Phase 2 Clinical Trial of Ablation Therapy With Intravesical N-803 In Combination With BCG or Gemcitabine in Participants With Intermediate-Risk Non-Muscle Invasive Papillary Bladder Cancer

This is an open-label, phase 2, randomized study of intravesical N-803 plus BCG (experimental arm A) and intravesical N-803 plus gemcitabine (experimental arm B) in participants who have intermediate-risk Ta/T1 papillary disease. The primary objective of this study is to evaluate the efficacy of these experimental therapies without the need for surgical intervention by CR rate at month 3 or month 6 (for re-inducted participants).

开始日期2025-05-01

申办/合作机构

ImmunityBio, Inc.

NCT05642195

/ Suspended临床1/2期IIT

Phase I/II Evaluation of a Cancer Lysate Vaccine and Montanide(R) ISA-51 VG With or Without the IL-15 Super-Agonist N-803 as Adjuvant Therapy for PD-L1 Negative Non-Small Cell Lung Cancer

Background:
Surgery is the primary treatment for non-small cell lung cancer (NSCLC) that is diagnosed in its earlier stages. But the tumors often return. Radiation and chemotherapy can improve survival in some people who have had surgery for NSCLC, but these treatments also cause serious side effects. A new approach, called immunotherapy, may be a better way to stop NSCLC tumors from coming back.
Objective:
To test a new treatment (H1299 lung cancer cell vaccine combined with the drug N-803) in people who received surgery for NSCLC.
Eligibility:
Adults aged 18 years or older with no sign of disease after surgery for NSCLC.
Design:
Participants will be screened. They will have a physical exam with blood tests. They will have tests of their heart and lung function. They will have imaging scans.
Study treatment will be given in 28-day cycles. Participants will visit the clinic on the first day of each cycle. They will receive 2 treatments at each visit:
The study vaccine is given as 2-4 small shots under the skin of the thigh or arm.
N-803 is given as a shot under the skin of the abdomen.
Treatment will continue for 6 cycles. Blood tests and imaging scans will be repeated throughout the study.
Participants will have a blood test 1 month after receiving the 6th vaccine. Some participants may then resume taking N-803; they may also receive 2 more vaccinations at 3 and 6 months after their previous treatment.
Follow-up visits will continue for up to 5 years.

开始日期2025-04-16

申办/合作机构

National Cancer Institute

NCT06161545

/ Not yet recruiting临床2期IIT

A Phase II Sequential Window of Opportunity Trial of Pembrolizumab + N-803 Alone or in Combination With PD-L1 t-haNK Cells for Resectable Head and Neck Squamous Cell Carcinoma

Background:
Squamous cell carcinoma is a type of cancer that can cause tumors on the head and neck (HNSCC). Even with treatment, less than 50% of people with certain types of HNSCC survive for 5 years.
Objective:
To test a new drug treatment (N-803 and pembrolizumab, with or without PD-L1 t-haNK cells) in people with HNSCC. These drugs may help the immune system to fight cancer.
Eligibility:
People aged 18 years and older who have HNSCC that is not linked to human papillomavirus infection. They must not yet have received any treatment and be scheduled for surgery to remove the tumors.
Design:
Participants will be screened. They will have a physical exam with blood and urine tests. They will have imaging scans and a test of their heart function. They will have a biopsy: A sample of tissue will be removed from the tumor.
Pembrolizumab is given through a tube attached to a needle inserted into a vein in the arm (intravenous infusion). N-803 is injected under the skin of the abdomen. All participants will receive these 2 treatments on day 1. They will have follow-up visits on days 8 and 15.
Some participants will also receive PD-L1 t-haNK cells by intravenous infusion. These are cells that attack cancer cells. These participants will receive this treatment on days 1, 5, 8, 12, and 15.
All participants will have a clinic visit on day 21. They will have a second biopsy.
Follow-up visits will occur on days 49 and 105. Visits will continue by phone or email every 9 weeks for 2 years....

开始日期2025-04-16

申办/合作机构

National Cancer Institute

100 项与诺格白介素α-因奇西普相关的临床结果

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100 项与诺格白介素α-因奇西普相关的转化医学

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100 项与诺格白介素α-因奇西普相关的专利（医药）

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100

项与诺格白介素α-因奇西普相关的文献（医药）

2025-03-10·ONCOLOGIST

Recurrent pancreatic cancer treated with N-803 and PD-L1 t-haNK followed by an EGFR-targeted nanocell drug conjugate

Article

作者: Brahmbhatt, Himanshu ; Nangia, Chaitali ; Seery, Tara ; Sender, Lennie ; Spilman, Patricia ; Moini, Katayoun ; MacDiarmid, Jennifer ; Soon-Shiong, Patrick

Abstract:

Multimodal temporal therapy orchestrated to leverage immunotherapy, tumor-targeted chemotherapy, and natural killer (NK) cell therapy may provide an opportunity to induce immunogenic cell death for tumor response and increased survival in patients with recurrent cancer. The interleukin-15 (IL-15) superagonist N-803, an enhancer of NK cells, CD4 + T cells, cytotoxic CD8 + T cells, and memory T-cell activity, combined with off-the-shelf PD-L1-targeted high-affinity NK (PD-L1 t-haNK) cells represent novel immunotherapies designed to overcome an immunosuppressive tumor microenvironment (TME). The epidermal growth factor receptor-targeted antibody-nanocell conjugate E-EDV-D682 provides tumor-targeted chemotherapy in the form of its anthracycline metabolite PNU159682 (nemorubicin) cargo and is currently being studied in combination with immunomodulatory EDVs delivering the adjuvant α-galactosyl ceramide (GC). Here, we report the compassionate use treatment of this combination in a patient with recurrent, metastatic pancreatic cancer (mPC) after 3 lines of therapy. Under the initial single-patient Investigational New Drug (spIND) protocol, the patient received N-803, PD-L1 t-haNK cells, and the albumin doxorubicin conjugate aldoxorubicin for ~27 months. The patient’s disease became stable on this regimen, and a transient complete response was observed by ~14 months of therapy. Due to progression, a second spIND protocol was designed whereby the patient received E-EDV-D682 plus EDV-GC for more than 24 months, which resulted in stable disease and the patient’s continued survival at the time this report was written. The patient’s extended survival despite the dire prognosis associated with recurrent mPC points to the merits of this temporal combination regimen in overcoming immuno-chemo resistance with enhanced immune activity required for tumor response and extended survival.

