Amarin Corporation plc。 (AMRN) 首席执行官 John Thero 在 2019年BMO 处方成功医疗保健会议上发表 (成绩单)

[机器翻译] 电话会议 · 2019年06月26日 · 41 次阅读

Amarin Corporation plc. (NASDAQ:AMRN) 2019 BMO Prescription For Success Healthcare Conference June 25, 2019 11:20 AM ET

Amarin Corporation plc。 (纳斯达克股票代码:[AMRN])2019年BMO处方成功医疗会议2019年6月25日美国东部时间上午11:20

公司参与者

John Thero - President and CEO

  • John Thero - 总裁兼首席执行官

电话会议参与者

Hello. President and CEO of Amarin, who'll give a presentation for about 15-20 minutes, I think, and then we'll follow-up with some Q&A. John has been at Amarin for almost 10 years, and CEO for the past five years, and I guess the old adage, patience is a virtue, couldn't be more fitting, considering the great results he got in the fall from the long-awaited REDUCE-IT outcomes study for your key product Vascepa, so congrats on that. We look forward to hearing about all that, and thank you so much for being here. So, hand it over to you.

  • 你好。 Amarin的总裁兼首席执行官,我想,他会做出约15-20分钟的演讲,然后我们会跟进一些问答。 约翰已经在Amarin工作了将近10年,在过去的五年中担任首席执行官,我猜这句古老的谚语,耐心是一种美德,不可能更合适,考虑到他在秋季从长期获得的巨大成果 期待您的关键产品Vascepa的REDUCE-IT成果研究,所以恭喜。 我们期待听到所有这些,并非常感谢您的光临。 所以,把它交给你。

John Thero

Thanks, Gary [ph], thanks for the welcome. As an adage I tend to like the one over here, if you follow the data it leads to good things, and particularly when you're working with excellent people, but I am here to talk about Amarin. I will be making forward-looking statements in conjunction with this presentation. Anybody considering investing in the company should review all risk factors and described in our SEC filings before investing. For those of you who have followed us for a while, we'll start with one comment which will be really the only update that I'll be providing here, which is the response to the question that I hear first from most investors today, the question of have we received any word from FDA with respect to a AdCom, and the answer to that is, no. It's been silent at this point in time. They've not told us whether they are considering one, whether they're not considering one, or maybe.
As a reminder, the sNDA that we filed with them was filed on late March, PDUFA date is September 28th, mid-cycle review is the 28th of this month, and while it is not without precedent for companies to be notified by FDA of an AdCom yea or nay before this, there is plenty of examples of companies being notified beyond this point in the review cycle, so Amarin continues to prepare for the potential AdCom. Some people would say, well, why would there be an AdCom. I would say because we're talking about the first therapy in a patient population that's measured in the millions -- potentially tens of millions of patient, and because there are some theses out there at, at least I could hypothetically come up with that the FDA might want to look at like such as emphasizing that the REDUCE-IT result is not a validation of triglyceride lowering, but rather what we studied is a patient population we elevated triglycerides and showed the multifactorial affect of Vascepa lowered cardiovascular risk, which we would agree with.
Or they might want to take on the fact that dietary supplement Omega-3's have repeatedly not worked, and want to emphasize that. The flipside of course is that Vascepa has already been in the market, been prescribed over five million times, and has had results of the study that's under review by the FDA published now into leading journals; the New England Journal of Medicine, and JAC. So we'll find out, in any case we continue to believe that we're in the range where it would be quite reasonable for the FDA to still declare an AdCom, so we are preparing, but the one update here is that we have not heard. The rest of what I will be commenting on in my prepared remarks, for those who've been following us, will not be new. I do look forward to Q&A afterwards.
As an overview, Amarin is working towards addressing a very large unmet medical need in cardiovascular risk. Cardiovascular disease, as you probably know, is an enormous and growing health burden. Current therapy, so the standard of care, whether that be for cholesterol management, blood pressure, diabetes, all are wonderful, but significant cardiovascular risk remains. We have been pioneering, as Gary has talked about, for 10 years a solution to this. Many companies have failed along the way. We, through a robust outcomes study, have shown that our drug, Vascepa, provides a very significant cardiovascular risk reduction beyond existing therapies.

谢谢,加里[ph],谢谢你们的欢迎。作为一句格言,我倾向于喜欢这里的那个,如果你按照数据导致好事,特别是当你和优秀的人一起工作时,我会在这里谈论Amarin。我将结合本演示文稿制作前瞻性陈述。任何考虑投资该公司的人都应该审查所有风险因素,并在投资前在我们的SEC文件中进行描述。对于那些关注我们一段时间的人,我们将从一条评论开始,这将是我将在这里提供的唯一更新,这是我今天大多数投资者首先听到的问题的回复,我们是否收到了FDA关于AdCom的任何消息的问题,答案是否定的。在这个时候,它一直保持沉默。他们没有告诉我们他们是否正在考虑一个,他们是不是在考虑一个,或者可能。

提醒一下,我们向他们提交的sNDA是在3月下旬提交的,PDUFA日期是9月28日,周期中期审查是本月28日,虽然公司被FDA通知并非先例AdCom是的,或者在此之前,有很多公司在审核周期之后被通知超过这一点,因此Amarin继续为潜在的AdCom做准备。有些人会说,为什么会有一个AdCom。我想说的是因为我们谈论的是患者人群中的第一次治疗,数百万人 - 可能是数千万患者,并且因为有一些论文在那里,至少我可以假设得出美国食品和药物管理局可能希望看到像强调REDUCE-IT结果不是甘油三酯降低的验证,而是我们研究的是患者群体,我们提高甘油三酯并显示Vascepa的多因素影响降低心血管风险,我们会同意。

或者他们可能想要承担膳食补充剂Omega-3一再无效的事实,并希望强调这一点。当然,另一方面是Vascepa已经进入市场,已经处方超过500万次,并且已经将FDA正在审查的研究结果发表在主要期刊上;新英格兰医学杂志和JAC。所以我们会发现,无论如何我们仍然认为我们处于FDA仍然宣布AdCom是合理的范围内,所以我们正在准备,但这里的一个更新是我们有没听见对于那些一直关注我们的人,我将在准备好的评论中评论的其余内容将不会是新的。我确实期待之后的问答。

