通过视网膜下注射将功能性的人类GUCY2D基因引入光感受器。ATSN-101已获得美国FDA授予的罕见儿科疾病认定、再生医学先进疗法认定和孤儿药资格，用于治疗GUCY2D相关LCA1。该研究中，15名经过确认的LCA1患者接受了单侧视网膜下注射，以确定ATSN-101的安全性和初步有效性。结果显示，在高剂量组中，暗适应全视场刺激测试（FST）的平均变化为20.3 dB，这意味着接受治疗眼睛的暗适应FST改善了100倍。这种改善在第28天时首次被观察到，并持续了12个月（p=0.012）。在最佳矫正视力（BCVA）方面也观察到轻微的改善，治疗后第12个月时高剂量组的平均改善为0.16 logMAR，或相当于视力表上的8个字母（p=0.10）。安全性方面，所有不良事件的严重程度均为轻度（91%）或中度（9%）。没有报告与药物相关的严重不良事件，且大多数治疗伴发不良事件与手术过程相关。眼部炎症为轻度，并可通过类固醇治疗逆转。 DYNE-251：公布1/2期临床试验的新数据 Dyne Therapeutics公司公布了其进行中的1/2期试验DELIVER的最新临床数据。该试验旨在评估其在研抗体偶联寡核苷酸疗法DYNE-251在适合进行外显子51跳跃治疗的杜氏肌营养不良患者中的疗效与安全性。DYNE-251由磷酸二氨基甲酰吗啉寡核苷酸（PMO）与靶向转铁蛋白受体1（TfR1）的抗体片段（Fab）偶联而成，TfR1在肌肉组织中高度表达。该疗法旨在实现靶向肌肉组织递送，促进核内外显子跳跃，从而使肌肉细胞生成截短但功能正常的抗肌萎缩蛋白，目标是停止或逆转疾病的进展。此次公布的结果显示，在接受DYNE-251治疗后，多个队列患者的抗肌萎缩蛋白表达达到前所未有的水平，且患者的临床功能获得改善。接受20 mg/kg剂量治疗患者的平均绝对抗肌萎缩蛋白表达为正常值的3.71%（未根据肌肉含量进行调整），比接受每周一次标准治疗的临床试验中所报告的0.3%高出10倍以上。接受20 mg/kg和10 mg/kg剂量治疗的患者在多个临床功能终点均有显著改善，包括北极星步行评估（NSAA）、步速95百分位数（SV95C）、10米步行/跑步时间（10-MWR）、从地面起身的时间。在试验6个月到12个月期间，10 mg/kg组患者在所有的指标中均显示出持续改善。基于这些积极数据和与监管单位讨论的结果，Dyne正在准备启动DELIVER试验中的注册性队列。 VAX-31：公布1/2期临床试验数据 Vaxcyte公司宣布，其候选31价肺炎球菌结合疫苗VAX-31在一项针对1015名50岁及以上健康成人的1/2期临床试验中取得了积极的顶线结果。基于这一积极结果，该公司已决定将VAX-31推进至成人3期临床试验。在这项1/2期研究中，VAX-31在所有研究剂量中均显示出良好的耐受性，并在整个六个月的评估期间展现了与获批20价肺炎球菌结合疫苗相似的安全性。VAX-31在所有研究剂量中对所有31种血清型均表现出强劲的调理吞噬作用（OPA）免疫反应。在中剂量和高剂量下，VAX-31与获批疫苗相比，针对两种疫苗共享的20种血清型，均达到或超过了OPA反应非劣效性标准。高剂量的VAX-31与获批20价疫苗相比，20种血清型中18种的平均OPA免疫反应更强，其中7种血清型的免疫反应显著高于活性对照。对于VAX-31独有的11种血清型，所有三种剂量与获批20价疫苗相比均达到了优效性标准。 JNT-517：公布1/2期临床试验的新数据 Jnana Therapeutics公司公布了其在研疗法JNT-517治疗苯丙酮尿症（PKU）成人患者的1/2期临床试验中的第二剂量组（150 mg，每日两次）的试验结果。JNT-517是一种针对苯丙氨酸（Phe）转运蛋白SLC6A19的小分子抑制剂，为潜在“first-in-class”口服疗法，适用于所有年龄段和基因型的PKU患者。此次公布的150 mg组数据基于18名受试者，其中11人服用JNT-517，7人服用安慰剂，历时28天。结果显示，JNT-517在第14、21、28天将患者平均血液Phe水平较基线降低了60%（p<0.0001）。相比之下，之前研究的75 mg剂量组在相同时间段内的平均血液Phe水平降低了44%。患者在用药开始后7天内观察到快速的疗效，血液Phe水平显著降低，并在28天的用药期间持续维持。无论基线Phe水平或之前的PKU治疗史如何，所有参与者均表现出显著的疗效。安全性方面，JNT-517耐受性良好，无严重不良事件，无临床显著的实验室参数变化，除Phe外，未观察到血液氨基酸的临床显著变化，与75 mg剂量组和1a期健康志愿者研究的安全性一致。基于这些积极结果，该公司计划在2025年初启动关键性3期临床试验，评估JNT-517治疗PKU的效果。 BX004：公布1b/2a期临床试验的新数据 BiomX公司公布了其用于治疗铜绿假单胞菌（PsA）引起慢性肺部感染的囊性纤维化（CF）患者的固定多噬菌体组合疗法BX004的1b/2a期研究的新数据。PsA是引起CF患者肺部感染和死亡的主要因素。BX004旨在靶向和破坏PsA。此次公布的结果显示，在研究的第一部分，接受BX004-A鸡尾酒疗法的受试者在第15天时，其痰液中的PsA菌落形成单位（CFU）/g的减少幅度较基线相比更大，而安慰剂组没有这种效果。在试验的第二部分，14%的BX004-A治疗组受试者在第10天（治疗结束）时痰液中的PsA培养结果为阴性，而安慰剂组的这一比例为0%。此外，在持续使用相同吸入抗生素且未更换或交替使用抗生素、正在接受elexacaftor/tezacaftor/ivacaftor（ETI）标准治疗、1秒用力呼气量（FEV1）＜70%的肺功能较差的患者亚组中，BX004-A治疗组患者的肺功能（以FEV1衡量）增加了5.66%，而安慰剂组则下降了3.23%。此外，所有接受BX004-A治疗的受试者痰液中均检测到了噬菌体，治疗过程中未发现与治疗相关的噬菌体耐药性，也没有严重的不良事件。 