2025-02-01·Annals of Medicine and Surgery

Transforming non-muscle invasive bladder cancer (NMIBC) treatment: FDA approval of Anktiva in combination with BCG

作者: Mukhtar, Sameen ; Pokhrel, Prakriti ; Khattak, Aimal Alam ; Javed, Ahmed

2025-02-01·European Urology Oncology

Unmet Need in Non–muscle-invasive Bladder Cancer Failing Bacillus Calmette-Guérin Therapy: A Systematic Review and Cost-effectiveness Analyses from the International Bladder Cancer Group

Review

作者: Burger, Maximilian ; D'Andrea, David ; Masson-Lecomte, Alexandra ; Kamat, Ashish ; Mostafid, Hugh ; Gontero, Paolo ; Shariat, Shahrokh ; Rouprêt, Morgan

BACKGROUND AND OBJECTIVE:

Non-muscle-invasive bladder cancer (NMIBC) poses a significant clinical challenge, particularly when failing bacillus Calmette-Guérin (BCG) therapy, necessitating alternative treatments. Despite radical cystectomy being the recommended treatment, many patients are unfit or unwilling to undergo this invasive procedure, highlighting the need for effective bladder-sparing therapies. This review aims to summarize and report the evidence on the efficacy and to estimate the costs of bladder-preserving strategies used in NMIBC recurrence after failure of intravesical BCG therapy.

METHODS:

We systematically searched online databases for prospective studies investigating intravesical therapy, systemic therapy, or combination of both in patients treated previously with BCG. Owing to significant heterogeneity across the studies, a meta-analysis was inappropriate. A sensitivity analysis was performed in an exploratory manner. We used a decision-analytic Markov model to compare novel U.S. Food and Drug Administration-approved treatments with a 2-yr time horizon.

KEY FINDINGS AND LIMITATIONS:

A total of 57 studies published between 1998 and 2024, with 68 unique study arms and consisting of 2589 patients, were identified. The 3-mo overall response rate (ORR) across all studies, complete response rate (CRR) in concomitant carcinoma in situ (CIS) or CIS only disease, and recurrence-free rate (RFR) in papillary disease were estimated to be 52.4% (95% confidence interval [CI]: 45.4-59.2), 52.8% (95% CI: 42.9-62.6), and 26.4% (95% CI: 13.3-45.6), respectively. The 12-mo ORR, CRR, and RFR were estimated to be 78% (95% CI: 52.9-91.8), 27.8% (95% CI: 21.3-35.4), and 25.4% (95% CI: 18.2-34.2), respectively. The progression rate was estimated to be 13% (95% CI: 9-18.2). The mean proportion of patients treated with radical cystectomy was estimated to be 24.7 (range 0-85.7). The reported toxicity grades were overall mild, with a median of 3.4% (range 0-33.3%) participants experiencing a dose limiting toxicity. Compared with using radical cystectomy to treat patients failing BCG therapy, at a willingness-to-pay threshold of 100 000 USD, nadofaragene firadenovec was cost effective, with an incremental cost-effectiveness ratio (ICER) of 10 014 USD per quality-adjusted life year (QALY), while nogapendekin alfa inbakicept was less cost effective than nadofaragene firadenovec (ICER of 44 602 USD per QALY). Pembrolizumab, which dominated, was both less costly and more effective than the other strategies.

CONCLUSIONS AND CLINICAL IMPLICATIONS:

Salvage bladder-sparing therapies show a response rate of around 50% at 3 mo in patients with NMIBC failing BCG. However, long-term data are heterogeneous. Nevertheless, recently developed agents show promising tumor control activity. In the rapidly evolving landscape of urothelial cancer, some of these treatment strategies might be cost effective and improve patients' quality of life. The findings of our review highlight the need for novel, more effective therapeutic strategies.

PATIENT SUMMARY:

In this study, we reviewed the evidence on the efficacy of bladder-preserving strategies used in patients with bladder cancer recurrence after failing bacillus Calmette-Guérin (BCG) therapy. We found that these strategies show a response rate of around 50% at 3 mo. However, long-term data are heterogeneous. Nevertheless, recently developed agents show promising tumor control activity. In the rapidly evolving landscape of urothelial cancer, some of these treatment strategies might be cost effective and improve patients' quality of life.

