Celyad SA (CYAD) 首席执行官 Filippo Petti 于 2019年 第二季度 - 收益电话会议记录

[机器翻译] 电话会议 · 2019年08月26日 · 152 次阅读 · Celyad SA

Celyad SA (NASDAQ:CYAD) Q2 2019 Results Earnings Conference Call August 23, 2019 8:00 AM ET

Celyad SA(纳斯达克股票代码:[CYAD])2019年第二季度业绩收益电话会议2019年8月23日美国东部时间上午8:00

公司参与者

Anne Moore - VP Corporate Strategy
Filippo Petti - CEO and Interim CFO
David Gilham - VP of Research and Development
Frédéric Lehmann - VP of Global Clinical Development and Medical Affairs

  • Anne Moore - 企业战略副总裁
  • Filippo Petti - 首席执行官兼临时首席财务官
  • David Gilham - 研发副总裁
  • FrédéricLehmann - 全球临床开发和医疗事务副总裁

电话会议参与者

Raju Prasad - William Blair
Jim Birchenough - Wells Fargo
Ming Wong - Suntrust

  • 拉朱普拉萨德 - 威廉布莱尔
  • Jim Birchenough - 富国银行
  • 明王 - Suntrust

会议主持员

Ladies and gentlemen, thank you for standing by and welcome to Celyad's Second Quarter 2019 Financial and Business Update. At this time, all participants are in a listen-only mode. There will be prepared remarks by Celyad's Management, followed by a question-and-answer session. I must advise you all that the conference is being recorded.
And with that, I'd like to turn over the call to Dr. Anne Moore, Celyad's Vice President of Corporate Strategy. Please go ahead, madam.

女士们,先生们,感谢您的支持,并欢迎Celyad 2019年第二季度财务和业务更新。 此时,所有参与者都处于只听模式。 Celyad管理层将准备一些评论,随后是问答环节。 我必须告诉大家记录会议的所有内容。

有了这个,我想把这个电话交给Celyad公司战略副总裁Anne Moore博士。 夫人,请继续。

Anne Moore

安妮摩尔

Thank you, operator. And thank you, everyone, for joining us for our first half 2019 results call today. Joining me today is Filippo Petti, our CEO and Interim CFO; Dr. David Gilham, Vice President of Research & Development; and Dr. Frédéric Lehmann, Vice President of Global Clinical Development and Medical Affairs.
We will start the call with the business and clinical update, followed by an overview of the financials. And then we will open the line for your questions.
With that, I'd like to turn over the call to Filippo Petti. Please go ahead, Filippo.

谢谢运营商。 感谢大家今天参加2019年上半年的结果电话会议。 今天加入我的是我们的首席执行官兼临时首席财务官Filippo Petti; David Gilham博士,研发副总裁; 和全球临床开发和医疗事务副总裁FrédéricLehmann博士。

我们将通过业务和临床更新开始调用,然后概述财务状况。 然后我们将为您的问题打开界限。

有了这个,我想把这个电话交给Filippo Petti。 请继续,菲利普。

Filippo Petti

Filippo Petti

Thank you, Anne. And thank you, everyone, for joining us today. We have had a steady flow of news over the past few months, and the momentum in our business has been building steadily since the beginning of the year.
I am pleased to say that Celyad team has continued to execute on the development strategy for our NKG2D-based autologous CAR-T candidates, CYAD-01 and CYAD-02; as well as our allogeneic NKG2D-based candidates, CYAD-101 and our shRNA based allogeneic CYAD-200 series of pre-clinical candidates.
In addition to advancing our pipeline, we have also made strategic enhancements to our platform, including the introduction of a proprietary OptimAb manufacturing process. T cells continued to be a promising area of oncology therapeutic development, and we believe that Celyad is increasingly helping to advance the landscape.
In the past few months, we announced promising data from our ongoing Phase 1 clinical trials, both for the treatment of hematological malignancies and solid tumors. In June, we presented an update to our autologous relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome program at the European Hematology Association, or EHA meeting, in Amsterdam, Netherlands; shortly followed by the presentation of preliminary data and our refractory metastatic colorectal cancer program at the European Society of Medical Oncology World Gastrointestinal Congress or ESMO GI in Barcelona, Spain, in early July.
We continue to be pleased with the preliminary data generated to date, for both our autologous and allogeneic NKG2D-based CAR-T product candidates, CYAD-01 and CYAD-101 respectively, and look forward to future updates for the program in the months to come.
Importantly, in June, we also introduced our updated manufacturing process, OptimAb; which brings together the best attributes from previous manufacturing processes that we have – well, that we have a high level of manufacturing reliability with, in line with our clinical development strategy needs for our autologous program, CYAD-01 and CYAD-02.
With that as an intro, I will turn the call over to Dr. David Gilham, to discuss our updates on the Food and Drug Administration's clearance of the investigational new drug application or IND, for our next generation NKG2D-based CAR-T therapy, CYAD-02; and to provide our additional thoughts on our manufacturing process, OptimAb.
We will then turn the call over to Dr. Frédéric Lehmann, to highlight details around our clinical updates that we provided during the quarter, including the preliminary data presented at EHA Congress in June for the THINK and DEPLETHINK Phase 1 trials, evaluating CYAD-01 in the treatment of relapsed/refractory acute myeloid leukemia myelodysplastic syndrome, as well as the preliminary data announced from the SHRINK and alloSHRINK trial for CYAD-01 and CYAD-101 respectively in the treatment of metastatic colorectal cancer.
I will then provide the financial overview for the first half of the year and walk through the next milestones for Celyad, before we open the call for questions.

安妮,谢谢你。感谢大家今天加入我们。过去几个月我们的新闻源源不断,自年初以来,我们的业务发展势头稳步增长。

我很高兴地说,Celyad团队继续执行基于NKG2D的自体CAR-T候选人CYAD-01和CYAD-02的发展战略;以及我们的同种异体NKG2D候选物,CYAD-101和我们基于shRNA的同种异体CYAD-200系列临床前候选物。

除了推进我们的产品线,我们还对我们的平台进行了战略性改进,包括引入专有的OptimAb制造工艺。 T细胞仍然是肿瘤治疗发展的一个有前景的领域,我们相信Celyad正在越来越多地帮助推进这一领域。

在过去的几个月里,我们公布了我们正在进行的1期临床试验的有希望的数据,这些试验都用于治疗血液系统恶性肿瘤和实体瘤。 6月,我们在荷兰阿姆斯特丹的欧洲血液学协会或EHA会议上介绍了我们的自体复发/难治性急性髓细胞白血病和骨髓增生异常综合征计划的最新情况;随后在7月初,在西班牙巴塞罗那举行的欧洲肿瘤内科学会世界胃肠大会或ESMO GI上,提交了初步数据和我们的难治性转移性结直肠癌计划。

对于我们的自体和同种异体NKG2D CAR-T候选产品CYAD-01和CYAD-101,我们对迄今为止产生的初步数据仍然感到满意,并期待未来几个月内对该计划的更新。来。

重要的是,在6月份,我们还介绍了我们最新的制造工艺OptimAb;它汇集了我们以前制造工艺的最佳属性 - 我们拥有高水平的制造可靠性,符合我们的自体程序CYAD-01和CYAD-02的临床开发战略需求。

以此作为介绍,我将把呼叫转交给David Gilham博士,讨论我们关于食品和药物管理局对研究性新药申请或IND的批准的更新,以用于我们的下一代基于NKG2D的CAR-T疗法, CYAD-02;并提供我们对制造过程OptimAb的额外想法。

然后,我们将把呼叫转交给FrédéricLehmann博士,以突出我们在本季度提供的临床更新的详细信息,包括6月份EHA大会上针对THINK和DEPLETHINK第1阶段试验提交的初步数据,评估CYAD-01治疗复发/难治性急性髓性白血病骨髓增生异常综合征,以及SHRINK和alloSHRINK试验中分别对CYAD-01和CYAD-101治疗转移性结直肠癌的初步数据。

然后,我将提供今年上半年的财务概况,并在我们打开问题征集之前,逐步了解Celyad的下一个里程碑。

I will now turn the call over to David, the floor is yours.