作为概述,Amarin正在努力解决心血管风险中非常大的未满足的医疗需求。您可能知道,心血管疾病是一种巨大且不断增长的健康负担。目前的治疗,所以护理的标准,无论是胆固醇管理,血压,糖尿病,都是美妙的,但仍然存在显着的心血管风险。正如加里所说的那样,我们已经开拓了10年的解决方案。许多公司在此过程中都失败了。通过强有力的结果研究,我们已经证明,我们的药物Vascepa在现有疗法之外提供了非常显着的心血管风险降低。

As these results have been published we have submitted to the FDA for approval of this expanded indication to build upon the indication that we have in the marketplace place, which is an important but niche indication for treating patients with triglycerides of 500 mgs per deciliter or greater which is more of a pancreatitis rather than a cardiovascular indication. We believe that the opportunity to be first in the market to be -- is an opportunity to address millions if not tens of millions of patients, and is an opportunity to be measured in the billions.
In terms of recent highlights, I've a little bit about the sNDA already; this is under priority review with the FDA. Again, the PDUFA date is September 28th. We, after the results of REDUCE-IT were known we expanded our commercial presence a bit. We will be expanding our commercial presence further upon the expanded label continuing to review what that expansion should be, but envisioning it being in multiple steps of starting with and increasing our sales force in the United States to between 600 and 800 reps. So now we're currently at 400, so that would be up by another 50% or potentially a doubling, and then we would be promptly submitting to OPDP to allow for consumer promotion which in their normal course typically takes about five to six months, but we would be envisioning a substantial DTC commending on a branded basis in the second quarter of next year.
We, based upon the positive results of REDUCE-IT, we have seen significant prescription increases. Greater than 50% of our prescriptions currently are all off-label. Fortunately our managed care coverage is already good. I think it will get better upon label approval, but the approval rate of our prescriptions is at right around 80%. We look forward to continuing to see prescription growth here based upon the label, and then hopefully further upon label expansion in the DTC, so it's multiple points of inflection there, and very pleasing to me is that here, in approximately seven months, since the time when we had these results presented we've already seen a market shift from early months of people trying to understand, and I'm talking about KOLs here because most docs don't yet know Vascepa.
We're looking forward to that broader education, but docs who have studied the REDUCE-IT results early on tried to understand the multifactorial effect. I think they predominantly get it at this point in time, and that the conversations at society meetings has so shifted to not to does it work but rather how do we best implemented this in our practices as therapy sort of the next treatment beyond statins, and I am very happy to be American Diabetes Association here even before the label has been expanded has provided a type A recommendation which is the high evidence recommendation for the use of the active ingredient. Our drug recommends specific drug, but they recommend the use of the active ingredient of the drug for patients who are statin treated and continue to have triglyceride levels of 135 mg/dl or greater. So that transition here even before the label approval to adoption of Vascepa is happening.

由于这些结果已经公布,我们已经向FDA提交了批准这一扩展适应症的建议,以建立我们在市场上的指示,这是治疗甘油三酯500 mg / dl或更高的患者的一个重要但适当的适应症。这更多的是胰腺炎而不是心血管疾病。我们相信,成为市场第一的机会 - 是一个机会,可以解决数百万甚至数千万患者,并且是一个以数十亿计量的机会。

就最近的亮点而言,我已经对sNDA有所了解;这是与FDA的优先审查。同样,PDUFA日期是9月28日。在我们知道REDUCE-IT的结果后,我们扩大了我们的商业存在。我们将继续扩大我们的商业存在,扩大后的品牌继续审查扩张应该是什么,但设想它在多个步骤中开始并将我们在美国的销售人员增加到600到800个销售代表。所以现在我们目前处于400,所以这将增加50%或可能增加一倍,然后我们将立即提交OPDP以允许消费者促销,这在正常过程中通常需要大约五到六个月,但我们将设想在明年第二季度以品牌为基础的大量DTC表彰。

我们基于REDUCE-IT的积极成果,我们看到了显着的处方增加。目前超过50%的处方都是标签外的。幸运的是,我们的管理式医疗服务已经很好。我认为在标签批准后它会变得更好,但我们处方的批准率大约在80%左右。我们期待继续看到基于标签的处方增长,然后希望进一步在DTC的标签扩展,所以这是多个拐点,我很高兴在这里,大约七个月,自从当我们得到这些结果的时候,我们已经看到了市场从早期人们试图理解的转变,我在这里谈论KOL,因为大多数文档还不知道Vascepa。

我们期待更广泛的教育,但是早期研究REDUCE-IT结果的文档试图理解多因素效应。我认为他们现在主要得到它,并且社会会议上的谈话已经转变为不会起作用,而是我们如何在我们的实践中最好地实施这种治疗,作为他汀类药物以外的下一种治疗方法,以及我很高兴在这里成为美国糖尿病协会,甚至在标签扩大之前提供了A型推荐,这是使用活性成分的高证据推荐。我们的药物推荐使用特定药物,但他们建议将这种药物的有效成分用于接受他汀类药物治疗的患者,并继续使用135 mg / dl或更高的甘油三酯水平。因此,即使在标签批准采用Vascepa之前,这种转变正在发生。