203

项与诺格白介素α-因奇西普相关的新闻（医药）

2025-04-10

·生物制品圈

2024 年的新药物靶点

2024 年，美国、欧盟和日本药品机构批准了 55 种新药。对相应的药品说明书和原始文献进行分析发现，约 80% 的这些药物具有明确的作用机制（MoA）靶点（《自然综述：药物发现》16，19 - 34；2017）。此外，其中 9 个 MoA 靶点尚未被任何先前获批的药物调节。在此，我们重点介绍这些靶点以及相应的 8 种药物（表 1）。表1 | 2024 年获批的具有新作用机制靶点的药物在近二十年来致力于癌症治疗的 menin 抑制剂研究后，小分子药物 revumenib 获得美国食品药品监督管理局（FDA）批准。Revumenib 阻断野生型赖氨酸甲基转移酶 2A（KMT2A）和 KMT2A 融合蛋白与 menin 在 KMT2A 重排急性白血病中的相互作用，抑制白血病生成性转录。表 1 中的另一种小分子药物 danicopan 是补体因子 D（一种红细胞形成 C3 转化酶所需的丝氨酸蛋白酶）的抑制剂，可减少成人阵发性夜间血红蛋白尿的血管外溶血。两种基于抗体的疗法 tarlatamab 和 zolbetuximab 分别获批用于广泛期小细胞肺癌和胃或胃食管结合部腺癌。Tarlatamab 是一种双特异性抗体，可结合肿瘤细胞表面的 DLL3（delta - like 配体 3）和细胞毒性 T 细胞表面的 CD3，促进 T 细胞介导的肿瘤细胞杀伤。Zolbetuximab 是一种针对 claudin 18 亚型 2（CLDN18.2）的单克隆抗体，CLDN18.2 是一种在多种胃肠道癌症中过表达的紧密连接蛋白。Sotatercept 和 nogapendekin alfa inbakicept 是融合蛋白。Sotatercept 由激活素受体 IIA 的细胞外结构域与免疫球蛋白 G1（IgG1）Fc 结构域连接而成。该药物可捕获转化生长因子 - β超家族成员，如激活素 A 和 B，抑制激活素信号传导，降低肺动脉高压患者的肺血管阻力。Nogapendekin alfa inbakicept 是由两个 nogapendekin alfa（IL - 15N72D 变体）部分和 inbakicept（一种二聚体 IL - 15Rα sushi 结构域 - IgG1 Fc 结构域融合蛋白）组成的复合体。它作为 IL - 15 受体激动剂，旨在激活并促进自然杀伤细胞、CD8 + T 细胞和记忆 T 细胞的增殖。Nogapendekin alfa inbakicept 通过导管注入膀胱，获批与卡介苗（BCG）疫苗联合用于治疗对 BCG 无反应的非肌层浸润性膀胱癌成人患者。共价脂化 13 - 聚合寡核苷酸 imetelstat 可结合人端粒酶 RNA 组分的模板区域。它抑制在骨髓增生异常综合征患者中增加的端粒酶逆转录酶（TERT）的活性。最后，flurpiridaz F 18 是线粒体复合体 1（MC-1）抑制剂吡啶苯（一种杀虫剂）的类似物，可结合具有活性线粒体的心脏组织，使正电子发射断层扫描成像能够评估心肌缺血和梗死。从治疗领域角度看，6 种药物针对罕见病，5 种用于癌症，2 种用于心血管疾病，1 种用于血液病。参考来源：https://www.nature.com/articles/d41573-025-00069-z识别微信二维码，添加生物制品圈小编，符合条件者即可加入生物制品微信群！请注明：姓名+研究方向！版权声明本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观不本站。