我现在将电话转到大卫,地板是你的。

David Gilham

大卫吉尔罕

Thank you, Filippo. And thank you, everybody, again, for joining us today. As Filippo mentioned earlier, our programs and the work we are focused on, strategically positions Celyad at the front of the pack in terms of the advancements we're making in T cell therapies. And this is truly an exciting period for us.
Our vision is to help enhance our autologous NKG2D-based CAR-T cell therapy programs, to potentially deepen the breadth, the frequency and the duration of the clinical response in these refractory, highly rapidly progressing patient populations.
Over the past year, we have been working towards increasing the potency of CYAD-01 through different means. In June, we were excited to announce our new manufacturing process called OptimAb, which brings together the best attributes from previous manufacturing processes being worked on Celyad.
This includes an eight-day culture period, which is two days shorter than the previous antibody based manufacturing process, the incorporation of an NKG2D blocking antibody, and the inclusion of a selective PI3 kinase inhibitor that helps us to enrich T cells with a memory-like phenotype.
The OptimAb process is being developed in response to the increased understanding of the role of memory-like cells in CAR-T cell therapy, and center around in-house expertise in cell therapy, R&D and manufacturing.
Based on research data from out in vivo experiments, the OptimAb manufacturing process led to an improved antitumor activity in a highly aggressive AML model, compared to our standard antibody manufacturing process.
CMC amendments relating to OptimAb were recently accepted by regulators in both the United States and Belgium, and are now in effect under the current IND application for CYAD-01. Looking ahead, we will be treating all future patients in our autologous relapsed/refractory AML and MDS program with an OptimAb manufactured product.
With the recent introduction of the OptimAb manufacturing process, we are continuing to tap into our deep expertise and knowledge in cell therapy manufacturing, which is providing tremendous flexibility in our platform and the ability to produce autologous novel CAR-T cell therapies with a memory-like phenotype for increased persistence and CAR-T potency.
We're excited by the opportunity to initially assess out new OptimAb manufacturing process, CYAD-01 in the DEPLETHINK trial, under the current IND application for CYAD-01. Following additional assessment of our relapsed/refractory AML and MDS program for CYAD-01, we now plan to treat the first patients using the OptimAb manufacturing process, the CYAD-01 in Cohort 3 of the trial, which will evaluate 300 billion cells of CYAD-01 following preconditioning.
We expect to treat the first patient with CYAD-01 using the OptimAb manufacturing process by the end of September.
In addition to optimizing the manufacturing process, we have focused our efforts to optimize the persistence of our NKG2D-based autologous program by using short hairpin RNA or shRNA, to modulate the expression of the NKG2D ligands. This brings me to our novel next generation NKG2D-based CAR-T cell therapy, CYAD-02, which we announced at our R&D day earlier this year.

谢谢你,菲利波。再次感谢大家今天加入我们。正如Filippo之前提到的,我们的计划和我们所关注的工作,战略性地将Celyad定位在我们在T细胞疗法中取得的进步方面。对我们来说这真是一个激动人心的时期。

我们的愿景是帮助增强我们的基于NKG2D的自体CAR-T细胞治疗计划,以潜在地加深这些难治的,高度快速发展的患者群体的临床反应的广度,频率和持续时间。

在过去一年中,我们一直致力于通过不同方式提高CYAD-01的效力。 6月,我们很高兴地宣布我们的新制造工艺OptimAb,它汇集了Celyad先前制造工艺的最佳属性。

这包括8天的培养期,比之前的抗体制造工艺短两天,加入NKG2D阻断抗体,并加入选择性PI3激酶抑制剂,帮助我们用记忆增强T细胞。像表型一样。

OptimAb工艺的开发是为了响应对记忆样细胞在CAR-T细胞治疗中作用的更多了解,并以细胞治疗,研发和制造方面的内部专业知识为中心。

基于来自体内实验的研究数据,与我们的标准抗体制造工艺相比,OptimAb制造工艺在高度侵袭性AML模型中导致改善的抗肿瘤活性。

最近,美国和比利时的监管机构都接受了与OptimAb有关的CMC修订,现在根据目前的CYAD-01 IND申请生效。展望未来,我们将使用OptimAb制造的产品治疗我们的自体复发/难治性AML和MDS计划中的所有未来患者。

随着最近推出的OptimAb制造工艺,我们将继续利用我们在细胞疗法制造方面的深厚专业知识和知识,为我们的平台提供极大的灵活性,并能够生产具有记忆功能的自体新型CAR-T细胞疗法。像表型增加持久性和CAR-T效力。

我们很高兴有机会根据目前的CYAD-01 IND申请,在DEPLETHINK试验中初步评估新的OptimAb制造工艺CYAD-01。在对CYAD-01的复发/难治性AML和MDS计划进行额外评估后,我们现在计划使用OptimAb制造工艺治疗第一批患者,该试验的第3组中的CYAD-01将评估3000亿细胞的CYAD -01预处理后。

我们希望在9月底之前使用OptimAb制造工艺治疗首例CYAD-01患者。

除了优化制造过程外,我们还致力于通过使用短发夹RNA或shRNA来优化基于NKG2D的自体程序的持久性,以调节NKG2D配体的表达。这让我想到了我们新一代基于NKG2D的CAR-T细胞疗法CYAD-02,我们在今年早些时候的研发日宣布了这一疗法。

CYAD-02 and CYAD-01 are very similar in terms of being NKG2D-based therapies. The key difference is that CYAD-02 benefits from Horizon Discovery's shRNA SMARTvector technology, which is designed to deliver efficient knockdown with high specificity for NKG2D ligands MICA/MICB.
We believe these similarities and the preclinical work we have performed to-date, comparing CYAD-01 and CYAD-02, have positioned 02 to benefit from both the preclinical and clinical work we are already performing for CYAD-01 with respect to safety and dosing.
In addition, CYAD-02 has the potential to further enhance the antitumor activity beyond with CYAD-01 where both the manufacturers are using the OptimAb manufacturing process.
Regarding next steps for CYAD-02, the FDA has cleared the IND application for CYAD-02 for the treatment of relapsed/refractory AML and MDS. We are currently planning to move ahead with Phase I trial, to evaluate the safety and clinical activity of CYAD-02 with preconditioning chemotherapy of cyclophosphamide and fludarabine, or CyFlu, in the United States and in Europe in early 2020.
We believe that this will be the first example of the clinical testing of a single shRNA, targeting the expression of two independent genes, that provide some insight into the opportunities afforded by this technology.
With that, I would now like to turn over the call to Frédéric, Celyad's VP of Clinical Development. Frédéric?
Frédéric Lehmann
Thank you, David. I will start with the clinical update for our lead product candidate, CYAD-01 autologous NKG2D-based CAR-T in the THINK and DEPLETHINK trials for the treatment of relapsed/refractory AML and MDS patients. Then I will review the clinical data presented at ESMO World GI Congress for CYAD-01 in the SHRINK and CYAD-101 in the alloSHRINK trial for the treatment of refractory metastatic colorectal cancer patients, which continues to see encouraging clinical activity.
Starting with the Phase 1, THINK trial, evaluating multiple injections of CYAD-01 without any preconditioning chemotherapy, we announced in June at the AHA conference, preliminary data from the Cohort 10, assessing a more frequent schedule of CYAD-01 at 1 billion of cells that was generally reported to be well tolerated and showed better cell engraftment compared to biweekly injections of CYAD-01 without preconditioning.
We are currently enrolling patient Cohort 11 of the THINK trial, which is evaluating a more frequent dose schedule of 3 billion of cells of CYAD-01.
Important to note, both of these cohorts use CYAD-01 cell generate with the antibody as called mAb manufacturing process. We expect preliminary results from Cohort 11 in the fourth quarter of 2019.
In addition, we are also investigating CYAD-01, following preconditioning chemotherapy regimen, cyclophosphamide and fludarabine, for the treatment of relapsed/refractory AML and MDS in the dose escalation depleting trial.
Initial safety data from the first dose level of 100 million of cells of CYAD-01 have shown the regimen to be well tolerated and lead to a better cell engraftments of 01 cells, compared to the dose escalation segment of the THINK trial, evaluating the cycle of three infusions of CYAD-01 without preconditioning chemotherapy.

