Idorsia Pharmaceuticals Ltd

Dear ladies and gentlemen, welcome to the IDOSIA conference call. At our customer's request, this conference will be recorded. As a reminder, all participants will be in a listen-only mode, and after the presentation, there will be an opportunity to ask questions. If any participant has difficulties hearing the conference, please press star 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.

Good morning, good afternoon, everyone, and welcome to today's conference call slash webcast. My name is Andrew Weiss. I'm the Head of Industrial Relations and Corporate Communications here at Eidosium. We are here to discuss our full year 2020 results, performance, as well as give you an outlook on 2021. With me on the call are our CEO, Jean-Paul Closel, and our CFO, André Muller. They'll be presenting in the first part of this session. For the Q&A, we will also have our Chief Commercial Officer, Simon Joes, join us. So let's dive right in. Next slide. As customary, I need to remind everybody that we will be making forward-looking statements. We have therefore been adequately warned about the risks and benefits of investing in IDORSIA. Next slide. We're now on slide three. Jean-Paul, the floor is all yours.

Thank you, Andrew. So good morning and good afternoon to everyone. 2020 has been a very bizarre year and quite a dangerous one if we think about this terrible epidemic. But despite these very difficult and tough conditions, Eidosia has done extremely well. And I have to say, much better than many would have expected and that personally I would have expected. It's very rare, as you will see, to have met nearly all our goals and fulfill all what we were expecting for 2020. So, next slide. Just let's have a look at the highlights. Clearly, in 2020, we have announced positive pivotal results for diuretics in insomnia. And we have been able at the beginning of this year to file this NDA, which has been submitted to the FDA. We have been able in 2020 to obtain very positive results for the two registration studies, which are required for the approval in Japan. What we have done also in 2020 is to strengthen our liquidity, and we have been able to raise 865 million Swiss francs. What we have also done in 2020 was to establish U.S. commercial operations under the leadership of Simon Jones. And also, just we happen to know very recently, we have won the case against the previous Axovan shareholders who were requesting the payment of the milestones after the G&G acquisitions. Next slide. What makes really the engine of any biotech company is, of course, the pipeline. And these slides summarize our pipeline, which is comprising 12 products without mentioning the T-channel calcium blocker, which is now being licensed, which has been licensed to Neurocrime and which is in phase two. of clinical development for rare form of epilepsy. But as you can see, our pipeline from daridorexant already submitted to four and three additional phase three programs, and clearly now with selatogrel, which is very soon, which is going to start the phase three very soon, and of course, Senerimod, where we are waiting the results before starting the final pivotal trial. You see that this pipeline is very rich, is also not only having late phase product, but also phase two products and early project, and we have been able to start new clinical developments of some very new innovative drugs. But we will today focus on the later phase program, which are going to bring new products to the market within the next three years for Aidosia. Next slide. So Dari-Durexan for insomnia. Dari-Durexan is quite a unique product. Because as an orexin receptor antagonist with a short half-life, we have been able to show that this drug could improve the sleep, but also could improve daytime functioning. And this improvement of daytime functioning has been measured using a validated instrument developed in collaboration with the FDA. So as I mentioned, the NDA has been submitted in January of this year, and we are going very soon, in the coming months, to submit the market authorization for the EU. And we are planning to be able to launch Dairy Dorexan in the U.S. in Q2 of 2022. and hopefully in Europe for the end of 2022. Next slide. So, clazosantan has done also extremely well. As you know, this endothelin receptor antagonist, it's a selective ETA receptor antagonist, has been able to show in the two-pivotal study, pivotal trial study in Japan has been able to show that it could prevent the cerebral ischemia, sorry, it could prevent the vasospasm and its clinical consequences following cerebral ischemia post-subarachnoidal hemorrhage. So the two studies, one was in patients treated with surgery or clipping, and one, with a patient with coiling, so without surgery. Both studies showed nearly the same very strong effect and a prevention of not only the vasospasm, but its clinical consequences. So we are planning in the coming months to file Clazozentan in Japan, and we are also pursuing in Europe and in U.S. Another study which will be required for the fighting in U.S. and in Europe, this study is called REACT, and we are halfway through the recruitment. This study is the one from all our programs which is suffering most of this COVID crisis because all the patients are treated within intensive care, And a lot of intensive care units are extremely busy with the COVID crisis and situation. But we hope to be able to finish maybe this year or first half of next year, finish the recruitment within REACT. Next slide. So, lucerastat. Lucerastat is an oral drug. which is distributing extremely well within the whole body. It can penetrate the tissue, and it can particularly penetrate the CNS, central nervous system, and