疫苗上市批准

2025-03-27

·抗体圈

全球药企创新力榜单揭榜，这些“新”值得关注

近日，商业媒体《快公司》（Fast Company）对2024年全球最具创新力的医药公司进行梳理总结，10家药企为此前研发进展甚微的疾病推出突破性疗法，并推动现有疗法可及性。疗法涉及阿尔茨海默病（AD）、精神分裂症、血友病、癌症等多领域。 ARS公司肾上腺素开启鼻喷时代当前，治疗过敏性休克的唯一疗法是肾上腺素，长期以来仅可通过注射给药。但有不少患者无法及时接受注射，甚至因针头过大而犹豫不决。2024年8月，ARS公司的肾上腺素鼻喷雾剂Neffy获美国FDA批准上市。据悉，该药的喷雾容器无需预热，只需插入并按下，且在高达122°F（50℃）的温度下其保质期为30个月。这比自动注射器有很大优势，后者保质期为18~20个月，且对温度的要求更严格。 BMS 精分新机制药物绽放 2024年9月，FDA批准百时美施贵宝（BMS）的Cobenfy成为几十年来首个治疗精神分裂症的主要新药。Cobenfy的使用机制与现有治疗方法完全不同，通过触发毒蕈碱受体而不是多巴胺受体起作用，是首个通过这种方式治疗精神分裂症的药物。 Cobenfy在临床试验中总体耐受性良好，与常用的非典型抗精神病药物不同，没有关于某些老年患者死亡率增加的黑框警告。 BMS目前正在努力扩大该药的适应症：可能会在2026年公布该药治疗AD的试验数据，还计划针对双相情感障碍Ⅰ型和AD适应症启动另外3项Ⅲ期试验。礼来 AD疗法新秀善补短板 2024年7月，FDA批准了礼来的AD药物Kisunla上市，这是一种用于治疗早期AD症状的单克隆抗体。Kisunla由礼来开发，是首个也是唯一一个使用有限给药疗程靶向清除淀粉样斑块的疗法。在1736名患者的Ⅲ期试验中，与安慰剂相比，Kisunla在18个月时将认知和功能衰退减缓了35%，并将疾病进展到下一临床阶段的风险降低了39%。 Kisunla与其竞争对手，即百健和卫材的Leqembi相比，给药间隔更长。前者每4周给药一次，后者每2周给药一次。除了AD领域创新，礼来还继续巩固其利润丰厚的GLP-1产品Mounjaro和Zepbound的市场地位。该公司于2024年1月推出了直接面向消费者的渠道，以折扣价提供Zepbound处方药，并提供送货上门服务。礼来也在不断扩大GLP-1药物适应症，例如在2024年12月23日，FDA批准减肥药Zepbound成为首个专门用于治疗阻塞性睡眠呼吸暂停的处方药，据估计，该病影响超过2200万名美国人。 Aurion公司细胞治疗预防失明该公司的Vyznova于2023年5月在日本获批，并于2024年9月在日本上市，是首个经过临床验证的角膜护理细胞疗法。Vyznova用于治疗角膜内皮疾病（如大泡性角膜病变），这是一种影响全球数百万人的渐进性疾病，如果不及时治疗，会导致失明。在Vyznova之前，大泡性角膜病变的唯一治疗方法是使用供体组织进行角膜移植，但每70只患病眼睛中，只有1个供体角膜可用，数量稀少。Aurion公司开发了一种制造工艺，可以从单个供体的角膜内皮细胞中生产多达1000剂Vyznova。 2024年6月，FDA授予Vyznova突破性疗法认定和再生医学先进疗法认定，为该细胞疗法提供了快速审批途径。2024年12月公司发布的美国Ⅰ/Ⅱ期临床试验数据显示，其最高剂量可显著改善视力。 Iovance公司冲破实体瘤治疗壁垒 2024年2月，FDA加速批准Iovance公司的Amtagvi用于治疗晚期黑色素瘤（正在进行Ⅲ期试验）。Amtagvi是首个获批用于治疗实体瘤的一次性个体化T细胞疗法。据悉，目前CAR-T细胞疗法虽然对血液肿瘤较为有效，但在实体瘤中表现不佳。Amtagvi是一种一次性疗法，使用肿瘤浸润淋巴细胞（TIL），持久治疗更多实体瘤。 Amtagvi获批使TIL疗法有望成为治疗实体肿瘤的新型药物。目前，该药正在进行肺癌、宫颈癌等临床试验。这种一次性疗法每位患者的费用为51.5万美元（未扣除返款和折扣）。该公司预计，许多保险公司将以与CAR-T细胞疗法相同的水平纳入对应报销范围。 Adaptimune公司滑膜肉瘤患者获新希望 2024年8月，Adaptimmune公司工程化T细胞疗法Tecelra获FDA加速批准。该药用于治疗之前接受过化疗的特定类型滑膜肉瘤成人患者。这项批准使Tecelra成为美国批准的首个针对实体瘤癌症的自体T细胞免疫疗法，也是十多年来首个针对滑膜肉瘤的新治疗选择。滑膜肉瘤是一种罕见的软组织癌，最常见于年轻人。2024年12月，公司宣布第一位患者已接受了治疗。该疗法定价为727,000美元，在美国癌症细胞药物中每剂成本最高。该公司第二款在研T细胞疗法lete-cel已在Ⅱ期试验达到主要终点。公司估计，这两种疗法结合可在美国市场产生4亿美元的销售额年峰值。 Verona公司畅通慢阻肺呼吸通道 2024年6月，FDA批准Verona公司慢性阻塞性肺病（COPD）疗法Ohtuvayre上市，用于成人患者。该疗法是20多年来治疗该病的首个具有新型作用机制的吸入维持疗法，有望为美国约1600万患有COPD的成年人带来新治疗选择。 Ohtuvayre是一种单一小分子药物，具有独特的双重作用机制，可选择性抑制磷酸二酯酶3/4（PDE3/4），同时增强支气管扩张（打开肺部小气道）并减少与COPD相关的炎症，无需使用类固醇。据悉，该公司目前正在进行Ohtuvayre的Ⅱ期临床试验，用于治疗其他有未满足医疗需求的呼吸系统疾病，包括慢性非囊性纤维化支气管扩张、囊性纤维化和哮喘。辉瑞释放血友病前沿产品活力 2024年10月，FDA批准了辉瑞的Hympavzi上市，这是首个每周注射一次的药物，用于预防或减少患有A型血友病（缺少凝血因子Ⅷ）和B型血友病（缺少凝血因子Ⅸ）成人和12岁及以上儿童患者的出血发作频率。在其他治疗领域，辉瑞已进军抗体药物偶联物（ADC）市场，2024年4月，FDA全面批准了Tivdak，该药物此前已于2021年获得加速批准，用于治疗复发性或转移性宫颈癌患者。 ImmunityBio公司 IL-15受体激动剂攻克膀胱癌多达40%的非肌层浸润性膀胱癌（NMIBC）患者对传统的免疫疗法（即卡介苗，BCG疗法）没有反应。2024年4月，FDA批准了ImmunityBio公司的Anktiva上市，是对BCG无应答的NMIBC患者的首创疗法和罕见突破。Anktiva被批准用于与标准护理BCG疗法联合进行维持治疗，以提高疗效。 Anktiva的作用方式新颖，其为一种IL-15受体激动剂，可激活自然杀伤（NK）和T细胞攻击肿瘤和癌细胞。2024年第三季度，Anktiva创造了约600万美元的净产品收入。据公司披露，截至2024年11月，已有100名患者接受了Anktiva-BCG联合疗法治疗，完全缓解率为71%。Anktiva在对检查点抑制剂无反应的非小细胞肺癌患者中进行的Ⅱ期试验数据显示，中位总生存期几乎是标准化疗的2倍。 Zevra公司小分子药穿越血脑屏障 2024年9月，FDA批准了Zevra公司的小分子口服药物Miplyffa与酶稳定剂miglustat联用，治疗C型尼曼匹克病（NPC）。NPC是一种极为罕见的遗传性疾病，会导致进行性神经系统症状和器官功能障碍。Miplyffa是FDA批准的首个用于治疗成人和2岁及以上儿童NPC相关神经系统症状的药物，可穿越血脑屏障。 NPC会阻止人体对细胞中的胆固醇和其他脂质的使用，进而导致这些物质在体内病理性积累。该疾病会对患者的言语、认知、吞咽、运动和精细运动技能造成损害，患有该疾病的人平均寿命约为13年。来源：From cancer vaccines to COPD treatments,these 10 companies are developing innovative medicines，FAST COMPANY 识别微信二维码，添加抗体圈小编，符合条件者即可加入抗体圈微信群！请注明：姓名+研究方向！本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观点，不代表本站立场。

