Idorsia Pharmaceuticals Ltd

Dear ladies and gentlemen, welcome to the conference call regarding the Half Year Financial Results Presentation 2021. At our customer's request, this conference will be recorded. As a reminder, all participants will be in a listen-only mode. After the presentation, there will be an opportunity to ask questions. If any participant has difficulties hearing the conference, Please press the bar key followed by zero on your telephone for operator assistance. May I now hand you over to Andrew Weiss who will lead you through this conference. Please go ahead.

Thank you, Angelo. Good morning, good afternoon, everyone, and welcome to the iDoors webcast to discuss the first half 21 results published this morning at 7 a.m. Central European Summertime. With me on the call to provide additional granularity on our operational, clinical, and financial advancements over the first half are with me, our CEO, Jean-Paul Cosell, our CFO, Andre Muller. They will be making prepared remarks. For the Q&A session, we will be joined by our Chief Commercial Officer, Simon Joes. Next slide. Before handing over the mic, a few customary remarks, i.e., we will be making forward-looking statements in this call, which may or may not turn out to happen. Hence, you have been appropriately warned about the risks and benefits of investing in Eidortia shares. Next slide. Thus, without further ado, Jean-Paul, the floor is yours.

Thank you very much, Andrew. During the first six months of this year, Eidortia has made tremendous progress to become a fully fledged biopharmaceutical company. Next slide. If I see the main highlights of this first half, you will understand why I feel so confident that we're on the good track. As you have, as we have discussed already with you, has been submitted in the U.S., the MEA has been submitted in Europe, and the submission has also been made with Swiss milk. Ponezimod, which is a drug from Rory sold by Johnson & Johnson, has been approved in the U.S. and in Europe, which shows the quality of the clinical program which was set up by Actelion and by the team which is now within IDORSIA. The Clasodentine NDA has been submitted in Japan and also we have initiated the phase three study with the for suspected myocardial infarction. In addition, within a few months, we are going to get the results of the study, the Phase III study, the pivotal study with Ducerastat and the Phase IIb of Senerimod, where we will get the results of aproxifentan in the middle of next year. But for aproxifentan, all the patients have been recruited. Next slide. So when I mentioned to become a fully-fetched biopharmaceutical company, what does it mean for us? It means mainly to fulfill five strategic priorities. These are the priorities we have from day one of the creation of Adortia, and we have not changed these priorities. So first, we wanted to deliver three products to the market, and I am not counting into these three products polygamous. And you will see in the next slide the progress of the pipeline and why we believe we can reach and maybe be better than this goal. Number two, or the second strategy priority is to build a world-class commercial organization. This task has been given to Simon Jones, and we are progressing extremely well in order to have this infrastructure, which will allow us to market our drugs. Number three is to bring Eidosia as soon as possible to a sustainable, and I would say also to a growing profitability. We are doing everything that in the midterm, we can really become financially independent. Number four's goal is to continue to fuel our pipeline with new discovery from our own research. I always say that drug discovery is the engine of a biopharmaceutical company. It's like in a car. In a car, you can stop the engine and you can continue to run, but you won't run very far. Without new drug discoveries, a company cannot really grow and cannot have a future. This is why it is so important for us to continue to build our pipeline by bringing new drugs within this clinical pipeline. Finally, the fifth goal of Idorsia was to utilize state-of-the-art technologies in every, I would say, department or in every area of the company. So you will see that we are using these new technologies not only in drug discovery but also in clinical development and in marketing. Next slide. So let's look at the pipeline. Our goal was to put on the market three new drugs, and we are really one of the way. Darylorexant, our dual orexin receptor antagonist, has been signed, as I've mentioned. The review is ongoing, and it's going, I would say, very well. Aprocitantan is a dual endothelin receptor antagonist. It is for the treatment of difficult-to-treat or resistant hypertension. And at Roquitentan, the study is fully recruited, and we will get the results in the middle of next year. What I have to say, I think that we've got more than five DSMB review, and we have not heard of any safety concern. Clasocentan is a non-dotating receptor antagonist intravenous. for the treatment of vasospasm and prevention of, sorry, prevention of vasospasm with aneurysm, after, sorry, aneurysm subacnoidal hemorrhage. You have heard that we had very positive phase three in Japan, and we are going to get the results next year of the REACT study, which is, more or less a similar study performed in