CYAD-02和CYAD-01在基于NKG2D的疗法方面非常相似。关键的区别在于CYAD-02受益于Horizo​​n Discovery的shRNA SMARTvector技术,该技术旨在为NKG2D配体MICA / MICB提供高效特性的敲除。

我们相信这些相似之处以及我们迄今为止所进行的临床前工作,比较CYAD-01和CYAD-02,已经确定02可以从我们在CYAD-01安全和剂量方面已经开展的临床前和临床工作中受益。

此外,CYAD-02有可能进一步增强CYAD-01的抗肿瘤活性,其中两家制造商都在使用OptimAb制造工艺。

关于CYAD-02的后续步骤,FDA已经批准了CYAD-02的IND应用,用于治疗复发/难治性AML和MDS。我们目前正计划推进I期试验,以评估CYAD-02在2020年初在美国和欧洲进行环磷酰胺和氟达拉滨(CyFlu)预处理化疗的安全性和临床活性。

我们相信这将是单个shRNA的临床测试的第一个例子,其针对两个独立基因的表达,这提供了对该技术提供的机会的一些了解。

有了这个,我现在想把这个电话交给Celyad的临床开发副总裁Frédéric。弗雷德里克?

FrédéricLehmann

谢谢你,大卫。我将从THINK和DEPLETHINK试验中用于治疗复发/难治性AML和MDS患者的主要候选产品CYAD-01自体NKG2D CAR-T的临床更新开始。然后,我将回顾ESMO世界GI大会在SHRINK和CYAD-101中为CYAD-01提交的alloSHRINK试验中用于治疗难治性转移性结直肠癌患者的临床数据,该试验继续看到令人鼓舞的临床活动。

从第1阶段,THINK试验开始,评估多次注射CYAD-01而不进行任何预处理化疗,我们在6月份的AHA会议上宣布了第10组的初步数据,评估了更频繁的CYAD-01计划在10亿通常报道细胞耐受良好并且与没有预处理的每两周注射CYAD-01相比表现出更好的细胞植入。

我们目前正在招募THINK试验的患者队列11,该试验正在评估30亿个CYAD-01细胞的更频繁的剂量时间表。

重要的是要注意,这两个队列都使用CYAD-01细胞与抗体一起产生,称为mAb制造过程。我们预计2019年第四季度的第11组的初步结果。

此外,我们还在预处理化疗方案,环磷酰胺和氟达拉滨后,调查CYAD-01,用于在剂量递增消耗试验中治疗复发/难治性AML和MDS。

来自1亿个CYAD-01细胞的第一剂量水平的初始安全性数据显示,与THINK试验的剂量递增部分相比,该方案具有良好的耐受性并导致01细胞更好的细胞植入,评估该循环三次输注CYAD-01无预处理化疗

As David highlighted earlier, we plan to move the OptimAb manufacturing process for CYAD-01, seamless into depleting trial and expect to have the results from the Cohort 3, evaluating 300 million of cells following CyFlu, by the year end 2019.
I would now like to turn to the recent data from the SHRINK and alloSHRINK trials, which were recently presented at ESMO World GI Congress, in July by Professor Van Cutsem. As a reminder, these two trials are similar to Phase 1 dose escalation study design that are evaluating the safety, the clinical activity and the CAR-T cell kinetics of the CYAD-01 and it's sister, CYAD-101 respectively for the treatment of refractory metastatic colorectal cancer patients.
The SHRINK study is an open label Phase 1 trial, evaluating multiple dose of the autologous CAR-T, CYAD-01, administered concurrently with the standard FOLFOX chemotherapy, while the sister study, the alloSHRINK trial, it's also an open label Phase 1 trial assisting multiple dose of the allogeneic CAR-T, CYAD-101 administered also concurrently with the FOLFOX chemotherapy.
Looking at the primary endpoint of these two trials, we have observed both regimens appear very well tolerated. Importantly, in the alloSHRINK trial, CYAD-101 show no clinical evidence of graft versus host disease across the patients treated to-date, which is important that it support the ability of our novel inhibitory peptide TIM or T cells inhibitory molecules to reduce signaling of the TCR complex in allogeneic cells.
With respect to the alloSHRINK trial, we continue to observe encouraging preliminary antitumor activity, as reported at ESMO GI in Barcelona in July, including one patient's experience partial critical response and treat patients with stable disease, out of the six patients that have been heavily pretreated relapsed/refractory colorectal cancer patients.
Of note, all patients in the alloSHRINK trial have previously received the FOLFOX chemotherapy. As previously announced, we have decided to enroll additional patients at the 1 billion cell dose per injections in the alloSHRINK trial, and now anticipated up to nine patients in this cohort. We anticipate to announce the additional data for the dose escalation of alloSHRINK trial by the year end 2019.
I think it's also worth mentioning that, to our knowledge, the alloSHRINK trial is a very first study with an allogeneic CAR-T cell therapy from the treatment of a solid cancer indications.
With that, let me now turn the call back to Filippo. Filippo?

正如David先前强调的那样,我们计划将针对CYAD-01的OptimAb制造流程无缝地转移到耗尽试验中,并期望在2019年年底之前得到第3组的结果,即CyFlu之后的3亿个细胞。

我现在想谈谈SHRINK和alloSHRINK试验的最新数据,这些试验最近于7月由Van Cutsem教授在ESMO世界地理标志大会上提出。提醒一下,这两项试验类似于第一阶段剂量递增研究设计,分别评估CYAD-01及其姐妹CYAD-101的安全性,临床活性和CAR-T细胞动力学,用于治疗耐火材料。转移性结直肠癌患者。

SHRINK研究是一项开放标签的1期试验,评估多剂量的自体CAR-T,CYAD-01,与标准FOLFOX化疗同时进行,而姐妹研究,alloSHRINK试验,它也是一个开放标签的1期试验辅助多剂量的同种异体CAR-T,CYAD-101也与FOLFOX化学疗法同时给药。

观察这两项试验的主要终点,我们观察到两种方案似乎都具有良好的耐受性。重要的是,在alloSHRINK试验中,CYAD-101在迄今为止治疗的患者中没有显示移植物抗宿主病的临床证据,这一点很重要,它支持我们的新型抑制性肽TIM或T细胞抑制分子减少信号传导的能力。同种异体细胞中的TCR复合物。

关于alloSHRINK试验,我们继续观察到令人鼓舞的初步抗肿瘤活性,正如7月在巴塞罗那的ESMO GI所报告的那样,其中包括一名患者经历部分临界反应并治疗病情稳定的患者,其中六名患者经过大量预处理复发/难治性结直肠癌患者。

值得注意的是,alloSHRINK试验中的所有患者之前都接受过FOLFOX化疗。正如之前所宣布的那样,我们决定在alloSHRINK试验中以每次注射10亿细胞剂量招募更多患者,现在预计该队列中将有9名患者。我们预计到2019年年底将公布alloSHRINK试验剂量递增的额外数据。

我认为值得一提的是,据我们所知,alloSHRINK试验是第一项使用同种异体CAR-T细胞疗法治疗实体癌症适应症的研究。

有了这个,现在让我把电话转回菲利波。菲利波?