突破性疗法临床3期上市批准临床结果细胞疗法

2025-03-04

·PRNewswire

Cryoport Reports Fourth Quarter and Full Year 2024 Financial Results

FY 2024 revenue of $228.4 million, in-line with company guidance Commercial Cell & Gene Therapy revenue rose to $26 million in FY 2024, up 20% year-over-year Supporting a record total of 701 global clinical trials as of December 31, 2024 NASHVILLE, Tenn., March 4, 2025 /PRNewswire/ -- Cryoport, Inc. (NASDAQ: CYRX) (Cryoport), a global leader in supply chain solutions for the life sciences industry, today announced financial results for the fourth quarter (Q4) and year ended (FY) December 31, 2024. Jerrell Shelton, CEO of Cryoport, commented, "Cryoport ended 2024 with solid results across the company including total full year revenue of $228.4 million, which was in-line with our expectations. We continued to see considerable revenue growth from our support of commercial Cell & Gene therapies where revenue rose 37% for the fourth quarter and 20% for the full year compared to the prior year periods. "Our Life Sciences Services business continued its expansion, partially attributed to the double-digit year-over-year growth in BioStorage/BioServices revenue for both the fourth quarter and full year periods. In the fourth quarter our Life Sciences Products business began to show signs of market demand stability and continued to provide positive free cash flow. "As previously reported, during 2024, we implemented cost reduction and capital realignment strategies, making significant progress in improving our cost structure. Notably, our gross margin improved to 45.8% in Q4 2024, up from 40.6% in the same period last year. We remain confident that our actions will lead us to a return to positive adjusted EBITDA during 2025 as we further implement our pathway to profitability. "We believe that as we enter 2025, we are prepared to capitalize on the anticipated growth in the Cell & Gene Therapy market. We intend to grow our leading market position and open additional revenue streams that have been under development through new services and product introductions. We will supplement this through potential strategic collaborations and partnerships. We are excited about our prospects for this year, and we believe we have all the necessary tools in place to execute on our growth plans and to reach our long-term objective of sustainable profitability," concluded Mr. Shelton. In tabular form, Q4 2024 and FY 2024 revenue compared to Q4 2023 and FY 2023, respectively, was as follows: BioStorage/BioServices revenue continues to grow double digits year-over-year, increasing 11% in FY 2024 as we continue to introduce our expanded capabilities to existing customers, as well as add new customers into our global network, and as more allogeneic clinical and commercial therapies progress in the number of patients treated. Revenue from the support of commercially approved Cell & Gene therapies grew to $25.9 million, up 20% year-over-year, for FY 2024 and increased to $7.9 million, up 37% year-over-year, for Q4 2024. During FY 2024, five (5) new therapies were approved including Mesoblast's Ryoncil® for the treatment of graft versus host disease, Adaptimmune's Tecelra® for the treatment of adults with unresectable or metastatic synovial sarcoma, ImmunityBio's Anktiva® for BCG-unresponsive non-muscle invasive bladder cancer, Iovance Biotherapeutics' Amtagvi™ therapy for advanced melanoma, and Immuneel's Qartemi® for the treatment of non-Hodgkin Lymphoma. Qartemi® is the first cell therapy developed and approved in India and is supported by CRYOPDP's logistics network of 14 facilities inside the country. Our total commercial therapy count was nineteen (19) as of December 31, 2024. As of December 31, 2024, Cryoport supported a total of 701 global clinical trials, a net increase of 26 clinical trials over December 31, 2023, with 81 trials in Phase 3. The number of trials by phase and region are as follows: A total of eleven (11) Cryoport supported Biologic License Applications (BLA)/Marketing Authorization Applications (MAA) were filed in 2024, of which three (3) were filed during the fourth quarter. Following the end of the year, three (3) filings occurred in January 2025. For 2025, we anticipate up to an additional twenty-three (23) application filings, five (5) new therapy approvals and an additional five (5) approvals for label/geographic expansions or moves to earlier lines of treatment. Financial Highlights Revenue Total revenue for Q4 2024 was $59.5 million compared to $57.3 million for Q4 2023, a year-over-year increase of 4.0% or $2.3 million. Life Sciences Services revenue for Q4 2024 was $39.6 million compared to $37.0 million for Q4 2023, up 6.8% year-over-year, including BioStorage/BioServices revenue of $4.0 million, up 10.4% year-over-year. Life Sciences Products revenue for Q4 2024 was $20.0 million compared to $20.2 million for Q4 2023, down 1.3% year-over-year. Total revenue for FY 2024 was $228.4 million, compared to $233.3 million for FY 2023, a year-over-year decrease of 2.1%. Life Sciences Services revenue for FY 2024 was $153.7 million compared to $144.1 million for FY 2023, up 6.6% year-over-year, including BioStorage/BioServices revenue of $15.0 million, up 10.6% year-over-year. Life Sciences Products revenue for FY 2024 was $74.7 million compared to $89.2 million for FY 2023, down 16.2%. Gross Margin Total gross margin was 45.8% for Q4 2024 compared to 40.6% for Q4 2023. Gross margin for Life Sciences Services was 46.2% for Q4 2024 compared to 40.8% for Q4 2023. Gross margin for Life Sciences Products was 45.1% for Q4 2024 compared to 40.4% for Q4 2023. Total gross margin was 43.6% for FY 2024 compared to 42.6% for FY 2023. Gross margin for Life Sciences Services was 44.5% for FY 2024 compared to 43.2% for FY 2023. Gross margin for Life Sciences Products was 41.7% for FY 2024 compared to 41.6% for FY 2023. Operating Costs and Expenses Operating costs and expenses decreased to $41.2 million for Q4 2024 compared to operating costs and expenses of $93.1 million for Q4 2023, which includes a non-cash impairment charge to goodwill of $49.6 million related to the MVE Biological Solutions business