prevent the buildup of lipids within this central nervous system. And as you know, within Fabry, there is an accumulation of some of this lipid and this accumulation within the nerves in particular is producing neuropathic pain. A lot of also consequences of the Fabry disease is the accumulation of this lipid within the heart, within the kidney, within the gastrointestinal systems. So by having a drug which distributes everywhere in the body, we think we have a drug which is going to have a major clinical effect on the disease and on its symptoms. And the main symptoms, which is a primary endpoint of the study, which is now going on and where we have finished the recruitment and we have the result in the second half of this year, this study called MODIFIED, is evaluating neuropathic pain with a patient-reported outcome, evaluating the complaints about the pain from the patients. Next slide. So, abrocitantin is a neural endothelin receptor antagonist, and we are evaluating its effects in patients which are non-responsive to most of the generic anti-hypertensive drugs. So the patients who can not be controlled with existing anti-hypertensive drugs are included in the study called PRECISIONS. And once we have shown that these patients are true resistant hypertensive patients, we are testing on them afrocitantin at two doses. And we are also evaluating the long-term effect on blood pressure of afrocitantin during an eight-month chronic treatment. At the end of these end months, we are doing a randomized withdrawal to be able to show that the drug is still very effective after nine months of treatment. So the study is going extremely well. We are finishing the screening and the inclusion of the patients will be done in the coming months. And the results are expected in the first half of 2022. We already had, I think, five review of the safety committee. So the safety, which is very important in this very severe patient, is extremely, seems to be posing no problems. And we are very optimistic that this drug is going to fulfill a very high medical need. As you know, a lot of patients, because of diabetes, because of genetic background, Because of obesity, in the U.S., there is an increasing number of patients which are not controlled for their blood pressure, and a new drug effective in these patients is going to be very important. As you know, Janssen will have the rights, commercial rights about this drug, and we will get royalties. Next slide. Thelatograv. Thelatograv is also, I would think, a revolution in cardiology because it's a self-injected device where you can, the patient, in case of a recurring myocardial infection. So these are patients who already got a myocardial infection. And once they have another pain, and therefore they are at risk of a second myocardial infection, they can inject themselves with the seratogrel and then be protected for four hours, which gives them the time to call an ambulance and to go to the hospital where they will be treated. The drug is not long active, so once they are within the hospital, Every intervention, every treatment can be done without the risk of an interaction with a . I think it's very important, and we have seen that in the time of COVID, for example, ambulances delay is a huge problem in New York. Patients have to wait sometimes three, four hours to get an ambulance to be hospitalized, and such a drug would be extremely useful and will be extremely useful. The clinical program phase three is going to be initiated within, I would say, the first patient of this program are going to be treated in the second half of this year, but we're already contacting the centers and preparing for this phase three program, which has an SBA, which means that we have agreed with the FDA of the program and its endpoints. Next slide. SceneryMod, so SceneryMod is the third generation sphingosine 1-phosphate receptor modulator. This drug has been selected not only because of its selectivity, its potency, because of a very specific interaction with the S1P1 receptor, which leads to really a very good safety profile And I think that with this drug, SceneryMod, we'll be able to show that we are avoiding some side effects of the first generation or second generation S1P1 receptor. It received a fast-track designation from the FDA. And the study, the first pivotal study called CARE, which is also a dose response, but which has a clinical endpoint and therefore can be used if it's positive as a pivotal trial, this CARE study will have its recruitment completed by the end of February, and we will get this, we get the results before the end of the year of 2021, and we are planning to have basically a discussion before the end of the year with the FDA in order to start in 2022 the second pivotal trial. Next slide. So as you see, we are at an infection point. Aidosia is really, because of this fantastic 2020 year, we are in a very good situation. We are going to see many, many important milestones crucial for the company. In 2021, you have seen that we have five dairy directions in FDA, soon with the MEA. Cladocentan will be filed also soon in Japan. Ceratogrel phase three will be initiated. Lucerastat results, phase three results, final phase three results will be available second half of this year. And Cenarimod also, SceneryMod results we know will be available before the end of 2021. But also in 2022, we are going to have crucial results, crucial milestone, hopefully with the approval and the launch of Daridorexan, of course, in the US, but also with the results of Aprocitantan, also the launch of Clasodentan in Japan, and the results of Clazozentan in the, sorry, in Clazozentan in Japan, that was in Japan. Sorry, Andrew, I don't see the slide anymore.