Filippo Petti

Filippo Petti

Thank you, Frédéric. Turning to the financials. I'd just like to remind you all that our full financial details are available on the Celyad website in both French and English. For the first half of 2019, research and development expenses totaled €12.7 million, compared to €11.1 million for the same period in the previous year.
The increase in 2019 expense reflects the organic growth of the Company's operations for both preclinical and clinical activities, driven primarily by increased spending related to our key clinical studies for CYAD-01 and CYAD-101, as well as increased spending associated with the development of our shRNA allogeneic platform and CYAD-200 series.
General and administrative expenses totaled €4.5 million in the first half of 2019, compared to €5.5 million for the same period of 2018. For the first half of 2019, the Company's other income and other expense, mainly include non-expense, cash expenses related to liability assessment required by International Financial Reporting Standards or IFRS, related to the advancement of the Company's NKG2D-based CAR-T candidates.
Overall, the Company has posted €0.4 million in net income for the first half of 2019, against a €3.9 million net loss for the first half of 2018.
Net operational cash burn, which excludes non-cash events, was €16.0 million in the first half of 2019, compared to €13.9 million for the same period of 2018. The Company's loss for the first half of 2019 amounts to €16.0 million versus a net loss of €18.5 million for the same period of 2018.
The Company ended the first half of 2019 with a treasury position of €33.7 million, compared to €62.4 million as of December 31, 2018. Celyad confirms it previous position, and its treasury position based on the current scope of activities should be sufficient to fund operating expenses and capital expenditure requirements until mid-2020.
In closing, the start of 2019 has the Celyad team very exciting. We look forward to achieving additional milestones over the next several months, including the treatment of the first patient with the CYAD-01 OptimAb process at 300 million produced with the OptimAb manufacturing process and the DEPLETHINK Phase 1 trial, which is expected by the end of September.
Also we expect results from the Cohort 11 of the THINK Phase1 trial and Cohort 3 of the DEPLETHINK trial, availing CYAD-01 produced with the manufacturing process for the treatment of relapsed refractory AML and MDS by the end of the year as well. Additional results from the dose escalation Phase 1 alloSHRINK trial, evaluating CYAD-101 for the treatment of metastatic colorectal cancer are anticipated by year end 2019.
Looking into 2020, we plan to announce the initiation of the Phase 1 dose escalation trial, availing CYAD-02, following preconditioning chemotherapy for the treatment of relapsed refractory AML and MDS which is expected in early 2020 as well as the submission of the IND application for CYAD-211, our shRNA based allogeneic BCMA CAR-T candidate for the treatment of patients with multiple myeloma, which is expected during the first half of 2020.

谢谢你,Frédéric。转向财务。我想提醒大家,我们的完整财务细节可以在Celyad网站上以法语和英语提供。 2019年上半年,研发费用总计1270万欧元,而去年同期为1110万欧元。

2019年费用的增加反映了公司在临床前和临床活动中的有机增长,主要是由于我们对CYAD-01和CYAD-101的关键临床研究的支出增加,以及与开发相关的支出增加我们的shRNA同种异体平台和CYAD-200系列。

2019年上半年的一般和行政费用总计为450万欧元,而2018年同期为550万欧元.2019年上半年,公司的其他收入和其他费用主要包括非费用,现金支出与国际财务报告准则或国际财务报告准则要求的与本公司基于NKG2D的CAR-T候选人晋升有关的责任评估有关。

总体而言,公司2019年上半年的净收入为40万欧元,而2018年上半年的净亏损为390万欧元。

不包括非现金事件的净运营现金燃烧在2019年上半年为1,600万欧元,而2018年同期为1390万欧元。公司2019年上半年的亏损为1600万欧元。 2018年同期净亏损1850万欧元。

截至2019年上半年,公司的财务状况为3370万欧元,而截至2018年12月31日为6240万欧元.Celyad确认其先前的立场,其基于当前活动范围的财务状况应足以资金运营费用和资本支出要求,直到2020年中期。

最后,2019年的开始让Celyad团队非常兴奋。我们期待在未来几个月内实现额外的里程碑,包括使用OptimAb制造工艺和DEPLETHINK第一阶段试验生产的第一批使用CYAD-01 OptimAb工艺的患者治疗3亿,预计到年底九月。

我们还期望THINK Phase1试验的队列11和DEPLETHINK试验的队列3的结果,利用CYAD-01生产的复合难治性AML和MDS的制造工艺也将在年底前生产。剂量递增的第1阶段alloSHRINK试验的其他结果预计将于2019年年底评估CYAD-101治疗转移性结直肠癌。

展望2020年,我们计划宣布启动第一阶段剂量递增试验,利用CYAD-02,预处理化疗治疗复发难治性AML和MDS,预计在2020年初以及IND申请提交对于CYAD-211,我们基于shRNA的同种异体BCMA CAR-T候选物用于治疗多发性骨髓瘤患者,预计将在2020年上半年进行。

And with that, I'll now turn the call over to your questions. Operator?

有了这个,我现在将把电话转到你的问题上。操作?

问答环节

[Operator Instructions] We will now take our first question. Please go ahead. Your line is now open.

[操作员说明]我们现在提出第一个问题。 请继续。 你的生产线现已开放。

Raju Prasad

Hey, it's Raju Prasad from William Blair. Thanks for taking the question. Just a couple for me. Just how you think about how you are going to take CYAD-101 forward and potentially hemalignancies and given your producing next generation autologus NKGD2
mica/micb. How are you thinking about potentially using CYAD-02 construct in the allo platform moving forward? Thanks.

嘿,这是William Blair的Raju Prasad。 谢谢你提出这个问题。 对我来说只是一对。 您是如何思考如何使用CYAD-101前进和潜在的血管生成并为您生产下一代自动化NKGD2

MICA / B。 您是如何考虑在allo平台中使用CYAD-02构建的? 谢谢。

Filippo Petti

Filippo Petti

Very good. I think they're great questions and once that we've been discussing over the last few months in terms of how maybe 101 could continue to be evaluating an additional malignancies perhaps and hematological malignancies or other solid tumors. I think we've seen encouraging data so far from the 101 program that Fredrick described as ESMO GI in the alloSHRINK trial we'll have an additional update at the end of the year for that trail.
I think based on that assessment we will look to see how we could pivot that and is it something we could look at in AML, is it something we should further investigate into colorectal cancer what might be the right approach in colorectal cancer, I think we'll let the data set kind of dictate that. But I do believe there is an opportunity for us to leverage CYAD-101 and based on the data that we've seen so far, what that will entail what indications that will to your point also would be led little bit by the initial OptimAb data that will have for 01 and perhaps the 02 program.
So I think, as you know it's never just a single data point that will lead you in a specific direction. We'll take the -- look at the totality of the data and strategically how that resolve for us to think about next steps but 101 is top of mind for us and making sure that we fully evaluate that and look at its true prospects as a potential of shelf NKG2D- CAR-T.

很好。我认为这些都是很好的问题,而且一旦我们在过去几个月里一直在讨论101可能会继续评估其他恶性肿瘤,也许是血液系统恶性肿瘤或其他实体肿瘤。我认为迄今为止我们已经看到了令人鼓舞的数据,这个数据来自于Fredrick在alloSHRINK试验中称为ESMO GI的101计划,我们将在今年年底对该计划进行额外更新。

我认为,基于这一评估,我们将看看我们如何能够实现这一目标,这是我们在AML中可以看到的东西,我们是否应该进一步研究结肠直肠癌可能是结直肠癌的正确方法,我想我们让数据集有点决定。但我确实认为我们有机会利用CYAD-101并基于我们目前所见的数据,这将导致最初的OptimAb数据会导致什么样的迹象。那将是01和02程序。

所以我认为,正如您所知道的那样,从来没有一个数据点可以引导您走向特定的方向。我们将采取 - 查看数据的总体情况,并从战略角度考虑如何决定我们考虑后续步骤,但101是我们的首要考虑因素,并确保我们对此进行全面评估,并将其真实前景看作是货架NKG2D-CAR-T的潜力。

Raju Prasad

It seems although you guys have obviously executed pretty well in regulatory filings, that was very pretty quick responses from the agencies. As you are thinking about moving in Phase 2 or kind of more of a registrational focus data set, when do you plan engagement latency on the data that you're producing from kind of all these different durations, would you plan on kind of moving over to Phase 2 first and then to Phase 2 meeting or plan to data to them before kind of initiating a Phase 2 study next year? Thanks.