unit. Operating costs and expenses increased to $230.5 million for FY 2024 (which includes a non-cash impairment charge of $63.8 million), compared to $214.5 million for FY 2023 (which includes a non-cash impairment charge of $49.6 million). Net Loss Net loss for Q4 2024 and FY 2024 was $18.7 million and $114.8 million, respectively, compared to a net loss of $62.4 million and $99.6 million for the same periods in 2023, respectively. Net loss attributable to common stockholders was $20.7 million, or $0.42 per share, and $122.8 million, or $2.49 per share, for Q4 2024 and FY 2024, respectively. This compares to a net loss attributable to common stockholders of $64.4 million, or $1.31 per share, and $107.6 million, or $2.21 per share, for Q4 2023 and FY 2023, respectively. Adjusted EBITDA Adjusted EBITDA was a negative $1.3 million for Q4 2024, compared to a negative $6.6 million for Q4 2023. Adjusted EBITDA for FY 2024 was a negative $15.1 million, compared to a negative $8.3 million for FY 2023. Cash, Cash equivalents, and Short-Term Investments Cryoport held $261.7 million in cash, cash equivalents, and short-term investments as of December 31, 2024. Convertible Debt repurchases During FY 2024, the Company repurchased $185.0 million in aggregate principal amount of its Convertible Senior Notes due in 2026 for an aggregate repurchase price of $163.2 million. The Company has approximately $73.9 million in total of repurchase authorization available under its repurchase programs as of December 31, 2024. Note: All reconciliations of GAAP to adjusted (non-GAAP) figures above are detailed in the reconciliation tables included later in the press release. Outlook The Company is providing full year 2025 revenue guidance in the range of $240 - $250 million. The Company's 2025 guidance is dependent on its current business and expectations, which may be further impacted by, among other things, factors that are outside of our control, such as national economic factors, the global macroeconomic and geopolitical environment, supply chain constraints, inflationary pressures, and/or the effects of foreign currency fluctuations, as well as the other factors described in the Company's filings with the Securities and Exchange Commission ("SEC"), including in the "Risk Factors" section of its most recently filed periodic reports on Form 10-K and Form 10-Q, as well as in its subsequent filings with the SEC. Additional Information Further information on Cryoport's financial results is included in the attached condensed consolidated balance sheets and statements of operations, and additional explanations of Cryoport's financial performance are provided in the Company's Annual Report on Form 10-K for the year ended December 31, 2024, which is expected to be filed with the SEC on March 7, 2025. Additionally, the full report will be available in the SEC Filings section of the Investor Relations section of Cryoport's website at . Earnings Conference Call Information IMPORTANT INFORMATION: In addition to the earnings release, a document titled "Cryoport Fourth Quarter and Full Year 2024 in Review", providing a review of Cryoport's financial and operational performance and a general business update, will be issued at 4:05 p.m. ET on Tuesday, March 4, 2025. The document is designed to be read in advance of the questions and answers conference call and will be accessible at . Cryoport management will host a conference call at 5:00 p.m. ET on March 4, 2025. The conference call will be in the format of a questions and answers session and will address any queries investors have regarding the Company's reported results. A slide deck will accompany the call. Conference Call Information Please allow 10 minutes prior to the call to visit this site to download and install any necessary audio software. The questions and answers call will be recorded and available approximately three hours after completion of the live event in the Investor Relations section of the Company's website at for a limited time. To access the replay of the questions and answers click here. A dial-in replay of the call will also be available to those interested, until March 11, 2025. To access the replay, dial 1-844-512-2921 (United States) or 1-412-317-6671 (International) and enter replay entry code: 1116296#. About Cryoport, Inc. Cryoport, Inc. (Nasdaq: CYRX), is a global leader in supply chain solutions for the Life Sciences with an emphasis on cell & gene therapies. Cryoport enables manufacturers, contract manufacturers (CDMOs), contract research organizations (CROs), developers, and researchers to carry out their respective business with products and services that are designed to derisk services and provide certainty. We provide a broad array of supply chain solutions for the life sciences industry. Through our platform of critical products and solutions including advanced temperature-controlled packaging, informatics, specialized bio-logistics services, bio-storage, bio-services, and cryogenic systems, we are "Enabling the Future of Medicine™" worldwide, through our innovative systems, compliant procedures, and agile approach to superior supply chain management. Our corporate headquarters, located in Nashville, Tennessee, is complemented by over 50 global locations in 17 countries, with key sites in the United States, United Kingdom, France, the Netherlands, Belgium, Portugal, Germany, Japan, Australia, India, and China. For more information, visit or follow via LinkedIn at or @cryoport on X, formerly known as Twitter at for live updates. Forward-Looking Statements Statements in this press release which are not purely historical, including statements regarding Cryoport's intentions, hopes, beliefs, expectations, representations, projections, plans, or predictions of the future, are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, those related to Cryoport's industry, business, long-term growth prospects, plans, strategies, acquisitions, future financial results and financial condition, such as Cryoport's outlook and guidance for full year 2025 revenue and the related assumptions and factors expected to drive revenue, projected growth trends in the markets