Europe and in the US. Leucerastat is tested in Fabry disease with a very interesting clinical protocol, which is evaluating not only the effect on the biomarkers, but the effect also on Leucerastat on the clinical symptoms, especially the neuropathic pain, which is the main problem, clinical problem in this patient with Fabry. Stella Togrell has been starting the phase three study, has been started. We had an SPA with the FDA, which means that the FDA agrees with the protocol that we are following for the phase three. and also with the way we are going to evaluate the effect of Sedatogrel in these patients. Senerimod is our drug for lupus. It's a third-generation S1P1 receptor modulator, an optimized S1P1 receptor, and we are waiting the result of a phase 2b, which could, if the study is positive and show consistent results, which could be used as a pivotal trial, which means that if the study is positive, we might have only one more study to perform before the NDA. We have also other drugs which are moving within our pipeline. I just can mention a selective orexin-1 receptor antagonist, for binge eating disorder, which is recruiting in phase two, and some other products coming from our drug discovery, which are in phase one or close to initiate phase two. Next slide. Coming back to garygorescent. I have mentioned and we have mentioned many times that the results were very consistent and showed an effect of the drug during the night, improving sleep, but also showing effects during the day consistent with an improvement of daytime functioning. The drug is now in discussion, so we are answering questions from the regulatory authorities, and I think the process is going very well. And we really should be in a position to launch in the U.S. in the beginning of next year this drug and in Europe and Switzerland later on during the year 2022. Next slide. I mentioned also that Senatogrel I started a study in a very interesting new indication, which is really to give the patient the chance to prevent his myocardial infarction or to have it in a much less severe form. And this can be obtained by having him injecting himself the platelet aggregation inhibitor, which is . It can inject with a device, and after that, it can call the ambulance, it can call the doctor, and then go into the hospital to be treated. But usually, the time between the first pain and the time the patient is treated within the hospital is in average three to four hours, depending, of course, if you are living in a big city or in the country. But it can be, and you can imagine in the United States, especially with very long distances, it can be six hours. So we have agreed with the FDA that we are going to do a treatment, sorry, doing a study where half of the patients will be treated with placebo, half of the patients will get this auto-injector, and will be able to treat themselves while they have the first signs of a new myocardial infarction, because we are dealing with patients who already had a myocardial infarction, so they can recognize a second incident. Next slide. So the second goal of iGrossia is to build a world-class commercial organization. We clearly are putting priorities on three regions, the U.S., Europe, and the main countries in Europe, and Japan. We have established affiliates in the U.S., in Rennes Nord, in France, in Paris, in Germany, in Munich, in Italy, in Milan, Spain in Madrid, in the United Kingdom in London, and for Japan in Tokyo. We have recruited the general manager, and I have to say that I am very happy to see how many top talents we can recruit. And sometimes I wonder why we are able to attract so experienced people who have already launched several drugs, who know the markets, who know the regulatory authorities and who know the processes needed to succeed for launch of new drugs. And frankly, I think that the recent measures being set in with BMS, being annexed on both by AstraZeneca, all these changes have created some opportunities for people to either choose staying within large companies or joining companies like maybe with a little bit more entrepreneurial, maybe a little bit more risk, but certainly with a very different culture, more close to startup. Next slide. Finally, we want to utilize state-of-the-art technologies to drive innovation. And this is, for example, used in drug discovery. Intelligence artificial is used now to select new drugs to help the drug discovery process. We try to make it something that is available for all the different departments in the drug discovery department. It is also Artificial intelligence, for example, is also used in clinical development to be able to best use our data, our data sets, and the millions of data points we have created by doing studies and, for example, chronic studies like . We are trying to use the most modern technology to analyze this data and also We also use this information to discuss, for example, with regulatory authorities. But we are also using modern technologies for marketing. Today, social media have a huge impact. Today, it's possible to address, to select the doctors who are more likely to prescribe our drugs, but we can also inform the patients and select the patient by using the most modern tools which can now allow to have a much better selection of the patient who require the type of drugs which we are developing.