APTN-101：IND申请获得FDA许可 Adaptin Bio公司宣布其主打项目APTN-101的IND申请已获美国FDA批准，该公司即将推动该候选药物的“first-in-human”临床1期试验，以评估其用于治疗胶质母细胞瘤（GBM）的疗效与安全性。APTN-101是该公司专有的BRiTE（大脑双特异性T细胞接合剂）疗法，设计靶向EGFRvIII分子，这是一种与侵袭性脑肿瘤相关的蛋白。在临床前研究中，APTN-101具有超过对照药物7倍的大脑分布增强的效果，并在消除恶性胶质瘤肿瘤方面表现强劲，具有成为“best-in-class”药物的潜力。 MDG1015：IND申请获得FDA许可 Medigene公司宣布，其主打项目MDG1015的IND申请已获得美国FDA的批准，可启动1期临床试验，针对晚期胃癌、卵巢癌、粘液样/圆细胞脂肪肉瘤和滑膜肉瘤患者。MDG1015是一种潜在“first-in-class”的第三代T细胞受体（TCR）工程化T细胞疗法，靶向NY-ESO-1/LAGE-1a，其TCR能够特异性识别靶点，同时具有较高的安全性和敏感性。这些TCR-T细胞通过添加专有的PD1-41BB共刺激开关蛋白（CSP）技术得到进一步增强，对表达不同水平PD-L1的肿瘤细胞均具有显著增强的抗肿瘤活性。与第一代TCR-T疗法相比，MDG1015的生产过程更快，只需要6天就可以扩增出治疗所需的细胞。这些细胞更年轻、更健康，在治疗时需要的细胞数量可能更少。整个治疗过程从采集患者的细胞到输注治疗的时间缩短到大约20天。此外，生成的MDG1015细胞几乎都是CD8+细胞，其中大约95%的细胞都具有类似干细胞的特质，这意味着它们可以在体内存活更长时间，提供更持续的治疗效果，同时可能减少不良事件。 OpCT-001：IND申请获得FDA许可 BlueRock Therapeutics公司宣布OpCT-001的IND申请已获得美国FDA的批准。OpCT-001是一种在研诱导多能干细胞（iPSC）衍生的细胞疗法，旨在治疗原发性光感受器疾病。原发性光感受器疾病是遗传性视网膜病变的一个亚组，影响视网膜中光感受器细胞的结构和功能，导致儿童和成人视力的不可逆丧失。新闻稿指出，OpCT-001是首个针对原发性光感受器疾病进行临床测试的iPSC衍生细胞疗法。 LA-GLA：IND申请获得FDA许可 Hanmi Pharmaceutical公司宣布，其用于治疗法布里病的候选药物LA-GLA的IND申请已获得美国FDA的批准。目前，大多数法布里病患者通过使用基因重组技术开发的酶替代疗法进行治疗，患者每两周需要到医院接受长时间的静脉注射。LA-GLA是一种每月一次皮下注射的下一代长效酶替代疗法，使用更具便利性。在非临床研究中，LA-GLA在改善肾功能、血管疾病和外周神经障碍方面显示出优于现有治疗药物的效果。基于这些成果，该药物于2024年5月获得了美国FDA授予的孤儿药资格。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看） [1] Three Complete Responses in Azer-Cel Allogeneic CD19 CAR T Phase 1b Trial in Blood Cancer (Diffuse Large B-Cell Lymphoma). Retrieved September 6, 2024, from https://app.sharelinktechnologies.com/announcement/asx/9ec9d95529ab3adb318296ee5b344164 [2] Cartesian Therapeutics Announces First Patient Dosed in First-In-Human Phase 1 Trial of Next-Generation mRNA CAR-T Cell Therapy Descartes-15. Retrieved September 6, 2024, from https://ir.cartesiantherapeutics.com/news-releases/news-release-details/cartesian-therapeutics-announces-first-patient-dosed-first-human [3] BlueRock Therapeutics announces FDA clearance of IND application for investigational iPSC-derived cell therapy OpCT-001 for the treatment of primary photoreceptor diseases. Retrieved September 6, 2024, from https://www.globenewswire.com/news-release/2024/09/03/2939564/0/en/BlueRock-Therapeutics-announces-FDA-clearance-of-IND-application-for-investigational-iPSC-derived-cell-therapy-OpCT-001-for-the-treatment-of-primary-photoreceptor-diseases.html [4] GC녹십자-한미, ‘파브리병 치료제’ 1/2상 “美 IND 승인” . Retrieved September 6, 2024, from GC녹십자-한미, ‘파브리병 치료제’ 1/2상 “美 IND 승인” [5] Dyne Therapeutics Announces