in which Cryoport operates, and Cryoport's plans and expectations regarding the launch of new products and services, such as the expected timing and benefits of such products and services launches. It is important to note that Cryoport's actual results could differ materially from those in any such forward-looking statements. Factors that could cause actual results to differ materially include, but are not limited to, risks and uncertainties associated with the effect of changing economic and geopolitical conditions, supply chain constraints, inflationary pressures, the effects of foreign currency fluctuations, trends in the products markets, variations in Cryoport's cash flow, market acceptance risks, and technical development risks. Cryoport's business could be affected by other factors discussed in Cryoport's SEC reports, including in the "Risk Factors" section of its most recently filed periodic reports on Form 10-K and Form 10-Q, as well as in its subsequent filings with the SEC. The forward-looking statements contained in this press release speak only as of the date hereof and Cryoport cautions investors not to place undue reliance on these forward-looking statements. Except as required by law, Cryoport disclaims any obligation, and does not undertake to update or revise any forward-looking statements in this press release. Note Regarding Use of Non-GAAP Financial Measures To supplement our financial statements, which are presented on the basis of U.S. generally accepted accounting principles (GAAP), the following non-GAAP measures of financial performance as defined in Regulation G of the Securities Exchange Act of 1934 are included in this release: revenue at constant currency, revenue growth rate at constant currency, and adjusted EBITDA. Non-GAAP financial measures are not calculated in accordance with GAAP, are not based on any comprehensive set of accounting rules or principles and may be different from non-GAAP financial measures presented by other companies. Non-GAAP financial measures, including revenue at constant currency, revenue growth rate at constant currency and adjusted EBITDA, should not be considered as a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP. We believe that revenue growth is a key indicator of how Cryoport is progressing from period to period, and we believe that the non-GAAP financial measures, revenue at constant currency and revenue growth rate at constant currency, are useful to investors in analyzing the underlying trends in revenue. Under GAAP, revenue received in local (non-U.S. dollar) currency is translated into U.S. dollars at the average exchange rate for the period presented. As a result, fluctuations in foreign currency exchange rates affect the results of our operations and the value of our foreign assets and liabilities, which in turn may adversely affect results of operations and cash flows and the comparability of period-to-period results of operations. When we use the term "constant currency," it means that we have translated local currency revenue for the current reporting period into U.S. dollars using the same average foreign currency exchange rates for the conversion of revenue into U.S. dollars that we used to translate local currency revenue for the comparable reporting period of the prior year. Revenue growth rate at constant currency refers to the measure of comparing the current reporting period revenue at constant currency with the reported GAAP revenue for the comparable reporting period of the prior year. However, we also believe that data on constant currency period-over-period changes have limitations, particularly as the currency effects that are eliminated could constitute a significant element of our revenue and could significantly impact our performance. We therefore limit our use of constant currency period-over-period changes to a measure for the impact of currency fluctuations on the translation of local currency revenue into U.S. dollars. We do not evaluate our results and performance without considering both period-over-period changes in non-GAAP constant currency revenue on the one hand and changes in revenue prepared in accordance with GAAP on the other. We caution the readers of this press release to follow a similar approach by considering revenue on constant currency period-over-period changes only in addition to, and not as a substitute for, or superior to, changes in revenue prepared in accordance with GAAP. Adjusted EBITDA is defined as net loss adjusted for net interest expense, income taxes, depreciation and amortization expense, stock-based compensation expense, acquisition and integration costs, cost reduction initiatives, investment income, unrealized (gain)/loss on investments, foreign currency (gain)/loss, gain on insurance claim, net gain on extinguishment of debt, impairment loss, changes in fair value of contingent consideration and charges or gains resulting from non-recurring events, as applicable. Management believes that adjusted EBITDA provides a useful measure of Cryoport's operating results, a meaningful comparison with historical results and with the results of other companies, and insight into Cryoport's ongoing operating performance. Further, management and the Company's board of directors utilize adjusted EBITDA to gain a better understanding of Cryoport's comparative operating performance from period to period and as a basis for planning and forecasting future periods. Adjusted EBITDA is also a significant performance measure used by Cryoport in connection with its incentive compensation programs. Management believes adjusted EBITDA, when read in conjunction with Cryoport's GAAP financials, is useful to investors because it provides a basis for meaningful period-to-period comparisons of Cryoport's ongoing operating results, including results of operations, against investor and analyst financial models, helps identify trends in Cryoport's underlying business and in performing related trend analyses, and it provides a better understanding of how management plans and measures Cryoport's underlying business. SOURCE Cryoport, Inc. 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财报上市批准细胞疗法

