Good day, and thank you for standing by. Welcome to the Regulatory Updates from the FDA Conference Call. At this time, all participants are in listen-only mode. After the speaker's presentation, there will be a question-and-answer session. To ask a question during the session, you will need to press star one on your telephone. Please be advised that today's conference is being recorded. If you're requiring further assistance, please press star zero. I would now like to hand the conference over to speaker today. Molly Poarch, Head of Global Communications for Orphazyme. Please go ahead. Thank you, Bernard. Good morning, everyone, and thank you for joining us today. On the call with me from Orphazyme are Christophe Bourdon, Chief Executive Officer, and Anders Vadsholt, Chief Financial Officer. Before we begin, I would like to remind you that this call will contain forward-looking statements concerning Orphazyme's future expectations, plans, prospects, corporate strategy, and performance, which constitute forward-looking statements for the purposes of the safe harbor provision under the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in our filings with the SEC. In addition, any forward-looking statements represent our views only as of the date of this webcast and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligations to update such statements. Now, I'll turn the call over to Christophe. Christophe? Thank you, Molly, and thanks for joining us on the call today. As you have seen from our press release, we received yesterday night a complete response letter from the U.S. FDA for investigational product arimoclomol for Niemann-Pick disease type C or NPC. Obviously, we are very disappointed by this outcome given the urgent need for new therapeutic options for NPC. We had hoped to deliver better news to this patient community, who has been eagerly awaiting for a PDUFA date. While we are not able to share any details outside of what it is in the press release, we felt it was important to hold this call today to communicate our resiliency and dedication to NPC patients and our key stakeholders. We do remain committed to working with the regulators with the goal of delivering arimoclomol to families managing this challenging disease. The CRL stated that the FDA is unable to approve the NDA in its present form based on needing additional qualitative and quantitative evidence to further substantiate the validity and interpretation of the five-domain NPC Clinical Severity Scale, NPC CSS, and in particular, the swallow domain. Our primary endpoint of the phase II/III clinical trial was progression in disease severity as measured by the five-domain NPC CSS. This is a disease-specific measure of disease progression consisting of the five clinically most relevant domains to patients with NPC caregivers and physicians. The FDA noted in the CRL that additional data are needed to bolster confirmatory evidence beyond the single phase II/III clinical trial to support the benefit risk assessment of the NDA. Right now, we plan to focus our efforts on pursuing the European regulatory approval with CHMP opinion expected in Q4 of 2021 and potential marketing authorization in Q1 of 2022. We will assess a potential path forward in the U.S. in partnership with the FDA. In the near term, we will need and we will reduce our costs substantially and freeze all company activities not related to clinical and regulatory activities to support approval for NPC. We have updated our financial outlook for the year. I'll turn it over to Anders to comment on this further. Anders? Thank you, Christophe. Today is a very difficult day for the NPC community and Orphazyme. The FDA decision has significant influence on our business and the outlook for the full year of 2021. We now expect operating expenses in the range of DKK 700 million-DKK 720 million compared to previously expected DKK 800 million-DKK 850 million, resulting in an expected operating loss of DKK 670 million-DKK 700 million, previously DKK 100 million-DKK 150 million. Our cash position at the end of 2021 is expected to be around DKK 50 million, previously DKK 350 million in excess of, which remove our previous stated revenue guidance for arimoclomol in the U.S. as well as the monetization of the priority review voucher. We expect to provide further updates in the coming weeks. With that, I'll turn over the call to Molly to begin Q&A. Molly? Thank you, Anders. Operator, we're now ready to take questions. Thank you. As a reminder, if you wish to ask a question, please press star and one on your telephone keypad and wait for your name to be announced. Once again, for questions, please press star one on your telephone. Your first question comes on the line of Thomas Bowers from Danske Bank. Please ask your question. Yes. Thank you very much. Very sorry and surprised about the FDA decision here. You had a very close dialogue with the FDA when deciding the trial and also the endpoints, then you would say maybe even over the last two years have had in the pre- and post-filing process and communication. Just help me understand what exactly differs from the communication now regarding the five-domain scale. Just secondly, on the swallow domain, what difference did you see compared to placebo in this part? Because as I remember, it was primarily on the cognitive domain that you only saw maybe at best a