New Clinical Data from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne Muscular Dystrophy Demonstrating Unprecedented Dystrophin Expression and Functional Improvement in Multiple Cohorts. Retrieved September 3, 2024 from https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-new-clinical-data-phase-12-deliver [6] BiomX to Present Positive Phase 1b/2a Clinical Trial Data for BX004 at the European Respiratory Society (ERS) Congress 2024. Retrieved September 3, 2024 from https://ir.biomx.com/news-events/press-releases/detail/120/biomx-to-present-positive-phase-1b2a-clinical-trial-data [7] Vaxcyte Reports Positive Topline Data from Phase 1/2 Study of VAX-31, its 31-Valent Pneumococcal Conjugate Vaccine Candidate, in Adults Aged 50 and Older. Retrieved September 3, 2024 from https://www.globenewswire.com/news-release/2024/09/03/2939548/0/en/Vaxcyte-Reports-Positive-Topline-Data-from-Phase-1-2-Study-of-VAX-31-its-31-Valent-Pneumococcal-Conjugate-Vaccine-Candidate-in-Adults-Aged-50-and-Older.html [8] Jnana Therapeutics Presents New Positive Data from Phase 1/2 Study of JNT-517 in Individuals with Phenylketonuria – Results Support Planned Pivotal Study in Early 2025. Retrieved September 4, 2024, from https://www.globenewswire.com/news-release/2024/09/04/2940409/0/en/Jnana-Therapeutics-Presents-New-Positive-Data-from-Phase-1-2-Study-of-JNT-517-in-Individuals-with-Phenylketonuria-Results-Support-Planned-Pivotal-Study-in-Early-2025.html [9] 7 Hills Pharma Safely Doses First Patient in Phase 1b/2a Clinical Trial Testing Alintegimod Against aPD-1-resistant Solid Tumors. Retrieved September 3, 2024 from https://www.globenewswire.com/news-release/2024/09/03/2940018/0/en/7-Hills-Pharma-Safely-Doses-First-Patient-in-Phase-1b-2a-Clinical-Trial-Testing-Alintegimod-Against-aPD-1-resistant-Solid-Tumors.html [10] Allyx Therapeutics Announces First Parkinson’s Disease Patient Treated with ALX-001. Retrieved September 3, 2024 from https://www.globenewswire.com/news-release/2024/09/04/2940463/0/en/Allyx-Therapeutics-Announces-First-Parkinson-s-Disease-Patient-Treated-with-ALX-001.html#:~:text=NEW%20HAVEN%2C%20Conn.%2C%20Sept.