Background, of course, cardiovascular risk is huge. I think people who have elevated LDL or bad cholesterol should get it managed. If you lower your cholesterol, you are lowering your cardiovascular risk by about 25% to 35%. That's great. Of course, that still leaves 65% to 75% of the risk remaining. Someone dies in the United States every 38 seconds from cardiovascular disease. It's number one cause of death more than all cancers combined.
Enormous cost here, and there is no proven therapy. We will be the first assuming that we get approved for this indication, so a huge need and huge opportunity. Identification of risk, LDL and triglycerides have been shown in various studies to be somewhat equal identifiers of risk. Lowering of LDL, bad cholesterol is shown to be directly co-related to lowering risk of -- lowering of triglycerides alone has not had as direct a correlation.
And as we get further into this presentation, you will see that we are using triglycerides as an identifier of risk, but with Vascepa addressing some of the underlying factors. You can see from this chart that risk begins to increase with triglyceride levels well below a 100 mgs/dl and then continues to go up. So, the most definitive way of evaluating the effectiveness of a therapy is through an outcome study.
And we conducted a very robust outcome study called the REDUCE-IT study which results were presented at the American Heart Association in November of last year and published in New England Journal of Medicine for Primary Results in Cardiology Watch called the number one story for 2018 in the cardiovascular area. This is a study where we took patients who were all treated with the current standard of care. So they were on statins, had their LDLs well controlled, baseline of 100 -- excuse me, 75 mg/dl. They were all below 100, and if they were diabetic, they were on diabetic meds. If they were hypertensive, they were on blood pressure meds et cetera. We took those patients, and if they had elevated triglycerides of 135 mg/dl or greater and other cardiovascular risk factors, we enrolled them in a study.
We randomized them on a one for one basis between staying on standard of care therapy plus Vascepa, or staying on standard of care therapy plus placebo, and we evaluated the results over what turned out to be an average of 4.9 years. Saw over 35,000 patients in years of study that was conducted under a special protocol assessment agreement with the FDA, and the results were terrific. So, the primary results were on the first occurrence of events. The primary endpoint was a composite 5-point MACE. So 5-point MACE consisting of heart attack, stroke, cardiovascular death, revascularization things like bypass and stents, or hospitalization for angina, and they have exceeded 25% relative risk reduction versus placebo and a lots of zeros in the P value. For those who like Hard MACE which is stroke, heart attack, and death, it jumps up to 26%, and if go to death which you don't see for most studies. In fact, you don't see in most of the statins study didn't the statistical significance on a SCV death reduction by 20%, statistically significant death, heart attack, down 31%; stroke down 28%.

当然,背景,心血管风险是巨大的。我认为LDL升高或胆固醇水平升高的人应该对其进行管理。如果你降低胆固醇,你的心血管风险降低了约25%至35%。那很棒。当然,这仍然留下65%至75%的风险。有人因心血管疾病每38秒就在美国死亡。它是死亡的头号原因,超过了所有癌症的总和。

这里的成本非常高,而且没有经过证实的治疗方法。我们将首先假设我们获得了此指示的批准,因此需要巨大的机会。各种研究表明,风险,低密度脂蛋白和甘油三酯的鉴定在某种程度上是相同的风险标识。降低低密度脂蛋白,坏胆固醇被证明与降低风险直接相关 - 单独降低甘油三酯没有直接相关性。

随着我们进一步深入讨论,您将看到我们使用甘油三酸酯作为风险的标识符,但Vascepa解决了一些潜在因素。从这张图表中可以看出,随着甘油三酯水平远低于100 mgs / dl,风险开始增加,然后继续上升。因此,评估治疗有效性的最明确方法是通过结果研究。

我们进行了一项非常有力的结果研究,称为REDUCE-IT研究,该研究结果于去年11月在美国心脏协会上发表,并发表在新英格兰医学杂志的心脏病学初级结果观察中称为2018年的第一个故事。心血管区。这是一项研究,我们采取了所有接受现有护理标准治疗的患者。所以他们服用他汀类药物,他们的LDL控制良好,基线为100 - 对不起,75 mg / dl。他们都低于100,如果他们是糖尿病患者,他们就是糖尿病患者。如果他们是高血压患者,他们就是血压药等。我们服用这些患者,如果他们的甘油三酯升高135 mg / dl或更高以及其他心血管危险因素,我们将他们纳入研究。

我们在保持标准治疗和Vascepa之间一对一地随机分组,或者继续使用标准治疗加上安慰剂,我们评估了结果,结果平均为4。9年。根据与FDA达成的特殊方案评估协议,在多年的研究中看到超过35,000名患者,结果非常好。因此,主要结果是第一次发生事件。主要终点是复合5点MACE。因此5点MACE包括心脏病发作,中风,心血管死亡,血运重建等事件,如旁路和支架,或住院治疗心绞痛,相对于安慰剂和P值中的大量零点,它们的相对风险降低超过25%。对于那些喜欢中风,心脏病和死亡的硬性MACE的人来说,它会上升到26%,如果你去大多数研究都没有看到死亡。事实上,你没有看到在大多数他汀类药物研究中没有统计学意义,SCV死亡率降低20%,统计学显着死亡,心脏病发作,下降31%;中风下降28%。

The principal investigator for the study was Dr. Deepak Bhatt, out of Harvard. He has discussed these results as being the most significant breakthrough in preventive cardiovascular care since statin, so 25 years ago. For those who like this graphically see the divide occurred pretty early throughout the one year point and what you like to see is that consistent separation there afterwards number needed to treat 21, but the result continued. So if people had heart attack or stroke or other event and survived those might add another event. So there are second events 32% reduction, third event 31%, 48% beyond that on a relative basis for an overall reduction of 30% but this translates into one fewer major adverse cardiovascular event for every six patients treated over a five year period and for those of you who like it graphically see again divide occurred early and was consistent.
The results were consistent male, female, diabetic, non-diabetic, secondary and primary prevention. Just to put the number needed to treat into perspective and we're not competing with cholesterol management drugs but our number needed to treat at 21. If you look at Lipitor or Atorvastatin you know, there's 45. You look at Repatha PCSK9 there is 67 again, no head to head studies here, but this is an important result and again that those numbers are all against first occurrence of events we go for first and recurrent events down to one in six, not seen that anywhere else.
An interesting finding of the result and again sort of speaks to the fact that Vascepa is addressing underlying factors to triglycerides is that while all the patients in the study had elevated triglycerides, the reduction was relatively consistent across triglyceride levels whether the triglycerides were 135 to 150 or 150 and above, we had seen the Japanese study the same active ingredient that you had at 19% relative risk reduction slightly lower risk patient population with only a 6% change in triglycerides. We had a pretty good idea coming into the study that this was about more than just triglyceride reduction and for those who want to go on our Web site, we have a couple of dozen publications on mechanism of action that deal with effects on inflammation to endothelial cell function to plaque formation, plaque regression, oxidation, anti-platelet et cetera sort of multifactorial effect.
The safety profile of Vascepa is pretty well-established that the drug has been prescribed over five million times and in the REDUCE-IT study with 35,000 patient years of study. The overall event rate between the Vascepa arm of the study and the placebo arm of the study was similar. There were no events of serious adverse, no serious adverse events greater than 2% greater than placebo. Keep digging you have to show something in the New England Journal of Medicine had us keep digging and we did report a few events that they considered to be low but to sort of round out the picture but overall a safety profile that's placebo like.