numerical difference compared to placebo. Where are you exactly on this swallow domain, and why is the agency potentially even highly focused on this specific domain? Just on the additional data, I know you can't say much, most likely, but do you have any feeling on whether the FDA actually thinks that they need a new confirmatory clinical trial, or is there some sort of way to look at maybe existing data or confirmatory pre-clinical work or any additional biopsy work or something like that that could be supported for resubmission? Just lastly, just on safety profile, is there anything also, I know you submitted, for example, the QTc data in December, so anything here that may have tilted the benefit-risk assessment from the FDA? Thank you. Thanks so much, Christophe, here. Thanks for your questions. Clearly, I'm going to really focus on what's on the press release. At this stage, I won't be able to really disclose much more details. I think, the FDA, they clearly stated that their CRL is really based at the moment on needing additional qualitative and quantitative evidence. We will really assess in the coming days what's the path forward with the FDA. That's what the team will be focusing on. Again, on the additional evidence beyond clinical, there could be some kind of also like, for example, pharmacodynamic data. At this stage, it's very important for us to ensure that we find the most constructive path forward with the FDA, and as soon as we have new information here, we're going to come back and we're going to update you. Okay. can you just remind me on the swallow domain, how did your data look for that domain alone? Maybe I will pass over to, yeah, Thomas Blaettler, Chief Medical Officer. Yeah. Thank you for the question, Thomas. We had good effect on the swallow domain. It was one of three domains that mostly carried the effect. Okay. That should be supported for your case if they focus more highly on the swallow domain, basically. Also meaning that you have a p-value of less than 0.05, I guess. I think what is still important to emphasize here, it is a composite outcome measure Of course. where all five domain should be considered in totality. Certainly, the swallow domain is one of the strong domains for arimoclomol and is associated with survival. Okay, perfect. just maybe a little also a little bit premature, but, and you also stated you'll give some more details on the financials, but just to understand the commercial approach here, so related to your updated guidance. does this include preparation ahead for a commercial launch in Europe? also, I remember, well, you have the whole team in place in the U.S., of course, for the U.S. launch, and then you will sort of take a little different approach in Europe. is this still the case, or are you going to maybe make some more preparation for Europe in order to actually have a launch with salespeople in the field? Is that how I should see it? Thomas, as indicated by Anders, I think our key priority is to ensure that we will close the year around DKK 15 million in cash, and we will really ensure a prioritization of all the key activities that are critical to ensure ongoing dialogue with the EMA and find a path forward with the FDA. we're going to reassess as time goes by, but that's really the focus of the core activities and this stage in the coming months. Okay, got it. Okay, great. Thank you very much. Thank you, Thomas. Your next question comes on the line of Jamie Johnny from TD Cowen. Please ask your question. Sorry, couldn't hear the question. We just lost Jamie on the line. We have next question from the line of Samir Devani from Rx Securities. Please ask your question Yeah. Hi, thanks for taking my question. I'm just quite interested in trying to understand how a drug with breakthrough designation, where you're supposed to obviously have intensive FDA interactions, how do you get to this point where the FDA is questioning the validity of the endpoint? Surely this must have been part of the dialogue ahead of filing. Perhaps you can just explain how you got to this point. Thanks very much. Christophe here. We are going to reassess clearly the detail of the CRL, and look what the path forward here. I think at this stage, as Thomas was saying, I think the decision of the FDA is based on totality of evidence. We're going to revert back, as soon as we know more, and then address your specific issue here. I'm not going to comment on how the FDA came to its conclusion. I guess, then just as a follow-up, in terms of Europe and regulatory interaction there, can you just comment on any interactions regarding this endpoint with them? Thanks very much. this is Thomas Blaettler. We are in review with EMA and conversations and dialogue is ongoing with EMA. Okay, thanks very much. Once again, for any questions, please press star one on your telephone keypad. There are no further questions at this time. Please continue. All right. Thanks everyone for joining us today. I think while we delivered disappointing news today, I just wanted to assure you that we are focused on partnering with the FDA to evaluate the path forward, while also we continue pursuing registration in Europe. We look forward to keeping you informed on our progress. Operator, now we are ready to close the call. Thank you. That does conclude our call for today. Thank you for participating. You may all disconnect.