看起来虽然你们在监管文件中表现得相当不错,但这些机构的回复非常快。 当您考虑进入第2阶段或更多注册焦点数据集时,您何时计划从所有这些不同持续时间生成的数据的参与延迟,您是否计划移动 首先进行第2阶段,然后进入第2阶段会议或计划向他们提供数据,然后再开始明年的第2阶段研究? 谢谢。

Filippo Petti

Filippo Petti

Yes, that's a good question. I think the way we think about it is we're – I think first for us is to let's kick into the DEPLETHINK trial begin to enroll that first patient with the OptimAb process, and that makes its way through the channel we begin to collect the patients there.
I think we will base our next step into a Phase 2 based on let's say 12 to 15 patients on that OptimAb product to make an assessment as to how we move the forward with the program. And I think on that and that data set, as we kick into maybe perhaps the Phase 2, we will go out to the agency and speak to them and thinking about how we could leverage that initial data set and that how that can move towards not only in initial Phase 2 but then eventually registrational as well.
So I think for us, what we're looking for and the ideas not only from the OptimAb process and DEPLETHINK, but in general, I think we want to get through a 12 to 15 patient data set for us to say its full speed ahead and let's have a discussion with the agency in thinking about what exactly needs to be in process, and next steps for us to get through registration. Maybe I'll just turn it over to Fred, to provide some additional thoughts as well.

是的,这是一个很好的问题。我认为我们考虑的方式是我们 - 我认为首先让我们进入DEPLETHINK试验,开始为第一位患者注册OptimAb流程,然后通过我们开始收集的渠道那里的病人。

我认为我们的下一步将基于让我们说这个OptimAb产品的12到15名患者进行评估,以评估我们如何推进计划的进展。我想在那个和那个数据集上,当我们可能进入第二阶段时,我们会去机构并与他们交谈并思考我们如何利用初始数据集以及如何将其转向仅在初始阶段2,但最终也是注册。

因此,我认为对于我们来说,我们正在寻找的是什么以及不仅来自OptimAb流程和DEPLETHINK的想法,但总的来说,我认为我们希望通过一个12到15个患者数据集来让我们说明它的全速前进让我们与代理商讨论在过程中究竟需要什么,以及我们通过注册接下来的步骤。也许我会把它交给弗雷德,以提供一些额外的想法。

Frédéric Lehmann

FrédéricLehmann

Just an additional thoughts, the way to interact with authorities will be obviously depending of the data that we will generate and so as mentioned by Filippo, the objective response rate and the duration of the response rate with OptimAb out of those 12, 14 patient, 14 patient will be OptimAb and as you know when we talk about registration study, we have to knock on the door and the authority is not asking about advice only but to come with some proposal, and therefore the registration study design will depending obviously of the rate of the objective response and the processes of those.
Do we have for example for United States, do we have a breakthrough type designation, do we have to go for randomization versus best supportive, etcetera. So all about that to say that we need to first generate data and therefore accordingly we will know how to approach and what will be our tactic to discuss with the to the folks.

只是一个额外的想法,与当局互动的方式显然取决于我们将产生的数据,正如Filippo所提到的,客观响应率和OptimAb对12,14名患者的响应率持续时间, 14名患者将是OptimAb,正如您所知,当我们谈论注册研究时,我们必须敲门,权威人员不仅仅询问建议,而是提出一些建议,因此注册研究设计将明显取决于 客观反应的速度和过程。

例如,对于美国,我们是否有突破性的类型指定,我们是否必须进行随机化而不是最佳支持等等。 所以关于这一点,我们需要首先生成数据,因此我们将知道如何处理以及与人们讨论的策略是什么。

Filippo Petti

Filippo Petti

And I would, the last thing I would add to that Raj, for us by the end of the year we will have on the monotherapy approach and using the schedule authorization on top of the THINK dose escalation, somewhere between 15 to 20 patients worth of data. We'll look to have a similar size in terms of the initial dose cohorts and DEPLETHINK as well as some additional incremental data from OptimAB and we'll have somewhere between 15 to 20 patients there as well.
We'll have a more wholesome data set for the OptimAB in the first half of 2020 but I would just say to note the way that DEPLETHINK and THINK are set up is that they are seamless in design and that they can go from a Phase 1 into a Phase 2 for allow us to pursue the signal before really having to go out to the agencies and having a discussion with full data. We can quickly move through and collect as much data if we're seeing the right signals.

对于我们来说,最后一件事我会加入到Raj中,对于我们来说,我们将采用单药治疗方法并在THINK剂量递增的基础上使用计划授权,大约15至20名患者 数据。 我们看起来在初始剂量组和DEPLETHINK方面具有相似的大小以及来自OptimAB的一些额外增量数据,我们也将在那里有15到20名患者。

我们将在2020年上半年为OptimAB提供更加健康的数据集,但我只想说明设置DEPLETHINK和THINK的方式是它们在设计上是无缝的,并且它们可以从第1阶段开始 进入第2阶段,允许我们在真正不得不前往代理商并与全部数据进行讨论之前追踪信号。 如果我们看到正确的信号,我们可以快速移动并收集尽可能多的数据。

会议主持员

We will take our next question, caller please go ahead. Your line is now open.

我们将采取下一个问题,来电者请继续。 你的生产线现已开放。

Jim Birchenough

Its Jim Birchenough from Wells Fargo. Hi, Phil. Thanks for the call and all the detail, so what sounds like you know pretty broad plans for development and continued efforts to pursue next generation candidates here, how do you think about ending the cash runway and perhaps you could talk to non-dilutive means to give more space with the cash runway and if you might be able to elaborate some of your TCR, IP and allogeneic T-Cell IP?

来自富国银行的Jim Birchenough。 嗨,菲尔。 感谢您的电话和所有细节,所以听起来您知道相当广泛的发展计划,并继续努力在这里寻找下一代候选人,您如何考虑结束现金跑道,也许您可以通过非稀释手段与 如果你能够详细说明你的一些TCR,IP和同种异体T-Cell IP,可以在现金跑道上留出更多空间吗?

Filippo Petti

Filippo Petti

Yes, great question, Jim. You know as we announced it in the press release and in the prepared remarks, our cash run way to goes to mid-2020. We're obviously looking at various opportunities to how to extend that as we kick into 2020 and we'll see data throughout the year.
I think to your point, can we get credit for the data we've generated so far from the AML program. That is certainly top of mind and thinking that maybe areas you know, we would unlikely go out and commercialize maybe territories such as Asia. I think top of mind is also we have - we had a collaboration and an agreement with a previous pharma company around 101, and can build on the data that we have now and continue to pursue that as well as an opportunity to bring in some non-dilutive business development cash.
And I think to your last point around the IP, I think for us, we're certainly encouraged by the activity we're seeing in the allogeneic space. I think we want more of that because we do believe that we're sitting on a patent, a state that could be very helpful in terms of us bringing in non-dilutive cash from potential strategic partners similar to what we did in May of 2017 with Novartis around the two targets that we provided access to.
So I think we're looking at multiple avenues. We recognize that we have lofty plans and we need to fund that. And we're looking at different instruments that can get us there.