该研究的主要研究者是哈佛大学的Deepak Bhatt博士。他将这些结果作为25年前自他汀类药物以来预防性心血管护理的最重大突破进行了讨论。对于那些以图形方式喜欢这种情况的人来说,看到分歧在整个一年的时间点发生得非常早,而你希望看到的是,之后需要一致的分离,需要治疗21,但结果仍然存在。因此,如果人们心脏病发作或中风或其他事件并幸存下来,那么可能会增加另一个事件。因此,第二次事件减少32%,第三次事件减少31%,相对基础减少48%,总体减少30%,但这相当于每五名患者在五年内治疗的主要不良心血管事件减少一次,对于那些喜欢它的人,以图形方式再次看到分裂发生在早期并且是一致的。

结果是男性,女性,糖尿病,非糖尿病,二级和一级预防的一致性。只是把治疗所需的数量放在一边,我们不与胆固醇管理药物竞争,但是我们的数量需要在21治疗。如果你看看立普妥或阿托伐他汀,你知道,有45个。你看看Repatha PCSK9有67个再次,这里没有头对头的研究,但这是一个重要的结果,而且这些数字都反对我们首先发生的事件的第一次发生和反复发生的事件下降到六分之一,其他任何地方都看不到。

结果的一个有趣发现再次说明Vascepa正在解决甘油三酯的潜在因素这一事实是,虽然研究中的所有患者都有甘油三酯升高,但甘油三酯水平的降低相对一致,甘油三酯是否为135至150或者150以上,我们看到日本人研究的相同活性成分相同,相对风险降低19%,风险患者人数略低,甘油三酯仅有6%的变化。我们有一个很好的想法进入研究,这不仅仅是甘油三酯减少,对于那些想要访问我们网站的人,我们有几十篇关于内皮炎症影响机制的出版物。细胞功能以斑块形成,斑块消退,氧化,抗血小板等多种因子作用。

Vascepa的安全性非常明确,该药物已被处方超过500万次,并且在REDUCE-IT研究中有35,000患者年的研究。该研究的Vascepa组与该研究的安慰剂组之间的总体事件发生率相似。没有严重不良事件,没有严重不良事件比安慰剂大2%以上。继续挖掘你必须在“新英格兰医学杂志”中展示一些东西,让我们继续挖掘,我们确实报告了一些他们认为很低的事件,但有点整理了图片,但整体上是安慰剂的安全概况。

Just to put into perspective, the accomplishment that we're showing here again statins, lower cardiovascular risk by about 25% to 35% before going generic that was an over $20 billion market in the U.S. alone over $30 billion. Globally many people have tried to show cardiovascular risk reduction beyond statins CETP inhibitors particularly niacin firebreaks all which have failed in studies saying CDL lowering drugs that we don't compete with them but you know, is that a PCSK9 that sixth and 15%, the Qantas study an interesting study on inflammation drug probably would never really be used for this purpose to its cost and safety profile but 15% Omega-3 mixtures whether that be Lovaza has a prescription therapy which completed two outcome studies last year failed them both or the dietary supplements have been shown not to work probably in part due to the LDL increasing effect of DHA which is included in those products, but the science goes beyond that.
The only success is sort of beyond the LDL lowering here has been with pure EPA both in the Japanese study and then very significantly in this robust study that I'm talking about reduced it with 25% risk reduction. So we think we're going to be able to help millions of patients with these results and the active ingredient is unique, it isn't ripe in nature but it's been declared a new chemical entity by FDA. It is a fragile molecule. Isolating it and keeping it stable is not a trivial exercise. We've put a lot of science into being able to do that. Its smaller size allows it get into the endothelial cell in a way that other Omega-3s and other agents haven't been shown to be able to do, which then improves endothelial cell function, endothelial cell signaling, which seems to have a downstream effect of lower inflammation and plaque formation, et cetera.
Conversely, something like DHA, which is a little bit longer, has a more difficult time getting into the endothelial cells and tends to be disruptive, doesn't have the reduction in inflation, doesn't have the -- in fact, ends up resulting in LDL going up, and I'll save time for Q&A here by not going into mechanism of action in more detail, but somebody who is interested in the multifactorial affect please look at our Web site, we have dozens of publications on these. This slide really lists the eight key steps in the formation of Atherosclerosis and EPA are their active ingredient. Eicosapentaenoic acid is the only molecule that's been shown to have a positive affect on all of these eight key factors.
Our primary focus today is the U.S. market. We have increased our sales presence modestly to start, this year. As I described earlier, we intend to increase it further, both in terms of the reach that we will have to docs, but also the frequency with which we're calling on physicians then to be complimented by DTC. The feedback we're hearing from physicians is very positive. Managed care coverage is already very good, and we look forward that to that expanding further upon our label approval expansion. We have been building our supply chain and staying out ahead of this, we came into this year with the guidance on revenues in which guidance was based upon assumed standard review of 10 months, rather than a priority review of revenues of $350 million, we came in with capacity to support about a billion dollars in revenue. So, from a supply perspective we're looking forward to expanding that and working with our suppliers to do so.