100 项与诺格白介素α-因奇西普相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D12888	-	-	-

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
非肌层浸润性膀胱肿瘤	美国	Altor BioScience Corp.	2024-04-22

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
转移性非小细胞肺癌	临床3期	美国	ImmunityBio, Inc.	2018-05-18
膀胱癌	临床3期	美国	ImmunityBio, Inc.	2017-06-02
铂耐药性卵巢癌	临床2期	美国	ImmunityBio, Inc.	2025-03-01
复发性铂耐药性卵巢癌	临床2期	美国	ImmunityBio, Inc.	2025-03-01
前列腺腺癌	临床2期	-	ImmunityBio, Inc.	2025-01-31
胶质母细胞瘤	临床2期	美国	ImmunityBio, Inc.	2024-08-07
头颈癌转移	临床2期	美国	ImmunityBio, Inc.	2024-02-16
复发性头颈癌	临床2期	美国	ImmunityBio, Inc.	2024-02-16
复发性头颈部鳞状细胞癌	临床2期	美国	ImmunityBio, Inc.	2024-02-16
转移性头颈部鳞状细胞癌	临床2期	美国	ImmunityBio, Inc.	2024-02-16

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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


NCT03381586 (CTgov)	临床1期	-	20	ALT-803 (Group A)	窪衊網構淵構鏇鑰艱簾(選糧窪願餘願艱簾鬱憲) = 構選蓋壓鏇醖廠廠繭簾衊繭獵構構淵夢襯餘膚 (鹹鑰夢顧顧鏇製網選繭, 0.813) 更多	-	2025-03-24
				ALT-803 (Group B)	窪衊網構淵構鏇鑰艱簾(選糧窪願餘願艱簾鬱憲) = 淵觸淵淵製獵範簾簾遞衊繭獵構構淵夢襯餘膚 (鹹鑰夢顧顧鏇製網選繭, 0.095) 更多
NCT03563157 (CTgov)	临床1/2期	转移性结直肠癌	2	SBRT+Aldoxorubicin Hydrochloride+GI-6301+GI-4000+ETBX-061+Cetuximab+ETBX-051+Leucovorin+ETBX-011+5-Fluorouracil+ALT-803+ETBX-021+nab-paclitaxel+CYCLOPHOSPHAMIDE+Avelumab+haNK+GI-6207+Regorafenib+Oxaliplatin+Capecitabine (NANT Colorectal Cancer (CRC) Vaccine)	遞鹽築壓積齋選積淵獵 = 鹽獵製觸糧選衊餘襯鑰艱餘鑰積顧鏇淵艱鹽鏇 (鑰鑰襯廠製艱膚構簾廠, 衊築鏇夢獵簾鏇繭網範 ~ 糧鏇鹹鬱糧艱鹹簾壓鏇) 更多	-	2024-12-11
				Regorafenib (Regorafenib)	餘襯鹽遞網夢廠網壓膚(衊範簾糧蓋壓範觸衊糧) = 網顧醖選簾齋鏇憲積築襯鏇構觸鏇鹽餘糧鬱蓋 (構鹽製獵窪鹽構糧餘觸, 願範夢鑰繭願鹽構顧繭 ~ 選衊膚餘蓋構鏇顧築遞) 更多
NCT02099539 (CTgov)	临床1期	复发性多发性骨髓瘤 \| 难治性多发性骨髓瘤	19	N-803 (Cohort 1: N-803 - IV 1 ug/kg)	壓願繭鹹窪膚夢醖壓鹹 = 遞選繭範積壓簾構膚鬱鏇鬱選繭網膚觸憲製願 (衊製積願鏇廠窪憲簾選, 選憲範蓋鹹願膚觸衊衊 ~ 願鹹繭積廠簾淵願醖範) 更多	-	2024-09-26
				N-803 (Cohort 2: N-803 - IV 3 ug/kg)	壓願繭鹹窪膚夢醖壓鹹 = 憲製鹽觸鹹齋夢範夢鹹鏇鬱選繭網膚觸憲製願 (衊製積願鏇廠窪憲簾選, 糧艱顧夢廠製網憲艱鹹 ~ 顧壓範廠鹽蓋製艱範衊) 更多
NCT03493945 (QuEST1, ESMO2024)	临床1/2期	去势抵抗性前列腺癌 dMMR \| pMMR	25	BNVax + BA + Anktiva	憲顧遞鏇鏇夢選網獵築(鑰製願簾鬱顧選夢廠顧) = 選鹽餘觸餘構簾鹹齋淵鬱鏇憲獵遞繭鏇製壓範 (構壓網糧築壓築繭糧繭, 39.9 ~ 83.9) 更多	积极	2024-09-15
NCT03228667 (QUILT 3.055, WCLC2024) 人工标引	临床2期	非小细胞肺癌二线 \| 末线 \| 三线 PDL1+	86	Anktiva 15mcg/knivolumabvolupembrolizumab	範製構簾壓窪窪繭鏇蓋(糧顧憲醖鬱範製觸齋願) = injection site reaction 78 (91%), fatigue 46 (53%), chills 36 (42%). 鏇鑰簾憲廠淵糧艱壓鑰 (簾艱膚積醖窪製蓋鏇衊 )	积极	2024-09-08
				Anktiva + CPI (nivolumab or pembrolizumab)pembrolizumab) (failure of CPI + 3rd line)
NCT04385849 (CTgov)	临床1期	新型冠状病毒感染	1	N-803 (Experimental Arm)	鏇憲獵顧鹹顧蓋獵齋廠 = 簾範淵壓繭淵壓積製醖選鏇醖築窪廠繭積網艱 (簾築範顧願衊構糧遞鹽, 獵鏇餘觸鏇繭艱鑰憲艱 ~ 願衊窪襯簾醖遞網積齋) 更多	-	2024-09-05
				Saline (Placebo Arm)	鏇憲獵顧鹹顧蓋獵齋廠 = 獵鹹範廠醖醖膚艱顧餘選鏇醖築窪廠繭積網艱 (簾築範顧願衊構糧遞鹽, 鏇壓願夢積窪醖顧夢膚 ~ 衊範鹽廠鹹構醖憲選願) 更多
NCT03853317 (QUILT-3.063, CTgov)	临床2期	转移性Merkel细胞癌 \| 梅克尔细胞癌	9	N-803+Avelumab+haNK™	網觸壓衊艱壓淵積獵衊 = 鏇範築範築鏇夢齋積醖製顧鏇膚鏇衊鑰選淵鹽 (觸夢顧觸鏇製糧齋糧鹹, 顧構鑰餘鹹觸獵鹹簾齋 ~ 網製網齋艱繭製網遞鏇) 更多	-	2024-08-09
NCT03127098 (CTgov)	临床1/2期	肺癌 \| 卵巢癌 \| 结肠癌 ... 更多	3	ALT-803+ETBX-011	製繭鏇廠鑰壓齋壓簾淵 = 遞艱遞願膚淵夢糧壓簾網範膚鹹膚簾遞製襯鹽 (鏇鬱窪積壓醖衊夢糧願, 願網淵構壓衊醖顧壓廠 ~ 餘蓋鑰膚夢醖蓋範鬱襯) 更多	-	2024-08-06
NCT02384954 (CTgov)	临床1/2期	难治性惰性非霍奇金淋巴瘤 \| 惰性B细胞非霍奇金淋巴瘤	43	N-803+Rituximab (Phase 1 Cohort 1: N-803 - IV 1 ug/kg)	膚餘餘範齋鹽構壓獵遞 = 顧糧糧蓋繭廠艱鏇願顧遞鑰鬱獵積夢鑰觸鏇壓 (窪糧構糧淵選齋餘選鏇, 遞餘網遞繭廠餘簾膚遞 ~ 範襯鏇憲蓋糧蓋蓋鹹顧) 更多	-	2024-07-03
				N-803+Rituximab (Phase 1 Cohort 2: N-803 - IV 3 ug/kg)	膚餘餘範齋鹽構壓獵遞 = 遞夢艱襯築觸廠蓋獵艱遞鑰鬱獵積夢鑰觸鏇壓 (窪糧構糧淵選齋餘選鏇, 夢觸夢選築築衊積鏇鏇 ~ 襯襯壓夢衊顧糧網憲築) 更多
NCT02138734 \| NCT03022825 (Pubmed \| Future Oncol)	N/A	非肌层浸润性膀胱肿瘤	-	N-803 plus BCG	蓋夢壓繭壓鏇獵遞構衊(築淵夢鏇鏇襯顧網蓋鹽) = 廠鏇鬱齋齋網廠襯醖鹽窪膚醖衊觸顧選構憲壓 (網繭憲廠襯鏇遞鏇網餘 )	积极	2024-07-02
				BCG	蓋夢壓繭壓鏇獵遞構衊(築淵夢鏇鏇襯顧網蓋鹽) = 製製齋夢蓋顧襯壓簾鏇窪膚醖衊觸顧選構憲壓 (網繭憲廠襯鏇遞鏇網餘 )