%2004%2C%202024%20%28GLOBE%20NEWSWIRE%29,potential%20therapeutic%20response%20in%20patients%20with%20Parkinson%E2%80%99s%20disease. [11] Boehringer Ingelheim announces plans to advance potential new treatment for Geographic Atrophy, following positive Phase I results. Retrieved September 6, 2024 from https://www.globenewswire.com/news-release/2024/09/05/2941540/0/en/Boehringer-Ingelheim-announces-plans-to-advance-potential-new-treatment-for-Geographic-Atrophy-following-positive-Phase-I-results.html [12] Atsena Therapeutics Announces 12-Month Safety and Efficacy Data from Phase I/II Clinical Trial of ATSN-101 in LCA1 Published in The Lancet. Retrieved September 6, 2024 from https://www.globenewswire.com/news-release/2024/09/05/2941896/0/en/Atsena-Therapeutics-Announces-12-Month-Safety-and-Efficacy-Data-from-Phase-I-II-Clinical-Trial-of-ATSN-101-in-LCA1-Published-in-The-Lancet.html [13] Medigene Announces U.S. FDA Clearance of IND Application for MDG1015, a First-in-Class, Third Generation T Cell Receptor Engineered T Cell (TCR-T) Therapy for Multiple Solid Tumor Indications. Retrieved September 6, 2024 from https://www.globenewswire.com/news-release/2024/09/05/2941272/0/en/Medigene-Announces-U-S-FDA-Clearance-of-IND-Application-for-MDG1015-a-First-in-Class-Third-Generation-T-Cell-Receptor-Engineered-T-Cell-TCR-T-Therapy-for-Multiple-Solid-Tumor-Indic.html [14] ALX Oncology Announces First Patients Dosed with Evorpacept and Sarclisa As Part of Randomized Phase 1/2 UMBRELLA Study with Sanofi. Retrieved September 6, 2024 from https://ir.alxoncology.com/news-releases/news-release-details/alx-oncology-announces-first-patients-dosed-evorpacept-and [15] OncoC4 Announces First Patient Dosed in Phase 1 Trial for Novel SIGLEC10 Immune Checkpoint Inhibitor ONC-841 for Solid Tumors. Retrieved September 6, 2024 from https://www.oncoc4.com/index.php/blog/news-releases/item/23-oncoc4-announces-first-patient-dosed-in-phase-1-trial-for-novel-siglec10-immune-checkpoint-inhibitor-onc-841-for-solid-tumors [16] Adaptin Bio Announces FDA Clearance of IND Application for APTN-101 in Glioblastoma. Retrieved September 5, 2024 from https://www.businesswire.com/news/home/20240905978578/en/Adaptin-Bio-Announces-FDA-Clearance-of-IND-Application-for-APTN-101-in-Glioblastoma 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

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2024年11月05日管线快照

管线布局中药物为当前组织机构及其子机构作为药物机构进行统计，早期临床1期并入临床1期，临床1/2期并入临床2期，临床2/3期并入临床3期

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临床1期

临床2期

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药物(靶点)	适应症	全球最高研发状态

帕博利珠单抗 ( PD-1 )	转移性胃癌更多	临床2期
IMU-201 ( PD-1 )	错配修复缺陷或微高卫星不稳定性结直肠癌更多	临床2期
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Azercabtagene Zapreleucel ( CD19 )	伯基特淋巴瘤更多	临床1期
OnCARlytics (Imugene) ( CD19 )	实体瘤更多	临床1期