只是为了说明我们在这里再次显示他汀类药物的成就,在通用之前将心血管风险降低了约25%至35%,仅在美国就超过300亿美元,超过300亿美元。在全球范围内,许多人试图显示心血管风险降低超过他汀类药物CETP抑制剂,特别是烟酸防火剂,所有这些都没有在研究中说CDL降低药物,我们不与他们竞争,但你知道,是PCSK9,第六和15%, Qantas研究一项关于炎症药物的有趣研究可能永远不会真正用于此目的的成本和安全性,但15%的Omega-3混合物,无论是Lovaza是否有处方疗法,去年完成了两项结果研究,他们两者或膳食补充剂已被证明不起作用可能部分是由于LDL增加了DHA的作用,这些作用包括在那些产品中,但科学超越了这一点。

唯一的成功是超越LDL降低,这在日本的研究中一直是纯EPA,然后在这个强有力的研究中非常显着,我正在谈论减少风险降低25%。因此,我们认为我们将能够帮助数百万患者获得这些结果,并且活性成分是独特的,它本质上不成熟,但它已被FDA宣布为新的化学实体。它是一种脆弱的分子。隔离并保持稳定并非易事。我们已经投入了大量的科学才能做到这一点。它的较小尺寸允许它以其他Omega-3和其他药物未能显示的方式进入内皮细胞,从而改善内皮细胞功能,内皮细胞信号传导,这似乎具有下游效应较低的炎症和斑块形成,等等。

相反,像DHA这样的东西,有点长,进入内皮细胞更困难,往往是破坏性的,没有减少通货膨胀,没有 - 事实上,最终导致LDL上升,我将通过更详细地讨论行动机制来节省问答时间,但是对于多因素影响感兴趣的人请查看我们的网站,我们有很多关于这些的出版物。这张幻灯片确实列出了动脉粥样硬化形成的八个关键步骤,EPA是其活性成分。二十碳五烯酸是唯一被证明对所有这八个关键因素产生积极影响的分子。

我们今天的主要焦点是美国市场。今年我们开始适度增加销售业绩。正如我之前所描述的那样,我们打算进一步增加它,无论是在我们对文档的覆盖范围方面,还是我们呼吁医生随后被DTC称赞的频率。我们从医生那里听到的反馈非常积极。管理式医疗保险已经非常好,我们期待在我们的标签批准扩展后进一步扩大。我们一直在建立我们的供应链并且在此之前保持领先,我们今年进入了收入指导,其中指导基于10个月的假设标准审查,而不是对3.5亿美元的收入进行优先审查,我们来到能够支持大约10亿美元的收入。因此,从供应角度来看,我们期待扩大这一范围并与供应商合作。

We've got now over 200 patents globally most with expiries in 2030, and internationally, we have a partner in Canada that has priority review there. We have some approvals in the Middle East that started, and other applications there, a trial going on with a partner in China where we're positioned to be first to market, and we're considering various pathways in Europe with an aim towards having a submission to EMA sometime around the end of this year, and just a sort of a near closing caption, so if we think about cholesterol management 25 years ago you're moving from resins and other therapies which weren't particularly convenient and weren't entirely proven without outcomes data to statin therapy. We think that the opportunity is similar here for risk management beyond LDL, and we're moving away from therapies that have been used for millions of patients but that haven't been proven successful, to now finally having a drug where there is a successful preventative result, and we've priced the drug affordably, like statins are with the hope that this can be a mass use to help millions of patients. Financially, we ended last quarter with about $211 million in cash. We do have a little bit of a royalty remaining, but no traditional debt, and we've got some loss carry forwards that are substantial, about $800 million, that I look forward to working through.
And with that I conclude my prepared comments, and I'll sit down and see what questions I might --

我们现在在全球拥有200多项专利,其中2030年到期,而在国际上,我们在加拿大有一个合作伙伴,在那里有优先审查。我们在中东开始获得一些批准,其他应用程序在中国与我们的合作伙伴在中国进行试验,我们将首先推向市场,我们正在考虑在欧洲开展各种途径,目标是拥有在今年年底的某个时候提交给EMA,并且只是一个接近关闭的标题,所以如果我们考虑25年前的胆固醇管理,你将从树脂和其他不太特别方便的疗法转向完全证实没有结果数据他汀类药物治疗。我们认为这里的机会类似于LDL以外的风险管理,我们正在摆脱已经用于数百万患者的疗法,但尚未被证明是成功的,现在终于有了成功的药物预防性结果,我们已经为这种药物的价格定价,就像他汀类药物一样,希望这可以成为大量使用,帮助数百万患者。从财务角度来看,我们上个季度以约2.11亿美元现金结束。我们确实还有一些特许权使用费,但没有传统的债务,而且我们有一些可观的亏损结转,大约8亿美元,我期待着继续努力。

然后我结束了我准备好的评论,我会坐下来看看我可能会有什么问题 -

身份不明的分析师

Yes, excellent. Thanks for that overview. John, that was great.

是的,非常好。 感谢您的概述。 约翰,那很棒。

问答环节

So my first question is do you have a sense from FDA if you're going to have an advisory panel yet or not?

所以我的第一个问题是,如果你想要一个咨询小组,你是否有FDA的意识?

John Thero

Zero words so far.

到目前为止零字。

身份不明的分析师

I'm joking. You only said that for about five minutes.

我在开玩笑。 你只说了大约五分钟。

John Thero

I spoke for a while. I thought maybe that was last of the music.

我说了一会儿。 我想也许那是最后的音乐。

身份不明的分析师

I was awake in the beginning. No, the real question is --

我一开始很清醒。 不,真正的问题是 -

John Thero

I will by the way, just for the audience, comment that because we get this question almost daily. If we find out something definitive on that topic from the FDA we will let -- we will publicly state it, but until we have something definitive, you know.

顺便说一下,我会为观众发表评论,因为我们几乎每天都会收到这个问题。 如果我们从FDA那里找到关于该主题的确定性内容,我们会公开说明 - 但是在我们确定了一些确定的内容之前,你知道。

身份不明的分析师

Okay. The question is more around what is the likelihood that you do get the expanded indication? And like just looking at other analogues for other cardiovascular drugs or even drugs in other categories where they have outcomes studies, the kind of data that you have, is there precedent that makes you pretty confident that it's just a question of when and not if?