是的,很好的问题,吉姆。你知道,正如我们在新闻稿和准备好的评论中宣布的那样,我们的现金运行方式将持续到2020年中期。我们显然正在寻找各种机会,以便在我们进入2020年时如何扩展它们,我们将全年看到数据。

我认为,对于我们迄今为止从AML计划中生成的数据,我们能否获得一致好评。这当然是最重要的,并且认为可能你知道的领域,我们不太可能将亚洲等地区商业化。我认为最重要的是我们 - 我们与101之前的一家制药公司进行了合作和协议,并且可以利用我们现在拥有的数据并继续追求这一目标,并有机会引入一些非 - 稀薄的业务发展现金。

我认为,关于知识产权的最后一点,我认为对于我们来说,我们在同种异体空间中看到的活动肯定会让我们感到鼓舞。我认为我们还需要更多,因为我们确实认为我们正在申请专利,这个国家对于我们从潜在的战略合作伙伴那里引入非稀释性现金非常有帮助,类似于我们在2017年5月所做的诺华与我们提供访问的两个目标。

所以我认为我们正在寻找多种途径。我们认识到我们有高尚的计划,我们需要为此提供资金。我们正在寻找可以让我们在那里的不同仪器。

Jim Birchenough

And then just maybe on the technology side, can you just speak or maybe quantify the benefit of knocking out MICA/MICB in terms of reducing fratricide potentially. and how you think that's impacted prior results and how you think could be improved with that second generation effort when you reduce the potential for fratricide?

然后可能只是在技术方面,你能说出或者可能量化MICA / MICB在减少自相残杀方面的好处。 以及当你减少自相残杀的可能性时,你认为这会影响先前的结果以及如何通过第二代努力改善你的想法?

David Gilham

大卫吉尔罕

I think that's a pretty complicated question to ask, I'm afraid. Because the question of fratricide that we see in vitro, there is a real question of whether this actually applies in vivo. And of course, our clinical trials were helped define that over time.
But aside from fratricide, one of the – I think, potential advantage is knocking down MICA/MICB may have is that they may also reduce the sensitivity of these cells to being targeted themselves by other natural killer cells resident in the patient.
So while we can see in vitro that there is certainly an effect on fratricide in terms of the fact that there is a protection against that in vitro phenomena. And what happens in the patient, I'm afraid, we'll really have to depend on whether fratricide is an important or not. And we really have very little evidence to say that fratricide is important in the patients.
But I think there's also other added benefits knocking down MICA/MICB could have that go beyond that straight forward - that phenomenon. So without being able to really give you a hard quantification, we're expecting to see some potentially different impacts.
And, of course, one of the broader things is whether the impact of MICA/MICB expression on T cells may be applicable beyond just the NKG2D approach.

我认为这是一个非常复杂的问题,我担心。因为我们在体外看到了自相残杀的问题,所以存在一个真正的问题,即这实际上是否适用于体内。当然,随着时间的推移,我们的临床试验得到了帮助。

但除了自相残杀之外,其中一个 - 我认为,潜在的优势是击倒MICA / MICB可能会降低这些细胞对居住在患者体内的其他自然杀伤细胞的敏感性。

因此,虽然我们可以在体外看到,对于自相残杀肯定会对这种体外现象有保护作用。而且,患者会发生什么,我担心,我们真的要依赖于自相残杀是否重要。我们确实很少有证据表明自相残杀对患者很重要。

但是我认为还有其他额外的好处,可以让MICA / MICB超越直线 - 这种现象。因此,如果不能真正为您量化,我们期望看到一些可能不同的影响。

当然,更广泛的事情之一是MICA / MICB表达对T细胞的影响是否可能适用于NKG2D方法。

Jim Birchenough

And then just one final question just on cost of goods, it's becoming an important consideration. And maybe you could speak in general terms on the OptimAb process, how competitive your cost of goods are with – what we know about existing CAR-T's that are out there on the market?

然后只是关于商品成本的最后一个问题,它正成为一个重要的考虑因素。 也许您可以就OptimAb流程概括地说一下,您的商品成本与竞争力有多大 - 我们对市场上现有CAR-T的了解程度如何?

David Gilham

大卫吉尔罕

It's very hard to quantify at the moment, because we're still in the Phase 1 stage of our clinical trials. And what I can say at least from our own experience is that we've not seen a particular impact of the OptimAb process as compared to our mAb process. Mainly, because there were maybe an additional small cost of a reagent, we're culturing the cells for a shorter period of time.
But I just really can't provide you with hard quantifiable data at the moment, because we're just at such an early stage and continuing to develop these. And we're also not sure what our recommended dose will be for the future. And so, I don't think we can really make a fair comparison as yet to what's really being discussed in the - particularly with respect to licensed products at the moment.

目前很难量化,因为我们仍处于临床试验的第一阶段。 至少根据我们自己的经验,我能说的是,与我们的mAb工艺相比,我们没有看到OptimAb工艺的特殊影响。 主要是因为试剂可能需要额外的小成本,我们将培养细胞的时间更短。

但我现在真的无法为您提供可量化的数据,因为我们正处于这样一个早期阶段并继续开发这些数据。 而且我们也不确定我们推荐的剂量将来会是什么。 因此,我认为我们不能真正做出公正的比较,特别是目前正在讨论的许可产品。

Filippo Petti

Filippo Petti

Yes, the only thing I would add to that, Jim, is obviously I think we're well hedged. Obviously, our targets in 01 and 02 are our lead programs. But we are looking at non-gene edited allogeneic approaches and led by CYAD-101 and certainly the focus to drive forward our shRNA platform in the CYAD-200 series. As we look to the future of that, we do believe that an allogeneic product specifically for treating patients with perhaps solid tumors may have to be allogeneic.
So I think we're well positioned both across autologous and allogeneic, and certainly keeping top of mind the cost benefit to patients and to the system here.

是的,我唯一想补充的是,吉姆,显然我认为我们已经很好地对冲了。 显然,我们在01和02的目标是我们的主要计划。 但是我们正在研究由CYAD-101领导的非基因编辑的同种异体方法,并且肯定是在CYAD-200系列中推动我们的shRNA平台的重点。 当我们展望未来时,我们确实认为专门用于治疗患有实体瘤的患者的同种异体产品可能必须是同种异体的。

因此,我认为我们在自体和同种异体方面都处于良好的位置,并且当然首先要考虑到患者和系统的成本效益。

David Gilham

大卫吉尔罕

Sorry, Jim, can I just add one further caveat to that as well. And the fact that you have manufacturing costs, and cost of goods are really related to manufacturing success rates. And the reason why we work through the iterations of our manufacturing process, and particularly the moment to the mAb and Ab building on the mAb has really been around improving our manufacturing success rate, which I think is very high, particularly for where we are and the patients we're treating.
And so that, and that we will have a beneficial effect and the cost of goods in the end. So when we're thinking about this as we go through these modifications, one of the things that are top of mind really is to ensure that we maintain that manufacturing success rate. So we're not looking to generate cells that are particularly free not tight but then compromising in our manufacturing success. And so I think that's an important thing to remember.

对不起,吉姆,我还可以再添加一个警告。 而且,制造成本和商品成本与制造成功率密切相关。 我们之所以能够完成制造过程的迭代,特别是mAb和Ab建立在mAb上的时刻,实际上是在提高我们的制造成功率,我认为这是非常高的,特别是对于我们现在和 我们正在治疗的病人。

因此,我们将最终产生有利影响和商品成本。 因此,当我们在进行这些修改时考虑到这一点时,最重要的事情之一就是确保我们保持制造成功率。 因此,我们不打算生产特别自由而不紧张的电池,但却会影响我们的制造成功。 所以我认为这是一件值得记住的重要事情。

会议主持员

Thank you. We will take our next question. Please go ahead. Your line is now open.