好的。 问题在于您获得扩展指示的可能性有多大? 就像只看其他心血管药物的其他类似物,甚至是他们有结果研究的其他类别的药物,你拥有的那种数据,是否有先例让你相信它只是一个什么时候的问题?

John Thero

So this trial was conducted under a special protocol assessment agreement with the FDA. They -- we along the way because we had a long trial reaffirm that SPA with them, and the results have now been presented in multiple scientific congresses, and published in two leading journals. We were taken over the coals with the reviewers at those various journals, I think we're in very good stead, but there's always the unknown, but we've got a high degree of confidence in the likely approval of this sNDA.

因此,该试验是在与FDA的特殊方案评估协议下进行的。 他们 - 我们一路走来,因为我们进行了长时间的试验,重申SPA与他们一起,结果现已在多个科学大会上发表,并在两个主要期刊上发表。 我们在各种期刊上与审稿人一起接管了煤炭,我认为我们处于非常好的状态,但总有一些未知,但我们对这个sNDA的可能获得批准有很高的信心。

身份不明的分析师

Okay. And you highlighted the prescription trends have accelerated very nicely, so it's being used off label. Physicians obviously are grabbing on to the data and a lot more comfortable with the benefits of Vascepa, but so even if you don't get the expanded label how much does that really matter? Obviously it helped, and then you could promote it more aggressively, but within the physician community it seems like it's out there.

好的。 并且你强调处方趋势已经非常好地加速,所以它被用于标签之外。 医生显然正在抓住数据,并且对Vascepa的好处更加舒服,但即使你没有得到扩展标签,这真的有多重要? 显然它有所帮助,然后你可以更积极地推广它,但在医生社区内,它似乎就在那里。

John Thero

Physicians are beginning to learn about it, and as they are learning about it beginning to use it more. There's about 660,000 docs who prescribe statins, we're calling on a little over 50,000 of them. We look forward to being able to expand our promotion on call on more of them. The hypothetical of our not getting approved and what that would mean, I really can't go down that rabbit hole because I cant think of the reason why we wouldn't be approved, and not knowing what that reason is I'd have a hard time saying what the impact would be where -- we are planning for success, and we think that we're well positioned for that.

医生们开始了解它,并且正在学习它开始更多地使用它。 大约有660,000名医生开了他汀类药物,我们要求他们服用超过50,000名。 我们期待能够在更多的电话会议上扩大我们的促销活动。 假设我们没有得到批准,这意味着什么,我真的不能说那个兔子洞,因为我无法想到我们不会被批准的原因,也不知道我的原因是什么 很难说影响会在哪里 - 我们正在计划成功,我们认为我们已经做好了准备。

身份不明的分析师

Okay. And then you touched briefly on the patents that you have on the product, but maybe just spend another minute or so, just how comfortable you are? I forget if there are ANDA filers already on a product and you're in the middle of litigation. So just give us an update there, and is there actually any potential lifecycle extension beyond the REDUCE-IT data, which I'm sure you could try and file for patents around that I would imagine, but is there anything else in terms of the ingredient itself or things that you're working on to try and extend the lifecycle of the franchise even further?

好的。 然后你简要介绍了你对产品的专利,但也许只花了一分钟左右,你有多舒服? 我忘了如果产品上已经有ANDA申报者而你正处于诉讼中。 那么请在那里给我们一个更新,实际上除了REDUCE-IT数据之外还有任何潜在的生命周期扩展,我相信你可以尝试为我想象的专利申请,但是还有什么其他方面的 成分本身或你正在努力尝试延长特许经营生命周期的东西?

John Thero

So regarding patents, the majority of our patents go to 2030, and some potentially beyond. Regarding the tail-end of your question about lifecycle management, we've got plenty of time to think about that. In terms of the here and now, there are ANDA filers, ironically sort of insulting if there wouldn't be ANDA filers around a good drug. There were four; Apotex sort of removed themselves from the process relatively early. Teva settled. They could come into the market with a generic in the second-half of 2029, so a little over 10 years from now. There are two remaining ANDA filers, Dr. Reddy's and Hikma. Claims, construction, the Markman hearings are done. We did very well in those, more recently procedurally. The court has allowed us to introduce the results of the REDUCE-IT study, which we think supports the uniqueness of Vascepa.
There is a court data scheduled for opening hearings on January 13th, if it goes to court then we likely would have a judgment coming out of that two to three months later. We've asserted 14 of our Orange Book list of patents, each have multiple claims and we intend to defend those vigorously.

因此,对于专利,我们的大多数专利都是在2030年,有些可能超出专利。关于生命周期管理问题的尾声,我们有充足的时间来考虑这个问题。就现在和现在而言,有一些ANDA申报者,具有讽刺意味的是侮辱性的,如果没有一个好药物的ANDA申报者。有四个; Apotex相对较早地从这个过程中解脱出来。特瓦安顿下来。他们可以在2029年的下半年以通用的方式进入市场,所以从现在起10多年。还有两个ANDA申报者,Reddy博士和Hikma。索赔,建设,马克曼听证会已经完成。我们在那些方面做得很好,最近在程序上做得很好。法院允许我们介绍REDUCE-IT研究的结果,我们认为该研究支持Vascepa的独特性。

有一个法庭数据计划在1月13日举行听证会,如果它上法庭,那么我们可能会在两到三个月后作出判决。我们已经断言了我们的橙皮书专利清单中的14个,每个都有多个索赔,我们打算大力捍卫这些专利。

身份不明的分析师

Okay. By the way, if there are any questions in the audience just raise your hand, we'll get a mic over to you. Or we have an app. You could always put it through the app. Okay, I will keep going. So, you also briefly touched on the manufacturing and the capacity, and you're working towards a billion. So, how much of a natural barrier is that, like how difficult is the manufacturing process see of you to get from where you are to the $1 billion, and then potentially to get to the next level in order to really satisfy the market?