谢谢。 我们将接受下一个问题。 请继续。 你的生产线现已开放。

身份不明的分析师

Thanks for the update. I was wondering if you could clarify again. I understand that you would have about 12 to 14 patients in the Cohort 3 by mid-2020 and about 15 to 20 in Cohort 11 around the same timing, how many patients would you be able to given up an update by 4Q 2019 for the hemato arm.
For the alloSHRINK trial, so I understood that to date you have not seen any [indiscernible] this to date, so after the ESMO meeting reporting, with regard to the reporting 2019 do you plan to again present the data on conferences like ASH or SITC or do you have another timing for reporting and then a very final last questions on the R&D spending going forward, if you could give us roughly a great on the existing or the ongoing clinical programs hemato and CYAD-01, solid CYAD-101 and then the next generation programs. Thanks.

感谢更新。 我想知道你是否可以再次澄清。 据我所知,到2020年中期,你将在队列3中有大约12到14名患者,在同一时间内大约有15到20名患者,你能在2019年4季度之前放弃更新的患者数量 臂。

对于alloSHRINK试验,所以我理解到目前为止您还没有看到任何[音频不清晰],所以在ESMO会议报告之后,关于报告2019,您是否计划再次提供ASH或SITC等会议的数据 或者你有另一个报告的时机,然后是关于研发支出的最后一个最后的问题,如果你能给我们大致了解现有的或正在进行的临床项目hemato和CYAD-01,固体CYAD-101然后 下一代计划。 谢谢。

Filippo Petti

Filippo Petti

Yes, so I would say from the hematological standpoint we're on target for let's say the monotherapy the THINK combined with the THINK schedule optimization cohorts to have somewhere between 15 to 18 patients work to data by the end of the year. So that's the first one.
And again remember those are based on the MAB antibody, they are the MAB manufacturing process. For the DEPLETHINK trial, we provided an update on six patients so far in the first dose cohort, we are going to three issue now we've treated going through, ongoing through the MAB 300 Cohort 3 process where we'll look to have an additional three patients there and then layering the OptimAB manufacturing CYAD-01 product to somewhere in three additional patients report perhaps thinking about dose escalating up. So I think from an overall perspective and that we think we'll have somewhere maybe between 12 patients or so 10 to 12 patients by year end.
Now the comment around the OptimAB, I think what we're trying to strive is they think for us to make a fair assessment on OptimAB. We would like to have somewhere between 12 and 15 patients to make kind of go, no go decision on that approach to using 01.
You know, based on what we would see for the program on folding over the next several months and recruitment rates, we think by maybe mid-next year could we get to, it's probably going to be less than that but our goal is to get to maybe nine patients or so or a little bit more from that process. But I would say, the overall goal for OptimAB is 12 to 15. Where that falls exactly in the timelines will be remain to be seen depending on how we look at the program, see the initial data from 300 and if it's necessary for us to access grade up to 1 billion dose level. So its kind of a moving target at this point but I think overall the picture is we won't have a dozen patients from the OptimAB process from us to make a decision.
Now as we kick into 02, 02 study will kick in early next year, that's a dose escalation three plus three study design, that data set is most likely probably a year end 2020 update in terms of the AML program. And so I think that's kind of how that fold for how we think about AML over the next several quarters.
With respect to the CYAD-101 data, as you saw we did have the updated ESMO GI. We are recruiting as Frederic said at that high dose level an additional up to nine patients there and we will hope to have update by year end 2019 and we are focused on a potential medical conference to present that data. And may not be for the full data set that we're looking for the dose level but we hope to have a more meaningful update to build upon what was presented at ESMO GI at Barcelona.

是的,所以我从血液学的角度说我们的目标是让我们说THINK与THINK计划优化队列相结合的单药治疗可以让年龄在15到18之间的患者在数据之前工作。这是第一个。

再次记住那些基于MAB抗体,它们是MAB制造过程。对于DEPLETHINK试验,我们提供了迄今为止在第一剂量队列中的六名患者的最新情况,我们现在将讨论三个问题,我们已经通过MAB 300队列3进程进行治疗,我们将寻求在那里另外三名患者,然后将OptimAB制造CYAD-01产品分层到另外三名患者的某个地方,可能会考虑剂量上升。因此,从整体的角度来看,我们认为到年底,我们可能会在12名患者之间或者10到12名患者之间。

现在关于OptimAB的评论,我认为我们正在努力争取的是他们认为我们对OptimAB进行了公平的评估。我们希望在12到15名患者之间做一些事情,没有决定使用01的方法。

你知道,根据我们对未来几个月的折叠计划以及招聘率的看法,我们认为可能会在明年年中到达,它可能会比这少,但我们的目标是获得大约九个病人或者那个过程中的一点点。但我想说,OptimAB的总体目标是12到15.根据我们对程序的看法,在时间轴上完全落后的情况还有待观察,请参阅300的初始数据以及是否有必要访问等级高达10亿剂量水平。所以它在这一点上是一个动人的目标,但我认为总的来说,我们不会有来自我们的OptimAB流程的十几名患者做出决定。

现在,当我们进入02,02研究将在明年初开始,这是一个剂量升级三加三研究设计,该数据集最有可能是在AML计划方面的2020年末更新。所以我认为这对我们未来几个季度对AML的看法有多么重要。

关于CYAD-101数据,如您所见,我们确实拥有更新的ESMO GI。我们正在招募弗雷德里克说,在高剂量水平,在那里增加了多达9名患者,我们希望在2019年年底之前进行更新,我们将专注于一个潜在的医疗会议来提供这些数据。并且可能不是我们正在寻找剂量水平的完整数据集,但我们希望有更有意义的更新,以建立在巴塞罗那ESMO GI上呈现的内容。

身份不明的分析师

And with regard to some of the R&D spending, breakdown for the second half of 2019 on the ongoing clinical trials versus next generation program, if its possible to give us some indication there?

关于一些研发支出,2019年下半年正在进行的临床试验与下一代计划的细分,是否有可能在那里给我们一些指示?

Filippo Petti

Filippo Petti

Yes, it's a good question. I don't think we can provide the full granularity but I think we continued to have cash runway as we set to mid-2020. As we think about the programs, the larger set of the burn is sitting with the 01 program. As you can imagine because that's already up in running.
Now obviously on the 02 we are working on some planning of that which is certainly taking a bite into the R&D spend as well as the work that David and the team are focused on with respect to getting the CYAD-220 series and particular, CYAD-211 our BCMA candidate filed for IND by middle of next year. So it's a fair balance, you know between clinical and preclinical but those are really the major levers for us in terms of how we think about the programs over the next several quarters.

是的,这是一个很好的问题。 我认为我们不能提供完整的粒度,但我认为我们将在2020年中期继续拥有现金跑道。 当我们考虑这些程序时,更大的一组刻录就是01程序。 你可以想象,因为那已经在运行了。

现在显然在02上我们正在制定一些计划,这肯定会影响到研发支出以及David和团队在获得CYAD-220系列和特别是CYAD-方面所关注的工作。 211我们的BCMA候选人将在明年年中之前申请IND。 所以这是一个公平的平衡,你知道在临床和临床前,但对于我们如何考虑未来几个季度的计划,这些确实是我们的主要杠杆。

会议主持员

We will now take the next question. Caller please go ahead. Your line is now open. We can hear you please go ahead.

我们现在将讨论下一个问题。 来电者请继续。 你的生产线现已开放。 我们可以听到你请继续。

身份不明的分析师

Hi. This is Ming Wong on for Peter Lawson, at Suntrust. Thank you so much for taking our questions. Lets really quick talking about OptimAB, it sounds like you guys are now planning on to Cohort 3 after additional effects can you talk a little bit more about what you saw in this assessment that made you say Cohort 3 as oppose I think previously you guys mentioned in Cohort 4 and also the timing can you certain push back little from August end of September?

你好。 这是Ming Wong在Suntrust的Peter Lawson。 非常感谢你提出我们的问题。 让我们快速谈论OptimAB,听起来你们现在正计划进入第3组,在额外的效果之后,你可以多谈一下你在这次评估中所看到的内容,这让你说群组3反对我想以前你们提到过 在队列4中,你可以确定从9月底开始的时间很少吗?

Filippo Petti

Filippo Petti

So I think with respect to the OptimAB and obviously we provided an update in late June following the clearance of the amendment from the FDI with respect to using OptimAB manufactured product in the CYAD-01 program. So really I would say there's a few reasons as to why we move to Cohort 3 of the DEPLETHINK trial.
I think overall we were looking at the CYAD 01 program broadly and since we've in the process of treating three patients at the 300 million dose level in Cohort 3 of DEPLETHINK, it provides us an opportunity to evaluate OptimAB at that dose level and allows us for a reasonable comparison between the patients with the MAB or antibody manufacturing process compared to the OptimAB manufacturing process both from a safety and a translational readout perspective. So I think that's really the first element.
I would say the second one is based on, we are, from the OptimAB manufacturing process producing in rich memory like phenotype cells and the profile for CYAD-01 cells produced with this process. At 300 million dose level it may offer us the opportunity to evaluate what maybe the recommended dose level and then thirdly I think as I touched upon before is the idea that starting at 300 allows us also to think about dose escalating up to the 1 billion dose level, if necessary.
If we stick with the 300 we could provide some additional patients and quickly go through that but I think first and foremost it's the ability of we'll have three patients from 300 from the MAB process and we'll get an obviously an opportunity to compare that to what a OptimAB manufacture process looks like translational for those three patients as well.

因此,我认为就OptimAB而言,显然我们在外国直接投资修订后,在CYAD-01计划中使用OptimAB制造产品后,于6月底提供了更新。所以我真的想说为什么我们转向DEPLETHINK试验的第3组的原因有几点。

我认为总的来说,我们正在广泛地研究CYAD 01计划,因为我们正在DEPLETHINK的第3组中处理3个剂量水平的3名患者,它为我们提供了在该剂量水平评估OptimAB的机会并允许从安全性和平移读数的角度来看,与OptimAB制造过程相比,我们可以对患者进行MAB或抗体制造过程的合理比较。所以我认为这是第一个元素。

我想说第二个是基于我们来自OptimAB制造过程,产生丰富的记忆,如表型细胞和用这个过程产生的CYAD-01细胞的特征。在3亿剂量水平下,它可能为我们提供评估推荐剂量水平的机会,然后我认为,之前我认为从300开始让我们也考虑剂量逐渐增加至10亿剂量的想法等级,如有必要。

如果我们坚持300,我们可以提供一些额外的患者,并快速通过,但我认为首先,我们将有三个患者来自MAB过程300的能力,我们将有一个明显的机会比较对于这三位患者来说,OptimAB制造过程看起来像是转化的。

身份不明的分析师

I guess, then moving forward hopefully you'll be looking for, I think you had mentioned about looking for things for moving on to dose escalation or Cohort 4 maybe dependent on some readout from presumably Cohort 3 you are looking at, what would you be looking for data readout there?

我想,然后继续前进希望你会寻找,我想你已经提到要寻找继续进行剂量升级的事情,或者队列4可能依赖于你想要的队列3的一些读数,你会是什么样的 在那里寻找数据读数?

Filippo Petti

Filippo Petti

Yes, I think well its going to be -- its we will look to and have three patients before making a decision. What are some of the initial clinical act -- perhaps clinical activity obviously we're making sure that safety is in check as well some of the translational work and that's why we really did go one of the reasons why we decided to go with the 300 is that we'll get a better assessment between the two processes.
I think, what does that look like compared to the mAb process and then thinking about, do we need to dose escalate upper [indiscernible] with continuing enrollment. Really, the goal of the DEPLETHINK trial is for us to find the recommended dose. And we felt that it was necessary to evaluate OptimAb at 300 before making the decision to just quickly start at the 1 billion dose level.

是的,我认为它将成为现实 - 我们将在做出决定之前寻找并拥有三名患者。 什么是最初的临床行为 - 也许是临床活动显然我们确保安全性以及一些转化工作,这就是为什么我们真的去了我们决定采用300的原因之一 是我们将在两个过程之间得到更好的评估。

我认为,与单克隆抗体过程相比,看起来是什么样的,然后再考虑,我们是否需要通过持续注册来增加上层[音频不清晰]。 实际上,DEPLETHINK试验的目标是让我们找到推荐的剂量。 我们认为有必要在决定快速启动10亿剂量水平之前评估OptimAb为300。

身份不明的分析师

I guess last question from us. With the cash runaway being mid-2020 and everything going on, it seems like you may possibly get meaningful data beyond 2020; so is it going to be that you guys will keep all the trials running and then possibly - or how are you thinking about focusing on certain trials over others around the timing around your cash runway?

我想我们的最后一个问题。 随着2020年中期的现金失控以及一切正在进行,看起来您可能会在2020年之后获得有意义的数据; 所以,你们将保持所有的试验运行,然后可能 - 或者你是如何考虑围绕现金跑道的时间关注某些试验?

Filippo Petti

Filippo Petti

It's a good question. I think the way we think about the program right now, strategically, we have the funding to go through what we have in plan for 01, and obviously some of preclinical activities for the 02 process, bringing on some additional resources. It's certainly top of mind for us, as we talked about earlier and with one of the other questions, looking at all avenues.
And I think one of the things we can pursue there is non-dilutive financing that we can help to extend out the cash runway in a partnership or perhaps other means and instruments that we can think about.
Overall, we have a kind of excitement across both what's in the clinic now, what will soon be in the clinic, as well as what we plan to have in the clinic. And as you know, as a biotech company, we need to continue to think about where we can source funds to continue to drive the investigation of our programs and our pipeline.

这是一个很好的问题。 我认为,就目前我们对该计划的思考方式而言,战略上,我们有资金来完成我们在01计划中所拥有的资金,显然还有一些针对02流程的临床前活动,带来了一些额外的资源。 对于我们来说,这当然是我们的首要考虑因素,正如我们之前所讨论的那样,以及其他一个问题,并着眼于所有途径。

我认为我们可以追求的其中一件事是非稀释性融资,我们可以帮助扩大合作伙伴关系中的现金跑道,或者我们可以考虑的其他手段和工具。

总的来说,我们对现在的诊所,即将到来的诊所,以及我们计划在诊所拥有的东西都有一种兴奋。 如您所知,作为一家生物技术公司,我们需要继续思考我们可以在哪里寻找资金来继续推动对我们的计划和管道的调查。

会议主持员

Thank you. [Operator Instructions] There are no further questions at this time. Please continue.

谢谢。 [操作员说明]目前没有其他问题。 请继续。

Filippo Petti

Filippo Petti

Great, thank you, operator. So that will bring our call to a close. To briefly recap, we are very excited with the progress made in the first half of 2019, which as you've heard, include some significant advances in our clinical development programs, particularly around our lead candidates CYAD-01, as well as our non-gene edited allogeneic clinical candidate CYAD-101 across a number of different trials.
Thank you everyone for joining the call, and we look forward to speaking with you all again soon.

太棒了,谢谢你,运营商。 这将使我们的呼吁结束。 简要回顾一下,我们对2019年上半年所取得的进展感到非常兴奋,正如您所听到的那样,我们的临床开发计划取得了一些重大进展,尤其是我们的主要候选人CYAD-01,以及我们的非 -gene在多个不同的试验中编辑了同种异体临床候选人CYAD-101。

感谢大家加入我们的电话,我们期待很快再次与您交谈。

会议主持员

Ladies and gentlemen, this does conclude your conference for today. Thank you very much for participating. You may now all disconnect.

女士们,先生们,这确实结束了今天的会议。 非常感谢您的参与。 你现在可能都断开了。

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