好的。 顺便说一句,如果观众有任何问题只需举手,我们就会给你一个麦克风。 或者我们有一个应用程序。 你可以随时通过应用程序。 好的,我会坚持下去。 所以,你还简要地谈到了制造业和产能,你正努力达到十亿。 那么,一个天然的障碍是多少,比如制造过程看到你从10亿美元到10亿美元的难度,然后为了真正满足市场需要更上一层楼?

John Thero

So learning -- and our manufacturing process is complex. So we've got a very fragile molecule and isolating it and keeping it stable, and we are very proud that we've got a proven four-year shelf life is important, right? So if somebody thinks about sort of normal fish oil, you open it and you then smell it, the odor you know, get a little bit of fishy taste, that's rotting basically, but it's oxidation and you tend to take a product from antioxidant or pro-oxidant which we don't think is necessarily helpful. Being able to isolate this unique molecule in a way that doesn't damage it and then being able to keep it stable over an extended period of time is tricky. You can think about a drug like Lovaza, which is easy to manufacture. They left in 16% of other stuff. Either they didn't know that that other stuff was not necessarily helpful or I think more likely it was just too difficult to remove.
Our view is this is a important drug and we shouldn't have 16% of other stuff in there. That volume in the body is important relative to our active ingredient. So, we want our drug to be all of this active ingredient. While many -- rather there is a limited number of companies in the world who can manufacture it, and some of those have failed as we have tried to work with them including some large industrial concerns. The companies that we have been working with are suppliers and now have been working with them multiple years have figured it out, and learning how to do it to begin with is tricky. Replicating it once you have figured it out seems to be relatively straightforward. So, we are using a model of having multiple suppliers competing with one another. We are pleased that they are each looking to find ways to continue to improve on the cost side of things although this is never going to be a cheap product to manufacture.
Our margins right now are around 76%, but also they are also trying to impress us with their ability to expand supply with the belief that if they are able to have more supply that we will commit to be purchasing greater percentages of them as we grow, and we are pleased to be working with such great suppliers.

所以学习 - 和我们的制造过程是复杂的。所以我们有一个非常脆弱的分子并隔离它并保持稳定,我们非常自豪我们已经证明了四年的保质期很重要,对吗?因此,如果有人想到正常的鱼油,你打开它,然后你闻到它,你知道的气味,得到一点鱼腥味,基本上腐烂,但它是氧化,你倾向于从抗氧化剂或我们认为不能帮助的促氧化剂。能够以不损害它的方式分离这种独特的分子,然后能够在长时间内保持稳定是很棘手的。您可以考虑像Lovaza这样易于制造的药物。他们留下了16%的其他东西。要么他们不知道其他东西不一定有用,或者我认为更难以删除。

我们认为这是一种重要的药物,我们不应该有16%的其他东西。相对于我们的活性成分,体内的体积是重要的。因此,我们希望我们的药物都是这种活性成分。虽然很多 - 而且世界上有限数量的公司可以制造它,其中一些公司已经失败了,因为我们试图与它们合作,包括一些大的工业问题。我们一直与之合作的公司是供应商,现在已与他们合作多年已经弄明白,并且学习如何开始这样做是非常棘手的。一旦你弄明白它就复制它似乎相对简单。因此,我们使用的模型是让多个供应商相互竞争。我们很高兴他们每个人都希望找到继续改进成本方面的方法,尽管这绝不是一个廉价的制造产品。

我们现在的利润率约为76%,但他们也试图通过扩大供应的能力给我们留下深刻印象,他们相信如果他们能够拥有更多的供应,我们将承诺随着我们的成长购买更多的供应量。 ,我们很高兴与这些优秀的供应商合作。

身份不明的分析师

Okay, great. On the commercial side, so you recently increased the sale force to 400 reps, and you are considering taking that up to 600 - 800. So how many -- so I think the target physician audience could be 50,000, right, or north of that, but how many of these incremental physicians are primary care? And how important is that segment for the overall use of the product going forward?

好,太棒了。 在商业方面,所以你最近将销售力量增加到400次,而你正在考虑将其提高到600 - 800.那么有多少 - 所以我认为目标医生的观众可能是50,000,正确或北部 ,但这些增量医生中有多少是初级保健? 这个细分市场对整个产品的使用有多重要?

John Thero

So, what we are doing right now is continue to do analysis. We are seeing that with the doctors that we are calling on, which is today about 50,000 or little over 50,000, about 80% of those are primary care. About 12% of those are cardiologist. About 7% of those are endocrinologist, and then there is a rounding in there for other disciplines. Those who tend to the more data drive particularly the cardiologists and endocrinologists have shown the greatest percentage growth, but many of the GPs are showing growth as well. With our existing sales force, we are actually getting to see some of the no-see docs much more than what we were expecting. The overall balance is such that with our current staffing that many doctors have been to sort of our target of blocks me seven times to roughly the third quarter of this year.
So we are seeing growth for that is terrific, but we do think we need to have greater frequency of calls on our existing targets, and there are many additional targets out there that we are addressing. Some of those will be addressed through education -- medical education programs. Some of them addressed through digital means but we do think calling on additional docs. So we are seeing that there are trends. Urban docs tend to prescribe or change practice behaviors faster than suburban docs; group practice docs faster than single practitioners.
Younger docs tend to change faster than older docs, but there is exceptions to all these. We are trying to take all that data and figure out the best path is going forward, but the consistent feedback we are getting is that these results are robust and regularly hearing from docs that they want to use Vascepa on many of their patients. Some of them are doing it already. Others are looking for the label to be expanded before acting more aggressively.

所以,我们现在正在做的是继续做分析。我们正在看到我们正在呼吁的医生,即今天大约50,000或略多于50,000,其中约80%是初级保健。其中约12%是心脏病专家。其中大约7%是内分泌学家,然后其他学科有一个四舍五入。那些倾向于更多数据驱动的人,尤其是心脏病专家和内分泌学家,已经表现出最大的百分比增长,但许多全科医生也表现出增长。凭借我们现有的销售人员,我们实际上看到的一些看不见的文档比我们期望的要多得多。整体平衡是这样的,在我们目前的人员配备下,许多医生已经将我的目标定为七次,大约在今年第三季度。

所以我们看到增长非常好,但我们确实认为我们需要对现有目标进行更频繁的调用,并且我们正在解决许多其他目标。其中一些将通过教育 - 医学教育计划来解决。其中一些通过数字方式解决,但我们确实认为需要额外的文档。所以我们看到有趋势。城市文档倾向于比郊区文档更快地规定或改变实践行为;小组练习文档比单个练习者更快。

较年轻的文档往往比旧文档更快地更改,但所有这些文档都有例外。我们正在尝试获取所有这些数据,并找出最佳路径,但我们得到的一致反馈是,这些结果非常强大,并且经常从文档中听到他们希望在许多患者身上使用Vascepa。他们中的一些人已经这样做了。其他人正在寻求扩大标签,然后才采取更积极的行动。

身份不明的分析师

Okay. And how much additional work needs to be done on the payer side? It sounds like it's been going through in most cases even if it's for off-label use, but do you need to get this expanded indication before I don't know, the gates really open up in terms of reimbursement? Or the way you described it, it sounds like really isn't that much pushback, and maybe just throw in there the price on a relative basis and how payers are thinking about that in the context of the whole pharmaco-economic analysis with this outcome study?

好的。 还有多少工作需要在付款方面完成? 在大多数情况下,即使是在标签外使用,这听起来似乎已经过去了,但是在我不知道之前,你是否需要获得这个扩展的指示,这些大门在报销方面是否真的开放了? 或者你描述它的方式,听起来真的不是那么多的推迟,也许只是在相关的基础上投入价格以及付款人如何在整个药物经济分析的背景下考虑这个结果 研究?

John Thero

So, we have been competing for six years against generic products, and our approval rate and coverage is pretty good, but there are opportunities for improvement. So, today greater than 50% of our prescriptions are off-label. The approval rate overall for our drug with managed care is just slightly below 80%, which is neck in neck with generics and of level that most drugs we are very pleased with.
In terms of coverage and Medicare Part D, we have got over 90% adult lives in the United States covered predominantly on tier 2 unrestricted and over 80% of commercial lives covered predominantly on tier 2 unrestricted, but within that, you have some holdouts and there are some managed care plans that just by their policies won't provide coverage until you have a label. So there is opportunity to improve there.
Many physicians are still very new to Vascepa, and where the drug is new to them, they will assume that coverage doesn't exist even if it does exist, and of course, if they have 10 patients and one doesn't get coverage, they remember the one that doesn't. So, I think -- I am on Vascepa. I have been taking it for six years now. Unfortunately off-label and my insurance company has never pushback on myself or my physician. I am happy to be taking it. Many payers are that way, but there are some that where coverage could improve, and we are looking forward to that happening.

因此,我们与仿制药竞争了六年,我们的认可率和覆盖率非常好,但仍有改进的机会。因此,今天超过50%的处方药都是标签外的。对于我们的管理式医疗药物,整体的批准率略低于80%,这与普通药物和我们非常满意的大多数药物的水平相当。

就覆盖范围和Medicare D部分而言,我们在美国有超过90%的成年人生活,主要是在第2层无限制,超过80%的商业生活主要在第2层无限制,但在此之内,你有一些坚持和有一些管理式医疗计划,只有他们的政策不会提供保险,直到你有一个标签。所以有机会在那里改进。

许多医生对Vascepa来说仍然是一个新手,并且药物对他们来说是新的,他们会认为即使它确实存在也不存在,当然,如果他们有10名患者且其中一名没有得到报道,他们记得没有的那个。所以,我想 - 我在Vascepa。我已经服用了六年了。不幸的是,不合格的我和我的保险公司从未对自己或我的医生施压。我很高兴接受它。许多付款人都是这样,但也有一些付款人可以改善,我们期待着这种情况发生。

身份不明的分析师

Okay. And just in terms of net pricing and I don't know there is a change at all given all the robustness of the data and the value prop that you could bring to the payers?

好的。 就净定价而言,我不知道数据的稳健性和您可以带给付款人的价值支柱是否会发生变化?

John Thero

There will likely be at least one if not two pharmaco-economic analyses presented this year. If those economic analysis are consistent with and some people argue whether they are always consistent or not, but if they are consistent with the methodologies that have used say for PCSK9s, where I price is about a fifth of where the PCSK9s were when those analysis were done and our number we didn't treat is considerable lower as we talk. Those analyses should turn out favorable.
That being said, our current pricing is similar to where statins were you to adjust for inflation. Like this is more like where Atorvastatin would be. A little bit lower than where Crestor or Rosuvastatin was, and we think by doing that, we are positioned for mass use as an affordable product, and this is much of a high volume opportunity than it is pricing opportunity.

今年可能会有至少一项(如果不是两项)药物经济学分析。 如果这些经济分析是一致的,并且有些人认为它们是否始终是一致的,但是如果它们与PCSK9使用的方法一致,那么我的价格大约是PCSK9的五分之一。 在我们谈话时,我们没有对待的数量相当低。 那些分析应该是有利的。

话虽如此,我们目前的定价与他汀类药物的通胀调整相似。 像这样更像阿托伐他汀。 比Crestor或Rosuvastatin低一点,我们认为,通过这样做,我们可以作为一种经济实惠的产品进行大规模使用,这是一个很大的机会,而不是定价机会。

身份不明的分析师

Okay. Excellent. We will end on that note. Thank you so much for being here, John. We all appreciate it, and thanks everyone for coming. Enjoy the rest of the conference.

好的。 优秀。 我们将以此说明结束。 非常感谢你在这里,约翰。 我们都很感激,并感谢大家的到来。 享受会议的其余部分。

John Thero

Thanks everybody.

谢谢大家。

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